Trial reportThe Journal of clinical endocrinology and metabolism2013
Metreleptin improves blood glucose in patients with insulin receptor mutations.
Trial report in The Journal of clinical endocrinology and metabolism, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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.
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.
Who cites it
19 citing papers in PubMed, 43 citations in OpenAlex.
- Long-Term Effects of Metreleptin in Rabson-Mendenhall Syndrome on Glycemia, Growth, and Kidney Function.The Journal of clinical endocrinology and metabolism · 2022Trial
- Thyroid Hormone Effects on Glucose Disposal in Patients With Insulin Receptor Mutations.The Journal of clinical endocrinology and metabolism · 2020Trial
- Effects of leptin replacement therapy on pancreatic β-cell function in patients with lipodystrophy.Diabetes care · 2014Trial
- Clinical spectrum of extreme insulin resistance syndromes treated with rhIGF-1: A single-center experience.The Journal of clinical endocrinology and metabolism · 2026Observational
- Reprogramming insulin receptor activation with a de novo agonist to overcome severe insulin resistance.bioRxiv : the preprint server for biology · 2026Article
- Severe Insulin Resistance: Diagnosis and Management.Current diabetes reviews · 2026Review
- Metreleptin In SHORT Syndrome With Uncontrolled Diabetes.JCEM case reports · 2025Article
- INSR novel mutations identified in a Chinese family with severe INSR-related insulin resistance syndromes: A case report.Medicine · 2022Article
- Ovarian Hyperandrogenism and Response to Gonadotropin-releasing Hormone Analogues in Primary Severe Insulin Resistance.The Journal of clinical endocrinology and metabolism · 2021Article
- Anti-Insulin Receptor Antibodies Improve Hyperglycemia in a Mouse Model of Human Insulin Receptoropathy.Diabetes · 2020Article
- Coexistence of ovarian serous papillary cystadenofibroma and type A insulin resistance syndrome in a 14-year-old girl: A case report.World journal of clinical cases · 2020Article
- Thyroid Abnormalities in Patients With Extreme Insulin Resistance Syndromes.The Journal of clinical endocrinology and metabolism · 2019Article
- Glimepiride treatment in a patient with type A insulin resistance syndrome due to a novel heterozygous missense mutation in the insulin receptor gene.Journal of diabetes investigation · 2018Article
- One Novel 2.43Kb Deletion and One Single Nucleotide Mutation of theJournal of clinical research in pediatric endocrinology · 2018Article
- Mecasermin in Insulin Receptor-Related Severe Insulin Resistance Syndromes: Case Report and Review of the Literature.International journal of molecular sciences · 2018Review
- A Mutation in INSR in a Child Presenting with Severe Acanthosis Nigricans.Journal of clinical research in pediatric endocrinology · 2017Article
- Glucagon receptor inhibition normalizes blood glucose in severe insulin-resistant mice.Proceedings of the National Academy of Sciences of the United States of America · 2017Article
- Metreleptin for injection to treat the complications of leptin deficiency in patients with congenital or acquired generalized lipodystrophy.Expert review of clinical pharmacology · 2016Review
- Leptin hormone and its effectiveness in reproduction, metabolism, immunity, diabetes, hopes and ambitions.Journal of medicine and lifeReview
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 2 institutions in 1 country.
Funding
Abstract
contextRabson-Mendenhall syndrome (RMS) is caused by mutations of the insulin receptor and results in extreme insulin resistance and dysglycemia. Hyperglycemia in RMS is very difficult to treat, and patients are at risk for early morbidity and mortality from complications of diabetes.
objectiveOur objective was to study 1-year effects of recombinant human methionyl leptin (metreleptin) in 5 patients with RMS and 10-year effects in 2 of these patients. DESIGN AND
settingWe conducted an open-label nonrandomized study at the National Institutes of Health. PATIENTS: Patients were adolescents with RMS and poorly controlled diabetes.
interventionTwo patients were treated with escalating doses (0.02 up to 0.22 mg/kg/d) of metreleptin for 10 years, including 3 cycles of metreleptin withdrawal and reinitiation. In all 5 patients, 1-year effects of metreleptin (0.22 mg/kg/d) were studied. OUTCOME MEASURES: Hemoglobin A1c (HbA1c) and body mass index (BMI) z-scores were evaluated every 6 months.
resultsHbA1c decreased from 11.4% ± 1.1% at baseline to 9.3% ± 1.9% after 6 months and 9.7% ± 1.6% after 12 months of metreleptin (P = .007). In patients treated for 10 years, HbA1c declined with each cycle of metreleptin and rose with each withdrawal. BMI z-scores declined from -1.4 ± 1.8 at baseline, to -2.6 ± 1.6 after 12 months of metreleptin (P = .0006). Changes in BMI z-score correlated with changes in HbA1c (P < .0001).
conclusionsMetreleptin treatment for 12 months was associated with a 1.7% reduction in HbA1c; part of this improvement was likely mediated via decreased BMI. Metreleptin is a promising treatment option for RMS, but additional therapies are needed to achieve HbA1c targets.
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Registered trials
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 OpenQuestion graph.