Rilzabrutinib, an oral BTK inhibitor, in immune thrombocytopenia
The result's identifiers
Result code in IS VaVaI
<a href="https://www.isvavai.cz/riv?ss=detail&h=RIV%2F00843989%3A_____%2F22%3AE0109660" target="_blank" >RIV/00843989:_____/22:E0109660 - isvavai.cz</a>
Alternative codes found
RIV/65269705:_____/22:00076001 RIV/00064165:_____/22:10445097 RIV/00216208:11110/22:10445097 RIV/00216208:11150/22:10445097 and 3 more
Result on the web
<a href="https://www.nejm.org/doi/10.1056/NEJMoa2110297" target="_blank" >https://www.nejm.org/doi/10.1056/NEJMoa2110297</a>
DOI - Digital Object Identifier
<a href="http://dx.doi.org/10.1056/NEJMoa2110297" target="_blank" >10.1056/NEJMoa2110297</a>
Alternative languages
Result language
angličtina
Original language name
Rilzabrutinib, an oral BTK inhibitor, in immune thrombocytopenia
Original language description
Background: Rilzabrutinib, an oral, reversible covalent inhibitor of Bruton's tyrosine kinase, may increase platelet counts in patients with immune thrombocytopenia by means of dual mechanisms of action: decreased macrophage (Fc? receptor)-mediated platelet destruction and reduced production of pathogenic autoantibodies. Methods: In an international, adaptive, open-label, dose-finding, phase 1-2 clinical trial, we evaluated rilzabrutinib therapy in previously treated patients with immune thrombocytopenia. We used intrapatient dose escalation of oral rilzabrutinib over a period of 24 weeks; the lowest starting dose was 200 mg once daily, with higher starting doses of 400 mg once daily, 300 mg twice daily, and 400 mg twice daily. The primary end points were safety and platelet response (defined as at least two consecutive platelet counts of ?50×103 per cubic millimeter and an increase from baseline of ?20×103 per cubic millimeter without the use of rescue medication). Results: Sixty patients were enrolled. At baseline, the median platelet count was 15×103 per cubic millimeter, the median duration of disease was 6.3 years, and patients had received a median of four different immune thrombocytopenia therapies previously. All the treatment-related adverse events were of grade 1 or 2 and transient. There were no treatment-related bleeding or thrombotic events of grade 2 or higher. At a median of 167.5 days (range, 4 to 293) of treatment, 24 of 60 patients (40%) overall and 18 of the 45 patients (40%) who had started rilzabrutinib treatment at the highest dose met the primary end point of platelet response. The median time to the first platelet count of at least 50×103 per cubic millimeter was 11.5 days. Among patients with a primary platelet response, the mean percentage of weeks with a platelet count of at least 50×103 per cubic millimeter was 65%. Conclusions: Rilzabrutinib was active and associated with only low-level toxic effects at all dose levels. The dose of...
Czech name
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Czech description
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Classification
Type
J<sub>imp</sub> - Article in a specialist periodical, which is included in the Web of Science database
CEP classification
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OECD FORD branch
30205 - Hematology
Result continuities
Project
—
Continuities
N - Vyzkumna aktivita podporovana z neverejnych zdroju
Others
Publication year
2022
Confidentiality
S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů
Data specific for result type
Name of the periodical
New England Journal of Medicine
ISSN
0028-4793
e-ISSN
1533-4406
Volume of the periodical
386
Issue of the periodical within the volume
15
Country of publishing house
US - UNITED STATES
Number of pages
11
Pages from-to
1421-1431
UT code for WoS article
000798829500005
EID of the result in the Scopus database
2-s2.0-85128335503