Background Exercise is important for children with hemophilia to maintain a healthy musculoskeletal system and to restore function as a consequence of muscle and joint bleeding. This study investigated the views of physiotherapists and children with hemophilia, along with their parents, on the delivery of an online muscle-strengthening intervention. The intervention involved a 12-week program intended to master movement control and emphasize body weight strength development, initiated by one in-person study visit at a care center when participants were randomized. The children conducted the program twice weekly, once with a physiotherapist and a second independently, but with the support of a parent. Methods The study involved qualitative exploration using online focus groups with the study’s physiotherapists and telephone interviews with children/parent dyads. Two focus groups involving eight physiotherapists and 12 interviews with children were conducted, with a parent present, both based at home. The interviews and focus groups were conducted by an experienced qualitative researcher, Ferhana Hashem (FH). The recordings of the focus groups and the interviews were then transcribed. Qualitative data were analyzed thematically. The analysis was supported using a qualitative software data management program (NVIVO 15). In terms of patient reported outcomes (PRO) selected, parents and children felt it important to ensure that the children’s confidence and feelings were supported to do the exercises, which were captured by the Child’s and parents’/caregivers’ satisfaction of the exercise programe and choice of outcome measures on the questionnaire. Results Qualitative themes were identified: (i) initial contact – face to face session, recruitment, and adherence; (ii) access and engagement; (iii) guidance from physiotherapists for sessions; (iv) multi-modal systems supporting exercise program; and (v) changing physiotherapy practice. Conclusion Physiotherapy care for children with hemophilia can be successfully supported and adapted to home-based settings. Physiotherapists were agile and innovative in providing sessions exclusively online. The real-time online sessions, physitrack application, and supporting resources were age-appropriate and accessible to both children and parents. Online physiotherapy care has the potential for ongoing engagement and accessibility, opportunities for collaborative practice through family centered approaches, and ultimately, greater sustainability in this rare diseases group.

Background: Advances in hemophilia A (HA) treatment have led to lower bleeding rates (Srivastava et al. Haemophilia 2020), shifting focus toward functional endpoints such as joint health. Despite treatment with factor (F)VIII prophylaxis, joint health has been observed to deteriorate over time (Arvanitakis et al. Haemophilia 2024). BEYOND ABR (NCT05181618) aims to evaluate joint health and physical activity outcomes in people with HA (PwHA) switching from FVIII prophylaxis to emicizumab. This second interim analysis reports data analyzed after 12 months of treatment with emicizumab. Methods: BEYOND ABR is a Phase IV, multicenter, open-label study in PwHA aged 13–69 years with moderate/severe HA without FVIII inhibitors. Joint health for ankles, knees, and elbows is evaluated in participants' joints without pre-study surgery/procedures using the Hemophilia Joint Health Score (HJHS) 2.1. Numerical changes in problem joint counts (defined as per Chowdary et al. Haemophilia 2023) from baseline are captured by both participants and investigators. Target joint resolution is assessed in participants with ≥52 weeks of follow-up. Physical activity is measured using the International Physical Activity Questionnaire (IPAQ). The number of participants with zero treated bleeds is determined using data from a Bleeds and Medication Questionnaire. The Emicizumab Preference Survey was carried out following 6 months of emicizumab treatment. Results: Overall, 136 PwHA were enrolled, all male with varying levels of joint status/impairment. At the single joint level, mean (standard deviation [SD]) HJHS total score was 2.3 (3.5) at baseline and improved by -0.3 (1.7) at Month 6 and -0.5 (1.9) at Month 12. At Month 6, 93/726 (12.8%) joints showed an improvement of ≥2 points, increasing to 119/696 (17.1%) at Month 12; 41/726 (5.6%) joints and 42/696 (6.0%) joints showed a worsening of ≥2 points at Month 6 and Month 12, respectively. At the participant level, the mean (SD) HJHS sum of joints (excluding Global Gait Score; maximum score: 120, calculated as 6 joints x 10 points each) was 10.1 (13.2) at baseline and improved by -2.0 (6.4) at Month 6 and -2.8 (7.9) at Month 12. In total, 23/88 (26.1%) participants reported an improvement of ≥4 points in HJHS sum of joints from baseline to Month 12 and 5/88 (5.7%) reported worsening of ≥4 points at Month 12. At Month 12, the number of participant-reported problem joints had decreased from baseline in 35/117 (29.9%) participants, including resolution of ≥2 problem joints in 16 (13.7%) participants. Conversely, 16/117 (13.7%) participants reported an increase in the number of problem joints from baseline, including an increase of ≥2 problem joints in 8 participants (6.8%). The number of investigator-reported problem joints reduced in 33/131 (25.2%) participants at Month 12; 13 (9.9%) participants had ≥2 resolved problem joints. Overall, 19/131 (14.5%) participants had an increase in investigator-reported problem joints, including 14 (10.7%) with an increase of ≥2 problem joints. In participants who remained in the study for at least one year, 27/27 (100%) baseline target joints in 15 participants had resolved at Month 12. In participants with valid IPAQ data at baseline and/or 3 months and/or 12 months after switching to emicizumab, the proportion of participants in the low physical activity category decreased from 30.8% (32/104) at baseline to 22.3% (23/103) at Month 3, and remained stable at Month 12 (23.4% [22/94]). The proportion of participants in the high physical activity category increased from 44.2% (46/104) to 52.4% (54/103) at Month 3, and was 50.0% (47/94) at Month 12. Zero treated bleeds were reported by 110/136 (80.9%) participants between Weeks 1 and 24, and 105/134 (78.4%) between Weeks 25 and 48. At Month 6, 125/130 (96.2%) participants preferred emicizumab to their previous FVIII prophylaxis, while only 1/130 (0.8%) preferred their previous treatment; 4/130 (3.1%) had no preference. Conclusions: In the first 12 months after switching from FVIII prophylaxis to emicizumab, participants had low bleeding rates associated with numerical improvements in joint health, as measured with HJHS, plus a reduction in the number of problem and target joints. Overall, physical activity levels assessed with IPAQ were stable or showed a shift towards higher activity levels, and most participants preferred emicizumab compared with their previous treatment. Follow-up will continue for 3 years.

