Randy Dotinga

A pair of new studies offers more evidence for the value of vegetables and the risk of red meat on the cancer prevention front. Researchers report that high consumption of vegetables – especially lettuce, legumes, and cruciferous varieties – appears to lower the risk of liver cancer/liver disease. A separate team suggests that high consumption of red meat, organ meats, and processed meats boosts the risk of gastric cancer.

The findings of the latter study “reinforce the idea that avoidance of red meat and processed meat is probably good beyond [the prevention of] colorectal cancer,” said corresponding author and epidemiologist Paolo Boffetta, MD, MPH, of Stony Brook University Cancer Center, New York, in an interview. “The possible carcinogenic effect may extend beyond the colon.”

Both studies were released at the annual meeting of the American Association for Cancer Research.

For the red meat study, researchers examined statistics from the Golestan cohort study, which is prospectively tracking 50,045 people aged 40-75 from northeastern Iran. The study focuses on esophageal cancer due to the region’s high rate of the disease.

Red meat consumption is fairly rare in the region, where residents typically prefer chicken, said study lead author Giulia Collatuzzo, MD, a resident physician in occupational medicine at the University of Bologna, Italy, in an interview. On average, participants reported eating 18.4 grams daily of red meat and 72.1 grams daily of white meat.

The researchers tracked study participants for a median 12-year follow-up, during which 369 developed esophageal cancer and 368 developed gastric cancer. Red meat was only linked to more esophageal cancer in women (hazard ratio, 1.13, 95% confidence interval, 1.00-1.18, for each quintile increase in consumption).

Overall red meat consumption (including red meat, organ meat, and processed meat) was linked to higher rates of gastric cancer (HR, 1.08, 95% CI, 1.00-1.17) for each quartile increase in consumption, as was consumption of the red meat subtype alone (HR, 1.09, 95% CI, 1.00-1.18).

According to Dr. Collatuzzo, the findings suggest that those in the highest quartile of overall red meat consumption may have around a 25% increase in risk, compared with the lowest quartile.

Overall, she said, the study findings aren’t surprising. The lack of a connection between red meat consumption and esophageal cancer may be due to the fact that meat only temporarily transits through the esophagus, she said.

For the liver cancer/liver disease study, researchers examined the medical records of 470,653 subjects in the NIH-AARP Diet and Health Study. They were recruited in 1995-1996 when they were 50-71 years old. Over a median follow-up of 15.5 years, 899 developed liver cancer, and 934 died of chronic liver disease.

The median intakes of vegetables in quintile 5 (highest) and quintile 1 (lowest) were 3.7 cups daily and 1.0 cups daily, respectively, said study lead author Long-Gang Zhao, MS, a graduate student at Harvard University.

After adjusting for possible cofounders, those in the highest quintile of vegetable consumption were a third less likely to develop liver cancer, compared with the lowest quintile (HR, 0.66, 95% CI, 0.53-0.82, P < 0.01). Several types of vegetables appeared to be the strongest cancer fighters: cruciferous (broccoli, cauliflower), lettuce, legumes, and carrots. These kinds of vegetables were also linked to lower rates of chronic liver disease mortality (all P < 0.01), as was total vegetable intake for the top quintile versus the lowest quintile (HR, 0.60, 95% CI, 0.49-0.74, P = < 0.01).

“A one-cup increase (8 oz or 225 g) in vegetable intake was associated with about 20% decreased risk of liver cancer incidence and chronic liver mortality,” Zhao said.

There was no statistically significant link between fruit consumption and liver cancer or chronic liver disease mortality.

The findings provide more insight into diet and liver disease, Zhao said. “Chronic liver disease, which predisposes to liver cancer, is the tenth cause of death worldwide, causing two million deaths each year. It shares some etiological processes with liver cancer. Therefore, examining both chronic liver disease mortality and liver cancer incidence in our study may provide a more general picture for the prevention of liver diseases.”

As for limitations, both studies are based on self-reports about food consumption, which can be unreliable, and the subjects in the fruit/vegetable analysis were mainly of European origin.

The authors of both studies report no relevant disclosures. No funding is reported for either study.

A pair of new studies offers more evidence for the value of vegetables and the risk of red meat on the cancer prevention front. Researchers report that high consumption of vegetables – especially lettuce, legumes, and cruciferous varieties – appears to lower the risk of liver cancer/liver disease. A separate team suggests that high consumption of red meat, organ meats, and processed meats boosts the risk of gastric cancer.

The findings of the latter study “reinforce the idea that avoidance of red meat and processed meat is probably good beyond [the prevention of] colorectal cancer,” said corresponding author and epidemiologist Paolo Boffetta, MD, MPH, of Stony Brook University Cancer Center, New York, in an interview. “The possible carcinogenic effect may extend beyond the colon.”

Both studies were released at the annual meeting of the American Association for Cancer Research.

For the red meat study, researchers examined statistics from the Golestan cohort study, which is prospectively tracking 50,045 people aged 40-75 from northeastern Iran. The study focuses on esophageal cancer due to the region’s high rate of the disease.

Red meat consumption is fairly rare in the region, where residents typically prefer chicken, said study lead author Giulia Collatuzzo, MD, a resident physician in occupational medicine at the University of Bologna, Italy, in an interview. On average, participants reported eating 18.4 grams daily of red meat and 72.1 grams daily of white meat.

The researchers tracked study participants for a median 12-year follow-up, during which 369 developed esophageal cancer and 368 developed gastric cancer. Red meat was only linked to more esophageal cancer in women (hazard ratio, 1.13, 95% confidence interval, 1.00-1.18, for each quintile increase in consumption).

Overall red meat consumption (including red meat, organ meat, and processed meat) was linked to higher rates of gastric cancer (HR, 1.08, 95% CI, 1.00-1.17) for each quartile increase in consumption, as was consumption of the red meat subtype alone (HR, 1.09, 95% CI, 1.00-1.18).

According to Dr. Collatuzzo, the findings suggest that those in the highest quartile of overall red meat consumption may have around a 25% increase in risk, compared with the lowest quartile.

Overall, she said, the study findings aren’t surprising. The lack of a connection between red meat consumption and esophageal cancer may be due to the fact that meat only temporarily transits through the esophagus, she said.

For the liver cancer/liver disease study, researchers examined the medical records of 470,653 subjects in the NIH-AARP Diet and Health Study. They were recruited in 1995-1996 when they were 50-71 years old. Over a median follow-up of 15.5 years, 899 developed liver cancer, and 934 died of chronic liver disease.

The median intakes of vegetables in quintile 5 (highest) and quintile 1 (lowest) were 3.7 cups daily and 1.0 cups daily, respectively, said study lead author Long-Gang Zhao, MS, a graduate student at Harvard University.

After adjusting for possible cofounders, those in the highest quintile of vegetable consumption were a third less likely to develop liver cancer, compared with the lowest quintile (HR, 0.66, 95% CI, 0.53-0.82, P < 0.01). Several types of vegetables appeared to be the strongest cancer fighters: cruciferous (broccoli, cauliflower), lettuce, legumes, and carrots. These kinds of vegetables were also linked to lower rates of chronic liver disease mortality (all P < 0.01), as was total vegetable intake for the top quintile versus the lowest quintile (HR, 0.60, 95% CI, 0.49-0.74, P = < 0.01).

“A one-cup increase (8 oz or 225 g) in vegetable intake was associated with about 20% decreased risk of liver cancer incidence and chronic liver mortality,” Zhao said.

There was no statistically significant link between fruit consumption and liver cancer or chronic liver disease mortality.

The findings provide more insight into diet and liver disease, Zhao said. “Chronic liver disease, which predisposes to liver cancer, is the tenth cause of death worldwide, causing two million deaths each year. It shares some etiological processes with liver cancer. Therefore, examining both chronic liver disease mortality and liver cancer incidence in our study may provide a more general picture for the prevention of liver diseases.”

As for limitations, both studies are based on self-reports about food consumption, which can be unreliable, and the subjects in the fruit/vegetable analysis were mainly of European origin.

The authors of both studies report no relevant disclosures. No funding is reported for either study.

A pair of new studies offers more evidence for the value of vegetables and the risk of red meat on the cancer prevention front. Researchers report that high consumption of vegetables – especially lettuce, legumes, and cruciferous varieties – appears to lower the risk of liver cancer/liver disease. A separate team suggests that high consumption of red meat, organ meats, and processed meats boosts the risk of gastric cancer.

The findings of the latter study “reinforce the idea that avoidance of red meat and processed meat is probably good beyond [the prevention of] colorectal cancer,” said corresponding author and epidemiologist Paolo Boffetta, MD, MPH, of Stony Brook University Cancer Center, New York, in an interview. “The possible carcinogenic effect may extend beyond the colon.”

Both studies were released at the annual meeting of the American Association for Cancer Research.

For the red meat study, researchers examined statistics from the Golestan cohort study, which is prospectively tracking 50,045 people aged 40-75 from northeastern Iran. The study focuses on esophageal cancer due to the region’s high rate of the disease.

Red meat consumption is fairly rare in the region, where residents typically prefer chicken, said study lead author Giulia Collatuzzo, MD, a resident physician in occupational medicine at the University of Bologna, Italy, in an interview. On average, participants reported eating 18.4 grams daily of red meat and 72.1 grams daily of white meat.

The researchers tracked study participants for a median 12-year follow-up, during which 369 developed esophageal cancer and 368 developed gastric cancer. Red meat was only linked to more esophageal cancer in women (hazard ratio, 1.13, 95% confidence interval, 1.00-1.18, for each quintile increase in consumption).

Overall red meat consumption (including red meat, organ meat, and processed meat) was linked to higher rates of gastric cancer (HR, 1.08, 95% CI, 1.00-1.17) for each quartile increase in consumption, as was consumption of the red meat subtype alone (HR, 1.09, 95% CI, 1.00-1.18).

According to Dr. Collatuzzo, the findings suggest that those in the highest quartile of overall red meat consumption may have around a 25% increase in risk, compared with the lowest quartile.

Overall, she said, the study findings aren’t surprising. The lack of a connection between red meat consumption and esophageal cancer may be due to the fact that meat only temporarily transits through the esophagus, she said.

For the liver cancer/liver disease study, researchers examined the medical records of 470,653 subjects in the NIH-AARP Diet and Health Study. They were recruited in 1995-1996 when they were 50-71 years old. Over a median follow-up of 15.5 years, 899 developed liver cancer, and 934 died of chronic liver disease.

The median intakes of vegetables in quintile 5 (highest) and quintile 1 (lowest) were 3.7 cups daily and 1.0 cups daily, respectively, said study lead author Long-Gang Zhao, MS, a graduate student at Harvard University.

After adjusting for possible cofounders, those in the highest quintile of vegetable consumption were a third less likely to develop liver cancer, compared with the lowest quintile (HR, 0.66, 95% CI, 0.53-0.82, P < 0.01). Several types of vegetables appeared to be the strongest cancer fighters: cruciferous (broccoli, cauliflower), lettuce, legumes, and carrots. These kinds of vegetables were also linked to lower rates of chronic liver disease mortality (all P < 0.01), as was total vegetable intake for the top quintile versus the lowest quintile (HR, 0.60, 95% CI, 0.49-0.74, P = < 0.01).

“A one-cup increase (8 oz or 225 g) in vegetable intake was associated with about 20% decreased risk of liver cancer incidence and chronic liver mortality,” Zhao said.

There was no statistically significant link between fruit consumption and liver cancer or chronic liver disease mortality.

The findings provide more insight into diet and liver disease, Zhao said. “Chronic liver disease, which predisposes to liver cancer, is the tenth cause of death worldwide, causing two million deaths each year. It shares some etiological processes with liver cancer. Therefore, examining both chronic liver disease mortality and liver cancer incidence in our study may provide a more general picture for the prevention of liver diseases.”

As for limitations, both studies are based on self-reports about food consumption, which can be unreliable, and the subjects in the fruit/vegetable analysis were mainly of European origin.

The authors of both studies report no relevant disclosures. No funding is reported for either study.

Because they have detected sluggishness in hospital diagnoses, researchers have developed an algorithm to help clinicians determine whether patients have acquired hemophilia A (AHA), a rare bleeding disorder that mainly affects elderly men with medical problems.

“A simple algorithm for unexplained bleeding might be helpful to emergency department physicians and other frontline workers to improve recognition of the disease,” said Amar Kelkar, MD, a fellow at the Dana-Farber Cancer Institute, Boston, and corresponding author of “Time is blood: The impact of diagnostic delays on acquired hemophilia A,” a report that appeared in the journal Cureus.

According to Dr. Kelkar, AHA is an autoimmune disease caused by the formation of autoantibodies against factor VIII (FVIII). “Classically, patients present with various forms of bleeding symptoms, including extensive bruising, spontaneous prolonged or persistent bleeding, and blood in the urine. These symptoms are usually accompanied by a prolonged activated partial thromboplastin time (aPTT) test,” he said in an interview. “While this is a rare diagnosis to be seen in primary, critical, or emergency care, it’s a disease that most hematologists should have seen and managed before.”

For the new study, researchers retrospectively tracked patients with AHA at the OSF Healthcare System in Illinois from 2010 to 2017. They focused on six patients (mean age, 79.5; male = 5). Cancer was considered a cause in four cases, and autoimmune disease in one. The sixth case was idiopathic. Five of the six patients died, with all but one death related to bleeding.

