Anemia

“Petechial rash often prompts further investigation into hematological, dermatological, or vasculitis causes. However, if the above investigations are negative and skin biopsy has not revealed a cause, there is a Renaissance-era diagnosis that is often overlooked but is easily investigated and treated,” wrote Andrew Dermawan, MD, and colleagues from Sir Charles Gairdner Hospital in Nedlands, Australia, in BMJ Case Reports. The diagnosis they highlight is scurvy, a disease that has faded from common medical concern but is reemerging, partly because of the rise in bariatric surgery.

Diagnosing Scurvy in the 2020s

In their article, Dermawan and colleagues present the case of a 50-year-old man with a bilateral petechial rash on his lower limbs, without any history of trauma. The patient, who exhibited no infectious symptoms, also had gross hematuria, microcytic anemia, mild neutropenia, and lymphopenia. Tests for autoimmune and hematological diseases were negative, as were abdominal and leg CT scans, ruling out abdominal hemorrhage and vasculitis. Additionally, a skin biopsy showed no causative findings.

The doctors noted that the patient had undergone sleeve gastrectomy, prompting them to inquire about his diet. They discovered that, because of financial difficulties, his diet primarily consisted of processed foods with little to no fruits or vegetables, and he had stopped taking supplements recommended by his gastroenterologist. Further tests revealed a vitamin D deficiency and a severe deficiency in vitamin C. With the diagnosis of scurvy confirmed, the doctors treated the patient with 1000 mg of ascorbic acid daily, along with cholecalciferol, folic acid, and a multivitamin complex, leading to a complete resolution of his symptoms.
 

Risk Factors Then and Now

Scurvy can present with a range of symptoms, including petechiae, perifollicular hemorrhage, ecchymosis, gingivitis, edema, anemia, delayed wound healing, malaise, weakness, joint swelling, arthralgia, anorexia, neuropathy, and vasomotor instability. It can cause mucosal and gastric hemorrhages, and if left untreated, it can lead to fatal bleeding.

Historically known as “sailors’ disease,” scurvy plagued men on long voyages who lacked access to fresh fruits or vegetables and thus did not get enough vitamin C. In 1747, James Lind, a British physician in the Royal Navy, demonstrated that the consumption of oranges and lemons could combat scurvy.

Today’s risk factors for scurvy include malnutrition, gastrointestinal disorders (eg, chronic inflammatory bowel diseases), alcohol and tobacco use, eating disorders, psychiatric illnesses, dialysis, and the use of medications that reduce the absorption of ascorbic acid (such as corticosteroids and proton pump inhibitors).

Scurvy remains more common among individuals with unfavorable socioeconomic conditions. The authors of the study emphasize how the rising cost of living — specifically in Australia but applicable elsewhere — is changing eating habits, leading to a high consumption of low-cost, nutritionally poor foods.

Poverty has always been a risk factor for scurvy, but today there may be an additional cause: bariatric surgery. Patients undergoing these procedures are at a risk for deficiencies in fat-soluble vitamins A, D, E, and K, and if their diet is inadequate, they may also experience a vitamin C deficiency. Awareness of this can facilitate the timely diagnosis of scurvy in these patients.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

“Petechial rash often prompts further investigation into hematological, dermatological, or vasculitis causes. However, if the above investigations are negative and skin biopsy has not revealed a cause, there is a Renaissance-era diagnosis that is often overlooked but is easily investigated and treated,” wrote Andrew Dermawan, MD, and colleagues from Sir Charles Gairdner Hospital in Nedlands, Australia, in BMJ Case Reports. The diagnosis they highlight is scurvy, a disease that has faded from common medical concern but is reemerging, partly because of the rise in bariatric surgery.

Diagnosing Scurvy in the 2020s

In their article, Dermawan and colleagues present the case of a 50-year-old man with a bilateral petechial rash on his lower limbs, without any history of trauma. The patient, who exhibited no infectious symptoms, also had gross hematuria, microcytic anemia, mild neutropenia, and lymphopenia. Tests for autoimmune and hematological diseases were negative, as were abdominal and leg CT scans, ruling out abdominal hemorrhage and vasculitis. Additionally, a skin biopsy showed no causative findings.

The doctors noted that the patient had undergone sleeve gastrectomy, prompting them to inquire about his diet. They discovered that, because of financial difficulties, his diet primarily consisted of processed foods with little to no fruits or vegetables, and he had stopped taking supplements recommended by his gastroenterologist. Further tests revealed a vitamin D deficiency and a severe deficiency in vitamin C. With the diagnosis of scurvy confirmed, the doctors treated the patient with 1000 mg of ascorbic acid daily, along with cholecalciferol, folic acid, and a multivitamin complex, leading to a complete resolution of his symptoms.
 

Risk Factors Then and Now

Scurvy can present with a range of symptoms, including petechiae, perifollicular hemorrhage, ecchymosis, gingivitis, edema, anemia, delayed wound healing, malaise, weakness, joint swelling, arthralgia, anorexia, neuropathy, and vasomotor instability. It can cause mucosal and gastric hemorrhages, and if left untreated, it can lead to fatal bleeding.

Historically known as “sailors’ disease,” scurvy plagued men on long voyages who lacked access to fresh fruits or vegetables and thus did not get enough vitamin C. In 1747, James Lind, a British physician in the Royal Navy, demonstrated that the consumption of oranges and lemons could combat scurvy.

Today’s risk factors for scurvy include malnutrition, gastrointestinal disorders (eg, chronic inflammatory bowel diseases), alcohol and tobacco use, eating disorders, psychiatric illnesses, dialysis, and the use of medications that reduce the absorption of ascorbic acid (such as corticosteroids and proton pump inhibitors).

Scurvy remains more common among individuals with unfavorable socioeconomic conditions. The authors of the study emphasize how the rising cost of living — specifically in Australia but applicable elsewhere — is changing eating habits, leading to a high consumption of low-cost, nutritionally poor foods.

Poverty has always been a risk factor for scurvy, but today there may be an additional cause: bariatric surgery. Patients undergoing these procedures are at a risk for deficiencies in fat-soluble vitamins A, D, E, and K, and if their diet is inadequate, they may also experience a vitamin C deficiency. Awareness of this can facilitate the timely diagnosis of scurvy in these patients.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

“Petechial rash often prompts further investigation into hematological, dermatological, or vasculitis causes. However, if the above investigations are negative and skin biopsy has not revealed a cause, there is a Renaissance-era diagnosis that is often overlooked but is easily investigated and treated,” wrote Andrew Dermawan, MD, and colleagues from Sir Charles Gairdner Hospital in Nedlands, Australia, in BMJ Case Reports. The diagnosis they highlight is scurvy, a disease that has faded from common medical concern but is reemerging, partly because of the rise in bariatric surgery.

Diagnosing Scurvy in the 2020s

In their article, Dermawan and colleagues present the case of a 50-year-old man with a bilateral petechial rash on his lower limbs, without any history of trauma. The patient, who exhibited no infectious symptoms, also had gross hematuria, microcytic anemia, mild neutropenia, and lymphopenia. Tests for autoimmune and hematological diseases were negative, as were abdominal and leg CT scans, ruling out abdominal hemorrhage and vasculitis. Additionally, a skin biopsy showed no causative findings.

The doctors noted that the patient had undergone sleeve gastrectomy, prompting them to inquire about his diet. They discovered that, because of financial difficulties, his diet primarily consisted of processed foods with little to no fruits or vegetables, and he had stopped taking supplements recommended by his gastroenterologist. Further tests revealed a vitamin D deficiency and a severe deficiency in vitamin C. With the diagnosis of scurvy confirmed, the doctors treated the patient with 1000 mg of ascorbic acid daily, along with cholecalciferol, folic acid, and a multivitamin complex, leading to a complete resolution of his symptoms.
 

Risk Factors Then and Now

Scurvy can present with a range of symptoms, including petechiae, perifollicular hemorrhage, ecchymosis, gingivitis, edema, anemia, delayed wound healing, malaise, weakness, joint swelling, arthralgia, anorexia, neuropathy, and vasomotor instability. It can cause mucosal and gastric hemorrhages, and if left untreated, it can lead to fatal bleeding.

Historically known as “sailors’ disease,” scurvy plagued men on long voyages who lacked access to fresh fruits or vegetables and thus did not get enough vitamin C. In 1747, James Lind, a British physician in the Royal Navy, demonstrated that the consumption of oranges and lemons could combat scurvy.

Today’s risk factors for scurvy include malnutrition, gastrointestinal disorders (eg, chronic inflammatory bowel diseases), alcohol and tobacco use, eating disorders, psychiatric illnesses, dialysis, and the use of medications that reduce the absorption of ascorbic acid (such as corticosteroids and proton pump inhibitors).

Scurvy remains more common among individuals with unfavorable socioeconomic conditions. The authors of the study emphasize how the rising cost of living — specifically in Australia but applicable elsewhere — is changing eating habits, leading to a high consumption of low-cost, nutritionally poor foods.

Poverty has always been a risk factor for scurvy, but today there may be an additional cause: bariatric surgery. Patients undergoing these procedures are at a risk for deficiencies in fat-soluble vitamins A, D, E, and K, and if their diet is inadequate, they may also experience a vitamin C deficiency. Awareness of this can facilitate the timely diagnosis of scurvy in these patients.

This story was translated from Univadis Italy using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Ferritin is the parameter most often used in primary care to diagnose iron deficiency. The cutoff value of ferritin can affect the number of cases diagnosed, however. A study published in JAMA Network Open  investigated how different cutoff values affect the diagnosis of iron deficiency.

The study, which included 255,351 adult primary care patients in Switzerland, showed that ferritin cutoff values of 15, 30, and 45 ng/mL were associated with incidences of iron deficiency diagnoses of 10.9, 29.9, and 48.3 cases per 1000 patient-years, respectively. In other words, as the cutoff value increases, the frequency of diagnosis also increases.

“It is a study to take into account, especially because of the number of patients it includes, and it can guide primary care clinical practice. As expected, as the cutoff point increases with respect to ferritin values, the incidence percentages of both iron deficiency and iron-deficiency anemia also increase,” Miguel Turégano-Yedro, MD, a family physician at the Casar de Cáceres Health Center in Spain, and coordinator of the Hematology Working Group of the Spanish Society of Primary Care Physicians, told this news organization. Ferritin is the most sensitive parameter for diagnosing iron deficiency and iron-deficiency anemia, he added. “When it is necessary to supplement a patient with iron, other parameters are taken into account, such as hemoglobin, to see if there is anemia.”
 

Ferritin Level

The ferritin level associated with iron deficiency in primary care is usually 15 ng/mL, said Dr. Turégano-Yedro. “If we assess patients with a ferritin level of 15 or less than 15, then we know that many cases will be symptomatic (with fatigue, tiredness, or lack of appetite) and, therefore, will need iron treatment. But if the ferritin cutoff value is increased to 30 ng/mL or 45 ng/mL, the incidence will be higher, although in many cases they will be asymptomatic and iron supplementation will not be necessary.”

He also pointed out that he does not consider it necessary to raise the cutoff to 45 ng/mL; however, “establishing the cutoff at 30 ng/mL, in a certain population at risk of iron deficiency or iron-deficiency anemia, may be interesting, for example in women of childbearing age, women with very heavy menstruation, children, frail elderly, people with gastrointestinal bleeding, or those who engage in physical exercise.”

Iron deficiency must be distinguished from anemia. “If the ferritin is below 15 ng/mL, there is iron deficiency, which may or may not be accompanied by symptoms, although usually most patients will have symptoms. Normally, to diagnose a patient with iron-deficiency anemia, on the one hand, they must have low hemoglobin, which indicates anemia, and on the other hand, low ferritin, which indicates iron deficiency.” Taking these parameters into account, the study does have a weakness. “It is striking that a percentage of patients in the study requested ferritin analysis without including hemoglobin, when hemoglobin is part of the basic analysis performed in Spain,” said Dr. Turégano-Yedro.
 

When to Supplement

The study highlights the incidence of nonanemic iron deficiency diagnoses associated with the choice of ferritin cutoff value. However, as Dr. Turégano-Yedro explained, the percentage of patients who have iron deficiency but do not have anemia is not very relevant. “In the case of family physicians in Spain, it is not usually taken into account, because if a patient has iron deficiency with or without anemia and is symptomatic, they should be given iron supplements.”

