Scobie Sarah, Jowett Sue, Lambe Tosin, Patel Smitaa, Woolley Rebecca, Ives Natalie, Rick Caroline, Smith Christina, Brady Marion C, Clarke Carl, Sackley Cath
Nuffield, Trust, London, UK.
Health Economics Unit, Institute for Applied Health Research, University of Birmingham, Birmingham, UK.
Pilot Feasibility Stud. 2021 Aug 9;7(1):154. doi: 10.1186/s40814-021-00888-y.
The PD COMM pilot randomised controlled trial compared Lee Silverman Voice Treatment (LSVT® LOUD) with standard NHS speech and language therapy (SLT) and a control arm in people with Parkinson's disease (PwPD) with self-reported problems with voice or speech. This analysis compares costs and quality of life outcomes between the trial arms, and considers the validity of the alternative outcome measures for economic evaluations.
A comparison of costs and outcomes was undertaken alongside the PD COMM pilot trial involving three arms: LSVT® LOUD treatment (n = 30); standard NHS SLT (n = 30); and a control arm (n = 29) excluded from receiving therapy for at least 6 months after randomisation unless deemed medically necessary. For all trial arms, resource use and NHS, social care and patient costs and quality of life were collected prospectively at baseline, 3, 6, and 12 months. Total economic costs and outcomes (EQ-5D-3L, ICECAP-O) were considered over the 12-month follow-up period from an NHS payer perspective. Quality of life measures for economic evaluation of SLT for people with Parkinson's disease were compared.
Whilst there was no difference between arms in voice or quality of life outcomes at 12 months, there were indications of differences at 3 months in favour of SLT, which need to be confirmed in the main trial. The estimated mean cost of NHS care was £3288 per patient per year for the LSVT® LOUD arm, £2033 for NHS SLT, and £1788 for the control arm. EQ-5D-3L was more strongly correlated to voice impairment than ICECAP-O, and was sensitive to differences in voice impairment between arms.
The pilot did not identify an effect of SLT on disease-specific or economic outcomes for PwPD at 12 months; however, there appeared to be improvements at 3 months. In addition to the sample size not powered to detect difference in cost-consequence analysis, many patients in the control arm started SLT during the 12-month period used for economic analysis, in line with the study protocol. The LSVT® LOUD intervention was more intense and therefore more costly. Early indications suggest that the preferred economic outcome measure for the full trial is EQ-5D-3L; however, the ICECAP-O should still be included to capture a broader measure of wellbeing.
International Standard Randomised Controlled Trial Number Register: ISRCTN75223808. Registered 22 March 2012.
帕金森病语音与沟通干预（PD COMM）试点随机对照试验比较了李氏言语疗法（LSVT® LOUD）、英国国家医疗服务体系（NHS）标准言语和语言治疗（SLT）以及对照组对有自我报告的声音或言语问题的帕金森病患者（PwPD）的效果。本分析比较了试验组之间的成本和生活质量结果，并考虑了经济评估替代结果指标的有效性。
在PD COMM试点试验中同时进行成本和结果比较，试验包括三个组：LSVT® LOUD治疗组（n = 30）；NHS标准SLT组（n = 30）；以及对照组（n = 29），对照组在随机分组后至少6个月内不接受治疗，除非被认为有医疗必要。对于所有试验组，在基线、3个月、6个月和12个月时前瞻性收集资源使用情况、NHS、社会护理和患者成本以及生活质量。从NHS支付方的角度考虑12个月随访期内的总经济成本和结果（EQ-5D-3L、ICECAP-O）。比较了帕金森病患者SLT经济评估的生活质量指标。
虽然12个月时各组在声音或生活质量结果方面没有差异，但在3个月时有迹象表明SLT组更具优势，这需要在主要试验中得到证实。LSVT® LOUD组NHS护理的估计平均成本为每年每位患者3288英镑，NHS SLT组为2033英镑，对照组为1788英镑。EQ-5D-3L与声音障碍的相关性比ICECAP-O更强，并且对各组之间声音障碍的差异敏感。
该试点未发现SLT在12个月时对帕金森病患者的疾病特异性或经济结果有影响；然而，在3个月时似乎有改善。除了样本量不足以检测成本效益分析中的差异外，对照组的许多患者在用于经济分析的12个月期间开始接受SLT治疗，这符合研究方案。LSVT® LOUD干预强度更大，因此成本更高。早期迹象表明，完整试验中首选的经济结果指标是EQ-5D-3L；然而，仍应纳入ICECAP-O以更全面地衡量健康状况。
国际标准随机对照试验编号注册库：ISRCTN75223808。2012年3月22日注册。
