Safety and efficacy of turoctocog alfa in the prevention and treatment of bleeds in previously untreated paediatric patients with severe haemophilia A: Results from the guardian 4 multinational clinical trial.

Hassan YaishTadashi MatsushitaMeriem BelhaniVictor Jimenez Yuste Kaan KavakliLars KorsholmIrina MatytsinaClaire PhilippKirsten ReichwaldRunhui Wu

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2019)

Turoctocog alfa was effective at preventing and stopping bleeds and was well tolerated. Inhibitor development was within the expected range for this PUP population.

Keyphrases

clinical trial
replacement therapy
emergency department
intensive care unit
early onset
open label
combination therapy
double blind