Update on imatinib for gastrointestinal stromal tumors: Duration of treatment

OncoTargets and Therapy

Volumn 6, Issue , 2013, Pages 1011-1023

  Linch, Mark ^a,b   Claus, Jeroen ^b   Benson, Charlotte ^a  

^a ROYAL MARSDEN HOSPITAL   (United Kingdom)

^b IMPERIAL CANCER RESEARCH FUND   (United Kingdom)

Author keywords

Adjuvant; C kIT; Gastrointestinal stromal tumors; Imatinib; Mutations; Resistance; Treatment

Indexed keywords

BUPARLISIB; EVEROLIMUS; IMATINIB; PANOBINOSTAT; PERIFOSINE; RETASPIMYCIN; STEM CELL FACTOR RECEPTOR;

CANCER ADJUVANT THERAPY; CANCER RECURRENCE; CHRONIC MYELOID LEUKEMIA; DIARRHEA; DRUG ACTIVITY; DRUG BLOOD LEVEL; DRUG CLEARANCE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG ERUPTION; DRUG INTERMITTENT THERAPY; DRUG MEGADOSE; DRUG SAFETY; DRUG SENSITIVITY; DRUG TOLERABILITY; EDEMA; FATIGUE; GASTROINTESTINAL STROMAL TUMOR; HUMAN; LIVER METASTASIS; LOW DRUG DOSE; OVERALL SURVIVAL; PATIENT COMPLIANCE; PEROPERATIVE COMPLICATION; PROGRESSION FREE SURVIVAL; PROTEIN BINDING; PROTEIN STRUCTURE; RADIOFREQUENCY ABLATION; RECURRENCE FREE SURVIVAL; REVIEW; RISK REDUCTION; SIGNAL TRANSDUCTION; TREATMENT DURATION;

EID: 84881250045     PISSN: 11786930     EISSN: None     Source Type: Journal    
DOI: 10.2147/OTT.S31260     Document Type: Review

References (91)

