Tyrosine kinase inhibitors in the treatment of systemic mastocytosis

Leukemia Research

Volumn 35, Issue 9, 2011, Pages 1143-1152

  Ustun, Celalettin ^a   DeRemer, David L ^b   Akin, Cem ^c  

^a UNIVERSITY OF MINNESOTA MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF GEORGIA   (United States)

^c BRIGHAM AND WOMEN S HOSPITAL   (United States)

Author keywords

Dasatinib; Imatinib; Masitinib; Midostaurin (PKC412); Nilotinib; Systemic mastocytosis; Tyrosine kinase inhibitors

Indexed keywords

CLADRIBINE; CYTARABINE; DASATINIB; EXEL 0862; IMATINIB; INTERFERON; MASITINIB; MIDOSTAURIN; NILOTINIB; OBATOCLAX; PROTEIN TYROSINE KINASE INHIBITOR; SEMAXANIB; STEM CELL FACTOR; STEM CELL FACTOR RECEPTOR; TANDUTINIB; UNCLASSIFIED DRUG;

ANEMIA; BONE MARROW SUPPRESSION; BONE PAIN; CHRONIC MYELOID LEUKEMIA; CONGESTIVE HEART FAILURE; DIARRHEA; DOSE RESPONSE; DRUG ERUPTION; DRUG INDUCED HEADACHE; DRUG MECHANISM; DRUG MEGADOSE; DRUG POTENTIATION; DRUG TARGETING; DYSPNEA; EDEMA; FATIGUE; FEVER; FLUID RETENTION; GENE; GENE MUTATION; HUMAN; HYPOGLYCEMIA; HYPOPHOSPHATEMIA; KIT GENE; LOW DRUG DOSE; MAST CELL; MASTOCYTOMA; MASTOCYTOSIS; NAUSEA; NEUTROPENIA; NONHUMAN; PAIN; PANCREATITIS; PLEURA EFFUSION; PRIORITY JOURNAL; PROTEIN FUNCTION; QT PROLONGATION; REVIEW; SIDE EFFECT; SIGNAL TRANSDUCTION; SUDDEN DEATH; SYSTEMIC MASTOCYTOSIS; SYSTEMIC MASTOCYTOSIS WITH ASSOCIATED CLONAL HEMATOLOGICAL NON MAST CELL LINEAGE DISEASE; THROMBOCYTOPENIA; URTICARIA PIGMENTOSA; VOMITING;

ANIMALS; HUMANS; MASTOCYTOSIS, SYSTEMIC; MUTATION; PIPERAZINES; PROTEIN KINASE INHIBITORS; PROTO-ONCOGENE PROTEINS C-KIT; PYRIMIDINES; STAUROSPORINE; THIAZOLES;

EID: 80051475544     PISSN: 01452126     EISSN: 18735835     Source Type: Journal    
DOI: 10.1016/j.leukres.2011.05.006     Document Type: Review

References (153)

1. Valent P., Horny H.P., Escribano L., Longley B.J., Li C.Y., Schwartz L.B., et al. Diagnostic criteria and classification of mastocytosis: a consensus proposal. Leuk Res 2001, 25:603-625.
2. Horny H.P., Akin C., Metcalfe D.D., Escribano L., Bennett J.M., Valent P., et al. Mastocytosis (Mast cell disease). World Health Organization (WHO) Classification of tumours. Pathology & genetics. Tumours of haematopoietic and lymphoid tissues 2008, 54-63. IARC Press, Lyon. S.H. Swerdlow, E. Campo, N.L. Harris, E.S. Jaffe, S.A. Pileri, H. Stein, J. Thiele, J.W. Vardiman (Eds.).
3. Lim K.H., Tefferi A., Lasho T.L., Finke C., Patnaik M., Butterfield J.H., et al. Systemic mastocytosis in 342 consecutive adults: survival studies and prognostic factors. Blood 2009, 113:5727-5736.
4. Escribano L., Alvarez-Twose I., Sanchez-Munoz L., Garcia-Montero A., Nunez R., Almeida J., et al. Prognosis in adult indolent systemic mastocytosis: a long-term study of the Spanish Network on Mastocytosis in a series of 145 patients. J Allergy Clin Immunol 2009, 124:514-521.
5. Valent P., Sperr W.R., Akin C. How I treat patients with advanced systemic mastocytosis. Blood 2010, 6, Robyn J, Metcalfe DD. Systemic mastocytosis. Adv Immunol 2006, 89:169-243.
6. Robyn J., Metcalfe D.D. Systemic mastocytosis. Adv Immunol 2006, 89:169-243.
7. Hennessy B., Giles F., Cortes J., O'Brien S., Ferrajoli A., Ossa G., et al. Management of patients with systemic mastocytosis: review of M.D. Anderson Cancer Center experience. Am J Hematol 2004, 77:209-214.
8. Bains S.N., Hsieh F.H. Current approaches to the diagnosis and treatment of systemic mastocytosis. Ann Allergy Asthma Immunol 2010, 104:1-10.
9. Bunimovich O., Grassi M., Baer M.R. Systemic mastocytosis: classification, pathogenesis, diagnosis, and treatment. Cutis 2009, 83:29-36.
