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. 2019 Nov 1;104(11):5120-5135.
doi: 10.1210/jc.2018-02730.

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

Baris Akinci  1 , Elif A Oral  2 , Adam Neidert  2 , Diana Rus  2 , Wendy Y Cheng  3 , Philippe Thompson-Leduc  3 , Hoi Ching Cheung  3 , Pamela Bradt  4 , Maria Cristina Foss de Freitas  5 , Renan Magalhães Montenegro  6 , Virgínia Oliveira Fernandes  6 , Elaine Cochran  7 , Rebecca J Brown  7
Affiliations

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

Baris Akinci et al. J Clin Endocrinol Metab. .

Abstract

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.

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Conflict of interest statement

Current Affiliation: P. Bradt’s current affiliation is the Institute for Clinical and Economic Review, Boston, Massachusetts 02109.

Disclosure Summary: B.A. is or has been on the advisory board of Aegerion Pharmaceuticals and a speaker for AstraZeneca, Eli Lilly and Company, Novartis, Novo Nordisk, Boehringer Ingelheim, Servier, and Sanofi Aventis. E.A.O. has received consulting fees indirectly through fees paid to the University of Michigan from Aegerion Pharmaceuticals, Akcea Therapeutics, and Regeneron Pharmaceuticals, has received grants from Akcea Therapeutics, Ionis Pharmaceuticals, Inc., Aegerion Pharmaceuticals, Gemphire Therapeutics, GI Dynamics, and Regeneron Pharmaceuticals, and had writing support relationships with Aegerion Pharmaceuticals. A.N., D.R., M.C.F.d.F., V.O.F., E.C., and R.J.B. have writing support relationships with Aegerion Pharmaceuticals. W.Y.C. and P.T.-L. are employees of Analysis Group, which has received consulting fees from Aegerion Pharmaceuticals to conduct this study. P.B. is a former employee of Aegerion Pharmaceuticals. R.M.M. serves as consultant and speaker for and received a research grant from Aegerion Pharmaceuticals.

Restrictions apply to the availability of data generated or analyzed during this study to preserve patient confidentiality or because they were used under license. The corresponding author will on request detail the restrictions and any conditions under which access to some data may be provided.

Figures

Figure 1.
Figure 1.
Study design. The study observation period spanned from the patients' birth until loss to follow-up, death, or date of chart abstraction, whichever occurred first, and includes the baseline and follow-up periods. The baseline period spanned the time from birth until the date of diagnosis with GL or PL. The follow-up period spanned the time from date of diagnosis with GL or PL to the end of the observation period. Durations of the various study periods in years are reported as means (SD).
Figure 2.
Figure 2.
Time to diabetes and/or insulin resistance, organ abnormalities, and overall survival, stratified by GL and PL. Time to diagnosis of (a) diabetes and/or insulin resistance (IR), (b) first organ abnormality, and (c) abnormality in subsequent organ (proxy for disease progression), stratified by GL and PL are shown. (d) Overall survival stratified by GL and PL is also shown.
See this image and copyright information in PMC

References

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