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


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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
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