Abstract | CONTEXT: OBJECTIVE: To study the effects of recombinant human methionyl leptin ( metreleptin) on bone mineral content (BMC) and mineral metabolism. DESIGN AND SETTING: An open-label nonrandomized study at the National Institutes of Health. PATIENTS: Thirty-one patients with CGL (ages 4.3 to 46.7 y). INTERVENTION:
Metreleptin (0.06 to 0.24 mg/kg/d) for 6 months to 11 years. OUTCOME MEASURES: RESULTS: At baseline, patients demonstrated significantly increased total body less head BMC (mean SDS, 1.8 ± 0.7), height (mean SDS, 1.3 ± 1.3), and lean mass index, defined as lean body mass per height squared (mean SDS, 1.5 ± 0.83), vs population normative data. No change in total body less head BMC was observed after metreleptin. Lean mass index decreased with metreleptin. Serum calcium decreased with metreleptin, but remained within normal limits. No changes were seen in phosphorus, PTH, or vitamin D. CONCLUSIONS: In contrast to rodent models, CGL patients have increased BMC in the leptin-deficient state, which does not change with leptin replacement. The high BMC in these patients is partially explained by high lean mass and tall stature.
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Authors | John D Christensen, Andreea O Lungu, Elaine Cochran, Michael T Collins, Rachel I Gafni, James C Reynolds, Kristina I Rother, Phillip Gorden, Rebecca J Brown |
Journal | The Journal of clinical endocrinology and metabolism
(J Clin Endocrinol Metab)
Vol. 99
Issue 8
Pg. E1493-500
(Aug 2014)
ISSN: 1945-7197 [Electronic] United States |
PMID | 25070319
(Publication Type: Controlled Clinical Trial, Journal Article, Research Support, N.I.H., Intramural)
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Chemical References |
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Topics |
- Absorptiometry, Photon
- Adolescent
- Adult
- Body Composition
(drug effects)
- Bone Density
(drug effects)
- Bone Development
(drug effects)
- Child
- Child, Preschool
- Female
- Hormone Replacement Therapy
- Humans
- Leptin
(analogs & derivatives, pharmacology, therapeutic use)
- Lipodystrophy, Congenital Generalized
(drug therapy, metabolism, physiopathology)
- Male
- Young Adult
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