Time-dependent endpoints as predictors of overall survival in multiple myeloma

BMC Cancer

Volumn 13, Issue , 2013, Pages

  Félix, Jorge ^a   Aragão, Filipa ^a   Almeida, João M ^a   Calado, Frederico J M ^a   Ferreira, Diana ^a   Parreira, António B S ^b   Rodrigues, Ricardo ^c   Rijo, João F R ^d  

^a Exigo Consultores   (Portugal)

^b PORTUGUESE ONCOLOGY INSTITUTE   (Portugal)

^c RESEARCH INSTITUTE FOR MEDICINES IMED ULISBOA   (Portugal)

^d HOSPITAL EGAS MONIZ   (Portugal)

Author keywords

Multiple myeloma; Overall survival; Survival predictors; Time to progression; Time dependent endpoint

Indexed keywords

BORTEZOMIB; CYCLOPHOSPHAMIDE; DEXAMETHASONE; LENALIDOMIDE; MELPHALAN; PREDNISONE; THALIDOMIDE;

ARTICLE; CORRELATION COEFFICIENT; EVENT FREE SURVIVAL; HUMAN; LOW DRUG DOSE; MULTIPLE MYELOMA; OVERALL SURVIVAL; PHASE 3 CLINICAL TRIAL (TOPIC); PREDICTION; PROGRESSION FREE SURVIVAL; QUANTITATIVE ANALYSIS; RANDOMIZED CONTROLLED TRIAL (TOPIC); SYSTEMATIC REVIEW; TIME;

AGED; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; DISEASE-FREE SURVIVAL; FEMALE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; MALE; MIDDLE AGED; MULTIPLE MYELOMA; RANDOMIZED CONTROLLED TRIALS AS TOPIC; TRANSPLANTATION, HOMOLOGOUS;

EID: 84874943920     PISSN: None     EISSN: 14712407     Source Type: Journal    
DOI: 10.1186/1471-2407-13-122     Document Type: Article

References (92)

