Modernizing Clinical Trial Eligibility Criteria: Recommendations of the ASCO-Friends of Cancer Research Performance Status Work Group.
Modernizing Clinical Trial Eligibility Criteria: Recommendations of the ASCO-Friends of Cancer Research Performance Status Work Group.
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DOI：
10.1158/1078-0432.ccr-20-3868
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发表时间：
2021-05-01
期刊：
影响因子：
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通讯作者：
Garrett-Mayer E
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文献类型：
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作者：
Magnuson A;Bruinooge SS;Singh H;Wilner KD;Jalal S;Lichtman SM;Kluetz PG;Lyman GH;Klepin HD;Fleury ME;Hirsch B;Melemed A;Arnaldez FI;Basu Roy U;Schenkel C;Sherwood S;Garrett-Mayer E
绩效状态(PS)是最常见的资格标准之一。许多试验仅限于高功能PS患者，导致试验参与者和疾病患者群体之间存在重要差异。此外，现有的PS测量具有主观性，容易受到调查者偏见的影响。美国临床肿瘤学协会和癌症研究之友的一个多学科工作组评估了PS资格标准如何能够更具包容性。工作组的建议基于文献搜索、试验回顾、模拟研究和多方利益相关者共识。工作组优先考虑包容性和获得研究治疗的机会，同时平衡患者安全和研究完整性。扩大PS资格标准可能会增加特定临床试验的潜在合格患者数量，从而缩短应计时间。它还可能导致更大的参与者多样性，潜在地减少试验参与者和患者的差异，并使临床医生能够更容易地将试验结果转化为功能低下的PS患者。通过一项模拟试验探索了对结果的潜在影响，该模拟试验表明，当东部合作肿瘤学小组(ECOG)PS2参与者的数量相对较少时，即使PS2患者没有获得治疗益处，对估计的风险比和功率的影响也不大。在许多情况下，将PS资格标准扩大到更具包容性可能是合理的，并可能导致更快的累计率和更具代表性的试验人群。
Performance status (PS) is one of the most common eligibility criteria. Many trials are limited to patients with high-functioning PS, resulting in important differences between trial participants and patient populations with the disease. Additionally, existing PS measures are subjective and susceptible to investigator bias. A multidisciplinary working group of the American Society of Clinical Oncology and Friends of Cancer Research evaluated how PS eligibility criteria could be more inclusive. The working group recommendations are based on a literature search, review of trials, simulation study, and multistakeholder consensus. The working group prioritized inclusiveness and access to investigational therapies, while balancing patient safety and study integrity. Broadening PS eligibility criteria may increase the number of potentially eligible patients for a given clinical trial, thus shortening accrual time. It may also result in greater participant diversity, potentially reduce trial participant and patient disparities, and enable clinicians to more readily translate trial results to patients with low-functioning PS. Potential impact on outcomes was explored through a simulation trial demonstrating that when the number of Eastern Cooperative Oncology Group (ECOG) PS2 participants was relatively small, the effect on the estimated hazard ratio and power were modest, even when PS2 patients did not derive a treatment benefit. Expanding PS eligibility criteria to be more inclusive may be justified in many cases and could result in faster accrual rates and more representative trial populations.