Wang Yue, Xiong Yaqin, Gu Shiyang, Wang Wenjing, Yang Yang, Liu Peng
Department of Hematology, Zhongshan Hospital, Fudan University, 180 Fenglin Road, Shanghai, China.
Discov Oncol. 2025 Apr 16;16(1):534. doi: 10.1007/s12672-025-02356-y.
Autologous hematopoietic stem cell transplantation (ASCT) is a recommended treatment for multiple myeloma (MM). Currently, with multiple treatment alternatives, patients' prognosis has improved significantly compared to the pre-proteasome inhibitor period. However, this has raised deliberations on the value and timing of ASCT. Compared with Western countries, the proportion of Chinese patients undergoing ASCT is relatively low. Nevertheless, this situation allows us to observe the treatment outcomes of transplant-eligible (TE) patients who haven't received ASCT and explore the role of ASCT in patients with distinct clinicopathological features. This real-world analysis encompassed 1059 newly diagnosed MM patients from 2012 to 2022, among whom 480 were TE. These patients were categorized into the TE-ASCT group (158 received ASCT) and the TE-no-ASCT group (322 did not receive ASCT). Disease progression and treatment response are evaluated based on the definition of IMWG. We found that the progression-free survival (PFS) was significantly prolonged in TE-ASCT group compared to TE-no-ASCT group, but there was no significant difference in overall survival (OS). Further exploratory analyses revealed that ASCT conspicuously augmented the PFS of patients aged ≤ 60 years, featuring ISS stage II/III, 1q gain/amplification, and positive minimal residual disease (MRD). Nevertheless, for patients aged > 60 years, presenting with ISS stage I, high-risk cytogenetics, renal insufficiency, and negative MRD, ASCT did not confer PFS benefits. Our investigation furnishes evidence of the clinical superiority of ASCT for MM patients with disparate clinicopathological characteristics in the contemporary treatment era, laying a groundwork for individualized ASCT selection.
自体造血干细胞移植（ASCT）是多发性骨髓瘤（MM）的推荐治疗方法。目前，有多种治疗选择，与蛋白酶体抑制剂应用前的时期相比，患者的预后有了显著改善。然而，这引发了关于ASCT的价值和时机的讨论。与西方国家相比，接受ASCT的中国患者比例相对较低。尽管如此，这种情况使我们能够观察未接受ASCT的移植 eligible（TE）患者的治疗结果，并探索ASCT在具有不同临床病理特征患者中的作用。这项真实世界分析纳入了2012年至2022年期间1059例新诊断的MM患者，其中480例为TE患者。这些患者被分为TE-ASCT组（158例接受了ASCT）和TE-no-ASCT组（322例未接受ASCT）。根据国际骨髓瘤工作组（IMWG）的定义评估疾病进展和治疗反应。我们发现，与TE-no-ASCT组相比，TE-ASCT组的无进展生存期（PFS）显著延长，但总生存期（OS）无显著差异。进一步的探索性分析显示，ASCT显著延长了年龄≤60岁、ISS分期为II/III期、1q获得/扩增且微小残留病（MRD）阳性患者的PFS。然而，对于年龄>60岁、ISS分期为I期、具有高危细胞遗传学、肾功能不全且MRD阴性的患者，ASCT并未带来PFS获益。我们的研究为当代治疗时代不同临床病理特征的MM患者ASCT的临床优势提供了证据，为个体化ASCT选择奠定了基础。
