Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!
Recruitment & retention program for the NeuroNEXT SMA Biomarker Study: Super Babies for SMA!
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DOI：
10.1016/j.conctc.2018.07.002
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发表时间：
2018-09-01
影响因子：
1.5
通讯作者：
Kissel, John T.
中科院分区：
文献类型：
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作者：
Bartlett, Amy;Kolb, Stephen J.;Kissel, John T.
背景/目标：研究参与者的招募和保留是成功的临床试验和自然历史研究的挑战和关键组成部分。患有脊髓性肌萎缩症 (SMA) 的婴儿由于其脆弱而复杂的医疗问题、预后不良以及直到 2016 年仍缺乏有效的治疗方法，因此一直是一个特别具有挑战性的研究人群。招募健康婴儿参加临床试验和自然史研究也具有挑战性，有时被认为是不可行的。方法：2011年，我们小组启动了一项为期两年的、针对SMA婴儿和健康婴儿对照的纵向自然史研究，以提供数据来协助分析和解释计划中的SMA婴儿临床试验。招募目标是在两年的招募期内招募27名小于6个月的SMA婴儿和27名年龄匹配的健康婴儿。为此制定了详细的招聘和保留计划。此外，还对参与者家庭进行了一项调查，以了解参与研究的决定因素。结果：研究第一年招募了所有健康婴儿，两年招募期内招募了 26 名 SMA 婴儿。 38 个参与家庭对招募决定因素调查做出了回应。近一半的受访者（18/38，48%）表示，他们是从医生或神经科医生那里第一次听说这项研究的。决定让婴儿入组（22/38，58%）并继续参与研究（28/38，74%）的最常见原因是他们对研究重要性的理解。据报道，研究手册、社交媒体上的视频和宣传会议上的演示等主题招募工具可以对入组决定产生积极影响。结论：一项积极主动、主题性和包容性的招募和保留计划，可以有效地传达临床研究的基本原理，并与患者、宣传团体和当地社区合作，可以有效地招募弱势群体的参与者。提供了将招募和保留计划主动纳入临床试验方案制定的建议。
Background/Aims: Recruitment and retention of research participants are challenging and critical components of successful clinical trials and natural history studies. Infants with spinal muscular atrophy (SMA) have been a particularly challenging population to study due to their fragile and complex medical issues, poor prognosis and, until 2016, a lack of effective therapies. Recruitment of healthy infants into clinical trials and natural history studies is also challenging and sometimes assumed to not be feasible.Methods: In 2011, our group initiated a two-year, longitudinal natural history study of infants with SMA and healthy infant controls to provide data to assist in the analysis and interpretation of planned clinical trials in infants with SMA. The recruitment goal was to enroll 27 infants less than 6 months of age with SMA and 27 age matched healthy infants within the two-year enrollment period. A detailed recruitment and retention plan was developed for this purpose. In addition, a survey was administered to participant families to understand the determinants of participation in the study.Results: All healthy infants were recruited within the study's first year and 26 SMA infants were recruited within the two-year recruitment period. Thirty-eight participant families responded to the recruitment determinants survey. Nearly half of respondents (18/38, 48%) reported that they first heard of the study from their physician or neurologist. The most common reason to decide to enroll their infant (22/38, 58%) and to remain in the study (28/38, 74%) was their understanding of the importance of the study. Thematic recruitment tools such as a study brochure, video on social media, and presentations at advocacy meetings were reported to positively influence the decision to enroll.Conclusions: A proactive, thematic and inclusive recruitment and retention plan that effectively communicates the rationale of a clinical study and partners with patients, advocacy groups and the local communities can effectively recruit participants in vulnerable populations. Recommendations for the proactive integration of recruitment and retention plans into clinical trial protocol development are provided.