Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2018-04-12

AUTHORS

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

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. More... »

PAGES

479-489

Identifiers

URI

http://scigraph.springernature.com/pub.10.1007/s12020-018-1589-1

DOI

http://dx.doi.org/10.1007/s12020-018-1589-1

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1103239955

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/29644599


Indexing Status Check whether this publication has been indexed by Scopus and Web Of Science using the SN Indexing Status Tool
Incoming Citations Browse incoming citations for this publication using opencitations.net

JSON-LD is the canonical representation for SciGraph data.

TIP: You can open this SciGraph record using an external JSON-LD service: JSON-LD Playground Google SDTT

[
  {
    "@context": "https://springernature.github.io/scigraph/jsonld/sgcontext.json", 
    "about": [
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/11", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "name": "Medical and Health Sciences", 
        "type": "DefinedTerm"
      }, 
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/1103", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "name": "Clinical Sciences", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Adolescent", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Adult", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Aged", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Child", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Child, Preschool", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Dose-Response Relationship, Drug", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Female", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Humans", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Hypertriglyceridemia", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Infant", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Insulin Resistance", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Leptin", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Lipodystrophy, Congenital Generalized", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Male", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Middle Aged", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Treatment Outcome", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Young Adult", 
        "type": "DefinedTerm"
      }
    ], 
    "author": [
      {
        "affiliation": {
          "alternateName": "National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA", 
          "id": "http://www.grid.ac/institutes/grid.419635.c", 
          "name": [
            "National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Brown", 
        "givenName": "Rebecca J.", 
        "id": "sg:person.01067352261.31", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01067352261.31"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Department of Internal Medicine, University of Michigan Medical School and Health Systems, Ann Arbor, MI, USA", 
          "id": "http://www.grid.ac/institutes/grid.214458.e", 
          "name": [
            "Department of Internal Medicine, University of Michigan Medical School and Health Systems, Ann Arbor, MI, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Oral", 
        "givenName": "Elif A.", 
        "id": "sg:person.01026535277.58", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01026535277.58"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA", 
          "id": "http://www.grid.ac/institutes/grid.419635.c", 
          "name": [
            "National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Cochran", 
        "givenName": "Elaine", 
        "id": "sg:person.01071763263.76", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01071763263.76"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Department of Medicine, University of Santiago de Compostela, Santiago de Compostela, Spain", 
          "id": "http://www.grid.ac/institutes/grid.11794.3a", 
          "name": [
            "Department of Medicine, University of Santiago de Compostela, Santiago de Compostela, Spain"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Ara\u00fajo-Vilar", 
        "givenName": "David", 
        "id": "sg:person.0725020315.61", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0725020315.61"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "The University of Cambridge Metabolic Research Laboratories, Wellcome Trust-MRC Institute of Metabolic Science, Cambridge, UK", 
          "id": "http://www.grid.ac/institutes/grid.5335.0", 
          "name": [
            "The University of Cambridge Metabolic Research Laboratories, Wellcome Trust-MRC Institute of Metabolic Science, Cambridge, UK"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Savage", 
        "givenName": "David B.", 
        "id": "sg:person.0607043561.67", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0607043561.67"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Aegerion Pharmaceuticals, Cambridge, MA, USA", 
          "id": "http://www.grid.ac/institutes/grid.488226.2", 
          "name": [
            "Aegerion Pharmaceuticals, Cambridge, MA, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Long", 
        "givenName": "Alison", 
        "id": "sg:person.016176172742.69", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.016176172742.69"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Aegerion Pharmaceuticals, Cambridge, MA, USA", 
          "id": "http://www.grid.ac/institutes/grid.488226.2", 
          "name": [
            "Aegerion Pharmaceuticals, Cambridge, MA, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Fine", 
        "givenName": "Gregory", 
        "id": "sg:person.07473121542.70", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.07473121542.70"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Aegerion Pharmaceuticals, Cambridge, MA, USA", 
          "id": "http://www.grid.ac/institutes/grid.488226.2", 
          "name": [
            "Aegerion Pharmaceuticals, Cambridge, MA, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Salinardi", 
        "givenName": "Taylor", 
        "id": "sg:person.01346406420.81", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01346406420.81"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA", 
          "id": "http://www.grid.ac/institutes/grid.419635.c", 
          "name": [
            "National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Gorden", 
        "givenName": "Phillip", 
