Efficacy of BTK inhibitor administered for central high-risk systemic diffuse large B cell lymphoma and primary central nervous system lymphoma: A single-center retrospective study

Abstract Purpose This study was to explore the efficacy of Bruton's tyrosine kinase inhibitor (BTKi) in treating patients with central high-risk systemic diffuse large B cell lymphoma (DLBCL) and primary central nervous system lymphoma (PCNSL), while also assessing the influence of genomic variants on treatment outcomes. Materials and methods The safety, efficacy, and prognosis of patients treated with BTKi-containing regimens were analyzed. Genetic variants on treatment efficacy were analyzed using whole-exome sequencing (WES). Results Of the 10 patients with central high-risk systemic DLBCL, nine completed treatment and were available for efficacy evaluation. The overall response rate (ORR) was 55.6%. And the 1-year central nervous system infiltration rate was 11.1% (1/9) after receiving prophylactic treatment with BTKi. In a separate group of 16 patients with PCNSL, which included three patients with relapsed PCNSL, the median overall survival (OS) was 9 (9–16) months, and six patients with primary refractory disease had a median OS of 18 (2–31) months. Besides, seven patients with PCNSL newly treated with a combination of chemotherapy and BTKi, the ORR was 100% after two courses of treatment. WES was performed on 18 patients with PCNSL, including five with R/R PCNSL who received a BTKi-containing regimen. The median PFS of 7 (3–9) months and a median OS of 16 (9–29) months. Conclusion The findings of this study indicate that BTKi-containing regimens are safe and effective for treating central high-risk systemic DLBCL and PCNSL.