Rilzabrutinib, a monoclonal antibody, has demonstrated rapid and durable platelet responses, improved health-related quality of life, and was generally well-tolerated in adults with difficult-to-treat Immune Thrombocytopenia (ITP). The findings, from the phase 3 LUNA 3 study, were presented at the American Society of Hematology Annual Meeting.
The study enrolled adults and children aged 10 years or older with primary persistent/chronic ITP and platelet counts less than 30×10^9/L. Participants received either rilzabrutinib 400mg twice daily or placebo for up to 24 weeks. The results showed that 31% of the rilzabrutinib group achieved a durable response, with significant improvements in fatigue and bleeding events.
Rilzabrutinib significantly reduced the need for rescue therapy by 52% compared to placebo, and improved fatigue scores, even among non-durable platelet responders. The medication’s anti-inflammatory action may contribute to these benefits.
The study found that rilzabrutinib was well-tolerated during the double-blind period, with most adverse events being grade 1 or 2. However, one participant experienced a serious AE of peripheral embolism and another due to unrelated pneumonia.
Overall, the results suggest that rilzabrutinib is a promising treatment option for adults with difficult-to-treat ITP, offering rapid and durable platelet responses, improved quality of life, and favorable safety profiles.
Source: https://www.hcplive.com/view/rilzabrutinib-improves-platelet-response-quality-of-life-immune-thrombocytopenia-compared-to-placebo