Department of Hematology and Oncology, NewYork-Presbyterian Hospital/Weill Cornell Medical College, New York, NY.
Department of Lymphoma/Myeloma, The University of Texas MD Anderson Cancer Center, Houston, TX.
Blood Adv. 2022 Aug 9;6(15):4413-4423. doi: 10.1182/bloodadvances.2022007687.
Cancer clinical trial eligibility criteria may create patient populations studied in trials that do not reflect the patient populations treated in the real-world setting. Follicular lymphoma (FL) is an indolent lymphoma with heterogeneous presentations across a broad range of individuals, resulting in many acceptable management strategies. We evaluated how first-line clinical trial eligibility criteria impacted the demographic makeup and outcomes of patients with FL for whom systemic therapy might be considered. We compared the characteristics of 196 patients with FL from a single institution to eligibility criteria from 10 first-line FL trials on clinicaltrials.gov. Next, we tabulated eligibility criteria from 24 first-line FL protocols and evaluated their impact on 1198 patients with FL with stages II to IV disease from the prospective Molecular Epidemiology Resource (MER) and Lymphoma Epidemiology of Outcomes (LEO) cohort studies. We found that 39.8% and 52.7% of patients with FL might be excluded from clinical trials based on eligibility criteria derived from clinicaltrials.gov and protocol documents, respectively. Patients excluded because of renal function, prior malignancy, and self-reported serious health conditions tended to be older. Expanding stage requirement from III-IV to II-IV, and platelet requirement from ≥150 000 to ≥75 000 increased population size by 21% and 8%, respectively, in MER and by 16% and 13%, respectively, in LEO, without impacting patient demographics or outcomes. These data suggest that management of older individuals with FL may not be fully informed by recent clinical trials. Moreover, liberalizing stage and platelet criteria might expand the eligible population and allow for quicker trial accrual without impacting outcomes.
癌症临床试验的入选标准可能会导致研究人群与实际治疗环境中的患者人群存在差异。滤泡性淋巴瘤（FL）是一种惰性淋巴瘤，在广泛的个体中表现出异质性，导致有许多可接受的治疗策略。我们评估了一线临床试验入选标准如何影响可能考虑接受系统治疗的 FL 患者的人口统计学构成和结局。我们将来自单一机构的 196 例 FL 患者的特征与 clinicaltrials.gov 上 10 项一线 FL 试验的入选标准进行了比较。接下来，我们列出了 24 项一线 FL 方案的入选标准，并评估了它们对来自前瞻性分子流行病学资源（MER）和淋巴瘤结局的流行病学（LEO）队列研究的 1198 例 II-IV 期 FL 患者的影响。我们发现，根据来自 clinicaltrials.gov 和方案文件的入选标准，39.8%和 52.7%的 FL 患者可能会被排除在临床试验之外。由于肾功能、先前的恶性肿瘤和自我报告的严重健康状况而被排除的患者往往年龄较大。将分期要求从 III-IV 期扩展到 II-IV 期，以及将血小板要求从≥150000 增加到≥75000，分别使 MER 中的人群规模增加了 21%和 8%，LEO 中的人群规模增加了 16%和 13%，而不会影响患者的人口统计学特征或结局。这些数据表明，最近的临床试验可能无法充分指导老年 FL 患者的治疗。此外，放宽分期和血小板标准可能会扩大合格人群，并允许更快地招募试验患者，而不会影响结局。