What if they do not have a deficiency but do have anemia? “In principle, iron supplementation is not necessary, because that anemia may be due to chronic disorders or it may be hemolytic anemia, so the case should be studied,” Dr. Turégano-Yedro concluded.

This story was translated from Univadis Spain, which is part of the Medscape Professional Network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Ferritin is the parameter most often used in primary care to diagnose iron deficiency. The cutoff value of ferritin can affect the number of cases diagnosed, however. A study published in JAMA Network Open  investigated how different cutoff values affect the diagnosis of iron deficiency.

The study, which included 255,351 adult primary care patients in Switzerland, showed that ferritin cutoff values of 15, 30, and 45 ng/mL were associated with incidences of iron deficiency diagnoses of 10.9, 29.9, and 48.3 cases per 1000 patient-years, respectively. In other words, as the cutoff value increases, the frequency of diagnosis also increases.

“It is a study to take into account, especially because of the number of patients it includes, and it can guide primary care clinical practice. As expected, as the cutoff point increases with respect to ferritin values, the incidence percentages of both iron deficiency and iron-deficiency anemia also increase,” Miguel Turégano-Yedro, MD, a family physician at the Casar de Cáceres Health Center in Spain, and coordinator of the Hematology Working Group of the Spanish Society of Primary Care Physicians, told this news organization. Ferritin is the most sensitive parameter for diagnosing iron deficiency and iron-deficiency anemia, he added. “When it is necessary to supplement a patient with iron, other parameters are taken into account, such as hemoglobin, to see if there is anemia.”
 

Ferritin Level

The ferritin level associated with iron deficiency in primary care is usually 15 ng/mL, said Dr. Turégano-Yedro. “If we assess patients with a ferritin level of 15 or less than 15, then we know that many cases will be symptomatic (with fatigue, tiredness, or lack of appetite) and, therefore, will need iron treatment. But if the ferritin cutoff value is increased to 30 ng/mL or 45 ng/mL, the incidence will be higher, although in many cases they will be asymptomatic and iron supplementation will not be necessary.”

He also pointed out that he does not consider it necessary to raise the cutoff to 45 ng/mL; however, “establishing the cutoff at 30 ng/mL, in a certain population at risk of iron deficiency or iron-deficiency anemia, may be interesting, for example in women of childbearing age, women with very heavy menstruation, children, frail elderly, people with gastrointestinal bleeding, or those who engage in physical exercise.”

Iron deficiency must be distinguished from anemia. “If the ferritin is below 15 ng/mL, there is iron deficiency, which may or may not be accompanied by symptoms, although usually most patients will have symptoms. Normally, to diagnose a patient with iron-deficiency anemia, on the one hand, they must have low hemoglobin, which indicates anemia, and on the other hand, low ferritin, which indicates iron deficiency.” Taking these parameters into account, the study does have a weakness. “It is striking that a percentage of patients in the study requested ferritin analysis without including hemoglobin, when hemoglobin is part of the basic analysis performed in Spain,” said Dr. Turégano-Yedro.
 

When to Supplement

The study highlights the incidence of nonanemic iron deficiency diagnoses associated with the choice of ferritin cutoff value. However, as Dr. Turégano-Yedro explained, the percentage of patients who have iron deficiency but do not have anemia is not very relevant. “In the case of family physicians in Spain, it is not usually taken into account, because if a patient has iron deficiency with or without anemia and is symptomatic, they should be given iron supplements.”

What if they do not have a deficiency but do have anemia? “In principle, iron supplementation is not necessary, because that anemia may be due to chronic disorders or it may be hemolytic anemia, so the case should be studied,” Dr. Turégano-Yedro concluded.

This story was translated from Univadis Spain, which is part of the Medscape Professional Network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Ferritin is the parameter most often used in primary care to diagnose iron deficiency. The cutoff value of ferritin can affect the number of cases diagnosed, however. A study published in JAMA Network Open  investigated how different cutoff values affect the diagnosis of iron deficiency.

The study, which included 255,351 adult primary care patients in Switzerland, showed that ferritin cutoff values of 15, 30, and 45 ng/mL were associated with incidences of iron deficiency diagnoses of 10.9, 29.9, and 48.3 cases per 1000 patient-years, respectively. In other words, as the cutoff value increases, the frequency of diagnosis also increases.

“It is a study to take into account, especially because of the number of patients it includes, and it can guide primary care clinical practice. As expected, as the cutoff point increases with respect to ferritin values, the incidence percentages of both iron deficiency and iron-deficiency anemia also increase,” Miguel Turégano-Yedro, MD, a family physician at the Casar de Cáceres Health Center in Spain, and coordinator of the Hematology Working Group of the Spanish Society of Primary Care Physicians, told this news organization. Ferritin is the most sensitive parameter for diagnosing iron deficiency and iron-deficiency anemia, he added. “When it is necessary to supplement a patient with iron, other parameters are taken into account, such as hemoglobin, to see if there is anemia.”
 

Ferritin Level

The ferritin level associated with iron deficiency in primary care is usually 15 ng/mL, said Dr. Turégano-Yedro. “If we assess patients with a ferritin level of 15 or less than 15, then we know that many cases will be symptomatic (with fatigue, tiredness, or lack of appetite) and, therefore, will need iron treatment. But if the ferritin cutoff value is increased to 30 ng/mL or 45 ng/mL, the incidence will be higher, although in many cases they will be asymptomatic and iron supplementation will not be necessary.”

He also pointed out that he does not consider it necessary to raise the cutoff to 45 ng/mL; however, “establishing the cutoff at 30 ng/mL, in a certain population at risk of iron deficiency or iron-deficiency anemia, may be interesting, for example in women of childbearing age, women with very heavy menstruation, children, frail elderly, people with gastrointestinal bleeding, or those who engage in physical exercise.”

Iron deficiency must be distinguished from anemia. “If the ferritin is below 15 ng/mL, there is iron deficiency, which may or may not be accompanied by symptoms, although usually most patients will have symptoms. Normally, to diagnose a patient with iron-deficiency anemia, on the one hand, they must have low hemoglobin, which indicates anemia, and on the other hand, low ferritin, which indicates iron deficiency.” Taking these parameters into account, the study does have a weakness. “It is striking that a percentage of patients in the study requested ferritin analysis without including hemoglobin, when hemoglobin is part of the basic analysis performed in Spain,” said Dr. Turégano-Yedro.
 

When to Supplement

The study highlights the incidence of nonanemic iron deficiency diagnoses associated with the choice of ferritin cutoff value. However, as Dr. Turégano-Yedro explained, the percentage of patients who have iron deficiency but do not have anemia is not very relevant. “In the case of family physicians in Spain, it is not usually taken into account, because if a patient has iron deficiency with or without anemia and is symptomatic, they should be given iron supplements.”

What if they do not have a deficiency but do have anemia? “In principle, iron supplementation is not necessary, because that anemia may be due to chronic disorders or it may be hemolytic anemia, so the case should be studied,” Dr. Turégano-Yedro concluded.

This story was translated from Univadis Spain, which is part of the Medscape Professional Network, using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article appeared on Medscape.com.

Nearly one-third of US adults may have low iron levels that can add to problems ranging from fatigue to heart failure. 

Researchers in a new study estimated that 7% of US adults have anemia, a blood disorder that can be iron related and is particularly well-known in part because of screenings given during pregnancy. But more striking was the finding in this latest study that a significant portion of the population may have less severe iron deficiencies that have been linked to serious health problems.

The body gets iron from food, and it can store iron for times when there isn’t enough it can access right away. The research team looked at test results that show whether people have enough iron stored, as well as whether their bodies could effectively use available iron. If you don’t have enough iron stored, you have a condition called absolute iron deficiency. And if you have stored iron but problems using it, you have what’s called functional iron deficiency. The study found that an estimated 14% of adults have absolute iron deficiency, and another 15% have functional iron deficiency.

The findings, published in JAMA Network Open, are based on data from more than 8,000 people who had laboratory iron levels on file as part of the National Health and Nutrition Examination Survey that was done from 2017 to 2020.

Besides anemia, iron deficiency is linked to other serious health problems, including restless legs syndrome, mental and thinking difficulties, reduced physical abilities, and heart failure, the authors noted. The effects of iron deficiency can significantly impact a person’s quality of life.

Routine blood work as part of an annual physical doesn’t typically include a check of iron levels unless there is a cause for concern. The out-of-pocket cost without using insurance for a blood test to check iron levels is typically around $60. 

“This is a common yet underappreciated public health problem,” study author Leo Buckley, PharmD, MPH, a clinical pharmacology specialist at Brigham and Women’s Hospital in Boston, Massachusetts, told NBC News. “What’s unique about our study is we were looking at regular people who would not otherwise have been screened or tested.”

Treatment for low iron levels can include changes to your diet, as well as intravenous or oral supplements. Taking an iron supplement should be done under the guidance of a health care provider because of the risk of iron toxicity.

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

Nearly one-third of US adults may have low iron levels that can add to problems ranging from fatigue to heart failure. 

Researchers in a new study estimated that 7% of US adults have anemia, a blood disorder that can be iron related and is particularly well-known in part because of screenings given during pregnancy. But more striking was the finding in this latest study that a significant portion of the population may have less severe iron deficiencies that have been linked to serious health problems.

The body gets iron from food, and it can store iron for times when there isn’t enough it can access right away. The research team looked at test results that show whether people have enough iron stored, as well as whether their bodies could effectively use available iron. If you don’t have enough iron stored, you have a condition called absolute iron deficiency. And if you have stored iron but problems using it, you have what’s called functional iron deficiency. The study found that an estimated 14% of adults have absolute iron deficiency, and another 15% have functional iron deficiency.

The findings, published in JAMA Network Open, are based on data from more than 8,000 people who had laboratory iron levels on file as part of the National Health and Nutrition Examination Survey that was done from 2017 to 2020.

Besides anemia, iron deficiency is linked to other serious health problems, including restless legs syndrome, mental and thinking difficulties, reduced physical abilities, and heart failure, the authors noted. The effects of iron deficiency can significantly impact a person’s quality of life.

Routine blood work as part of an annual physical doesn’t typically include a check of iron levels unless there is a cause for concern. The out-of-pocket cost without using insurance for a blood test to check iron levels is typically around $60. 

“This is a common yet underappreciated public health problem,” study author Leo Buckley, PharmD, MPH, a clinical pharmacology specialist at Brigham and Women’s Hospital in Boston, Massachusetts, told NBC News. “What’s unique about our study is we were looking at regular people who would not otherwise have been screened or tested.”

Treatment for low iron levels can include changes to your diet, as well as intravenous or oral supplements. Taking an iron supplement should be done under the guidance of a health care provider because of the risk of iron toxicity.

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

Nearly one-third of US adults may have low iron levels that can add to problems ranging from fatigue to heart failure. 

Researchers in a new study estimated that 7% of US adults have anemia, a blood disorder that can be iron related and is particularly well-known in part because of screenings given during pregnancy. But more striking was the finding in this latest study that a significant portion of the population may have less severe iron deficiencies that have been linked to serious health problems.

The body gets iron from food, and it can store iron for times when there isn’t enough it can access right away. The research team looked at test results that show whether people have enough iron stored, as well as whether their bodies could effectively use available iron. If you don’t have enough iron stored, you have a condition called absolute iron deficiency. And if you have stored iron but problems using it, you have what’s called functional iron deficiency. The study found that an estimated 14% of adults have absolute iron deficiency, and another 15% have functional iron deficiency.

The findings, published in JAMA Network Open, are based on data from more than 8,000 people who had laboratory iron levels on file as part of the National Health and Nutrition Examination Survey that was done from 2017 to 2020.

Besides anemia, iron deficiency is linked to other serious health problems, including restless legs syndrome, mental and thinking difficulties, reduced physical abilities, and heart failure, the authors noted. The effects of iron deficiency can significantly impact a person’s quality of life.

Routine blood work as part of an annual physical doesn’t typically include a check of iron levels unless there is a cause for concern. The out-of-pocket cost without using insurance for a blood test to check iron levels is typically around $60. 

“This is a common yet underappreciated public health problem,” study author Leo Buckley, PharmD, MPH, a clinical pharmacology specialist at Brigham and Women’s Hospital in Boston, Massachusetts, told NBC News. “What’s unique about our study is we were looking at regular people who would not otherwise have been screened or tested.”

