Uncovered needs in the management of inherited bleeding disorders in Italy.

Abstract

Haemophilia is an X-linked bleeding disorder1 and is characterised by repeated bleeding, especially in joints. The current management of haemophilia is based on the replacement therapy with intravenous clotting factor and includes plasma-derived and recombinant factor concentrates2. The early treatment in pediatrics age has been recognised as an important determinant of better physical development3, and several studies have demonstrated that primary prophylaxis has a positive and significant impact on the quality of life and the prevention of arthropathy in haemophiliac children4,5. Also, home treatment has shown to improve both life expectancy and quality of life of patients with haemophilia6. The global treatment of haemophilia, however, requires a multidisciplinary team including haematologists, physicians, surgeons, orthopaedics and other specialists in several medical areas7. This level of clinical assistance is or can be guaranteed only in some countries, and this influences the life expectancy of haemophiliacs8. To ensure a high quality of haemophilia care, an appropriate health organisation and economic resources are necessary. The European Principles of Haemophilia Care (EPHC) were published in 20089. These Principles detail the standard of haemophilia care that should be achieved for an appropriate global care of persons with haemophilia (PWH). The aim of this review is to highlight uncovered needs of Italian haemophilia community, relative to the implementation of each of the EPHC.