Single agent vemurafenib or rituximab-vemurafenib combination for the treatment of relapsed/refractory hairy cell leukemia, a multicenter experience

• Published in: Leukemia research Vol. 140; p. 107495
• Main Authors: Yiğit Kaya, Süreyya, Mutlu, Yaşa Gül, Malkan, Ümit Yavuz, Mehtap, Özgür, Keklik Karadağ, Fatma, Korkmaz, Gülten, Elverdi, Tuğrul, Saydam, Güray, Özet, Gülsüm, Ar, Muhlis Cem, Melek, Elif, Maral, Senem, Kaynar, Leylagül, Sevindik, Ömür Gökmen
• Format: Journal Article
• Language: English
• Published: England Elsevier Ltd 01.05.2024
• Subjects: BRAF inhibitor; Hairy cell leukemia; Rituximab; Vemurafenib; Rituximab; Vemurafenib; BRAF inhibitor; Hairy cell leukemia
• ISSN: 0145-2126; 1873-5835; 1873-5835
• DOI: 10.1016/j.leukres.2024.107495

Hairy cell leukemia (HCL) is a rare mature B-cell malignancy that is primarily treated with purine analogues. However, relapse remains a significant challenge, prompting the search for alternative therapies. The BRAF V600E mutation prevalent in HCL patients provides a target for treatment with vemurafenib. This multicenter retrospective study included nine patients with relapsed/refractory (R/R) HCL from six different centers. Patient data included demographics, prior treatments, clinical outcomes, and adverse events. Patients received different treatment regimens between centers, including vemurafenib alone or in combination with rituximab. Despite the differences in protocols, all patients achieved at least a partial response, with seven patients achieving a complete response. Adverse events were generally mild with manageable side effects. The absence of myelotoxic effects and manageable side effects make BRAF inhibitors attractive, especially for patients ineligible for purine analogues or those with severe neutropenia. Single agent vemurafenib or in combination with rituximab appears to be a promising therapeutic option for R/R HCL. Further research is needed to establish standardized treatment protocols and to investigate long-term outcomes. •Evaluate the use of vemurafenib for relapsed/refractory HCL with BRAF V600E mutation.•There are potential benefits of vemurafenib treatment, particularly in patients who are ineligible for purine analogues or who have severe neutropenia.•Further research is needed to establish standardized treatment protocols for vemurafenib monotherapy or vemurafenib in combination with rituximab in HCL.