Results of high-dose imatinib mesylate in intermediate Sokal risk chronic myeloid leukemia patients in early chronic phase: A phase 2 trial of the GIMEMA CML Working Party

Blood

Volumn 113, Issue 15, 2009, Pages 3428-3434

  Castagnetti, Fausto ^a,i   Palandri, Francesca ^a   Amabile, Marilina ^a   Testoni, Nicoletta ^a   Luatti, Simona ^a   Soverini, Simona ^a   Iacobucci, Ilaria ^b   Breccia, Massimo ^b   Cambrin, Giovanna Rege ^c   Stagno, Fabio ^d   Specchia, Giorgina ^e   Galieni, Piero ^f   Iuliano, Franco ^g   Pane, Fabrizio ^h   Saglio, Giuseppe ^c   Alimena, Giuliana ^b   Martinelli, Giovanni ^a   Baccarani, Michele ^a   Rosti, Gianantonio ^a  

^a UNIVERSITY OF BOLOGNA   (Italy)

^b SAPIENZA UNIVERSITY OF ROME   (Italy)

^c SAN LUIGI GONZAGA HOSPITAL   (Italy)

^d UNIVERSITY OF CATANIA   (Italy)

^e UNIVERSITY OF BARI   (Italy)

^f Mazzoni Hospital   (Italy)

^g Giannettasio Hospital   (Italy)

^h UNIVERSITY OF NAPLES FEDERICO II   (Italy)

ⁱ UNIVERSITY OF BOLOGNA   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

IMATINIB;

ADULT; AGED; ANEMIA; ARTICLE; ASTHENIA; BONE PAIN; CANCER PATIENT; CHRONIC MYELOID LEUKEMIA; CLINICAL EVALUATION; CLINICAL TRIAL; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; CYTOGENETICS; DIARRHEA; DISEASE COURSE; DRUG DOSE REDUCTION; DRUG EFFICACY; DRUG ERUPTION; DRUG MEGADOSE; DRUG RESPONSE; DRUG SAFETY; DRUG TOLERABILITY; DRUG WITHDRAWAL; EDEMA; FATIGUE; FEMALE; FOLLOW UP; GASTROINTESTINAL SYMPTOM; HUMAN; INCIDENCE; LEUKOPENIA; LIVER TOXICITY; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; MUSCLE CRAMP; MYALGIA; NAUSEA; OUTCOME ASSESSMENT; PATIENT COMPLIANCE; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; PROSPECTIVE STUDY; RISK ASSESSMENT; SCORING SYSTEM; THROMBOCYTOPENIA; TREATMENT DURATION; VOMITING;

EID: 65349140717     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2007-08-103499     Document Type: Article

References (64)

