Metreleptin treatment of non-HIV lipodystrophy syndromes
Tài liệu tham khảo
Vantyghem, 2012, How to diagnose a lipodystrophy syndrome, Ann d’Endocrinol, 73, 170, 10.1016/j.ando.2012.04.010
Araújo-Vilar, 2019, Diagnosis and treatment of lipodystrophy: a step-by-step approach, J Endocrinol Investig, 42, 61, 10.1007/s40618-018-0887-z
Akinci, 2019, Comorbidities and survival in patients with lipodystrophy: an international chart review study, J Clin Endocrinol Metab, 16
Zhang, 1994, Positional cloning of the mouse obese gene and its human homologue, Nature, 372, 425, 10.1038/372425a0
Halaas, 1995, Weight-reducing effects of the plasma protein encoded by the obese gene, Science, 269, 543, 10.1126/science.7624777
Schoeller, 1997, Entrainment of the diurnal rhythm of plasma leptin to meal timing, J Clin Investig, 100, 1882, 10.1172/JCI119717
Minocci, 2000, Leptin plasma concentrations are dependent on body fat distribution in obese patients, Int J Obes, 24, 1139, 10.1038/sj.ijo.0801385
Wauters, 1998, Are leptin levels dependent on body fat distribution in obese men and women?, Eat Weight Disord, 3, 124, 10.1007/BF03339999
Klein, 1996, Adipose tissue leptin production and plasma leptin kinetics in humans, Diabetes, 45, 984, 10.2337/diab.45.7.984
Ahima, 1996, Role of leptin in the neuroendocrine response to fasting, Nature, 382, 250, 10.1038/382250a0
Tartaglia, 1995, Identification and expression cloning of a leptin receptor, OB-R. Cell., 83, 1263, 10.1016/0092-8674(95)90151-5
Friedman, 1998, Leptin and the regulation of body weight in mammals, Nature, 395, 763, 10.1038/27376
Flier, 2019, Starvation in the Midst of Plenty: Reflections on the History and Biology of Insulin and Leptin, Endocrine Rev, 40, 1, 10.1210/er.2018-00179
Seufert, 1999, Leptin inhibits insulin gene transcription and reverses hyperinsulinemia in leptin-deficient ob/ob mice, Proc Natl Acad Sci, 96, 674, 10.1073/pnas.96.2.674
Baykal, 2020, Leptin decreases de novo lipogenesis in patients with lipodystrophy, JCI Insight, 5, 10.1172/jci.insight.137180
Chan, 2003, The role of falling leptin levels in the neuroendocrine and metabolic adaptation to short-term starvation in healthy men, J Clin Invest, 111, 1409, 10.1172/JCI200317490
DePaoli, 2014, 20 YEARS OF LEPTIN: leptin in common obesity and associated disorders of metabolism, J Endocrinol, 223, T71, 10.1530/JOE-14-0258
Heymsfield, 1999, Recombinant leptin for weight loss in obese and lean adults: a randomized, controlled, dose-escalation trial, JAMA, 282, 1568, 10.1001/jama.282.16.1568
Wilding, 2021, Once-weekly semaglutide in adults with overweight or obesity, N Engl J Med, 384, 989, 10.1056/NEJMoa2032183
Frederich, 1995, Leptin levels reflect body lipid content in mice: evidence for diet-induced resistance to leptin action, Nat Med, 1, 1311, 10.1038/nm1295-1311
Moon, 2011, Efficacy of metreleptin in obese patients with type 2 diabetes: cellular and molecular pathways underlying leptin tolerance, Diabetes, 60, 1647, 10.2337/db10-1791
Moon, 2015, Identification and saturable nature of signaling pathways induced by metreleptin in humans: comparative evaluation of in vivo, ex vivo, and in vitro administration, Diabetes, 64, 828, 10.2337/db14-0625
Park, 2015, Physiology of leptin: energy homeostasis, neuroendocrine function and metabolism, Metabolism., 64, 24, 10.1016/j.metabol.2014.08.004
Cook, 2021, Effect of leptin therapy on survival in generalized and partial lipodystrophy: a matched cohort analysis, J Clin Endocrinol Metab, dgab216
Guillín-Amarelle, 2018, Lipodystrophic laminopathies: diagnostic clues, Nucleus, 9, 277, 10.1080/19491034.2018.1454167
Shimomura, 1999, Leptin reverses insulin resistance and diabetes mellitus in mice with congenital lipodystrophy, Nature, 401, 73, 10.1038/43448
Oral, 2002, Leptin-replacement therapy for lipodystrophy, N Engl J Med, 346, 570, 10.1056/NEJMoa012437
Vatier, 2016, One-year metreleptin improves insulin secretion in patients with diabetes linked to genetic lipodystrophic syndromes, Diabetes Obes Metab, 18, 693, 10.1111/dom.12606
