Hydrotherapy in mucopolysaccharidosis II

Authors
Category Primary study
Registry of TrialsISRCTN registry
Year 2023
INTERVENTION: Participants are randomised into two groups A and B. While group A initially receives the hydrotherapy treatment, group B receives nothing and completes pain and quality of life questionnaires. Then there is a washout period. Then group B receives the treatment whilst group A receives no therapy but completes the questionnaires. The outcome is measured before each treatment and between the treatments. This study design will give 10 data points for analysis. The hydrotherapy lasts 12 weeks and the control arm will last 12 weeks. Overall each patient will participate in the study over 24 weeks. CONDITION: Mucopolysaccharidosis type II ; Nutritional, Metabolic, Endocrine ; Mucopolysaccharidosis, type II PRIMARY OUTCOME: Quality of life assessed using the Short Form Health Survey 36 scale (performed by nurse) at baseline, 6 weeks and 12 weeks while undergoing hydrotherapy and then separately while in the non‐intervention arm SECONDARY OUTCOME: Assessed at baseline, at a 6‐ and 12‐week follow‐up unless stated otherwise:; 1. Risk assessment at baseline ; 2. Symptom severity assessed using the Brief Pain Inventory and pain‐related questionnaires related to anxiety (GAD‐7), quality of life (PHQ9) and kinesiophobia (TSK11) (performed by pain specialist/nurse); 3. The objective LANSS score will be used to identify and exclude patients who suffer from predominantly neuropathic pain (rather than musculoskeletal nociceptive‐driven pain). This assessment will be performed by a pain specialist.; 4. Global impression of change for patients with learning difficulties as assessed by the MDT team (consultant, physiotherapist & nurse); 5. Physiotherapy assessment including joint range of movement, muscle strength, functional mobility using ‘Timed Up and Go’ and either a 6‐minute walk test or 10‐m walk test as appropriate (performed by a specialist physiotherapist); 6. Medication utilization assessed using questionnaires before and after the intervention (performed by a nurse); 7. Adverse event log from starting hydrotherapy e.g. balance problems after each hydrotherapy session; 8. Patient satisfaction assessed using a questionnaire at the end of the study INCLUSION CRITERIA: 1. Adult MPS II patients (Hunter syndrome; all males aged 18‐58 years), who suffer from joint pain and stiffness and/or post‐surgical musculoskeletal pain, not responding to analgesic agents 2. Patients with reduced mobility and wheelchair reliance 3. Patients are able to give consent and feedback. In patients with limited capacity, their carers/parents may be asked to help complete the questionnaires. 4. Participants who pass the Trust hydrotherapy risk assessment at baseline
Epistemonikos ID: a03b39f44e7bea9904f3d5265b035632e4d86d6c
First added on: Aug 26, 2024