Introduction: Concizumab is a humanised, monoclonal anti-tissue factor pathway inhibitor antibody. In explorer7, a phase 3 study (NCT04083781), patients with haemophilia A or B with inhibitors (HAwI/HBwI) received once-daily subcutaneous prophylactic treatment with concizumab for prevention of bleeding episodes. Here, we present data on patient-reported health-related quality of life (HRQoL) after concizumab treatment for 24 weeks in explorer7. Methods: Male patients aged ≥12 years (n = 133) were randomised to no prophylaxis (arm 1) or concizumab (arm 2) or assigned to non-randomised concizumab arms (arms 3 or 4). All patients could complete the 36-item Short Form Health Survey (SF-36v2) and patients ≥17 years could complete the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL) at Weeks 0, 4, 8, 16 and 24. SF-36v2 health scales “bodily pain” (BP) and “physical functioning”(PF) were analysed as key secondary endpoints. Several patient-reported outcomes recordings were not collected due to technical reasons. Therefore, a post-hoc mixed model for repeated measures (MMRM) was used for analysis. The MMRM analysis included patients from arms 1 and 2 completing the questionnaire at baseline and at least once on the new dosing regimen. Results: The estimated treatment difference (ETD) on the SF-36v2 BP scale between arm 1 (n = 9) and arm 2 (concizumab, n = 23) from baseline to Week 24 was 7.0 points (95% CI: -1.6; 15.6). On the PF scale, the ETD was 3.3 points (95% CI: -3.8; 10.4). While BP and PF scores were not significantly different between both arms, the scales “general health”, “mental health”, “role-emotional”and “vitality”showed statistically significant differences in favour of concizumab. The total Haem-A-QoL score was significantly improved in arm 2 (n = 13) compared to arm 1 (n = 4, ETD: -22.6 points [95% CI: -42.5; -2.7]). Significant differences were observed for the domains “feeling”, “treatment”, “view of yourself”, and “sport and leisure”. Discussion/Conclusion: Patients on daily subcutaneous concizumab prophylaxis reported better scores on generic (SF-36v2) and disease-specific (Haem-A-QoL) questionnaires compared to patients treated on-demand. The largest ETDs were related to mental and general health. This reflects the potential of concizumab prophylaxis to improve HRQoL in patients with HAwI/HBwI.

BackgroundBackground: Spontaneous bleeding in haemophilia shows a predilection for joints, and repeated hemarthrosis induce synovial inflammation, cartilage degeneration and bone damage, leading to hemophilic arthropathy. The hip is not a target joint and its involvement is not easy to be distinguished from other conditions which may provoke pain referred to the hip.ObjectivesObjectives: Aim of the study was to analyse the different sonographic findings related to different pain producing conditions.MethodsMethods: Thirty patients with haemophilia A and monolateral coxalgia were examined. Sonographic exam (US) was performed in all patients by Esaote equipment My Lab 70 with linear probe 4–13 MHz and power Doppler. The iliopsoas muscle and its bursa, the greater trochanteric bursa, and the hip joint were analysed. Sonographic findings in the joint were scored (score ranging from 0 to 21) for effusion, bone remodelling, cartilage damage, synovial hypertrophy, hemosiderin, osteophytes, hemarthrosis, erosion and fibrotic septa. The World Federation of Haemophilia (WFH) orthopaedic joint scale score and the radiologic Pettersson score were evaluated in all patients.ResultsResults: US showed effusion in 20 hip joints (67%), bone remodelling in 18 (60%), cartilage damage in 10 (33%), synovial hypertrophy in 12 (40%), hemosiderin in 4 (13%), osteophytes in 7 (23%), hemarthrosis in 10 (33%) with power Doppler activity (>3 flags). In no patient erosion or fibrotic septa were observed. Fifteen out of 30 joints showed US score ≤5, and 15 US score >5. In 8 patients a hematoma of iliopsoas muscle was present, in two patients great trochanteric bursitis was detected. WFH mean score value was 39.5 (range 12–57) and the mean Pettersson score value was 10.4 (range 6–12).ConclusionsConclusions: Surprisingly, in most cases (67%) coxalgia was not related to trochanteric bursitis or iliopsoas muscle involvement but was due to hemophilic arthropathy of the hip which is not a target joint. Moreover, power Doppler allowed to detect hemarthrosis in 33% of patients, supporting the hypothesis that vascular changes and remodelling in haemophilia are associated with bleeding and joint damage1.Reference[1] Bhat V, et al. Vascular remodeling underlies rebleeding in hemophilic arthropathy. Am J Hematol2015;90:1027–1035.Disclosure of InterestNone declared

IntroductionHaemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available.AimTo provide an overview of the FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) across Europe.MethodsNon‐interventional, 12‐month retrospective study where anonymized data were retrieved from haemophilia centres/registers in Belgium, France, Germany, Italy, Spain, Sweden and the United Kingdom. Male patients (all ages) receiving coagulation factor treatment 24 months prior to the study, with basal FVIII/FIX levels ≤5 IU dL−1, without inhibitors, were included. Data were summarized descriptively.ResultsIn total, 1346 patients with HA and 312 with HB were included in the analysis; 75% and 57% had severe disease (FVIII/FIX < 1 IU dL−1) respectively. Prophylaxis was most common for severe haemophilia, especially for children, whereas on‐demand treatment was more common for moderate haemophilia in most countries. The mean (SD) prescribed prophylactic treatment ranged from 67.9 (30.4) to 108.4 (78.1) (HA) and 32.3 (10.2) to 97.7 (32.1) (HB) IU kg−1 per week, across countries. Most patients on prophylaxis were treated ≥3 times/week (HA) or two times/week (HB). The median annual bleeding rate (ABR) for patients on prophylaxis ranged from 1.0 to 4.0 for severe HA, and from 1.0 to 6.0 for severe HB, while those with moderate haemophilia generally had slightly higher ABRs. Median ABRs for on‐demand‐treated severe HA ranged from 4.5 to 18.0, and for HB, 1.5 to 14.0.ConclusionTreatment practice varied greatly between centres and countries and patients treated on‐demand and prophylactically both experienced bleeds, emphasizing the need for further optimization of care.

Haemophilic synovitis is a disabling complication of hemarthrosis that leads to destruction of the articular cartilage and the emergence of hemophilic arthropathy. Produces disability, chronic pain, depression, social and physical limitation. Selective embolization of the knee can prevent joint bleeds and joint damage. Aim: Assess selective embolization in patient with knee synovitis in relation to episodes of bleeding. Material and Method: Thirty four knee embolization in 33 patients were performed. Mean age 20 years old. Thirty one type A, two type A with inhibitors and 2 haemophilia type B. Evaluation of the number/year of bleeding episodes and consumption of factor VIII before and after embolization was performed. Results: Thirty patients (91%) had a satisfactory results. Three patients (9%) needed another embolization, two with favourable outcome, and one need arthroscopic synovectomy for poor outcome. The Average bleeding episodes were 1.21 per month (range 0-3) before the procedure and was reduced to 0 (range 0-1.67, p: 0.001) at three months, 0.17 (range 0-1.67, p: 0.001) at 6 months and 0.33 (range 0-1.67; p: 0.024) at twelve months. Four patients were free of bleeding for more than six months. Also after the procedure there was a significant reduction in the use of factor VIII in the first year. No significant adverse effects were reported. Conclusions: Selective embolization of arteries of the knee, is a safe procedure that prevents repetitive episodes of bleeding, which translates into a better joint function, with decreased cartilage damage and less consume of factor VIII.

Background The hip is not a target joint in haemophilic arthropathy (HA). Patients (pts) with Haemophilia have hip pain without obvious signs of joint involvement. They have, very often, haematoma or bursitis. Objectives To investigate the role of ultrasonography (US) to detect bleeding and joint damage HA of the hip. Methods In 30 pts, with Haemophilia and monolateral hip pain, the iliopsoas and its bursa, the greater trochanteric bursae, and the joint were studied by US. The 30 joints were evaluated and scored (score ranging from 0 to 21) for effusion, bone remodelling, cartilage damage, synovial hypertrophy, haemosiderin, osteophytes, haemarthrosis, erosion and fibrotic septa. The clinical evaluation [World Federation Haemophiliac orthopaedic score (WFHO)] was done in all pts. Power Doppler US (PDUS) was performed in all patients. Results US showed effusion in 20 joints, bone remodelling in 18, cartilage damage in 10, synovial hypertrophy in 12, haemosiderin in 4, osteophytes in 7, haemarthrosis in 10 with PDUS activity, erosion in 0 and fibrotic septa in 0. In haemophiliacs 15 out of 30 joints showed US score ≤5, and 15 US score >5 (1,2). In 8 pts was present haematoma of iliopsoas, in two pts great trocanteric bursitis. Conclusions US detected bone and cartilage alterations and synovitis in the hip. Indeed PDUS identified the bleeding in the joints and was able to show different entity of haemarthrosis. US may be useful to evaluate joint modifications in HA in no target joint, too. References Melchiorre D, et al. Haemophilia 2011,17:112-7 Querol F, et al. Haemophilia 2012,18:e215-e226 Disclosure of Interest None declared DOI 10.1136/annrheumdis-2014-eular.4391