Araujo-Vilar, 2015, Recombinant human leptin treatment in genetic lipodystrophic syndromes: the long-term Spanish experience, Endocrine, 49, 139, 10.1007/s12020-014-0450-4
Rodríguez, 2014, Leptin replacement therapy for the treatment of non-HAART associated lipodystrophy syndromes: a meta-analysis into the effects of leptin on metabolic and hepatic endpoints, Arq Bras Endocrinol Metab, 58, 783, 10.1590/0004-2730000003174
Ebihara, 2007, Efficacy and safety of leptin-replacement therapy and possible mechanisms of leptin actions in patients with generalized lipodystrophy, J Clin Endocrinol Metab, 92, 532, 10.1210/jc.2006-1546
Chan, 2011, Clinical effects of long-term metreleptin treatment in patients with lipodystrophy, Endocr Pract, 17, 922, 10.4158/EP11229.OR
Brown, 2018, Long-term effectiveness and safety of metreleptin in the treatment of patients with generalized lipodystrophy, Endocrine, 60, 479, 10.1007/s12020-018-1589-1
Oral, 2019, Long-term effectiveness and safety of metreleptin in the treatment of patients with partial lipodystrophy, Endocrine, 64, 500, 10.1007/s12020-019-01862-8
Moahad Dar, 2016, Efficacy and safety of metreleptin in patients with partial lipodystrophy: lessons from an expanded access program, J Diabetes Metab [Internet], 10.4172/2155-6156.1000659
Park, 2007, Long-term efficacy of leptin replacement in patients with Dunnigan-type familial partial lipodystrophy, Metabolism, 56, 508, 10.1016/j.metabol.2006.11.010
Simha, 2012, 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, 97, 785, 10.1210/jc.2011-2229
Sekizkardes, 2019, Efficacy of metreleptin treatment in familial partial lipodystrophy due to PPARG vs LMNA pathogenic variants, J Clin Endocrinol Metab, 104, 3068, 10.1210/jc.2018-02787
Khan, 2001, Long-term leptin treatment of ob/ob mice improves glucose-induced insulin secretion, Int J Obes, 25, 816, 10.1038/sj.ijo.0801628
Brown, 2018, Metreleptin-mediated improvements in insulin sensitivity are independent of food intake in humans with lipodystrophy, J Clin Invest, 128, 3504, 10.1172/JCI95476
Simha, 2003, Effect of leptin replacement on intrahepatic and intramyocellular lipid content in patients with generalized lipodystrophy, Diabetes Care, 26, 30, 10.2337/diacare.26.1.30
Diker-Cohen, 2015, Partial and generalized lipodystrophy: comparison of baseline characteristics and response to metreleptin, J Clin Endocrinol Metab, 100, 1802, 10.1210/jc.2014-4491
Kassai, 2016, Effect of leptin administration on circulating apolipoprotein CIII levels in patients with lipodystrophy, J Clin Endocrinol Metab, 101, 1790, 10.1210/jc.2015-3891
Muniyappa, 2017, Metreleptin therapy lowers plasma angiopoietin-like protein 3 in patients with generalized lipodystrophy, J Clin Lipidol, 11, 543, 10.1016/j.jacl.2017.02.002
Lightbourne, 2021, Apolipoprotein CIII and angiopoietin-like protein 8 are elevated in lipodystrophy and decrease after metreleptin, J Endocrine Soc, 5, bvaa191, 10.1210/jendso/bvaa191
Joseph, 2014, Lipid regulation in lipodystrophy versus the obesity-associated metabolic syndrome: the dissociation of HDL-C and triglycerides, J Clin Endocrinol Metab, 99, E1676, 10.1210/jc.2014-1878
Leguier, 2020, Improvement in triglycerides and HDL-cholesterol with metreleptin in lipodystrophic syndromes with mild metabolic syndrome, Ann d’Endocrinol, 81, 176, 10.1016/j.ando.2020.07.111
Levenson, 2016, Effect of leptin replacement on PCSK9 in ob/ob mice and female lipodystrophic patients, Endocrinology, 157, 1421, 10.1210/en.2015-1624
Vatier, 2017, One-year metreleptin therapy decreases PCSK9 serum levels in diabetic patients with monogenic lipodystrophy syndromes, Diabetes Metab, 43, 275, 10.1016/j.diabet.2016.08.004
Kinzer, 2019, Advanced lipoprotein analysis shows atherogenic lipid profile that improves after metreleptin in patients with lipodystrophy, J Endocrine Soc, 3, 1503, 10.1210/js.2019-00103
Bruder-Nascimento, 2019, Recent advances in understanding lipodystrophy: a focus on lipodystrophy-associated cardiovascular disease and potential effects of leptin therapy on cardiovascular function, F1000Res, 8, 1756, 10.12688/f1000research.20150.1
