Metreleptin for metabolic disorders associated with generalized or partial lipodystrophy

References

• Garg A. Clinical review: lipodystrophies: genetic and acquired body fat disorders. J Clin Endocrinol Metab 2011;96(11):3313-25
   PubMed Web of Science ®Google Scholar
• Chan JL, Oral EA. Clinical classification and treatment of congenital and acquired lipodystrophy. Endocr Pract 2010;16(2):310-23
   Google Scholar
• Capeau J, Magre J, Lascols O, et al. Diseases of adipose tissue: genetic and acquired lipodystrophies. Biochem Soc Trans 2005;33(Pt 5):1073-7
   Google Scholar
• Moon HS, Dalamaga M, Kim SY, et al. Leptin's role in lipodystrophic and nonlipodystrophic insulin-resistant and diabetic individuals. Endocr Rev 2013;34(3):377-412
   PubMed Web of Science ®Google Scholar
• Vantyghem MC, Balavoine AS, Douillard C, et al. How to diagnose a lipodystrophy syndrome. Ann Endocrinol (Paris) 2012;73(3):170-89
   PubMed Web of Science ®Google Scholar
• Krahmer N, Farese RV Jr, Walther TC. Balancing the fat: lipid droplets and human disease. EMBO Mol Med 2013;5(7):905-15
   Google Scholar
• Simha V, Garg A. Phenotypic heterogeneity in body fat distribution in patients with congenital generalized lipodystrophy caused by mutations in the AGPAT2 or seipin genes. J Clin Endocrinol Metab 2003;88(11):5433-7
   PubMed Web of Science ®Google Scholar
• Agarwal AK, Simha V, Oral EA, et al. Phenotypic and genetic heterogeneity in congenital generalized lipodystrophy. J Clin Endocrinol Metab 2003;88(10):4840-7
   PubMed Web of Science ®Google Scholar
• Haque WA, Shimomura I, Matsuzawa Y, Garg A. Serum adiponectin and leptin levels in patients with lipodystrophies. J Clin Endocrinol Metab 2002;87(5):2395
   PubMed Web of Science ®Google Scholar
• Misra A, Garg A. Clinical features and metabolic derangements in acquired generalized lipodystrophy: case reports and review of the literature. Medicine (Baltimore) 2003;82(2):129-46
   PubMed Web of Science ®Google Scholar
• Misra A, Peethambaram A, Garg A. Clinical features and metabolic and autoimmune derangements in acquired partial lipodystrophy: report of 35 cases and review of the literature. Medicine (Baltimore) 2004;83(1):18-34
   PubMed Web of Science ®Google Scholar
• Handelsman Y, Oral EA, Bloomgarden ZT, et al. The clinical approach to the detection of lipodystrophy - an AACE consensus statement. Endocr Pract 2013;19(1):107-16
   PubMed Web of Science ®Google Scholar
• Rother KI, Brown RJ. Novel forms of lipodystrophy: why should we care? Diabetes Care 2013;36(8):2142-5
   Google Scholar
• Bingham A, Mamyrova G, Rother KI, et al. Predictors of acquired lipodystrophy in juvenile-onset dermatomyositis and a gradient of severity. Medicine (Baltimore) 2008;87(2):70-86
   PubMed Web of Science ®Google Scholar
• Grinspoon S, Carr A. Cardiovascular risk and body-fat abnormalities in HIV-infected adults. N Engl J Med 2005;352(1):48-62
   PubMed Web of Science ®Google Scholar
• Gavrilova O, Marcus-Samuels B, Graham D, et al. Surgical implantation of adipose tissue reverses diabetes in lipoatrophic mice. J Clin Invest 2000;105(3):271-8
   Google Scholar
• Colombo C, Cutson JJ, Yamauchi T, et al. Transplantation of adipose tissue lacking leptin is unable to reverse the metabolic abnormalities associated with lipoatrophy. Diabetes 2002;51(9):2727-33
   PubMed Web of Science ®Google Scholar
