Comorbidities and Survival in Patients With Lipodystrophy: An International Chart Review Study.

Autor:	Akinci B; Dokuz Eylül University, Izmir, Turkey., Oral EA; Division of Metabolism, Endocrine & Diabetes and Brehm Center for Diabetes Research, Department of Internal Medicine, Michigan Medicine, University of Michigan, Ann Arbor, Michigan., Neidert A; Division of Metabolism, Endocrine & Diabetes and Brehm Center for Diabetes Research, Department of Internal Medicine, Michigan Medicine, University of Michigan, Ann Arbor, Michigan., Rus D; Division of Metabolism, Endocrine & Diabetes and Brehm Center for Diabetes Research, Department of Internal Medicine, Michigan Medicine, University of Michigan, Ann Arbor, Michigan., Cheng WY; Analysis Group Inc., Boston, Massachusetts., Thompson-Leduc P; Analysis Group Inc., Boston, Massachusetts., Cheung HC; Analysis Group Inc., Boston, Massachusetts., Bradt P; Aegerion Pharmaceuticals Inc., Cambridge, Massachusetts., Foss de Freitas MC; University of São Paulo-Ribeirao Preto Medical School, São Paulo, Brazil., Montenegro RM; Federal University of Ceará, Ceará, Brazil., Fernandes VO; Federal University of Ceará, Ceará, Brazil., Cochran E; National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, Maryland., Brown RJ; National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, Maryland.
Jazyk:	angličtina
Zdroj:	The Journal of clinical endocrinology and metabolism [J Clin Endocrinol Metab] 2019 Nov 01; Vol. 104 (11), pp. 5120-5135.
DOI:	10.1210/jc.2018-02730
Abstrakt:	Context: Limited natural history data are available in patients with non-HIV-related lipodystrophy syndromes who never received disease-specific therapies, making interpretation of benefits of therapies in lipodystrophy syndromes challenging. Objective: We assessed the natural history of non-HIV-related generalized lipodystrophy (GL) and partial lipodystrophy (PL) in patients who have never received leptin or other lipodystrophy-specific therapies. Design/setting/patients: We conducted an international chart review of 230 patients with confirmed GL or PL at five treatment centers who never received leptin or other lipodystrophy-specific therapies. Patients were observed from birth to loss to follow-up, death, or date of chart abstraction. Outcome Measures: Lifetime prevalence of diabetes/insulin resistance and select organ abnormalities, time to diabetes/insulin resistance, first organ abnormality, disease progression, and mortality were described. Results: Diabetes/insulin resistance was identified in 58.3% of patients. Liver abnormalities were the most common organ abnormality (71.7%), followed by kidney (40.4%), heart (30.4%), and pancreatitis (13.0%). Kaplan-Meier estimates of mean (SE) time to first organ abnormality were 7.7 years (0.9) in GL and 16.1 years (1.5) in PL (P < 0.001). Mean time to diabetes/insulin resistance was 12.7 years (1.2) in GL and 19.1 years (1.7) in PL (P = 0.131). Mean time to disease progression was 7.6 years (0.8) and comparable between GL and PL subgroups (P = 0.393). Mean time to death was 51.2 years (3.5) in GL and 66.6 years (1.0) in PL (P < 0.001). Conclusions: This large-scale study provides comprehensive, long-term data across multiple countries on the natural history of non-HIV-related lipodystrophy. (Copyright © 2019 Endocrine Society.)
Databáze:	MEDLINE
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