Use of new drugs within primary therapy of multiple myeloma

1. Dušek L, Mužík J, Malúšková D et al. Epidemiologie zhoubných nádorů cílených screeningem dle nových dat Národního onkologického registru České republiky. Klin Onkol 2014; 27(Suppl 2): S19-S39.

2. Hájek R, Krejčí M, Pour L et al. Multiple myeloma. Klin Onkol 2011; 24(Suppl): S10-S13.

3. Kyle R, Gertz M, Witzig T et al. Review of 1027 patients with newly diagnosed multiple myeloma, Mayo Clin Proc 2003; 78(1): 21–33.

4. National Comprehensive Cancer Network. NCCN Guidelines: multiple myeloma. Dostupné z WWW: <http://www.nccn.org/professionals/physician_gls/f_guidelines.asp#myeloma>. [March 12, 2014].

5. Straub J, Adam Z, Gregora E et al. Mnohočetný myelom – časná diagnostika. Med Pro Praxi 2009; 6(4): 197–199.

6. Bladé J, Dimopoulos M, Rosinol L et al. Smoldering (Asymptomatic) Multiple Myeloma: Current Diagnostic Criteria, New Predictors of Outcome, and Follow-Up Recommendations. J Clin Oncol 2010; 28(4): 690–697.

7. Kumar S, Rajkumar S, Dispenzieri A et al. Improved survival in multiple myeloma and the impact of novel therapies. Blood 2008; 111(5): 2516–2520.

8. San-Miguel JF, Mateos MV. Can multiple myeloma become a curable disease? Haematologica 2011; 96(9): 1246–1248.

9. Hajek R, Zahradova L. Možnosti a limitace farmakoterapie mnohočetného myelomu. Farmakoterapie 2012; 8(3): 287–293.

10. Hájek R, Adam Z, Ščudla V et al. Diagnostika a léčba mnohočetného myelomu. Doporučení vypracované Českou myelomovou skupinou, Myelomovou sekcí České hematologické společnosti a Slovenskou myelómovou spoločnosti pro diagnostiku a léčbu mnohočetného myelomu. Transfuze a hematologie dnes 2012; 18: 6–89.

11. Harousseau J, Attal M, Avet-Loiseau H et al. Bortezomib plus dexamethasone is superior to vincristine plus doxorubicin plus dexamethasone as induction treatment prior to autologous stem-cell transplantation in newly diagnosed multiple myeloma: results of the IFM 2005–01 phase III trial. J Clin Oncol 2010; 28(30): 4621–4629.

12. Cavo M, Zamagni E, Tosi P et al. Superiority of thalidomide and dexamethasone over vincristine-doxorubicin-dexamethasone (VAD) as primary therapy in preparation for autologous transplantation for multiple myeloma. Blood 2005; 106(1): 35–39.

13. Rajkumar S, Rosiñol L, Hussein M et al. Multicenter, randomized, double-blind, placebo-controlled study of thalidomide plus dexamethasone compared with dexamethasone as initial therapy for newly diagnosed multiple myeloma. J Clin Oncol 2008; 26(13): 2171–2177.

14. Lokhorst H, van der Holt B, Zweegman S et al. Final Analysis of HOVON-50 Randomized Phase III Study on the Effect of Thalidomide combined with adriamycine, dexamethasone (AD) and high dose melphalan (HDM) in Patients with Multiple Myeloma. Blood 2010; 115(6): 1113–1120.

15. Cavo M, Pantani L, Pettruci MT et al. Bortezomib-thalidomide-dexamethasone is superior to thalidomide-dexamethasone as consolidation therapy after autologous hematopoietic stem cell transplantation in patients with newly diagnosed multiple myeloma. Blood 2012; 120(1): 9–19.

16. Kumar S, Flinn I, Richardson P et al. Randomized, multicenter, phase 2 study (EVOLUTION) of combinations of bortezomib, dexamethasone, cyclophosphamide, and lenalidomide in previously untreated multiple myeloma. Blood 2012; 119(19): 4375–4382.

