Emerging drugs for Cushing’s disease

Bibliography

• Newell-Price J, Bertagna X, Grossman AB, Nieman LK. Cushing’s syndrome. Lancet 2006;367(9522):1605-17
   PubMed Web of Science ®Google Scholar
• Nieman LK, Biller BM, Findling JW, et al. The diagnosis of Cushing’s syndrome: an endocrine society clinical practice guideline. J Clin Endocrinol Metab 2008;93(5):1526-40
   PubMed Web of Science ®Google Scholar
• Cuevas-Ramos D, Fleseriu M. Treatment of Cushing’s disease: a mechanistic update. J Endocrinol 2014;223(2):R19-39
   PubMed Web of Science ®Google Scholar
• Sharma ST, Nieman LK. Cushing’s syndrome: all variants, detection, and treatment. Endocrinol Metabol Clin North Am 2011;40(2):379-91.viii-x
   PubMed Web of Science ®Google Scholar
• Mazziotti G, Gazzaruso C, Giustina A. Diabetes in Cushing syndrome: basic and clinical aspects. Trends Endocrinol Metab 2011;22(12):499-506
   PubMed Web of Science ®Google Scholar
• Bertagna X, Guignat L, Groussin L, Bertherat J. Cushing’s disease. Best Pract Res Clin Endocrinol Metab 2009;23(5):607-23
   PubMed Web of Science ®Google Scholar
• Biller BM, Grossman AB, Stewart PM, et al. Treatment of adrenocorticotropin-dependent Cushing’s syndrome: a consensus statement. J Clin Endocrinol Metab 2008;93(7):2454-62
   PubMed Web of Science ®Google Scholar
• Patil CG, Prevedello DM, Lad SP, et al. Jr. Late recurrences of Cushing’s disease after initial successful transsphenoidal surgery. J Clin Endocrinol Metab 2008;93(2):358-62
   PubMed Web of Science ®Google Scholar
• Atkinson AB, Kennedy A, Wiggam MI, et al. Long-term remission rates after pituitary surgery for Cushing’s disease: the need for long-term surveillance. Clin Endocrinol 2005;63(5):549-59
   PubMed Web of Science ®Google Scholar
• Friedman RB, Oldfield EH, Nieman LK, et al. Repeat transsphenoidal surgery for Cushing’s disease. J Neurosurg 1989;71(4):520-7
   PubMed Web of Science ®Google Scholar
• Patil CG, Veeravagu A, Prevedello DM, et al. Jr. Outcomes after repeat transsphenoidal surgery for recurrent Cushing’s disease. Neurosurg 2008;63(2):266-70; discussion 270-61
   PubMed Web of Science ®Google Scholar
• Tritos NA, Biller BM, Swearingen B. Management of Cushing disease. Nat Rev Endocrinol 2011;7(5):279-89
   PubMed Web of Science ®Google Scholar
• Sheehan JP, Xu Z, Salvetti DJ, et al. Results of gamma knife surgery for Cushing’s disease. J Neurosurg 2013;119(6):1486-92
   PubMed Web of Science ®Google Scholar
• Castinetti F, Fassnacht M, Johanssen S, et al. Merits and pitfalls of mifepristone in Cushing’s syndrome. Eur J Endocrinol 2009;160(6):1003-10
   PubMed Web of Science ®Google Scholar
• Grossman AB. Clinical review#: the diagnosis and management of central hypoadrenalism. J Clin Endocrinol Metab 2010;95(11):4855-63
   PubMed Web of Science ®Google Scholar
• Assie G, Bahurel H, Coste J, et al. Corticotroph tumor progression after adrenalectomy in Cushing’s disease: A reappraisal of nelson’s syndrome. J Clin Endocrinol Metab 2007;92(1):172-9
   PubMed Web of Science ®Google Scholar
• Mancini T, Casanueva FF, Giustina A. Hyperprolactinemia and prolactinomas. Endocrinol Metabol Clin North Am 2008;37(1):67-99.viii
   PubMed Web of Science ®Google Scholar
• Giustina A, Chanson P, Bronstein MD, et al. A consensus on criteria for cure of acromegaly. J Clin Endocrinol Metab 2010;95(7):3141-8
   PubMed Web of Science ®Google Scholar
• Sarlis NJ, Chanock SJ, Nieman LK. Cortisolemic indices predict severe infections in Cushing syndrome due to ectopic production of adrenocorticotropin. J Clin Endocrinol Metab 2000;85(1):42-7
   PubMed Web of Science ®Google Scholar
• Sherlock M, Ayuk J, Tomlinson JW, et al. Mortality in patients with pituitary disease. Endocr Rev 2010;31(3):301-42
   PubMed Web of Science ®Google Scholar
