Reliability and construct validity of the ACTIVLIM-Hemo and Haemophilia Activities List (HAL) questionnaires in individuals with haemophilia.

OBJECTIVE: The objective of this study is to assess the reliability and construct validity of ACTIVLIM-Hemo, a newly developed Rasch-built questionnaire designed to evaluate activity limitations in people with haemophilia (PwH), in comparison with the Haemophilia Activities List (HAL), which was developed using Classical Test Theory. METHODS: A total of 130 participants with haemophilia A or B were included. They underwent various assessments, including joint health scoring (HJHS), functional tests (TUG and 2MWT) and completed questionnaires such as the BPI, IPAQ, HAL and ACTIVLIM-Hemo. Reliability indices and the minimum detectable change (MDC95) were determined for ACTIVLIM-Hemo and for HAL. Construct validity was evaluated through correlations and multiple linear regression, considering demographic and clinical factors. RESULTS: Both ACTIVLIM-Hemo (Person Separation Index = 0.92) and HAL (Cronbach's α = 0.98) demonstrated high reliability. The MDC95 for ACTIVLIM-Hemo represented 11.6% of its measurement range, while for HAL, it amounted to 18/100 score points. Activity limitations measured by both instruments were significantly correlated with demographic and clinical factors. Joint health (HJHS), pain severity (BPI) and walking performance (2MWT) emerged as the main predictors of activity limitations, explaining 75% of the variance in ACTIVLIM-Hemo and 60% in HAL. CONCLUSION: ACTIVLIM-Hemo stands as a reliable and valid instrument for assessing activity limitations in PwH. Both instruments exhibited significant correlations with demographic and clinical factors, but ACTIVLIM-Hemo displayed a more homogeneous construct. Given its linear scale and lower MDC95 and better targeting, ACTIVLIM-Hemo shows promise as a patient-centric instrument for assessing responsiveness to treatment during individual follow-up.

Building a haemophilia-specific navigator in Epic® Electronic Health Records: Single-centre preliminary experience.

INTRODUCTION: With the increasing complexity of haemophilia care and the advent of numerous therapeutic innovations, there is an unmet need for documentation and data collection tools tailored to people with haemophilia (PwH). To date, no fully integrated haemophilia-specific electronic health record (EHR) has been described in the literature. AIM: To evaluate the feasibility of integrating a haemophilia-specific navigator into the Epic EHR. METHODS: Based on clinical experience and registry datasets, we identified key variables describing both PwH and carriers of haemophilia. These were then incorporated into a REDCap database, which served as a starting point for the development of a comprehensive haemophilia flowsheet. We built a dedicated haemophilia navigator within Epic that includes a flowsheet featuring up to 212 variables, as well as customised note templates and patient lists integrating data from the haemophilia flowsheet. RESULTS: It was feasible to develop a haemophilia navigator within Epic over the course of 12 months. The navigator's flowsheet enables systematic and comprehensive clinical assessment of PwH and carriers, while customised patient lists provide a quick summary of each patient's profile to the haemophilia treatment centre staff and highlight issues that require an intervention. In our clinical practice, patients actively participated in the new documentation process and responded positively to the navigator. CONCLUSION: Adapting EHRs to the needs of PwH and carriers promotes holistic care for this population and provides an opportunity for patient empowerment. Such haemophilia-specific EHRs are expected to promote standardisation of care and facilitate the collection of registry data on a national and international level.

Synovitis and joint health in patients with haemophilia: Statements from a European e-Delphi consensus study.

INTRODUCTION: Synovitis, a common feature in haemophilia, is triggered by the presence of blood in joints, and represents the first step towards the development of chronic arthropathy. Synovitis may be detected early by means of ultrasound or magnetic resonance imaging scan; clinical joint scores are less sensitive in this setting. Regular long-term prophylaxis with clotting factor concentrates, as primary prophylaxis and tailored to individual needs, has high efficacy in preventing synovitis. In general, higher factor levels lower bleeding risk, but no direct correlation between factor levels and synovitis incidence has been demonstrated. AIM: This study aimed to develop an expert consensus relating to the definition, pathophysiology, diagnosis, prevention, follow-up and treatment of synovitis, recognising its relevance for joint health and taking into account existing knowledge gaps. METHODS: A Delphi consensus study was designed and performed. An expert group prepared 22 statements based on existing literature; a wider expert panel subsequently voted on these. RESULTS: Retention of panellists was high. Four statements required amending and consensus on all statements was achieved after three rounds of voting. CONCLUSION: This e-Delphi consensus study addressed the importance of synovitis in joint health of people with haemophilia and highlighted knowledge gaps in this field. Studies on the natural course of synovitis are lacking and the biological mechanisms underlying this process are not yet fully elucidated. While basic and clinical research proceeds in this field, expert consensus can help guide clinicians in their routine clinical practice, and Delphi methodology is often used to produce best-practice guidelines.