The researchers note that they saw more cases than expected (6 per 2.1 million vs. an estimated incidence of 1.48 per 1 million per year), although they attributed this high incidence to the population being made up of older hospitalized patients. In fact, Dr. Kelkar said, researchers believe this is an undercount reflecting diagnostic misses.

The median time to diagnosis was 14 days, the authors report, reflecting other studies that have also shown delays. Pseudo-thrombosis and preexisting anticoagulant therapy likely contribute to the diagnostic delays, they write.

In their new report, the authors developed an algorithm to speed diagnosis.

“The initial step is the identification of a patient with new, unexplained bleeding,” they write. “In the setting of unexplained bleeding, a detailed clinical history, including medication use, along with a thorough physical examination is critical. Prompt primary laboratory testing should include a complete blood count, a metabolic panel including creatinine and bilirubin, and coagulation testing including aPTT and prothrombin time with international normalized ratio (PT/INR). A resulting isolated aPTT elevation will initiate subsequent steps. Early inpatient hematology consultation is recommended.”

The authors add: “An important point to highlight is that we recommend concurrently ordering an aPTT mixing study and a factor VIII activity (FVIII:C) once a prolonged aPTT is confirmed. This may decrease the time to initiate treatment and improve patient outcomes. If the mixing study result is abnormal with low FVIII:C, hemostatic treatment could be initiated with concurrent confirmatory Bethesda assay or anti-FVIII ELISA, preventing further delay in patient recovery and hopefully reducing potential complications. If there is limited availability of specialty testing or prolonged delays in getting test results, such as for FVIII:C, or an inability to confirm a diagnosis at any stage of the algorithm, transferring the patient to a higher level of care with these laboratory and hematology services should be strongly considered.”

The authors also note that “when the diagnostic delay is greater than 1 month, there will be a significant increase in the days that the patient is required to be on hemostatic therapy, compared to diagnosis before 1 month (23.8 ± 13 vs. 7.6 ± 5.7 days, respectively; P = .003).”

The algorithm is meant to be widely available, Dr. Kelkar said. “That is why we targeted an open-source, general medicine journal like Cureus.”

Jerome Teitel, MD, a hematologist with St. Michael’s Hospital in Toronto, said in an interview that the algorithm “might be a useful guide for initial investigation at community hospitals.”

However, he recommended against emphasizing the use of mixing studies. They are “often ambiguous and just delay ordering the definitive tests (FVIII activity and inhibitor assay), which will need to be done regardless,” he said. “The most important message should be that patients with AHA should be referred to, or at least comanaged with, a hematologist who has specific experience and expertise in the field, and who will likely have access to specialized coagulation tests with short turnaround times.”

Another hematologist, George M. Rodgers III, MD, of the University of Utah, Salt Lake City, said in an interview that the algorithm is appropriate for the evaluation of possible AHA. “Patients with the disorder who present with minor bleeding are not evaluated with high priority by physicians,” he said. “Patients with bleeding and a prolonged PTT should be taken very seriously because AHA patients can develop spontaneous fatal bleeding.”

No study funding is reported. The authors, Dr. Teitel, and Dr. Rodgers report no relevant disclosures.

Because they have detected sluggishness in hospital diagnoses, researchers have developed an algorithm to help clinicians determine whether patients have acquired hemophilia A (AHA), a rare bleeding disorder that mainly affects elderly men with medical problems.

“A simple algorithm for unexplained bleeding might be helpful to emergency department physicians and other frontline workers to improve recognition of the disease,” said Amar Kelkar, MD, a fellow at the Dana-Farber Cancer Institute, Boston, and corresponding author of “Time is blood: The impact of diagnostic delays on acquired hemophilia A,” a report that appeared in the journal Cureus.

According to Dr. Kelkar, AHA is an autoimmune disease caused by the formation of autoantibodies against factor VIII (FVIII). “Classically, patients present with various forms of bleeding symptoms, including extensive bruising, spontaneous prolonged or persistent bleeding, and blood in the urine. These symptoms are usually accompanied by a prolonged activated partial thromboplastin time (aPTT) test,” he said in an interview. “While this is a rare diagnosis to be seen in primary, critical, or emergency care, it’s a disease that most hematologists should have seen and managed before.”

For the new study, researchers retrospectively tracked patients with AHA at the OSF Healthcare System in Illinois from 2010 to 2017. They focused on six patients (mean age, 79.5; male = 5). Cancer was considered a cause in four cases, and autoimmune disease in one. The sixth case was idiopathic. Five of the six patients died, with all but one death related to bleeding.

The researchers note that they saw more cases than expected (6 per 2.1 million vs. an estimated incidence of 1.48 per 1 million per year), although they attributed this high incidence to the population being made up of older hospitalized patients. In fact, Dr. Kelkar said, researchers believe this is an undercount reflecting diagnostic misses.

The median time to diagnosis was 14 days, the authors report, reflecting other studies that have also shown delays. Pseudo-thrombosis and preexisting anticoagulant therapy likely contribute to the diagnostic delays, they write.

In their new report, the authors developed an algorithm to speed diagnosis.

“The initial step is the identification of a patient with new, unexplained bleeding,” they write. “In the setting of unexplained bleeding, a detailed clinical history, including medication use, along with a thorough physical examination is critical. Prompt primary laboratory testing should include a complete blood count, a metabolic panel including creatinine and bilirubin, and coagulation testing including aPTT and prothrombin time with international normalized ratio (PT/INR). A resulting isolated aPTT elevation will initiate subsequent steps. Early inpatient hematology consultation is recommended.”

The authors add: “An important point to highlight is that we recommend concurrently ordering an aPTT mixing study and a factor VIII activity (FVIII:C) once a prolonged aPTT is confirmed. This may decrease the time to initiate treatment and improve patient outcomes. If the mixing study result is abnormal with low FVIII:C, hemostatic treatment could be initiated with concurrent confirmatory Bethesda assay or anti-FVIII ELISA, preventing further delay in patient recovery and hopefully reducing potential complications. If there is limited availability of specialty testing or prolonged delays in getting test results, such as for FVIII:C, or an inability to confirm a diagnosis at any stage of the algorithm, transferring the patient to a higher level of care with these laboratory and hematology services should be strongly considered.”

The authors also note that “when the diagnostic delay is greater than 1 month, there will be a significant increase in the days that the patient is required to be on hemostatic therapy, compared to diagnosis before 1 month (23.8 ± 13 vs. 7.6 ± 5.7 days, respectively; P = .003).”

The algorithm is meant to be widely available, Dr. Kelkar said. “That is why we targeted an open-source, general medicine journal like Cureus.”

Jerome Teitel, MD, a hematologist with St. Michael’s Hospital in Toronto, said in an interview that the algorithm “might be a useful guide for initial investigation at community hospitals.”

However, he recommended against emphasizing the use of mixing studies. They are “often ambiguous and just delay ordering the definitive tests (FVIII activity and inhibitor assay), which will need to be done regardless,” he said. “The most important message should be that patients with AHA should be referred to, or at least comanaged with, a hematologist who has specific experience and expertise in the field, and who will likely have access to specialized coagulation tests with short turnaround times.”

Another hematologist, George M. Rodgers III, MD, of the University of Utah, Salt Lake City, said in an interview that the algorithm is appropriate for the evaluation of possible AHA. “Patients with the disorder who present with minor bleeding are not evaluated with high priority by physicians,” he said. “Patients with bleeding and a prolonged PTT should be taken very seriously because AHA patients can develop spontaneous fatal bleeding.”

No study funding is reported. The authors, Dr. Teitel, and Dr. Rodgers report no relevant disclosures.

Because they have detected sluggishness in hospital diagnoses, researchers have developed an algorithm to help clinicians determine whether patients have acquired hemophilia A (AHA), a rare bleeding disorder that mainly affects elderly men with medical problems.

“A simple algorithm for unexplained bleeding might be helpful to emergency department physicians and other frontline workers to improve recognition of the disease,” said Amar Kelkar, MD, a fellow at the Dana-Farber Cancer Institute, Boston, and corresponding author of “Time is blood: The impact of diagnostic delays on acquired hemophilia A,” a report that appeared in the journal Cureus.

According to Dr. Kelkar, AHA is an autoimmune disease caused by the formation of autoantibodies against factor VIII (FVIII). “Classically, patients present with various forms of bleeding symptoms, including extensive bruising, spontaneous prolonged or persistent bleeding, and blood in the urine. These symptoms are usually accompanied by a prolonged activated partial thromboplastin time (aPTT) test,” he said in an interview. “While this is a rare diagnosis to be seen in primary, critical, or emergency care, it’s a disease that most hematologists should have seen and managed before.”

For the new study, researchers retrospectively tracked patients with AHA at the OSF Healthcare System in Illinois from 2010 to 2017. They focused on six patients (mean age, 79.5; male = 5). Cancer was considered a cause in four cases, and autoimmune disease in one. The sixth case was idiopathic. Five of the six patients died, with all but one death related to bleeding.

The researchers note that they saw more cases than expected (6 per 2.1 million vs. an estimated incidence of 1.48 per 1 million per year), although they attributed this high incidence to the population being made up of older hospitalized patients. In fact, Dr. Kelkar said, researchers believe this is an undercount reflecting diagnostic misses.

The median time to diagnosis was 14 days, the authors report, reflecting other studies that have also shown delays. Pseudo-thrombosis and preexisting anticoagulant therapy likely contribute to the diagnostic delays, they write.

In their new report, the authors developed an algorithm to speed diagnosis.

“The initial step is the identification of a patient with new, unexplained bleeding,” they write. “In the setting of unexplained bleeding, a detailed clinical history, including medication use, along with a thorough physical examination is critical. Prompt primary laboratory testing should include a complete blood count, a metabolic panel including creatinine and bilirubin, and coagulation testing including aPTT and prothrombin time with international normalized ratio (PT/INR). A resulting isolated aPTT elevation will initiate subsequent steps. Early inpatient hematology consultation is recommended.”

The authors add: “An important point to highlight is that we recommend concurrently ordering an aPTT mixing study and a factor VIII activity (FVIII:C) once a prolonged aPTT is confirmed. This may decrease the time to initiate treatment and improve patient outcomes. If the mixing study result is abnormal with low FVIII:C, hemostatic treatment could be initiated with concurrent confirmatory Bethesda assay or anti-FVIII ELISA, preventing further delay in patient recovery and hopefully reducing potential complications. If there is limited availability of specialty testing or prolonged delays in getting test results, such as for FVIII:C, or an inability to confirm a diagnosis at any stage of the algorithm, transferring the patient to a higher level of care with these laboratory and hematology services should be strongly considered.”

The authors also note that “when the diagnostic delay is greater than 1 month, there will be a significant increase in the days that the patient is required to be on hemostatic therapy, compared to diagnosis before 1 month (23.8 ± 13 vs. 7.6 ± 5.7 days, respectively; P = .003).”

The algorithm is meant to be widely available, Dr. Kelkar said. “That is why we targeted an open-source, general medicine journal like Cureus.”

Jerome Teitel, MD, a hematologist with St. Michael’s Hospital in Toronto, said in an interview that the algorithm “might be a useful guide for initial investigation at community hospitals.”

However, he recommended against emphasizing the use of mixing studies. They are “often ambiguous and just delay ordering the definitive tests (FVIII activity and inhibitor assay), which will need to be done regardless,” he said. “The most important message should be that patients with AHA should be referred to, or at least comanaged with, a hematologist who has specific experience and expertise in the field, and who will likely have access to specialized coagulation tests with short turnaround times.”

Another hematologist, George M. Rodgers III, MD, of the University of Utah, Salt Lake City, said in an interview that the algorithm is appropriate for the evaluation of possible AHA. “Patients with the disorder who present with minor bleeding are not evaluated with high priority by physicians,” he said. “Patients with bleeding and a prolonged PTT should be taken very seriously because AHA patients can develop spontaneous fatal bleeding.”

No study funding is reported. The authors, Dr. Teitel, and Dr. Rodgers report no relevant disclosures.

More than one in seven patients with breast cancer saw their financial status deteriorate within the first years after their diagnosis, a new study found. Factors like disease severity and treatment type didn’t seem to have an impact on financial status.

The findings, presented at the annual meeting of the American Association for Cancer Research, were unexpected. “We were surprised that we did not find that patients who received more aggressive therapies were more likely to experience worsening financial concerns,” said corresponding author and medical oncologist Kathryn J. Ruddy, MD, of the Mayo Clinici in Rochester, Minn.

The study was undertaken to understand the financial stress facing patients with breast cancer. The question was whether individual or disease factors, or both, were at play.

The study is based on results from the Mayo Clinic Breast Disease Registry, a prospective cohort of patient who were at Mayo Clinic Rochester. Participants answered questions about their finances at baseline and then again at annual follow-ups.

Researchers examined survey findings from 1,957 patients (mean age 58.5, 99.1% female, 95.4% White, 54.9% bachelor degree or higher) who answered questions at least twice from 2015-2020. The average time between diagnosis and the most recent follow-up was 25.6 months.

Of the 1,957 patients, 357 (18.2%) said their finances deteriorated as measured by a 1 point or higher decline on a 10-point scale.