• Shimomura I, Hammer RE, Ikemoto S, et al. Leptin reverses insulin resistance and diabetes mellitus in mice with congenital lipodystrophy. Nature 1999;401(6748):73-6
   PubMed Web of Science ®Google Scholar
• Oral EA, Simha V, Ruiz E, et al. Leptin-replacement therapy for lipodystrophy. N Engl J Med 2002;346(8):570-8
   PubMed Web of Science ®Google Scholar
• Chan JL, Wong SL, Orlova C, et al. Pharmacokinetics of recombinant methionyl human leptin after subcutaneous administration: variation of concentration-dependent parameters according to assay. J Clin Endocrinol Metab 2007;92(6):2307-11
   Google Scholar
• Chou K, Perry CM. Metreleptin: first global approval. Drugs 2013;73(9):989-97
   PubMed Web of Science ®Google Scholar
• Bristol Myers Squibb Press release. FDA Advisory Committee Votes on Investigational Medicine Metreleptin. Available from: http://news.bms.com/press-release/rd-news/fda-advisory-committee-votes-investigational-medicine-metreleptin [Last accessed 11 December 2013]
   Google Scholar
• Javor ED, Cochran EK, Musso C, et al. Long-term efficacy of leptin replacement in patients with generalized lipodystrophy. Diabetes 2005;54(7):1994-2002
   PubMed Web of Science ®Google Scholar
• Chan JL, Lutz K, Cochran E, et al. Clinical effects of long-term metreleptin treatment in patients with lipodystrophy. Endocr Pract 2011;17(6):922-32
   PubMed Web of Science ®Google Scholar
• Ebihara K, Kusakabe T, Hirata M, et al. Efficacy and safety of leptin-replacement therapy and possible mechanisms of leptin actions in patients with generalized lipodystrophy. J Clin Endocrinol Metab 2007;92(2):532-41
   PubMed Web of Science ®Google Scholar
• Javor ED, Moran SA, Young JR, et al. Proteinuric nephropathy in acquired and congenital generalized lipodystrophy: baseline characteristics and course during recombinant leptin therapy. J Clin Endocrinol Metab 2004;89(7):3199-207
   PubMed Web of Science ®Google Scholar
• Beltrand J, Beregszaszi M, Chevenne D, et al. Metabolic correction induced by leptin replacement treatment in young children with Berardinelli-Seip congenital lipoatrophy. Pediatrics 2007;120(2):e291-6
   Google Scholar
• Kamran F, Rother KI, Cochran E, et al. Consequences of stopping and restarting leptin in an adolescent with lipodystrophy. Horm Res Paediatr 2012;78(5-6):320-5
   Google Scholar
• Simha V, Szczepaniak LS, Wagner AJ, et al. Effect of leptin replacement on intrahepatic and intramyocellular lipid content in patients with generalized lipodystrophy. Diabetes Care 2003;26(1):30-5
   Google Scholar
• Petersen KF, Oral EA, Dufour S, et al. Leptin reverses insulin resistance and hepatic steatosis in patients with severe lipodystrophy. J Clin Invest 2002;109(10):1345-50
   PubMed Web of Science ®Google Scholar
• Javor ED, Ghany MG, Cochran EK, et al. Leptin reverses nonalcoholic steatohepatitis in patients with severe lipodystrophy. Hepatology 2005;41(4):753-60
   PubMed Web of Science ®Google Scholar
• Safar Zadeh E, Lungu AO, Cochran EK, et al. The liver diseases of lipodystrophy: the long-term effect of leptin treatment. J Hepatol 2013;59(1):131-7
   PubMed Web of Science ®Google Scholar
• Casey SP, Lokan J, Testro A, et al. Post-liver transplant leptin results in resolution of severe recurrence of lipodystrophy-associated nonalcoholic steatohepatitis. Am J Transplant 2013;13(11):3031-4
   Google Scholar
• Simha V, Subramanyam L, Szczepaniak L, et al. Comparison of efficacy and safety of leptin replacement therapy in moderately and severely hypoleptinemic patients with familial partial lipodystrophy of the Dunnigan variety. J Clin Endocrinol Metab 2012;97(3):785-92
