title: Efficacy and safety of highly purified, doubly virus-inactivated VWF/FVIII concentrates in inherited von Willebrand's disease: results of an Italian cohort study on 120 patients characterized by bleeding severity score.
abstract: The efficacy of highly purified VWF/FVIII concentrates with standardized ristocetin cofactor content (VWF:RCo) has been already proven in patients with von Willebrand's disease (VWD). Aim of this retrospective study is to confirm efficacy and safety of two highly purified, doubly virus-inactivated VWF/FVIII concentrates in a large cohort of patients with VWD who were characterized at enrolment by bleeding severity score. Study drugs Alphanate or Fanhdi were given to 120 cases (51 males, 69 females, median age 50 years, range 6-83 years). Patients had VWD3 (10), VWD2A (19), VWD2B (25), VWD2M (10) and DDAVP-unresponsive VWD1 (56) and a median bleeding severity score of 8 (range 0-27). A total of 114 bleeding episodes in 55 cases and 131 surgical procedures in 85 cases could be analysed. Excellent-good clinical responses were seen in 97% of bleeding episodes and in 99% of surgical procedures. To prevent recurrent gastrointestinal (GI) bleeding, cerebral (CNS) haemorrhage, haemarthroses, urogenital or multisite bleeding in more severe patients, secondary prophylaxis was also carried out in 15 cases with VWD3 (3), VWD2A (3), VWD2B (2), VWD1 (7). A median dose of 42 IU VWF:RCo kg(-1) given every other day or twice a week over a median period of 334 days (range 24-799) prevented bleeding completely in 13 cases and reduced its incidence in the remaining two. These results confirm the efficacy and safety of the study concentrates, not only in the management of bleeding and surgery but also in secondary prophylaxis of severe VWD.
authors.affiliation: Angelo Bianchi Bonomi Hemophilia Thrombosis Center, Department of Medicine and Medical Specialties, IRCCS Foundation Ospedale Maggiore, Mangiagalli, Regina Elena and Universita degli Studi di Milano, Via Pace 9, Milan, Italy. augusto.federici@unimi.it
authors.forename: A B
authors.initials: AB
authors.lastname: Federici
authors.forename: G
authors.initials: G
authors.lastname: Barillari
authors.forename: E
authors.initials: E
authors.lastname: Zanon
authors.forename: M G
authors.initials: MG
authors.lastname: Mazzucconi
authors.forename: R
authors.initials: R
authors.lastname: Musso
authors.forename: R
authors.initials: R
authors.lastname: Targhetta
authors.forename: P M
authors.initials: PM
authors.lastname: Mannucci
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D016063:Blood Loss, Surgical / Q000517:prevention & control; D002648:Child; D015331:Cohort Studies; D004338:Drug Combinations; D005169:Factor VIII / Q000627:therapeutic use*; D005260:Female; D006470:Hemorrhage / Q000453:epidemiology / Q000517:prevention & control; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D012720:Severity of Illness Index; D038301:Virus Inactivation; D055815:Young Adult; D014842:von Willebrand Diseases / Q000188:drug therapy*; D014841:von Willebrand Factor / Q000627:therapeutic use*
chemical_list: D004338:Drug Combinations; D014841:von Willebrand Factor; D005169:Factor VIII