Department of Health Services Research, University of Liverpool, Liverpool, UK
Department of Health Services Research, University of Liverpool, Liverpool, UK.
BMJ Open. 2020 Apr 16;10(4):e035516. doi: 10.1136/bmjopen-2019-035516.
To determine the feasibility and optimal design of a randomised controlled trial (RCT) of Seizure First Aid Training For Epilepsy (SAFE).
Pilot RCT with embedded microcosting.
Three English hospital emergency departments (EDs).
Patients aged ≥16 with established epilepsy reporting ≥2 ED visits in the prior 12 months and their significant others (SOs).
Patients (and their SOs) were randomly allocated (1:1) to SAFE plus treatment-as-usual (TAU) or TAU alone. SAFE is a 4-hour group course.
Two criteria evaluated a definitive RCT's feasibility: (1) ≥20% of eligible patients needed to be consented into the pilot trial; (2) routine data on use of ED over the 12 months postrandomisation needed securing for ≥75%. Other measures included eligibility, ease of obtaining routine data, availability of self-report ED data and comparability, SAFE's effect and intervention cost.
Of ED attendees with a suspected seizure, 424 (10.6%) patients were eligible; 53 (12.5%) patients and 38 SOs consented. Fifty-one patients (and 37 SOs) were randomised. Routine data on ED use at 12 months were secured for 94.1% patients. Self-report ED data were available for 66.7% patients. Patients reported more visits compared with routine data. Most (76.9%) patients randomised to SAFE received it and no related serious adverse events occurred. ED use at 12 months was lower in the SAFE+TAU arm compared with TAU alone, but not significantly (rate ratio=0.62, 95% CI 0.33 to 1.17). A definitive trial would need ~674 patient participants and ~39 recruitment sites. Obtaining routine data was challenging, taking ~8.5 months.
In satisfying only one predetermined 'stop/go' criterion, a definitive RCT is not feasible. The low consent rate in the pilot trial raises concerns about a definitive trial's finding's external validity and means it would be expensive to conduct. Research is required into how to optimise recruitment from the target population.
ISRCTN13871327.
确定癫痫急救培训（SAFE）随机对照试验（RCT）的可行性和最佳设计。
具有嵌入式微观成本的试点 RCT。
三家英国医院急诊科（ED）。
年龄≥16 岁，有明确癫痫病史，在过去 12 个月内至少有 2 次 ED 就诊经历，且有其重要他人（SO）的患者。
患者（及其 SO）被随机分配（1:1）接受 SAFE 加常规治疗（TAU）或仅 TAU。SAFE 是一个 4 小时的小组课程。
两个标准评估了确定性 RCT 的可行性：（1）需要有≥20%的合格患者同意参与试验；（2）需要确保≥75%的患者在随机分组后 12 个月内获得常规 ED 数据。其他措施包括合格性、常规数据的获取难易程度、ED 自我报告数据的可用性和可比性、SAFE 的效果和干预成本。
在疑似癫痫发作的 ED 就诊者中，有 424 名（10.6%）患者符合条件；53 名（12.5%）患者及其 38 名 SO 同意。51 名患者（和 37 名 SO）被随机分配。94.1%的患者在 12 个月时获得了 ED 使用的常规数据。66.7%的患者可提供 ED 自我报告数据。与常规数据相比，患者报告的就诊次数更多。大多数（76.9%）随机分配到 SAFE+TAU 组的患者接受了 SAFE 培训，且未发生任何相关严重不良事件。SAFE+TAU 组患者在 12 个月时的 ED 使用量低于 TAU 组，但无显著差异（率比=0.62，95%CI 0.33 至 1.17）。一项确定性试验需要约 674 名患者参与者和 39 个招募地点。常规数据的获取极具挑战性，耗时约 8.5 个月。
在仅满足一个预定的“停止/继续”标准的情况下，一项确定性 RCT 是不可行的。试点试验中较低的同意率引起了人们对确定性试验结果外部有效性的担忧，这意味着开展这项试验代价高昂。需要研究如何优化目标人群的招募。
ISRCTN8551437。
