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自体干细胞移植治疗多发性骨髓瘤的长期疗效。

Long-term outcomes after autologous stem cell transplantation for multiple myeloma.

机构信息

Cancer Research and Biostatistics, Seattle, WA.

Tisch Cancer Institute of the Icahn School of Medicine at Mount Sinai, New York, NY.

出版信息

Blood Adv. 2020 Jan 28;4(2):422-431. doi: 10.1182/bloodadvances.2019000524.

Abstract

As multiple myeloma (MM) treatments evolve, frequent updates are required to monitor the long-term effect of changes in approach. Traditionally, MM is considered an incurable disease, with most patients eventually relapsing. However, improvements in treatments has raised the possibility that MM might be functionally curable. To examine improvements in long-term survival, we followed 4329 patients with newly diagnosed MM treated with autologous stem cell transplantation (ASCT) at the University of Arkansas for Medical Sciences from 1989 through 2018. Overall survival (OS) and progression-free survival (PFS) were evaluated using Kaplan-Meier analysis, Cox proportional hazards models, relative survival analysis, and cure modeling among different time periods, risk groups, and demographic traits. Steady improvements in OS were found, with patients treated in 2014 or later having superior OS (hazard ratio, 0.35; 95% confidence interval [CI], 0.27-0.45) and reduced excess risk for MM death (relative excess risk, 0.30; 95% CI, 0.22-0.41) compared with patients treated in 1997 or earlier. Patients treated during intervening time periods often had intermediate survival, but trends in OS, PFS, and landmarked analyses were inconsistent. Cure models support the potential for cure, ranging from 6.3% to 31.3%, depending on the year of treatment, with 10.0% to 18.6% of patients achieving their normal life expectancy across multiple periods. There was some evidence of reductions in early mortality within 3 years of diagnosis, longer complete response (CR) duration, and reductions in relapse after achieving CR. However, results differed depending on age, risk group, and cytogenetic characteristics.

摘要

随着多发性骨髓瘤 (MM) 治疗方法的不断发展,需要频繁更新以监测方法改变的长期效果。传统上,MM 被认为是一种无法治愈的疾病,大多数患者最终会复发。然而,治疗方法的改进提高了 MM 可能在功能上可治愈的可能性。为了研究长期生存的改善情况,我们随访了 1989 年至 2018 年在阿肯色大学医学科学中心接受自体干细胞移植 (ASCT) 治疗的 4329 例新诊断 MM 患者。使用 Kaplan-Meier 分析、Cox 比例风险模型、相对生存分析和不同时间段、风险组和人口统计学特征的治愈模型评估总生存 (OS) 和无进展生存 (PFS)。发现 OS 稳步改善,2014 年或之后接受治疗的患者 OS 更高(风险比,0.35;95%置信区间 [CI],0.27-0.45),死于 MM 的超额风险降低(相对超额风险,0.30;95%CI,0.22-0.41)与 1997 年或更早接受治疗的患者相比。接受治疗的患者生存时间往往处于中间水平,但 OS、PFS 和里程碑分析的趋势不一致。治愈模型支持治愈的可能性,范围从 6.3%到 31.3%,具体取决于治疗年份,多个时期有 10.0%至 18.6%的患者达到其正常预期寿命。在诊断后 3 年内有减少早期死亡率、更长的完全缓解 (CR) 持续时间和 CR 后减少复发的一些证据。然而,结果因年龄、风险组和细胞遗传学特征而异。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/dd1d/6988393/ef2fcea605a4/advancesADV2019000524absf1.jpg

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