Acute Leukemias - Republican Scientific Medical Library

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16 Chapter 1 · Therapy of AML treatment. Thus, there may be more reason to protect against a false negative than a false positive result. At the least, it might be reasonable to specify type 1 and type 2 errors of 20% each. Such a change together with an interest in detecting only larger, more clinically meaningful, differences would enable fewer patients to be entered per treatment arm, thus allowing more treatments to be studied [119]. While such trials would be nominally “underpowered,” this ignores the false negative rate inherent in the selection of which, of many, investigational regimens to study. For example, if there are three potential regimens that could be investigated and if preclinical rationale is an imperfect predictor of clinical results, limiting investigation to one regimen potentially entails a false negative rate of 67%. In particular, the most egregious false negative results when a treatment is not studied at all [119]. This is problematic since, although by no means exhaustive, Table 1.5 lists nine different classes of drugs undergoing investigation. Use of each class rests on a presumably sound preclinical rationale, and each class may contain many drugs. Furthermore, there may eventually be combinations of drugs across classes. Patients with poor performance status or other factors likely to increase TRD are often ineligible for clinical trials, although the development of new NCT agents might particularly benefit such patients. Even when eligible, patients may be excluded because it is believed they will do poorly. Although the extent of this subtler form of selection bias is unknown, papers reporting results of clinical trials only infrequently note that consecutive patients were entered and treated. Obviously the greater the extent of such bias the less likely it becomes that results will be reproducible in a more representative group of patients. References 1. Freireich EJ, Gehan EA, Sulma D, et al. (1961) The effect of chemotherapy on acute leukemia in the human. J Chron Dis 14: 593–608 2. Jaffe ES, Harris NL, Stein H, Vardiman JW (eds) (2001) World Health Organization Classification of Tumours. Pathology and Genetics of Tumours of Haematopoietic and Lymphoid Tissues. IARC Press, Lyon 3. Bennett JM, Catovsky D, Daniel MT, Flandrin G, Galton DA, Gralnick HR (1985) Proposed revised criteria for the classification of acute myeloid leukemia. A report of the French-American-British Cooperative Group. Ann Intern Med 103:620–625 4. de Lima M, Strom S, Keating M, et al. (1997) Implications of potential cure in acute myelogenous leukemia: Development of subsequent cancer and return to work. Blood 90:4719–4724 5. Buchner T, Urbanitz D, Hiddemann W, et al. (1985) Intensified induction and consolidation with or without maintenance chemotherapy for acute myeloid leukemia (AML): Two multicenter studies of the German AML Cooperative Group. J Clin Oncol 3:1583–1589 6. Buchner T, Hiddemann W, Berdel B (2001) Requestioning the role of prolonged maintenance chemotherapy in AML: A randomized trial by the German AML Cooperative Group. Blood 98:462a 7. Goldstone AH, Burnett AK, Wheatley K, Smith AG, Hutchinson RM, Clark RE (2001) Attempts to improve treatment outcomes in AML in older patients: The results of the United Kingdom Medical Research Council AML11 trial. Blood 98:1302–1311 8. Charlson ME, Pompei P, Ales KL, et al. (1987) A new method of classifying prognostic comorbidity in longitudinal studies: Development and validation. J Chr Dis 40:373–383 9. Sorror MI, Maris MB, Storer B, et al. (2004) Comparing morbidity and mortality of HLA-matched unrelated donor hematopoietic cell transplantation after nonmyeloablative and myeloablative conditioning: Influence of pretransplantation comorbidities. Blood 104:961–968 10. Grimwade D, Walker H, Oliver F, et al. (1998) The importance of diagnostic cytogenetics on outcome in AML: Analysis of 1,612 patients entered into the MRC AML 10 trial. The Medical Research Council Adult and Children’s Leukemia Working Parties. Blood 92:2322–2333 11. Grimwade D, Walker H, Harrison G, et al. (2001) The predictive value of hierarchical cytogenetic classification in older adults with acute myeloid leukemia: Analysis of 1065 patients entered into the United Kingdom Medical Research Council AML 11 trial. Blood 98:1312–1320 12. Slovak ML, Kopecky KJ, Cassileth PA, et al. (2000) Karyotypic analysis predicts outcome of preremission and postremission therapy in adult acute myeloid leukemia: A Southwest Oncology Group/ Eastern Cooperative Oncology Group Study. Blood 96:4075–4083 13. Downing JR (2003) The core-binding factor leukemias: Lessons learned from murine models. Curr Opin Genet Dev 13:48–54 14. Bloomfield CD, Lawrence D, Byrd JC, et al. (1998) Frequency of prolonged remission duration after high-dose cytarabine intensification in acute myeloid leukemia varies by cytogenetic