PDF(Pubmed)
Abstract：
BACKGROUND: The main aim of this paper is to present the feasibility of rigorously designed multiple N-of-1 design in prosthetics research. While research of adequate power and high quality is often lacking in rehabilitation, N-of-1 trials can offer a feasible alternative to randomized controlled group trials, both increasing design power at group level and allowing a rigorous, statistically confirmed evaluation of effectiveness at a single patient level. The paper presents a multiple N-of-1 trial protocol, which aim is to evaluate the effectiveness of Unity, a prosthetic add-on suspension system for amputees, on patient-reported comfort during daily activities (main outcome measure), prosthesis wearing time, perception of limb-prosthesis fitting and stump volume and functional walking parameters.
METHODS: Multicenter, randomized, prospective, double-blind multiple N-of-1 trial using an introduction/withdrawal design alternating Unity connected/disconnected phases of randomized length on twenty patients with unilateral transtibial amputation. The primary outcome measure is the Prosthetic Socket Comfort Score (SCS), a validated measure of comfort, administered daily by an phone app designed for the study. Secondary outcomes measures will be collected during the 50 days period of the N-of-1 trial: (1) by the same app, daily for patient-reported limb-prosthesis fitting, stump volume variation, and daily wearing time of the prosthesis; (2) by a pedometer for the number of steps per day; (3) by blind assessors in the rehabilitation center during adjustment visits for functional walking parameter (L-Test, 6-minute walk test), and by the patient for the QUEST, and ABC-S. Effectiveness of the Unity system regarding SCS and daily secondary outcome measures will be tested by randomization test. The secondary outcome measures assessed during visits in the rehabilitation center will be analyzed by Non Overlap of All pairs. An estimate of the effect on the amputee population will be generated by aggregating each individual clinical trial (N-of-1 trial) by Hierarchical Bayesian methods.
CONCLUSIONS: This study protocol was designed to answer the question \"which device is best for THIS patient\" and to conclude at a group level on the effectiveness of a new devic, using a Multiple N-of-1 trial, which is promising but underused in prosthetics research so far.
BACKGROUND: N° ID-RCB 2020-A01309-30 Clintrial.gov : NCT04804150 - Retrospectively registered March 20th 2021.
METHODS: Multicenter, randomized, prospective, double-blind multiple N-of-1 trial using an introduction/withdrawal design alternating Unity connected/disconnected phases of randomized length on twenty patients with unilateral transtibial amputation. The primary outcome measure is the Prosthetic Socket Comfort Score (SCS), a validated measure of comfort, administered daily by an phone app designed for the study. Secondary outcomes measures will be collected during the 50 days period of the N-of-1 trial: (1) by the same app, daily for patient-reported limb-prosthesis fitting, stump volume variation, and daily wearing time of the prosthesis; (2) by a pedometer for the number of steps per day; (3) by blind assessors in the rehabilitation center during adjustment visits for functional walking parameter (L-Test, 6-minute walk test), and by the patient for the QUEST, and ABC-S. Effectiveness of the Unity system regarding SCS and daily secondary outcome measures will be tested by randomization test. The secondary outcome measures assessed during visits in the rehabilitation center will be analyzed by Non Overlap of All pairs. An estimate of the effect on the amputee population will be generated by aggregating each individual clinical trial (N-of-1 trial) by Hierarchical Bayesian methods.
CONCLUSIONS: This study protocol was designed to answer the question \"which device is best for THIS patient\" and to conclude at a group level on the effectiveness of a new devic, using a Multiple N-of-1 trial, which is promising but underused in prosthetics research so far.
BACKGROUND: N° ID-RCB 2020-A01309-30 Clintrial.gov : NCT04804150 - Retrospectively registered March 20th 2021.
摘要：
背景：本文的主要目的是提出在假肢研究中严格设计的多个N-of-1设计的可行性。虽然在康复中往往缺乏足够功率和高质量的研究，N-of-1试验可以为随机对照试验提供可行的替代方案，既增加了集团层面的设计能力，又允许严格的，统计上证实了单个患者水平的有效性评估。本文提出了一种多N-of-1试验协议，其目的是评估团结的有效性，用于截肢者的假肢附加悬挂系统，患者在日常活动中报告的舒适度(主要结果衡量标准)，假肢佩戴时间,假肢拟合和残端体积和功能性步行参数的感知。
方法：多中心，随机化，prospective,在20例单侧胫骨截肢患者中，使用引入/退出设计交替Unity连接/断开阶段的随机长度的双盲多项N-of-1试验。主要结果测量是假肢睡眠舒适评分(SCS)，一种经过验证的舒适度，每天通过为研究设计的手机应用程序进行管理。次要结局指标将在N-of-1试验的50天期间收集：（1）由同一应用程序，每日用于患者报告的假肢装配，树桩体积变化,和假体的每日佩戴时间；（2）通过计步器测量每天的步数；（3）在康复中心的盲人评估人员对功能性步行参数进行调整访问（L-Test，6分钟步行测试),由病人寻求，ABC-SUnity系统有关SCS和每日次要结果指标的有效性将通过随机测试进行测试。在康复中心访视期间评估的次要结果指标将通过所有配对的非重叠进行分析。对截肢者群体的影响的估计将通过分层贝叶斯方法汇总每个单独的临床试验(N-of-1试验)来产生。
结论：这项研究方案旨在回答“哪种设备最适合该患者”的问题，并在小组水平上得出结论，使用多次N-of-1试验，到目前为止，这是有希望的，但在假肢研究中应用不足。
背景：N°ID-RCB2020-A01309-30Clintrial.gov：NCT04804150-回顾性注册于2021年3月20日。
方法：多中心，随机化，prospective,在20例单侧胫骨截肢患者中，使用引入/退出设计交替Unity连接/断开阶段的随机长度的双盲多项N-of-1试验。主要结果测量是假肢睡眠舒适评分(SCS)，一种经过验证的舒适度，每天通过为研究设计的手机应用程序进行管理。次要结局指标将在N-of-1试验的50天期间收集：（1）由同一应用程序，每日用于患者报告的假肢装配，树桩体积变化,和假体的每日佩戴时间；（2）通过计步器测量每天的步数；（3）在康复中心的盲人评估人员对功能性步行参数进行调整访问（L-Test，6分钟步行测试),由病人寻求，ABC-SUnity系统有关SCS和每日次要结果指标的有效性将通过随机测试进行测试。在康复中心访视期间评估的次要结果指标将通过所有配对的非重叠进行分析。对截肢者群体的影响的估计将通过分层贝叶斯方法汇总每个单独的临床试验(N-of-1试验)来产生。
结论：这项研究方案旨在回答“哪种设备最适合该患者”的问题，并在小组水平上得出结论，使用多次N-of-1试验，到目前为止，这是有希望的，但在假肢研究中应用不足。
背景：N°ID-RCB2020-A01309-30Clintrial.gov：NCT04804150-回顾性注册于2021年3月20日。
