AHP Oral Abstracts
NU01 Training of nursing staff from non-haematology units in the reconstitution and administration of coagulation factors for haemophilia treatment
L. Moralejo Cubero*; Y. Echarte Buil, C. Campoy Martinez
Hospital Universitario Miguel Servet, Zaragoza, Spain
Introduction: The increase in life expectancy among individuals with haemophilia in recent years has led to a rise in age-related comorbidities. Alongside the emergence of new treatments and their more frequent use in various clinical scenarios such as surgeries, invasive diagnostic procedures, and on-demand treatment for bleeding episodes, there is a need to train nursing staff from units other than haematology in the handling, preparation and administration of haemostatic treatment.
Methods: An educational workshop was conducted with eight nurses from different departments, divided into two parts:
A theoretical presentation using a PowerPoint to introduce haemostasis, haemophilia, types of treatment and various treatment formulations.
After explaining the process of reconstituting coagulation factors, the students practiced using demo-kits provided by laboratories.
At the end, participants were given a 10-question test to assess their understanding of the workshop's content, which was later reviewed, discussed and any questions were resolved.
This workshop is repeated twice a year.
Results: Improved knowledge among nurses regarding haemophilia and the reconstitution and administration of coagulation factors.
Increased self-confidence among these nurses, enhancing their self-assurance.
Enhanced quality of care for patients with haemophilia.
Discussion/Conclusion: Adequate training of nursing staff leads to improved quality and safety of care provided to patients with haemophilia.
Greater satisfaction among nurses due to their proficient execution of this technique and overcoming the fear associated with it.
Disclosure of Interest: None declared.
NU02 Unicentric analysis of the electronic record of health data reported by patients in clinical trials in coagulopathies
S. García-Barcenilla1,*; M. T. Álvarez-Román1,2; S. Rochas-López1; M. Martín-Salces1; I. Rivas-Pollmar1; E. Monzón-Manzano1; E. García Arias-Salgado1; P. Acuña-Butta1; N. Butta-Coll1; L. Rodríguez-González1; V. Jiménez-Yuste1,2
1Thrombosis and Hemostasis Unit, Hospital Universitario La Paz—IdiPAZ; 2Medicine department, Autonomous University of Madrid, Madrid, Spain
Introduction: Recording patient-reported health data on electronic devices and mobile apps is an indirect method to measure concordance with treatment. Real-time availability of ambulatory reported data is essential, especially in the context of investigational molecules. However, certain barriers hinder adequate data collection, leading to incomplete information. The aim was to determine the level of concordance between the prescribed treatment and the electronic medical records.
Methods: Data were collected from different platforms used by 28 patients included in trials in our centre in May 2023. The variables selected were age, type of treatment, who entered the data and whether a mobile app or PDA-type electronic device was used. We also analysed the percentage of correspondence of the data entered with the prescribed treatment. We reviewed the clinical history to detect different types of incidents in the inclusion of information.
Results: Half of the 28 patients used an app, while the other 50% used a PDA. 21 patients (75%) self-reported, and 25% were reported by the caregiver. Caregivers also reported data for paediatric patients < 14 years of age. Regarding the correspondence, 71.4% (n = 20) reported 100%. 17.8% (n = 5) had 0%, and 10.7% (n = 3) had 50%−63%. All caregivers in the study (n = 7) had a 100%. Of the patients who had 100% match, 25% (5) had problems with data recording; of the patients who had 50%−63%, only one. All patients with 0% had problems with data collection like failures in data transmission or forgotten passwords among others. 71.4% of the sample recorded more than 80% of the doses administered in the last month, compared to 20% who did not report them.
Discussion/Conclusion: Problems arising from the electronic health data recording system itself have a considerable impact on the continuity and quality of record entry. In addition, they can lead to a loss of interest in data collection, decreasing commitment to recording important health data. Therefore, missing data is not only the responsibility of the person reporting the data. Electronic data recording has several important advantages, among others, real-time monitoring, specially important with an investigational drug. However, there are certain barriers to the use of these devices in daily practice that, if overcome, would improve the data collection. Experience for all parties involved.