subtype. Cancer Res 58:4173–4179 15. Goldstone AH, Burnett AK, Avivi I, et al. (2002) Secondary AML has a worse outcome than de novo AML even taking into account cytogenetics and age. AML 10,11,12 MRC trials. Blood 100:88a (abstr) 16. Nguyen S, Leblanc T, Fenaux P, et al. (2002) A white blood cell index as the main prognostic factor in t(8;21) acute myeloid leukemia (AML): A survey of 161 cases from the French AML Intergroup. Blood 99:3517–3523 17. Schlenk RF, Benner A, Krauter J, et al. (2004) Individual patient data-based meta-analysis of patients aged 16–60 years with core binding factor acute myeloid leukemia: A survey of the German Acute Myeloid Leukemia Intergroup. J Clin Oncol 15:3741–3750 18. Marcucci G, Mrózek K, Ruppero AS, et al. (2005) Prognostic factors and outcome of core binding factor acute myeloid leukemia patients with t(8;21) differ from those of patients with inv(16): A Cancer and Leukemia Group B Study. J Clin Oncol 23:5705–5717
a References 17 19. Care RS, Valk PJ, Goodeve AC, et al. (2003) Incidence and prognosis of c-KIT and FLT3 mutations in core binding factor (CBF) acute myeloid leukaemias. Br J Haematol 121:775–777 20. Carioli R, Beghini A, Grillo G, et al. (2006) Prognostic impact of c- KIT mutations in core binding factor leukemias: An Italian retrospective study. Blood 107:3463–3468 21. Schnittger S, Kohl TM, Haferlach T, et al. (2006) KIT-D816 mutations in AML1-ETO positive AML are associated with impaired event-free and overall survival. Blood 107:1791–1799 22. Paschka P, Marcucci G, Ruppert A, et al. (2006) Mutations of KIT tyrosine kinase (TK) gene predict relapse in adult patients (pts) with core binding factor acute myeloid leukemia (CBF AML): A cancer and leukemia group B (CALGB) study. Proc Am Soc Clin Oncol 24:1 (abstr) 23. Yokota S, Kiyoi H, Nakao M, et al. (1997) Internal tandem duplication of the FLT3 gene is preferentially seen in acute myeloid leukemia and myelodysplastic syndrome among various hematological malignancies: A study on a large series of patients and cell lines. Leukemia 10:1605–1609 24. Kottaridis PD, Gale RE, Frew ME, et al. (2001) 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 98:1752–1759 25. Fröhling S, Schlenk RF, Breitruck J, et al. (2002) Prognostic significance of activating FLT3 mutations in younger adults (16 to 60 years) with acute myeloid leukemia and normal cytogenetics: A study of the AML Study Group Ulm. Blood 100:4372–4380 26. Thiede C, Steudel C, Mohr B, et al. (2002) Analysis of FLT3-activating mutations in 979 patients with acute myelogenous leukemia: Association with FAB subtypes and identification of subgroups with poor prognosis. Blood 99:4326–4335 27. Whitman SP, Archer KJ, Feng L, et al. (2001) Absence of the wildtype allele predicts poor prognosis in adult de novo acute myeloid leukemia with normal cytogenetics and the internal tandem duplication of FLT3: A cancer and leukemia group B study. Cancer Res 61:7233–7239 28. Yamamoto Y, Kiyoi H, Nakano Y, et al. (2001) Activating mutation of D835 within the activation loop of FLT3 in human hematologic malignancies. Blood 97:2434–2439 29. Fröhling S, Scholl C, Gilliland DG, Levine RL (2005) Genetics of myeloid malignancies7Pathogenetic and clinical implications. J Clin Oncol 23:6285–6295 30. Yanada M, Matsuo K, Suzuki T, Kiyoi H, Naoe T (2005) Prognostic significance of FLT3 internal tandem duplication and tyrosine kinase domain mutations for acute myeloid leukemia: A meta-analysis. Leukemia 19:1345–1349 31. Mead A, Linch D, Hills R, et al. (2005) Favorable prognosis associated with FLT3 tyrosine kinase domain mutations in AML in contrast to the adverse outcome associated with internal tandem duplications. Blood 106:334 (abstr) 32. Falini B, Mecucci C, Tiacci E, et al. (2005) Gimema Acute Leukemia Working Party. Cytoplasmic nucleophosmin in acute myelogenous leukemia with a normal karyotype. N Engl J Med 352:254–266 33. Döhner K, Schlenk RF, Habdank M, et al. (2005) Mutant nucleophosmin (NPM1) predicts favorable prognosis in younger adults with acute myeloid leukemia and normal cytogenetics – Interaction with other gene mutations. Blood 106:3740–3746 34. Verhaak RGW, Goudswaard CS, van Putten W, et al. (2005) Mutations in nucleophosmin NPM1 in acute myeloid leukemia (AML): Association with other genetic abnormalities and previously established gene expression signatures and their favorable prognostic significance. Blood 106:3747–3754 35. Schnittger S, Schoch C, Kern W, et al. (2005) Nucleophosmin gene mutations are predictors of favorable prognosis in acute myelogenous leukemia with a normal karyotype. Blood 106: 3733–3739 36. Suzuki T, Kiyoi H, Ozeki K, et al. (2005) Clinical characteristics and prognostic implications of NPM1 mutations in acute myeloid leukemia. Blood 106:2854–2861 37. Bowen DT, Frew ME, Hills R, et al. (2005) RAS mutation in acute myeloid leukemia is associated with distinct cytogenetic subgroups but does not influence outcome in patients
Page 1 and 2: E.H. Estey · S.H. Faderl · H. M.
