Current standards and progress in understanding and treatment of GIST

Swiss Medical Weekly

Volumn 139, Issue 7-8, 2009, Pages 90-102

  Dirnhofef, Stephan ^a   Leyvraz, Serge ^b  

^a UNIVERSITY HOSPITAL BASEL   (Switzerland)

^b LAUSANNE UNIVERSITY HOSPITAL   (Switzerland)

Author keywords

c KIT; Cancer; Diagnosis; Dosage; Gastrointestinal stromal tumour; GIST; Imatinib; Metastasis; Mutation analysis; PDGFR ; Review; Sunitinib; Surgery; Treatment; Tyrosine kinase inhibitor

Indexed keywords

IMATINIB; PLATELET DERIVED GROWTH FACTOR ALPHA RECEPTOR; STEM CELL FACTOR; SUNITINIB; TUMOR MARKER;

CANCER ADJUVANT THERAPY; CANCER DIAGNOSIS; CANCER EPIDEMIOLOGY; CANCER INCIDENCE; CANCER SURGERY; CANCER SURVIVAL; DRUG COST; DRUG DOSE COMPARISON; DRUG DOSE ESCALATION; DRUG MEGADOSE; DRUG SAFETY; DRUG TOLERABILITY; GASTROINTESTINAL STROMAL TUMOR; HISTOPATHOLOGY; HUMAN; METASTASIS; PATHOPHYSIOLOGY; PATIENT COMPLIANCE; PRACTICE GUIDELINE; PROGNOSIS; REVIEW; TREATMENT OUTCOME;

EID: 61549093107     PISSN: 14247860     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (133)

1. Hirota S, Isozaki K, Moriyama Y, Hashimoto K, Nishida T, Ishiguro S, et al. Gain-of-function mutations of c-kit in human gastrointestinal stromal tumors. Science. 1998;279(5350):577-80.
2. Kindblom LG, Remotti HE, Aldenborg F, Meis-Kindblom JM. Gastrointestinal pacemaker cell tumor (GIPACT): gastrointestinal stromal tumors show phenotypic characteristics of the interstitial cells of Cajal. Am J Pathol. 1998;152(5):1259-69.
3. Steigen SE, Eide TJ. Trends in incidence and survival of mesenchymal neoplasms of the digestive tract within a defined population of northern Norway. APMIS. 2006;114(3):192-200.
4. Blay JY, Le Cesne A, Verweij J, Scurr M, Seynaeve C, Bonvalot S, et al. A phase II study of ET-743/trabectedin ("Yondelis") for patients with advanced gastrointestinal stromal tumours. Eur J Cancer. 2004:40(9):1327-31.
5. Ryan DP, Puchalski T, Supko JG, Harmon D, Maki R, Garcia-Carbonero R, et al. A phase II and pharmacokinetic study of ecteinascidin 743 in patients with gastrointestinal stromal tumors. Oncologist. 2002;7(6):531-8.
6. Ng EH, Pollock RE, Munsell MF, Atkinson EN, Romsdahl MM. Prognostic factors influencing survival in gastrointestinal leiomyosarcomas. Implications for surgical management and staging. Ann Surg. 1992;215(1):68-77.
7. Ng EH, Pollock RE, Romsdahl MM. Prognostic implications of patterns of failure for gastrointestinal leiomyosarcomas. Cancer. 1992;69(6):1334-41.
8. DeMatteo RP, Lewis JJ, Leung D, Mudan SS, Woodruff JM, Brennan MF. Two hundred gastrointestinal stromal tumors: recurrence patterns and prognostic factors for survival. Ann Surg. 2000;231(1):51-8.
9. Gold JS, van der Zwan SM, Gonen M, Maki RG, Singer S, Brennan MF, et al. Outcome of metastatic GIST in the era before tyrosine kinase inhibitors. Ann Surg Oncol. 2007;14(1):134-42.
10. Sarlomo-Rikala M, Kovadch AJ, Barusevicius A, Miettinen M. CD117: a sensitive marker for gastrointestinal stromal tumors that is more specific than CD34. Mod Pathol. 1998;11(8):728-34.
11. Joensuu H, Roberts PJ, Sarlomo-Rikala M, Andersson LC, Tervahartiala P, Tuveson D, et al. Effect of the tyrosine kinase inhibitor STI571 in a patient with a metastatic gastrointestinal stromal rumor. N Engl J Med. 2001;344(14):1052-6.
12. Gold JS, Dematteo RP. Combined surgical and molecular therapy: the gastrointestinal stromal tumor model. Ann Surg. 2006;244(2): 176-84.
