Clinical activity and safety of rilzabrutinib in patients with previously treated immune thrombocytopenia (LUNA3): results from the open-label period of a phase 3 trial.
Lancet HaematolSep 17, 2026 (epub)
Clinical ResearchHematology
David J Kuter, Waleed Ghanima, Nichola Cooper et al.
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In 30 seconds
The LUNA3 trial, a multicenter, randomized phase 3 study, evaluated the long-term efficacy and safety of rilzabrutinib in adults with previously treated immune thrombocytopenia. Among 180 patients, 46 (23%) achieved stable platelet responses, with treatment-related adverse events occurring in 26% of patients. The median follow-up was 196 days.
Key findings
- 46 (23%) of 198 patients had stable platelet responses by the data cutoff date.
- 42 (23%) of 180 patients achieved a complete response.
- Treatment-related adverse events occurred in 46 (26%) of 180 patients.
- 14 (22%) of 65 patients in the placebo group had a durable platelet response.
Why it matters
The findings suggest that rilzabrutinib may provide a viable treatment option for patients with immune thrombocytopenia, demonstrating both efficacy in achieving stable platelet counts and a manageable safety profile.