AVAHO

Idiopathic thrombocytopenic purpura (ITP) is an immune-mediated acquired condition affecting both adults and children.¹ Acute ITP is the most common form, which happens in the presence of a precipitant, leading to a drop in platelet counts. However, chronic ITP can occur when all the causes that might precipitate thrombocytopenia have been ruled out, and it is persistent for ≥ 12 months.² Its presence can mask other diseases that exhibit somewhat similar signs and symptoms. We present a case of a patient presenting with chronic ITP with diffuse rash and was later diagnosed with idiopathic leukocytoclastic vasculitis (LCV).

Case Presentation

A 79-year-old presented to the hospital with 2-day history of a rash. The rash was purpureal and petechial and located on the trunk and bilateral upper and lower extremities. The rash was associated with itchiness and pain in the wrists, ankles, and small joints of the hands. The patient reported no changes in medication or diet, no recent upper respiratory tract or gastrointestinal infections, fever or chills, night sweats, or weight loss. The patient’s medical history consisted of thrombocytopenia about 5 years before and since then had been following up with a hematologist and underwent an extensive workup, including bone marrow biopsy without a definite diagnosis.

The patient mentioned that at the time of diagnosis the platelet count was about 90,000 but had been fluctuating between 50 and 60,000 recently. The patient also reported no history of gum bleeding, nosebleeds, hemoptysis, hematemesis, or any miscarriages. She also had difficulty voiding for 2 to 3 days but no dysuria, frequency, urgency, or incontinence.

The patient was diagnosed with a urinary tract infection (UTI) 1 day before presentation and was started on ciprofloxacin 500 mg daily for 5 days. Her home medications included diphenhydramine as needed, metoprolol, and levothyroxine 125 µg. Her medical history was significant for hypertension, bradycardia with pacemaker placement, and obstructive sleep apnea. There were no noteworthy elements in her family and social history.

Laboratory results were significant for 57,000/µL platelet count (normal range, 150,000-450,000), elevated d-dimer (6.07), < 6 mg/dL C4 (normal range, 88-201). Hemoglobin level, coagulation panel, hemolytic panel, and fibrinogen level results were unremarkable. The hepatitis panel, Lyme disease, and HIV test were negative. The peripheral blood smear showed moderate thrombocytopenia, mild monocytosis, and borderline normochromic normocytic anemia without schistocytes. The autoimmune panel to evaluate thrombocytopenia showed platelet antibody against glycoprotein (GP) IIb/IIIa, GP Ib/Ix, GP Ia/IIa, suggestive toward a diagnosis of chronic idiopathic ITP. However, the skin biopsy of the rash was indicative of LCV.

An autoimmune panel for vasculitis, including antinuclear antibody and antidouble-stranded DNA, was negative. While in the hospital, the patient completed the course of ciprofloxacin for the UTI, the rash started to fade without any intervention, and the platelet count improved to 69,000/µL. The patient was discharged after 3 days with the recommendation to follow up with her hematologist.

Discussion

LCV is a small vessel vasculitis of the dermal capillaries and venules. Histologically, LCV is characterized by fibrinoid necrosis of the vessel wall with frequent neutrophils, nuclear dust, and extravasated erythrocytes.³

Although a thorough evaluation is recommended to determine etiology, about 50% of cases are idiopathic. The most common precipitants are acute infection or a new medication. Postinfectious LCV is most commonly seen after streptococcal upper respiratory tract infection. Among other infectious triggers, Mycobacterium, Staphylococcus aureus, chlamydia, Neisseria, HIV, hepatitis B, hepatitis C, and syphilis are noteworthy. Foods, autoimmune disease, collagen vascular disease, and malignancy are also associated with LCV.⁴

In our patient we could not find any specific identifiable triggers. However, the presence of a UTI as a precipitating factor cannot be ruled out.⁵ Moreover, the patient received ciprofloxacin and there have been several case reports of LCV associated with use of a fluroquinolone.⁶ Nevertheless, in the presence of chronic ITP, which also is an auto-immune condition, an idiopathic cause seemed a reasonable explanation for the patient’s etiopathogenesis.

The cutaneous manifestations of LCV may appear about 1 to 3 weeks after the triggering event if present. The major clinical findings include palpable purpura and/or petechiae that are nonblanching. These findings can easily be confused with other diagnoses especially in the presence of a similar preexisting diagnosis. For example, our patient already had chronic ITP, and in such circumstances, a diagnosis of superimposed LCV can be easily missed without a thorough investigation. Extracutaneous manifestations with LCV are less common. Systemic symptoms may include low-grade fevers, malaise, weight loss, myalgia, and arthralgia. These findings have been noted in about 30% of affected patients, with arthralgia the most common manifestation.⁷ Our patient also presented with pain involving multiple joints.

The mainstay of diagnosis for LCV is a skin biopsy with direct immunofluorescence. However, a workup for an underlying condition should be considered based on clinical suspicion. If a secondary cause is found, management should target treating the underlying cause, including withdrawal of the offending drug, treatment or control of the underlying infection, malignancy, or connective tissue disease. Most cases of idiopathic cutaneous LCV resolve with supportive measures, including leg elevation, rest, compression stockings, and antihistamines. In resistant cases, a 4- to 6-week tapering dose of corticosteroids and immunosuppressive steroid-sparing agents may be needed.⁸

Conclusions

Although most cases of LCV are mild and resolve without intervention, many cases go undiagnosed due to a delay in performing a biopsy. However, we should always look for the root cause of a patient’s condition to rule out underlying contributing conditions. Differentiating LCV from any other preexisting condition presenting similarly is important.

Idiopathic thrombocytopenic purpura (ITP) is an immune-mediated acquired condition affecting both adults and children.¹ Acute ITP is the most common form, which happens in the presence of a precipitant, leading to a drop in platelet counts. However, chronic ITP can occur when all the causes that might precipitate thrombocytopenia have been ruled out, and it is persistent for ≥ 12 months.² Its presence can mask other diseases that exhibit somewhat similar signs and symptoms. We present a case of a patient presenting with chronic ITP with diffuse rash and was later diagnosed with idiopathic leukocytoclastic vasculitis (LCV).

Case Presentation

A 79-year-old presented to the hospital with 2-day history of a rash. The rash was purpureal and petechial and located on the trunk and bilateral upper and lower extremities. The rash was associated with itchiness and pain in the wrists, ankles, and small joints of the hands. The patient reported no changes in medication or diet, no recent upper respiratory tract or gastrointestinal infections, fever or chills, night sweats, or weight loss. The patient’s medical history consisted of thrombocytopenia about 5 years before and since then had been following up with a hematologist and underwent an extensive workup, including bone marrow biopsy without a definite diagnosis.

The patient mentioned that at the time of diagnosis the platelet count was about 90,000 but had been fluctuating between 50 and 60,000 recently. The patient also reported no history of gum bleeding, nosebleeds, hemoptysis, hematemesis, or any miscarriages. She also had difficulty voiding for 2 to 3 days but no dysuria, frequency, urgency, or incontinence.

The patient was diagnosed with a urinary tract infection (UTI) 1 day before presentation and was started on ciprofloxacin 500 mg daily for 5 days. Her home medications included diphenhydramine as needed, metoprolol, and levothyroxine 125 µg. Her medical history was significant for hypertension, bradycardia with pacemaker placement, and obstructive sleep apnea. There were no noteworthy elements in her family and social history.

Laboratory results were significant for 57,000/µL platelet count (normal range, 150,000-450,000), elevated d-dimer (6.07), < 6 mg/dL C4 (normal range, 88-201). Hemoglobin level, coagulation panel, hemolytic panel, and fibrinogen level results were unremarkable. The hepatitis panel, Lyme disease, and HIV test were negative. The peripheral blood smear showed moderate thrombocytopenia, mild monocytosis, and borderline normochromic normocytic anemia without schistocytes. The autoimmune panel to evaluate thrombocytopenia showed platelet antibody against glycoprotein (GP) IIb/IIIa, GP Ib/Ix, GP Ia/IIa, suggestive toward a diagnosis of chronic idiopathic ITP. However, the skin biopsy of the rash was indicative of LCV.

An autoimmune panel for vasculitis, including antinuclear antibody and antidouble-stranded DNA, was negative. While in the hospital, the patient completed the course of ciprofloxacin for the UTI, the rash started to fade without any intervention, and the platelet count improved to 69,000/µL. The patient was discharged after 3 days with the recommendation to follow up with her hematologist.

Discussion

LCV is a small vessel vasculitis of the dermal capillaries and venules. Histologically, LCV is characterized by fibrinoid necrosis of the vessel wall with frequent neutrophils, nuclear dust, and extravasated erythrocytes.³

Although a thorough evaluation is recommended to determine etiology, about 50% of cases are idiopathic. The most common precipitants are acute infection or a new medication. Postinfectious LCV is most commonly seen after streptococcal upper respiratory tract infection. Among other infectious triggers, Mycobacterium, Staphylococcus aureus, chlamydia, Neisseria, HIV, hepatitis B, hepatitis C, and syphilis are noteworthy. Foods, autoimmune disease, collagen vascular disease, and malignancy are also associated with LCV.⁴

In our patient we could not find any specific identifiable triggers. However, the presence of a UTI as a precipitating factor cannot be ruled out.⁵ Moreover, the patient received ciprofloxacin and there have been several case reports of LCV associated with use of a fluroquinolone.⁶ Nevertheless, in the presence of chronic ITP, which also is an auto-immune condition, an idiopathic cause seemed a reasonable explanation for the patient’s etiopathogenesis.

The cutaneous manifestations of LCV may appear about 1 to 3 weeks after the triggering event if present. The major clinical findings include palpable purpura and/or petechiae that are nonblanching. These findings can easily be confused with other diagnoses especially in the presence of a similar preexisting diagnosis. For example, our patient already had chronic ITP, and in such circumstances, a diagnosis of superimposed LCV can be easily missed without a thorough investigation. Extracutaneous manifestations with LCV are less common. Systemic symptoms may include low-grade fevers, malaise, weight loss, myalgia, and arthralgia. These findings have been noted in about 30% of affected patients, with arthralgia the most common manifestation.⁷ Our patient also presented with pain involving multiple joints.

The mainstay of diagnosis for LCV is a skin biopsy with direct immunofluorescence. However, a workup for an underlying condition should be considered based on clinical suspicion. If a secondary cause is found, management should target treating the underlying cause, including withdrawal of the offending drug, treatment or control of the underlying infection, malignancy, or connective tissue disease. Most cases of idiopathic cutaneous LCV resolve with supportive measures, including leg elevation, rest, compression stockings, and antihistamines. In resistant cases, a 4- to 6-week tapering dose of corticosteroids and immunosuppressive steroid-sparing agents may be needed.⁸

Conclusions

Although most cases of LCV are mild and resolve without intervention, many cases go undiagnosed due to a delay in performing a biopsy. However, we should always look for the root cause of a patient’s condition to rule out underlying contributing conditions. Differentiating LCV from any other preexisting condition presenting similarly is important.

Idiopathic thrombocytopenic purpura (ITP) is an immune-mediated acquired condition affecting both adults and children.¹ Acute ITP is the most common form, which happens in the presence of a precipitant, leading to a drop in platelet counts. However, chronic ITP can occur when all the causes that might precipitate thrombocytopenia have been ruled out, and it is persistent for ≥ 12 months.² Its presence can mask other diseases that exhibit somewhat similar signs and symptoms. We present a case of a patient presenting with chronic ITP with diffuse rash and was later diagnosed with idiopathic leukocytoclastic vasculitis (LCV).

Case Presentation

A 79-year-old presented to the hospital with 2-day history of a rash. The rash was purpureal and petechial and located on the trunk and bilateral upper and lower extremities. The rash was associated with itchiness and pain in the wrists, ankles, and small joints of the hands. The patient reported no changes in medication or diet, no recent upper respiratory tract or gastrointestinal infections, fever or chills, night sweats, or weight loss. The patient’s medical history consisted of thrombocytopenia about 5 years before and since then had been following up with a hematologist and underwent an extensive workup, including bone marrow biopsy without a definite diagnosis.

The patient mentioned that at the time of diagnosis the platelet count was about 90,000 but had been fluctuating between 50 and 60,000 recently. The patient also reported no history of gum bleeding, nosebleeds, hemoptysis, hematemesis, or any miscarriages. She also had difficulty voiding for 2 to 3 days but no dysuria, frequency, urgency, or incontinence.

The patient was diagnosed with a urinary tract infection (UTI) 1 day before presentation and was started on ciprofloxacin 500 mg daily for 5 days. Her home medications included diphenhydramine as needed, metoprolol, and levothyroxine 125 µg. Her medical history was significant for hypertension, bradycardia with pacemaker placement, and obstructive sleep apnea. There were no noteworthy elements in her family and social history.

Laboratory results were significant for 57,000/µL platelet count (normal range, 150,000-450,000), elevated d-dimer (6.07), < 6 mg/dL C4 (normal range, 88-201). Hemoglobin level, coagulation panel, hemolytic panel, and fibrinogen level results were unremarkable. The hepatitis panel, Lyme disease, and HIV test were negative. The peripheral blood smear showed moderate thrombocytopenia, mild monocytosis, and borderline normochromic normocytic anemia without schistocytes. The autoimmune panel to evaluate thrombocytopenia showed platelet antibody against glycoprotein (GP) IIb/IIIa, GP Ib/Ix, GP Ia/IIa, suggestive toward a diagnosis of chronic idiopathic ITP. However, the skin biopsy of the rash was indicative of LCV.

An autoimmune panel for vasculitis, including antinuclear antibody and antidouble-stranded DNA, was negative. While in the hospital, the patient completed the course of ciprofloxacin for the UTI, the rash started to fade without any intervention, and the platelet count improved to 69,000/µL. The patient was discharged after 3 days with the recommendation to follow up with her hematologist.

Discussion

LCV is a small vessel vasculitis of the dermal capillaries and venules. Histologically, LCV is characterized by fibrinoid necrosis of the vessel wall with frequent neutrophils, nuclear dust, and extravasated erythrocytes.³

Although a thorough evaluation is recommended to determine etiology, about 50% of cases are idiopathic. The most common precipitants are acute infection or a new medication. Postinfectious LCV is most commonly seen after streptococcal upper respiratory tract infection. Among other infectious triggers, Mycobacterium, Staphylococcus aureus, chlamydia, Neisseria, HIV, hepatitis B, hepatitis C, and syphilis are noteworthy. Foods, autoimmune disease, collagen vascular disease, and malignancy are also associated with LCV.⁴

In our patient we could not find any specific identifiable triggers. However, the presence of a UTI as a precipitating factor cannot be ruled out.⁵ Moreover, the patient received ciprofloxacin and there have been several case reports of LCV associated with use of a fluroquinolone.⁶ Nevertheless, in the presence of chronic ITP, which also is an auto-immune condition, an idiopathic cause seemed a reasonable explanation for the patient’s etiopathogenesis.

The cutaneous manifestations of LCV may appear about 1 to 3 weeks after the triggering event if present. The major clinical findings include palpable purpura and/or petechiae that are nonblanching. These findings can easily be confused with other diagnoses especially in the presence of a similar preexisting diagnosis. For example, our patient already had chronic ITP, and in such circumstances, a diagnosis of superimposed LCV can be easily missed without a thorough investigation. Extracutaneous manifestations with LCV are less common. Systemic symptoms may include low-grade fevers, malaise, weight loss, myalgia, and arthralgia. These findings have been noted in about 30% of affected patients, with arthralgia the most common manifestation.⁷ Our patient also presented with pain involving multiple joints.

The mainstay of diagnosis for LCV is a skin biopsy with direct immunofluorescence. However, a workup for an underlying condition should be considered based on clinical suspicion. If a secondary cause is found, management should target treating the underlying cause, including withdrawal of the offending drug, treatment or control of the underlying infection, malignancy, or connective tissue disease. Most cases of idiopathic cutaneous LCV resolve with supportive measures, including leg elevation, rest, compression stockings, and antihistamines. In resistant cases, a 4- to 6-week tapering dose of corticosteroids and immunosuppressive steroid-sparing agents may be needed.⁸

Conclusions

Although most cases of LCV are mild and resolve without intervention, many cases go undiagnosed due to a delay in performing a biopsy. However, we should always look for the root cause of a patient’s condition to rule out underlying contributing conditions. Differentiating LCV from any other preexisting condition presenting similarly is important.

