Title: A multi-centre randomised controlled trial to compare the clinical and cost effectiveness of Lee Silverman Voice Treatment versus standard NHS speech and language therapy versus control in Parkinson's disease (PD COMM)
Funder: Department of Health and Social Care

Abstract: PHASE III PRAGMATIC MULTICENTRE 3 GROUP RCT with economic evaluation. The randomisation programme will use a minimisation procedure (with a random element) with the following variables: age, disease severity (as estimated by Hoehn and Yahr staging), and severity of speech impairment measured by the Voice Handicap Index. Analysis will be by intention-to-treat. SETTING: Patients will be recruited from ~40 neurology and elderly care outpatient clinics nationwide, building on the 11 pilot sites (Including; City Hospital, Birmingham; Fairfield Hospital, Bury, Royal Devon and Exeter Hospital, Devon, Southern General Hospital, Glasgow). SLT interventions will be delivered within both outpatient and community-based settings as per local practice. Over forty centres from our PD trial network have expressed an interest in taking part. TARGET POPULATION: Patients with a confirmed diagnosis of idiopathic PD (using the UK Parkinson’s UK (PUK) Brain Bank Criteria), and self or carer-reported problems with speech when asked the simple question “Do you have any problems with your speech or voice?” Participants should not have had SLT within the last year or have a history of laryngeal pathology or surgery. HEALTH TECHNOLOGIES BEING ASSESSED: LSVT: Therapists are formally trained in this technique and a list of practitioners is held. The treatment focuses on improving intelligibility through vocal loudness. Four 50 minute sessions are delivered each week over 4 weeks and daily home practice is completed. Standard UK NHS SLT: This includes the provision of exercises, techniques and therapeutic devices targeting prosody, respiratory capacity and control, and the articulatory muscles. Dose will be determined by the individuals’ needs, but is most likely to reflect the dose reported in the pilot and a survey of current UK SLT practice for PD by Miller et al (2010) of 6-8 sessions over 6-8 weeks. Control: This group will continue with their standard PD care (avoiding benign neglect) and will not be referred for SLT within the 12 months of the study. A process evaluation will be carried out. MEASUREMENT OF COSTS AND OUTCOMES: Baseline characteristics: Age/ date of birth, gender, date of PD diagnosis, Hoehn and Yahr stage and current medication will be recorded by the referring physician. Our pilot study has informed the final choice of outcomes measures for the main trial. Measures will be collected at baseline, 3 (to allow for the varying intervention lengths), 6 and 12 months from randomisation. The primary outcome measure will be the self-completed Voice Handicap Index (VHI) at 3 months. Secondary measures will include; PDQ-39 & Voice-Related QoL scale for the patient, PDQ-39 for the carer. Resource use, EQ5D and ICECAP O for health economic evaluation will be collected at 3, 6 and 12 months.

Lay abstract: THE PROBLEM Around two thirds of people with Parkinson's disease (PD) have problems with their speech. However, in the UK, less than a third of these people are offered speech and language therapy (PUK Membership Survey 2008). The lack of concrete evidence of any benefit of therapy is a barrier to the commissioning of the service. THE STUDY We propose to address this problem by following the HTA brief to evaluate the effectiveness and value for money of speech and language therapy (including the 2 types of therapy most frequently used in the NHS currently) compared with a standard care control group (of no treatment). To do this a physician, nurse or research facilitator will ask people with PD who feel they have difficulty with their speech or voice (or perhaps their carer feels they have problems) to take part in a study. Once the person has consented to take part, we will use a computer to decide which group they will be asked to join. The three groups will be: • Lee Silverman Voice Treatment. This will be delivered through face to face sessions with one therapist, usually in the speech therapy clinic. Treatment will be delivered for 50 minutes, 4 times a week for 4 weeks, with a focus on vocal loudness. • Traditional NHS therapy. This is more variable but is commonly 6 - 8 sessions, once a week, in the speech and language therapy clinic. • Standard care for PD but with no therapy referral during the study (approx 1 year). All participants will be asked to complete assessments at the start, 3, 6 and 12 months later. Their carer (or relative) will also be asked to complete a questionnaire at the same 4 time-points. The assessments will cover general ability to communicate and the general effects on their well-being. The study team will compare treatment with the control, and then explore the two different speech and language therapy treatments. The costs and value for money will also be examined. ETHICAL ISSUES We do not know if speech and language therapy works for people with PD, and if it does which therapy works best and so it is not to the person’s disadvantage to be allocated to any of the three groups. However, we would hope that therapy has some positive effect. At the end of the study (approximately 1 year later) people in the control group will be referred to therapy if they so wish. This is the reason why we are not using a longer period of follow up. If at any time the clinician caring for the participant feels that speech and language therapy becomes essential they can be referred via usual NHS routes. SERVICE USER INVOLVEMENT We have extensive experience of running successful trials in this area, and have included thousands of people with PD in trials. Through these studies we have linked with Parkinson's UK to establish a panel of people and their families who work with us on our studies (including this one). This is invaluable as it helps us to incorporate their views and ideas and it enhances our ability to deliver the study in a way that reflects their views. In addition, we have included a service user and a representative from Parkinson's UK as collaborators in order to optimise service user involvement. We also work hard to incorporate the views of people who may not be well represented within PDUK. Birmingham is second only to London for the proportion of people from ethnic minorities and the lead applicant's department specialises in methods of enhancing inclusion. THE TEAM Expert input in the area of Speech and Language therapy has been provided by Dr Christina Smith (Senior Lecturer, University College London; Specialist practitioner, Moseley Hall Hospital) and Professor Marian Brady (Programme director,pan-Scotland Nursing, Midwifery and Allied Health Professions Research Unit). Professor Lorraine Ramig, University of Colorado (who was instrumental in developing and evaluating LSVT) will also advise. The applicants have worked together on several large clinical trials of Parkinson’s disease and other rehabilitation studies. The University of Birmingham Clinical Trials Unit is very experienced in running large NHS-based studies of healthcare and has a proven record of recruiting successfully to rehabilitation trials. Sackley, Clarke, Rick and Ives have successfully completed a pilot study, exceeding our target of 60 people by 29. We have recently completed a trial of physiotherapy and occupational therapy in PD (PD REHAB) recruiting more than the 750 participants required, within the time and budget constraints. Our relationships with therapy managers will further enhance our ability to recruit in PD COMM and Prof Brady's links will continue to enhance recruitment in Scotland. Dr Burton is an expert in study process evaluation. The trials unit also has a team of experienced data managers, computer programmers and administrators in addition to the scientists, statisticians, health economists and therapists involved in the application. THE COST AND OUTCOME People with PD have substantial differences in their symptoms and experiences of their disease. Communication practices are also very variable. In order to reflect this variability we have to include a large number of people. Our pilot study indicates we will require 500 people to be able to adequately answer this question reliably. The results from high quality studies (such as the one we propose) can be directly incorporated into the NHS and can improve care. However, the large number of people with Parkinson’s disease, the complexity of the study, the number of experienced people in the study team and the length of the study all contribute to the cost. We feel that our proposed study offers the best chance of answering the questions and represents good value for money.

Research activity codes: 6.7 PHYSICAL
Health categories: NEUROLOGICAL