1. American Cancer Society Cancer Facts & Figures 2010, http://www.cancer.org/acs/groups/content/@nho/documents/document/acspc-024113.pdf, American Cancer Society.
2. National Comprehensive Cancer Network NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines™): Multiple myeloma, Version 2. 2010 http://www.nccn.org/professionals/physician_gls/f_guidelines.asp, National Comprehensive Cancer Network.
3. National Cancer Institute Surveillance Epidemiology and End Results. SEER Stat Fact Sheets: Myeloma 2010, http://seer.cancer.gov/statfacts/html/mulmy.html, National Cancer Institute.
4. Sirohi B, Powles R. Epidemiology and outcomes research for MGUS, myeloma and amyloidosis. Eur J Cancer 2006, 42:1671-1683. 10.1016/j.ejca.2006.01.065, 16870424.
5. Rajkumar SV. Treatment of myeloma: cure vs control. Mayo Clin Proc 2008, 83:1142-1145. 10.4065/83.10.1142, 18828974.
6. Shi Q, Sargent DJ. Meta-analysis for the evaluation of surrogate endpoints in cancer clinical trials. Int J Clin Oncol 2009, 14:102-111. 10.1007/s10147-009-0885-4, 19390940.
7. Fleming TR, Rothmann MD, Lu HL. Issues in using progression-free survival when evaluating oncology products. J Clin Oncol 2009, 27:2874-2880. 10.1200/JCO.2008.20.4107, 2698020, 19414672.
8. Korn EL, Freidlin B, Abrams JS. Overall survival as the outcome for randomized clinical trials with effective subsequent therapies. J Clin Oncol 2011, 29:2439-2442. 10.1200/JCO.2011.34.6056, 3107757, 21555691.
9. Zhuang SH, Xiu L, Elsayed YA. Overall survival: a gold standard in search of a surrogate: the value of progression-free survival and time to progression as end points of drug efficacy. Cancer J 2009, 15:395-400. 10.1097/PPO.0b013e3181be231d, 19826359.
10. Cooper R, Kaanders JH. Biological surrogate end-points in cancer trials: potential uses, benefits and pitfalls. Eur J Cancer 2005, 41:1261-1266. 10.1016/j.ejca.2005.03.002, 15939261.
11. Buyse M, Sargent DJ, Grothey A. Biomarkers and surrogate end points-the challenge of statistical validation. Nat Rev Clin Oncol 2010, 7:309-317. 10.1038/nrclinonc.2010.43, 20368727.
12. Baker SG. Surrogate endpoints: wishful thinking or reality?. J Natl Cancer Inst 2006, 98:502-503. 10.1093/jnci/djj153, 16622112.
13. Tuma R. Progression-free survival remains debatable endpoint in cancer trials. J Natl Cancer Inst 2009, 101:1439-1441. 10.1093/jnci/djp399, 19828873.
14. Elston J, Taylor RS. Use of surrogate outcomes in cost-effectiveness models: a review of United Kingdom health technology assessment reports. Int J Technol Assess Health Care 2009, 25:6-13. 10.1017/S0266462309090023, 19126246.
15. Velasco Garrido M, Mangiapane S. Surrogate outcomes in health technology assessment: an international comparison. Int J Technol Assess Health Care 2009, 25:315-322. 10.1017/S0266462309990213, 19619350.
16. Johnson JR, Williams G, Pazdur R. End points and United States food and drug administration approval of oncology drugs. J Clin Oncol 2003, 21:1404-1411. 10.1200/JCO.2003.08.072, 12663734.
17. Anderson KC, Kyle RA, Rajkumar SV. Clinically relevant end points and new drug approvals for myeloma. Leukemia 2008, 22:231-239. 10.1038/sj.leu.2405016, 17972944.
18. Greene WH. Econometric analysis 2003, New Jersey: Prentice Hall, 5.
19. Hansen LP. Large sample properties of generalized method of moments estimators. Econometrica 1982, 50:1029-1054.
20. Huber PJ. The behavior of maximum likelihood estimates under nonstandard conditions. Fifth Berkeley Symposium on Mathematical Statistics and Probability. Volume 1 1967, 221-233. Berkeley, CA: University of California Press, Le Cam LM, Neyman J.
21. White H. A heteroskedasticity-consistent covariance matrix estimator and a direct test for heteroskedasticity. Econometrica 1980, 48:817-830.
