Quality of Life in Hemophilia

Abstract

Quality of life (QoL) assessment is increasingly considered an important outcome measure in medicine and is better known as health-related quality of life (HRQoL). HRQoL can be assessed with generic and disease-specific instruments, which must be differentiated according to age groups as measures for adults and measures for children. Recently, the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA) have recommended that HRQoL instruments be developed based on an appropriate and clearly defined framework according to specific guidelines. Only in the last decade, disease-specific QoL instruments have been developed for children and adults with hemophilia. Due to the multiplicity and diversity of instruments, physicians are uncertain which of the existing hemophilia-specific instruments to use. The choice should be based on study-related and instrument-related issues. To interpret HRQoL data precisely, knowledge of the content, scoring, reliability, and validity of a questionnaire is important. Recently, there is a growing interest in the assessment of HRQoL in the field of hemophilia; results are presented for pediatric and adult hemophilia patients.