A clinical practice guideline for primary care physiotherapy in patients with haemophilia.

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.

Joint health and pain in the changing hemophilia treatment landscape.

INTRODUCTION: Hemophilia is an inherited bleeding disorder. Bleeding, and in particular joint hemorrhage results in chronic arthropathy and disability. Acute and chronic pain are frequent and limit activity and participation and result in decreased health-related quality of life. Remarkable progress has been made in the diagnosis and treatment of hemophilia but bleeding continues to prove recalcitrant to currently available treatments and joint disease remains problematic. Physiotherapy and pain management are mainstays of current multidisciplinary integrated care of people with hemophilia (PWH). The focus of this review is on preservation of joint health in the era of new and innovative therapies. AREAS COVERED: A search of the PubMed Central was conducted on 1 February 2024 using the MeSH Major Topic terms identified as keywords for the manuscript. This review will highlight what is known and unknown about joint bleeding and arthropathy, including insights on pain as a related complication. EXPERT OPINION: Recent advances in therapeutic interventions aimed at promoting healthy joints in PWH will be discussed, including both the pharmacological treatment landscape and related strategies to promote joint health.

The Effects of Wastewater Reuse on Smallmouth Bass (Micropterus dolomieu) Relative Abundance in the Shenandoah River Watershed, USA.

Municipal and industrial wastewater effluent is an important source of water for lotic systems, especially during periods of low flow. The accumulated wastewater effluent flows-expressed as a percentage of total streamflow (ACCWW%)-contain chemical mixtures that pose a risk to aquatic life; fish may be particularly vulnerable when chronically exposed. Although there has been considerable focus on individual-level effects of exposure to chemical mixtures found in wastewater effluent, scaling up to population-level effects remains a challenging component needed to better understand the potential consequences of exposure in wild populations. This may be particularly important under a changing climate in which wastewater reuse could be essential to maintain river flows. We evaluated the effects of chronic exposure to wastewater effluent, as measured by ACCWW%, on the relative abundance of young-of-year (YOY), juvenile, and adult smallmouth bass (Micropterus dolomieu) populations in the Shenandoah River Watershed (USA). We found that increases in ACCWW% in the previous year and during the prespawn period were negatively correlated with the relative abundance of YOY, resulting in an average 41% predicted decrease in abundance (range = 0.5%-94% predicted decrease in abundance). This lagged effect suggests that adult fish reproductive performance may be compromised by chemical exposure during periods of high ACCWW%. No relationships between ACCWW% and juvenile or adult relative abundance were found, suggesting that negative effects of ACCWW% on YOY abundance may be offset due to compensatory mechanisms following higher ACCWW% exposure. Understanding the effects of wastewater effluent exposure at multiple levels of biological organization will help in the development of management strategies aimed at protecting aquatic life. Environ Toxicol Chem 2024;43:1138-1148. © 2024 The Authors. Environmental Toxicology and Chemistry published by Wiley Periodicals LLC on behalf of SETAC. This article has been contributed to by U.S. Government employees and their work is in the public domain in the USA.

Expert United Kingdom consensus on the preservation of joint health in people with moderate and severe haemophilia A: A modified Delphi panel.

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.

Using theory of change to co-create a programme theory for a telerehabilitation intervention for pain management in people with haemophilia.

BACKGROUND: Improved approaches for chronic pain management are a clinical and research priority for people with haemophilia (PWH). Involving people with lived experience in the design of a complex rehabilitation intervention strengthens the credibility and plausibility of the intervention, particularly in relation to rare disorders. Here we describe using a 'Theory of Change' (ToC) dialogue-based stakeholder process to create a programme theory for a telerehabilitation intervention. METHODS: An online workshop was convened and stakeholders received a briefing document in advance. Five stakeholders took part (3 PWH and 2 physiotherapists). At the workshop the group first agreed the overall aim of the intervention. Discussions then identified the resources, activities, barriers and enablers needed to achieve this outcome. All discussions were recorded and annotated by the workshop moderator. Behaviour change techniques were mapped for inclusion in the theory. RESULTS: A programme theory and narrative report were produced. All stakeholders reviewed these for clarity and to ensure a true reflection of the workshop discussions. Agreement was based on how meaningful, well-defined, do-able, plausible, credible, and testable each component was. Stakeholders highlighted the importance of issues unique to PWH. Key components included the need for physiotherapists to be knowledgeable of the condition, a range of exercises that were inclusive of all abilities, and the need for people to feel safe and supported whilst taking part. CONCLUSIONS: Co-developed theory based approaches to intervention design offer an inclusive and transparent way to develop novel and meaningful interventions for people with complex health conditions. The ToC is wholly transparent in its design and content. Together with the identified behaviour change techniques, the theory informs the protocol for a feasibility study evaluating a telerehabilitation intervention. Importantly, it allows the opportunity to revise, adapt and improve the programme theory for further implementation and evaluation.

