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SIMTI Servizi srl, Blood Transfusion, 11, p. s82-s85

DOI: 10.2450/2013.013s

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Present and future challenges in the treatment of haemophilia: The patient's perspective

Journal article published in 2013 by Molinari Ac, Romano Arcieri, Stefania Farace, Giuseppe Mazza, Emily Oliovecchio, Angelo C. Molinari, Alberto Garnero, Gabriele Calizzani, Paola Giordano, Lorenzo Mantovani ORCID, Lamberto Manzoli, Paul Giangrande
This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

Haemophilia is a rare bleeding disorder, caused by a mutation in the genes for factor VIII (Haemophilia A) and factor IX (Haemophilia B). Patients with severe haemophilia, with a factor plasma level of 1% or less, are affected by frequent episodes of spontaneous or excessive bleeding into joints and muscles. The current management of haemophilia is based on treatment with plasma-derived and recombinant factor VIII (FVIII) or factor IX products (FIX)1. Home treatment has shown to improve both life expectancy and quality of life of patients with haemophilia and other inherited coagulation disorders (PWH), with a reduction of musculoskeletal damage2.