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Background: Limited research has been published regarding movement behaviour of adult persons with haemophilia (PWH). It is hypothesized that avoidance of activities and more sedentary behaviour cause poorer physical functioning. Aim: To determine differences in movement behaviour between PWH and healthy adults. Methods: Movement behaviour was measured with an accelerometer distinguishing between; lying/non‐wear, sitting, standing, walking, running and cycling. Time spent on activities was compared between PWH and healthy adults, using absolute time spent on activities and activities as percentage of wear time. Results: One hundred and five PWH (32 mild/moderate with a mean age of 42.8 ± 15.1, severe 42.1 ± 13.6) and 98 healthy adults (mean age 41.9 ± 15.5) showed that adults with severe haemophilia sit and stand more than healthy adults (4.5 [CI 0.6‐8.4] and 4.2 [CI 1.8‐6.6] h/wk, respectively) and walk and run less (3.4 [CI 1.4‐5.3] hours and 33.6 [CI 19.0‐41.7] min/wk, respectively). Patients with mild/moderate haemophilia stand more than healthy adults (3.3 [CI 0.1‐6.4] h/wk). Differences in sitting between severe haemophilia and healthy adults and differences in standing between mild/moderate haemophilia and healthy adults disappeared when using activities as percentage of wear time. Conclusion: Movement behaviour of adults with severe haemophilia differs from healthy adults, mainly due to less walking and less running. No differences were found in other activities and postures or the distribution of movement behaviour over the day. No significant differences were found between adults with mild/moderate haemophilia and healthy adults. [ABSTRACT FROM AUTHOR]

Introduction: Monitoring clinical outcome in persons with haemophilia (PWH) is essential in order to provide optimal treatment for individual patients and compare effectiveness of treatment strategies. Experience with measurement of activities and participation in haemophilia is limited and consensus on preferred tools is lacking. Aim: The aim of this study was to give a comprehensive overview of the measurement properties of a selection of commonly used tools developed to assess activities and participation in PWH. Methods: Electronic databases were searched for articles that reported on reliability, validity or responsiveness of predetermined measurement tools (5 self‐reported and 4 performance based measurement tools). Methodological quality of the studies was assessed according to the COSMIN checklist. Best evidence synthesis was used to summarize evidence on the measurement properties. Results: The search resulted in 3453 unique hits. Forty‐two articles were included. The self‐reported Haemophilia Acitivity List (HAL), Pediatric HAL (PedHAL) and the performance based Functional Independence Score in Haemophilia (FISH) were studied most extensively. Methodological quality of the studies was limited. Measurement error, cross‐cultural validity and responsiveness have been insufficiently evaluated. Conclusion: Albeit based on limited evidence, the measurement properties of the PedHAL, HAL and FISH are currently considered most satisfactory. Further research needs to focus on measurement error, responsiveness, interpretability and cross‐cultural validity of the self‐reported tools and validity of performance based tools which are able to assess limitations in sports and leisure activities. [ABSTRACT FROM AUTHOR]

Haemophilia is characterized by a spontaneous bleeding tendency, affecting mainly the synovial joints. Recurrent joint bleeds induce a cascade of inflammatory as well as degenerative processes injuring synovium, cartilage and bone. These processes affect each other and may occur in parallel and/or sequentially. Clinically, the effects of joint bleeds are heterogeneous. A marked variability in joint damage is observed in patients with a similar bleeding history. Also late stage effects differ with some patients developing chronic synovitis, and others suffering from osteochondral degeneration called haemophilic arthropathy. This article reviews the current understanding of the pathogenesis of blood-induced joint damage, elaborates on potential explanations for the differential effects of a bleed, and discusses challenges for future research. [ABSTRACT FROM AUTHOR]

Background Joint bleeds are reported by 23% of von Willebrand disease (VWD) patients and associated with orthopaedic surgery. Limited data are available on joint surgery in VWD. Aim To assess the prevalence, indications, management and complications of joint surgery in VWD patients. Methods 804 VWD patients with historically lowest von Willebrand factor (VWF) activity ≤30 U dL−1 completed a questionnaire on joint bleeds, joint damage and orthopaedic surgery. We retrieved additional medical file data of patients who underwent surgery on large joints (shoulder, elbow, hip, knee or ankle). Results 116 out of 804 patients (14%) reported large joint surgery. Compared to VWD patients without previous orthopaedic surgery, these 116 patients reported more frequently a history of joint bleeds and joint damage (41% vs. 20%, P < 0.001 and 61% vs. 20%, P < 0.001). Medical file data on 126 large joint surgeries in 79 VWD patients revealed that this surgery was associated with joint damage due to prior joint bleeds in 24% of the procedures. Preoperative clotting factor correction (CFC) to prevent bleeding was administered in most cases (81%). Documentation on postoperative bleeding was found in 23 surgeries (18%). Conclusions Large joint surgery is reported by 14% of VWD patients, related to joint bleeds in 24% and seems associated with bleeding complications frequently despite perioperative CFC. [ABSTRACT FROM AUTHOR]

