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

J 2025

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

COOPER, Nichola; A J Gerard JANSEN; Robert BIRD; Jiří MAYER; Michelle SHOLZBERG et. al.

Basic information

Original name

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

Authors

COOPER, Nichola; A J Gerard JANSEN; Robert BIRD; Jiří MAYER (203 Czech Republic, belonging to the institution); Michelle SHOLZBERG; Michael D TARANTINO; Mamta GARG; Paula F YPMA; Vickie MCDONALD; Charles PERCY; Milan KOSTAL; Isaac GONCALVES; Lachezar H BOGDANOV; Terry B GERNSHEIMER; Remco DIAB; Mengjie YAO; Ahmed DAAK and David J KUTER

Edition

American Journal of Hematology, Hoboken, WILEY, 2025, 0361-8609

Other information

Language

English

Type of outcome

Article in a journal

Field of Study

30205 Hematology

Country of publisher

United States of America

Confidentiality degree

is not subject to a state or trade secret

References:

URL

Impact factor

Impact factor: 10.100 in 2023

Organization unit

Faculty of Medicine

DOI

http://dx.doi.org/10.1002/ajh.27539

UT WoS

001404176200001

EID Scopus

2-s2.0-85215668973

Keywords in English

adults; immune thrombocytopenia; platelets; quality of life; response

Abstract

V originále

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.

Cite

COOPER, Nichola; A J Gerard JANSEN; Robert BIRD; Jiří MAYER; Michelle SHOLZBERG; Michael D TARANTINO; Mamta GARG; Paula F YPMA; Vickie MCDONALD; Charles PERCY; Milan KOSTAL; Isaac GONCALVES; Lachezar H BOGDANOV; Terry B GERNSHEIMER; Remco DIAB; Mengjie YAO; Ahmed DAAK and David J KUTER. Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study. American Journal of Hematology. Hoboken: WILEY, 2025, vol. 100, No 3, p. 439-449. ISSN 0361-8609. Available from: https://dx.doi.org/10.1002/ajh.27539.

@article{2474837,
   author = {Cooper, Nichola and Jansen, A J Gerard and Bird, Robert and Mayer, Jiří and Sholzberg, Michelle and Tarantino, Michael D and Garg, Mamta and Ypma, Paula F and Mcdonald, Vickie and Percy, Charles and Kostal, Milan and Goncalves, Isaac and Bogdanov, Lachezar H and Gernsheimer, Terry B and Diab, Remco and Yao, Mengjie and Daak, Ahmed and Kuter, David J},
   article_location = {Hoboken},
   article_number = {3},
   doi = {http://dx.doi.org/10.1002/ajh.27539},
   keywords = {adults; immune thrombocytopenia; platelets; quality of life; response},
   language = {eng},
   issn = {0361-8609},
   journal = {American Journal of Hematology},
   title = {Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study},
   url = {https://onlinelibrary.wiley.com/doi/10.1002/ajh.27539},
   volume = {100},
   year = {2025}
}

TY  - JOUR
ID  - 2474837
AU  - 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
PY  - 2025
TI  - Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study
JF  - American Journal of Hematology
VL  - 100
IS  - 3
SP  - 439-449
EP  - 439-449
PB  - WILEY
SN  - 03618609
KW  - adults
KW  - immune thrombocytopenia
KW  - platelets
KW  - quality of life
KW  - response
UR  - https://onlinelibrary.wiley.com/doi/10.1002/ajh.27539
N2  - 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.
ER  -

COOPER, Nichola; A J Gerard JANSEN; Robert BIRD; Jiří MAYER; Michelle SHOLZBERG; Michael D TARANTINO; Mamta GARG; Paula F YPMA; Vickie MCDONALD; Charles PERCY; Milan KOSTAL; Isaac GONCALVES; Lachezar H BOGDANOV; Terry B GERNSHEIMER; Remco DIAB; Mengjie YAO; Ahmed DAAK and David J KUTER. Efficacy and Safety Results With Rilzabrutinib, an Oral Bruton Tyrosine Kinase Inhibitor, in Patients With Immune Thrombocytopenia: Phase 2 Part B Study. \textit{American Journal of Hematology}. Hoboken: WILEY, 2025, vol.~100, No~3, p.~439-449. ISSN~0361-8609. Available from: https://dx.doi.org/10.1002/ajh.27539.

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