Women with polycystic ovary syndrome (PCOS) may be at a higher risk of developing pancreatic cancer, say researchers reporting a single-center case-control study.

A diagnosis of PCOS was associated with a 1.9-fold higher risk of pancreatic cancer after adjusting for age, race, ethnicity, estrogen level, and diabetes.

This is the second study to find such an association.

“Our study findings combined with those from the 2019 Swedish Registry study offer compelling evidence that PCOS may be a novel risk factor for pancreatic cancer,” said corresponding author Mengmeng Du, ScD, department of epidemiology and biostatistics, Memorial Sloan Kettering Cancer Center.

“These data suggest some individuals may have unknown metabolic derangements that may underlie the development of both conditions,” the team concluded.

The findings were published in JAMA Oncology.

Approached for comment, Srinivas Gaddam, MD, MPH, associate director of pancreatic biliary research medicine, Cedars-Sinai, suggested that the findings may pave the way for a better understanding of the two diseases, but he emphasized that more research is needed.

“I think there’s more research to be done because now we’re seeing more younger women get pancreatic cancer,” Dr. Gaddam said. “So that makes it interesting whether PCOS itself contributes to pancreatic cancer. I still think the jury is out there.”

Dr. Gaddam drew attention to the confidence interval for the finding – the adjusted odds ratio was 1.88 (95% confidence interval, 1.02-3.46). “Because their odds ratio includes 1, I’m left with the question as to whether or not this is truly associated. I’m not certain that we can draw any conclusions based on this,” he commented.

The investigators acknowledge that they did “not observe statistically significant interactions” and comment that “prospective studies are needed to examine underlying biologic mechanisms and confirm our findings.”

For the study, the team used data from the Memorial Sloan Kettering Cancer Center Pancreatic Tumor Registry. They identified patients with pancreatic cancer who also self-reported a diagnosis of PCOS.

The investigators compared data from 446 women with pathologically or cytologically confirmed pancreatic adenocarcinoma with 209 women who had no history of cancer. The mean age at cancer diagnosis or enrollment was 63.8 years among patients with pancreatic cancer and 57.7 years in the control group.

The study found that having PCOS nearly doubled a person’s risk of developing pancreatic cancer.

When adjusted for type 2 diabetes diagnosis, the odds ratio fell slightly to 1.78 (95% CI, 0.95-3.34).

Dr. Du, along with lead author Noah Peeri, PhD, were surprised that even after adjusting for body mass index and the presence of type 2 diabetes, PCOS remained strongly associated with pancreatic cancer risk.

“We originally thought type 2 diabetes may drive this association, given more than half of those with PCOS develop type 2 diabetes by age 40, according to the CDC, and type 2 diabetes has also been linked with increased pancreatic cancer risk,” said Dr. Du.

“While the association was slightly weaker and no longer statistically significant after we controlled for type 2 diabetes, the magnitude of the association remained largely unchanged,” he said.

Dr. Peeri believes that some of the factors that have been causally related to PCOS may increase an individual’s pancreatic cancer risk.

“PCOS itself does not likely cause pancreatic cancer, but metabolic problems (for example, improper breakdown of insulin) and chronic inflammation can contribute to both PCOS and pancreatic cancer risk,” Dr. Peeri said.

He concluded that the study results “suggest other underlying metabolic dysfunction may increase an individual’s pancreatic cancer risk.”

An important limitation of this study was that women in the study self-reported PCOS and may have incorrectly recalled their diagnosis. However, the authors believe it is unlikely that that had a bearing on the study findings.

The study was supported by National Cancer Institute grants, the Geoffrey Beene Foundation, and the Arnold and Arlene Goldstein Family Foundation.

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

Women with polycystic ovary syndrome (PCOS) may be at a higher risk of developing pancreatic cancer, say researchers reporting a single-center case-control study.

A diagnosis of PCOS was associated with a 1.9-fold higher risk of pancreatic cancer after adjusting for age, race, ethnicity, estrogen level, and diabetes.

This is the second study to find such an association.

“Our study findings combined with those from the 2019 Swedish Registry study offer compelling evidence that PCOS may be a novel risk factor for pancreatic cancer,” said corresponding author Mengmeng Du, ScD, department of epidemiology and biostatistics, Memorial Sloan Kettering Cancer Center.

“These data suggest some individuals may have unknown metabolic derangements that may underlie the development of both conditions,” the team concluded.

The findings were published in JAMA Oncology.

Approached for comment, Srinivas Gaddam, MD, MPH, associate director of pancreatic biliary research medicine, Cedars-Sinai, suggested that the findings may pave the way for a better understanding of the two diseases, but he emphasized that more research is needed.

“I think there’s more research to be done because now we’re seeing more younger women get pancreatic cancer,” Dr. Gaddam said. “So that makes it interesting whether PCOS itself contributes to pancreatic cancer. I still think the jury is out there.”

Dr. Gaddam drew attention to the confidence interval for the finding – the adjusted odds ratio was 1.88 (95% confidence interval, 1.02-3.46). “Because their odds ratio includes 1, I’m left with the question as to whether or not this is truly associated. I’m not certain that we can draw any conclusions based on this,” he commented.

The investigators acknowledge that they did “not observe statistically significant interactions” and comment that “prospective studies are needed to examine underlying biologic mechanisms and confirm our findings.”

For the study, the team used data from the Memorial Sloan Kettering Cancer Center Pancreatic Tumor Registry. They identified patients with pancreatic cancer who also self-reported a diagnosis of PCOS.

The investigators compared data from 446 women with pathologically or cytologically confirmed pancreatic adenocarcinoma with 209 women who had no history of cancer. The mean age at cancer diagnosis or enrollment was 63.8 years among patients with pancreatic cancer and 57.7 years in the control group.

The study found that having PCOS nearly doubled a person’s risk of developing pancreatic cancer.

When adjusted for type 2 diabetes diagnosis, the odds ratio fell slightly to 1.78 (95% CI, 0.95-3.34).

Dr. Du, along with lead author Noah Peeri, PhD, were surprised that even after adjusting for body mass index and the presence of type 2 diabetes, PCOS remained strongly associated with pancreatic cancer risk.

“We originally thought type 2 diabetes may drive this association, given more than half of those with PCOS develop type 2 diabetes by age 40, according to the CDC, and type 2 diabetes has also been linked with increased pancreatic cancer risk,” said Dr. Du.

“While the association was slightly weaker and no longer statistically significant after we controlled for type 2 diabetes, the magnitude of the association remained largely unchanged,” he said.

Dr. Peeri believes that some of the factors that have been causally related to PCOS may increase an individual’s pancreatic cancer risk.

“PCOS itself does not likely cause pancreatic cancer, but metabolic problems (for example, improper breakdown of insulin) and chronic inflammation can contribute to both PCOS and pancreatic cancer risk,” Dr. Peeri said.

He concluded that the study results “suggest other underlying metabolic dysfunction may increase an individual’s pancreatic cancer risk.”

An important limitation of this study was that women in the study self-reported PCOS and may have incorrectly recalled their diagnosis. However, the authors believe it is unlikely that that had a bearing on the study findings.

The study was supported by National Cancer Institute grants, the Geoffrey Beene Foundation, and the Arnold and Arlene Goldstein Family Foundation.

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

Women with polycystic ovary syndrome (PCOS) may be at a higher risk of developing pancreatic cancer, say researchers reporting a single-center case-control study.

A diagnosis of PCOS was associated with a 1.9-fold higher risk of pancreatic cancer after adjusting for age, race, ethnicity, estrogen level, and diabetes.

This is the second study to find such an association.

“Our study findings combined with those from the 2019 Swedish Registry study offer compelling evidence that PCOS may be a novel risk factor for pancreatic cancer,” said corresponding author Mengmeng Du, ScD, department of epidemiology and biostatistics, Memorial Sloan Kettering Cancer Center.

“These data suggest some individuals may have unknown metabolic derangements that may underlie the development of both conditions,” the team concluded.

The findings were published in JAMA Oncology.

Approached for comment, Srinivas Gaddam, MD, MPH, associate director of pancreatic biliary research medicine, Cedars-Sinai, suggested that the findings may pave the way for a better understanding of the two diseases, but he emphasized that more research is needed.

“I think there’s more research to be done because now we’re seeing more younger women get pancreatic cancer,” Dr. Gaddam said. “So that makes it interesting whether PCOS itself contributes to pancreatic cancer. I still think the jury is out there.”

Dr. Gaddam drew attention to the confidence interval for the finding – the adjusted odds ratio was 1.88 (95% confidence interval, 1.02-3.46). “Because their odds ratio includes 1, I’m left with the question as to whether or not this is truly associated. I’m not certain that we can draw any conclusions based on this,” he commented.

The investigators acknowledge that they did “not observe statistically significant interactions” and comment that “prospective studies are needed to examine underlying biologic mechanisms and confirm our findings.”

For the study, the team used data from the Memorial Sloan Kettering Cancer Center Pancreatic Tumor Registry. They identified patients with pancreatic cancer who also self-reported a diagnosis of PCOS.

The investigators compared data from 446 women with pathologically or cytologically confirmed pancreatic adenocarcinoma with 209 women who had no history of cancer. The mean age at cancer diagnosis or enrollment was 63.8 years among patients with pancreatic cancer and 57.7 years in the control group.

The study found that having PCOS nearly doubled a person’s risk of developing pancreatic cancer.

When adjusted for type 2 diabetes diagnosis, the odds ratio fell slightly to 1.78 (95% CI, 0.95-3.34).

Dr. Du, along with lead author Noah Peeri, PhD, were surprised that even after adjusting for body mass index and the presence of type 2 diabetes, PCOS remained strongly associated with pancreatic cancer risk.

“We originally thought type 2 diabetes may drive this association, given more than half of those with PCOS develop type 2 diabetes by age 40, according to the CDC, and type 2 diabetes has also been linked with increased pancreatic cancer risk,” said Dr. Du.

“While the association was slightly weaker and no longer statistically significant after we controlled for type 2 diabetes, the magnitude of the association remained largely unchanged,” he said.

Dr. Peeri believes that some of the factors that have been causally related to PCOS may increase an individual’s pancreatic cancer risk.

“PCOS itself does not likely cause pancreatic cancer, but metabolic problems (for example, improper breakdown of insulin) and chronic inflammation can contribute to both PCOS and pancreatic cancer risk,” Dr. Peeri said.

He concluded that the study results “suggest other underlying metabolic dysfunction may increase an individual’s pancreatic cancer risk.”

An important limitation of this study was that women in the study self-reported PCOS and may have incorrectly recalled their diagnosis. However, the authors believe it is unlikely that that had a bearing on the study findings.

The study was supported by National Cancer Institute grants, the Geoffrey Beene Foundation, and the Arnold and Arlene Goldstein Family Foundation.

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

Response-adapted de-escalation of immunotherapy in patients with advanced melanoma may lead to considerable savings for payers – close to $20,000 per patient – compared with standard immunotherapy, an economic analysis found.

“The rising costs of cancer therapies are becoming untenable for both patients and payers, and there is both clinical and economic benefit to finding less expensive treatment alternatives,” Wolfgang Kunz, MD, University Hospital, Ludwig Maximilian University of Munich, told this news organization.

This economic analysis “highlights that leveraging modern diagnostic capabilities can do just that: Pairing drug regimens with CT-image analysis to optimize dosages can reduce health care costs and improve clinical outcomes,” Dr. Kunz said.

The study was published online in JAMA Dermatology.

While the use of immunotherapies over the past decade has improved the prognosis for patients with advanced melanoma, these drugs come with a hefty price tag.

One potential way to help reduce costs: de-escalate therapy. The ADAPT-IT trial demonstrated similar progression-free and overall survival among patients who received response-adapted ipilimumab discontinuation and those who received standard of care.

In the current analysis, Dr. Kunz and colleagues wanted to understand whether this response-adapted approach was also cost effective.

The team applied economic modeling to data from the ADAPT-IT trial as well as CheckMate 067, in which patients received standard of care four doses of combination ipilimumab-nivolumab followed by nivolumab monotherapy. In the ADAPT-IT trial, patients also initially received the immunotherapy combination but had CT scans to determine their response after two doses; if they responded, patients discontinued ipilimumab and continued with nivolumab monotherapy.

Overall, ADAPT-IT showed that responders could forgo the additional two doses of ipilimumab plus nivolumab while maintaining similar progression-free survival and overall survival seen at 18 months in the CheckMate 067 trial.

The current economic analysis, based on 41 patients from ADAPT-IT and 314 from CheckMate 067, showed a potential reduction in health care costs of $19,891 per patient with the response-adapted approach.

Response-adapted treatment was the cost-effective option in 94% of simulated scenarios.

When extrapolated to 2019 incidence rates of distant melanoma cases, yearly national savings could reach about $58 million.

“In the relatively small space of immunotherapies in advanced melanoma, we hope this analysis motivates clinicians to consider response-adapted treatment,” Dr. Kunz told this news organization.

“On the larger scale, this analysis serves as a stepping stone to more response-guided treatment protocols,” Dr. Kunz added. “With drug costs rising and imaging capabilities growing, more frequent image-guided adjustments are a perfect fit into the personalized care model.”

When applying the cost savings noted in this analysis across all treated patients, “the economic impact may be profound,” said Joseph Skitzki, MD, surgical oncologist, Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., who wasn’t involved in the study. The “financial toxicity of cancer care is increasingly recognized as a potential barrier to optimal outcomes and any measures to mitigate cost may be impactful.”

However, Dr. Skitzki said several caveats need to be considered.

One is that the data included from ADAPT-IT only included 41 patients, compared with 314 patients from CheckMate 067.

“It is possible that a larger real-world study utilizing the ADAPT-IT protocol may not be as favorable in terms of outcomes and could lessen the economic impact of de-escalation, although any form of de-escalation is likely to have a cost benefit,” Dr. Skitzki said in an interview.

A real-world response–adapted de-escalation clinical trial, with an emphasis on costs and a benchmark of similar progression-free and overall survival, should be conducted before the de-escalated option becomes “practice changing,” Dr. Skitzki said.

Jeffrey Weber, MD, PhD, deputy director, Perlmutter Cancer Center, NYU Langone Health, New York, also urged caution in interpreting the results.

“I would not base treatment decisions on a small sampling of 41 patients in the absence of a randomized comparison,” Dr. Weber told this news organization. “Without a proper comparison, I would not advocate using only two doses of ipilimumab-nivolumab to make decisions on treatment.”

Dr. Skitzki added that, while “studies like this one are desperately needed to lessen the economic impact of new and emerging combination immunotherapies,” there is likely also a “disincentive for pharmaceutical companies to conduct this type of research.”

This research had no specific funding. Dr. Kunz and Dr. Skitzki reported no relevant conflicts of interest. Dr. Weber disclosed relationships with Merck, Genentech, AstraZeneca, Pfizer, Regeneron, and GSK, among others, and holds equity in Cytomx, Biond, NexImmune, and Immunomax.

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

Response-adapted de-escalation of immunotherapy in patients with advanced melanoma may lead to considerable savings for payers – close to $20,000 per patient – compared with standard immunotherapy, an economic analysis found.

“The rising costs of cancer therapies are becoming untenable for both patients and payers, and there is both clinical and economic benefit to finding less expensive treatment alternatives,” Wolfgang Kunz, MD, University Hospital, Ludwig Maximilian University of Munich, told this news organization.

This economic analysis “highlights that leveraging modern diagnostic capabilities can do just that: Pairing drug regimens with CT-image analysis to optimize dosages can reduce health care costs and improve clinical outcomes,” Dr. Kunz said.

The study was published online in JAMA Dermatology.

While the use of immunotherapies over the past decade has improved the prognosis for patients with advanced melanoma, these drugs come with a hefty price tag.

One potential way to help reduce costs: de-escalate therapy. The ADAPT-IT trial demonstrated similar progression-free and overall survival among patients who received response-adapted ipilimumab discontinuation and those who received standard of care.

In the current analysis, Dr. Kunz and colleagues wanted to understand whether this response-adapted approach was also cost effective.

