Quality of life in haemophilia

Summary.  There is a growing interest in patient‐reported outcomes as measures for evaluating the benefits of new and existing treatments. Health‐related quality of life (HRQoL) is one of these patient‐reported outcomes and represents the individual experience and perception of illness/health together with the psychosocial response to disease‐related and treatment‐related symptoms. Generic and disease‐specific HRQoL questionnaires enable us to assess and quantify the multi‐dimensional perception of well‐being, namely the physical components and the psychological (emotional, mental, social and behavioural) components of patient’s well‐being and functioning. These instruments should be standardized and validated and they should prove to be reliable, valid, specific and sensitive in a similar manner to instruments created for objective parameters. HRQoL assessment can help us to evaluate the benefits of new treatments from the perspective of patient’s values and expectations. It can also help to evaluate the quality of care provided, in order to be able to improve it at a local and national level. Moreover, HRQoL assessment can be routinely assessed to monitor improvement and progress or deterioration and decline from the global point of view of each single patient, integrating the otherwise limited angle of objective signs and instrumental or lab parameters.