Analysis of Common Eligibility Criteria of Randomized Controlled Trials in Newly Diagnosed Multiple Myeloma Patients and Extrapolating Outcomes
Analysis of Common Eligibility Criteria of Randomized Controlled Trials in Newly Diagnosed Multiple Myeloma Patients and Extrapolating Outcomes
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DOI：
10.1016/j.clml.2017.06.013
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发表时间：
2017-09-01
影响因子：
2.7
通讯作者：
Rifkin, Robert M.
中科院分区：
文献类型：
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作者：
Shah, Jatin J.;Abonour, Rafat;Rifkin, Robert M.
多发性骨髓瘤（MM）临床试验的严格排除标准限制了反映一般患者人群的患者入组。使用Connect MM登记研究数据代表了新诊断的MM人群，1406例患者中有563例（40.0%）被确定为不符合临床试验条件。这项研究提供了深入了解潜在的修改标准的资格标准，可以导致改善trial design.Background：的性能多发性骨髓瘤（MM）治疗在一般患者人群和特定的资格标准，可能会限制入组随机对照试验（RCT）尚未进行深入评估。本研究旨在确定在RCT中观察到的MM治疗改善是否反映在一般患者人群中，并确定可以修改以增加入组的合格性标准。患者和方法：Connect MM登记研究是一项在美国新诊断MM（NDMM）患者中开展的前瞻性观察性队列研究。使用从16项已发表研究中收集的常见RCT排除标准，根据纳入RCT的资格对登记研究中的患者进行分类。结果：根据通用标准，1406例登记患者中有563例（40.0%）不符合RCT的条件。导致排除的标准包括M蛋白2.5 mg/dL（13.9%）、中性粒细胞绝对计数低（10.0%）和血红蛋白低（9.6%）。不符合RCT条件的患者中，高钙血症（11.0% vs. 5.5%）、肌酐水平升高（38.9% vs. 6.2%）、血红蛋白水平降低（59.5% vs. 39.5%）或国际分期系统III期疾病（40.1% vs. 22.1%; P
The strict exclusion criteria of clinical trials in multiple myeloma (MM) limit the enrollment of patients reflective of a general patient population. Using the Connect MM Registry data representative of an unselected newly diagnosed MM population, 563 of 1406 patients (40.0%) were identified as ineligible for clinical trials. This study provides insight into potential modifications of standard eligibility criteria that can lead to improved trial design.Background: The performance of multiple myeloma (MM) therapies in a general patient population and specific eligibility criteria that might limit enrollment into randomized controlled trials (RCTs) have not been evaluated in depth. This study aimed to determine if improvements seen with MM therapies in RCTs are reflected in the general patient population and to identify eligibility criteria that can be modified to increase enrollment. Patients and Methods: The Connect MM Registry is a prospective observational cohort study of patients with newly diagnosed MM (NDMM) in the United States. Using common RCT exclusion criteria collected from 16 published studies, patients in the registry were categorized according to their eligibility for inclusion in RCTs. Results: On the basis of common criteria, 563 of 1406 of registry patients (40.0%) are ineligible for RCTs. Criteria leading to exclusion included M-protein 2.5 mg/dL (13.9%), low absolute neutrophil count (10.0%), and low hemoglobin (9.6%). Significantly more RCT-ineligible versus RCT-eligible patients had hypercalcemia (11.0% vs. 5.5%), elevated creatinine levels (38.9% vs. 6.2%), low hemoglobin levels (59.5% vs. 39.5%), or International Staging System stage III disease (40.1% vs. 22.1%; P