To reach early diagnoses and improve outcomes in cases of mucosal and esophageal lichen planus (ELP), patient education along with a multidisciplinary approach centered on collaboration among dermatologists, gastroenterologists, gynecologists, and dental practitioners should be a priority. Tofacitinib therapy should be considered in the treatment of patients presenting with cutaneous lichen planus (CLP), mucosal lichen planus, and ELP.

Lichen planus is a papulosquamous disease of the skin and mucous membranes that is most common on the skin and oral mucosa. Typical lesions of CLP present as purple, pruritic, polygonal papules and plaques on the flexural surfaces of the wrists and ankles as well as areas of friction or trauma due to scratching such as the shins and lower back. Various subtypes of lichen planus can present simultaneously, resulting in extensive involvement that worsens through koebnerization and affects the oral cavity, esophagus, larynx, sclera, genitalia, scalp, and nails.^1,2

Esophageal lichen planus can develop with or without the presence of CLP, oral lichen planus (OLP), or genital lichen planus.³ It typically affects women older than 50 years and is linked to OLP and vulvar lichen planus, with 1 study reporting that 87% (63/72) of ELP patients were women with a median age of 61.9 years at the time of diagnosis (range, 22–85 years). Almost all ELP patients in the study had lichen planus symptoms in other locations; 89% (64/72) had OLP, and 42% (30/72) had vulvar lichen planus.⁴ Consequently, a diagnosis of ELP should be followed by a thorough full-body examination to check for lichen planus at other sites. Studies that examined lichen planus patients for ELP found that 25% to 50% of patients diagnosed with orocutaneous lichen planus also had ELP, with ELP frequently presenting without symptoms.^3,5 These findings indicate that ELP likely is underdiagnosed and often misdiagnosed, resulting in an underestimation of its prevalence.

Our case highlights a frequently misdiagnosed condition and underscores the importance of close examination of patients presenting with CLP and OLP for signs and symptoms of ELP. Furthermore, we discuss the importance of patient education and collaboration among different specialties in attaining an early diagnosis to improve patient outcomes. Finally, we review the clinical presentation, diagnosis, and treatment of CLP, OLP, and ELP, as well as the utility of tofacitinib for ELP.

Case Report

An emaciated 89-year-old woman with an 11-year history of CLP, OLP, and genital lichen planus that had been successfully treated with topicals presented with an OLP recurrence alongside difficulties eating and swallowing. Her symptoms lasted 1 year and would recur when treatment was paused. Her medical history included rheumatoid arthritis, hypothyroidism, and hypertension, and she was taking levothyroxine, olmesartan, and vitamin D supplements. Dentures and olmesartan previously were ruled out as potential triggers following a 2-month elimination. None of her remaining natural teeth had fillings. She also reported that neither she nor her partner had ever smoked or chewed tobacco.

The patient’s lichen planus involvement first manifested as red, itchy, polygonal, lichenoid papules on the superior and inferior mid back 11 years prior to the current presentation (Figure 1). Further examination noted erosions on the genitalia, and a subsequent biopsy of the vulva confirmed a diagnosis of lichen planus (Figure 2). Treatment with halobetasol propionate ointment and tacrolimus ointment 0.1% twice daily (BID) resulted in remission of the CLP and vulvar lichen planus. She presented a year later with oral involvement revealing Wickham striae on the buccal mucosa and erosions on the upper palate that resolved after 2 months of treatment with cyclosporine oral solution mixed with a 5-times-daily nystatin swish-and-spit (Figure 3). The CLP did not recur but OLP was punctuated by remissions and recurrences on a yearly basis, often related to the cessation of mouthwash and topical creams. The OLP and vulvar lichen planus were successfully treated with as-needed use of a cyclosporine mouthwash swish-and-spit 3 times daily as well as halobetasol ointment 0.05% 3 times daily, respectively. Six years later, the patient was hospitalized for unrelated causes and was lost to follow-up for 2 years.

The patient experienced worsening dysphagia and odynophagia over a period of 2 years (mild dysphagia was first recorded 7 years prior to the initial presentation) and reported an unintentional weight loss of 20 pounds. An endoscopy was performed 3 years after the initial report of dysphagia and noted esophageal erosions (Figure 4A) and a stricture (Figure 4B), but all abnormal involvement was attributed to active gastroesophageal reflux disease. She underwent 8 esophageal dilations to treat the stricture but noted that the duration of symptomatic relief decreased with every subsequent dilation. An esophageal stent was placed 4 years after the initial concern of dysphagia, but it was not well tolerated and had to be removed soon thereafter. A year later, the patient underwent an esophageal bypass with a substernal gastric conduit that provided relief for 2 months but failed to permanently resolve the condition. In fact, her condition worsened over the next 1.5 years when she presented with extreme emaciation attributed to a low appetite and pain while eating. A review of the slides from a prior hospital esophageal biopsy revealed lichen planus (Figure 5). She was prescribed tofacitinib 5 mg BID as a dual-purpose treatment for the rheumatoid arthritis and OLP/ELP. At 1-month follow-up she noted that she had only taken one 5-mg pill daily without notable improvement, and after the visit she started the initial recommendation of 5 mg BID. Over the next several months, her condition continued to consistently improve; the odynophagia resolved, and she regained the majority of her lost weight. Tofacitinib was well tolerated across the course of treatment, and no adverse side effects were noted. Furthermore, the patient regained a full range of motion in the previously immobile arthritic right shoulder. She has experienced no recurrence of the genital lichen planus, OLP, or CLP since starting tofacitinib. To date, the patient is still taking only tofacitinib 5 mg BID with no recurrence of the cutaneous, mucosal, or esophageal lichen planus and has experienced no adverse events from the medication.

Clinical Presentation—Lichen planus—CLP and OLP—most frequently presents between the ages of 40 and 60 years, with a slight female predilection.^1,2 The lesions typically present with the 5 P’s—purple, pruritic, polygonal papules and plaques—with some lesions revealing white lacy lines overlying them called Wickham striae.⁶ The lesions may be red at first before turning purple. They often present on the flexural surfaces of the wrists and ankles as well as the shins and back but rarely affect the face, perhaps because of increased chronic sun exposure.^2,6 Less common locations include the scalp, nails, and mucosal areas (eg, oral, vulvar, conjunctival, laryngeal, esophageal, anal).¹

If CLP is diagnosed, the patient likely will also have oral lesions, which occur in 50% of patients.² Once any form of lichen planus is found, it is important to examine all of the most frequently involved locations—mucocutaneous and cutaneous as well as the nails and scalp. Special care should be taken when examining OLP and genital lichen planus, as long-standing lesions have a 2% to 5% chance of transforming into squamous cell carcinoma.²

Although cases of traditional OLP and CLP are ubiquitous in the literature, ELP rarely is documented because of frequent misdiagnoses. Esophageal lichen planus has a closer histopathologic resemblance to OLP compared to CLP, and its highly variable presentation often results in an inconclusive diagnosis.³ A review of 27 patients with lichen planus highlighted the difficult nature of diagnosing ELP; ELP manifested up to 20 years after initial lichen planus diagnosis, and patients underwent an average of 2.5 dilations prior to the successful diagnosis of ELP. Interestingly, 2 patients in the study presented with ELP in isolation, which emphasizes the importance of secondary examination for lichen planus in the presence of esophageal strictures.⁷ The eTable provides common patient demographics and symptoms to more effectively identify ELP.Differential Diagnosis—Because lichen planus can present anywhere on the body, it may be difficult to differentiate it from other skin conditions. Clinical appearance alone often is insufficient for diagnosing lichen planus, and a punch biopsy often is needed.^2,20 Cutaneous lichen planus may resemble eczema, lichen simplex chronicus, pityriasis rosea, prurigo nodularis, and psoriasis, while OLP may resemble bite trauma, leukoplakia, pemphigus, and thrush.²⁰ Dermoscopy of the tissue makes Wickham striae easier to visualize and assists in the diagnosis of lichen planus. Furthermore, thickening of the stratum granulosum, a prevalence of lymphocytes in the dermoepidermal junction, and vacuolar alteration of the stratum basale help to distinguish between lichen planus and other inflammatory dermatoses.²⁰ A diagnosis of lichen planus merits a full-body skin examination—hair, nails, eyes, oral mucosa, and genitalia—to rule out additional involvement.

Esophageal lichen planus most frequently presents as dysphagia, odynophagia, and weight loss, but other symptoms including heartburn, hoarseness, choking, and epigastric pain may suggest esophageal involvement.⁴ Typically, ELP presents in the proximal and/or central esophagus, assisting in the differentiation between ELP and other esophageal conditions.³ Special consideration should be taken when both ELP and gastroesophageal reflux disease are considered in a differential diagnosis, and it is recommended to pair an upper endoscopy with pH monitoring to avoid misdiagnosis.⁸ Screening endoscopies also are helpful, as they assist in identifying the characteristic white webs, skin peeling, skin surface erosion, and strictures of ELP.⁴ Taken together, dermatologists should encourage patients with cutaneous or mucocutaneous lichen planus to undergo an esophagogastroduodenoscopy, especially in the presence of any of ELP’s common symptoms (eTable).

Etiology—Although the exact etiology of lichen planus is not well established, there are several known correlative factors, including hepatitis C; increased stress; dental materials; oral medications, most frequently antihypertensives and nonsteroidal anti-inflammatory drugs; systemic diseases; and tobacco usage.^6,21

Dental materials used in oral treatments such as silver amalgam, gold, cobalt, palladium, chromium, epoxy resins, and dentures can trigger or exacerbate OLP, and patch testing of a patient’s dental materials can help determine if the reaction was caused by the materials.^6,22 The removal of material contributing to lesions often will cause OLP to resolve.²²

It also has been suggested that the presence of thyroid disorders, autoimmune disease, various cancers, hypertension, type 2 diabetes mellitus, hyperlipidemia, oral sedative usage, and/or vitamin D deficiency may be associated with OLP.^21,23 Although OLP patients who were initially deficient in vitamin D demonstrated marked improvement with supplementation, it is unlikely that vitamin D supplements impacted our patient’s presentation of OLP, as she had been consistently taking them for more than 5 years with no change in OLP presentation.²⁴

Pathogenesis—Lichen planus is thought to be a cytotoxic CD8⁺ T cell–mediated autoimmune disease to a virally modified epidermal self-antigen on keratinocytes. The cytotoxic T cells target the modified self-antigens on basal keratinocytes and induce apoptosis.²⁵ The cytokine-mediated lymphocyte homing mechanism is human leukocyte antigen dependent and involves tumor necrosis factor α as well as IFN-γ and IL-1. The latter cytokines lead to upregulation of vascular adhesion molecules on endothelial vessels of subepithelial vascular plexus as well as a cascade of nonspecific mechanisms such as mast cell degranulation and matrix metalloproteinase activation, resulting in increased basement membrane disruption.⁶

Shao et al¹⁹ underscored the role of IFN-γ in CD8⁺ T cell–mediated cytotoxic cellular responses, noting that the Janus kinase (JAK)–signal transducer and activator of transcription pathway may play a key role in the pathogenesis of lichen planus. They proposed using JAK inhibitors for the treatment of lichen planus, specifically tofacitinib, a JAK1/JAK3 inhibitor, and baricitinib, a JAK1/JAK2 inhibitor, as top therapeutic agents for lichen planus (eTable).¹⁹ Tofacitinib has been reported to successfully treat conditions such as psoriasis, psoriatic arthritis, alopecia areata, vitiligo, atopic dermatitis, sarcoidosis, pyoderma gangrenosum, and lichen planopilaris.²⁶ Additionally, the efficacy of tofacitinib has been established in patients with erosive lichen planus; tofacitinib resulted in marked improvement while prednisone, acitretin, methotrexate, mycophenolate mofetil, and cyclosporine treatment failed.²⁷ Although more studies on tofacitinib’s long-term efficacy, cost, and safety are necessary, tofacitinib may soon play an integral role in the battle against inflammatory dermatoses.

Conclusion

Esophageal lichen planus is an underreported form of lichen planus that often is misdiagnosed. It frequently causes dysphagia and odynophagia, resulting in a major decrease in a patient’s quality of life. We present the case of an 89-year-old woman who underwent procedures to dilate her esophagus that worsened her condition. We emphasize the importance of considering ELP in the differential diagnosis of patients presenting with lichen planus in another region. In our patient, tofacitinib 5 mg BID resolved her condition without any adverse effects.

To reach early diagnoses and improve outcomes in cases of mucosal and esophageal lichen planus (ELP), patient education along with a multidisciplinary approach centered on collaboration among dermatologists, gastroenterologists, gynecologists, and dental practitioners should be a priority. Tofacitinib therapy should be considered in the treatment of patients presenting with cutaneous lichen planus (CLP), mucosal lichen planus, and ELP.

Lichen planus is a papulosquamous disease of the skin and mucous membranes that is most common on the skin and oral mucosa. Typical lesions of CLP present as purple, pruritic, polygonal papules and plaques on the flexural surfaces of the wrists and ankles as well as areas of friction or trauma due to scratching such as the shins and lower back. Various subtypes of lichen planus can present simultaneously, resulting in extensive involvement that worsens through koebnerization and affects the oral cavity, esophagus, larynx, sclera, genitalia, scalp, and nails.^1,2

Esophageal lichen planus can develop with or without the presence of CLP, oral lichen planus (OLP), or genital lichen planus.³ It typically affects women older than 50 years and is linked to OLP and vulvar lichen planus, with 1 study reporting that 87% (63/72) of ELP patients were women with a median age of 61.9 years at the time of diagnosis (range, 22–85 years). Almost all ELP patients in the study had lichen planus symptoms in other locations; 89% (64/72) had OLP, and 42% (30/72) had vulvar lichen planus.⁴ Consequently, a diagnosis of ELP should be followed by a thorough full-body examination to check for lichen planus at other sites. Studies that examined lichen planus patients for ELP found that 25% to 50% of patients diagnosed with orocutaneous lichen planus also had ELP, with ELP frequently presenting without symptoms.^3,5 These findings indicate that ELP likely is underdiagnosed and often misdiagnosed, resulting in an underestimation of its prevalence.

Our case highlights a frequently misdiagnosed condition and underscores the importance of close examination of patients presenting with CLP and OLP for signs and symptoms of ELP. Furthermore, we discuss the importance of patient education and collaboration among different specialties in attaining an early diagnosis to improve patient outcomes. Finally, we review the clinical presentation, diagnosis, and treatment of CLP, OLP, and ELP, as well as the utility of tofacitinib for ELP.

Case Report

An emaciated 89-year-old woman with an 11-year history of CLP, OLP, and genital lichen planus that had been successfully treated with topicals presented with an OLP recurrence alongside difficulties eating and swallowing. Her symptoms lasted 1 year and would recur when treatment was paused. Her medical history included rheumatoid arthritis, hypothyroidism, and hypertension, and she was taking levothyroxine, olmesartan, and vitamin D supplements. Dentures and olmesartan previously were ruled out as potential triggers following a 2-month elimination. None of her remaining natural teeth had fillings. She also reported that neither she nor her partner had ever smoked or chewed tobacco.

The patient’s lichen planus involvement first manifested as red, itchy, polygonal, lichenoid papules on the superior and inferior mid back 11 years prior to the current presentation (Figure 1). Further examination noted erosions on the genitalia, and a subsequent biopsy of the vulva confirmed a diagnosis of lichen planus (Figure 2). Treatment with halobetasol propionate ointment and tacrolimus ointment 0.1% twice daily (BID) resulted in remission of the CLP and vulvar lichen planus. She presented a year later with oral involvement revealing Wickham striae on the buccal mucosa and erosions on the upper palate that resolved after 2 months of treatment with cyclosporine oral solution mixed with a 5-times-daily nystatin swish-and-spit (Figure 3). The CLP did not recur but OLP was punctuated by remissions and recurrences on a yearly basis, often related to the cessation of mouthwash and topical creams. The OLP and vulvar lichen planus were successfully treated with as-needed use of a cyclosporine mouthwash swish-and-spit 3 times daily as well as halobetasol ointment 0.05% 3 times daily, respectively. Six years later, the patient was hospitalized for unrelated causes and was lost to follow-up for 2 years.

The patient experienced worsening dysphagia and odynophagia over a period of 2 years (mild dysphagia was first recorded 7 years prior to the initial presentation) and reported an unintentional weight loss of 20 pounds. An endoscopy was performed 3 years after the initial report of dysphagia and noted esophageal erosions (Figure 4A) and a stricture (Figure 4B), but all abnormal involvement was attributed to active gastroesophageal reflux disease. She underwent 8 esophageal dilations to treat the stricture but noted that the duration of symptomatic relief decreased with every subsequent dilation. An esophageal stent was placed 4 years after the initial concern of dysphagia, but it was not well tolerated and had to be removed soon thereafter. A year later, the patient underwent an esophageal bypass with a substernal gastric conduit that provided relief for 2 months but failed to permanently resolve the condition. In fact, her condition worsened over the next 1.5 years when she presented with extreme emaciation attributed to a low appetite and pain while eating. A review of the slides from a prior hospital esophageal biopsy revealed lichen planus (Figure 5). She was prescribed tofacitinib 5 mg BID as a dual-purpose treatment for the rheumatoid arthritis and OLP/ELP. At 1-month follow-up she noted that she had only taken one 5-mg pill daily without notable improvement, and after the visit she started the initial recommendation of 5 mg BID. Over the next several months, her condition continued to consistently improve; the odynophagia resolved, and she regained the majority of her lost weight. Tofacitinib was well tolerated across the course of treatment, and no adverse side effects were noted. Furthermore, the patient regained a full range of motion in the previously immobile arthritic right shoulder. She has experienced no recurrence of the genital lichen planus, OLP, or CLP since starting tofacitinib. To date, the patient is still taking only tofacitinib 5 mg BID with no recurrence of the cutaneous, mucosal, or esophageal lichen planus and has experienced no adverse events from the medication.

Clinical Presentation—Lichen planus—CLP and OLP—most frequently presents between the ages of 40 and 60 years, with a slight female predilection.^1,2 The lesions typically present with the 5 P’s—purple, pruritic, polygonal papules and plaques—with some lesions revealing white lacy lines overlying them called Wickham striae.⁶ The lesions may be red at first before turning purple. They often present on the flexural surfaces of the wrists and ankles as well as the shins and back but rarely affect the face, perhaps because of increased chronic sun exposure.^2,6 Less common locations include the scalp, nails, and mucosal areas (eg, oral, vulvar, conjunctival, laryngeal, esophageal, anal).¹

If CLP is diagnosed, the patient likely will also have oral lesions, which occur in 50% of patients.² Once any form of lichen planus is found, it is important to examine all of the most frequently involved locations—mucocutaneous and cutaneous as well as the nails and scalp. Special care should be taken when examining OLP and genital lichen planus, as long-standing lesions have a 2% to 5% chance of transforming into squamous cell carcinoma.²

Although cases of traditional OLP and CLP are ubiquitous in the literature, ELP rarely is documented because of frequent misdiagnoses. Esophageal lichen planus has a closer histopathologic resemblance to OLP compared to CLP, and its highly variable presentation often results in an inconclusive diagnosis.³ A review of 27 patients with lichen planus highlighted the difficult nature of diagnosing ELP; ELP manifested up to 20 years after initial lichen planus diagnosis, and patients underwent an average of 2.5 dilations prior to the successful diagnosis of ELP. Interestingly, 2 patients in the study presented with ELP in isolation, which emphasizes the importance of secondary examination for lichen planus in the presence of esophageal strictures.⁷ The eTable provides common patient demographics and symptoms to more effectively identify ELP.Differential Diagnosis—Because lichen planus can present anywhere on the body, it may be difficult to differentiate it from other skin conditions. Clinical appearance alone often is insufficient for diagnosing lichen planus, and a punch biopsy often is needed.^2,20 Cutaneous lichen planus may resemble eczema, lichen simplex chronicus, pityriasis rosea, prurigo nodularis, and psoriasis, while OLP may resemble bite trauma, leukoplakia, pemphigus, and thrush.²⁰ Dermoscopy of the tissue makes Wickham striae easier to visualize and assists in the diagnosis of lichen planus. Furthermore, thickening of the stratum granulosum, a prevalence of lymphocytes in the dermoepidermal junction, and vacuolar alteration of the stratum basale help to distinguish between lichen planus and other inflammatory dermatoses.²⁰ A diagnosis of lichen planus merits a full-body skin examination—hair, nails, eyes, oral mucosa, and genitalia—to rule out additional involvement.