Psychophysical assessment of pain in adults with moderate and severe haemophilia: A cross-sectional study.

BACKGROUND: Joint pain is the hallmark of haemophilia; therefore it seems clinically rather a musculoskeletal than a bleeding disorder. Although joint pain in people with haemophilia (PwH) is a complex and multidimensional problem, pain assessment remains primarily focused on the structural evaluation of their joints. Whereas, only few data are available on the potential implication of psychophysical and psychological factors. OBJECTIVE: This study aimed to perform a psychophysical pain assessment including quantitative sensory testing (QST) and an evaluation of psychological factors in a large sample of PwH, to get insight into the individuals' pain system. METHODS: Ninety-nine adults (36.9 ± 13.5 years) with moderate/severe haemophilia A/B and 46 healthy controls filled in self-reported pain and psychological questionnaires and underwent a QST evaluation including static and dynamic tests. Static tests focused on the determination of thermal detection and pain thresholds and mechanical pressure pain thresholds. Dynamic tests evaluated pain facilitation and the efficacy of endogenous pain inhibition. Besides comparing PwH and healthy controls, between-subgroup differences were studied in PwH based on their pain distribution. RESULTS: The study revealed increased thermal and mechanical pain sensitivity and the presence of unhelpful psychological factors such as anxiety/depression in PwH. Among the subgroups, especially PwH with widespread pain showed altered somatosensory functioning. Enhanced pain facilitation and impaired efficacy of endogenous pain inhibition in PwH could not be observed. CONCLUSION: Altered somatosensory functioning and unhelpful psychological factors, appear to play an important role in the pathophysiology of pain in PwH, especially in PwH with widespread pain.

ACTIVLIM-Hemo: A new self-reported, unidimensional and linear measure of activity limitations in persons with haemophilia.

INTRODUCTION: To assess activity limitations in people with haemophilia (PwH), the self-reported Haemophilia Activity List (HAL) is widely employed, despite several methodological limitations impacting the interpretation of categorical scores. Modern psychometric approaches avoid these limitations by using a probabilistic model, such as the Rasch model. The ACTIVLIM is a Rasch-built measurement of activity limitations previously validated in several clinical conditions like neuromuscular disorders. AIMS: This study sought to develop the ACTIVLIM-Hemo, meaning an ACTIVLIM scale version specifically adapted to assess daily activity limitations in adult PwH. METHODS: Daily activities were assessed as "impossible," "difficult" or "easy" by 114 PwH (median age of 44 years) with 63 of them reassessed after 12 days. The Rasch Rating Scale model was used to identify activities delineating a unidimensional and linear scale unbiased by demographic and clinical status. Concurrent validity was determined through correlation with the HAL sub-scores and sum score. RESULTS: The ACTIVLIM-Hemo included 22 pertinent activities, with difficulties independent of demographic and clinical conditions, allowing a reliable measure of activity limitations (PSI = .92) expressed on a linear and unidimensional scale in PwH (7%-100 % range, ceiling effect of 1/114) with excellent test-retest reliability (ICC = .978). Spearman rank correlations between ACTIVLIM-Hemo and HAL sub-scores ranged between .623 and .869. CONCLUSIONS: The ACTIVLIM-Hemo is an easy-to-administer, valid and reliable alternative to HAL in assessing activity limitations in PwH. Its invariant scale can be used across conditions and time to compare the functional status of PwH over a wide measurement range.

Pain interferes with daily activities, emotions and sleep in adults with severe, moderate and mild haemophilia: A national cross-sectional survey.