The team applied economic modeling to data from the ADAPT-IT trial as well as CheckMate 067, in which patients received standard of care four doses of combination ipilimumab-nivolumab followed by nivolumab monotherapy. In the ADAPT-IT trial, patients also initially received the immunotherapy combination but had CT scans to determine their response after two doses; if they responded, patients discontinued ipilimumab and continued with nivolumab monotherapy.

Overall, ADAPT-IT showed that responders could forgo the additional two doses of ipilimumab plus nivolumab while maintaining similar progression-free survival and overall survival seen at 18 months in the CheckMate 067 trial.

The current economic analysis, based on 41 patients from ADAPT-IT and 314 from CheckMate 067, showed a potential reduction in health care costs of $19,891 per patient with the response-adapted approach.

Response-adapted treatment was the cost-effective option in 94% of simulated scenarios.

When extrapolated to 2019 incidence rates of distant melanoma cases, yearly national savings could reach about $58 million.

“In the relatively small space of immunotherapies in advanced melanoma, we hope this analysis motivates clinicians to consider response-adapted treatment,” Dr. Kunz told this news organization.

“On the larger scale, this analysis serves as a stepping stone to more response-guided treatment protocols,” Dr. Kunz added. “With drug costs rising and imaging capabilities growing, more frequent image-guided adjustments are a perfect fit into the personalized care model.”

When applying the cost savings noted in this analysis across all treated patients, “the economic impact may be profound,” said Joseph Skitzki, MD, surgical oncologist, Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., who wasn’t involved in the study. The “financial toxicity of cancer care is increasingly recognized as a potential barrier to optimal outcomes and any measures to mitigate cost may be impactful.”

However, Dr. Skitzki said several caveats need to be considered.

One is that the data included from ADAPT-IT only included 41 patients, compared with 314 patients from CheckMate 067.

“It is possible that a larger real-world study utilizing the ADAPT-IT protocol may not be as favorable in terms of outcomes and could lessen the economic impact of de-escalation, although any form of de-escalation is likely to have a cost benefit,” Dr. Skitzki said in an interview.

A real-world response–adapted de-escalation clinical trial, with an emphasis on costs and a benchmark of similar progression-free and overall survival, should be conducted before the de-escalated option becomes “practice changing,” Dr. Skitzki said.

Jeffrey Weber, MD, PhD, deputy director, Perlmutter Cancer Center, NYU Langone Health, New York, also urged caution in interpreting the results.

“I would not base treatment decisions on a small sampling of 41 patients in the absence of a randomized comparison,” Dr. Weber told this news organization. “Without a proper comparison, I would not advocate using only two doses of ipilimumab-nivolumab to make decisions on treatment.”

Dr. Skitzki added that, while “studies like this one are desperately needed to lessen the economic impact of new and emerging combination immunotherapies,” there is likely also a “disincentive for pharmaceutical companies to conduct this type of research.”

This research had no specific funding. Dr. Kunz and Dr. Skitzki reported no relevant conflicts of interest. Dr. Weber disclosed relationships with Merck, Genentech, AstraZeneca, Pfizer, Regeneron, and GSK, among others, and holds equity in Cytomx, Biond, NexImmune, and Immunomax.

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

Response-adapted de-escalation of immunotherapy in patients with advanced melanoma may lead to considerable savings for payers – close to $20,000 per patient – compared with standard immunotherapy, an economic analysis found.

“The rising costs of cancer therapies are becoming untenable for both patients and payers, and there is both clinical and economic benefit to finding less expensive treatment alternatives,” Wolfgang Kunz, MD, University Hospital, Ludwig Maximilian University of Munich, told this news organization.

This economic analysis “highlights that leveraging modern diagnostic capabilities can do just that: Pairing drug regimens with CT-image analysis to optimize dosages can reduce health care costs and improve clinical outcomes,” Dr. Kunz said.

The study was published online in JAMA Dermatology.

While the use of immunotherapies over the past decade has improved the prognosis for patients with advanced melanoma, these drugs come with a hefty price tag.

One potential way to help reduce costs: de-escalate therapy. The ADAPT-IT trial demonstrated similar progression-free and overall survival among patients who received response-adapted ipilimumab discontinuation and those who received standard of care.

In the current analysis, Dr. Kunz and colleagues wanted to understand whether this response-adapted approach was also cost effective.

The team applied economic modeling to data from the ADAPT-IT trial as well as CheckMate 067, in which patients received standard of care four doses of combination ipilimumab-nivolumab followed by nivolumab monotherapy. In the ADAPT-IT trial, patients also initially received the immunotherapy combination but had CT scans to determine their response after two doses; if they responded, patients discontinued ipilimumab and continued with nivolumab monotherapy.

Overall, ADAPT-IT showed that responders could forgo the additional two doses of ipilimumab plus nivolumab while maintaining similar progression-free survival and overall survival seen at 18 months in the CheckMate 067 trial.

The current economic analysis, based on 41 patients from ADAPT-IT and 314 from CheckMate 067, showed a potential reduction in health care costs of $19,891 per patient with the response-adapted approach.

Response-adapted treatment was the cost-effective option in 94% of simulated scenarios.

When extrapolated to 2019 incidence rates of distant melanoma cases, yearly national savings could reach about $58 million.

“In the relatively small space of immunotherapies in advanced melanoma, we hope this analysis motivates clinicians to consider response-adapted treatment,” Dr. Kunz told this news organization.

“On the larger scale, this analysis serves as a stepping stone to more response-guided treatment protocols,” Dr. Kunz added. “With drug costs rising and imaging capabilities growing, more frequent image-guided adjustments are a perfect fit into the personalized care model.”

When applying the cost savings noted in this analysis across all treated patients, “the economic impact may be profound,” said Joseph Skitzki, MD, surgical oncologist, Roswell Park Comprehensive Cancer Center, Buffalo, N.Y., who wasn’t involved in the study. The “financial toxicity of cancer care is increasingly recognized as a potential barrier to optimal outcomes and any measures to mitigate cost may be impactful.”

However, Dr. Skitzki said several caveats need to be considered.

One is that the data included from ADAPT-IT only included 41 patients, compared with 314 patients from CheckMate 067.

“It is possible that a larger real-world study utilizing the ADAPT-IT protocol may not be as favorable in terms of outcomes and could lessen the economic impact of de-escalation, although any form of de-escalation is likely to have a cost benefit,” Dr. Skitzki said in an interview.

A real-world response–adapted de-escalation clinical trial, with an emphasis on costs and a benchmark of similar progression-free and overall survival, should be conducted before the de-escalated option becomes “practice changing,” Dr. Skitzki said.

Jeffrey Weber, MD, PhD, deputy director, Perlmutter Cancer Center, NYU Langone Health, New York, also urged caution in interpreting the results.

“I would not base treatment decisions on a small sampling of 41 patients in the absence of a randomized comparison,” Dr. Weber told this news organization. “Without a proper comparison, I would not advocate using only two doses of ipilimumab-nivolumab to make decisions on treatment.”

Dr. Skitzki added that, while “studies like this one are desperately needed to lessen the economic impact of new and emerging combination immunotherapies,” there is likely also a “disincentive for pharmaceutical companies to conduct this type of research.”

This research had no specific funding. Dr. Kunz and Dr. Skitzki reported no relevant conflicts of interest. Dr. Weber disclosed relationships with Merck, Genentech, AstraZeneca, Pfizer, Regeneron, and GSK, among others, and holds equity in Cytomx, Biond, NexImmune, and Immunomax.

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

Adults with chronic gastroesophageal reflux disease (GERD) are at increased risk for Barrett’s esophagus (BE) – the precursor to esophageal adenocarcinoma (EAC) – but most don’t undergo recommended screening, new research shows.

Jennifer Kolb, MD, with the University of California, Los Angeles, and colleagues surveyed 472 adults with chronic GERD who qualify for BE screening and had a recent visit with their primary care provider.

In this diverse population of people at risk for BE and EAC, only 13% had ever been advised to undergo endoscopy to screen for BE and only 5% actually had a prior screening, the study notes.

“These results make it clear that screening is rarely done,” Dr. Kolb told this news organization.

The results of the survey are published online in the American Journal of Gastroenterology.
 

Concern high, understanding low

Esophageal cancer and BE have increased among middle-aged adults over roughly the past 5 years, and this increase is not because of better or more frequent screening, as reported previously by this news organization.

In fact, the majority of patients who develop EAC do not have a prior diagnosis of BE, which highlights the failure of current BE screening practices, Dr. Kolb and colleagues point out.

Professional gastroenterology society guidelines recommend screening for BE using upper endoscopy for at-risk individuals, which includes those with chronic GERD, along with other risk factors such as age older than 50 years, male sex, white race, smoking, obesity, and family history of BE or EAC.

In the current survey, most individuals said early detection of BE/EAC is important and leads to better outcomes, but most had poor overall knowledge of risk factors and indications for screening.

Only about two-thirds of respondents correctly identified BE risk factors, and only about 20% believed BE screening was necessary with GERD, the researchers note.

Roughly two-thirds of individuals wanted to prioritize BE screening and felt that getting an upper endoscopy would ease their concern.

Yet, 40% had no prior esophagogastroduodenoscopy. These individuals were less knowledgeable about BE/EAC risk and screening recommendations and identified more barriers to completing endoscopy.

“While minorities were most concerned about developing Barrett’s esophagus and cancer, they reported more barriers to screening compared to White participants,” Dr. Kolb said.
 

Addressing knowledge gaps

The primary care clinician is often the first line for patients with symptomatic acid reflux and the gateway for preventive cancer screening.

Yet, research has shown that primary care clinicians often have trouble identifying who should be screened for BE, and competing clinical issues make it challenging to implement BE screening.

“As gastroenterologists, we must partner with our primary care colleagues to help increase awareness of this lethal disease and improve recognition of risk factors so that eligible patients can be identified and referred for screening,” Dr. Kolb said.

Reached for comment, Seth Gross, MD, clinical chief of the division of gastroenterology and hepatology at New York University Langone Health, said the results “shed light on the fact that patients with GERD don’t have the knowledge of when they should get medical attention and possibly endoscopy.”

“We may need to do a better job of educating our colleagues and patients to know when to seek specialists to potentially get an endoscopy,” Dr. Gross said.

About 90% of esophageal cancers are diagnosed outside of surveillance programs, noted Prasad G. Iyer, MD, a gastroenterologist at Mayo Clinic in Rochester, Minn.

“Patients didn’t even know that they had Barrett’s [esophagus], so they were never under surveillance. They only come to attention after they have trouble with food sticking, and they can’t swallow solid food,” said Dr. Iyer, who wasn’t involved in the survey.

“Unfortunately, there are just so many cancers and so many issues that primary care providers have to deal with that I think this may not be getting the attention it deserves,” he said.

Access to endoscopy is also likely a barrier, Dr. Iyer noted.

“The waiting list may be several months, and I think providers may focus on other things,” he said.
 

Less-invasive screening options

Fear of endoscopy may be another issue.

In their survey, Dr. Kolb and colleagues found that 20% of respondents reported fear of discomfort with endoscopy as a barrier to completing screening.

But less-invasive screening options are increasingly available or in development.

This includes Cytosponge, a swallowable capsule containing a compressed sponge attached to a string. When withdrawn, the sponge contains esophageal cytology samples that can be used to identify biomarkers for BE.

In a guideline released last spring, the American College of Gastroenterology endorsed Cytosponge as a nonendoscopic BE screening modality, as published in the American Journal of Gastroenterology.

“The strength of the recommendation is conditional, but it’s the first time where [the ACG] is saying that this may be an option for people,” Dr. Gross said.

This research was funded by the American College of Gastroenterology. Dr. Kolb, Dr. Gross, and Dr. Iyer report no relevant financial relationships.

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

Adults with chronic gastroesophageal reflux disease (GERD) are at increased risk for Barrett’s esophagus (BE) – the precursor to esophageal adenocarcinoma (EAC) – but most don’t undergo recommended screening, new research shows.

Jennifer Kolb, MD, with the University of California, Los Angeles, and colleagues surveyed 472 adults with chronic GERD who qualify for BE screening and had a recent visit with their primary care provider.

In this diverse population of people at risk for BE and EAC, only 13% had ever been advised to undergo endoscopy to screen for BE and only 5% actually had a prior screening, the study notes.

“These results make it clear that screening is rarely done,” Dr. Kolb told this news organization.

The results of the survey are published online in the American Journal of Gastroenterology.
 

Concern high, understanding low

Esophageal cancer and BE have increased among middle-aged adults over roughly the past 5 years, and this increase is not because of better or more frequent screening, as reported previously by this news organization.

In fact, the majority of patients who develop EAC do not have a prior diagnosis of BE, which highlights the failure of current BE screening practices, Dr. Kolb and colleagues point out.

Professional gastroenterology society guidelines recommend screening for BE using upper endoscopy for at-risk individuals, which includes those with chronic GERD, along with other risk factors such as age older than 50 years, male sex, white race, smoking, obesity, and family history of BE or EAC.

In the current survey, most individuals said early detection of BE/EAC is important and leads to better outcomes, but most had poor overall knowledge of risk factors and indications for screening.

Only about two-thirds of respondents correctly identified BE risk factors, and only about 20% believed BE screening was necessary with GERD, the researchers note.

Roughly two-thirds of individuals wanted to prioritize BE screening and felt that getting an upper endoscopy would ease their concern.

Yet, 40% had no prior esophagogastroduodenoscopy. These individuals were less knowledgeable about BE/EAC risk and screening recommendations and identified more barriers to completing endoscopy.

“While minorities were most concerned about developing Barrett’s esophagus and cancer, they reported more barriers to screening compared to White participants,” Dr. Kolb said.
 

Addressing knowledge gaps

The primary care clinician is often the first line for patients with symptomatic acid reflux and the gateway for preventive cancer screening.

Yet, research has shown that primary care clinicians often have trouble identifying who should be screened for BE, and competing clinical issues make it challenging to implement BE screening.

“As gastroenterologists, we must partner with our primary care colleagues to help increase awareness of this lethal disease and improve recognition of risk factors so that eligible patients can be identified and referred for screening,” Dr. Kolb said.

Reached for comment, Seth Gross, MD, clinical chief of the division of gastroenterology and hepatology at New York University Langone Health, said the results “shed light on the fact that patients with GERD don’t have the knowledge of when they should get medical attention and possibly endoscopy.”

“We may need to do a better job of educating our colleagues and patients to know when to seek specialists to potentially get an endoscopy,” Dr. Gross said.

About 90% of esophageal cancers are diagnosed outside of surveillance programs, noted Prasad G. Iyer, MD, a gastroenterologist at Mayo Clinic in Rochester, Minn.

“Patients didn’t even know that they had Barrett’s [esophagus], so they were never under surveillance. They only come to attention after they have trouble with food sticking, and they can’t swallow solid food,” said Dr. Iyer, who wasn’t involved in the survey.

“Unfortunately, there are just so many cancers and so many issues that primary care providers have to deal with that I think this may not be getting the attention it deserves,” he said.

Access to endoscopy is also likely a barrier, Dr. Iyer noted.

“The waiting list may be several months, and I think providers may focus on other things,” he said.
 

Less-invasive screening options

Fear of endoscopy may be another issue.

In their survey, Dr. Kolb and colleagues found that 20% of respondents reported fear of discomfort with endoscopy as a barrier to completing screening.

But less-invasive screening options are increasingly available or in development.

This includes Cytosponge, a swallowable capsule containing a compressed sponge attached to a string. When withdrawn, the sponge contains esophageal cytology samples that can be used to identify biomarkers for BE.

In a guideline released last spring, the American College of Gastroenterology endorsed Cytosponge as a nonendoscopic BE screening modality, as published in the American Journal of Gastroenterology.

“The strength of the recommendation is conditional, but it’s the first time where [the ACG] is saying that this may be an option for people,” Dr. Gross said.

This research was funded by the American College of Gastroenterology. Dr. Kolb, Dr. Gross, and Dr. Iyer report no relevant financial relationships.

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

Adults with chronic gastroesophageal reflux disease (GERD) are at increased risk for Barrett’s esophagus (BE) – the precursor to esophageal adenocarcinoma (EAC) – but most don’t undergo recommended screening, new research shows.