Esophageal lichen planus most frequently presents as dysphagia, odynophagia, and weight loss, but other symptoms including heartburn, hoarseness, choking, and epigastric pain may suggest esophageal involvement.⁴ Typically, ELP presents in the proximal and/or central esophagus, assisting in the differentiation between ELP and other esophageal conditions.³ Special consideration should be taken when both ELP and gastroesophageal reflux disease are considered in a differential diagnosis, and it is recommended to pair an upper endoscopy with pH monitoring to avoid misdiagnosis.⁸ Screening endoscopies also are helpful, as they assist in identifying the characteristic white webs, skin peeling, skin surface erosion, and strictures of ELP.⁴ Taken together, dermatologists should encourage patients with cutaneous or mucocutaneous lichen planus to undergo an esophagogastroduodenoscopy, especially in the presence of any of ELP’s common symptoms (eTable).

Etiology—Although the exact etiology of lichen planus is not well established, there are several known correlative factors, including hepatitis C; increased stress; dental materials; oral medications, most frequently antihypertensives and nonsteroidal anti-inflammatory drugs; systemic diseases; and tobacco usage.^6,21

Dental materials used in oral treatments such as silver amalgam, gold, cobalt, palladium, chromium, epoxy resins, and dentures can trigger or exacerbate OLP, and patch testing of a patient’s dental materials can help determine if the reaction was caused by the materials.^6,22 The removal of material contributing to lesions often will cause OLP to resolve.²²

It also has been suggested that the presence of thyroid disorders, autoimmune disease, various cancers, hypertension, type 2 diabetes mellitus, hyperlipidemia, oral sedative usage, and/or vitamin D deficiency may be associated with OLP.^21,23 Although OLP patients who were initially deficient in vitamin D demonstrated marked improvement with supplementation, it is unlikely that vitamin D supplements impacted our patient’s presentation of OLP, as she had been consistently taking them for more than 5 years with no change in OLP presentation.²⁴

Pathogenesis—Lichen planus is thought to be a cytotoxic CD8⁺ T cell–mediated autoimmune disease to a virally modified epidermal self-antigen on keratinocytes. The cytotoxic T cells target the modified self-antigens on basal keratinocytes and induce apoptosis.²⁵ The cytokine-mediated lymphocyte homing mechanism is human leukocyte antigen dependent and involves tumor necrosis factor α as well as IFN-γ and IL-1. The latter cytokines lead to upregulation of vascular adhesion molecules on endothelial vessels of subepithelial vascular plexus as well as a cascade of nonspecific mechanisms such as mast cell degranulation and matrix metalloproteinase activation, resulting in increased basement membrane disruption.⁶

Shao et al¹⁹ underscored the role of IFN-γ in CD8⁺ T cell–mediated cytotoxic cellular responses, noting that the Janus kinase (JAK)–signal transducer and activator of transcription pathway may play a key role in the pathogenesis of lichen planus. They proposed using JAK inhibitors for the treatment of lichen planus, specifically tofacitinib, a JAK1/JAK3 inhibitor, and baricitinib, a JAK1/JAK2 inhibitor, as top therapeutic agents for lichen planus (eTable).¹⁹ Tofacitinib has been reported to successfully treat conditions such as psoriasis, psoriatic arthritis, alopecia areata, vitiligo, atopic dermatitis, sarcoidosis, pyoderma gangrenosum, and lichen planopilaris.²⁶ Additionally, the efficacy of tofacitinib has been established in patients with erosive lichen planus; tofacitinib resulted in marked improvement while prednisone, acitretin, methotrexate, mycophenolate mofetil, and cyclosporine treatment failed.²⁷ Although more studies on tofacitinib’s long-term efficacy, cost, and safety are necessary, tofacitinib may soon play an integral role in the battle against inflammatory dermatoses.

Conclusion

Esophageal lichen planus is an underreported form of lichen planus that often is misdiagnosed. It frequently causes dysphagia and odynophagia, resulting in a major decrease in a patient’s quality of life. We present the case of an 89-year-old woman who underwent procedures to dilate her esophagus that worsened her condition. We emphasize the importance of considering ELP in the differential diagnosis of patients presenting with lichen planus in another region. In our patient, tofacitinib 5 mg BID resolved her condition without any adverse effects.

To reach early diagnoses and improve outcomes in cases of mucosal and esophageal lichen planus (ELP), patient education along with a multidisciplinary approach centered on collaboration among dermatologists, gastroenterologists, gynecologists, and dental practitioners should be a priority. Tofacitinib therapy should be considered in the treatment of patients presenting with cutaneous lichen planus (CLP), mucosal lichen planus, and ELP.

Lichen planus is a papulosquamous disease of the skin and mucous membranes that is most common on the skin and oral mucosa. Typical lesions of CLP present as purple, pruritic, polygonal papules and plaques on the flexural surfaces of the wrists and ankles as well as areas of friction or trauma due to scratching such as the shins and lower back. Various subtypes of lichen planus can present simultaneously, resulting in extensive involvement that worsens through koebnerization and affects the oral cavity, esophagus, larynx, sclera, genitalia, scalp, and nails.^1,2

Esophageal lichen planus can develop with or without the presence of CLP, oral lichen planus (OLP), or genital lichen planus.³ It typically affects women older than 50 years and is linked to OLP and vulvar lichen planus, with 1 study reporting that 87% (63/72) of ELP patients were women with a median age of 61.9 years at the time of diagnosis (range, 22–85 years). Almost all ELP patients in the study had lichen planus symptoms in other locations; 89% (64/72) had OLP, and 42% (30/72) had vulvar lichen planus.⁴ Consequently, a diagnosis of ELP should be followed by a thorough full-body examination to check for lichen planus at other sites. Studies that examined lichen planus patients for ELP found that 25% to 50% of patients diagnosed with orocutaneous lichen planus also had ELP, with ELP frequently presenting without symptoms.^3,5 These findings indicate that ELP likely is underdiagnosed and often misdiagnosed, resulting in an underestimation of its prevalence.

Our case highlights a frequently misdiagnosed condition and underscores the importance of close examination of patients presenting with CLP and OLP for signs and symptoms of ELP. Furthermore, we discuss the importance of patient education and collaboration among different specialties in attaining an early diagnosis to improve patient outcomes. Finally, we review the clinical presentation, diagnosis, and treatment of CLP, OLP, and ELP, as well as the utility of tofacitinib for ELP.

Case Report

An emaciated 89-year-old woman with an 11-year history of CLP, OLP, and genital lichen planus that had been successfully treated with topicals presented with an OLP recurrence alongside difficulties eating and swallowing. Her symptoms lasted 1 year and would recur when treatment was paused. Her medical history included rheumatoid arthritis, hypothyroidism, and hypertension, and she was taking levothyroxine, olmesartan, and vitamin D supplements. Dentures and olmesartan previously were ruled out as potential triggers following a 2-month elimination. None of her remaining natural teeth had fillings. She also reported that neither she nor her partner had ever smoked or chewed tobacco.

The patient’s lichen planus involvement first manifested as red, itchy, polygonal, lichenoid papules on the superior and inferior mid back 11 years prior to the current presentation (Figure 1). Further examination noted erosions on the genitalia, and a subsequent biopsy of the vulva confirmed a diagnosis of lichen planus (Figure 2). Treatment with halobetasol propionate ointment and tacrolimus ointment 0.1% twice daily (BID) resulted in remission of the CLP and vulvar lichen planus. She presented a year later with oral involvement revealing Wickham striae on the buccal mucosa and erosions on the upper palate that resolved after 2 months of treatment with cyclosporine oral solution mixed with a 5-times-daily nystatin swish-and-spit (Figure 3). The CLP did not recur but OLP was punctuated by remissions and recurrences on a yearly basis, often related to the cessation of mouthwash and topical creams. The OLP and vulvar lichen planus were successfully treated with as-needed use of a cyclosporine mouthwash swish-and-spit 3 times daily as well as halobetasol ointment 0.05% 3 times daily, respectively. Six years later, the patient was hospitalized for unrelated causes and was lost to follow-up for 2 years.

The patient experienced worsening dysphagia and odynophagia over a period of 2 years (mild dysphagia was first recorded 7 years prior to the initial presentation) and reported an unintentional weight loss of 20 pounds. An endoscopy was performed 3 years after the initial report of dysphagia and noted esophageal erosions (Figure 4A) and a stricture (Figure 4B), but all abnormal involvement was attributed to active gastroesophageal reflux disease. She underwent 8 esophageal dilations to treat the stricture but noted that the duration of symptomatic relief decreased with every subsequent dilation. An esophageal stent was placed 4 years after the initial concern of dysphagia, but it was not well tolerated and had to be removed soon thereafter. A year later, the patient underwent an esophageal bypass with a substernal gastric conduit that provided relief for 2 months but failed to permanently resolve the condition. In fact, her condition worsened over the next 1.5 years when she presented with extreme emaciation attributed to a low appetite and pain while eating. A review of the slides from a prior hospital esophageal biopsy revealed lichen planus (Figure 5). She was prescribed tofacitinib 5 mg BID as a dual-purpose treatment for the rheumatoid arthritis and OLP/ELP. At 1-month follow-up she noted that she had only taken one 5-mg pill daily without notable improvement, and after the visit she started the initial recommendation of 5 mg BID. Over the next several months, her condition continued to consistently improve; the odynophagia resolved, and she regained the majority of her lost weight. Tofacitinib was well tolerated across the course of treatment, and no adverse side effects were noted. Furthermore, the patient regained a full range of motion in the previously immobile arthritic right shoulder. She has experienced no recurrence of the genital lichen planus, OLP, or CLP since starting tofacitinib. To date, the patient is still taking only tofacitinib 5 mg BID with no recurrence of the cutaneous, mucosal, or esophageal lichen planus and has experienced no adverse events from the medication.

Clinical Presentation—Lichen planus—CLP and OLP—most frequently presents between the ages of 40 and 60 years, with a slight female predilection.^1,2 The lesions typically present with the 5 P’s—purple, pruritic, polygonal papules and plaques—with some lesions revealing white lacy lines overlying them called Wickham striae.⁶ The lesions may be red at first before turning purple. They often present on the flexural surfaces of the wrists and ankles as well as the shins and back but rarely affect the face, perhaps because of increased chronic sun exposure.^2,6 Less common locations include the scalp, nails, and mucosal areas (eg, oral, vulvar, conjunctival, laryngeal, esophageal, anal).¹

If CLP is diagnosed, the patient likely will also have oral lesions, which occur in 50% of patients.² Once any form of lichen planus is found, it is important to examine all of the most frequently involved locations—mucocutaneous and cutaneous as well as the nails and scalp. Special care should be taken when examining OLP and genital lichen planus, as long-standing lesions have a 2% to 5% chance of transforming into squamous cell carcinoma.²

Although cases of traditional OLP and CLP are ubiquitous in the literature, ELP rarely is documented because of frequent misdiagnoses. Esophageal lichen planus has a closer histopathologic resemblance to OLP compared to CLP, and its highly variable presentation often results in an inconclusive diagnosis.³ A review of 27 patients with lichen planus highlighted the difficult nature of diagnosing ELP; ELP manifested up to 20 years after initial lichen planus diagnosis, and patients underwent an average of 2.5 dilations prior to the successful diagnosis of ELP. Interestingly, 2 patients in the study presented with ELP in isolation, which emphasizes the importance of secondary examination for lichen planus in the presence of esophageal strictures.⁷ The eTable provides common patient demographics and symptoms to more effectively identify ELP.Differential Diagnosis—Because lichen planus can present anywhere on the body, it may be difficult to differentiate it from other skin conditions. Clinical appearance alone often is insufficient for diagnosing lichen planus, and a punch biopsy often is needed.^2,20 Cutaneous lichen planus may resemble eczema, lichen simplex chronicus, pityriasis rosea, prurigo nodularis, and psoriasis, while OLP may resemble bite trauma, leukoplakia, pemphigus, and thrush.²⁰ Dermoscopy of the tissue makes Wickham striae easier to visualize and assists in the diagnosis of lichen planus. Furthermore, thickening of the stratum granulosum, a prevalence of lymphocytes in the dermoepidermal junction, and vacuolar alteration of the stratum basale help to distinguish between lichen planus and other inflammatory dermatoses.²⁰ A diagnosis of lichen planus merits a full-body skin examination—hair, nails, eyes, oral mucosa, and genitalia—to rule out additional involvement.

Esophageal lichen planus most frequently presents as dysphagia, odynophagia, and weight loss, but other symptoms including heartburn, hoarseness, choking, and epigastric pain may suggest esophageal involvement.⁴ Typically, ELP presents in the proximal and/or central esophagus, assisting in the differentiation between ELP and other esophageal conditions.³ Special consideration should be taken when both ELP and gastroesophageal reflux disease are considered in a differential diagnosis, and it is recommended to pair an upper endoscopy with pH monitoring to avoid misdiagnosis.⁸ Screening endoscopies also are helpful, as they assist in identifying the characteristic white webs, skin peeling, skin surface erosion, and strictures of ELP.⁴ Taken together, dermatologists should encourage patients with cutaneous or mucocutaneous lichen planus to undergo an esophagogastroduodenoscopy, especially in the presence of any of ELP’s common symptoms (eTable).

Etiology—Although the exact etiology of lichen planus is not well established, there are several known correlative factors, including hepatitis C; increased stress; dental materials; oral medications, most frequently antihypertensives and nonsteroidal anti-inflammatory drugs; systemic diseases; and tobacco usage.^6,21

Dental materials used in oral treatments such as silver amalgam, gold, cobalt, palladium, chromium, epoxy resins, and dentures can trigger or exacerbate OLP, and patch testing of a patient’s dental materials can help determine if the reaction was caused by the materials.^6,22 The removal of material contributing to lesions often will cause OLP to resolve.²²

It also has been suggested that the presence of thyroid disorders, autoimmune disease, various cancers, hypertension, type 2 diabetes mellitus, hyperlipidemia, oral sedative usage, and/or vitamin D deficiency may be associated with OLP.^21,23 Although OLP patients who were initially deficient in vitamin D demonstrated marked improvement with supplementation, it is unlikely that vitamin D supplements impacted our patient’s presentation of OLP, as she had been consistently taking them for more than 5 years with no change in OLP presentation.²⁴

Pathogenesis—Lichen planus is thought to be a cytotoxic CD8⁺ T cell–mediated autoimmune disease to a virally modified epidermal self-antigen on keratinocytes. The cytotoxic T cells target the modified self-antigens on basal keratinocytes and induce apoptosis.²⁵ The cytokine-mediated lymphocyte homing mechanism is human leukocyte antigen dependent and involves tumor necrosis factor α as well as IFN-γ and IL-1. The latter cytokines lead to upregulation of vascular adhesion molecules on endothelial vessels of subepithelial vascular plexus as well as a cascade of nonspecific mechanisms such as mast cell degranulation and matrix metalloproteinase activation, resulting in increased basement membrane disruption.⁶

Shao et al¹⁹ underscored the role of IFN-γ in CD8⁺ T cell–mediated cytotoxic cellular responses, noting that the Janus kinase (JAK)–signal transducer and activator of transcription pathway may play a key role in the pathogenesis of lichen planus. They proposed using JAK inhibitors for the treatment of lichen planus, specifically tofacitinib, a JAK1/JAK3 inhibitor, and baricitinib, a JAK1/JAK2 inhibitor, as top therapeutic agents for lichen planus (eTable).¹⁹ Tofacitinib has been reported to successfully treat conditions such as psoriasis, psoriatic arthritis, alopecia areata, vitiligo, atopic dermatitis, sarcoidosis, pyoderma gangrenosum, and lichen planopilaris.²⁶ Additionally, the efficacy of tofacitinib has been established in patients with erosive lichen planus; tofacitinib resulted in marked improvement while prednisone, acitretin, methotrexate, mycophenolate mofetil, and cyclosporine treatment failed.²⁷ Although more studies on tofacitinib’s long-term efficacy, cost, and safety are necessary, tofacitinib may soon play an integral role in the battle against inflammatory dermatoses.

Conclusion

Esophageal lichen planus is an underreported form of lichen planus that often is misdiagnosed. It frequently causes dysphagia and odynophagia, resulting in a major decrease in a patient’s quality of life. We present the case of an 89-year-old woman who underwent procedures to dilate her esophagus that worsened her condition. We emphasize the importance of considering ELP in the differential diagnosis of patients presenting with lichen planus in another region. In our patient, tofacitinib 5 mg BID resolved her condition without any adverse effects.

Dermatology providers are at an increased risk for blood-borne pathogen (BBP) exposures during procedures in clinical practice.^1-3 Current data regarding the characterization of these exposures are limited. Prior studies are based on surveys that result in low response rates and potential for selection bias. Donnelly et al¹ reported a 26% response rate in a national survey-based study evaluating BBP exposures in resident physicians, fellows, and practicing dermatologists, with 85% of respondents reporting at least 1 injury. Similarly, Goulart et al² reported a 35% response rate in a survey evaluating sharps injuries in residents and medical students, with 85% reporting a sharps injury. In addition, there are conflicting data regarding characteristics of these exposures, including common implicated instruments and procedures.^1-3 Prior studies also have not evaluated exposures in all members of dermatologic staff, including resident physicians, practicing dermatologists, and ancillary staff.

To make appropriate quality improvements in dermatologic procedures, a more comprehensive understanding of BBP exposures is needed. We conducted a retrospective review of BBP incidence reports to identify the incidence of BBP events among all dermatologic staff, including resident physicians, practicing dermatologists, and ancillary staff. We further investigated the type of exposure, the type of procedure associated with each exposure, anatomic locations of exposures, and instruments involved in each exposure.

Methods

Data on BBP exposures in the dermatology departments were obtained from the occupational health departments at each of 3 Mayo Clinic sites—Scottsdale, Arizona; Jacksonville, Florida; and Rochester, Minnesota—from March 2010 through January 2021. The institutional review board at Mayo Clinic, Scottsdale, Arizona, granted approval of this study (IRB #20-012625). A retrospective review of each exposure was conducted to identify the incidence of BBP exposures. Occupational BBP exposure was defined as any percutaneous injury or mucosal exposure with foreign blood, tissue, or other bodily fluids that placed the health care worker at risk for communicable infections. Secondary aims included identification of the type of exposure, type of procedure associated with each exposure, common anatomic locations of exposures, and common instruments involved in each exposure.

Statistical Analysis—Variables were summarized using counts and percentages. The 3 most common categories for each variable were then compared among occupational groups using the Fisher exact test. All other categories were grouped for analysis purposes. Medical staff were categorized into 3 occupational groups: practicing dermatologists; resident physicians; and ancillary staff, including nurse/medical assistants, physician assistants, and clinical laboratory technologists. All analyses were 2 sided and considered statistically significant at P<.05. Analyses were performed using SAS 9.4 (SAS Institute Inc).

Results

Type of Exposure—A total of 222 BBP exposures were identified through the trisite retrospective review from March 2010 through January 2021. One hundred ninety-nine (89.6%) of 222 exposures were attributed to needlesticks and medical sharps, while 23 (10.4%) of 222 exposures were attributed to splash incidents (Table).

Anatomic Sites Affected—The anatomic location most frequently involved was the thumb (130/217 events [59.9%]), followed by the hand (39/217 events [18.0%]) and finger (22/217 events [10.1%]). The arm, face, and knee were affected with the lowest frequency, with only 1 event reported at each anatomic site (0.5%)(eTable). Five incidents were excluded from the analysis of anatomic location because of insufficient details of events.

Incident Tasks and Tools—Most BBP exposures occurred during suturing or assisting with suturing (64/210 events [30.5%]), followed by handling of sharps, wires, or instruments (40/210 events [19.0%]) and medication administration (37/210 events [17.6%])(eTable). Twelve incidents were excluded from the analysis of implicated tasks because of insufficient details of events.

The tools involved in exposure events with the greatest prevalence included the suture needle (76/201 events [37.8%]), injection syringe/needle (43/201 events [21.4%]), and shave biopsy razor (24/201 events [11.9%])(eTable). Twenty-one incidents were excluded from the analysis of implicated instruments because of insufficient details of events.

