Editorial

Abstract

The articles in this supplement report the lectures given at the Bayer Schering Pharma Conference entitled ‘Haemophilia: new challenges and winning directions’ held in Milan on 30 January, 2009. The focus of this meeting was to discuss some hot topics in the field of haemophilia, such as the safety issue and venous access management, and to establish a vision for the future development of haemophilia treatment. The opening lecture was given by Prof. Di Minno on the strategies adopted to achieve longer-acting FVIII molecules that may potentially facilitate enhanced treatment compliance because of the need for less frequent infusions. Preclinical and phase I clinical studies have shown that the most promising formulation is the recombinant FVIII molecule conjugated with pegylated liposomes (BAY 79-4980) and the results of the ongoing phase II multicentre double-blind randomized trial are awaited with great anticipation. The first session of this conference was focused on the issue of viral safety. Professor Zanetti assessed the issue of emerging infections, from severe acute respiratory syndrome (SARS) to influenza pandemics and vector-borne (i.e., West Nile and Chikungunya viruses) viruses. These pathogens, along with prions, may represent a new challenge for haemophilia treatment centres. Consequently, the manufacturing process of recombinant FVIII products has further evolved during the last few years to minimize the risk of blood-borne infections by improving the techniques for purification of the protein, by implementing further viral inactivation steps and by avoiding the use of human or animal proteins at any stage of the production process. In the second session, Dr Coppola analysed the literature data on the genetic and non-genetic risk factors for inhibitor development in haemophilia A patients. The presence of null mutations (large deletions, nonsense mutations and inversions) in the FVIII gene, a positive family history of inhibitors and intensive treatment have been identified as the most relevant risk factors for inhibitor development; on the other hand, several studies have recently outlined the potential protective effect of regular early prophylaxis on inhibitor formation. The second session was dedicated to the management of venous access in haemophilia patients. Dr Santagostino discussed the choice and management of venous access in haemophilic children. Although peripheral veins are the preferred option, central venous access devices (CVADs) are often necessary in children especially in the home setting when frequent infusions for prolonged periods (i.e. prophylaxis or immune tolerance induction regimens) are required. Fully implantable devices have a lower incidence of infections than external CVADs, although they carry the inconvenience of needle sticks. Arteriovenous fistula (AVF) has been recently introduced in haemophilic children as a suitable alternative to central catheters. This latter option was discussed by Professor Berardinelli. Based on the published literature data and on her personal experience, the author concluded that AVF is a suitable venous access for haemophilia children because it is easy to use, is well accepted by children and their parents and has a lower rate of complication compared with CVADs. Professor Izzi reviewed the current literature on the use of CVADs in haemophilia patients. Finally, he presented his personal experience on the use of external tunnelled central venous catheter (CVC) Broviac in haemophilic children documenting the usefulness and safety of this device. M.F. has received fees for the manuscript. E.S. has no conflicts of interest.