Clinical use of Haemate(R) P in inherited von Willebrand's disease: a cohort study on 100 Italian patients

From the Centro Emofilia e Trombosi "Angelo Bianchi Bonomi", Fondazione IRCCS Ospedale Maggiore, Mangiagalli e Regina Elena, Milano (ABF, PMM); Dipartimento di Terapie cellulari ed Ematologia, Ospedale San Bortolo, Vicenza (GC); Immunoematologia e Trasfusione, Centro Emofilia, Azienda Ospedaliera di Verona, Verona (MF); Agenzia per l’Emofilia, Azienda Ospedaliera Universitaria Careggi, Firenze (MM); Centro Emofilia, Clinica Medica II, Azienda Ospedaliera di Padova, Padova (EZ); Centro di Riferimento Regionale per le Emocoagulopatie, Dipartimento di Medicina Clinica e Sperimentale, Università "Federico II", Napoli (AC); Centro di Riferimento Regionale per la Cura dell’Emofilia e delle Malattie Emorragiche Congenite, Azienda Ospedaliero, Universitaria di Parma, Parma (AT); Centro Emofilia, Istituto Gianna Gaslini, Genova (EB); Ematologia, Dipartimento Biotecnologie Cellulari ed Ematologia, Università "La Sapienza", Roma (MGM); Centro Emofilia, Servizio di Immunoematologia e Trasfusione, Ospedale S. Chiara, Trento (GR) Correspondence: Augusto B. Federici, MD, Centro Emofilia e Trombosi "Angelo Bianchi Bonomi", Dipartimento Medicina Interna e Specialità Mediche, Fondazione IRCCS Ospedale Maggiore, Mangiagalli e Regina Elena, Milano. E-mail: augusto.federici{at}unimi.it Background and Objectives: Plasma-derived concentrates containing von Willebrand factor and factor VIII (VWF/FVIII concentrates) are the mainstay of treatment of patients with inherited von Willebrand’s disease (VWD) who are unresponsive or have a contraindication to desmopressin (DDAVP) therapy. Only a few clinical studies are available on the use of these VWF/FVIII concentrates in large numbers of cases and within the same country. The aim of our study was to collect retrospective data on the efficacy and safety of Haemate ® P (CSL Behring, Marburg, Germany) in a large cohort of well-characterized VWD patients after the introduction of the guidelines for VWD management in Italy. Design and Methods: A retrospective survey of data records was organized among ten Italian Hemophilia Centers in order to retrieve information on the clinical use of Haemate ® P. Data on 100 VWD patients (44 males and 56 females, median age 41.5, range 2–87 years) were available relating to the period from January 2002 to December 2004. All patients were diagnosed according to the criteria proposed by the Italian guidelines for VWD management. Results: Of the 100 VWD patients enrolled, 23 had type 1 VWD, 40 had type 2