10. Pardanani A., Tefferi A. Systemic mastocytosis in adults: a review on prognosis and treatment based on 342 Mayo Clinic patients and current literature. Curr Opin Hematol 2010, 17:125-132.
11. Horny H.P. Mastocytosis: an unusual clonal disorder of bone marrow-derived hematopoietic progenitor cells. Am J Clin Pathol 2009, 132:438-447.
12. Valent P. Systemic mastocytosis. Cancer Treat Res 2008, 142:399-419.
13. Horny H.P., Sotlar K., Valent P. Mastocytosis: state of the art. Pathobiology 2007, 74:121-132.
14. Pardanani A., Akin C., Valent P. Pathogenesis, clinical features, and treatment advances in mastocytosis. Best Pract Res Clin Haematol 2006, 19:595-615.
15. Valent P., Akin C., Sperr W.R., Mayerhofer M., Fodinger M., Fritsche-Polanz R., et al. Mastocytosis: pathology, genetics, and current options for therapy. Leuk Lymphoma 2005, 46:35-48.
16. Akin C. Clonality and molecular pathogenesis of mastocytosis. Acta Haematol 2005, 114:61-69.
17. Kitamura Y., Hirotab S. Kit as a human oncogenic tyrosine kinase. Cell Mol Life Sci 2004, 61:2924-2931.
18. Boissan M., Feger F., Guillosson J.J., Arock M. c-Kit and c-kit mutations in mastocytosis and other hematological diseases. J Leukoc Biol 2000, 67:135-148.
19. Ronnstrand L. Signal transduction via the stem cell factor receptor/c-kit. Cell Mol Life Sci 2004, 61:2535-2548.
20. Fukuda T., Kamishima T., Tsuura Y., Suzuki T., Kakihara T., Naito M., et al. Expression of the c-kit gene product in normal and neoplastic mast cells but not in neoplastic basophil/mast cell precursors from chronic myelogenous leukaemia. J Pathol 1995, 177:139-146.
21. 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, 13:205-220.
22. Dym M., Jia M.C., Dirami G., Price J.M., Rabin S.J., Mocchetti I., et al. Expression of c-kit receptor and its autophosphorylation in immature rat type A spermatogonia. Biol Reprod 1995, 52:8-19.
23. Blume-Jensen P., Janknecht R., Hunter T. The kit receptor promotes cell survival via activation of PI 3-kinase and subsequent Akt-mediated phosphorylation of Bad on Ser136. Curr Biol 1998, 8:779-782.
24. Pawson T. Specificity in signal transduction: from phosphotyrosine-SH2 domain interactions to complex cellular systems. Cell 2004, 116:191-203.
25. Mol C.D., Lim K.B., Sridhar V., Zou H., Chien E.Y., Sang B.C., et al. Structure of a c-kit product complex reveals the basis for kinase transactivation. J Biol Chem 2003, 278:31461-31464.
26. Masson K., Ronnstrand L. Oncogenic signaling from the hematopoietic growth factor receptors c-kit and Flt3. Cell Signal 2009, 21:1717-1726.
27. Yarden Y., Kuang W.J., Yang-Feng T., Coussens L., Munemitsu S., Dull T.J., et al. Human proto-oncogene c-kit: a new cell surface receptor tyrosine kinase for an unidentified ligand. EMBO J 1987, 6:3341-3351.
28. Qiu F.H., Ray P., Brown K., Barker P.E., Jhanwar S., Ruddle F.H., et al. Primary structure of c-kit: relationship with the CSF-1/PDGF receptor kinase family-oncogenic activation of v-kit involves deletion of extracellular domain and C terminus. EMBO J 1988, 7:1003-1011.
29. Zakut R., Perlis R., Eliyahu S., Yarden Y., Givol D., Lyman S.D., et al. KIT ligand (mast cell growth factor) inhibits the growth of KIT-expressing melanoma cells. Oncogene 1993, 8:2221-2229.
30. Lemmon M.A., Pinchasi D., Zhou M., Lax I., Schlessinger J. Kit receptor dimerization is driven by bivalent binding of stem cell factor. J Biol Chem 1997, 272:6311-6317.
31. Linnekin D. Early signaling pathways activated by c-Kit in hematopoietic cells. Int J Biochem Cell Biol 1999, 31:1053-1074.
32. Karin M. Signal transduction from cell surface to nucleus in development and disease. FASEB J 1992, 6:2581-2590.
33. Irani A.M., Nilsson G., Miettinen U., Craig S.S., Ashman L.K., Ishizaka T., et al. Recombinant human stem cell factor stimulates differentiation of mast cells from dispersed human fetal liver cells. Blood 1992, 80:3009-3021.
34. Valent P., Spanblochl E., Sperr W.R., Sillaber C., Zsebo K.M., Agis H., et al. Induction of differentiation of human mast cells from bone marrow and peripheral blood mononuclear cells by recombinant human stem cell factor/kit-ligand in long-term culture. Blood 1992, 80:2237-2245.