13. Siddiqui MA, Scott LJ. Imatinib: a review of its use in the management of gastrointestinal stromal tumours. Drugs. 2007;67(5):805-20.
14. Lasota J, Miettinen M. Clinical significance of oncogenic KIT and PDGFRA mutations in gastrointestinal stromal tumours. Histopathology. 2008;53:245-66.
15. Sleijfer S, Wiemer E, Verweij J. Drug Insight: gastrointestinal stromal tumors (GIST) - the solid tumor model for cancer-specific treatment. Nat Clin Pract Oncol. 2008;5(2):102-11.
16. Tran T, Davila JA, El-Serag HB. The epidemiology of malignant gastrointestinal stromal tumors: an analysis of 1,458 cases from 1992 to 2000. Am J Gastroenterol. 2005;100(1):162-8.
17. Nilsson B, Bumming P, Meis-Kindblom JM, Oden A, Dortok A, Gustavsson B, et al. Gastrointestinal stromal rumors: the incidence, prevalence, clinical course, and prognostication in the preimatinib mesylate era - a population-based study in western Sweden. Cancer. 2005;103(4):821-9.
18. Tryggvason G, Gislason HG, Magnusson MK, Jonasson JG. Gastrointestinal stromal tumors in Iceland, 1990-2003: the Icelandic GIST study, a population-based incidence and pathologic risk stratification study. Int J Cancer. 2005;117(2):289-93.
19. Rubio J, Marcos-Gragera R, Ortiz MR, Miro J, Vilardell L, Girones J, et al. Population-based incidence and survival of gastrointestinal stromal tumours (GIST) in Girona, Spain. Eur J Cancer. 2007;43(1): 144-8.
20. Goettsch WG, Bos SD, Breekveldt-Postma N, Casparie M, Herings
21. RM, Hogendoorn PC. Incidence of gastrointestinal stromal tumours is underestimated: results of a nation-wide study. Eur J Cancer. 2005;41(18):2868-72.
22. Mucciarini C, Rossi G, Bertolini F, Valli R, Cirilli C, Rashid I, et al. Incidence and clinicopathologic features of gastrointestinal stromal tumors. A population-based study. BMC Cancer. 2007;7:230.
23. Fletcher CD, Berman JJ, Corless C, Gorstein F, Lasota J, Longley BJ, et al. Diagnosis of gastrointestinal stromal tumors: A consensus approach. Hum Pathol. 2002;33(5):459-65.
24. Chetty R. Small and microscopically detected gastrointestinal stromal tumours: an overview. Pathology. 2008;40(1):9-12.
25. Agaimy A, Wunsch PH, Hofstaedter F, Blaszyk H, Rummele P, Gaumann A, et al. Minute gastric sclerosing stromal tumors (GIST tumorlets) are common in adults and frequently show c-KIT mutations. Am J Surg Pathol. 2007;31(1):113-20.
26. Kawanowa K, Sakuma Y, Sakurai S, Hishima T, Iwasaki Y, Saito K, et al. High incidence of microscopic gastrointestinal stromal tumors in the stomach. Hum Pathol. 2006;37(12):1527-35.
27. Agaimy A, Wunsch PH, Dirnhofer S, Bihl MP, Terracciano LM, Tornillo L. Microscopic gastrointestinal stromal tumors in esophageal and intestinal surgical resection specimens: a clinicopathologic, immunohistochemical, and molecular study of 19 lesions. Am J Surg Pathol. 2008;32(6):867-73.
28. Carney JA. The triad of gastric epithelioid leiomyosarcoma, pulmonary chondroma, and functioning extra-adrenal paraganglioma: a five-year review. Medicine (Baltimore). 1983;62(3):159-69.
29. Chompret A, Kannengiesser C, Barrois M, Terrier P, Dahan P, Tursz T, et al. PDGFRA germline mutation in a family with multiple cases of gastrointestinal stromal tumor. Gastroenterology. 2004;126(1): 318-21.
30. Nishida T, Hirota S, Taniguchi M, Hashimoto K, Isozaki K, Nakamura H, et al. Familial gastrointestinal stromal tumours with germline mutation of the KIT gene. Nat Genet. 1998;19(4):323-4.
31. Rubin BP, Heinrich MC, Corless CL. Gastrointestinal stromal tumour. Lancet. 2007;369(9574):1731-41.
32. Agaimy A, Wuensch PH. Gastrointestinal stromal tumours in patients with other-type cancer: a mere coincidence or an etiological association? A study of 97 GIST cases. Z Gastroenterol. 2005;43(9): 1025-30.