ABSTRACT:Hemophilia is a rare inherited bleeding disorder associated with recurrent musculoskeletal bleeding, chronic pain, and functional decline. Advances in prophylactic therapies, including extended half-life factors, non-factor therapies like emicizumab, and gene therapies, have fundamentally transformed care, enabling a paradigmatic shift from activity restriction to promotion. This review synthesizes recent evidence to examine the multifaceted role of physical activity (PA) in people with hemophilia (PwH), current challenges, and strategies to optimize health outcomes. PA confers substantial benefits for PwH, improving muscle strength, coordination, bone density, cardiovascular fitness, mental health, and quality of life. It modulates chronic pain through neuroplastic, anti-inflammatory, and neuroendocrine mechanisms, while exercise-derived molecules (exerkines) may directly influence cartilage health. With individualized prophylaxis and structured programming, both resistance and aerobic exercise can be prescribed safely using evidence-based risk stratification (categories I-III) and comprehensive monitoring approaches, including accelerometers, questionnaires, and subjective effort scales. Persistent barriers include chronic pain, fear of injury, insufficient professional guidance, and treatment inequities, while facilitators encompass enjoyment, social support, and adequate prophylactic coverage. Therapies providing continuous hemostatic protection have further expanded safe PA opportunities, supporting participation in previously restricted activities. In this new therapeutic era, PA should be recognized as a fundamental pillar alongside medical care. Not only for musculoskeletal preservation, but also as a strategy promoting metabolic and mental health. Ensuring access for all patients to both advanced therapies and tailored PA prescription, supported by education, enabling policies, and multidisciplinary care, is essential for democratizing active lifestyles within the global hemophilia community.