There was no statistically significant link between deteriorating finances and age, race, employment status, stage of cancer at diagnosis, type of cancer, or treatment type. There was a slight link between deteriorating finances and reporting that they were in the category of “pay bills, no money for special things” near diagnosis.

Other research has suggested that breast cancer may not disrupt finances to a large extent, at least early on. Earlier in 2022, Stanford (Calif.) University researchers reported the results of a survey of 273 breast and gynecologic cancer patients who were surveyed about their finances at a mean of 3.4 years after diagnosis. While one-third said their cancer caused career changes, the study described overall financial toxicity as mild.

In regard to limitations, the subject population of the new study is overwhelmingly White, and the finances were self-reported by those who participated in the survey. Also, “because our participants were recruited at a tertiary medical center, there were relatively financially secure at baseline,” Dr. Ruddy said. “More financial hardship would be expected in a more financially diverse population.”

In an interview, Cathy Bradley, PhD, associate dean for research at the University of Colorado at Denver and deputy director of the University of Colorado Cancer Center, both in Aurora, praised the study as “an important start toward assessing financial burden in the clinic. Having more universal assessments in the clinic would remove stigma.”

She cautioned about interpreting a seemingly low number of patients whose financial situation worsened. “This was for a single site where there is a high rate of health insurance either through Medicare or Medicaid. There may be some selection bias as well given that Mayo may attract a wealthier patient population. Most women completed treatment and may not have been on long-term therapies.”

Moving forward, Dr. Ruddy said, “we hope to study cost of oncologic care in more geographically and financially diverse populations with breast cancer and other cancers.”

The study was funded by the Breast Cancer Research Foundation and National Cancer Institute. The study authors and Dr. Ruddy report no relevant disclosures.

More than one in seven patients with breast cancer saw their financial status deteriorate within the first years after their diagnosis, a new study found. Factors like disease severity and treatment type didn’t seem to have an impact on financial status.

The findings, presented at the annual meeting of the American Association for Cancer Research, were unexpected. “We were surprised that we did not find that patients who received more aggressive therapies were more likely to experience worsening financial concerns,” said corresponding author and medical oncologist Kathryn J. Ruddy, MD, of the Mayo Clinici in Rochester, Minn.

The study was undertaken to understand the financial stress facing patients with breast cancer. The question was whether individual or disease factors, or both, were at play.

The study is based on results from the Mayo Clinic Breast Disease Registry, a prospective cohort of patient who were at Mayo Clinic Rochester. Participants answered questions about their finances at baseline and then again at annual follow-ups.

Researchers examined survey findings from 1,957 patients (mean age 58.5, 99.1% female, 95.4% White, 54.9% bachelor degree or higher) who answered questions at least twice from 2015-2020. The average time between diagnosis and the most recent follow-up was 25.6 months.

Of the 1,957 patients, 357 (18.2%) said their finances deteriorated as measured by a 1 point or higher decline on a 10-point scale.

There was no statistically significant link between deteriorating finances and age, race, employment status, stage of cancer at diagnosis, type of cancer, or treatment type. There was a slight link between deteriorating finances and reporting that they were in the category of “pay bills, no money for special things” near diagnosis.

Other research has suggested that breast cancer may not disrupt finances to a large extent, at least early on. Earlier in 2022, Stanford (Calif.) University researchers reported the results of a survey of 273 breast and gynecologic cancer patients who were surveyed about their finances at a mean of 3.4 years after diagnosis. While one-third said their cancer caused career changes, the study described overall financial toxicity as mild.

In regard to limitations, the subject population of the new study is overwhelmingly White, and the finances were self-reported by those who participated in the survey. Also, “because our participants were recruited at a tertiary medical center, there were relatively financially secure at baseline,” Dr. Ruddy said. “More financial hardship would be expected in a more financially diverse population.”

In an interview, Cathy Bradley, PhD, associate dean for research at the University of Colorado at Denver and deputy director of the University of Colorado Cancer Center, both in Aurora, praised the study as “an important start toward assessing financial burden in the clinic. Having more universal assessments in the clinic would remove stigma.”

She cautioned about interpreting a seemingly low number of patients whose financial situation worsened. “This was for a single site where there is a high rate of health insurance either through Medicare or Medicaid. There may be some selection bias as well given that Mayo may attract a wealthier patient population. Most women completed treatment and may not have been on long-term therapies.”

Moving forward, Dr. Ruddy said, “we hope to study cost of oncologic care in more geographically and financially diverse populations with breast cancer and other cancers.”

The study was funded by the Breast Cancer Research Foundation and National Cancer Institute. The study authors and Dr. Ruddy report no relevant disclosures.

More than one in seven patients with breast cancer saw their financial status deteriorate within the first years after their diagnosis, a new study found. Factors like disease severity and treatment type didn’t seem to have an impact on financial status.

The findings, presented at the annual meeting of the American Association for Cancer Research, were unexpected. “We were surprised that we did not find that patients who received more aggressive therapies were more likely to experience worsening financial concerns,” said corresponding author and medical oncologist Kathryn J. Ruddy, MD, of the Mayo Clinici in Rochester, Minn.

The study was undertaken to understand the financial stress facing patients with breast cancer. The question was whether individual or disease factors, or both, were at play.

The study is based on results from the Mayo Clinic Breast Disease Registry, a prospective cohort of patient who were at Mayo Clinic Rochester. Participants answered questions about their finances at baseline and then again at annual follow-ups.

Researchers examined survey findings from 1,957 patients (mean age 58.5, 99.1% female, 95.4% White, 54.9% bachelor degree or higher) who answered questions at least twice from 2015-2020. The average time between diagnosis and the most recent follow-up was 25.6 months.

Of the 1,957 patients, 357 (18.2%) said their finances deteriorated as measured by a 1 point or higher decline on a 10-point scale.

There was no statistically significant link between deteriorating finances and age, race, employment status, stage of cancer at diagnosis, type of cancer, or treatment type. There was a slight link between deteriorating finances and reporting that they were in the category of “pay bills, no money for special things” near diagnosis.

Other research has suggested that breast cancer may not disrupt finances to a large extent, at least early on. Earlier in 2022, Stanford (Calif.) University researchers reported the results of a survey of 273 breast and gynecologic cancer patients who were surveyed about their finances at a mean of 3.4 years after diagnosis. While one-third said their cancer caused career changes, the study described overall financial toxicity as mild.

In regard to limitations, the subject population of the new study is overwhelmingly White, and the finances were self-reported by those who participated in the survey. Also, “because our participants were recruited at a tertiary medical center, there were relatively financially secure at baseline,” Dr. Ruddy said. “More financial hardship would be expected in a more financially diverse population.”

In an interview, Cathy Bradley, PhD, associate dean for research at the University of Colorado at Denver and deputy director of the University of Colorado Cancer Center, both in Aurora, praised the study as “an important start toward assessing financial burden in the clinic. Having more universal assessments in the clinic would remove stigma.”

She cautioned about interpreting a seemingly low number of patients whose financial situation worsened. “This was for a single site where there is a high rate of health insurance either through Medicare or Medicaid. There may be some selection bias as well given that Mayo may attract a wealthier patient population. Most women completed treatment and may not have been on long-term therapies.”

Moving forward, Dr. Ruddy said, “we hope to study cost of oncologic care in more geographically and financially diverse populations with breast cancer and other cancers.”

The study was funded by the Breast Cancer Research Foundation and National Cancer Institute. The study authors and Dr. Ruddy report no relevant disclosures.

Researchers have demonstrated that diet supplementation with high-dose omega-3 fatty acids can lead to a decrease in potentially dangerous FOXA1 in benign breast tissue, potentially pointing the way toward the use of the pioneer transcription factor as a helpful biomarker for breast cancer researchers.

The findings were released at the annual meeting of the American Association for Cancer Research.

In the study, researchers who were led by Bruce F. Kimler, PhD, a radiation biologist and breast cancer researcher at the University of Kansas Medical Center, Kansas City, examined benign breast tissue cells aspirated from 12 women (mean age, 53 years; 7 on low-dose hormone replacement) before and after 6 months of high-dose omega-3 fatty acid supplementation. After the supplementation, FOXA1 positive cells fell in 11 of 12 women (P = .019). “There was a robust linear relationship between stain positivity for FOXA1 and AGR2,” the researchers reported (P < .001).

Increased FOXA1 activity along with GRHL2) transcription factor can boost endocrine resistance, while omega-3 fatty acids can reduce it.

In an interview, Robert S. Chapkin, PhD, the Allen Endowed Chair in Nutrition and Chronic Disease Prevention at Texas A&M University, College Station, said it’s important to examine the value of omega-3 fatty acid supplementation, and the understanding of biomarkers is crucial. “Omega 3 fatty acids are pleiotropic, dose dependent, and likely impact multiple signaling mechanisms in select cells types and cancer contexts. The key is to dissect out the highest impact targets and pursue them in the context of preclinical and clinical studies.”

However, he said, “in many cases, the lack of a mechanistic understanding detracts from the merit of the work.”

Studies like this are useful in the development of clinical trials to test the value of high-dose omega-3 fatty acids in breast cancer prevention trials, said Carol Fabian, MD, a breast medical oncologist with the University of Kansas Medical Center, and the study’s first author.

“They help us understand both what dose will be needed and biomarkers that will likely be helpful in predicting response. Early-phase trials with biomarker modulation as a primary endpoint are generally necessary to make sure you have the right dose for the target population prior to committing to a long-term cancer incidence study involving thousands of women and tens of millions of dollars,” she said.

What’s next? “This work was done on reserved specimens from a prior pilot trial,” Dr. Fabian said. “We need a placebo-controlled study to know for sure that omega-3 FA in a dose of about 3.2g daily, or about 2% of calories, modulates FOXA1 and/or AGR2 in postmenopausal women.”

Previously, she said, the researchers “found that high dose omega-3 administered to overweight peri- and postmenopausal high-risk women undergoing a 6-month weight loss intervention increased the number of systemic risk biomarkers which were favorably modulated compared to placebo despite the same median weight loss in each group [–10%],” Dr. Fabian said. “We want to duplicate that finding in a larger study as well as determine if omega-3 fatty acids can block tamoxifen-induced increases in AGR2 associated with endocrine resistance.”

The study was funded by the Breast Cancer Research Foundation, the Morris Family Foundation, and the University of Kansas Cancer Center. The authors and Chapkin report no relevant disclosures.

Researchers have demonstrated that diet supplementation with high-dose omega-3 fatty acids can lead to a decrease in potentially dangerous FOXA1 in benign breast tissue, potentially pointing the way toward the use of the pioneer transcription factor as a helpful biomarker for breast cancer researchers.

The findings were released at the annual meeting of the American Association for Cancer Research.

In the study, researchers who were led by Bruce F. Kimler, PhD, a radiation biologist and breast cancer researcher at the University of Kansas Medical Center, Kansas City, examined benign breast tissue cells aspirated from 12 women (mean age, 53 years; 7 on low-dose hormone replacement) before and after 6 months of high-dose omega-3 fatty acid supplementation. After the supplementation, FOXA1 positive cells fell in 11 of 12 women (P = .019). “There was a robust linear relationship between stain positivity for FOXA1 and AGR2,” the researchers reported (P < .001).

Increased FOXA1 activity along with GRHL2) transcription factor can boost endocrine resistance, while omega-3 fatty acids can reduce it.

In an interview, Robert S. Chapkin, PhD, the Allen Endowed Chair in Nutrition and Chronic Disease Prevention at Texas A&M University, College Station, said it’s important to examine the value of omega-3 fatty acid supplementation, and the understanding of biomarkers is crucial. “Omega 3 fatty acids are pleiotropic, dose dependent, and likely impact multiple signaling mechanisms in select cells types and cancer contexts. The key is to dissect out the highest impact targets and pursue them in the context of preclinical and clinical studies.”

However, he said, “in many cases, the lack of a mechanistic understanding detracts from the merit of the work.”

Studies like this are useful in the development of clinical trials to test the value of high-dose omega-3 fatty acids in breast cancer prevention trials, said Carol Fabian, MD, a breast medical oncologist with the University of Kansas Medical Center, and the study’s first author.

“They help us understand both what dose will be needed and biomarkers that will likely be helpful in predicting response. Early-phase trials with biomarker modulation as a primary endpoint are generally necessary to make sure you have the right dose for the target population prior to committing to a long-term cancer incidence study involving thousands of women and tens of millions of dollars,” she said.

What’s next? “This work was done on reserved specimens from a prior pilot trial,” Dr. Fabian said. “We need a placebo-controlled study to know for sure that omega-3 FA in a dose of about 3.2g daily, or about 2% of calories, modulates FOXA1 and/or AGR2 in postmenopausal women.”

Previously, she said, the researchers “found that high dose omega-3 administered to overweight peri- and postmenopausal high-risk women undergoing a 6-month weight loss intervention increased the number of systemic risk biomarkers which were favorably modulated compared to placebo despite the same median weight loss in each group [–10%],” Dr. Fabian said. “We want to duplicate that finding in a larger study as well as determine if omega-3 fatty acids can block tamoxifen-induced increases in AGR2 associated with endocrine resistance.”