Disclosure of Interest: S. García-Barcenilla Grant/Research support from: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche, Consultant for: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche, Speaker Bureau of: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche, M. T. Álvarez-Román Grant/Research support from: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche Novartis, Speaker Bureau of: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche Novartis, S. Rochas-López: None declared, M. Martín-Salces: None declared, I. Rivas-Pollmar: None declared, E. Monzón-Manzano: None declared, E. García Arias-Salgado: None declared, P. Acuña-Butta: None declared, N. Butta-Coll: None declared, L. Rodríguez-González: None declared, V. Jiménez-Yuste Grant/Research support from: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche Novartis, Speaker Bureau of: NovoNordisk Biomarin CSL Behring Bayer Takeda Sobi Roche Novartis.
NU03 Adult transition program led by clinical nurse specialist: The case of congenital coagulopathies
C. Benedicto1,2,*; N. Caballero1,2; M. Rambla2,3; R. Macho2,3; C. Burriel2,3; R. Berrueco1,2; N. Vilalta2,3; M. Carrasco2,3; J. Mateo2,3; M. Triquell2,3
1Pediatric Hematology Department, Hospital Sant Joan de Déu; 2CSUR Coagulopaties, EuroBloodNet Member, Unitat Integrada Hemofília SJD-HSP; 3Thrombosis and Hemostasis Unit, Hemaology Department, Hospital de la Santa Creu i Sant Pau, Barcelona, Spain
Introduction: Life expectancy in patients with Congenital Coagulopathies (CC) has increased during last decades. Patients face new challenges that make mandatory the existence of expert CC units including specialists in paediatric and adult diseases apart from haemophilia. Clinical nurses’ specialist (CNSs) plays a key role for patient management, including the transition process
Aim: To describe the experience of an Integrated Unit (composed by a paediatric and an adult hospital).
Methods: Descriptive analysis of the experience in the transition program from paediatric to adult care during last 4 years. A transition model composed with two transversal programs was used: “A10” program (“goodbye” in Catalan) in the Paediatric Unit (PU) and “Hola!” program (“hello” in Catalan) in the Adult Unit (AU). Both follow the same methodology and have common objectives: to support young patients and their families during adulthood transition; to empower adolescents to self-manage their disease involving the patient in the health care process; and to offer a follow up. This process starts at the age of 14 years, when patient information is shared in a multidisciplinary session, and finishes in the first visit at AU.
The experience of the CNSs was evaluated using an ad-hoc questionnaire carried out by the three CNSs of both centres.
Results: From 2019 to 2022 a total of 25 patients with CC completed transition: eight with severe haemorrhagic phenotype and prophylactic treatment and 17 with low-moderate haemorrhagic profile.
Severe haemorrhagic patients were autonomous in self-administration treatment. When they leave PU, only 20% were autonomous to manage themselves in health care environment. This percentage increases up to 60% after 6 months in the AU. During the transition process, two cases (5%) of poor adherence during first months were seen.
We observed that a 100% of patients with low-moderate haemorrhagic phenotype have lower knowledge about their disease and how to manage it.
Discussion/Conclusion: Empowering CC patients and families in self-care is challenging.
It is necessary to promote adherence during this process, especially in those with severe haemorrhagic phenotype.
It is mandatory to educate and reinforce knowledge in low-moderate haemorrhagic patients.
Specialised integrated units and CNSs are essential with cross-cutting transition programs.
Disclosure of Interest: None declared.
