On 2 March 2015, the European Haemophilia Consortium (EHC) hosted its first Round Table of the year at the Royal Windsor Hotel in Brussels. The event aimed to present an overview of the current findings on the risks associated with inhibitor development when using different types of factor concentrates in previously untreated patients (PUPs). The event featured several presentations summing up findings from studies such as the Research Of Determinants of INhibitor Development among PUPs with haemophilia (RODIN study), the UKHCDO, FranceCoag and the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET study).
Prof Michael Makris, member of the EHC Medical Advisory Group and chair of the event, stated in his opening address that inhibitors are the real challenge in haemophilia care today. In fact, the mechanism and factors contributing to inhibitor formation are still poorly understood by the medical and scientific community globally. The development of inhibitors has devastating consequences for patients both physically and emotionally as it leaves people affected by inhibitors at greater risk of disability and death. Furthermore, inhibitors are extremely expensive to treat, which results in even poorer access to treatment in resource-limited countries.
The Round Table showed that it is believed that inhibitor development is a multi-factorial event in which both genetic and external factors, such as treatment regimens, contribute to inhibitor formation. Professor Pier-Mannuccio Mannucci (University of Milan) discussed the controversial issue of whether recombinant concentrates cause more inhibitors in PUPs than plasma derived products. He ended by presenting the SIPPET trial, which is examining this issue in a randomised manner and the results of which are awaited with great interest.
The issue of whether different recombinant concentrates are associated with different inhibitor risks was discussed by the other speakers. Professor Jenny Goudemand (University of Lille) presented the data of the RODIN, UKHCDO and FranceCoag studies which suggested that second generation concentrates had a higher inhibitor risk in PUPs. Dr Kathelijn Fischer (University of Utrecht) showed the EUHASS data, which did not find a difference between the different brands. Dr Alfonso Iorio (McMaster University) discussed all the studies to date and suggested that the issue may not be as clear cut as has been suggested. Prof Frits Rosendaal (University of Leiden), invoked the in dubio abstine principle of modern medicine and recommended to avoid using any product against which concern has been raised, if an alternative is available. Other speakers, however, claimed that the only way to be sure about whether or not a particular product has an increased effect on inhibitor formation is to run a randomised clinical trial.
The documents of the event can be consulted here.