Providers Affected by BBP Exposures—Resident physicians experienced the greatest number of BBP exposures (105/222 events [47.3%]), followed by ancillary providers (84/222 events [37.8%]) and practicing dermatologists (33/222 events [14.9%]). All occupational groups experienced more BBP exposures through needlesticks/medical sharps compared with splash incidents (resident physicians, 88.6%; ancillary staff, 91.7%; practicing dermatologists, 87.9%; P=.725)(Table).

Among resident physicians, practicing dermatologists, and ancillary staff, the most frequent site of injury was the thumb. Suturing/assisting with suturing was the most common task leading to injury, and the suture needle was the most common instrument of injury for both resident physicians and practicing dermatologists. Handling of sharps, wires, or instruments was the most common task leading to injury for ancillary staff, and the injection syringe/needle was the most common instrument of injury in this cohort.

Resident physicians experienced the lowest rate of BBP exposures during administration of medications (12.7%; P=.003). Ancillary staff experienced the highest rate of BBP exposures with an injection needle (35.5%; P=.001). There were no statistically significant differences among occupational groups for the anatomic location of injury (P=.074)(eTable).

Comment

In the year 2000, the annual global incidence of occupational BBP exposures among health care workers worldwide for hepatitis B virus, hepatitis C virus, and HIV was estimated at 2.1 million, 926,000, and 327,000, respectively. Most of these exposures were due to sharps injuries.⁴ Dermatologists are particularly at risk for BBP exposures given their reliance on frequent procedures in practice. During an 11-year period, 222 BBP exposures were documented in the dermatology departments at 3 Mayo Clinic institutions. Most exposures were due to needlestick/sharps across all occupational groups compared with splash injuries. Prior survey studies confirm that sharps injuries are frequently implicated, with 75% to 94% of residents and practicing dermatologists reporting at least 1 sharps injury.¹

Among occupational groups, resident physicians had the highest rate of BBP exposures, followed by nurse/medical assistants and practicing dermatologists, which may be secondary to lack of training or experience. Data from other surgical fields, including general surgery, support that resident physicians have the highest rate of sharps injuries.⁵ In a survey study (N=452), 51% of residents reported that extra training in safe techniques would be beneficial.² Safety training may be beneficial in reducing the incidence of BBP exposures in residency programs.

The most common implicated task in resident physicians and practicing dermatologists was suturing or assisting with suturing, and the most common implicated instrument was the suture needle. Prior studies showed conflicting data regarding common implicated tasks and instruments in this cohort.^1,2 The task of suturing and the suture needle also were the most implicated means of injury among other surgical specialties.⁶ Ancillary staff experienced most BBP exposures during handling of sharps, wires, or instruments, as well as the use of an injection needle. The designation of tasks among dermatologic staff likely explains the difference among occupational groups. This new information may provide the opportunity to improve safety measures among all members of the dermatologic team.

Limitations—There are several limitations to this study. This retrospective review was conducted at a single health system at 3 institutions. Hence, similar safety protocols likely were in place across all sites, which may reduce the generalizability of the results. In addition, there is risk of nonreporting bias among staff, as only documented incidence reports were evaluated. Prior studies demonstrated a nonreporting prevalence of 33% to 64% among dermatology staff.^1-3 We also did not evaluate whether injuries resulted in BBP exposure or transmission. The rates of postexposure prophylaxis also were not studied. This information was not available for review because of concerns for privacy. Demographic features, such as gender or years of training, also were not evaluated.

Conclusion

This study provides additional insight on the incidence of BBP exposures in dermatology, as well as the implicated tasks, instruments, and anatomic locations of injury. Studies show that implementing formal education regarding the risks of BBP exposure may result in reduction of sharps injuries.⁷ Formal education in residency programs may be needed in the field of dermatology to reduce BBP exposures. Quality improvement measures should focus on identified risk factors among occupational groups to reduce BBP exposures in the workplace.

Dermatology providers are at an increased risk for blood-borne pathogen (BBP) exposures during procedures in clinical practice.^1-3 Current data regarding the characterization of these exposures are limited. Prior studies are based on surveys that result in low response rates and potential for selection bias. Donnelly et al¹ reported a 26% response rate in a national survey-based study evaluating BBP exposures in resident physicians, fellows, and practicing dermatologists, with 85% of respondents reporting at least 1 injury. Similarly, Goulart et al² reported a 35% response rate in a survey evaluating sharps injuries in residents and medical students, with 85% reporting a sharps injury. In addition, there are conflicting data regarding characteristics of these exposures, including common implicated instruments and procedures.^1-3 Prior studies also have not evaluated exposures in all members of dermatologic staff, including resident physicians, practicing dermatologists, and ancillary staff.

To make appropriate quality improvements in dermatologic procedures, a more comprehensive understanding of BBP exposures is needed. We conducted a retrospective review of BBP incidence reports to identify the incidence of BBP events among all dermatologic staff, including resident physicians, practicing dermatologists, and ancillary staff. We further investigated the type of exposure, the type of procedure associated with each exposure, anatomic locations of exposures, and instruments involved in each exposure.

Methods

Data on BBP exposures in the dermatology departments were obtained from the occupational health departments at each of 3 Mayo Clinic sites—Scottsdale, Arizona; Jacksonville, Florida; and Rochester, Minnesota—from March 2010 through January 2021. The institutional review board at Mayo Clinic, Scottsdale, Arizona, granted approval of this study (IRB #20-012625). A retrospective review of each exposure was conducted to identify the incidence of BBP exposures. Occupational BBP exposure was defined as any percutaneous injury or mucosal exposure with foreign blood, tissue, or other bodily fluids that placed the health care worker at risk for communicable infections. Secondary aims included identification of the type of exposure, type of procedure associated with each exposure, common anatomic locations of exposures, and common instruments involved in each exposure.

Statistical Analysis—Variables were summarized using counts and percentages. The 3 most common categories for each variable were then compared among occupational groups using the Fisher exact test. All other categories were grouped for analysis purposes. Medical staff were categorized into 3 occupational groups: practicing dermatologists; resident physicians; and ancillary staff, including nurse/medical assistants, physician assistants, and clinical laboratory technologists. All analyses were 2 sided and considered statistically significant at P<.05. Analyses were performed using SAS 9.4 (SAS Institute Inc).

Results

Type of Exposure—A total of 222 BBP exposures were identified through the trisite retrospective review from March 2010 through January 2021. One hundred ninety-nine (89.6%) of 222 exposures were attributed to needlesticks and medical sharps, while 23 (10.4%) of 222 exposures were attributed to splash incidents (Table).

Anatomic Sites Affected—The anatomic location most frequently involved was the thumb (130/217 events [59.9%]), followed by the hand (39/217 events [18.0%]) and finger (22/217 events [10.1%]). The arm, face, and knee were affected with the lowest frequency, with only 1 event reported at each anatomic site (0.5%)(eTable). Five incidents were excluded from the analysis of anatomic location because of insufficient details of events.

Incident Tasks and Tools—Most BBP exposures occurred during suturing or assisting with suturing (64/210 events [30.5%]), followed by handling of sharps, wires, or instruments (40/210 events [19.0%]) and medication administration (37/210 events [17.6%])(eTable). Twelve incidents were excluded from the analysis of implicated tasks because of insufficient details of events.

The tools involved in exposure events with the greatest prevalence included the suture needle (76/201 events [37.8%]), injection syringe/needle (43/201 events [21.4%]), and shave biopsy razor (24/201 events [11.9%])(eTable). Twenty-one incidents were excluded from the analysis of implicated instruments because of insufficient details of events.

Providers Affected by BBP Exposures—Resident physicians experienced the greatest number of BBP exposures (105/222 events [47.3%]), followed by ancillary providers (84/222 events [37.8%]) and practicing dermatologists (33/222 events [14.9%]). All occupational groups experienced more BBP exposures through needlesticks/medical sharps compared with splash incidents (resident physicians, 88.6%; ancillary staff, 91.7%; practicing dermatologists, 87.9%; P=.725)(Table).

Among resident physicians, practicing dermatologists, and ancillary staff, the most frequent site of injury was the thumb. Suturing/assisting with suturing was the most common task leading to injury, and the suture needle was the most common instrument of injury for both resident physicians and practicing dermatologists. Handling of sharps, wires, or instruments was the most common task leading to injury for ancillary staff, and the injection syringe/needle was the most common instrument of injury in this cohort.

Resident physicians experienced the lowest rate of BBP exposures during administration of medications (12.7%; P=.003). Ancillary staff experienced the highest rate of BBP exposures with an injection needle (35.5%; P=.001). There were no statistically significant differences among occupational groups for the anatomic location of injury (P=.074)(eTable).

Comment

In the year 2000, the annual global incidence of occupational BBP exposures among health care workers worldwide for hepatitis B virus, hepatitis C virus, and HIV was estimated at 2.1 million, 926,000, and 327,000, respectively. Most of these exposures were due to sharps injuries.⁴ Dermatologists are particularly at risk for BBP exposures given their reliance on frequent procedures in practice. During an 11-year period, 222 BBP exposures were documented in the dermatology departments at 3 Mayo Clinic institutions. Most exposures were due to needlestick/sharps across all occupational groups compared with splash injuries. Prior survey studies confirm that sharps injuries are frequently implicated, with 75% to 94% of residents and practicing dermatologists reporting at least 1 sharps injury.¹

Among occupational groups, resident physicians had the highest rate of BBP exposures, followed by nurse/medical assistants and practicing dermatologists, which may be secondary to lack of training or experience. Data from other surgical fields, including general surgery, support that resident physicians have the highest rate of sharps injuries.⁵ In a survey study (N=452), 51% of residents reported that extra training in safe techniques would be beneficial.² Safety training may be beneficial in reducing the incidence of BBP exposures in residency programs.

The most common implicated task in resident physicians and practicing dermatologists was suturing or assisting with suturing, and the most common implicated instrument was the suture needle. Prior studies showed conflicting data regarding common implicated tasks and instruments in this cohort.^1,2 The task of suturing and the suture needle also were the most implicated means of injury among other surgical specialties.⁶ Ancillary staff experienced most BBP exposures during handling of sharps, wires, or instruments, as well as the use of an injection needle. The designation of tasks among dermatologic staff likely explains the difference among occupational groups. This new information may provide the opportunity to improve safety measures among all members of the dermatologic team.

Limitations—There are several limitations to this study. This retrospective review was conducted at a single health system at 3 institutions. Hence, similar safety protocols likely were in place across all sites, which may reduce the generalizability of the results. In addition, there is risk of nonreporting bias among staff, as only documented incidence reports were evaluated. Prior studies demonstrated a nonreporting prevalence of 33% to 64% among dermatology staff.^1-3 We also did not evaluate whether injuries resulted in BBP exposure or transmission. The rates of postexposure prophylaxis also were not studied. This information was not available for review because of concerns for privacy. Demographic features, such as gender or years of training, also were not evaluated.

Conclusion

This study provides additional insight on the incidence of BBP exposures in dermatology, as well as the implicated tasks, instruments, and anatomic locations of injury. Studies show that implementing formal education regarding the risks of BBP exposure may result in reduction of sharps injuries.⁷ Formal education in residency programs may be needed in the field of dermatology to reduce BBP exposures. Quality improvement measures should focus on identified risk factors among occupational groups to reduce BBP exposures in the workplace.

Dermatology providers are at an increased risk for blood-borne pathogen (BBP) exposures during procedures in clinical practice.^1-3 Current data regarding the characterization of these exposures are limited. Prior studies are based on surveys that result in low response rates and potential for selection bias. Donnelly et al¹ reported a 26% response rate in a national survey-based study evaluating BBP exposures in resident physicians, fellows, and practicing dermatologists, with 85% of respondents reporting at least 1 injury. Similarly, Goulart et al² reported a 35% response rate in a survey evaluating sharps injuries in residents and medical students, with 85% reporting a sharps injury. In addition, there are conflicting data regarding characteristics of these exposures, including common implicated instruments and procedures.^1-3 Prior studies also have not evaluated exposures in all members of dermatologic staff, including resident physicians, practicing dermatologists, and ancillary staff.

To make appropriate quality improvements in dermatologic procedures, a more comprehensive understanding of BBP exposures is needed. We conducted a retrospective review of BBP incidence reports to identify the incidence of BBP events among all dermatologic staff, including resident physicians, practicing dermatologists, and ancillary staff. We further investigated the type of exposure, the type of procedure associated with each exposure, anatomic locations of exposures, and instruments involved in each exposure.

Methods

Data on BBP exposures in the dermatology departments were obtained from the occupational health departments at each of 3 Mayo Clinic sites—Scottsdale, Arizona; Jacksonville, Florida; and Rochester, Minnesota—from March 2010 through January 2021. The institutional review board at Mayo Clinic, Scottsdale, Arizona, granted approval of this study (IRB #20-012625). A retrospective review of each exposure was conducted to identify the incidence of BBP exposures. Occupational BBP exposure was defined as any percutaneous injury or mucosal exposure with foreign blood, tissue, or other bodily fluids that placed the health care worker at risk for communicable infections. Secondary aims included identification of the type of exposure, type of procedure associated with each exposure, common anatomic locations of exposures, and common instruments involved in each exposure.

Statistical Analysis—Variables were summarized using counts and percentages. The 3 most common categories for each variable were then compared among occupational groups using the Fisher exact test. All other categories were grouped for analysis purposes. Medical staff were categorized into 3 occupational groups: practicing dermatologists; resident physicians; and ancillary staff, including nurse/medical assistants, physician assistants, and clinical laboratory technologists. All analyses were 2 sided and considered statistically significant at P<.05. Analyses were performed using SAS 9.4 (SAS Institute Inc).

Results

Type of Exposure—A total of 222 BBP exposures were identified through the trisite retrospective review from March 2010 through January 2021. One hundred ninety-nine (89.6%) of 222 exposures were attributed to needlesticks and medical sharps, while 23 (10.4%) of 222 exposures were attributed to splash incidents (Table).

Anatomic Sites Affected—The anatomic location most frequently involved was the thumb (130/217 events [59.9%]), followed by the hand (39/217 events [18.0%]) and finger (22/217 events [10.1%]). The arm, face, and knee were affected with the lowest frequency, with only 1 event reported at each anatomic site (0.5%)(eTable). Five incidents were excluded from the analysis of anatomic location because of insufficient details of events.

Incident Tasks and Tools—Most BBP exposures occurred during suturing or assisting with suturing (64/210 events [30.5%]), followed by handling of sharps, wires, or instruments (40/210 events [19.0%]) and medication administration (37/210 events [17.6%])(eTable). Twelve incidents were excluded from the analysis of implicated tasks because of insufficient details of events.

The tools involved in exposure events with the greatest prevalence included the suture needle (76/201 events [37.8%]), injection syringe/needle (43/201 events [21.4%]), and shave biopsy razor (24/201 events [11.9%])(eTable). Twenty-one incidents were excluded from the analysis of implicated instruments because of insufficient details of events.

Providers Affected by BBP Exposures—Resident physicians experienced the greatest number of BBP exposures (105/222 events [47.3%]), followed by ancillary providers (84/222 events [37.8%]) and practicing dermatologists (33/222 events [14.9%]). All occupational groups experienced more BBP exposures through needlesticks/medical sharps compared with splash incidents (resident physicians, 88.6%; ancillary staff, 91.7%; practicing dermatologists, 87.9%; P=.725)(Table).

Among resident physicians, practicing dermatologists, and ancillary staff, the most frequent site of injury was the thumb. Suturing/assisting with suturing was the most common task leading to injury, and the suture needle was the most common instrument of injury for both resident physicians and practicing dermatologists. Handling of sharps, wires, or instruments was the most common task leading to injury for ancillary staff, and the injection syringe/needle was the most common instrument of injury in this cohort.

Resident physicians experienced the lowest rate of BBP exposures during administration of medications (12.7%; P=.003). Ancillary staff experienced the highest rate of BBP exposures with an injection needle (35.5%; P=.001). There were no statistically significant differences among occupational groups for the anatomic location of injury (P=.074)(eTable).

Comment

In the year 2000, the annual global incidence of occupational BBP exposures among health care workers worldwide for hepatitis B virus, hepatitis C virus, and HIV was estimated at 2.1 million, 926,000, and 327,000, respectively. Most of these exposures were due to sharps injuries.⁴ Dermatologists are particularly at risk for BBP exposures given their reliance on frequent procedures in practice. During an 11-year period, 222 BBP exposures were documented in the dermatology departments at 3 Mayo Clinic institutions. Most exposures were due to needlestick/sharps across all occupational groups compared with splash injuries. Prior survey studies confirm that sharps injuries are frequently implicated, with 75% to 94% of residents and practicing dermatologists reporting at least 1 sharps injury.¹

Among occupational groups, resident physicians had the highest rate of BBP exposures, followed by nurse/medical assistants and practicing dermatologists, which may be secondary to lack of training or experience. Data from other surgical fields, including general surgery, support that resident physicians have the highest rate of sharps injuries.⁵ In a survey study (N=452), 51% of residents reported that extra training in safe techniques would be beneficial.² Safety training may be beneficial in reducing the incidence of BBP exposures in residency programs.

The most common implicated task in resident physicians and practicing dermatologists was suturing or assisting with suturing, and the most common implicated instrument was the suture needle. Prior studies showed conflicting data regarding common implicated tasks and instruments in this cohort.^1,2 The task of suturing and the suture needle also were the most implicated means of injury among other surgical specialties.⁶ Ancillary staff experienced most BBP exposures during handling of sharps, wires, or instruments, as well as the use of an injection needle. The designation of tasks among dermatologic staff likely explains the difference among occupational groups. This new information may provide the opportunity to improve safety measures among all members of the dermatologic team.

Limitations—There are several limitations to this study. This retrospective review was conducted at a single health system at 3 institutions. Hence, similar safety protocols likely were in place across all sites, which may reduce the generalizability of the results. In addition, there is risk of nonreporting bias among staff, as only documented incidence reports were evaluated. Prior studies demonstrated a nonreporting prevalence of 33% to 64% among dermatology staff.^1-3 We also did not evaluate whether injuries resulted in BBP exposure or transmission. The rates of postexposure prophylaxis also were not studied. This information was not available for review because of concerns for privacy. Demographic features, such as gender or years of training, also were not evaluated.

Conclusion

This study provides additional insight on the incidence of BBP exposures in dermatology, as well as the implicated tasks, instruments, and anatomic locations of injury. Studies show that implementing formal education regarding the risks of BBP exposure may result in reduction of sharps injuries.⁷ Formal education in residency programs may be needed in the field of dermatology to reduce BBP exposures. Quality improvement measures should focus on identified risk factors among occupational groups to reduce BBP exposures in the workplace.

Two diabetes management devices that aid in the precision of insulin delivery have been recently cleared by the Food and Drug Administration.

On March 2, the FDA cleared the Android version of Bigfoot Biomedical’s Unity Mobile App for use with its system of smart pen caps that are compatible with different disposable insulin pens for administering both long-acting and rapid-acting insulin.

The system, which has been compatible with iOS devices since May 2021, is “the first and only FDA-cleared smart injection system that turns CGM [continuous glucose monitoring] data into dosing recommendations displayed right on the pen cap for people using multiple daily [insulin] injection therapy,” according to a company statement.

The Bigfoot app allows users to input and review provider treatment recommendations, displays current glucose ranges, and delivers real-time alerts.

Once it is commercially launched, the Android phone application will be available via the Google Play Store. “Given that 41% of U.S. smartphone users choose Android devices, this clearance enables expanded access to a large group of people with diabetes,” the company said.

On March 6, the FDA cleared the Abbott FreeStyle Libre 2 and FreeStyle Libre 3 devices as “integrated” CGM sensors. This means that they can now be used as components in automated insulin delivery systems, along with insulin pumps and connectivity software.

Abbott is working with insulin pump manufacturers Insulet and Tandem in the United States for integration with the FreeStyle Libre versions 2 and 3. Outside the United States, the Libre 3 is already authorized to work with mylife Loop from Ypsomed and CamDiab in Germany. Further launches are expected in the United Kingdom, Switzerland, and the Netherlands later this year.

The modified FreeStyle Libre 2 and FreeStyle Libre 3 sensors have been cleared for use by patients as young as age 2 years and for up to 15 days, in contrast to the previous versions, which were available for patients as young as 4 years for use up to 14 days. The FDA has cleared all Libre sensors – 2 and 3, current and future versions – for use by pregnant women with any type of diabetes.