• Feelders RA, Pulgar SJ, Kempel A, Pereira AM. The burden of Cushing’s disease: Clinical and health-related quality of life aspects. Eur J Endocrinol 2012;167(3):311-26
   PubMed Web of Science ®Google Scholar
• Pereira AM, Tiemensma J, Romijn JA. Neuropsychiatric disorders in Cushing’s syndrome. Neuroendocrinology 2010;92(Suppl 1):65-70
   PubMed Web of Science ®Google Scholar
• Sonino N, Fava GA, Raffi AR, et al. Clinical correlates of major depression in Cushing’s disease. Psychopathology 1998;31(6):302-6
   PubMed Web of Science ®Google Scholar
• Pivonello R, Faggiano A, Lombardi G, Colao A. The metabolic syndrome and cardiovascular risk in Cushing’s syndrome. Endocrinol Metabol Clin North Am 2005;34(2):327-39, viii
   PubMed Web of Science ®Google Scholar
• van der Pas R, de Bruin C, Leebeek FW, et al. The hypercoagulable state in Cushing’s disease is associated with increased levels of procoagulant factors and impaired fibrinolysis, but is not reversible after short-term biochemical remission induced by medical therapy. J Clin Endocrinol Metab 2012;97(4):1303-10
   PubMed Web of Science ®Google Scholar
• Juszczak A, Ertorer ME, Grossman A. The therapy of Cushing’s disease in adults and children: an update. Horm Metab Res 2013;45(2):109-17
   PubMed Web of Science ®Google Scholar
• Hamrahian AH, Yuen KC, Hoffman AR; for the Aace N, Pituitary Scientific C. Aace/ace disease state clinical review: medical management of Cushing disease. Endocr Pract 2014;20(7):746-57
   PubMed Web of Science ®Google Scholar
• Gadelha MR, Vieira Neto L. Efficacy of medical treatment in Cushing’s disease: a systematic review. Clin Endocrinol 2014;80(1):1-12
   PubMed Web of Science ®Google Scholar
• AG. NP. Signifor summary of product characteristics. 2012. Available from: http://www.signifor.com/european-product-characteristics.jsp
   Google Scholar
• Pharmaceuticals N. Signifor prescribing information. 2012. Available from: https://www.pharma.us.novartis.com/product/pi/pdf/exelon.pdf
   Google Scholar
• Therapeutics C. Mifepristone (korlym) prescribing information. 2012. Available from: http://www.korlym.com/docs/KorlymPrescribingInformation.pdf
   Google Scholar
• Valassi E, Crespo I, Gich I, et al. A reappraisal of the medical therapy with steroidogenesis inhibitors in Cushing’s syndrome. Clin Endocrinol 2012;77(5):735-42
   PubMed Web of Science ®Google Scholar
• Preda VA, Sen J, Karavitaki N, Grossman AB. Etomidate in the management of hypercortisolaemia in Cushing’s syndrome: a review. Eur J Endocrinol 2012;167(2):137-43
   PubMed Web of Science ®Google Scholar
• Baudry C, Coste J, Bou Khalil R, et al. Efficiency and tolerance of mitotane in Cushing’s disease in 76 patients from a single center. Eur J Endocrinol 2012;167(4):473-81
   PubMed Web of Science ®Google Scholar
• Donadille B, Groussin L, Waintrop C, et al. Management of Cushing’s syndrome due to ectopic adrenocorticotropin secretion with 1,ortho-1, para’-dichloro-diphenyl-dichloro-ethane: findings in 23 patients from a single center. J Clin Endocrinol Metab 2010;95(2):537-44
   PubMed Web of Science ®Google Scholar
• Gentilin E, Tagliati F, Terzolo M, et al. Mitotane reduces human and mouse acth-secreting pituitary cell viability and function. J Endocrinol 2013;218(3):275-85
   PubMed Web of Science ®Google Scholar
• Pivonello R, Ferone D, de Herder WW, et al. Dopamine receptor expression and function in corticotroph pituitary tumors. J Clin Endocrinol Metab 2004;89(5):2452-62
   PubMed Web of Science ®Google Scholar
• Fleseriu M, Loriaux DL, Ludlam WH. Second-line treatment for Cushing’s disease when initial pituitary surgery is unsuccessful. Curr Opin Endocrinol Diabetes Obes 2007;14(4):323-8
   PubMedGoogle Scholar
• Godbout A, Manavela M, Danilowicz K, et al. Cabergoline monotherapy in the long-term treatment of Cushing’s disease. Eur J Endocrinol 2010;163(5):709-16