35. Kirshenbaum A.S., Goff J.P., Kessler S.W., Mican J.M., Zsebo K.M., Metcalfe D.D. Effect of IL-3 and stem cell factor on the appearance of human basophils and mast cells from CD34+ pluripotent progenitor cells. J Immunol 1992, 148:772-777.
36. Mitsui H., Furitsu T., Dvorak A.M., Irani A.M., Schwartz L.B., Inagaki N., et al. Development of human mast cells from umbilical cord blood cells by recombinant human and murine c-kit ligand. Proc Natl Acad Sci USA 1993, 90:735-739.
37. Shelburne C.P., McCoy M.E., Piekorz R., et al. Stat5 expression is critical for mast cell development and survival. Blood 2003, 102:1290-1297.
38. Iemura A., Tsai M., Ando A., Wershil B.K., Galli S.J. The c-kit ligand, stem cell factor, promotes mast cell survival by suppressing apoptosis. Am J Pathol 1994, 144:321-328.
39. Lohning M., Stroehmann A., Coyle A.J., Grogan J.L., Lin S., Gutierrez-Ramos J.C., et al. T1/ST2 is preferentially expressed on murine Th2 cells, independent of interleukin 4, interleukin 5, and interleukin 10, and important for Th2 effector function. Proc Natl Acad Sci USA 1998, 95:6930-6935.
40. Hayakawa H., Hayakawa M., Kume A., Tominaga S. Soluble ST2 blocks interleukin-33 signaling in allergic airway inflammation. J Biol Chem 2007, 282:26369-26380.
41. Kuehn H.S., Gilfillan A.M. G protein-coupled receptors and the modification of FcepsilonRI-mediated mast cell activation. Immunol Lett 2007, 113:59-69.
42. Drube S., Heink S., Walter S., Lohn T., Grusser M., Gerbaulet A., et al. The receptor tyrosine kinase c-Kit controls IL-33 receptor signaling in mast cells. Blood 2010, 115:3899-3906.
43. 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, 279:577-580.
44. Naeem M., Dahiya M., Clark J.I., Creech S.D., Alkan S. Analysis of c-kit protein expression in small-cell lung carcinoma and its implication for prognosis. Hum Pathol 2002, 33:1182-1187.
45. Bellone G., Silvestri S., Artusio E., Tibaudi D., Turletti A., Geuna M., et al. Growth stimulation of colorectal carcinoma cells via the c-kit receptor is inhibited by TGF-beta 1. J Cell Physiol 1997, 172:1-11.
46. Nakata Y., Kimura A., Katoh O., Kawaishi K., Hyodo H., Abe K., et al. c-kit point mutation of extracellular domain in patients with myeloproliferative disorders. Br J Haematol 1995, 91:661-663.
47. Cohen P.S., Chan J.P., Lipkunskaya M., Biedler J.L., Seeger R.C. Expression of stem cell factor and c-kit in human neuroblastoma, The Children's Cancer Group. Blood 1994, 84:3465-3472.
48. Natali P.G., Nicotra M.R., Sures I., Mottolese M., Botti C., Ullrich A. Breast cancer is associated with loss of the c-kit oncogene product. Int J Cancer 1992, 52:713-717.
49. Patnaik M.M., Tefferi A., Pardanani A. Kit: molecule of interest for the diagnosis and treatment of mastocytosis and other neoplastic disorders. Curr Cancer Drug Targets 2007, 7:492-503.
50. Piao X., Bernstein A. A point mutation in the catalytic domain of c-kit induces growth factor independence, tumorigenicity, and differentiation of mast cells. Blood 1996, 87:3117-3123.
51. Tsujimura T., Furitsu T., Morimoto M., Isozaki K., Nomura S., Matsuzawa Y., et al. Ligand-independent activation of c-kit receptor tyrosine kinase in a murine mastocytoma cell line P-815 generated by a point mutation. Blood 1994, 83:2619-2626.
52. Furitsu T., Tsujimura T., Tono T., Ikeda H., Kitayama H., Koshimizu U., et al. Identification of mutations in the coding sequence of the proto-oncogene c-kit in a human mast cell leukemia cell line causing ligand-independent activation of c-kit product. J Clin Invest 1993, 92:1736-1744.
53. Kitayama H., Kanakura Y., Furitsu T., Tsujimura T., Oritani K., Ikeda H., et al. Constitutively activating mutations of c-kit receptor tyrosine kinase confer factor-independent growth and tumorigenicity of factor-dependent hematopoietic cell lines. Blood 1995, 85:790-798.
54. Tsujimura T., Furitsu T., Morimoto M., Kanayama Y., Nomura S., Matsuzawa Y., et al. Substitution of an aspartic acid results in constitutive activation of c-kit receptor tyrosine kinase in a rat tumor mast cell line RBL-2H3. Int Arch Allergy Immunol 1995, 106:377-385.
55. Hashimoto K., Tsujimura T., Moriyama Y., Yamatodani A., Kimura M., Tohya K., et al. Transforming and differentiation-inducing potential of constitutively activated c-kit mutant genes in the IC-2 murine interleukin-3-dependent mast cell line. Am J Pathol 1996, 148:189-200.