Prevalence and perceptions of pain in people with haemophilia: A UK study.

INTRODUCTION: Joint bleeds in haemophilia cause destruction of articular structures, impaired function and pain. Up to 70% of people with haemophilia (PWH) report chronic pain. Little is known about the pain experiences in PWH in the UK. AIM: To identify prevalence and perceptions of pain among PWH living in the UK. METHODS: A cross-sectional, non-interventional survey study conducted among PWH (all severities). The survey incorporated elements from validated tools (EQ-5D; EQ-VAS) and was distributed via participating treatment centres. RESULTS: Five hundred and ninety-nine PWH responded, 91% aged > 18. 81% used factor prophylactically or on demand. More pain was reported by those treated on demand versus prophylaxis particularly in those who reported daily pain. 65% reported 'problem joints' based on individual impact rather than medically defined 'target joints', 2/3 reported multiple joint issues. The ankle was most commonly affected. 59% reported frequent pain, with 56% aware of pain constantly or most of the time and were more likely to report less favourable EQ-5D or EQ-VAS scores (p < .001). Pain frequency/awareness was consistent across all severities. Most discussed pain with care teams, 31% only when asked; 25% did not discuss it. Pain discussions resulted in physiotherapy referral (63%) analgesia prescription (48%), and a minority specialist pain referral (9%). Most felt well supported with regard to their pain, but 70% reported learning to live with it. CONCLUSION: Pain affects PWH of all ages and severities even in a well-resourced country significantly impacting quality of life. Clinicians must be more aware of chronic pain in PWH. Biopsychosocial approaches to pain assessment and management are recommended.

Humanistic burden of problem joints for children and adults with haemophilia.

INTRODUCTION: The "problem joint" (PJ) concept was developed to address patient-centric needs for a more holistic assessment of joint morbidity for people with haemophilia (PwH). AIM: To quantify the humanistic burden of PJs in PwH to further support validation of the PJ outcome measure. METHODS: Multivariable regression models evaluated the relationship between PJs and health-related quality of life (HRQoL, EQ-5D-5L) and overall work productivity loss (WPL) using data from the 'Cost of HaEmophilia: a Socioeconomic Survey' population studies (adults: CHESS II, CHESS US+; children/adolescents: CHESS-Paeds). Covariates included were haemophilia severity, age, comorbidities and education. RESULTS: The CHESS II sample included 292 and 134 PwH for HRQoL and WPL analyses, mean age 38.6 years (39% ≥1 PJ, 61% none). CHESS US+ included 345 and 239 PwH for HRQoL and WPL, mean age 35 years (43% ≥1 PJ, 57% none). CHESS-Paeds included 198 PwH aged 4-17 (HRQoL only), mean age 11.5 years (19% ≥1 PJ, 81% none). In CHESS II and CHESS US+, presence of PJs was associated with worse HRQoL (Both p < .001). Few CHESS-Paeds participants had PJs, with no significant correlation with HRQoL. In CHESS II, upper body PJs were significantly correlated to WPL (p < .05). In CHESS US+, having ≥1 PJ or upper and lower body PJs were significantly correlated to WPL (vs. none; both p < .05). CONCLUSION: This study has shown a meaningful burden of PJs on PwH, which should be considered in clinical and health policy assessments of joint health.

Promoting physical activity for people with haemophilia in the age of new treatments.

Haemophilia treatment has seen great advances in recent years with an accompanied reduced risk of physical activity (PA) related bleeds. Based on its known health benefits, people with haemophilia (PWH) are currently encouraged to regularly engage in PA. However, this may not always translate to increased levels of PA. In this narrative review we aim to provide a brief overview of what is currently understood regarding PA levels and influences for the three broad age groups of children and adolescents, adults and older adults. We also provide recommendations for members of the haemophilia team on important aspects related to promotion of PA in their clinical practice. We highlight that PA behaviour is multifactorial and that many PWH still have limited access to adequate care. Whilst some still face unique challenges to being more physically active, overall, the barriers and facilitators to activity are very similar to that of the general population.

