Beyond tyrosine kinase inhibitors: Combinations and other agents

Best Practice & Research Clinical Haematology - Tập 29 - Trang 271-283 - 2016
Emilie Cayssials1, Francois Guilhot1
1INSERM CIC 1402, CHU de Poitiers, Poitiers, France

Tài liệu tham khảo

Deininger, 2009, International randomized study of interferon vs STI571 (IRIS) 8-year follow up: sustained survival and low risk for progression or events in patients with newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP) treated with imatinib, Blood (ASH Annual Meeting Abstracts), 114, 1126 Baccarani, 2013, European LeukemiaNet recommendations for the management of chronic myeloid leukemia: 2013, Blood, 122, 872, 10.1182/blood-2013-05-501569 O'Brien, 2003, Imatinib compared with interferon and low-dose cytarabine for newly diagnosed chronic-phase chronic myeloid leukemia, N Engl J Med, 348, 994, 10.1056/NEJMoa022457 Druker, 2006, Five-year follow-up of patients receiving imatinib for chronic myeloid leukaemia, N Engl J Med, 355, 2408, 10.1056/NEJMoa062867 Mahon, 2010, Discontinuation of imatinib in patients with chronic myeloid leukaemia who have maintained complete molecular remission for at least 2 years: the prospective, multicentre Stop Imatinib (STIM) trial, Lancet Oncol, 11, 1029, 10.1016/S1470-2045(10)70233-3 Ross, 2010, Patients with chronic myeloid leukemia who maintain a complete molecular response after stopping imatinib treatment have evidence of persistent leukemia by DNA PCR, Leukemia, 24, 1719, 10.1038/leu.2010.185 Hu, 2006, Targeting multiple kinase pathways in leukemic progenitors and stem cells is essential for improved treatment of Ph+ leukemia in mice, Proc Natl Acad Sci U S A, 103, 16870, 10.1073/pnas.0606509103 Jamieson, 2008, Chronic myeloid leukemia stem cells, Hematol Am Soc Hematol Educ Program, 436, 10.1182/asheducation-2008.1.436 Rice, 2010, Molecular pathways to CML stem cells, Int J Hematol, 91, 748, 10.1007/s12185-010-0615-8 Zhao, 2009, Hedgehog signalling is essential for maintenance of cancer stem cells in myeloid leukemia, Nature, 458, 776, 10.1038/nature07737 Bhatia, 2003, Persistence of malignant hematopoietic progenitors in chronic myelogenous leukemia patients in complete cytogenetic remission following imatinib mesylate treatment, Blood, 101, 4701, 10.1182/blood-2002-09-2780 Chomel, 2000, Persistence of BCR-ABL genomic rearrangement in chronic myeloid leukemia patients in complete and sustained cytogenetic remission after interferon-alpha therapy or allogeneic bone marrow transplantation, Blood, 95, 404, 10.1182/blood.V95.2.404 Talpaz, 1994, Persistence of dormant leukemic progenitors during interferon-induced remission in chronic myelogenous leukemia. Analysis by polymerase chain reaction of individual colonies, J Clin Invest, 94, 1383, 10.1172/JCI117473 Bocchia, 2008, CD34+/Ph+ cells are still detectable in chronic myeloid leukemia patients with sustained and prolonged complete cytogenetic remission during treatment with imatinib mesylate, Leukemia, 22, 426, 10.1038/sj.leu.2404893 Mustjoki, 2010, Low or undetectable numbers of Philadelphia chromosome-positive leukemic stem cells (Ph(+)CD34(+)CD38(neg)) in chronic myeloid leukemia patients in complete cytogenetic remission after tyrosine kinase inhibitor therapy, Leukemia, 24, 219, 10.1038/leu.2009.190 Nair, 2010, The bone marrow microenvironment as a sanctuary for minimal residual disease in CML, Biochem Pharmacol, 80, 602, 10.1016/j.bcp.2010.04.003 Ben-Neriah, 1986, The chronic myelogenous leukaemia-specific P210 protein is the product of the bcr/abl hybrid gene, Science, 233, 212, 10.1126/science.3460176 Shtivelman, 1985, Fused transcript of abl and bcr genes in chronic myelogenous leukaemia, Nature, 315, 550, 10.1038/315550a0 Smit, 1998, T cells recognizing leukemic CD34(+) progenitor cells mediate the antileukemic effect of donor lymphocyte infusions for relapsed chronic myeloid leukaemia after allogeneic stem cell transplantation, Proc Natl Acad Sci