INTRODUCTION: Pain is a major issue in people with haemophilia (PwH). Few studies comprehensively assessed pain in PwH using a biopsychosocial framework and studies in mild PwH are lacking. AIM: To assess pain prevalence, pain interference and their relationship with health-related quality of life (HR-QoL) in male adults with haemophilia. METHODS: A survey was initiated by the Belgian national member organisation. Pain in the last 24 h, pain severity (BPI-PS) and pain interference (BPI-PI) scores were obtained with the Brief Pain Inventory short-form (BPI). HR-QoL was evaluated with the EQ-5D-3L, giving the health utility index (EQ-HUI). Associations between EQ-HUI, BPI-PS and BPI-PI were analysed using Pearson's correlation test. A multiple regression analysed the relationship between HR-QoL and BPI-PS, with age and haemophilia severity as confounding factors. RESULTS: Within 185 respondents (97, 31 and 57 respectively severe, moderate and mild PwH), 67% (118/177) reported pain. In severe, moderate and mild PwH, respectively 86% (79/92), 71% (22/31) and 32% (17/54) reported pain. Median [IQR] BPI-PS, BPI-PI and EQ-HUI scores were respectively 1.5 [.0; 4.0], 1.6 [.0; 3.6] and .81 [.69; 1.00]. PwH reported pain interference with general activity (56% (99/176)), psychosocial factors such as mood (53% (93/175)), and sleep (51% (90/177)). Moderate correlations were found between EQ-HUI, BPI-PS and BPI-PI. After adjusting for age and haemophilia severity, BPI-PS explained 14% of HR-QoL variance. CONCLUSIONS: Pain is a major issue amongst PwH, including people with mild haemophilia. Pain interferes with activities, emotions, sleep and HR-QoL, arguing for a comprehensive biopsychosocial approach of pain.

Discordance between joint pain and imagery severity in the ankle joint and contributors of lower limb activity limitations in adults with haemophilia: A cross-sectional study.

INTRODUCTION: People with haemophilia (PwH) suffer from knee and ankle joint pain, but the association with structural damage remains underexplored. They report activity limitations but it is unclear which factors contribute to lower limb activity limitations (LL-AL). AIMS: This study aimed (i) to analyse the association between ankle joint pain and structure and (ii) explore the contribution of haemophilia-related, individual and psychological factors to LL-AL in PwH. METHODS: This study included 104 moderate/severe PwH. Ankle pain intensity was assessed with a numeric rating scale and pain sensitivity with algometry (pressure pain threshold (PPTA )). Ankle structure was assessed with MRI (IPSG-MRI) and ultrasound (HEAD-US), joint health with the Haemophilia Joint Health Score (HJHS). The HAL-LOWCOM subscore evaluated LL-AL. A Spearman correlation analysed the correlation between ankle pain and structure. The contribution of haemophilia-related factors (joint health, overall pain (Brief Pain Inventory-Pain Severity (BPI-PS)), functional status (2-Minute-Walking-Distance, Timed Up and Go); individual factors (age, BMI) and psychological factors (fear and avoidance beliefs over physical activity (FABQ-PA) and work (FABQ-Work), anxiety and depression) to LL-AL was explored using a regression analysis. RESULTS: Only low correlations were found between ankle pain intensity and structure (IPSG-MRI, HEAD-US). PPTA was unrelated to structure. Altogether, HJHS, overall pain (BPI-PS), FABQ-Work and age explained 69% of HAL-LOWCOM variance, with 65% explained by the combination of HJHS and BPI-PS. CONCLUSION: No meaningful associations were found between ankle pain and structural damage, suggesting that other factors may contribute to PwH's ankle pain. In contrast, mainly haemophilia-related factors explained LL-AL variance.

Joint status, pain and quality of life in elderly people with haemophilia: A case-control study.