The Hemophilia Joint Health Score version 2.1 Validation in Adult Patients Study: A multicenter international study.

Background: The Hemophilia Joint Health Score (HJHS) was developed and validated to detect arthropathy in children. Additional evidence is required to show validity in adults. We studied the convergent and discriminant construct validity of the HJHS version 2.1(HJHSv2.1) in adults with hemophilia. A secondary aim was to define age-related normative adult HJHSv2.1 reference values. Methods: We studied 192 adults with hemophilia, and 120 healthy adults in four age-matched groups-18 to 29, 30 to 40, 41 to 50, and >50 years-at nine centers. Trained physiotherapists scored the HJHS and World Federation of Hemophilia (WFH) joint score. Health history, the Functional Independence Scale of Hemophilia (FISH), Hemophilia Activities List (HAL), and Short-Form McGill Pain Questionnaire (SF-MPQ) were also collected. Results: The median age was 35.0 years. Of participants with hemophilia, 68% had severe, 14% moderate, and 18% mild disease. The HJHS correlated strongly with WFH score (Spearman's rho [rs ] = .95, P < .001). Moderate correlations were seen between the FISH (rs  = .50, P < .001) and SF-MPQ Present Pain Intensity (rs  = .50, P < .001), while a modest correlation was found with the HAL (rs  = -.37, P < .001). The HJHS significantly differentiated between age groups (Kruskal-Wallis T = 35.02, P < .001) and disease severity in participants with hemophilia. The HJHS had high internal reliability (Cronbach's α = .88). We identified duration of swelling as a redundant item in the HJHS. Conclusions: The HJHS shows evidence of strong convergent and discriminant construct validity to detect arthropathy in adults with hemophilia and is well suited for use in this population.

The importance of casting an eye beyond the dental arch: A case report.

Study models are frequently used in orthodontics and are a key part of both initial assessment and treatment planning. They are an important part of comprehensive orthodontic records; furthermore, study models are an essential diagnostic aid that can be studied in detail without the patient being present. We present a case of a 14-year-old boy with an abnormal palatal swelling detected by his specialist orthodontist after review of study models taken at the initial appointment. At the assessment alone, the significance of the abnormality was not recognised and without the help of study models, diagnosis and treatment of his fibrous dysplasia may have been significantly delayed.

The statistical power to detect regional temporal trends in riverine contaminants in the Chesapeake Bay Watershed, USA.

Chemical contamination of riverine ecosystems is largely a result of urbanization, industrialization, and agricultural activities occurring on adjacent terrestrial landscapes. Land management activities (e.g., Best Management Practices) are an important tool used to reduce point and non-point sources of pollution. However, the ability to confidently make inferences about the efficacy of land management activities on reducing in-stream chemical concentrations is poorly understood. We estimated regional temporal trends and components of variation for commonly used herbicides (atrazine and metolachlor), total estrogenicity, and riverine sediment concentrations of total PCBs for rivers in the Chesapeake Bay Watershed, USA. We then used the estimated variance components to perform a power analysis and evaluated the statistical power to detect regional temporal trends under different monitoring scenarios. Scenarios included varying the magnitude of the annual contaminant decline, the number of sites sampled each year, the number of years sampled, and sampling frequency. Monitoring for short time periods (e.g., 5 years) was inadequate for detecting regional temporal trends, regardless of the number of sites sampled or the magnitude of the annual declines. Even when monitoring over a 20-year period, sampling a relatively large number of sites each year was required (e.g., >50 sites) to achieve adequate statistical power for smaller trend magnitudes (declines of 5-7%/year). Annual sampling frequency had little impact on power for any monitoring scenario. All sampling scenarios were underpowered for sediment total PCBs. Power was greatest for total estrogenicity, suggesting that this aggregate measure of estrogenic activity may be a useful indicator. This study provides information that can be used to help (1) guide the development of monitoring programs aimed at detecting regional declines in riverine chemical contaminant concentrations in response to land management actions, and (2) set expectations for the ability to detect changes over time.

