Faculty of Health and Human Sciences, School of Health Professions, University of Plymouth, Peninsula Allied Health Centre, Plymouth, UK.
Faculty of Health and Human Sciences, School of Health Professions, University of Plymouth, Peninsula Allied Health Centre, Plymouth, UK; Norwich MS Centre, Norwich, UK.
Lancet Neurol. 2019 Aug;18(8):736-747. doi: 10.1016/S1474-4422(19)30190-5.
People severely impaired with progressive multiple sclerosis spend much of their day sitting, with very few options to improve motor function. As a result, secondary physical and psychosocial complications can occur. Effective and feasible self-management strategies are needed to reduce sedentary behaviour and enhance motor function. In this study, we aimed to assess the clinical and cost effectiveness of a home-based, self-managed, standing frame programme.
SUMS was a pragmatic, multicentre, randomised controlled superiority trial of people with progressive multiple sclerosis and severe mobility impairment, undertaken in eight centres from two regions in the UK. The study had assessor-blinded outcome assessments with use of clinician-rated and patient-rated measures at baseline, 20 weeks, and 36 weeks. After baseline assessment, participants were randomised (1:1) by computer-generated assignment to either a standing frame programme plus usual care or usual care alone. The intervention consisted of two home-based physiotherapy sessions (60 min each) to set up the standing frame programme, supported by six follow-up telephone calls (15 min per call). Participants were asked to stand for 30 min, three times per week over 20 weeks, and encouraged to continue in the longer term, although no further physiotherapy support was provided. The primary clinical outcome was motor function measured by the Amended Motor Club Assessment (AMCA) score at week 36, analysed in the modified intention-to-treat population (excluding only patients who were deemed ineligible after randomisation, those who withdrew from the trial and were unwilling for their previously collected data to be used, or those who did not provide baseline and week 36 measurements). A 9-point AMCA score change was considered clinically meaningful a priori. Adverse events were collected through a daily preformatted patient diary throughout the 36 weeks and analysed in the modified intention-to-treat population. An economic assessment established the resources required to provide the standing frame programme, estimated intervention costs, and estimate cost effectiveness. This trial is registered with the International Standard Randomised Controlled Trials, number ISRCTN69614598.
Between Sept 16, 2015, and April 28, 2017, 285 people with progressive multiple sclerosis were screened for eligibility, and 140 were randomly assigned to either the standing frame group (n=71) or the usual care group (n=69). Of these, 122 completed the primary outcome assessment (61 participants in both groups) for the modified intention-to-treat analysis. The use of the standing frame resulted in a significant increase in AMCA score compared with that for usual care alone, with a fully adjusted between-group difference in AMCA score at 36 weeks of 4·7 points (95% CI 1·9-7·5; p=0·0014). For adverse events collected through patient diaries, we observed a disparity between the two groups in the frequency of short-term musculoskeletal pain (486 [41%] of 1188 adverse events in the standing frame group vs 160 [22%] of 736 adverse events in the usual care group), which was potentially related to the intervention. The musculoskeletal pain lasted longer than 7 days in five participants (two in the standing frame group and three in the usual care group). No serious adverse events related to the study occurred. The standing frame group had a mean 0·018 (95% CI -0·014 to 0·051) additional quality-adjusted life-years (QALYs) compared with those of the usual care group, and the estimated incremental cost-per-QALY was approximately £14 700.
The standing frame programme significantly increased motor function in people with severe progressive multiple sclerosis, although not to the degree that was considered a priori as clinically meaningful. The standing frame is one of the first physiotherapy interventions to be effective in this population. We suggest that the programme is feasible as a home-based, self-managed intervention that could be routinely implemented in clinical practice in the UK.
UK National Institute of Health Research.
患有进行性多发性硬化症且严重受损的人一天大部分时间都在坐着，几乎没有改善运动功能的选择。因此，可能会出现继发性身体和心理社会并发症。需要有效的、可行的自我管理策略来减少久坐行为并增强运动功能。在这项研究中，我们旨在评估一种基于家庭的、自我管理的站立框架计划在临床和成本效益方面的效果。
SUMS 是一项在英国两个地区的八个中心进行的针对患有进行性多发性硬化症和严重运动障碍的人群的实用、多中心、随机对照优势试验。该研究采用盲法评估结果，使用临床医生评定和患者评定的措施在基线、20 周和 36 周进行评估。在基线评估后，参与者通过计算机生成的分配按 1:1 的比例随机分为站立框架计划加常规护理组或常规护理组。干预措施包括两次家庭基础物理治疗（每次 60 分钟）来设置站立框架计划，并通过六次随访电话（每次 15 分钟）提供支持。参与者被要求每周站立 30 分钟，每天三次，共 20 周，并鼓励他们在更长时间内继续站立，尽管不再提供进一步的物理治疗支持。主要临床结果是在第 36 周时使用改良的运动俱乐部评估（AMCA）评分测量的运动功能，在改良的意向治疗人群中进行分析（仅排除随机分组后被认为不合格的患者、退出试验且不愿意使用之前收集的数据的患者，或未提供基线和第 36 周测量值的患者）。预先设定的临床意义是 AMCA 评分改变 9 分。通过 36 周期间的每日预格式化患者日记收集不良事件，并在改良的意向治疗人群中进行分析。经济评估确定了提供站立框架计划所需的资源，估算了干预成本，并估计了成本效益。该试验在国际标准随机对照试验（ISRCTN）注册，编号为 ISRCTN69614598。
2015 年 9 月 16 日至 2017 年 4 月 28 日，对 285 名进展性多发性硬化症患者进行了筛选，以确定其是否符合条件，其中 140 名患者被随机分配至站立框架组（n=71）或常规护理组（n=69）。在这些患者中，有 122 名完成了改良意向治疗分析的主要结局评估（两组各有 61 名参与者）。与单独常规护理相比，使用站立框架导致 AMCA 评分显著增加，36 周时 AMCA 评分的完全调整组间差异为 4.7 分（95%CI 1.9-7.5；p=0.0014）。对于通过患者日记收集的不良事件，我们观察到两组之间短期肌肉骨骼疼痛的频率存在差异（站立框架组 1188 次不良事件中有 486 次[41%]，常规护理组 736 次不良事件中有 160 次[22%]），这可能与干预措施有关。有五名参与者（站立框架组 2 名，常规护理组 3 名）的肌肉骨骼疼痛持续时间超过 7 天。没有发生与研究相关的严重不良事件。站立框架组比常规护理组平均多获得 0.018（95%CI -0.014 至 0.051）个质量调整生命年（QALY），估计增量成本-每质量调整生命年约为 14700 英镑。
站立框架计划显著增加了严重进行性多发性硬化症患者的运动功能，尽管其程度没有达到预先设定的临床意义上的显著程度。站立框架是第一种对该人群有效的物理治疗干预措施之一。我们建议该方案作为一种基于家庭的、自我管理的干预措施是可行的，可以在英国的临床实践中常规实施。
英国国家卫生研究院。
