Gidwani Risha, Franks Jeffrey A, Enogela Ene M, Caston Nicole E, Williams Courtney P, Aswani Monica S, Azuero Andres, Rocque Gabrielle B
Department of Health Management and Policy, University of California, Los Angeles, Los Angeles, CA.
Division of Hematology and Oncology, Department of Medicine, University of Alabama at Birmingham, Birmingham, AL.
JCO Oncol Pract. 2022 Feb;18(2):e235-e249. doi: 10.1200/OP.21.00274. Epub 2021 Sep 24.
Many patient population groups are not proportionally represented in clinical trials, including patients of color, at age extremes, or with comorbidities. It is therefore unclear how treatment outcomes may differ for these patients compared with those who are well-represented in trials.
This retrospective cohort study included women diagnosed with stage I-III breast cancer between 2005 and 2015 in the national CancerLinQ Discovery electronic medical record-based data set. Patients with comorbidities or concurrent cancer were considered in clinical trials. Non-White patients and/or those age < 45 or ≥ 70 years were considered . Patients who were White, age 45-69 years, and without comorbidities were considered . Cox proportional hazards models were used to evaluate 5-year mortality by representation group and patient characteristics, adjusting for cancer stage, subtype, chemotherapy, and diagnosis year.
Of 11,770 included patients, 48% were considered well-represented in trials, 45% under-represented, and 7% unrepresented. Compared with well-represented patients, unrepresented patients had almost three times the hazard of 5-year mortality (adjusted hazard ratio [aHR], 2.71; 95% CI, 2.08 to 3.52). There were no significant differences in the hazard of 5-year mortality for under-represented patients compared with well-represented patients (aHR, 1.19; 95% CI, 0.98 to 1.45). However, among under-represented patients, those age < 45 years had a lower hazard of 5-year mortality (aHR, 0.63; 95% CI, 0.48 to 0.84) and those age ≥ 70 years had a higher hazard of 5-year mortality (aHR, 2.21; 95% CI, 1.76 to 2.77) compared with those age 45-69 years.
More than half of the patients were under-represented or unrepresented in clinical trials, because of age, comorbidity, or race. Some of these groups experienced poorer survival compared with those well-represented in trials. Trialists should ensure that study participants reflect the disease population to support evidence-based decision making for all individuals with cancer.
许多患者群体在临床试验中的代表性不均衡，包括有色人种患者、极端年龄患者或患有合并症的患者。因此，与在试验中具有充分代表性的患者相比，这些患者的治疗结果可能有何不同尚不清楚。
这项回顾性队列研究纳入了2005年至2015年期间在基于国家CancerLinQ发现电子病历的数据集里被诊断为I-III期乳腺癌的女性患者。临床试验中纳入了患有合并症或同时患有癌症的患者。纳入了非白人患者和/或年龄<45岁或≥70岁的患者。纳入了白人、年龄在45-69岁且无合并症的患者。采用Cox比例风险模型，根据代表性分组和患者特征评估5年死亡率，并对癌症分期、亚型、化疗和诊断年份进行调整。
在纳入的11770名患者中，48%被认为在试验中有充分代表性，45%代表性不足，7%未被纳入。与有充分代表性的患者相比，未被纳入的患者5年死亡风险几乎高出两倍（调整后风险比[aHR]，2.71；95%置信区间，2.08至3.52）。与有充分代表性的患者相比，代表性不足的患者5年死亡风险无显著差异（aHR，1.19；95%置信区间，0.98至1.45）。然而，在代表性不足的患者中，年龄<45岁的患者5年死亡风险较低（aHR，0.63；95%置信区间，0.48至0.84），年龄≥70岁的患者5年死亡风险高于年龄在45-69岁的患者（aHR，2.21；95%置信区间，1.76至2.77）。
由于年龄、合并症或种族原因，超过一半的患者在临床试验中的代表性不足或未被纳入。与试验中具有充分代表性的患者相比，其中一些群体的生存率较差。试验者应确保研究参与者反映疾病人群，以支持对所有癌症患者进行基于证据的决策。