22. Smith RJ, Blundell RW. An exogeneity test for a simultaneous equation Tobit model with an application to labor supply. Econometrica 1986, 54:679-685.
23. Cragg JG. More efficient estimation in the presence of heteroskedasticity of unknown form. Econometrica 1983, 51:751-763.
24. Durbin J. Errors in variables. Rev Int Stat Inst 1954, 22:23-32.
25. Hausman J. Specification tests in econometrics. Econometrica 1978, 46:1251-1271.
26. Wu DM. Alternative tests of independence between stochastic regressors and disturbances. Econometrica 1973, 41:733-750.
27. Hansen L. Proofs for large sample properties of generalized method of moments estimators. J Econ 2012, 170(2):325-330.
28. Breusch TS, Pagan AR. A simple test for heteroskedasticity and random coefficient variation. Econometrica 1979, 47:1287-1294.
29. Ramsey JB. Tests for specification errors in classical linear least squares regression analysis. J Roy Stat Soc B 1969, 31:350-371.
30. Sherrill B, Amonkar M, Wu Y. Relationship between effects on time-to-disease progression and overall survival in studies of metastatic breast cancer. Br J Cancer 2008, 99:1572-1578. 10.1038/sj.bjc.6604759, 2584937, 19002178.
31. Tang PA, Bentzen SM, Chen EX. Surrogate end points for median overall survival in metastatic colorectal cancer: literature-based analysis from 39 randomized controlled trials of first-line chemotherapy. J Clin Oncol 2007, 25:4562-4568. 10.1200/JCO.2006.08.1935, 17876010.
32. Little R, Rubin D. Statistical Analysis with Missing Data 2002, New York: John Wiley and Sons, 2.
33. Mateos MV, San-Miguel J. Treatment of newly diagnosed myeloma in patients not eligible for transplantation. Curr Hematol Malig Rep 2011, 6:113-119. 10.1007/s11899-011-0080-3, 21347656.
34. Ludwig H, Beksac M, Blade J. Current multiple myeloma treatment strategies with novel agents: a European perspective. Oncologist 2010, 15:6-25. 10.1634/theoncologist.2009-0203, 3227886, 20086168.
35. Reece DE. Posttransplantation maintenance therapy and optimal frontline therapy in myeloma. Hematology Am Soc Hematol Educ Program 2011, 2011:197-204. 10.1182/asheducation-2011.1.197, 22160034.
36. Ludwig H, Hajek R, Tothova E. Thalidomide-dexamethasone compared with melphalan-prednisolone in elderly patients with multiple myeloma. Blood 2009, 113:3435-3442. 10.1182/blood-2008-07-169565, 18955563.
37. Morgan GJ, Davies FE, Gregory WM. Cyclophosphamide, thalidomide, and dexamethasone (CTD) as initial therapy for patients with multiple myeloma unsuitable for autologous transplantation. Blood 2011, 118:1231-1238. 10.1182/blood-2011-02-338665, 3152492, 21652683.
38. Wijermans P, Schaafsma M, Termorshuizen F. Phase III study of the value of thalidomide added to melphalan plus prednisone in elderly patients with newly diagnosed multiple myeloma: the HOVON 49 study. J Clin Oncol 2010, 28:3160-3166. 10.1200/JCO.2009.26.1610, 20516439.
39. Waage A, Gimsing P, Fayers P. Melphalan and prednisone plus thalidomide or placebo in elderly patients with multiple myeloma. Blood 2010, 116:1405-1412. 10.1182/blood-2009-08-237974, 20448107.
40. Beksac M, Haznedar R, Firatli-Tuglular T. Addition of thalidomide to oral melphalan/prednisone in patients with multiple myeloma not eligible for transplantation: results of a randomized trial from the Turkish myeloma study group. Eur J Haematol 2010, 86:16-22.
41. Palumbo A, Bringhen S, Liberati AM. Oral melphalan, prednisone, and thalidomide in elderly patients with multiple myeloma: updated results of a randomized controlled trial. Blood 2008, 112:3107-3114. 10.1182/blood-2008-04-149427, 18505783.