33. Emory TS, Sobin LH, Lukes L, Lee DH, O'Leary TJ. Prognosis of gastrointestinal smooth-muscle (stromal) tumors: dependence on anatomic site. Am J Surg Pathol. 1999;23(1):82-7.
34. Demetri GD, Benjamin RS, Blanke CD, Blay JY, Casali P, Choi H, et al. NCCN Task Force report: management of patients with gastrointestinal stromal tumor (GIST) - update of the NCCN clinical practice guidelines. J Natl Compr Cane Netw. 2007;5(Suppl 2):S1-29; quiz S30.
35. Blay JY, Le Cesne A, ESMO Guidelines Working Group. Gastrointestinal stromal tumors: ESMO Clinical Recommendations for diagnosis, treatment and follow-up. Ann Oncol. 2007;18(Suppl_2): ii27-9.
36. Casali PG, Jost L, Reichardt P, Schlemmer M, Blay JY. Gastrointestinal stromal tumors: ESMO clinical recommendations for diagnosis, treatment and follow-up. Ann Oncol. 2008;19(Suppl 2):ii35-8.
37. Montemurro M, Dirnhofer S, Borner M, Burkhard R, Demartines N, Furrer M, et al. Diagnosis and treatment of GIST in Switzerland. [Guidelines]. Schweiz Med Forum. 2008;8(30-31):544-9.
38. Wu JJ, Rothman TP, Gershon MD. Development of the interstitial cell of Cajal: origin, kit dependence and neuronal and nonneuronal sources of kit ligand. J Neurosci Res. 2000;59(3):384-401.
39. Huizinga JD, Thuneberg L, Kluppel M, Malysz J, Mikkelsen HB, Bernstein A. W/kit gene required for interstitial cells of Cajal and for intestinal pacemaker activity. Nature. 1995;373(6512):347-9.
40. Maeda H, Yamagata A, Nishikawa S, Yoshinaga K, Kobayashi S, Nishi K. Requirement of c-kit for development of intestinal pacemaker system. Development. 1992;116(2):369-75.
41. Miettinen M, Sarlomo-Rikala M, Lasota J. Gastrointestinal stromal tumors: recent advances in understanding of their biology. Hum Pathol. 1999;30(10):1213-20.
42. Went PT, Dirnhofer S, Bundi M, Mirlacher M, Schraml P, Mangialaio S, et al. Prevalence of KIT expression in human tumors. J Clin Oncol. 2004;22(22):4514-22.
43. Sircar K, Hewlett BR, Huizinga JD, Chorneyko K, Berezin I, Riddell RH. Interstitial cells of Cajal as precursors of gastrointestinal stromal tumors. Am J SurgPathol. 1999;23(4):377-89.
44. Miettinen M, Lasota J. Gastrointestinal stromal tumors: review on morphology, molecular pathology, prognosis, and differential diagnosis. Arch Pathol Lab Med. 2006;130(10):1466-78.
45. Hasegawa T, Matsuno Y, Shimoda T, Hirohashi S. Gastrointestinal stromal tumor: consistent CD 117 immunostaining for diagnosis, and prognostic classification based on tumor size and MIB-1 grade. Hum Pathol. 2002;33(6):669-76.
46. Miettinen M, Virolainen M, Maarit Sarlomo R. Gastrointestinal stromal tumors - value of CD34 antigen in their identification and separation from true leiomyomas and schwannomas. Am J Surg Pathol. 1995;19(2):207-16.
47. West RB, Corless CL, Chen X, Rubin BP, Subramanian S, Montgomery K, et al. The novel marker, DOG1, is expressed ubiquitously in gastrointestinal stromal tumors irrespective of KIT or PDGFRA mutation status. Am J Pathol. 2004;165(1):107-13.
48. Espinosa I, Lee CH, Kim MK, Rouse BT, Subramanian S, Montgomery K, et al. A novel monoclonal antibody against DOG1 is a sensitive and specific marker for gastrointestinal stromal tumors. Am J Surg Pathol. 2008;32(2):210-8.
49. Blay P, Astudillo A, Buesa JM, Campo E, Abad M, Garcia-Garcia J, et al. Protein kinase C theta is highly expressed in gastrointestinal stromal tumors but not in other mesenchynial neoplasias. Clin Cancer Res. 2004;10(12 Pt 1):4089-95.
50. Debiec-Rychter M, Wasag B, Stul M, De Wever I, Van Oosterom A, Hagemeijer A, et al. Gastrointestinal stromal tumours (GISTs) negative for KIT (CD117 antigen) immunoreactivity. J Pathol. 2004;202(4):430-8.