        "id": "sg:person.011046672577.26", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.011046672577.26"
        ], 
        "type": "Person"
      }
    ], 
    "citation": [
      {
        "id": "sg:pub.10.1007/s40265-013-0074-7", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1021225560", 
          "https://doi.org/10.1007/s40265-013-0074-7"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1007/s12020-014-0450-4", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1021575959", 
          "https://doi.org/10.1007/s12020-014-0450-4"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/onc.2017.107", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1085099160", 
          "https://doi.org/10.1038/onc.2017.107"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1023/a:1021827520301", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1040136713", 
          "https://doi.org/10.1023/a:1021827520301"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/nrendo.2015.123", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1014292895", 
          "https://doi.org/10.1038/nrendo.2015.123"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/372425a0", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1052657184", 
          "https://doi.org/10.1038/372425a0"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1007/s00125-009-1502-9", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1008963706", 
          "https://doi.org/10.1007/s00125-009-1502-9"
        ], 
        "type": "CreativeWork"
      }
    ], 
    "datePublished": "2018-04-12", 
    "datePublishedReg": "2018-04-12", 
    "description": "PurposeThe purpose of this study is to summarize the effectiveness and safety of metreleptin in patients with congenital or acquired generalized lipodystrophy.MethodsPatients (n\u2009=\u200966) aged \u22656 months had lipodystrophy, low circulating leptin, and \u22651 metabolic abnormality (diabetes mellitus, insulin resistance, or hypertriglyceridemia). Metreleptin dose (once or twice daily) was titrated to a mean dose of 0.10\u2009mg/kg/day with a maximum of 0.24\u2009mg/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 (\u20132.2%, n\u2009=\u200959) and FPG (\u20133.0\u2009mmol/L, n\u2009=\u200959) and mean percent change in fasting TGs (\u201332.1%, n\u2009=\u200957) (all p\u2009\u2264\u20090.001). Reductions from baseline over time in these parameters were also significant at month 36 (all p\u2009<\u20090.001, n\u2009=\u200914). At month 4, 34.8% of patients had a \u22651% reduction in HbA1c and 62.5% had a \u226530% reduction in fasting TGs; at month 12, 80% of patients had a \u22651% decrease in HbA1c or \u226530% decrease in TGs, and 66% had a decrease of \u22652% in HbA1c or \u226540% 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\u2009<\u20090.001, n\u2009=\u200912). 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.", 
    "genre": "article", 
    "id": "sg:pub.10.1007/s12020-018-1589-1", 
    "isAccessibleForFree": true, 
    "isFundedItemOf": [
      {
        "id": "sg:grant.2438968", 
        "type": "MonetaryGrant"
      }, 
      {
        "id": "sg:grant.2500127", 
        "type": "MonetaryGrant"
      }, 
      {
        "id": "sg:grant.4579876", 
        "type": "MonetaryGrant"
      }, 
      {
        "id": "sg:grant.2724868", 
        "type": "MonetaryGrant"
      }
    ], 
    "isPartOf": [
      {
        "id": "sg:journal.1112251", 
        "issn": [
          "1355-008X", 
          "1559-0100"
        ], 
        "name": "Endocrine", 
        "publisher": "Springer Nature", 
        "type": "Periodical"
      }, 
      {
        "issueNumber": "3", 
        "type": "PublicationIssue"
      }, 
      {
        "type": "PublicationVolume", 
        "volumeNumber": "60"
      }
    ], 
    "keywords": [
      "treatment-emergent adverse events", 
      "month 12", 
      "generalized lipodystrophy", 
      "month 4", 
      "liver volume", 
      "Most treatment-emergent adverse events", 
      "proportion of patients", 
      "oral antidiabetic medications", 
      "lipid-lowering medications", 
      "treatment of patients", 
      "long-term treatment", 
      "long-term effectiveness", 
      "ConclusionsIn patients", 
      "medication changes", 
      "month 36", 
      "antidiabetic medications", 
      "adverse events", 
      "glycemic control", 
      "metabolic abnormalities", 
      "mean dose", 
      "glycated hemoglobin", 
      "plasma glucose", 
      "moderate severity", 
      "sustained improvement", 
      "patients", 
      "liver size", 
      "PurposeThe purpose", 
      "percent change", 
      "metreleptin", 
      "triglycerides", 
      "lipodystrophy", 
      "medications", 
      "additional assessment", 
      "baseline", 
      "HbA1c", 
      "dose", 
      "treatment", 
      "days", 
      "safety", 
      "MethodsPatients", 
      "hypertriglyceridemia", 
      "decrease", 
      "congenital", 
      "FPG", 
      "abnormalities", 
      "insulin", 
      "severity", 
      "months", 
      "hemoglobin", 
      "ResultsSignificant", 
      "reduction", 
      "glucose", 
      "changes", 
      "volume", 
      "proportion", 
      "assessment", 
      "control", 
      "study", 
      "effectiveness", 
      "events", 
      "improvement", 
      "purpose", 
      "target decreases", 
      "time", 
      "means", 
      "size", 
      "estimates", 
      "parameters", 
      "maximum"
    ], 
    "name": "Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy", 
    "pagination": "479-489", 
    "productId": [
      {
        "name": "dimensions_id", 
        "type": "PropertyValue", 
        "value": [
          "pub.1103239955"
        ]
      }, 
      {
        "name": "doi", 
        "type": "PropertyValue", 
        "value": [
          "10.1007/s12020-018-1589-1"
        ]
      }, 
      {
        "name": "pubmed_id", 
        "type": "PropertyValue", 
        "value": [
          "29644599"
        ]
      }
    ], 
    "sameAs": [
      "https://doi.org/10.1007/s12020-018-1589-1", 
      "https://app.dimensions.ai/details/publication/pub.1103239955"
    ], 
    "sdDataset": "articles", 
    "sdDatePublished": "2022-09-02T16:02", 
    "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
    "sdPublisher": {
      "name": "Springer Nature - SN SciGraph project", 
      "type": "Organization"
    }, 
    "sdSource": "s3://com-springernature-scigraph/baseset/20220902/entities/gbq_results/article/article_760.jsonl", 
    "type": "ScholarlyArticle", 
    "url": "https://doi.org/10.1007/s12020-018-1589-1"
  }
]
 