42. Facon T, Mary JY, Hulin C. Melphalan and prednisone plus thalidomide versus melphalan and prednisone alone or reduced-intensity autologous stem cell transplantation in elderly patients with multiple myeloma (IFM 99-06): a randomised trial. Lancet 2007, 370:1209-1218. 10.1016/S0140-6736(07)61537-2, 17920916.
43. Hulin C, Facon T, Rodon P. Efficacy of melphalan and prednisone plus thalidomide in patients older than 75 years with newly diagnosed multiple myeloma: IFM 01/01 trial. J Clin Oncol 2009, 27:3664-3670. 10.1200/JCO.2008.21.0948, 19451428.
44. San Miguel JF, Schlag R, Khuageva NK. Bortezomib plus melphalan and prednisone for initial treatment of multiple myeloma. N Engl J Med 2008, 359:906-917. 10.1056/NEJMoa0801479, 18753647.
45. Niesvizky R, Flinn IW, Rifkin RM. Phase 3b UPFRONT Study: safety and efficacy of weekly bortezomib maintenance therapy after bortezomib-based induction regimens in elderly, newly diagnosed multiple myeloma patients [abstract]. Blood 2010, 116:619.
46. Mateos MV, Oriol A, Martinez-Lopez J. 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, 11:934-941. 10.1016/S1470-2045(10)70187-X, 20739218.
47. Palumbo A, Bringhen S, Rossi D. 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, 28:5101-5109. 10.1200/JCO.2010.29.8216, 20940200.
48. Palumbo A, Delforge M, Catalano J. A phase 3 study evaluating the efficacy and safety of lenalidomide combined with melphalan and prednisone in patients ≥65 years with newly diagnosed multiple myeloma (NDMM): continuous use of lenalidomide vs fixed-duration regimens [abstract]. Blood 2010, 116:622.
49. Rajkumar SV, Jacobus S, Callander NS. 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, 11:29-37. 10.1016/S1470-2045(09)70284-0, 3042271, 19853510.
50. Dimopoulos MA, Chen C, Spencer A. Long-term follow-up on overall survival from the MM-009 and MM-010 phase III trials of lenalidomide plus dexamethasone in patients with relapsed or refractory multiple myeloma. Leukemia 2009, 23:2147-2152. 10.1038/leu.2009.147, 19626046.
51. Richardson PG, Sonneveld P, Schuster M. Extended follow-up of a phase 3 trial in relapsed multiple myeloma: final time-to-event results of the APEX trial. Blood 2007, 110:3557-3560. 10.1182/blood-2006-08-036947, 17690257.
52. Orlowski RZ, Nagler A, Sonneveld P. Randomized phase III study of pegylated liposomal doxorubicin plus bortezomib compared with bortezomib alone in relapsed or refractory multiple myeloma: combination therapy improves time to progression. J Clin Oncol 2007, 25:3892-3901. 10.1200/JCO.2006.10.5460, 17679727.
53. Stewart AK, Trudel S, Bahlis NJ. A randomized phase III trial of thalidomide and prednisone as maintenance therapy following autologous stem cell transplantation (ASCT) in patients with multiple myeloma (MM): the NCIC CTG MY.10 trial [abstract]. Blood 2010, 116:39.
54. Spencer A, Prince HM, Roberts AW. 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, 27:1788-1793. 10.1200/JCO.2008.18.8573, 19273705.
55. Morgan GJ, Gregory WM, Davies FE. The role of maintenance thalidomide therapy in multiple myeloma: MRC Myeloma IX results and meta-analysis. Blood 2012, 119:7-15. 10.1182/blood-2011-06-357038, 22021371.
56. Lokhorst HM, van der Holt B, Zweegman S. 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 2009, 115:1113-1120.
57. Sonneveld P, Schmidt-Wolf I, van der Holt B. HOVON-65/GMMG-HD4 randomized phase III trial comparing bortezomib, doxorubicin, dexamethasone (PAD) vs VAD followed by high-dose melphalan (HDM) and maintenance with bortezomib or thalidomide in patients with newly diagnosed multiple myeloma (MM) [abstract]. Blood 2010, 116:40.
58. McCarthy P, Owzar K, Anderson K. Phase III intergroup study of lenalidomide versus placebo maintenance therapy following single autologous stem cell transplant (ASCT) for multiple myeloma (MM): CALGB ECOG BMT-CTN 100104 [abstract]. Haematologica 2011, 96:S23.