Background: Joint bleeds ( JB) are reported in a minority of patients with von Willebrand disease ( VWD) but may lead to structural joint damage. Prevalence, severity and impact of JB in VWD are largely unknown. Objectives: The aim of this study was to assess JB prevalence, onset, treatment and impact on health-related quality of life ( HR-QoL) and joint integrity in moderate and severe VWD. Methods: In the Willebrand in the Netherlands study 804 moderate and severe VWD patients [von Willebrand factor ( VWF) activity ≤30U dL−1] completed a questionnaire on occurrence, sites and consequences of JB. To analyse JB number, onset, treatment and impact on joint integrity we additionally performed a patient-control study on medical file data comparing patients with JB to age, gender, factor VIII (FVIII)- and VWF activity matched VWD patients without JB. Results: Of all VWD patients 23% (184/804) self-reported JB. These 184 patients reported joint damage more often (54% vs. 18%, P < 0.001) and had lower HR-QoL (SF36, P < 0.05) compared to VWD patients not reporting JB. Of 55 patients with available JB data, 65% had the first JB before age 16. These 55 patients used more clotting factor concentrate ( CFC; median dose 43 vs. 0 IE FVIII kg−1 year−1, P < 0.001), more often had X-ray joint damage (44% vs. 11%, P = 0.001] and chronic joint pain (44% vs. 18%, P = 0.008) compared to 55 control VWD patients without JB. Conclusion: In conclusion, joint bleeds are reported by 23% of moderate and severe VWD patients, mostly start in childhood, are associated with more CFC use, joint pain, lower HR-QoL and significantly more radiological and self-reported joint damage. [ABSTRACT FROM AUTHOR]

The effects of coagulation factor concentrate infusion on restoring secondary haemostasis in patients with haemophilia are obvious. It is not known whether coagulation factor concentrate infusion affects primary haemostasis or induces an acute inflammatory response. In this study, the influence of a factor VIII (FVIII) concentrate bolus infusion on platelet activation and responsiveness, endothelial activation, and inflammation in adult patients with severe haemophilia A was assessed. VWF showed a mild, but significant decrease 15 min after FVIII infusion (85.02 IU dL−1) vs. before infusion (92.04 IU dL−1; P = 0.017), while ADAMTS-13 levels also show a mild but significant decrease from 66.1 ng mL−1 before infusion, to 53.9 ng mL−1 ( P = 0.012) 15 min after and 50.8 ng mL−1 ( P = 0.050) 60 min after infusion. Platelet P-selectin expression decreased 15 min (33.3 AU) and 60 min (38.7 AU) after infusion compared to before infusion (41.3 AU; P = 0.018 and 0.036). In conclusion, a single infusion of a high dose FVIII concentrate in haemophilia A patients may influence primary haemostasis by decreasing VWF, ADAMTS-13 and the number of circulating activated platelets. These effects possibly occur as a consequence of binding of the infused FVIII to VWF, influencing its processing. When treating severe haemophilia A patients with coagulation concentrate infusion, one should realize this does not merely correct FVIII levels but also may influence primary haemostasis. [ABSTRACT FROM AUTHOR]

The article comments on a study on the influence of joint bleeds on the antibody response towards recombinant human FVIII in a recently developed haemophilia A rat model by researchers K. M. Lovgren and others published in the current issue. Topics include the role of inflammation in inhibitor development, and the finding that a joint bleed contributed to an accelerated antibody response with higher inhibitor levels.

Overall life expectancy and quality of life among persons with haemophilia have increased in recent years, primarily because of the advances in factor replacement therapy and better treatment of infectious diseases. Older haemophilic patients now face aging co-morbidities that are common in the general male population, such as cardiovascular or metabolic diseases, prostate hypertrophy and hepatic, prostate and other cancers. The prevalence of cardiovascular disease and incidence of vascular events among older haemophilic patients can be expected to increase and haemophilic patients may become prone to some cardiovascular risk factors, warranting preventative measures. The treatment of long-term complications of hepatitis C virus infection such as liver cirrhosis and hepatic cancer can be expected to be required in a large portion of the older haemophilia population for some years to come. Appropriate antiviral treatment and close monitoring for possible disease advancement will constitute an important part of routine medical care, and special considerations may be appropriate in conjunction with invasive procedures, chemo- or radiotherapy. At the moment, hard data on which to base the management of these conditions are largely lacking, but can be expected to increase dramatically in the coming decades. In the meantime, the ageing population of haemophilia patients should be offered the same comprehensive health care offered to the general population, which may require a restructuring of health care delivery. [ABSTRACT FROM AUTHOR]

A letter to the editor is presented on whether haemophilia protect against ischaemic cardiovascular disease.