The modified sensors will be available in the United States later this year and will eventually replace the Libre sensors in current use, the company said in a statement.

“The FreeStyle Libre portfolio is still the most affordable CGM on the market,” an Abbott representative said in an interview.

A version of this article first appeared on Medscape.com.

Two diabetes management devices that aid in the precision of insulin delivery have been recently cleared by the Food and Drug Administration.

On March 2, the FDA cleared the Android version of Bigfoot Biomedical’s Unity Mobile App for use with its system of smart pen caps that are compatible with different disposable insulin pens for administering both long-acting and rapid-acting insulin.

The system, which has been compatible with iOS devices since May 2021, is “the first and only FDA-cleared smart injection system that turns CGM [continuous glucose monitoring] data into dosing recommendations displayed right on the pen cap for people using multiple daily [insulin] injection therapy,” according to a company statement.

The Bigfoot app allows users to input and review provider treatment recommendations, displays current glucose ranges, and delivers real-time alerts.

Once it is commercially launched, the Android phone application will be available via the Google Play Store. “Given that 41% of U.S. smartphone users choose Android devices, this clearance enables expanded access to a large group of people with diabetes,” the company said.

On March 6, the FDA cleared the Abbott FreeStyle Libre 2 and FreeStyle Libre 3 devices as “integrated” CGM sensors. This means that they can now be used as components in automated insulin delivery systems, along with insulin pumps and connectivity software.

Abbott is working with insulin pump manufacturers Insulet and Tandem in the United States for integration with the FreeStyle Libre versions 2 and 3. Outside the United States, the Libre 3 is already authorized to work with mylife Loop from Ypsomed and CamDiab in Germany. Further launches are expected in the United Kingdom, Switzerland, and the Netherlands later this year.

The modified FreeStyle Libre 2 and FreeStyle Libre 3 sensors have been cleared for use by patients as young as age 2 years and for up to 15 days, in contrast to the previous versions, which were available for patients as young as 4 years for use up to 14 days. The FDA has cleared all Libre sensors – 2 and 3, current and future versions – for use by pregnant women with any type of diabetes.

The modified sensors will be available in the United States later this year and will eventually replace the Libre sensors in current use, the company said in a statement.

“The FreeStyle Libre portfolio is still the most affordable CGM on the market,” an Abbott representative said in an interview.

A version of this article first appeared on Medscape.com.

Two diabetes management devices that aid in the precision of insulin delivery have been recently cleared by the Food and Drug Administration.

On March 2, the FDA cleared the Android version of Bigfoot Biomedical’s Unity Mobile App for use with its system of smart pen caps that are compatible with different disposable insulin pens for administering both long-acting and rapid-acting insulin.

The system, which has been compatible with iOS devices since May 2021, is “the first and only FDA-cleared smart injection system that turns CGM [continuous glucose monitoring] data into dosing recommendations displayed right on the pen cap for people using multiple daily [insulin] injection therapy,” according to a company statement.

The Bigfoot app allows users to input and review provider treatment recommendations, displays current glucose ranges, and delivers real-time alerts.

Once it is commercially launched, the Android phone application will be available via the Google Play Store. “Given that 41% of U.S. smartphone users choose Android devices, this clearance enables expanded access to a large group of people with diabetes,” the company said.

On March 6, the FDA cleared the Abbott FreeStyle Libre 2 and FreeStyle Libre 3 devices as “integrated” CGM sensors. This means that they can now be used as components in automated insulin delivery systems, along with insulin pumps and connectivity software.

Abbott is working with insulin pump manufacturers Insulet and Tandem in the United States for integration with the FreeStyle Libre versions 2 and 3. Outside the United States, the Libre 3 is already authorized to work with mylife Loop from Ypsomed and CamDiab in Germany. Further launches are expected in the United Kingdom, Switzerland, and the Netherlands later this year.

The modified FreeStyle Libre 2 and FreeStyle Libre 3 sensors have been cleared for use by patients as young as age 2 years and for up to 15 days, in contrast to the previous versions, which were available for patients as young as 4 years for use up to 14 days. The FDA has cleared all Libre sensors – 2 and 3, current and future versions – for use by pregnant women with any type of diabetes.

The modified sensors will be available in the United States later this year and will eventually replace the Libre sensors in current use, the company said in a statement.

“The FreeStyle Libre portfolio is still the most affordable CGM on the market,” an Abbott representative said in an interview.

A version of this article first appeared on Medscape.com.

The American Psychiatric Association has released updated practice guidelines for the management of eating disorders, the first update in 16 years.

The updated guidelines focus primarily on anorexia nervosa (AN), bulimia nervosa (BN), and binge-eating disorder (BED) and include recommendations for screening and treatment.

“Eating disorders often are unrecognized and untreated,” Catherine Crone, MD, chair of the guideline writing group, said in a statement from APA. “This guideline and supplementary resources are intended to serve as a practical tool for clinicians, to help with screening, diagnosis, and providing evidence-based treatment for eating disorders.”

Approximately one in five children worldwide are at risk for developing an eating disorder and U.S. medical admissions for adolescents with restrictive eating disorders more than doubled during the pandemic.

The economic cost of eating disorders in the United States from 2018 to 2019 was an estimated $64.7 billion, the report notes, with an additional $326.5 billion attributable to reductions in well-being associated with eating disorders.

The executive summary of the updated guidelines was published online in The American Journal of Psychiatry.

The practice guideline, which was approved at the 2021 APA annual meeting, features 16 recommendations for clinicians, including screening patients for eating disorders as part of an initial psychiatric evaluation and conducting comprehensive patient evaluations that incorporate laboratory tests and electrocardiograms.

Recommendations also include setting individualized weight goals for patients with anorexia and incorporating family-based therapy as part of a treatment plan for adolescents with anorexia or bulimia.

“This practice guideline aims to help clinicians improve care for their patients by reviewing current evidence and providing evidence-based statements that are intended to enhance knowledge, increase assessment, and optimize treatment of eating disorders,” the authors wrote.

A range of other resources were released with the new guidelines to provide clinicians with support to implement the recommendations, including a pocket guide for clinicians, continuing medical education activities, and slides. The association is also launching a pocket guide for patients and families and an interactive tool kit with a screening assessment calculator.

The APA guidelines follow the 2021 release by the American Academy of Pediatrics on diagnosing and managing eating disorders in children and adolescents.

The development of the guidelines was supported by a grant from the Council of Medical Specialty Societies.

A version of this article first appeared on Medscape.com.

The American Psychiatric Association has released updated practice guidelines for the management of eating disorders, the first update in 16 years.

The updated guidelines focus primarily on anorexia nervosa (AN), bulimia nervosa (BN), and binge-eating disorder (BED) and include recommendations for screening and treatment.

“Eating disorders often are unrecognized and untreated,” Catherine Crone, MD, chair of the guideline writing group, said in a statement from APA. “This guideline and supplementary resources are intended to serve as a practical tool for clinicians, to help with screening, diagnosis, and providing evidence-based treatment for eating disorders.”

Approximately one in five children worldwide are at risk for developing an eating disorder and U.S. medical admissions for adolescents with restrictive eating disorders more than doubled during the pandemic.

The economic cost of eating disorders in the United States from 2018 to 2019 was an estimated $64.7 billion, the report notes, with an additional $326.5 billion attributable to reductions in well-being associated with eating disorders.

The executive summary of the updated guidelines was published online in The American Journal of Psychiatry.

The practice guideline, which was approved at the 2021 APA annual meeting, features 16 recommendations for clinicians, including screening patients for eating disorders as part of an initial psychiatric evaluation and conducting comprehensive patient evaluations that incorporate laboratory tests and electrocardiograms.

Recommendations also include setting individualized weight goals for patients with anorexia and incorporating family-based therapy as part of a treatment plan for adolescents with anorexia or bulimia.

“This practice guideline aims to help clinicians improve care for their patients by reviewing current evidence and providing evidence-based statements that are intended to enhance knowledge, increase assessment, and optimize treatment of eating disorders,” the authors wrote.

A range of other resources were released with the new guidelines to provide clinicians with support to implement the recommendations, including a pocket guide for clinicians, continuing medical education activities, and slides. The association is also launching a pocket guide for patients and families and an interactive tool kit with a screening assessment calculator.

The APA guidelines follow the 2021 release by the American Academy of Pediatrics on diagnosing and managing eating disorders in children and adolescents.

The development of the guidelines was supported by a grant from the Council of Medical Specialty Societies.

A version of this article first appeared on Medscape.com.

The American Psychiatric Association has released updated practice guidelines for the management of eating disorders, the first update in 16 years.

The updated guidelines focus primarily on anorexia nervosa (AN), bulimia nervosa (BN), and binge-eating disorder (BED) and include recommendations for screening and treatment.

“Eating disorders often are unrecognized and untreated,” Catherine Crone, MD, chair of the guideline writing group, said in a statement from APA. “This guideline and supplementary resources are intended to serve as a practical tool for clinicians, to help with screening, diagnosis, and providing evidence-based treatment for eating disorders.”

Approximately one in five children worldwide are at risk for developing an eating disorder and U.S. medical admissions for adolescents with restrictive eating disorders more than doubled during the pandemic.

The economic cost of eating disorders in the United States from 2018 to 2019 was an estimated $64.7 billion, the report notes, with an additional $326.5 billion attributable to reductions in well-being associated with eating disorders.

The executive summary of the updated guidelines was published online in The American Journal of Psychiatry.

The practice guideline, which was approved at the 2021 APA annual meeting, features 16 recommendations for clinicians, including screening patients for eating disorders as part of an initial psychiatric evaluation and conducting comprehensive patient evaluations that incorporate laboratory tests and electrocardiograms.

Recommendations also include setting individualized weight goals for patients with anorexia and incorporating family-based therapy as part of a treatment plan for adolescents with anorexia or bulimia.

“This practice guideline aims to help clinicians improve care for their patients by reviewing current evidence and providing evidence-based statements that are intended to enhance knowledge, increase assessment, and optimize treatment of eating disorders,” the authors wrote.

A range of other resources were released with the new guidelines to provide clinicians with support to implement the recommendations, including a pocket guide for clinicians, continuing medical education activities, and slides. The association is also launching a pocket guide for patients and families and an interactive tool kit with a screening assessment calculator.

The APA guidelines follow the 2021 release by the American Academy of Pediatrics on diagnosing and managing eating disorders in children and adolescents.

The development of the guidelines was supported by a grant from the Council of Medical Specialty Societies.

A version of this article first appeared on Medscape.com.

An incisionless surgical procedure that uses focused ultrasound ablation (FUSA) to target the globus pallidus internus of patients with Parkinson’s disease significantly reduced tremors and improved mobility for those with advanced disease, new research shows.

The technique requires no sedation or brain implants. Surgeons use MRI to identify the globus pallidus internus, a part of the basal ganglia involved in movement disorders, and a focused ultrasound beam to heat and destroy the tissue.

Investigators performed the procedure with a device called Exablate Neuro, which was first approved by the Food and Drug Administration in 2016 to treat essential tremor.

On the basis of the results of a multicenter, randomized, sham-controlled trial, the agency expanded the indication in 2021 to include unilateral pallidotomy to treat advanced Parkinson’s disease for patients with mobility, rigidity, or dyskinesia symptoms.

“In some patients with Parkinson’s disease, you get dyskinesias, and ablation of the globus pallidus significantly reduces those dyskinesias and motor impairment,” said lead investigator Vibhor Krishna, MD, associate professor of neurosurgery at the University of North Carolina at Chapel Hill. “It could be used to treat patients when other surgical procedures can’t be applied.”

The study was published online in the New England Journal of Medicine.
 

Strong response

For the study, 94 patients with advanced Parkinson’s disease who had dyskinesias or motor fluctuations and motor impairment in the off-medication state wore transducer helmets while lying in an MRI scanner. Patients were awake during the entire procedure.

The treatment group received unilateral FUSA on the side of the brain with the greatest motor impairment. The device initially delivered target temperatures of 40°-45° C. Ablative temperatures were gradually increased following evaluations to test for improvement of motor symptoms. The maximum temperature used was 54.3° C.

Patients in the control group underwent an identical procedure with the sonication energy disabled.

The primary outcome was a response to therapy at 3 months, defined as a decrease of at least three points from baseline either in the score on the Movement Disorders Society–Unified Parkinson’s Disease Rating Scale (MDS-UPDRS), part III, while off medication or in the score on the Unified Dyskinesia Rating Scale (UDRS) while on medication.

At 3 months, 69% of the treatment group reported a response, compared with 32% of the control group (P = .003).

When researchers analyzed MDS-UPDRS scores, they found that 29% of the treatment group and 27% of the control group showed improvement. For UDRS scores, 12% of the treatment group demonstrated improvement. In the control group, there was no improvement on this score. Improvements in both scores were reported in 28% of the treatment group and 5% of the control group.

Among those who reported a response at 3 months, 77% continued to show a response at 12 months.
 

‘Unforgiving’ area of the brain

While the response rate was a promising sign of this finding, it was not what interested Dr. Krishna the most. “The most surprising finding of this trial is how safe focused ultrasound pallidotomy is in treating patients with Parkinson’s disease,” he said.

The globus pallidus internus is an area of the brain that Dr. Krishna calls “unforgiving.”

“One side is motor fibers, and any problem with that can paralyze the patient, and just below that is the optic tract, and any problem there, you would lose vision,” Dr. Krishna said. “It is a very tough neighborhood to be in.”

By using MRI-guided ultrasound, surgeons can change the target and temperature of the ultrasound beam during the procedure to allow more precise treatment.

Pallidotomy-related adverse events in the treatment group included dysarthria, gait disturbance, loss of taste, visual disturbance, and facial weakness. All were mild to moderate, Dr. Krishna said.
 

More study is needed

Dyskinesia is a challenge in the management of Parkinson’s disease. Patients need antiparkinsonian medications to slow deterioration of motor function, but those medications can cause the involuntary movements that are a hallmark of dyskinesia.

The most common treatment for this complication, deep-brain stimulation (DBS), has its own drawbacks. It’s an open procedure, and there is a low-level risk for intracranial bleeding and infection. In addition, the electrode implants require ongoing maintenance and adjustment.

But the findings of this study show that, for patients who aren’t candidates for other therapies, such as DBS and ablative radiofrequency, FUSA may be an alternative, wrote Anette Schrag, PhD, professor of clinical neurosciences at University College London, in an accompanying commentary.

“The results confirm that it is effective in reducing motor complications of Parkinson’s disease, at least in the short term,” Dr. Schrag wrote. However, more long-term studies are needed, she added.

One-third of patients in the treatment group had no response to the treatment, and investigators aren’t sure why. Dr. Krishna noted that the benefits of the procedure waned in about a quarter of patients within a year of treatment.

Investigators plan to probe these questions in future trials.

“The results of this trial are promising,” Dr. Schrag wrote, “but given the nonreversible nature of the intervention and the progressive nature of the disease, it will be important to establish whether improvements in motor complications are maintained over longer periods and whether treatment results in improved overall functioning and quality of life for patients.”

The study was funded by Insightec. Disclosure forms for Dr. Krishna and Dr. Schrag are provided on the journal’s website.

A version of this article originally appeared on Medscape.com.

An incisionless surgical procedure that uses focused ultrasound ablation (FUSA) to target the globus pallidus internus of patients with Parkinson’s disease significantly reduced tremors and improved mobility for those with advanced disease, new research shows.

The technique requires no sedation or brain implants. Surgeons use MRI to identify the globus pallidus internus, a part of the basal ganglia involved in movement disorders, and a focused ultrasound beam to heat and destroy the tissue.

Investigators performed the procedure with a device called Exablate Neuro, which was first approved by the Food and Drug Administration in 2016 to treat essential tremor.

On the basis of the results of a multicenter, randomized, sham-controlled trial, the agency expanded the indication in 2021 to include unilateral pallidotomy to treat advanced Parkinson’s disease for patients with mobility, rigidity, or dyskinesia symptoms.

“In some patients with Parkinson’s disease, you get dyskinesias, and ablation of the globus pallidus significantly reduces those dyskinesias and motor impairment,” said lead investigator Vibhor Krishna, MD, associate professor of neurosurgery at the University of North Carolina at Chapel Hill. “It could be used to treat patients when other surgical procedures can’t be applied.”

The study was published online in the New England Journal of Medicine.
 

Strong response

For the study, 94 patients with advanced Parkinson’s disease who had dyskinesias or motor fluctuations and motor impairment in the off-medication state wore transducer helmets while lying in an MRI scanner. Patients were awake during the entire procedure.

The treatment group received unilateral FUSA on the side of the brain with the greatest motor impairment. The device initially delivered target temperatures of 40°-45° C. Ablative temperatures were gradually increased following evaluations to test for improvement of motor symptoms. The maximum temperature used was 54.3° C.

Patients in the control group underwent an identical procedure with the sonication energy disabled.

The primary outcome was a response to therapy at 3 months, defined as a decrease of at least three points from baseline either in the score on the Movement Disorders Society–Unified Parkinson’s Disease Rating Scale (MDS-UPDRS), part III, while off medication or in the score on the Unified Dyskinesia Rating Scale (UDRS) while on medication.

At 3 months, 69% of the treatment group reported a response, compared with 32% of the control group (P = .003).

When researchers analyzed MDS-UPDRS scores, they found that 29% of the treatment group and 27% of the control group showed improvement. For UDRS scores, 12% of the treatment group demonstrated improvement. In the control group, there was no improvement on this score. Improvements in both scores were reported in 28% of the treatment group and 5% of the control group.

Among those who reported a response at 3 months, 77% continued to show a response at 12 months.
 

‘Unforgiving’ area of the brain

While the response rate was a promising sign of this finding, it was not what interested Dr. Krishna the most. “The most surprising finding of this trial is how safe focused ultrasound pallidotomy is in treating patients with Parkinson’s disease,” he said.

The globus pallidus internus is an area of the brain that Dr. Krishna calls “unforgiving.”

“One side is motor fibers, and any problem with that can paralyze the patient, and just below that is the optic tract, and any problem there, you would lose vision,” Dr. Krishna said. “It is a very tough neighborhood to be in.”

By using MRI-guided ultrasound, surgeons can change the target and temperature of the ultrasound beam during the procedure to allow more precise treatment.

Pallidotomy-related adverse events in the treatment group included dysarthria, gait disturbance, loss of taste, visual disturbance, and facial weakness. All were mild to moderate, Dr. Krishna said.
 

More study is needed

Dyskinesia is a challenge in the management of Parkinson’s disease. Patients need antiparkinsonian medications to slow deterioration of motor function, but those medications can cause the involuntary movements that are a hallmark of dyskinesia.

The most common treatment for this complication, deep-brain stimulation (DBS), has its own drawbacks. It’s an open procedure, and there is a low-level risk for intracranial bleeding and infection. In addition, the electrode implants require ongoing maintenance and adjustment.

But the findings of this study show that, for patients who aren’t candidates for other therapies, such as DBS and ablative radiofrequency, FUSA may be an alternative, wrote Anette Schrag, PhD, professor of clinical neurosciences at University College London, in an accompanying commentary.

“The results confirm that it is effective in reducing motor complications of Parkinson’s disease, at least in the short term,” Dr. Schrag wrote. However, more long-term studies are needed, she added.

One-third of patients in the treatment group had no response to the treatment, and investigators aren’t sure why. Dr. Krishna noted that the benefits of the procedure waned in about a quarter of patients within a year of treatment.

Investigators plan to probe these questions in future trials.

“The results of this trial are promising,” Dr. Schrag wrote, “but given the nonreversible nature of the intervention and the progressive nature of the disease, it will be important to establish whether improvements in motor complications are maintained over longer periods and whether treatment results in improved overall functioning and quality of life for patients.”

The study was funded by Insightec. Disclosure forms for Dr. Krishna and Dr. Schrag are provided on the journal’s website.

A version of this article originally appeared on Medscape.com.

An incisionless surgical procedure that uses focused ultrasound ablation (FUSA) to target the globus pallidus internus of patients with Parkinson’s disease significantly reduced tremors and improved mobility for those with advanced disease, new research shows.

The technique requires no sedation or brain implants. Surgeons use MRI to identify the globus pallidus internus, a part of the basal ganglia involved in movement disorders, and a focused ultrasound beam to heat and destroy the tissue.

Investigators performed the procedure with a device called Exablate Neuro, which was first approved by the Food and Drug Administration in 2016 to treat essential tremor.