The study was funded by the Breast Cancer Research Foundation, the Morris Family Foundation, and the University of Kansas Cancer Center. The authors and Chapkin report no relevant disclosures.

Researchers have demonstrated that diet supplementation with high-dose omega-3 fatty acids can lead to a decrease in potentially dangerous FOXA1 in benign breast tissue, potentially pointing the way toward the use of the pioneer transcription factor as a helpful biomarker for breast cancer researchers.

The findings were released at the annual meeting of the American Association for Cancer Research.

In the study, researchers who were led by Bruce F. Kimler, PhD, a radiation biologist and breast cancer researcher at the University of Kansas Medical Center, Kansas City, examined benign breast tissue cells aspirated from 12 women (mean age, 53 years; 7 on low-dose hormone replacement) before and after 6 months of high-dose omega-3 fatty acid supplementation. After the supplementation, FOXA1 positive cells fell in 11 of 12 women (P = .019). “There was a robust linear relationship between stain positivity for FOXA1 and AGR2,” the researchers reported (P < .001).

Increased FOXA1 activity along with GRHL2) transcription factor can boost endocrine resistance, while omega-3 fatty acids can reduce it.

In an interview, Robert S. Chapkin, PhD, the Allen Endowed Chair in Nutrition and Chronic Disease Prevention at Texas A&M University, College Station, said it’s important to examine the value of omega-3 fatty acid supplementation, and the understanding of biomarkers is crucial. “Omega 3 fatty acids are pleiotropic, dose dependent, and likely impact multiple signaling mechanisms in select cells types and cancer contexts. The key is to dissect out the highest impact targets and pursue them in the context of preclinical and clinical studies.”

However, he said, “in many cases, the lack of a mechanistic understanding detracts from the merit of the work.”

Studies like this are useful in the development of clinical trials to test the value of high-dose omega-3 fatty acids in breast cancer prevention trials, said Carol Fabian, MD, a breast medical oncologist with the University of Kansas Medical Center, and the study’s first author.

“They help us understand both what dose will be needed and biomarkers that will likely be helpful in predicting response. Early-phase trials with biomarker modulation as a primary endpoint are generally necessary to make sure you have the right dose for the target population prior to committing to a long-term cancer incidence study involving thousands of women and tens of millions of dollars,” she said.

What’s next? “This work was done on reserved specimens from a prior pilot trial,” Dr. Fabian said. “We need a placebo-controlled study to know for sure that omega-3 FA in a dose of about 3.2g daily, or about 2% of calories, modulates FOXA1 and/or AGR2 in postmenopausal women.”

Previously, she said, the researchers “found that high dose omega-3 administered to overweight peri- and postmenopausal high-risk women undergoing a 6-month weight loss intervention increased the number of systemic risk biomarkers which were favorably modulated compared to placebo despite the same median weight loss in each group [–10%],” Dr. Fabian said. “We want to duplicate that finding in a larger study as well as determine if omega-3 fatty acids can block tamoxifen-induced increases in AGR2 associated with endocrine resistance.”

The study was funded by the Breast Cancer Research Foundation, the Morris Family Foundation, and the University of Kansas Cancer Center. The authors and Chapkin report no relevant disclosures.

New research suggests that chemotherapy treatments for recurrent high-grade gliomas indicated by an assay-guided tool called ChemoID can boost median survival, compared with physician choice.

The randomized, phase 3 trial results were presented at the annual meeting of the American Association for Cancer Research.

Over a median follow-up of 9 months, median overall survival in the ChemoID group was 12.5 months (95% confidence interval, 10.2-14.7), compared with 9 months (95% CI, 4.2-13.8) in the group whose treatments were chosen by physicians (P = .010).

“While the prognosis is very dismal, we’re still providing a 3.5-month benefit in the guided arm versus physician choice,” said study coauthor Jagan Valluri, PhD, professor of cellular biology and integrative medicine at Marshall University, Huntington, W. Va.

As Dr. Valluri noted, patients with recurrent high-grade gliomas typically have failed radiation and are left with poor prognoses. Fewer than one in four patients respond to chemotherapy at this point, he said, and the response is inconsistent from patient to patient.

“We developed ChemoID since cancer is very unique,” he said, “and any kind of chemotherapy should be tailored to each individual patient on a case-by-case basis.”

The ChemoID tool, a proprietary assay, tests the response of patient cells to various chemotherapy treatments. A test costs $3,500, and some insurers cover it, Dr. Valluri said.

For the new study, researchers randomly assigned 50 patients with grade III/IV recurrent glioma to be treated with chemotherapy chosen by physicians or chemotherapy recommended by the ChemoID tool.

Risk of death in the ChemoID group was lower than in the physician-guided group (hazard ratio, 0.44; 95% CI, 0.24-0.81; P = .008), and median progression-free survival was higher in the ChemoID group (10.1 months vs. 3.5 months; 95% CI, 4.8-15.4 vs. 1.9-5.1; HR, 0.25; 95% CI, 0.14-0.44; P < .001).

“We want the treating physician to have actionable tools in front of them before they treat the patient,” Dr. Valluri said. “We want this assay to become mainstream and part of the standard care workup.”

The study is funded by Cordgenics, where Dr. Valluri serves as chief operating officer.

New research suggests that chemotherapy treatments for recurrent high-grade gliomas indicated by an assay-guided tool called ChemoID can boost median survival, compared with physician choice.

The randomized, phase 3 trial results were presented at the annual meeting of the American Association for Cancer Research.

Over a median follow-up of 9 months, median overall survival in the ChemoID group was 12.5 months (95% confidence interval, 10.2-14.7), compared with 9 months (95% CI, 4.2-13.8) in the group whose treatments were chosen by physicians (P = .010).

“While the prognosis is very dismal, we’re still providing a 3.5-month benefit in the guided arm versus physician choice,” said study coauthor Jagan Valluri, PhD, professor of cellular biology and integrative medicine at Marshall University, Huntington, W. Va.

As Dr. Valluri noted, patients with recurrent high-grade gliomas typically have failed radiation and are left with poor prognoses. Fewer than one in four patients respond to chemotherapy at this point, he said, and the response is inconsistent from patient to patient.

“We developed ChemoID since cancer is very unique,” he said, “and any kind of chemotherapy should be tailored to each individual patient on a case-by-case basis.”

The ChemoID tool, a proprietary assay, tests the response of patient cells to various chemotherapy treatments. A test costs $3,500, and some insurers cover it, Dr. Valluri said.

For the new study, researchers randomly assigned 50 patients with grade III/IV recurrent glioma to be treated with chemotherapy chosen by physicians or chemotherapy recommended by the ChemoID tool.

Risk of death in the ChemoID group was lower than in the physician-guided group (hazard ratio, 0.44; 95% CI, 0.24-0.81; P = .008), and median progression-free survival was higher in the ChemoID group (10.1 months vs. 3.5 months; 95% CI, 4.8-15.4 vs. 1.9-5.1; HR, 0.25; 95% CI, 0.14-0.44; P < .001).

“We want the treating physician to have actionable tools in front of them before they treat the patient,” Dr. Valluri said. “We want this assay to become mainstream and part of the standard care workup.”

The study is funded by Cordgenics, where Dr. Valluri serves as chief operating officer.

New research suggests that chemotherapy treatments for recurrent high-grade gliomas indicated by an assay-guided tool called ChemoID can boost median survival, compared with physician choice.

The randomized, phase 3 trial results were presented at the annual meeting of the American Association for Cancer Research.

Over a median follow-up of 9 months, median overall survival in the ChemoID group was 12.5 months (95% confidence interval, 10.2-14.7), compared with 9 months (95% CI, 4.2-13.8) in the group whose treatments were chosen by physicians (P = .010).

“While the prognosis is very dismal, we’re still providing a 3.5-month benefit in the guided arm versus physician choice,” said study coauthor Jagan Valluri, PhD, professor of cellular biology and integrative medicine at Marshall University, Huntington, W. Va.

As Dr. Valluri noted, patients with recurrent high-grade gliomas typically have failed radiation and are left with poor prognoses. Fewer than one in four patients respond to chemotherapy at this point, he said, and the response is inconsistent from patient to patient.

“We developed ChemoID since cancer is very unique,” he said, “and any kind of chemotherapy should be tailored to each individual patient on a case-by-case basis.”

The ChemoID tool, a proprietary assay, tests the response of patient cells to various chemotherapy treatments. A test costs $3,500, and some insurers cover it, Dr. Valluri said.

For the new study, researchers randomly assigned 50 patients with grade III/IV recurrent glioma to be treated with chemotherapy chosen by physicians or chemotherapy recommended by the ChemoID tool.

Risk of death in the ChemoID group was lower than in the physician-guided group (hazard ratio, 0.44; 95% CI, 0.24-0.81; P = .008), and median progression-free survival was higher in the ChemoID group (10.1 months vs. 3.5 months; 95% CI, 4.8-15.4 vs. 1.9-5.1; HR, 0.25; 95% CI, 0.14-0.44; P < .001).

“We want the treating physician to have actionable tools in front of them before they treat the patient,” Dr. Valluri said. “We want this assay to become mainstream and part of the standard care workup.”

The study is funded by Cordgenics, where Dr. Valluri serves as chief operating officer.

New research suggests that the casirivimab-imdevimab monoclonal antibody treatment for COVID-19 could have been delivered via injection instead of intravenously. There was no statistically significant difference in 28-day hospitalization or death in those treated intravenously and via subcutaneous injection.

The findings, published in JAMA Network Open, aren’t directly relevant at the moment, since the casirivimab-imdevimab treatment was abandoned when it failed to work during the Omicron outbreak. However, they point toward the importance of studying multiple routes of administration, said study lead author and pharmacist Erin K. McCreary, PharmD, of the University of Pittsburgh, in an interview.

“It would be beneficial for all future monoclonal antibodies for COVID-19 to be studied subcutaneously or intramuscularly, if possible, since that’s logistically easier than IV in the outpatient setting,” she said.

According to Dr. McCreary, an outpatient casirivimab-imdevimab treatment was used from 2020 to 2022 to treat higher-risk patients with mild to moderate COVID-19. The treatment was typically given intravenously as recommended by the federal government’s Emergency Use Authorization, she said. Clinical trials of the treatment, according to the study, allowed only IV administration.

“However, during the Delta surge, we were faced with so many patient referrals for treatment and staffing shortages that we couldn’t accommodate every patient unless we switched to [the] subcutaneous route,” Dr. McCreary said. This approach shortened appointment times by 30 minutes vs. infusion, she said.

There are many benefits to subcutaneous administration versus IV, Dr. McCreary said. “You don’t need to start an intravenous line, so you avoid the line kit and the nursing time needed for that. You draw up the drug directly into syringes and inject under the skin, so you avoid the need for a fluid bag to mix the drug in and run intravenously,” she said. “The appointment times are shorter, so you can accommodate more patients per day. Pharmacy interns can give subcutaneous injections, so you avoid the need for a nurse trained in placing intravenous lines.”

The researchers prospectively assigned 1,959 matched adults with mild to moderate COVID-19 to subcutaneous or intravenous treatment. Of 969 patients who received the subcutaneous treatment (mean age, 53.8; 56.4% women), the 28-day rate of hospitalization or death was 3.4%. Of 1,216 patients who received intravenous treatment (mean age, 54.3; 54.4% women), the rate was 1.7%. The difference was not statistically significant (P = .16).

Among 1,306 nontreated controls, 7.0% were hospitalized or died within 28 days (risk ratio = 0.48 vs. subcutaneous treatment group; 95% confidence interval, 0.30-0.80; P = .002).

“We did not find any patients where IV is a must,” Dr. McCreary said. “However, our study wasn’t powered to see a difference in certain subgroups.”

In an interview, University of Toronto internal medicine and pharmacology/toxicology physician Peter Wu, MD, said he agrees that the study has value because it emphasizes the importance of testing whether monoclonal antibodies can be administered in ways other than intravenously.

However, in the larger picture, he said, this may be irrelevant since it’s clear that anti-spike treatments are not holding up against COVID-19 variants.

No study funding is reported. Some study authors reported disclosures outside the submitted work. Dr. Wu has disclosed no relevant financial relationships.

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

New research suggests that the casirivimab-imdevimab monoclonal antibody treatment for COVID-19 could have been delivered via injection instead of intravenously. There was no statistically significant difference in 28-day hospitalization or death in those treated intravenously and via subcutaneous injection.

The findings, published in JAMA Network Open, aren’t directly relevant at the moment, since the casirivimab-imdevimab treatment was abandoned when it failed to work during the Omicron outbreak. However, they point toward the importance of studying multiple routes of administration, said study lead author and pharmacist Erin K. McCreary, PharmD, of the University of Pittsburgh, in an interview.

“It would be beneficial for all future monoclonal antibodies for COVID-19 to be studied subcutaneously or intramuscularly, if possible, since that’s logistically easier than IV in the outpatient setting,” she said.

According to Dr. McCreary, an outpatient casirivimab-imdevimab treatment was used from 2020 to 2022 to treat higher-risk patients with mild to moderate COVID-19. The treatment was typically given intravenously as recommended by the federal government’s Emergency Use Authorization, she said. Clinical trials of the treatment, according to the study, allowed only IV administration.

