Background: Despite major treatment advances, multiple myeloma remains incurable. The outcome of patients who are refractory to immunomodulatory agents, proteasome inhibitors, and anti-CD38 monoclonal antibodies is poor, and improved treatment strategies for this difficult-to-treat patient population are an unmet medical need. Methods: This retrospective, unicentric analysis included 38 patients with relapsed/refractory multiple myeloma or plasma cell leukemia who underwent allogeneic stem cell transplantation (allo-HSCT) between 2013 and 2022. Survival outcomes, relapse incidence, and non-relapse mortality were calculated according to remission status, date of allo-HSCT, cytogenetic risk status, timing, and number of previous autologous HSCTs. Results: The median PFS was 13.6 months (95% CI, 7.7–30.4) and the median OS was 51.4 months (95% CI, 23.5–NA) in the overall cohort. The cumulative incidence of relapse at 3 years was 57%, and non-relapse mortality was 16%. The median PFS and OS were significantly longer in patients with very good partial remission (VGPR) or better compared to patients with less than VGPR at the time of allo-HSCT (mPFS 29.7 months (95% CI, 13.7–NA) vs. 6.5 months (95% CI, 2.6–17.0);p= 0.009 and mOS not reached vs. 18.6 months (95% CI, 7.0–NA);p= 0.006). Conclusion: For selected patients, allo-HSCT may result in favorable overall survival, in part by providing an appropriate hemato-immunological basis for subsequent therapies.
背景：尽管治疗手段取得重大进展，多发性骨髓瘤仍属不可治愈疾病。对免疫调节剂、蛋白酶体抑制剂及抗CD38单克隆抗体均产生耐药的患者预后较差，针对这一难治性患者群体的优化治疗策略仍是亟待满足的临床需求。方法：本回顾性单中心研究纳入2013年至2022年间接受异基因造血干细胞移植的38例复发/难治性多发性骨髓瘤或浆细胞白血病患者。根据移植时缓解状态、移植时间、细胞遗传学风险分层、既往自体移植时机及次数等参数，计算生存结局、复发率及非复发死亡率。结果：全组患者中位无进展生存期为13.6个月（95% CI：7.7-30.4），中位总生存期为51.4个月（95% CI：23.5-未达到）。3年累积复发率为57%，非复发死亡率为16%。移植时达到非常好的部分缓解及以上疗效的患者，其中位无进展生存期与总生存期均显著优于未达该缓解标准的患者（中位无进展生存期：29.7个月[95% CI：13.7-未达到] vs 6.5个月[95% CI：2.6-17.0]，p=0.009；中位总生存期：未达到 vs 18.6个月[95% CI：7.0-未达到]，p=0.006）。结论：对于经筛选的患者，异基因造血干细胞移植可能获得良好的总生存获益，其部分机制在于为后续治疗提供了适宜的血液免疫学基础。
肿瘤(癌症)患者之家