INTRODUCTION: Elderly people with haemophilia (PwH) develop haemophilic arthropathy, pain, and reduced health-related quality of life (HR-QoL). The condition of elderly mild haemophilia patients have rarely been evaluated. This study aimed to compare joint status, pain, and HR-QoL between elderly with mild, moderate/severe haemophilia and healthy elderlies. METHODS: Knee/ankle abnormalities were assessed by ultrasound (HEAD-US) and physical examination (HJHS 2.1). Pain severity and pain interference were investigated using the Brief Pain Inventory. Pressure pain thresholds (PPTs) were obtained at knees/ankles and forehead. Functional limitations were evaluated using the 2-Minute-Walking-Test, Timed-Up-and-Go and HAL. The EQ-5D-5L questionnaire evaluated HR-QoL. Healthy controls (HCs) and elderly individuals with moderate/severe and mild haemophilia were compared using Kruskal-Wallis and Mann-Whitney U tests. RESULTS: From the 46 elderly PwH approached, 40 individuals (≥60 years) with haemophilia A/B (17 moderate/severe; 23 mild) and 20 age-matched HCs were recruited. Moderate/severe PwH displayed worse joint status, lower PPTs, and poorer HR-QoL than mild PwH and HCs (p-value = .010-<.001). HEAD-US abnormalities were observed in 100% of knees and 94% of ankles in moderate/severe PwH, versus 50% of knees and 61% of ankles in mild PwH. Pain was reported by 80% and 57% of moderate/severe and mild PwH, respectively. Low PPTs, functional limitations, and poor HR-QoL scores were likewise observed in some mild PwH, yet without significantly differing from HCs. CONCLUSION: This study highlights poor joint/functional status, pain, and HR-QoL outcomes in elderly with moderate/severe haemophilia. A few mild haemophilia subjects presented joint abnormalities, pain, functional limitations, and poor HR-QoL, without significantly differing from HCs. HIGHLIGHTS: Elderly individuals with mild haemophilia have not yet been extensively studied, whereas moderate/severe haemophilia individuals have proven to suffer from haemophilic arthropathy, pain, and poor health-related quality of life (HR-QoL). Using a case-control design, joint status, pain, and HR-QoL outcomes were examined in elderly haemophilia individuals and compared with those of healthy controls (HCs). Elderly moderate/severe haemophilia individuals exhibited worse joint status, increased joint pain sensitivity, and reduced HR-QoL compared with both mild haemophilia subjects and HCs. A subset of mild haemophilia subjects exhibited poor joint status, pain, and HR-QoL outcomes, without any differences noted when compared with HCs.

Pain coping behaviour strategies in people with haemophilia: A systematic literature review.

INTRODUCTION: Despite the fact that joint bleeds (haemarthrosis) frequently occur in people with haemophilia (PwH) with invalidating arthropathies as result, the clinical pain experience has received only limited attention. A sudden increase in pain intensity can be linked to a bleed, but in most cases, no acute bleed is confirmed. Nevertheless, a patient's perception of an acute bleed as cause of the pain might impact the patients' behaviour in response to pain. It is therefore essential to gain more insight into pain coping strategies seen in PwH. AIM: This systematic review aims to identify the range of pain coping behaviour strategies used among PwH and the factors associated with pain coping behaviour. METHODS: This review was reported according to the Preferred Reporting Items for Systematic reviews and Meta-analyses guidelines (PRISMA). PubMed and Web of Science were systematically screened for relevant literature using keyword combinations related to adult PwH, pain and pain coping behaviour strategies. Risk of bias was assessed with the modified Newcastle-Ottowa Scale. RESULTS: Eleven full text articles (nine cross-sectional and two comparative studies) consisting of 1832 PwH met the inclusion criteria. Due to the heterogeneity of the study samples, quality of evaluation instruments and varying risk of bias, it was difficult to draw conclusions regarding the used pain coping behaviour strategies and associated factors. CONCLUSION: Literature on pain coping behaviour strategies and associated factors in PwH is still scarce and describes heterogenous results. Validated haemophilia-specific instruments are warranted to inventory pain coping behaviour in a standardized way.

Effects of a supervised therapeutic exercise program on musculoskeletal health and gait in patients with haemophilia: A pilot study.

INTRODUCTION: Episodes of bleeding in patients with haemophilia (PwH) are associated with haemophilic arthropathy, limitations in physical performance, reduced quality of life (QoL), and gait disorders. AIM: This non-randomized, controlled, interventional, prospective, single-centre pilot study aimed to assess the effects of an 8-week supervised therapeutic exercise program on musculoskeletal health, gait kinematic parameters (GKP), functional capacity, and QoL in adult PwH. METHODS: Nineteen PwH were allocated to an exercise group (n = 10) or a control group (n = 9). The patients in the exercise group followed an 8-week supervised therapeutic exercise program. The Haemophilia Joint Health Score (HJHS), a two-dimensional video-based gait kinematic analysis (2D-GKA), the 6-min walking test (6MWT), and the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-Qol) were used as the outcome measures at baseline, after the exercise program (at the 8th week), and at the 6th-month follow-up. RESULTS: A significant improvement was observed in the exercise group in the HJHS-Total and Haem-A-Qol Total scores and the 6MWT value after the exercise program. Moreover, the 2D-GKA revealed improvement in most of the GKP (knee extension during the midstance and late swing phases, ankle dorsiflexion during the midstance phase, and ankle plantar flexion during the preswing phase). However, the gain obtained by the exercise program was not maintained at the 6th-month follow-up for the HJHS-Total and Hem-A-QoL-Total scores and GKP. CONCLUSION: The 8-week supervised therapeutic exercise program was successful in achieving improvement in joint health, GKP, functional capacity, and QoL in PwH.

