Patient-reported outcomes in adults with congenital heart disease: What have we learned from APPROACH-IS?

Patient-reported outcomes (PROs) are “any outcome based on data provided by patients or patient proxy as opposed to data provided from other sources”. Together with clinician-reported, physiological, and caregiver-reported outcomes, PROs are important sources of data to assess health and well-being. Examples of PROs include quality of life, psychosocial well-being, functional status, symptoms, health behaviours, and adherence to treatment. PRO research requires specific methodologies, for which dedicated expertise is needed. In congenital heart disease (CHD), PRO research has received increasing attention over the past decade. This paper summarizes knowledge advancements achieved by the ‘Assessment of Patterns of Patient-Reported Outcomes in Adults with Congenital Heart disease-International Study’ (APPROACH-IS). This was an international project conducted in 15 countries situated on 5 continents, and enrolled more than 4000 patients. Using a uniform research methodology, a broad set of correlates of PROs in CHD was investigated, both at the individual and contextual level. APPROACH-IS confirmed intercountry variation in PROs. Furthermore, several country-level characteristics were associated with PROs in CHD, although they explained little of the variance. Indeed, most of the variance in PROs was explained by individual medical, demographic, behavioural, psychological, and social factors. Although much has been learned about PROs in CHD as a result of APPROACH-IS, this project also generated new questions. The assessment of PROs must thus remain a research priority