Download the RDF metadata as:  json-ld nt turtle xml License info

HOW TO GET THIS DATA PROGRAMMATICALLY:

JSON-LD is a popular format for linked data which is fully compatible with JSON.

curl -H 'Accept: application/ld+json' 'https://scigraph.springernature.com/pub.10.1007/s12020-018-1589-1'

N-Triples is a line-based linked data format ideal for batch operations.

curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/pub.10.1007/s12020-018-1589-1'

Turtle is a human-readable linked data format.

curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1007/s12020-018-1589-1'

RDF/XML is a standard XML format for linked data.

curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1007/s12020-018-1589-1'


 

This table displays all metadata directly associated to this object as RDF triples.

302 TRIPLES      21 PREDICATES      118 URIs      103 LITERALS      24 BLANK NODES

Subject Predicate Object
1 sg:pub.10.1007/s12020-018-1589-1 schema:about N0187d08431a34d599bc8d0c59c48dbb3
2 N22197a24a46942fd94bb9d00a2957a40
3 N22c036ca20e04abaa7fadcb0be2bc66a
4 N237e26b7b702495ab73de472bd92c960
5 N3fe34473df9140de8911beeb4aa3994d
6 N55887ef6a86244db851d556bfeba971c
7 N6f11499061c24cb9b7a80c3be44d1dd4
8 N75e269fcc4b240bb9752fd396bcf0b8c
9 N789158bbda5a4a66909914ae29a9ea76
10 Na32e3992e1d54ece8af5daf8834d82a1
11 Nbcc70bfda8fc4b58aa680686e41f7387
12 Nc8788053343c43978714eb03f9dbde77
13 Nd6eeeea360c74c49b8ded485628ba527
14 Ne201b9c08e6746c787d391e965f537c7
15 Ne22f49a3a1d3482dac189359773c4bed
16 Nfe88908e88d243fb80caf2051878c96e
17 Nfe9fa6f517c5481aba8e63b2e098a7f1
18 anzsrc-for:11
19 anzsrc-for:1103
20 schema:author Naf57a5a5d0184ab0b109ffe9a2ebcdd1
21 schema:citation sg:pub.10.1007/s00125-009-1502-9
22 sg:pub.10.1007/s12020-014-0450-4
23 sg:pub.10.1007/s40265-013-0074-7
24 sg:pub.10.1023/a:1021827520301
25 sg:pub.10.1038/372425a0
26 sg:pub.10.1038/nrendo.2015.123
27 sg:pub.10.1038/onc.2017.107
28 schema:datePublished 2018-04-12
29 schema:datePublishedReg 2018-04-12
30 schema:description 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.
31 schema:genre article
32 schema:isAccessibleForFree true
33 schema:isPartOf N4587a372c9714479b8bcf185691f57cb
34 Nb3305a523d074c41b921c3798889c260
35 sg:journal.1112251
36 schema:keywords ConclusionsIn patients
37 FPG
38 HbA1c
39 MethodsPatients
40 Most treatment-emergent adverse events
41 PurposeThe purpose
42 ResultsSignificant
43 abnormalities
44 additional assessment
45 adverse events
46 antidiabetic medications
47 assessment
48 baseline
49 changes
50 congenital
51 control
52 days
53 decrease
54 dose
55 effectiveness
56 estimates
57 events
58 generalized lipodystrophy
59 glucose
60 glycated hemoglobin
61 glycemic control
62 hemoglobin
63 hypertriglyceridemia
64 improvement
65 insulin
66 lipid-lowering medications
67 lipodystrophy
68 liver size
69 liver volume
70 long-term effectiveness
71 long-term treatment
72 maximum
73 mean dose
74 means
75 medication changes
76 medications
77 metabolic abnormalities
78 metreleptin
79 moderate severity
80 month 12
81 month 36
82 month 4
83 months
84 oral antidiabetic medications
85 parameters
86 patients
87 percent change
88 plasma glucose
89 proportion
90 proportion of patients
91 purpose
92 reduction
93 safety
94 severity
95 size
96 study
97 sustained improvement
98 target decreases
99 time
100 treatment
101 treatment of patients
102 treatment-emergent adverse events
103 triglycerides
104 volume
105 schema:name Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy
106 schema:pagination 479-489
107 schema:productId Nac2db4b6fbee4c519ba7582ad4452c72
