Abstract：
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.
摘要：
这项II期研究评估了新诊断（NDWM；n=9）和复发性/难治性（RRWM；n=12）Waldenström巨球蛋白血症（WM）的依鲁替尼和伊沙佐米的限时（24个周期）治疗。21名可评估患者的总反应率（ORR）为76.2％（n=16），没有患者达到完全反应（CR）。中位治疗时间为15.6个月，在中位随访时间为25.7个月后，中位无进展生存期(PFS)为22.9个月.虽然未达到主要终点（任何时间的CR率），并且28.5％由于毒性而停止治疗，伊布替尼联合艾沙佐米可导致具有临床意义的ORR和PFS.联合布鲁顿酪氨酸激酶（BTK）和蛋白酶体抑制值得在WM中进一步评估。