1. Gatta G, van der Zwan JM, Casali PG, et al. Rare cancers are not so rare: the rare cancer burden in Europe. Eur J Cancer. 2011;47(17): 2493-2511.
2. Druker BJ, Talpaz M, Resta DJ, et al. Efficacy and safety of a specific inhibitor of the BCR-ABL tyrosine kinase in chronic myeloid leukemia. N Engl J Med. 2001;344(14):1031-1037.
3. Druker BJ, Tamura S, Buchdunger E, et al. Effects of a selective inhibitor of the Abl tyrosine kinase on the growth of Bcr-Abl positive cells. Nat Med. 1996;2(5):561-566.
4. Heinrich MC, Griffith DJ, Druker BJ, Wait CL, Ott KA, Zigler AJ. Inhibition of c-kit receptor tyrosine kinase activity by STI 571, a selective tyrosine kinase inhibitor. Blood. 2000;96(3):925-932.
5. Tuveson DA, Willis NA, Jacks T, et al. STI571 inactivation of the gastrointestinal stromal tumor c-KIT oncoprotein: biological and clinical implications. Oncogene. 2001;20(36):5054-5058.
6. Joensuu H, Roberts PJ, Sarlomo-Rikala M, et al. Effect of the tyrosine kinase inhibitor STI571 in a patient with a metastatic gastrointestinal stromal tumor. N Engl J Med. 2001;344(14):1052-1056.
7. Besmer P, Murphy JE, George PC, et al. A new acute transforming feline retrovirus and relationship of its oncogene v-kit with the protein kinase gene family. Nature. 1986;320(6061):415-421.
8. Yarden Y, Kuang WJ, Yang-Feng T, et al. Human proto-oncogene c-kit: a new cell surface receptor tyrosine kinase for an unidentified ligand. EMBO J. 1987;6(11):3341-3351.
9. DiNitto JP, Deshmukh GD, Zhang Y, et al. Function of activation loop tyrosine phosphorylation in the mechanism of c-Kit auto-activation and its implication in sunitinib resistance. J Biochem. 2010;147(4): 601-609.
10. Mol CD, Lim KB, Sridhar V, et al. Structure of a c-kit product complex reveals the basis for kinase transactivation. J Biol Chem. 2003;278(34): 31461-31464.
11. Mol CD, Fabbro D, Hosfield DJ. Structural insights into the conformational selectivity of STI-571 and related kinase inhibitors. Curr Opin Drug Discov Devel. 2004;7(5):639-648.
12. Mol CD, Dougan DR, Schneider TR, et al. Structural basis for the autoinhibition and STI-571 inhibition of c-Kit tyrosine kinase. J Biol Chem. 2004;279(30):31655-31663.
13. Lennartsson J, Ronnstrand L. Stem cell factor receptor/c-Kit: from basic science to clinical implications. Physiol Rev. 2012;92(4): 1619-1649.
14. Roskoski R Jr. Signaling by Kit protein-tyrosine kinase - the stem cell factor receptor. Biochem Biophys Res Commun. 2005;337(1):1-13.
15. Jahn T, Seipel P, Urschel S, Peschel C, Duyster J. Role for the adaptor protein Grb10 in the activation of Akt. Mol Cell Biol. 2002;22(4): 979-991.
16. van Oosterom AT, Judson I, Verweij J, et al. Safety and efficacy of imatinib (STI571) in metastatic gastrointestinal stromal tumours: a Phase I study. Lancet. 2001;358(9291):1421-1423.
17. Demetri GD, von Mehren M, Blanke CD, et al. Efficacy and safety of imatinib mesylate in advanced gastrointestinal stromal tumors. N Engl J Med. 2002;347(7):472-480.
18. Verweij J, van Oosterom A, Blay JY, et al. Imatinib mesylate (STI-571 Glivec, Gleevec) is an active agent for gastrointestinal stromal tumours, but does not yield responses in other soft-tissue sarcomas that are unselected for a molecular target. Results from an EORTC Soft Tissue and Bone Sarcoma Group Phase II study. Eur J Cancer. 2003;39(14): 2006-2011.
19. Verweij J, Casali PG, Zalcberg J, et al. Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: randomised trial. Lancet. 2004;364(9440):1127-1134.
20. Blanke CD, Rankin C, Demetri GD, et al. Phase III randomized, intergroup trial assessing imatinib mesylate at two dose levels in patients with unresectable or metastatic gastrointestinal stromal tumors expressing the kit receptor tyrosine kinase: S0033. J Clin Oncol. 2008;26(4):626-632.
