BAY 81-8973 Efficacy and Safety in Previously Untreated and Minimally Treated Children with Severe Hemophilia A: The LEOPOLD Kids Trial.

Rolf LjungAnthony K C ChanHeidi GlosliOlubunmi AfonjaBastian BeckerDespina Tseneklidou-StoeterMaria Elisa MancusoSonata Saulyte-TrakymieneGili Kenet

Published in: Thrombosis and haemostasis (2023)

BAY 81-8973 was effective for bleed prevention and treatment in PUPs/MTPs. The observed inhibitor rate was strongly influenced by a cluster of inhibitor cases, and consequently, slightly higher than in other PUP/MTP studies. Overall, the BAY 81-8973 benefit-risk profile remains unchanged and supported by ongoing safety surveillance. Immune tolerance can be achieved with BAY 81-8973.

Keyphrases

water quality
public health
young adults
clinical trial
study protocol
randomized controlled trial
phase iii
combination therapy