AL淀粉样变性病患者的患者报告结局尚未得到良好研究。我们从第3期TOURMALINE-AL1试验（NCT01659658）（伊扎莫司-地塞米松，n = 85；医师选择的化疗[PC]，n = 83）中分析了与健康相关的生命质量（HRQOL）和AL淀粉样变性病症状的数据。利用SF-36v2、癌症治疗功能评估/妇科肿瘤学组神经毒性子量表（FACT/GOG-Ntx）和淀粉样变性症状问卷（ASQ）进行HRQOL和症状负担的测量。治疗期间的得分变化进行了描述性的分析和重复测量线性混合模型的分析；分析没有针对多重性进行调整。基线的最小二乘（LS）均值变化在某些周期内伊扎莫司-地塞米松的SF-36v2角色和生命力量表上显著较高（更好的HRQOL）（p < .05）；没有子量表表明PC有显著优势。对于FACT/GOG-Ntx，LS均值变化的微小但显著差异有利于伊扎莫司-地塞米松在多个周期中的七个项目和两个汇总得分；一个项目（听力障碍）的LS均值变化有利于PC在多个周期中。ASQ总分在两组中呈下降趋势（负担较轻）；一些周期内显著LS均值差异有利于伊扎莫司-地塞米松（p < .05）。与接受PC治疗的患者相比，接受伊扎莫司-地塞米松治疗的复发/难治性AL淀粉样变性病患者体验到的HRQOL和症状相似或有所改善，尽管治疗时间更长。© 2023 武田制药公司和作者。由Wiley Periodicals LLC出版的《美国血液学杂志》。

Patient-reported outcomes in AL amyloidosis have not been well-studied. We analyzed health-related quality of life (HRQOL) and AL amyloidosis symptoms data from the phase 3 TOURMALINE-AL1 trial (NCT01659658) (ixazomib-dexamethasone, n = 85; physician's choice of chemotherapy [PC], n = 83). HRQOL and symptom burden were measured with the SF-36v2, Functional Assessment of Cancer Therapy/Gynecologic Oncology Group Neurotoxicity subscale (FACT/GOG-Ntx), and an amyloidosis symptom questionnaire (ASQ). Score changes during treatment were analyzed descriptively and using repeated-measures linear mixed models; analyses were not adjusted for multiplicity. Least-squares (LS) mean changes from baseline were significantly higher (better HRQOL) for ixazomib-dexamethasone at several cycles for SF-36v2 Role Physical and Vitality subscales (p < .05); no subscales demonstrated significant differences favoring PC. For FACT/GOG-Ntx, small but significant differences in LS mean changes favored ixazomib-dexamethasone over PC at multiple cycles for seven items and both summary scores; significant differences favored PC for one item (trouble hearing) at multiple cycles. ASQ total score trended downward (lower burden) in both arms; significant LS mean differences favored ixazomib-dexamethasone over PC at some cycles (p < .05). Patients with relapsed/refractory AL amyloidosis treated with ixazomib-dexamethasone experienced HRQOL and symptoms that were similar to or trended better than patients treated with PC despite longer duration of therapy.© 2023 Takeda Pharmaceutical Company and The Authors. American Journal of Hematology published by Wiley Periodicals LLC.