56. Nagata H., Worobec A.S., Oh C.K., Chowdhury B.A., Tannenbaum S., Suzuki Y., et al. Identification of a point mutation in the catalytic domain of the protooncogene c-kit in peripheral blood mononuclear cells of patients who have mastocytosis with an associated hematologic disorder. Proc Natl Acad Sci USA 1995, 92:10560-10564.
57. Longley B.J., Tyrrell L., Lu S.Z., Ma Y.S., Langley K., Ding T.G., et al. Somatic c-KIT activating mutation in urticaria pigmentosa and aggressive mastocytosis: establishment of clonality in a human mast cell neoplasm. Nat Genet 1996, 12:312-314.
58. Garcia-Montero A.C., Jara-Acevedo M., Teodosio C., Sanchez M.L., Nunez R., Prados A., et al. KIT mutation in mast cells and other bone marrow hematopoietic cell lineages in systemic mast cell disorders: a prospective study of the Spanish Network on Mastocytosis (REMA) in a series of 113 patients. Blood 2006, 108:2366-2372.
59. Bodemer C., Hermine O., Palmerini F., Yang Y., Grandpeix-Guyodo C., Leventhal P.S., et al. Pediatric mastocytosis is a clonal disease associated with D816V and other activating c-KIT mutations. J Invest Dermatol 2010, 130:804-815.
60. Pignon J.M., Giraudier S., Duquesnoy P., Jouault H., Imbert M., Vainchenker W., et al. A new c-kit mutation in a case of aggressive mast cell disease. Br J Haematol 1997, 96:374-376.
61. Longley B.J., Metcalfe D.D., Tharp M., Wang X., Tyrrell L., Lu S.Z., et al. Activating and dominant inactivating c-KIT catalytic domain mutations in distinct clinical forms of human mastocytosis. Proc Natl Acad Sci USA 1999, 96:1609-1614.
62. Pullarkat V.A., Bueso-Ramos C., Lai R., Kroft S., Wilson C.S., Pullarkat S.T., et al. Systemic mastocytosis with associated clonal hematological non-mast-cell lineage disease: analysis of clinicopathologic features and activating c-kit mutations. Am J Hematol 2003, 73:12-17.
63. Sotlar K., Escribano L., Landt O., Mohrle S., Herrero S., Torrelo A., et al. One-step detection of c-kit point mutations using peptide nucleic acid-mediated polymerase chain reaction clamping and hybridization probes. Am J Pathol 2003, 162:737-746.
64. Nakagomi N., Hirota S. Juxtamembrane-type c-kit gene mutation found in aggressive systemic mastocytosis induces imatinib-resistant constitutive KIT activation. Lab Invest 2007, 87:365-371.
65. Buttner C., Henz B.M., Welker P., Sepp N.T., Grabbe J. Identification of activating c-kit mutations in adult-, but not in childhood-onset indolent mastocytosis: a possible explanation for divergent clinical behavior. J Invest Dermatol 1998, 111:1227-1231.
66. Akin C., Fumo G., Yavuz A.S., Lipsky P.E., Neckers L., Metcalfe D.D. A novel form of mastocytosis associated with a transmembrane c-kit mutation and response to imatinib. Blood 2004, 103:3222-3225.
67. Ozer O., Zhao Y.D., Ostler K.R., Akin C., Anastasi J., Vardiman J.W., et al. The identification and characterisation of novel KIT transcripts in aggressive mast cell malignancies and normal CD34+ cells. Leuk Lymp 2008, 49:1567-1577.
68. Tang X., Boxer M., Drummond A., Ogston P., Hodgins M., Burden A.D. A germline mutation in KIT in familial diffuse cutaneous mastocytosis. J Med Genet 2004, 41:e88.
69. Zhang L.Y., Smith M.L., Schultheis B., Fitzgibbon J., Lister T.A., Melo J.V., et al. A novel K509I mutation of KIT identified in familial mastocytosis-in vitro and in vivo responsiveness to imatinib therapy. Leuk Res 2006, 30:373-378.
70. Hartmann K., Wardelmann E., Ma Y., Merkelbach-Bruse S., Preussner L.M., Woolery C., et al. Novel germline mutation of KIT associated with familial gastrointestinal stromal tumors and mastocytosis. Gastroenterology 2005, 129:1042-1046.
71. Hoffmann K.M., Moser A., Lohse P., Winkler A., Binder B., Sovinz P., et al. Successful treatment of progressive cutaneous mastocytosis with imatinib in a 2-year-old boy carrying a somatic KIT mutation. Blood 2008, 112:1655-1657.
72. Yanagihori H., Oyama N., Nakamura K., Kaneko F. c-kit Mutations in patients with childhood-onset mastocytosis and genotype-phenotype correlation. J Mol Diagn 2005, 7:252-257.
73. Verzijl A., Heide R., Oranje A.P., van Schaik R.H. C-kit Asp-816-Val mutation analysis in patients with mastocytosis. Dermatology 2007, 214:15-20.
74. Teodosio C., Garcia-Montero A.C., Jara-Acevedo M., Sanchez-Munoz L., Alvarez-Twose I., Nunez R., et al. Mast cells from different molecular and prognostic subtypes of systemic mastocytosis display distinct immunophenotypes. J Allergy Clin Immunol 2010, 125:719-726.