51. Duensing A, Joseph NE, Medeiros F, Smith F, Hornick JL, Heinrich MC, et al. Protein Kinase C theta (PKCtheta) expression and constitutive activation in gastrointestinal stromal tumors (GISTs). Cancer Res. 2004;64(15):5127-31.
52. Motegi A, Sakurai S, Nakayama H, Sano T, Oyama T, Nakajima T PKC theta, a novel immunohistochemical marker for gastrointestinal stromal tumors (GIST), especially useful for identifying KIT-negative tumors. Pathol Int. 2005;55(3):106-12.
53. Zhu MJ, Ou WB, Fletcher CD, Cohen PS, Demetri GD, Fletcher JA. KIT oncoprotein interactions in gastrointestinal stromal tumors: therapeutic relevance. Oncogene. 2007;26(44):6386-95.
54. Pauls K, Merkelbach-Bruse S, Thal D, Buttner R, Wardelmann E. PDGFRalpha- and c-kit-mutated gastrointestinal stromal tumours (GISTs) are characterized by distinctive histological and immunohistochemical features. Histopathology. 2005;46(2):166-75.
55. Perona R. Cell signalling: growth factors and tyrosine kinase receptors. Clin Transl Oncol. 2006;8(2):77-82.
56. Vliagoftis H, Worobec AS, Metcalfe DD. The protooncogene c-kit and c-kit ligand in human disease. J Allergy Clin Immunol. 1997; 100(4):435-40.
57. Miettinen M, Lasota J. KIT (CD117): a review on expression in normal and neoplastic tissues, and mutations and their clinicopathologic correlation. Appl Immunohistochem Mol Morphol. 2005;13(3): 205-20.
58. Hornick JL, Fletcher CD. Immunohistochemical staining for KIT (CD 117) in soft tissue sarcomas is very limited in distribution. Am J Clin Pathol. 2002;117(2):188-93.
59. Miettinen M, Makhlouf H, Sobin LH, Lasota J. Gastrointestinal stromal tumors of the jejunum and ileum: a clinicopathologic, immunohistochemical, and molecular genetic study of 906 cases before imatinib with long-term follow-up. Am J Surg Pathol. 2006;30(4): 477-89.
60. Miettinen M, Sobin LH, Lasota J. Gastrointestinal stromal tumors of the stomach: a clinicopathologic, immunohistochemical, and molecular genetic study of 1765 cases with long-term follow-up. Am J Surg Pathol. 2005;29(1):52-68.
61. Lasota J, Wozniak A, Sarlomo-Rikala M, Rys J, Kordek R, Nassar A, et al. Mutations in exons 9 and 13 of KIT gene are rare events in gastrointestinal stromal tumors. A study of 200 cases. Am J Pathol. 2000;157(4):1091-5.
62. Lux ML, Rubin BP, Biase TL, Chen CJ, Maclure T, Demetri G, et al. KIT extracellular and kinase domain mutations in gastrointestinal stromal tumors. Am J Pathol. 2000;156(3):791-5.
63. Rubin BP, Singer S, Tsao C, Duensing A, Lux ML, Ruiz R, et al. KIT activation is a ubiquitous feature of gastrointestinal stromal tumors. Cancer Res. 2001;61(22):8118-21.
64. Hartmann K, Wardelmann E, Ma Y, Merkelbach-Bruse S, Preussner LM, Woolery C, et al. Novel germline mutation of KTT associated with familial gastrointestinal stromal tumors and mastocytosis. Gastroenterology. 2005;129(3):1042-6.
65. Heinrich MC, Corless CL, Demetri GD, Blanke CD, von Mehren M, Joensuu H, et al. Kinase mutations and imatinib response in patients with metastatic gastrointestinal stromal tumor. J Clin Oncol. 2003;21(23):4342-9.
66. Heinrich MC, Rubin BP, Longley BJ, Fletcher JA. Biology and genetic aspects of gastrointestinal stromal tumors: KIT activation and cytogenetic alterations. Hum Pathol. 2002;33(5):484-95.
67. Corless CL, Fletcher JA, Heinrich MC. Biology of gastrointestinal stromal tumors. J Clin Oncol. 2004;22(18):3813-25.
68. Duensing A, Medeiros F, McConarty B, Joseph NE, Panigrahy D, Singer S, et al. Mechanisms of oncogenic KTT signal transduction in primary gastrointestinal stromal tumors (GISTs). Oncogene. 2004; 23(22):3999-4006.
