Venetoclax for the treatment of multiple myeloma: Outcomes outside of clinical trials

M. Hasib Sidiqi, Abdullah S. Al Saleh, Shaji K. Kumar, Nelson Leung, Dragan Jevremovic, Eli Muchtar, Wilson I. Gonsalves, Taxiarchis V. Kourelis, Rahma Warsame, Francis K. Buadi, Martha Q. Lacy, Robert A. Kyle, Ronald Go, Miriam Hobbs, Angela Dispenzieri, David Dingli, Suzanne R. Hayman, Morie A. Gertz, S. Vincent Rajkumar, Prashant Kapoor

Research output: Contribution to journalArticlepeer-review


Multiple myeloma (MM) remains an incurable disease despite incorporation of novel agents. Venetoclax, a B-cell lymphoma 2 (BCL-2) inhibitor is approved for some hematologic malignancies but not yet for MM, although clinical trials have shown efficacy in patients with MM, particularly those harboring t(11;14). We reviewed the medical records of relapsed and/or refractory MM patients to study the efficacy and safety of venetoclax used outside of clinical trials at Mayo Clinic between December, 2016 and March, 2019. The data cut-off date was August 06, 2020. We identified 56 patients of whom 42 (75%) harbored t(11;14). The median number of prior therapies was six (range 1–15) and 14% of patients had received ≥10 prior lines of therapy. Fifty-three (95%) patients were refractory to an immunomodulatory drug and proteasome inhibitor. Venetoclax was used as monotherapy or doublet, in combination with dexamethasone in 55% (n = 31) and a triplet or quadruplet in 45% of patients. No patient experienced tumor lysis syndrome. Overall response rate in 52 evaluable patients was 44%. The median time to best response was 2 months and median duration of response was 13.6 months. The median PFS for the entire cohort was 5.8 (95% CI 4.9–10.3) months and median OS was 28.4 (95% CI 14.6-not reached) months. The presence of t(11;14) was associated with improved PFS (median 9.7 months vs. 4.2 months, p = 0.019) and OS (median not reached vs. 10.8 9 months, p = 0.015). Venetoclax demonstrates encouraging activity in heavily-treated patients with relapsed/refractory MM, particularly the t(11;14) patient-population.

Original languageEnglish (US)
Pages (from-to)1131-1136
Number of pages6
JournalAmerican journal of hematology
Issue number9
StatePublished - Sep 1 2021

ASJC Scopus subject areas

  • Hematology


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