Abstract

Introduction
The SIPPET (Survey of Inhibitors in Plasma-Products Exposed Toddlers) trial1 provided evidence that, in previously untreated patients with severe haemophilia A, recombinant factor VIII increases the risk of developing high-titre inhibitors as compared with plasma-derived factor VIII. This multicentre, international study enrolled 264 previously untreated patients (mean age, around 20 months) who were randomised to receive either recombinant factor VIII or plasma-derived factor VIII. Inhibitors developed in 29/125 patients treated with plasma-derived factor VIII (high-titre inhibitors: 20 patients) and in 47/126 patients treated with recombinant factor VIII (high-titre inhibitors: 30 patients). The cumulative rates of all inhibitors were 26.8% with plasma-derived factor VIII (high-titre inhibitors: 18.6%; 95% confidence interval [CI]: 11.2 to 26.0) and 44.5% with recombinant factor VIII (high-titre inhibitors: 28.4%; 95% CI: 19.6 to 37.2). This implies that, in the SIPPET trial, the relative risk reduction for the incidence of high-titre inhibitors was 34.5% for plasma-derived factor VIII compared with recombinant products. All inhibitors occurred before 39 exposure days; all high-titre inhibitors occurred before 34 exposure days (median: 7 to 8 exposure days). [...]

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Authors

Andrea Messori - HTA Unit, ESTAR, Regional Health Service, Florence

Flora Peyvandi - "A. Bianchi Bonomi" Haemophilia and Thrombosis Centre, "Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico", Milan; "Luigi Villa" Foundation, Milan; Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

Sabrina Trippoli - HTA Unit, ESTAR, Regional Health Service, Florence

Roberta Palla - Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

Frits R. Rosendaal - Department of Clinical Epidemiology, Leiden University Medical Centre, Leiden, the Netherlands

Pier Mannuccio Mannucci - "A. Bianchi Bonomi" Haemophilia and Thrombosis Centre, "Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico", Milan; "Luigi Villa" Foundation, Milan; Department of Pathophysiology and Transplantation, University of Milan, Milan, Italy

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