Authors' objectives: Speech impairments are common with Parkinson’s disease (reported prevalence 68%), increasing conversational demands, reliance on family and social withdrawal. The PD COMM trial compared the clinical and cost-effectiveness of two speech and language therapy approaches: Lee Silverman Voice Treatment LOUD and National Health Service speech and language therapy for the treatment of speech or voice problems in people with Parkinson’s disease to no speech and language therapy (control) and against each other. Parkinson’s disease (PD) is a complex neurodegenerative disorder experienced by 6.1 million people worldwide in 2016. PD is more common in men (1.4 : 1.0) and mostly affects people over 50, with peak incidence between 85 and 89 years old. PD affects approximately 145,000 people in the UK. Symptoms of PD are classified as motor or non-motor. The initial diagnostic motor symptoms are unilateral tremor, slowness, stiffness and mild imbalance. However, as the condition progresses, more severe motor decline occurs with imbalance leading to falls and unpredictable freezing episodes, all of which are unresponsive to medication. Speech and voice problems (known as dysarthria) are common with a reported prevalence of 68%. These problems increase physical and mental demands during conversation, reliance on family and/or carers and the likelihood of social withdrawal reducing quality of life. Speech and language therapy (SLT) in the UK aims to improve communication for people with PD-related dysarthria and their families. The NHS SLT or Lee Silverman Voice Treatment (LSVT) LOUD are two SLT approaches typically available in the UK, but evidence of their effectiveness is inconclusive. The primary objective of the PD COMM trial was to evaluate the clinical and cost-effectiveness of two SLT approaches: LSVT LOUD and NHS SLT compared with no SLT (control) for people with PD-related dysarthria. The primary comparisons were: LSVT LOUD versus no SLT control and NHS SLT versus no SLT (control). An additional objective was to evaluate and compare the clinical and cost-effectiveness of two types of SLT (LSVT LOUD vs. NHS SLT) in people with PD.

Authors' results and conclusions: Three hundred and eighty-eight participants were randomised to Lee Silverman Voice Treatment LOUD (n = 130), National Health Service speech and language therapy (n = 129) and control (n = 129). The impact of voice problems at 3 months after randomisation was lower for Lee Silverman Voice Treatment LOUD participants than control [−8.0 (99% confidence interval: −13.3, −2.6); p = 0.001]. There was no evidence of improvement for those with access to National Health Service speech and language therapy when compared to control [1.7 (99% confidence interval: −3.8, 7.1); p = 0.4]. Participants randomised to Lee Silverman Voice Treatment LOUD reported a lower impact of their voice problems than participants randomised to National Health Service speech and language therapy [99% confidence interval: −9.6 (−14.9, −4.4); p 

