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Achieving the unimaginable: Health equity in haemophilia
Skinner, Mark W. Nugent, Diane Wilton, Pam O’Mahony, Brian Dolan, Gerry O’Hara, Jamie Berntorp, Erik
Skinner, Mark W.
Nugent, Diane
Wilton, Pam
O’Mahony, Brian
Dolan, Gerry
O’Hara, Jamie
Berntorp, Erik
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Editors
Other Contributors
EPub Date
Publication Date
2019-11-13
Submitted Date
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Article - VoR
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Abstract
Historically, treatment based on the availability of clotting factor replacement has resulted in an arcane guideline for the correction of factor deficiencies in people with haemophilia (PwH). While all other disease entities seek to restore function to a normal level, PwH are restricted to factor nadirs still equivalent to mild or moderate disease, resulting in continued risk of bleeding. A new treatment paradigm is needed based on the defined needs of PwH. A treatment model was developed by a panel of haemophilia providers, patient advocates and health economists to establish specific treatment milestones and targeted outcomes. The panel defined a series of treatment milestones to characterize the activity and outcomes linked to level of factor deficiency correction. All agreed that the ultimate goal should be ‘functional cure’ and ‘health equity’. Seven levels to achieving a functional cure were identified, (a) Sustain life; (b) Minimal joint impairment; (c) Freedom from any spontaneous bleeds; (d) Attainment of ‘normal’ mobility; (e) Able to sustain minor trauma without additional intervention; (f) Ability to sustain major surgery or trauma; and (g) Normal haemostasis. A parallel set of patient-reported outcomes to achieve health equity was identified. These guidelines are now comparable with other disorders where the goal is to replace missing proteins to attain normal activity levels. As we are no longer limited by plasma supply due to the manufacture of recombinant factors, mimetics, and the early success of gene therapy, health equity is now achievable.
Citation
Skinner, M. W., Nugent, D., Wilton, P., O’Mahony, B., Dolan, G., O’Hara, J., & Berntorp, E. (2020). Achieving the unimaginable: Health equity in haemophilia. Haemophilia, 26(1), 17-24. https://doi.org/10.1111/hae.13862
Publisher
Wiley
Journal
Haemophilia
Research Unit
DOI
10.1111/hae.13862
PubMed ID
PubMed Central ID
Type
Article
Language
Description
© 2019 The Authors.
Series/Report no.
ISSN
1351-8216
EISSN
1365-2516