108 Nb734bb77e51443798a39bf810330a7ff
109 Nefb3c801ecc24354bcbae26574326de5
110 schema:sameAs https://app.dimensions.ai/details/publication/pub.1103239955
111 https://doi.org/10.1007/s12020-018-1589-1
112 schema:sdDatePublished 2022-09-02T16:02
113 schema:sdLicense https://scigraph.springernature.com/explorer/license/
114 schema:sdPublisher N63c3052826d7403e8402d1c11307c842
115 schema:url https://doi.org/10.1007/s12020-018-1589-1
116 sgo:license sg:explorer/license/
117 sgo:sdDataset articles
118 rdf:type schema:ScholarlyArticle
119 N0187d08431a34d599bc8d0c59c48dbb3 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
120 schema:name Lipodystrophy, Congenital Generalized
121 rdf:type schema:DefinedTerm
122 N07990cce48c44cb89ec6297ab7940464 rdf:first sg:person.07473121542.70
123 rdf:rest N13552e12c9e94642bb0759f68a0ea1b1
124 N13552e12c9e94642bb0759f68a0ea1b1 rdf:first sg:person.01346406420.81
125 rdf:rest N3a8c3a33c3d24675a754b7cab12c69e9
126 N22197a24a46942fd94bb9d00a2957a40 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
127 schema:name Adult
128 rdf:type schema:DefinedTerm
129 N22c036ca20e04abaa7fadcb0be2bc66a schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
130 schema:name Middle Aged
131 rdf:type schema:DefinedTerm
132 N237e26b7b702495ab73de472bd92c960 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
133 schema:name Adolescent
134 rdf:type schema:DefinedTerm
135 N327b6257e5844cff94650cd59602cebc rdf:first sg:person.01071763263.76
136 rdf:rest N8031c8fa76654e0e8d6cb3d715f79802
137 N3a8c3a33c3d24675a754b7cab12c69e9 rdf:first sg:person.011046672577.26
138 rdf:rest rdf:nil
139 N3fe34473df9140de8911beeb4aa3994d schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
140 schema:name Child
141 rdf:type schema:DefinedTerm
142 N4587a372c9714479b8bcf185691f57cb schema:issueNumber 3
143 rdf:type schema:PublicationIssue
144 N4cf678296ca04ed69bd1cf7e6aa24bca rdf:first sg:person.016176172742.69
145 rdf:rest N07990cce48c44cb89ec6297ab7940464
146 N526123353421428b9e8dd86098e093bf rdf:first sg:person.0607043561.67
147 rdf:rest N4cf678296ca04ed69bd1cf7e6aa24bca
148 N55887ef6a86244db851d556bfeba971c schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
149 schema:name Child, Preschool
150 rdf:type schema:DefinedTerm
151 N63c3052826d7403e8402d1c11307c842 schema:name Springer Nature - SN SciGraph project
152 rdf:type schema:Organization
153 N6f11499061c24cb9b7a80c3be44d1dd4 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
154 schema:name Dose-Response Relationship, Drug
155 rdf:type schema:DefinedTerm
156 N75e269fcc4b240bb9752fd396bcf0b8c schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
157 schema:name Leptin
158 rdf:type schema:DefinedTerm
159 N789158bbda5a4a66909914ae29a9ea76 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
160 schema:name Young Adult
161 rdf:type schema:DefinedTerm
162 N8031c8fa76654e0e8d6cb3d715f79802 rdf:first sg:person.0725020315.61
163 rdf:rest N526123353421428b9e8dd86098e093bf
164 N88ff2321199741c78ccbc417c97dc03b rdf:first sg:person.01026535277.58
165 rdf:rest N327b6257e5844cff94650cd59602cebc
166 Na32e3992e1d54ece8af5daf8834d82a1 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
167 schema:name Treatment Outcome
168 rdf:type schema:DefinedTerm
169 Nac2db4b6fbee4c519ba7582ad4452c72 schema:name pubmed_id
170 schema:value 29644599
171 rdf:type schema:PropertyValue
172 Naf57a5a5d0184ab0b109ffe9a2ebcdd1 rdf:first sg:person.01067352261.31
173 rdf:rest N88ff2321199741c78ccbc417c97dc03b
174 Nb3305a523d074c41b921c3798889c260 schema:volumeNumber 60
