Rilzabrutinib, an oral BTK inhibitor, in immune thrombocytopenia

Kód výsledku v 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>

Nalezeny alternativní kódy

RIV/65269705:_____/22:00076001 RIV/00064165:_____/22:10445097 RIV/00216208:11110/22:10445097 RIV/00216208:11150/22:10445097 a 3 dalších

Výsledek na webu

<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>

Jazyk výsledku

angličtina

Název v původním jazyce

Rilzabrutinib, an oral BTK inhibitor, in immune thrombocytopenia

Popis výsledku v původním jazyce

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...

Název v anglickém jazyce

Rilzabrutinib, an oral BTK inhibitor, in immune thrombocytopenia

Popis výsledku anglicky

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...

Druh

J_imp - Článek v periodiku v databázi Web of Science

CEP obor

—

OECD FORD obor

30205 - Hematology

Projekt

—

Návaznosti

N - Vyzkumna aktivita podporovana z neverejnych zdroju

Rok uplatnění

2022

Kód důvěrnosti údajů

S - Úplné a pravdivé údaje o projektu nepodléhají ochraně podle zvláštních právních předpisů

Název periodika

New England Journal of Medicine

ISSN

0028-4793

e-ISSN

1533-4406

Svazek periodika

386

Číslo periodika v rámci svazku

15

Stát vydavatele periodika

US - Spojené státy americké

Počet stran výsledku

11

Strana od-do

1421-1431

Kód UT WoS článku

000798829500005

EID výsledku v databázi Scopus

2-s2.0-85128335503