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Fig. 2 | Orphanet Journal of Rare Diseases

Fig. 2

From: Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet–Biedl syndrome: phase 3 trial results

Fig. 2

Change in PedsQL and IWQOL-Lite scores after 52 weeks. Spaghetti plots of individual patient HRQOL course in A pediatric and adolescent patients (n = 9) and B adults (n = 11) from active treatment baseline to 52 weeks of treatment. Solid blue lines represent patient-level scores. Dashed pink lines represent the group mean at each visit. Shading indicates threshold for HRQOL impairment (PedsQL: impairment < 68.2; IWQOL-Lite: mild impairment range 79.5–87.0, moderate impairment range 71.9–79.4, severe impairment < 71.8). Only patients with active treatment baseline and 52-week data were included. aIncludes patients who reported PedsQL-Child or PedsQL-Teen. HRQOL, health-related quality of life; IWQOL-Lite, Impact of Weight on Quality of Life Questionnaire-Lite; PedsQL, Pediatric Quality of Life Inventory

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Orphanet Journal of Rare Diseases

ISSN: 1750-1172