Voices of children and adolescents on phase 1 or phase 2 cancer trials: A new trial endpoint?
Voices of children and adolescents on phase 1 or phase 2 cancer trials: A new trial endpoint?
复制标题
儿童和青少年在第一阶段或第二阶段癌症试验中的声音：新的试验终点？
DOI：
10.1002/cncr.30782
复制
发表时间：
2017-10-01
期刊：
影响因子：
6.2
通讯作者：
Jacobs S
中科院分区：
文献类型：
--
作者：
Hinds PS;Wang J;Stern ED;Macpherson CF;Wharton CM;Okorosobo R;Cheng YI;Gross HE;Meany HJ;Jacobs S
参与I期或II期临床试验的儿童患者存在发生与参与试验相关的毒性（不良事件，AE）的风险。多起AE是主观的;因此，除非征集患者主观报告，否则无法了解试验治疗的真实的影响。我们评估了在化疗的第一个疗程期间，从4个癌症中心参加I/II期临床试验的8- 18岁参与者中征集症状、功能和生活质量（QoL）报告的可行性和可接受性。我们还在两个时间点（试验入组，T1和3 - 4周后，T2）评估了6个自我报告PROMIS儿科指标和4个开放式访谈问题的可靠性和有效性。75.9%（n=20）的入组率超过了我们的可行性标准，T1和T2时按人、测量和项目划分的测量缺失率低于我们的可接受标准。新的QoL主题仅限于治疗对家庭的影响以及离家、家人和朋友接受治疗。T1时除一项测量外，所有测量均符合可靠性标准，T2时所有测量均符合可靠性标准。有效性支持是有限的，虽然从理论上讲，随着AE的增加，移动性降低，疲劳增加。征集和记录参加I/II期临床试验的8- 18岁儿童的症状、功能和生活质量报告是可行的，也是参与者可以接受的，特别是在试验中。可以得出可靠和有效的结果，使患者自我报告的结局成为可能的新试验终点。
Pediatric participants on Phase I or Phase II clinical trials for incurable cancer are at risk of experiencing toxicities (adverse events, AEs) related to trial participation. Multiple AEs are subjective; thus, the real impact of trial treatment cannot be known unless patient subjective reports are solicited. We assessed the feasibility and acceptability of soliciting symptom, function and quality of life (QoL) reports from 8- to 18-year-old participants enrolled on Phase I/II clinical trials at four cancer centers during the first course of chemotherapy. We also assessed the reliability and validity of six self-report PROMIS pediatric measures and four open-ended interview questions at two time points (trial enrollment, T1 and 3 to 4 weeks later, T2). The enrollment rate of 75.9% (n=20) exceeded our feasibility criterion, and missingness of measures by person, measure and items at T1 and T2 were lower than our acceptability criteria. New QoL themes were limited to the impact of treatment on families and being away from home, family and friends for treatment. All but one measure at T1 met the reliability criterion and all did at T2. Validity support was limited though as theorized, mobility decreased and fatigue increased as AEs increased. Soliciting and documenting symptom, function and QoL reports from 8- to 18-year-olds enrolled on a Phase I/II clinical trial is feasible and acceptable to participants, particularly when embedded in trials. Reliable and valid findings can result, making patient self-reported outcomes a possible new trial endpoint.
登录
查看更多内容
影响因子：
3.2
作者：
Hinds, Pamela S.;Nuss, Suzanne L.;DeWalt, Darren A.
通讯作者：
DeWalt, Darren A.
影响因子：
158.5
作者：
Krueger, Darcy A.;Care, Marguerite M.;Franz, David Neal
通讯作者：
Franz, David Neal
影响因子：
3.5
作者：
Hinds, PS;Gattuso, JS;Pui, CH
通讯作者：
Pui, CH
影响因子：
3.6
作者：
Hinds, Pamela S.;Brandon, Jennifer;Kane, Javier R.
通讯作者：
Kane, Javier R.
影响因子：
2.6
作者：
Menard JC;Hinds PS;Jacobs SS;Cranston K;Wang J;DeWalt DA;Gross HE
通讯作者：
Gross HE