179 Psychometrics of the pediatric PROMIS measures in hemophilia 1. Introduction Hemophilia A or B are x-linked bleeding disorders that are caused by a deficiency of the coagulation proteins factor (F) VIII (haemophilia A) or FIX (haemophilia B), resulting in excessive bleeding typically in joints and muscles, spontaneously or after minor trauma. The risk of bleeding is related to the severity of the factor deficiency, and repeated bleeds can cause pain, functional impairment, and acute and longterm disabilities, especially when treated inadequately [1-3]. In recent decades, the treatment of hemophilia has greatly improved. In children with a severe phenotype of hemophilia the treatment is now mainly focused on the prevention of bleeding by prophylactic therapy with factor concentrates (eg, prophylaxis) or non-factor alternatives (eg, emicizumab) [1,4-6]. With these treatment advancements, health outcomes and the health-related quality of life (HRQoL) of children with hemophilia have significantly improved. Children now have a near-normal life expectancy and HRQoL, experience a lower treatment burden, and are less limited in activities of daily living [5,7-10]. However, hemophilia treatment still has an impact on the lives of these children. Therefore, comprehensive care focusing on both physical and psychosocial outcomes is standard in highincome countries [4,11,12]. The use of patient-reported outcomes measures (PROMs) are of important value in comprehensive care to gain insight into the consequences of hemophilia treatment [13]. PROMs are self-reported questionnaires that measure patients’ perspectives on their health, well-being, and the impact of disease and treatment on their life [14,15]. PROMs can be used both at a group level to study differences between populations or to measure the effect of treatment modalities in clinical research, or at an individual level to increase awareness for patients’ problems and concerns, facilitate communication, and to guide clinical decision-making [16-18]. PROMs can be disease-specific (ie, applicable to patients with a specific disease) or generic (ie, applicable to everyone, regardless of disease) and are preferably standardized and validated [19]. For measuring outcomes in children, it is important to have PROMs available for different age ranges and parent proxy questionnaires. In hemophilia research, a wide variety of PROMs are used which makes comparisons difficult due to differences in content, age ranges, and scoring methods [13,15,20-22]. Specifically for pediatric hemophilia care, a wide variety of disease-specific PROMs (eg, CHO-KLAT, Haemo-QoL) are used without established psychometric properties 7
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