Page 3 and 4: E. H. Estey Department of Leukemia
Page 5 and 6: VI Table of Contents 14 Philadelphi
Page 7 and 8: VIII Contributors S. H. Faderl Depa
Page 9 and 10: X Contributors D. Wartenberg Depart
Page 11 and 12: Therapy of AML Elihu Estey Contents
Page 13 and 14: a 1.2 · Standard Therapy 3 Table 1
Page 15 and 16: a 1.2 · Standard Therapy 5 Table 1
Page 17 and 18: a 1.2 · Standard Therapy 7 tween G
Page 19 and 20: a 1.2 · Standard Therapy 9 Fig. 1.
Page 21 and 22: a 1.2 · Standard Therapy 11 of tre
Page 23 and 24: a 1.2 · Standard Therapy 13 Table
Page 25: a 1.2 · Standard Therapy 15 permit
Page 29 and 30: a References 19 6-thioguanine in th
Page 31 and 32: Acute Myeloid Leukemia: Epidemiolog
Page 33 and 34: a 3.1 · Epidemiology 49 3.1.4 Gend
Page 35 and 36: a 3.2 · Etiology 51 part of the in
Page 37 and 38: a 3.2 · Etiology 53 for the develo
Page 39 and 40: a References 55 38. Crane MM, Stom
Page 41 and 42: Relapsed and Refractory Acute Myelo
Page 43 and 44: a 4.2 · Prognostic Factors in Pati
Page 45 and 46: a 4.3 · Treatment of Relapsed and
Page 47 and 48: a 4.4 · Hematopoietic Stem Cell Tr
Page 49 and 50: a 4.6 · Gemtuzumab Ozogamicin 65 T
Page 51 and 52: a 4.7 · Relapsed and Refractory Ac
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Page 55 and 56: a References 71 The ability of immu
Page 57 and 58: a References 73 39. Vogler WR, McCa
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Page 61 and 62: Part II - Acute Lymphoblastic Leuke
Page 63 and 64: 78 Chapter 5 · Acute Lymphoblastic
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92 Chapter 5 · Acute Lymphoblastic
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Molecular Biology and Genetics Meir
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a 6.3 · Structural Aberrations 97
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a 6.3 · Structural Aberrations 99
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a 6.5 · Molecular Aberrations 101
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a References 103 clear that homozyg
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a References 105 53. Chim CS, Tam C
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a References 107 122. Lennard L, Li
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Diagnosis of Acute Lymphoblastic Le
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a 8.3 · Cytochemistry and Immunoph
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a 8.5 · Cytogenetic and Molecular
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a 8.5 · Cytogenetic and Molecular
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a References 127 including the reti
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a References 129 47. Kita K, Shirak
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Acute Lymphoblastic Leukemia: Clini
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a 7.3 · Diagnosis 111 or neoplasti
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a 7.3 · Diagnosis 113 Fig. 7.2. Hi
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a 7.3 · Diagnosis 115 The vast maj
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a References 117 dren: Biologic bas
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General Approach to the Therapy of
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a 9.3 · New Agents 133 dose methot
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a References 135 4. Gökbuget N, Ho
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138 Chapter 10 · Recent Clinical T
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146 Chapter 11 · Conventional Ther
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ALL Therapy: Review of the MD Ander
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a 12.2 · The Hyper-CVAD Regimen in
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a 12.3 · Subset-Specific Approache
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Treatment of Adult ALL According to
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a 13.2 · Therapy for Younger (15-6
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a 13.3 · Therapy of Ph/BCR-ABL-Pos
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a 13.5 · Prognostic Factors 173 13
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a References 175 Only patients with
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Philadelphia Chromosome-Positive Ac
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a 14.2 · Molecular Biology of the
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a 14.3 · Treatment of Ph+ ALL 181
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a 14.4 · Hematopoietic Stem Cell T
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a References 185 2. Kurzrock R, Sht
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a References 187 56. Mori T, Manabe
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a References 189 acute lymphoblasti
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192 Chapter 15 · Burkitt’s Acute
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204 Chapter 16 · Treatment of Lymp
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216 Chapter 17 · The Role of Allog
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230 Chapter 18 · The Role of Autol
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238 Chapter 19 · Novel Therapies i
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248 Chapter 20 · Minimal Residual
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264 Chapter 21 · Central Nervous S
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276 Chapter 22 · Relapsed Acute Ly
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278 Chapter 22 · Relapsed Acute Ly
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Emergencies in Acute Lymphoblastic
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a 23.5 · Hyperleukocytosis 283 LA
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a 23.9 · Neurological Complication
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a References 287 29. Hingorani AD,
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Subject Index A aberrant methylatio
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a Subject Index 291 CREB, see enhan
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a Subject Index 293 MUD, see matche
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