Project GYM: A randomized feasibility study investigating effect on motivation of personal trainer-led exercise in young men with hemophilia.

INTRODUCTION: Many young men with hemophilia engage in physical activity and sport but face challenges to participation because of their hemophilia. Project GYM aimed to investigate the feasibility of a hemophilia-specific fitness program led by a personal trainer (PT) and its impact on gym activity, motivation, and adherence to exercise. METHODS: This was a nonblinded, randomized feasibility study, recruiting participants aged 18 to 25 years with hemophilia A or B (all severities, ± inhibitor) from three London hemophilia centers. All participants were given an activity tracker and free gym membership. Participants were randomized to a "gym only" or "gym and PT" arm. Participants completed questionnaires evaluating motivation to exercise, quality of life, physical activity levels, self-efficacy, and self-esteem at study start and study end. RESULTS: Of 142 eligible individuals, 19 agreed to participate. Participants were healthy, with mean body mass index and adiposity slightly lower than the UK average. They reported low bleed numbers and had good joint health (median Hemophilia Joint Health Score [HJHS], 0; range, 0-13). The gym and PT group had more gym attendance than the gym-only group. Seven participants increased their activity levels and nine stayed the same, with no statistical difference between groups. HJHS scores improved in 3 participants and were unchanged in 12. There was no bleeding associated with gym activity. CONCLUSION: Project GYM has demonstrated the safety and feasibility of a tailored physical training program in young men with hemophilia. Increased gym attendance, with and without support from a PT, is associated with increased physical activity.

Fitness enhances psychosocial well-being and self-confidence in young men with hemophilia: Results from Project GYM.

INTRODUCTION: Contemporary hemophilia care supports physical activity, its benefits being well recognized. Despite recognition of the psychological challenges encountered by people with hemophilia, little is known about the psychological impact of physical fitness in this population. AIM: To identify changes in psychological well-being in young men with hemophilia through participation in a gym program. METHODS: This observational feasibility study of a 6-month gym participation program used validated questionnaires pre- and poststudy to evaluate motivation to exercise, physical activity levels, self-efficacy, self-esteem, and quality of life. Individual audio-recorded interviews about study participation and impact were transcribed verbatim and analyzed for recurring themes using thematic analysis. RESULTS: Nineteen participants aged 18-25 years with hemophilia A or B (all severities ± inhibitor) consented to the study; two were lost to follow-up. There was a shift in motivation to exercise as shown by the Stages of Change grouping moving from contemplation to action and maintenance phases (p = 0.03). Self-efficacy overall scores showed a trend (p < 0.06) towards improvement. Median self-esteem scores improved from 22 (range 12-30, n = 19) to 25 (range 13-30, n = 17), a statistically significant change (p = 0.02). Three participants recorded scores below the accepted normal range before study, of whom two improved at study end. The key themes identified from the interviews were: fear, self-confidence, "being normal," pain, weight loss, ability, getting fitter. CONCLUSIONS: The psychological wellbeing of young men with hemophilia improved during this study. This may have been related to participating in a gym-based, physical exercise program.

The Role of Physiotherapy in the New Treatment Landscape for Haemophilia.

The physiotherapist plays an essential role for people with haemophilia, an inherited bleeding disease responsible for musculoskeletal complications. Yet, with the advent of new and advanced therapies, the medical landscape is changing, and physiotherapy must adapt alongside. This paper considers whether there will still be a need for physiotherapy in the era of advanced therapies, and discusses ways in which services should evolve to complement emerging treatment paradigms for haemostasis in people with haemophilia. Ultimately, physiotherapy will remain an important element of care, even for people with little joint damage and low risks in the era of the new mild phenotype. However, competencies will need to evolve, and physiotherapists in both primary care and specialist treatment centres should work with haematology colleagues to develop more sensitive tools for detecting early joint changes. Physiotherapists will also play a crucial role in counselling and physically coaching, monitoring the musculoskeletal status of people with haemophilia who have transitioned to new treatments.

Physiotherapy interventions for pain management in haemophilia: A systematic review.

