title: Treatment of hemophilia: a review of current advances and ongoing issues.
abstract: Replacement of the congenitally deficient factor VIII or IX through plasma-derived or recombinant concentrates is the mainstay of treatment for hemophilia. Concentrate infusions when hemorrhages occur typically in joint and muscles (on-demand treatment) is able to resolve bleeding, but does not prevent the progressive joint deterioration leading to crippling hemophilic arthropathy. Therefore, primary prophylaxis, ie, regular infusion of concentrates started after the first joint bleed and/or before the age of two years, is now recognized as first-line treatment in children with severe hemophilia. Secondary prophylaxis, whenever started, aims to avoid (or delay) the progression of arthropathy and improve patient quality of life. Interestingly, recent data suggest a role for early prophylaxis also in preventing development of inhibitors, the most serious complication of treatment in hemophilia, in which multiple genetic and environmental factors may be involved. Treatment of bleeds in patients with inhibitors requires bypassing agents (activated prothrombin complex concentrates, recombinant factor VIIa). However, eradication of inhibitors by induction of immune tolerance should be the first choice for patients with recent onset inhibitors. The wide availability of safe factor concentrates and programs for comprehensive care has now resulted in highly satisfactory treatment of hemophilia patients in developed countries. Unfortunately, this is not true for more than two-thirds of persons with hemophilia, who live in developing countries.
authors.affiliation: Regional Reference Center for Coagulation Disorders, Federico II University Hospital, Naples, Italy.
authors.forename: Antonio
authors.initials: A
authors.lastname: Coppola
authors.forename: Mirko
authors.initials: M
authors.lastname: Di Capua
authors.forename: Matteo Nicola Dario
authors.initials: MN
authors.lastname: Di Minno
authors.forename: Mariagiovanna
authors.initials: M
authors.lastname: Di Palo
authors.forename: Emiliana
authors.initials: E
authors.lastname: Marrone
authors.forename: Paola
authors.initials: P
authors.lastname: Ieranò
authors.forename: Claudia
authors.initials: C
authors.lastname: Arturo
authors.forename: Antonella
authors.initials: A
authors.lastname: Tufano
authors.forename: Anna Maria
authors.initials: AM
authors.lastname: Cerbone