175 rdf:type schema:PublicationVolume
176 Nb734bb77e51443798a39bf810330a7ff schema:name dimensions_id
177 schema:value pub.1103239955
178 rdf:type schema:PropertyValue
179 Nbcc70bfda8fc4b58aa680686e41f7387 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
180 schema:name Aged
181 rdf:type schema:DefinedTerm
182 Nc8788053343c43978714eb03f9dbde77 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
183 schema:name Male
184 rdf:type schema:DefinedTerm
185 Nd6eeeea360c74c49b8ded485628ba527 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
186 schema:name Infant
187 rdf:type schema:DefinedTerm
188 Ne201b9c08e6746c787d391e965f537c7 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
189 schema:name Insulin Resistance
190 rdf:type schema:DefinedTerm
191 Ne22f49a3a1d3482dac189359773c4bed schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
192 schema:name Humans
193 rdf:type schema:DefinedTerm
194 Nefb3c801ecc24354bcbae26574326de5 schema:name doi
195 schema:value 10.1007/s12020-018-1589-1
196 rdf:type schema:PropertyValue
197 Nfe88908e88d243fb80caf2051878c96e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
198 schema:name Hypertriglyceridemia
199 rdf:type schema:DefinedTerm
200 Nfe9fa6f517c5481aba8e63b2e098a7f1 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
201 schema:name Female
202 rdf:type schema:DefinedTerm
203 anzsrc-for:11 schema:inDefinedTermSet anzsrc-for:
204 schema:name Medical and Health Sciences
205 rdf:type schema:DefinedTerm
206 anzsrc-for:1103 schema:inDefinedTermSet anzsrc-for:
207 schema:name Clinical Sciences
208 rdf:type schema:DefinedTerm
209 sg:grant.2438968 http://pending.schema.org/fundedItem sg:pub.10.1007/s12020-018-1589-1
210 rdf:type schema:MonetaryGrant
211 sg:grant.2500127 http://pending.schema.org/fundedItem sg:pub.10.1007/s12020-018-1589-1
212 rdf:type schema:MonetaryGrant
213 sg:grant.2724868 http://pending.schema.org/fundedItem sg:pub.10.1007/s12020-018-1589-1
214 rdf:type schema:MonetaryGrant
215 sg:grant.4579876 http://pending.schema.org/fundedItem sg:pub.10.1007/s12020-018-1589-1
216 rdf:type schema:MonetaryGrant
217 sg:journal.1112251 schema:issn 1355-008X
218 1559-0100
219 schema:name Endocrine
220 schema:publisher Springer Nature
221 rdf:type schema:Periodical
222 sg:person.01026535277.58 schema:affiliation grid-institutes:grid.214458.e
223 schema:familyName Oral
224 schema:givenName Elif A.
225 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01026535277.58
226 rdf:type schema:Person
227 sg:person.01067352261.31 schema:affiliation grid-institutes:grid.419635.c
228 schema:familyName Brown
229 schema:givenName Rebecca J.
230 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01067352261.31
231 rdf:type schema:Person
232 sg:person.01071763263.76 schema:affiliation grid-institutes:grid.419635.c
233 schema:familyName Cochran
234 schema:givenName Elaine
235 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01071763263.76
236 rdf:type schema:Person
237 sg:person.011046672577.26 schema:affiliation grid-institutes:grid.419635.c
238 schema:familyName Gorden
239 schema:givenName Phillip
240 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.011046672577.26
241 rdf:type schema:Person
242 sg:person.01346406420.81 schema:affiliation grid-institutes:grid.488226.2
243 schema:familyName Salinardi
244 schema:givenName Taylor
245 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01346406420.81
246 rdf:type schema:Person
247 sg:person.016176172742.69 schema:affiliation grid-institutes:grid.488226.2
248 schema:familyName Long
249 schema:givenName Alison