“However, during the Delta surge, we were faced with so many patient referrals for treatment and staffing shortages that we couldn’t accommodate every patient unless we switched to [the] subcutaneous route,” Dr. McCreary said. This approach shortened appointment times by 30 minutes vs. infusion, she said.

There are many benefits to subcutaneous administration versus IV, Dr. McCreary said. “You don’t need to start an intravenous line, so you avoid the line kit and the nursing time needed for that. You draw up the drug directly into syringes and inject under the skin, so you avoid the need for a fluid bag to mix the drug in and run intravenously,” she said. “The appointment times are shorter, so you can accommodate more patients per day. Pharmacy interns can give subcutaneous injections, so you avoid the need for a nurse trained in placing intravenous lines.”

The researchers prospectively assigned 1,959 matched adults with mild to moderate COVID-19 to subcutaneous or intravenous treatment. Of 969 patients who received the subcutaneous treatment (mean age, 53.8; 56.4% women), the 28-day rate of hospitalization or death was 3.4%. Of 1,216 patients who received intravenous treatment (mean age, 54.3; 54.4% women), the rate was 1.7%. The difference was not statistically significant (P = .16).

Among 1,306 nontreated controls, 7.0% were hospitalized or died within 28 days (risk ratio = 0.48 vs. subcutaneous treatment group; 95% confidence interval, 0.30-0.80; P = .002).

“We did not find any patients where IV is a must,” Dr. McCreary said. “However, our study wasn’t powered to see a difference in certain subgroups.”

In an interview, University of Toronto internal medicine and pharmacology/toxicology physician Peter Wu, MD, said he agrees that the study has value because it emphasizes the importance of testing whether monoclonal antibodies can be administered in ways other than intravenously.

However, in the larger picture, he said, this may be irrelevant since it’s clear that anti-spike treatments are not holding up against COVID-19 variants.

No study funding is reported. Some study authors reported disclosures outside the submitted work. Dr. Wu has disclosed no relevant financial relationships.

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

New research suggests that the casirivimab-imdevimab monoclonal antibody treatment for COVID-19 could have been delivered via injection instead of intravenously. There was no statistically significant difference in 28-day hospitalization or death in those treated intravenously and via subcutaneous injection.

The findings, published in JAMA Network Open, aren’t directly relevant at the moment, since the casirivimab-imdevimab treatment was abandoned when it failed to work during the Omicron outbreak. However, they point toward the importance of studying multiple routes of administration, said study lead author and pharmacist Erin K. McCreary, PharmD, of the University of Pittsburgh, in an interview.

“It would be beneficial for all future monoclonal antibodies for COVID-19 to be studied subcutaneously or intramuscularly, if possible, since that’s logistically easier than IV in the outpatient setting,” she said.

According to Dr. McCreary, an outpatient casirivimab-imdevimab treatment was used from 2020 to 2022 to treat higher-risk patients with mild to moderate COVID-19. The treatment was typically given intravenously as recommended by the federal government’s Emergency Use Authorization, she said. Clinical trials of the treatment, according to the study, allowed only IV administration.

“However, during the Delta surge, we were faced with so many patient referrals for treatment and staffing shortages that we couldn’t accommodate every patient unless we switched to [the] subcutaneous route,” Dr. McCreary said. This approach shortened appointment times by 30 minutes vs. infusion, she said.

There are many benefits to subcutaneous administration versus IV, Dr. McCreary said. “You don’t need to start an intravenous line, so you avoid the line kit and the nursing time needed for that. You draw up the drug directly into syringes and inject under the skin, so you avoid the need for a fluid bag to mix the drug in and run intravenously,” she said. “The appointment times are shorter, so you can accommodate more patients per day. Pharmacy interns can give subcutaneous injections, so you avoid the need for a nurse trained in placing intravenous lines.”

The researchers prospectively assigned 1,959 matched adults with mild to moderate COVID-19 to subcutaneous or intravenous treatment. Of 969 patients who received the subcutaneous treatment (mean age, 53.8; 56.4% women), the 28-day rate of hospitalization or death was 3.4%. Of 1,216 patients who received intravenous treatment (mean age, 54.3; 54.4% women), the rate was 1.7%. The difference was not statistically significant (P = .16).

Among 1,306 nontreated controls, 7.0% were hospitalized or died within 28 days (risk ratio = 0.48 vs. subcutaneous treatment group; 95% confidence interval, 0.30-0.80; P = .002).

“We did not find any patients where IV is a must,” Dr. McCreary said. “However, our study wasn’t powered to see a difference in certain subgroups.”

In an interview, University of Toronto internal medicine and pharmacology/toxicology physician Peter Wu, MD, said he agrees that the study has value because it emphasizes the importance of testing whether monoclonal antibodies can be administered in ways other than intravenously.

However, in the larger picture, he said, this may be irrelevant since it’s clear that anti-spike treatments are not holding up against COVID-19 variants.

No study funding is reported. Some study authors reported disclosures outside the submitted work. Dr. Wu has disclosed no relevant financial relationships.

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

There’s a strong likelihood that the antidepressant fluvoxamine (Luvox) may moderately lower rates of hospitalization caused by COVID-19 in unvaccinated patients, a new systematic review and meta-analysis has found. But outside experts differ over whether the evidence from just three studies is strong enough to warrant adding the drug to the COVID-19 armamentarium.

The report, published online in JAMA Network Open, looked at three studies and estimated that the drug could reduce the relative risk of hospitalization by around 25% (likelihood of moderate effect, 81.6%-91.8%), depending on the type of analysis used.

“This research might be valuable, but the jury remains out until several other adequately powered and designed trials are completed,” said infectious disease specialist Carl J. Fichtenbaum, MD, of the University of Cincinnati, who’s familiar with the findings. “I’m not sure how useful this is given we have several antiviral agents available. Why would we choose this over Paxlovid, remdesivir, or molnupiravir?”

According to Dr. Fichtenbaum, researchers began focusing on fluvoxamine after case reports about patients improving while on the medication. This led to further interest, he said, boosted by the drug’s known ability to dampen the immune system.

A Silicon Valley investor and antivaccine activist named Steve Kirsch has been pushing the drug along with the debunked treatment hydroxychloroquine. He’s accused the government of a cover-up of fluvoxamine’s worth, according to MIT Technology Review, and he wrote a commentary that referred to the drug as “the fast, easy, safe, simple, low-cost solution to COVID that works 100% of the time that nobody wants to talk about.”

For the new analysis, researchers examined three randomized clinical trials with a total of 2,196 participants. The most extensive trial, the TOGETHER study in Brazil (n = 1,497), focused on an unusual outcome: It linked the drug to a 32% reduction in relative risk of patients with COVID-19 being hospitalized in an ED for fewer than 6 hours or transferred to a tertiary hospital because of the disease.

Another study, the STOP COVID 2 trial in the United States and Canada (n = 547), was stopped because too few patients could be recruited to provide useful results. The initial phase of this trial, STOP COVID 1 (n = 152), was also included in the analysis.

All participants in the three studies were unvaccinated. Their median age was 46-50 years, 55%-72% were women, and 44%-56% were obese. Most were multiracial due to the high number of participants from Brazil.

“In the Bayesian analyses, the pooled risk ratio in favor of fluvoxamine was 0.78 (95% confidence interval, 0.58-1.08) for the weakly neutral prior and 0.73 (95% CI, 0.53-1.01) for the moderately optimistic prior,” the researchers reported, referring to a reduction in risk of hospitalization. “In the frequentist meta-analysis, the pooled risk ratio in favor of fluvoxamine was 0.75 (95% CI, 0.58-0.97; I², 0.2%).”

Two of the authors of the new analysis were also coauthors of the TOGETHER trial and both STOP COVID trials.

Corresponding author Emily G. McDonald, MD, division of experimental medicine at McGill University, Montreal, said in an interview that the findings show fluvoxamine “very likely reduces hospitalization in high-risk outpatient adults with COVID-19. This effect varies depending on your baseline risk of developing complications in the first place.”

Dr. McDonald added that “fluvoxamine is an option to reduce hospitalizations in high-risk adults. It is likely effective, is inexpensive, and has a long safety track record.” She also noted that “not all countries have access to Paxlovid, and some people have drug interactions that preclude its use. Existing monoclonals are not effective with newer variants.”

The drug’s apparent anti-inflammatory properties seem to be key, she said. According to her, the next steps should be “testing lower doses to see if they remain effective, following patients long term to see what impact there is on long COVID symptoms, testing related medications in the drug class to see if they also show an effect, and testing in vaccinated people and with newer variants.”

In an interview, biostatistician James Watson, PhD, of the Mahidol Oxford Tropical Medicine Research Unit, Bangkok, Thailand, and Nuffield department of medicine, University of Oxford, England, said the findings of the analysis are “not overwhelming data.”

He noted the TOGETHER study’s unusual focus on ED visits that latest fewer than 6 hours, which he described as “not a very objective endpoint.” The new meta-analysis focused instead on “outcome data on emergency department visits lasting more than 24 hours and used this as a more representative proxy for hospital admission than an ED visit alone.”

Dr. Fichtenbaum also highlighted the odd endpoint. “Most of us would have chosen something like use of oxygen, requirement for ventilation, or death,” he said. “There are many reasons why people go to the ED. This endpoint is not very strong.”

He also noted that the three studies “are very different in design and endpoints.”

Jeffrey S. Morris, PhD, a biostatistician at the University of Pennsylvania, Philadelphia, offered a different perspective about the findings in an interview. “There’s good evidence that it helps some,” he said, and may reduce hospitalizations by 10%. “If the pill is super cheap and toxicity is very acceptable, it’s not adding additional risk. Most clinicians would say that: ‘If I’m reducing risk by 10%, it’s worthwhile.’ ”

No funding was reported. Two authors report having a patent application filed by Washington University for methods of treating COVID-19 during the conduct of the study. Dr. Watson is an investigator for studies analyzing antiviral drugs and Prozac as COVID-19 treatments. Dr. Fichtenbaum and Dr. Morris disclosed no relevant financial relationships.

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

There’s a strong likelihood that the antidepressant fluvoxamine (Luvox) may moderately lower rates of hospitalization caused by COVID-19 in unvaccinated patients, a new systematic review and meta-analysis has found. But outside experts differ over whether the evidence from just three studies is strong enough to warrant adding the drug to the COVID-19 armamentarium.

The report, published online in JAMA Network Open, looked at three studies and estimated that the drug could reduce the relative risk of hospitalization by around 25% (likelihood of moderate effect, 81.6%-91.8%), depending on the type of analysis used.

“This research might be valuable, but the jury remains out until several other adequately powered and designed trials are completed,” said infectious disease specialist Carl J. Fichtenbaum, MD, of the University of Cincinnati, who’s familiar with the findings. “I’m not sure how useful this is given we have several antiviral agents available. Why would we choose this over Paxlovid, remdesivir, or molnupiravir?”

According to Dr. Fichtenbaum, researchers began focusing on fluvoxamine after case reports about patients improving while on the medication. This led to further interest, he said, boosted by the drug’s known ability to dampen the immune system.

A Silicon Valley investor and antivaccine activist named Steve Kirsch has been pushing the drug along with the debunked treatment hydroxychloroquine. He’s accused the government of a cover-up of fluvoxamine’s worth, according to MIT Technology Review, and he wrote a commentary that referred to the drug as “the fast, easy, safe, simple, low-cost solution to COVID that works 100% of the time that nobody wants to talk about.”

For the new analysis, researchers examined three randomized clinical trials with a total of 2,196 participants. The most extensive trial, the TOGETHER study in Brazil (n = 1,497), focused on an unusual outcome: It linked the drug to a 32% reduction in relative risk of patients with COVID-19 being hospitalized in an ED for fewer than 6 hours or transferred to a tertiary hospital because of the disease.

Another study, the STOP COVID 2 trial in the United States and Canada (n = 547), was stopped because too few patients could be recruited to provide useful results. The initial phase of this trial, STOP COVID 1 (n = 152), was also included in the analysis.

All participants in the three studies were unvaccinated. Their median age was 46-50 years, 55%-72% were women, and 44%-56% were obese. Most were multiracial due to the high number of participants from Brazil.

“In the Bayesian analyses, the pooled risk ratio in favor of fluvoxamine was 0.78 (95% confidence interval, 0.58-1.08) for the weakly neutral prior and 0.73 (95% CI, 0.53-1.01) for the moderately optimistic prior,” the researchers reported, referring to a reduction in risk of hospitalization. “In the frequentist meta-analysis, the pooled risk ratio in favor of fluvoxamine was 0.75 (95% CI, 0.58-0.97; I², 0.2%).”

Two of the authors of the new analysis were also coauthors of the TOGETHER trial and both STOP COVID trials.

Corresponding author Emily G. McDonald, MD, division of experimental medicine at McGill University, Montreal, said in an interview that the findings show fluvoxamine “very likely reduces hospitalization in high-risk outpatient adults with COVID-19. This effect varies depending on your baseline risk of developing complications in the first place.”

Dr. McDonald added that “fluvoxamine is an option to reduce hospitalizations in high-risk adults. It is likely effective, is inexpensive, and has a long safety track record.” She also noted that “not all countries have access to Paxlovid, and some people have drug interactions that preclude its use. Existing monoclonals are not effective with newer variants.”