17. Sonneveld P, Schmidt-Wolf IG, van der Holt B et al. Bortezomib induction and maintenance treatment in patients with newly diagnosed multiple myeloma: results of the randomized phase III HOVON-65/GMMG-HD4 trial. J Clin Oncol 2012; 30(24): 2946–2955. Erratum in J Clin Oncol 2012; 30(29): 3654.

18. Uttervall K, Adil D, Lund J et al. The use of novel drugs can effectively improve response, delay relapse and enhance overall survival in multiple myeloma patients with renal impairment. PLoS One 2014; 9(7): e101819. Dostupné z DOI: <http://dx.doi.org/10.1371/journal.pone.0101819>.

19. Heinz L, Adam Z, Hajek R et al. Light Chain–Induced Acute Renal Failure Can Be Reversed by Bortezomib-Doxorubicin-Dexamethasone in Multiple Myeloma: Results of a Phase II Study. J Clin Oncol 2010; 28(30): 4635–4641.

20. Sonneveld P, Goldschmidt H, Rosinol L et al. Bortezomib-based versus Nobortezomib- based induction treatment before autologous stem-cell transplantation in patients with previously untreated multiple myeloma: a meta-analysis of phase III randomized, controlled trials. J Clin Oncol 2013; 31(26): 3279–3287.

21. Weiss BM, Abadie J, Verma P et al. A monoclonal gammopathy precedes multiple myeloma in most patients. Blood 2009; 113(22): 5418–5422.

22. Moreau P, Facon T, Attal M et al. [Intergroupe Francophone du Myélome]. Comparison of 200 mg/m2 melphalan and 8 Gy total body irradiation plus 140 mg/m2 melphalan as conditioning regimens for peripheral blood stem cell transplantation in patients with newly diagnosed multiple myeloma: final analysis of the Intergroupe Francophone du Myélome 9502 randomized trial. Blood 2002; 99(3): 731–735.

23. Bjorkstrand B. European Group for Blood and Marrow Transplantation Registry studies in multiple myeloma. Semin Hematol 2001; 38(3): 219–225.

24. Carreras E, Rosińol L, Terol MJ et al. Veno-occlusive disease of the liver after highdose cytoreductive therapy with busulfan and melphalan for autologous blood stem cell transplantation in multiple myeloma patients. Biol Blood Marrow Transplant 2007; 13(12): 1448–1454.

25. Badros A, Barlogie B, Siegel E et al. Autologous stem cell transplantation in elderly multiple myeloma patients over the age of 70 years. Br J Haematol 2001; 114(3): 600–607.

26. Bourhis JH, Bouko Y, Koscielny S et al. Relapse risk after autologous transplantation in patients with newly diagnose myeloma is not related with infused tumor cell load and the outcome is not improved by CD34+ cell selection: long term follow-up of an EBMT phase III randomized study. Haematologica 2007; 92(8): 1083–1090.

27. Attal M, Harousseau JL, Facon T et al. Single versus double autologous stem-cell transplantation for multiple myeloma. N Engl J Med 2003; 349(26): 2495–2502. Erratum in N Engl J Med 2004; 350(25): 2628.

28. Cavo M, Tosi P, Zamagni E et al. Prospective, randomized study of single compared with double autologous stem-cell transplantation for multiple myeloma: Bologna 96 clinical study. J Clin Oncol 2007; 25(17): 2434–2441.

29. Kumar A, Kharfan-Dabaja MA, Glasmacher A et al. Tandem versus single autologous hematopoietic cell transplantation for the treatment of multiple myeloma: A systematic review and meta-analysis. J Natl Cancer Inst 2009; 101(2): 100–106.

30. Rajkumar SV, Callander NS, Jacobus S et al. Eastern Cooperative Oncology Group. Lenalidomide plus high-dose dexamethasone versus lenalidomide plus low-dose dexamethasone as initial therapy for newly diagnosed multiple myeloma: an open-label randomised controlled trial. Lancet Oncol 2010; 11(1): 29–37.

