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Achieving the unima...
Achieving the unimaginable : Health equity in haemophilia
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- Skinner, Mark W. (author)
- Institute for Policy Advancement Ltd,McMaster University
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- Nugent, Diane (author)
- University of California, Irvine,Children's Hospital of Orange County
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- Wilton, Pam (author)
- Canadian Hemophilia Society (CHS)
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- O’Mahony, Brian (author)
- Irish Haemophilia Society,Trinity College Dublin
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- Dolan, Gerry (author)
- Guy's and St Thomas' NHS Foundation Trust
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- O’Hara, Jamie (author)
- University of Chester,HCD Economics Ltd
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- Berntorp, Erik (author)
- Lund University,Lunds universitet,Klinisk koagulationsmedicin, Malmö,Forskargrupper vid Lunds universitet,Clinical Coagulation, Malmö,Lund University Research Groups,Skåne University Hospital
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(creator_code:org_t)
- 2019-11-13
- 2020
- English 8 s.
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In: Haemophilia. - : Wiley. - 1351-8216 .- 1365-2516. ; 26:1, s. 17-24
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Abstract
Subject headings
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Klinisk medicin -- Hematologi (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Clinical Medicine -- Hematology (hsv//eng)
Keyword
- haemophilia
- outcomes
- prophylaxis
- replacement factor
Publication and Content Type
- for (subject category)
- ref (subject category)
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