Lorynn Teela

184 Chapter 7 Table 1. (Predefined) correlations between the PROMIS pediatric measures and the legacy instruments PROMIS pediatric measures Legacy instruments Version Predefined hypothesized correlations Spearman’s correlation Confirmed Pain Interference Haemo-QoL Physical Health 8-12 years ≥0.40 0.49 Yes 13-17 years ≥0.40 0.42 Yes Anxiety Haemo-QoL Feeling 8-12 years ≥0.10 0.60 Yes 13-17 years ≥0.10 0.35 Yes Depressive Symptoms Haemo-QoL Feeling 8-12 years ≥0.40 0.63 Yes 13-17 years ≥0.40 0.29 No Mobility PedHAL 8-17 years ≥0.40 0.41 Yes Peer Relationships Haemo-QoL Other People 8-12 years ≥-0.10 -0.33 Yes 13-17 years ≥-0.10 -0.04 No Anger Haemo-QoL Feeling 8-17 years ≥0.10 0.44 Yes 13-17 years ≥0.10 0.48 Yes Global Health Haemo-QoL total score 8-12 years ≥-0.40 -0.51 Yes 13-17 years ≥-0.40 -0.20 No Note: Predefined correlations were either weak (> 0.10) or moderate (>0.40) based on the content of the items and the domains assessed. Haemo-QoL, Haemophilia Quality of Life Questionnaire for Children; PedHAL, Pediatric Hemophilia Activities List; PROMIS, patient-reported outcomes measurement information system. 3. Results 3.1. Patient characteristics A total of 77 boys with hemophilia participated (response rate: 47.5%). Of these, 70 participants (90.9%) completed all PROMs. The data of one participant was excluded, because this participant ticked the first answer for almost all questions in the PROMs (N=76). Patient characteristics are shown in Table 2. The mean age was 13.5 years (range, 8-17 years). The majority of the participants (86.8%) had hemophilia A. In addition, 40.8% of the participants had a severe form of hemophilia. These participants were treated with prophylaxis with factor concentrates (19 participants) or with emicizumab

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