Improving treatment for an autoimmune bleeding condition
Penn Medicine researchers find that an investigational monoclonal antibody may provide extended relief for immune thrombocytopenia.
2 min. read
More than half of patients in a phase III clinical trial who received a limited course of the experimental monoclonal antibody ianalumab for primary immune thrombocytopenia (ITP), an autoimmune disorder that can cause life-threatening bleeding, were able to maintain safe platelet counts without serious bleeding episodes for at least one year. The results are published in the New England Journal of Medicine, by researchers from the Perelman School of Medicine and presented by collaborators at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition.
ITP is an autoimmune condition where the body’s immune cells mistakenly attack platelets, the blood cells responsible for clotting. Some patients with ITP do not require treatment, but for those with low platelet counts or repeated or severe bleeding, initial treatment involves steroids, which work well for some patients. However, for patients who continue to have bleeding issues or low platelet counts with—or after tapering off—steroids, another form of treatment is required. While there are currently three FDA-approved second-line therapies for ITP, they all generally require treatment for life, either in the form of a daily pill or weekly injections, which come with their own side effects and costs.
“As a hematologist, I’m glad that we have effective therapies for ITP, but they’re not necessarily ideal for chronic disease management or long-term quality of life,” says lead author Adam Cuker, section chief for hematology and clinical director of the Penn Blood Disorders Center. “This study shows that prolonged, durable responses to ITP treatment, without the need for ongoing therapy, are possible—and that’s a huge advantage for patients.”
Additional clinical trials for ianalumab are ongoing, including in studies for other autoimmune conditions, and it is not yet FDA-approved for patients. The researchers will continue to follow the patients from this study to track long-term response to treatment.
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