On the basis of the results of a multicenter, randomized, sham-controlled trial, the agency expanded the indication in 2021 to include unilateral pallidotomy to treat advanced Parkinson’s disease for patients with mobility, rigidity, or dyskinesia symptoms.

“In some patients with Parkinson’s disease, you get dyskinesias, and ablation of the globus pallidus significantly reduces those dyskinesias and motor impairment,” said lead investigator Vibhor Krishna, MD, associate professor of neurosurgery at the University of North Carolina at Chapel Hill. “It could be used to treat patients when other surgical procedures can’t be applied.”

The study was published online in the New England Journal of Medicine.
 

Strong response

For the study, 94 patients with advanced Parkinson’s disease who had dyskinesias or motor fluctuations and motor impairment in the off-medication state wore transducer helmets while lying in an MRI scanner. Patients were awake during the entire procedure.

The treatment group received unilateral FUSA on the side of the brain with the greatest motor impairment. The device initially delivered target temperatures of 40°-45° C. Ablative temperatures were gradually increased following evaluations to test for improvement of motor symptoms. The maximum temperature used was 54.3° C.

Patients in the control group underwent an identical procedure with the sonication energy disabled.

The primary outcome was a response to therapy at 3 months, defined as a decrease of at least three points from baseline either in the score on the Movement Disorders Society–Unified Parkinson’s Disease Rating Scale (MDS-UPDRS), part III, while off medication or in the score on the Unified Dyskinesia Rating Scale (UDRS) while on medication.

At 3 months, 69% of the treatment group reported a response, compared with 32% of the control group (P = .003).

When researchers analyzed MDS-UPDRS scores, they found that 29% of the treatment group and 27% of the control group showed improvement. For UDRS scores, 12% of the treatment group demonstrated improvement. In the control group, there was no improvement on this score. Improvements in both scores were reported in 28% of the treatment group and 5% of the control group.

Among those who reported a response at 3 months, 77% continued to show a response at 12 months.
 

‘Unforgiving’ area of the brain

While the response rate was a promising sign of this finding, it was not what interested Dr. Krishna the most. “The most surprising finding of this trial is how safe focused ultrasound pallidotomy is in treating patients with Parkinson’s disease,” he said.

The globus pallidus internus is an area of the brain that Dr. Krishna calls “unforgiving.”

“One side is motor fibers, and any problem with that can paralyze the patient, and just below that is the optic tract, and any problem there, you would lose vision,” Dr. Krishna said. “It is a very tough neighborhood to be in.”

By using MRI-guided ultrasound, surgeons can change the target and temperature of the ultrasound beam during the procedure to allow more precise treatment.

Pallidotomy-related adverse events in the treatment group included dysarthria, gait disturbance, loss of taste, visual disturbance, and facial weakness. All were mild to moderate, Dr. Krishna said.
 

More study is needed

Dyskinesia is a challenge in the management of Parkinson’s disease. Patients need antiparkinsonian medications to slow deterioration of motor function, but those medications can cause the involuntary movements that are a hallmark of dyskinesia.

The most common treatment for this complication, deep-brain stimulation (DBS), has its own drawbacks. It’s an open procedure, and there is a low-level risk for intracranial bleeding and infection. In addition, the electrode implants require ongoing maintenance and adjustment.

But the findings of this study show that, for patients who aren’t candidates for other therapies, such as DBS and ablative radiofrequency, FUSA may be an alternative, wrote Anette Schrag, PhD, professor of clinical neurosciences at University College London, in an accompanying commentary.

“The results confirm that it is effective in reducing motor complications of Parkinson’s disease, at least in the short term,” Dr. Schrag wrote. However, more long-term studies are needed, she added.

One-third of patients in the treatment group had no response to the treatment, and investigators aren’t sure why. Dr. Krishna noted that the benefits of the procedure waned in about a quarter of patients within a year of treatment.

Investigators plan to probe these questions in future trials.

“The results of this trial are promising,” Dr. Schrag wrote, “but given the nonreversible nature of the intervention and the progressive nature of the disease, it will be important to establish whether improvements in motor complications are maintained over longer periods and whether treatment results in improved overall functioning and quality of life for patients.”

The study was funded by Insightec. Disclosure forms for Dr. Krishna and Dr. Schrag are provided on the journal’s website.

A version of this article originally appeared on Medscape.com.

New national data on the occurrence of triple-negative breast cancer (TNBC) among different racial groups confirms that the disease is more common among Black women nationwide. A state-by-state analysis in the study, published online  in JAMA Oncology, shows that these trends persist at the state level.

The analysis revealed that incidence rate ratios of TNBC were significantly higher among Black women, compared with White women, in all states with data on this population. Rates ranged from a low of 1.38 in Colorado to a high of 2.32 in Delaware.

The state-level disparities highlight gaps in physicians’ understanding of how social factors contribute to disparities in TNBC risk and the need “to develop effective preventative measures,” the study authors explain.

“We’ve realized for a long time that Black women have a higher incidence of TNBC. This is related to the genetic signature of the cancer. So that is not at all surprising,” said Arnold M. Baskies, MD, past chairman of the national board of directors of the American Cancer Society, Atlanta, who was not involved in the research. However, “the variance of TNBC among women from state to state is somewhat surprising.”

Existing research shows that TNBC is diagnosed more frequently among non-Hispanic Black women than among other populations in the United States, but it’s unclear whether these racial and ethnic disparities differ at the state level.

The authors identified 133,579 women with TNBC from the U.S. Cancer Statistics Public Use Research Database whose conditions were diagnosed from January 2015 through the end of December 2019. Most patients (64.5%) were White, 21.5% were Black, nearly 10% were Hispanic, 3.7% were Asian or Pacific Islander, and 0.6% were American Indian or Alaska Native. States with fewer than 30 cases were excluded, as was Nevada, owing to concerns regarding data quality. That left eight states for American Indian or Alaska Natives, 22 for Asian or Pacific Islanders, 35 for Hispanic women, 38 for Black women, and 50 for White women.

Overall, the incidence ratios of TNBC were highest among Black women (IR, 25.2 per 100,000), followed by White women (IR, 12.9 per 100,000), American Indian or Alaska Native women (IR, 11.2 per 100,000), Hispanic women (IR, 11.1 per 100,000 women), and Asian or Pacific Islander (IR, 9.0 per 100,000) women.

The authors also uncovered significant state-by-state variations in TNBC incidence by racial and ethnic groups. The lowest IR rates occurred among Asian or Pacific Islander women in Oregon and Pennsylvania – fewer than 7 per 100,000 women – and the highest occurred among Black women in Delaware, Missouri, Louisiana, and Mississippi – more than 29 per 100,000 women.

In the 38 states for which data on Black women were available, IR rates were significantly higher among Black women in all 38, compared with White women. The IR rates ranged from a low of 1.38 (IR, 17.4 per 100 000 women) in Colorado to a high of 2.32 (IR, 32.0 per 100 000 women) in Delaware.

While genetics play a role in TNBC risk, “the substantial geographic variation we found within each racial and ethnic group is highly suggestive that there are structural, environmental, and social factors at play in determining women’s risk of TNBC,” said lead study author Hyuna Sung, PhD, senior principal scientist and cancer epidemiologist at the American Cancer Society, Atlanta.

Existing evidence indicates that Black and White women living in socioeconomically disadvantaged neighborhoods are at higher risk of developing more aggressive subtypes of breast cancer, Dr. Sung said. Another factor, Dr. Sung and co-authors note, is breastfeeding. Across races, women who breastfeed have lower rates of TNBC.

Getting more definitive answers as to what causes differences in TNBC rates across states and what strategies can help reduce these disparities will be difficult and requires more research. “We really need to do a better job at researching and treating TNBC to improve health care equality for all women,” Dr. Baskies said. “The mortality rates from this cancer are high, and we rely heavily on surgery and toxic chemotherapy to treat it.”

Dr. Sung agreed, noting that “the observed state variation in TNBC rates merits further studies with risk factor data at multiple levels to better understand the associations of social exposures with the risk of TNBC.”

In states such as Louisiana and Mississippi, which are known to have a disproportionately higher burden of many types of cancers, “addressing barriers to access to preventive care and empowering public health efforts to promote a healthy living environment are the best policy prescription that could be deduced from our results,” Dr. Sung concluded.

Dr. Baskies is on the board of directors of Anixa Biosciences, which is currently conducting a clinical trial of a TNBC vaccine at the Cleveland Clinic. Dr. Sung has disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

New national data on the occurrence of triple-negative breast cancer (TNBC) among different racial groups confirms that the disease is more common among Black women nationwide. A state-by-state analysis in the study, published online  in JAMA Oncology, shows that these trends persist at the state level.

The analysis revealed that incidence rate ratios of TNBC were significantly higher among Black women, compared with White women, in all states with data on this population. Rates ranged from a low of 1.38 in Colorado to a high of 2.32 in Delaware.

The state-level disparities highlight gaps in physicians’ understanding of how social factors contribute to disparities in TNBC risk and the need “to develop effective preventative measures,” the study authors explain.

“We’ve realized for a long time that Black women have a higher incidence of TNBC. This is related to the genetic signature of the cancer. So that is not at all surprising,” said Arnold M. Baskies, MD, past chairman of the national board of directors of the American Cancer Society, Atlanta, who was not involved in the research. However, “the variance of TNBC among women from state to state is somewhat surprising.”

Existing research shows that TNBC is diagnosed more frequently among non-Hispanic Black women than among other populations in the United States, but it’s unclear whether these racial and ethnic disparities differ at the state level.

The authors identified 133,579 women with TNBC from the U.S. Cancer Statistics Public Use Research Database whose conditions were diagnosed from January 2015 through the end of December 2019. Most patients (64.5%) were White, 21.5% were Black, nearly 10% were Hispanic, 3.7% were Asian or Pacific Islander, and 0.6% were American Indian or Alaska Native. States with fewer than 30 cases were excluded, as was Nevada, owing to concerns regarding data quality. That left eight states for American Indian or Alaska Natives, 22 for Asian or Pacific Islanders, 35 for Hispanic women, 38 for Black women, and 50 for White women.

Overall, the incidence ratios of TNBC were highest among Black women (IR, 25.2 per 100,000), followed by White women (IR, 12.9 per 100,000), American Indian or Alaska Native women (IR, 11.2 per 100,000), Hispanic women (IR, 11.1 per 100,000 women), and Asian or Pacific Islander (IR, 9.0 per 100,000) women.

The authors also uncovered significant state-by-state variations in TNBC incidence by racial and ethnic groups. The lowest IR rates occurred among Asian or Pacific Islander women in Oregon and Pennsylvania – fewer than 7 per 100,000 women – and the highest occurred among Black women in Delaware, Missouri, Louisiana, and Mississippi – more than 29 per 100,000 women.

In the 38 states for which data on Black women were available, IR rates were significantly higher among Black women in all 38, compared with White women. The IR rates ranged from a low of 1.38 (IR, 17.4 per 100 000 women) in Colorado to a high of 2.32 (IR, 32.0 per 100 000 women) in Delaware.

While genetics play a role in TNBC risk, “the substantial geographic variation we found within each racial and ethnic group is highly suggestive that there are structural, environmental, and social factors at play in determining women’s risk of TNBC,” said lead study author Hyuna Sung, PhD, senior principal scientist and cancer epidemiologist at the American Cancer Society, Atlanta.

Existing evidence indicates that Black and White women living in socioeconomically disadvantaged neighborhoods are at higher risk of developing more aggressive subtypes of breast cancer, Dr. Sung said. Another factor, Dr. Sung and co-authors note, is breastfeeding. Across races, women who breastfeed have lower rates of TNBC.

Getting more definitive answers as to what causes differences in TNBC rates across states and what strategies can help reduce these disparities will be difficult and requires more research. “We really need to do a better job at researching and treating TNBC to improve health care equality for all women,” Dr. Baskies said. “The mortality rates from this cancer are high, and we rely heavily on surgery and toxic chemotherapy to treat it.”

Dr. Sung agreed, noting that “the observed state variation in TNBC rates merits further studies with risk factor data at multiple levels to better understand the associations of social exposures with the risk of TNBC.”

In states such as Louisiana and Mississippi, which are known to have a disproportionately higher burden of many types of cancers, “addressing barriers to access to preventive care and empowering public health efforts to promote a healthy living environment are the best policy prescription that could be deduced from our results,” Dr. Sung concluded.

Dr. Baskies is on the board of directors of Anixa Biosciences, which is currently conducting a clinical trial of a TNBC vaccine at the Cleveland Clinic. Dr. Sung has disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

New national data on the occurrence of triple-negative breast cancer (TNBC) among different racial groups confirms that the disease is more common among Black women nationwide. A state-by-state analysis in the study, published online  in JAMA Oncology, shows that these trends persist at the state level.

The analysis revealed that incidence rate ratios of TNBC were significantly higher among Black women, compared with White women, in all states with data on this population. Rates ranged from a low of 1.38 in Colorado to a high of 2.32 in Delaware.

The state-level disparities highlight gaps in physicians’ understanding of how social factors contribute to disparities in TNBC risk and the need “to develop effective preventative measures,” the study authors explain.

“We’ve realized for a long time that Black women have a higher incidence of TNBC. This is related to the genetic signature of the cancer. So that is not at all surprising,” said Arnold M. Baskies, MD, past chairman of the national board of directors of the American Cancer Society, Atlanta, who was not involved in the research. However, “the variance of TNBC among women from state to state is somewhat surprising.”

Existing research shows that TNBC is diagnosed more frequently among non-Hispanic Black women than among other populations in the United States, but it’s unclear whether these racial and ethnic disparities differ at the state level.

The authors identified 133,579 women with TNBC from the U.S. Cancer Statistics Public Use Research Database whose conditions were diagnosed from January 2015 through the end of December 2019. Most patients (64.5%) were White, 21.5% were Black, nearly 10% were Hispanic, 3.7% were Asian or Pacific Islander, and 0.6% were American Indian or Alaska Native. States with fewer than 30 cases were excluded, as was Nevada, owing to concerns regarding data quality. That left eight states for American Indian or Alaska Natives, 22 for Asian or Pacific Islanders, 35 for Hispanic women, 38 for Black women, and 50 for White women.

Overall, the incidence ratios of TNBC were highest among Black women (IR, 25.2 per 100,000), followed by White women (IR, 12.9 per 100,000), American Indian or Alaska Native women (IR, 11.2 per 100,000), Hispanic women (IR, 11.1 per 100,000 women), and Asian or Pacific Islander (IR, 9.0 per 100,000) women.

The authors also uncovered significant state-by-state variations in TNBC incidence by racial and ethnic groups. The lowest IR rates occurred among Asian or Pacific Islander women in Oregon and Pennsylvania – fewer than 7 per 100,000 women – and the highest occurred among Black women in Delaware, Missouri, Louisiana, and Mississippi – more than 29 per 100,000 women.

In the 38 states for which data on Black women were available, IR rates were significantly higher among Black women in all 38, compared with White women. The IR rates ranged from a low of 1.38 (IR, 17.4 per 100 000 women) in Colorado to a high of 2.32 (IR, 32.0 per 100 000 women) in Delaware.

While genetics play a role in TNBC risk, “the substantial geographic variation we found within each racial and ethnic group is highly suggestive that there are structural, environmental, and social factors at play in determining women’s risk of TNBC,” said lead study author Hyuna Sung, PhD, senior principal scientist and cancer epidemiologist at the American Cancer Society, Atlanta.

Existing evidence indicates that Black and White women living in socioeconomically disadvantaged neighborhoods are at higher risk of developing more aggressive subtypes of breast cancer, Dr. Sung said. Another factor, Dr. Sung and co-authors note, is breastfeeding. Across races, women who breastfeed have lower rates of TNBC.

Getting more definitive answers as to what causes differences in TNBC rates across states and what strategies can help reduce these disparities will be difficult and requires more research. “We really need to do a better job at researching and treating TNBC to improve health care equality for all women,” Dr. Baskies said. “The mortality rates from this cancer are high, and we rely heavily on surgery and toxic chemotherapy to treat it.”

Dr. Sung agreed, noting that “the observed state variation in TNBC rates merits further studies with risk factor data at multiple levels to better understand the associations of social exposures with the risk of TNBC.”

In states such as Louisiana and Mississippi, which are known to have a disproportionately higher burden of many types of cancers, “addressing barriers to access to preventive care and empowering public health efforts to promote a healthy living environment are the best policy prescription that could be deduced from our results,” Dr. Sung concluded.

Dr. Baskies is on the board of directors of Anixa Biosciences, which is currently conducting a clinical trial of a TNBC vaccine at the Cleveland Clinic. Dr. Sung has disclosed no relevant financial relationships.

A version of this article first appeared on Medscape.com.

Consumption of a low-carbohydrate, high-fat diet, dubbed a “keto-like” diet, was associated with an increase in LDL levels and a twofold increase in the risk for future cardiovascular events, in a new observational study.

“To our knowledge this is the first study to demonstrate an association between a carbohydrate-restricted dietary platform and greater risk of atherosclerotic cardiovascular disease,” said study investigator Iulia Iatan, MD, PhD, University of British Columbia, Vancouver.

a_namenko/Getty Images

“Hypercholesterolemia occurring during a low-carb, high-fat diet should not be assumed to be benign,” she concluded.

Dr. Iatan presented the study March 5 at the joint scientific sessions of the American College of Cardiology and the World Heart Federation.

The presentation received much media attention, with headlines implying a causal relationship with cardiac events based on these observational results. But lipid expert Steven Nissen, MD, of the Cleveland Clinic, warned against paying much attention to the headlines or to the study’s conclusions.

In an interview, Dr. Nissen pointed out that the LDL increase in the “keto-like” diet group was relatively small and “certainly not enough to produce a doubling in cardiovascular risk.

“The people who were on the ‘keto-like’ diet in this study were different than those who were on the standard diet,” he said. “Those on the ‘keto-like’ diet were on it for a reason – they were more overweight, they had a higher incidence of diabetes, so their risk profile was completely different. Even though the researchers tried to adjust for other cardiovascular risk factors, there will be unmeasured confounding in a study like this.”

He said he doesn’t think this study “answers any significant questions in a way that we want to have them answered. I’m not a big fan of this type of diet, but I don’t think it doubles the risk of adverse cardiovascular events, and I don’t think this study tells us one way or another.” 

For the study, Dr. Iatan and colleagues defined a low-carbohydrate, high-fat diet as consisting of no more than 25% of total daily energy from carbohydrates and more than 45% of total daily calories from fat. This is somewhat higher in carbohydrates and lower in fat than a strict ketogenic diet but could be thought of as a ‘keto-like’ diet.

They analyzed data from the UK Biobank, a large-scale prospective database with health information from over half a million people living in the United Kingdom who were followed for at least 10 years.

On enrollment in the Biobank, participants completed a one-time, self-reported 24-hour diet questionnaire and, at the same time, had blood drawn to check their levels of cholesterol. The researchers identified 305 participants whose questionnaire responses indicated that they followed a low-carbohydrate, high-fat diet. These participants were matched by age and sex with 1,220 individuals who reported being on a standard diet.

Of the study population, 73% were women and the average age was 54 years. Those on a low carbohydrate/high fat diet had a higher average body mass index (27.7 vs. 26.7) and a higher incidence of diabetes (4.9% vs. 1.7%).

Results showed that compared with participants on a standard diet, those on the “keto-like” diet had significantly higher levels of both LDL cholesterol and apolipoprotein B (ApoB).

Levels of LDL were 3.80 mmol/L (147 mg/dL) in the keto-like group vs. 3.64 mmol/L (141 mg/dL) in the standard group (P = .004).  Levels of ApoB were 1.09 g/L (109 mg/dL) in the keto-like group and 1.04 g/L (104 mg/dL) in the standard group (P < .001).

After an average of 11.8 years of follow-up, 9.8% of participants on the low-carbohydrate/high-fat diet vs. 4.3% in the standard diet group experienced one of the events included in the composite event endpoint: Angina, myocardial infarction, coronary artery disease, ischemic stroke, peripheral arterial disease, or coronary/carotid revascularization.

After adjustment for other risk factors for heart disease – diabetes, hypertension, obesity, and smoking – individuals on a low-carbohydrate, high-fat diet were found to have a twofold risk of having a cardiovascular event (HR, 2.18; P < .001).
 

