UNAIDS: WHO Classification of Tumors of the Haemopoietic and Lymphoid Tissues. Edited by Swerdlow CE, Harris SH. WHO; 2008.
Druker BJ, et al.: Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med 2006, 355:2408–2417.CrossRefPubMed
Hochhaus A, et al.: Dasatinib induces durable cytogenetic responses in patients with chronic myelogenous leukemia in chronic phase with resistance or intolerance to imatinib. Leukemia 2008, 22:1200–1206.CrossRefPubMed
Kantarjian HM, et al.: Nilotinib (formerly AMN107), a highly selective BCR-ABL tyrosine kinase inhibitor, is effective in patients with Philadelphia chromosome-positive chronic myelogenous leukemia in chronic phase following imatinib resistance and intolerance. Blood 2007, 110:3540–3546.CrossRefPubMed
Weisberg E, et al.: Characterization of AMN107, a selective inhibitor of native and mutant Bcr-Abl. Cancer Cell 2005, 7:129–141.CrossRefPubMed
Shah NP, et al.: Overriding imatinib resistance with a novel ABL kinase inhibitor. Science 2004, 305:399–401.CrossRefPubMed
Kantarjian HM, et al.: Optimizing therapy for patients with chronic myelogenous leukemia in chronic phase. Cancer 2010, 116:1419–1430.CrossRefPubMed
•• Saglio G, et al.: Nilotinib versus imatinib for newly diagnosed chronic myeloid leukemia. N Engl J Med 2010, 362(24):2251–2259.
•• Kantarjian H, et al.: Dasatinib versus imatinib in newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med 2010, 362(24):2260–2270.
Dameshek W: Some speculations on the myeloproliferative syndromes. Blood 1951, 6:372–375.PubMed
Baxter EJ, et al.: Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet 2005, 365:1054–1061.PubMed
Kralovics R, et al.: A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med 2005, 352:1779–1790.CrossRefPubMed
Levine RL, et al.: Activating mutation in the tyrosine kinase JAK2 in polycythemia vera, essential thrombocythemia, and myeloid metaplasia with myelofibrosis. Cancer Cell 2005, 7:387–397.CrossRefPubMed
James C, et al.: A unique clonal JAK2 mutation leading to constitutive signalling causes polycythaemia vera. Nature 2005, 434:1144–1148.CrossRefPubMed
Hexner EO, et al.: Lestaurtinib (CEP701) is a JAK2 inhibitor that suppresses JAK2/STAT5 signaling and the proliferation of primary erythroid cells from patients with myeloproliferative disorders. Blood 2008, 111:5663–5671.CrossRefPubMed
Pardanani A, et al.: TG101209, a small molecule JAK2-selective kinase inhibitor potently inhibits myeloproliferative disorder-associated JAK2V617F and MPLW515L/K mutations. Leukemia 2007, 21:1658–1668.CrossRefPubMed
Quintas-Cardama A, et al.: Preclinical characterization of the selective JAK1/2 inhibitor INCB018424: therapeutic implications for the treatment of myeloproliferative neoplasms. Blood 2010, 115:3109–3117.CrossRefPubMed
Verstovsek S, Kantarjian H, Mesa RA, et al.: Long-term follow up and optimized dosing regimen of INCB018424 in patients with myelofibrosis: durable clinical, functional and symptomatic responses with improved hematological safety. In 51st American Society of Hematology. New Orleans: 2009.
Pardanani A, Gotlib JR, Jamieson C, et al.: A phase I evaluation of TG101348, a selective JAK2 inhibitor, in myelofibrosis: clinical response is accompanied by significant reduction in JAK2V617F allele burden. In 51st American Society of Hematology. New Orleans: 2009.
Santos FP, et al.: Phase 2 study of CEP-701, an orally available JAK2 inhibitor, in patients with primary or post-polycythemia vera/essential thrombocythemia myelofibrosis. Blood 2010, 115:1131–1136.CrossRefPubMed
Hexner E, Goldberg JD, Prchal JT, et al.: A multicenter, open label phase I/II study of CEP701 (lestaurtinib) in adults with myelofibrosis; a report on phase I: A Study of the Myeloproliferative Disorders Research Consortium (MPD-RC). In 51st American Society of Hematology. New Orleans: 2009.
Mesa RA, et al.: The Myelofibrosis Symptom Assessment Form (MFSAF): an evidence-based brief inventory to measure quality of life and symptomatic response to treatment in myelofibrosis. Leuk Res 2009, 33:1199–1203.CrossRefPubMed
Moliterno A, Hexner E, Roboz GJ, et al.: An open-label study of CEP-701 in patients with JAK2 V617F-positive PV and ET: update of 39 enrolled patients. In 51st American Society of Hematology. New Orleans: 2009.
Verstovsek S, Passamonti F, Rambaldi A, et al.: A Phase 2 study of INCB018424, an oral, selective JAK1/JAK2 inhibitor, in patients with advanced polycythemia vera (PV) and essential thrombocythemia (ET) refractory to hydroxyurea. In 51st American Society of Hematology. New Orleans: 2009.
Swerdlow SH, Harris CN: WHO Classification of Tumors of Haematopoietic and Lymphoid Tissues. Lyon: IARC; 2008.