Poetsch, 2020, Role of leptin in cardiovascular diseases, Front Endocrinol, 10.3389/fendo.2020.00354
Pearson, 2020, Metreleptin restores vascular endothelial function in a mouse model of acquired lipodystrophy likely via PPARγ-dependent reduction in reactive oxygen species, FASEB J, 34, 1
Kwapich, 2019, Cardiometabolic assessment of lamin A/C gene mutation carriers: a phenotype–genotype correlation, Diabetes Metab, 45, 382, 10.1016/j.diabet.2018.09.006
Brown, 2015, Leptin does not mediate hypertension associated with human obesity, Cell, 162, 465, 10.1016/j.cell.2015.07.007
Cook, 2021, Effects of metreleptin on patient outcomes and quality of life in generalized and partial lipodystrophy, J Endocrine Soc, bvab019, 10.1210/jendso/bvab019
Akinci, 2020, The complicated clinical course in a case of atypical lipodystrophy after development of neutralizing antibody to metreleptin: treatment with setmelanotide, Endocrinol Diabetes Metab Case Rep, 2020
Fukaishi, 2020, A case of generalized lipodystrophy-associated progeroid syndrome treated by leptin replacement with short and long-term monitoring of the metabolic and endocrine profiles, Endocr J, 67, 211, 10.1507/endocrj.EJ19-0226
Javor, 2005, Leptin reverses nonalcoholic steatohepatitis in patients with severe lipodystrophy, Hepatology, 41, 753, 10.1002/hep.20672
Petersen, 2002, Leptin reverses insulin resistance and hepatic steatosis in patients with severe lipodystrophy, J Clin Investig, 109, 1345, 10.1172/JCI0215001
Safar Zadeh, 2013, The liver diseases of lipodystrophy: the long-term effect of leptin treatment, J Hepatol, 59, 131, 10.1016/j.jhep.2013.02.007
Misra, 2003, Clinical Features and metabolic derangements in acquired generalized lipodystrophy: case reports and review of the literature, Medicine, 82, 129, 10.1097/00005792-200303000-00007
Lima, 2018, Causes of death in patients with Berardinelli-Seip congenital generalized lipodystrophy, PLoS One, 13, 10.1371/journal.pone.0199052
Polyzos, 2019, Fatty liver in lipodystrophy: a review with a focus on therapeutic perspectives of adiponectin and/or leptin replacement, Metabolism, 96, 66, 10.1016/j.metabol.2019.05.001
Akinci, 2018, Renal complications of lipodystrophy: a closer look at the natural history of kidney disease, Clin Endocrinol, 89, 65, 10.1111/cen.13732
Lee, 2019, Effects of metreleptin on proteinuria in patients with lipodystrophy, J Clin Endocrinol Metab, 104, 4169, 10.1210/jc.2019-00200
Javor, 2004, Proteinuric nephropathy in acquired and congenital generalized lipodystrophy: baseline characteristics and course during recombinant leptin therapy, J Clin Endocrinol Metab, 89, 3199, 10.1210/jc.2003-032140
Sienkiewicz, 2011, Long-term metreleptin treatment increases bone mineral density and content at the lumbar spine of lean hypoleptinemic women, Metabolism., 60, 1211, 10.1016/j.metabol.2011.05.016
Christensen, 2014, Bone mineral content in patients with congenital generalized lipodystrophy is unaffected by metreleptin replacement therapy, J Clin Endocrinol Metab, 99, E1493, 10.1210/jc.2014-1353
Moran, 2004, Changes in body composition in patients with severe lipodystrophy after leptin replacement therapy, Metabolism, 53, 513, 10.1016/j.metabol.2003.10.019
McDuffie, 2004, Effects of exogenous leptin on satiety and satiation in patients with lipodystrophy and leptin insufficiency, J Clin Endocrinol Metab, 89, 4258, 10.1210/jc.2003-031868
Püschel, 2019, Beneficial effects of leptin substitution on impaired eating behavior in lipodystrophy are sustained beyond 150 weeks of treatment, Cytokine, 113, 400, 10.1016/j.cyto.2018.10.012
Schlögl, 2016, Leptin substitution in patients with lipodystrophy: neural correlates for long-term success in the normalization of eating behavior, Diabetes, 65, 2179, 10.2337/db15-1550
Vatier, 2019, Adherence with metreleptin therapy and health self-perception in patients with lipodystrophic syndromes, Orphanet J Rare Dis, 14, 177, 10.1186/s13023-019-1141-2
Oral EA, Ruiz E, Andewelt A, Sebring N, Wagner AJ, Depaoli AM, et al. Effect of leptin replacement on pituitary hormone regulation in patients with severe lipodystrophy. 8.
