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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