Recently, the Progress in Dermatomyositis (ProDERM) study published in the New England Journal of Medicine reported a milestone in managing adult myositis. With the trial’s favorable results, adult myositis patients have new hope with the approval of Octagam® 10%.
After decades of juvenile myositis first-line treatments being conducted “off-label,” we may be on the verge of progress regarding FDA compliance on intravenous immunoglobulin (IVIg). For many juvenile myositis patients, IVIg plays a vital role in short and long-term treatment cycles. IVIg treatment has become standard practice amongst myositis doctors internationally despite being administered unapproved in varying stages of the disease. In many cases, IVIg is used to help patients taper harsh drugs, including corticosteroids, in the hope of improving outcomes.
“The ProDERM study will have a significant impact on clinical practice because IVIg is likely to become an important treatment option for patients with dermatomyositis,” said Rohit Aggarwal, M.D., MS, Medical Director of the Arthritis and Autoimmunity Center at the University of Pittsburgh School of Medicine and a member of the ProDERM study Steering Committee. “The study gives clinicians much more confidence in the efficacy and safety of intravenous immunoglobulin and provides valuable information about what type of patient is best suited for the treatment.”
The pivotal international phase III clinical trial investigated the efficacy, safety, and tolerability of Octagam 10% in adults with dermatomyositis. The study’s findings have led to the approval in the U.S. to treat adults with dermatomyositis and in Europe in adults with the active disease treated with immunosuppressive drugs. The preliminary 16-week, double-blind, placebo-controlled study enrolled 95 patients from 36 sites in ten countries. Patients were randomly selected to receive the drug or a placebo.
During the initial 16-week phase, 78.7% of patients receiving Octagam® 10% responded positively to treatment compared to 43.8% of those receiving the placebo. The second phase of the trial included an open-label extension, where patients on a placebo in the first 16 weeks could switch to Octagam® 10%. After transitioning to IVIg in the extension period, the placebo group achieved a similar response rate at week 40 as did the Octagam® 10% treated patients at week 16. The drug was generally well tolerated, but there were a few adverse effects, including thrombosis.
What Does This Study Mean for Juvenile Myositis Patients?
The implications for the trial are hopeful.
“There are so few approved drugs for dermatomyositis that the approval for Octagam® 10% is an important milestone, especially for our adult population with JDM,” says Susan Kim, M.D., at the University of California, San Francisco.
Experts from the Cure JM Medical Advisory Board hope to bring similar clinical trials to juvenile myositis patients, as future FDA approvals for IVIg could have game-changing implications on insurance compliance, along with greater efficacy and more standardized care across the U.S. and abroad, with safety and tolerability being primary considerations.
With a growing number of our juvenile myositis patient population entering adulthood and currently one-third of JM patients already into adulthood, the new approval might make the transition of care smoother.
After 20 years of JM advocacy and research progress, we are optimistic for continued scientific milestones and better treatments for JM patients as more promising clinical trials conclude.
For more information on other exciting 2023 research projects being funded and supported by Cure JM’s Clinical Care Network, visit https://www.curejm.org/newresearchgrants/.