250 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.016176172742.69
251 rdf:type schema:Person
252 sg:person.0607043561.67 schema:affiliation grid-institutes:grid.5335.0
253 schema:familyName Savage
254 schema:givenName David B.
255 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0607043561.67
256 rdf:type schema:Person
257 sg:person.0725020315.61 schema:affiliation grid-institutes:grid.11794.3a
258 schema:familyName Araújo-Vilar
259 schema:givenName David
260 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0725020315.61
261 rdf:type schema:Person
262 sg:person.07473121542.70 schema:affiliation grid-institutes:grid.488226.2
263 schema:familyName Fine
264 schema:givenName Gregory
265 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.07473121542.70
266 rdf:type schema:Person
267 sg:pub.10.1007/s00125-009-1502-9 schema:sameAs https://app.dimensions.ai/details/publication/pub.1008963706
268 https://doi.org/10.1007/s00125-009-1502-9
269 rdf:type schema:CreativeWork
270 sg:pub.10.1007/s12020-014-0450-4 schema:sameAs https://app.dimensions.ai/details/publication/pub.1021575959
271 https://doi.org/10.1007/s12020-014-0450-4
272 rdf:type schema:CreativeWork
273 sg:pub.10.1007/s40265-013-0074-7 schema:sameAs https://app.dimensions.ai/details/publication/pub.1021225560
274 https://doi.org/10.1007/s40265-013-0074-7
275 rdf:type schema:CreativeWork
276 sg:pub.10.1023/a:1021827520301 schema:sameAs https://app.dimensions.ai/details/publication/pub.1040136713
277 https://doi.org/10.1023/a:1021827520301
278 rdf:type schema:CreativeWork
279 sg:pub.10.1038/372425a0 schema:sameAs https://app.dimensions.ai/details/publication/pub.1052657184
280 https://doi.org/10.1038/372425a0
281 rdf:type schema:CreativeWork
282 sg:pub.10.1038/nrendo.2015.123 schema:sameAs https://app.dimensions.ai/details/publication/pub.1014292895
283 https://doi.org/10.1038/nrendo.2015.123
284 rdf:type schema:CreativeWork
285 sg:pub.10.1038/onc.2017.107 schema:sameAs https://app.dimensions.ai/details/publication/pub.1085099160
286 https://doi.org/10.1038/onc.2017.107
287 rdf:type schema:CreativeWork
288 grid-institutes:grid.11794.3a schema:alternateName Department of Medicine, University of Santiago de Compostela, Santiago de Compostela, Spain
289 schema:name Department of Medicine, University of Santiago de Compostela, Santiago de Compostela, Spain
290 rdf:type schema:Organization
291 grid-institutes:grid.214458.e schema:alternateName Department of Internal Medicine, University of Michigan Medical School and Health Systems, Ann Arbor, MI, USA
292 schema:name Department of Internal Medicine, University of Michigan Medical School and Health Systems, Ann Arbor, MI, USA
293 rdf:type schema:Organization
294 grid-institutes:grid.419635.c schema:alternateName National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA
295 schema:name National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD, USA
296 rdf:type schema:Organization
297 grid-institutes:grid.488226.2 schema:alternateName Aegerion Pharmaceuticals, Cambridge, MA, USA
298 schema:name Aegerion Pharmaceuticals, Cambridge, MA, USA
299 rdf:type schema:Organization
300 grid-institutes:grid.5335.0 schema:alternateName The University of Cambridge Metabolic Research Laboratories, Wellcome Trust-MRC Institute of Metabolic Science, Cambridge, UK
301 schema:name The University of Cambridge Metabolic Research Laboratories, Wellcome Trust-MRC Institute of Metabolic Science, Cambridge, UK
302 rdf:type schema:Organization
 




Preview window. Press ESC to close (or click here)


...