U S A, 95, 10152, 10.1073/pnas.95.17.10152 Drobyski, 1993, Salvage immunotherapy using donor leukocyte infusions as treatment for relapsed chronic myelogenous leukaemia after allogeneic bone marrow transplantation: efficacy and toxicity of a defined T-cell dose, Blood, 82, 2310, 10.1182/blood.V82.8.2310.2310 Kolb, 1990, Donor leukocyte transfusions for treatment of recurrent chronic myelogenous leukaemia in marrow transplant patients, Blood, 76, 2462, 10.1182/blood.V76.12.2462.2462 Kolb, 1995, Graft-versus-leukaemia effect of donor lymphocyte transfusions in marrow grafted patients, Blood, 86, 2041, 10.1182/blood.V86.5.2041.bloodjournal8652041 Mar, 2011, The controversial role of the Hedgehog pathway in normal and malignant hematopoiesis, Leukemia, 25, 1665, 10.1038/leu.2011.143 Dierks, 2008, Expansion of Bcr-Abl-positive leukemic stem cells is dependent on Hedgehog pathway activation, Cancer Cell, 14, 238, 10.1016/j.ccr.2008.08.003 Irvine, 2009, Combination of the hedgehog pathway inhibitor LDE225 and nilotinib eliminates chronic myeloid leukemia stem and progenitor cells Novartis Pharmaceuticals, 2012 Shah, 2014, Dasatinib plus smoothened (SMO) inhibitor BMS-833923 in chronic myeloid leukemia (CML) with resistance or suboptimal response to a prior tyrosine kinase inhibitor (TKI): phase I study CA180323, Blood, 124, 4539, 10.1182/blood.V124.21.4539.4539 Samanta, 2011, Janus kinase 2 regulates BCR-ABL signaling in chronic myeloid leukemia, Leukemia, 25, 463, 10.1038/leu.2010.287 Bewry, 2008, Stat3 contributes to resistance toward BCR-ABL inhibitors in a bone marrow microenvironment model of drug resistance, Mol Cancer Ther, 7, 3169, 10.1158/1535-7163.MCT-08-0314 Carlesso, 1996, Tyrosyl phosphorylation and DNA binding activity of signal transducers and activators of transcription (STAT) proteins in hematopoietic cell lines trans-formed by BCR/ABL, J Exp Med, 183, 811, 10.1084/jem.183.3.811 Warsch, 2013, JAK of all trades: JAK2-STAT5 as novel therapeutic targets in BCR-ABL1+ chronic myeloid leukemia, Blood, 122, 2167, 10.1182/blood-2013-02-485573 Gallipoli, 2014, JAK2/STAT5 inhibition by nilotinib with ruxolitinib contributes to the elimination of CML CD34+ cells in vitro and in vivo, Blood, 124, 1492, 10.1182/blood-2013-12-545640 Iurlo, 2014, Imatinib and ruxolitinib association: first experience in two patients, Haematologica, 99, 10.3324/haematol.2013.102525 Knoche, 2012, Concomitant JAK2 V617F-positive polycythemia vera and BCR-ABL-positive chronic myelogenous leukemia treated with ruxolitinib and dasatinib, Blood (ASH Annual Meeting Abstracts), 120, 2832 Neviani, 2005, The tumor suppressor PP2A is functionally inactivated in blast crisis CML through the inhibitory activity of the BCR/ABL-regulated SET protein, Cancer Cell, 8, 355, 10.1016/j.ccr.2005.10.015 Perrotti, 2013, Protein phosphatase 2A: a target for anticancer therapy, Lancet Oncol, 14, e229, 10.1016/S1470-2045(12)70558-2 Neviani, 2007, FTY720, a new alternative for treating blast crisis chronic myelogenous leukemia and Philadelphia chromosome-positive acute lymphocytic leukemia, J Clin Invest, 117, 2408, 10.1172/JCI31095 Agarwal, 2014, Antagonism of SET using OP449 enhances the efficacy of tyrosine kinase inhibitors and overcomes drug resistance in myeloid leukemia, Clin cancer Res, 20, 2092, 10.1158/1078-0432.CCR-13-2575 Yoshida, 1992, A novel tetrapeptide, trapoxin, induces phenotypic change from transformed to normal in sis-oncogene-transformed NIH3T3 cells, Jpn J Cancer Res, 83, 324, 10.1111/j.1349-7006.1992.tb00109.x Nimmanapalli, 2003, Histone deacetylase inhibitor LAQ824 both lowers expression and promotes proteasomal degradation of Bcr-Abl and induces apoptosis of imatinib mesylate-sensitive or -refractory chronic myelogenous leukemia-blast crisis cells, Cancer Res, 63, 5126 Guilhot, 2008, A phase II study of oral panobinostat (LBH589) in accelerated phase (AP) or blast crisis (BC) chronic myeloid leukemia (CML) patients resistant to ≥2 BCR-ABL tyrosine kinase inhibitors, Blood, 112, 4263, 10.1182/blood.V112.11.4263.4263 Zaritskey, 2008, A phase II study of oral panobinostat (LBH589) for chronic phase chronic myeloid leukemia (CML) with resistance to ≥2 BCR-ABL tyrosine kinase inhibitors, Blood, 112, 4254, 10.1182/blood.V112.11.4254.4254 Carter, 2005, Inhibition of drug-resistant mutants of ABL, KIT, and EGF receptor kinases, Proc Natl Acad Sci U S A, 102, 11011, 10.1073/pnas.0504952102 Seymour, 2014, A phase 2 study of MK-0457 in patients with BCR-ABL T315I mutant chronic myelogenous leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia, Blood Cancer J, 4, e238, 10.1038/bcj.2014.60 Pietarinen, 2015, Novel drug candidates for blast phase chronic myeloid leukemia from high-throughput drug sensitivity and resistance testing, Blood Cancer J, 5, e309, 10.1038/bcj.2015.30 Gumireddy, 2005, A non-ATP-competitive inhibitor of BCR-ABL overrides imatinib resistance, Proc Natl Acad Sci U S A, 102, 1992, 10.1073/pnas.0408283102 Wu, 2010, ON012380, a putative BCR-ABL kinase inhibitor with a unique mechanism of action in imatinib-resistant cells, Leukemia, 24, 869, 10.1038/leu.2009.300 Heidel, 2012, Genetic and pharmacologic inhibition of beta-catenin tar-gets imatinib-resistant leukemia stem cells in CML, Cell Stem Cell, 10, 412, 10.1016/j.stem.2012.02.017 Kantarjian, 2010, Phase 1 study of INNO-406, a dual Abl/Lyn kinase inhibitor, in Philadelphia chromosome-positive leukemias after imatinib resistance or intolerance, Cancer, 116, 2665 Sellers, 2015 Thiesing, 2000, Efficacy of STI571, an Abl tyrosine kinase inhibitor, in conjunction with other antileukemic agents against Bcr-Abl–positive cells, Blood, 96, 3195, 10.1182/blood.V96.9.3195 Kano, 2001, In vitro cytotoxic effects of a tyrosine kinase inhibitor STI571 in combination with commonly used antileukemic agents, Blood, 97, 1999, 10.1182/blood.V97.7.1999 Guilhot, 2009, Interferon in chronic myeloid leukaemia : past and future, Best Pract Res Clin Haematol, 22, 315, 10.1016/j.beha.2009.10.005 Lipton, 2007, Phase II, randomized, multicenter, comparative study of peginterferon alfa-2a(40KD) versus interferon alfa -2a (Roferon®-A) in patients with treatment-naïve, chronic-phase chronic myelogenous leukemia, Leuk Lymphoma, 48, 497, 10.1080/10428190601175393 Michallet, 2004, Pegylated recombinant interferon alpha-2b vs recombinant interferon alpha-2b for the initial treatment of chronic-phase chronic myelogenous leukemia: a phase III study, Leukemia, 18, 309, 10.1038/sj.leu.2403217 Essers, 2009, IFNalpha activates dormant haematopoietic stem cells in vivo, Nature, 458, 904, 10.1038/nature07815 Sato, 2009, Interferon regulatory factor-2 protects quiescent hematopoietic stem cells from type I interferon-dependent exhaustion, Nat Med, 15, 696, 10.1038/nm.1973 Bhattacharya, 2011, Bcr-abl signals to desensitize chronic myeloid leukemia cells to interferon alpha via accelerating the degradation of its receptor, Blood, 118, 4179, 10.1182/blood-2010-12-325373 Baccarani, 2004, Imatinib an pegylated human recombinant interferon-α2b in early chronic-phase chronic myeloid leukemia, Blood, 104, 4245, 10.1182/blood-2004-03-0826 Palandri, 2008, Front line treatment of Philadelphia positive chronic myeloid leukemia with imatinib and interferon-α: 5 year outcome, Haematologica, 93, 770, 10.3324/haematol.12265 O'Brien, 2001, PEGIntron and STI571 combination evaluation study (PISCES) in chronic phase Chronic Myeloïd Leukemia, Blood, 98, 3512a Preudhomme, 2010, Imatinib plus peginterferon alfa-2a in chronic myeloid leukemia, N Engl J Med, 363, 2511, 10.1056/NEJMoa1004095 Johnson-Ansah, 2013, Tolerability and efficacy of pegylated interferon-α-2a in combination with imatinib for patients with chronic-phase chronic myeloid leukemia, Cancer, 119, 4284, 10.1002/cncr.28328 Guilhot, 2014, Blood, 124, 1793, 10.1182/blood.V124.21.1793.1793 Hehlmann, 2011, Tolerability-adapted imatinib 800 mg versus 400 mg versus. 