Comparing the burden of illness of haemophilia between resource-constrained and unconstrained countries: the São Paulo-Toronto Hemophilia Study

J D A Carneiro; V Blanchette; M C Ozelo; S V Antunes; P R Villaca; N L Young; D Castro; L R Brandão; M Carcao; A Abad; B M Feldman; São Paulo-Toronto Hemophilia Study Group

doi:10.1111/hae.13230

Comparing the burden of illness of haemophilia between resource-constrained and unconstrained countries: the São Paulo-Toronto Hemophilia Study

Haemophilia. 2017 Sep;23(5):682-688. doi: 10.1111/hae.13230. Epub 2017 Apr 24.

Authors

J D A Carneiro¹, V Blanchette^{2

3}, M C Ozelo⁴, S V Antunes⁵, P R Villaca⁶, N L Young⁷, D Castro⁸, L R Brandão⁹, M Carcao⁹, A Abad¹⁰, B M Feldman^{11

12}; São Paulo-Toronto Hemophilia Study Group

Affiliations

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.

PMID: 28440005
DOI: 10.1111/hae.13230

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.

Keywords: factor VIII and IX; global health; haemophilia; outcome measures; prophylaxis.

MeSH terms

Adolescent
Brazil / epidemiology
Canada / epidemiology
Child
Cost of Illness*
Cross-Sectional Studies
Developing Countries*
Health Resources*
Health Status Indicators
Hemophilia A / diagnosis
Hemophilia A / epidemiology*
Hemophilia A / therapy
Humans
Male
Quality of Life
Severity of Illness Index