1. Sawyers CL. Chronic myeloid leukemia. N Engl J Med. 1999;340:1330-1340.
2. Nowell PC, Hungerford DA. A minute chromosome in human chronic granulocytic leukemia. Science. 1960;132:1497.
3. RowleyJD. A new consistent chromosomal abnormality in chronic myelogenous leukaemia identified by quinacrine fluorescence and Giemsa staining. Nature. 1973;243:290-293.
4. Shtivelman E, Lifshitz B, Gale RP, et al. Fused transcript of abl and bcr genes in chronic myelog- enous leukaemia. Nature. 1985;315:550-554.
5. Lugo TG, Pendergast AM, Muller AJ, et al.Ty-rosine kinase activity and transformation potency of bcr-abl oncogene products. Science. 1990; 247:1079-1082.
6. Druker BJ, Tamura S, Buchdunger E, et al. Effects of a selective inhibitor of the ABL tyrosine kinase on the growth of BCR/ABL positive cells. Nat Med. 1996;2:561-566.
7. Buchdunger E, Zimmermann J, Mett H, et al. Inhibition of the Abl protein-tyrosine kinase in vitro and in vivo by a 2-phenylaminopyrimidine derivative. Cancer Res. 1996;56:100-104.
8. Druker BJ, Talpaz M, Resta DJ, et al. Efficacy and safety of a specific inhibitor of the BCR-ABL ty- rosine kinase in chronic myeloid leukemia. N Engl J Med. 2001;344:1031-1037.
9. Druker BJ, Sawyers CL, Kantarjian H, et al. Activity of a specific inhibitor of the BCR-ABL tyrosine kinase in the blast crisis of chronic myeloid leukemia and acute lymphoblastic leukemia with the Philadelphia chromosome. N Engl J Med. 2001; 344:1038-1042.
10. Kantarjian H, Sawyers C, Hochhaus A, et al. He- matologic and cytogenetic responses to imatinib mesylate in chronic myelogenous leukemia. N Engl J Med. 2002;346:645-52.
11. Sawyers CL, Hochhaus A, Feldman E, et al. Ima- tinib induces hematologic and cytogenetic responses in patients with chronic myeloid leukemia in myeloid blast crisis: results of a phase II study. Blood. 2002;99:3530-3539.
12. Talpaz M, Silver RT, Druker BJ, et al. Imatinib induces durable hematologic and cytogenetic responses in patients with accelerated phase chronic myeloid leukemia: results of a phase 2 study. Blood. 2002;99:1928-1937.
13. Kantarjian HM, O'Brien S, Cortes JE, et al. Treatment of Philadelphia chromosome-positive, accelerated-phase chronic myelogenous leukemia with imatinib mesylate. Clin Cancer Res. 2002;8: 2167-2176.
14. O'Brien SG, Guilhot F, Larson RA, et al. Imatinib compared with interferon and low-dose cytara- bine for newly diagnosed chronic-phase chronic myeloid leukemia. N Engl J Med. 2003;348:994- 1004.
15. Hughes TP, Kaeda J, Branford S, et al. Frequency of major molecular responses to imatinib or interferon alfa plus cytarabine in newlydiag- nosed chronic myeloid leukemia. N Engl J Med. 2003;349:1423-1432.
16. Druker BJ, Guilhot F, O'Brien SG, et al. Five-year follow-up of patients receiving imatinib for chronic myeloid leukemia. N Engl J Med. 2006;355:2408- 2417.
17. Sokal JE, Cox EB, Baccarani M, et al. Prognostic discrimination in "good risk" chronic granulocytic leukemia. Blood. 1984;63:789-799.
18. Hasford J, Pfirmann M, Hehlmann R, et al. Anew prognostic score for survival of patients with chronic myeloid leukemia treated with interferon α. J Natl Cancer Inst. 1998;90:850-858.
19. Hehlmann R, Heimpel H, Hasford J, et al. Randomized comparison of busulfan and hydroxyu- rea in chronic myelogenous leukemia: prolongation of survival by hydroxyurea. The German CML Study Group. Blood. 1993;82:398-407.
20. Allan NC, Richards SM, Shepherd PC. UK Medical Research Council randomised, multicentre trial of interferon-alpha n1 for chronic myeloid leukaemia: improved survival irrespective of cytoge- netic response. Lancet. 1995;345:1392-1397.
21. Italian Cooperative Study Group on Chronic Myeloid Leukemia. Interferon alfa-2a as compared with conventional chemotherapy for the treatment of chronic myeloid leukemia. N Engl J Med. 1994; 330:820-825.
22. Hehlmann R, Heimpel H, Hasford J, et al. Randomized comparison of interferon alpha with busulfan and hydroxyurea in chronic myelog- enous leukemia. Blood. 1994;84:4064-4077.
23. Italian Cooperative Study Group on Chronic My- eloid Leukemia and Italian Group for Bone Marrow Transplantation. Monitoring treatment and survival in chronic myeloid leukemia. J Clin Oncol. 1999;17:1858-1868.
24. Bonifazi F, de Vivo A, Rosti G, et al. Chronic myeloid leukemia and interferon a: a study of complete cytogenetic responders. Blood. 2001;98: 3074-3081.
25. Guilhot F, Chastang C, Michallet M, et al. Inter- feron alfa-2b combined with cytarabine versus interferon alone in chronic myelogenous leukemia. N Engl J Med. 1997;337:223-229.
26. Baccarani M, Rosti G, de Vivo A, et al. A randomized study of interferon-alpha versus interferon- alpha and low-dose arabinosyl cytosine in chronic myeloid leukemia. Blood. 2002;99:1527-1535.
27. Rosti G, Trabacchi E, Bassi S, et al. Risk and early cytogenetic response to imatinib and inter- feron in chronic myeloid leukemia. Haemato-logica. 2003;88:256-259.
28. Baccarani M, Martinelli G, Rosti G, et al. Imatinib and pegylated human recombinant interferon- alpha2b in early chronic-phase chronic myeloid leukemia. Blood. 2004;104:4245-4251.
29. Kantarjian HM, Talpaz M, O'Brien S, et al. Dose escalation of imatinib mesylate can overcome resistance to standard-dose therapy in patients with chronic myelogenous leukemia. Blood. 2003; 101:473-475.
30. Zonder JA, Pemberton P, Brandt H, Mohamed AN, Schiffer CA. The effect of dose increase of imatinib mesylate in patients with chronic or accelerated phase chronic myelogenous leukemia with inadequate hematologic or cytogenetic response to initial treatment. Clin Cancer Res. 2003;9:2092-2097.
31. Cortes J, Giles F, O'Brien, et al. Result of high- dose imatinib mesylate in patients with Philadelphia chromosome-positive chronic myeloid leukemia after failure of interferon-alpha. Blood. 2003; 102:83-86.
32. Kantarjian H, Talpaz M, O'Brien S, et al. High- dose imatinib mesylate therapy in newly diagnosed Philadelphia chromosome-positive chronic phase chronic myeloid leukemia. Blood. 2004; 103:2873-2878.
33. Aoki T, Kantarjian H, O'Brien S, et al. High-dose (HD) imatinib provides better responses in patients with untreated early chronic phase (CP) CML [abstract]. Blood. 2006;108:2143a.
34. Cortes J, Giles F, Salvado AJ, et al. Molecular responses in newly diagnosed chronic myelocytic leukemia (CML) patients treated with 800 mg imatinib daily: an update from the RIGHT Trial Study Group [abstract]. Blood. 2006;108:2149a.
35. Hughes TP, Branford S, White DL, et al. Impact of early dose intensity on cytogenetic and molecular responses in chronic-phase CML patients receiving 600 mg/day of imatinib as initial therapy Blood. 2008;112:3965-3973.
36. Gorre ME, Mohammed M, Ellwood K, et al. Clinical resistance to STI-571 cancer therapy caused by BCR-ABL gene mutation or amplification. Science. 2001;293:876-880.
37. Von Bubnoff N, Schneller F, Peschel C, et al. BCR-ABL gene mutations in relation to clinical resistance of Philadelphia-chromosome-positive leukaemia to STI571: a prospective study. Lancet. 2002;359:487-491.
38. Shah NP, Nicoll JM, Nagar B, et al. Multiple BCR-ABL kinase domain mutations confer polyclonal resistance to the tyrosine kinase inhibitor imatinib (STI571) in chronic phase and blast crisis chronic myeloid leukemia. Cancer Cell. 2002;2:117-125.
39. Corbin AS, La Rosee P, Stoffregen EP, et al. Several Bcr-Abl kinase domain mutants associated with imatinib mesylate resistance remain sensitive to imatinib. Blood. 2003;101:4611-4614.
40. O'Hare T, Eide CA, Deininger MW. Bcr-Abl kinase domain mutations, drug resistance and the road to a cure of chronic myeloid leukemia. Blood. 2007;110:2242-2249.
41. Peng B, Hayes M, Resta D, et al. Pharmacokinet- ics and pharmacodynamics of imatinib in a phase I trial with chronic myeloid leukemia patients. J Clin Oncol. 2004;22:935-942.
42. Larson RA, Druker BJ, Guilhot F, et al. Correlation of pharmacokinetic data with cytogenetic and molecular response in newly diagnosed patients with chronic myeloid leukemia in chronic phase (CML-CP) treated with imatinib: an analysis of IRIS Study Data [abstract]. Blood. 2006;108:429a.
43. Haiat S, Decleves X, Mittaine B, et al. Determination of imatinib plasma levels by high performance liquid chromatography (HPLC): evaluation of pharmacokinetic variability and haematological consequences [abstract]. Blood. 2006;108:1375a.
44. Picard S, Titier K, Etienne G, et al. Through ima- tinib plasma levels are associated with both cyto- genetic and molecular responses to standarddose imatinib in chronic myeloid leukemia. Blood. 2007;109:3496-3499.
45. Gabert J, Beillard E, van der Velden VH, et al. Standardization and quality control studies of "real-time" quantitative reverse transcriptase polymerase chain reaction of fusion gene transcripts for residual disease detection in leukemia: a Europe Against Cancer program. Leukemia. 2003;17:2318-2357.
