E Berntorp1, G Dolan2, C Hay3, S Linari4, E Santagostino5, A Tosetto6, G Castaman4,6, M T Álvarez-Román7, R Parra Lopez8, J Oldenburg9, T Albert9, U Scholz10, M Holmström11,12, J-F Schved13, M Trossaërt14, C Hermans15, A Boban15,16, C Ludlam17,18, S Lethagen17,19. 1. Clinical Coagulation Research Unit, Lund University, Malmö, Sweden. 2. St Thomas' Hospital Haemophilia Centre, St Thomas' Hospital, London, UK. 3. Department of Haematology, Manchester Royal Infirmary, Manchester, UK. 4. Center for Bleeding Disorders, Careggi University Hospital, Florence, Italy. 5. Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, IRCCS Ca' Granda Foundation, Maggiore Hospital Policlinico, Milan, Italy. 6. Department of Cell Therapy and Hematology, Hemophilia and Thrombosis Center, San Bortolo Hospital, Vicenza, Italy. 7. Hospital Universitario La Paz, Thrombosis and Haemostasis Unit, Madrid, Spain. 8. Unidad de Hemofilia, Hospital Vall d'Hebrón, Barcelona, Spain. 9. Institut für Experimentelle Hämatologie und Transfusionsmedizin, Universitätsklinik Bonn, Bonn, Germany. 10. Center of Haemostasis, Leipzig, Germany. 11. Coagulation Unit, Hematology Centre Karolinska, Karolinska University Hospital, Stockholm, Sweden. 12. Department of Medicine, Karolinska Institutet, Solna, Sweden. 13. Hôpital Saint Eloi, Laboratoire d'hématologie, Montpellier, France. 14. Centre Régional de Traitement de l'Hémophilie CHU, Nantes, France. 15. Haemostasis and Thrombosis Unit, Haemophilia Clinic St-Luc University Hospital, Brussels, Belgium. 16. Department of Hematology, Medical School of Zagreb, University Hospital Center Zagreb, Zagreb, Croatia. 17. Sobi, Stockholm, Sweden. 18. University of Edinburgh, Edinburgh, UK. 19. Copenhagen University, Copenhagen, Denmark.
Abstract
INTRODUCTION: Haemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available. AIM: To provide an overview of the FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) across Europe. METHODS: Non-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. RESULTS: In 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. CONCLUSION: Treatment 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.
INTRODUCTION:Haemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available. AIM: To provide an overview of the FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) across Europe. METHODS: Non-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. RESULTS: In 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. CONCLUSION: Treatment 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.
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Authors: Gili Kenet; Yeu-Chin Chen; Gillian Lowe; Charles Percy; Huyen Tran; Annette von Drygalski; Marc Trossaërt; Mark Reding; Johannes Oldenburg; Maria Eva Mingot-Castellano; Young-Shil Park; Flora Peyvandi; Margareth C Ozelo; Johnny Mahlangu; Jennifer Quinn; Mei Huang; Divya B Reddy; Benjamin Kim Journal: J Clin Med Date: 2021-12-18 Impact factor: 4.241
Authors: Stacy E Croteau; Michael U Callaghan; Joanna Davis; Amy L Dunn; Michael Guerrera; Osman Khan; Ellis J Neufeld; Leslie J Raffini; Michael Recht; Michael Wang; Alfonso Iorio Journal: Res Pract Thromb Haemost Date: 2018-05-27