A rare autoimmune disease that causes bone-crushing fatigue and can flatten even elite athletes may soon have its first approved treatment. For patients with warm autoimmune hemolytic anemia, or wAIHA, the standard of care has long relied on steroids and off-label drugs, with no FDA-approved therapies available. That could change after a phase 2/3 clinical trial found that the experimental drug nipocalimab-aahu (Imaavy) produced a statistically significant and durable hemoglobin response with rapid onset, with many patients seeing improvement within one week.
wAIHA occurs when the immune system creates antibodies that destroy healthy red blood cells at normal body temperature. Symptoms include profound fatigue, dizziness, shortness of breath, and heart rate spikes. The disease can strike at any age but is most common in people between 50 and 70. About half of cases are idiopathic, while the rest are secondary to conditions such as lupus or rheumatoid arthritis. Diagnosis can take up to two years because early symptoms are often mistaken for dehydration or overexertion. Standard care begins with corticosteroids, then moves to broader immunosuppressants or B-cell-directed therapies, and sometimes splenectomy. There is no cure.
The ENERGY trial results offer a new reason for hope. The drug not only raised hemoglobin levels quickly but also reduced fatigue and lowered patients’ need for steroids. Johnson & Johnson, which developed the therapy, reported the first comprehensive data from the trial last month. For a patient community that has long relied on a patchwork of treatments, the prospect of a targeted, approved option marks a significant step forward.
Patient Advocacy Fills Critical Gaps
While pharmaceutical research advances, patient advocacy groups are working to address the isolation and lack of awareness that surround wAIHA. The group wAIHA Warriors, founded by patients and led by Dr. Irina Murakhovskaya, has grown its private Facebook community from fewer than 70 members to nearly 600. The organization also hosts an annual patient meeting, a Warriors on the Hill event in Washington, DC, and participates in AIHA Awareness Day. Its president and CEO, Karen A. Jones, who is herself a wAIHA patient, said the group’s mission is simple: “Our job is to get patients the best treatment for them at the time that they need it.”
Lisa Shea, director of global patient advocacy and engagement at Johnson & Johnson, emphasized the importance of understanding the patient experience. “We want improved quality of life for wAIHA patients. For example, they shouldn’t have to be afraid to get on a plane to visit their grandchildren because they were immunosuppressed. Social isolation can be an additional stressor.” The group also works to educate not just hematologists but primary care physicians, emergency doctors, and transfusion specialists, many of whom see only a handful of wAIHA cases in their careers.
Looking ahead, the wAIHA community is pushing for better research into the relationship between the disease and cancer. One patient leader, Sean Powell, was diagnosed with non-Hodgkin’s lymphoma six years after his wAIHA diagnosis. He is now in remission and continues to lead support sessions. “One physician told us it always appears autoimmune until we find something else,” he said. “So, do we have enough testing to determine if we’re truly autoimmune, or if there’s some underlying process causing us to exhibit wAIHA that hasn’t been found yet?” For a group built to fill gaps the medical establishment has overlooked, that question is exactly the kind they are suited to keep pushing for.