Musso, 2005, The long-term effect of recombinant methionyl human leptin therapy on hyperandrogenism and menstrual function in female and pituitary function in male and female hypoleptinemic lipodystrophic patients, Metabolism, 54, 255, 10.1016/j.metabol.2004.08.021
Abel, 2016, Effects of recombinant human leptin (Metreleptin) on nocturnal luteinizing hormone secretion in lipodystrophy patients, NEN, 103, 402
Corvillo, 2019, An overview of lipodystrophy and the role of the complement system, Mol Immunol, 112, 223, 10.1016/j.molimm.2019.05.011
Shibata, 2018, Acquired partial lipoatrophy as graft-versus-host disease and treatment with metreleptin: two case reports, J Med Case Rep, 12, 368, 10.1186/s13256-018-1901-y
Park, 2008, Type 1 diabetes associated with acquired generalized lipodystrophy and insulin resistance: the effect of long-term leptin therapy, J Clin Endocrinol Metab, 93, 26, 10.1210/jc.2007-1856
Lebastchi, 2015, A report of three cases with acquired generalized lipodystrophy with distinct autoimmune conditions treated with metreleptin, J Clin Endocrinol Metab, 100, 3967, 10.1210/jc.2015-2589
Chan, 2005, Recombinant methionyl human leptin administration to achieve high physiologic or pharmacologic leptin levels does not alter circulating inflammatory marker levels in humans with leptin sufficiency or excess, J Clin Endocrinol Metab, 90, 1618, 10.1210/jc.2004-1921
Ziegler, 2019, Leptin induces TNFα-dependent inflammation in acquired generalized lipodystrophy and combined Crohn's disease, Nat Commun, 10, 5629, 10.1038/s41467-019-13559-7
Brown, 2016, The diagnosis and management of lipodystrophy syndromes: a multi-society practice guideline, J Clin Endocrinol Metab, 101, 4500, 10.1210/jc.2016-2466
Maguire, 2012, Pregnancy in a woman with congenital generalized lipodystrophy: leptin’s vital role in reproduction, Obstetr Gynecol, 119, 452, 10.1097/AOG.0b013e31822cecf7
Brown, 2017, Effects of metreleptin in pediatric patients with lipodystrophy, J Clin Endocrinol Metab, 102, 1511, 10.1210/jc.2016-3628
Gupta, 2017, Clinical features and management of non-HIV–related lipodystrophy in children: a systematic review, J Clin Endocrinol Metab, 102, 363, 10.1210/jc.2016-2271
Kamran, 2012, Consequences of stopping and restarting leptin in an adolescent with lipodystrophy, Horm Res Paediatr, 78, 320, 10.1159/000341398
Brown, 2016, Lymphoma in acquired generalized lipodystrophy, Leuk Lymphoma, 57, 45, 10.3109/10428194.2015.1040015
Beltrand, 2010, Resistance to leptin-replacement therapy in Berardinelli–Seip congenital lipodystrophy: an immunological origin, Eur J Endocrinol, 162, 1083, 10.1530/EJE-09-1027
Chan, 2016, Immunogenicity associated with metreleptin treatment in patients with obesity or lipodystrophy, Clin Endocrinol (Oxf), 85, 137, 10.1111/cen.12980
Oliveira, 2017, Glucagon-like peptide-1 analogues - an efficient therapeutic option for the severe insulin resistance of lipodystrophic syndromes: two case reports, J Med Case Reports, 11, 12, 10.1186/s13256-016-1175-1
Hosokawa, 2020, SGLT2 inhibitors for genetic and acquired insulin resistance: considerations for clinical use, J Diabetes Investig, 11, 1431, 10.1111/jdi.13309
Banning, 2017, Insulin secretory defect in familial partial lipodystrophy Type 2 and successful long-term treatment with a glucagon-like peptide 1 receptor agonist, Diabet Med, 34, 1792, 10.1111/dme.13527
González-Clavijo, 2020, Uso de metformina y un inhibidor de SGLT2 en el manejo de lipodistrofia congénita generalizada. Reporte de caso, Rev Fac Med, 68, 10.15446/revfacmed.v68n4.77325