Results of the randomized phase 3 LUNA 3 study showed that rilzabrutinib induced rapid and lasting platelet count expansion for patients with immune thrombocytopenia. The drug also improved overall response rate and quality of life compared to placebo. This BTK inhibitor had a low adverse event rate and demonstrated significant efficacy in a refractory patient population. Patients experienced improvements in physical fatigue and bleeding, with a reduced need for rescue therapy. Rilzabrutinib is likely to become a new standard of care for immune thrombocytopenia and may have implications for other autoimmune diseases. Further research will explore its potential in earlier-stage disease.
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