Curing myeloma at last: Defining criteria and providing the evidence

Blood

Volumn 124, Issue 20, 2014, Pages 3043-3051

  Barlogie, Bart ^a   Mitchell, Alan ^b   Van Rhee, Frits ^a   Epstein, Joshua ^a   Morgan, Gareth J ^a   Crowley, John ^b  

^a UNIVERSITY OF ARKANSAS FOR MEDICAL SCIENCES   (United States)

^b USA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTINEOPLASTIC AGENT; THALIDOMIDE; TRANSCRIPTOME;

APOPTOSIS; CANCER CHEMOTHERAPY; CANCER RECURRENCE; CANCER SURVIVAL; DISEASE MARKER; FOLLOW UP; GENE EXPRESSION PROFILING; HEREDITY; HUMAN; INTRATUMORAL HETEROGENEITY; MINIMAL RESIDUAL DISEASE; MULTIPLE MYELOMA; PHASE 3 CLINICAL TRIAL (TOPIC); PLASMA CELL; PROGRESSION FREE SURVIVAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; RISK FACTOR; TREATMENT RESPONSE; CLINICAL TRIAL (TOPIC); DISEASE FREE SURVIVAL; GENETICS; KAPLAN MEIER METHOD; STATISTICAL MODEL; TREATMENT OUTCOME;

CLINICAL TRIALS AS TOPIC; DISEASE-FREE SURVIVAL; FOLLOW-UP STUDIES; HUMANS; KAPLAN-MEIER ESTIMATE; MODELS, STATISTICAL; MULTIPLE MYELOMA; NEOPLASM, RESIDUAL; TRANSCRIPTOME; TREATMENT OUTCOME;

EID: 84911913452     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2014-07-552059     Document Type: Review

References (68)

