Back to the future: a recent history of haemophilia treatment.
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.
Department of Medicine and Medical Specialties, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, University of Milan and IRCCS Maggiore Hospital, Milan, Italy. piermannuccio.mannucci@unimi.it
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D000293:Adolescent; D000328:Adult; D000818:Animals; D002648:Child; D002986:Clinical Trials as Topic; D005169:Factor VIII / Q000627:therapeutic use*; D015942:Factor VIIa / Q000627:therapeutic use*; D018014:Gene Transfer Techniques / Q000639:trends; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D006467:Hemophilia A / Q000266:history / Q000628:therapy*; D006470:Hemorrhage / Q000266:history / Q000628:therapy*; D049673:History, 20th Century; D006801:Humans; D011994:Recombinant Proteins
D011994:Recombinant Proteins; D005169:Factor VIII; D015942:Factor VIIa