Gaining more insight into ankle pain in haemophilia: A study exploring pain, structural and functional evaluation of the ankle joint.

INTRODUCTION: Ankle arthropathy is highly prevalent among people with haemophilia (PwH), even with prophylaxis, and leads to pain and disability. Mechanisms and consequences of painful symptoms related to ankle arthropathy have not been extensively studied. METHODS: A consecutive sample of 30 adult PwH was included (60 ankles). Ankle structure was assessed with magnetic resonance imaging (IPSG-MRI) and ultrasound (HEAD-US). The HJHS 2.1 assessed function of ankles and knees. Physical functioning was assessed with the Timed Up and Go test, the 2-Minute Walking Test and activity limitations with the HAL questionnaire. Health-related quality of life was evaluated using the EQ-5D-5L questionnaire. Overall pain severity was examined using the Brief Pain Inventory questionnaire and ankle pain intensity with a visual analogue scale. Pressure pain thresholds with an algometer assessed pain sensitivity. Spearman correlations were used to calculate interrelations between joint structure, function and pain. RESULTS: Twenty-five PwH (83%) reported ≥1 painful joint, with 67% reporting the ankle as most painful joint. MRI-confirmed abnormalities were seen in 76% of talocrural and 55% of subtalar joints. HEAD-US abnormalities were seen in 93% of the ankles. A large variation was seen in pain sensitivity at the ankle. While moderate to high correlations were observed between ankle structure and HJHS, no meaningful correlations were found between MRI-scores and pain intensity or sensitivity. CONCLUSIONS: Structural joint damage is present in many ankles but is not related to pain in PwH. Further studies should consider somatosensory nervous system dysfunction in PwH as contributing factor to painful ankle arthropathy.

Feasibility and outcomes of low-dose and low-frequency prophylaxis with recombinant extended half-life products (Fc-rFVIII and Fc-rFIX) in Ivorian children with hemophilia: Two-year experience in the setting of World Federation of Haemophilia humanitarian aid programme.

INTRODUCTION: In sub-Saharan Africa, access to clotting factor concentrates (CFCs) is often extremely limited and published data on people with haemophilia on prophylaxis are almost not existent. AIMS AND METHODS: To assess the feasibility, barriers and outcomes of a low-dose and low-frequency prophylaxis with extended half-life (EHL) recombinant Fc fusion FVIII and FIX in Ivorian children on a two-year period in the setting of the World Federation of Hemophilia's (WFH) humanitarian aid programme. RESULTS: Twenty-five boys with haemophilia were included. Mean (SD) age at inclusion was 5.6 (2.5) years. The median [range] follow-up duration was 17 [11-24] months. Regimen of prophylaxis was 20 IU kg-1 1×/week in haemophilia A and every 10 days in haemophilia B. We observed a maximal reduction by 87.6% of the annual spontaneous joint bleeding rate and a slight decrease in the total HJHS scores (p = .047). Adherence problems related to parents' low education level and shortage in CFCs were the main issues to carry out the programme. Inhibitors occurred in 12.5%. CONCLUSION: This study confirms the feasibility and efficacy of low-dose and low-frequency prophylaxis in young Ivorian children with haemophilia treated with EHL CFCs donated through the WFH humanitarian aid programme. This work also highlights the crucial role of adherence and the need for appropriate education to achieve prophylaxis. Finally, it reminds the paramount objective of achieving self-sufficient, sustainable and available haemophilia replacement therapy for all worldwide.

The biomechanical behaviour of ankle and foot joints during walking with shoes in patients with haemophilia.