Treatment for low iron levels can include changes to your diet, as well as intravenous or oral supplements. Taking an iron supplement should be done under the guidance of a health care provider because of the risk of iron toxicity.

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

TOPLINE: 

Intravenous iron reduced iron deficiency more effectively than oral iron, which is often distasteful, among pregnant women in Nigeria. However, no significant difference was found in the prevalence of anemia or preterm birth between the two groups.

METHODOLOGY:

• A total of 1056 pregnant women aged 15-49 years with hemoglobin concentrations 10 g/dL at 20-32 weeks’ gestation were included in the trial.
• Participants were randomly assigned to receive either a single dose of intravenous ferric carboxymaltose (20 mg/kg to a maximum of 1000 mg) or oral ferrous sulphate (200 mg; 65 mg elemental iron) three times daily until 6 weeks postpartum.
• Primary outcomes were maternal anemia (hemoglobin, < 11 g/dL) at 36 weeks’ gestation and preterm birth before 37 weeks’ gestation.
• Secondary outcomes were iron deficiency, iron deficiency anemia, maternal depression, infections, immunization, and breastfeeding practices.
• The trial was conducted in 11 health facilities in Lagos and Kano, Nigeria, with follow-up visits at 2 weeks and 6 weeks postpartum.

TAKEAWAY:

• No significant difference was found in the prevalence of anemia at 36 weeks’ gestation between the intravenous and oral iron groups (58% vs 61%; P = .36).
• Intravenous iron was more effective at reducing iron deficiency (5% vs 16%; P = .0001) and iron deficiency anemia (2% vs 10%; P = .0001) at 36 weeks’ gestation.
• The incidence of preterm birth did not significantly differ between the intravenous and oral iron groups (14% vs 15%; P = .66).
• Intravenous iron led to a higher mean hemoglobin concentration from baseline to 4 weeks in both iron-deficient and non–iron-deficient subgroups.

IN PRACTICE:

“Although the effect on overall anaemia did not differ, intravenous iron reduced the prevalence of iron deficiency to a greater extent than oral iron and was considered to be safe. We recommend that intravenous iron be considered for anaemic pregnant women in Nigeria and similar settings,” wrote the authors of the study.

SOURCE:

This study was led by Bosede B. Afolabi, Department of Obstetrics and Gynaecology, Faculty of Clinical Sciences, College of Medicine, University of Lagos, Nigeria. It was published online in The Lancet Global Health.

LIMITATIONS:

The study’s sample size estimation assumed a 25% rate of preterm births, but the actual rate was only 14.5%, which potentially underpowered the study to measure this outcome. Most participants were enrolled after 20 weeks’ gestation, which limited the ability to explore the effect of treatment duration. The interpretation of postpartum hemorrhage was limited by the use of visual assessment to determine blood loss, which is subjective.

DISCLOSURES:

A coathor, Kristi S. Annerstedt, PhD, reported participation on the ALERT project Data Safety Monitoring Board. Additional disclosures are noted in the original article. The study was supported by grants from the Bill & Melinda Gates Foundation.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE: 

Intravenous iron reduced iron deficiency more effectively than oral iron, which is often distasteful, among pregnant women in Nigeria. However, no significant difference was found in the prevalence of anemia or preterm birth between the two groups.

METHODOLOGY:

• A total of 1056 pregnant women aged 15-49 years with hemoglobin concentrations 10 g/dL at 20-32 weeks’ gestation were included in the trial.
• Participants were randomly assigned to receive either a single dose of intravenous ferric carboxymaltose (20 mg/kg to a maximum of 1000 mg) or oral ferrous sulphate (200 mg; 65 mg elemental iron) three times daily until 6 weeks postpartum.
• Primary outcomes were maternal anemia (hemoglobin, < 11 g/dL) at 36 weeks’ gestation and preterm birth before 37 weeks’ gestation.
• Secondary outcomes were iron deficiency, iron deficiency anemia, maternal depression, infections, immunization, and breastfeeding practices.
• The trial was conducted in 11 health facilities in Lagos and Kano, Nigeria, with follow-up visits at 2 weeks and 6 weeks postpartum.

TAKEAWAY:

• No significant difference was found in the prevalence of anemia at 36 weeks’ gestation between the intravenous and oral iron groups (58% vs 61%; P = .36).
• Intravenous iron was more effective at reducing iron deficiency (5% vs 16%; P = .0001) and iron deficiency anemia (2% vs 10%; P = .0001) at 36 weeks’ gestation.
• The incidence of preterm birth did not significantly differ between the intravenous and oral iron groups (14% vs 15%; P = .66).
• Intravenous iron led to a higher mean hemoglobin concentration from baseline to 4 weeks in both iron-deficient and non–iron-deficient subgroups.

IN PRACTICE:

“Although the effect on overall anaemia did not differ, intravenous iron reduced the prevalence of iron deficiency to a greater extent than oral iron and was considered to be safe. We recommend that intravenous iron be considered for anaemic pregnant women in Nigeria and similar settings,” wrote the authors of the study.

SOURCE:

This study was led by Bosede B. Afolabi, Department of Obstetrics and Gynaecology, Faculty of Clinical Sciences, College of Medicine, University of Lagos, Nigeria. It was published online in The Lancet Global Health.

LIMITATIONS:

The study’s sample size estimation assumed a 25% rate of preterm births, but the actual rate was only 14.5%, which potentially underpowered the study to measure this outcome. Most participants were enrolled after 20 weeks’ gestation, which limited the ability to explore the effect of treatment duration. The interpretation of postpartum hemorrhage was limited by the use of visual assessment to determine blood loss, which is subjective.

DISCLOSURES:

A coathor, Kristi S. Annerstedt, PhD, reported participation on the ALERT project Data Safety Monitoring Board. Additional disclosures are noted in the original article. The study was supported by grants from the Bill & Melinda Gates Foundation.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

TOPLINE: 

Intravenous iron reduced iron deficiency more effectively than oral iron, which is often distasteful, among pregnant women in Nigeria. However, no significant difference was found in the prevalence of anemia or preterm birth between the two groups.

METHODOLOGY:

• A total of 1056 pregnant women aged 15-49 years with hemoglobin concentrations 10 g/dL at 20-32 weeks’ gestation were included in the trial.
• Participants were randomly assigned to receive either a single dose of intravenous ferric carboxymaltose (20 mg/kg to a maximum of 1000 mg) or oral ferrous sulphate (200 mg; 65 mg elemental iron) three times daily until 6 weeks postpartum.
• Primary outcomes were maternal anemia (hemoglobin, < 11 g/dL) at 36 weeks’ gestation and preterm birth before 37 weeks’ gestation.
• Secondary outcomes were iron deficiency, iron deficiency anemia, maternal depression, infections, immunization, and breastfeeding practices.
• The trial was conducted in 11 health facilities in Lagos and Kano, Nigeria, with follow-up visits at 2 weeks and 6 weeks postpartum.

TAKEAWAY:

• No significant difference was found in the prevalence of anemia at 36 weeks’ gestation between the intravenous and oral iron groups (58% vs 61%; P = .36).
• Intravenous iron was more effective at reducing iron deficiency (5% vs 16%; P = .0001) and iron deficiency anemia (2% vs 10%; P = .0001) at 36 weeks’ gestation.
• The incidence of preterm birth did not significantly differ between the intravenous and oral iron groups (14% vs 15%; P = .66).
• Intravenous iron led to a higher mean hemoglobin concentration from baseline to 4 weeks in both iron-deficient and non–iron-deficient subgroups.

IN PRACTICE:

“Although the effect on overall anaemia did not differ, intravenous iron reduced the prevalence of iron deficiency to a greater extent than oral iron and was considered to be safe. We recommend that intravenous iron be considered for anaemic pregnant women in Nigeria and similar settings,” wrote the authors of the study.

SOURCE:

This study was led by Bosede B. Afolabi, Department of Obstetrics and Gynaecology, Faculty of Clinical Sciences, College of Medicine, University of Lagos, Nigeria. It was published online in The Lancet Global Health.

LIMITATIONS:

The study’s sample size estimation assumed a 25% rate of preterm births, but the actual rate was only 14.5%, which potentially underpowered the study to measure this outcome. Most participants were enrolled after 20 weeks’ gestation, which limited the ability to explore the effect of treatment duration. The interpretation of postpartum hemorrhage was limited by the use of visual assessment to determine blood loss, which is subjective.

DISCLOSURES:

A coathor, Kristi S. Annerstedt, PhD, reported participation on the ALERT project Data Safety Monitoring Board. Additional disclosures are noted in the original article. The study was supported by grants from the Bill & Melinda Gates Foundation.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication. A version of this article first appeared on Medscape.com.

Three different definitions of nonanemic iron deficiency (ID), a common disorder causing substantial morbidity in women, were significantly associated with different population prevalence estimates, a data analysis of the cross-sectional Hemochromatosis and Iron Overload Screening Study (HEIRS) study found.

These differences held, regardless of self-reported age, pregnancy, or race and ethnicity, according to HEIRS researchers led by James C. Barton, MD, professor of hematology at the University of Alabama at Birmingham.

“Using higher serum ferritin thresholds to define ID could lead to diagnosis and treatment of more women with ID and greater reduction of related morbidity,” the investigators wrote. The study was published in JAMA Network Open.

The authors noted that ID affects about 2 billion people worldwide, mainly women and children, increasing risks of fatigue, impaired muscular performance, cold intolerance, mucosal and epithelial abnormalities, pica, disturbances of menstruation, and adverse pregnancy outcomes.

Manifestations of ID, including anemia, are less prevalent or less severe in adults with higher serum ferritin (SF), and the three definitions correspond, in sequence, to ID of increasing prevalence and decreasing severity, they explained.
 

The Study

HEIRS conducted multiethnic, primary care–based screening for iron disorders during 2001-2003 at four field centers in the United States and one in Canada at primary care venues.

In data for the current study analyzed from June to December, 2023, the three ID definitions were: combined transferrin saturation less than 10% and SF less than 15 ng/mL (HEIRS); SF less than 15 ng/mL (World Health Organization [WHO]); and SF less than 25 ng/mL, the threshold for ID-deficient erythropoiesis [IDE).

Among the cohort’s 62,685 women (mean age, 49.58 years, 27,072 White, 17,272 Black), the estimated prevalence of ID emerged as follows across the different definitions:

• 1957 (3.12%) according to HEIRS
• 4659 (7.43%) according to WHO
• 9611 (15.33%) according to IDE

Those figures translated to an increased relative ID prevalence of 2.4-fold (95% CI, 2.3-2.5; P < .001) according to the WHO standard and 4.9-fold (95% CI, 4.7-5.2; P < .001) according IDEs.

In addition, prevalence was higher in younger women, and within each racial and ethnic subgroup of participants aged 25-54 years, prevalence rose significantly from the HEIRS definition to the WHO and IDE definitions.

Notably, ID was significantly higher among Black and Hispanic participants than Asian and White participants.

An accompanying editorial pointed to gender-based health equity issues raised by the HEIRS analysis and argued that a similar passive acceptance of laboratory definitions of a debilitating but correctable condition in White males would be “frankly unimaginable.”

“Iron deficiency is the leading cause of years lived with disability among women of reproductive age,” wrote hematologist Michelle Sholzberg, MDCM, MSc, and Grace H. Tang, MSc, of St. Michael’s Hospital in Toronto, Canada. “It is a factor clearly associated with maternal death and morbidity (including diminished IQ), and it is correctable, and, thus, unnecessary, in high-income, middle-income, and low-income geographic settings.”

The authors listed no specific funding for this analysis of HEIRS data. Dr. Barton reported contracts from the National Institutes of Health, National Human Genome Research Institute, outside of the submitted work. A coauthor reported grants from the National Heart, Lung, and Blood Institute and the National Human Genome Research Institute outside of the submitted work. Dr. Sholzberg reported unrestricted research funding to her institution from Octapharma and Pfizer and speakers’ honoraria from Takeda, Sobi, and Medison outside of the submitted work.

Three different definitions of nonanemic iron deficiency (ID), a common disorder causing substantial morbidity in women, were significantly associated with different population prevalence estimates, a data analysis of the cross-sectional Hemochromatosis and Iron Overload Screening Study (HEIRS) study found.

