{"database":"biostudies-literature","file_versions":[],"scores":null,"additional":{"submitter":["Kreitman RJ"],"funding":["NCI NIH HHS"],"pagination":["996-1006"],"full_dataset_link":["https://www.ebi.ac.uk/biostudies/studies/S-EPMC10163281"],"repository":["biostudies-literature"],"omics_type":["Unknown"],"volume":["141(9)"],"pubmed_abstract":["BRAF V600E is the key oncogenic driver mutation in hairy cell leukemia (HCL). We report the efficacy and safety of dabrafenib plus trametinib in patients with relapsed/refractory BRAF V600E mutation-positive HCL. This open-label, phase 2 study enrolled patients with BRAF V600E mutation-positive HCL refractory to first-line treatment with a purine analog or relapsed after ≥2 prior lines of treatment. Patients received dabrafenib 150 mg twice daily plus trametinib 2 mg once daily until disease progression, unacceptable toxicity, or death. The primary endpoint was investigator-assessed objective response rate (ORR) per criteria adapted from National Comprehensive Cancer Network-Consensus Resolution guidelines. Secondary endpoints included duration of response (DOR), progression-free survival "],"journal":["Blood"],"pubmed_title":["Dabrafenib plus trametinib in patients with relapsed/refractory BRAF V600E mutation-positive hairy cell leukemia."],"pmcid":["PMC10163281"],"funding_grant_id":["P30 CA016672","R01 CA242845"],"pubmed_authors":["Gazzah A","De Greve J","Arons E","Hutchings M","Subbiah V","Moreau P","Kreitman RJ","de Jonge MJA","Ilankumaran P","Gasal E","Michallet AS","Wainberg ZA","Burgess P","Stein A","Willenbacher W","Dietrich S","Ravandi F"],"additional_accession":[]},"is_claimable":false,"name":"Dabrafenib plus trametinib in patients with relapsed/refractory BRAF V600E mutation-positive hairy cell leukemia.","description":"BRAF V600E is the key oncogenic driver mutation in hairy cell leukemia (HCL). We report the efficacy and safety of dabrafenib plus trametinib in patients with relapsed/refractory BRAF V600E mutation-positive HCL. This open-label, phase 2 study enrolled patients with BRAF V600E mutation-positive HCL refractory to first-line treatment with a purine analog or relapsed after ≥2 prior lines of treatment. Patients received dabrafenib 150 mg twice daily plus trametinib 2 mg once daily until disease progression, unacceptable toxicity, or death. The primary endpoint was investigator-assessed objective response rate (ORR) per criteria adapted from National Comprehensive Cancer Network-Consensus Resolution guidelines. Secondary endpoints included duration of response (DOR), progression-free survival ","dates":{"release":"2023-01-01T00:00:00Z","publication":"2023 Mar","modification":"2026-05-28T18:32:48.84Z","creation":"2025-04-19T02:42:50.328Z"},"accession":"S-EPMC10163281","cross_references":{"pubmed":["36108341"],"doi":["10.1182/blood.2021013658"]}}