75. Yavuz A.S., Lipsky P.E., Yavuz S., Metcalfe D.D., Akin C. Evidence for the involvement of a hematopoietic progenitor cell in systemic mastocytosis from single-cell analysis of mutations in the c-kit gene. Blood 2002, 100:661-665.
76. Akin C., Kirshenbaum A.S., Semere T., Worobec A.S., Scott L.M., Metcalfe D.D. Analysis of the surface expression of c-kit and occurrence of the c-kit Asp816Val activating mutation in T cells, B cells, and myelomonocytic cells in patients with mastocytosis. Exp Hematol 2000, 28:140-147.
77. Baumgartner C., Cerny-Reiterer S., Sonneck K., Mayerhofer M., Gleixner K.V., Fritz R., et al. Expression of activated STAT5 in neoplastic mast cells in systemic mastocytosis: subcellular distribution and role of the transforming oncoprotein KIT D816V. Am J Pathol 2009, 175:2416-2429.
78. Shelburne C.P., McCoy M.E., Piekorz R., Sexl V., Roh K.H., Jacobs-Helber S.M., et al. Stat5 expression is critical for mast cell development and survival. Blood 2003, 102:1290-1297.
79. Shelburne C.P., McCoy M.E., Piekorz R., Sexl V.V., Gillespie S.R., Bailey D.P., et al. Stat5: an essential regulator of mast cell biology. Mol Immunol 2002, 38:1187-1191.
80. Harir N., Boudot C., Friedbichler K., et al. Oncogenic kit controls neoplastic mast cell growth through a Stat5/PI3-kinase signaling cascade. Blood 2008, 112:2463-2473.
81. Ning Z.Q., Li J., Arceci R.J. Signal transducer and activator of transcription 3 activation is required for Asp(816) mutant c-Kit-mediated cytokine-independent survival and proliferation in human leukemia cells. Blood 2001, 97:3559-3567.
82. Chaix A., Lopez S., Voisset E., et al. Mechanisms of STAT protein activation by oncogenic KIT mutants in neoplastic mast cells. J Biol Chem 2011, 286:5956-5966.
83. Mekori Y.A., Gilfillan A.M., Akin C., et al. Human mast cell apoptosis is regulated through Bcl-2 and Bcl-XL. J Clin Immunol 2001, 21:171-174.
84. Cervero C., Escribano L., San Miguel J.F., et al. Expression of Bcl-2 by human bone marrow mast cells and its overexpression in mast cell leukemia. Am J Hematol 1999, 60:191-195.
85. Aichberger K.J., Gleixner K.V., Mirkina I., Cerny-Reiterer S., Peter B., Ferenc V., et al. Identification of proapoptotic Bim as a tumor suppressor in neoplastic mast cells: role of KIT D816V and effects of various targeted drugs. Blood 2009, 114:5342-5351.
86. Moller C., Alfredsson J., Engstrom M., et al. Stem cell factor promotes mast cell survival via inactivation of FOXO3a-mediated transcriptional induction and MEK-regulated phosphorylation of the proapoptotic protein Bim. Blood 2005, 106:1330-1336.
87. Chian R., Young S., Danilkovitch-Miagkova A., et al. Phosphatidylinositol 3 kinase contributes to the transformation of hematopoietic cells by the D816V c-Kit mutant. Blood 2001, 98:1365-1373.
88. Blume-Jensen P., Siegbahn A., Stabel S., Heldin C.H., Ronnstrand L. Increased Kit/SCF receptor induced mitogenicity but abolished cell motility after inhibition of protein kinase C. EMBO J 1993, 12:4199-4209.
89. Blume-Jensen P., Wernstedt C., Heldin C.H., Ronnstrand L. Identification of the major phosphorylation sites for protein kinase C in kit/stem cell factor receptor in vitro and in intact cells. J Biol Chem 1995, 270:14192-14200.
90. Mauro M.J., Deininger M. Management of drug toxicities in chronic myeloid leukaemia. Best Pract Res Clin Haematol 2009, 22:409-429.
91. Gleevec dosing and administration. http://www.gleevec.com/health-care-professional/dosing-for-gleevec.jsp.
92. Dasatinib dosing. http://www.sprycel.com/hcp/dosing.aspx.
93. Nilotinib. http://www.us.tasigna.com/health-care-professional.
94. Chen H., Isozaki K., Kinoshita K., Ohashi A., Shinomura Y., Matsuzawa Y., et al. Imatinib inhibits various types of activating mutant kit found in gastrointestinal stromal tumors. Int J Cancer 2003, 105:130-135.
95. Akin C., Brockow K., D'Ambrosio C., Kirshenbaum A.S., Ma Y., Longley B.J., et al. Effects of tyrosine kinase inhibitor STI571 on human mast cells bearing wild-type or mutated c-kit. Exp Hematol 2003, 31:686-692.
96. Ma Y., Zeng S., Metcalfe D.D., Akin C., Dimitrijevic S., Butterfield J.H., et al. The c-KIT mutation causing human mastocytosis is resistant to STI571 and other KIT kinase inhibitors; kinases with enzymatic site mutations show different inhibitor sensitivity profiles than wild-type kinases and those with regulatory-type mutations. Blood 2002, 99:1741-1744.