Jennifer Kolb, MD, with the University of California, Los Angeles, and colleagues surveyed 472 adults with chronic GERD who qualify for BE screening and had a recent visit with their primary care provider.

In this diverse population of people at risk for BE and EAC, only 13% had ever been advised to undergo endoscopy to screen for BE and only 5% actually had a prior screening, the study notes.

“These results make it clear that screening is rarely done,” Dr. Kolb told this news organization.

The results of the survey are published online in the American Journal of Gastroenterology.
 

Concern high, understanding low

Esophageal cancer and BE have increased among middle-aged adults over roughly the past 5 years, and this increase is not because of better or more frequent screening, as reported previously by this news organization.

In fact, the majority of patients who develop EAC do not have a prior diagnosis of BE, which highlights the failure of current BE screening practices, Dr. Kolb and colleagues point out.

Professional gastroenterology society guidelines recommend screening for BE using upper endoscopy for at-risk individuals, which includes those with chronic GERD, along with other risk factors such as age older than 50 years, male sex, white race, smoking, obesity, and family history of BE or EAC.

In the current survey, most individuals said early detection of BE/EAC is important and leads to better outcomes, but most had poor overall knowledge of risk factors and indications for screening.

Only about two-thirds of respondents correctly identified BE risk factors, and only about 20% believed BE screening was necessary with GERD, the researchers note.

Roughly two-thirds of individuals wanted to prioritize BE screening and felt that getting an upper endoscopy would ease their concern.

Yet, 40% had no prior esophagogastroduodenoscopy. These individuals were less knowledgeable about BE/EAC risk and screening recommendations and identified more barriers to completing endoscopy.

“While minorities were most concerned about developing Barrett’s esophagus and cancer, they reported more barriers to screening compared to White participants,” Dr. Kolb said.
 

Addressing knowledge gaps

The primary care clinician is often the first line for patients with symptomatic acid reflux and the gateway for preventive cancer screening.

Yet, research has shown that primary care clinicians often have trouble identifying who should be screened for BE, and competing clinical issues make it challenging to implement BE screening.

“As gastroenterologists, we must partner with our primary care colleagues to help increase awareness of this lethal disease and improve recognition of risk factors so that eligible patients can be identified and referred for screening,” Dr. Kolb said.

Reached for comment, Seth Gross, MD, clinical chief of the division of gastroenterology and hepatology at New York University Langone Health, said the results “shed light on the fact that patients with GERD don’t have the knowledge of when they should get medical attention and possibly endoscopy.”

“We may need to do a better job of educating our colleagues and patients to know when to seek specialists to potentially get an endoscopy,” Dr. Gross said.

About 90% of esophageal cancers are diagnosed outside of surveillance programs, noted Prasad G. Iyer, MD, a gastroenterologist at Mayo Clinic in Rochester, Minn.

“Patients didn’t even know that they had Barrett’s [esophagus], so they were never under surveillance. They only come to attention after they have trouble with food sticking, and they can’t swallow solid food,” said Dr. Iyer, who wasn’t involved in the survey.

“Unfortunately, there are just so many cancers and so many issues that primary care providers have to deal with that I think this may not be getting the attention it deserves,” he said.

Access to endoscopy is also likely a barrier, Dr. Iyer noted.

“The waiting list may be several months, and I think providers may focus on other things,” he said.
 

Less-invasive screening options

Fear of endoscopy may be another issue.

In their survey, Dr. Kolb and colleagues found that 20% of respondents reported fear of discomfort with endoscopy as a barrier to completing screening.

But less-invasive screening options are increasingly available or in development.

This includes Cytosponge, a swallowable capsule containing a compressed sponge attached to a string. When withdrawn, the sponge contains esophageal cytology samples that can be used to identify biomarkers for BE.

In a guideline released last spring, the American College of Gastroenterology endorsed Cytosponge as a nonendoscopic BE screening modality, as published in the American Journal of Gastroenterology.

“The strength of the recommendation is conditional, but it’s the first time where [the ACG] is saying that this may be an option for people,” Dr. Gross said.

This research was funded by the American College of Gastroenterology. Dr. Kolb, Dr. Gross, and Dr. Iyer report no relevant financial relationships.

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

SAN DIEGO—As patients with cancer live longer, pain is going to become an even bigger challenge for clinicians, a palliative care specialist told cancer specialists in a presentation at the annual meeting of the Association of VA Hematology/Oncology (AVAHO) in September, and decisions about treatment are becoming more complicated amid the opioid epidemic.

Fortunately, guidelines and clinical experience offer helpful insight into the best practices, said hematologist/oncologist Andrea Ruskin, MD, medical director of palliative care at Veterans Administration (VA) Connecticut Healthcare System (VACHS).

As Ruskin pointed out, two-thirds of newly diagnosed cancer patients are living for at least 5 years, “but with this progress comes challenges.” More than one-third (37%) of cancer survivors report cancer pain, 21% have noncancer pain, and 45% have both. About 5% to 8% of VA cancer survivors use opioids for the long term, she said, although there have been few studies in this population.

Among patients with head and neck cancer, specifically, chronic pain affects 45%, and severe pain affects 11%. Subclinical PTSD, depression, anxiety, and low quality of life are common in this population. “We may cure them, but they have a lot of issues going forward.”

One key strategy is to perform a comprehensive pain assessment at the first visit, and then address pain at every subsequent visit. She recommended a physician resource from the American Society of Clinical Oncology, and a template may be useful to provide helpful questions, Ruskin said.

At VACHS certain questions are routine. “Is pain interfering with your function? Sometimes people say it’s always a 10, but it’s not affecting function at all. Ask if the medicine is working. And how are they taking it? Sometimes they say, ‘I’m taking that for sleep,’ and we say ‘No, Mr. Smith, that is not a sleep medication.’”

Be aware that some patients may use nonmedical opioids, she said. And set expectations early on. “Safe opioid use starts with the very first prescription,” she said. “If I have somebody with myeloma or head and neck cancer, I make it very clear that my goal is that we want you off the opioids after the radiation or once the disease is in remission. I really make an effort at the very beginning to make sure that we're all on the same page.”

As you continue to see a patient, consider ordering urine tests, she said, not as a punitive measure but to make sure you’re offering the safest and most effective treatment. “We don’t do it to say ‘no, no, no.’ We do it for safety and to make sure they’re not getting meds elsewhere.”

What are the best practices when pain doesn’t go away? Should they stay on opioids? According to Ruskin, few evidence-based guidelines address the “more nuanced care” that patients need when their pain lasts for months or years.

But there are useful resources. Ruskin highlighted the National Comprehensive Cancer Network’s survivorship guidelines, and she summarized a few of the available painkiller options. “Opioids are great, and adjuvants are so-so. They work in some people, but we definitely have room for improvement.”

What if patients have persistent opioid use after cancer recovery? “I try to taper if I can, and I try to explain why I’m tapering. It could take months or years to taper patients,” she said. And consider transitioning the patient to buprenorphine, a drug that treats both pain and opioid use disorder, if appropriate. “You don’t need a waiver if you use it for pain. It’s definitely something we’re using more of.”

One important step is to bring in colleagues to help. Psychologists, chiropractors, physical therapists, physiatrists, and pain pharmacists can all be helpful, she said. “Learn about your VA resources and who can partner with you to help these complicated patients. They’re all at your fingertips.”

SAN DIEGO—As patients with cancer live longer, pain is going to become an even bigger challenge for clinicians, a palliative care specialist told cancer specialists in a presentation at the annual meeting of the Association of VA Hematology/Oncology (AVAHO) in September, and decisions about treatment are becoming more complicated amid the opioid epidemic.

Fortunately, guidelines and clinical experience offer helpful insight into the best practices, said hematologist/oncologist Andrea Ruskin, MD, medical director of palliative care at Veterans Administration (VA) Connecticut Healthcare System (VACHS).

As Ruskin pointed out, two-thirds of newly diagnosed cancer patients are living for at least 5 years, “but with this progress comes challenges.” More than one-third (37%) of cancer survivors report cancer pain, 21% have noncancer pain, and 45% have both. About 5% to 8% of VA cancer survivors use opioids for the long term, she said, although there have been few studies in this population.

Among patients with head and neck cancer, specifically, chronic pain affects 45%, and severe pain affects 11%. Subclinical PTSD, depression, anxiety, and low quality of life are common in this population. “We may cure them, but they have a lot of issues going forward.”

One key strategy is to perform a comprehensive pain assessment at the first visit, and then address pain at every subsequent visit. She recommended a physician resource from the American Society of Clinical Oncology, and a template may be useful to provide helpful questions, Ruskin said.

At VACHS certain questions are routine. “Is pain interfering with your function? Sometimes people say it’s always a 10, but it’s not affecting function at all. Ask if the medicine is working. And how are they taking it? Sometimes they say, ‘I’m taking that for sleep,’ and we say ‘No, Mr. Smith, that is not a sleep medication.’”

Be aware that some patients may use nonmedical opioids, she said. And set expectations early on. “Safe opioid use starts with the very first prescription,” she said. “If I have somebody with myeloma or head and neck cancer, I make it very clear that my goal is that we want you off the opioids after the radiation or once the disease is in remission. I really make an effort at the very beginning to make sure that we're all on the same page.”

As you continue to see a patient, consider ordering urine tests, she said, not as a punitive measure but to make sure you’re offering the safest and most effective treatment. “We don’t do it to say ‘no, no, no.’ We do it for safety and to make sure they’re not getting meds elsewhere.”

What are the best practices when pain doesn’t go away? Should they stay on opioids? According to Ruskin, few evidence-based guidelines address the “more nuanced care” that patients need when their pain lasts for months or years.

But there are useful resources. Ruskin highlighted the National Comprehensive Cancer Network’s survivorship guidelines, and she summarized a few of the available painkiller options. “Opioids are great, and adjuvants are so-so. They work in some people, but we definitely have room for improvement.”

What if patients have persistent opioid use after cancer recovery? “I try to taper if I can, and I try to explain why I’m tapering. It could take months or years to taper patients,” she said. And consider transitioning the patient to buprenorphine, a drug that treats both pain and opioid use disorder, if appropriate. “You don’t need a waiver if you use it for pain. It’s definitely something we’re using more of.”

One important step is to bring in colleagues to help. Psychologists, chiropractors, physical therapists, physiatrists, and pain pharmacists can all be helpful, she said. “Learn about your VA resources and who can partner with you to help these complicated patients. They’re all at your fingertips.”

SAN DIEGO—As patients with cancer live longer, pain is going to become an even bigger challenge for clinicians, a palliative care specialist told cancer specialists in a presentation at the annual meeting of the Association of VA Hematology/Oncology (AVAHO) in September, and decisions about treatment are becoming more complicated amid the opioid epidemic.

Fortunately, guidelines and clinical experience offer helpful insight into the best practices, said hematologist/oncologist Andrea Ruskin, MD, medical director of palliative care at Veterans Administration (VA) Connecticut Healthcare System (VACHS).

As Ruskin pointed out, two-thirds of newly diagnosed cancer patients are living for at least 5 years, “but with this progress comes challenges.” More than one-third (37%) of cancer survivors report cancer pain, 21% have noncancer pain, and 45% have both. About 5% to 8% of VA cancer survivors use opioids for the long term, she said, although there have been few studies in this population.

Among patients with head and neck cancer, specifically, chronic pain affects 45%, and severe pain affects 11%. Subclinical PTSD, depression, anxiety, and low quality of life are common in this population. “We may cure them, but they have a lot of issues going forward.”

One key strategy is to perform a comprehensive pain assessment at the first visit, and then address pain at every subsequent visit. She recommended a physician resource from the American Society of Clinical Oncology, and a template may be useful to provide helpful questions, Ruskin said.

At VACHS certain questions are routine. “Is pain interfering with your function? Sometimes people say it’s always a 10, but it’s not affecting function at all. Ask if the medicine is working. And how are they taking it? Sometimes they say, ‘I’m taking that for sleep,’ and we say ‘No, Mr. Smith, that is not a sleep medication.’”

Be aware that some patients may use nonmedical opioids, she said. And set expectations early on. “Safe opioid use starts with the very first prescription,” she said. “If I have somebody with myeloma or head and neck cancer, I make it very clear that my goal is that we want you off the opioids after the radiation or once the disease is in remission. I really make an effort at the very beginning to make sure that we're all on the same page.”

As you continue to see a patient, consider ordering urine tests, she said, not as a punitive measure but to make sure you’re offering the safest and most effective treatment. “We don’t do it to say ‘no, no, no.’ We do it for safety and to make sure they’re not getting meds elsewhere.”

What are the best practices when pain doesn’t go away? Should they stay on opioids? According to Ruskin, few evidence-based guidelines address the “more nuanced care” that patients need when their pain lasts for months or years.

But there are useful resources. Ruskin highlighted the National Comprehensive Cancer Network’s survivorship guidelines, and she summarized a few of the available painkiller options. “Opioids are great, and adjuvants are so-so. They work in some people, but we definitely have room for improvement.”

What if patients have persistent opioid use after cancer recovery? “I try to taper if I can, and I try to explain why I’m tapering. It could take months or years to taper patients,” she said. And consider transitioning the patient to buprenorphine, a drug that treats both pain and opioid use disorder, if appropriate. “You don’t need a waiver if you use it for pain. It’s definitely something we’re using more of.”

One important step is to bring in colleagues to help. Psychologists, chiropractors, physical therapists, physiatrists, and pain pharmacists can all be helpful, she said. “Learn about your VA resources and who can partner with you to help these complicated patients. They’re all at your fingertips.”

Among high-risk early breast cancer patients, delivery of a radiation boost to the tumor bed during whole breast irradiation was just as safe and effective as delivering the boost sequentially after whole breast irradiation ended. The findings from the phase 3 clinical trial are a boon to patient convenience.

“These findings are indeed practice changing. This was a well-designed trial that looked at shortening treatment from six to three weeks. They showed equivalent local control and importantly, a good cosmetic outcome over time,” said Kathleen Horst, MD, who served as a discussant during a presentation given by Frank Vicini, MD, FASTRO, GenesisCare, during the annual meeting of the American Society for Radiation Oncology.

“This is substantially more convenient. It’s cost effective both for the health care system and individual patients. Importantly, our patients come in for treatment every day and they’re taking time from work which means they have to arrange for childcare and transportation. So, this makes a big difference for these patients,” said Dr. Horst, who is a professor of radiation oncology at Stanford (Calif.) Medicine and director of well-being in the radiation department at Stanford Medicine.

“One of the things that was surprising is that I think all of us were thinking this might be a more toxic regimen, but as Dr. Vicini showed, it was equally effective over time with minimal toxicity and cosmesis was stable over time, which is important. Importantly, it included patient-reported outcomes, not just the physician-reported outcomes. Broadly, I think these findings are applicable for many patients, including all patients who are receiving whole breast radiotherapy with an added boost. I think over time this is going to improve the quality of life of our patients. It represents an innovative change that everyone is going to be excited to embrace,” Dr. Horst said.

Previous randomized controlled trials showed that an additional radiation dose to the tumor bed following lumpectomy and whole breast irradiation reduces the relative risk of local recurrence by about 35%. However, this increases treatment time for patients who have already endured an extensive regimen. For whole breast irradiation, hypofractionated radiation in 15-16 fractions over 3 weeks has comparable recurrence rates as a 5-week regimen, but the relevant trials did not examine the effect hypofractionation may have on a radiation boost to the tumor bed of high-risk patients. Because of this lack of evidence, current practice calls for the boost to remain sequential in five to eight fractions after completion of whole breast irradiation, which adds 1 week to a 1.5 week–long treatment.

The study included 2,262 patients who were randomized to receive a sequential boost or a concomitant boost. After a median follow-up of 7.4 years, there were 54 ipsilateral breast recurrence (IBR) events. The estimated 7-year risk of IBR was 2.2% in the sequential boost and 2.6% in the concurrent risk group (hazard ratio, 1.32; noninferiority test P = .039). Approximately 60% of patients received adjuvant chemotherapy.

