J D A Carneiro1, V Blanchette2,3, M C Ozelo4, S V Antunes5, P R Villaca6, N L Young7, D Castro8, L R Brandão9, M Carcao9, A Abad10, B M Feldman11,12. 1. Centro de Hemofilia e Instituto da Criança, Hospital das Clínicas da Faculdade Medcina da Universidade de São Paulo, São Paulo, Brazil. 2. Division of Hematology/Oncology, The Hospital for Sick Children, Toronto, ON, Canada. 3. Department of Pediatrics, University of Toronto, Toronto, ON, Canada. 4. Unit of Hemophilia IHTC 'Cláudio L.P. Correa', INCT do Sangue Hemocentro Unicamp, University of Campinas, Campinas, São Paulo, Brazil. 5. Department of Hematology, Universidade Federal de São Paulo (UNIFESP), São Paulo, Brazil. 6. Service of Hematology, Hospital das Clínicas da Faculdade de Medicina da Universidade de São Paulo, São Paulo, Brazil. 7. School of Rural and Northern Health, Laurentian University, Sudbury, ON, Canada. 8. Department of Diagnostic Imaging, The Hospital for Sick Children, Toronto, ON, Canada. 9. Division of Hematology/Oncology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada. 10. Department of Child Health Evaluative Sciences, The Hospital for Sick Children, Toronto, ON, Canada. 11. Division of Rheumatology, Department of Pediatrics, The Hospital for Sick Children, Toronto, ON, Canada. 12. Institute of Health Policy, Management and Evaluation, The Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada.
Abstract
INTRODUCTION: Although the regular replacement of clotting factor concentrates (prophylaxis) has been well established as the standard of care for severe haemophilia, the high cost of factor concentrates has limited access to prophylaxis in countries with under-developed or developing economies. AIMS: We studied the health gap that could be addressed by providing unlimited access to clotting factor concentrates with implementation of long-term prophylaxis initiated from an early age in life. METHODS: We performed a cross-sectional study of a random, representative sample of boys with moderate and severe haemophilia at three haemophilia treatment centres in Sao Paulo, Brazil, and one centre in Toronto, Canada. RESULTS: Canadian subjects were more often treated with prophylaxis, and began treatment at an earlier age. Fewer Canadian subjects had bleeds within the preceding 6 months (19 vs. 34, P = 0.003). Canadian subjects had lower (better) Pettersson radiographic scores (1.5 vs. 6.0, P = 0.0016), lower (better) Hemophilia Joint Health Scores (5.5 vs. 10.5, P = 0.0038), higher (better) Activity Scale for Kids scores (96.6 vs. 92.0, P = 0.033), more time spent in vigorous activity, and higher (better) social participation scores. CONCLUSIONS: Our findings suggest that increasing access to clotting factor concentrates for young boys with severe haemophilia is a global imperative.
INTRODUCTION: Although the regular replacement of clotting factor concentrates (prophylaxis) has been well established as the standard of care for severe haemophilia, the high cost of factor concentrates has limited access to prophylaxis in countries with under-developed or developing economies. AIMS: We studied the health gap that could be addressed by providing unlimited access to clotting factor concentrates with implementation of long-term prophylaxis initiated from an early age in life. METHODS: We performed a cross-sectional study of a random, representative sample of boys with moderate and severe haemophilia at three haemophilia treatment centres in Sao Paulo, Brazil, and one centre in Toronto, Canada. RESULTS: Canadian subjects were more often treated with prophylaxis, and began treatment at an earlier age. Fewer Canadian subjects had bleeds within the preceding 6 months (19 vs. 34, P = 0.003). Canadian subjects had lower (better) Pettersson radiographic scores (1.5 vs. 6.0, P = 0.0016), lower (better) Hemophilia Joint Health Scores (5.5 vs. 10.5, P = 0.0038), higher (better) Activity Scale for Kids scores (96.6 vs. 92.0, P = 0.033), more time spent in vigorous activity, and higher (better) social participation scores. CONCLUSIONS: Our findings suggest that increasing access to clotting factor concentrates for young boys with severe haemophilia is a global imperative.
Authors: Koyo Usuba; Victoria E Price; Victor Blanchette; Audrey Abad; Carmen Altisent; Loretta Buchner-Daley; Jorge D A Carneiro; Brian M Feldman; Kathelijn Fischer; John Grainger; Susanne Holzhauer; Koon-Hung Luke; Sandrine Meunier; Margareth Ozelo; Ling Tang; Sandra V Antunes; Paula Villaça; Cindy Wakefield; Gilian Wharfe; Runhui Wu; Nancy L Young Journal: Res Pract Thromb Haemost Date: 2019-04-23
Authors: Isolde A R Kuijlaars; Janjaap van der Net; Brian M Feldman; Magnus Aspdahl; Melanie Bladen; Wypke de Boer; Rubén Cuesta-Barriuso; Ruth E D Matlary; Sharon M Funk; Pamela Hilliard; Judy A John; Christine L Kempton; Piet de Kleijn; Marilyn Manco-Johnson; Pia Petrini; Pradeep Poonnoose; Jean St-Louis; Sylvia Thomas; Merel A Timmer; Sonata Saulyte Trakymiene; Leo van Vlimmeren; Kathelijn Fischer Journal: Haemophilia Date: 2020-10-15 Impact factor: 4.287