97. Frost M.J., Ferrao P.T., Hughes T.P., Ashman L.K. Juxtamembrane mutant V560GKit is more sensitive to Imatinib (STI571) compared with wild-type c-kit whereas the kinase domain mutant D816VKit is resistant. Mol Cancer Ther 2002, 1:1115-1124.
98. Zermati Y., De Sepulveda P., Feger F., Letard S., Kersual J., Casteran N., et al. Effect of tyrosine kinase inhibitor STI571 on the kinase activity of wild-type and various mutated c-kit receptors found in mast cell neoplasms. Oncogene 2003, 22:660-664.
99. Longley B.J., Ma Y., Carter E., McMahon G. New approaches to therapy for mastocytosis. A case for treatment with kit kinase inhibitors. Hematol Oncol Clin North Am 2000, 14:689-695.
100. Vega-Ruiz A., Cortes J.E., Sever M., Manshouri T., Quintas-Cardama A., Luthra R., et al. Phase II study of imatinib mesylate as therapy for patients with systemic mastocytosis. Leuk Res 2009, 33:1481-1484.
101. Pagano L., Valentini C.G., Caira M., Rondoni M., Van Lint M.T., Candoni A., et al. Advanced mast cell disease: an Italian hematological multicenter experience. Int J Hematol 2008, 88:483-488.
102. Pardanani A., Elliott M., Reeder T., Li C.Y., Baxter E.J., Cross N.C., et al. Imatinib for systemic mast-cell disease. Lancet 2003, 362:535-536.
103. Lim K.H., Pardanani A., Butterfield J.H., Li C.Y., Tefferi A. Cytoreductive therapy in 108 adults with systemic mastocytosis: outcome analysis and response prediction during treatment with interferon-alpha, hydroxyurea, imatinib mesylate or 2-chlorodeoxyadenosine. Am J Hematol 2009, 84:790-794.
104. Droogendijk H.J., Kluin-Nelemans H.J., van Doormaal J.J., Oranje A.P., van de Loosdrecht A.A., van Daele P.L. Imatinib mesylate in the treatment of systemic mastocytosis: a phase II trial. Cancer 2006, 107:345-351.
105. Heinrich M.C., Joensuu H., Demetri G.D., Corless C.L., Apperley J., Fletcher J.A., et al. Phase II, open-label study evaluating the activity of imatinib in treating life-threatening malignancies known to be associated with imatinib-sensitive tyrosine kinases. Clin Cancer Res 2008, 14:2717-2725.
106. Gotlib J., Cools J., Malone J.M., Schrier S.L., Gilliland D.G., Coutre S.E. The FIP1L1-PDGFRalpha fusion tyrosine kinase in hypereosinophilic syndrome and chronic eosinophilic leukemia: implications for diagnosis, classification, and management. Blood 2004, 103:2879-2891.
107. Klion A.D., Noel P., Akin C., Law M.A., Gilliland D.G., Cools J., et al. Elevated serum tryptase levels identify a subset of patients with a myeloproliferative variant of idiopathic hypereosinophilic syndrome associated with tissue fibrosis, poor prognosis, and imatinib responsiveness. Blood 2003, 101:4660-4666.
108. Pardanani A., Ketterling R.P., Brockman S.R., Flynn H.C., Paternoster S.F., Shearer B.M., et al. CHIC2 deletion, a surrogate for FIP1L1-PDGFRA fusion, occurs in systemic mastocytosis associated with eosinophilia and predicts response to imatinib mesylate therapy. Blood 2003, 102:3093-3096.
109. Yamada Y., Cancelas J.A. FIP1L1/PDGFR alpha-associated systemic mastocytosis. Int Arch Allergy Immunol 2010, 152(Suppl. 1):101-105.
110. Lahortiga I., Akin C., Cools J., Wilson T.M., Mentens N., Arthur D.C., et al. Activity of imatinib in systemic mastocytosis with chronic basophilic leukemia and a PRKG2-PDGFRB fusion. Haematologica 2008, 93:49-56.
111. Pardanani A., Verstovsek S. Hypereosinophilic syndrome, chronic eosinophilic leukemia, and mast cell disease. Cancer J 2007, 13:384-391.
112. Florian S., Esterbauer H., Binder T., Mullauer L., Haas O.A., Sperr W.R., et al. Systemic mastocytosis (SM) associated with chronic eosinophilic leukemia (SM-CEL): detection of FIP1L1/PDGFRalpha, classification by WHO criteria, and response to therapy with imatinib. Leuk Res 2006, 30:1201-1205.
113. Bain B.J., Gilliand D.G., Horny H.P., Vardiman J.W. Myeloid and lymphoid neoplasms with eosinophilia and abnormalities of PDGFRA, PDGFRB or FGFR1. World Health Organization (WHO) Classification of tumours. Pathology & genetics. Tumours of haematopoietic and lymphoid tissues 2008, 68-73. IARC Press, Lyon. S.H. Swerdlow, E. Campo, N.L. Harris, E.S. Jaffe, S.A. Pileri, H. Stein, J. Thiele, J.W. Vardiman (Eds.).
