Impact of the sippet study on management of Australian pups

Abstract

Background : The SIPPET (Survey of Inhibitors in Plasma- Products- Exposed Toddlers) Trial appeared to clearly demonstrate a higher rate of inhibitor development in patients initially exposed to a recombinant FVIII product, however the impact of these findings on real- world clinical practice has not been formally assessed. Aims : The aim of this study was to examine Australian prescribing practices in previously untreated patients (PUPs) with severe haemophilia A (SHA) before and after the SIPPET publication. Methods : Patients with SHA on the Australian Bleeding Disorder Registry (ABDR) who received their first ever exposure to FVIII concentrate between 2011 and 2017 were included. Demographic and clinical data (date and indication for first FVIII exposure, product used at first exposure, prophylaxis regimen, inhibitor development, F8 mutation, family history of inhibitor) were collected. Registry data were supplemented by a questionnaire- based survey of haemophilia centre directors regarding their opinion of the relevance of the SIPPET findings. Results : A recombinant FVIII was the initial product choice in 103 PUPs (95.4%). Only 5 PUPs were initially prescribed plasma- derived FVIII (2 before and 3 after release of the SIPPET findings) and these patients appeared to be selected based on higher risk F8 mutation and/or family history. Of the 5 PUPs prescribed plasma- derived FVIII, 4 developed an inhibitor. Only 3 of 8 haemophilia centre directors surveyed felt that SIPPET findings were potentially relevant to their own practice. Conclusions : The findings of the SIPPET study have not had any overt influence over Australian practice with regard to initial product choice for PUPs.L6288139472019-08-09 <br />