INTRODUCTION: Patients with haemophilia (PwH) often prefer shod walking over barefoot walking as footwear offers ankle joint stability and comfort during gait. Yet, the biomechanical mechanisms contributing to the latter remain poorly understood. AIM: To explore the effect of shoes on the biomechanical functioning of the ankle and foot complex in PwH with and without haemophilic ankle arthropathy and to determine the amount of ankle joint loading during shod walking. METHODS: We analysed data of PwH without haemophilic ankle arthropathy (n = 5) and PwH with severe haemophilic ankle arthropathy (n = 17) and a control group (n = 17). During 3D gait analysis, a four-segment kinetic foot model was used to calculate kinematic and kinetic parameters of the ankle, Chopart, Lisfranc and first metatarsophalangeal (MTP 1) joints during both barefoot and shod walking. RESULTS: We found a significantly greater ankle joint power generation during shod walking compared to barefoot walking in PwH with severe haemophilic ankle arthropathy (P < .001). Chopart joint biomechanics were significantly lowered in all three groups during shod walking compared to barefoot walking. During shod walking, the ankle joint load was significantly lowered in both PwH groups (P = .039 and P = .002), but not in the control group (P = .952). CONCLUSION: Explorations in this study uncover a tendency that shoes alter the biomechanical functioning of the ankle and foot complex in PwH and simultaneously lower the ankle joint load during walking.

Cross-cultural adaptation and validation of Haem-A-QoL in Côte d'Ivoire.

INTRODUCTION: Health-related quality (HRQoL) evaluations are considered essential outcomes in the assessment of people with haemophilia. In developing countries, reliable HRQoL data are even more critical whilst enabling government agencies to develop national haemophilia care programmes. However, validated tools are not yet available in sub-Saharan African countries. AIMS: This study sought to perform a cultural adaptation and validation of the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) in Côte d'Ivoire. METHODS: The process comprised several steps, such as linguistic adaptation, cognitive debriefing interviews with adult haemophilia patients and psychometric testing, including reliability (internal consistency, test-retest reliability) and validity assessments (convergent with EQ-5D-5L, criterion with HJHS 2.1, known-groups). RESULTS: The final Ivoirian Haem-A-QoL version was obtained in December 2017 following linguistic adaptation and cognitive debriefings with six participants. The validation process included 25 patients, mainly haemophilia A patients (88%) with severe forms (80%). All participants received on-demand treatment, with joint impairment observed in 92%. Internal consistency and test-retest reliability of the Ivoirian Haem-A-QoL were very good. A Pearson correlation analysis revealed a moderate negative correlation between EQ-VAS and total Haem-A-QoL scores and a moderate positive correlation between HJHS 2.1 and total Haem-A-QoL scores. CONCLUSIONS: A cross-culturally adapted and validated Haem-A-QoL version in Côte d'Ivoire is now available, enabling measurement of intervention outcomes in the targeted population and Ivorian participation to multisite international trials. However, further work is needed to ensure optimal understanding of HRQoL questionnaires, previously developed in culturally distinct countries, with almost unlimited access to different treatment regimens.

Acquired multi-segment foot kinematics in haemophilic children, adolescents and young adults with or without haemophilic ankle arthropathy.

INTRODUCTION AND AIM: The ankle joint remains vulnerable in children with haemophilia and is the primary joint affected. The purpose of this study was to dynamically characterize the segmental foot and ankle kinematics of male children, adolescents and young adults with or without ankle arthropathy. METHODS: The barefoot multi-segment foot kinematics of 70 ankles from 35 haemophilia subjects between 6 and 20 years old were captured with the Rizzoli Multi-Segment Foot Model. Joint damage of the tibiotalar and subtalar joints was scored using the IPSG-MRI score. The feet of patients with or without evidence of ankle arthropathy were compared with those of matched typically developing boys via a nonpaired comparison. The differences between the affected and nonaffected sides of patients with unilateral ankle arthropathy were assessed using a paired comparison. RESULTS: Subjects without arthropathy demonstrated a nonsignificant trend towards a higher frontal plane range of motion (RoM) at the midfoot upon loading response and a lower sagittal plane RoM at the midfoot during midstance. No differences were observed between the affected side group and their matched control group. The affected side of unilaterally affected subjects exhibited a nonsignificant tendency towards a higher frontal plane RoM at the ankle joint upon loading response and terminal stance compared to the healthy side. CONCLUSION: Most patients maintained physiological rocker function of the ankle and had no (mal)adaptive motion patterns in the more distal joints of the foot. Therefore, established structural lesions may remain subclinical with respect to moderate functional activities like walking.

Clinical gait features are associated with MRI findings in patients with haemophilic ankle arthropathy.

