Impact of a rare disease on Public Health today and tomorrow : the case of haemophilia
(2013)
Files
These_Henrard.pdf
Restricted Access - Adobe PDF
- 2.69 MB
Details
- Authors
- Supervisors
- Abstract
- The aim of this thesis was to study some implications of a rare disease on Public Health in Belgium, taking the example of haemophilia, a rare but well-known haemorrhagic X-linked disease. If untreated, the bleeding tendency in patients with haemophiliac may induce spontaneous bleeding episodes leading to serious complications, including premature death. However, substitutive treatments that consist of intravenous infusions of factor VIII or factor IX clotting concentrates which can be administered in a preventive or curative way are presently available. The first section of this thesis addresses the optimal substitutive treatment of haemophilia, which allows the treatment or the prevention of haemorrhagic complications. In this context, a correct dosing of exogenous coagulation factor VIII is crucial as under-dosing puts patients at risk of haemorrhage, and over-dosing results in a waste of expensive concentrates. We estimated the correct dosing of factor VIII concentrates in patients with haemophilia A in relation to morphometric variables. The second section of this thesis evaluated some current limitations of developing new treatments for haemophilia. Developments of new treatments are indeed only possible through clinical studies requiring the active collaboration of patients. We assessed factors influencing the motivation of patients to participate in clinical studies. Finally, we integrated this work in a general public health perspective by investigating the burden and cost of haemophilia. The consumption of coagulation factors needed to treat haemophilia and its associated cost is steadily increasing in Belgium and other developed countries. Since no global cost estimation of haemophilia is available in our country, we estimated for the first time the total financial burden of haemophilia in Belgium. In addition to cost calculations, we quantified the disease burden related to haemophilia in Belgium, considering the physical and psychological impacts of this rare disease, through Disability-Adjusted Life Year (DALY) calculations.
- Affiliations
Citations
Henrard, S. (2013). Impact of a rare disease on Public Health today and tomorrow : the case of haemophilia. https://hdl.handle.net/2078.5/216214