NU04 Oasis phase 1: Current state of nursing practices for patient management in the hemophilia treatment centers (HTC) in France
S. Delienne1,*; C. Chenuel2; N. Bouvet3; G. Tuaillon4; P. Senechal5; C. Cerato-Blanc6; N. Guerin7; P. Lezeau8; M.-A. Champiat9; O. Rouault3; S. Gerard10; G. Minnaert11; C. Massimino11
1HTC, Hospital, Dijon; 2HTC, Hospital, Nancy; 3HTC, Hospital, Rennes; 4HTC, Hospital, Bordeaux; 5HTC, Hospital, Amiens; 6HTC, Hospital, Nice; 7HTC, Hospital, Caen; 8HTC, Hospital, Paris; 9HTC, Hospital, Montpellier; 10HTC, Hospital, Toulouse; 11Science, Roche Laboratory employee, Paris, France
Introduction: In France, 38 HTC and their multidisciplinary teams manage haemophilia patients under a rare disease network, Mhemo. In 2021, Roche and Chugai, with expertise from Fidel'hem, the French Association of Haemophilia Nurses, initiated the OASIS project. The OASIS project examines the nurse's role within the HTC in order to enhance quality of care and improve patient management of people living with haemophilia. The project aims to raise awareness of the role of nurse coordinators within HTCs and allow better recognition of their specific and complex skills.
Methods: A steering committee with 11 HTC nurses has designed a questionnaire about: (a) the scope of nurses’ activities and practices that are implemented in HTCs; (b) the prospective and professional evolution of nursing in haemophilia. The questionnaire has been sent to all members of Fidel'Hem.
Results: A total of 19/26 nurses (73%) who worked in 17/33 HTCs (51.5%) answered the questionnaire. The scope of potential nurses’ activities covers 34 activities. Each activity is implemented in at least one HTC. Twenty-one activities are implemented in most of the HTCs and 13 activities in every HTC. Respondents appraise all of the 34 activities of the scope as important or very important; 14/21 activities win unanimous agreement. Respondents point that it is essential to lead a combined approach, both locally in each HTC and nationwide with MHEMO.
Discussion/Conclusion: This initial review of nurses’ practices among HTCs in France highlights a common foundation on one hand and a heterogeneity in the practices on the other hand. There is a need to share and extend these practices across different centres. Accordingly, a second phase of experimentation will begin across various centres upon receiving local and national support. This is with the goal of enhancing patient care pathways.
Disclosure of Interest: None declared.
NU05 Effect of nordic walking on risk factors for cardiovascular disease in people with haemophilia
J. Belev1,*; M. Marinic2; A. Boban1
1Hematology, Hemophilia Centre Zagreb, UHC Zagreb; 2Croatian Haemophilia Society & Institute of Social Sciences Ivo Pilar, Zagreb, Croatia
Introduction: Physical activity has several benefits for people with haemophilia (PWH), including reduction of risk factors for development of cardiovascular diseases (CVD). However, due to the increased risk of bleeding, physical activity was avoided by PWH in the past. Now, with the availability of efficacious prophylaxis, PWH are encouraged to be actively involved in sports, although ones avoiding physical contact. Nordic walking (NW) is specific type of walking that uses specially designed poles with the aim to correct the posture and to include different muscle, and at the same time to relieve the pressure from the hips, knees and ankles. The aim of this study was to assess the efficacy and safety of NW on reducing risk factors for CVD in PWH.
Methods: Prospective study was conducted at the Haemophilia Centre in University Hospital Centre Zagreb in 2022. People with either haemophilia A (HA) and B (HB) of all severities were eligible for the study. Each participant had to do NW at minimum of 40 min three times a week during the 3 months.
Results: We included 20 participants in the study, however, only eight of them finished the program as planned. Among these, 18(90%) had HA and 2(10%) HB, and 90% had severe and 10% mild disease, respectively. Median (range) age was 52 (26–69) years. BMI decreased in 75% participants, blood pressure level in 75%, pulse rate in 75% and the waste girth in 100%. Moreover, the number of steps and the speed of walking improved in majority of participants (62%). Finally, no participant experienced bleed during walking, not had the need for analgesic therapy.
Discussion/Conclusion: We have demonstrated that NW has a potentially beneficial effect on reducing risk factors for CVD in PWH; however, further studies with higher number of participants are needed to confirm this result. Based on our results, the lack of motivation is the biggest obstacle for NW in PWH.
