Back to the future: a recent history of haemophilia treatment
- 16 May 2008
- journal article
- review article
- Published by Wiley in Haemophilia
- Vol. 14 (s3), 10-18
- https://doi.org/10.1111/j.1365-2516.2008.01708.x
Abstract
In the last few decades, the management of patients with haemophilia has witnessed dramatic improvements, through the larger availability of safe plasma-derived and recombinant products for replacement therapy. Another important step forward is the progressively larger-scale implementation of primary prophylaxis in children. Currently, the main problem in patients with haemophilia is the onset of antibodies inactivating the infused clotting factor (inhibitors), even though immune tolerance regimens that are able to eradicate inhibitors and the availability of products that bypass the intrinsic coagulation defects have dramatically improved the management of these patients. Cure of haemophilia through gene transfer is being attempted, but relatively, it is far from being implemented on a large scale. It is likely that further improvements in replacement therapy will occur in the near future, through the availability of new-therapeutic tools such as factors VIII and IX with longer half-lives, more potent bypassing agents and factors extracted from the milk of transgenic animals.Keywords
This publication has 62 references indexed in Scilit:
- Prophylaxis versus Episodic Treatment to Prevent Joint Disease in Boys with Severe HemophiliaThe New England Journal of Medicine, 2007
- Early factor VIII exposure and subsequent inhibitor development in children with severe haemophilia AHaemophilia, 2007
- Update on Progress and Hurdles in Novel Genetic Therapies for HemophiliaHematology, 2007
- Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodiesJCI Insight, 2006
- Ethical issues in haemophiliaHaemophilia, 2006
- Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatmentBlood, 2003
- Use of Recombinant Antihemophilic Factor in the Treatment of Two Patients with Classic HemophiliaThe New England Journal of Medicine, 1989
- T-Lymphocyte Subpopulations in Patients with Classic Hemophilia Treated with Cryoprecipitate and Lyophilized ConcentratesThe New England Journal of Medicine, 1983
- Impaired Cell-Mediated Immunity in Patients with Classic HemophiliaThe New England Journal of Medicine, 1983
- The Effect of Activated Prothrombin-Complex Concentrate (FEIBA) on Joint and Muscle Bleeding in Patients with Hemophilia A and Antibodies to Factor VIIIThe New England Journal of Medicine, 1981