INTRODUCTION: Haemophilic ankle arthropathy due to repeated joint bleeds often leads to altered gait in adult patients with haemophilia. AIM: To investigate the association between clinical gait features and blood-induced ankle joint damage scored using MRI findings in patients with haemophilic ankle arthropathy. METHODS: This observational study investigated 48 ankles of 24 patients with severe haemophilia (median age of 33 years). Blood-induced ankle joint damage was scored by an experienced radiologist using the International Prophylaxis Study Group (IPSG-)MRI score which evaluates the presence or absence of effusion, synovial hypertrophy, haemosiderin, surface erosions, subchondral cysts and cartilage degeneration. Using 3D gait analysis, peak ankle joint power generation and absorption (W/kg) were measured for each ankle since these are surrogate measures for joint loading during walking. Associations between MRI findings and these two clinical gait features were calculated using Spearman's ρ correlation with an α-level correction (α = 0.01) for multiple tests. RESULTS: We found large negative associations between ankle joint peak power generation and IPSG-MRI score (ρ = -0.631; P = <.001), IPSG-MRI osteochondral subscore (ρ = -0.701; P = <.001), severity of synovial hypertrophy (ρ = -0.507; P = <.001) and haemosiderin (ρ = -0.400; P = .005). Associations were also found for ankle joint peak power absorption and IPSG-MRI score (ρ = -0.425; P = .003) and IPSG-MRI osteochondral subscore (ρ = -0.556; P = <.001). CONCLUSION: Severe blood-induced ankle joint damage relates to a lowered tolerance towards ankle joint mechanical loading during walking in patients with haemophilia.

Paediatric patients with blood-induced ankle joint arthritis demonstrate physiological foot joint mechanics and energetics during walking.

AIM: To compare foot joint kinetics and energetics in male paediatric boys with and without blood-induced ankle joint destruction to these of matched control groups. METHODS: A cross-sectional study was conducted in which 3D gait analysis data were collected from thirty-five male children (6-21 years) with severe or moderate haemophilia and twenty-six typically developing boys. Structural integrity of the tarsal foot joints of all haemophilic patients was assessed using the IPSG-MRI scale. All participants walked barefoot while adopting a physiological gait pattern. Three subgroups were created based on the IPSG-MRI scores: a group with no joint involvement (HealthyHaemo), with uni- or bilaterally involvement (PathoHaemo) and with only unilaterally involvement (Haemo_Unilateral_Patho). RESULTS: The PathoHaemo group presented a significant lower Lisfranc peak dorsiflexion angular velocity (34.7°/s vs 71.4°/s, P = .000, Cohen d = 1.31) and a significantly higher Lisfranc peak plantarflexion angular velocity (-130.5°/s vs -51.8°/s, P = .000, Cohen d = 0.98) compared to the control group. The Haemo_Unilateral_Patho side had a significant higher Chopart peak dorsiflexion angular velocity compared to the Haemo_Unilateral_Healthy side (41.7°/s vs 31.9°/s, P = .002, Cohen d = 1.16). CONCLUSION: No evidence for mild and severe gait deviations could be demonstrated. Internal moments, used as a surrogate measure of joint loading, quantified by the multi-segment foot model were found to be similar within the three subanalyses. We suggest that the ongoing musculoskeletal development in children compensates for structural damage to the ankle joint.

Cross-cultural adaptation and validation of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) in Côte d'Ivoire (the Ivory Coast).

INTRODUCTION: Health-related quality of life evaluation is recognized as an important outcome in the assessment of boys with haemophilia. In fact, reliable health-related quality of life data are even more critical in developing countries to advocate for government agencies to develop national haemophilia care programmes. However, validated tools are not yet available in sub-Saharan African countries. AIMS: The purpose of this study was to complete the cultural adaptation and validation of the Canadian Haemophilia Outcomes-Kids' Life Assessment Tool version2.0 (CHO-KLAT2.0) in Côte d'Ivoire. METHODS: The process included four steps: a linguistic adaptation, cognitive debriefing interviews with children and their parents, a validity assessment with the Pediatric Quality of Life Inventory (PedsQL) as a comparator, and a test-retest reliability assessment. RESULTS: The initial Ivoirian version of the CHO-KLAT2.0 was developed through a linguistic adaptation performed in close collaboration with members of the local medical team and haemophilia community. Cognitive debriefings were completed with five boys and their parents, with the final Ivoirian version of the CHO-KLAT2.0 developed in September 2017. The validation process included 37 boys with haemophilia (mean age: 11.4 years; 34 with severe and three with moderate forms of haemophilia, all treated on demand) and their parents. Among the child-reported population (n = 20), we observed a mean CHO-KLAT2.0 score of 51.3 ± 9.2; there was a moderate correlation between the CHO-KLAT2.0 and PedsQL scores (r = 0.581; p = 0.007) and an inverse correlation of the CHO-KLAT2.0 and PedsQL scores with the global rating of the degree to which the boys were bothered by their haemophilia. The mean parent proxy CHO-KLAT2.0 score (n = 17) was 53.5 ± 9.8. Among the parents, we found no significant correlation between the Ivoirian CHO-KLAT2.0 and PedsQL scores or between the parent-reported scores and the parent global ratings of bother. The test-retest intraclass correlation coefficient was 0.879 (95% CI: 0.673; 0.954) for the child-reported questionnaires and 0.880 (95% CI: 0.694; 0.955) for the proxy-reported questionnaires. CONCLUSIONS: A cross-culturally adapted and validated version of the CHO-KLAT2.0 for Côte d'Ivoire is now available that enables baseline values to be obtained and intervention outcomes (namely, prophylaxis) to be measured in Ivoirian boys with haemophilia.