‘Closer monitoring needed’

“Our results have shown, I think for the first time, that there is an association between this increasingly popular dietary pattern and high LDL cholesterol and an increased future risk of cardiovascular events,” senior author Liam Brunham, MD, of the University of British Columbia, said in an interview. “This is concerning as there are many people out there following this type of diet, and I think it suggests there is a need for closer monitoring of these people.”

He explained that while it would be expected for cholesterol levels to rise on a high-fat diet, “there has been a perception by some that this is not worrisome as it is reflecting certain metabolic changes. What we’ve shown in this study is that if your cholesterol does increase significantly on this diet then you should not assume that this is not a problem.

“For some people with diabetes this diet can help lower blood sugar and some people can lose weight on it,” he noted, “but what our data show is that there is a subgroup of people who experience high levels of LDL and ApoB and that seems to be driving the risk.”

He pointed out that overall the mean level of LDL was only slightly increased in the individuals on the low-carb/high-fat diet but severe high cholesterol (more than 5 mmol/L or 190 mg/dL) was about doubled in that group (10% vs. 5%). And these patients had a sixfold increase in risk of cardiovascular disease (P < .001). 

“This suggests that there is a subgroup of people who are susceptible to this exacerbation of hypercholesterolemia in response to a low-carb/high-fat diet.”

Dr. Brunham said his advice would be that if people choose to follow this diet, they should have their cholesterol monitored, and manage their cardiovascular risk factors.

“I wouldn’t say it is not appropriate to follow this diet based on this study,” he added. “This is just an observational study. It is not definitive. But if people do want to follow this dietary pattern because they feel there would be some benefits, then they should be aware of the potential risks and take steps to mitigate those risks.”
 

Jury still out

Dr. Nissen said in his view “the jury was still out” on this type of diet. “I’m open to the possibility that, particularly in the short run, a ‘keto-like’ diet may help some people lose weight and that’s a good thing. But I do not generally recommend this type of diet.”

Rather, he advises patients to follow a Mediterranean diet, which has been proven to reduce cardiovascular events in a randomized study, the PREDIMED trial.  

“We can’t make decisions on what type of diet to recommend to patients based on observational studies like this where there is a lot of subtlety missing. But when studies like this are reported, the mass media seize on it. That’s not the way the public needs to be educated,” Dr. Nissen said. 

“We refer to this type of study as hypothesis-generating. It raises a hypothesis. It doesn’t answer the question. It is worth looking at the question of whether a ketogenic-like diet is harmful. We don’t know at present, and I don’t think we know any more after this study,” he added.

The authors of the study reported no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

Consumption of a low-carbohydrate, high-fat diet, dubbed a “keto-like” diet, was associated with an increase in LDL levels and a twofold increase in the risk for future cardiovascular events, in a new observational study.

“To our knowledge this is the first study to demonstrate an association between a carbohydrate-restricted dietary platform and greater risk of atherosclerotic cardiovascular disease,” said study investigator Iulia Iatan, MD, PhD, University of British Columbia, Vancouver.

a_namenko/Getty Images

“Hypercholesterolemia occurring during a low-carb, high-fat diet should not be assumed to be benign,” she concluded.

Dr. Iatan presented the study March 5 at the joint scientific sessions of the American College of Cardiology and the World Heart Federation.

The presentation received much media attention, with headlines implying a causal relationship with cardiac events based on these observational results. But lipid expert Steven Nissen, MD, of the Cleveland Clinic, warned against paying much attention to the headlines or to the study’s conclusions.

In an interview, Dr. Nissen pointed out that the LDL increase in the “keto-like” diet group was relatively small and “certainly not enough to produce a doubling in cardiovascular risk.

“The people who were on the ‘keto-like’ diet in this study were different than those who were on the standard diet,” he said. “Those on the ‘keto-like’ diet were on it for a reason – they were more overweight, they had a higher incidence of diabetes, so their risk profile was completely different. Even though the researchers tried to adjust for other cardiovascular risk factors, there will be unmeasured confounding in a study like this.”

He said he doesn’t think this study “answers any significant questions in a way that we want to have them answered. I’m not a big fan of this type of diet, but I don’t think it doubles the risk of adverse cardiovascular events, and I don’t think this study tells us one way or another.” 

For the study, Dr. Iatan and colleagues defined a low-carbohydrate, high-fat diet as consisting of no more than 25% of total daily energy from carbohydrates and more than 45% of total daily calories from fat. This is somewhat higher in carbohydrates and lower in fat than a strict ketogenic diet but could be thought of as a ‘keto-like’ diet.

They analyzed data from the UK Biobank, a large-scale prospective database with health information from over half a million people living in the United Kingdom who were followed for at least 10 years.

On enrollment in the Biobank, participants completed a one-time, self-reported 24-hour diet questionnaire and, at the same time, had blood drawn to check their levels of cholesterol. The researchers identified 305 participants whose questionnaire responses indicated that they followed a low-carbohydrate, high-fat diet. These participants were matched by age and sex with 1,220 individuals who reported being on a standard diet.

Of the study population, 73% were women and the average age was 54 years. Those on a low carbohydrate/high fat diet had a higher average body mass index (27.7 vs. 26.7) and a higher incidence of diabetes (4.9% vs. 1.7%).

Results showed that compared with participants on a standard diet, those on the “keto-like” diet had significantly higher levels of both LDL cholesterol and apolipoprotein B (ApoB).

Levels of LDL were 3.80 mmol/L (147 mg/dL) in the keto-like group vs. 3.64 mmol/L (141 mg/dL) in the standard group (P = .004).  Levels of ApoB were 1.09 g/L (109 mg/dL) in the keto-like group and 1.04 g/L (104 mg/dL) in the standard group (P < .001).

After an average of 11.8 years of follow-up, 9.8% of participants on the low-carbohydrate/high-fat diet vs. 4.3% in the standard diet group experienced one of the events included in the composite event endpoint: Angina, myocardial infarction, coronary artery disease, ischemic stroke, peripheral arterial disease, or coronary/carotid revascularization.

After adjustment for other risk factors for heart disease – diabetes, hypertension, obesity, and smoking – individuals on a low-carbohydrate, high-fat diet were found to have a twofold risk of having a cardiovascular event (HR, 2.18; P < .001).
 

‘Closer monitoring needed’

“Our results have shown, I think for the first time, that there is an association between this increasingly popular dietary pattern and high LDL cholesterol and an increased future risk of cardiovascular events,” senior author Liam Brunham, MD, of the University of British Columbia, said in an interview. “This is concerning as there are many people out there following this type of diet, and I think it suggests there is a need for closer monitoring of these people.”

He explained that while it would be expected for cholesterol levels to rise on a high-fat diet, “there has been a perception by some that this is not worrisome as it is reflecting certain metabolic changes. What we’ve shown in this study is that if your cholesterol does increase significantly on this diet then you should not assume that this is not a problem.

“For some people with diabetes this diet can help lower blood sugar and some people can lose weight on it,” he noted, “but what our data show is that there is a subgroup of people who experience high levels of LDL and ApoB and that seems to be driving the risk.”

He pointed out that overall the mean level of LDL was only slightly increased in the individuals on the low-carb/high-fat diet but severe high cholesterol (more than 5 mmol/L or 190 mg/dL) was about doubled in that group (10% vs. 5%). And these patients had a sixfold increase in risk of cardiovascular disease (P < .001). 

“This suggests that there is a subgroup of people who are susceptible to this exacerbation of hypercholesterolemia in response to a low-carb/high-fat diet.”

Dr. Brunham said his advice would be that if people choose to follow this diet, they should have their cholesterol monitored, and manage their cardiovascular risk factors.

“I wouldn’t say it is not appropriate to follow this diet based on this study,” he added. “This is just an observational study. It is not definitive. But if people do want to follow this dietary pattern because they feel there would be some benefits, then they should be aware of the potential risks and take steps to mitigate those risks.”
 

Jury still out

Dr. Nissen said in his view “the jury was still out” on this type of diet. “I’m open to the possibility that, particularly in the short run, a ‘keto-like’ diet may help some people lose weight and that’s a good thing. But I do not generally recommend this type of diet.”

Rather, he advises patients to follow a Mediterranean diet, which has been proven to reduce cardiovascular events in a randomized study, the PREDIMED trial.  

“We can’t make decisions on what type of diet to recommend to patients based on observational studies like this where there is a lot of subtlety missing. But when studies like this are reported, the mass media seize on it. That’s not the way the public needs to be educated,” Dr. Nissen said. 

“We refer to this type of study as hypothesis-generating. It raises a hypothesis. It doesn’t answer the question. It is worth looking at the question of whether a ketogenic-like diet is harmful. We don’t know at present, and I don’t think we know any more after this study,” he added.

The authors of the study reported no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

Consumption of a low-carbohydrate, high-fat diet, dubbed a “keto-like” diet, was associated with an increase in LDL levels and a twofold increase in the risk for future cardiovascular events, in a new observational study.

“To our knowledge this is the first study to demonstrate an association between a carbohydrate-restricted dietary platform and greater risk of atherosclerotic cardiovascular disease,” said study investigator Iulia Iatan, MD, PhD, University of British Columbia, Vancouver.

a_namenko/Getty Images

“Hypercholesterolemia occurring during a low-carb, high-fat diet should not be assumed to be benign,” she concluded.

Dr. Iatan presented the study March 5 at the joint scientific sessions of the American College of Cardiology and the World Heart Federation.

The presentation received much media attention, with headlines implying a causal relationship with cardiac events based on these observational results. But lipid expert Steven Nissen, MD, of the Cleveland Clinic, warned against paying much attention to the headlines or to the study’s conclusions.

In an interview, Dr. Nissen pointed out that the LDL increase in the “keto-like” diet group was relatively small and “certainly not enough to produce a doubling in cardiovascular risk.

“The people who were on the ‘keto-like’ diet in this study were different than those who were on the standard diet,” he said. “Those on the ‘keto-like’ diet were on it for a reason – they were more overweight, they had a higher incidence of diabetes, so their risk profile was completely different. Even though the researchers tried to adjust for other cardiovascular risk factors, there will be unmeasured confounding in a study like this.”

He said he doesn’t think this study “answers any significant questions in a way that we want to have them answered. I’m not a big fan of this type of diet, but I don’t think it doubles the risk of adverse cardiovascular events, and I don’t think this study tells us one way or another.” 

For the study, Dr. Iatan and colleagues defined a low-carbohydrate, high-fat diet as consisting of no more than 25% of total daily energy from carbohydrates and more than 45% of total daily calories from fat. This is somewhat higher in carbohydrates and lower in fat than a strict ketogenic diet but could be thought of as a ‘keto-like’ diet.

They analyzed data from the UK Biobank, a large-scale prospective database with health information from over half a million people living in the United Kingdom who were followed for at least 10 years.

On enrollment in the Biobank, participants completed a one-time, self-reported 24-hour diet questionnaire and, at the same time, had blood drawn to check their levels of cholesterol. The researchers identified 305 participants whose questionnaire responses indicated that they followed a low-carbohydrate, high-fat diet. These participants were matched by age and sex with 1,220 individuals who reported being on a standard diet.

Of the study population, 73% were women and the average age was 54 years. Those on a low carbohydrate/high fat diet had a higher average body mass index (27.7 vs. 26.7) and a higher incidence of diabetes (4.9% vs. 1.7%).

Results showed that compared with participants on a standard diet, those on the “keto-like” diet had significantly higher levels of both LDL cholesterol and apolipoprotein B (ApoB).

Levels of LDL were 3.80 mmol/L (147 mg/dL) in the keto-like group vs. 3.64 mmol/L (141 mg/dL) in the standard group (P = .004).  Levels of ApoB were 1.09 g/L (109 mg/dL) in the keto-like group and 1.04 g/L (104 mg/dL) in the standard group (P < .001).

After an average of 11.8 years of follow-up, 9.8% of participants on the low-carbohydrate/high-fat diet vs. 4.3% in the standard diet group experienced one of the events included in the composite event endpoint: Angina, myocardial infarction, coronary artery disease, ischemic stroke, peripheral arterial disease, or coronary/carotid revascularization.

After adjustment for other risk factors for heart disease – diabetes, hypertension, obesity, and smoking – individuals on a low-carbohydrate, high-fat diet were found to have a twofold risk of having a cardiovascular event (HR, 2.18; P < .001).
 

‘Closer monitoring needed’

“Our results have shown, I think for the first time, that there is an association between this increasingly popular dietary pattern and high LDL cholesterol and an increased future risk of cardiovascular events,” senior author Liam Brunham, MD, of the University of British Columbia, said in an interview. “This is concerning as there are many people out there following this type of diet, and I think it suggests there is a need for closer monitoring of these people.”

He explained that while it would be expected for cholesterol levels to rise on a high-fat diet, “there has been a perception by some that this is not worrisome as it is reflecting certain metabolic changes. What we’ve shown in this study is that if your cholesterol does increase significantly on this diet then you should not assume that this is not a problem.

“For some people with diabetes this diet can help lower blood sugar and some people can lose weight on it,” he noted, “but what our data show is that there is a subgroup of people who experience high levels of LDL and ApoB and that seems to be driving the risk.”

He pointed out that overall the mean level of LDL was only slightly increased in the individuals on the low-carb/high-fat diet but severe high cholesterol (more than 5 mmol/L or 190 mg/dL) was about doubled in that group (10% vs. 5%). And these patients had a sixfold increase in risk of cardiovascular disease (P < .001). 

“This suggests that there is a subgroup of people who are susceptible to this exacerbation of hypercholesterolemia in response to a low-carb/high-fat diet.”

Dr. Brunham said his advice would be that if people choose to follow this diet, they should have their cholesterol monitored, and manage their cardiovascular risk factors.

“I wouldn’t say it is not appropriate to follow this diet based on this study,” he added. “This is just an observational study. It is not definitive. But if people do want to follow this dietary pattern because they feel there would be some benefits, then they should be aware of the potential risks and take steps to mitigate those risks.”
 

Jury still out

Dr. Nissen said in his view “the jury was still out” on this type of diet. “I’m open to the possibility that, particularly in the short run, a ‘keto-like’ diet may help some people lose weight and that’s a good thing. But I do not generally recommend this type of diet.”

Rather, he advises patients to follow a Mediterranean diet, which has been proven to reduce cardiovascular events in a randomized study, the PREDIMED trial.  

“We can’t make decisions on what type of diet to recommend to patients based on observational studies like this where there is a lot of subtlety missing. But when studies like this are reported, the mass media seize on it. That’s not the way the public needs to be educated,” Dr. Nissen said. 

“We refer to this type of study as hypothesis-generating. It raises a hypothesis. It doesn’t answer the question. It is worth looking at the question of whether a ketogenic-like diet is harmful. We don’t know at present, and I don’t think we know any more after this study,” he added.

The authors of the study reported no relevant financial relationships.

A version of this article originally appeared on Medscape.com.

Patients with advanced renal cell carcinoma who are taking a tyrosine kinase inhibitor (TKI) to prolong their lives will typically keep going without a break until the disease progresses or toxicities such as severe fatigue and diarrhea become intolerable.

That might soon change with the publication of a unique study. Lasting 10 years, the phase 3 STAR trial involved 920 patients across 60 cancer centers. These patients had advanced kidney cancer and were taking either sunitinib (Sutent) or pazopanib (Votrient).

The results showed that taking an occasional respite from TKI therapy had little impact on the patient’s survival.

The study was published online in The Lancet Oncology.

The study was funded by the United Kingdom’s National Institute for Health and Care Research because drug companies never run studies on how to reduce the use of their drug, commented lead author Janet Brown, MD, of the University of Sheffield (England).

“We rely on the NIHR to do these important trials that … companies wouldn’t do,” she commented to this news organization.

Commenting on the rationale for STAR, coauthor Jenny Hewison, PhD, of Leeds (England) University School of Medicine, explained that patients often find it difficult to tolerate TKIs. “Although these patients are getting the best treatment that we can offer them, it’s very demanding. … It could make them feel tired, quite unwell. And there can be a range of other effects including sickness and diarrhea.”

As an example, 77% of patients in the pivotal trial of sunitinib in kidney cancer experienced grade 3 or 4 adverse events such as hypertension (13%), fatigue (15%), diarrhea (10%) and hand-foot syndrome (8%).

Both sunitinib and pazopanib carry label warnings of severe and fatal hepatotoxicity.

Also, in contrast to conventional chemotherapy, which is usually given in a finite number of courses, treatment with TKIs carries on indefinitely.

“It feels like you’re taking [TKIs] for the whole of the rest of your life,” said Dr. Brown.
 

Study details

The STAR trial, an open-label, noninferiority, randomized controlled study, is the first phase 3 study of treatment breaks in renal cell carcinoma. The participants had inoperable locoregional or metastatic clear cell renal cell carcinoma (ccRCC) and had received no systemic therapy for advanced disease.

They were randomly assigned before TKI treatment to a conventional continuation strategy or a drug-free interval approach. The treating physician decided whether a patient would take sunitinib or pazopanib.

All participants took their drugs for four cycles (6 weeks each cycle). At the 24-week point, those with a complete response, partial response, or stable disease began their randomized assignment.

Individuals who took a break continued until their disease progressed, at which point therapy was resumed. They could take further treatment breaks once their disease was back under control. The group on continuous treatment kept going until disease progression or intolerable toxicities. Median follow up was 58 months.

In both the per-protocol and intent-to-treat (ITT) populations, overall survival was 28 months for the people who received continuous treatment vs. 27 months for those who took a break. Statistical noninferiority was established in the ITT population but not in the per-protocol population.

The median length of all treatment breaks was 87 days. Many people took two or more breaks; one patient took nine breaks overall. The breaks were popular: only 3% of participants who were meant to stop therapy withdrew from the study in order to continue their treatment.

Said Dr. Hewison: “In the very early days of planning the study there were some doubts as to whether it would succeed because of potential unwillingness of people to stop treatment for a while.”

Dr. Brown agreed: “People did worry about that initially, but it actually seemed to be more the other way around. By that time – 6 months – people were relieved to be there. …We actually had some people from the other arm asking, could they also have a break?”

To understand better the benefits of treatment breaks to patients, Janine Bestall, PhD, a senior research fellow in applied health research at the University of Leeds, conducted a qualitative study in parallel with the main trial.

Summing up the patients’ experiences, Dr. Bestall said the drug-free periods “gave them more time.”

Dr. Bestall quoted one patient who said: “I know that things can happen and it grows back, but you’ve always got the buffer there knowing that you can go back and get help. But you actually lead a normal life and the advantage is, yeah, you can go on holiday, you can actually do more things in the garden, cleaning up, painting, whatever needs doing, you do it.”

Dr. Brown said, “I had a lady who, when she was on the trial, had four breaks in total, one when her daughter got married, and [she said] that was really nice for her to do all the shopping and all the normal things that you do, and not be on something that was making her tired and causing sore hands and diarrhea.” 

The drug-free interval strategy provided annual cost savings of 3,235 pounds sterling ($3,850) and a noninferior quality-adjusted life-year (QALY) benefit in both the ITT and per-protocol populations.

Serious adverse reactions occurred in 9% of patients in the treatment-break group versus 12% of the continuous-treatment group.

The authors of the study concluded, “Treatment breaks might be a feasible and cost-effective option with lifestyle benefits for patients during tyrosine kinase inhibitor therapy in patients with renal cell carcinoma.”
 

Changes in treatment strategies

The STAR trial started recruiting in January 2012.

Since that time, immunotherapy has taken over as first-line treatment for many patients with advanced ccRCC in both the United Kingdom and the United States.

However, TKIs still have a place. The NCCN Kidney Cancer 2022 Guidelines recommend both sunitinib and pazopanib as options for first-line therapy in advanced disease. The 2022 ASCO Metastatic ccRCC guidelines recommend either drug as first-line treatment in combination with an immune checkpoint inhibitor or in monotherapy if there are “coexisting medical problems.”

In the United States, intermittent sunitinib in metastatic RCC was tested in a small study in 2017 with little activity in the literature since then. The authors, led by Moshe Ornstein, MD, from the Cleveland Clinic, concluded at the time that sunitinib treatment breaks were feasible and “clinical efficacy does not seem to be compromised.” Dr. Ornstein was approached for comment on this latest U.K. study but declined.

Back in the United Kingdom, the results of STAR arrived just in time.

