自体造血干细胞移植（ASCT）后进行Lenalidomide维持治疗可以提高多发性骨髓瘤（MM）的无进展生存期和总生存期。然而，高危多发性骨髓瘤（HRMM）患者与标准风险患者相比，从Lenalidomide维持治疗中获得的生存益处并不相同。作者试图确定与Lenalidomide维持治疗相比，基于Bortezomib的维持治疗在接受ASCT的HRMM患者中的结果。总共，作者在国际血液和骨髓移植研究中心的数据库中确定了503名HRMM患者，在诊断后12个月内接受三联新型药物诱导后接受ASCT治疗，期间从2013年1月至2018年12月。HRMM被定义为删除17p、t(14;16)、t(4;14)、t(14;20)或染色体1q增益。其中，357名患者（67%）仅接受Lenalidomide治疗，而146名（33%）接受基于Bortezomib的维持治疗（仅58名患者接受Bortezomib单药）。Bortezomib-based维持治疗组的患者更可能携带两个或更多高危异常，且国际分期系统（ISS）分期为III期疾病（30% vs. 22%; p = .01），相对于Lenalidomide组（24% vs. 15%; p < .01）。接受Lenalidomide维持治疗的患者在2年内的无进展生存期优于接受Bortezomib单药或联合治疗的患者（75% vs. 63%; p = .009）。2年内总生存率在Lenalidomide组中也更高（93% vs. 84%; p = .001）。与Lenalidomide单独治疗相比，接受Bortezomib单药或联合治疗的HRMM患者未观察到优越的治疗结果（维持治疗）。在有随机临床试验的前景数据之前，应根据每个患者的情况量身定制移植后治疗，考虑治疗临床试验的患者，以针对HRMM的新颖治疗策略为目标。Lenalidomide应继续作为治疗的基石。©2023 American Cancer Society。

Lenalidomide maintenance after autologous stem cell transplant (ASCT) in multiple myeloma (MM) results in superior progression-free survival and overall survival. However, patients with high-risk multiple myeloma (HRMM) do not derive the same survival benefit from lenalidomide maintenance compared with standard-risk patients. The authors sought to determine the outcomes of bortezomib-based maintenance compared with lenalidomide maintenance in patients with HRMM undergoing ASCT.In total, the authors identified 503 patients with HRMM who were undergoing ASCT within 12 months of diagnosis from January 2013 to December 2018 after receiving triplet novel-agent induction in the Center for International Blood and Marrow Transplant Research database. HRMM was defined as deletion 17p, t(14;16), t(4;14), t(14;20), or chromosome 1q gain.Three hundred fifty-seven patients (67%) received lenalidomide alone, and 146 (33%) received bortezomib-based maintenance (with bortezomib alone in 58%). Patients in the bortezomib-based maintenance group were more likely to harbor two or more high-risk abnormalities and International Staging System stage III disease (30% vs. 22%; p = .01) compared with the lenalidomide group (24% vs. 15%; p < .01). Patients who were receiving lenalidomide maintenance had superior progression-free survival at 2 years compared with those who were receiving either bortezomib monotherapy or combination therapy (75% vs. 63%; p = .009). Overall survival at 2 years was also superior in the lenalidomide group (93% vs. 84%; p = .001).No superior outcomes were observed in patients with HRMM who received bortezomib monotherapy or (to a lesser extent) in those who received bortezomib in combination as maintenance compared with lenalidomide alone. Until prospective data from randomized clinical trials are available, post-transplant therapy should be tailored to each patient with consideration for treating patients in clinical trials that target novel therapeutic strategies for HRMM, and lenalidomide should remain a cornerstone of treatment.© 2023 American Cancer Society.