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“真实世界”研究中新药时代新诊断的非分泌型多发性骨髓瘤患者的预后因素和临床特征:波兰骨髓瘤研究组的经验

Prognostic factors and clinical characteristics of patients with newly diagnosed non-secretory multiple myeloma in the era of new drugs in "real-world" study: Experiences of the Polish Myeloma Group.

作者信息

Charliński Grzegorz, Szudy-Szczyrek Aneta, Podgajna Martyna, Mielnik Michał, Kopińska Anna, Tyczyńska Agata, Usnarska-Zubkiewicz Lidia, Bołkun Łukasz, Wiater Elżbieta, Krzystański Mateusz, Vesole David H, Jurczyszyn Artur

机构信息

Department of Nephrology, Hypertension and Internal Medicine, Faculty of Medicine, University of Warmia and Mazury, Olsztyn, Poland.

Department of Hematology and Bone Marrow Transplantation, Nicolaus Copernicus Hospital, Toruń, Poland.

出版信息

Adv Clin Exp Med. 2025 Mar;34(3):369-378. doi: 10.17219/acem/189390.

DOI:10.17219/acem/189390
PMID:39206811
Abstract

BACKGROUND

Non-secretory multiple myeloma (NSMM) accounts for approx. 2-3% of multiple myeloma (MM) cases. Due to the rare occurrence and ineligibility of patients with NSMM to participate in clinical trials, we have limited data on treatment efficacy and the clinical course in these patients. Most of the literature consists of case reports and small retrospective studies.

OBJECTIVES

The study aimed to analyze patient characteristics, prognostic factors and treatment outcomes in newly diagnosed (ND) NSMM.

MATERIAL AND METHODS

This is a multicenter, retrospective analysis of 43 patients with NSMM diagnosed between June 2010 and September 2021, conducted in 8 Polish hematology centers.

RESULTS

The median overall survival (OS) was 103 months (95% confidence interval (95% CI): 20-72). The most common cause of death was MM disease progression. The overall response rate (ORR) was 84.6%; complete response (CR), very good partial response (VGPR), partial response (PR), and no response (NR) rates were 20.5%, 46.2%, 17.9%, and 15.4%, respectively. In multivariable analysis, factors contributing to worse OS included International Staging System stage 3 (ISS-3) (p = 0.0277), anemia (Hb <10 g/dL or >2 below upper limit of normal value (ULN), p = 0.0270), renal insufficiency (RI, serum creatinine >2 mg/dL, p = 0.0476), and serum albumin <5.5 mg/L (0.0408).

CONCLUSIONS

Non-secretory multiple myeloma is a rare subtype of MM. This small study demonstrates that outcomes are comparable to secretory MM. However, the inclusion of this subset of patients in clinical trials is essential to assess prognosis, treatment efficacy and clinical outcomes.

摘要

背景

非分泌型多发性骨髓瘤(NSMM)约占多发性骨髓瘤(MM)病例的2%-3%。由于NSMM患者发病率低且不符合参与临床试验的条件,我们对这些患者的治疗效果和临床病程的数据有限。大多数文献由病例报告和小型回顾性研究组成。

目的

本研究旨在分析新诊断(ND)NSMM患者的特征、预后因素和治疗结果。

材料与方法

这是一项在8个波兰血液学中心进行的多中心回顾性分析,纳入了2010年6月至2021年9月期间诊断的43例NSMM患者。

结果

中位总生存期(OS)为103个月(95%置信区间(95%CI):20-72)。最常见的死亡原因是MM疾病进展。总缓解率(ORR)为84.6%;完全缓解(CR)、非常好的部分缓解(VGPR)、部分缓解(PR)和无缓解(NR)率分别为20.5%、46.2%、17.9%和15.4%。在多变量分析中,导致OS较差的因素包括国际分期系统3期(ISS-3)(p = 0.0277)、贫血(血红蛋白<10 g/dL或高于正常上限(ULN)2倍以上,p = 0.0270)、肾功能不全(RI,血清肌酐>2 mg/dL,p = 0.0476)和血清白蛋白<5.5 mg/L(0.0408)。

结论

非分泌型多发性骨髓瘤是MM的一种罕见亚型。这项小型研究表明,其结果与分泌型MM相当。然而,将这部分患者纳入临床试验对于评估预后、治疗效果和临床结果至关重要。

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