The drug’s apparent anti-inflammatory properties seem to be key, she said. According to her, the next steps should be “testing lower doses to see if they remain effective, following patients long term to see what impact there is on long COVID symptoms, testing related medications in the drug class to see if they also show an effect, and testing in vaccinated people and with newer variants.”

In an interview, biostatistician James Watson, PhD, of the Mahidol Oxford Tropical Medicine Research Unit, Bangkok, Thailand, and Nuffield department of medicine, University of Oxford, England, said the findings of the analysis are “not overwhelming data.”

He noted the TOGETHER study’s unusual focus on ED visits that latest fewer than 6 hours, which he described as “not a very objective endpoint.” The new meta-analysis focused instead on “outcome data on emergency department visits lasting more than 24 hours and used this as a more representative proxy for hospital admission than an ED visit alone.”

Dr. Fichtenbaum also highlighted the odd endpoint. “Most of us would have chosen something like use of oxygen, requirement for ventilation, or death,” he said. “There are many reasons why people go to the ED. This endpoint is not very strong.”

He also noted that the three studies “are very different in design and endpoints.”

Jeffrey S. Morris, PhD, a biostatistician at the University of Pennsylvania, Philadelphia, offered a different perspective about the findings in an interview. “There’s good evidence that it helps some,” he said, and may reduce hospitalizations by 10%. “If the pill is super cheap and toxicity is very acceptable, it’s not adding additional risk. Most clinicians would say that: ‘If I’m reducing risk by 10%, it’s worthwhile.’ ”

No funding was reported. Two authors report having a patent application filed by Washington University for methods of treating COVID-19 during the conduct of the study. Dr. Watson is an investigator for studies analyzing antiviral drugs and Prozac as COVID-19 treatments. Dr. Fichtenbaum and Dr. Morris disclosed no relevant financial relationships.

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

There’s a strong likelihood that the antidepressant fluvoxamine (Luvox) may moderately lower rates of hospitalization caused by COVID-19 in unvaccinated patients, a new systematic review and meta-analysis has found. But outside experts differ over whether the evidence from just three studies is strong enough to warrant adding the drug to the COVID-19 armamentarium.

The report, published online in JAMA Network Open, looked at three studies and estimated that the drug could reduce the relative risk of hospitalization by around 25% (likelihood of moderate effect, 81.6%-91.8%), depending on the type of analysis used.

“This research might be valuable, but the jury remains out until several other adequately powered and designed trials are completed,” said infectious disease specialist Carl J. Fichtenbaum, MD, of the University of Cincinnati, who’s familiar with the findings. “I’m not sure how useful this is given we have several antiviral agents available. Why would we choose this over Paxlovid, remdesivir, or molnupiravir?”

According to Dr. Fichtenbaum, researchers began focusing on fluvoxamine after case reports about patients improving while on the medication. This led to further interest, he said, boosted by the drug’s known ability to dampen the immune system.

A Silicon Valley investor and antivaccine activist named Steve Kirsch has been pushing the drug along with the debunked treatment hydroxychloroquine. He’s accused the government of a cover-up of fluvoxamine’s worth, according to MIT Technology Review, and he wrote a commentary that referred to the drug as “the fast, easy, safe, simple, low-cost solution to COVID that works 100% of the time that nobody wants to talk about.”

For the new analysis, researchers examined three randomized clinical trials with a total of 2,196 participants. The most extensive trial, the TOGETHER study in Brazil (n = 1,497), focused on an unusual outcome: It linked the drug to a 32% reduction in relative risk of patients with COVID-19 being hospitalized in an ED for fewer than 6 hours or transferred to a tertiary hospital because of the disease.

Another study, the STOP COVID 2 trial in the United States and Canada (n = 547), was stopped because too few patients could be recruited to provide useful results. The initial phase of this trial, STOP COVID 1 (n = 152), was also included in the analysis.

All participants in the three studies were unvaccinated. Their median age was 46-50 years, 55%-72% were women, and 44%-56% were obese. Most were multiracial due to the high number of participants from Brazil.

“In the Bayesian analyses, the pooled risk ratio in favor of fluvoxamine was 0.78 (95% confidence interval, 0.58-1.08) for the weakly neutral prior and 0.73 (95% CI, 0.53-1.01) for the moderately optimistic prior,” the researchers reported, referring to a reduction in risk of hospitalization. “In the frequentist meta-analysis, the pooled risk ratio in favor of fluvoxamine was 0.75 (95% CI, 0.58-0.97; I², 0.2%).”

Two of the authors of the new analysis were also coauthors of the TOGETHER trial and both STOP COVID trials.

Corresponding author Emily G. McDonald, MD, division of experimental medicine at McGill University, Montreal, said in an interview that the findings show fluvoxamine “very likely reduces hospitalization in high-risk outpatient adults with COVID-19. This effect varies depending on your baseline risk of developing complications in the first place.”

Dr. McDonald added that “fluvoxamine is an option to reduce hospitalizations in high-risk adults. It is likely effective, is inexpensive, and has a long safety track record.” She also noted that “not all countries have access to Paxlovid, and some people have drug interactions that preclude its use. Existing monoclonals are not effective with newer variants.”

The drug’s apparent anti-inflammatory properties seem to be key, she said. According to her, the next steps should be “testing lower doses to see if they remain effective, following patients long term to see what impact there is on long COVID symptoms, testing related medications in the drug class to see if they also show an effect, and testing in vaccinated people and with newer variants.”

In an interview, biostatistician James Watson, PhD, of the Mahidol Oxford Tropical Medicine Research Unit, Bangkok, Thailand, and Nuffield department of medicine, University of Oxford, England, said the findings of the analysis are “not overwhelming data.”

He noted the TOGETHER study’s unusual focus on ED visits that latest fewer than 6 hours, which he described as “not a very objective endpoint.” The new meta-analysis focused instead on “outcome data on emergency department visits lasting more than 24 hours and used this as a more representative proxy for hospital admission than an ED visit alone.”

Dr. Fichtenbaum also highlighted the odd endpoint. “Most of us would have chosen something like use of oxygen, requirement for ventilation, or death,” he said. “There are many reasons why people go to the ED. This endpoint is not very strong.”

He also noted that the three studies “are very different in design and endpoints.”

Jeffrey S. Morris, PhD, a biostatistician at the University of Pennsylvania, Philadelphia, offered a different perspective about the findings in an interview. “There’s good evidence that it helps some,” he said, and may reduce hospitalizations by 10%. “If the pill is super cheap and toxicity is very acceptable, it’s not adding additional risk. Most clinicians would say that: ‘If I’m reducing risk by 10%, it’s worthwhile.’ ”

No funding was reported. Two authors report having a patent application filed by Washington University for methods of treating COVID-19 during the conduct of the study. Dr. Watson is an investigator for studies analyzing antiviral drugs and Prozac as COVID-19 treatments. Dr. Fichtenbaum and Dr. Morris disclosed no relevant financial relationships.

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

Results of the phase 2 ZUMA-12 trial suggest that axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor (CAR) T-cell therapy approved to treat certain types of lymphoma, also shows promise as a treatment for another group of lymphoma patients – those with high-risk large B-cell lymphoma (LBCL) who failed two rounds of standard chemoimmunotherapy. In fact, a study author said, first-line treatment with this therapy could help usher some patients toward a cure.

The results appeared March 21, 2022, in Nature Medicine.

“The high efficacy with manageable safety profile suggest that further evaluation of axi-cel in first-line setting in patients with high-risk LBCL is warranted in a randomized, phase 3 trial comparing it to standard chemoimmunotherapy,” study lead author Sattva S. Neelapu, MD, of the University of Texas MD Anderson Cancer Center, Houston, said in an interview.

According to Dr. Neelapu, “patients with high-risk LBCL include those with high-intermediate or high International Prognostic Index score and those with certain molecular subtypes such as double- or triple-hit lymphoma. These patients have lower response rates and lower progression-free and overall survival with standard chemoimmunotherapy.”

Treatment of these patients can be especially challenging because they are underrepresented in clinical research, hematologist Michael Dickinson, MBBS, of the Peter MacCallum Cancer Center in Melbourne, said in an interview. “They often have disease that requires urgent treatment, so there is no time to recruit them into trials. A feature of ZUMA-12 is that it allowed patients to be recruited after short exposure to chemotherapy, which means that higher-risk patients could successfully be recruited into the trial.”

Axi-cel is already Food and Drug Administration approved for treatment of relapsed or refractory LBCL after 2 or more lines of systemic therapy plus relapsed or refractory follicular lymphoma, also after two or more lines of systemic therapy, Dr. Neelapu said.

For this study, researchers administered the treatment to 40 subjects with high-risk disease from 2019-2020 (median age, 61 years; 68% male; 95% at disease stage III or IV).

The researchers reported that 78% of 37 patients in the primary efficacy analysis reached complete response rate (95% confidence interval, 62-90); the median time to first complete response rate was 30 days (range, 27-207). About 89% of these subjects reached the secondary endpoint of objective response rate (95% CI, 75-97); the median time to first objective response was 29 days (range, 27-207).

At a median follow-up of 15.9 months, 73% were still in objective response.

“This is quite remarkable,” Dr. Neelapu said. “The durability of more than 70% is far higher than what would be expected with standard chemoimmunotherapy in these patients – under 40% durability with standard chemoimmunotherapy. Also, axi-cel induces durable responses in about 40% of patients in second- and third-line setting. However, when used as part of first-line therapy in this study, durable responses were observed in more than 70% of patients, suggesting that the efficacy of axi-cel may be much higher when used in first-line setting.”

Dr. Neelapu added: “Although the follow-up is short, it is highly likely that the majority of the patients with ongoing response beyond 1 year will likely be cured of their lymphoma.”

As for side effects, no treatment-related grade 5 events occurred, but 18 patients (45%) experienced serious adverse events. Grade 3 or higher cytokine release syndrome occurred in three patients (8%) and nine experienced neurologic events (23%).

“The majority of the higher-grade adverse events observed were due to cytopenias, which were expected due to the conditioning therapy,” Dr. Neelapu said. “Such cytopenias would also have been expected if these patients had received standard chemoimmunotherapy.”

Six patients (15%) died, 4 of progressive disease after going forward to other therapies.

As for cost, Dr. Neelapu said it should be similar to that of axi-cel as an FDA-approved third-line therapy. Axi-cel is highly expensive. Research has suggested that CAR T-cell therapy can boost costs beyond standard chemotherapy by $350,000-$490,000 with gains of 2-8 years of life (J Med Econ. Jan-Dec 2021;24[1]:458-68).

The study was funded by Kite. The authors reported various disclosures.

Results of the phase 2 ZUMA-12 trial suggest that axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor (CAR) T-cell therapy approved to treat certain types of lymphoma, also shows promise as a treatment for another group of lymphoma patients – those with high-risk large B-cell lymphoma (LBCL) who failed two rounds of standard chemoimmunotherapy. In fact, a study author said, first-line treatment with this therapy could help usher some patients toward a cure.

The results appeared March 21, 2022, in Nature Medicine.

“The high efficacy with manageable safety profile suggest that further evaluation of axi-cel in first-line setting in patients with high-risk LBCL is warranted in a randomized, phase 3 trial comparing it to standard chemoimmunotherapy,” study lead author Sattva S. Neelapu, MD, of the University of Texas MD Anderson Cancer Center, Houston, said in an interview.

According to Dr. Neelapu, “patients with high-risk LBCL include those with high-intermediate or high International Prognostic Index score and those with certain molecular subtypes such as double- or triple-hit lymphoma. These patients have lower response rates and lower progression-free and overall survival with standard chemoimmunotherapy.”

Treatment of these patients can be especially challenging because they are underrepresented in clinical research, hematologist Michael Dickinson, MBBS, of the Peter MacCallum Cancer Center in Melbourne, said in an interview. “They often have disease that requires urgent treatment, so there is no time to recruit them into trials. A feature of ZUMA-12 is that it allowed patients to be recruited after short exposure to chemotherapy, which means that higher-risk patients could successfully be recruited into the trial.”

Axi-cel is already Food and Drug Administration approved for treatment of relapsed or refractory LBCL after 2 or more lines of systemic therapy plus relapsed or refractory follicular lymphoma, also after two or more lines of systemic therapy, Dr. Neelapu said.

For this study, researchers administered the treatment to 40 subjects with high-risk disease from 2019-2020 (median age, 61 years; 68% male; 95% at disease stage III or IV).

The researchers reported that 78% of 37 patients in the primary efficacy analysis reached complete response rate (95% confidence interval, 62-90); the median time to first complete response rate was 30 days (range, 27-207). About 89% of these subjects reached the secondary endpoint of objective response rate (95% CI, 75-97); the median time to first objective response was 29 days (range, 27-207).

At a median follow-up of 15.9 months, 73% were still in objective response.

“This is quite remarkable,” Dr. Neelapu said. “The durability of more than 70% is far higher than what would be expected with standard chemoimmunotherapy in these patients – under 40% durability with standard chemoimmunotherapy. Also, axi-cel induces durable responses in about 40% of patients in second- and third-line setting. However, when used as part of first-line therapy in this study, durable responses were observed in more than 70% of patients, suggesting that the efficacy of axi-cel may be much higher when used in first-line setting.”