Development and evaluation of appropriate, culturally adapted educational tools for Ivoirian patients with haemophilia, haemophilia carriers and their families.

INTRODUCTION: Patient education is the cornerstone of the management of chronic diseases like haemophilia. The education of patients with haemophilia (PWH), haemophilia carriers and their families requires educational materials adapted to their socio-cultural situations for maximum effectiveness. These tools are currently lacking in developing countries like Côte d'Ivoire. AIMS: We sought to develop educational materials adapted to the Ivoirian context, assess their short- and long-term impacts on knowledge about haemophilia and evaluate the participants' motivation and their satisfaction with the tools. METHODS: Following their elaboration, the materials were administered to 71 participants (37 PWH, 29 carriers and 5 fathers), whose level of knowledge was assessed before (T0), just after (T1), and 1 year following the intervention (T2). We evaluated, analysed and compared the scores at T0, T1 and T2 and evaluated motivation at T0 and satisfaction at T1. RESULTS: All participants significantly improved their skills at T1 (P < 0.001), maintaining a sustained and significant improvement at T2 in comparison with T0 (P < 0.001). In all participants, we observed a high degree of motivation towards improving their knowledge and a high degree of satisfaction with the materials. CONCLUSIONS: Appropriate, culturally adapted educational tools focused on haemophilia are now available in Côte d'Ivoire. These materials will likely contribute to improving haemophilia awareness, to implementing screening, prevention and self-management of the disease and to positively impacting the outcomes of Ivoirian PWH in the long term.

Haemophilia in Côte d'Ivoire (the Ivory Coast) in 2017: Extensive data collection as part of the World Federation of Hemophilia's twinning programme.

INTRODUCTION: In sub-Saharan African countries, research on haemophilia is limited. Since 2015, a partnership has been established through the World Federation of Hemophilia (WFH)'s twinning programme between the haemophilia treatment centre (HTC) of the Centre Hospitalier universitaire of Yopougon in Abidjan, Côte d'Ivoire, and the Cliniques universitaires Saint-Luc of Brussels, Belgium. AIM: This study sought to collect accurate, and detailed demographic, clinical, and laboratory data on the whole identified Ivorian haemophilia population. METHODS: A prospective study was conducted in 2017 in Yopougon's HTC. Participants were assessed through multidisciplinary workups including interviews, logbook review, pedigree establishment, clinical examination and laboratory testing. RESULTS: Data on 81 patients with haemophilia (PWH) (78 severe and moderate) were collected. Postcircumcision bleeding was the most common diagnosis reason (32%). Mouth bleeds and skin wounds accounted for 55.2% of bleeds. Pedigrees revealed 63 deaths in affected relatives among 33 families. Most PWHs (76.5%) were treated on demand, and 21% had never been exposed to clotting factor. Non-substitutive therapies (tranexamic acid [43%], physiotherapy [11%] and DDAVP [0%]) were underused. Overweight was uncommon. Knees were the most clinically affected joints at the Hemophilia Joint Health Score. Inhibitors were present in 7.8% of previously treated PWHs. CONCLUSIONS: This study highlights the value of simple, feasible and inexpensive tools to collect data in the Ivorian haemophilia population and provides the basis for developing and implementing locally appropriate strategies to improve screening, diagnosis, preventive care, treatment and education. It demonstrated the WFH twinning programme benefits for haemophilia care in the developing world.