INTRODUCTION:The accurate physical examination of musculoskeletal (MSK) health is an essential part of proactive best practice care of people with hemophilia and must be consistently monitored in clinic. In response to current MSK tool limitations, the Musculoskeletal Health Expert Working Group (MSKH EWG) of the International Prophylaxis Study Group (IPSG) has identified developing an improved MSK scoring system a priority. AIM:To identify the priority needs for healthcare professionals examining the MSK health of people with hemophilia in clinic. METHODS:Priorities were identified through a modified Delphi consensus process and an expert meeting with the members of the MSKH EWG. Three iterative surveys were distributed worldwide to healthcare professionals who were participants of the World Federation of Haemophilia (WFH) MSK Committee meetings. RESULTS:The modified Delphi process identified priorities of these healthcare professionals (n = 78) through surveys and an expert meeting (n = 13). The three surveys achieved response rates of 58%, 54% and 55%. The top two priorities, as to why a new MSK scoring system should be developed, were 1) current tools cannot detect the earliest and subtle changes of hemophilia, and 2) current tools are too time consuming. The top two attributes and purposes of a new MSK scoring system were to 1) measure the earliest changes of hemophilia, and 2) measure joint health changes over the course of time. CONCLUSION:The Delphi process successfully identified which future MSK examination priorities should be considered in the development of an improved MSK scoring system for clinical use.

PURPOSE:Swimming is one form of exercise advised to people with chronic low back pain (CLBP), there is limited research, however, supporting this recommendation and describing the experience and use of swimming in this population. The aim of this study was to explore the experience of people who use swimming to manage CLBP. METHODS:Semi-structure interviews were conducted with 14 swimmers who were using swimming to manage CLBP. Thematic analysis was used to analyse the interview data, and the themes were mapped onto the capability, opportunity, motivation and behaviour (COM-B) model to understand the behavioural factors. RESULTS:Five common themes were developed during the analysis: (1) My back pain journey; (2) Learning to swim with back pain; (3) How swimming looks for me; (4) What I gain from swimming; and (5) Keep calm and carry-on swimming. The themes mapped onto all three COM-B domains. CONCLUSIONS:The participants found that swimming was a valuable self-management tool for CLBP. The findings from the thematic and COM-B analysis indicate that learning to swim with CLBP can be a complex journey, influenced by several interrelated behavioural factors. In the absence of multi-professional support, inclusive swimming communities and accessible swimming venues, swimming participation rates may be affected.

Hemophilia is a bleeding disorder characterized by recurrent bleeding into muscles and joints. Many people with hemophilia experience multiple traumatic painful bleeding episodes, meaning that pain is often a significant problem for people with hemophilia, with a potentially high prevalence of posttraumatic stress and posttraumatic stress disorder symptoms. Current pain treatments are often ineffective and do not consider pain memories, which are experienced by almost half of people with posttraumatic stress disorder and which has not been explored in people with hemophilia. To fill this gap, 14 semistructured interviews with men with hemophilia were completed between November 2022 and January 2023 to explore their lived experiences of pain relating to their hemophilia. Data were analyzed using reflexive thematic analysis. Three overarching themes were constructed from the data: “trauma histories,” “pain management,” and “impact on the present.” Findings show that pain experienced by people with hemophilia is complex and does include a memory element for many individuals. Pain memories are clear and vivid and include visual, somatic, and emotional elements in intricate detail. Pain can also be experienced in the present when experiencing a pain flashback, and pain in the present can be a trigger to recalling and re-experiencing pain memories from the past. Self-taught active dissociation from pain, imagery, and distraction were described as useful pain management strategies. Findings from this study have implications for clinicians and service providers because current pharmacological and physical pain management techniques may be ineffective when pain memory is involved. Future interventions should consider how pain management is advanced for people with hemophilia and how hemophilia services become trauma informed.

INTRODUCTION:People with haemophilia continue to experience pain impacting activities of daily living despite the advances in haemostatic treatments. Although guidelines exist, pain management in the clinical setting remains inadequate. It is unclear why this gap in practice persists. This study aimed to explore the practice and experience of pain management by haemophilia healthcare professionals (HCPs) to better understand perceived competency and barriers/facilitators to appropriate care. METHODS:European HCPs working ≥ 3 years in haemophilia care were invited to participate (haematologists, nurses, physiotherapists and psychosocial professionals). Data were collected using profession-specific focus groups and semi-structured individual interviews, recorded and transcribed. The data were analysed using reflexive thematic analysis. RESULTS:Twenty-three HCPs (6 haematologists, 7 nurses, 6 physiotherapists, 4 psychosocial professionals) from 10 European countries agreed to participate. Four themes were constructed: (1) Defining who is responsible: the challenges in multidisciplinary team provision for good pain management; (2) the concept of pain and its management exists in multiple temporalities; (3) aspirations for better pain management are limited by prevailing biomedicalism; (4) pain management can be better-knowledge and experience improves confidence. DISCUSSION:These data demonstrate a gap in confidence in the delivery of effective pain management, influenced by individual knowledge and skills and wider geographical determinants of healthcare delivery. There is a need for services to establish who is responsible for pain management and better understand professional role identity. Mechanisms to support the implementation of better pain care in haemophilia need to be explored.