Grade 3 or higher adverse events were similar with a frequency of 3.3% in the sequential group and 3.5% in the concurrent group (P = .79). The researchers used the Global Cosmetic Score (GCS) to assess outcomes from the perspective of both physicians and patients. 86% of physicians rated the outcome as excellent/good in the sequential group versus 82% in the concurrent group (P = .33).

“For high-risk early-stage breast cancer patients undergoing breast conservation, a concurrent boost with hypofractionated whole breast irradiation – compared to a sequential boost – results in noninferior local recurrence rates with no significant difference in toxicity, noninferior patient rated cosmesis, and no significant difference in physician rated cosmesis. The entire treatment was delivered in three weeks, even for high-risk patients. Just as critical, the use of target volume based radiation planning for [three-dimensional conformal or IMRT whole breast irradiation assessed by dose volume analysis is feasible, and resulted in very low toxicity in the treatment arms, regardless of the fractionation schedule, or the boost delivery,” Dr. Vincini said.

No conflicts of interest were disclosed for Dr. Horst or Dr. Vicini.

Among high-risk early breast cancer patients, delivery of a radiation boost to the tumor bed during whole breast irradiation was just as safe and effective as delivering the boost sequentially after whole breast irradiation ended. The findings from the phase 3 clinical trial are a boon to patient convenience.

“These findings are indeed practice changing. This was a well-designed trial that looked at shortening treatment from six to three weeks. They showed equivalent local control and importantly, a good cosmetic outcome over time,” said Kathleen Horst, MD, who served as a discussant during a presentation given by Frank Vicini, MD, FASTRO, GenesisCare, during the annual meeting of the American Society for Radiation Oncology.

“This is substantially more convenient. It’s cost effective both for the health care system and individual patients. Importantly, our patients come in for treatment every day and they’re taking time from work which means they have to arrange for childcare and transportation. So, this makes a big difference for these patients,” said Dr. Horst, who is a professor of radiation oncology at Stanford (Calif.) Medicine and director of well-being in the radiation department at Stanford Medicine.

“One of the things that was surprising is that I think all of us were thinking this might be a more toxic regimen, but as Dr. Vicini showed, it was equally effective over time with minimal toxicity and cosmesis was stable over time, which is important. Importantly, it included patient-reported outcomes, not just the physician-reported outcomes. Broadly, I think these findings are applicable for many patients, including all patients who are receiving whole breast radiotherapy with an added boost. I think over time this is going to improve the quality of life of our patients. It represents an innovative change that everyone is going to be excited to embrace,” Dr. Horst said.

Previous randomized controlled trials showed that an additional radiation dose to the tumor bed following lumpectomy and whole breast irradiation reduces the relative risk of local recurrence by about 35%. However, this increases treatment time for patients who have already endured an extensive regimen. For whole breast irradiation, hypofractionated radiation in 15-16 fractions over 3 weeks has comparable recurrence rates as a 5-week regimen, but the relevant trials did not examine the effect hypofractionation may have on a radiation boost to the tumor bed of high-risk patients. Because of this lack of evidence, current practice calls for the boost to remain sequential in five to eight fractions after completion of whole breast irradiation, which adds 1 week to a 1.5 week–long treatment.

The study included 2,262 patients who were randomized to receive a sequential boost or a concomitant boost. After a median follow-up of 7.4 years, there were 54 ipsilateral breast recurrence (IBR) events. The estimated 7-year risk of IBR was 2.2% in the sequential boost and 2.6% in the concurrent risk group (hazard ratio, 1.32; noninferiority test P = .039). Approximately 60% of patients received adjuvant chemotherapy.

Grade 3 or higher adverse events were similar with a frequency of 3.3% in the sequential group and 3.5% in the concurrent group (P = .79). The researchers used the Global Cosmetic Score (GCS) to assess outcomes from the perspective of both physicians and patients. 86% of physicians rated the outcome as excellent/good in the sequential group versus 82% in the concurrent group (P = .33).

“For high-risk early-stage breast cancer patients undergoing breast conservation, a concurrent boost with hypofractionated whole breast irradiation – compared to a sequential boost – results in noninferior local recurrence rates with no significant difference in toxicity, noninferior patient rated cosmesis, and no significant difference in physician rated cosmesis. The entire treatment was delivered in three weeks, even for high-risk patients. Just as critical, the use of target volume based radiation planning for [three-dimensional conformal or IMRT whole breast irradiation assessed by dose volume analysis is feasible, and resulted in very low toxicity in the treatment arms, regardless of the fractionation schedule, or the boost delivery,” Dr. Vincini said.

No conflicts of interest were disclosed for Dr. Horst or Dr. Vicini.

Among high-risk early breast cancer patients, delivery of a radiation boost to the tumor bed during whole breast irradiation was just as safe and effective as delivering the boost sequentially after whole breast irradiation ended. The findings from the phase 3 clinical trial are a boon to patient convenience.

“These findings are indeed practice changing. This was a well-designed trial that looked at shortening treatment from six to three weeks. They showed equivalent local control and importantly, a good cosmetic outcome over time,” said Kathleen Horst, MD, who served as a discussant during a presentation given by Frank Vicini, MD, FASTRO, GenesisCare, during the annual meeting of the American Society for Radiation Oncology.

“This is substantially more convenient. It’s cost effective both for the health care system and individual patients. Importantly, our patients come in for treatment every day and they’re taking time from work which means they have to arrange for childcare and transportation. So, this makes a big difference for these patients,” said Dr. Horst, who is a professor of radiation oncology at Stanford (Calif.) Medicine and director of well-being in the radiation department at Stanford Medicine.

“One of the things that was surprising is that I think all of us were thinking this might be a more toxic regimen, but as Dr. Vicini showed, it was equally effective over time with minimal toxicity and cosmesis was stable over time, which is important. Importantly, it included patient-reported outcomes, not just the physician-reported outcomes. Broadly, I think these findings are applicable for many patients, including all patients who are receiving whole breast radiotherapy with an added boost. I think over time this is going to improve the quality of life of our patients. It represents an innovative change that everyone is going to be excited to embrace,” Dr. Horst said.

Previous randomized controlled trials showed that an additional radiation dose to the tumor bed following lumpectomy and whole breast irradiation reduces the relative risk of local recurrence by about 35%. However, this increases treatment time for patients who have already endured an extensive regimen. For whole breast irradiation, hypofractionated radiation in 15-16 fractions over 3 weeks has comparable recurrence rates as a 5-week regimen, but the relevant trials did not examine the effect hypofractionation may have on a radiation boost to the tumor bed of high-risk patients. Because of this lack of evidence, current practice calls for the boost to remain sequential in five to eight fractions after completion of whole breast irradiation, which adds 1 week to a 1.5 week–long treatment.

The study included 2,262 patients who were randomized to receive a sequential boost or a concomitant boost. After a median follow-up of 7.4 years, there were 54 ipsilateral breast recurrence (IBR) events. The estimated 7-year risk of IBR was 2.2% in the sequential boost and 2.6% in the concurrent risk group (hazard ratio, 1.32; noninferiority test P = .039). Approximately 60% of patients received adjuvant chemotherapy.

Grade 3 or higher adverse events were similar with a frequency of 3.3% in the sequential group and 3.5% in the concurrent group (P = .79). The researchers used the Global Cosmetic Score (GCS) to assess outcomes from the perspective of both physicians and patients. 86% of physicians rated the outcome as excellent/good in the sequential group versus 82% in the concurrent group (P = .33).

“For high-risk early-stage breast cancer patients undergoing breast conservation, a concurrent boost with hypofractionated whole breast irradiation – compared to a sequential boost – results in noninferior local recurrence rates with no significant difference in toxicity, noninferior patient rated cosmesis, and no significant difference in physician rated cosmesis. The entire treatment was delivered in three weeks, even for high-risk patients. Just as critical, the use of target volume based radiation planning for [three-dimensional conformal or IMRT whole breast irradiation assessed by dose volume analysis is feasible, and resulted in very low toxicity in the treatment arms, regardless of the fractionation schedule, or the boost delivery,” Dr. Vincini said.

No conflicts of interest were disclosed for Dr. Horst or Dr. Vicini.

Among patients with advanced hepatocellular carcinoma (HCC), and especially those with macrovascular invasion, stereotactic body radiation therapy (SBRT) appears to grant a survival benefit when added to systemic therapy. That was the finding from a phase 3 clinical trial presented at the annual meeting of the American Society for Radiation Oncology.

For unresectable HCC or cases that cannot be treated with thermal ablation or regional therapy, the current standard of care is systemic therapy. When the study was conducted, the recommended therapy was the sorafenib, a tyrosine kinase inhibitor. But with the publication of the IMbrave 150 study in 2021, atezolizumab plus bevacizumab is now increasingly preferred by some oncologists.

In 2008, the SHARP study found that sorafenib improved median survival, but it provided less benefit for patients with macrovascular invasion. Various studies have addressed the question of whether radiation could improve survival among this patient population, but results have not been encouraging. Direct comparisons between sorafenib and radiotherapy in the SARAH and SIRveNIB studies showed no significant differences in outcomes.

To determine the efficacy of combined SBRT and sorafenib, researchers randomized 177 patients with locally advanced HCC to receive 400 mg sorafenib every 12 hours or SBRT of 27.5-50 Gy in five fractions, followed by 200 mg sorafenib every 12 hours for 4 weeks, then 400 mg sorafenib every 12 hours thereafter. The median age was 66 years, 85% of patients were male, 74% had macrovascular invasion. The study included patients with locally advanced tumors up to a 20-cm sum of diameters or up to a 20-cm conglomerate tumor, as well as those with metastases of 3 cm size or smaller.

After a median follow-up of 13.2 months, median overall survival was 15.8 months in the combination group, versus 12.3 months in sorafenib group (hazard ratio, 0.77; 1-sided P = .055). After a multivariable analysis, the combined treatment was associated with better overall survival (HR, 0.72; P = .042).

“This overall survival is greater than expected and impressive even in the era now of immunotherapy trials,” said Laura Dawson, MD, who presented the results of the study during a press conference at the meeting. Dr. Dawson is a professor of radiation oncology at University of Toronto and a radiation oncologist at Princess Margaret Hospital in Toronto.

Median progression-free survival was 9.2 months in the combined group versus 5.5 months in the sorafenib-only group (HR, 0.55; P = .0001). At 24 months, 17% of the combination group had 7% of the sorafenib group remained had not progressed. The median time to progression was 18.5 months in the combination group and 9.5 months in the sorafenib group (HR, 0.69; P = .034). The frequency of adverse events was similar in both groups. The study admitted patients with any level of vascular invasion, which contrasted with many earlier trials that excluded those with involvement of the main portal vein.

“I think this is really one of the most important studies that’s come out in many years in terms of practice changing outcomes. We’ve seen that with patients who have very high-risk HCC, especially patients who have portal vein or macrovascular vascular invasion, there’s been a significant improvement in overall survival for these patients, and this is a very difficult patient population. Adding SBRT in this group improved both the progression free survival and overall survival, so I think we’re really at a point where we can call this a standard of care for patients,” Karyn A. Goodman, MD, professor and vice chair of clinical research and radiation oncology at the Icahn School of Medicine at Mount Sinai, New York, said at the press conference.

A limitation of the study is that it closed early to accrual because of a change in the standard of care.

Dr. Goodman has served on advisory boards for Novartis, Philips Healthcare, and Genentech, and has consulted for RenovoRx and Syntactx. Dr. Dawson has received research grants from Merck and received patent/license fees or copyright compensation from RaySearch.

Among patients with advanced hepatocellular carcinoma (HCC), and especially those with macrovascular invasion, stereotactic body radiation therapy (SBRT) appears to grant a survival benefit when added to systemic therapy. That was the finding from a phase 3 clinical trial presented at the annual meeting of the American Society for Radiation Oncology.

For unresectable HCC or cases that cannot be treated with thermal ablation or regional therapy, the current standard of care is systemic therapy. When the study was conducted, the recommended therapy was the sorafenib, a tyrosine kinase inhibitor. But with the publication of the IMbrave 150 study in 2021, atezolizumab plus bevacizumab is now increasingly preferred by some oncologists.

In 2008, the SHARP study found that sorafenib improved median survival, but it provided less benefit for patients with macrovascular invasion. Various studies have addressed the question of whether radiation could improve survival among this patient population, but results have not been encouraging. Direct comparisons between sorafenib and radiotherapy in the SARAH and SIRveNIB studies showed no significant differences in outcomes.

To determine the efficacy of combined SBRT and sorafenib, researchers randomized 177 patients with locally advanced HCC to receive 400 mg sorafenib every 12 hours or SBRT of 27.5-50 Gy in five fractions, followed by 200 mg sorafenib every 12 hours for 4 weeks, then 400 mg sorafenib every 12 hours thereafter. The median age was 66 years, 85% of patients were male, 74% had macrovascular invasion. The study included patients with locally advanced tumors up to a 20-cm sum of diameters or up to a 20-cm conglomerate tumor, as well as those with metastases of 3 cm size or smaller.

After a median follow-up of 13.2 months, median overall survival was 15.8 months in the combination group, versus 12.3 months in sorafenib group (hazard ratio, 0.77; 1-sided P = .055). After a multivariable analysis, the combined treatment was associated with better overall survival (HR, 0.72; P = .042).

“This overall survival is greater than expected and impressive even in the era now of immunotherapy trials,” said Laura Dawson, MD, who presented the results of the study during a press conference at the meeting. Dr. Dawson is a professor of radiation oncology at University of Toronto and a radiation oncologist at Princess Margaret Hospital in Toronto.

Median progression-free survival was 9.2 months in the combined group versus 5.5 months in the sorafenib-only group (HR, 0.55; P = .0001). At 24 months, 17% of the combination group had 7% of the sorafenib group remained had not progressed. The median time to progression was 18.5 months in the combination group and 9.5 months in the sorafenib group (HR, 0.69; P = .034). The frequency of adverse events was similar in both groups. The study admitted patients with any level of vascular invasion, which contrasted with many earlier trials that excluded those with involvement of the main portal vein.

“I think this is really one of the most important studies that’s come out in many years in terms of practice changing outcomes. We’ve seen that with patients who have very high-risk HCC, especially patients who have portal vein or macrovascular vascular invasion, there’s been a significant improvement in overall survival for these patients, and this is a very difficult patient population. Adding SBRT in this group improved both the progression free survival and overall survival, so I think we’re really at a point where we can call this a standard of care for patients,” Karyn A. Goodman, MD, professor and vice chair of clinical research and radiation oncology at the Icahn School of Medicine at Mount Sinai, New York, said at the press conference.

A limitation of the study is that it closed early to accrual because of a change in the standard of care.

Dr. Goodman has served on advisory boards for Novartis, Philips Healthcare, and Genentech, and has consulted for RenovoRx and Syntactx. Dr. Dawson has received research grants from Merck and received patent/license fees or copyright compensation from RaySearch.

Among patients with advanced hepatocellular carcinoma (HCC), and especially those with macrovascular invasion, stereotactic body radiation therapy (SBRT) appears to grant a survival benefit when added to systemic therapy. That was the finding from a phase 3 clinical trial presented at the annual meeting of the American Society for Radiation Oncology.

For unresectable HCC or cases that cannot be treated with thermal ablation or regional therapy, the current standard of care is systemic therapy. When the study was conducted, the recommended therapy was the sorafenib, a tyrosine kinase inhibitor. But with the publication of the IMbrave 150 study in 2021, atezolizumab plus bevacizumab is now increasingly preferred by some oncologists.

In 2008, the SHARP study found that sorafenib improved median survival, but it provided less benefit for patients with macrovascular invasion. Various studies have addressed the question of whether radiation could improve survival among this patient population, but results have not been encouraging. Direct comparisons between sorafenib and radiotherapy in the SARAH and SIRveNIB studies showed no significant differences in outcomes.

To determine the efficacy of combined SBRT and sorafenib, researchers randomized 177 patients with locally advanced HCC to receive 400 mg sorafenib every 12 hours or SBRT of 27.5-50 Gy in five fractions, followed by 200 mg sorafenib every 12 hours for 4 weeks, then 400 mg sorafenib every 12 hours thereafter. The median age was 66 years, 85% of patients were male, 74% had macrovascular invasion. The study included patients with locally advanced tumors up to a 20-cm sum of diameters or up to a 20-cm conglomerate tumor, as well as those with metastases of 3 cm size or smaller.

