More data, if not clarity
By Haemnet
It was probably one of the most hotly anticipated sessions of EAHAD 2016. Saturday afternoon the meeting concluded with a Hot Topics session on type of FVIII concentrate and inhibitor formation in PUPs chaired by Jan Astermark, Philippe de Moerloose and Cedric Hermans.” The session contained updates on new data from the randomized SIPPET study (by Flora Peyvandi), and the observational France Coag and PedNet studies (Jenny Goudemand and Johannes Oldenburg, respectively). And Alfonso Iorio was there to comment on how to interpret the new data. It promised so much – could it possibly deliver?
SIPPET, of course, found that the rate of inhibitor formation was higher among those previously untreated patients treated with recombinant products. The France Coag data seemed to support this. But the PedNet data found no difference between plasma-derived and recombinant concentrates with respect to inhibitor formation. But it did seem to offer a reprieve to the second-generation concentrates slated in its earlier analysis (presumably reflecting a higher rate of inhibitors in more recent products).
Spotting the elephant in the room Mike Makris from Sheffield put the cat firmly among the pigeons. He urged the esteemed panel to state how they would treat the next PUP who walked into each of their clinics. Understandably, each panel member wanted to know more detail of the data unearthed by each study, but it seemed clear that none felt inclined to change their practice. Professor Oldenburg said his clinic was likely to remain in the plasma camp, while Professor Astermark thought it unlikely that Malmo would abandon the recombinant world at the present time.
In most disciplines of medicine, well-conducted randomized controlled trials have the power to change practice. Several panelists noted that in many countries, recombinant products cost less than plasma-products. Given that inhibitor formation remains one of the most serious and costly complications of clotting factor use by those with haemophilia we can probably expect a glut of cost-benefit analyses to follow SIPPET. In the current issue of The Journal of Haemophilia Practice, Cedric Hermans summarises his approach to interpreting SIPPET.