The History and Evolution of the Clinical Effectiveness of Haemophilia Type A Treatment: A Systematic Review

Abstract

First evidence of cases of haemophilia dates from ancient Egypt, but it was when Queen Victoria from England in the 19th century transmitted this illness to her descendants, when it became known as the ''royal disease''. Last decades of the 20th century account for major discoveries that improved the life expectancy and quality of life of these patients. The history and evolution of haemophilia healthcare counts ups and downs. The introduction of prophylactic schemes during the 1970s have proved to be more effective that the classic on-demand replacement of clotting factors, nevertheless many patients managed with frequent plasma transfusions or derived products became infected with the Human Immunodeficiency Virus (HIV) and Hepatitis C virus during the 1980s and 1990s. Recombinant factor VIII inception has decreased the risk of blood borne infections and restored back longer life expectancies. Main concerns for haemophilia healthcare are shifting from the pure clinical aspects to the economic considerations of long-term replacement therapy. Nowadays researchers' attention has been placed on the future costs and cost-effectiveness of costly long-term treatment. Equity considerations are relevant as well, and alternative options for less affluent countries are under the scope of further research. The aim of this review was to assess the evidence of different treatment options for haemophilia type A over the past four decades, focusing on the most important technological advances that have influenced the natural course of this ''royal disease''.

Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII

“…In moderate cases, patients bleed after injuries, and also suffer spontaneous bleeding episodes without obvious cause. Patients with severe hemophilia have recurrent spontaneous bleeding episodes, in addition to their bleeding complications from normal injuries 9,10 , that can be life-threatening and require immediate medical attention.…”

“…This replacement therapy, however, is limited by frequent intravenous infusion and potential immune response to the infused concentrate, resulting in inhibitory antibodies that leave patients susceptible to increased morbidity and disability 11 . Currently, relatively longer half-lives of recombinant products 12,13 and complex remedy using a combination of multiple activated blood clotting factors 10,11 have been developed to improve the outcomes of replacement therapies.…”

Niños con hemofilia y su atención odontológica por estomatología pediátrica. Revisión de la literatura / Children with Hemophilia and their Dental Care by Pediatric Stomatology. Review of Literature

“…De un total de 115 publicaciones, solo 29 artículos cumplieron con el criterio de inclusión. De estos, 15 se utilizaron para identificar lo más reciente y relevante con respecto al tratamiento estomatológico de niños con hemofilia A (4,6,7,9,(11)(12)(13)(14)(15)(16)(17)(18)(19)(20)(21)(22)(23)(24)(25)(26)(27). Ello incluye adecuada evidencia y guías de práctica (tabla 1).…”

Data Analysis Protocol for the Development and Evaluation of Population Pharmacokinetic Models for Incorporation Into the Web-Accessible Population Pharmacokinetic Service - Hemophilia (WAPPS-Hemo)

“…Modern hemophilia treatment consists of replacement of the deficient factor [3]. Replacement therapy began with the introduction of plasma-derived clotting factor concentrates in the 1960s, and advances in DNA technologies in the 1990s propelled the development of recombinant coagulation factors and the more recent design of longer-lasting recombinant products [4,5].…”