Said Dr. Brown: “This has … been really helpful in the U.K. in the pandemic when people said, can these patients have extra breaks? At the worst of the pandemic we were able to say, sure, if it’s stable, we can keep them off for 3-6 months. …And so that’s already had a powerful impact.”

Dr. Brown concluded, “I think what the trial does allow us to do, as individual oncologists, is to look at the patients that this might be suitable for – it won’t be everybody – and to say yes, it’s okay to personalize things.”

The study was funded by the U.K.’s National Institute for Health and Care Research. Dr. Bestall reported no relevant financial relationships. Dr. Hewison reported funding to her institution from the NIHR Health Technology Assessment. Dr. Brown reports having served as a consultant or adviser for Novartis, Ipsen, Amgen, Merck Sharp & Dohme, Bristol-Myers Squibb, and Bayer; honoraria from Novartis, Ipsen, Amgen, Merck Sharp & Dohme, Bristol-Myers Squibb, and Bayer; research funding paid to their institution from the National Institute for Health and Care Research; and travel expenses from Ipsen. Other coauthors reported numerous relationships with industry.

A version of this article first appeared on Medscape.com.

Patients with advanced renal cell carcinoma who are taking a tyrosine kinase inhibitor (TKI) to prolong their lives will typically keep going without a break until the disease progresses or toxicities such as severe fatigue and diarrhea become intolerable.

That might soon change with the publication of a unique study. Lasting 10 years, the phase 3 STAR trial involved 920 patients across 60 cancer centers. These patients had advanced kidney cancer and were taking either sunitinib (Sutent) or pazopanib (Votrient).

The results showed that taking an occasional respite from TKI therapy had little impact on the patient’s survival.

The study was published online in The Lancet Oncology.

The study was funded by the United Kingdom’s National Institute for Health and Care Research because drug companies never run studies on how to reduce the use of their drug, commented lead author Janet Brown, MD, of the University of Sheffield (England).

“We rely on the NIHR to do these important trials that … companies wouldn’t do,” she commented to this news organization.

Commenting on the rationale for STAR, coauthor Jenny Hewison, PhD, of Leeds (England) University School of Medicine, explained that patients often find it difficult to tolerate TKIs. “Although these patients are getting the best treatment that we can offer them, it’s very demanding. … It could make them feel tired, quite unwell. And there can be a range of other effects including sickness and diarrhea.”

As an example, 77% of patients in the pivotal trial of sunitinib in kidney cancer experienced grade 3 or 4 adverse events such as hypertension (13%), fatigue (15%), diarrhea (10%) and hand-foot syndrome (8%).

Both sunitinib and pazopanib carry label warnings of severe and fatal hepatotoxicity.

Also, in contrast to conventional chemotherapy, which is usually given in a finite number of courses, treatment with TKIs carries on indefinitely.

“It feels like you’re taking [TKIs] for the whole of the rest of your life,” said Dr. Brown.
 

Study details

The STAR trial, an open-label, noninferiority, randomized controlled study, is the first phase 3 study of treatment breaks in renal cell carcinoma. The participants had inoperable locoregional or metastatic clear cell renal cell carcinoma (ccRCC) and had received no systemic therapy for advanced disease.

They were randomly assigned before TKI treatment to a conventional continuation strategy or a drug-free interval approach. The treating physician decided whether a patient would take sunitinib or pazopanib.

All participants took their drugs for four cycles (6 weeks each cycle). At the 24-week point, those with a complete response, partial response, or stable disease began their randomized assignment.

Individuals who took a break continued until their disease progressed, at which point therapy was resumed. They could take further treatment breaks once their disease was back under control. The group on continuous treatment kept going until disease progression or intolerable toxicities. Median follow up was 58 months.

In both the per-protocol and intent-to-treat (ITT) populations, overall survival was 28 months for the people who received continuous treatment vs. 27 months for those who took a break. Statistical noninferiority was established in the ITT population but not in the per-protocol population.

The median length of all treatment breaks was 87 days. Many people took two or more breaks; one patient took nine breaks overall. The breaks were popular: only 3% of participants who were meant to stop therapy withdrew from the study in order to continue their treatment.

Said Dr. Hewison: “In the very early days of planning the study there were some doubts as to whether it would succeed because of potential unwillingness of people to stop treatment for a while.”

Dr. Brown agreed: “People did worry about that initially, but it actually seemed to be more the other way around. By that time – 6 months – people were relieved to be there. …We actually had some people from the other arm asking, could they also have a break?”

To understand better the benefits of treatment breaks to patients, Janine Bestall, PhD, a senior research fellow in applied health research at the University of Leeds, conducted a qualitative study in parallel with the main trial.

Summing up the patients’ experiences, Dr. Bestall said the drug-free periods “gave them more time.”

Dr. Bestall quoted one patient who said: “I know that things can happen and it grows back, but you’ve always got the buffer there knowing that you can go back and get help. But you actually lead a normal life and the advantage is, yeah, you can go on holiday, you can actually do more things in the garden, cleaning up, painting, whatever needs doing, you do it.”

Dr. Brown said, “I had a lady who, when she was on the trial, had four breaks in total, one when her daughter got married, and [she said] that was really nice for her to do all the shopping and all the normal things that you do, and not be on something that was making her tired and causing sore hands and diarrhea.” 

The drug-free interval strategy provided annual cost savings of 3,235 pounds sterling ($3,850) and a noninferior quality-adjusted life-year (QALY) benefit in both the ITT and per-protocol populations.

Serious adverse reactions occurred in 9% of patients in the treatment-break group versus 12% of the continuous-treatment group.

The authors of the study concluded, “Treatment breaks might be a feasible and cost-effective option with lifestyle benefits for patients during tyrosine kinase inhibitor therapy in patients with renal cell carcinoma.”
 

Changes in treatment strategies

The STAR trial started recruiting in January 2012.

Since that time, immunotherapy has taken over as first-line treatment for many patients with advanced ccRCC in both the United Kingdom and the United States.

However, TKIs still have a place. The NCCN Kidney Cancer 2022 Guidelines recommend both sunitinib and pazopanib as options for first-line therapy in advanced disease. The 2022 ASCO Metastatic ccRCC guidelines recommend either drug as first-line treatment in combination with an immune checkpoint inhibitor or in monotherapy if there are “coexisting medical problems.”

In the United States, intermittent sunitinib in metastatic RCC was tested in a small study in 2017 with little activity in the literature since then. The authors, led by Moshe Ornstein, MD, from the Cleveland Clinic, concluded at the time that sunitinib treatment breaks were feasible and “clinical efficacy does not seem to be compromised.” Dr. Ornstein was approached for comment on this latest U.K. study but declined.

Back in the United Kingdom, the results of STAR arrived just in time.

Said Dr. Brown: “This has … been really helpful in the U.K. in the pandemic when people said, can these patients have extra breaks? At the worst of the pandemic we were able to say, sure, if it’s stable, we can keep them off for 3-6 months. …And so that’s already had a powerful impact.”

Dr. Brown concluded, “I think what the trial does allow us to do, as individual oncologists, is to look at the patients that this might be suitable for – it won’t be everybody – and to say yes, it’s okay to personalize things.”

The study was funded by the U.K.’s National Institute for Health and Care Research. Dr. Bestall reported no relevant financial relationships. Dr. Hewison reported funding to her institution from the NIHR Health Technology Assessment. Dr. Brown reports having served as a consultant or adviser for Novartis, Ipsen, Amgen, Merck Sharp & Dohme, Bristol-Myers Squibb, and Bayer; honoraria from Novartis, Ipsen, Amgen, Merck Sharp & Dohme, Bristol-Myers Squibb, and Bayer; research funding paid to their institution from the National Institute for Health and Care Research; and travel expenses from Ipsen. Other coauthors reported numerous relationships with industry.

A version of this article first appeared on Medscape.com.

Patients with advanced renal cell carcinoma who are taking a tyrosine kinase inhibitor (TKI) to prolong their lives will typically keep going without a break until the disease progresses or toxicities such as severe fatigue and diarrhea become intolerable.

That might soon change with the publication of a unique study. Lasting 10 years, the phase 3 STAR trial involved 920 patients across 60 cancer centers. These patients had advanced kidney cancer and were taking either sunitinib (Sutent) or pazopanib (Votrient).

The results showed that taking an occasional respite from TKI therapy had little impact on the patient’s survival.

The study was published online in The Lancet Oncology.

The study was funded by the United Kingdom’s National Institute for Health and Care Research because drug companies never run studies on how to reduce the use of their drug, commented lead author Janet Brown, MD, of the University of Sheffield (England).

“We rely on the NIHR to do these important trials that … companies wouldn’t do,” she commented to this news organization.

Commenting on the rationale for STAR, coauthor Jenny Hewison, PhD, of Leeds (England) University School of Medicine, explained that patients often find it difficult to tolerate TKIs. “Although these patients are getting the best treatment that we can offer them, it’s very demanding. … It could make them feel tired, quite unwell. And there can be a range of other effects including sickness and diarrhea.”

As an example, 77% of patients in the pivotal trial of sunitinib in kidney cancer experienced grade 3 or 4 adverse events such as hypertension (13%), fatigue (15%), diarrhea (10%) and hand-foot syndrome (8%).

Both sunitinib and pazopanib carry label warnings of severe and fatal hepatotoxicity.

Also, in contrast to conventional chemotherapy, which is usually given in a finite number of courses, treatment with TKIs carries on indefinitely.

“It feels like you’re taking [TKIs] for the whole of the rest of your life,” said Dr. Brown.
 

Study details

The STAR trial, an open-label, noninferiority, randomized controlled study, is the first phase 3 study of treatment breaks in renal cell carcinoma. The participants had inoperable locoregional or metastatic clear cell renal cell carcinoma (ccRCC) and had received no systemic therapy for advanced disease.

They were randomly assigned before TKI treatment to a conventional continuation strategy or a drug-free interval approach. The treating physician decided whether a patient would take sunitinib or pazopanib.

All participants took their drugs for four cycles (6 weeks each cycle). At the 24-week point, those with a complete response, partial response, or stable disease began their randomized assignment.

Individuals who took a break continued until their disease progressed, at which point therapy was resumed. They could take further treatment breaks once their disease was back under control. The group on continuous treatment kept going until disease progression or intolerable toxicities. Median follow up was 58 months.

In both the per-protocol and intent-to-treat (ITT) populations, overall survival was 28 months for the people who received continuous treatment vs. 27 months for those who took a break. Statistical noninferiority was established in the ITT population but not in the per-protocol population.

The median length of all treatment breaks was 87 days. Many people took two or more breaks; one patient took nine breaks overall. The breaks were popular: only 3% of participants who were meant to stop therapy withdrew from the study in order to continue their treatment.

Said Dr. Hewison: “In the very early days of planning the study there were some doubts as to whether it would succeed because of potential unwillingness of people to stop treatment for a while.”

Dr. Brown agreed: “People did worry about that initially, but it actually seemed to be more the other way around. By that time – 6 months – people were relieved to be there. …We actually had some people from the other arm asking, could they also have a break?”

To understand better the benefits of treatment breaks to patients, Janine Bestall, PhD, a senior research fellow in applied health research at the University of Leeds, conducted a qualitative study in parallel with the main trial.

Summing up the patients’ experiences, Dr. Bestall said the drug-free periods “gave them more time.”

Dr. Bestall quoted one patient who said: “I know that things can happen and it grows back, but you’ve always got the buffer there knowing that you can go back and get help. But you actually lead a normal life and the advantage is, yeah, you can go on holiday, you can actually do more things in the garden, cleaning up, painting, whatever needs doing, you do it.”

Dr. Brown said, “I had a lady who, when she was on the trial, had four breaks in total, one when her daughter got married, and [she said] that was really nice for her to do all the shopping and all the normal things that you do, and not be on something that was making her tired and causing sore hands and diarrhea.” 

The drug-free interval strategy provided annual cost savings of 3,235 pounds sterling ($3,850) and a noninferior quality-adjusted life-year (QALY) benefit in both the ITT and per-protocol populations.

Serious adverse reactions occurred in 9% of patients in the treatment-break group versus 12% of the continuous-treatment group.

The authors of the study concluded, “Treatment breaks might be a feasible and cost-effective option with lifestyle benefits for patients during tyrosine kinase inhibitor therapy in patients with renal cell carcinoma.”
 

Changes in treatment strategies

The STAR trial started recruiting in January 2012.

Since that time, immunotherapy has taken over as first-line treatment for many patients with advanced ccRCC in both the United Kingdom and the United States.

However, TKIs still have a place. The NCCN Kidney Cancer 2022 Guidelines recommend both sunitinib and pazopanib as options for first-line therapy in advanced disease. The 2022 ASCO Metastatic ccRCC guidelines recommend either drug as first-line treatment in combination with an immune checkpoint inhibitor or in monotherapy if there are “coexisting medical problems.”

In the United States, intermittent sunitinib in metastatic RCC was tested in a small study in 2017 with little activity in the literature since then. The authors, led by Moshe Ornstein, MD, from the Cleveland Clinic, concluded at the time that sunitinib treatment breaks were feasible and “clinical efficacy does not seem to be compromised.” Dr. Ornstein was approached for comment on this latest U.K. study but declined.

Back in the United Kingdom, the results of STAR arrived just in time.

Said Dr. Brown: “This has … been really helpful in the U.K. in the pandemic when people said, can these patients have extra breaks? At the worst of the pandemic we were able to say, sure, if it’s stable, we can keep them off for 3-6 months. …And so that’s already had a powerful impact.”

Dr. Brown concluded, “I think what the trial does allow us to do, as individual oncologists, is to look at the patients that this might be suitable for – it won’t be everybody – and to say yes, it’s okay to personalize things.”

The study was funded by the U.K.’s National Institute for Health and Care Research. Dr. Bestall reported no relevant financial relationships. Dr. Hewison reported funding to her institution from the NIHR Health Technology Assessment. Dr. Brown reports having served as a consultant or adviser for Novartis, Ipsen, Amgen, Merck Sharp & Dohme, Bristol-Myers Squibb, and Bayer; honoraria from Novartis, Ipsen, Amgen, Merck Sharp & Dohme, Bristol-Myers Squibb, and Bayer; research funding paid to their institution from the National Institute for Health and Care Research; and travel expenses from Ipsen. Other coauthors reported numerous relationships with industry.

A version of this article first appeared on Medscape.com.

A physician’s specialty can make a difference when it comes to having suicidal thoughts. Doctors who specialize in family medicine, obstetrics-gynecology, and psychiatry reported double the rates of suicidal thoughts than doctors in oncology, rheumatology, and pulmonary medicine, according to Doctors’ Burden: Medscape Physician Suicide Report 2023.

“The specialties with the highest reporting of physician suicidal thoughts are also those with the greatest physician shortages, based on the number of job openings posted by recruiting sites,” said Peter Yellowlees, MD, professor of psychiatry and chief wellness officer at UC Davis Health.

Doctors in those specialties are overworked, which can lead to burnout, he said. “While burnout doesn’t cause depression, it’s correlated with depression and suicidal ideation.”

There’s also a generational divide among physicians who reported suicidal thoughts. Millennials (age 27-41) and Gen-X physicians (age 42-56) were more likely to report these thoughts than were Baby Boomers (age 57-75) and the Silent Generation (age 76-95).

“Younger physicians are more burned out – they may have less control over their lives and less meaning than some older doctors who can do what they want,” said Dr. Yellowlees.

One millennial respondent commented that being on call and being required to chart detailed notes in the EHR has contributed to her burnout. “I’m more impatient and make less time and effort to see my friends and family.”

One Silent Generation respondent commented, “I am semi-retired, I take no call, I work no weekends, I provide anesthesia care in my area of special expertise, I work clinically about 46 days a year. Life is good, particularly compared to my younger colleagues who are working 60-plus hours a week with evening work, weekend work, and call. I feel really sorry for them.”    

When young people enter medical school, they’re quite healthy, with low rates of depression and burnout, said Dr. Yellowlees. Yet, studies have shown that rates of burnout and suicidal thoughts increased within 2 years. “That reflects what happens when a group of idealistic young people hit a horrible system,” he said.
 

Who’s responsible?

Millennials were three times as likely as baby boomers to say that a medical school or health care organization should be responsible when a student or physician commits suicide.

“Young physicians may expect more of their employers than my generation did, which we see in residency programs that have unionized,” said Dr. Yellowlees, a Baby Boomer.

“As more young doctors are employed by health care organizations, they also may expect more resources to be available to them, such as wellness programs,” he added.

Younger doctors also focus more on work-life balance than older doctors, including time off and having hobbies, he said. “They are much more rational in terms of their overall beliefs and expectations than the older generation.”
 

Whom doctors confide in

Nearly 60% of physician-respondents with suicidal thoughts said they confided in a professional or someone they knew. Men were just as likely as women to reach out to a therapist (38%), whereas men were slightly more likely to confide in a family member and women were slightly more likely to confide in a colleague.

“It’s interesting that women are more active in seeking support at work – they often have developed a network of colleagues to support each other’s careers and whom they can confide in,” said Dr. Yellowlees.

He emphasized that 40% of physicians said they didn’t confide in anyone when they had suicidal thoughts. Of those, just over half said they could cope without professional help.

One respondent commented, “It’s just a thought; nothing I would actually do.” Another commented, “Mental health professionals can’t fix the underlying reason for the problem.”

Many doctors were concerned about risking disclosure to their medical boards (42%); that it would show up on their insurance records (33%); and that their colleagues would find out (25%), according to the report.

One respondent commented, “I don’t trust doctors to keep it to themselves.”

Another barrier doctors mentioned was a lack of time to seek help. One commented, “Time. I have none, when am I supposed to find an hour for counseling?”

A version of this article originally appeared on Medscape.com.

A physician’s specialty can make a difference when it comes to having suicidal thoughts. Doctors who specialize in family medicine, obstetrics-gynecology, and psychiatry reported double the rates of suicidal thoughts than doctors in oncology, rheumatology, and pulmonary medicine, according to Doctors’ Burden: Medscape Physician Suicide Report 2023.

“The specialties with the highest reporting of physician suicidal thoughts are also those with the greatest physician shortages, based on the number of job openings posted by recruiting sites,” said Peter Yellowlees, MD, professor of psychiatry and chief wellness officer at UC Davis Health.

Doctors in those specialties are overworked, which can lead to burnout, he said. “While burnout doesn’t cause depression, it’s correlated with depression and suicidal ideation.”

There’s also a generational divide among physicians who reported suicidal thoughts. Millennials (age 27-41) and Gen-X physicians (age 42-56) were more likely to report these thoughts than were Baby Boomers (age 57-75) and the Silent Generation (age 76-95).

“Younger physicians are more burned out – they may have less control over their lives and less meaning than some older doctors who can do what they want,” said Dr. Yellowlees.

One millennial respondent commented that being on call and being required to chart detailed notes in the EHR has contributed to her burnout. “I’m more impatient and make less time and effort to see my friends and family.”

One Silent Generation respondent commented, “I am semi-retired, I take no call, I work no weekends, I provide anesthesia care in my area of special expertise, I work clinically about 46 days a year. Life is good, particularly compared to my younger colleagues who are working 60-plus hours a week with evening work, weekend work, and call. I feel really sorry for them.”    

When young people enter medical school, they’re quite healthy, with low rates of depression and burnout, said Dr. Yellowlees. Yet, studies have shown that rates of burnout and suicidal thoughts increased within 2 years. “That reflects what happens when a group of idealistic young people hit a horrible system,” he said.
 

Who’s responsible?

Millennials were three times as likely as baby boomers to say that a medical school or health care organization should be responsible when a student or physician commits suicide.

“Young physicians may expect more of their employers than my generation did, which we see in residency programs that have unionized,” said Dr. Yellowlees, a Baby Boomer.

“As more young doctors are employed by health care organizations, they also may expect more resources to be available to them, such as wellness programs,” he added.

Younger doctors also focus more on work-life balance than older doctors, including time off and having hobbies, he said. “They are much more rational in terms of their overall beliefs and expectations than the older generation.”
 

Whom doctors confide in

Nearly 60% of physician-respondents with suicidal thoughts said they confided in a professional or someone they knew. Men were just as likely as women to reach out to a therapist (38%), whereas men were slightly more likely to confide in a family member and women were slightly more likely to confide in a colleague.

“It’s interesting that women are more active in seeking support at work – they often have developed a network of colleagues to support each other’s careers and whom they can confide in,” said Dr. Yellowlees.