PURPOSE: Approximately 35%-50% of people with haemophilia (PWH) report living with chronic musculoskeletal pain. Although exercise based rehabilitation is effective for pain in other arthritises, there are no published guidelines for management of chronic pain in PWH. This review aims to evaluate and appraise the current evidence of effectiveness of physiotherapy interventions on (a) pain intensity, (b) quality of life (QoL) and (c) function in PWH. METHODS: A systematic review of five databases AMED and CINAHL, EMBASE and MEDLINE and PEDro, as well as trial registries, grey literature and hand searching key journals was completed. Included studies were critically appraised and evaluated for risk of bias. The GRADE approach was used to rate the quality of the evidence. RESULTS: Nine trials consisting of 235 participants met the inclusion criteria. All studies had an overall risk of bias with low methodological quality. Meta-analysis was not possible due to heterogeneity across trials. Studies comparing a range of physiotherapy interventions against no intervention showed no clear beneficial effect on pain intensity or QoL. Only one study, investigating hydrotherapy or land-based exercise against control, showed positive effect for pain intensity, but rated very low on GRADE assessment. Studies comparing one physiotherapy intervention against another showed no clear benefit on pain intensity, QoL or function. LASER with exercise and hydrotherapy were shown to have some positive effects on pain intensity, but no clear benefit on function. CONCLUSIONS: At present, there is limited evidence for the use of physiotherapy interventions in addressing the issue of pain in PWH. Better designed trials with higher quality and explicit methodology along with user involvement are needed to assess the efficacy of any proposed intervention.

Scope of practice of haemophilia physiotherapists: A European survey.

INTRODUCTION: European guidelines on the care of haemophilia recommend ready access to a range of services provided by a multidisciplinary team of specialists including physiotherapy. However, the scope of physiotherapy provided is unknown. METHODS: The Physiotherapists Committee of the European Association for Haemophilia and Allied Disorders (EAHAD) conducted a web-based survey to quantify the role and scope of practice of physiotherapists involved in haemophilia care. The survey was sent to more than 200 physiotherapists registered on the EAHAD database. Questions concerned their work practices including assessment and treatment activities and level of autonomy. RESULTS: Eighty physiotherapists from twenty-four European countries responded. Considerable heterogeneity exists in roles, responsibilities, and clinical practice of physiotherapists, particularly in access to and type of physiotherapy treatment provided, as well as the skill set and autonomy of physiotherapists to make independent assessment and treatment decisions. DISCUSSION: This pan-European survey establishes a context to support physiotherapy role development and professional identity. Key recommendations include the following: (a) establishing a pan-European network to support collaboration and education for physiotherapists working in haemophilia, (b) developing a core skills and capability framework to ensure person-centred approaches are central as well as working in partnership with those with the condition to maximize early recovery, support self-management and enablement in remaining active and independent, (c) regular training, standardized validation and maintenance of competency for assessment tools, (d) well-designed randomized clinical studies with larger numbers of participants from multiple sites should be the focus of future research.

Investigating the relationship between the HJHS and HAL in routine clinical practice: A retrospective review.

INTRODUCTION: Comprehensive musculoskeletal assessment for monitoring joint health in haemophilia includes both physical assessment with Haemophilia Joint Health Score (HJHS) and assessment of self-reported function by Haemophilia Activities List (HAL). METHODS: Correlation between physical assessment and joint function was undertaken between HJHS and HAL in patients with SHA and SHB who had both assessments at the same visit over a one-year period. RESULTS: Data from 120 patients (96-SHA/24 = SHB) with a median age 33 years (range 19-73) were included. Median total HJHS was 19, increasing with age: 18-30 years-7, 31-50 years-25 and 51-73 years-44. Similarly, median total HAL score was 80 with decreased function associated with increasing age: 18-30 years-90.4, 31-59 years-71.7, 51-73 years-49.5. Median Total HJHS and HAL demonstrated strong correlation (rs  = 0.66, P < 0.01). Moderate-to-strong correlations were seen across the entire age group between the HJHS LL and UL subtotals and corresponding limb HAL domains. Within age groups, correlations were less significant particularly for the upper UL domains in HAL and the UL HJHS score. The wide range of ROM in joints categorized as markedly affected (ie, ROM loss score = 3) highlights the potential ceiling effect of this domain score and its use in chronically damaged joints. CONCLUSION: HJHS and HAL showed moderate-to-strong correlation with discrepancy in some individual patients. Prospective studies are required to better understand the clinical utility of both especially in severe joint disease where HAL may have a potential advantage.