Evidence map›Paper›PMID 29644599›Full record

ArticleEndocrine2018

Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy.

Rebecca J Brown, Elif A Oral, Elaine Cochran, David Araújo-Vilar, David B Savage, Alison Long, Gregory Fine, Taylor Salinardi, Phillip Gorden

Open access · greenAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Endocrine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 69 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
69citing papers in PubMed, 2 pooled it
5.6field-weighted citation impact, top 3% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

69 citing papers in PubMed, 2 syntheses or guidelines pooled it, 135 citations in OpenAlex.

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  6. Evaluating the therapeutic impact of tirzepatide in people with partial lipodystrophy.The Journal of clinical endocrinology and metabolism · 2026
    Observational
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9 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

9 authors at 6 institutions in 3 countries.

Rebecca J BrownNational Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA. brownrebecca@niddk.nih.gov.
Elif A OralDepartment of Internal Medicine, University of Michigan Medical School and Health Systems, Ann Arbor, MI, USA.
Elaine CochranNational Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA.
David Araújo-VilarDepartment of Medicine, University of Santiago de Compostela, Santiago de Compostela, Spain.
David B SavageThe University of Cambridge Metabolic Research Laboratories, Wellcome Trust-MRC Institute of Metabolic Science, Cambridge, UK.
Alison LongAegerion Pharmaceuticals, Cambridge, MA, USA.
Gregory FineAegerion Pharmaceuticals, Cambridge, MA, USA.
Taylor SalinardiAegerion Pharmaceuticals, Cambridge, MA, USA.
Phillip GordenNational Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA.
Aegerion Pharmaceuticals (United States) · USNational Institutes of Health · USNational Institute of Diabetes and Digestive and Kidney Diseases · USUniversidade de Santiago de Compostela · ESUniversity of Michigan–Ann Arbor · USWellcome Trust · GB

Funding

Regional Pilot And Feasibility Study Grants ProgramP30DK020572 · NIDDK · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI DAVID P OLSON · 2013 to 2026
$24.3M
Clinical utility of leptin therapy in syndromic forms of insulin resistance.ZIADK047052 · NIDDK · NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES · PI BROWN, REBECCA · 2009 to 2025
$5.5M
Effects of recombinant human leptin in nonalcoholic fatty liver disease (NAFLD)R01DK088114 · NIDDK · UNIVERSITY OF MICHIGAN AT ANN ARBOR · PI CONJEEVARAM, HARI S, ORAL, ELIF ARIOGLU · 2011 to 2015
$1.7M
Intramural NIH HHS Z99 DK999999NIDDK NIH HHS P30 DK020572NIDDK NIH HHS R01 DK088114
6 · The paper itself

Abstract

purposeThe purpose of this study is to summarize the effectiveness and safety of metreleptin in patients with congenital or acquired generalized lipodystrophy.

methodsPatients (n = 66) aged ≥6 months had lipodystrophy, low circulating leptin, and ≥1 metabolic abnormality (diabetes mellitus, insulin resistance, or hypertriglyceridemia). Metreleptin dose (once or twice daily) was titrated to a mean dose of 0.10 mg/kg/day with a maximum of 0.24 mg/kg/day. Means and changes from baseline to month 12 were assessed for glycated hemoglobin (HbA1c), fasting triglycerides (TGs), and fasting plasma glucose (FPG). Additional assessments included the proportions of patients achieving target decreases in HbA1c or fasting TGs at months 4, 12, and 36, medication changes, and estimates of liver size. Treatment-emergent adverse events (TEAEs) were recorded.

resultsSignificant mean reductions from baseline were seen at month 12 for HbA1c (-2.2%, n = 59) and FPG (-3.0 mmol/L, n = 59) and mean percent change in fasting TGs (-32.1%, n = 57) (all p ≤ 0.001). Reductions from baseline over time in these parameters were also significant at month 36 (all p < 0.001, n = 14). At month 4, 34.8% of patients had a ≥1% reduction in HbA1c and 62.5% had a ≥30% reduction in fasting TGs; at month 12, 80% of patients had a ≥1% decrease in HbA1c or ≥30% decrease in TGs, and 66% had a decrease of ≥2% in HbA1c or ≥40% decrease in TGs. Of those on medications, 41%, 22%, and 24% discontinued insulin, oral antidiabetic medications, or lipid-lowering medications, respectively. Mean decrease in liver volume at month 12 was 33.8% (p < 0.001, n = 12). Most TEAEs were of mild/moderate severity.

conclusionsIn patients with generalized lipodystrophy, long-term treatment with metreleptin was well tolerated and resulted in sustained improvements in hypertriglyceridemia, glycemic control, and liver volume.

Indexed as

AdolescentAdultAgedChildChild, PreschoolDose-Response Relationship, DrugFemaleHumansHypertriglyceridemiaInfantInsulin ResistanceLeptinLipodystrophy, Congenital GeneralizedMaleMiddle AgedTreatment OutcomeLeptinmetreleptinDiabetesInsulin resistanceLeptinLipodystrophyMetreleptin

Identifiers

PMID29644599
PMCPMC5936645
OpenAlexW2797973053

What Socratic holds

Textmetadata
LicenceTDM
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the Socratic graph.