多发性骨髓瘤长寿患者的特征和治疗模式:连接Ⓡ MM 登记处超过 13 年的随访。

IF 2.7 4区 医学 Q2 HEMATOLOGY
Howard R. Terebelo , James Omel , Lynne I. Wagner , James W. Hardin , Robert M. Rifkin , Sikander Ailawadhi , Brian G.M. Durie , Mohit Narang , Kathleen Toomey , Cristina J. Gasparetto , Prashant Joshi , Edward Yu , E. Dawn Flick , Ying-Ming Jou , Hans C. Lee , Rafat Abonour , Sundar Jagannath
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引用次数: 0

摘要

背景:在过去的 15 年中,患者管理和治疗方法的改进延长了多发性骨髓瘤(MM)患者的生存期。Connect MM登记处是美国一项长期开展的多中心前瞻性观察性队列研究,研究对象是新诊断的多发性骨髓瘤(NDMM)患者。我们评估了登记在册的长期幸存者(LTS)的人口统计学、临床特征和治疗模式:2009年至2016年期间,全美250个社区、学术和政府机构登记了NDMM成人患者(n = 3,011)。研究了LTS的基线特征、治疗模式、生活质量(QoL)和总生存率(OS),LTS是指入选后随访≥8年的患者:截至 2023 年 2 月 7 日,518 名患者为 LTS,2493 名患者为非 LTS。与非 LTS 相比,LTS 一般更年轻,入组时的表现状态更好。大多数(65%)LTS患者接受了干细胞移植,很少(2%)患者在开始一线治疗后6个月内出现疾病进展。数据截止时,63%的LTS在最近一次就诊时仍在接受治疗。LTS 的 QoL 评分和 QoL 问卷完成率一直高于非 LTS。登记的所有患者的估计8年OS率为40%,与监测、流行病学和最终结果(SEER)数据库中观察到的39%的8年生存率相当:这项分析利用真实世界的数据对长期存活的 MM 患者进行了深入分析,因此其普遍性超越了临床试验长期随访所获得的数据,强调了通过登记处进行纵向随访的必要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Characteristics and Treatment Patterns of Long-surviving Patients With Multiple Myeloma: Over 13 Years of Follow-up in the ConnectⓇ MM Registry

Background

Over the last 15 years, improvements in patient management and treatments have been associated with longer survival in patients with multiple myeloma (MM). The Connect MM Registry is a long-running, US, multicenter, prospective observational cohort study of patients with newly diagnosed MM (NDMM). We assessed the demographics, clinical characteristics, and treatment patterns of long-term survivors (LTS) enrolled in this registry.

Methods

Adults with NDMM (n = 3,011) were enrolled from 250 community, academic, and government sites across the US from 2009-2016. Baseline characteristics, treatment patterns, quality of life (QoL), and overall survival (OS) were examined among LTS, defined as patients with follow-up of ≥ 8 years after enrollment.

Results

As of February 7, 2023, 518 patients were LTS and 2,493 were non-LTS. LTS were generally younger and had better performance status at enrollment compared with non-LTS. Most (65%) LTS received stem cell transplants and few (2%) experienced disease progression within 6 months of starting first line of therapy. At data cutoff, 63% of LTS were still on treatment at their most recent visit. QoL scores and QoL questionnaire completion rates were consistently higher among LTS than non-LTS. The estimated 8-year OS rate of all patients enrolled in the registry was 40%, comparable to an observed 8-year survival of 39% from the Surveillance, Epidemiology, and End Results (SEER) database.

Conclusion

This analysis provides insights on long-surviving patients with MM using real-world data and therefore presents generalizability beyond data obtained in long-term follow-up of clinical trials, underscoring the need for longitudinal follow-up through registries.
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来源期刊
CiteScore
2.70
自引率
3.70%
发文量
1606
审稿时长
26 days
期刊介绍: Clinical Lymphoma, Myeloma & Leukemia is a peer-reviewed monthly journal that publishes original articles describing various aspects of clinical and translational research of lymphoma, myeloma and leukemia. Clinical Lymphoma, Myeloma & Leukemia is devoted to articles on detection, diagnosis, prevention, and treatment of lymphoma, myeloma, leukemia and related disorders including macroglobulinemia, amyloidosis, and plasma-cell dyscrasias. The main emphasis is on recent scientific developments in all areas related to lymphoma, myeloma and leukemia. Specific areas of interest include clinical research and mechanistic approaches; drug sensitivity and resistance; gene and antisense therapy; pathology, markers, and prognostic indicators; chemoprevention strategies; multimodality therapy; and integration of various approaches.
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