Three-year follow-up analysis of phase 1/2 study on tirabrutinib in patients with relapsed or refractory primary central nervous system lymphoma.
Neurooncol Adv
; 6(1): vdae037, 2024.
Article
in En
| MEDLINE
| ID: mdl-38690230
ABSTRACT
Background:
The ONO-4059-02 phase 1/2 study showed favorable efficacy and acceptable safety profile of tirabrutinib, a second-generation Bruton's tyrosine kinase inhibitor, for relapsed/refractory primary central nervous system lymphoma (PCNSL). Here, we report the long-term efficacy and safety after a 3-year follow-up.Methods:
Eligible patients were agedâ ≥â 20 years with histologically diagnosed PCNSL and KPS ofâ ≥â 70. Patients received oral tirabrutinib once daily at 320 or 480 mg, or 480 mg under fasted conditions.Results:
Between October 19, 2017, and June 13, 2019, 44 patients were enrolled 33 and 9 had relapsed and refractory, respectively. The 320, 480, and 480 mg fasted groups included 20, 7, and 17 patients, respectively. The median follow-up was 37.1 months. The overall response rate was 63.6% (95% CI 47.8-77.6) with complete response (CR), unconfirmed CR, and partial response in 9, 7, and 12 patients, respectively. The median duration of response (DOR) was 9.2 months, with a DOR rate of 19.8%; the median progression-free survival (PFS) and median overall survival (OS) were 2.9 months and not reached, respectively, with PFS and OS rates of 13.9% and 56.7%, respectively. Adverse events occurred in 38 patients (86.4%) gradeâ ≥â 3 in 23 (52.3%) including 1 patient with grade 5 events. KPS and quality of life (QoL) scores were well maintained among patients receiving long-term treatment.Conclusions:
The results demonstrated the long-term clinical benefit of tirabrutinib, with deep and durable response in a subset of patients and acceptable safety profile, while KPS and QoL scores were maintained.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Language:
En
Journal:
Neurooncol Adv
Year:
2024
Document type:
Article
Affiliation country:
Japón
Country of publication:
Reino Unido