文章：

多发性骨髓瘤中自体干细胞移植的“超级应答者”特征

Characteristics of exceptional responders to autologous stem cell transplantation in multiple myeloma

原文发布日期：2020-08-28

DOI: 10.1038/s41408-020-00353-8

类型: Article

开放获取: 是

英文摘要：

Autologous stem cell transplantation (ASCT) is an important treatment modality in multiple myeloma (MM). However, relapse following ASCT is considered almost inevitable. This study aimed to characterize exceptional responders to ASCT, defined as progression-free survival (PFS) >8 years in the absence of maintenance therapy. We retrospectively analyzed patients treated at Mayo Clinic between August 1, 1998 and January 3, 2006, and included those with symptomatic MM, treated with an ASCT within 12 months of diagnosis. We found that 46 (9%) of the 509 patients who underwent ASCT during the study period were exceptional responders. The median duration of follow-up from diagnosis was 16.2 (interquartile range 14.3–17.7) years. The best response to therapy was a complete response (CR) or better in 34 (74%) of patients, and less than a CR in 12 (26%) of patients. The median PFS was 13.8 (95% confidence interval 10.5–18.5) years, and at the time of the last hematology assessment, 24 of 46 (52%) patients remained in remission. In conclusion, we showed that a small subset of patients with MM attains durable disease control without maintenance therapy post ASCT. Pre-emptive identification of these patients may help prevent undue toxicities and costs of subsequent therapy.
 

摘要翻译： 

自体干细胞移植（ASCT）是多发性骨髓瘤（MM）的重要治疗手段。然而，移植后的复发几乎被认为是不可避免的。本研究旨在明确ASCT后特殊应答者的特征，其定义为在无维持治疗的情况下无进展生存期（PFS）超过8年的患者。我们回顾性分析了1998年8月1日至2006年1月3日期间在梅奥诊所接受治疗的患者，纳入标准为确诊后12个月内接受ASCT治疗的症状性多发性骨髓瘤患者。研究发现，在509例接受ASCT治疗的患者中，46例（9%）属于特殊应答者。从诊断开始的中位随访时间为16.2年（四分位距14.3-17.7年）。治疗最佳反应为完全缓解（CR）或更佳水平的患者34例（74%），未达到完全缓解的患者12例（26%）。中位无进展生存期为13.8年（95%置信区间10.5-18.5年），截至末次血液学评估时，46例患者中仍有24例（52%）处于缓解状态。研究表明，一小部分多发性骨髓瘤患者在接受ASCT后无需维持治疗即可获得持久的疾病控制。对这些患者进行预先识别可能有助于避免后续治疗带来的不当毒性和经济负担。

原文链接：

Characteristics of exceptional responders to autologous stem cell transplantation in multiple myeloma