Autologous stem cell transplantation (autoHCT) is considered standard of care for newly diagnosed multiple myeloma (MM). Although most patients eventually progress after autoHCT, a small proportion achieve a durable response. In this retrospective study we included 1576 patients, 244 (15%) of whom were long-term responders (LTR), defined as having a progression-free survival (PFS) of ≥8 years after transplant. Patients in the LTR group were younger than the non-LTR group (median age 58.4 vs. 59.5 years; p = 0.012), less likely to have high-risk cytogenetics (4% vs. 14%; p < 0.001), more often had <50% bone marrow plasma cells (67% vs. 58%; p = 0.018) and R-ISS stage I disease (43% vs. 34%). More patients in the LTR group received post-transplant maintenance (63% vs. 52%; p = 0.002). Patients in the LTR group had higher rates of complete response (CR) at day100 (41% vs. 27%; p < 0.001) and at best post-transplant response (70% vs. 37%; p < 0.001), compared to the non-LTR group. Patients in the LTR groups had a median PFS of 169.3 months and the median overall survival (OS) had not been reached. The leading cause of death in the LTR was disease progression. In conclusion, 15% of patients in the cohort were LTR after upfront autoHCT, with distinct characteristics and a median PFS of more than 14 years.
自体干细胞移植（autoHCT）被视为新诊断多发性骨髓瘤（MM）的标准治疗方法。尽管大多数患者在autoHCT后最终会进展，但一小部分患者可获得持久应答。在这项回顾性研究中，我们纳入了1576名患者，其中244名（15%）为长期应答者（LTR），定义为移植后无进展生存期（PFS）≥8年。LTR组患者比非LTR组更年轻（中位年龄58.4岁 vs. 59.5岁；p=0.012），更少具有高危细胞遗传学（4% vs. 14%；p<0.001），更多患者骨髓浆细胞<50%（67% vs. 58%；p=0.018）且为R-ISS分期I期疾病（43% vs. 34%）。LTR组中更多患者接受了移植后维持治疗（63% vs. 52%；p=0.002）。与非LTR组相比，LTR组在第100天完全缓解（CR）率更高（41% vs. 27%；p<0.001），且在移植后最佳应答时CR率更高（70% vs. 37%；p<0.001）。LTR组患者的中位PFS为169.3个月，中位总生存期（OS）尚未达到。LTR组中死亡的主要原因是疾病进展。总之，该队列中15%的患者在前期autoHCT后成为LTR，具有独特特征，且中位PFS超过14年。
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