Journal
LEUKEMIA
Volume 36, Issue 2, Pages 532-539Publisher
SPRINGERNATURE
DOI: 10.1038/s41375-021-01417-9
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Funding
- Pharmacyclics
- David and Janet Bingham Research Fund of the International Waldenstrom's Macroglobulinemia Foundation
- Yang Family Research Fund of the International Waldenstrom's Macroglobulinemia Foundation
- Leukemia and Lymphoma Society [R6507-18]
- NIH SPORE in Multiple Myeloma [2P50CA100707-16A1]
- Edward and Linda Nelson Fund for WM Research
- Kerry Robertson Fund for WM Research
- Bauman Family Trust
- Siegel Family Fund for WM
- Kinogen Inc.
- WMR Fund
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Ibrutinib monotherapy showed durable responses in treatment-naive patients with Waldenstrom macroglobulinemia, with CXCR4 mutations impacting VGPR attainment, time to major response, and 4-year PFS rate.
Herein, we present the final report of a single-center, prospective phase II study evaluating ibrutinib 420 mg once daily in 30 treatment-naive patients with Waldenstrom macroglobulinemia (WM). The present study is registered with ClinicalTrials.Gov (NCT02604511). With a median follow-up of 50 months, the overall, major, and VGPR response rates were 100%, 87%, and 30%. The VGPR rate was numerically but not significantly lower in patients with than without CXCR4 mutations (14% vs. 44%; p = 0.09). The median time to a minor response was 0.9 months, and to a major response was 1.9 months, though were longer in those with mutated CXCR4 at 1.7 months (p = 0.07) and 7.3 months (p = 0.01). Six patients had disease progression. The median progression-free survival (PFS) was not reached, and the 4-year PFS rate was 76%. There was also a non-significant lower 4-year PFS rate in patients with than without CXCR4 mutations (59% vs. 92%; p = 0.06). The most common treatment-related adverse events were fatigue, upper respiratory infection, and hematoma. Atrial fibrillation occurred in 20% of patients. Ibrutinib monotherapy induced durable responses in treatment-naive patients with WM. CXCR4 mutations impacted VGPR attainment, time to major response, and 4-year PFS rate.
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