21. Casali PG, Verweij J, Kotasek D, et al. Imatinib mesylate in advanced gastrointestinal stromal tumors (GIST): survival analysis of the intergroup EORTC/ISG/AGITG randomized trial in 946 patients. Eur J Cancer Suppl. 2005;3(2):201-202.
22. Gastrointestinal Stromal Tumor Meta-Analysis Group (MetaGIST). Comparison of two doses of imatinib for the treatment of unresectable or metastatic gastrointestinal stromal tumors: a meta-analysis of 1,640 patients. J Clin Oncol. 2010;28(7):1247-1253.
23. Pierotti MA, Tamborini E, Negri T, Pricl S, Pilotti S. Targeted therapy in GIST: in silico modeling for prediction of resistance. Nat Rev Clin Oncol. 2011;8(3):161-170.
24. Tamborini E, Pricl S, Negri T, et al. Functional analyses and molecular modeling of two c-Kit mutations responsible for imatinib secondary resistance in GIST patients. Oncogene. 2006;25(45):6140-6146.
25. Zalcberg JR, Verweij J, Casali PG, et al. Outcome of patients with advanced gastro-intestinal stromal tumours crossing over to a daily imatinib dose of 800 mg after progression on 400 mg. Eur J Cancer. 2005;41(12):1751-1757.
26. Blanke CD, Demetri GD, von Mehren M, et al. Long-term results from a randomized Phase II trial of standard- versus higher-dose imatinib mesylate for patients with unresectable or metastatic gastrointestinal stromal tumors expressing KIT. J Clin Oncol. 2008;26(4): 620-625.
27. Debiec-Rychter M, Sciot R, Le Cesne A, et al. KIT mutations and dose selection for imatinib in patients with advanced gastrointestinal stromal tumours. Eur J Cancer. 2006;42(8):1093-1103.
28. Judson I, Ma P, Peng B, et al. Imatinib pharmacokinetics in patients with gastrointestinal stromal tumour: a retrospective population pharmacokinetic study over time. EORTC Soft Tissue and Bone Sarcoma Group. Cancer Chemother Pharmacol. 2005;55(4):379-386.
29. Demetri GD,Wang Y, Wehrle E, et al., Imatinib plasma levels are correlated with clinical benefit in patients with unresectable/metastatic gastrointestinal stromal tumors. J Clin Oncol. 2009;27(19):3141-3147.
30. Eechoute K, Fransson MN, Reyners AK, et al. A long-term prospective population pharmacokinetic study on imatinib plasma concentrations in GIST patients. Clin Cancer Res. 2012;18(20):5780-5787.
31. Bartolovic K, Balabanov S, Hartmann U, et al. Inhibitory effect of imatinib on normal progenitor cells in vitro. Blood. 2004;103(2):523-529.
32. Druker BJ, Guilhot F, O'Brien SG, et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med. 2006;355(23):2408-2417.
33. Song MK, Chung JS, Seol YM, et al. Mean cell volume can be an early predictor for the cytogenetic response of chronic myeloid leukemia patients treated with imatinib? Leuk Res. 2009;33(11):1459-1462.
34. Constantinidou A, Krikelis D, Olmos D, et al. Early assessment of MCV to predict clinical outcome in patients with advanced gastrointestinal stromal tumors (GIST) receiving imatinib. J Clin Oncol. 2012;30 Suppl:S10086.
35. Le Cesne A, Ray-Coquard I, Bui BN, et al. Discontinuation of imatinib in patients with advanced gastrointestinal stromal tumours after 3 years of treatment: an open-label multicentre randomised Phase 3 trial. Lancet Oncol. 2010;11(10):942-949.
36. Patrikidou A, Chabaud S, Ray-Coquard I, et al. Influence of imatinib interruption and rechallenge on the residual disease in patients with advanced GIST: results of the BFR14 prospective French Sarcoma Group randomised, Phase III trial. Ann Oncol. 2013;24(4):1087-1093.
37. Conley AP, Araujo D, Ludwig J, et al. A randomized Phase II study of perifosine (P) plus imatinib for patients with imatinib-resistant gastrointestinal stromal tumor (GIST). J Clin Oncol. 2009;27(Suppl 15): 10563.
