Understanding the patient experience of treatment toxicities and their impact on health-related quality of life (HRQoL) of cancer treatments requires asking patients themselves using patient-reported outcomes (PROs). Over the past twenty years, the National Institutes of Health (NIH) sponsored several tools, namely Patient-Reported Outcome Measurement Information System (PROMIS) measures and the Patient-Reported Outcomes version of the Common Terminology Criteria for Adverse Events (PRO-CTCAE) for precisely this purpose: to ensure valid, reliable tools to collect and detect patient-reported toxicities or adverse events and their impact on HRQoL. These PRO measures have been widely incorporated in clinical trials for adults with cancer. Yet, despite considerable work developing and validating developmentally appropriate versions of these measures for pediatric and adolescent self-report, PRO inclusion in pediatric and adolescent and young adult (AYA) clinical trials has lagged. Here we discuss optimal strategies to integrate validated PRO tools and sound analytic methodologies in clinical trials for children and AYAs with cancer, highlighting lessons learned from recent successes and ongoing experiences developing and opening cross-network trials for children and AYAs through the Children’s Oncology Group (COG) for patients with classic Hodgkin lymphoma (cHL), osteosarcoma (OS), and acute lymphoblastic leukemia (ALL).

中文翻译：

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将患者报告的结果纳入儿科癌症临床试验：设计、实施和传播

了解患者对治疗毒性的体验及其对癌症治疗的健康相关生活质量 （HRQoL） 的影响需要使用患者报告的结果 （PRO） 询问患者自己。在过去的二十年里，美国国立卫生研究院 （NIH） 赞助了多种工具，即患者报告结果测量信息系统 （PROMIS） 测量和不良事件通用术语标准 （PRO-CTCAE） 的患者报告结果版本，正是为了这个目的：确保有效、可靠的工具来收集和检测患者报告的毒性或不良事件及其对 HRQoL 的影响。这些 PRO 措施已广泛纳入成人癌症临床试验中。然而，尽管在开发和验证这些用于儿科和青少年自我报告的措施的适合发育的版本方面做了大量工作，但 PRO 纳入儿科和青少年和年轻人 （AYA） 临床试验仍然滞后。在这里，我们讨论了在患有癌症的儿童和 AYA 的临床试验中整合经过验证的 PRO 工具和合理的分析方法的最佳策略，强调了从最近的成功中吸取的经验教训以及通过儿童肿瘤学组 （COG） 为经典霍奇金淋巴瘤 （cHL）、骨肉瘤 （OS） 患者开发和开设儿童和 AYA 跨网络试验的持续经验， 和急性淋巴细胞白血病 （ALL）。