46. van der Velden VH, Hochhaus A, Cazzaniga G, et al. Detection of minimal residual disease in hema- tologic malignancies by real-time quantitative PCR: principles, approaches, and laboratory aspects. Leukemia. 2003;17:1013-1034.
47. van Dongen JJ, Macintyre EA, Gabert JA, et al. Standardized RT-PCR analysis of fusion gene transcripts from chromosome aberrations in acute leukemia for detection of minimal residual disease. Report of the BIOMED-1 Concerted Action: investigation of minimal residual disease in acute leukemia. Leukemia. 1999;13:1901-1928.
48. Hughes T, Deininger M, Hochhaus A, et al. Monitoring CML patients responding to treatment with tyrosine kinase inhibitors: review and recommendations for harmonizing current methodology for detecting BCR-ABL transcripts and kinase domain mutations and for expressing results. Blood. 2006;108:28-37.
49. Branford S, Fletcher L, Cross NC, et al. Desirable performance characteristics for BCR-ABL measurement on an international reporting scale to allow consistent interpretation of individual patient response and comparison of response rates between clinical trials. Blood. 2008;112:3330- 3338.
50. Fleming TR. One-sample multiple testing procedure for phase II clinical trials. Biometrics. 1982; 38:143-151.
51. Ottmann OG, Druker BJ, Sawyers CL, et al. A phase 2 study of imatinib in patients with relapsed or refractory Philadelphia chromosome-positive acute lymphoid leukemias. Blood. 2002;100: 1965-1971.
52. Thomas DA, Faderl S, Cortes J, et al. Treatment of Philadelphia chromosome-positive acute lym- phocytic leukemia with hyper-CVAD and imatinib mesylate. Blood. 2004;103:4396-4407.
53. Towatari M, Yanada M, Usui N, et al. Combination of intensive chemotherapy and imatinib can rapidly induce high-quality complete remission for a majority of patients with newly diagnosed BCR- ABL-positive acute lymphoblastic leukemia. Blood. 2004;104:3507-3512.
54. Yanada M, Takeuchi J, Sugiura I, et al. High complete remission rate and promising outcome by combination of imatinib and chemotherapy for newly diagnosed BCR-ABL-positive acute lym- phoblastic leukemia: a phase II study by the Japan Adult Leukemia Study Group. J Clin Oncol. 2006;24:460-466.
55. Ottmann OG, Wassmann B, Pfeifer H, et al. Ima- tinib compared with chemotherapy as front-line treatment of elderly patients with Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). Cancer. 2007;109:2068-2076.
56. Kantarjian HM, O'Brien S, Cortes JE, et al. Complete cytogenetic and molecular responses to in- terferon-alpha-based therapy for chronic myelog- enous leukemia are associated with excellent long-term prognosis. Cancer. 2003;97:1033-1041.
57. Rosti G, Testoni N, Martinelli G, et al. The cytoge- netic response as a surrogate marker of survival. Semin Hematol. 2003;40:56-61.
58. Merx K, Muller MC, Kreil S, et al. Early reduction of BCR-ABL mRNAtranscript levels predicts cyto- genetic response in chronic phase CML patients treated with imatinib after failure of interferon alpha. Leukemia. 2002;16:1579-1583.
59. Wang L, Pearson K, Ferguson JE, et al. The early molecular response to imatinib predicts cytoge- netic and clinical outcome in chronic myeloid leukaemia. Br J Haematol. 2003;120:990-999.
60. Press RD, Love Z,Tronnes AA, et al. BCR-ABL mRNA levels at and after the time of a complete cytogenetic response (CCR) predict the duration of CCR in imatinib mesylate-treated patients with CML. Blood. 2006;107:4250-4256.
61. Cortes J, Talpaz M, O'Brien S, et al. Molecular responses in patients with chronic myelogenous leukemia in chronic phase treated with imatinib mesylate. Clin Cancer Res. 2005;11:3425-3432.
62. Iacobucci I, Rosti G, Amabile M, et al. Comparison between patients with Philadelphia-positive chronic phase chronic myeloid leukemia who obtained a complete cytogenetic response within 1 year of imatinib therapy and those who achieved such a response after 12 months of treatment. J Clin Oncol. 2006;24:454-459.
63. Baccarani M, Saglio G, Goldman J, et al. Evolving concepts in the management of chronic my- eloid leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood. 2006;108:1809-1820.
64. Baccarani M, Castagnetti F, Simonsson B, et al. Cytogenetic and molecular response to imatinib in high risk (Sokal) chronic myeloid leukemia (CML): results of a European Leukemianet Prospective Study comparing 400 mg and 800 mg front-line [abstract]. Blood. 2008;112:185a.