114. Copland M., Hamilton A., Elrick L.J., et al. Dasatinib (BMS-354825) targets an earlier progenitor population than imatinib in primary CML but does not eliminate the quiescent fraction. Blood 2006, 107:4532-4539.
115. Schittenhelm M.M., Shiraga S., Schroeder A., 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, 66:473-481.
116. Fabarius A., Giehl M., Rebacz B., Kramer A., Frank O., Haferlach C., et al. Centrosome aberrations and G1 phase arrest after in vitro and in vivo treatment with the SRC/ABL inhibitor dasatinib. Haematologica 2008, 93:1145-1154.
117. Shah N.P., Lee F.Y., Luo R., Jiang Y., Donker M., Akin C. Dasatinib (BMS-354825) inhibits KITD816V, an imatinib-resistant activating mutation that triggers neoplastic growth in most patients with systemic mastocytosis. Blood 2006, 108:286-291.
118. Gleixner K.V., Mayerhofer M., Sonneck K., et al. Synergistic growth-inhibitory effects of two tyrosine kinase inhibitors, dasatinib and PKC412, on neoplastic mast cells expressing the D816V-mutated oncogenic variant of KIT. Haematologica 2007, 92:1451-1459.
119. Aichberger K.J., Sperr W.R., Gleixner K.V., et al. Treatment responses to cladribine and dasatinib in rapidly progressing aggressive mastocytosis. Eur J Clin Invest 2008, 38:869-873.
120. Gleixner K.V., Mayerhofer M., Aichberger K.J., Derdak S., Sonneck K., Bohm A., et al. PKC412 inhibits in vitro growth of neoplastic human mast cells expressing the D816V-mutated variant of KIT: comparison with AMN107, imatinib, and cladribine (2CdA) and evaluation of cooperative drug effects. Blood 2006, 107:752-759.
121. Verstovsek S., Tefferi A., Cortes J., O'Brien S., Garcia-Manero G., Pardanani A., et al. Phase II study of dasatinib in Philadelphia chromosome-negative acute and chronic myeloid diseases, including systemic mastocytosis. Clin Cancer Res 2008, 14:3906-3915.
122. Purtill D., Cooney J., Sinniah R., Carnley B., Cull G., Augustson B., et al. Dasatinib therapy for systemic mastocytosis: four cases. Eur J Haematol 2008, 80:456-458.
123. Benz R., Boesiger J., Fehr J. Systemic mastocytosis with c-KITD816V mutation treated with dasatinib. Blood 2007, 110(258b). [suppl., abstr 4656].
124. Ustun C., Corless C.L., Savage N., Fiskus W., Manaloor E., Heinrich M.C., et al. Chemotherapy and dasatinib induce long-term hematologic and molecular remission in systemic mastocytosis with acute myeloid leukemia with KIT D816V. Leuk Res 2009, 33:735-741.
125. Kneidinger M., Schmidt U., Rix U., et al. The effects of dasatinib on IgE receptor-dependent activation and histamine release in human basophils. Blood 2008, 111:3097-3107.
126. Schade A.E., Schieven G.L., Townsend R., et al. Dasatinib, a small-molecule protein tyrosine kinase inhibitor, inhibits T-cell activation and proliferation. Blood 2008, 111:1366-1377.
127. Weichsel R., Dix C., Wooldridge L., et al. Profound inhibition of antigen-specific T-cell effector functions by dasatinib. Clin Cancer Res 2008, 14:2484-2491.
128. Sillaber C., Herrmann H., Bennett K., et al. Immunosuppression and atypical infections in CML patients treated with dasatinib at 140mg daily. Eur J Clin Invest 2009, 39:1098-1109.
129. Quintas-Cardama A., Kantarjian H., brien O., et al. Pleural effusion in patient with chronic myelogenous leukemia (CML) treated with dasatinib after imatinib failure. J Clin Oncol 2007, 25:3908-3914.
130. Hochhaus A., Kantarjian H.M., Baccarani M., et al. Dasatinib induces notable hematologic and cytogenetic responses in chronic-phase chronic myeloid leukemia after failure of imatinib therapy. Blood 2007, 109:2303-2309.
131. Rix U., Hantschel O., Durnberger G., Remsing Rix L.L., Planyavsky M., Fernbach N.V., et al. Chemical proteomic profiles of the BCR-ABL inhibitors imatinib, nilotinib, and dasatinib reveal novel kinase and nonkinase targets. Blood 2007, 110:4055-4063.
132. Deremer D.L., Ustun C., Natarajan K. Nilotinib: a second-generation tyrosine kinase inhibitor for the treatment of chronic myelogenous leukemia. Clin Ther 2008, 30:1956-1975.
133. Deremer D.L., Katsanevas K., Ustun C. Critical appraisal of nilotinib in frontline treatment of chronic myeloid leukemia. Cancer Manag Res 2011, 3:65-78.
134. von Bubnoff N., Gorantla S.H., Kancha R.K., Lordick F., Peschel C., Duyster J. The systemic mastocytosis-specific activating cKit mutation D816V can be inhibited by the tyrosine kinase inhibitor AMN107. Leukemia 2005, 19:1670-1671.
