Skip to main content

Table 2 Impact of setmelanotide in children (< 18 years old; self-reported) with baseline and week-52 PedsQL data

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

 

Baseline

Change from baseline at week 52

All patients

(n = 9)

Patients without cognitive impairment

(n = 3)

All patients

(n = 9)

Patients without cognitive impairment

(n = 3)

PedsQL total score, mean (SD) [range]

67.2 (18.9)

[33.7–90.2]

83.3 (2.7)

[80.4–87.0]

 + 11.2 (14.3)

[− 5.2 to 45.6]

 + 3.3 (6.6)

[− 5.2 to 10.9]

PedsQL physical function score, mean (SD) [range]

60.4 (28.1)

[0–93.8]

83.3 (7.8)

[75.0–93.8]

 + 14.0 (27.7)

[− 18.8–68.8]

 + 2.1 (14.7)

[− 18.8–12.5]

PedsQL psychosocial score, mean (SD) [range]

70.7 (16.3)

[46.7–90.0]

83.3 (5.4)

[76.7–90.0]

 + 9.3 (9.9)

[0.0–33.3]

 + 3.9 (4.4)

[0.0–10.0]

BMI Z-score, mean (SD) [range]

3.7 (1.5)

[1.8–7.1]

2.9 (1.4)

[1.8–4.8]

 − 0.7 (0.5)a

[− 0.2 to − 1.9]

 − 1.0 (0.7)a

[− 0.2 to − 1.9]

Maximal hunger, mean (SD) [range]

6.0 (0.8)

[5.0–7.0]

 − 52.7% (23.2)

[− 71.4% to − 20.0%]

  1. aAbsolute change. BMI body mass index, PedsQL pediatric quality of life inventory, SD standard deviation