Voices of children and adolescents with incurable cancer on Phase I or II Trials

患有无法治愈癌症的儿童和青少年在一期或二期试验中的声音

• 批准号: 8078390
• 负责人: Pamela S Hinds
• 金额: $ 26.64万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2010
• 资助国家: 美国
• 起止时间: 2010-09-24 至 2012-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8078390
• 关键词: Voices; children; adolescents; incurable; cancer

DESCRIPTION (provided by applicant): In the United States, cancer is the number one disease-related cause of death in children between the ages of 1 and 19 years; approximately 25% of the 12,400 children newly diagnosed with cancer will die of their disease. A priority of the National Institute of Nursing Research and the National Cancer Institute is to improve the quality of life of pediatric cancer patients at end of life in part by measuring patient- reported outcomes (PROs). Inviting and measuring the pediatric cancer patients' symptom and quality of life reports when their disease is incurable will help clinicians to anticipate and better manage their symptoms secondary to cancer and reduce patient suffering. PROs are included in multiple Phase III therapeutic trials where the objective is cure. No PRO is a component of pediatric Phase I (drug dose finding and toxicity) or Phase II (drug activity and safety) trials, yet it is these two trial types that contribute to new drug indications and labeling. Quite recently, the U.S. Food and Drug Administration (FDA) has released guidance concerning the use of PROs in trials to support drug labeling, including clear language requiring PROs in pediatrics and not parent or clinician proxy reports. To date, no pediatric oncology drug indication has been secured on the basis of a PRO; organ and system toxicities are documented but not the child's voice in terms of symptom and QoL experiences while enrolled on the experimental drug trial. We now have pediatric instrumentation to measure symptoms and aspects of quality of life (QoL) by patient report in 8 to 18 year olds, developed and validated as part of the PROMIS (Patient-Reported Outcomes Measurement Information System) initiative sponsored by the NIH Roadmap for Medical Research. These instruments combined with an interview will allow us to go beyond the single traditionally measured benefit (tumor response) of Phase I or Phase II participation by children and adolescents with incurable cancer to documenting their symptoms and QoL during this phase of care. With these tools to measure pediatric PROs, we will provide knowledge to help clinicians diminish patient suffering and we could position pediatric oncology to meet FDA standards for drug labeling and in this two-site, longitudinal validity study (T1, time of trial enrollment; T2, 3 to 4 weeks later), we will assess the feasibility, acceptability, reliability, validity and responsiveness of the PROMIS pediatric instruments for the first time ever in up to 80, 8 to 18 year olds with incurable or refractory cancer enrolled on Phase I or Phase II trials. PUBLIC HEALTH RELEVANCE: Children and adolescents with incurable cancer can receive experimental drugs that cause them troubling symptoms. Clinicians could better manage these symptoms and reduce patient suffering if the troubling symptoms were measured directly in reports from these young patients. This study will allow us to measure direct symptom and quality of life reports of children and adolescents with incurable cancer who are receiving experimental drugs.

描述（由申请人提供）：在美国，癌症是1至19岁儿童死亡的头号疾病相关原因;新诊断患有癌症的12，400名儿童中约有25%将死于疾病。国家护理研究所和国家癌症研究所的一个优先事项是通过测量患者报告的结果（PRO）来改善儿童癌症患者在生命结束时的生活质量。邀请和测量儿科癌症患者的症状和生活质量报告时，他们的疾病是不可治愈的，将有助于临床医生预测和更好地管理他们的症状继发于癌症，并减少患者的痛苦。PRO包括在多个III期治疗试验中，其目的是治愈。没有PRO是儿科I期（药物剂量发现和毒性）或II期（药物活性和安全性）试验的组成部分，但正是这两种试验类型有助于新药适应症和标签。最近，美国食品药品监督管理局（FDA）发布了关于在试验中使用PRO以支持药物标签的指南，包括明确要求儿科PRO而不是父母或临床医生代理报告的语言。迄今为止，尚未根据PRO确定儿科肿瘤药物适应症;记录了器官和系统毒性，但未记录入组实验性药物试验时儿童在症状和QoL体验方面的声音。我们现在有儿科仪器来测量症状和生活质量（QoL）方面的患者报告在8至18奥尔兹的人，开发和验证的PROMIS（患者报告的结果测量信息系统）倡议的一部分，由NIH医学研究路线图。这些工具与访谈相结合，将使我们能够超越I期或II期参与的儿童和青少年的单一传统测量获益（肿瘤缓解），以记录他们在此护理阶段的症状和生活质量。有了这些测量儿科PRO的工具，我们将提供知识，帮助临床医生减少患者的痛苦，我们可以定位儿科肿瘤，以满足FDA的药物标签标准，并在这两个网站，纵向有效性研究（T1，试验入组时间; T2，3至4周后），我们将评估可行性，可接受性，可靠性，PROMIS儿科仪器的有效性和反应性首次在80岁，8至18奥尔兹的不可治愈或难治性癌症患者中进行了I期或II期试验。 公共卫生相关性：患有不可治愈癌症的儿童和青少年可以接受导致他们出现令人不安症状的实验性药物。 临床医生可以更好地管理这些症状，并减少患者的痛苦，如果这些令人不安的症状是直接测量这些年轻患者的报告。这项研究将使我们能够测量接受实验性药物治疗的儿童和青少年患有不可治愈癌症的直接症状和生活质量报告。