Table 3.
Changes in Anthropometric and Metabolic Parameters Compared With Baseline After 52 Weeks of Setmelanotide Treatment: Pivotal Patients With BBS (n=32)
| ≥12 years old (n=28) | ||||
|---|---|---|---|---|
|
| ||||
| ATB | Week 52 | Change from ATB | % change from ATB | |
| Body weight, kg | 115·9 (26·7) | 108·5 (27·0) | −7·4 (8·2) [−10·6, −4·2] p<0·0001 | −6·5 (7·0) [−9·2, −3·8] p<0 p<0·001 |
| Maximal hunger scorea | 7·0 (1·9) | 4·9 (2·5) | −21 (2·0) [−3·3, −0·9] p=0·0010 | −30·5 (26 ·5) [−45·7, −15·2] p=0 ·0004 |
| Achieved ≥25% reduction in | — | 571c [28·9, 82·3] | — | — |
| body weightb, % | p<0·0001 | |||
| ≥1-point reduction, % | — | 71·4 | — | — |
| ≥2-point reduction, % | — | 42·9 | — | — |
|
| ||||
| All ages, ≥6 years old (n=31) | ||||
|
| ||||
| ATB | Week 52 | Change from ATB | % change from ATB | |
|
| ||||
| Waist circumference, cmd | 117·9 (18·0) | 110·3 (21·0) | −7·2 (7·4) [−10·2, −4·1] | −6·3 (7·4) [−9·4, −3·3] |
| Body fat, kge | 51·1 (18·9) | 43·1(16·3) | −5·6 (12·0) [−11·8, 0·5] | −11·3 (26·3) [−24·9, 2·2] |
| Lean muscle, kge | 58·9 (14·1) | 57·6 (12·4) | −12 (3·9) [−3·2, 0·8] | −2·0 (6·5) [−5·4, 1·3] |
| Lipids, mmol/Lf | ||||
| Total cholesterol | 4·4 (1·0) | 3·9 (0·9) | −0·3 (0·4) | −6·1 (10·6) [−22·6, 19·2] |
| HDL cholesterol | 1·1 (0·2) | 1·1 (0·2) | 0·1 (0·1) | 5·3 (11·6) [−14·3, |
| LDL cholesterol | 3·0 (10) | 2·6 (0·9) | −0·2 (0·4) | 30·8] −7·8 (168) [−33·3, |
| Triglycerides | 1·9 (0·9) | 1·4 (0·8) | −0·2 (0·6) | 33·3] −9·6 (32·5) [−69·9,67·2] |
|
| ||||
| ≥18 years old (n=15) | ||||
|
| ||||
| Body weight, kg | 128·4 (16·6) | 119·0 (20·6) | −9·4 (9·4) [−14·6, −4·2] p=0·0008 | −7·6 (−7·1) [−11·5, −36] p=0·0005 |
| BMI, kg/m2g | 46·4 (5·9) | 43·3 (7·2) | −4·2 (3·3) [−6·3, −21] | −9·1(6·8) [−13·4, −4·8] |
|
| ||||
| <18 years old (n=16) | ||||
|
| ||||
| BMI,h kg/m2 | 37·4 (9·4) | 34·2 (10·1) | −3·4 (2·1) [−4·6, −2·2] | −95 (6·4) [−13·2, −5·8] |
| BMI Z scoreh | 3·7 (1·3) | 3·0 (1·5) | −0·8 (0·5) [−1·0, −0·5] | — |
| % 95th BMI percentile | 144·5 (35·8) | 126·8 (37·1) | −17·3 (7·7) [−21·7, −12·9] | — |
Data are the mean (SD), mean [95% CI], or mean (SD) [95% CI] unless otherwise specified. ATB, active treatment baseline; BBS; Bardet-Biedl syndrome; BMI, body mass index; CI, confidence interval; HDL, high-density lipoprotein; LDL, low-density lipoprotein; SD, standard deviation.
Patients ≥12 years old without cognitive impairment and with hunger scores at ATB and Week 52 (n=14).
Multiple imputation model used to impute measurements for patients with <52 weeks of treatment.
Estimated proportion based on qualifying patients in the full analysis set (ie, ≥12 years old without cognitive impairment; n=16).
Patients with measurements at ATB (n=31) and Week 52 (n=25).
Patients with measurements at ATB (n=29), Week 52 (n=18), and both (n=17).
Patients with measurements at ATB (n=31) and Week 52 (n=23).
Patients with measurements at ATB (n=15) and Week 52 (n=12).
Patients with measurements at ATB (n=16) and Week 52 (n=14).