Disclosure of Interest: None declared.
PT01 Using evidence-based co-design to develop a virtual based exercise intervention that aims to increase confidence to exercise in persons with haemophilia
S. Taylor1,2,*; K. Barker3,4; D. Stephensen5; E. Williamson4
1Oxford Haemophilia and Thrombosis Centre, Oxford University Hospitals Foundation Trust, UK; 2NIHR Applied Research Collaboration, Oxford University, UK; 3Physiotherapy, Oxford University Hospitals Foundation Trust, UK; 4Nuffield Department of Orthopaedics and Rhuematology and Musculoskeletal Sciences, Oxford University, Oxford, UK; 5Applied Clinical Research Institution, Canterbury Christ Church University, Canterbury, UK
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 ageing through promotion of physical activity and exercise. Changing patient beliefs and increasing physical literacy and confidence to move are thought to be key to helping PWH become more active. 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.
1: Identify possible intervention elements from the literature and by engagement with stakeholders. Existing online exercises classes in haemophilia centres were visited to review exercises and gain stakeholder feedback.
2: Produce a draft exercise programme for stakeholder review.
3: Develop a theory of change model to guide the behaviour change elements with stakeholders.
4: Finalise the intervention.
Stakeholders included 17 PWH and seven physiotherapists working in haemophilia. Seven online focus group meetings were held.
Results: The final intervention is a hybrid 12-week physiotherapist led progressive exercise programme. Classes are 45 min including pilates, HIIT and balance elements, together with discussion sessions focusing on physical activity recommendations, the types and benefits of different exercise styles and the effects of physical activity, together with the effects of aging for PWH. PWH expressed the need for a physiotherapist expert in haemophilia to teach the class and for their individual musculoskeletal abilities to be considered. They were keen to exercise with other PWH and gain confidence/social support from group interaction. The COM-B model of behaviour change was used to develop the intervention.
Discussion/Conclusion: Co-design helps to produce an intervention that understands the stakeholders needs. Through this process the intervention developed to incorporate not only increasing physical activity 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.
Disclosure of Interest: None declared.
PT02 Treatment using the Blazepod® to improve balance an reaction time in hemophilia patients: A pilot study
R. Tiktinsky*; L. Landa; G. Kenet
National Hemophilia Center, Sheba Medical Center, Tel Hashomer, Ramat Gan, Israel
Introduction: The hallmark of haemophilia is bleeding into joints and muscles. Although prophylaxis is recommended to patients many still suffer intra-articular bleeds, even with the proper use of factor concentrate. These bleeds damage knee and ankle joints leading to pain, muscle weakness and shortening of the muscles, altering the proper mechanisms of gait pattern and balance in older adults. Balance in adults with haemophilia has been tested showing abnormal balance patterns and monitor natural disease progression and early biomechanical changes that occur in older haemophilia patients. The object of this pilot study was to show that treatment with the Blazepod® will help improve balance and reaction time.
Methods: Five haemophilia A and B patients ages 40−65 participated in this pilot study. They were assessed to determine their muscle strength, range of motion, Berg Balance and one leg stance tests. The program took place twice a week for 8 weeks while working with the BlazePod® 15−20 min each session. Blazepod® by Play Coyotta Ltd. and BlazePod Inc is a smart reaction training program. There are three phases: recognition, processing and reaction. The pods and software generate the activity. The pods light up according to a preset program and the patient has to press on the pod to extinguish the light either on the wall while standing and using the upper extremities or on the floor while using the lower extremities. These could also be used to reduce fall risk in the geriatric population. The pods are placed around the room, while the patient scans the surroundings looking for the lit pod and navigating to hit the target. Strengthening and core stability can be achieved by having the pod timed to go on or off at a certain time and holding an isometric activity such as single-leg balance.
