文章：

新型疗法时代下年轻多发性骨髓瘤患者的临床特征、细胞遗传学风险及预后分析

Clinical Characteristics, Cytogenetic Risks, and Prognoses of Young Multiple Myeloma Patients in the Era of Novel Therapies

原文发布日期：6 December 2024

DOI: 10.3390/cancers16234090

类型: Article

开放获取: 是

英文摘要：

Background/Objectives:In recent years, there have been significant advances in the understanding and treatment of multiple myeloma (MM). Despite this progress, there is still limited information on the disease in patients aged 50 or younger, including the impact of young age on disease characteristics, treatment, and outcome.Methods:In this retrospective study, we analyzed 68 newly diagnosed MM patients aged ≤ 50 years (y) who had undergone at least one peripheral blood stem cell transplantation (PBSCT). Additionally, we reviewed data published during 2008–2022 and compared these to our cohort.Results:Of note, the disease characteristics in our cohort were similar to those in older patients. However, the incidence of bone lesions was higher in younger patients (84%). Moreover, 33% had LC-only MM and 7% had high-risk (del17p, t(14;16), t(4;14)) cytogenetics. Advanced ISS and R-ISS II/III were observed in 57% and 78%, respectively. Therapy was intense, with 53% of patients undergoing ≥2 SCTs. Median follow-up was 75 months, median progression-free survival was 57 months, and median overall survival (OS) was not reached. The 10-year OS rate was 72%, with only 19% succumbing to the disease. Notably, no specific therapeutic regimen or risk factors for worse outcomes were identified through uni- or bivariate analyses, even in subgroup analyses of younger patients aged ≤ 40 y.Conclusions:Our, and prior, results of young (<50 y) and very young (<40 y) MM patients underscore the need for further comprehensive studies focused on this significantly affected cohort.

摘要翻译： 

背景/目的：近年来，多发性骨髓瘤（MM）的认知与治疗取得了显著进展。尽管取得这些进步，关于50岁及以下患者群体的疾病信息仍然有限，包括年轻年龄对疾病特征、治疗及预后的影响。 方法：在这项回顾性研究中，我们分析了68例年龄≤50岁、至少接受过一次外周血干细胞移植（PBSCT）的新诊断MM患者。此外，我们回顾了2008年至2022年间发表的数据，并将其与我们队列的数据进行比较。 结果：值得注意的是，我们队列的疾病特征与老年患者相似。然而，年轻患者的骨病变发生率更高（84%）。此外，33%的患者为仅轻链型MM，7%具有高危（del17p、t(14;16)、t(4;14)）细胞遗传学特征。分别有57%和78%的患者处于晚期ISS分期和R-ISS II/III期。治疗强度较高，53%的患者接受了≥2次干细胞移植。中位随访时间为75个月，中位无进展生存期为57个月，中位总生存期（OS）尚未达到。10年OS率为72%，仅19%的患者死于该疾病。值得注意的是，通过单变量或双变量分析，甚至在对≤40岁更年轻患者的亚组分析中，均未发现与不良预后相关的特定治疗方案或风险因素。 结论：我们及既往关于年轻（<50岁）和极年轻（<40岁）MM患者的研究结果强调，有必要针对这一显著受影响的群体开展进一步全面研究。

原文链接：

Clinical Characteristics, Cytogenetic Risks, and Prognoses of Young Multiple Myeloma Patients in the Era of Novel Therapies