The recently published SIPPET study which looked at the relative risk of developing inhibitors using recombinant factor VIII as opposed to plasma-derived factor VIII containing von Willebrand’s factor demonstrated a significantly higher risk of factor VIII inhibitors when using recombinant factor VIII. This has led to the need for discussion of the best treatment option for newly diagnosed previously untreated patients with severe factor VIII deficiency, especially for the period of their first 50 exposure days. This led to a recommendation stating that there is increasing evidence that the incidence of inhibitors amongst previously–untreated patients (PUP’s) varies between products. Steps should be taken to understand and minimise this risk. (patients, or their parents, should be involved in discussions related to product choice). In Ireland, the decision has been taken to not change treatment practice across the board but the results of this study will be discussed with parents of newly diagnosed children with severe factor VIII deficiency by the clinicians and a decision taken on a case by case basis.
As should be evident from these recommendations and the current reality of care in Ireland, we are fortunate to have standards of treatment and care which greatly exceed the minimum and in most cases approach the optimum level of care. There are improvements to be made and gaps to close but it is useful to be able to benchmark our care with the current reality in Europe and against the new recommendations.
Please click on this link to read the response to the publication of this study, from the European Haemophilia Consortium.