135. Verstovsek S., Akin C., Manshouri T., Quintas-Cardama A., Huynh L., Manley P., et al. Effects of AMN107, a novel aminopyrimidine tyrosine kinase inhibitor, on human mast cells bearing wild-type or mutated codon 816 c-kit. Leuk Res 2006, 30:1365-1370.
136. Hochhaus A., Ottmann O.G., Lauber S., Hughes T., Verhoef G., Schwarer A.P., et al. A phase II study of nilotinib, a novel inhibitor of c-Kit, PDGFR, and Bcr-Abl, administered to patients with systemic mastocytosis. Blood 2006, 108. [abstract 2703].
137. Schenk P.W., Snaar-Jagalska B.E. Signal preception and transduction: the role protein kinases. Biochim Biophys Acta 1999, 1449:1-24.
138. Fishman D.D., Segal S., Livneh E. The role of protein kinase C in G1 and G2/M phases of the cell cycle (review). Int J Oncol 1998, 12:181-186.
139. Meyer T., Regenass U., Fabbro D., et al. A derivative of staurosporine (CGP 41251) shows selectivity of protein kinase C inhibition and in vitro anti-proliferative as well as in vivo anti-tumor activity. Int J Cancer 1989, 43:851-856.
140. Propper D.J., McDonal A.C., Man A., et al. Phase I and pharmacokinetic study of PKC412, an inhibitor of protein kinase C. J Clin Oncol 2001, 19:1485-1492.
141. Levis M., Brown P., Smith B.D., et al. Plasma inhibitory activity (PIA): a pharmacodynamid assay reveals insights into the basis for cytotoxic response to FLT3 inhibitors. Blood 2006, 108:3477-3483.
142. Gotlib J., Berube C., Growney J.D., Chen C.C., George T.I., Williams C., et al. Activity of the tyrosine kinase inhibitor PKC412 in a patient with mast cell leukemia with the D816V KIT mutation. Blood 2005, 106:2865-2870.
143. Krauth M.T., Mirkina I., Herrmann H., Baumgartner C., Kneidinger M., Valent P. Midostaurin (PKC412) inhibits immunoglobulin E-dependent activation and mediator release in human blood basophils and mast cells. Clin Exp Allergy 2009, 39:1711-1720.
144. Gotlib J., Angelo D.J., George T.I., Corless C.L., Linder A., Langford C., et al. KIT inhibitor midostaurin exhibits a high rate of clinically meaningful and durable responses in advanced systemic mastocytosis: report of a fully accrued phase II trial. Blood 2010, 116. [abstract 316].
145. Dubreuil P., Letard S., Ciufolini M., Gros L., Humbert M., Casteran N., et al. Masitinib (AB1010), a potent and selective tyrosine kinase inhibitor targeting KIT. PLoS One 2009, 4:e7258.
146. Hahn K.A., Ogilvie G., Rusk T., Devauchelle P., Leblanc A., Legendre A., et al. Masitinib is safe and effective for the treatment of canine mast cell tumors. J Vet Intern Med 2008, 22:1301-1309.
147. Hahn K.A., Legendre A.M., Shaw N.G., Phillips B., Ogilvie G.K., Prescott D.M., et al. Evaluation of 12- and 24-month survival rates after treatment with masitinib in dogs with nonresectable mast cell tumors. Am J Vet Res 2010, 71:1354-1361.
148. Corbin A.S., Griswold I.J., La Rosee P., Yee K.W., Heinrich M.C., Reimer C.L., et al. Sensitivity of oncogenic KIT mutants to the kinase inhibitors MLN518 and PD180970. Blood 2004, 104:3754-3757.
149. Kosmider O., Denis N., Dubreuil P., Moreau-Gachelin F. Semaxinib (SU5416) as a therapeutic agent targeting oncogenic Kit mutants resistant to imatinib mesylate. Oncogene 2007, 26:3904-3908.
150. Pan J., Quintas-Cardama A., Kantarjian H.M., Akin C., Manshouri T., Lamb P., et al. EXEL-0862, a novel tyrosine kinase inhibitor, induces apoptosis in vitro and ex vivo in human mast cells expressing the KIT D816V mutation. Blood 2007, 109:315-322.
151. Peter B., Hadzijusufovic E., Blatt K., Gleixner K.V., Pickl W.F., Thaiwong T., et al. KIT polymorphisms and mutations determine responses of neoplastic mast cells to bafetinib (INNO-406). Exp Hematol 2010, 38:782-791.
152. Corbin A.S., Demehri S., Griswold I.J., Wang Y., Metcalf C.A., Sundaramoorthi R., et al. In vitro and in vivo activity of ATP-based kinase inhibitors AP23464 and AP23848 against activation-loop mutants of Kit. Blood 2005, 106:227-234.
153. Faderl S., Pal A., Bornmann W., Albitar M., Maxwell D., Van Q., et al. Kit inhibitor APcK110 induces apoptosis and inhibits proliferation of acute myeloid leukemia cells. Cancer Res 2009, 69:3910-3917.