   PubMed Web of Science ®Google Scholar
• Schade R, Andersohn F, Suissa S, et al. Dopamine agonists and the risk of cardiac-valve regurgitation. N Engl J Med 2007;356(1):29-38
   PubMed Web of Science ®Google Scholar
• Woo I, Ehsanipoor RM. Cabergoline therapy for Cushing disease throughout pregnancy. Obstet Gynecol 2013;122(2 Pt 2):485-7
   PubMed Web of Science ®Google Scholar
• Batista DL, Zhang X, Gejman R, et al. The effects of som230 on cell proliferation and adrenocorticotropin secretion in human corticotroph pituitary adenomas. J Clin Endocrinol Metab 2006;91(11):4482-8
   PubMed Web of Science ®Google Scholar
• Liu JK, Fleseriu M, Delashaw JBJr, et al. Treatment options for Cushing disease after unsuccessful transsphenoidal surgery. Neurosurg Focus 2007;23(3):E8
   PubMedGoogle Scholar
• Altucci L, Leibowitz MD, Ogilvie KM, et al. Rar and rxr modulation in cancer and metabolic disease. Nat Rev Drug Discov 2007;6(10):793-810
   PubMed Web of Science ®Google Scholar
• Paez-Pereda M, Kovalovsky D, Hopfner U, et al. Retinoic acid prevents experimental Cushing syndrome. J Clin Invest 2001;108(8):1123-31
   PubMed Web of Science ®Google Scholar
• Castillo V, Giacomini D, Paez-Pereda M, et al. Retinoic acid as a novel medical therapy for Cushing’s disease in dogs. Endocrinology 2006;147(9):4438-44
   PubMed Web of Science ®Google Scholar
• Pecori Giraldi F, Ambrogio AG, Andrioli M, et al. Potential role for retinoic acid in patients with Cushing’s disease. J Clin Endocrinol Metab 2012;97(10):3577-83
   PubMed Web of Science ®Google Scholar
• Heaney AP, Fernando M, Yong WH, Melmed S. Functional ppar-gamma receptor is a novel therapeutic target for ACTH-secreting pituitary adenomas. Nat Med 2002;8(11):1281-7
   PubMed Web of Science ®Google Scholar
• Suri D, Weiss RE. Effect of pioglitazone on adrenocorticotropic hormone and cortisol secretion in Cushing’s disease. J Clin Endocrinol Metab 2005;90(3):1340-6
   PubMed Web of Science ®Google Scholar
• Mancini T, Mazziotti G, Doga M, et al. Vertebral fractures in males with type 2 diabetes treated with rosiglitazone. Bone 2009;45(4):784-8
   PubMed Web of Science ®Google Scholar
• Syro LV, Ortiz LD, Scheithauer BW, et al. Treatment of pituitary neoplasms with temozolomide: a review. Cancer 2011;117(3):454-62
   PubMed Web of Science ®Google Scholar
• Ortiz LD, Syro LV, Scheithauer BW, et al. Temozolomide in aggressive pituitary adenomas and carcinomas. Clinics (Sao Paulo) 2012;67(Suppl 1):119-23
   PubMed Web of Science ®Google Scholar
• Bode H, Seiz M, Lammert A, et al. Som230 (pasireotide) and temozolomide achieve sustained control of tumour progression and acth secretion in pituitary carcinoma with widespread metastases. Exp Clin Endocrinol Diabetes 2010;118(10):760-3
   PubMed Web of Science ®Google Scholar
• Fleseriu M, Petersenn S. New avenues in the medical treatment of Cushing’s disease: Corticotroph tumor targeted therapy. J Neurooncol 2013;114(1):1-11
   PubMed Web of Science ®Google Scholar
• Scheithauer BW, Gaffey TA, Lloyd RV, et al. Pathobiology of pituitary adenomas and carcinomas. Neurosurgery 2006;59(2):341-53; discussion 341-53
   PubMed Web of Science ®Google Scholar
• Hague K, Post KD, Morgello S. Absence of Peritumoral Crooke’s change is associated with recurrence in surgically treated Cushing’s disease. Surg Neurol 2000;53(1):77-81
   PubMed Web of Science ®Google Scholar
• Arakaki R, Welles B. Ketoconazole enantiomer for the treatment of diabetes mellitus. Expert Opin Investig Drugs 2010;19(2):185-94
   PubMed Web of Science ®Google Scholar
• Stalla GK, Stalla J, Huber M, et al. Ketoconazole inhibits corticotropic cell function in vitro. Endocrinology 1988;122(2):618-23
   PubMed Web of Science ®Google Scholar
• Sonino N, Boscaro M, Paoletta A, et al. Ketoconazole treatment in Cushing’s syndrome: experience in 34 patients. Clin Endocrinol 1991;35(4):347-52
   PubMed Web of Science ®Google Scholar
• Verhelst JA, Trainer PJ, Howlett TA, et al. Short and long-term responses to metyrapone in the medical management of 91 patients with Cushing’s syndrome. Clin Endocrinol 1991;35(2):169-78
   PubMed Web of Science ®Google Scholar
• Castinetti F, Conte-Devolx B, Brue T. Medical treatment of Cushing’s syndrome: Glucocorticoid receptor antagonists and mifepristone. Neuroendocrinology 2010;92(Suppl 1):125-30
   PubMed Web of Science ®Google Scholar
• Bertagna X, Pivonello R, Fleseriu M, et al. Lci699, a potent 11beta-hydroxylase inhibitor, normalizes urinary cortisol in patients with Cushing’s disease: Results from a multicenter, proof-of-concept study. J Clin Endocrinol Metab 2014;99(4):1375-83
   PubMed Web of Science ®Google Scholar
• Amar L, Azizi M, Menard J, et al. Aldosterone synthase inhibition with lci699: A proof-of-concept study in patients with primary aldosteronism. Hypertension 2010;56(5):831-8
   PubMed Web of Science ®Google Scholar
• Karns AD, Bral JM, Hartman D, et al. Study of aldosterone synthase inhibition as an add-on therapy in resistant hypertension. J Clin Hypertens (Greenwich) 2013;15(3):186-92
   PubMed Web of Science ®Google Scholar
• Bertagna X, Bertagna C, Luton JP, et al. The new steroid analog ru 486 inhibits glucocorticoid action in man. J Clin Endocrinol Metab 1984;59(1):25-8
   PubMed Web of Science ®Google Scholar
• McMaster A, Ray DW. Drug insight: selective agonists and antagonists of the glucocorticoid receptor. Nat Clin Pract Endocrinol Metab 2008;4(2):91-101
   PubMedGoogle Scholar
• PM Stewart NK. The adrenal cortex. In: Melmed S, Polonsky KS, Larsen PR, Kronenberg HM, editors. Williams textbook of endocrinology. Elsevier/Saunders; Philadelphia: 2012. p. chapter 15
   Google Scholar
• Sartor O, Cutler GBJr. Mifepristone: treatment of Cushing’s syndrome. Clin Obstet Gynecol 1996;39(2):506-10
   PubMed Web of Science ®Google Scholar
• Giustina G, Peracchi M, Reschini E, et al. Dose-response study of the inhibiting effect of somatostatin on growth hormone and insulin secretion in normal subjects and acromegalic patients. Metabolism 1975;24(7):807-15
   PubMed Web of Science ®Google Scholar
• van der Hoek J, Waaijers M, van Koetsveld PM, et al. Distinct functional properties of native somatostatin receptor subtype 5 compared with subtype 2 in the regulation of acth release by corticotroph tumor cells. Am J Physiol Endocrinol Metab 2005;289(2):E278-87
   PubMed Web of Science ®Google Scholar
• Bruns C, Lewis I, Briner U, et al. Som230: a novel somatostatin peptidomimetic with broad somatotropin release inhibiting factor (srif) receptor binding and a unique antisecretory profile. Eur J Endocrinol 2002;146(5):707-16
   PubMed Web of Science ®Google Scholar
• Hofland LJ, van der Hoek J, Feelders R, et al. The multi-ligand somatostatin analogue som230 inhibits acth secretion by cultured human corticotroph adenomas via somatostatin receptor type 5. Eur J Endocrinol 2005;152(4):645-54
   PubMed Web of Science ®Google Scholar
• Hofland LJ, Lamberts SW. Somatostatin receptors in pituitary function, diagnosis and therapy. Front Horm Res 2004;32:235-52
   PubMed Web of Science ®Google Scholar
• Susini C, Buscail L. Rationale for the use of somatostatin analogs as antitumor agents. Ann Oncol 2006;17(12):1733-42
   PubMed Web of Science ®Google Scholar
• de Bruin C, Hofland LJ, Nieman LK, et al. Mifepristone effects on tumor somatostatin receptor expression in two patients with Cushing’s syndrome due to ectopic adrenocorticotropin secretion. J Clin Endocrinol Metab 2012;97(2):455-62
   PubMed Web of Science ®Google Scholar
• Castinetti F, Morange I, Jaquet P, et al. Ketoconazole revisited: A preoperative or postoperative treatment in cushing’s disease. Eur J Endocrinol 2008;158(1):91-9
   PubMed Web of Science ®Google Scholar
• Castinetti F, Guignat L, Giraud P, et al. Ketoconazole in Cushing’s disease: is it worth a try? J Clin Endocrinol Metab 2014;99(5):1623-30
   PubMed Web of Science ®Google Scholar
• Nieman LK, Chrousos GP, Kellner C, et al. Successful treatment of Cushing’s syndrome with the glucocorticoid antagonist ru 486. J Clin Endocrinol Metab 1985;61(3):536-40
   PubMed Web of Science ®Google Scholar
• Fleseriu M, Biller BM, Findling JW, et al. Mifepristone, a glucocorticoid receptor antagonist, produces clinical and metabolic benefits in patients with Cushing’s syndrome. J Clin Endocrinol Metab 2012;97(6):2039-49
   PubMed Web of Science ®Google Scholar
• Fleseriu M, Petersenn S. Medical management of Cushing’s disease: what is the future? Pituitary 2012;15(3):330-41
   PubMed Web of Science ®Google Scholar
• Colao A, Petersenn S, Newell-Price J, et al. A 12-month phase 3 study of pasireotide in Cushing’s disease. N Eng J Med 2012;366(10):914-24
   PubMed Web of Science ®Google Scholar
• Boscaro M, Ludlam WH, Atkinson B, et al. Treatment of pituitary-dependent Cushing’s disease with the multireceptor ligand somatostatin analog pasireotide (som230): A multicenter, phase ii trial. J Clin Endocrinol Metab 2009;94(1):115-22
   PubMed Web of Science ®Google Scholar
• Feelders RA, de Bruin C, Pereira AM, et al. Pasireotide alone or with cabergoline and ketoconazole in Cushing’s disease. N Eng J Med 2010;362(19):1846-8
   PubMed Web of Science ®Google Scholar
• Vilar L, Naves LA, Azevedo MF, et al. Effectiveness of cabergoline in monotherapy and combined with ketoconazole in the management of Cushing’s disease. Pituitary 2010;13(2):123-9
   PubMed Web of Science ®Google Scholar
• Kamenicky P, Droumaguet C, Salenave S, et al. Mitotane, metyrapone, and ketoconazole combination therapy as an alternative to rescue adrenalectomy for severe acth-dependent Cushing’s syndrome. J Clin Endocrinol Metab 2011;96(9):2796-804
   PubMed Web of Science ®Google Scholar
• Henry RR MS, Wetli-Hermosillo K, Ligueros-Saylan M, et al. Mechanism and management of hyperglycemia associated with pasireotide: results from studies in healthy volunteers. Endocr Rev 2011;32(03):abst P3-274
   Google Scholar
• Shenouda M, Maldonado M, Wang Y, et al. An open-label dose-escalation study of once-daily and twice-daily pasireotide in healthy volunteers: safety, tolerability, and effects on glucose, insulin, and glucagon levels. Am J Ther 2014;21(3):164-73
   PubMed Web of Science ®Google Scholar
• Barbot M, Albiger N, Ceccato F, et al. Combination therapy for Cushing’s disease: effectiveness of two schedules of treatment: should we start with cabergoline or ketoconazole? Pituitary 2014;17(2):109-17
   PubMed Web of Science ®Google Scholar
• de Bruin C, Pereira AM, Feelders RA, et al. Coexpression of dopamine and somatostatin receptor subtypes in corticotroph adenomas. J Clin Endocrinol Metab 2009;94(4):1118-24
   PubMed Web of Science ®Google Scholar
• Ren SG, Kim S, Taylor J, et al. Suppression of rat and human growth hormone and prolactin secretion by a novel somatostatin/dopaminergic chimeric ligand. J Clin Endocrinol Metab 2003;88(11):5414-21
   PubMed Web of Science ®Google Scholar
• Ferone D, Pivonello C, Vitale G, et al. Molecular basis of pharmacological therapy in Cushing’s disease. Endocrine 2014;46(2):181-98
   PubMed Web of Science ®Google Scholar
• Fleseriu M PR, Pedroncelli AM, Patino H, et al. Study design of a phase ii trial of subcutaneous pasireotide alone or combined with cabergoline in patients with Cushing’s disease. Presented at The 96th Endocrine Society’s Annual Meeting/16th International Congress of Endocrinology; 21–24 June 2014; Chicago, IL, USA; Poster Board MON-0715
   Google Scholar