1. Gahrton G, Tura S, Ljungman P, et al; European Group for Bone Marrow Transplantation. Allogeneic bone marrow transplantation in multiple myeloma. N Engl J Med. 1991;325(18):1267-1273.
2. Barlogie B. Toward a cure for multiple myeloma? N Engl J Med. 1991;325(18):1304-1306.
3. Fassas A, Shaughnessy J, Barlogie B. Cure of myeloma: hype or reality? Bone Marrow Transplant. 2005;35(3):215-224.
4. Martinez-Lopez J, Blade J, Mateos MV, et al; Grupo Español de MM; Programa para el Estudio de la Terapé utica en Hemopatía Maligna. Long-term prognostic significance of response in multiple myeloma after stem cell transplantation. Blood. 2011;118(3):529-534.
5. Martino M, Postorino M, Gallo GA, et al. Long-term results in multiple myeloma after high-dose melphalan and autologous transplantation according to response categories in the era of old drugs. Clin Lymphoma Myeloma Leuk. 2014;14(2):148-154.
6. Brioli A, Tacchetti P, Zamagni E, Cavo M. Maintenance therapy in newly diagnosed multiple myeloma: current recommendations. Expert Rev Anticancer Ther. 2014;14(5):581-594.
7. Alexanian R, Delasalle K, Wang M, Thomas S, Weber D. Curability of multiple myeloma. Bone Marrow Res. 2012;2012:916479.
8. Roschewski M, Korde N, Wu SP, Landgren O. Pursuing the curative blueprint for early myeloma. Blood. 2013;122(4):486-490.
9. Chapman MA, Lawrence MS, Keats JJ, et al. Initial genome sequencing and analysis of multiple myeloma. Nature. 2011;471(7339):467-472.
10. Morgan GJ, Walker BA, Davies FE. The genetic architecture of multiple myeloma. Nat Rev Cancer. 2012;12(5):335-348.
11. Kyle RA, Therneau TM, Rajkumar SV, et al. A long-term study of prognosis in monoclonal gammopathy of undetermined significance. N Engl J Med. 2002;21:346(8):564-9.
12. Kyle RA, Remstein ED, Therneau TM, et al. Clinical course and prognosis of smoldering (asymptomatic) multiple myeloma. N Engl J Med. 2007;356(25):2582-2590.
13. International Myeloma Working Group. Criteria for the classification of monoclonal gammopathies, multiple myeloma and related disorders: a report of the International Myeloma Working Group. Br J Haematol. 2003;121(5):749-757.
14. Cavo M, Tacchetti P, Patriarca F, et al; GIMEMA Italian Myeloma Network. Bortezomib with thalidomide plus dexamethasone compared with thalidomide plus dexamethasone as induction therapy before, and consolidation therapy after, double autologous stem-cell transplantation in newly diagnosed multiple myeloma: a randomised phase 3 study. Lancet. 2010;376(9758): 2075-2085.
15. Harousseau JL, Attal M, Avet-Loiseau H. The role of complete response in multiple myeloma. Blood. 2009;114(15):3139-3146.
16. Lonial S, Anderson KC. Association of response endpoints with survival outcomes in multiple myeloma. Leukemia. 2014;28(2):258-268.
17. Chanan-Khan AA, Giralt S. Importance of achieving a complete response in multiple myeloma, and the impact of novel agents. J Clin Oncol. 2010;28(15):2612-2624.
18. Jakubowiak AJ, Dytfeld D, Griffith KA, et al. A phase 1/2 study of carfilzomib in combination with lenalidomide and low-dose dexamethasone as a frontline treatment for multiple myeloma. Blood. 2012;120(9):1801-1809.
19. Paiva B, Vidriales MB, Cerveró J, et al; GEM (Grupo Español de MM)/PETHEMA (Programa para el Estudio de la Terapéutica en Hemopatías Malignas) Cooperative Study Groups. Multiparameter flow cytometric remission is the most relevant prognostic factor for multiple myeloma patients who undergo autologous stem cell transplantation. Blood. 2008;112(10): 4017-4023.
20. Barlogie B, Epstein J, Shaughnessy JD Jr. Going with the flow, and beyond, in myeloma. Blood. 2008;112(10):3917-3918.
21. Merlo LM, Pepper JW, Reid BJ, Maley CC. Cancer as an evolutionary and ecological process. Nat Rev Cancer. 2006;6(12):924-935.
22. Walker BA, Wardell CP, Melchor L, et al. Intraclonal heterogeneity is a critical early event in the development of myeloma and precedes the development of clinical symptoms. Leukemia. 2014;28(2):384-390.
23. Melchor L, Brioli A, Wardell CP, et al. Single-cell genetic analysis reveals the composition of initiating clones and phylogenetic patterns of branching and parallel evolution in myeloma. Leukemia. 2014;28(8):1705-1715.
24. Zhao S, Choi M, Heuck C, et al. Serial exome analysis of disease progression in premalignant gammopathies. Leukemia. 2014;28(7): 1548-1552.
25. Walker BA, Leone PE, Chiecchio L, et al. A compendium of myeloma-associated chromosomal copy number abnormalities and their prognostic value. Blood. 2010;116(15): e56-e65.