69. Heinrich MC, Corless CL, Duensing A, McGreevey L, Chen CJ, Joseph N, et al. PDGFRA activating mutations in gastrointestinal stromal tumors. Science. 2003;299(5607):708-10.
70. Corless CL, Schroeder A, Griffith D, Town A, McGreevey L, Harrell P, et al. PDGFRA mutations in gastrointestinal stromal tumors: frequency, spectrum and in vitro sensitivity to imatinib. J Clin Oncol. 2005;23(23):5357-64.
71. Medeiros F, Corless CL, Duensing A, Hornick JL, Oliveira AM, Heinrich MC, et al. KIT-negative gastrointestinal stromal tumors: proof of concept and therapeutic implications. Am J Surg Pathol. 2004;28(7):889-94.
72. Lasota J, Stachura J, Miettinen M. GISTs with PDGFRA exon 14 mutations represent subset of clinically favorable gastric tumors with epithelioid morphology. Lab Invest. 2006;86(1):94-100.
73. Miettinen M, Lasota J. Gastrointestinal stromal tumors: pathology and prognosis at different sites. Semin Diagn Pathol. 2006;23(2): 70-83.
74. Rutkowski P, Nowecki ZI, Michej W, Debiec-Rychter M, Wozniak A, Limon J, et al. Risk criteria and prognostic factors for predicting recurrences after resection of primary gastrointestinal stromal tumor. Ann Surg Oncol. 2007;14(7):2018-27.
75. Van Glabbeke MM, Owzar K, Rankin C, Simes J, Crowley J, Gist Meta-analysis Group (MetaGIST). Comparison of two doses of imatinib for the treatment of unresectable or metastatic gastrointestinal stromal tumors (GIST): A meta-analyis based on 1,640 patients (pts). J Clin Oncol. 2007;25(18S June 20 Supplement): 10004.
76. Huang HY, Li CF, Huang WW, Hu TH, Lin CN, Uen YH, et al. A modification of NIH consensus criteria to better distinguish the highly lethal subset of primary localized gastrointestinal stromal tumors: a subdivision of the original high-risk group on the basis of outcome. Surgery. 2007;141(6):748-56.
77. Takahashi T, Nakajima K, Nishitani A, Souma Y, Hirota S, Sawa Y, et al. An enhanced risk-group stratification system for more practical prognostication of clinically malignant gastrointestinal stromal tumors. Int J Clin Oncol. 2007;12(5):369-74.
78. Hassan I, You YN, Shyyan R, Dozois EJ, Smyrk TC, Okuno SH, et al. Surgically managed gastrointestinal stromal tumors: a comparative and prognostic analysis. Ann Surg Oncol. 2008;15(1):52-9.
79. Blay JY, Bonvalot S, Casali P, Choi H, Debiec-Richter M, Dei Tos AP, et al. Consensus meeting for the management of gastrointestinal stromal tumors. Report of the GIST Consensus Conference of 20-21 March 2004, under the auspices of ESMO. Ann Oncol. 2005;16(4): 566-78.
80. Raut CP, Ashley SW. How I Do It: Surgical Management of Gastrointestinal Stromal Tumors. J Gastrointest Surg. 2008.
81. Novitsky YW, Kercher KW, Sing RF, Heniford BT. Long-term outcomes of laparoscopic resection of gastric gastrointestinal stromal tumors. Ann Surg. 2006;243(6):738-45; discussion 45-7.
82. Nguyen SQ, Divino CM, Wang JL, Dikman SH. Laparoscopic management of gastrointestinal stromal tumors. Surg Endosc. 2006;20(5):713-6.
83. Sexton JA, Pierce RA, Halpin VJ, Eagon JC, Hawkins WG, Linehan DC, et al. Laparoscopic gastric resection for gastrointestinal stromal tumors. Surg Endosc. 2008.
84. Blackstein ME, Blay JY, Corless C, Driman DK, Riddell R, Soulieres D, et al. Gastrointestinal stromal tumours: consensus statement on diagnosis and treatment. Can J Gastroenterol. 2006;20(3):157-63.
85. von Mehren M, Watson JC. Gastrointestinal stromal tumors. Hematol Oncol Clin North Am. 2005;19(3):547-64, vii.
86. Singer S, Rubin BP, Lux ML, Chen CJ, Demetri GD, Fletcher CD, et al. Prognostic value of KIT mutation type, mitotic activity, and histologic subtype in gastrointestinal stromal tumors. J Clin Oncol. 2002;20(18):3898-905.