He emphasized that 40% of physicians said they didn’t confide in anyone when they had suicidal thoughts. Of those, just over half said they could cope without professional help.

One respondent commented, “It’s just a thought; nothing I would actually do.” Another commented, “Mental health professionals can’t fix the underlying reason for the problem.”

Many doctors were concerned about risking disclosure to their medical boards (42%); that it would show up on their insurance records (33%); and that their colleagues would find out (25%), according to the report.

One respondent commented, “I don’t trust doctors to keep it to themselves.”

Another barrier doctors mentioned was a lack of time to seek help. One commented, “Time. I have none, when am I supposed to find an hour for counseling?”

A version of this article originally appeared on Medscape.com.

A physician’s specialty can make a difference when it comes to having suicidal thoughts. Doctors who specialize in family medicine, obstetrics-gynecology, and psychiatry reported double the rates of suicidal thoughts than doctors in oncology, rheumatology, and pulmonary medicine, according to Doctors’ Burden: Medscape Physician Suicide Report 2023.

“The specialties with the highest reporting of physician suicidal thoughts are also those with the greatest physician shortages, based on the number of job openings posted by recruiting sites,” said Peter Yellowlees, MD, professor of psychiatry and chief wellness officer at UC Davis Health.

Doctors in those specialties are overworked, which can lead to burnout, he said. “While burnout doesn’t cause depression, it’s correlated with depression and suicidal ideation.”

There’s also a generational divide among physicians who reported suicidal thoughts. Millennials (age 27-41) and Gen-X physicians (age 42-56) were more likely to report these thoughts than were Baby Boomers (age 57-75) and the Silent Generation (age 76-95).

“Younger physicians are more burned out – they may have less control over their lives and less meaning than some older doctors who can do what they want,” said Dr. Yellowlees.

One millennial respondent commented that being on call and being required to chart detailed notes in the EHR has contributed to her burnout. “I’m more impatient and make less time and effort to see my friends and family.”

One Silent Generation respondent commented, “I am semi-retired, I take no call, I work no weekends, I provide anesthesia care in my area of special expertise, I work clinically about 46 days a year. Life is good, particularly compared to my younger colleagues who are working 60-plus hours a week with evening work, weekend work, and call. I feel really sorry for them.”    

When young people enter medical school, they’re quite healthy, with low rates of depression and burnout, said Dr. Yellowlees. Yet, studies have shown that rates of burnout and suicidal thoughts increased within 2 years. “That reflects what happens when a group of idealistic young people hit a horrible system,” he said.
 

Who’s responsible?

Millennials were three times as likely as baby boomers to say that a medical school or health care organization should be responsible when a student or physician commits suicide.

“Young physicians may expect more of their employers than my generation did, which we see in residency programs that have unionized,” said Dr. Yellowlees, a Baby Boomer.

“As more young doctors are employed by health care organizations, they also may expect more resources to be available to them, such as wellness programs,” he added.

Younger doctors also focus more on work-life balance than older doctors, including time off and having hobbies, he said. “They are much more rational in terms of their overall beliefs and expectations than the older generation.”
 

Whom doctors confide in

Nearly 60% of physician-respondents with suicidal thoughts said they confided in a professional or someone they knew. Men were just as likely as women to reach out to a therapist (38%), whereas men were slightly more likely to confide in a family member and women were slightly more likely to confide in a colleague.

“It’s interesting that women are more active in seeking support at work – they often have developed a network of colleagues to support each other’s careers and whom they can confide in,” said Dr. Yellowlees.

He emphasized that 40% of physicians said they didn’t confide in anyone when they had suicidal thoughts. Of those, just over half said they could cope without professional help.

One respondent commented, “It’s just a thought; nothing I would actually do.” Another commented, “Mental health professionals can’t fix the underlying reason for the problem.”

Many doctors were concerned about risking disclosure to their medical boards (42%); that it would show up on their insurance records (33%); and that their colleagues would find out (25%), according to the report.

One respondent commented, “I don’t trust doctors to keep it to themselves.”

Another barrier doctors mentioned was a lack of time to seek help. One commented, “Time. I have none, when am I supposed to find an hour for counseling?”

A version of this article originally appeared on Medscape.com.

Sam Chavarría said her doctor was clear about the birth defects her medication could cause if she became pregnant but agreed to keep her on it as long as she had an IUD.

As she was waiting to get her contraceptive intrauterine device replaced at her local clinic, however, the billing nurse told her that her insurance wouldn’t cover the removal – or a new IUD. Chavarría didn’t understand why not.

“Then she said very delicately, ‘Well, people on this insurance typically tend to be older,’ ” Chavarría recalled.

Although Chavarría is 34, she is enrolled in Medicare, the government insurance program designed for those 65 and older. Chavarría, who lives in Houston, is disabled by fibromyalgia, rheumatoid arthritis, and mental health issues. Medicare automatically enrolls anyone who has received Social Security disability benefits for two years and this was her first time getting an IUD while in the government program.

Without insurance, just removing her expired IUD would cost Chavarría $350 out of pocket; exchanging it for a new one would be $2,000. She left the clinic in tears.

Chavarría’s experience is not rare. Medicare was originally intended for people of retirement age. Over the years, the program has evolved to include new populations, such as those who have disabilities or are critically ill, said Jennifer Lea Huer, a public health expert at Yale University, New Haven, Conn. In 2020, 1.7 million people ages 18-44 were enrolled in Medicare.

An estimated 70% of childbearing-age women on Medicare are also eligible for Medicaid, a state and federal program for those with low incomes, which should fill the gap for contraception. It’s not clear how many transgender or nonbinary people – who also might need contraception – are on Medicare or are eligible for Medicaid.

Medicaid, like the plans offered via the federal Affordable Care Act, mandates coverage of birth control. But those who aren’t eligible for Medicaid are left in the lurch – Medicare’s origins mean it does not require access to birth control.

Traditional Medicare includes two parts: Part A covers hospital costs, while Part B covers physicians’ care and certain other services, such as ambulance rides. Neither ordinarily includes contraception.

People can get contraception through a Medicare Advantage plan or Part D of Medicare, which covers prescription drugs, but those come at a cost. And even people who pay for Part D often aren’t covered for some types of birth control, such as IUDs.

“So, if you are disabled, if you are locked outside of the labor market, if you do not have the means or any other way to financially support yourself, you were likely still on traditional Medicare, which is Part A and Part B,” Huer said. “In which case, your access to contraception is incredibly difficult.”

Contraception for those with traditional Medicare is given on a case-by-case basis, Huer said. It can be covered only if a doctor can make a credible case that the patient needs it for medical reasons – because their body cannot sustain a pregnancy – as opposed to merely wanting to avoid one.

“You have to have a champion physician who’s willing to partner with you and make those arguments,” Huer said.

That’s what Chavarría’s doctor tried to do. Before she left the clinic, staffers there told her they would try to make the case she needed the IUD for medical reasons. The IUD exchange was scheduled almost 10 weeks later, but during those weeks, she got pregnant. Her body couldn’t sustain a pregnancy, so she and her partner rushed to get an abortion just before Texas tightened its rules Sept. 1, 2021.

“If Medicare had just covered the IUD removal or exchange to begin with, none of this would have happened,” Chavarría said. “It would have saved me having to make a really tough decision that I never thought I’d have to make.”

Women with disabilities often face a stigma from health care practitioners, especially when it comes to birth control, said Willi Horner-Johnson, a public health researcher specializing in disabilities at Oregon Health & Science University, Portland. In her research, women with disabilities have described being treated like children or having to go to multiple doctors to find someone with whom they felt comfortable.

“We don’t want to acknowledge that disabled people have sex,” said Miriam Garber, a 36-year-old sex worker who lives in Rhode Island and is also on Medicare because of her disabilities. Garber got an IUD from Planned Parenthood because her insurance wouldn’t cover it.

Even those who pay for Part D to have their prescription drugs covered and have a “champion physician” face difficulties. Liz Moore, a nonbinary person in their 30s who lives in the Washington, D.C., area, could not get Medicare to pay for the Mirena IUD their doctor prescribed for their polycystic ovary syndrome. Moore is disabled with fibromyalgia and dysautonomia, a condition of the autonomic nervous system, which regulates breathing, heart rate, and more.

“After literally months of phone calls, it seemed like my Medicare Part D and original Medicare could not agree on who should pay for my IUD,” they wrote in a direct message. “Was it a prescription or durable medical equipment?”

When Moore finally learned it would cost $800 upfront, they said, they decided to get a hysterectomy – which Medicare would pay for – instead.

Chavarría’s doctor told her a tubal ligation also was more likely to be approved by Medicare than an IUD, because older people have that procedure more often. Like all surgeries, both come with risks of complications and recovery.

Even for those on both Medicare and Medicaid, getting contraception also isn’t always easy, as in Katie Elizabeth Walsh’s case.

Walsh, 34, who lives in northeastern Connecticut, is disabled by a traumatic brain injury, depression, and chronic fatigue syndrome. She got an IUD at an ob.gyn. clinic and was told there her insurance would cover it.

Then she got a bill for nearly $2,000.

Medicaid should cover contraceptive devices for dual-eligibility people, according to Centers for Medicare & Medicaid Services policy guidance, but when Walsh tried to get her bill covered, Medicare and Medicaid could not agree on which of them should pay.

“Every single time I have called one of the insurance offices, they are like, ‘Oh, no, you have to talk to the other one, and we don’t really talk to each other,’ ” Walsh said.

Walsh said the hassle to get her contraception covered feels like a kick in the stomach: “Like truly you do not have a place in this world, and your insurance is telling you that.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Sam Chavarría said her doctor was clear about the birth defects her medication could cause if she became pregnant but agreed to keep her on it as long as she had an IUD.

As she was waiting to get her contraceptive intrauterine device replaced at her local clinic, however, the billing nurse told her that her insurance wouldn’t cover the removal – or a new IUD. Chavarría didn’t understand why not.

“Then she said very delicately, ‘Well, people on this insurance typically tend to be older,’ ” Chavarría recalled.

Although Chavarría is 34, she is enrolled in Medicare, the government insurance program designed for those 65 and older. Chavarría, who lives in Houston, is disabled by fibromyalgia, rheumatoid arthritis, and mental health issues. Medicare automatically enrolls anyone who has received Social Security disability benefits for two years and this was her first time getting an IUD while in the government program.

Without insurance, just removing her expired IUD would cost Chavarría $350 out of pocket; exchanging it for a new one would be $2,000. She left the clinic in tears.

Chavarría’s experience is not rare. Medicare was originally intended for people of retirement age. Over the years, the program has evolved to include new populations, such as those who have disabilities or are critically ill, said Jennifer Lea Huer, a public health expert at Yale University, New Haven, Conn. In 2020, 1.7 million people ages 18-44 were enrolled in Medicare.

An estimated 70% of childbearing-age women on Medicare are also eligible for Medicaid, a state and federal program for those with low incomes, which should fill the gap for contraception. It’s not clear how many transgender or nonbinary people – who also might need contraception – are on Medicare or are eligible for Medicaid.

Medicaid, like the plans offered via the federal Affordable Care Act, mandates coverage of birth control. But those who aren’t eligible for Medicaid are left in the lurch – Medicare’s origins mean it does not require access to birth control.

Traditional Medicare includes two parts: Part A covers hospital costs, while Part B covers physicians’ care and certain other services, such as ambulance rides. Neither ordinarily includes contraception.

People can get contraception through a Medicare Advantage plan or Part D of Medicare, which covers prescription drugs, but those come at a cost. And even people who pay for Part D often aren’t covered for some types of birth control, such as IUDs.

“So, if you are disabled, if you are locked outside of the labor market, if you do not have the means or any other way to financially support yourself, you were likely still on traditional Medicare, which is Part A and Part B,” Huer said. “In which case, your access to contraception is incredibly difficult.”

Contraception for those with traditional Medicare is given on a case-by-case basis, Huer said. It can be covered only if a doctor can make a credible case that the patient needs it for medical reasons – because their body cannot sustain a pregnancy – as opposed to merely wanting to avoid one.

“You have to have a champion physician who’s willing to partner with you and make those arguments,” Huer said.

That’s what Chavarría’s doctor tried to do. Before she left the clinic, staffers there told her they would try to make the case she needed the IUD for medical reasons. The IUD exchange was scheduled almost 10 weeks later, but during those weeks, she got pregnant. Her body couldn’t sustain a pregnancy, so she and her partner rushed to get an abortion just before Texas tightened its rules Sept. 1, 2021.

“If Medicare had just covered the IUD removal or exchange to begin with, none of this would have happened,” Chavarría said. “It would have saved me having to make a really tough decision that I never thought I’d have to make.”

Women with disabilities often face a stigma from health care practitioners, especially when it comes to birth control, said Willi Horner-Johnson, a public health researcher specializing in disabilities at Oregon Health & Science University, Portland. In her research, women with disabilities have described being treated like children or having to go to multiple doctors to find someone with whom they felt comfortable.

“We don’t want to acknowledge that disabled people have sex,” said Miriam Garber, a 36-year-old sex worker who lives in Rhode Island and is also on Medicare because of her disabilities. Garber got an IUD from Planned Parenthood because her insurance wouldn’t cover it.

Even those who pay for Part D to have their prescription drugs covered and have a “champion physician” face difficulties. Liz Moore, a nonbinary person in their 30s who lives in the Washington, D.C., area, could not get Medicare to pay for the Mirena IUD their doctor prescribed for their polycystic ovary syndrome. Moore is disabled with fibromyalgia and dysautonomia, a condition of the autonomic nervous system, which regulates breathing, heart rate, and more.

“After literally months of phone calls, it seemed like my Medicare Part D and original Medicare could not agree on who should pay for my IUD,” they wrote in a direct message. “Was it a prescription or durable medical equipment?”

When Moore finally learned it would cost $800 upfront, they said, they decided to get a hysterectomy – which Medicare would pay for – instead.

Chavarría’s doctor told her a tubal ligation also was more likely to be approved by Medicare than an IUD, because older people have that procedure more often. Like all surgeries, both come with risks of complications and recovery.

Even for those on both Medicare and Medicaid, getting contraception also isn’t always easy, as in Katie Elizabeth Walsh’s case.

Walsh, 34, who lives in northeastern Connecticut, is disabled by a traumatic brain injury, depression, and chronic fatigue syndrome. She got an IUD at an ob.gyn. clinic and was told there her insurance would cover it.

Then she got a bill for nearly $2,000.

Medicaid should cover contraceptive devices for dual-eligibility people, according to Centers for Medicare & Medicaid Services policy guidance, but when Walsh tried to get her bill covered, Medicare and Medicaid could not agree on which of them should pay.

“Every single time I have called one of the insurance offices, they are like, ‘Oh, no, you have to talk to the other one, and we don’t really talk to each other,’ ” Walsh said.

Walsh said the hassle to get her contraception covered feels like a kick in the stomach: “Like truly you do not have a place in this world, and your insurance is telling you that.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.

Sam Chavarría said her doctor was clear about the birth defects her medication could cause if she became pregnant but agreed to keep her on it as long as she had an IUD.

As she was waiting to get her contraceptive intrauterine device replaced at her local clinic, however, the billing nurse told her that her insurance wouldn’t cover the removal – or a new IUD. Chavarría didn’t understand why not.

“Then she said very delicately, ‘Well, people on this insurance typically tend to be older,’ ” Chavarría recalled.

Although Chavarría is 34, she is enrolled in Medicare, the government insurance program designed for those 65 and older. Chavarría, who lives in Houston, is disabled by fibromyalgia, rheumatoid arthritis, and mental health issues. Medicare automatically enrolls anyone who has received Social Security disability benefits for two years and this was her first time getting an IUD while in the government program.

Without insurance, just removing her expired IUD would cost Chavarría $350 out of pocket; exchanging it for a new one would be $2,000. She left the clinic in tears.

Chavarría’s experience is not rare. Medicare was originally intended for people of retirement age. Over the years, the program has evolved to include new populations, such as those who have disabilities or are critically ill, said Jennifer Lea Huer, a public health expert at Yale University, New Haven, Conn. In 2020, 1.7 million people ages 18-44 were enrolled in Medicare.

An estimated 70% of childbearing-age women on Medicare are also eligible for Medicaid, a state and federal program for those with low incomes, which should fill the gap for contraception. It’s not clear how many transgender or nonbinary people – who also might need contraception – are on Medicare or are eligible for Medicaid.

Medicaid, like the plans offered via the federal Affordable Care Act, mandates coverage of birth control. But those who aren’t eligible for Medicaid are left in the lurch – Medicare’s origins mean it does not require access to birth control.

Traditional Medicare includes two parts: Part A covers hospital costs, while Part B covers physicians’ care and certain other services, such as ambulance rides. Neither ordinarily includes contraception.

People can get contraception through a Medicare Advantage plan or Part D of Medicare, which covers prescription drugs, but those come at a cost. And even people who pay for Part D often aren’t covered for some types of birth control, such as IUDs.

“So, if you are disabled, if you are locked outside of the labor market, if you do not have the means or any other way to financially support yourself, you were likely still on traditional Medicare, which is Part A and Part B,” Huer said. “In which case, your access to contraception is incredibly difficult.”

Contraception for those with traditional Medicare is given on a case-by-case basis, Huer said. It can be covered only if a doctor can make a credible case that the patient needs it for medical reasons – because their body cannot sustain a pregnancy – as opposed to merely wanting to avoid one.

“You have to have a champion physician who’s willing to partner with you and make those arguments,” Huer said.

That’s what Chavarría’s doctor tried to do. Before she left the clinic, staffers there told her they would try to make the case she needed the IUD for medical reasons. The IUD exchange was scheduled almost 10 weeks later, but during those weeks, she got pregnant. Her body couldn’t sustain a pregnancy, so she and her partner rushed to get an abortion just before Texas tightened its rules Sept. 1, 2021.

“If Medicare had just covered the IUD removal or exchange to begin with, none of this would have happened,” Chavarría said. “It would have saved me having to make a really tough decision that I never thought I’d have to make.”

Women with disabilities often face a stigma from health care practitioners, especially when it comes to birth control, said Willi Horner-Johnson, a public health researcher specializing in disabilities at Oregon Health & Science University, Portland. In her research, women with disabilities have described being treated like children or having to go to multiple doctors to find someone with whom they felt comfortable.

“We don’t want to acknowledge that disabled people have sex,” said Miriam Garber, a 36-year-old sex worker who lives in Rhode Island and is also on Medicare because of her disabilities. Garber got an IUD from Planned Parenthood because her insurance wouldn’t cover it.

Even those who pay for Part D to have their prescription drugs covered and have a “champion physician” face difficulties. Liz Moore, a nonbinary person in their 30s who lives in the Washington, D.C., area, could not get Medicare to pay for the Mirena IUD their doctor prescribed for their polycystic ovary syndrome. Moore is disabled with fibromyalgia and dysautonomia, a condition of the autonomic nervous system, which regulates breathing, heart rate, and more.

“After literally months of phone calls, it seemed like my Medicare Part D and original Medicare could not agree on who should pay for my IUD,” they wrote in a direct message. “Was it a prescription or durable medical equipment?”

When Moore finally learned it would cost $800 upfront, they said, they decided to get a hysterectomy – which Medicare would pay for – instead.

Chavarría’s doctor told her a tubal ligation also was more likely to be approved by Medicare than an IUD, because older people have that procedure more often. Like all surgeries, both come with risks of complications and recovery.

Even for those on both Medicare and Medicaid, getting contraception also isn’t always easy, as in Katie Elizabeth Walsh’s case.

Walsh, 34, who lives in northeastern Connecticut, is disabled by a traumatic brain injury, depression, and chronic fatigue syndrome. She got an IUD at an ob.gyn. clinic and was told there her insurance would cover it.

Then she got a bill for nearly $2,000.

Medicaid should cover contraceptive devices for dual-eligibility people, according to Centers for Medicare & Medicaid Services policy guidance, but when Walsh tried to get her bill covered, Medicare and Medicaid could not agree on which of them should pay.

“Every single time I have called one of the insurance offices, they are like, ‘Oh, no, you have to talk to the other one, and we don’t really talk to each other,’ ” Walsh said.

Walsh said the hassle to get her contraception covered feels like a kick in the stomach: “Like truly you do not have a place in this world, and your insurance is telling you that.”

KHN (Kaiser Health News) is a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.