These differences held, regardless of self-reported age, pregnancy, or race and ethnicity, according to HEIRS researchers led by James C. Barton, MD, professor of hematology at the University of Alabama at Birmingham.

“Using higher serum ferritin thresholds to define ID could lead to diagnosis and treatment of more women with ID and greater reduction of related morbidity,” the investigators wrote. The study was published in JAMA Network Open.

The authors noted that ID affects about 2 billion people worldwide, mainly women and children, increasing risks of fatigue, impaired muscular performance, cold intolerance, mucosal and epithelial abnormalities, pica, disturbances of menstruation, and adverse pregnancy outcomes.

Manifestations of ID, including anemia, are less prevalent or less severe in adults with higher serum ferritin (SF), and the three definitions correspond, in sequence, to ID of increasing prevalence and decreasing severity, they explained.
 

The Study

HEIRS conducted multiethnic, primary care–based screening for iron disorders during 2001-2003 at four field centers in the United States and one in Canada at primary care venues.

In data for the current study analyzed from June to December, 2023, the three ID definitions were: combined transferrin saturation less than 10% and SF less than 15 ng/mL (HEIRS); SF less than 15 ng/mL (World Health Organization [WHO]); and SF less than 25 ng/mL, the threshold for ID-deficient erythropoiesis [IDE).

Among the cohort’s 62,685 women (mean age, 49.58 years, 27,072 White, 17,272 Black), the estimated prevalence of ID emerged as follows across the different definitions:

• 1957 (3.12%) according to HEIRS
• 4659 (7.43%) according to WHO
• 9611 (15.33%) according to IDE

Those figures translated to an increased relative ID prevalence of 2.4-fold (95% CI, 2.3-2.5; P < .001) according to the WHO standard and 4.9-fold (95% CI, 4.7-5.2; P < .001) according IDEs.

In addition, prevalence was higher in younger women, and within each racial and ethnic subgroup of participants aged 25-54 years, prevalence rose significantly from the HEIRS definition to the WHO and IDE definitions.

Notably, ID was significantly higher among Black and Hispanic participants than Asian and White participants.

An accompanying editorial pointed to gender-based health equity issues raised by the HEIRS analysis and argued that a similar passive acceptance of laboratory definitions of a debilitating but correctable condition in White males would be “frankly unimaginable.”

“Iron deficiency is the leading cause of years lived with disability among women of reproductive age,” wrote hematologist Michelle Sholzberg, MDCM, MSc, and Grace H. Tang, MSc, of St. Michael’s Hospital in Toronto, Canada. “It is a factor clearly associated with maternal death and morbidity (including diminished IQ), and it is correctable, and, thus, unnecessary, in high-income, middle-income, and low-income geographic settings.”

The authors listed no specific funding for this analysis of HEIRS data. Dr. Barton reported contracts from the National Institutes of Health, National Human Genome Research Institute, outside of the submitted work. A coauthor reported grants from the National Heart, Lung, and Blood Institute and the National Human Genome Research Institute outside of the submitted work. Dr. Sholzberg reported unrestricted research funding to her institution from Octapharma and Pfizer and speakers’ honoraria from Takeda, Sobi, and Medison outside of the submitted work.

Three different definitions of nonanemic iron deficiency (ID), a common disorder causing substantial morbidity in women, were significantly associated with different population prevalence estimates, a data analysis of the cross-sectional Hemochromatosis and Iron Overload Screening Study (HEIRS) study found.

These differences held, regardless of self-reported age, pregnancy, or race and ethnicity, according to HEIRS researchers led by James C. Barton, MD, professor of hematology at the University of Alabama at Birmingham.

“Using higher serum ferritin thresholds to define ID could lead to diagnosis and treatment of more women with ID and greater reduction of related morbidity,” the investigators wrote. The study was published in JAMA Network Open.

The authors noted that ID affects about 2 billion people worldwide, mainly women and children, increasing risks of fatigue, impaired muscular performance, cold intolerance, mucosal and epithelial abnormalities, pica, disturbances of menstruation, and adverse pregnancy outcomes.

Manifestations of ID, including anemia, are less prevalent or less severe in adults with higher serum ferritin (SF), and the three definitions correspond, in sequence, to ID of increasing prevalence and decreasing severity, they explained.
 

The Study

HEIRS conducted multiethnic, primary care–based screening for iron disorders during 2001-2003 at four field centers in the United States and one in Canada at primary care venues.

In data for the current study analyzed from June to December, 2023, the three ID definitions were: combined transferrin saturation less than 10% and SF less than 15 ng/mL (HEIRS); SF less than 15 ng/mL (World Health Organization [WHO]); and SF less than 25 ng/mL, the threshold for ID-deficient erythropoiesis [IDE).

Among the cohort’s 62,685 women (mean age, 49.58 years, 27,072 White, 17,272 Black), the estimated prevalence of ID emerged as follows across the different definitions:

• 1957 (3.12%) according to HEIRS
• 4659 (7.43%) according to WHO
• 9611 (15.33%) according to IDE

Those figures translated to an increased relative ID prevalence of 2.4-fold (95% CI, 2.3-2.5; P < .001) according to the WHO standard and 4.9-fold (95% CI, 4.7-5.2; P < .001) according IDEs.

In addition, prevalence was higher in younger women, and within each racial and ethnic subgroup of participants aged 25-54 years, prevalence rose significantly from the HEIRS definition to the WHO and IDE definitions.

Notably, ID was significantly higher among Black and Hispanic participants than Asian and White participants.

An accompanying editorial pointed to gender-based health equity issues raised by the HEIRS analysis and argued that a similar passive acceptance of laboratory definitions of a debilitating but correctable condition in White males would be “frankly unimaginable.”

“Iron deficiency is the leading cause of years lived with disability among women of reproductive age,” wrote hematologist Michelle Sholzberg, MDCM, MSc, and Grace H. Tang, MSc, of St. Michael’s Hospital in Toronto, Canada. “It is a factor clearly associated with maternal death and morbidity (including diminished IQ), and it is correctable, and, thus, unnecessary, in high-income, middle-income, and low-income geographic settings.”

The authors listed no specific funding for this analysis of HEIRS data. Dr. Barton reported contracts from the National Institutes of Health, National Human Genome Research Institute, outside of the submitted work. A coauthor reported grants from the National Heart, Lung, and Blood Institute and the National Human Genome Research Institute outside of the submitted work. Dr. Sholzberg reported unrestricted research funding to her institution from Octapharma and Pfizer and speakers’ honoraria from Takeda, Sobi, and Medison outside of the submitted work.

TOPLINE:

Patients with early-onset colorectal cancer (EOCRC) often present with hematochezia or abdominal pain, symptoms frequently overlooked in younger populations, leading to delays in diagnosis of 4-6 months, a new analysis showed.

METHODOLOGY:

• As the number of cases of EOCRC, defined as colorectal cancer (CRC) diagnosed before age 50, continues to rise, early detection has become increasingly important. Improved recognition of presenting signs and symptoms associated with EOCRC could lead to a more timely diagnosis and better clinical outcomes.
• In a systematic review and meta-analysis of 81 studies with 24.9 million EOCRC cases, researchers sought to determine the most common presenting signs and symptoms, their association with EOCRC risk, and the time from presentation to diagnosis.
• Data extraction and quality assessment were performed independently in duplicate using PRISMA guidelines, and Joanna Briggs Institute critical appraisal tools were used to measure the risk of bias.

TAKEAWAY:

• Hematochezia was the most common presenting sign/symptom, with a pooled prevalence of 45%, followed by abdominal pain, with a pooled prevalence of 40%.
• Altered bowel habits, which included constipation, diarrhea, and alternating bowel habits, were the third most common presenting sign/symptom (pooled prevalence of 27%), followed by unexplained weight loss (pooled prevalence of 17%).
• The likelihood of EOCRC was estimated to be fivefold to 54-fold higher with hematochezia and 1.3-fold to sixfold higher with abdominal pain.
• The mean time from sign or symptom onset to EOCRC diagnosis was 6.4 months (range, 1.8-13.7 months).

IN PRACTICE:

“These findings and the increasing risk of CRC in individuals younger than 50 years highlight the urgent need to educate clinicians and patients about these signs and symptoms to ensure that diagnostic workup and resolution are not delayed. Adapting current clinical practice to identify and address these signs and symptoms through careful clinical triage and follow-up could help limit morbidity and mortality associated with EOCRC,” the authors wrote.

SOURCE:

The study, with Joshua Demb, PhD, MPH, division of gastroenterology, department of medicine, University of California, San Diego, was published online May 24 in JAMA Network Open.

LIMITATIONS:

Significant heterogeneity across studies affected the ability to meta-analyze some results. The cross-sectional data limited the ability to stratify by age, sex, race and ethnicity, or genetic ancestry. It was not possible to evaluate the impact of time to diagnosis on CRC outcomes due to a limited number of studies answering this question. Researchers were unable to examine the constellation of signs and symptoms because they lacked individual-level data from each study.

DISCLOSURES:

The authors disclosed no relevant conflicts of interest. No specific funding was disclosed.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Patients with early-onset colorectal cancer (EOCRC) often present with hematochezia or abdominal pain, symptoms frequently overlooked in younger populations, leading to delays in diagnosis of 4-6 months, a new analysis showed.

METHODOLOGY:

• As the number of cases of EOCRC, defined as colorectal cancer (CRC) diagnosed before age 50, continues to rise, early detection has become increasingly important. Improved recognition of presenting signs and symptoms associated with EOCRC could lead to a more timely diagnosis and better clinical outcomes.
• In a systematic review and meta-analysis of 81 studies with 24.9 million EOCRC cases, researchers sought to determine the most common presenting signs and symptoms, their association with EOCRC risk, and the time from presentation to diagnosis.
• Data extraction and quality assessment were performed independently in duplicate using PRISMA guidelines, and Joanna Briggs Institute critical appraisal tools were used to measure the risk of bias.

TAKEAWAY:

• Hematochezia was the most common presenting sign/symptom, with a pooled prevalence of 45%, followed by abdominal pain, with a pooled prevalence of 40%.
• Altered bowel habits, which included constipation, diarrhea, and alternating bowel habits, were the third most common presenting sign/symptom (pooled prevalence of 27%), followed by unexplained weight loss (pooled prevalence of 17%).
• The likelihood of EOCRC was estimated to be fivefold to 54-fold higher with hematochezia and 1.3-fold to sixfold higher with abdominal pain.
• The mean time from sign or symptom onset to EOCRC diagnosis was 6.4 months (range, 1.8-13.7 months).

IN PRACTICE:

“These findings and the increasing risk of CRC in individuals younger than 50 years highlight the urgent need to educate clinicians and patients about these signs and symptoms to ensure that diagnostic workup and resolution are not delayed. Adapting current clinical practice to identify and address these signs and symptoms through careful clinical triage and follow-up could help limit morbidity and mortality associated with EOCRC,” the authors wrote.

SOURCE:

The study, with Joshua Demb, PhD, MPH, division of gastroenterology, department of medicine, University of California, San Diego, was published online May 24 in JAMA Network Open.

LIMITATIONS:

Significant heterogeneity across studies affected the ability to meta-analyze some results. The cross-sectional data limited the ability to stratify by age, sex, race and ethnicity, or genetic ancestry. It was not possible to evaluate the impact of time to diagnosis on CRC outcomes due to a limited number of studies answering this question. Researchers were unable to examine the constellation of signs and symptoms because they lacked individual-level data from each study.

DISCLOSURES:

The authors disclosed no relevant conflicts of interest. No specific funding was disclosed.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Patients with early-onset colorectal cancer (EOCRC) often present with hematochezia or abdominal pain, symptoms frequently overlooked in younger populations, leading to delays in diagnosis of 4-6 months, a new analysis showed.

METHODOLOGY:

• As the number of cases of EOCRC, defined as colorectal cancer (CRC) diagnosed before age 50, continues to rise, early detection has become increasingly important. Improved recognition of presenting signs and symptoms associated with EOCRC could lead to a more timely diagnosis and better clinical outcomes.
• In a systematic review and meta-analysis of 81 studies with 24.9 million EOCRC cases, researchers sought to determine the most common presenting signs and symptoms, their association with EOCRC risk, and the time from presentation to diagnosis.
• Data extraction and quality assessment were performed independently in duplicate using PRISMA guidelines, and Joanna Briggs Institute critical appraisal tools were used to measure the risk of bias.