38. Schoffski P, Reichardt P, Blay JY, et al. A Phase I-II study of everolimus (RAD001) in combination with imatinib in patients with imatinib-resistant gastrointestinal stromal tumors. Ann Oncol. 2010;21(10):1990-1998.
39. Demetri GD, Le-Cesne A, von Mehren M, et al. Final results form a Phase III study of IPI-504 (retaspimycin hydrochloride) versus placebo in patients (pts) with gastrointestinal stromal tumors (GIST) following failure of kinase inhibitor therapies. Abstract presented at the Gastrointestinal Cancers Symposium, Orlando, FL, January 22-24, 2010.
40. Wagner AJ, Morgan JA, Chugh R, et al. Inhibition of heat shock protein 90 (Hsp90) with the novel agent IPI-504 in metastatic GIST following failure of tyrosine kinase inhibitors (TKIs) or other sarcomas: clinical results from Phase I trial. J Clin Oncol. 2008;26(Suppl 15):10503.
41. Bauer S, Hilger R, Grabellus F, et al. Phase I trial of panobinostat (P) and imatinib (IM) in patients with treatment-refractory gastrointestinal stromal tumors (GIST). J Clin Oncol. 2012;30 Suppl:10032.
42. Raut CP, Posner M, Desai J, et al. Surgical management of advanced gastrointestinal stromal tumors after treatment with targeted systemic therapy using kinase inhibitors. J Clin Oncol. 2006;24(15): 2325-2331.
43. Mussi C, Ronellenfitsch U, Jakob J, et al. Post-imatinib surgery in advanced/metastatic GIST: is it worthwhile in all patients? Ann Oncol. 2010;21(2):403-408.
44. Al-Batran SE, Hartmann JT, Heidel F, et al. Focal progression in patients with gastrointestinal stromal tumors after initial response to imatinib mesylate: a three-center-based study of 38 patients. Gastric Cancer. 2007;10(3):145-152.
45. Demetri GD, van Oosterom AT, Garrett CR, et al. Efficacy and safety of sunitinib in patients with advanced gastrointestinal stromal tumour after failure of imatinib: a randomised controlled trial. Lancet. 2006;368(9544):1329-1338.
46. Jones RL, McCall J, Adam A, et al. Radiofrequency ablation is a feasible therapeutic option in the multi modality management of sarcoma. Eur J Surg Oncol. 2010;36(5):477-482.
47. Dileo P, Randhawa R, Vansonnenberg E, et al. Safety and efficacy of percutaneous radio-frequency ablation (RFA) in patients (pts) with metastatic gastrointestinal stromal tumor (GIST) with clonal evolution of lesions refractory to imatinib mesylate (IM). J Clin Oncol. 2004;22(Suppl 14):9024.
48. Miettinen M, Lasota J. Gastrointestinal stromal tumors: review on morphology, molecular pathology, prognosis, and differential diagnosis. Arch Pathol Lab Med. 2006;130(10):1466-1478.
49. Fletcher CD, Berman JJ, Corless C, et al. Diagnosis of gastrointestinal stromal tumors: a consensus approach. Hum Pathol. 2002;33(5): 459-465.
50. Miettinen M, Lasota J. Gastrointestinal stromal tumors: pathology and prognosis at different sites. Semin Diagn Pathol. 2006;23(2):70-83.
51. Gold JS, Gonen M, Gutierrez A, et al. Development and validation of a prognostic nomogram for recurrence-free survival after complete surgical resection of localised primary gastrointestinal stromal tumour: a retrospective analysis. Lancet Oncol. 2009;10(11):1045-1052.
52. Joensuu H, Vehtari A, Riihimaki J, et al. Risk of recurrence of gastrointestinal stromal tumour after surgery: an analysis of pooled population-based cohorts. Lancet Oncol. 2012;13(3):265-274.
53. Dematteo RP, Ballman KV, Antonescu CR, et al. Adjuvant imatinib mesylate after resection of localised, primary gastrointestinal stromal tumour: a randomised, double-blind, placebo-controlled trial. Lancet. 2009;373(9669):1097-1104.
54. Joensuu H, Eriksson M, Sundby Hall K, et al. One vs three years of adjuvant imatinib for operable gastrointestinal stromal tumor: a randomized trial. JAMA. 2012;307(12):1265-1272.
55. [No authors listed]. Gastrointestinal stromal tumors: ESMO clinical practice guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2012;23 Suppl 7:vii49-vii55.