87. Langer C, Gunawan B, Schuler P, Huber W, Fuzesi L, Becker H. Prognostic factors influencing surgical management and outcome of gastrointestinal stromal tumours. Br J Surg. 2003;90(3):332-9.
88. DeMatteo RP, Owzar K, Antonescu CR, Maki R, Demetri GD, McCarter M, et al. Efficacy of adjuvant imatinib mesylate following complete resection of localized, primary gastrointestinal stromal tumor (GIST) at high risk of recurrence: The U.S. Intergroup phase II trial ACOSOG Z9000. [2008 Gastrointestinal Cancers Symposium Conference Abstract] Orlando, FL, USA: ASCO; 2008 [updated 2008; cited 2008 Mar 01]; 8. Available from: http://www.asco.org/ ASCO/Abstracts+%26+Virtual+Meeting/Abstracts?&vm view= abst_detail_view&confID=53&abstractID=10450.
89. Dematteo RP. Adjuvant imatinib mesylate increases recurrence free survival (RFS) in patients with completely resected localized primary gastrointestinal stromal tumor (GIST): North American Intergroup Phase III trial ACOSOG Z9001. [Conference Abstract]: ASCO; 2007 [updated 2007; cited 2008 Mar 01]; 10079. Available from: http://www.asco.org/ASCO/ Abstracts+%26+Virtual+Meeting/Abstracts?&vmview=abst_detail_view&confID= 47 &abstractID=100001.
90. Demetri GD, van Oosterom AT, Garrett CR, Blackstein ME, Shah MH, Verweij J, 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):1329-38.
91. Demetri GD, Wang Y, Wehrle E, Blanke C, Joensuu H, von Mehren M. Correlation of imatinib plasma levels with clinical benefit in patients (Pts) with unresectable/metastatic gastrointestinal stromal tumors (GIST). [Conference Abstract] Orlando, FL, USA: ASCO; 2008 [updated 2008; cited 2008 Mar 01]; 3. Available from: http://www.asco.org/ASCO/Abstracts+%26+Virtual+Meeting/ Abstracts?&vmview=abst_detail_view&confID=53&abstractID= 10635.
92. Blanke CD, Demetri GD, von Mehren M, Heinrich MC, Eisenberg B, Fletcher JA, 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):620-5.
93. Verweij J, Casali PG, Zalcberg J, LeCesne A, Reichardt P, Blay JY, et al. Progression-free survival in gastrointestinal stromal tumours with high-dose imatinib: randomised trial. Lancet. 2004;364 (9440):1127-34.
94. Blanke CD, Rankin C, Demetri GD, Ryan CW, von Mehren M, Benjamin RS, 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):626-32.
95. Debiec-Rychter M, Sciot R, Le Cesne A, Schlemmer M, Hohenberger P, van Oosterom AT, et al. KIT mutations and dose selection for imatinib in patients with advanced gastrointestinal stromal tumours. Eur J Cancer. 2006;42(8):1093-103.
96. Blay JY, Le Cesne A, Ray-Coquard I, Bui B, Duffaud F, Delbaldo C, 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): 1107-13.
97. Le Cesne A, Ray-Coquard I, Bui B, Rios M, Adenis A, Bertucci F, et al. Continuous versus interruption of imatinib (IM) in responding patients with advanced GIST after three years of treatment: A prospective randomized phase III trial of the French Sarcoma Group. Proceedings of the American Society of Clinical Oncology. 2007;25(18S(June 20 Supplement)):10005.
98. Richter KK, Schmid C, Thompson-Fawcett M, Settmacher U, Altendorf-Hofmann A. Long-term follow-up in 54 surgically treated patients with gastrointestinal stromal tumours. Langenbecks Arch Surg. 2008.
99. Debiec-Rychter M, Cools J, Dumez H, Sciot R, Stul M, Mentens N, et al. Mechanisms of resistance to imatinib mesylate in gastrointestinal stromal tumors and activity of the PKC412 inhibitor against imatinib-resistant mutants. Gastroenterology. 2005;128(2): 270-9.
100. Wardelmann E, Merkelbach-Bruse S, Pauls K, Thomas N, Schildhaus HU, Heinicke T, et al. Polyclonal evolution of multiple secondary KIT mutations in gastrointestinal stromal tumors under treatment with imatinib mesylate. Clin Cancer Res. 2006; 12(6):1743-9.
101. Branford S, Rudzki Z, Walsh S, Parkinson I, Grigg A, Szer J, et al. Detection of BCR-ABL mutations in patients with CML treated with imatinib is virtually always accompanied by clinical resistance, and mutations in the ATP phosphate-binding loop (P-loop) are associated with a poor prognosis. Blood. 2003;102(1):276-83.
