Development and validation of a measure of disease-specific quality of life in young children with haemophilia.

No disease-specific tool for measuring health-related quality of life (HRQL), an important outcome when assessing medical treatment, has been developed for children with haemophilia. The goal of this study was to develop a parent-administered questionnaire for evaluating quality of life (QOL) in paediatric haemophilia patients between 2 and 6 years of age. After interviewing physicians (5), nurses (5) and parents (10) of children with haemophilia aged between 2 and 6 years, 92 questions were developed and pilot-tested with parents (44) of children with haemophilia to create a 39-question instrument that assessed somatic symptoms, physical functioning, sleep disturbance, stigma, social functioning, fear/resentment, mood/behaviour, restrictions, treatment upset, haemophilia concern and energy level.