400 mg + IFN in newly diagnosed chronic myeloid leukemia, J Clin Oncol, 29, 1634, 10.1200/JCO.2010.32.0598 Hehlmann, 2014, Deep molecular response is reached by the majority of patients treated with imatinib, predicts survival, and is achieved more quickly by optimized high-dose imatinib: results from the randomized CML-study IV, J Clin Oncol, 32, 415, 10.1200/JCO.2013.49.9020 Simonsson, 2011, Combination of pegylated IFN-α2b with imatinib increases molecular response rates in patients with low- or intermediate-risk chronic myeloid leukemia, Blood, 118, 3228, 10.1182/blood-2011-02-336685 Cortes, 2011, Cancer, 117, 572, 10.1002/cncr.25438 Nicolini, 2015, Nilotinib and peginterferon alfa-2a for newly diagnosed chronic-phase chronic myeloid leukaemia (NiloPeg): a multicenter, non randomized, open-label phase 2 study, Lancet, 2, e37 Hjorth-Hansen, 2016, Safety and efficacy of the combination of pegylated interferon-α2b and dasatinib in newly diagnosed chronic-phase chronic myeloid leukemia patients, Leukemia, 30, 1853, 10.1038/leu.2016.121 Roy, 2015, Combination of dasatinib and peg-interferon alpha 2b in chronic phase chronic myeloid leukemia (CP-CML) first line: preliminary results of a phase II trial, from the French Intergroup of CML (Fi-LMC), Blood, 126, 134, 10.1182/blood.V126.23.134.134 El Eit, 2013, Effective targeting of chronic myeloid leukemia initiating activity with the combination of arsenic trioxide and interferon alpha, Int J Cancer, 134, 988, 10.1002/ijc.28427 Guilhot, 2008, Immunotherapeutic approaches in chronic myelogenous Leukemia, Leukemia Lymphoma, 49, 629, 10.1080/10428190801927510 Bocchia, 1996, Specific human cellular immunity to BCR-ABL oncogene-derived peptides, Blood, 87, 3587, 10.1182/blood.V87.9.3587.bloodjournal8793587 Norbury, 2000, b3a2 BCR-ABL fusion peptides as targets for cytotoxic T cells in chronic myeloid leukemia, Br J Haematol, 109, 616, 10.1046/j.1365-2141.2000.02090.x Livingston, 1994, Phase I trial of immunological adjuvant QS-21 with a GM2 ganglioside-keyhole limpet haemocyanin conjugate vaccine in patients with malignant melanoma, Vaccine, 12, 1275, 10.1016/S0264-410X(94)80052-2 Cathcart, 2003, A multivalent bcr-abl fusion peptide vaccination trial in patients with chronic myeloid leukemia, Blood, 103, 1037, 10.1182/blood-2003-03-0954 Bocchia, 2005, Effect of a p210 multipeptide vaccine associated with imatinib or interferon in patients with chronic myeloid leukaemia and persistent residual disease: a multicentre observational trial, Lancet, 365, 657, 10.1016/S0140-6736(05)70931-4 Rojas, 2007, Clinical evaluation of BCR-ABL peptide immunisation in chronic myeloid leukaemia: results of the EPIC study, Leukemia, 21, 2287, 10.1038/sj.leu.2404858 Rezvani, 2008, Leukemia-associated antigen-specific T-cell responses following combined PR1 and WT1 peptide vaccination in patients with myeloid malignancies, Blood, 111, 236, 10.1182/blood-2007-08-108241 Quintarelli, 2008, Cytotoxic T lymphocytes directed to the preferentially expressed antigen of melanoma (PRAME) target chronic myeloid leukemia, Blood, 112, 1876, 10.1182/blood-2008-04-150045 Dubrovsky, 2014, A TCR-mimic antibody to WT1 bypasses tyrosine kinase inhibitor resistance in human BCR-ABL1 leukemias, Blood, 123, 3296, 10.1182/blood-2014-01-549022 Goff, 2013, A Pan-BCL2 inhibitor renders bone-marrow-resident human leukemia stem cells sensitive to tyrosine kinase inhibition, Cell Stem Cell, 12, 316, 10.1016/j.stem.2012.12.011 Ko, 2014, The BCL2 inhibitor ABT-199 significantly enhances imatinib induced cell death in chronic myeloid leukemia progenitors, Oncotarget, 5, 9033, 10.18632/oncotarget.1925 Heiblig, 2014, Subcutaneous omacetaxine mepesuccinate in patients with chronic myeloid leukemia in tyrosine kinase inhibitor-resistant patients: review and perspectives, Leu Res, 38, 1145, 10.1016/j.leukres.2014.05.007 Prost, 2015, Erosion of the chronic myeloid leukaemia stem cell pool by PPARγ agonists, Nature, 10.1038/nature15248