56. Casali PG, Fumagalli E, Gronchi A. Adjuvant therapy of gastrointestinal stromal tumors (GIST). Curr Treat Options Oncol. 2012;13(3): 277-284.
57. Chen J, Guo T, Zhang L, et al. CD133 and CD44 are universally overexpressed in GIST and do not represent cancer stem cell markers. Genes Chromosomes Cancer. 2012;51(2):186-195.
58. Blay JY, Le Cesne A, Ray-Coquard I, et al. Prospective multicentric randomized Phase III study of imatinib in patients with advanced gastrointestinal stromal tumors comparing interruption versus continuation of treatment beyond 1 year: the French Sarcoma Group. J Clin Oncol. 2007;25(9):1107-1113.
59. Eisenberg BL, Harris J, Blanke CD, et al. Phase II trial of neoadjuvant/ adjuvant imatinib mesylate (IM) for advanced primary and metastatic/ recurrent operable gastrointestinal stromal tumor (GIST): early results of RTOG 0132/ACRIN 6665. J Surg Oncol. 2009;99(1):42-47.
60. Wang D, Zhang Q, Blanke CD, et al. Phase II trial of neoadjuvant/ adjuvant imatinib mesylate for advanced primary and metastatic/ recurrent operable gastrointestinal stromal tumors: long-term follow-up results of Radiation Therapy Oncology Group 0132. Ann Surg Oncol. 2012;19(4):1074-1080.
61. Doyon C, Sideris L, Leblanc G, Leclerc YE, Boudreau D, Dube P. Prolonged therapy with imatinib mesylate before surgery for advanced gastrointestinal stromal tumor results of a Phase II trial. Int J Surg Oncol. 2012;2012:761576.
62. Hohenberger P, Langer C, Wendtner CM, et al. Neoadjuvant treatment of locally advanced GIST: results of APOLLON, a prospective, open label Phase II study in KIT- or PDGFRA-positive tumors. J Clin Oncol. 2012;30 Suppl:10031.
63. Reichardt P, Blay JY, Boukovinas I, et al. Adjuvant therapy in primary GIST: state-of-the-art. Ann Oncol. 2012;23(11):2776-2781.
64. Marin D, Bazeos A, Mahon FX, et al. Adherence is the critical factor for achieving molecular responses in patients with chronic myeloid leukemia who achieve complete cytogenetic responses on imatinib. J Clin Oncol. 2010;28(14):2381-2388.
65. Kerkela R, Grazette L, Yacobi R, et al. Cardiotoxicity of the cancer therapeutic agent imatinib mesylate. Nat Med. 2006;12(8):908-916.
66. Berman E, Nicolaides M, Maki RG, et al. Altered bone and mineral metabolism in patients receiving imatinib mesylate. N Engl J Med. 2006;354(19):2006-2013.
67. Berman E, Girotra M, Cheng C, et al. Effect of long term imatinib on bone in adults with chronic myelogenous leukemia and gastrointestinal stromal tumors. Leuk Res. 2013;37(7):790-794.
68. Hamberg P,de Jong FA, Boonstra JG, van Doorn J, Verweij J, Sleijfer S., Non-islet-cell tumor induced hypoglycemia in patients with advanced gastrointestinal stromal tumor possibly worsened by imatinib. J Clin Oncol. 2006;24(18):e30-e31.
69. Lux ML, Rubin BP, Biase TL, et al. KIT extracellular and kinase domain mutations in gastrointestinal stromal tumors. Am J Pathol. 2000;156(3): 791-795.
70. Miettinen M, Lasota J. Gastrointestinal stromal tumors -definition, clinical, histological, immunohistochemical, and molecular genetic features and differential diagnosis. Virchows Arch. 2001;438(1): 1-12.
71. Conca E, Miranda C, Col VD, et al. Are two better than one? A novel double-mutant KIT in GIST that responds to imatinib. Mol Oncol. March 21, 2013. [Epub ahead of print.]
72. Roskoski R Jr. Structure and regulation of Kit protein-tyrosine kinase - the stem cell factor receptor. Biochem Biophys Res Commun. 2005;338(3):1307-1315.
73. Conca E, Negri T, Gronchi A, et al. Activate and resist: L576P-KIT in GIST. Mol Cancer Ther. 2009;8(9):2491-2495.
74. Tamborini E, Bonadiman L, Greco A, et al. A new mutation in the KIT ATP pocket causes acquired resistance to imatinib in a gastrointestinal stromal tumor patient. Gastroenterology. 2004;127(1):294-299.
