Gene delivery of AAV2-neurturin for Parkinson's disease: a double-blind, randomised, controlled trial

Abstract Background 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. Methods 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. Results 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. Conclusions 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. Trial registration International Standard Randomised Controlled Trial Number Register: ISRCTN75223808. Registered 22 March 2012.

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The effectiveness of peroneal nerve functional electrical simulation for the reduction of bradykinesia in Parkinson’s disease: A feasibility study for a randomised control trial

Clinical Rehabilitation ◽

10.1177/0269215520972519 ◽

2020 ◽

pp. 026921552097251

Author(s):

Paul N Taylor ◽

Trish Sampson ◽

Ben Beare ◽

Maggie Donavon-Hall ◽

Peter W Thomas ◽

...

Keyword(s):

Parkinson’S Disease ◽

Parkinson's Disease ◽

Electrical Stimulation ◽

Randomised Controlled Trial ◽

Study Design ◽

Functional Electrical Stimulation ◽

Standard Care ◽

Peroneal Nerve ◽

Controlled Trial ◽

Randomised Controlled

Objectives: To assess the feasibility of a multi-site randomised controlled trial to evaluate the effect of functional electrical stimulation on bradykinesia in people with Parkinson’s disease. Design: A two-arm assessor blinded randomised controlled trial with an 18 weeks intervention period and 4 weeks post-intervention follow-up. Setting: Two UK hospitals; a therapy outpatient department in a district general hospital and a specialist neuroscience centre. Participants: A total of 64 participants with idiopathic Parkinson’s disease and slow gait <1.25 ms−1. Interventions: Functional electrical stimulation delivered to the common peroneal nerve while walking in addition to standard care compared with standard care alone. Main measures: Feasibility aims included the determination of sample size, recruitment and retention rates, acceptability of the protocol and confirmation of the primary outcome measure. The outcome measures were 10 m walking speed, Unified Parkinson’s Disease Rating Scale (UPDRS), Mini Balance Evaluation Systems Test, Parkinson’s Disease Questionnaire-39, EuroQol 5-dimension 5-level, New Freezing of Gait questionnaire, Falls Efficacy Score International and falls diary. Participants opinion on the study design and relevance of outcome measures were evaluated using an embedded qualitative study. Results: There was a mean difference between groups of 0.14 ms−1 (CI 0.03, 0.26) at week 18 in favour of the treatment group, which was maintained at week 22, 0.10 ms−1 (CI –0.05, 0.25). There was a mean difference in UPDRS motor examination score of –3.65 (CI –4.35, 0.54) at week 18 which was lost at week 22 –0.91 (CI –2.19, 2.26). Conclusion: The study design and intervention were feasible and supportive for a definitive trial. While both the study protocol and intervention were acceptable, recommendations for modifications are made.

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