Results: The results showed an improvement in: Muscle strength improved from 4- and 4 to 4+ and 5-. Range of motion improved between 10−15° in the hip and knee. Results of the Berg Balance test improved by 20% and the one leg stand test by 50%. Reaction time improved by 25%.
Discussion/Conclusion: In conclusion, it has been shown that as individuals age their risk of falls increases. In our pilot study, the results have shown an improvement of strength, range of motion, balance and reaction time.
Our aim is to include a control group and increase the number of participants in order to reinforce our findings.
Disclosure of Interest: None declared.
PT03 The DASH project—developing AHP services for people with haemophlia
F. Sayers1,*; C. Forde1; C. Macartney2; G. Benson3
1Haemophilia Comprehensive Care Centre, UK; 2Children's Haematology Unit, Royal Belfast Hospital for Sick Children, UK; 3Haemophilia Comprehensive Care Centre, Belfast City Hospital, Belfast Health & Social Care Trust, Belfast, UK
Introduction: DASH 1, a quality improvement initiative aimed at development of Allied Health Profession (AHP) services for People with Haemophilia (PWH), involved the introduction of Occupational therapy (OT) in conjunction with a review of Physiotherapy (PT) at the Belfast Adult Haemophilia Centre.1 The aim of DASH 2 was to complete outstanding actions from DASH 1 and carry out a similar review at the Paediatric Centre.
Methods: A key objective identified in DASH 1 was to develop a leaflet to help PWH prepare for clinic appointments, including questions they may be asked, or wish to ask, at clinic. Several drafts were reviewed and amended by Staff, service users, the ‘Reader Group’ and Corporate Communications before the final leaflet (Getting Clinic Ready—what matters to you) was agreed and circulated in advance of clinic appointments. A questionnaire was developed and circulated to parents (n = 58 families) of all children with Haemophilia (n = 82) addressing areas including knowledge, awareness of, and satisfaction with, existing AHP services and willingness to participate in focus groups. AHP involvement in multi-disciplinary team (MDT) training and Service User education sessions was reviewed.
Results: Eighteen families (31%) responded to the questionnaire. About 83% were aware of the OT service and 94% understood the role. About 100% were aware of the PT service and the PT role. When asked how it felt meeting team members at clinic parents said that staff were friendly, helpful, reassuring, accommodating and welcoming. One noted that it can be overwhelming and stressful. Suggested improvements included, improved parking, shorter wait times, virtual appointments, fans in waiting areas and entertainment for children. About 50% were willing to consider participation in a focus group. AHPs have participated in delivery of staff training sessions and NI Haemophilia ‘Family Days’ providing information on topics including physical activity, sedentary behaviour, physical literacy, sleep, reducing screen time and supporting participation in daily activities.
Discussion/Conclusion: Several PDSA cycles have taken place with patient involvement. Next steps will involve a Focus Group with parents of children with Haemophilia, to consider survey results and priorities regarding AHP involvement in their child's care within the Haemophilia service.
Reference
1. Forde C et al. Occupational Therapy a new voice in haemophilia. Haemophilia, 2021. 10.1111/hae.14236
Disclosure of Interest: F. Sayers Speaker Bureau of: SOBI; Novo Nordisk; CSL Behring, C. Forde: None declared, C. Macartney Speaker Bureau of: SOBI, G. Benson: None declared.
PT04 Effects of blended physical therapy intervention on joint health in persons with haemophilia: A controlled trial
F. Yadolahi*; N. hosseini
Rehabilitation, Shahid Beheshti University of Medical Sciences, Teharn, Iran
Introduction: The physical therapist is essential for persons with haemophilia (PWH). Regular physical therapy can improve joint stability and flexibility and reduce the risk of bleeding in patients with haemophilia. However, regular physical therapy with a physical therapist experienced in this area is not feasible for many patients with haemophilia.
We, therefore, developed a blended physiotherapy intervention for PWH, integrating face-to-face physical therapy with a web-based application to observe the effect on the frequency of bleeds, pain and ankle joint Haemophilia Joint Health Scores (HJHS).
