Effects of Metreleptin in Patients With Generalized Lipodystrophy Before vs After the Onset of Severe Metabolic Disease
Authors' conclusion
Patients with GLD who initiated metreleptin before the onset of severe metabolic complications had better long-term control of diabetes, proteinuria, and hypertriglyceridemia. Early treatment may also result is less severe progression of liver fibrosis, but further histological studies are needed to determine the effects of metreleptin therapy on liver disease.
Study context
- Population
- Patients ≥6 months old with congenital or acquired generalized lipodystrophy (GLD) treated with metreleptin at the National Institutes of Health since 2001Patients ≥6 months old with congenital 47 · or acquired 16
- Exposure
- Early vs late metreleptin initiation, defined by baseline metabolic health
- Outcome
- Least squares means of metabolic measures during follow-up, early vs late
- Follow-up
- Median 72 (24-108) months'
Methodological detailssingle-centre
- Single-centre
Main finding
As reported in the abstract
| Group | Value | Insulin AUC | TG (mg/dL) | Urine protein excretion (mg/24 h) | ALT (U/L) | AST (U/L) | AST to Platelet Ratio Index | Platelets (K/µL) |
|---|---|---|---|---|---|---|---|---|
| Early | mean 5.3 (SE 0.3) % | 20 831 ± 1 | 101 ± 1 | 85 ± 1.5 | 30 ± 1 | 23 ± 1 | 0.22 ± 1.3 | 257 ± 24 |
| Late | mean 6.8 (SE 0.3) % | 11 948 ± 1 | 193 ± 1 | 404 ± 1.4 | 53 ± 1 | 40 ± 1 | 0.78 ± 1.3 | 152 ± 28 |
Value
Earlymean 5.3 (SE 0.3) %
Latemean 6.8 (SE 0.3) %
Insulin AUC
Early20 831 ± 1
Late11 948 ± 1
TG (mg/dL)
Early101 ± 1
Late193 ± 1
Urine protein excretion (mg/24 h)
Early85 ± 1.5
Late404 ± 1.4
ALT (U/L)
Early30 ± 1
Late53 ± 1
AST (U/L)
Early23 ± 1
Late40 ± 1
AST to Platelet Ratio Index
Early0.22 ± 1.3
Late0.78 ± 1.3
Platelets (K/µL)
Early257 ± 24
Late152 ± 28
- Early vs Late: P < 0.05 — Early: higher insulin AUC and platelets; lower HbA1c, TG, urine protein, ALT, AST, AST to Platelet Ratio Index
Observational data: associations, not effects.
Further findings
HbA1c and AST by age at baseline (<6 vs ≥6 years) · during follow-up · <6 years: lower HbA1c, higher AST
P < 0.05
AST (U/L) <6 years 40 ± 1 · ≥6 years 23 ± 1
- HbA1c and AST by age at baseline (<6 vs ≥6 years): per_group value = HbA1c
Original publication
Effects of Metreleptin in Patients With Generalized Lipodystrophy Before vs After the Onset of Severe Metabolic Disease. J Clin Endocrinol Metab. Published 17 May 2024. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →