Pasireotide for the treatment of cushing's disease

Expert Opinion on Investigational Drugs

Volumn 19, Issue 7, 2010, Pages 889-898

  Arnaldi, Giorgio ^a   Boscaro, Marco ^a  

^a OSPEDALI RIUNITI   (Italy)

Author keywords

Cushing's disease; Medical therapy; Pasireotide (SOM230); Pituitary directed drugs; Somatostatin receptors

Indexed keywords

ANGIOPEPTIN; CORTICOTROPIN; GROWTH HORMONE; OCTREOTIDE; PASIREOTIDE; SOMATOMEDIN C; SOMATOSTATIN RECEPTOR 1; SOMATOSTATIN RECEPTOR 2; SOMATOSTATIN RECEPTOR 3; SOMATOSTATIN RECEPTOR 4; SOMATOSTATIN RECEPTOR 5;

ADRENALECTOMY; AREA UNDER THE CURVE; BINDING AFFINITY; CLINICAL TRIAL; CUSHING DISEASE; CUSHING SYNDROME; DISEASE SEVERITY; DRUG BLOOD LEVEL; DRUG EFFICACY; FOLLOW UP; GASTROINTESTINAL DISEASE; GROWTH HORMONE RELEASE; HUMAN; HYPERCORTISOLISM; HYPERGLYCEMIA; HYPOPHYSECTOMY; INHIBITION KINETICS; INJECTION SITE REACTION; MAXIMUM PLASMA CONCENTRATION; MEDICAL DECISION MAKING; MEDICAL RESEARCH; MORBIDITY; MORTALITY; NONHUMAN; PALLIATIVE THERAPY; RECEPTOR BINDING; RECURRENCE RISK; RELAPSE; REVIEW; SINGLE DRUG DOSE; TREATMENT FAILURE; TREATMENT OUTCOME;

ADRENOCORTICOTROPIC HORMONE; ANIMALS; CLINICAL TRIALS, PHASE III AS TOPIC; CUSHING SYNDROME; DRUG EVALUATION, PRECLINICAL; HUMANS; PROTEIN BINDING; RECEPTORS, SOMATOSTATIN; SOMATOSTATIN; TREATMENT OUTCOME;

EID: 77953413026     PISSN: 13543784     EISSN: None     Source Type: Journal    
DOI: 10.1517/13543784.2010.495943     Document Type: Review

References (89)

1. Arnaldi G, Angeli A, Atkinson AB, et al. Diagnosis and complications of Cushing's syndrome: a consensus statement. J Clin Endocrinol Metab 2003;19:5593-5602
2. 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;93:1526-1540
3. Arnaldi G, Mancini T, Polenta B, Boscaro M. Cardiovascular risk in Cushing's syndrome. Pituitary 2004;7(4):253-256
4. Pivonello R, Faggiano A, Lombardi G, et al. The metabolic syndrome and cardiovascular risk in Cushing's syndrome. Endocrinol Metab Clin North Am 2005;34:327-339
5. Biller BM, Grossman AB, Stewart PM, et al. Treatment of adrenocorticotropin-dependent Cushing's syndrome: a consensus statement. J Clin Endocrinol Metab 2008;93:2454-2462
6. Bochicchio D, Losa M, Buchfelder M. Factors influencing the immediate and late outcome of Cushing's disease treated by transsphenoidal surgery: a retrospective study by the European Cushing's Disease Survey Group. J Clin Endocrinol Metab 1995;80(11):3114-3120
7. Patil CG, Prevedello DM, Lad SP, et al. Late recurrences of Cushing's disease after initial successful transsphenoidal surgery. J Clin Endocrinol Metab 2008;93:358-362
8. Prevedello DM, Pouratian N, Sherman J, et al. Management of Cushing's disease: outcome in patients with microadenoma detected on pituitary magnetic resonance imaging. J Neurosurg 2008;109:751-759
9. Hammer GD, Tyrrell JB, Lamborn KR, et al. Transsphenoidal microsurgery for Cushing's disease: initial outcome and long-term results. J Clin Endocrinol Metab 2004;89:6348-6357
10. 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 (Oxf) 2005;63:549-559
11. Patil CG, Veeravagu A, Prevedello DM, et al. Outcomes after repeat transsphenoidal surgery for recurrent cushing's disease. Neurosurgery 2008;63:266-270
12. Aghi MK. Management of recurrent and refractory Cushing disease. Nat Clin Pract Endocrinol Metab 2008;4(10):560-568
13. Mullan KR, Atkinson B. Endocrine clinical update: Where are we in the therapeutic management of pituitary-dependent hypercortisolism? Clin Endocrinol 2008;68:327-337
14. Castinetti F, Nagai M, Dufour H, et al. Gamma knife radiosurgery is a successful adjunctive treatment in Cushing's disease. Eur J Endocrinol 2007;156:91-98
15. Jagannathan J, Sheehan JP, Pouratian N, et al. Gamma Knife surgery for Cushing's disease. J Neurosurg 2007;106:980-987
16. Petit JH, Biller BM, Yock TI, et al. Proton stereotactic radiotherapy for persistent adrenocorticotropin-producing adenomas. J Clin Endocrinol Metab 2008;93:393-399
17. Chow JT, Thompson GB, Grant CS, et al. Bilateral laparoscopic adrenalectomy for corticotrophin-dependent Cushing's syndrome: a review of the Mayo Clinic experience. Clin Endocrinol (Oxf) 2008;68:513-519
18. Thompson SK, Hayman AV, Ludlam WH, et al. Improved quality of life after bilateral laparoscopic adrenalectomy for Cushing's disease: a 10-year experience. Ann Surg 2007;245:790-794
19. 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;92:172-179
20. Miller JW, Crapo L. The medical treatment of Cushing's syndrome. Endocr Rev 1993;14(4):443-458
21. Nieman LK. Medical therapy of Cushing's disease. Pituitary 2002;5(2):77-82
22. Arnaldi G, Cardinaletti M, Boscaro M. Advances in medical treatment of Cushing's disease. Expert Rev Endocrinol Metab 2007;2(6):735-743
23. Alexandraki KI, Grossman AB. Pituitary-targeted medical therapy of Cushing's disease. Expert Opin Investig Drugs 2008;17(5):669-677
24. Castinetti F, Fassnacht M, Johanssen S, et al. Merits and pitfalls of mifepristone in Cushing's syndrome. Eur J Endocrinol 2009;160(6):1003-1010
25. 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):91-99
26. 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):1281-1287
27. Ambrosi B, Dall'Asta C, Cannavo S, et al. Effects of chronic administration of PPAR-gamma ligand rosiglitazone in Cushing's disease. Eur J Endocrinol 2004;151(2):173-178
28. Pivonello R, Ferone D, de Herder WW. Dopamine receptor expression and function in corticotroph pituitary tumors. J Clin Endocrinol Metab 2004;89(5):2452-2462 (Pubitemid 38619889)
29. Pivonello R, De Martino MC, Cappabianca P, et al. The medical treatment of Cushing's disease: effectiveness of chronic treatment with the dopamine agonist cabergoline in patients unsuccessfully treated by surgery. J Clin Endocrinol Metab 2008;94(1):223-230
30. 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):707-716
31. 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
32. Patel YC. Somatostatin and its receptor family. Front Neuroendocrinol 1999;20(3):157-198
33. Kraus J, Woltje M, Schonwetter N, Hollt V. Alternative pro-moter usage and tissue specific expression of the mouse somatostatin receptor 2 gene. FEBS Lett 1998;3:165-170
34. Weckbecker G, Lewis I, Albert R, et al. Opportunities in somatostatin research: biological, chemical and therapeutic aspects [review]. Nat Rev Drug Discov 2003;2(12):999-1017.
35. Erratum in: Nat Rev Drug Discov 2005;4(12):975
36. Ben-Shlomo A, Zhou C, Pichurin O, et al. Constitutive somatostatin receptor activity determines tonic pituitary cell response. Mol Endocrinol 2009;23(3):337-348
37. Ferone D, Gatto F, Arvigo M, et al. The clinical-molecular interface of somatostatin, dopamine and their receptors in pituitary pathophysiology. J Mol Endocrinol 2009;42(5):361-370
38. Liu Q, Bee MS, Schonbrunn A. Site specificity of agonist and second messenger-activated kinases for somatostatin receptor subtype 2A (Sst2A) phosphorylation. Mol Pharmacol 2009;76(1):68-80
39. Schonbrunn A. Selective agonism in somatostatin receptor signaling and regulation. Mol Cell Endocrinol 2008;286(1-2):35-39
40. Jacobs S, Schulz S. Intracellular trafficking of somatostatin receptors. Mol Cell Endocrinol 2008;286(1-2):58-62
41. Hofland LJ, Lamberts SW. The pathophysiological consequences of somatostatin receptor internalization and resistance. Endocr Rev 2003;24(1):28-47
42. Duran-Prado M, Malagon MM, Gracia-Navarro F, Castano JP. Dimerization of G protein-coupled receptors: new avenues for somatostatin receptor signalling,control and functioning. Mol Cell Endocrinol 2008;286(1-2):63-68
43. Grant M, Alturaihi H, Jaquet P, et al. Cell growth inhibition and functioning of human somatostatin receptor type 2 are modulated by receptor heterodimerization. Mol Endocrinol 2008;22:2278-2292
44. Ren SG, Kim S, Taylor J, et al. Functional association of somatostatin receptor subtypes 2 and 5 in inhibiting human growth hormone secretion. J Clin Endocrinol Metab 2003;88:4239-4245
45. Murray RD, Melmed S. A critical analysis of clinically available somatostatin analog formulations for therapy of acromegaly. J Clin Endocrinol Metab 2008;93(8):2957-2968
46. Modlin IM, Pavel M, Kidd M, Gustafsson BI. Review article: somatostatin analogues in the treatment of gastroenteropancreatic neuroendocrine (carcinoid) tumours. Aliment Pharmacol Ther 2010;31(2):169-188
47. Van der Hoek J, Lamberts SW, Hofland LJ. The role of somatostatin analogs in Cushing's disease. Pituitary 2004;7(4):257-264
48. Arnaldi G, Polenta B, Cardinaletti M, Boscaro M. Potential indications for somatostatin analogs in Cushing's syndrome. J Endocrinol Invest 2005;28(Suppl 11):106-110
49. Lamberts SW, Uitterlinden P, Klijn JM. The effect of the long-acting somatostatin analogue SMS 201-995 on ACTH secretion in Nelson's syndrome and Cushing's disease. Acta Endocrinol (Copenh) 1989;6:760-766
50. Stalla GK, Brockmeier SJ, Renner U, et al. Octreotide exerts different effects in vivo and in vitro in Cushing's disease. Eur J Endocrinol 1994;2:125-131
51. Ambrosi B, Bochicchio D, Fadin C, et al. Failure of somatostatin and octreotide to acutely affect the hypothalamic-pituitary-adrenal function in patients with corticotropin hypersecretion. J Endocrinol Invest 1990;3:257-261
52. Invitti C, de Martin M, Brunani A, et al. Treatment of Cushing's syndrome with the long-acting somatostatin analogue SM 201-995 (sandostatin). Clin Endocrinol (Oxf) 1990;32(3):275-281
53. 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):4482-4488
54. Schonbrunn A. Glucocorticoids down-regulate somatostatin receptors on pituitary cells in culture. Endocrinology 1982;4:1147-1154
55. Kraus J, Woltje M, Hollt V. Regulation of mouse somatostatin receptor type 2 gene expression by glucocorticoids. FEBS Lett 1999;2:200-204
56. 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
57. Strowski MZ, Dashkevicz MP, Parmar RM, et al. Somatostatin receptor subtypes 2 and 5 inhibit corticotropin-releasing hormone-stimulated adrenocorticotropin secretion from AtT-20 cells. Neuroendocrinology 2002;6:339-346
58. Cervia D, Nunn C, Fehlmann D, et al. Pharmacological characterisation of native somatostatin receptors in AtT-20 mouse tumour corticotrophs. Br J Pharmacol 2003;1:109-121
59. Stafford PJ, Kopelman PG, Davidson K, et al. The pituitary-adrenal response to CRF-41 is unaltered by intravenous somatostatin in normal subjects. Clin Endocrinol (Oxf) 1989;30(6):661-666
60. Fehm HL, Voigt KH, Lang R, et al. Somatostatin: a potent inhibitor of ACTH-hypersecretion in adrenal insufficiency. Klin Wochenschr 1976;4:173-175
61. Ben-Shlomo A, Wawrowsky KA, Proekt I, et al. Somatostatin receptor type 5 modulates somatostatin receptor type 2 regulation of adrenocorticotropin secretion. J Biol Chem 2005;280:24011-24021
62. Ben-Shlomo A, Schmid H, Wawrowsky K, et al. Differential ligand-mediated pituitary somatostatin receptor subtype signaling: implications for corticotroph tumor therapy. J Clin Endocrinol Metab 2009;94(11):4342-4350
63. De Bruin C, Feelders RA, Lamberts SW, Hofland LJ. Somatostatin and dopamine receptors as targets for medical treatment of Cushing's Syndrome. Rev Endocr Metab Disord 2009;10(2):91-102
64. Hofland LJ. Somatostatin and somatostatin receptors in Cushing's disease. Mol Cell Endocrinol 2008;286(1-2):199-205
65. Schmid HA. Pasireotide (SOM230): development, mechanism of action and potential applications. Mol Cell Endocrinol 2008;286(1-2):69-74
66. Lesche S, Lehmann D, Nagel F, et al. Differential effects of octreotide and pasireotide on somatostatin receptor internalization and trafficking in vitro. J Clin Endocrinol Metab 2009;94(2):654-661
67. Poll F, Lehmann D, Illing S, et al. Pasireotide and octreotide stimulate distinct patterns of sst2A somatostatin receptor phosphorylation. Mol Endocrinol 2010;24(2):436-446
68. Cescato R, Loesch KA, Waser B, et al. Agonist-biased signaling at the sst2A receptor: the multi-somatostatin analogs KE108 and SOM230 activate and antagonize distinct signaling pathways. Mol Endocrinol 2010;24(1):240-249
69. Schmid HA, Silva AP. Short-and long-term effects of octreotide and SOM230 on GH, IGF-I, ACTH, corticosterone and ghrelin in rats. J Endocrinol Invest 2005;28(Suppl 11):28-35
70. Weckbecker G, Briner U, Lewis I, Bruns C. SOM230: a new somatostatin peptidomimetic with potent inhibitory effects on the growth hormone/insulinlike growth factor-I axis in rats, primates, and dogs. Endocrinology 2002;143(10):4123-4130
71. Schmid HA, Brueggen J, Guitard P. Effect of a long acting release formulation of pasireotide (SOM230) on hormone secretion in rats. 89th Endocrine Society Annual Meeting ENDO2007, June 2-5, 2007, Toronto, Canada, Abstract Book, P3.337
72. van der Hoek J, de Herder WW, Feelders RA, et al. A single-dose comparison of the acute effects between the new somatostatin analog SOM230 and octreotide in acromegalic patients. J Clin Endocrinol Metab 2004;89:638-645
73. Ma P, Wang Y, van der Hoek J, et al. Pharmacokinetic-pharmacodynamic comparison of a novel multiligand somatostatin analog, SOM230, with octreotide in patients with acromegaly. Clin Pharmacol Ther 2005;78(1):69-80
74. Farral A, Glusman JE, Buchelt A, et al. Pasireotide (SOM230) effectively reduces pituitary tumor volume in patients with active acromegaly: results from a Phase II trial. 89th Endocrine Society Annual Meeting ENDO2007, June 2-5, 2007, Toronto, Canada, Abstract Book OR 53-54
75. Kvlos L, Wiedenmann B, Oberg K, et al. The SOM230 Carcinoid Study Group 2006. Safety and efficacy of pasireotide (SOM230) in patients with metastatic carcinoid tumors refractory or resistant to octreotide LAR: results from a Phase II trial. J Clin Oncol 2006 ASCO Annual Meeting Proceedings Post-Meeting Edition 2006;24(18S Suppl):4082
76. Fedele M, De Martino I, Pivonello R, et al. SOM230, a new somatostatin analogue, is highly effective in the therapy of growth hormone/prolactin- secreting pituitary adenomas. Clin Cancer Res 2007;13(9):2738-2744
77. Hofland LJ, van der Hoek J, van Koetsveld PM, et al. The novel somatostatin analog SOM230 is a potent inhibitor of hormone release by growth hormone-and prolactin-secreting pituitary adenomas in vitro. J Clin Endocrinol Metab 2004;89(4):1577-1585
78. Zatelli MC, Piccin D, Vignali C, et al. Pasireotide, a multiple somatostatin receptor subtypes ligand, reduces cell viability in non-functioning pituitary adenomas by inhibiting vascular endothelial growth factor secretion. Endocr Relat Cancer 2007;14(1):91-102
79. Danila DC, Haidar JN, Zhang X, et al. Somatostatin receptor-specific analogs: effects on cell proliferation and growth hormone secretion in human somatotroph tumors. J Clin Endocrinol Metab 2001;86:2976-2981
80. Nolan LA, Schmid HA, Levy A. Octreotide and the novel multireceptor ligand somatostatin receptor agonist pasireotide (SOM230) block the adrenalectomy-induced increase in mitotic activity in male rat anterior pituitary. Endocrinology 2007;148(6):2821-2827
81. Cukier K, Tewari R, Kurth F, et al. Significant response to pasireotide (SOM230) in the treatment of a patient with persistent, refractory Cushing's disease. Clin Endocrinol (Oxf) 2009;71(2):305-307
82. Dutreix C, Degen L, Schroller S, et al. The safety, tolerability,and pharmacokinetics of multiple daily doses of SOM230, a novel multiligand somatostatin analog [abstract]. Proceedings of the 12th International Congress of Endocrinology, 31 August-4 September, 2004. Lisbon, Portugal. Abstract p 761-765
83. Boscaro M, Ludlam WH, Atkinson B, et al. Treatment of pituitary-dependent Cushing's Disease with Multireceptor Ligand Somatostatin Analog Pasireotide (SOM230): a multicenter, Phase II Trial. J Clin Endocrinol Metab 2009;94(1):, 115-122
84. Boscaro M, Zhang Y, Sen K, et al. on behalf of the Pasireotide Cushing's Disease Study Group UFC response is related to pasireotide (SOM230) plasma concentrations in patients with Cushing's disease [abstract]. 26-30 March 2010 14th International Congress of Endocrinology (ICE), Kyoto, Japan; 2010. p. 7-6-4
85. Unger N, Serdiuk I, Sheu SY, et al. Immunohistochemical localization of somatostatin receptor subtypes in benign and malignant adrenal tumours. Clin Endocrinol (Oxf) 2008;68:850-857
86. De Bruin C, Pereira AM, Feelders RA, et al. Co-expression of dopamine and somatostatin receptor subtypes in corticotroph adenomas. J Clin Endocrinol Metab 2009;94(4):1118-1124
87. De Bruin C, Feelders RA, Waaijers AM, et al. Differential regulation of human dopamine D2 and somatostatin receptor subtype expression by glucocorticoids in vitro. J Mol Endocrinol 2009;42(1):47-56
88. Dalm VA, Hofland LJ, Lamberts SW. Future clinical prospects in somatostatin/cortistatin/somatostatin receptor field. Mol Cell Endocrinol 2008;286(1-2):262-277
89. Feelders RA, de Bruin C, Pereira AM, et al. Pasireotide alone or with cabergoline and ketoconazole in Cushing's disease. N Engl J Med 2010;362(19):1846-1848