Background: Emicizumab prophylaxis is restricted to severe hemophilia A in the UK. Treatment choice and safety remain a matter of debate. Objectives: This study was conducted to investigate factors influencing treatment choice, continued use, safety, and clinical outcomes associated with emicizumab in a national cohort of persons with severe hemophilia A without current inhibitors. Methods: A 3-year study was conducted in 618 persons with severe hemophilia A who switched to emicizumab and 413 who continued factor (F)VIII prophylaxis. Outcome measures included annualized bleed rates (ABRs), the Hemophilia Joint Health Score, and health-related quality of life. Results: Switchers and nonswitchers had a similar median age (26 and 28 years, respectively). Switchers had a significantly higher median (IQR) ABR than those continuing FVIII prophylaxis, but a significantly lower proportion had an inhibitor history (13.6% vs 20.5%; P = .0005). Thirty-one adverse events were reported, including 1 thrombosis (0.2%), 8/84 recurrent inhibitors (9.5%), 1 neutralizing antidrug antibody (0.2%), and 14/618 (2.3%) patients discontinued emicizumab. A higher prestudy median (IQR) ABR was observed in switchers compared with nonswitchers (2.05 [0.43, 6.06] vs 0.68 [0, 2.7]), reducing to a median (IQR) of 0 (0, 0) with emicizumab prophylaxis. The proportion with a zero-treated bleed rate increased from 35% to 71% (P = .001). An 82% reduction in bleeding into target joints was observed in favor of emicizumab. A modest (Δ= −2; P = .02) improvement in the total Hemophilia Joint Health Score was observed. Conclusions: Emicizumab selection was influenced by ABR and inhibitor history but not age. Emicizumab was generally well tolerated, with only 2.3% discontinuing the drug. A significant within-person improvement in all bleeding outcomes was observed with emicizumab.

INTRODUCTION:Currently, physical health assessments in persons with haemophilia focus on bleed-related events and after-effects. The aim of the systematic review was to review and apply standardised criteria to evaluate reliability, responsiveness and construct validity of performance-based instruments evaluating physical capability in persons with haemophilia. METHODS:Medline, CINAHL, Embase, EMCARE, and Cochrane (inception-March 2024) were searched using COSMIN filters for 7 performance-based tests in haemophilia, supplemented by manual searches. Reliability, responsiveness and construct validity of the six-minute walk test (6MWT), timed up and go test (TUG), timed up and down stairs (TUDS), 30-second sit-to-stand (30-STS), single leg stance (SLS), tandem stance (TS) and single hop for distance (SH) were evaluated. RESULTS:The search yielded 88 abstracts; 25 studies remained after full-text screening, covering 5 of 7 performance-based instruments: 6MWT, TUG, TUDS, SLS, and 30-STS. No performance-based test was evaluated for all properties across all ages. Only TUG in adults and older adults and 6MWT in children and adolescents has been tested for all properties. No test received a high grading. Low and very low grades were given mostly for indeterminate results, small or single studies and lack of a similar construct of comparator. The 6MWT in all age groups was the only performance-based test graded moderate, and this was for responsiveness. CONCLUSION:With increasing use of performance-based methods of physical function capacity, evaluating measurement properties is a priority. Until evidence is generated, we can only advocate the 6MWT to monitor responsiveness in adult persons with haemophilia affected with marked arthropathy. SUMMARY:Understanding Physical Health in People with Haemophilia Currently, when we check the physical health of people with haemophilia, we mostly look at problems caused by bleeding. But we wanted to see if there are better ways to measure how well people with haemophilia can move and do daily activities. What We Did We looked through a lot of medical studies (up to March 2024) to find information on 7 specific physical tests. These tests measure things like: How far someone can walk in six minutes (6-minute walk test or 6MWT) How long it takes to stand up, walk a short distance, and sit down (Timed Up and Go test or TUG) How long it takes to go up and down stairs (timed up and down stairs or TUDS) How many times someone can stand up from a chair in 30 seconds (30-second sit-to-stand or 30-STS) How long someone can stand on one leg (single leg stance or SLS) How long someone can stand with one foot directly in front of the other (Tandem Stance or TS) How far someone can hop on one leg (single hop for distance or SH) We wanted to see how reliable (consistent), responsive (can detect changes), and valid (measures what it's supposed to) these tests were for people with haemophilia. What We Found We found 25 studies that looked at 5 of the 7 tests (6MWT, TUG, TUDS, SLS, and 30-STS). Here's what stood out: No single test was good for everything and for all ages. Only the TUG test (for adults) and the 6MWT (for children) had been fully studied for all aspects (reliability, responsiveness, and validity). None of the tests were rated as highly effective overall. Most got low ratings because the results weren't clear, studies were small, or there wasn't enough good information to compare them to. The 6MWT was the only test that received a 'moderate' rating, but only for its ability to show changes in all age groups. What This Means It's becoming more common to use these kinds of physical tests to understand how well people function. So, it's very important to know if these tests work well for people with haemophilia. For now, based on the evidence, we can only suggest using the 6-minute walk test (6MWT) to track how much someone's physical ability changes over time, especially for adults with haemophilia who have severe joint problems. We need more research to find better tests.

