25 PROMs in autosomal inherited bleeding disorders: A systematic literature review 2 INTRODUCTION The trend towards a more value-based healthcare system has led to an increasing emphasis on outcome measurement. Especially in the last few years, the interest to incorporate patient-reported outcomes (PROs) in medical practice and research has grown exponentially. 1-3 PROs are defined as “any report coming directly from patients, without interpretation by physicians or persons, about how the patient functions or feels in relation to a health condition and its treatment” 4. There are several types of PROs with the most common being self-reported symptoms, self-reported functioning, and healthrelated quality of life (HRQoL) 5. HRQoL is a broad multidimensional concept that incorporates various domains (e.g., physical, psychologic and social functioning) related to the health status of an individual. 6 PROs are often assessed using questionnaires, otherwise known as patientreported outcomes measures (PROMs) 7. PROMs can be classified as either generic or as disease-specific. Generic PROMs, such as Euro-QoL EQ-5D, consist of questions relevant to multiple disease groups or a healthy population. In contrary, disease specific PROMs focus on particular patient groups and consist of questions that are only related to a given disease, disability or surgery. 7,8 Incorporating PROs in clinical practice enables 1) the evaluation of the effectiveness of a healthcare intervention, 2) the assessment of the quality of care and the needs of different populations, 3) the improvement of clinical decision making, and 4) a better understanding and causes of variations in health 7,9. In addition, monitoring PROs may enhance patient engagement and shared decision making, which subsequently leads to a higher quality of care and more patient-centered care 8,10,11. For example, studies in oncology have shown that the systematic collection of PROs result in better symptom control, fewer hospitalizations and better quality of life 12. The increased attention on more patient-centered approaches in healthcare has led to more studies examining PROs and PROMs in a variety of diseases, including in patients with inherited bleeding disorders. Inherited bleeding disorders consist of a heterogeneous group of diseases affecting the primary and secondary hemostasis that include abnormalities or deficiencies of platelets or coagulation proteins. As hemophilia A and B are X-linked, the autosomal inherited bleeding disorders include von Willebrand disease (VWD), inherited platelet function disorders and various coagulation factor
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