26. Keats JJ, Chesi M, Egan JB, et al. Clonal competition with alternating dominance in multiple myeloma. Blood. 2012;120(5):1067-1076.
27. Walker R, Barlogie B, Haessler J, et al. Magnetic resonance imaging in multiple myeloma: diagnostic and clinical implications. J Clin Oncol. 2007;25(9):1121-1128.
28. Paiva B, Gutiérrez NC, Rosiñol L, et al; PETHEMA/GEM (Programa para el Estudio de la Terapéutica en Hemopatías Malignas/Grupo Español de Mieloma) Cooperative Study Groups. High-risk cytogenetics and persistent minimal residual disease by multiparameter flow cytometry predict unsustained complete response after autologous stem cell transplantation in multiple myeloma. Blood. 2012;119(3):687-691.
29. Bartel TB, Haessler J, Brown TL, et al. F18-fluorodeoxyglucose positron emission tomography in the context of other imaging techniques and prognostic factors in multiple myeloma. Blood. 2009;114(10):2068-2076.
30. Zamagni E, Patriarca F, Nanni C, et al. Prognostic relevance of 18-F FDG PET/CT in newly diagnosed multiple myeloma patients treated with up-front autologous transplantation. Blood. 2011; 118(23):5989-5995.
31. Usmani SZ, Mitchell A, Waheed S, et al. Prognostic implications of serial 18-fluoro-deoxyglucose emission tomography in multiple myeloma treated with total therapy 3. Blood. 2013;121(10):1819-1823.
32. Lohr JG, Stojanov P, Carter SL, et al; Multiple Myeloma Research Consortium. Widespread genetic heterogeneity in multiple myeloma: implications for targeted therapy. Cancer Cell. 2014;25(1):91-101.
33. Landau DA, Carter SL, Getz G, Wu CJ. Clonal evolution in hematological malignancies and therapeutic implications. Leukemia. 2014;28(1): 34-43.
34. Pui CH, Campana D, Pei D, et al. Treating childhood acute lymphoblastic leukemia without cranial irradiation. N Engl J Med. 2009;360(26): 2730-2741.
35. Barlogie B, Jagannath S, Vesole DH, et al. Superiority of tandem autologous transplantation over standard therapy for previously untreated multiple myeloma. Blood. 1997;89(3):789-793.
36. Singhal S, Mehta J, Desikan R, et al. Antitumor activity of thalidomide in refractory multiple myeloma. N Engl J Med. 1999;341(21): 1565-1571.
37. Barlogie B, Tricot G, Anaissie E, et al. Thalidomide and hematopoietic-cell transplantation for multiple myeloma. N Engl J Med. 2006;354(10):1021-1030.
38. Richardson PG, Barlogie B, Berenson J, et al. A phase 2 study of bortezomib in relapsed, refractory myeloma. N Engl J Med. 2003;348(26): 2609-2617.
39. Barlogie B, Anaissie E, van Rhee F, et al. Incorporating bortezomib into upfront treatment for multiple myeloma: early results of total therapy 3. Br J Haematol. 2007;138(2):176-185.
40. van Rhee F, Bolejack V, Hollmig K, et al. High serum-free light chain levels and their rapid reduction in response to therapy define an aggressive multiple myeloma subtype with poor prognosis. Blood. 2007;110(3):827-832.
41. van Rhee F, Szymonifka J, Anaissie E, et al. Total Therapy 3 for multiple myeloma: prognostic implications of cumulative dosing and premature discontinuation of VTD maintenance components, bortezomib, thalidomide, and dexamethasone, relevant to all phases of therapy. Blood. 2010; 116(8):1220-1227.
42. Pineda-Roman M, Zangari M, Haessler J, et al. Sustained complete remissions in multiple myeloma linked to bortezomib in total therapy 3: comparison with total therapy 2. Br J Haematol. 2008;140(6):625-634.
43. Barlogie B, Attal M, Crowley J, et al. Long-term follow-up of autotransplantation trials for multiple myeloma: update of protocols conducted by the intergroupe francophone du myelome, southwest oncology group, and university of arkansas for medical sciences. J Clin Oncol. 2010;28(7): 1209-1214.
44. Ederer F, Axtell LM, Cutler SJ. The relative survival rate: a statistical methodology. Natl Cancer Inst Monogr. 1961;6:101-121.
45. Farewell VT. The use of mixture models for the analysis of survival data with long-term survivors. Biometrics. 1982;38(4):1041-1046.
46. Lahuerta JJ, Mateos MV, Martínez-López J, et al. Influence of pre-and post-transplantation responses on outcome of patients with multiple myeloma: sequential improvement of response and achievement of complete response are associated with longer survival. J Clin Oncol. 2008;26(35):5775-5782.
47. Moreau P, Attal M, Pégourié B, et al; IFM 2005-01 study investigators. Achievement of VGPR to induction therapy is an important prognostic factor for longer PFS in the IFM 2005-01 trial. Blood. 2011;117(11):3041-3044.