31. Palumbo A, Cavallo F, Gay F et al Autologous transplantation and maintenance therapy in multiple myeloma. N Engl J Med 2014; 371(10): 895–905.

32. Gay F, Larocca A, Vijermans P et al. Complete response correlates with long-term progression-free and overall survival in elderly myeloma treated with novel agents: analysis of 1175 patients. Blood 2011; 117(11): 3025–3031.

33. Ladetto M, Ferrero S, Pagliano G et al. Major tumor shrinking and persistent molecular remissions after consolidation with bortezomib, thalidomide, and dexamethasone in patients with autografted myeloma. J Clin Oncol 2010; 28(12): 2077–2084.

34. Mellqvist UH, Gimsing P, Hjertner O et al. Bortezomib consolidation after autologous stem cell transplantation in multiple myeloma: a Nordic Myeloma Study Group randomized phase 3 trial. Blood 2013; 121(23): 4647–4654.

35. Ludwig H, Durie BG, McCarthy P et al. IMWG consensus on maintenance therapy in multiple myeloma. Blood 2012; 119(13): 3003–3015.

36. Belch A, Shelley W, Bergsagel D et al. A randomized trial of maintenance versus no maintenance melphalan and prednisone in responding multiple myeloma patients. Br J Cancer 1988; 57(1): 94–99.

37. [Myeloma Trialists’ Collaborative Group]. Interferon as therapy for multiple myeloma: an individual patient data overview of 24 randomized trials and 4012 patients. Br J Haematol 2001; 113(4): 1020–1034.

38. Attal M, Harousseau JL, Leyvraz S et al. Maintenance therapy with thalidomide improves survival in multiple myeloma patients. Blood 2006; 108(10): 3289–3294.

39. Spencer A, Prince HM, Roberts P et al. Consolidation therapy with low-dose thalidomide and prednisolone prolongs the survival of multiple myeloma patients undergoing a single autologous stem-cell transplantation procedure. J Clin Oncol 2009; 27(11): 1788–1793.

40. Lokhorst HM, van der Holt B, Zweegman S et al. A randomized phase 3 study on the effect of thalidomide combined with adriamycin, dexamethasone, and high-dose melphalan, followed by thalidomide maintenance in patients with multiple myeloma. Blood 2010; 115(6): 1113–1120.

41. Morgan GJ, Gregory WM, Davies FE et al. The role of maintenance thalidomide therapy in multiple myeloma: MRC Myeloma IX results and meta-analysis. Blood 2012; 119(1): 7–15.

42. Goldschmidt H, Neben K, Bertsch U et al. Bortezomib-based induction therapy followed by autologous stem cell transplantation and maintenance therapy with bortezomib improves outcome in myeloma patients with gain 1q21 and t(4;14): a subgroup analysis of the HOVON-65/GMMG-HD4 trial. Blood 2010; 116(21): Abstract 305.

43. McCarthy PL, Owzar K, Hofmeister C et al. Lenalidomide after stem-cell transplantation for multiple myeloma. N Engl J Med 2012; 366(19): 1770–1781.

44. Attal M, Lauwers-Cances V, Marit G et al. Lenalidomide maintenance after stem-cell transplantation for multiple myeloma. N Engl J Med 2012; 366(19): 1782–1791.

45. Attal M, Lauwers-Cances V, Marit G et al. Lenalidomide Maintenance After Stem-Cell Transplantation For Multiple Myeloma: Follow-Up Analysis Of The IFM 2005–02 Trial. Blood 2013; 122(21): Abstract 406.

46. Palumbo A, Bringhen S, Kumar SK et al. Second primary malignancies with lenalidomide therapy for newly diagnosed myeloma: a meta-analysis of individual patient data. Lancet Oncol 2014; 15(3): 333–342.

