Long-term outcomes in patients with relapsed or refractory hairy cell leukemia treated with vemurafenib monotherapy Journal Article


Authors: Handa, S.; Lee, J. O.; Derkach, A.; Stone, R. M.; Saven, A.; Altman, J. K.; Grever, M. R.; Rai, K. R.; Shukla, M.; Vemuri, S.; Montoya, S.; Taylor, J.; Abdel-Wahab, O.; Tallman, M. S.; Park, J. H.
Article Title: Long-term outcomes in patients with relapsed or refractory hairy cell leukemia treated with vemurafenib monotherapy
Abstract: Vemurafenib, an oral BRAF inhibitor, has demonstrated high response rates in relapsed/refractory (R/R) hairy cell leukemia (HCL). However, little is known about long-term outcomes and response to retreatment. Herein, we report the results of 36 patients with R/R HCL treated with vemurafenib from the United States arm of the phase 2 clinical trial (NCT01711632). The best overall response rate was 86%, including 33% complete response (CR) and 53% partial response (PR). After a median follow-up of 40 months, 21 of 31 responders (68%) experienced relapse with a median relapse-free survival (RFS) of 19 months (range, 12.5-53.9 months). There was no significant difference in the RFS for patients with CR vs PR. Fourteen of 21 (67%) relapsed patients were retreated with vemurafenib, with 86% achieving complete hematologic response. Two patients acquired resistance to vemurafenib with the emergence of new KRAS and CDKN2A mutations, respectively. Six of 12 (50%) responders to vemurafenib retreatment experienced another relapse with a median RFS of 12.7 months. Overall survival (OS) was 82% at 4 years, with a significantly shorter OS in patients who relapsed within 1 year of initial treatment with vemurafenib. Higher cumulative doses or a longer duration of treatment did not lengthen the durability of response. All adverse events in the retreatment cohort were grade 1/2 except for 1 case of a grade 3 rash and 1 grade 3 fever/pneumonia. Our data suggest that vemurafenib retreatment is a safe and effective option for patients with R/R HCL. © 2022 The American Society of Hematology
Keywords: genetics; antineoplastic agents; antineoplastic agent; protein kinase inhibitor; protein kinase inhibitors; remission; remission induction; b raf kinase; proto-oncogene proteins b-raf; hairy cell leukemia; vemurafenib; leukemia, hairy cell; humans; human
Journal Title: Blood
Volume: 140
Issue: 25
ISSN: 0006-4971
Publisher: American Society of Hematology  
Date Published: 2022-12-22
Start Page: 2663
End Page: 2671
Language: English
DOI: 10.1182/blood.2022016183
PUBMED: 35930750
PROVIDER: scopus
PMCID: PMC9935554
DOI/URL:
Notes: Article -- Export Date: 3 January 2023 -- Source: Scopus
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MSK Authors
  1. Martin Stuart Tallman
    649 Tallman
  2. Jae Hong Park
    357 Park
  3. Shreya Vemuri
    15 Vemuri
  4. Andriy Derkach
    148 Derkach
  5. Madhulika Shukla
    6 Shukla