After a median follow-up of 13.2 months, median overall survival was 15.8 months in the combination group, versus 12.3 months in sorafenib group (hazard ratio, 0.77; 1-sided P = .055). After a multivariable analysis, the combined treatment was associated with better overall survival (HR, 0.72; P = .042).

“This overall survival is greater than expected and impressive even in the era now of immunotherapy trials,” said Laura Dawson, MD, who presented the results of the study during a press conference at the meeting. Dr. Dawson is a professor of radiation oncology at University of Toronto and a radiation oncologist at Princess Margaret Hospital in Toronto.

Median progression-free survival was 9.2 months in the combined group versus 5.5 months in the sorafenib-only group (HR, 0.55; P = .0001). At 24 months, 17% of the combination group had 7% of the sorafenib group remained had not progressed. The median time to progression was 18.5 months in the combination group and 9.5 months in the sorafenib group (HR, 0.69; P = .034). The frequency of adverse events was similar in both groups. The study admitted patients with any level of vascular invasion, which contrasted with many earlier trials that excluded those with involvement of the main portal vein.

“I think this is really one of the most important studies that’s come out in many years in terms of practice changing outcomes. We’ve seen that with patients who have very high-risk HCC, especially patients who have portal vein or macrovascular vascular invasion, there’s been a significant improvement in overall survival for these patients, and this is a very difficult patient population. Adding SBRT in this group improved both the progression free survival and overall survival, so I think we’re really at a point where we can call this a standard of care for patients,” Karyn A. Goodman, MD, professor and vice chair of clinical research and radiation oncology at the Icahn School of Medicine at Mount Sinai, New York, said at the press conference.

A limitation of the study is that it closed early to accrual because of a change in the standard of care.

Dr. Goodman has served on advisory boards for Novartis, Philips Healthcare, and Genentech, and has consulted for RenovoRx and Syntactx. Dr. Dawson has received research grants from Merck and received patent/license fees or copyright compensation from RaySearch.

CHARLOTTE, N.C.– Performing high-quality index colonoscopies may pay off later in your patients’ reduced risk for advanced neoplasia, investigators report.

A study of registry data on more than 2,200 patients who had an index colonoscopy showing no evidence of neoplasia found that, on repeat colonoscopy 10 years later, the absolute risk for advanced neoplasia outcomes was lower for those with a high-quality index exam, compared with those who had a lesser-quality index colonoscopy.

Neil Osterweil/MDedge News

The adjusted odds ratio for patients who underwent high-quality index exams was 0.59%, reported Joseph Anderson, MD, from the Geisel School of Medicine at Dartmouth, Hanover, N.H.

“These data demonstrate that high-quality index colonoscopy provides better protection from interval lesions than low-quality exams with no polyps detected at that index exam,” he said in an oral abstract presentation at the annual meeting of the American College of Gastroenterology.

“These data support the importance of high-quality index exams in the prevention of interval colorectal cancer, and support the 10-year interval for normal exams,” Dr. Anderson added.

He recommended that endoscopists focus on the quality of their exams by using adequate scope withdrawal time – 8-10 minutes – to ensure optimal adenoma detection, and by ensuring the use of optimal bowel preparation in their practices.

Registry study

Dr. Anderson and colleagues studied how the quality of index colonoscopies could affect the risk of advanced outcomes in low-risk patients at the 10-year or later follow-up. They used records from the New Hampshire Colonoscopy Registry, which includes data from 2004 to the present on more than 250,000 exams performed by more than 150 endoscopists in more than 30 Granite State practices.

The investigators also looked at data on patients with less than 5 years of follow-up, and those with follow-up from 5 to less than 10 years.

The study sample included patients with no adenoma or significant serrated polyps on their index exams who had at least one follow-up exam 12 months or more after the index exams. Patients with inflammatory bowel disease or familial colon cancer syndromes were excluded.

They defined a high-quality colonoscopy as an exam complete to cecum, with adequate bowel preparation, and performed by an endoscopist with an adenoma detection rate of 25 or higher.

The adenoma detection rate is calculated as the number of screening colonoscopies with at least one adenoma divided by the total number of screening colonoscopies.

The definition of advanced outcomes included advanced adenomas, colorectal cancer, and/or large serrated polyps (1 cm or greater).

Of the 14,011 patients in the sample, 2,283 had a follow-up exam at 10 years. The absolute risk for advanced outcomes among patients who had a high quality index exam was 4.0% vs. 6.7% for those with lower quality exams.

Among patients with low-quality index exams – but not patients with high quality exams – there was a statistically significant increase in the absolute risk for advanced outcomes at all time periods, from 5.1% in the less than 5-year follow-up group, to 6.7% in the 10-years or more follow-up group.

Patients with initial high-quality exams also had a lower risk for postcolonoscopy colorectal cancer, compared with patients who had low-quality index exams: 0.4% vs. 0.8%. This difference translated into an adjusted hazard ratio for colorectal cancer after a high-quality exam of 0.53.
 

It’s getting better all the time

In an interview, Daniel J. Pambianco, MD, FACG from Charlottesville (Va.) Gastroenterology Associates, who was not involved in the study, commented that Dr. Anderson and colleagues highlighted the importance of the quality of the bowel prep and the quality of the examination itself.

He noted that the use of devices such as colonoscopy caps can help further improve adenoma detection rates and pointed to up-and-coming developments such as the use of artificial intelligence algorithms to aid human endoscopists.

Dr. Pambianco comoderated the session where the data were presented.

The investigators did not report a study funding source. Dr. Anderson and Dr. Pambianco reported having no relevant financial disclosures.

CHARLOTTE, N.C.– Performing high-quality index colonoscopies may pay off later in your patients’ reduced risk for advanced neoplasia, investigators report.

A study of registry data on more than 2,200 patients who had an index colonoscopy showing no evidence of neoplasia found that, on repeat colonoscopy 10 years later, the absolute risk for advanced neoplasia outcomes was lower for those with a high-quality index exam, compared with those who had a lesser-quality index colonoscopy.

Neil Osterweil/MDedge News

The adjusted odds ratio for patients who underwent high-quality index exams was 0.59%, reported Joseph Anderson, MD, from the Geisel School of Medicine at Dartmouth, Hanover, N.H.

“These data demonstrate that high-quality index colonoscopy provides better protection from interval lesions than low-quality exams with no polyps detected at that index exam,” he said in an oral abstract presentation at the annual meeting of the American College of Gastroenterology.

“These data support the importance of high-quality index exams in the prevention of interval colorectal cancer, and support the 10-year interval for normal exams,” Dr. Anderson added.

He recommended that endoscopists focus on the quality of their exams by using adequate scope withdrawal time – 8-10 minutes – to ensure optimal adenoma detection, and by ensuring the use of optimal bowel preparation in their practices.

Registry study

Dr. Anderson and colleagues studied how the quality of index colonoscopies could affect the risk of advanced outcomes in low-risk patients at the 10-year or later follow-up. They used records from the New Hampshire Colonoscopy Registry, which includes data from 2004 to the present on more than 250,000 exams performed by more than 150 endoscopists in more than 30 Granite State practices.

The investigators also looked at data on patients with less than 5 years of follow-up, and those with follow-up from 5 to less than 10 years.

The study sample included patients with no adenoma or significant serrated polyps on their index exams who had at least one follow-up exam 12 months or more after the index exams. Patients with inflammatory bowel disease or familial colon cancer syndromes were excluded.

They defined a high-quality colonoscopy as an exam complete to cecum, with adequate bowel preparation, and performed by an endoscopist with an adenoma detection rate of 25 or higher.

The adenoma detection rate is calculated as the number of screening colonoscopies with at least one adenoma divided by the total number of screening colonoscopies.

The definition of advanced outcomes included advanced adenomas, colorectal cancer, and/or large serrated polyps (1 cm or greater).

Of the 14,011 patients in the sample, 2,283 had a follow-up exam at 10 years. The absolute risk for advanced outcomes among patients who had a high quality index exam was 4.0% vs. 6.7% for those with lower quality exams.

Among patients with low-quality index exams – but not patients with high quality exams – there was a statistically significant increase in the absolute risk for advanced outcomes at all time periods, from 5.1% in the less than 5-year follow-up group, to 6.7% in the 10-years or more follow-up group.

Patients with initial high-quality exams also had a lower risk for postcolonoscopy colorectal cancer, compared with patients who had low-quality index exams: 0.4% vs. 0.8%. This difference translated into an adjusted hazard ratio for colorectal cancer after a high-quality exam of 0.53.
 

It’s getting better all the time

In an interview, Daniel J. Pambianco, MD, FACG from Charlottesville (Va.) Gastroenterology Associates, who was not involved in the study, commented that Dr. Anderson and colleagues highlighted the importance of the quality of the bowel prep and the quality of the examination itself.

He noted that the use of devices such as colonoscopy caps can help further improve adenoma detection rates and pointed to up-and-coming developments such as the use of artificial intelligence algorithms to aid human endoscopists.

Dr. Pambianco comoderated the session where the data were presented.

The investigators did not report a study funding source. Dr. Anderson and Dr. Pambianco reported having no relevant financial disclosures.

CHARLOTTE, N.C.– Performing high-quality index colonoscopies may pay off later in your patients’ reduced risk for advanced neoplasia, investigators report.

A study of registry data on more than 2,200 patients who had an index colonoscopy showing no evidence of neoplasia found that, on repeat colonoscopy 10 years later, the absolute risk for advanced neoplasia outcomes was lower for those with a high-quality index exam, compared with those who had a lesser-quality index colonoscopy.

Neil Osterweil/MDedge News

The adjusted odds ratio for patients who underwent high-quality index exams was 0.59%, reported Joseph Anderson, MD, from the Geisel School of Medicine at Dartmouth, Hanover, N.H.

“These data demonstrate that high-quality index colonoscopy provides better protection from interval lesions than low-quality exams with no polyps detected at that index exam,” he said in an oral abstract presentation at the annual meeting of the American College of Gastroenterology.

“These data support the importance of high-quality index exams in the prevention of interval colorectal cancer, and support the 10-year interval for normal exams,” Dr. Anderson added.

He recommended that endoscopists focus on the quality of their exams by using adequate scope withdrawal time – 8-10 minutes – to ensure optimal adenoma detection, and by ensuring the use of optimal bowel preparation in their practices.

Registry study

Dr. Anderson and colleagues studied how the quality of index colonoscopies could affect the risk of advanced outcomes in low-risk patients at the 10-year or later follow-up. They used records from the New Hampshire Colonoscopy Registry, which includes data from 2004 to the present on more than 250,000 exams performed by more than 150 endoscopists in more than 30 Granite State practices.

The investigators also looked at data on patients with less than 5 years of follow-up, and those with follow-up from 5 to less than 10 years.

The study sample included patients with no adenoma or significant serrated polyps on their index exams who had at least one follow-up exam 12 months or more after the index exams. Patients with inflammatory bowel disease or familial colon cancer syndromes were excluded.

They defined a high-quality colonoscopy as an exam complete to cecum, with adequate bowel preparation, and performed by an endoscopist with an adenoma detection rate of 25 or higher.

The adenoma detection rate is calculated as the number of screening colonoscopies with at least one adenoma divided by the total number of screening colonoscopies.

The definition of advanced outcomes included advanced adenomas, colorectal cancer, and/or large serrated polyps (1 cm or greater).

Of the 14,011 patients in the sample, 2,283 had a follow-up exam at 10 years. The absolute risk for advanced outcomes among patients who had a high quality index exam was 4.0% vs. 6.7% for those with lower quality exams.

Among patients with low-quality index exams – but not patients with high quality exams – there was a statistically significant increase in the absolute risk for advanced outcomes at all time periods, from 5.1% in the less than 5-year follow-up group, to 6.7% in the 10-years or more follow-up group.

Patients with initial high-quality exams also had a lower risk for postcolonoscopy colorectal cancer, compared with patients who had low-quality index exams: 0.4% vs. 0.8%. This difference translated into an adjusted hazard ratio for colorectal cancer after a high-quality exam of 0.53.
 

It’s getting better all the time

In an interview, Daniel J. Pambianco, MD, FACG from Charlottesville (Va.) Gastroenterology Associates, who was not involved in the study, commented that Dr. Anderson and colleagues highlighted the importance of the quality of the bowel prep and the quality of the examination itself.

He noted that the use of devices such as colonoscopy caps can help further improve adenoma detection rates and pointed to up-and-coming developments such as the use of artificial intelligence algorithms to aid human endoscopists.

Dr. Pambianco comoderated the session where the data were presented.

The investigators did not report a study funding source. Dr. Anderson and Dr. Pambianco reported having no relevant financial disclosures.

CHARLOTTE, N.C. – In a real-world setting, a 1-liter polyethylene glycol and ascorbic acid combination produced a high level of adequate or better bowel cleansing for colonoscopy.

Among more than 13,000 patients who used the combination, abbreviated as 1L PEG+ASC (Plenvu), the overall rate of adequate quality bowel prep was 89.3%, reported Cátia Arieira, MD, from the Hospital da Senhora da Oliveira in Guimarães, Portugal.

The rate of adequate prep was significantly higher with a split-dose regimen (evening-morning) than with a same-day regimen, at 94.7% versus 86.7%, respectively.

“Results from this large study confirm the high cleansing effectiveness and good tolerability of 1 liter of polyethylene glycol and ascorbic acid in real-world settings,” she said in an oral abstract session during the annual meeting of the American College of Gastroenterology.

Designed for tolerability

The 1L PEG+ASC regimen is intended to make precolonoscopy bowel prep a little easier both to take, by reducing the volume of liquid patients need to ingest, and to reduce indigestion with two asymmetric doses, with the second dose having a high ascorbate content.

The 1-liter regimen has been shown to be safe and effective both in clinical trials and in smaller practice-based studies, Dr. Arieira said.

To see how well 1L PEG+ASC performs on a larger scale, the investigators conducted a retrospective observational study of patients underwent a colonoscopy from June 2019 to September 2021 at 12 centers in Spain and Portugal.

The sample included patients who had either a screening, diagnostic, or surveillance colonoscopy and used 1L PEG+ASC in either a split or same-day dose.

The investigators used the Boston Bowel Preparation Scale (BBPS) to evaluate the quality of cleansing. They defined an adequate cleansing as a total BBPS score of 6 or greater, with all segmental scores 2 or greater, and a high-quality cleansing as segmental scores of 3.

They enrolled a total of 13,169 patients, 6,406 men and 6,763 women. The same-day regimen was used by two-thirds of patients, and the split-dose regimen by one-third.

In all, 41.9% of procedures were for screening, 29.4% for diagnosis, 26.2% for surveillance, and 2.6% for other, unspecified reasons.

Results

As noted, the overall rate of adequate prep was 89.3%, with rates of 94.7% and 86.7% for the split and same-day doses, respectively.

A breakdown of cleansing by bowel segment showed that, for each segment, the split-dose regimen was numerically superior to the same-day regimen, with rates of 95.6% versus 89.5% for the right colon, 97.1% versus 91.9% for the left colon, and 97.8% versus 93.1% for the transverse colon, respectively.

Mean BBPS scores were significantly better with split dosing, at 8.02 versus 6.96. Higher scores were seen with split-dosing for each colon segment.

The incidence of adverse events was low, at 2.3% overall, 1.4% for same-day dosing, and 3.9% for split dosing, with nausea the most common.

Tolerability is key

Renee L. Williams, MD, MHPE, FACG, from New York University, who moderated the session but was not involved in the study, commented that the more convenient 1L PEG+ASC regimen may be helpful with improving compliance with bowel prep in underserved populations.

“My population of patients is very different from the one in this study,” she said in an interview. “Normally, if you’re looking at people who are not prepped, at least in the United States, people who have a lot of comorbidities, who are underserved, or have insurance uncertainty tend to have a lower level of bowel prep. So I’d be curious to see whether this would work in that population.”

Dr. Williams noted that she prefers split dosing for bowel prep because it offers better tolerability for patients, adding that when her center introduced split-dose prep, the percentage of adequate prep rose from around 60% to more than 90%.