47. Palumbo A, Rajkumar SV, San Miguel JF et al. International Myeloma Working Group consensus statement for the management, treatment, and supportive care of patients with myeloma not eligible for standard autologous stem-cell transplantation. J Clin Oncol 2014; 32(6): 587–600.

48. 2009 Ageing report: economic and budgetary projections for the EU-27 Member States (2008–2060). Joint Report prepared by the European Commission (DG ECFIN) and the Economic Policy Committee (AWG). European Economy 2009; 2. Office for Official Publications of the European Communities: Luxembourg 2009. ISBN 978–92–79–11363–9. Dostupné z WWW: <http://ec.europa.eu/economy_finance/publications/publication14992_en.pdf>.

49. Bringhen S, Mateos MV, Zveegman S et al. Age And Organ Damage Correlate With Poor Survival In Myeloma Patients: Meta-Analysis Of 1435 Individual Patient Data From 4 Randomized Trials. Haematologica 2013; 98(6): 980–987.

50. Larocca A, Bringhen S, Andrea E et al. A simple score, based on geriatric assessment, improves prediction of survival, and risk of serious adverse events in elderly newly diagnosed multiple myeloma patients. Blood 2013; 122(21): Abstract 687.

51. Gay F, Palumbo A. Management of older patients with multiple myeloma. Blood Rev 2011; 25(2): 65–73.

52. Gay F, Larocca A, Wijermans P et al. Complete response correlates with long-term progression-free and overall survival in elderly myeloma treated with novel agents: analysis of 1175 patients. Blood 2011; 117(11): 3025–3031.

53. [Myeloma Trialists’ Collaborative Group]. Combination chemotherapy versus melphalan plus prednisone as treatment for multiple myeloma: an overview of 6,633 patients from 27 randomized trials. J Clin Oncol 1998; 16(12): 3832–3842.

54. Fayers PM, Palumbo A, Hulin C et al. Thalidomide for previously untreated elderly patients with multiple myeloma: meta-analysis of 1685 individual patient data from 6 randomized clinical trials. Blood 2011; 118(5): 1239–1247.

55. Palumbo A, Waage A, Hulin C et al. Safety of thalidomide in newly diagnosed elderly myeloma patients: a meta-analysis of data from individual patients in six randomized trials. Haematologica 2013; 98(1): 87–94.

56. Morgan GJ, Davies FE, Gregory WM et al. Cyclophosphamide, thalidomide, and dexamethasone (CTD) as initial therapy for patients with multiple myeloma unsuitable for autologous transplantation. Blood 2011; 118(5): 1231–1238.

57. Mateos MV, Oriol A, Martínez-López J et al. Bortezomib, melphalan, and prednisone versus bortezomib, thalidomide, and prednisone as induction therapy followed by maintenance treatment with bortezomib and thalidomide versus bortezomib and prednisone in elderly patients with untreated multiple myeloma: a randomised trial. Lancet Oncol 2010; 11(10): 934–941.

58. Palumbo A, Bringhen S, Rossi H et al. Bortezomib-melphalan-prednisone-thalidomide followed by maintenance with bortezomib-thalidomide compared with bortezomib-melphalan-prednisone for initial treatment of multiple myeloma: a randomized controlled trial. J Clin Oncol 2010; 28(34): 5101–5109.

59. Benboubker L, Dimopoulos MA, Dispenzieri A et al Lenalidomide and Dexamethasone in Transplant-Ineligible Patients with Myeloma. N Engl J Med 2014; 371(10): 906–917.

60. Palumbo A, Hajek R, Delforge M et al. Continuous lenalidomide treatment for newly diagnosed multiple myeloma. N Engl J Med 2012; 366(19): 1759–1769. Erratum in N Engl J Med 2012; 367(3): 285.

61. San Miguel JF, Schlag R, Khuageva NK et al. Bortezomib plus Melphalan and Prednisone for Initial Treatment of Multiple Myeloma. N Engl J Med 2008; 359(9): 906–917.

