Long-term follow-up of patients with relapsed or refractory non-hodgkin lymphoma treated with venetoclax in a phase 1, first-in-human study
Journal Title
Clinical Cancer Research
Publication Type
epub ahead of print
PURPOSE: We previously reported a 44% overall response rate (ORR) with venetoclax in a phase 1 study of relapsed/refractory NHL. Complete remission (CR) was observed in patients with mantle cell lymphoma ([MCL], 21%, n=6/28) and follicular lymphoma ([FL], 17%, n=5/29), and partial response (PR) noted in several patients with Waldenström macroglobulinemia (WM), and marginal zone lymphoma (MZL). Here, we report the long-term outcomes of these four cohorts. PATIENTS AND METHODS: All patients (n=106) received venetoclax monotherapy in dose cohorts of 200 - 1200 mg daily until disease progression or unacceptable toxicity. ORR, PFS, DoR, and adverse events (AEs) were evaluated. RESULTS: At median follow-up of 38.5 months (range: 30.0 - 46.5), median PFS for 106 patients was 5.4 (95% CI: 3.5 - 8.4) months (FL: 10.8; MCL: 11.3; MZL: 21.2; WM: 30.4). Median DoR was 14.9 (95% CI: 9.7 - 27.6) months (FL: 26.6; MCL: 15.7; MZL: 20.1; WM: 25.3). Achievement of CR vs. PR predicted longer DoR in both MCL (31.5 vs. 10.1 months) and FL (37.6 vs. 9.7 months). All grade hematologic AEs were infrequent: neutropenia (19%), anemia (17%), and thrombocytopenia (15%), with no new cytopenias {greater than or equal to}2 years on therapy. Non-hematological AEs included nausea (47%), diarrhea (43%), fatigue (40%), with decreased incidence {greater than or equal to}1 year. CONCLUSIONS: Venetoclax monotherapy has a manageable safety profile and achieves durable responses in a subset of patients with FL, MCL, WM, and MZL, particularly in those who achieve CR. Further research is warranted on combination strategies to enhance the durability of response to venetoclax.
WEHI Research Division(s)
Blood Cells And Blood Cancer
PubMed ID
Open Access at Publisher's Site
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Creation Date: 2021-06-10 11:46:59
Last Modified: 2021-06-10 12:10:47
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