Purpose: It is common practice for health professionals to recommend swimming to people with low back pain (LBP). When prescribing swimming, preferences, barriers, and enablers should be considered. Research has found that for people with chronic pain, motivation, fatigue, pain, and comorbidities can be barriers to physical activity engagement whilst cost and suitable provision can be barriers to aquatic-based activities. Healthcare professionals can only address some of these barriers; uptake is also impacted by the pool operators, swimming professionals, and the national swimming bodies. A better understanding of the barriers, enablers, and preferences to swimming by health and swimming professionals and pool operators could improve service delivery, access to swimming, and reduce health inequalities in this population. The aim of this study was to identify the most common swimming barriers, enablers, and preferences for people with chronic LBP and map them onto the COM-B model.

Background and Purpose: It is common practice for health professionals to recommend swimming to people with chronic low back pain (CLBP) for rehabilitation and pain management and to improve levels of physical activity. Uptake and engagement in swimming involve a change in behavior, influenced by various factors including barriers, enablers, and preferences. The aim of this study was to identify the most common barriers, enablers, and preferences to swimming for people with CLBP and map them onto the Capability Opportunity Motivation-Behavior (COM-B) model. Methods: People with CLBP were invited to complete a survey collecting data regarding the barriers, enablers, and preferences in relation to swimming. The questions were scored on a 5-point Likert scale and mapped onto the COM-B model. Results: Eighty-two people completed the survey. More than 50% of participants agreed with the following barriers: I am not sure which swimming stroke is best for my back pain and I lack motivation to go swimming. These barriers mapped to the psychological capability and reflective motivation dimensions on the COM-B model. At least 50% of participants agreed with the following enablers: I would like to use swimming to improve my strength, flexibility, weight, fitness, health, mood, and well-being; setting goals and making an action plan could help me swim regularly, I am more likely to go swimming if a health professional advised me and I believe that swimming is good for my back. These enablers were also mapped to the reflective motivation and physical and social opportunity dimensions on the COM-B model. Discussion and Conclusions: The findings from this survey suggest that when recommending swimming, health professionals should discuss the wider health benefits of swimming, discuss specific swimming strokes and why swimming could be beneficial for back pain, and consider using motivational tools to encourage reflective motivation such as goal setting and action planning.

Chronic pain is reported by between 30 and 71

Purpose: Swimming is often advised by health professionals to people with low back pain (LBP) despite limited research and low levels of swimming ability in a proportion of the adult population. Unlike other forms of exercise recommended for LBP, swimming is a more complex skill to learn and master. There are several learn-to-swim frameworks and swimming manuals which aim to develop water confidence, aquatic skills, swimming strokes, and technique, however, they have not been evaluated systematically. There are currently no frameworks or guidelines for using swimming as a rehabilitation modality. The aim of the study was to use a modified Delphi technique to develop guidance for implementation of a swimming programme for people with LBP.

