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

Table 1 Baseline characteristics

	All patients^a	Patients without cognitive impairment
Patients who reported HRQOL assessments, n	20	10
Age, mean (SD) [range], years	22.0 (10.9) [10–44]	23.3 (10.7) [12–43]
Female sex, % [n]	65 (13)	70 (7)
BMI, mean (SD) [range], kg/m²	43.4 (9.6) [24.4–61.4]	45.3 (10.7) [24.4–57.8]
≥ 18 years	47.6 (5.8) [39.4–57.8]	50.2 (5.3) [43.0–57.8]
< 18 years	38.3 (10.7) [24.4–61.4]	34.8 (8.0) [24.4–49.1]
Maximal hunger score, mean (SD)^b	–	6.8 (1.2)

^aIncludes adults and children with and without cognitive impairment. Includes randomized patients who received ≥ 1 dose of setmelanotide or placebo and have baseline and Week 52 data. ^bHunger was evaluated in patients ≥ 12 years without cognitive impairment
BMI body mass index, HRQOL health-related quality of life, SD standard deviation

ISSN: 1750-1172