TAKEAWAY:

• Hematochezia was the most common presenting sign/symptom, with a pooled prevalence of 45%, followed by abdominal pain, with a pooled prevalence of 40%.
• Altered bowel habits, which included constipation, diarrhea, and alternating bowel habits, were the third most common presenting sign/symptom (pooled prevalence of 27%), followed by unexplained weight loss (pooled prevalence of 17%).
• The likelihood of EOCRC was estimated to be fivefold to 54-fold higher with hematochezia and 1.3-fold to sixfold higher with abdominal pain.
• The mean time from sign or symptom onset to EOCRC diagnosis was 6.4 months (range, 1.8-13.7 months).

IN PRACTICE:

“These findings and the increasing risk of CRC in individuals younger than 50 years highlight the urgent need to educate clinicians and patients about these signs and symptoms to ensure that diagnostic workup and resolution are not delayed. Adapting current clinical practice to identify and address these signs and symptoms through careful clinical triage and follow-up could help limit morbidity and mortality associated with EOCRC,” the authors wrote.

SOURCE:

The study, with Joshua Demb, PhD, MPH, division of gastroenterology, department of medicine, University of California, San Diego, was published online May 24 in JAMA Network Open.

LIMITATIONS:

Significant heterogeneity across studies affected the ability to meta-analyze some results. The cross-sectional data limited the ability to stratify by age, sex, race and ethnicity, or genetic ancestry. It was not possible to evaluate the impact of time to diagnosis on CRC outcomes due to a limited number of studies answering this question. Researchers were unable to examine the constellation of signs and symptoms because they lacked individual-level data from each study.

DISCLOSURES:

The authors disclosed no relevant conflicts of interest. No specific funding was disclosed.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Secondary cancers were flagged in 4.3% of all adverse event reports among patients who received CAR T therapy, with T-cell malignancies comprising only 0.15% of the total reports and 3.54% of all second primary malignancies, according to an analysis of adverse event reports submitted to the US Food and Drug Administration (FDA).

METHODOLOGY:

• In November 2023, the FDA announced its investigation into whether chimeric antigen receptor (CAR) T-cell immunotherapies can cause secondary blood cancers, specifically T-cell malignancies. At the time, the agency said: “Although the overall benefits of these products continue to outweigh their potential risks for their approved uses, FDA is investigating the identified risk of T-cell malignancy with serious outcomes.”
• In January 2024, the FDA issued boxed warnings on the six approved CART cell therapies, citing the possibility of second primary malignancies, including CAR-positive lymphomas, in patients who had received a CAR T agent.
• To evaluate the extent of these secondary cancers, researchers analyzed the FDA Adverse Event Reporting System database for CAR T-cell reports citing second primary malignancies.

TAKEAWAY:

• Overall, the authors identified 12,394 unique adverse events associated with CAR T therapy; of these, 536 adverse events (4.3%) were second primary malignancies.
• Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tis-cel) accounted for most of the second primary malignancies reports — 51.7% (277 of 536 patients) for axi-cel and 33% (177 of 536 patients) for tis-cel.
• The researchers identified 19 cases of T-cell malignancies, representing only 0.15% of all unique adverse events and 3.54% of all second primary malignancies (19 of 536 patients); 17 of these cases were T-cell non-Hodgkin lymphomas, and two were T-cell large granular lymphocytic leukemia.
• Among the reported 536 second primary malignancies, the most frequent cancers were leukemias (333 reports, or 62%), followed by skin neoplasms (54 reports, or 10.1%), hematopoietic neoplasms excluding leukemias and lymphomas (26 reports, 4.85%), nervous system tumors (21 reports, 3.92%), and respiratory neoplasms (20 reports, 3.73%).

IN PRACTICE:

“We will continue to monitor the data released by the FDA to learn more about CAR T-associated risks. However, it’s crucial to stress that the benefits of CAR T-cell therapies still outweigh the risks for the approved indications,” Magdi Elsallab, MD, the study’s co-lead author, said in a news release.

SOURCE:

This work, led by Dr. Elsallab from Harvard Medical School in Boston, was published online on March 14 in Blood.

LIMITATIONS:

The limitations of the analysis include the presence of duplicate report submissions, incomplete data, difficulty establishing causal relationships, and the potential for both underreporting and overreporting based on the severity of adverse events. Furthermore, without the total number of prescribed products, it was difficult to determine the adverse event frequency.

DISCLOSURES:

The study funding source was not disclosed. Some of the authors reported financial ties with various organizations outside this work, including Bristol Myers Squibb, Janssen Biotech, Johnson & Johnson, Kite Pharma, and Novartis.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Secondary cancers were flagged in 4.3% of all adverse event reports among patients who received CAR T therapy, with T-cell malignancies comprising only 0.15% of the total reports and 3.54% of all second primary malignancies, according to an analysis of adverse event reports submitted to the US Food and Drug Administration (FDA).

METHODOLOGY:

• In November 2023, the FDA announced its investigation into whether chimeric antigen receptor (CAR) T-cell immunotherapies can cause secondary blood cancers, specifically T-cell malignancies. At the time, the agency said: “Although the overall benefits of these products continue to outweigh their potential risks for their approved uses, FDA is investigating the identified risk of T-cell malignancy with serious outcomes.”
• In January 2024, the FDA issued boxed warnings on the six approved CART cell therapies, citing the possibility of second primary malignancies, including CAR-positive lymphomas, in patients who had received a CAR T agent.
• To evaluate the extent of these secondary cancers, researchers analyzed the FDA Adverse Event Reporting System database for CAR T-cell reports citing second primary malignancies.

TAKEAWAY:

• Overall, the authors identified 12,394 unique adverse events associated with CAR T therapy; of these, 536 adverse events (4.3%) were second primary malignancies.
• Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tis-cel) accounted for most of the second primary malignancies reports — 51.7% (277 of 536 patients) for axi-cel and 33% (177 of 536 patients) for tis-cel.
• The researchers identified 19 cases of T-cell malignancies, representing only 0.15% of all unique adverse events and 3.54% of all second primary malignancies (19 of 536 patients); 17 of these cases were T-cell non-Hodgkin lymphomas, and two were T-cell large granular lymphocytic leukemia.
• Among the reported 536 second primary malignancies, the most frequent cancers were leukemias (333 reports, or 62%), followed by skin neoplasms (54 reports, or 10.1%), hematopoietic neoplasms excluding leukemias and lymphomas (26 reports, 4.85%), nervous system tumors (21 reports, 3.92%), and respiratory neoplasms (20 reports, 3.73%).

IN PRACTICE:

“We will continue to monitor the data released by the FDA to learn more about CAR T-associated risks. However, it’s crucial to stress that the benefits of CAR T-cell therapies still outweigh the risks for the approved indications,” Magdi Elsallab, MD, the study’s co-lead author, said in a news release.

SOURCE:

This work, led by Dr. Elsallab from Harvard Medical School in Boston, was published online on March 14 in Blood.

LIMITATIONS:

The limitations of the analysis include the presence of duplicate report submissions, incomplete data, difficulty establishing causal relationships, and the potential for both underreporting and overreporting based on the severity of adverse events. Furthermore, without the total number of prescribed products, it was difficult to determine the adverse event frequency.

DISCLOSURES:

The study funding source was not disclosed. Some of the authors reported financial ties with various organizations outside this work, including Bristol Myers Squibb, Janssen Biotech, Johnson & Johnson, Kite Pharma, and Novartis.
 

A version of this article appeared on Medscape.com.

TOPLINE:

Secondary cancers were flagged in 4.3% of all adverse event reports among patients who received CAR T therapy, with T-cell malignancies comprising only 0.15% of the total reports and 3.54% of all second primary malignancies, according to an analysis of adverse event reports submitted to the US Food and Drug Administration (FDA).

METHODOLOGY:

• In November 2023, the FDA announced its investigation into whether chimeric antigen receptor (CAR) T-cell immunotherapies can cause secondary blood cancers, specifically T-cell malignancies. At the time, the agency said: “Although the overall benefits of these products continue to outweigh their potential risks for their approved uses, FDA is investigating the identified risk of T-cell malignancy with serious outcomes.”
• In January 2024, the FDA issued boxed warnings on the six approved CART cell therapies, citing the possibility of second primary malignancies, including CAR-positive lymphomas, in patients who had received a CAR T agent.
• To evaluate the extent of these secondary cancers, researchers analyzed the FDA Adverse Event Reporting System database for CAR T-cell reports citing second primary malignancies.

TAKEAWAY:

• Overall, the authors identified 12,394 unique adverse events associated with CAR T therapy; of these, 536 adverse events (4.3%) were second primary malignancies.
• Axicabtagene ciloleucel (axi-cel) and tisagenlecleucel (tis-cel) accounted for most of the second primary malignancies reports — 51.7% (277 of 536 patients) for axi-cel and 33% (177 of 536 patients) for tis-cel.
• The researchers identified 19 cases of T-cell malignancies, representing only 0.15% of all unique adverse events and 3.54% of all second primary malignancies (19 of 536 patients); 17 of these cases were T-cell non-Hodgkin lymphomas, and two were T-cell large granular lymphocytic leukemia.
• Among the reported 536 second primary malignancies, the most frequent cancers were leukemias (333 reports, or 62%), followed by skin neoplasms (54 reports, or 10.1%), hematopoietic neoplasms excluding leukemias and lymphomas (26 reports, 4.85%), nervous system tumors (21 reports, 3.92%), and respiratory neoplasms (20 reports, 3.73%).

IN PRACTICE:

“We will continue to monitor the data released by the FDA to learn more about CAR T-associated risks. However, it’s crucial to stress that the benefits of CAR T-cell therapies still outweigh the risks for the approved indications,” Magdi Elsallab, MD, the study’s co-lead author, said in a news release.

SOURCE:

This work, led by Dr. Elsallab from Harvard Medical School in Boston, was published online on March 14 in Blood.

LIMITATIONS:

The limitations of the analysis include the presence of duplicate report submissions, incomplete data, difficulty establishing causal relationships, and the potential for both underreporting and overreporting based on the severity of adverse events. Furthermore, without the total number of prescribed products, it was difficult to determine the adverse event frequency.

DISCLOSURES:

The study funding source was not disclosed. Some of the authors reported financial ties with various organizations outside this work, including Bristol Myers Squibb, Janssen Biotech, Johnson & Johnson, Kite Pharma, and Novartis.
 

A version of this article appeared on Medscape.com.

SAN DIEGO — The average age of death for Black U.S. patients with sickle cell disease (SCD) rose from 39 years (females = 40, males = 38) in 1999-2009 to 43 years (females = 44, males = 41) in 2010-2020, reflecting improvements in treatment, a new study finds.

But the news is not all positive. Mortality rates still jumped markedly as patients transitioned from pediatric to adult care, lead author Kristine A. Karkoska, MD, a pediatric hematology/oncologist with the University of Cincinnati College of Medicine, said at the annual meeting of the American Society of Hematology.

“This reflects that young adults are getting lost to care, and then they’re presenting with acute, life-threatening complications,” she said. “We still need more emphasis on comprehensive lifetime sickle-cell care and the transition to adult clinics to improve mortality in young adults.”

According to Dr. Karkoska, researchers launched the analysis of sickle-cell mortality rates to update previously available data up to the year 2009, which showed improvements as current standard-of-care treatments were introduced. Updated numbers, she said, would reflect the influence of a rise in dedicated SCD clinics and a 2014 National Heart, Lung, and Blood Institute recommendation that all children with SCD be treated with hydroxyurea starting at 9 months.

For the study, Dr. Karkoska and colleagues analyzed mortality statistics from the period of 1979-2020 via a CDC database. They found that 5272 Black patients died of SCD from 2010 to 2020. The crude mortality rate was 1.1 per 100,000 Black people, lower than the 1.2 per 100,000 rate of 1999-2009 (P < .0001).

The researchers also found that from 2010 to 2020, the mortality rate jumped for patients in the 15-19 to 20-24 age group: It rose from 0.9 per 100,000 to 1.4 per 100,000, P < .0001).

