多发性骨髓瘤临床试验将风险最高的患者排除在外:对试验排除标准的系统回顾。

IF 2.2 4区 医学 Q3 HEMATOLOGY
Leukemia & Lymphoma Pub Date : 2024-12-01 Epub Date: 2024-09-24 DOI:10.1080/10428194.2024.2395440
Sara Zhukovsky, Joshua White, Rajshekhar Chakraborty, Luciano J Costa, Oliver Van Oekelen, Douglas W Sborov, Edward R Scheffer Cliff, Ghulam Rehman Mohyuddin
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引用次数: 0

摘要

某些亚型多发性骨髓瘤患者的治疗效果仍然不佳,需要新的治疗方法。随机对照试验(RCT)的严格资格标准限制了这些患者参与临床试验的机会,也限制了试验结果的外部有效性。我们系统回顾了2006年至2023年新诊断骨髓瘤的RCT,以确定12项主要排除标准的流行程度和随时间变化的趋势。共纳入了 80 项 RCT。排除标准包括:43项试验(51%)的年龄;20项试验(24%)的预期寿命;74项试验(87%)的表现状态;47项试验(55%)的非分泌性和/或少分泌性疾病;64项试验(79%)的肝功能;63项试验(74%)的肾功能;50项试验(59%)的血液学阈值;68项试验(80%)的既往恶性肿瘤;50项试验(59%)的神经病变。在 53 项有详细排除标准的试验中,21 项(40%)排除了浆细胞白血病,5 项(9%)排除了髓外疾病,13 项(25%)排除了中枢神经系统疾病。在单变量回归分析中,采用上述各项排除标准的研究比例并没有随着时间的推移而显著提高,而与神经病变相关的排除标准则有所恶化。大多数骨髓瘤 RCT 的限制性资格标准使治疗具有挑战性的骨髓瘤亚型的数据有限的循环得以延续。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Multiple myeloma clinical trials exclude patients with the highest-risk disease: a systematic review of trial exclusion criteria.

Patients with certain subsets of multiple myeloma continue to have poor outcomes and are in need of novel treatment approaches. Strict eligibility criteria for randomized controlled trials (RCTs) limit access to clinical trials and limit the external validity of trial results for these patients. We systematically reviewed RCTs in newly diagnosed myeloma from 2006 to 2023 to ascertain the prevalence of 12 key exclusion criteria and trends over time. 80 RCTs were included. Exclusion criteria included: age in 43 (51%) trials; projected life expectancy in 20 (24%); performance status in 74 (87%); non-secretory and/or oligosecretory disease in 47 (55%), hepatic function in 64 (79%), renal function in 63 (74%), hematological thresholds in 50 (59%), prior malignancy in 68 (80%), and neuropathy in 50 (59%). For 53 trials which had detailed exclusion criteria available, plasma cell leukemia was excluded in 21 (40%), extramedullary disease in 5 (9%) and CNS disease in 13 (25%). The percentage of studies invoking each of these exclusion criteria did not significantly improve over time on univariate regression analysis, and exclusion criteria relating to neuropathy have worsened. The restrictive eligibility criteria of most myeloma RCTs perpetuate a cycle where limited data exists to treat challenging myeloma subtypes.

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来源期刊
Leukemia & Lymphoma
Leukemia & Lymphoma 医学-血液学
CiteScore
4.10
自引率
3.80%
发文量
384
审稿时长
1.8 months
期刊介绍: Leukemia & Lymphoma in its fourth decade continues to provide an international forum for publication of high quality clinical, translational, and basic science research, and original observations relating to all aspects of hematological malignancies. The scope ranges from clinical and clinico-pathological investigations to fundamental research in disease biology, mechanisms of action of novel agents, development of combination chemotherapy, pharmacology and pharmacogenomics as well as ethics and epidemiology. Submissions of unique clinical observations or confirmatory studies are considered and published as Letters to the Editor
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