Strassl Irene, Nikoloudis Alexander, Machherndl-Spandl Sigrid, Buxhofer-Ausch Veronika, Binder Michaela, Wipplinger Dagmar, Stiefel Olga, Kaynak Emine, Milanov Robert, Aichinger Christoph, Nocker Stefanie, Bauer Thomas, Kreissl Stefanie, Girschikofsky Michael, Petzer Andreas, Weltermann Ansgar, Clausen Johannes
Division of Hematology with Stem Cell Transplantation, Hemostaseology and Medical Oncology, Department of Internal Medicine I, Ordensklinikum Linz, Fadingerstrasse 1, 4020 Linz, Austria.
Medical Faculty, Johannes Kepler University Linz, Altenberger Strasse 69, 4040 Linz, Austria.
Cancers (Basel). 2023 Dec 7;15(24):5738. doi: 10.3390/cancers15245738.
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.
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.
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); = 0.009 and mOS not reached vs. 18.6 months (95% CI, 7.0-NA); = 0.006).
For selected patients, allo-HSCT may result in favorable overall survival, in part by providing an appropriate hemato-immunological basis for subsequent therapies.
尽管在治疗方面取得了重大进展，但多发性骨髓瘤仍然无法治愈。对免疫调节剂、蛋白酶体抑制剂和抗CD38单克隆抗体难治的患者预后较差，针对这一难以治疗的患者群体改进治疗策略是一项未满足的医疗需求。
这项回顾性单中心分析纳入了38例2013年至2022年间接受异基因干细胞移植（allo-HSCT）的复发/难治性多发性骨髓瘤或浆细胞白血病患者。根据缓解状态、allo-HSCT日期、细胞遗传学风险状态、时间以及既往自体HSCT的次数计算生存结局、复发率和非复发死亡率。
整个队列的中位无进展生存期（PFS）为13.6个月（95%置信区间，7.7 - 30.4），中位总生存期（OS）为51.4个月（95%置信区间，23.5 - 无数据）。3年时的累积复发率为57%，非复发死亡率为16%。与allo-HSCT时达到非常好的部分缓解（VGPR）或更好缓解的患者相比，未达到VGPR的患者中位PFS和OS显著更长（mPFS 29.7个月（95%置信区间，13.7 - 无数据）对6.5个月（95%置信区间，2.6 - 17.0）；P = 0.009，mOS未达到对18.6个月（95%置信区间，7.0 - 无数据）；P = 0.006）。
对于选定的患者，allo-HSCT可能带来良好的总生存期，部分原因是为后续治疗提供了合适的血液免疫基础。