62. San Miguel JF, Schlag R, Khuageva NK et al. Persistent overall survival benefit and no increased risk of second malignancies with bortezomib-melphalan-prednisone versus melphalan-prednisone in patients with previously untreated multiple myeloma. J Clin Oncol 2013; 31(4): 448–455.

63. Richardson PG, Briemberg H, Jagannath S et al. Frequency, characteristics, and reversibility of peripheral neuropathy during treatment of advanced multiple myeloma with bortezomib. J Clin Oncol 2006; 24(19): 3113–3120.

64. Bringhen S, Larocca A, Rossi D et al. Efficacy and safety of once-weekly bortezomib in multiple myeloma patients. Blood 2010; 116(23): 4745–4753.

65. Moreau P, Pylypenko H, Grosicki S et al. Subcutaneous versus intravenous administration of bortezomib in patients with relapsed multiple myeloma: a randomised, phase 3, non-inferiority study. Lancet Oncol 2011; 12(5): 431–440. Erratum in Lancet Oncol 2011 ;12(6): 522.

66. Minarik J, Pavlicek P, Pour L et al. Subcutaneous bortezomib in multiple myeloma patients induces similar therapeutic response rates as intravenous application but it does not reduce the incidence of peripheral neuropathy. PLoS One 2015; 10(4): e0123866. Dostupné z DOI: <http://dx.doi.org/10.1371/journal.pone.0123866>.

67. De Weers M, Tai YT, Van der Veer MS et al. Daratumumab, a novel therapeutic human CD38 monoclonal antibody, induces killing of multiple myeloma and other hematological tumors. J Immunol 2011; 1868š9: 1840–1848.

68. Jansen JH, Boross P, Overdijk MB et al. Daratumumab, a Human CD38 Antibody Induces Apoptosis of Myeloma Tumor Cells Via Fc Receptor-Mediated Crosslinking. American Society of Hematology Proceedings 2012. Blood 2012; 120(21): 2974.

69. Lokhorst HM, Plesner T, Gimsing P et al. Phase I/II dose-escalation study of Daratumumab in patients with relapsed or refractory multiple myeloma. J Clin Oncol 2013; 31(Suppl): Abstract 8512.

70. Van der Veer MS, de Weers M, van Kessel B et al. Towards effective immunotherapy of myeloma: enhanced elimination of myeloma cells by combination of lenalidomide with human CD38 monoclonal antibody daratumumab. Haematologica 2011; 96(2): 284–290.

71. [Janssen Research & Development LLC]. A Study of Combination of Daratumumab and Velcade (Bortezomib) Melphalan-Prednisone (DVMP) Compared to Velcade Melphalan-Prednisone (VMP) in Participants With Previously Untreated Multiple Myeloma. Dostupné z WWW: <https://clinicaltrials.gov/show/NCT02195479 NLM identifier NCT02195479>.

72. Collins SM, Courtney E. Bakan Swartzel GD et al. Elotuzumab directly enhances NK cell cytotoxicity against myeloma via CS1 ligation: evidence for augmented NK cell function complementing ADCC. Cancer Immunol Immunother 2013; 62(12): 1841–1849.

73. Lonial S, Dimopoulos M, Palumbo A et al. Elotuzumab therapy for relapsed or refractory multiple myeloma. N Engl J Med 2015; 373(7): 621–631.

74. Kuhn DJ, Chen Q, Voorhees PM et al. Potent activity of carfilzomib, a novel, irreversible inhibitor of the ubiquitin-proteasome pathway, against preclinical models of multiple myeloma. Blood 2007; 110(9): 3281–3290.

75. [Onyx Pharmaceuticals]. Phase 3 Study With Carfilzomib and Dexamethasone Versus Velcade and Dexamethasone for Relapsed Multiple Myeloma Patients (ENDEAVOR). Dostupné z WWW: <https://clinicaltrials.gov/ct2/show/NCT01568866>.