Authors' methods: PD COMM is a phase III, multicentre, three-arm, unblinded, randomised controlled trial. Participants were randomised in a 1 : 1 : 1 ratio to control, National Health Service speech and language therapy or Lee Silverman Voice Treatment LOUD via a central computer-generated programme, using a minimisation procedure with a random element, to ensure allocation concealment. Mixed-methods process and health economic evaluations were conducted. United Kingdom outpatient and home settings. People with idiopathic Parkinson’s disease, with self-reported or carer-reported speech or voice problems. We excluded people with dementia, laryngeal pathology and those within 24 months of previous speech and language therapy. The Lee Silverman Voice Treatment LOUD intervention included maximum effort drills and high-effort speech production tasks delivered over four 50-minute therapist-led personalised sessions per week, for 4 weeks with prescribed daily home practice. National Health Service speech and language therapy content and dosage reflected local non-Lee Silverman Voice Treatment speech and language therapy practices, usually 1 hour, once weekly, for 6 weeks. Trained, experienced speech and language therapists or assistants provided interventions. The control was no speech and language therapy until the trial was completed. Primary outcome: Voice Handicap Index total score at 3 months. Secondary outcomes: Voice Handicap Index subscales, Parkinson’s Disease Questionnaire-39; Questionnaire on Acquired Speech Disorders; EuroQol-5D-5L; ICEpop Capabilities Measure for Older Adults; Parkinson’s Disease Questionnaire – Carers; resource utilisation; and adverse events. Assessments were completed pre-randomisation and at 3, 6 and 12 months post randomisation. The number of participants recruited to the trial did not meet the pre-specified power. The PD COMM trial was a UK, multicentre, three-arm parallel group, unblinded, superiority, randomised controlled trial with a 12-month follow-up. Participants were randomised at the level of the individual to treatment with NHS SLT, LSVT LOUD or no SLT (control) in a 1 : 1 : 1 ratio. Participants randomised to the no SLT (control) group could be referred for SLT at the end of trial or if it became medically necessary, during the trial. The type and dose of SLT for those in the no SLT (control) group for whom it became necessary was determined by the therapists and clinicians responsible for the care plan of the participant. Recruitment took place at 41 sites throughout the UK and sites remained open until the trial finished. This trial was conducted in the UK in outpatient and home settings. The trial interventions were intended to be provided as follows: LSVT LOUD consisted of personalised maximum effort drills and high-effort speech production tasks. NHS SLT reflected local non-LSVT practices: content, dose and frequency determined by the therapist in response to participant’s individual needs. NHS SLT was tailored to the individuals’ needs as per local practice, typically consisting of 6 to 8 weekly sessions; LSVT LOUD comprised 16 sessions of individual treatment with home-based practice, over 4 weeks (16 hours dosage). Suitably trained speech and language therapists or therapist assistants administered the interventions. People with idiopathic PD where themselves or their carer reported speech or voice problems, were enrolled. We excluded people with a diagnosis of dementia, laryngeal pathology or those who have received SLT for speech or voice problems in the previous 2 years. Speech and language therapists or assistants trained in LSVT LOUD delivered the intervention. Speech and language therapists on the trial were provided with LSVT LOUD training by LSVT Global for free if they needed it to register or re-register as a LSVT therapist. Participants were recruited from their routine outpatient appointments in geriatric/elderly care, neurology or SLT secondary care settings. Interventions were provided through secondary-care outpatient community-based SLT departments. For some participants who had specific needs, the intervention was provided at home. Participants were randomised at the level of the individual via a central, secure, web-based computer-generated randomisation system developed and controlled by the Birmingham Clinical Trials Unit (BCTU), thus ensuring concealment of next treatment allocation. To randomise a patient into the trial, staff delegated the task of randomising patients into the trial either logged onto the trial database or rang the BCTU randomisation telephone line. The randomisation process used a minimisation procedure. The following minimisation variables were used: age (≤ 59, 60–70, > 70 years); disease severity measured using the Hoehn and Yahr staging (1.0–2.5, 3.0–5.0) and severity of speech measured using the Voice Handicap Index (VHI) total score (≤ 33, mild 34–44, moderate 45–61, severe > 61). To avoid any possibility of the treatment allocation becoming predictable, a random element was included within the randomisation process. Once the participant was randomised into the trial, they were given a unique trial identifier and the treatment allocation was confirmed by e-mail to the site. The primary outcome measure for the trial was patient-reported VHI total score at 3 months. The VHI measures the psychosocial consequences of voice disorders and provides an overall perception of effectiveness of voice-related communication. The VHI comprises of 30 questions (0–120 negatively scored) divided into emotional, functional and physical subscales (0–40 subscale score). Secondary outcomes were: the VHI subscales; the Parkinson’s Disease Questionnaire-39, a validated, health-related quality-of-life measure specific to PD; the Questionnaire on Acquired Speech Disorders, self-reported participation restriction related to speech and communication; the participant-rated EuroQol5D (5-level version) which provides a simple descriptive profile and a single index value for health status; the ICEpop Capabilities Measure for Older Adults (ICECAP-O), a measure of capability in older people for use in economic evaluations; a disease-specific participant resource usage questionnaire; carer quality of life, measured using the PD Questionnaire – carer; and adverse events. For participants in either SLT arm, any vocal strain or abuse believed to be associated with treatment was identified by the therapists at the participants’ SLT session and was reported in the adverse event log. All participant-reported resource usage forms completed for the trial were checked to ensure that no vocal strain or abuse had occurred in outpatient appointments with ENT specialists. At the 12-month clinical visit, the medical professional also checked whether any adverse events had occurred since entering the trial. There were two nested studies within this trial: a process evaluation and an economic evaluation. A process evaluation of the content of the interventions examined participants’ and therapists’ reported content of a subset of the two SLT interventions. Individual participant therapy data recorded by participants and therapists during the PD COMM trial were extracted from treatment record forms and therapy notes. Extraction category headings were piloted and reflected the Template for Intervention Description and Replication intervention reporting guidelines, with ongoing refinement as extraction progressed and components of the interventions were identified which were inadequately accounted for in the initial framework. The process evaluation of the implementation of the interventions used normalisation process theory which is highly attuned to the challenges of complex interventions as it encourages looking at systems as a whole. We considered that its conceptual framework would assist the interpretation and synthesis of data and analysis to explain what implementation processes took place and the interactions and gaps between the PD COMM interventions, the changing context, speech and language therapists and their practice. A within-trial economic evaluation in the form of cost–utility analysis was conducted from the perspective of the NHS. The primary results were expressed in terms of cost per quality-adjusted life-year (QALY) gained at 12 months for the three trial comparisons. Additional secondary analyses were performed from the NHS, personal social services perspective, broader societal perspective and using the capability approach, which uses broader measures of capability well-being.

Project Status: Completed

URL for project: https://www.journalslibrary.nihr.ac.uk/programmes/hta/10/135/02

Year Published: 2024

URL for published report: https://www.journalslibrary.nihr.ac.uk/hta/ADWP8001

URL for additional information: English

English language abstract: An English language summary is available

Publication Type: Full HTA

Country: England, United Kingdom

DOI: 10.3310/ADWP8001

Organisation Name: NIHR Health Technology Assessment programme

Contact Address: NIHR Journals Library, National Institute for Health and Care Research, Evaluation, Trials and Studies Coordinating Centre, Alpha House, University of Southampton Science Park, Southampton SO16 7NS, UK

Contact Name: journals.library@nihr.ac.uk

Contact Email: journals.library@nihr.ac.uk