Background: Prerequisite for optimal haemophilia care provision all around Europe is clearness on clinical status and treatment patterns. This was one of the aims of the European Study of Clinical, Health economic and Quality of Life outcomes in Haemophilia treatment(ESCHQoL). Methods: Retrospective/prospective, multicentre (43 haemophilia centres), multinational (21 European countries) cohort study. Data collection: chart extraction for retrospective data (6 months) and ad hoc patient diaries for prospective data (6 months). Results: Overall 1426 patients were enrolled, 26 rejected to participate, 1191 had haemophilia A and 202 haemophilia B (7 missing data). Out of 1003 adults (mean age ± SD: 35.3 ± 14.4 years), 701 (70.2%) were severely affected, 12% had an inhibitor history, 24.5% were on prophylaxis (225/701 severe patients and 12/278 mild patients), 55.3% received plasma-derived and 29.4% recombinant factor concentrates. Mean overall bleeds were 9.0 ± 12.5 over 6 months, prevalently joint bleeds (7.7 ± 11.4). The mean Orthopaedic Joint Score was 17.5 ± 16.8. Chronic hepatitis C was present in 55.8% of patients, HIV infection in 12.6%. Out of 423 children (mean age ± SD: 10.7 ± 3.4), 279 (67.6%) were severely affected, 16.5% had an inhibitor history, 55.2% were on prophylaxis (196/279 severe patients and 32/134 mild patients), 44.2% received plasma-derived and 43% recombinant factor concentrates. Mean overall bleeds were 4.1 ± 5.8 over 6 months, more than half of these were joint bleeds (2.4 ± 4.5). The mean age at the first bleed was 12.5 ± 12.4 months and at the first joint bleed 24.8 ± 19.6 months). The Paediatric Joint Score was 4.3 ± 9.7. Chronic hepatitis C was present in 2.8% of patients, hepatitis B in 0.9%. Conclusion: ESCHQoL will provide data on the relationship between the clinical status of patients with haemophilia and modalities of treatment. These findings will enable us to identify existing unmet medical needs and to develop European consensus on best practice in haemophilia care
The european study of clinical, health economic and quality of life outcomes in 1426 haemophilia patients / W. Schramm, K. Berger, B. Bullinger, A. Crispin, P. Giangrande, A. Giebl, A. Gringeri, R. Ljung, L. Nemes, S.V. Mackensen, L. Mantovani, M. Serban. - In: JOURNAL OF THROMBOSIS AND HAEMOSTASIS. - ISSN 1538-7933. - 7:Suppl. 2(2009 Jul), pp. 247-248. (Intervento presentato al 22. convegno Congress of the International Society of Thrombosis and Haemostasis tenutosi a Boston, USA nel 2009).
The european study of clinical, health economic and quality of life outcomes in 1426 haemophilia patients
A. Gringeri;L. MantovaniPenultimo
;
2009
Abstract
Background: Prerequisite for optimal haemophilia care provision all around Europe is clearness on clinical status and treatment patterns. This was one of the aims of the European Study of Clinical, Health economic and Quality of Life outcomes in Haemophilia treatment(ESCHQoL). Methods: Retrospective/prospective, multicentre (43 haemophilia centres), multinational (21 European countries) cohort study. Data collection: chart extraction for retrospective data (6 months) and ad hoc patient diaries for prospective data (6 months). Results: Overall 1426 patients were enrolled, 26 rejected to participate, 1191 had haemophilia A and 202 haemophilia B (7 missing data). Out of 1003 adults (mean age ± SD: 35.3 ± 14.4 years), 701 (70.2%) were severely affected, 12% had an inhibitor history, 24.5% were on prophylaxis (225/701 severe patients and 12/278 mild patients), 55.3% received plasma-derived and 29.4% recombinant factor concentrates. Mean overall bleeds were 9.0 ± 12.5 over 6 months, prevalently joint bleeds (7.7 ± 11.4). The mean Orthopaedic Joint Score was 17.5 ± 16.8. Chronic hepatitis C was present in 55.8% of patients, HIV infection in 12.6%. Out of 423 children (mean age ± SD: 10.7 ± 3.4), 279 (67.6%) were severely affected, 16.5% had an inhibitor history, 55.2% were on prophylaxis (196/279 severe patients and 32/134 mild patients), 44.2% received plasma-derived and 43% recombinant factor concentrates. Mean overall bleeds were 4.1 ± 5.8 over 6 months, more than half of these were joint bleeds (2.4 ± 4.5). The mean age at the first bleed was 12.5 ± 12.4 months and at the first joint bleed 24.8 ± 19.6 months). The Paediatric Joint Score was 4.3 ± 9.7. Chronic hepatitis C was present in 2.8% of patients, hepatitis B in 0.9%. Conclusion: ESCHQoL will provide data on the relationship between the clinical status of patients with haemophilia and modalities of treatment. These findings will enable us to identify existing unmet medical needs and to develop European consensus on best practice in haemophilia care
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