76. [Onyx Therapeutics, Inc]. Phase 3 Study of Carfilzomib, Melphalan, Prednisone vs Bortezomib, Melphalan, Prednisone in Newly Diagnosed Multiple Myeloma (CLARION). Dostupné z WWW: <https://clinicaltrials.gov/show/NCT01818752 NLM identifier NCT01818752>.

77. Hideshima T, Chauhan D, Shima Y et al. Thalidomide and its analogs overcome drug resistance of human multiple myeloma cells to conventional therapy. Blood 2000; 96(9): 2943–2950.

78. Zahra H., Beatriz F, Hemmings R et al. The European Medicines Agency Review of Pomalidomide in Combination with Low-Dose Dexamethasone for the Treatment of Adult Patients With Multiple Myeloma: Summary of the Scientific Assessment of the Committee for Medicinal Products for Human Use. Oncologist 2015; 20(3): 329–334.

79. Gozzetti A, Candi V, Papini G et al. Therapeutic advancements in multiple myeloma. Front Oncol 2014; 4: 241. Dostupné z DOI: <http://dx.doi.org/10.3389/fonc.2014.0024>.

80. Ludwig H, Durie BG, Bolejack V et al. Myeloma in patients younger than age 50 years presents with more favorable features and shows better survival: an analysis of 10 549 patients from the International Myeloma Working Group. Blood 2008; 111(8): 4039–4047.

81. Alexanian R, Haut A, Khan AU et al. Treatment for multiple myeloma. Combination chemotherapy with different melphalan dose regimens. JAMA 1969; 208(9): 1680–1605.

82. San-Miguel JF, Mateos MV. How to treat a newly diagnosed young patient with multiple myeloma. Hematology Am Soc Hematol Educ Program 2009: 555–565.

83. Lonial S. Presentation and risk stratification- improving prognosis for patients with multiple myeloma. Cancer Treat Reviews 2010; 36(Suppl 2): S12-S17.

84. Moreau P, Attal M, Facon T. Frontline therapy of multiple myeloma. Blood 2015; 125(20): 3076–3084.

85. Sonneveld P, Asselbergs E, Zweegman S et al. Phase 2 study of carfilzomib, thalidomide, and dexamethasone as induction/consolidation therapy for newly diagnosed multiple myeloma. Blood 2015; 125(3): 449–456.

86. Stewart K, Rajkumar V, Dimopoulos MA et al. Carfilzomib, Lenalidomide, and Dexamethasone for Relapsed Multiple Myeloma. N Engl J Med 2015; 372(2): 142–152.

87. Dimopoulos A, Moreau P, Palumbo A et al. Carfilzomib and dexamethasone (Kd) vs bortezomib and dexamethasone (Vd) in patients (pts) with relapsed multiple myeloma (RMM): Results from the phase III study ENDEAVOR. 2015 ASCO Annual Meeting. J Clin Oncol 2015; 33(Suppl.): Abstract 8509.

88. Cavo M, Pantani L, Pezzi A et al. Bortezomib-thalidomide-dexamethasone (VTD) is superior to bortezomib-cyclophosphamide-dexamethasone (VCD) as induction therapy prior to autologous stem cell transplantation in multiple myeloma. Leukemia 2015; 28(12): 2429–2431. Dostupné z DOI: <http://dx.doi.org/10.1038/leu.2015.274>.

89. Morabito F, Brighten S, Laroca A et al. Bortezomib, melphalan, prednisone (VMP) versus melphalan, prednisone, thalidomide (MPT) in erderly newly diagnosed multiple myeloma patients: A retrospective case-matched study. Am J Hematol 2014: 89(4): 355–362.

90. Ludwig H, Hajek R, Thotova E et al. Thalidomide-dexamethasone compared with melphalan-prednisolone in elderly patients with multiple myeloma. Blood 2009; 113(15): 3435–3442.

91. Curran MP, McKeage K. Bortezomib: a review of its use in patients with multiple myeloma. Drugs 2009; 69(7): 859–888.