59. Attal M, Olivier P, Cances-Lauwers V. Maintenance treatment with lenalidomide after transplantation for myeloma: analysis of secondary malignancies within the IFM 2005-02 trial. Haematologica 2011, 96:S23.
60. Berghmans T, Pasleau F, Paesmans M. Surrogate markers predicting overall survival for lung cancer: ELCWP recommendations. Eur Respir J 2012, 39:9-28. 10.1183/09031936.00190310, 21737547.
61. Buyse M. Use of meta-analysis for the validation of surrogate endpoints and biomarkers in cancer trials. Cancer J 2009, 15:421-425. 10.1097/PPO.0b013e3181b9c602, 19826362.
62. Buyse M, Burzykowski T, Carroll K. Progression-free survival is a surrogate for survival in advanced colorectal cancer. J Clin Oncol 2007, 25:5218-5224. 10.1200/JCO.2007.11.8836, 18024867.
63. Buyse M, Michiels S, Squifflet P. Leukemia-free survival as a surrogate end point for overall survival in the evaluation of maintenance therapy for patients with acute myeloid leukemia in complete remission. Haematologica 2011, 96:1106-1112. 10.3324/haematol.2010.039131, 3148903, 21546500.
64. Hackshaw A, Knight A, Barrett-Lee P. Surrogate markers and survival in women receiving first-line combination anthracycline chemotherapy for advanced breast cancer. Br J Cancer 2005, 93:1215-1221. 10.1038/sj.bjc.6602858, 2361525, 16278665.
65. Hotta K, Fujiwara Y, Matsuo K. Time to progression as a surrogate marker for overall survival in patients with advanced non-small cell lung cancer. J Thorac Oncol 2009, 4:311-317. 10.1097/JTO.0b013e3181989bd2, 19190515.
66. Johnson KR, Ringland C, Stokes BJ. Response rate or time to progression as predictors of survival in trials of metastatic colorectal cancer or non-small-cell lung cancer: a meta-analysis. Lancet Oncol 2006, 7:741-746. 10.1016/S1470-2045(06)70800-2, 16945769.
67. Lee L, Wang L, Crump M. Identification of potential surrogate end points in randomized clinical trials of aggressive and indolent non-Hodgkin's lymphoma: correlation of complete response, time-to-event and overall survival end points. Ann Oncol 2011, 22:1392-1403. 10.1093/annonc/mdq615, 3101365, 21266519.
68. Michiels S, Le Maitre A, Buyse M. Surrogate endpoints for overall survival in locally advanced head and neck cancer: meta-analyses of individual patient data. Lancet Oncol 2009, 10:341-350. 10.1016/S1470-2045(09)70023-3, 19246242.
69. Miksad RA, Zietemann V, Gothe R. Progression-free survival as a surrogate endpoint in advanced breast cancer. Int J Technol Assess Health Care 2008, 24:371-383.
70. Sargent DJ, Patiyil S, Yothers G. End points for colon cancer adjuvant trials: observations and recommendations based on individual patient data from 20,898 patients enrolled onto 18 randomized trials from the ACCENT Group. J Clin Oncol 2007, 25:4569-4574. 10.1200/JCO.2006.10.4323, 17876008.
71. Shitara K, Ikeda J, Yokota T. Progression-free survival and time to progression as surrogate markers of overall survival in patients with advanced gastric cancer: analysis of 36 randomized trials. Invest New Drugs 2012, 30:1224-1231. 10.1007/s10637-011-9648-y, 21350803.
72. Burzykowski T, Buyse M, Piccart-Gebhart MJ. Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer. J Clin Oncol 2008, 26:1987-1992. 10.1200/JCO.2007.10.8407, 18421050.
73. Matsubara Y, Sakabayashi S, Nishimura T. Surrogacy of tumor response and progression-free survival for overall survival in metastatic breast cancer resistant to both anthracyclines and taxanes. Int J Clin Oncol 2011, 16:623-629. 10.1007/s10147-011-0231-5, 21537884.
74. Chakravarty A, Sridhara R. Use of progression-free survival as a surrogate marker in oncology trials: some regulatory issues. Stat Methods Med Res 2008, 17:515-518. 10.1177/0962280207081862, 18285437.