Comoderator John R. Saltzman, MD, FACG, from Harvard Medical School and Brigham & Women’s Hospital, both in Boston, said that while he’s not familiar with this specific bowel prep formulation, “I’m looking for whatever is most palatable to patients and most effective in practice. Still, most of our patients tolerate these 2-liter overnight preps very well.”

The 1L-PEG+ASC regimen may be a suitable option for patients whose colonoscopies are scheduled for later in the day, Dr. Saltzman added.

The study was supported by Norgine and Xolomon Tree. Dr. Arieira, Dr. Williams, and Dr. Saltzman reported no relevant conflicts of interest.

CHARLOTTE, N.C. – In a real-world setting, a 1-liter polyethylene glycol and ascorbic acid combination produced a high level of adequate or better bowel cleansing for colonoscopy.

Among more than 13,000 patients who used the combination, abbreviated as 1L PEG+ASC (Plenvu), the overall rate of adequate quality bowel prep was 89.3%, reported Cátia Arieira, MD, from the Hospital da Senhora da Oliveira in Guimarães, Portugal.

The rate of adequate prep was significantly higher with a split-dose regimen (evening-morning) than with a same-day regimen, at 94.7% versus 86.7%, respectively.

“Results from this large study confirm the high cleansing effectiveness and good tolerability of 1 liter of polyethylene glycol and ascorbic acid in real-world settings,” she said in an oral abstract session during the annual meeting of the American College of Gastroenterology.

Designed for tolerability

The 1L PEG+ASC regimen is intended to make precolonoscopy bowel prep a little easier both to take, by reducing the volume of liquid patients need to ingest, and to reduce indigestion with two asymmetric doses, with the second dose having a high ascorbate content.

The 1-liter regimen has been shown to be safe and effective both in clinical trials and in smaller practice-based studies, Dr. Arieira said.

To see how well 1L PEG+ASC performs on a larger scale, the investigators conducted a retrospective observational study of patients underwent a colonoscopy from June 2019 to September 2021 at 12 centers in Spain and Portugal.

The sample included patients who had either a screening, diagnostic, or surveillance colonoscopy and used 1L PEG+ASC in either a split or same-day dose.

The investigators used the Boston Bowel Preparation Scale (BBPS) to evaluate the quality of cleansing. They defined an adequate cleansing as a total BBPS score of 6 or greater, with all segmental scores 2 or greater, and a high-quality cleansing as segmental scores of 3.

They enrolled a total of 13,169 patients, 6,406 men and 6,763 women. The same-day regimen was used by two-thirds of patients, and the split-dose regimen by one-third.

In all, 41.9% of procedures were for screening, 29.4% for diagnosis, 26.2% for surveillance, and 2.6% for other, unspecified reasons.

Results

As noted, the overall rate of adequate prep was 89.3%, with rates of 94.7% and 86.7% for the split and same-day doses, respectively.

A breakdown of cleansing by bowel segment showed that, for each segment, the split-dose regimen was numerically superior to the same-day regimen, with rates of 95.6% versus 89.5% for the right colon, 97.1% versus 91.9% for the left colon, and 97.8% versus 93.1% for the transverse colon, respectively.

Mean BBPS scores were significantly better with split dosing, at 8.02 versus 6.96. Higher scores were seen with split-dosing for each colon segment.

The incidence of adverse events was low, at 2.3% overall, 1.4% for same-day dosing, and 3.9% for split dosing, with nausea the most common.

Tolerability is key

Renee L. Williams, MD, MHPE, FACG, from New York University, who moderated the session but was not involved in the study, commented that the more convenient 1L PEG+ASC regimen may be helpful with improving compliance with bowel prep in underserved populations.

“My population of patients is very different from the one in this study,” she said in an interview. “Normally, if you’re looking at people who are not prepped, at least in the United States, people who have a lot of comorbidities, who are underserved, or have insurance uncertainty tend to have a lower level of bowel prep. So I’d be curious to see whether this would work in that population.”

Dr. Williams noted that she prefers split dosing for bowel prep because it offers better tolerability for patients, adding that when her center introduced split-dose prep, the percentage of adequate prep rose from around 60% to more than 90%.

Comoderator John R. Saltzman, MD, FACG, from Harvard Medical School and Brigham & Women’s Hospital, both in Boston, said that while he’s not familiar with this specific bowel prep formulation, “I’m looking for whatever is most palatable to patients and most effective in practice. Still, most of our patients tolerate these 2-liter overnight preps very well.”

The 1L-PEG+ASC regimen may be a suitable option for patients whose colonoscopies are scheduled for later in the day, Dr. Saltzman added.

The study was supported by Norgine and Xolomon Tree. Dr. Arieira, Dr. Williams, and Dr. Saltzman reported no relevant conflicts of interest.

CHARLOTTE, N.C. – In a real-world setting, a 1-liter polyethylene glycol and ascorbic acid combination produced a high level of adequate or better bowel cleansing for colonoscopy.

Among more than 13,000 patients who used the combination, abbreviated as 1L PEG+ASC (Plenvu), the overall rate of adequate quality bowel prep was 89.3%, reported Cátia Arieira, MD, from the Hospital da Senhora da Oliveira in Guimarães, Portugal.

The rate of adequate prep was significantly higher with a split-dose regimen (evening-morning) than with a same-day regimen, at 94.7% versus 86.7%, respectively.

“Results from this large study confirm the high cleansing effectiveness and good tolerability of 1 liter of polyethylene glycol and ascorbic acid in real-world settings,” she said in an oral abstract session during the annual meeting of the American College of Gastroenterology.

Designed for tolerability

The 1L PEG+ASC regimen is intended to make precolonoscopy bowel prep a little easier both to take, by reducing the volume of liquid patients need to ingest, and to reduce indigestion with two asymmetric doses, with the second dose having a high ascorbate content.

The 1-liter regimen has been shown to be safe and effective both in clinical trials and in smaller practice-based studies, Dr. Arieira said.

To see how well 1L PEG+ASC performs on a larger scale, the investigators conducted a retrospective observational study of patients underwent a colonoscopy from June 2019 to September 2021 at 12 centers in Spain and Portugal.

The sample included patients who had either a screening, diagnostic, or surveillance colonoscopy and used 1L PEG+ASC in either a split or same-day dose.

The investigators used the Boston Bowel Preparation Scale (BBPS) to evaluate the quality of cleansing. They defined an adequate cleansing as a total BBPS score of 6 or greater, with all segmental scores 2 or greater, and a high-quality cleansing as segmental scores of 3.

They enrolled a total of 13,169 patients, 6,406 men and 6,763 women. The same-day regimen was used by two-thirds of patients, and the split-dose regimen by one-third.

In all, 41.9% of procedures were for screening, 29.4% for diagnosis, 26.2% for surveillance, and 2.6% for other, unspecified reasons.

Results

As noted, the overall rate of adequate prep was 89.3%, with rates of 94.7% and 86.7% for the split and same-day doses, respectively.

A breakdown of cleansing by bowel segment showed that, for each segment, the split-dose regimen was numerically superior to the same-day regimen, with rates of 95.6% versus 89.5% for the right colon, 97.1% versus 91.9% for the left colon, and 97.8% versus 93.1% for the transverse colon, respectively.

Mean BBPS scores were significantly better with split dosing, at 8.02 versus 6.96. Higher scores were seen with split-dosing for each colon segment.

The incidence of adverse events was low, at 2.3% overall, 1.4% for same-day dosing, and 3.9% for split dosing, with nausea the most common.

Tolerability is key

Renee L. Williams, MD, MHPE, FACG, from New York University, who moderated the session but was not involved in the study, commented that the more convenient 1L PEG+ASC regimen may be helpful with improving compliance with bowel prep in underserved populations.

“My population of patients is very different from the one in this study,” she said in an interview. “Normally, if you’re looking at people who are not prepped, at least in the United States, people who have a lot of comorbidities, who are underserved, or have insurance uncertainty tend to have a lower level of bowel prep. So I’d be curious to see whether this would work in that population.”

Dr. Williams noted that she prefers split dosing for bowel prep because it offers better tolerability for patients, adding that when her center introduced split-dose prep, the percentage of adequate prep rose from around 60% to more than 90%.

Comoderator John R. Saltzman, MD, FACG, from Harvard Medical School and Brigham & Women’s Hospital, both in Boston, said that while he’s not familiar with this specific bowel prep formulation, “I’m looking for whatever is most palatable to patients and most effective in practice. Still, most of our patients tolerate these 2-liter overnight preps very well.”

The 1L-PEG+ASC regimen may be a suitable option for patients whose colonoscopies are scheduled for later in the day, Dr. Saltzman added.

The study was supported by Norgine and Xolomon Tree. Dr. Arieira, Dr. Williams, and Dr. Saltzman reported no relevant conflicts of interest.

Josie Tenore, MD, and Paul Hinds were introduced by a mutual friend in 2017 and hadn’t been going out long when she laid down the law: He had to get a physical.

“I don’t date people who don’t take care of their health,” said Dr. Tenore, who practices cosmetic dermatology and functional medicine in suburban Chicago.

One of Mr. Hinds’ blood tests that summer came back with an alarming result: His prostate-specific antigen (PSA), level was very high. A biopsy confirmed he had advanced prostate cancer.

There aren’t a lot of comfortable alternatives for treating prostate cancer, which generally progresses as long as testosterone levels remain high. Marijuana appears to lower testosterone levels, so after his diagnosis, he dosed a liquid form of cannabis for several weeks. That cut his PSA in half, but Mr. Hinds, a cybersecurity expert who likes yoga and bicycling, “was stoned out of his mind and couldn’t function,” Dr. Tenore recalled.

With Dr. Tenore guiding his decisions, Mr. Hinds next tried high-frequency ultrasound treatment, but it failed. And in the summer of 2019 doctors removed his prostate gland. Still, the PSA levels climbed again, and doctors assessed that the cancer had metastasized. The only alternative was to drastically lower Mr. Hinds’ testosterone levels – either via surgery or drugs that block all testosterone. In May 2021, he got his first intramuscular shot of Lupron Depot, a brand name for leuprolide, designed to suppress the prostate gland’s release of the hormone for 3 months. That August, he got his second shot.

And then the bills came.

The patient: Paul Hinds, now 60, is covered by United Healthcare through a COBRA plan from his former employer.

Medical service: Two 3-month Lupron Depot injections for metastatic prostate cancer.

Service provider: University of Chicago Medicine, a 900-physician nonprofit system that includes an 811-bed medical center, a suburban hospital, the Pritzker School of Medicine, and outpatient clinics and physician offices throughout the Chicago area.

Total bill: $73,812 for the two shots ($35,414 for the first, $38,398 for the second), including lab work and physician charges. United Healthcare’s negotiated rate for the two shots plus associated fees was $27,568, of which the insurer paid $19,567. After Mr. Hinds haggled with the hospital and insurer for more than a year, his share of the bills was determined to be nearly $7,000.

What gives: The first issue is unrelenting price increases on old drugs that have remained branded as manufacturers find ways to extend patents for decades and maintain sales through marketing.

Though Lupron was invented in 1973, its manufacturer got patent extensions in 1989 by offering a slow-release version. Drugmakers commonly use this tactic to extend their exclusive rights to sell a product.

The development of Lupron Depot as an intramuscular shot that suppressed testosterone for months at a time improved patient compliance and also enabled its maker, Abbott Laboratories, and its Japanese partner, Takeda, to extend their patents on the drug into the 2000s, said Gerald Weisberg, MD, a former Abbott scientist who has been critical of the company’s pricing policies.

In subsequent years, Abbott and Takeda, in a joint venture called TAP Pharmaceuticals, steadily marked up the price of their slow-release product. In 2000, the average wholesale U.S. price for a 3-month shot was $1,245; currently that figure is $5,866. (It is manufactured in the United States by AbbVie now.)

In the United Kingdom, where health care is generally free and Takeda sells the drug under the name Prostap, all physicians can purchase a 3-month dose for about $260.

It’s likely that Chicago Medicine, where Mr. Hinds got his shots, paid something close to the British price. That’s because the health system’s hospital on Chicago’s South Side participates in a federal program called 340B, which allows hospitals that serve low-income populations to purchase drugs at deep discounts.

Lupron Depot is given as a simple injection into the muscle. It takes minutes for a nurse or doctor to administer. Yet hospital systems like Chicago Medicine can and typically do charge lavishly for such services, to enhance revenue, said Morgan Henderson, principal data scientist at the Hilltop Institute at the University of Maryland-Baltimore County. Chicago Medicine declined to say what it paid for the drug.

While U.S. drugmakers can price their drugs however they please, TAP has gotten into trouble for its Lupron sales policies in the past. In 2001, after a Justice Department probe, it paid an $875 million settlement for illegally stimulating sales by giving urologists free and discounted vials of the drug while enabling them to charge Medicare full price.

Since then, many other drugs aimed at lowering testosterone levels have entered the market, including a pill, relugolix (Orgovyx). So why wouldn’t a patient use them?

Lupron Depot is long acting, is easy to prepare and store, and employs a small needle, which some patients prefer, said Brian McNeil, MD, chief of urology at University Hospital of Brooklyn. Orgovyx is convenient, but “a patient has to be very compliant. They have to take it every day around the same time,” he said. “Some people just forget.”

But there is another important factor that may well explain Lupron Depot’s ongoing popularity among medical providers: Doctors and hospitals can earn tens of thousands of dollars each visit by marking up its price and administration fees – as they did with Mr. Hinds. If they merely write a prescription for a drug that can be taken at home, they earn nothing.

Asked about this high patient charge and the possibility of using alternatives, United spokesperson Maria Gordon Shydlo said payment was “appropriately based on the hospital’s contract and the member’s benefit plan,” adding that the insurer encourages customers to shop around for the best quality and price.

Resolution: In addition to leaving Mr. Hinds listless, the Lupron Depot shots were, literally, a pain in the rear end. “Each time he was miserable for 2 weeks,” Dr. Tenore said. After looking over his first bill for the Lupron shot, Dr. Tenore told Mr. Hinds he should ask his doctor whether there was a less expensive drug that was easier to take.

After the second shot, in August 2021, a pharmacist told him he could instead receive the pill. His doctor prescribed Mr. Hinds 3 months’ worth of Orgovyx last November, for which he paid $216 and the insurer paid over $6,000. The drug’s list price is about $2,700 a month. There is evidence that Orgovyx works a little better than leuprolide.

Orgovyx was a “no-brainer,” Mr. Hinds said. “Why would you want a sore ass for two weeks when you can take a pill that kicks in sooner, functions the same way, and clears your body of testosterone faster?”

While Orgovyx is increasingly used for prostate cancer, Lupron and other injections usually remain the standard of care, hospital spokesperson Ashley Heher said. Clinicians “work with patients to determine what treatments are the most medically effective and, when necessary, to find reasonable alternatives that may be less financially burdensome due to insurance coverage limitations.”

Mr. Hinds was baffled by the size of the charges. During months of phone calls and emails, the hospital reversed and then reapplied part of the charge, and then in July agreed to a $666.34 monthly payment plan. After Hinds had made two payments, however, the hospital announced Aug. 29 it was canceling the agreement and sending the remainder of his bill to a collection agency. Two weeks later, the hospital reinstated the payment plan – after KHN asked about the cancellation.

As for Mr. Hinds, he remains active, though his bike rides have been shortened from 50 or 60 miles to about 30, he said.

He’s grateful to have Dr. Tenore as a free consultant and empathizes with those who lack a knowledgeable guide through their disease and health care’s financial maze.

“I’ve got Dr. Josie as an advocate who knows the system,” Mr. Hinds said.

The takeaway: First tip: If you are prescribed an infusion or injection, ask your physician if there are cheaper oral medications to treat your condition. Also, many drugs that are given by injection – ones that are given “subcutaneously,” rather than into a muscle – can be administered by a patient at home, avoiding hefty administration fees. Drugs like Dupixent for eczema fall into this category.

Keep in mind that where you get treatment could make a big difference in your charges: A study found that leading U.S. cancer centers charge enormous markups to private insurers for drug injections or infusions. Another study found that hospital systems charge an average of 86% more than private clinics for cancer drug infusions. And the percentage of cancer infusions done in hospital-operated clinics increased from 6% in 2004 to 43% in 2014, and has grown since.