Methods: Thirty-two patients with a mean age of 30.2 (SD: 8.89) years were randomized to the blended therapy group (n = 16) and control group (n = 12). A 12-week blended intervention combining face-to-face physical therapy sessions with a web-based app was used. The control group received face-to-face physical therapy sessions. The intervention consists of information modules for individuals with HA, a graded activity program with a self-selected activity, and personalized video-based exercises. Patients can receive pop-ups as reminders and provide feedback on how to perform the exercises. Retrospectively the number of bleedings during the intervention was compared to the number of bleedings before. Pain and Haemophilia Joint Health Scores (HJHS) together with a satisfaction questionnaire were evaluated before and after the interventions.
Results: Patients in the blended group exercised more in a median of 1.6 times a week. No training-related bleeds occurred. The blended group showed reduced joint pain (p < .05) and muscle bleeds (p < .02) comparing to control. HJHS scores post-intervention were significantly higher in the intervention group (p < .05). Self-reported satisfaction with blended therapy was good. Barriers to continuing included a busy lifestyle, anxiety or stress and poor internet.
Discussion/Conclusion: Blended physical therapy is an innovative intervention that can assist individuals with haemophilia to perform recommended physical activities and exercises and can help to improve physical fitness and coordination, reduce bleeding propensity, and improve patient adherence to physical activities.
KEYWORDS
eHealth, exercise, haemophilia, physiotherapy, telerehabilitation, telehealth
Disclosure of Interest: None declared
PT05 Utilization and treatment content of primary care physiotherapy for persons with bleeding disorders in the Netherlands: preliminary results
J. Blokzijl1,2,*; M. F. Pisters3,4; C. Veenhof3,5; R. E. Schutgens1; M. Timmer1
1Centre for Benign Haematology, Thrombosis and Haemostasis, Van Creveldkliniek, University Medical Centre Utrecht, The Netherlands; 2Physical Therapy Research, Department of Rehabilitation, Physiotherapy Science and Sport, Brain Center Rudolf Magnus, University Medical Center Utrecht, The Netherlands; 3Physical Therapy Research, Department of Rehabilitation, Physiotherapy Science and Sport, Brain Center Rudolf Magnus, University Medical Centre Utrecht, The Netherlands; 4Research Group Empowering Healthy Behaviour, Department of Health Innovations and Technology, Fontys University of Applied Sciences, Eindhoven, The Netherlands; 5Research Group Innovation of Human Movement Care, University of Applied Sciences Utrecht, Utrecht, The Netherlands
Introduction: Physiotherapy is mentioned in the WFH guideline for every musculoskeletal complication due to bleeding disorders. In the Netherlands, care for persons with bleeding disorders is often provided by primary care physiotherapists close to their home. However, to our knowledge it is unclear what the utilization and content of treatment is in primary care physiotherapy. Aim is to describe the utilization and content of primary care physiotherapy treatment in persons with bleeding disorders in the Netherlands.
Methods: All patients over 16 years old with haemophilia or VWD attending the clinic were asked if they received primary care physiotherapy treatment between 2015 and 2023 for a haemophilia related condition. Data was collected from primary care physiotherapists and the primary care medical records consisted of (among others) number of treatment sessions, content of treatment provided and effect of treatment.
Results: Between 2021 and 2023 141 persons with a bleeding disorder were asked if they had attended primary care physiotherapy. A total of 76 (54%) did not visit a primary care physiotherapist or only for a non-haemophilia related complaint. Of the group that indicated they did not attend primary care physiotherapy 8% (6/76) indicated this was caused by not having enough trust in a primary care physiotherapist and 3% (2/76) because they experienced financial barriers. Of the 37 medical files that were reviewed almost half (n = 17; 46%) were for haemophilic arthropathy, 30% (n = 11) for joint bleeds, 16% (n = 6) for muscle bleeds and 8% (n = 3) for chronic synovitis. A median of 18 (IQR: 13) treatment sessions were provided for persons with haemophilic arthropathy. Most commonly applied treatment was passive mobilisation techniques (77%), followed by exercise therapy (59%) and education (47%). In joint and muscle bleed a median of 11 (IQR 12) and 8.5 (IQR 11.2) treatment sessions were provided respectively. In contrast with arthropathy therapy in this field is more exercise based (83% and 82%) and rely less on passive techniques (37% and 0%).
