A single-center retrospective cohort analysis of venetoclax in relapsed/refractory multiple myeloma

Swetha Kambhampati1, Derek Galligan2, Chiung-Yu Huang3

  • 1Department of Medicine, Division of Hematology and Oncology, University of California San Francisco, San Francisco, CA, USA.

Leukemia & Lymphoma
|January 14, 2020
PubMed

Insights

Venetoclax shows promise for relapsed refractory multiple myeloma (RRMM). It demonstrated efficacy, particularly in patients with the t(11;14) genetic marker, suggesting a potential new treatment option.

Area of Science:

  • Hematology
  • Oncology
  • Pharmacology

Background:

  • Venetoclax, a BCL-2 inhibitor, shows preclinical and early clinical promise in relapsed refractory multiple myeloma (RRMM).
  • Clinical application of venetoclax in RRMM requires further investigation.

Purpose of the Study:

  • To evaluate the safety and efficacy of off-label venetoclax use in patients with RRMM.
  • To assess treatment outcomes based on the presence of the t(11;14) chromosomal translocation.

Main Methods:

  • Retrospective analysis of 47 RRMM patients treated with off-label venetoclax at a single center.
  • Analysis of patient demographics, prior therapies, treatment regimens, and clinical outcomes.
  • Subgroup analysis for patients with known t(11;14) translocation.

Main Results:

  • Overall response rate (ORR) was 39%, with 17% achieving very good partial response (VGPR).
  • In the t(11;14) subgroup, ORR was 71%, with 24% achieving VGPR.
  • Median progression-free survival was 2.1 months, and median overall survival was 15.6 months. One treatment-related death occurred.

Conclusions:

  • Venetoclax appears safe and effective in RRMM patients, especially those with t(11;14).
  • Further evaluation in prospective clinical trials is warranted to confirm these findings.

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