AIM:For people with haemophilia A (PwHA), bleeding in the joints leads to joint damage and haemophilia-related arthropathy, impacting range of motion and life expectancy. Existing guidelines for managing haemophilia A support healthcare professionals (HCPs) and PwHA in their efforts to preserve joint health. However, such guidance should be reviewed, considering emerging evidence and consensus as presented in this manuscript.METHODS:Fifteen HCPs experienced in the management of PwHA in the UK participated in a three-round Delphi panel. Consensus was defined at ≥70% of panellists agreeing or disagreeing for Likert-scale questions, and ≥70% selecting the same option for multiple- or single-choice questions. Questions not reaching consensus were revised for the next round.RESULTS:26.8% (11/41), 44.8% (13/29) and 93.3% (14/15) of statements reached consensus in Rounds 1, 2 and 3, respectively. HCPs agreed that prophylaxis should be offered to patients with a baseline factor VIII (FVIII) level of ≤5 IU/dL and that, where there is no treatment burden, the aim of prophylaxis should be to achieve a trough FVIII level ≥15 IU/dL and maintain a longer period with FVIII levels of ≥20-30 IU/dL to provide better bleed protection. The aspirational goal for PwHA is to prevent all joint bleeds, which may be achieved by maintaining normalised (50-150 IU/dL) FVIII levels.CONCLUSION:The panel of experts were largely aligned on approaches to preserving joint health in PwHA, and this consensus may help guide HCPs.

INTRODUCTION:As a result of centralisation of haemophilia care to a limited number of intramural settings, many persons with haemophilia have to travel long distances to attend their haemophilia specialised treatment centre. However, regular physiotherapy treatment can be provided by primary care physiotherapists in the person's own region. Due to the rarity of the disease most primary care physiotherapists have limited experience with this population. This study aims to provide a clinical practice guideline for primary care physiotherapists working with persons with bleeding disorders. METHOD:A list of the most urgent key-questions was derived from a previous study. Literature was summarised using the grading of recommendations assessment, development, and evaluation (GRADE) evidence-to-decision framework. Recommendations were drafted based on four 90 min consensus meetings with expert physiotherapists. Recommendations were finalised after feedback and >80% consensus of all stakeholders (including PWH, physiotherapists, haematologists and the corresponding societies). RESULTS:A list of 82 recommendations was formulated to support primary care physiotherapists when treating a person with a bleeding disorder. These recommendations could be divided into 13 categories: two including recommendations on organisation of care, six on therapy for adult patients with bleeding disorders and five on therapy adaptations for paediatric care. Therapy recommendations included treatment after a joint- or muscle bleed, haemophilic arthropathy, chronic synovitis, non-haemophilia related conditions and orthopaedic surgery. CONCLUSION:An evidence-based practice guideline, based on current evidence from literature and clinical expertise, has been developed for primary care physiotherapists treating a person with haemophilia. To improve care, the recommendations should be implemented in daily practice.

INTRODUCTION:Due to advances in treatments, people with haemophilia (PWH) are living longer. They are not as active as the general population due to joint damage and lack confidence to be active due to concerns about further bleeds and pain. There is a need to facilitate healthy aging through promotion of physical activity (PA) and exercise. Changing patient beliefs and increasing physical literacy and confidence to move are thought to be key to helping PWH become more active.AIM:This paper describes the development of an exercise and behaviour change intervention to improve confidence to exercise in PWH.METHODS:The 4-stage Medical Research Council framework for complex intervention development was used.RESULTS:Stakeholders included 17 PWH and 7 physiotherapists working in haemophilia. Seven online focus group meetings were held. The final intervention is a hybrid 12-week physiotherapist led progressive exercise programme. Classes are 45 min including Pilates, High intensity interval training and balance elements, together with discussion sessions focusing on PA recommendations, the types and benefits of different exercise styles and the effects of PA, together with the effects of aging for PWH. The COM-B model of behaviour change was used to develop the intervention.CONCLUSION:Co-design helps to produce an intervention that understands the stakeholders needs. Through this process the intervention developed to incorporate not only increasing PA but also confidence to exercise. The use of behaviour change theory identified the behaviour techniques included in the intervention and aims to increase physical literacy in this population.