Dr. Neelapu added: “Although the follow-up is short, it is highly likely that the majority of the patients with ongoing response beyond 1 year will likely be cured of their lymphoma.”

As for side effects, no treatment-related grade 5 events occurred, but 18 patients (45%) experienced serious adverse events. Grade 3 or higher cytokine release syndrome occurred in three patients (8%) and nine experienced neurologic events (23%).

“The majority of the higher-grade adverse events observed were due to cytopenias, which were expected due to the conditioning therapy,” Dr. Neelapu said. “Such cytopenias would also have been expected if these patients had received standard chemoimmunotherapy.”

Six patients (15%) died, 4 of progressive disease after going forward to other therapies.

As for cost, Dr. Neelapu said it should be similar to that of axi-cel as an FDA-approved third-line therapy. Axi-cel is highly expensive. Research has suggested that CAR T-cell therapy can boost costs beyond standard chemotherapy by $350,000-$490,000 with gains of 2-8 years of life (J Med Econ. Jan-Dec 2021;24[1]:458-68).

The study was funded by Kite. The authors reported various disclosures.

Results of the phase 2 ZUMA-12 trial suggest that axicabtagene ciloleucel (axi-cel), a chimeric antigen receptor (CAR) T-cell therapy approved to treat certain types of lymphoma, also shows promise as a treatment for another group of lymphoma patients – those with high-risk large B-cell lymphoma (LBCL) who failed two rounds of standard chemoimmunotherapy. In fact, a study author said, first-line treatment with this therapy could help usher some patients toward a cure.

The results appeared March 21, 2022, in Nature Medicine.

“The high efficacy with manageable safety profile suggest that further evaluation of axi-cel in first-line setting in patients with high-risk LBCL is warranted in a randomized, phase 3 trial comparing it to standard chemoimmunotherapy,” study lead author Sattva S. Neelapu, MD, of the University of Texas MD Anderson Cancer Center, Houston, said in an interview.

According to Dr. Neelapu, “patients with high-risk LBCL include those with high-intermediate or high International Prognostic Index score and those with certain molecular subtypes such as double- or triple-hit lymphoma. These patients have lower response rates and lower progression-free and overall survival with standard chemoimmunotherapy.”

Treatment of these patients can be especially challenging because they are underrepresented in clinical research, hematologist Michael Dickinson, MBBS, of the Peter MacCallum Cancer Center in Melbourne, said in an interview. “They often have disease that requires urgent treatment, so there is no time to recruit them into trials. A feature of ZUMA-12 is that it allowed patients to be recruited after short exposure to chemotherapy, which means that higher-risk patients could successfully be recruited into the trial.”

Axi-cel is already Food and Drug Administration approved for treatment of relapsed or refractory LBCL after 2 or more lines of systemic therapy plus relapsed or refractory follicular lymphoma, also after two or more lines of systemic therapy, Dr. Neelapu said.

For this study, researchers administered the treatment to 40 subjects with high-risk disease from 2019-2020 (median age, 61 years; 68% male; 95% at disease stage III or IV).

The researchers reported that 78% of 37 patients in the primary efficacy analysis reached complete response rate (95% confidence interval, 62-90); the median time to first complete response rate was 30 days (range, 27-207). About 89% of these subjects reached the secondary endpoint of objective response rate (95% CI, 75-97); the median time to first objective response was 29 days (range, 27-207).

At a median follow-up of 15.9 months, 73% were still in objective response.

“This is quite remarkable,” Dr. Neelapu said. “The durability of more than 70% is far higher than what would be expected with standard chemoimmunotherapy in these patients – under 40% durability with standard chemoimmunotherapy. Also, axi-cel induces durable responses in about 40% of patients in second- and third-line setting. However, when used as part of first-line therapy in this study, durable responses were observed in more than 70% of patients, suggesting that the efficacy of axi-cel may be much higher when used in first-line setting.”

Dr. Neelapu added: “Although the follow-up is short, it is highly likely that the majority of the patients with ongoing response beyond 1 year will likely be cured of their lymphoma.”

As for side effects, no treatment-related grade 5 events occurred, but 18 patients (45%) experienced serious adverse events. Grade 3 or higher cytokine release syndrome occurred in three patients (8%) and nine experienced neurologic events (23%).

“The majority of the higher-grade adverse events observed were due to cytopenias, which were expected due to the conditioning therapy,” Dr. Neelapu said. “Such cytopenias would also have been expected if these patients had received standard chemoimmunotherapy.”

Six patients (15%) died, 4 of progressive disease after going forward to other therapies.

As for cost, Dr. Neelapu said it should be similar to that of axi-cel as an FDA-approved third-line therapy. Axi-cel is highly expensive. Research has suggested that CAR T-cell therapy can boost costs beyond standard chemotherapy by $350,000-$490,000 with gains of 2-8 years of life (J Med Econ. Jan-Dec 2021;24[1]:458-68).

The study was funded by Kite. The authors reported various disclosures.

A new systematic literature review suggests that there may be – at most – a weak link between COVID-19 and alopecia areata.

If there is a connection, it’s likely not a strong one, said study author Rachel E. Christensen, a graduate student at Rutgers Robert Wood Johnson Medical School, in an interview. “Based on the reported number of cases following COVID-19, alopecia areata appears to be low on the list of common skin manifestations of COVID-19,” she said. Of 402 articles screened from three databases in the review, only 11 were identified as related to alopecia areata (AA) and COVID-19, and only 9 of those met the study inclusion criteria. “This number alone highlights the very low number of published articles investigating this connection.”

The review was published in JAAD International.

While COVID-19 has been linked to a variety of skin conditions, a 2021 South Korean study of 7,958 cases and 218,779 controls found no connection between infection and AA even after covariates such as age, gender, and income level were taken into account. In a letter to the editor published in 2020, dermatologists in Turkey reported that the percentage of patients with AA at the dermatology outpatient clinic jumped from 0.97% in May 2019 to 1.48% in May 2020. The number of patients in each group wasn’t reported.

Systematic review

The investigators launched the systematic review to gain a wider perspective, although there are still limitations. On the one hand, Ms. Christensen said, “we do know that COVID-19, like other viruses, has been linked to various dermatological disorders.”

However, “it is difficult to tease apart whether any worsening of alopecia areata we see following COVID-19 is due to the virus itself or the increased psychological burden related to the infection or to the pandemic in general,” she said. Indeed, the authors of the report in Turkey attributed the rise in cases to stress.

For the review, the researchers analyzed studies from Italy (four), Turkey (two), Brazil (one), the United States (one), and Poland (one).

Six of the studies reported cases of new-onset AA following COVID-19 infection (seven cases; average age, 37 years; females, three). Another study was a retrospective review of 32 patients with preexisting AA who developed COVID-19; none experienced significant worsening of AA within 6 months.

The review also included a study based on a survey of 389 patients with AA. The investigators found that, at a median 2.14 months after infection, 44% of those who had COVID-19 vs. 12% of those who were COVID negative had a relapse. Finally, a case report noted a patient with preexisting AA whose condition worsened following COVID infection.

The findings suggest that AA “could be a dermatological manifestation of COVID-19, with cases most often appearing 1-2 months following infection,” the authors wrote. “However, the heterogeneity of study designs and high proportion of case reports make it challenging to draw any conclusion.”

In an interview, dermatologist Brett King, MD, PhD, of the department of dermatology, Yale University, New Haven, Conn., said the review findings suggest that “there is little concern of alopecia areata following COVID infection.

Does new-onset AA happen, and are there exacerbations of preexisting disease related to COVID infection? Probably yes, but rarely.”

However, he noted that another form of alopecia, telogen effluvium (TE), is more common after COVID-19 infection. According to Dr. King, who was not involved with the systematic review, TE is typically time-limited, compared with AA’s more common chronic waxing-and-waning course.

“Distinguishing TE and AA is usually straightforward because AA typically presents with well-circumscribed patches of hair loss,” such as circular patches, “while TE manifests as diffuse hair loss,” he explained. “Rarely, however, AA does manifest diffuse hair loss without patches, similar to TE. In those cases, it may be difficult to distinguish them. A biopsy may be helpful if there is a question of the diagnosis.”

No study funding is reported. The review authors and Dr. King report no relevant disclosures.

A new systematic literature review suggests that there may be – at most – a weak link between COVID-19 and alopecia areata.

If there is a connection, it’s likely not a strong one, said study author Rachel E. Christensen, a graduate student at Rutgers Robert Wood Johnson Medical School, in an interview. “Based on the reported number of cases following COVID-19, alopecia areata appears to be low on the list of common skin manifestations of COVID-19,” she said. Of 402 articles screened from three databases in the review, only 11 were identified as related to alopecia areata (AA) and COVID-19, and only 9 of those met the study inclusion criteria. “This number alone highlights the very low number of published articles investigating this connection.”

The review was published in JAAD International.

While COVID-19 has been linked to a variety of skin conditions, a 2021 South Korean study of 7,958 cases and 218,779 controls found no connection between infection and AA even after covariates such as age, gender, and income level were taken into account. In a letter to the editor published in 2020, dermatologists in Turkey reported that the percentage of patients with AA at the dermatology outpatient clinic jumped from 0.97% in May 2019 to 1.48% in May 2020. The number of patients in each group wasn’t reported.

Systematic review

The investigators launched the systematic review to gain a wider perspective, although there are still limitations. On the one hand, Ms. Christensen said, “we do know that COVID-19, like other viruses, has been linked to various dermatological disorders.”

However, “it is difficult to tease apart whether any worsening of alopecia areata we see following COVID-19 is due to the virus itself or the increased psychological burden related to the infection or to the pandemic in general,” she said. Indeed, the authors of the report in Turkey attributed the rise in cases to stress.

For the review, the researchers analyzed studies from Italy (four), Turkey (two), Brazil (one), the United States (one), and Poland (one).

Six of the studies reported cases of new-onset AA following COVID-19 infection (seven cases; average age, 37 years; females, three). Another study was a retrospective review of 32 patients with preexisting AA who developed COVID-19; none experienced significant worsening of AA within 6 months.

The review also included a study based on a survey of 389 patients with AA. The investigators found that, at a median 2.14 months after infection, 44% of those who had COVID-19 vs. 12% of those who were COVID negative had a relapse. Finally, a case report noted a patient with preexisting AA whose condition worsened following COVID infection.

The findings suggest that AA “could be a dermatological manifestation of COVID-19, with cases most often appearing 1-2 months following infection,” the authors wrote. “However, the heterogeneity of study designs and high proportion of case reports make it challenging to draw any conclusion.”

In an interview, dermatologist Brett King, MD, PhD, of the department of dermatology, Yale University, New Haven, Conn., said the review findings suggest that “there is little concern of alopecia areata following COVID infection.

Does new-onset AA happen, and are there exacerbations of preexisting disease related to COVID infection? Probably yes, but rarely.”

However, he noted that another form of alopecia, telogen effluvium (TE), is more common after COVID-19 infection. According to Dr. King, who was not involved with the systematic review, TE is typically time-limited, compared with AA’s more common chronic waxing-and-waning course.

“Distinguishing TE and AA is usually straightforward because AA typically presents with well-circumscribed patches of hair loss,” such as circular patches, “while TE manifests as diffuse hair loss,” he explained. “Rarely, however, AA does manifest diffuse hair loss without patches, similar to TE. In those cases, it may be difficult to distinguish them. A biopsy may be helpful if there is a question of the diagnosis.”

No study funding is reported. The review authors and Dr. King report no relevant disclosures.

A new systematic literature review suggests that there may be – at most – a weak link between COVID-19 and alopecia areata.

If there is a connection, it’s likely not a strong one, said study author Rachel E. Christensen, a graduate student at Rutgers Robert Wood Johnson Medical School, in an interview. “Based on the reported number of cases following COVID-19, alopecia areata appears to be low on the list of common skin manifestations of COVID-19,” she said. Of 402 articles screened from three databases in the review, only 11 were identified as related to alopecia areata (AA) and COVID-19, and only 9 of those met the study inclusion criteria. “This number alone highlights the very low number of published articles investigating this connection.”

The review was published in JAAD International.

While COVID-19 has been linked to a variety of skin conditions, a 2021 South Korean study of 7,958 cases and 218,779 controls found no connection between infection and AA even after covariates such as age, gender, and income level were taken into account. In a letter to the editor published in 2020, dermatologists in Turkey reported that the percentage of patients with AA at the dermatology outpatient clinic jumped from 0.97% in May 2019 to 1.48% in May 2020. The number of patients in each group wasn’t reported.

Systematic review

The investigators launched the systematic review to gain a wider perspective, although there are still limitations. On the one hand, Ms. Christensen said, “we do know that COVID-19, like other viruses, has been linked to various dermatological disorders.”

However, “it is difficult to tease apart whether any worsening of alopecia areata we see following COVID-19 is due to the virus itself or the increased psychological burden related to the infection or to the pandemic in general,” she said. Indeed, the authors of the report in Turkey attributed the rise in cases to stress.

For the review, the researchers analyzed studies from Italy (four), Turkey (two), Brazil (one), the United States (one), and Poland (one).

Six of the studies reported cases of new-onset AA following COVID-19 infection (seven cases; average age, 37 years; females, three). Another study was a retrospective review of 32 patients with preexisting AA who developed COVID-19; none experienced significant worsening of AA within 6 months.