The researchers also examined contributors to death other than SCD. In 39% of cases, underlying causes were noted: cardiovascular disease (28%), accidents (7%), cerebrovascular disease (7%), malignancy (6%), septicemia (4.8%), and renal disease (3.8%). The population of people with SCD is “getting older, and they’re developing a combination of both sickle-related chronic organ damage as well as non-sickle-related chronic disease,” Dr. Karkoska said.

She noted that limitations include a reliance on data that can be incomplete or inaccurate. She also mentioned that the study only focuses on Black patients, who make up the vast majority of those with SCD.

How good is the news about improved mortality numbers? One member of the audience at the ASH presentation was disappointed that they hadn’t gotten even better. “I was hoping to come here to be cheered up,” he said, “and I’m not.”

Three physicians who didn’t take part in the research but are familiar with the new study spoke in interviews about the findings.

Michael Bender, MD, PhD, director of the Odessa Brown Comprehensive Sickle Cell Clinic in Seattle, pointed out that mortality rates improve slowly over time, as new treatments enter the picture. When new therapies come along, he said, “it’s tough if someone’s already 40 years old and their body has gone through a lot. They’re not going to have as much benefit as someone who started [on therapy] when they were 5 years old, and they grew up with that improvement.”

Sickle cell specialist Asmaa Ferdjallah, MD, MPH, of the Mayo Clinic in Rochester, Minnesota, said that the data showing a spike in mortality rates during the pediatric-adult transition are not surprising but still “really hard to digest.”

“It is a testament to the fact that we are not meeting patients where they are,” she said. “We struggle immensely with the transition period. This is something that is difficult across all providers all over the country,” she said. “There are different ways to ensure a successful transition from the pediatric side to the adult side. Here at Mayo Clinic, we use a slow transition, and we rotate appointments with peds and adults until age 30.”

Sophie Miriam Lanzkron, MD, MHS, director of the Sickle Cell Center for Adults at Johns Hopkins Hospital, Baltimore, said increases in mortality in the post-pediatric period appear to be due in part to “lack of access to high-quality sickle cell care for adults because there aren’t enough hematologists.” Worsening disease due to aging is another factor, she said, and “there might also be some behavioral changes. Young people think they will live forever. Sometimes they choose not to adhere to medical recommendations, which for this population is very risky.”

Dr. Lanzkron said her team is developing a long-term patient registry that should provide more insight.

No study funding was reported. Dr. Karkoska had no disclosures. The other coauthor disclosed research funding and safety advisory board relationships with Novartis. Dr. Ferdjallah, Dr. Lanzkron, and Dr. Bender reported no disclosures.

SAN DIEGO — The average age of death for Black U.S. patients with sickle cell disease (SCD) rose from 39 years (females = 40, males = 38) in 1999-2009 to 43 years (females = 44, males = 41) in 2010-2020, reflecting improvements in treatment, a new study finds.

But the news is not all positive. Mortality rates still jumped markedly as patients transitioned from pediatric to adult care, lead author Kristine A. Karkoska, MD, a pediatric hematology/oncologist with the University of Cincinnati College of Medicine, said at the annual meeting of the American Society of Hematology.

“This reflects that young adults are getting lost to care, and then they’re presenting with acute, life-threatening complications,” she said. “We still need more emphasis on comprehensive lifetime sickle-cell care and the transition to adult clinics to improve mortality in young adults.”

According to Dr. Karkoska, researchers launched the analysis of sickle-cell mortality rates to update previously available data up to the year 2009, which showed improvements as current standard-of-care treatments were introduced. Updated numbers, she said, would reflect the influence of a rise in dedicated SCD clinics and a 2014 National Heart, Lung, and Blood Institute recommendation that all children with SCD be treated with hydroxyurea starting at 9 months.

For the study, Dr. Karkoska and colleagues analyzed mortality statistics from the period of 1979-2020 via a CDC database. They found that 5272 Black patients died of SCD from 2010 to 2020. The crude mortality rate was 1.1 per 100,000 Black people, lower than the 1.2 per 100,000 rate of 1999-2009 (P < .0001).

The researchers also found that from 2010 to 2020, the mortality rate jumped for patients in the 15-19 to 20-24 age group: It rose from 0.9 per 100,000 to 1.4 per 100,000, P < .0001).

The researchers also examined contributors to death other than SCD. In 39% of cases, underlying causes were noted: cardiovascular disease (28%), accidents (7%), cerebrovascular disease (7%), malignancy (6%), septicemia (4.8%), and renal disease (3.8%). The population of people with SCD is “getting older, and they’re developing a combination of both sickle-related chronic organ damage as well as non-sickle-related chronic disease,” Dr. Karkoska said.

She noted that limitations include a reliance on data that can be incomplete or inaccurate. She also mentioned that the study only focuses on Black patients, who make up the vast majority of those with SCD.

How good is the news about improved mortality numbers? One member of the audience at the ASH presentation was disappointed that they hadn’t gotten even better. “I was hoping to come here to be cheered up,” he said, “and I’m not.”

Three physicians who didn’t take part in the research but are familiar with the new study spoke in interviews about the findings.

Michael Bender, MD, PhD, director of the Odessa Brown Comprehensive Sickle Cell Clinic in Seattle, pointed out that mortality rates improve slowly over time, as new treatments enter the picture. When new therapies come along, he said, “it’s tough if someone’s already 40 years old and their body has gone through a lot. They’re not going to have as much benefit as someone who started [on therapy] when they were 5 years old, and they grew up with that improvement.”

Sickle cell specialist Asmaa Ferdjallah, MD, MPH, of the Mayo Clinic in Rochester, Minnesota, said that the data showing a spike in mortality rates during the pediatric-adult transition are not surprising but still “really hard to digest.”

“It is a testament to the fact that we are not meeting patients where they are,” she said. “We struggle immensely with the transition period. This is something that is difficult across all providers all over the country,” she said. “There are different ways to ensure a successful transition from the pediatric side to the adult side. Here at Mayo Clinic, we use a slow transition, and we rotate appointments with peds and adults until age 30.”

Sophie Miriam Lanzkron, MD, MHS, director of the Sickle Cell Center for Adults at Johns Hopkins Hospital, Baltimore, said increases in mortality in the post-pediatric period appear to be due in part to “lack of access to high-quality sickle cell care for adults because there aren’t enough hematologists.” Worsening disease due to aging is another factor, she said, and “there might also be some behavioral changes. Young people think they will live forever. Sometimes they choose not to adhere to medical recommendations, which for this population is very risky.”

Dr. Lanzkron said her team is developing a long-term patient registry that should provide more insight.

No study funding was reported. Dr. Karkoska had no disclosures. The other coauthor disclosed research funding and safety advisory board relationships with Novartis. Dr. Ferdjallah, Dr. Lanzkron, and Dr. Bender reported no disclosures.

SAN DIEGO — The average age of death for Black U.S. patients with sickle cell disease (SCD) rose from 39 years (females = 40, males = 38) in 1999-2009 to 43 years (females = 44, males = 41) in 2010-2020, reflecting improvements in treatment, a new study finds.

But the news is not all positive. Mortality rates still jumped markedly as patients transitioned from pediatric to adult care, lead author Kristine A. Karkoska, MD, a pediatric hematology/oncologist with the University of Cincinnati College of Medicine, said at the annual meeting of the American Society of Hematology.

“This reflects that young adults are getting lost to care, and then they’re presenting with acute, life-threatening complications,” she said. “We still need more emphasis on comprehensive lifetime sickle-cell care and the transition to adult clinics to improve mortality in young adults.”

According to Dr. Karkoska, researchers launched the analysis of sickle-cell mortality rates to update previously available data up to the year 2009, which showed improvements as current standard-of-care treatments were introduced. Updated numbers, she said, would reflect the influence of a rise in dedicated SCD clinics and a 2014 National Heart, Lung, and Blood Institute recommendation that all children with SCD be treated with hydroxyurea starting at 9 months.

For the study, Dr. Karkoska and colleagues analyzed mortality statistics from the period of 1979-2020 via a CDC database. They found that 5272 Black patients died of SCD from 2010 to 2020. The crude mortality rate was 1.1 per 100,000 Black people, lower than the 1.2 per 100,000 rate of 1999-2009 (P < .0001).

The researchers also found that from 2010 to 2020, the mortality rate jumped for patients in the 15-19 to 20-24 age group: It rose from 0.9 per 100,000 to 1.4 per 100,000, P < .0001).

The researchers also examined contributors to death other than SCD. In 39% of cases, underlying causes were noted: cardiovascular disease (28%), accidents (7%), cerebrovascular disease (7%), malignancy (6%), septicemia (4.8%), and renal disease (3.8%). The population of people with SCD is “getting older, and they’re developing a combination of both sickle-related chronic organ damage as well as non-sickle-related chronic disease,” Dr. Karkoska said.

She noted that limitations include a reliance on data that can be incomplete or inaccurate. She also mentioned that the study only focuses on Black patients, who make up the vast majority of those with SCD.

How good is the news about improved mortality numbers? One member of the audience at the ASH presentation was disappointed that they hadn’t gotten even better. “I was hoping to come here to be cheered up,” he said, “and I’m not.”

Three physicians who didn’t take part in the research but are familiar with the new study spoke in interviews about the findings.

Michael Bender, MD, PhD, director of the Odessa Brown Comprehensive Sickle Cell Clinic in Seattle, pointed out that mortality rates improve slowly over time, as new treatments enter the picture. When new therapies come along, he said, “it’s tough if someone’s already 40 years old and their body has gone through a lot. They’re not going to have as much benefit as someone who started [on therapy] when they were 5 years old, and they grew up with that improvement.”

Sickle cell specialist Asmaa Ferdjallah, MD, MPH, of the Mayo Clinic in Rochester, Minnesota, said that the data showing a spike in mortality rates during the pediatric-adult transition are not surprising but still “really hard to digest.”

“It is a testament to the fact that we are not meeting patients where they are,” she said. “We struggle immensely with the transition period. This is something that is difficult across all providers all over the country,” she said. “There are different ways to ensure a successful transition from the pediatric side to the adult side. Here at Mayo Clinic, we use a slow transition, and we rotate appointments with peds and adults until age 30.”

Sophie Miriam Lanzkron, MD, MHS, director of the Sickle Cell Center for Adults at Johns Hopkins Hospital, Baltimore, said increases in mortality in the post-pediatric period appear to be due in part to “lack of access to high-quality sickle cell care for adults because there aren’t enough hematologists.” Worsening disease due to aging is another factor, she said, and “there might also be some behavioral changes. Young people think they will live forever. Sometimes they choose not to adhere to medical recommendations, which for this population is very risky.”

Dr. Lanzkron said her team is developing a long-term patient registry that should provide more insight.

No study funding was reported. Dr. Karkoska had no disclosures. The other coauthor disclosed research funding and safety advisory board relationships with Novartis. Dr. Ferdjallah, Dr. Lanzkron, and Dr. Bender reported no disclosures.

SAN DIEGO — A small group of adult and pediatric patients with sickle cell disease (SCD) reached high 2-year survival after undergoing reduced-intensity haploidentical stem cell transplantation, a new phase 2 trial reports. It is much easier to find eligible haploidentical donors — half-matched or partially matched — than eligible hematopoietic donors.

Of 42 patients aged 15-45 who were fully treated, 95% survived to 2 years post transplant (overall survival, (95% CI, 81.5%-98.7%), and 88% reached the primary endpoint of event-free survival at 2 years (95% CI, 73.5%-94.8%), according to the findings, which were released at the annual meeting of the American Society of Hematology.

At an ASH news briefing, study lead author Adetola A. Kassim, MBBS, MS, of Vanderbilt University Medical Center, in Nashville, Tennessee, said the results support haploidentical stem cell transplants “as a suitable and tolerable curative therapy for adults with sickle cell disease and severe end-organ toxicity such as stroke or pulmonary hypertension, a population typically excluded from participating in gene therapy.”

Dr. Kassim added that the findings are especially promising since there are so many potential donors in stem-cell transplants: “Your siblings can be donors, your parents can be donors, your cousins can be donors. First-, second-, and third-degree relatives can be donors. So there’s really endless donors within the family.”

In an interview, Mayo Clinic SCD specialist Asmaa Ferdjallah, MD, MPH, of Mayo Clinic in Rochester, Minnesota, who was not involved with the study but is familiar with its findings, said stem cell transplant is the only option to cure SCD.

