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

BRAF inhibitor Hairy cell leukemia Rituximab Vemurafenib

Journal

Leukemia research
ISSN: 1873-5835
Titre abrégé: Leuk Res
Pays: England
ID NLM: 7706787

Informations de publication

Date de publication:
29 Mar 2024
Historique:
received: 26 01 2024
revised: 07 03 2024
accepted: 21 03 2024
medline: 11 4 2024
pubmed: 11 4 2024
entrez: 10 4 2024
Statut: aheadofprint

Résumé

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.

Sections du résumé

BACKGROUND BACKGROUND
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.
PATIENTS AND METHODS METHODS
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.
RESULTS RESULTS
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.
CONCLUSION CONCLUSIONS
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.

Identifiants

pubmed: 38599153
pii: S0145-2126(24)00061-4
doi: 10.1016/j.leukres.2024.107495
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

107495

Informations de copyright

Copyright © 2024 Elsevier Ltd. All rights reserved.

Déclaration de conflit d'intérêts

Declaration of Competing Interest All of the authors declare that there is no conflict of interest.

Auteurs

Süreyya Yiğit Kaya (S)

Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Turkey. Electronic address: sureyya.yigitkaya@medipol.com.tr.

Yaşa Gül Mutlu (YG)

Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Istanbul, Turkey.

Ümit Yavuz Malkan (ÜY)

Department of Hematology, Faculty of Medicine, Hacettepe University, Turkey.

Özgür Mehtap (Ö)

Department of Hematology, Faculty of Medicine, Kocaeli University, Turkey.

Fatma Keklik Karadağ (F)

Department of Hematology, Faculty of Medicine, Ege University, Turkey.

Gülten Korkmaz (G)

Department of Hematology, Ankara Bilkent City Hospital, Turkey.

Tuğrul Elverdi (T)

Department of Hematology, Cerrahpaşa Faculty of Medicine, Istanbul University, Turkey.

Güray Saydam (G)

Department of Hematology, Faculty of Medicine, Ege University, Turkey.

Gülsüm Özet (G)

Department of Hematology, Ankara Bilkent City Hospital, Turkey.

Muhlis Cem Ar (MC)

Department of Hematology, Cerrahpaşa Faculty of Medicine, Istanbul University, Turkey.

Elif Melek (E)

Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Istanbul, Turkey.

Senem Maral (S)

Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Istanbul, Turkey.

Leylagül Kaynar (L)

Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Istanbul, Turkey.

Ömür Gökmen Sevindik (ÖG)

Department of Hematology, Faculty of Medicine, Istanbul Medipol University, Istanbul, Turkey.

Classifications MeSH