Discussion/Conclusion: Current study provides insight into the use of primary care physiotherapy in the Netherlands. There appears to be a high utilization of passive treatment techniques in arthropathy which is not in concordance with newly developed guidelines.
Disclosure of Interest: None declared.
PT06 A systematic review of measurement properties of physical function outcome measures applied in people with haemophilia
A. Bärlocher1,*; A. Juanós Solé1; N. Pecorelli2; R. H. Knols1,3; E. D. de Bruin3,4,5; P. Baschung Pfister1
1Directory of Research and Education, Physiotherapy-Occupational Research Center, Switzerland; 2Directory of Research and Education, Physiotherapy-Occupational research Center, University Hospital Zurich, Switzerland; 3Department of Health Sciences and Technology, Institute of Human Movement Sciences and Sport, ETH Zurich, Zurich, Switzerland; 4Eastern Swiss University of Applied Sciences, Department of Health, OST, St. Gallen, Switzerland; 5Division of Physiotherapy, Department of Neurobiology, Care Sciences and Society, Karolinska Institutet, Stockholm, Sweden
Introduction: The aim of this systematic review was to investigate the measurement properties of assessments, determining physical fitness and function in adult people with haemophilia (PwH), as well as to evaluate the methodological quality of the studies, and the level of evidence for the measurement properties.
Methods: Articles published in English or German in Medline, EMBASE, CINAHL, Cochrane, PsycINFO, Scopus, PEDRO or Cosmin were included if they evaluated performance-based or clinician-reported outcome measurement instruments (OMI) aiming to measure physical function in adult PwH. Studies were excluded if they only used these assessments as an OMI, or only included patient reported outcome measures or laboratory values. Data extraction and evaluation of the methodological quality was performed following the COSMIN guidelines: For each OMI, risk of bias was evaluated and measurement properties were rated. Then, results were summarized and the quality of evidence was defined following the modified GRADE approach.
Results: Fifteen articles describing 12 OMIs (Colorado Adult Joint Assessment Scale, Functional Independence Score in Haemophilia, Haemophilia Joint Health Score, Four Square Step Test, M3 diagnos, Microsoft Kinect V2 sensor, Three-Dimensional Gait Analysis, Timed Up and Go, and Short Physical Performance Battery) were included. Out of the nine possible measurement properties defined by COSMIN, six were examined: construct validity (convergent and discriminative), reliability, measurement error, internal consistency, criterion validity and responsiveness. None of the included OMIs has sufficiently described measurement properties. Overall, the quality of evidence of all evaluated measurement properties was ‘very low’ in 77%, ‘low’ in 8% and ‘moderate’ in 15%. Furthermore, the rating of the criteria for good measurement properties could not be determined in 54%.
Discussion/Conclusion: Only the Haemophilia Joint Health Score 2.1 showed ‘sufficient’ convergent validity with ‘moderate’ quality of evidence and can be recommended. Due to the unsatisfactory methodological quality of the included studies and low quality of evidence, it still remains a challenge for researchers and clinicians to select appropriate OMIs for adult PwH. Further clinimetric studies are urgently needed and it is highly recommended to follow the COSMIN reporting guidelines for their conduction.
Disclosure of Interest: A. Bärlocher Grant/Research support from: Novonordisk, A. Juanós Solé Grant/Research support from: Novonordisk, N. Pecorelli Grant/Research support from: Novonordisk, R. Knols: None declared, E. de Bruin: None declared, P. Baschung Pfister Grant/Research support from: Novonordisk.
