Mayo Clinic Cancer Center, Jacksonville, Florida, USA.
Department of Cancer Biology, Mayo Clinic, Jacksonville, Florida, USA.
Br J Haematol. 2024 May;204(5):1825-1829. doi: 10.1111/bjh.19320. Epub 2024 Jan 29.
This phase II study evaluated time-limited (24 cycles) treatment with ibrutinib and ixazomib in newly diagnosed (NDWM; n = 9) and relapsed/refractory (RRWM; n = 12) Waldenström macroglobulinaemia (WM). The overall response rate (ORR) was 76.2% (n = 16) in 21 evaluable patients with no patient achieving a complete response (CR). The median duration of treatment was 15.6 months, and after a median follow-up time of 25.7 months, the median progression-free survival (PFS) was 22.9 months. While the primary end-point was not met (CR rate at any time) and 28.5% discontinued treatment due to toxicity, ibrutinib plus ixazomib led to a clinically meaningful ORR and PFS. Combined Bruton's tyrosine kinase (BTK) and proteasome inhibition merits further evaluation in WM.
这项 II 期研究评估了伊布替尼和来那度胺联合治疗初诊（n = 9）和复发/难治性（n = 12）华氏巨球蛋白血症（WM）患者的限时（24 个周期）治疗。21 例可评估患者的总体缓解率（ORR）为 76.2%（n = 16），无患者达到完全缓解（CR）。中位治疗持续时间为 15.6 个月，中位随访时间为 25.7 个月后，中位无进展生存期（PFS）为 22.9 个月。尽管主要终点未达到（任何时间的 CR 率），且由于毒性有 28.5%的患者停止治疗，但伊布替尼联合来那度胺导致了具有临床意义的 ORR 和 PFS。联合 Bruton 酪氨酸激酶（BTK）和蛋白酶体抑制值得进一步评估在 WM 中的作用。