   PubMed Web of Science ®Google Scholar
• Park JY, Javor ED, Cochran EK, et al. Long-term efficacy of leptin replacement in patients with Dunnigan-type familial partial lipodystrophy. Metabolism 2007;56(4):508-16
   PubMed Web of Science ®Google Scholar
• Lee JH, Chan JL, Sourlas E, et al. Recombinant methionyl human leptin therapy in replacement doses improves insulin resistance and metabolic profile in patients with lipoatrophy and metabolic syndrome induced by the highly active antiretroviral therapy. J Clin Endocrinol Metab 2006;91(7):2605-11
   PubMed Web of Science ®Google Scholar
• Mulligan K, Khatami H, Schwarz JM, et al. The effects of recombinant human leptin on visceral fat, dyslipidemia, and insulin resistance in patients with human immunodeficiency virus-associated lipoatrophy and hypoleptinemia. J Clin Endocrinol Metab 2009;94(4):1137-44
   PubMed Web of Science ®Google Scholar
• Magkos F, Brennan A, Sweeney L, et al. Leptin replacement improves postprandial glycemia and insulin sensitivity in human immunodeficiency virus-infected lipoatrophic men treated with pioglitazone: a pilot study. Metabolism 2011;60(7):1045-9
   PubMed Web of Science ®Google Scholar
• Sekhar RV, Jahoor F, Iyer D, et al. Leptin replacement therapy does not improve the abnormal lipid kinetics of hypoleptinemic patients with HIV-associated lipodystrophy syndrome. Metabolism 2012;61(10):1395-403
   PubMed Web of Science ®Google Scholar
• McDuffie JR, Riggs PA, Calis KA, et al. Effects of exogenous leptin on satiety and satiation in patients with lipodystrophy and leptin insufficiency. J Clin Endocrinol Metab 2004;89(9):4258-63
   PubMed Web of Science ®Google Scholar
• Aotani D, Ebihara K, Sawamoto N, et al. Functional magnetic resonance imaging analysis of food-related brain activity in patients with lipodystrophy undergoing leptin replacement therapy. J Clin Endocrinol Metab 2012;97(10):3663-71
   Google Scholar
• Unger RH, Scherer PE. Gluttony, sloth and the metabolic syndrome: a roadmap to lipotoxicity. Trends Endocrinol Metab 2010;21(6):345-52
   PubMed Web of Science ®Google Scholar
• Miyamoto L, Ebihara K, Kusakabe T, et al. Leptin activates hepatic 5'-AMP-activated protein kinase through sympathetic nervous system and alpha1-adrenergic receptor: a potential mechanism for improvement of fatty liver in lipodystrophy by leptin. J Biol Chem 2012;287(48):40441-7
   PubMed Web of Science ®Google Scholar
• Moran SA, Patten N, Young JR, et al. Changes in body composition in patients with severe lipodystrophy after leptin replacement therapy. Metabolism 2004;53(4):513-19
   PubMed Web of Science ®Google Scholar
• Vadacca M, Margiotta DP, Navarini L, Afeltra A. Leptin in immuno-rheumatological diseases. Cell Mol Immunol 2011;8(3):203-12
   PubMed Web of Science ®Google Scholar
• Amylin Pharmaceuticals. Myalept (metreleptin for injection): FDA Endocrinologic and Metabolic Drugs advisory committee meeting. Available from: www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/EndocrinologicandMetabolicDrugsAdvisoryCommittee/UCM377928.pdf [Last accessed December 2013]
   Google Scholar
• Beltrand J, Lahlou N, Le Charpentier T, et al. Resistance to leptin-replacement therapy in Berardinelli-Seip congenital lipodystrophy: an immunological origin. Eur J Endocrinol 2010;162(6):1083-91
   Google Scholar