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Published on: December 9, 2015
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.
Abstract:
Venetoclax, a small molecule inhibitor of BCL-2, has promising pre-clinical and early clinical activity in relapsed refractory multiple myeloma (RRMM). However, the clinical use of venetoclax remains under evaluation. We performed a single-center, retrospective analysis of patients with RRMM treated with off-label venetoclax. Forty-seven patients with a median of seven lines of prior therapy were identified. Most patients (87%) received venetoclax plus proteasome inhibitor, though there was heterogeneity in the venetoclax-containing regimen patients received, and 38% had known t(11;14). The overall response rate (ORR) was 39%, with 17% achieving ≥ very good partial response (VGPR). In the t(11;14) cohort, the ORR was 71%, with 24% achieving ≥ VGPR. The median progression-free survival was 2.1 months and overall survival was 15.6 months. One treatment-related death (related to infection) occurred. Venetoclax appears safe and efficacious in patients with RRMM, especially in those with t(11;14). Prospective trials are ongoing to further evaluate this treatment.
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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