Assistant professor in the Department of Pediatrics at the Icahn School of Medicine at Mount Sinai.
Doctoral student in the Department of Biostatistics at the University of Washington School of Public Health.
Ethics Hum Res. 2023 Jan;45(1):29-38. doi: 10.1002/eahr.500154.
Inconsistent enrollment among hospitals for neonatal clinical trials may lead to study populations that are not representative of the patient population in the neonatal intensive care unit. The High-Dose Erythropoietin for Asphyxia and Encephalopathy (HEAL) trial was a multisite randomized clinical trial investigating erythropoietin as a neuroprotective treatment for term infants (those born between 37 and 42 complete weeks) with hypoxic ischemic encephalopathy. Substantial variability was noted in enrollment rate by hospital. We developed survey questions across five conceptual domains to understand systems-level issues that might contribute to variation in enrollment rate by hospital. Our study found that hospitals varied in their responses across these five domains. We propose three potential reasons that we found a lack of identifiable hospital-level factors that correlated with enrollment rates: sample-size limitations, methodological concerns, and confounding factors. Future studies with a larger sample size should be considered to evaluate contributors to hospital-level variability. This will lead to more robust recruitment strategies, improved enrollment, and decreases in the waste of research resources.
医院在新生儿临床试验中的入组情况不一致,可能导致研究人群不能代表新生儿重症监护病房的患者人群。高剂量促红细胞生成素治疗窒息和脑病(HEAL)试验是一项多中心随机临床试验,旨在研究促红细胞生成素作为一种神经保护治疗对患有缺氧缺血性脑病的足月婴儿(出生在 37 周到 42 周之间)的疗效。我们注意到医院的入组率存在显著差异。我们围绕五个概念领域制定了调查问题,以了解可能导致医院间入组率差异的系统层面问题。我们的研究发现,医院在这五个领域的反应存在差异。我们提出了三个潜在的原因,即我们没有发现与入组率相关的可识别的医院层面因素:样本量限制、方法学问题和混杂因素。未来应考虑进行更大样本量的研究,以评估医院层面变异性的贡献因素。这将导致更有效的招募策略、提高入组率,并减少研究资源的浪费。