75. Duensing A, Medeiros F, McConarty B, et al. Mechanisms of oncogenic KIT signal transduction in primary gastrointestinal stromal tumors (GISTs). Oncogene. 2004;23(22):3999-4006.
76. Rossi F, Ehlers I, Agosti V, et al. Oncogenic Kit signaling and therapeutic intervention in a mouse model of gastrointestinal stromal tumor. Proc Natl Acad Sci U S A. 2006;103(34):12843-12848.
77. Baird K, Davis S, Antonescu CR, et al. Gene expression profiling of human sarcomas: insights into sarcoma biology. Cancer Res. 2005;65(20):9226-9235.
78. Francis P, Namlos HM, Muller C, et al. Diagnostic and prognostic gene expression signatures in 177 soft tissue sarcomas: hypoxia-induced transcription profile signifies metastatic potential. BMC Genomics. 2007;8:73.
79. Astolfi A, Nannini M, Pantaleo MA, et al. A molecular portrait of gastrointestinal stromal tumors: an integrative analysis of gene expression profiling and high-resolution genomic copy number. Lab Invest. 2010;90(9):1285-1294.
80. Li F, Growney J, Battalagine L, Qiu S, Manley P, Monahan J. The effect combining the KIT inhibitor imatinib with the PI3K inhibitor BKM120 or the dual PI3K/mTOR inhibitor BEZ235 on the proliferation of gastrointestinal stromal tumor cell lines. Abstract 2239 presented at the 103rd Annual Meeting of the American Association for Cancer Research, Chicago, IL, March 31-April 4, 2012.
81. Floris G, Wozniak A, Sciot R, et al. A potent combination of the novel PI3K inhibitor, GDC-0941, with imatinib in gastrointestinal stromal tumor xenografts: long-lasting responses after treatment withdrawal. Clin Cancer Res. 2013;19(3):620-630.
82. Chang BS, Yang T, Cibas ES, Fletcher JA. An in vitro cytologic assay for evaluation of the KIT signaling pathway in gastrointestinal stromal tumors. Mod Pathol. 2007;20(5):579-583.
83. Pantaleo MA, Nicoletti G, Nanni C, et al. Preclinical evaluation of KIT/ PDGFRA and mTOR inhibitors in gastrointestinal stromal tumors using small animal FDG PET. J Exp Clin Cancer Res. 2010;29:173.
84. Duensing A, Joseph NE, Medeiros F, et al. Protein kinase C theta (PKCtheta) expression and constitutive activation in gastrointestinal stromal tumors (GISTs). Cancer Res. 2004;64(15):5127-5131.
85. Ou WB, Zhu MJ, Demetri GD, Fletcher CD, Fletcher JA. Protein kinase C-theta regulates KIT expression and proliferation in gastrointestinal stromal tumors. Oncogene. 2008;27(42):5624-5634.
86. Bauer S, Yu LK, Demetri GD, Fletcher JA. Heat shock protein 90 inhibition in imatinib-resistant gastrointestinal stromal tumor. Cancer Res. 2006;66(18):9153-9161.
87. Smyth T, Van Looy T, Curry JE, et al. The HSP90 inhibitor, AT13387, is effective against imatinib-sensitive and -resistant gastrointestinal stromal tumor models. Mol Cancer Ther. 2012;11(8):1799-1808.
88. Floris G, Debiec-Rychter M, Wozniak A, et al. The heat shock protein 90 inhibitor IPI-504 induces KIT degradation, tumor shrinkage, and cell proliferation arrest in xenograft models of gastrointestinal stromal tumors. Mol Cancer Ther. 2011;10(10):1897-1908.
89. Muhlenberg T, Zhang Y, Wagner AJ, et al. Inhibitors of deacetylases suppress oncogenic KIT signaling, acetylate HSP90, and induce apoptosis in gastrointestinal stromal tumors. Cancer Res. 2009;69(17): 6941-6950.
90. Edris B, Willingham SB, Weiskopf K, et al. Anti-KIT monoclonal antibody inhibits imatinib-resistant gastrointestinal stromal tumor growth. Proc Natl Acad Sci U S A. 2013;110(9):3501-3506.
91. Heinrich MC, Corless CL, Blanke CD, et al. Molecular correlates of imatinib resistance in gastrointestinal stromal tumors. J Clin Ocol: Official Journal of the American Society of Clinical Oncology. Oct 10 2006;24(29):4764-4774.