title: Rare bleeding disorders: worldwide efforts for classification, diagnosis, and management.
abstract: Rare bleeding disorders (RBDs) comprise the inherited deficiencies of coagulation factors such as fibrinogen, factor (F)II, FV, FV + FVIII, FVII, FX, FXI, and FXIII, and are usually transmitted as autosomal recessive disorders. RBDs are characterized by a wide variety of symptoms from mild to severe; however, due to their rarity, only little information is available on the adequate management of patients affected with these deficiencies. Moreover, the limitations of laboratory assays and the lack of a definitive consensus concerning their classification have prevented adoption of optimal approaches to their individual management. To overcome these limitations, new strategies are therefore necessary, such as the establishment of global collaborations and networks among treatment centers, as well as increasing support provided by public health organizations.
authors.affiliation: A. Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy. flora.peyvandi@unimi.it
authors.forename: Flora
authors.initials: F
authors.lastname: Peyvandi
authors.forename: Marzia
authors.initials: M
authors.lastname: Menegatti
authors.forename: Roberta
authors.initials: R
authors.lastname: Palla
mesh_terms: D001778:Blood Coagulation Disorders / Q000145:classification / Q000175:diagnosis* / Q000628:therapy*; D025861:Blood Coagulation Disorders, Inherited / Q000175:diagnosis / Q000628:therapy; D019411:Clinical Laboratory Techniques / Q000379:methods; D020147:Coagulation Protein Disorders / Q000175:diagnosis / Q000628:therapy; D006801:Humans; D007391:International Cooperation