Evidence-based recommendations on the treatment of von Willebrand disease in Italy
Pier Mannuccio Mannucci, Massimo Franchini, Giancarlo Castaman, Augusto B Federici, Italian Association of Hemophilia Centers, Laura Contino, Arianna Accorsi, Nicola Ciavarella, Mario Schiavoni, Francesco Antonio Scaraggi, Giuseppina Rodorigo, Lelia Valdre, Roberto Targhetta, Giuseppe Tagariello, Paolo Radossi, Roberto Musso, Dorina Cultrera, Gaetano Muleo, Piergiorgio Iannacaro, Chiara Biasioli, Sophie Testa, Adriano Alatri, Daniele Vincenzi, Gianluigi Scapoli, Massimo Morfini, Angelo Claudio Molinari, Elio Boeri, Daniela Caprino, Grazia Delios, Mauro Girotto, Guglielmo Mariani, Mario Lapecorella, Maria Teresa Carloni, Isabella Cantori, Elena Santagostino, Alessandro Gringeri, Marco Marietta, Paola Pedrazzoli, Giovanni Di Minno, Antonio Coppola, Corrado Perricone, Michele Schiavulli, Angiola Rocino, Mauro Berrettini, Ezio Zanon, Giacomo Mancuso, Sergio Siragusa, Alessandra Malato, Giorgia Saccullo, Annarita Tagliaferri, Franca Rivolta, Alfonso Iorio, Emily Oliovecchio, Francesca Ferrante, Alfredo Dragani, Agostino Rossi, Antonino Mancino, Patrizia Albertini, Silvia Macchi, Marco D'Incà, Giulio De Rossi, Matteo Luciani, Raffaele Landolfi, Maria Gabriella Mazzucconi, Cristina Santoro, Gavino Piseddu, Pier Carla Schinco, Gina Rossetti, Giovanni Barillari, Giulio Feola, Giorgio Gandini, Pier Mannuccio Mannucci, Massimo Franchini, Giancarlo Castaman, Augusto B Federici, Italian Association of Hemophilia Centers, Laura Contino, Arianna Accorsi, Nicola Ciavarella, Mario Schiavoni, Francesco Antonio Scaraggi, Giuseppina Rodorigo, Lelia Valdre, Roberto Targhetta, Giuseppe Tagariello, Paolo Radossi, Roberto Musso, Dorina Cultrera, Gaetano Muleo, Piergiorgio Iannacaro, Chiara Biasioli, Sophie Testa, Adriano Alatri, Daniele Vincenzi, Gianluigi Scapoli, Massimo Morfini, Angelo Claudio Molinari, Elio Boeri, Daniela Caprino, Grazia Delios, Mauro Girotto, Guglielmo Mariani, Mario Lapecorella, Maria Teresa Carloni, Isabella Cantori, Elena Santagostino, Alessandro Gringeri, Marco Marietta, Paola Pedrazzoli, Giovanni Di Minno, Antonio Coppola, Corrado Perricone, Michele Schiavulli, Angiola Rocino, Mauro Berrettini, Ezio Zanon, Giacomo Mancuso, Sergio Siragusa, Alessandra Malato, Giorgia Saccullo, Annarita Tagliaferri, Franca Rivolta, Alfonso Iorio, Emily Oliovecchio, Francesca Ferrante, Alfredo Dragani, Agostino Rossi, Antonino Mancino, Patrizia Albertini, Silvia Macchi, Marco D'Incà, Giulio De Rossi, Matteo Luciani, Raffaele Landolfi, Maria Gabriella Mazzucconi, Cristina Santoro, Gavino Piseddu, Pier Carla Schinco, Gina Rossetti, Giovanni Barillari, Giulio Feola, Giorgio Gandini
Abstract
Background: von Willebrand disease (VWD) is the most common hereditary bleeding disorder affecting both males and females. It arises from quantitative or qualitative defects of von Willebrand factor (VWF) and causes bleeding of mucous membranes and soft tissues. The aim of treatment is to correct the dual defect of haemostasis caused by the abnormal/reduced VWF and the concomitant deficiency of factor VIII (FVIII).
Material and methods: This document contains evidence-based recommendations for the management of VWD compiled by AICE (the Italian Association of Haemophilia Centres). All the evidence supporting these recommendations are based on non-randomised comparative studies or case series, because randomised controlled clinical trials or meta-analyses are not available for this disease.
Results and conclusions: Desmopressin (DDAVP) is the treatment of choice for patients with type 1 VWD with FVIII and VWF levels of 10 U/dL or more, while VWF/FVIII concentrates are indicated for those who are unresponsive or insufficiently responsive to DDAVP (severe type 1, type 2 and 3 VWD). VWF concentrates devoid of FVIII, not yet licensed in Italy, may be considered for short-term prophylaxis in elective surgery or for long-term secondary prophylaxis.
Keywords: desmopressin; von Willebrand disease; von Willebrand factor.
Source: PubMed