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Introduction: Considering the advances in haemophilia management and treatment observed in the last decades, a new set of value‐based outcome indicators is needed to assess the quality of care and the impact of these medical innovations. Aim: The Value‐Based Healthcare in Haemophilia project aimed to define a set of clinical outcome indicators (COIs) and patient‐reported outcome indicators (PROIs) to assess quality of care in haemophilia in high‐income countries with a value‐based approach to inform and guide the decision‐making process. Methods: A Value‐based healthcare approach based on the available literature, current guidelines and the involvement of a multidisciplinary group of experts was applied to generate a set of indicators to assess the quality of care of haemophilia. Results: A final list of three COIs and five PROIs was created and validated. The identified COIs focus on two domains: musculoskeletal health and function, and safety. The identified PROIs cover five domains: bleeding frequency, pain, mobility and physical activities, Health‐Related Quality of Life and satisfaction. Finally, two composite outcomes, one based on COIs, and one based on PROIs, were proposed as synthetic outcome indicators of quality of care. Conclusion: The presented standard set of health outcome indicators provides the basis for harmonised longitudinal and cross‐sectional monitoring and comparison. The implementation of this value‐based approach would enable a more robust assessment of quality of care in haemophilia, within a framework of continuous treatment improvements with potential added value for patients. Moreover, proposed COIs and PROIs should be reviewed and updated routinely. [ABSTRACT FROM AUTHOR]

Introduction: Haemophilia is a rare congenital bleeding disorder, and the most common manifestation is spontaneous bleeding in muscles and joints. Despite the benefits linked to recent and dramatic pharmacological advances at least in high income setting, many patients still develop musculoskeletal dysfunctions during their lifetime, which must be managed by physiotherapists in the frame of a multidisciplinary team. The aim of the scoping review is to map the available evidence by providing an overview on the past and present physiotherapy scenario in persons with haemophilia (PWH). Materials and methods: The review was conducted according to the guidelines of the PRISMA extension for scoping reviews. Scientific articles on physiotherapy and sport interventions for PWH published from 1960 up to September 2021 have been included. Search was conducted on the e‐databases PubMed and PEDro without restrictions for the study design. Results: Sixty eight articles were included, 52 related to rehabilitation and preventive physiotherapy, 16 to sport. The results have been reported in chronological order and divided into two categories: (1) rehabilitation and preventive physiotherapy; (2) sport activities. Conclusions: This is the first scoping review on physiotherapy in haemophilia, based on the existing evidence on this topic which allowed us to underline how the role of the physiotherapist changed over time. Historically this specialist did intervene only after an acute bleed or surgical operation, but now he has a pivotal role in the multidisciplinary team that acts to improve from birth the quality of life of the PWH. His activity is also closely intertwined with sport promotion and supervision. [ABSTRACT FROM AUTHOR]

Summary: Although synovitis is recognized as a marker of joint disease activity, its periodic assessment is not included in routine clinical surveillance of patients with haemophilia (PwH). In order to evaluate the current knowledge and to identify controversial issues, a preliminary literature search by the Musculoskeletal Committee of the Italian Association of Haemophilia Centres (AICE) has been conducted. Statements have been established and sent to the Italian AICE members to collect their level of agreement or disagreement by a Delphi process. Thirty‐seven consensus recommendations have been drafted. We found a general agreement on the indication to consider the presence of synovitis as a marker of joint disease activity in PwH. Accordingly, there was agreement on the indication to search for synovitis both in patients reporting joint pain and in asymptomatic ones, recognizing ultrasound as the most practical imaging technique to perform periodic joint screening. Interestingly, after detection of synovitis, there was agreement on the indication to modify the therapeutic approach, suggesting prophylaxis in patients treated on demand and tailoring treatment in patients already under prophylaxis. Whereas the need of an early consultation with a physiotherapist is recommended for PwH affected by chronic synovitis, the exact timing for an orthopaedic surgeon consultation is currently unknown. [ABSTRACT FROM AUTHOR]

In persons with haemophilia (PWH), rehabilitation programmes based upon physical exercise must be implemented as adjuvants to factor replacement or non-factor therapies,1,2 with the goal to improve proprioception, balance, resilience and overall the quality of life.3,4 Exercises should be simple enough to attain adherence but also enjoyable enough to avoid boredom, frustration and fatigue, that inevitably lead PWH to give them up. With this background and unmet needs, we chose to evaluate the feasibility and acceptability but also the preliminary efficacy of an exercise-based rehabilitation programme engaging at home young PWH. This programme, newly based upon the use of exercise videogames (exergames) specifically designed for telerehabilitation,5-8 was conceived to make physical activity amusing and appealing, but at the same time to allow physiotherapists to monitor and supervise at distance if and how PWH exercised. [Extracted from the article]

In persons with haemophilia (PWH), the importance of comprehensive disease management to prevent bleeding, joint damage and secondary diseases has been well established. However, because haemophilia is a chronic disease, intervention programmes carried out for prolonged periods of time may create problems of patient adherence. Driven by continuous technological innovation, telemedicine is being increasingly proposed as a way to provide PWH with a range of services designed to improve their health, saving the time and cost involved in going to the treatment centre, and increasing therapeutic adherence. The aim of this article is to identify and discuss the tools available for the management of PWH by means of telemedicine and information technology. Video conferences are helpful to obtain a rapid evaluation at a distance of the occurrence and severity of bleeding episodes by the personnel of the treatment centre. Cell phones and associated applications (apps) help to improve the regular implementation of replacement therapy and monitor any ensuing adverse effect. Portable sensors help to improve lifestyle and to monitor the degree of physical activity through the fulfilment of a given number of daily walking steps and other physical activities. In the context of telerehabilitation, exergames have the potential to improve the musculoskeletal function of PWH by exploiting the recreational features of videogames. Thus, telemedicine and its multiple applications may be useful in the management of haemophilia, especially for patients living far from specialised centres. However, since this is a recent and rapidly evolving field, published studies are few and have, so far, involved only a limited number of cases. Therefore, additional evidence needs to be obtained by means of accrual of cumulative data from multiple centres specialised in haemophilia.