Goldstone AH, et al.: Attempts to improve treatment outcomes in acute myeloid leukemia (AML) in older patients: the results of the United Kingdom Medical Research Council AML11 trial. Blood 2001, 98:1302–1311.CrossRefPubMed
Rowe JM, et al.: A phase 3 study of three induction regimens and of priming with GM-CSF in older adults with acute myeloid leukemia: a trial by the Eastern Cooperative Oncology Group. Blood 2004, 103:479–485.CrossRefPubMed
Mardis ER, et al.: Recurring mutations found by sequencing an acute myeloid leukemia genome. N Engl J Med 2009, 361:1058–1066.CrossRefPubMed
Rucker FG, et al.: Disclosure of candidate genes in acute myeloid leukemia with complex karyotypes using microarray-based molecular characterization. J Clin Oncol 2006, 24:3887–3894.CrossRefPubMed
Schlenk RF, et al.: Mutations and treatment outcome in cytogenetically normal acute myeloid leukemia. N Engl J Med 2008, 358:1909–1918.CrossRefPubMed
Suela J, et al.: DNA profiling analysis of 100 consecutive de novo acute myeloid leukemia cases reveals patterns of genomic instability that affect all cytogenetic risk groups. Leukemia 2007, 21:1224–1231.CrossRefPubMed
Small D, et al.: STK-1, the human homolog of Flk-2/Flt-3, is selectively expressed in CD34+ human bone marrow cells and is involved in the proliferation of early progenitor/stem cells. Proc Natl Acad Sci U S A 1994, 91:459–463.CrossRefPubMed
Small D: FLT3 mutations: biology and treatment. Hematol Am Soc Hematol Educ Program 2006, 178–184.
Yamamoto Y, et al.: Activating mutation of D835 within the activation loop of FLT3 in human hematologic malignancies. Blood 2001, 97:2434–2439.CrossRefPubMed
Griffith J, et al.: The structural basis for autoinhibition of FLT3 by the juxtamembrane domain. Mol Cell 2004, 13:169–178.CrossRefPubMed
Levis M, Small D: FLT3: It does matter in leukemia. Leukemia 2003, 17:1738–1752.CrossRefPubMed
Kiyoi H, et al.: Mechanism of constitutive activation of FLT3 with internal tandem duplication in the juxtamembrane domain. Oncogene 2002, 21:2555–2563.CrossRefPubMed
Abu-Duhier FM, et al.: Identification of novel FLT-3 Asp835 mutations in adult acute myeloid leukaemia. Br J Haematol 2001, 113:983–988.CrossRefPubMed
Kottaridis PD, et al.: The presence of a FLT3 internal tandem duplication in patients with acute myeloid leukemia (AML) adds important prognostic information to cytogenetic risk group and response to the first cycle of chemotherapy: analysis of 854 patients from the United Kingdom Medical Research Council AML 10 and 12 trials. Blood 2001, 98:1752–1759.CrossRefPubMed
Pratz K, Levis M: Incorporating FLT3 inhibitors into acute myeloid leukemia treatment regimens. Leuk Lymphoma 2008, 49:852–863.CrossRefPubMed
Stone RM, et al.: Patients with acute myeloid leukemia and an activating mutation in FLT3 respond to a small-molecule FLT3 tyrosine kinase inhibitor, PKC412. Blood 2005, 105:54–60.CrossRefPubMed
Smith BD, et al.: Single-agent CEP-701, a novel FLT3 inhibitor, shows biologic and clinical activity in patients with relapsed or refractory acute myeloid leukemia. Blood 2004, 103:3669–3676.CrossRefPubMed
Giles FJ, et al.: SU5416, a small molecule tyrosine kinase receptor inhibitor, has biologic activity in patients with refractory acute myeloid leukemia or myelodysplastic syndromes. Blood 2003, 102:795–801.CrossRefPubMed
O’Farrell AM, et al.: An innovative phase I clinical study demonstrates inhibition of FLT3 phosphorylation by SU11248 in acute myeloid leukemia patients. Clin Cancer Res 2003, 9:5465–5476.PubMed
• Ravandi F, et al.: Phase I/II study of combination therapy with sorafenib, idarubicin, and cytarabine in younger patients with acute myeloid leukemia. J Clin Oncol 2010, 28(11):1856–1862.
Pratz KW, et al.: A pharmacodynamic study of the FLT3 inhibitor KW-2449 yields insight into the basis for clinical response. Blood 2009, 113:3938–3946.CrossRefPubMed
Cortes JFJ, Ghirdaladze D, et al.: AC220, a potent, selective, second generation FLT3 receptor tyrosine kinase (RTK) inhibitor, in a first-in-human (FIH) phase 1 AML study. In 51st Meeting of the American Society of Hematology. New Orleans: 2009.
Levis M, Wang RF, et al.: Results from a randomized trial of salvage chemotherapy followed by lestaurtinib for FLT3 mutant AML patients in first relapse. In 51st Meeting of the American Society of Hematology. New Orleans: 2009.
Zarrinkar PP, et al.: AC220 is a uniquely potent and selective inhibitor of FLT3 for the treatment of acute myeloid leukemia (AML). Blood 2009, 114:2984–2992.CrossRefPubMed
Stone RM, et al.: A phase 1b study of midostaurin (PKC412) in combination with daunorubicin and cytarabine induction and high-dose cytarabine consolidation in patients under age 61 with newly diagnosed de novo acute myeloid leukemia: Overall survival of patients whose blasts have FLT3 mutations is similar to those with wild-type FLT3. In 51st Meeting of the American Society of Hematology. New Orleans: 2009.