The cost of severe haemophilia in Europe: the CHESS study

Background Severe haemophilia is associated with major psychological and economic burden for patients, caregivers, and the wider health care system. This burden has been quantified and documented for a number of European countries in recent years. However, few studies have taken a standardised methodology across multiple countries simultaneously, and sought to amalgamate all three levels of burden for severe disease. The overall aim of the ‘Cost of Haemophilia in Europe: a Socioeconomic Survey’ (CHESS) study was to capture the annualised economic and psychosocial burden of severe haemophilia in five European countries. A cross-section of haemophilia specialists (surveyed between January and April 2015) provided demographic and clinical information and 12-month ambulatory and secondary care activity for patients via an online survey. In turn, patients provided corresponding direct and indirect non-medical cost information, including work loss and out-of-pocket expenses, as well as information on quality of life and adherence. The direct and indirect costs for the patient sample were calculated and extrapolated to population level. Results Clinical reports for a total of 1,285 patients were received. Five hundred and fifty-two patients (43% of the sample) provided information on indirect costs and health-related quality of life via the PSC. The total annual cost of severe haemophilia across the five countries for 2014 was estimated at EUR 1.4 billion, or just under EUR 200,000 per patient. The highest per-patient costs were in Germany (mean EUR 319,024) and the lowest were in the United Kingdom (mean EUR 129,365), with a study average of EUR 199,541. As expected, consumption of clotting factor replacement therapy represented the vast majority of costs (up to 99%). Indirect costs are driven by patient and caregiver work loss. Conclusions The results of the CHESS study reflect previous research findings suggesting that costs of factor replacement therapy account for the vast majority of the cost burden in severe haemophilia. However, the importance of the indirect impact of haemophilia on the patient and family should not be overlooked. The CHESS study highlights the benefits of observational study methodologies in capturing a ‘snapshot’ of information for patients with rare diseases

Hemophilic arthropathy in patients with von Willebrand disease

von Willebrand disease (VWD) affects approximately 1% of the population. Joint bleeds are not a predominant symptom of VWD, however they do occur. Especially in patients with severe VWD, joint bleeds have been described in up to 45% of the patients. Recurrent hemarthroses can lead to joint destruction which is the main cause of morbidity, as is observed in hemophilia patients. In VWD however, the occurrence, severity, onset, treatment and impact of arthropathy have not been well described. This literature review summarizes all publications on arthropathy in patients with hereditary VWD, published until 1-5-2012. joint bleeds in VWD are associated with low FVIII levels and seem to occur at young age. Depending on the severity of VWD, arthropathy occurs in 2-30% of the patients. Radiological joint damage can be seen but has not systematically been studied. Arthropathy can affect daily functioning and interfere with the quality of life. For acute management of joint bleeds FVIII/VWF concentrates can be used in VWD patients unresponsive to desmopressin or desmopressin in responsive VWD patients. When severe joint bleeds have caused synovitis and arthropathy, chemical or radioactive synovectomy and surgical joint procedures seem feasible to prevent further joint damage. Additional research is needed to learn more about the severity, onset and impact of arthropathy in VWD. Ongoing studies will hopefully answer the question whether prophylactic treatment with coagulation factor concentrates should be considered standard of care in severe VWD to prevent arthropathy. (C) 2012 Elsevier Ltd. All rights reserved