48. Ladetto M, Pagliano G, Ferrero S, et al. Major shrinking of residual tumor cell burden and achievement of molecular remissions in myeloma patients undergoing post-transplant consolidation with bortezomib, thalidomide and dexamethasone a qualitative and quantitative PCR study [abstract]. Blood. 2008;112(11). Abstract 1261.
49. Palumbo A, Gay A, Musto P, et al. Continuous treatment (CT) versus fixed duration of therapy (FDT) in newly diagnosed myeloma patients: PFS1, PFS2, OS endpoints. [abstract] J Clin Oncol. 2014;32:8515a.
50. Barlogie B, van Rhee F, Shaughnessy JD Jr, Anaissie E, Crowley J. Making progress in treating multiple myeloma with total therapies: issue of complete remission and more. [letter] Leukemia. 2008;22(8):1633-1636.
51. Barlogie B, Anaissie E, van Rhee F, et al. The Arkansas approach to therapy of patients with multiple myeloma. Best Pract Res Clin Haematol. 2007;20(4):761-781.
52. Barlogie B, Tricot G. Complete response in myeloma: a Trojan horse? Blood. 2006;108(7): 2134.
53. Pineda-Roman M, Bolejack V, Arzoumanian V, et al. Complete response in myeloma extends survival without, but not with history of prior monoclonal gammopathy of undetermined significance or smouldering disease. Br J Haematol. 2007;136(3):393-399.
54. Zhan F, Barlogie B, Arzoumanian V, et al. Gene-expression signature of benign monoclonal gammopathy evident in multiple myeloma is linked to good prognosis. Blood. 2007;109(4): 1692-1700.
55. Paiva B, Vidriales MB, Rosiñol L, et al; Grupo Español de MM/Programa para el Estudio de la Terapéutica en Hemopatías Malignas Cooperative Study Group. A multiparameter flow cytometry immunophenotypic algorithm for the identification of newly diagnosed symptomatic myeloma with an MGUS-like signature and long-term disease control. Leukemia. 2013; 27(10):2056-2061.
56. Haessler J, Shaughnessy JD Jr, Zhan F, et al. Benefit of complete response in multiple myeloma limited to high-risk subgroup identified by gene expression profiling. Clin Cancer Res. 2007; 13(23):7073-7079.
57. Hoering A, Crowley J, Shaughnessy JD Jr, et al. Complete remission in multiple myeloma examined as time-dependent variable in terms of both onset and duration in Total Therapy protocols. Blood. 2009;114(7):1299-1305.
58. Li ZW, Dalton WS. Tumor microenvironment and drug resistance in hematologic malignancies. Blood Rev. 2006;20(6):333-342.
59. Vacca A, Ribatti D, Roccaro AM, Frigeri A, Dammacco F. Bone marrow angiogenesis in patients with active multiple myeloma. Semin Oncol. 2001;28(6):543-550.
60. Bhutani M, Turkbey B, Tan E, et al. Bone marrow angiogenesis in myeloma and its precursor disease: a prospective clinical trial. Leukemia. 2014;28(2):413-416.
61. Palumbo A, Bringhen S, Larocca A, et al. Bortezomib-melphalan-prednisone-thalidomide followed by maintenance with bortezomib-thalidomide compared with bortezomib-melphalan-prednisone for initial treatment of multiple myeloma: updated follow-up and improved survival. J Clin Oncol. 2014;32(7):634-640.
62. Moreau P, Kolb B, Hulin C, et al. CMP-carfilzomib (CFZ) plus melphalan-prednisone (CMP)-in elderly patients with newly diagnosed multiple myeloma (NDMM): results of a phase 1/2 trial. Presented at 18th Congress of the Congress of the European Hematology Association, Stockholm, Sweden, June 14, 2013. Abstract P224.
63. Bringhen S, Petrucci MT, Larocca A, et al. Carfilzomib, cyclophosphamide, and dexamethasone in patients with newly diagnosed multiple myeloma: a multicenter, phase 2 study. Blood. 2014;124(1):63-69.
64. Papanikolaou X, Heuck CJ, Barlogie B. Reply to "Metronomic chemotherapy beyond misconceptions." Haematologica. 2013;98(11): 149-150.
65. Annunziata CM, Hernandez L, Davis RE, et al. A mechanistic rationale for MEK inhibitor therapy in myeloma based on blockade of MAF oncogene expression. Blood. 2011;117(8):2396-2404.
66. Lionetti M, Biasiolo M, Agnelli L, et al. Identification of microRNA expression patterns and definition of a microRNA/mRNA regulatory network in distinct molecular groups of multiple myeloma. Blood. 2009;114(25):e20-e26.
67. Wang J, Hendrix A, Hernot S, et al. Bone marrow stromal cell-derived exosomes as communicators in drug resistance in multiple myeloma cells. Blood. 2014;124(4):555-566.
68. Abdi J, Qiu L, Chang H. Micro-RNAs, New performers in multiple myeloma bone marrow microenvironment [published online ahead of print May 30, 2014]. Biomark Res.