75. Prentice RL. Surrogate and mediating endpoints: current status and future directions. J Natl Cancer Inst 2009, 101:216-217. 10.1093/jnci/djn515, 19211455.
76. Rajkumar SV, Gahrton G, Bergsagel PL. Approach to the treatment of multiple myeloma: a clash of philosophies. Blood 2011, 118:3205-3211. 10.1182/blood-2011-06-297853, 3179390, 21791430.
77. Maroun JA. The significance of progression-free survival as an endpoint in evaluating the therapeutic value of antineoplastic agents. Curr Oncol 2011, 18(Suppl 2):S3-S4. 3176907, 21969809.
78. Verma S, McLeod D, Batist G. In the end what matters most? A review of clinical endpoints in advanced breast cancer. Oncologist 2011, 16:25-35.
79. Saad ED, Katz A, Hoff PM. Progression-free survival as surrogate and as true end point: insights from the breast and colorectal cancer literature. Ann Oncol 2010, 21:7-12. 10.1093/annonc/mdp523, 19901012.
80. Duffy SW, Treasure FP. Potential surrogate endpoints in cancer research-some considerations and examples. Pharm Stat 2011, 10:34-39. 10.1002/pst.406, 20029937.
81. Peninsula Technology Assessment Group The clinical and cost-effectiveness of lenalidomide for multiple myeloma in people who have received at least one prior therapy: An evidence review of the submission from Celgene 2008, http://www.hta.ac.uk/erg/reports/1746.pdf, Peninsula Technology Assessment Group.
82. Richardson PG, Sonneveld P, Schuster MW. Bortezomib or high-dose dexamethasone for relapsed multiple myeloma. N Engl J Med 2005, 352:2487-2498. 10.1056/NEJMoa043445, 15958804.
83. Freidlin B, Korn EL, George SL. Randomized clinical trial design for assessing noninferiority when superiority is expected. J Clin Oncol 2007, 25:5019-5023. 10.1200/JCO.2007.11.8711, 17971602.
84. Di Leo A, Bleiberg H, Buyse M. Overall survival is not a realistic end point for clinical trials of new drugs in advanced solid tumors: a critical assessment based on recently reported phase III trials in colorectal and breast cancer. J Clin Oncol 2003, 21:2045-2047. 10.1200/JCO.2003.99.078, 12743164.
85. Kumar SK, Rajkumar SV, Dispenzieri A. Improved survival in multiple myeloma and the impact of novel therapies. Blood 2008, 111:2516-2520. 10.1182/blood-2007-10-116129, 2254544, 17975015.
86. Colleoni M, Giobbie-Hurder A, Regan MM. Analyses adjusting for selective crossover show improved overall survival with adjuvant letrozole compared with tamoxifen in the BIG 1-98 study. J Clin Oncol 2011, 29:1117-1124. 10.1200/JCO.2010.31.6455, 3083867, 21321298.
87. Morden JP, Lambert PC, Latimer N. Assessing methods for dealing with treatment switching in randomised controlled trials: a simulation study. BMC Med Res Methodol 2011, 11:4. 10.1186/1471-2288-11-4, 3024998, 21223539.
88. Ishak KJ, Caro JJ, Drayson MT. Adjusting for patient crossover in clinical trials using external data: a case study of lenalidomide for advanced multiple myeloma. Value Health 2011, 14:672-678. 10.1016/j.jval.2011.02.1182, 21839405.
89. Pacou M, Gauthier A, Abrams K. Survival analysis adjusted for crossover in relapsed multiple myeloma: Results of the APEX trial [abstract]. Value Health 2011, 14:A437-A438.
90. Therasse P, Carbonnelle S, Bogaerts J. Clinical trials design and treatment tailoring: general principles applied to breast cancer research. Crit Rev Oncol Hematol 2006, 59:98-105. 10.1016/j.critrevonc.2005.11.003, 16431124.
91. Ellenberg SS. Accelerated approval of oncology drugs: can we do better?. J Natl Cancer Inst 2011, 103:616-617. 10.1093/jnci/djr104, 21422401.
92. Putter H, Fiocco M, Geskus RB. Tutorial in biostatistics: competing risks and multi-state models. Stat Med 2007, 26:2389-2430. 10.1002/sim.2712, 17031868.