The review also included a study based on a survey of 389 patients with AA. The investigators found that, at a median 2.14 months after infection, 44% of those who had COVID-19 vs. 12% of those who were COVID negative had a relapse. Finally, a case report noted a patient with preexisting AA whose condition worsened following COVID infection.

The findings suggest that AA “could be a dermatological manifestation of COVID-19, with cases most often appearing 1-2 months following infection,” the authors wrote. “However, the heterogeneity of study designs and high proportion of case reports make it challenging to draw any conclusion.”

In an interview, dermatologist Brett King, MD, PhD, of the department of dermatology, Yale University, New Haven, Conn., said the review findings suggest that “there is little concern of alopecia areata following COVID infection.

Does new-onset AA happen, and are there exacerbations of preexisting disease related to COVID infection? Probably yes, but rarely.”

However, he noted that another form of alopecia, telogen effluvium (TE), is more common after COVID-19 infection. According to Dr. King, who was not involved with the systematic review, TE is typically time-limited, compared with AA’s more common chronic waxing-and-waning course.

“Distinguishing TE and AA is usually straightforward because AA typically presents with well-circumscribed patches of hair loss,” such as circular patches, “while TE manifests as diffuse hair loss,” he explained. “Rarely, however, AA does manifest diffuse hair loss without patches, similar to TE. In those cases, it may be difficult to distinguish them. A biopsy may be helpful if there is a question of the diagnosis.”

No study funding is reported. The review authors and Dr. King report no relevant disclosures.

Armed with data from multiple studies about how to implement goal-setting in hemophilia, a national nonprofit organization has released a free app designed to help patients track their illness and develop and monitor their objectives.

Robust Health, available for the iPhone and Android, “can really enhance the physician-patient relationship. This is a good approach to capture the many facets of what’s important to patients and help them improve their therapy management in their lives,” Jonathan C. Roberts, MD, a hematologist/oncologist at the University of Illinois at Peoria, said in an interview.

Working with colleagues, Dr. Roberts helped the app developer, the American Thrombosis and Hemostasis Network, devise the app’s goal-setting tool. Researchers reported their findings on the tool – known as Goal Attainment Scaling for Hemophilia, or “GOAL‐Hēm” – in a series of studies that emphasized the importance of including the “patient voice.”

The tool was developed to monitor patient outcomes in terms of “meaningful change,” beyond data points such as annualized bleed rate, Dr. Roberts said.

“Metrics like this are definitely important to joint health and quality of life,” he said, but researchers hoped to expand to more outcomes that matter to patients.

Consider a pediatric patient, for example, who may set a goal of preparing his or her clotting-factor treatment and making one attempt at a puncture. The tool allows a benchmark and timetable to be set up, Dr. Roberts said, and can provide both positive reinforcement and a score that reflects how well the patient is doing. “That really can help the multidisciplinary treatment team measure improvements in the patient’s overall treatment adherence.”

For the most recent study, published in the January 2022 issue of Research and Practice in Thrombosis and Haemostasis, researchers interviewed 19 adult patients with hemophilia (mean age 35, 68% male) and 19 caregivers of children with hemophilia (mean age of children 13, 83% male) about the language used in the tool. They responded in surveys, interviews, and focus groups.

“Thematic analysis indicated that participants were enthusiastic about patient‐centric language, empowered through the goal‐setting process, and recognized GOAL‐Hēm could measure clinically meaningful change,” the researchers reported.

They wrote that the participants kept 15 of 48 goals unchanged (32%), modified or deleted the others, and added three new goals. Their revisions included renaming one goal “bleeds,” instead of both“muscle bleeds” and “bleeds.” They renamed “work attendance” and “career planning” as simply “work.” “Depression,” “feelings of anger” and “self-esteem” were consolidated as a new heading: “emotional well-being.”

Each goal provides answers that patients can use to respond to queries about how they’re doing. For example, under the pediatric goal of “independent self-care management,” a descriptor could be “Always sets their own reminders to self‐infuse. Mother never needs to remind them.” This answer would be considered “much better than expected.”

Out of 635 responses, known as “descriptors,” most (75%) were revised or deleted in response to input from patients and caregivers. In the end, the total number of answers was reduced to 368 – 218 in the adult section, and 150 in the pediatric section.

“Our study highlights the importance of patient engagement in developing the tool and how it can be used in day-to-day practice,” Dr. Roberts said.

Going forward, he said, “we’re hoping this tool could potentially be an important player in studies of new therapeutic options for patients. The metrics could be used as kind of a common language to measure how our patients are doing on a particular therapy.”

Jayson Stoffman, MD, a pediatric hematologist/oncologist at Children’s Hospital of Winnipeg and the University of Manitoba, who was not involved in the research, welcomed the new app.

“The big challenge is always how to balance hemophilia and its management against the lifestyle needs and wants of the individual,” Dr. Stoffman said in an interview. “We don’t want people to be held back by their hemophilia, so it’s important to find the best ways to support them in their choices while optimizing their management.”

An app that helps patients define and delineate goals will be a “great benchmark to use in making treatment decisions and adjustments,” he said.

The study was funded by Takeda. Dr. Roberts disclosed grants and/or contracts from Takeda and consulting fees from Sanofi Genzyme, Takeda, Octapharma, uniQure, Novo Nordisk, Pfizer, Spark, and CSL Behring. The other authors reported various disclosures. Dr. Stoffman disclosed a consulting agreement with F. Hoffman La Roche AG.

Armed with data from multiple studies about how to implement goal-setting in hemophilia, a national nonprofit organization has released a free app designed to help patients track their illness and develop and monitor their objectives.

Robust Health, available for the iPhone and Android, “can really enhance the physician-patient relationship. This is a good approach to capture the many facets of what’s important to patients and help them improve their therapy management in their lives,” Jonathan C. Roberts, MD, a hematologist/oncologist at the University of Illinois at Peoria, said in an interview.

Working with colleagues, Dr. Roberts helped the app developer, the American Thrombosis and Hemostasis Network, devise the app’s goal-setting tool. Researchers reported their findings on the tool – known as Goal Attainment Scaling for Hemophilia, or “GOAL‐Hēm” – in a series of studies that emphasized the importance of including the “patient voice.”

The tool was developed to monitor patient outcomes in terms of “meaningful change,” beyond data points such as annualized bleed rate, Dr. Roberts said.

“Metrics like this are definitely important to joint health and quality of life,” he said, but researchers hoped to expand to more outcomes that matter to patients.

Consider a pediatric patient, for example, who may set a goal of preparing his or her clotting-factor treatment and making one attempt at a puncture. The tool allows a benchmark and timetable to be set up, Dr. Roberts said, and can provide both positive reinforcement and a score that reflects how well the patient is doing. “That really can help the multidisciplinary treatment team measure improvements in the patient’s overall treatment adherence.”

For the most recent study, published in the January 2022 issue of Research and Practice in Thrombosis and Haemostasis, researchers interviewed 19 adult patients with hemophilia (mean age 35, 68% male) and 19 caregivers of children with hemophilia (mean age of children 13, 83% male) about the language used in the tool. They responded in surveys, interviews, and focus groups.

“Thematic analysis indicated that participants were enthusiastic about patient‐centric language, empowered through the goal‐setting process, and recognized GOAL‐Hēm could measure clinically meaningful change,” the researchers reported.

They wrote that the participants kept 15 of 48 goals unchanged (32%), modified or deleted the others, and added three new goals. Their revisions included renaming one goal “bleeds,” instead of both“muscle bleeds” and “bleeds.” They renamed “work attendance” and “career planning” as simply “work.” “Depression,” “feelings of anger” and “self-esteem” were consolidated as a new heading: “emotional well-being.”

Each goal provides answers that patients can use to respond to queries about how they’re doing. For example, under the pediatric goal of “independent self-care management,” a descriptor could be “Always sets their own reminders to self‐infuse. Mother never needs to remind them.” This answer would be considered “much better than expected.”

Out of 635 responses, known as “descriptors,” most (75%) were revised or deleted in response to input from patients and caregivers. In the end, the total number of answers was reduced to 368 – 218 in the adult section, and 150 in the pediatric section.

“Our study highlights the importance of patient engagement in developing the tool and how it can be used in day-to-day practice,” Dr. Roberts said.

Going forward, he said, “we’re hoping this tool could potentially be an important player in studies of new therapeutic options for patients. The metrics could be used as kind of a common language to measure how our patients are doing on a particular therapy.”

Jayson Stoffman, MD, a pediatric hematologist/oncologist at Children’s Hospital of Winnipeg and the University of Manitoba, who was not involved in the research, welcomed the new app.

“The big challenge is always how to balance hemophilia and its management against the lifestyle needs and wants of the individual,” Dr. Stoffman said in an interview. “We don’t want people to be held back by their hemophilia, so it’s important to find the best ways to support them in their choices while optimizing their management.”

An app that helps patients define and delineate goals will be a “great benchmark to use in making treatment decisions and adjustments,” he said.

The study was funded by Takeda. Dr. Roberts disclosed grants and/or contracts from Takeda and consulting fees from Sanofi Genzyme, Takeda, Octapharma, uniQure, Novo Nordisk, Pfizer, Spark, and CSL Behring. The other authors reported various disclosures. Dr. Stoffman disclosed a consulting agreement with F. Hoffman La Roche AG.

Armed with data from multiple studies about how to implement goal-setting in hemophilia, a national nonprofit organization has released a free app designed to help patients track their illness and develop and monitor their objectives.

Robust Health, available for the iPhone and Android, “can really enhance the physician-patient relationship. This is a good approach to capture the many facets of what’s important to patients and help them improve their therapy management in their lives,” Jonathan C. Roberts, MD, a hematologist/oncologist at the University of Illinois at Peoria, said in an interview.

Working with colleagues, Dr. Roberts helped the app developer, the American Thrombosis and Hemostasis Network, devise the app’s goal-setting tool. Researchers reported their findings on the tool – known as Goal Attainment Scaling for Hemophilia, or “GOAL‐Hēm” – in a series of studies that emphasized the importance of including the “patient voice.”

The tool was developed to monitor patient outcomes in terms of “meaningful change,” beyond data points such as annualized bleed rate, Dr. Roberts said.

“Metrics like this are definitely important to joint health and quality of life,” he said, but researchers hoped to expand to more outcomes that matter to patients.

Consider a pediatric patient, for example, who may set a goal of preparing his or her clotting-factor treatment and making one attempt at a puncture. The tool allows a benchmark and timetable to be set up, Dr. Roberts said, and can provide both positive reinforcement and a score that reflects how well the patient is doing. “That really can help the multidisciplinary treatment team measure improvements in the patient’s overall treatment adherence.”

For the most recent study, published in the January 2022 issue of Research and Practice in Thrombosis and Haemostasis, researchers interviewed 19 adult patients with hemophilia (mean age 35, 68% male) and 19 caregivers of children with hemophilia (mean age of children 13, 83% male) about the language used in the tool. They responded in surveys, interviews, and focus groups.

“Thematic analysis indicated that participants were enthusiastic about patient‐centric language, empowered through the goal‐setting process, and recognized GOAL‐Hēm could measure clinically meaningful change,” the researchers reported.

They wrote that the participants kept 15 of 48 goals unchanged (32%), modified or deleted the others, and added three new goals. Their revisions included renaming one goal “bleeds,” instead of both“muscle bleeds” and “bleeds.” They renamed “work attendance” and “career planning” as simply “work.” “Depression,” “feelings of anger” and “self-esteem” were consolidated as a new heading: “emotional well-being.”

Each goal provides answers that patients can use to respond to queries about how they’re doing. For example, under the pediatric goal of “independent self-care management,” a descriptor could be “Always sets their own reminders to self‐infuse. Mother never needs to remind them.” This answer would be considered “much better than expected.”

Out of 635 responses, known as “descriptors,” most (75%) were revised or deleted in response to input from patients and caregivers. In the end, the total number of answers was reduced to 368 – 218 in the adult section, and 150 in the pediatric section.

“Our study highlights the importance of patient engagement in developing the tool and how it can be used in day-to-day practice,” Dr. Roberts said.

Going forward, he said, “we’re hoping this tool could potentially be an important player in studies of new therapeutic options for patients. The metrics could be used as kind of a common language to measure how our patients are doing on a particular therapy.”

Jayson Stoffman, MD, a pediatric hematologist/oncologist at Children’s Hospital of Winnipeg and the University of Manitoba, who was not involved in the research, welcomed the new app.

“The big challenge is always how to balance hemophilia and its management against the lifestyle needs and wants of the individual,” Dr. Stoffman said in an interview. “We don’t want people to be held back by their hemophilia, so it’s important to find the best ways to support them in their choices while optimizing their management.”

An app that helps patients define and delineate goals will be a “great benchmark to use in making treatment decisions and adjustments,” he said.

The study was funded by Takeda. Dr. Roberts disclosed grants and/or contracts from Takeda and consulting fees from Sanofi Genzyme, Takeda, Octapharma, uniQure, Novo Nordisk, Pfizer, Spark, and CSL Behring. The other authors reported various disclosures. Dr. Stoffman disclosed a consulting agreement with F. Hoffman La Roche AG.