Title: Developing a rehabilitation intervention for the management of chronic arthritic joint pain in people with haemophilia.
Funder: Health Education England

Abstract: Rationale: Haemophilia is an inherited bleeding disorder characterised by recurrent and spontaneous bleeding into joints and muscles. Approximately 8300 people with haemophilia (PWH) A or B are registered in the UK, with 40% being classified as having severe or moderate disease. PWH experience acute pain from an early age from musculoskeletal bleeding episodes as well as from multiple repeated venipunctures. Due to lack of appropriate treatment in their youth, many adults with severe haemophilia have chronically painful, multi-joint arthritis in the elbows, knees and ankles. Between 35-50% of PWH report chronic musculoskeletal pain and this poorly managed phenomenon has significant debilitating personal and healthcare resource impacts. The cost to the NHS of prophylaxis with factor concentrate replacement for an average adult with haemophilia, is in the range of 70,000 to 500,000 per annum. Those with arthritis have greater costs in addition to this, as the arthritis makes it difficult to distinguish between the sensation of an acute joint bleed and that of arthritic pain.Most recommendations for pain management in haemophilia relate to pharmacological management of pain. It has only recently been highlighted that the use of physiotherapy and exercise is under-investigated and trials should be designed to gauge its potential benefits for pain management. Although it appears safe to do exercise in PWH, there has been no structured scientific research to evaluate the effect of a rehabilitation programme for management of chronic lower limb multi-joint pain in PWH, where pain, quality of life and function are primary outcomes. Knowledge of the potential benefits of rehabilitation as a management strategy for chronic joint pain appears limited. The lack of guideline for chronic pain management in PWH means the knowledge, confidence and pathways used by haemophilia clinicians such as haematologists, specialist nurses and physiotherapists, in managing chronic joint pain, is unclear.Aim: To develop and test the feasibility of a rehabilitation programme, that incorporates education and exercise, for the management of chronic arthritic lower limb joint pain in people with haemophilia. Methods: Phase I - Development and modelling Systematic review to evaluate pain management interventions in haemophilia and to define effectiveness of current interventions for chronic pain management in haemophilia Focus groups with PWH exploring their own experience of pain and strategies used and their views and opinions on the use of rehabilitation and pain education for joint pain in PWH. Develop a Theory of Change (ToC) model to inform the feasibility study. This causal framework map will map all elements of the intervention, by understanding how and why the intervention has a particular effect Phase II Feasibility testing Design: A multi-centre, parallel group, cross-sectional, randomized feasibility study. Population: 48 adults with severe Haemophilia A or B with chronic lower limb pain. Intervention: 6-week rehabilitation and education programme Control: Usual care, which can include physiotherapy assessment/contact within the haemophilia centre Feasibility outcomes: Number of patients screened for eligibility, proportion of eligible patients, number willing to be randomised, number completing programme, safety and suitability of outcome measures used. A questionnaire survey will investigate patient and physiotherapist experience of the trial.Potential Benefits: At the end of the study, we will not be able to answer the question Does exercise and education help reduce pain in people with haemophilia? However, we will know whether such a research study can be done, and what the best ways to do it will be. The potential for financial savings due to inappropriate use of factor concentrate to treat chronically painful joints is also significant, as are clinical implications of an improved and effective management strategy.

Lay abstract: People with haemophilia (PWH) experience pain on many occasions in their life. This can be from injecting their treatment or having a joint bleed. When a joint has had many bleeds, a painful arthritis can develop. The pain from this arthritis is there every day and can stay for many years this is called chronic pain. In haemophilia this usually in the knees and ankles, and often people find it painful to do even day to day things like walk or go to work. In studies where people with haemophilia have been asked it they have chronic pain, 50% say that they do. Most people use a lot of painkillers for this chronic pain but they are often not very effective. PWH who have this chronic pain tell us that they are scared to do exercise in case it makes their pain worse, or makes them have a joint bleed.Unlike haemophilia, people who have osteoarthritis (OA) usually get their arthritis in later life, and it is often only one joint in their leg that is affected. Studies have been done on patients with OA that have chronic knee pain. They show that rehabilitation (exercise), with some education about their arthritis and pain, helps them to manage and cope with their pain better. This effect has been so positive, that national guidelines for management of OA knee pain state that all patients with OA should have access to this kind of rehabilitation.As yet, we do not know if this kind of approach can work with PWH who also have multiple joint pain in their legs, as this kind of study has never been done. Although some studies now show general exercise is safe to do with PWH, we cannot assume that the programme that works in OA knee pain will work in a PWH. This is because PWH usually have more than one chronically painful joint in their legs (knees and ankles). PWH have usually had their arthritis from a much younger age than those with OA so they have lived more years with their pain, and may have their own thoughts and ideas about it.The aim of this project is to develop and then test a rehabilitation programme, that includes teaching about pain and arthritis, to see if it is safe to do in PWH that have chronic knee and ankle pain. It will be carried out in physiotherapy gyms at the hospital where the patients' haemophilia centre is, and will be done by physiotherapists who know about haemophilia.There are two main parts to the project: First we will develop the rehabilitation programme. We will review previous studies to see how chronic pain in haemophilia is currently being managed. We will assess which aspects of these are the most successful and if we should include them in our study. We will also hold focus groups. These are meetings where we will ask patients what they think of using rehabilitation for chronic joint pain, what their knowledge is of pain science and why arthritis is painful, and ask them what they think is important for us to consider when planning such an intervention. We will also have a group meeting with PWH and professionals who work in haemophilia. This will be to help us design what the rehabilitation and education programme will look like. Second, we will then test out the programme in a research study. We will recruit 48 patients. 50% of them will be randomly placed in to the rehabilitation arm of the study and the other 50% will just receive their normal care, that is, will not take part in the rehabilitation programme. The rehabilitation programme will last six weeks, with two sessions per week. We will measure success of the study by recording how many people completed the programme, how safe it was for them, how they felt about being part of it and how happy they were to be in either the rehabilitation or usual care group. We will also measure pain, quality of life, physical well-being and physical function before and after the programme. PWH have been involved in the design of this project, and will also be involved in the focus groups meetings and designing any patient information being used in the study. The findings from this study will help us decide if this type of rehabilitation approach is worth pursuing further, and which parts of the programme we should keep or change.

Research activity codes: 6.7 PHYSICAL
Health categories: BLOOD;MUSCULOSKELETAL