A prospective study of health-related quality of life of boys with severe haemophilia A in China: comparing on-demand to prophylaxis treatment.

R WuJ SunJ XiaoY LiuF XueH WangL TangY ZhaoK LiR YangY HuK-H LukeM-C PoonV S BlanchetteK UsubaNancy L Young

Published in: Haemophilia : the official journal of the World Federation of Hemophilia (2017)

Child-reported CHO-KLAT scores were lower in boys with severe HA in China than reported in countries with access to full-dose prophylaxis. Boys reported higher HR-QoL scores than their parents. Small improvements in ASK scores were noted following the prophylaxis phase. These changes were only significant in the parent-reported CHO-KLAT scores. Longer term prospective clinical trials are needed in China to determine the impact of prophylaxis on HR-QoL in boys with severe HA.

Keyphrases

clinical trial
early onset
preterm infants
mental health
randomized controlled trial
drug induced
study protocol
gestational age
smoking cessation