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Novel Insights from Clinical Practice

Rapid Improvement of Hepatic Steatosis after Initiation of Leptin Substitution in a Leptin-Deficient Girl

von Schnurbein J.a, b · Heni M.c · Moss A.a, b · Nagel S.A.a, b · Machann J.d, e · Muehleder H.f · Debatin K.M.b · Farooqi S.g · Wabitsch M.a, b

Author affiliations

aDivision of Pediatric Endocrinology and Diabetes at bDepartment of Pediatrics and Adolescent Medicine, University Medical Center Ulm, Ulm, cDivision of Endocrinology, Diabetology, Angiology, Nephrology and Clinical Chemistry, Department of Internal Medicine and dSection on Experimental Radiology, Department of Diagnostic Radiology, Eberhard Karls University Tübingen, and eInstitute for Diabetes Research and Metabolic Diseases - Metabolic Imaging, Helmholtz Center Munich, University of Tübingen, Paul Langerhans Institute, Tübingen, Germany; fChildren's Hospital Wels, Wels, Austria; gUniversity of Cambridge Metabolic Research Laboratories, Institute of Metabolic Science, Addenbrooke's Hospital, Cambridge, UK

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Horm Res Paediatr 2013;79:310-317

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Article / Publication Details

First-Page Preview
Abstract of Novel Insights from Clinical Practice

Received: October 23, 2012
Accepted: February 01, 2013
Published online: May 07, 2013
Issue release date: June 2013

Number of Print Pages: 8
Number of Figures: 1
Number of Tables: 1

ISSN: 1663-2818 (Print)
eISSN: 1663-2826 (Online)

For additional information: https://www.karger.com/HRP

Abstract

Background: Leptin deficiency is associated with severe obesity and metabolic disturbances. Increased liver fat content has been reported in only one case beforehand, even though hepatic steatosis is a typical comorbidity of common obesity. It is also frequent in patients with lipodystrophy where it resolves under leptin therapy. Subject and Methods: In 2010, we reported a leptin-deficient patient with a novel homozygous mutation in the leptin gene and severe hepatic steatosis. We have now studied serum changes and changes in liver fat content during the substitution with recombinant methionyl human leptin. Results: After 23 weeks of leptin substitution, elevated transaminases, total cholesterol and low-density lipoprotein levels normalized. After 62 weeks, homeostasis model assessment of insulin resistance improved from 10.7 to 6.0 and body fat mass dropped from 50.2 to 37.8%. Liver fat content was drastically reduced from 49.7 to 9.4%. The first changes in liver fat content were detectable after 3 days of therapy. Conclusion: Our patient showed a remarkable reduction of liver fat content during the treatment with recombinant methionyl human leptin. These changes occurred rapidly after initiation of the substitution, which implies that leptin has a direct effect on hepatic lipid metabolism in humans as it is seen in rodents.

© 2013 S. Karger AG, Basel


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Article / Publication Details

First-Page Preview
Abstract of Novel Insights from Clinical Practice

Received: October 23, 2012
Accepted: February 01, 2013
Published online: May 07, 2013
Issue release date: June 2013

Number of Print Pages: 8
Number of Figures: 1
Number of Tables: 1

ISSN: 1663-2818 (Print)
eISSN: 1663-2826 (Online)

For additional information: https://www.karger.com/HRP


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