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多发性骨髓瘤中的治疗特异性预测模型:当前证据的批判性综述与未来方向

英文原题:Treatment-Specific Prediction Models in Multiple Myeloma: A Critical Review of Current Evidence and Future Directions.

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Treatment-Specific Prediction Models in Multiple Myeloma: A Critical Review of Current Evidence and Future Directions.

PubMed 2026/03/30(内容时间) Eur J Haematol Q2 · IF 2.6(JCR 2025)

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研究概要

方法学和转化方面仍存在差距,包括透明度有限、外部验证稀缺,以及缺乏患者报告或纵向预测因子。尚无任何模型被实现为在线计算器或整合进电子决策支持系统,限制了真实世界中的应用。弥补这些差距对于开发具有临床意义的预测工具、以支持 MM 的个体化治疗至关重要。

研究思路结论见上方概要

多发性骨髓瘤(MM)具有显著的临床异质性,导致治疗反应和毒性存在很大差异。尽管已有众多预后工具,但基于特定治疗方案估计结局的模型相对较少。治疗方案特异性预测模型是迈向个体化治疗选择的重要一步。本综述概述了MM治疗方案特异性临床预测模型的现状。

通过对 PubMed 和 Embase/Scopus 进行结构化检索,筛选出在静态治疗框架内开发的多变量临床预测模型,这些模型用于评估 MM 中治疗特异性疗效或毒性相关结局。提取的信息包括治疗方案、预测因子、建模方法、验证策略以及临床实用性报告。

共识别出13个模型,评估的结局包括疗效(n = 10)或毒性相关(n = 3)结局,涵盖的治疗方案包括以硼替佐米为基础的诱导治疗、含daratumumab的联合方案、以ixazomib为基础的三联方案以及CAR-T 治疗。预测变量主要为常规临床和实验室变量,细胞遗传学或患者报告结局的整合有限。大多数模型采用传统回归方法;校准的报告不一致,7项研究进行了外部验证。仅2个模型纳入了决策曲线分析。

展开英文摘要原文

A structured search of PubMed and Embase/Scopus identified multivariable clinical prediction models developed within a static treatment framework, evaluating treatment-specific therapeutic or toxicity-related outcomes in MM. Information was extracted on treatment regimens, predictors, modeling methods, validation strategies, and reporting of clinical utility.

Thirteen models were identified, evaluating therapeutic (n = 10) or toxicity-related (n = 3) outcomes across regimens including bortezomib-based induction, daratumumab-containing combinations, ixazomib-based triplets, and CAR-T therapy. Predictors were mainly routine clinical and laboratory variables, with limited integration of cytogenetics or patient-reported outcomes. Most models used traditional regression methods; calibration was inconsistently reported, and external validation was performed in seven studies. Decision curve analysis was included in only two models.

Methodological and translational gaps remain, including limited transparency, scarce external validation, and lack of patient-reported or longitudinal predictors. None of the models have been implemented as online calculators or integrated into electronic decision-support systems, limiting real-world uptake. Addressing these gaps is essential for developing clinically meaningful prediction tools to support personalized treatment in MM.

论文信息

作者
Jarrah MM、Al-Shamsi HO、Abuhelwa Z、Bustanji Y、Semreen MH、McKinnon RA、Sorich MJ、Hopkins AM
单位
College of Pharmacy, University of Sharjah, Sharjah, UAE.
文献类型
综述
期刊
European journal of haematology2026 Jul
原文标识
PubMed 41909977 · DOI 10.1111/ejh.70177