Profile Response Detail

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Molecular Profile BRAF V600E
Therapy Vemurafenib
Indication/Tumor Type hairy cell leukemia
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
BRAF V600E hairy cell leukemia sensitive Vemurafenib Case Reports/Case Series Actionable In a clinical case study, Zelboraf (vemurafenib) treatment resulted in complete resolution of the brain lesions after 3 months of treatment, decreased spleen size, and resolution of leukemic retinopathy in a patient with hairy cell leukemia harboring BRAF V600E (PMID: 36713531). 36713531
BRAF V600E hairy cell leukemia sensitive Vemurafenib Phase II Actionable In two Phase II clinical studies, patients with refractory hairy cell leukemia harboring BRAF V600E responded to Zelboraf (vemurafenib) with overall response rates of 96% (25/26) and 100% (24/24) as well as complete response rates of 35% (9/26) and 42% (10/24) with median follow up times of 8 and 12 weeks, respectively (PMID: 26352686). 26352686
PubMed Id Reference Title Details
(26352686) Targeting Mutant BRAF in Relapsed or Refractory Hairy-Cell Leukemia. Full reference...
(36713531) Case report: A case of classic hairy cell leukemia with CNS involvement treated with vemurafenib. Full reference...