Informace o publikaci

Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study

Autoři	COOPER Nichola JANSEN A J Gerard BIRD Robert MAYER Jiří SHOLZBERG Michelle TARANTINO Michael D GARG Mamta YPMA Paula F MCDONALD Vickie PERCY Charles KOSTAL Milan GONCALVES Isaac BOGDANOV Lachezar H GERNSHEIMER Terry B DIAB Remco YAO Mengjie DAAK Ahmed KUTER David J
Rok publikování	2025
Druh	Článek v odborném periodiku
Časopis / Zdroj	American Journal of Hematology
Fakulta / Pracoviště MU	Lékařská fakulta
Citace
www	https://onlinelibrary.wiley.com/doi/10.1002/ajh.27539
Doi	http://dx.doi.org/10.1002/ajh.27539
Klíčová slova	adults; immune thrombocytopenia; platelets; quality of life; response
Popis	Current treatments for persistent or chronic immune thrombocytopenia (ITP) are limited by inadequate response, toxicity, and impaired quality of life. The Bruton tyrosine kinase inhibitor rilzabrutinib was evaluated to further characterize safety and durability of platelet response. LUNA2 Part B is a multicenter, phase 1/2 study in adults with ITP (??3?months duration, platelet count <?30?×?109/L) who failed ??1 ITP therapy (NCT03395210, EudraCT 2017–004012-19). Oral rilzabrutinib 400?mg bid was given over 24?weeks, with optional long-term extension (LTE). Primary endpoints were safety and platelet counts ??50?×?109/L on ??8 of the last 12?weeks of main treatment without rescue medication. From 22 March2018 to 31 January2023, 26 patients were enrolled. Patients had baseline median platelet count 13?×?109/L, ITP duration 10.3?years, and six prior ITP therapies (46% splenectomized). Nine (35%) patients achieved the primary endpoint. Platelet counts ??50?×?109/L or ??30?×?109/L and doubling from baseline without rescue therapy were sustained for a mean 9.3?weeks. 11 (42%) LTE-eligible patients were ongoing with median LTE platelet >?80?×?109/L. Three (12%) patients received rescue medication during main treatment, none in LTE. Clinically meaningful improvements were observed in fatigue and women's health. With a median treatment duration of 167?days (main treatment), 16 (62%) patients had ??1 treatment-related adverse event (AE), mainly grade 1, including diarrhea (35%), headache (23%), and nausea (15%). There was no treatment-related grade ??2 bleeding/thrombotic events/infections, serious AE, or death. Rilzabrutinib continues to demonstrate durable platelet responses with favorable safety profile in previously treated ITP patients.