“This is advantageous because SCD is otherwise a chronic disease that is marked by chronic pain, risk of stroke, frequent interruptions of school/work due to sick days, and decreased life span,” she said. “Most patients, assuming they can tolerate the conditioning chemotherapy that is given before transplant, are eligible.”

Matched sibling donors are preferable, but they can be hard to find, she said. It hasn’t been clear whether half-matched donors are feasible options in SCD, she said. “This means that, if you are a patient with sickle cell disease, and you don’t have a suitable matched donor, haploidentical transplant is not a recommendation we can make outside of enrollment in a clinical trial.”

For the study, researchers enlisted 54 patients with SCD and prior stroke, recurrent acute chest syndrome or pain, chronic transfusion regimen, or tricuspid valve regurgitant jet velocity ≥2.7 m/sec. Participants had to have an HLA-haploidentical first-degree relative donor who would donate bone marrow.

“The median age was 22.8 years at enrollment; 47/54 (87%) of enrolled participants had hemoglobin SS disease, 40/54 (74.1%) had a Lansky/Karnofsky score of 90-100 at baseline, and 41/54 (75.9%) had an HLA match score of 4/8,” the researchers reported. “Recurrent vaso-occlusive pain episodes (38.9%), acute chest syndrome (16.8%), and overt stroke (16.7%) were the most common indications for transplant.”

“We knew going into this that we were going to get very high-risk patients,” Dr. Kassim said.

Forty-two patients went through with transplants. As for adverse events, 2 patients died, all within the first year, of organ failure and acute respiratory distress syndrome; 4.8% of participants had primary graft failure, and 2.4% had secondary graft failure before day 100. “The cumulative incidence of grades II-IV acute GVHD [graft-versus-host disease] at day 100 was 26.2% (95% CI, 14.0%-40.2%), and grades III-IV acute GVHD at day 100 was 4.8% (95% CI, 0.9%-14.4%).”

The outcomes are similar to those in transplants with matched sibling donors, Dr. Kassim said.

Dr. Ferdjallah said the new study is “robust” and impressive, although it’s small.

“As a clinician, these are the kind of outcomes I have been hoping for,” Dr. Ferdjallah said. “I have been very reluctant to suggest haploidentical transplant for my sickle cell disease patients. However, reviewing the results of this study with my motivated patients and families can help us both to use shared medical decision-making and come together with what is best for that specific patient.”

As for adverse events, she said they “confirm a fear of using haploidentical transplant, which is graft failure. Fortunately, out of 42 who proceeded to transplant, only 2 had primary graft failure and 1 had secondary graft failure. This is not overtly a large number. Of course, we would hope for more durable engraftment. The other side effects including GVHD and infection are all to be expected.”

As for cost, Dr. Kassim said the transplants run from $200,000 to $400,000 vs over $2 million for gene therapy, and Dr. Ferdjallah said insurance is likely to cover the treatment.

Moving ahead, Dr. Ferdjallah said she looks forward to getting study data about pediatric patients specifically. For now, “we should consider HLA-haploidentical seriously in patients with sickle cell disease and no available HLA-matched donors.”

Grants to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute and National Cancer Institute funded the study. Dr. Kassim had no disclosures. Some other authors disclosed various and multiple relationships with industry. Dr. Ferdjallah has no disclosures.

SAN DIEGO — A small group of adult and pediatric patients with sickle cell disease (SCD) reached high 2-year survival after undergoing reduced-intensity haploidentical stem cell transplantation, a new phase 2 trial reports. It is much easier to find eligible haploidentical donors — half-matched or partially matched — than eligible hematopoietic donors.

Of 42 patients aged 15-45 who were fully treated, 95% survived to 2 years post transplant (overall survival, (95% CI, 81.5%-98.7%), and 88% reached the primary endpoint of event-free survival at 2 years (95% CI, 73.5%-94.8%), according to the findings, which were released at the annual meeting of the American Society of Hematology.

At an ASH news briefing, study lead author Adetola A. Kassim, MBBS, MS, of Vanderbilt University Medical Center, in Nashville, Tennessee, said the results support haploidentical stem cell transplants “as a suitable and tolerable curative therapy for adults with sickle cell disease and severe end-organ toxicity such as stroke or pulmonary hypertension, a population typically excluded from participating in gene therapy.”

Dr. Kassim added that the findings are especially promising since there are so many potential donors in stem-cell transplants: “Your siblings can be donors, your parents can be donors, your cousins can be donors. First-, second-, and third-degree relatives can be donors. So there’s really endless donors within the family.”

In an interview, Mayo Clinic SCD specialist Asmaa Ferdjallah, MD, MPH, of Mayo Clinic in Rochester, Minnesota, who was not involved with the study but is familiar with its findings, said stem cell transplant is the only option to cure SCD.

“This is advantageous because SCD is otherwise a chronic disease that is marked by chronic pain, risk of stroke, frequent interruptions of school/work due to sick days, and decreased life span,” she said. “Most patients, assuming they can tolerate the conditioning chemotherapy that is given before transplant, are eligible.”

Matched sibling donors are preferable, but they can be hard to find, she said. It hasn’t been clear whether half-matched donors are feasible options in SCD, she said. “This means that, if you are a patient with sickle cell disease, and you don’t have a suitable matched donor, haploidentical transplant is not a recommendation we can make outside of enrollment in a clinical trial.”

For the study, researchers enlisted 54 patients with SCD and prior stroke, recurrent acute chest syndrome or pain, chronic transfusion regimen, or tricuspid valve regurgitant jet velocity ≥2.7 m/sec. Participants had to have an HLA-haploidentical first-degree relative donor who would donate bone marrow.

“The median age was 22.8 years at enrollment; 47/54 (87%) of enrolled participants had hemoglobin SS disease, 40/54 (74.1%) had a Lansky/Karnofsky score of 90-100 at baseline, and 41/54 (75.9%) had an HLA match score of 4/8,” the researchers reported. “Recurrent vaso-occlusive pain episodes (38.9%), acute chest syndrome (16.8%), and overt stroke (16.7%) were the most common indications for transplant.”

“We knew going into this that we were going to get very high-risk patients,” Dr. Kassim said.

Forty-two patients went through with transplants. As for adverse events, 2 patients died, all within the first year, of organ failure and acute respiratory distress syndrome; 4.8% of participants had primary graft failure, and 2.4% had secondary graft failure before day 100. “The cumulative incidence of grades II-IV acute GVHD [graft-versus-host disease] at day 100 was 26.2% (95% CI, 14.0%-40.2%), and grades III-IV acute GVHD at day 100 was 4.8% (95% CI, 0.9%-14.4%).”

The outcomes are similar to those in transplants with matched sibling donors, Dr. Kassim said.

Dr. Ferdjallah said the new study is “robust” and impressive, although it’s small.

“As a clinician, these are the kind of outcomes I have been hoping for,” Dr. Ferdjallah said. “I have been very reluctant to suggest haploidentical transplant for my sickle cell disease patients. However, reviewing the results of this study with my motivated patients and families can help us both to use shared medical decision-making and come together with what is best for that specific patient.”

As for adverse events, she said they “confirm a fear of using haploidentical transplant, which is graft failure. Fortunately, out of 42 who proceeded to transplant, only 2 had primary graft failure and 1 had secondary graft failure. This is not overtly a large number. Of course, we would hope for more durable engraftment. The other side effects including GVHD and infection are all to be expected.”

As for cost, Dr. Kassim said the transplants run from $200,000 to $400,000 vs over $2 million for gene therapy, and Dr. Ferdjallah said insurance is likely to cover the treatment.

Moving ahead, Dr. Ferdjallah said she looks forward to getting study data about pediatric patients specifically. For now, “we should consider HLA-haploidentical seriously in patients with sickle cell disease and no available HLA-matched donors.”

Grants to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute and National Cancer Institute funded the study. Dr. Kassim had no disclosures. Some other authors disclosed various and multiple relationships with industry. Dr. Ferdjallah has no disclosures.

SAN DIEGO — A small group of adult and pediatric patients with sickle cell disease (SCD) reached high 2-year survival after undergoing reduced-intensity haploidentical stem cell transplantation, a new phase 2 trial reports. It is much easier to find eligible haploidentical donors — half-matched or partially matched — than eligible hematopoietic donors.

Of 42 patients aged 15-45 who were fully treated, 95% survived to 2 years post transplant (overall survival, (95% CI, 81.5%-98.7%), and 88% reached the primary endpoint of event-free survival at 2 years (95% CI, 73.5%-94.8%), according to the findings, which were released at the annual meeting of the American Society of Hematology.

At an ASH news briefing, study lead author Adetola A. Kassim, MBBS, MS, of Vanderbilt University Medical Center, in Nashville, Tennessee, said the results support haploidentical stem cell transplants “as a suitable and tolerable curative therapy for adults with sickle cell disease and severe end-organ toxicity such as stroke or pulmonary hypertension, a population typically excluded from participating in gene therapy.”

Dr. Kassim added that the findings are especially promising since there are so many potential donors in stem-cell transplants: “Your siblings can be donors, your parents can be donors, your cousins can be donors. First-, second-, and third-degree relatives can be donors. So there’s really endless donors within the family.”

In an interview, Mayo Clinic SCD specialist Asmaa Ferdjallah, MD, MPH, of Mayo Clinic in Rochester, Minnesota, who was not involved with the study but is familiar with its findings, said stem cell transplant is the only option to cure SCD.

“This is advantageous because SCD is otherwise a chronic disease that is marked by chronic pain, risk of stroke, frequent interruptions of school/work due to sick days, and decreased life span,” she said. “Most patients, assuming they can tolerate the conditioning chemotherapy that is given before transplant, are eligible.”

Matched sibling donors are preferable, but they can be hard to find, she said. It hasn’t been clear whether half-matched donors are feasible options in SCD, she said. “This means that, if you are a patient with sickle cell disease, and you don’t have a suitable matched donor, haploidentical transplant is not a recommendation we can make outside of enrollment in a clinical trial.”

For the study, researchers enlisted 54 patients with SCD and prior stroke, recurrent acute chest syndrome or pain, chronic transfusion regimen, or tricuspid valve regurgitant jet velocity ≥2.7 m/sec. Participants had to have an HLA-haploidentical first-degree relative donor who would donate bone marrow.

“The median age was 22.8 years at enrollment; 47/54 (87%) of enrolled participants had hemoglobin SS disease, 40/54 (74.1%) had a Lansky/Karnofsky score of 90-100 at baseline, and 41/54 (75.9%) had an HLA match score of 4/8,” the researchers reported. “Recurrent vaso-occlusive pain episodes (38.9%), acute chest syndrome (16.8%), and overt stroke (16.7%) were the most common indications for transplant.”

“We knew going into this that we were going to get very high-risk patients,” Dr. Kassim said.

Forty-two patients went through with transplants. As for adverse events, 2 patients died, all within the first year, of organ failure and acute respiratory distress syndrome; 4.8% of participants had primary graft failure, and 2.4% had secondary graft failure before day 100. “The cumulative incidence of grades II-IV acute GVHD [graft-versus-host disease] at day 100 was 26.2% (95% CI, 14.0%-40.2%), and grades III-IV acute GVHD at day 100 was 4.8% (95% CI, 0.9%-14.4%).”

The outcomes are similar to those in transplants with matched sibling donors, Dr. Kassim said.

Dr. Ferdjallah said the new study is “robust” and impressive, although it’s small.

“As a clinician, these are the kind of outcomes I have been hoping for,” Dr. Ferdjallah said. “I have been very reluctant to suggest haploidentical transplant for my sickle cell disease patients. However, reviewing the results of this study with my motivated patients and families can help us both to use shared medical decision-making and come together with what is best for that specific patient.”

As for adverse events, she said they “confirm a fear of using haploidentical transplant, which is graft failure. Fortunately, out of 42 who proceeded to transplant, only 2 had primary graft failure and 1 had secondary graft failure. This is not overtly a large number. Of course, we would hope for more durable engraftment. The other side effects including GVHD and infection are all to be expected.”

As for cost, Dr. Kassim said the transplants run from $200,000 to $400,000 vs over $2 million for gene therapy, and Dr. Ferdjallah said insurance is likely to cover the treatment.