102. Nishida T, Kanda T, Nishitani A, Takahashi T, Nakajima K, Ishikawa T, et al. Secondary mutations in the kinase domain of the KIT gene are predominant in imatinib-resistant gastrointestinal stromal tumor. Cancer Sci. 2008;99(4):799-804.
103. Gronchi A, Fiore M, Miselli F, Lagonigro MS, Coco P, Messina A, et al. Surgery of residual disease following molecular-targeted therapy with imatinib mesylate in advanced/metastatic GIST. Ann Surg. 2007;245(3):341-6.
104. Hasegawa J, Kanda T, Hirota S, Fukuda M, Nishitani A, Takahashi T, et al. Surgical interventions for focal progression of advanced gastrointestinal stromal tumors during imatinib therapy. Int J Clin Oncol. 2007;12(3):212-7.
105. Al-Batran SE, Hartmann JT, Heidel F, Stoehlmacher J, Wardelmann E, Dechow C, 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):145-52.
106. Tuma RS. Disease progression in some cancers may be due to low blood levels of targeted therapies. J Natl Cancer Inst. 2008;100 (13):912-3.
107. George S, Blay JY, Casali PG, Le Cesne A, Morgan JA, Pokela J, et al. Continuous daily dosing (CDD) of sunitinib malate (SU) compares favorably with intermittent dosing in pts with advanced GISTJ Clin Oncol. 2007;25(18S June 20 Supplement):10015.
108. Abhyankar SA, Nair N. Highlighting the role of FDG PET scan in early response assessment of gastrointestinal stromal tumor treated with imatinib mesylate. Clin Nucl Med. 2008;33(3):213-4.
109. Kelloff GJ, Hoffman JM, Johnson B, Scher HI, Siegel BA, Cheng EY, et al. Progress and promise of FDG-PET imaging for cancer patient management and oncologic drug development. Clin Cancer Res. 2005;11(8):2785-808.
110. Prior JO, Montemurro M, Orcurto M-V, Michielin O, Luthi F, Benhattar J, et al. Early Prediction of Response to Sunitinib Therapy by F-18-FDG PET in Patients With Gastrointestinal Stromal Tumor (GIST) After Imatinib Failure. J Clin Oncol. 2008;in press.
111. Boss DS, Olmos RV, Sinaasappel M, Beijnen JH, Schellens JH. Application of PET/CT in the development of novel anticancer drugs. Oncologist. 2008;13(1):25-38.
112. Therasse P, Arbuck SG, Eisenhauer EA, Wanders J, Kaplan RS, Rubinstein L, et al. New guidelines to evaluate the response to treatment in solid tumors. European Organization for Research and Treatment of Cancer, National Cancer Institute of the United States, National Cancer Institute of Canada. J Natl Cancer Inst. 2000;92(3):205-16.
113. Green S, Weiss GR. Southwest Oncology Group standard response criteria, endpoint definitions and toxicity criteria. Invest New Drugs. 1992;10(4):239-53.
114. Choi H, Charnsangavej C, Faria SC, Macapinlac HA, Burgess MA, Patel SR, et al. Correlation of computed tomography and positron emission tomography in patients with metastatic gastrointestinal stromal tumor treated at a single institution with imatinib mesylate: proposal of new computed tomography response criteria. J Clin Oncol. 2007;25(13):1753-9.
115. Antoch G, Kanja J, Bauer S, Kuehl H, Renzing-Koehler K, Schuette J, et al. Comparison of PET, CT, and dual-modality PET/CT imaging for monitoring of imatinib (STI571) therapy in patients with gastrointestinal stromal rumors. J Nucl Med. 2004; 45(3):357-65.
116. Benjamin RS, Choi H, Macapinlac HA, Burgess MA, Patel SR, Chen LL, et al. We should desist using RECIST, at least in GISTJ Clin Oncol. 2007;25(13):1760-4.
117. von Mehren M. Beyond imatinib: second generation c-KIT inhibitors for the management of gastrointestinal stromal tumors. Clin Colorectal Cancer. 2006;6 Suppl 1:S30-4.
118. Verweij J, de Jonge M. Multitarget tyrosine kinase inhibition: [and the winner is...]. J Clin Oncol. 2007;25(17):2340-2.
119. Weisberg E, Wright RD, Jiang J, Ray A, Moreno D, Manley PW, et al. Effects of PKC412, nilotinib, and imatinib against GIST-associated PDGFRA mutants with differential imatinib sensitivity. Gastroenterology. 2006;131(6):1734-42.