Under a law that took effect in 2021, hospitals are required to list their charges, though they currently do so in a way that is not user friendly. But it’s worth taking a look at the price list – the hospital chargemaster – to try to decipher the pricing and markup for your medicine. If you’re about to get an injection, infusion, or procedure done in a hospital system, ask ahead of time for an estimate of what you will owe.

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.

Josie Tenore, MD, and Paul Hinds were introduced by a mutual friend in 2017 and hadn’t been going out long when she laid down the law: He had to get a physical.

“I don’t date people who don’t take care of their health,” said Dr. Tenore, who practices cosmetic dermatology and functional medicine in suburban Chicago.

One of Mr. Hinds’ blood tests that summer came back with an alarming result: His prostate-specific antigen (PSA), level was very high. A biopsy confirmed he had advanced prostate cancer.

There aren’t a lot of comfortable alternatives for treating prostate cancer, which generally progresses as long as testosterone levels remain high. Marijuana appears to lower testosterone levels, so after his diagnosis, he dosed a liquid form of cannabis for several weeks. That cut his PSA in half, but Mr. Hinds, a cybersecurity expert who likes yoga and bicycling, “was stoned out of his mind and couldn’t function,” Dr. Tenore recalled.

With Dr. Tenore guiding his decisions, Mr. Hinds next tried high-frequency ultrasound treatment, but it failed. And in the summer of 2019 doctors removed his prostate gland. Still, the PSA levels climbed again, and doctors assessed that the cancer had metastasized. The only alternative was to drastically lower Mr. Hinds’ testosterone levels – either via surgery or drugs that block all testosterone. In May 2021, he got his first intramuscular shot of Lupron Depot, a brand name for leuprolide, designed to suppress the prostate gland’s release of the hormone for 3 months. That August, he got his second shot.

And then the bills came.

The patient: Paul Hinds, now 60, is covered by United Healthcare through a COBRA plan from his former employer.

Medical service: Two 3-month Lupron Depot injections for metastatic prostate cancer.

Service provider: University of Chicago Medicine, a 900-physician nonprofit system that includes an 811-bed medical center, a suburban hospital, the Pritzker School of Medicine, and outpatient clinics and physician offices throughout the Chicago area.

Total bill: $73,812 for the two shots ($35,414 for the first, $38,398 for the second), including lab work and physician charges. United Healthcare’s negotiated rate for the two shots plus associated fees was $27,568, of which the insurer paid $19,567. After Mr. Hinds haggled with the hospital and insurer for more than a year, his share of the bills was determined to be nearly $7,000.

What gives: The first issue is unrelenting price increases on old drugs that have remained branded as manufacturers find ways to extend patents for decades and maintain sales through marketing.

Though Lupron was invented in 1973, its manufacturer got patent extensions in 1989 by offering a slow-release version. Drugmakers commonly use this tactic to extend their exclusive rights to sell a product.

The development of Lupron Depot as an intramuscular shot that suppressed testosterone for months at a time improved patient compliance and also enabled its maker, Abbott Laboratories, and its Japanese partner, Takeda, to extend their patents on the drug into the 2000s, said Gerald Weisberg, MD, a former Abbott scientist who has been critical of the company’s pricing policies.

In subsequent years, Abbott and Takeda, in a joint venture called TAP Pharmaceuticals, steadily marked up the price of their slow-release product. In 2000, the average wholesale U.S. price for a 3-month shot was $1,245; currently that figure is $5,866. (It is manufactured in the United States by AbbVie now.)

In the United Kingdom, where health care is generally free and Takeda sells the drug under the name Prostap, all physicians can purchase a 3-month dose for about $260.

It’s likely that Chicago Medicine, where Mr. Hinds got his shots, paid something close to the British price. That’s because the health system’s hospital on Chicago’s South Side participates in a federal program called 340B, which allows hospitals that serve low-income populations to purchase drugs at deep discounts.

Lupron Depot is given as a simple injection into the muscle. It takes minutes for a nurse or doctor to administer. Yet hospital systems like Chicago Medicine can and typically do charge lavishly for such services, to enhance revenue, said Morgan Henderson, principal data scientist at the Hilltop Institute at the University of Maryland-Baltimore County. Chicago Medicine declined to say what it paid for the drug.

While U.S. drugmakers can price their drugs however they please, TAP has gotten into trouble for its Lupron sales policies in the past. In 2001, after a Justice Department probe, it paid an $875 million settlement for illegally stimulating sales by giving urologists free and discounted vials of the drug while enabling them to charge Medicare full price.

Since then, many other drugs aimed at lowering testosterone levels have entered the market, including a pill, relugolix (Orgovyx). So why wouldn’t a patient use them?

Lupron Depot is long acting, is easy to prepare and store, and employs a small needle, which some patients prefer, said Brian McNeil, MD, chief of urology at University Hospital of Brooklyn. Orgovyx is convenient, but “a patient has to be very compliant. They have to take it every day around the same time,” he said. “Some people just forget.”

But there is another important factor that may well explain Lupron Depot’s ongoing popularity among medical providers: Doctors and hospitals can earn tens of thousands of dollars each visit by marking up its price and administration fees – as they did with Mr. Hinds. If they merely write a prescription for a drug that can be taken at home, they earn nothing.

Asked about this high patient charge and the possibility of using alternatives, United spokesperson Maria Gordon Shydlo said payment was “appropriately based on the hospital’s contract and the member’s benefit plan,” adding that the insurer encourages customers to shop around for the best quality and price.

Resolution: In addition to leaving Mr. Hinds listless, the Lupron Depot shots were, literally, a pain in the rear end. “Each time he was miserable for 2 weeks,” Dr. Tenore said. After looking over his first bill for the Lupron shot, Dr. Tenore told Mr. Hinds he should ask his doctor whether there was a less expensive drug that was easier to take.

After the second shot, in August 2021, a pharmacist told him he could instead receive the pill. His doctor prescribed Mr. Hinds 3 months’ worth of Orgovyx last November, for which he paid $216 and the insurer paid over $6,000. The drug’s list price is about $2,700 a month. There is evidence that Orgovyx works a little better than leuprolide.

Orgovyx was a “no-brainer,” Mr. Hinds said. “Why would you want a sore ass for two weeks when you can take a pill that kicks in sooner, functions the same way, and clears your body of testosterone faster?”

While Orgovyx is increasingly used for prostate cancer, Lupron and other injections usually remain the standard of care, hospital spokesperson Ashley Heher said. Clinicians “work with patients to determine what treatments are the most medically effective and, when necessary, to find reasonable alternatives that may be less financially burdensome due to insurance coverage limitations.”

Mr. Hinds was baffled by the size of the charges. During months of phone calls and emails, the hospital reversed and then reapplied part of the charge, and then in July agreed to a $666.34 monthly payment plan. After Hinds had made two payments, however, the hospital announced Aug. 29 it was canceling the agreement and sending the remainder of his bill to a collection agency. Two weeks later, the hospital reinstated the payment plan – after KHN asked about the cancellation.

As for Mr. Hinds, he remains active, though his bike rides have been shortened from 50 or 60 miles to about 30, he said.

He’s grateful to have Dr. Tenore as a free consultant and empathizes with those who lack a knowledgeable guide through their disease and health care’s financial maze.

“I’ve got Dr. Josie as an advocate who knows the system,” Mr. Hinds said.

The takeaway: First tip: If you are prescribed an infusion or injection, ask your physician if there are cheaper oral medications to treat your condition. Also, many drugs that are given by injection – ones that are given “subcutaneously,” rather than into a muscle – can be administered by a patient at home, avoiding hefty administration fees. Drugs like Dupixent for eczema fall into this category.

Keep in mind that where you get treatment could make a big difference in your charges: A study found that leading U.S. cancer centers charge enormous markups to private insurers for drug injections or infusions. Another study found that hospital systems charge an average of 86% more than private clinics for cancer drug infusions. And the percentage of cancer infusions done in hospital-operated clinics increased from 6% in 2004 to 43% in 2014, and has grown since.

Under a law that took effect in 2021, hospitals are required to list their charges, though they currently do so in a way that is not user friendly. But it’s worth taking a look at the price list – the hospital chargemaster – to try to decipher the pricing and markup for your medicine. If you’re about to get an injection, infusion, or procedure done in a hospital system, ask ahead of time for an estimate of what you will owe.

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.

Josie Tenore, MD, and Paul Hinds were introduced by a mutual friend in 2017 and hadn’t been going out long when she laid down the law: He had to get a physical.

“I don’t date people who don’t take care of their health,” said Dr. Tenore, who practices cosmetic dermatology and functional medicine in suburban Chicago.

One of Mr. Hinds’ blood tests that summer came back with an alarming result: His prostate-specific antigen (PSA), level was very high. A biopsy confirmed he had advanced prostate cancer.

There aren’t a lot of comfortable alternatives for treating prostate cancer, which generally progresses as long as testosterone levels remain high. Marijuana appears to lower testosterone levels, so after his diagnosis, he dosed a liquid form of cannabis for several weeks. That cut his PSA in half, but Mr. Hinds, a cybersecurity expert who likes yoga and bicycling, “was stoned out of his mind and couldn’t function,” Dr. Tenore recalled.

With Dr. Tenore guiding his decisions, Mr. Hinds next tried high-frequency ultrasound treatment, but it failed. And in the summer of 2019 doctors removed his prostate gland. Still, the PSA levels climbed again, and doctors assessed that the cancer had metastasized. The only alternative was to drastically lower Mr. Hinds’ testosterone levels – either via surgery or drugs that block all testosterone. In May 2021, he got his first intramuscular shot of Lupron Depot, a brand name for leuprolide, designed to suppress the prostate gland’s release of the hormone for 3 months. That August, he got his second shot.

And then the bills came.

The patient: Paul Hinds, now 60, is covered by United Healthcare through a COBRA plan from his former employer.

Medical service: Two 3-month Lupron Depot injections for metastatic prostate cancer.

Service provider: University of Chicago Medicine, a 900-physician nonprofit system that includes an 811-bed medical center, a suburban hospital, the Pritzker School of Medicine, and outpatient clinics and physician offices throughout the Chicago area.

Total bill: $73,812 for the two shots ($35,414 for the first, $38,398 for the second), including lab work and physician charges. United Healthcare’s negotiated rate for the two shots plus associated fees was $27,568, of which the insurer paid $19,567. After Mr. Hinds haggled with the hospital and insurer for more than a year, his share of the bills was determined to be nearly $7,000.

What gives: The first issue is unrelenting price increases on old drugs that have remained branded as manufacturers find ways to extend patents for decades and maintain sales through marketing.

Though Lupron was invented in 1973, its manufacturer got patent extensions in 1989 by offering a slow-release version. Drugmakers commonly use this tactic to extend their exclusive rights to sell a product.

The development of Lupron Depot as an intramuscular shot that suppressed testosterone for months at a time improved patient compliance and also enabled its maker, Abbott Laboratories, and its Japanese partner, Takeda, to extend their patents on the drug into the 2000s, said Gerald Weisberg, MD, a former Abbott scientist who has been critical of the company’s pricing policies.

In subsequent years, Abbott and Takeda, in a joint venture called TAP Pharmaceuticals, steadily marked up the price of their slow-release product. In 2000, the average wholesale U.S. price for a 3-month shot was $1,245; currently that figure is $5,866. (It is manufactured in the United States by AbbVie now.)

In the United Kingdom, where health care is generally free and Takeda sells the drug under the name Prostap, all physicians can purchase a 3-month dose for about $260.

It’s likely that Chicago Medicine, where Mr. Hinds got his shots, paid something close to the British price. That’s because the health system’s hospital on Chicago’s South Side participates in a federal program called 340B, which allows hospitals that serve low-income populations to purchase drugs at deep discounts.

Lupron Depot is given as a simple injection into the muscle. It takes minutes for a nurse or doctor to administer. Yet hospital systems like Chicago Medicine can and typically do charge lavishly for such services, to enhance revenue, said Morgan Henderson, principal data scientist at the Hilltop Institute at the University of Maryland-Baltimore County. Chicago Medicine declined to say what it paid for the drug.

While U.S. drugmakers can price their drugs however they please, TAP has gotten into trouble for its Lupron sales policies in the past. In 2001, after a Justice Department probe, it paid an $875 million settlement for illegally stimulating sales by giving urologists free and discounted vials of the drug while enabling them to charge Medicare full price.

Since then, many other drugs aimed at lowering testosterone levels have entered the market, including a pill, relugolix (Orgovyx). So why wouldn’t a patient use them?

Lupron Depot is long acting, is easy to prepare and store, and employs a small needle, which some patients prefer, said Brian McNeil, MD, chief of urology at University Hospital of Brooklyn. Orgovyx is convenient, but “a patient has to be very compliant. They have to take it every day around the same time,” he said. “Some people just forget.”

But there is another important factor that may well explain Lupron Depot’s ongoing popularity among medical providers: Doctors and hospitals can earn tens of thousands of dollars each visit by marking up its price and administration fees – as they did with Mr. Hinds. If they merely write a prescription for a drug that can be taken at home, they earn nothing.

Asked about this high patient charge and the possibility of using alternatives, United spokesperson Maria Gordon Shydlo said payment was “appropriately based on the hospital’s contract and the member’s benefit plan,” adding that the insurer encourages customers to shop around for the best quality and price.

Resolution: In addition to leaving Mr. Hinds listless, the Lupron Depot shots were, literally, a pain in the rear end. “Each time he was miserable for 2 weeks,” Dr. Tenore said. After looking over his first bill for the Lupron shot, Dr. Tenore told Mr. Hinds he should ask his doctor whether there was a less expensive drug that was easier to take.

After the second shot, in August 2021, a pharmacist told him he could instead receive the pill. His doctor prescribed Mr. Hinds 3 months’ worth of Orgovyx last November, for which he paid $216 and the insurer paid over $6,000. The drug’s list price is about $2,700 a month. There is evidence that Orgovyx works a little better than leuprolide.

Orgovyx was a “no-brainer,” Mr. Hinds said. “Why would you want a sore ass for two weeks when you can take a pill that kicks in sooner, functions the same way, and clears your body of testosterone faster?”

While Orgovyx is increasingly used for prostate cancer, Lupron and other injections usually remain the standard of care, hospital spokesperson Ashley Heher said. Clinicians “work with patients to determine what treatments are the most medically effective and, when necessary, to find reasonable alternatives that may be less financially burdensome due to insurance coverage limitations.”

Mr. Hinds was baffled by the size of the charges. During months of phone calls and emails, the hospital reversed and then reapplied part of the charge, and then in July agreed to a $666.34 monthly payment plan. After Hinds had made two payments, however, the hospital announced Aug. 29 it was canceling the agreement and sending the remainder of his bill to a collection agency. Two weeks later, the hospital reinstated the payment plan – after KHN asked about the cancellation.

As for Mr. Hinds, he remains active, though his bike rides have been shortened from 50 or 60 miles to about 30, he said.

He’s grateful to have Dr. Tenore as a free consultant and empathizes with those who lack a knowledgeable guide through their disease and health care’s financial maze.

“I’ve got Dr. Josie as an advocate who knows the system,” Mr. Hinds said.

The takeaway: First tip: If you are prescribed an infusion or injection, ask your physician if there are cheaper oral medications to treat your condition. Also, many drugs that are given by injection – ones that are given “subcutaneously,” rather than into a muscle – can be administered by a patient at home, avoiding hefty administration fees. Drugs like Dupixent for eczema fall into this category.

Keep in mind that where you get treatment could make a big difference in your charges: A study found that leading U.S. cancer centers charge enormous markups to private insurers for drug injections or infusions. Another study found that hospital systems charge an average of 86% more than private clinics for cancer drug infusions. And the percentage of cancer infusions done in hospital-operated clinics increased from 6% in 2004 to 43% in 2014, and has grown since.

Under a law that took effect in 2021, hospitals are required to list their charges, though they currently do so in a way that is not user friendly. But it’s worth taking a look at the price list – the hospital chargemaster – to try to decipher the pricing and markup for your medicine. If you’re about to get an injection, infusion, or procedure done in a hospital system, ask ahead of time for an estimate of what you will owe.

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.