Moving ahead, Dr. Ferdjallah said she looks forward to getting study data about pediatric patients specifically. For now, “we should consider HLA-haploidentical seriously in patients with sickle cell disease and no available HLA-matched donors.”

Grants to the Blood and Marrow Transplant Clinical Trials Network from the National Heart, Lung, and Blood Institute and National Cancer Institute funded the study. Dr. Kassim had no disclosures. Some other authors disclosed various and multiple relationships with industry. Dr. Ferdjallah has no disclosures.

SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.

“This is a one-time, truly transformative treatment with lovo-cel,” lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing at the annual meeting of the American Society of Hematology. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Dr. Kanter added. 

For “anybody who has rounded on the inpatient floor and taken care of adolescents admitted with a pain crisis multiple times a year,” seeing these results “is so compelling,” commented Sarah O’Brien, MD, a pediatric hematologist at Nationwide Children’s Hospital in Columbus, Ohio, who moderated the briefing but was not involved in the study.
 

One and Done

Sickle cell disease, a debilitating and potentially life-threatening blood disorder, affects an estimated 100,000 people in the US. 

People with the condition have a mutation in hemoglobin, which causes red blood cells to develop an abnormal sickle shape. These sickled cells block the flow of blood, ultimately depriving tissues of oxygen and leading to organ damage and severe pain, known as vaso-occlusive events. 

On Dec. 8, the U.S. Food and Drug Administration (FDA) approved lovo-cel for patients aged 12 years or older with severe sickle cell disease alongside another gene-editing therapy called exagamglogene autotemcel or exa-cel (Casgevy, Vertex Pharmaceuticals and Crispr Therapeutics). The two therapies use different gene-editing approaches — exa-cel is the first to use the gene-editing tool CRISPR while lovo-cel uses a lentiviral vector.

Both are one-time, single-dose cell-based gene therapies.

With lovo-cel, patients first undergo a transfusion regimen and myeloablative conditioning with busulfan to collect cells that can then be genetically modified. A patient’s harvested cells are modified with an anti-sickling version of hemoglobin A, HbAT87Q. Patients then receive an infusion of these edited cells and remain in the hospital during engraftment and reconstitution.

Dr. Kanter presented long-term follow-up data on 47 patients enrolled in phase 1/2 and phase 3 studies of lovo-cel. 

All patients had stable HbAT87Q levels from 6 months to their last follow-up at a median of 35.5 months. 

Most patients achieved a durable globin response through their final follow-up visit.

Among the 34 evaluable patients, 88% had complete resolution of vaso-occlusive events 6 to 18 months after their infusion, including all 10 adolescent patients. Almost all patients (94%) achieved complete resolution of serious vaso-occlusive events. 

In the few patients who experienced posttreatment vaso-occlusive events, these individuals still achieved major reductions in hospital admissions and hospital days.

Among 20 patients followed for at least 3 years, more than half had clinically meaningful improvements in pain intensity, pain interference, and fatigue.

Most treatment-related adverse events occurred within 1 year of lovo-cel infusions and were primarily related to busulfan conditioning. No cases of veno-occlusive liver disease, graft failure, or graft vs host disease occurred, and patients did not have complications related to the viral vector. No patients who had a history of stroke prior to lovo-cel therapy experienced a post-therapy stroke. 

One patient died at baseline from significant cardiopulmonary disease related to sickle cell disease, but the death was considered unrelated to lovo-cel therapy.

To see a one-time treatment that essentially eradicates vaso-occlusive events is “really unparalleled,” said Steven Pipe, MD, from the University of Michigan School of Medicine in Ann Arbor, who presented data on a different study at the briefing.

However, Dr. Kanter noted, “it’s important to highlight that many of these individuals come into this therapy with significant disease and end-organ complications, and this will be something we will really need to follow long-term to understand how much this therapy can stabilize or reverse these complications.”

The studies were funded by bluebird bio. Dr. Kanter disclosed honoraria from the company and consulting/advising activities and receipt of research funding from multiple other entities. Dr. O’Brien disclosed consultancy for AstraZeneca, honoraria from Pharmacosmos, and research funding from Bristol Myers Squibb. Dr. Pipe disclosed consulting activities from multiple companies, not including bluebird bio. 

A version of this article appeared on Medscape.com.

SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.

“This is a one-time, truly transformative treatment with lovo-cel,” lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing at the annual meeting of the American Society of Hematology. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Dr. Kanter added. 

For “anybody who has rounded on the inpatient floor and taken care of adolescents admitted with a pain crisis multiple times a year,” seeing these results “is so compelling,” commented Sarah O’Brien, MD, a pediatric hematologist at Nationwide Children’s Hospital in Columbus, Ohio, who moderated the briefing but was not involved in the study.
 

One and Done

Sickle cell disease, a debilitating and potentially life-threatening blood disorder, affects an estimated 100,000 people in the US. 

People with the condition have a mutation in hemoglobin, which causes red blood cells to develop an abnormal sickle shape. These sickled cells block the flow of blood, ultimately depriving tissues of oxygen and leading to organ damage and severe pain, known as vaso-occlusive events. 

On Dec. 8, the U.S. Food and Drug Administration (FDA) approved lovo-cel for patients aged 12 years or older with severe sickle cell disease alongside another gene-editing therapy called exagamglogene autotemcel or exa-cel (Casgevy, Vertex Pharmaceuticals and Crispr Therapeutics). The two therapies use different gene-editing approaches — exa-cel is the first to use the gene-editing tool CRISPR while lovo-cel uses a lentiviral vector.

Both are one-time, single-dose cell-based gene therapies.

With lovo-cel, patients first undergo a transfusion regimen and myeloablative conditioning with busulfan to collect cells that can then be genetically modified. A patient’s harvested cells are modified with an anti-sickling version of hemoglobin A, HbAT87Q. Patients then receive an infusion of these edited cells and remain in the hospital during engraftment and reconstitution.

Dr. Kanter presented long-term follow-up data on 47 patients enrolled in phase 1/2 and phase 3 studies of lovo-cel. 

All patients had stable HbAT87Q levels from 6 months to their last follow-up at a median of 35.5 months. 

Most patients achieved a durable globin response through their final follow-up visit.

Among the 34 evaluable patients, 88% had complete resolution of vaso-occlusive events 6 to 18 months after their infusion, including all 10 adolescent patients. Almost all patients (94%) achieved complete resolution of serious vaso-occlusive events. 

In the few patients who experienced posttreatment vaso-occlusive events, these individuals still achieved major reductions in hospital admissions and hospital days.

Among 20 patients followed for at least 3 years, more than half had clinically meaningful improvements in pain intensity, pain interference, and fatigue.

Most treatment-related adverse events occurred within 1 year of lovo-cel infusions and were primarily related to busulfan conditioning. No cases of veno-occlusive liver disease, graft failure, or graft vs host disease occurred, and patients did not have complications related to the viral vector. No patients who had a history of stroke prior to lovo-cel therapy experienced a post-therapy stroke. 

One patient died at baseline from significant cardiopulmonary disease related to sickle cell disease, but the death was considered unrelated to lovo-cel therapy.

To see a one-time treatment that essentially eradicates vaso-occlusive events is “really unparalleled,” said Steven Pipe, MD, from the University of Michigan School of Medicine in Ann Arbor, who presented data on a different study at the briefing.

However, Dr. Kanter noted, “it’s important to highlight that many of these individuals come into this therapy with significant disease and end-organ complications, and this will be something we will really need to follow long-term to understand how much this therapy can stabilize or reverse these complications.”

The studies were funded by bluebird bio. Dr. Kanter disclosed honoraria from the company and consulting/advising activities and receipt of research funding from multiple other entities. Dr. O’Brien disclosed consultancy for AstraZeneca, honoraria from Pharmacosmos, and research funding from Bristol Myers Squibb. Dr. Pipe disclosed consulting activities from multiple companies, not including bluebird bio. 

A version of this article appeared on Medscape.com.

SAN DIEGO — A newly approved gene therapy product for sickle cell disease, lovotibeglogene autotemcel (lovo-cel, marketed as Lyfgenia), led to durable disease remissions for up to 5 years and almost complete elimination of dangerous and debilitating vaso-occlusive events, according to results of a long-term follow-up study.

More specifically, a single infusion of lovo-cel led to complete resolution of vaso-occlusive events in 88% of patients, with 94% achieving complete resolution of severe events. All 10 adolescents in the study achieved complete resolution of vaso-occlusive events. Most patients remained free of vaso-occlusive events at their last follow-up.

“This is a one-time, truly transformative treatment with lovo-cel,” lead author Julie Kanter, MD, director of the adult sickle cell clinic at the University of Alabama in Birmingham, said in a media briefing at the annual meeting of the American Society of Hematology. The gene therapy can essentially eliminate vaso-occlusive events in patients with sickle cell disease and lead to normal hemoglobin levels, Dr. Kanter added. 

For “anybody who has rounded on the inpatient floor and taken care of adolescents admitted with a pain crisis multiple times a year,” seeing these results “is so compelling,” commented Sarah O’Brien, MD, a pediatric hematologist at Nationwide Children’s Hospital in Columbus, Ohio, who moderated the briefing but was not involved in the study.
 

One and Done

Sickle cell disease, a debilitating and potentially life-threatening blood disorder, affects an estimated 100,000 people in the US. 

People with the condition have a mutation in hemoglobin, which causes red blood cells to develop an abnormal sickle shape. These sickled cells block the flow of blood, ultimately depriving tissues of oxygen and leading to organ damage and severe pain, known as vaso-occlusive events. 

On Dec. 8, the U.S. Food and Drug Administration (FDA) approved lovo-cel for patients aged 12 years or older with severe sickle cell disease alongside another gene-editing therapy called exagamglogene autotemcel or exa-cel (Casgevy, Vertex Pharmaceuticals and Crispr Therapeutics). The two therapies use different gene-editing approaches — exa-cel is the first to use the gene-editing tool CRISPR while lovo-cel uses a lentiviral vector.

Both are one-time, single-dose cell-based gene therapies.

With lovo-cel, patients first undergo a transfusion regimen and myeloablative conditioning with busulfan to collect cells that can then be genetically modified. A patient’s harvested cells are modified with an anti-sickling version of hemoglobin A, HbAT87Q. Patients then receive an infusion of these edited cells and remain in the hospital during engraftment and reconstitution.

Dr. Kanter presented long-term follow-up data on 47 patients enrolled in phase 1/2 and phase 3 studies of lovo-cel. 

All patients had stable HbAT87Q levels from 6 months to their last follow-up at a median of 35.5 months. 

Most patients achieved a durable globin response through their final follow-up visit.

Among the 34 evaluable patients, 88% had complete resolution of vaso-occlusive events 6 to 18 months after their infusion, including all 10 adolescent patients. Almost all patients (94%) achieved complete resolution of serious vaso-occlusive events. 

In the few patients who experienced posttreatment vaso-occlusive events, these individuals still achieved major reductions in hospital admissions and hospital days.

Among 20 patients followed for at least 3 years, more than half had clinically meaningful improvements in pain intensity, pain interference, and fatigue.

Most treatment-related adverse events occurred within 1 year of lovo-cel infusions and were primarily related to busulfan conditioning. No cases of veno-occlusive liver disease, graft failure, or graft vs host disease occurred, and patients did not have complications related to the viral vector. No patients who had a history of stroke prior to lovo-cel therapy experienced a post-therapy stroke. 

One patient died at baseline from significant cardiopulmonary disease related to sickle cell disease, but the death was considered unrelated to lovo-cel therapy.

To see a one-time treatment that essentially eradicates vaso-occlusive events is “really unparalleled,” said Steven Pipe, MD, from the University of Michigan School of Medicine in Ann Arbor, who presented data on a different study at the briefing.

However, Dr. Kanter noted, “it’s important to highlight that many of these individuals come into this therapy with significant disease and end-organ complications, and this will be something we will really need to follow long-term to understand how much this therapy can stabilize or reverse these complications.”

The studies were funded by bluebird bio. Dr. Kanter disclosed honoraria from the company and consulting/advising activities and receipt of research funding from multiple other entities. Dr. O’Brien disclosed consultancy for AstraZeneca, honoraria from Pharmacosmos, and research funding from Bristol Myers Squibb. Dr. Pipe disclosed consulting activities from multiple companies, not including bluebird bio. 

A version of this article appeared on Medscape.com.