120. Schittenhelm MM, Shiraga S, Schroeder A, Corbin AS, Griffith D, Lee FY, et al. Dasatinib (BMS-354825), a dual SRC/ABL kinase inhibitor, inhibits the kinase activity of wild-type, juxtamembrane, and activation loop mutant KIT isoforms associated with human malignancies. Cancer Res. 2006;66(1):473-81.
121. Gridelli C, Maione P, Del Gaizo F, Colantuoni G, Guerriero C, Ferrara C, et al. Sorafenib and sunitinib in the treatment of advanced non-small cell lung cancer. Oncologist. 2007;12(2):191-200.
122. Bui BN, Blay J, Duffaud F, Hermine O, Hermine O, Le Cesne A. Preliminary efficacy and safety results of Masitinib administered, front line in patients with advanced GIST. A phase II study. J Clin Oncol. 2007;25(18S Part I, June 20 Supplement): Abstr. #10025.
123. Joensuu H, De Braud F, Coco P, De Pas T, Putzu C, Spreafico C, et al. Phase II, open-label study of PTK787/ZK222584 for the treatment of metastatic gastrointestinal stromal tumors resistant to imatinib mesylate. Ann Oncol. 2008;19(1):173-7.
124. Drevs J, Siegert P, Medinger M, Mross K, Strecker R, Zirrgiebel U, et al. Phase I clinical study of AZD2171, an oral vascular endothelial growth factor signaling inhibitor, in patients with advanced solid tumors. J Clin Oncol. 2007;25(21):3045-54.
125. Yamada Y, Sawaki A, Nishida T, Komatsu Y, Kanda T, Doi T, et al. Phase II study of motesanib diphosphate (AMG 706} in Japanese patients (pts) with advanced gastrointestinal stromal tumors (GISTs) who developed progressive disease or relapsed while on imatinib mesylate. [Conference Abstract] Orlando, FL, USA: ASCO; 2008 [updated 2008; cited 2008 Mar 01]; 3]. Available from: http://www.asco.org/ASCO/Abstracts+%26+Virtual+ Meeting/AbstractsP&vmview= abst_detail_view&confID=53&abstractID=10104.
126. Rosen LS, Kurzrock R, Mulay M, Van Vugt A, Purdom M, Ng C, et al. Safety, pharmacokinetics, and efficacy of AMG 706, an oral multikinase inhibitor, in patients with advanced solid tumors. J Clin Oncol. 2007;25(17):2369-76.
127. Di Cosimo S, Matar P, Rojo F, Guzman M, Rodriguez S, Jimenez J, et al. Schedule-dependent effects of the epidermal growth factor receptor (EGFR) tyrosine kinase inhibitor gefitinib in combination with the mammalian target of rapamycin (mTOR) inhibitor everolimus (RAD001). J Clin Oncol. 2004;22(14S Part I, July 15 Supplement):Abstr. #3074.
128. Demetri GD, George S, Morgan JA, Wagner A, Quigley MT, Polson K, et al. Inhibition of the Heat Shock Protein 90 (Hsp90) chaperone with the novel agent IPI-504 to overcome resistance to tyrosine kinase inhibitors (TKIs) in metastatic GIST: Updated results of a phase I trial. J Clin Oncol. 2007;25(18S June 20 Supplement):Abstr. #10024.
129. Huse DM, von Mehren M, Lenhart G, Joensuu H, Blanke C, Feng W, et al. Cost effectiveness of imatinib mesylate in the treatment of advanced gastrointestinal stromal tumours. Clin Drug Investig. 2007;27(2):85-93.
130. Wilson J, Connock M, Song F, Yao G, Fry-Smith A, Raftery J, et al. Imatinib for the treatment of patients with unresectable and/or metastatic gastrointestinal stromal tumours: systematic review and economic evaluation. Health Technol Assess. 2005;9(25): 1-142.
131. Braithwaite RS, Meltzer DO, King JT Jr. Leslie D, Roberts MS. What Does the Value of Modern Medicine Say About the $ 50,000 per Quality-Adjusted Life-Year Decision Rule? Med Care. 2008;46(4):349-56.
132. Brouwer W, Van Exel J, Van Baal P, Polder J. Economics and public health: engaged to be happily married! Eur J Public Health. 2007;17(2):122-3.
133. Mabasa V, Taylor SC, Chu CC, Moravan V, Peacock S, Knowling M. Verification of imatinib cost-effectiveness in advanced gastrointestinal stromal tumor in British Columbia (VINCE). J Clin Oncol. 2007;25(18S Part I, June 20 Supplement):Abstr.#10049.