Statistical aspects of prognostic factor studies in oncology

British Journal of Cancer

Volumn 69, Issue 6, 1994, Pages 979-985

  Simon, R ^a   Altman, D G ^b  

^a NATIONAL CANCER INSTITUTE   (United States)

^b IMPERIAL CANCER RESEARCH FUND   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

CALCULATION; CLINICAL TRIAL; COMPARATIVE STUDY; COST BENEFIT ANALYSIS; DEMOGRAPHY; DISEASE COURSE; FOLLOW UP; HUMAN; HYPOTHESIS; META ANALYSIS; METHODOLOGY; ONCOLOGY; PATHOGENESIS; PATIENT SELECTION; PRIORITY JOURNAL; PROBABILITY; PROGNOSIS; QUALITY CONTROL; REGRESSION ANALYSIS; RETROSPECTIVE STUDY; REVIEW; RISK FACTOR; SAMPLE; STATISTICAL ANALYSIS; STATISTICAL MODEL; SURVIVAL RATE;

HUMAN; NEOPLASMS; PROGNOSIS; PROPORTIONAL HAZARDS MODELS; REGRESSION ANALYSIS; RESEARCH; RESEARCH DESIGN; STATISTICS;

EID: 0028318390     PISSN: 00070920     EISSN: 15321827     Source Type: Journal    
DOI: 10.1038/bjc.1994.192     Document Type: Article

References (46)

1. ALTMAN, D.G. (1992). Categorizing continuous variables. Br. J. Cancer, 64, 975.
2. ALTMAN, D.G. & ANDERSEN, P.K. (1989). Bootstrap investigation of the stability of a Cox regression model. Stat. Med., 8, 771-783.
3. ALTMAN, D.G. & MACHIN, D. (1994). Practical problems with survival analysis. Br. J. Cancer (in press).
4. ALTMAN, D.G., LAUSEN, B., SAUERBREI, W. & SCHUMACHER, M. (1994). The dangers of using ‘optimal’ cutpoints in the evaluation of prognostic factors. J. Natl Cancer Inst, (in press).
5. BLAY, J.Y., LASSET, C., CARRIE, C., CHAUVIN, F., COIFFER, B., GISSELBRECHT, C., REBATTU, P., BENNAT-MENTIGNY, M., PHILIP, T. & BIRON, P. (1993). Multivariate analysis of prognostic factors in patients with non HIV-related primary cerebral lymphoma. A proposal for a prognostic scoring. Br. J. Cancer, 67, 1136-1141.
6. BLISS, J.M., ALTMAN, D.G. & MACHIN, D. (1994). Reporting research. Br. J. Cancer (in press).
7. BUYSE, M. (1989). Analysis of clinical trial outcomes: some comments on subgroup analyses. Control. Clin. Trials, 10, 187S-194S.
8. COURDI, A., HERY, M., CHAUVEL, P., GIOANNI, J., NAMER, M. & DEMARD, F. (1988). Prognostic value of continuous variables in breast cancer and head and neck cancer. Dependence on the cut-off level. Br. J. Cancer, 58, 88-90.
9. COX, D.R. (1972). Regression models and life tables (with discussion). J. R. Stat. Soc. B., 34, 187-220.
10. CUZICK, J., COOPER, E.H. & MACLENNAN, I.C.M. (1985). The prognostic value of serum 02 microglobulin compared with other presentation features in myelomatosis. Br. J. Cancer, 52, 1-6.
11. DIXON, D.D. & SIMON, R. (1992). Bayesian subset analysis in a colorectal cancer clinical trial. Stat. Med., 11, 13-22.
12. DORR, F.A. (1992). NIH Consensus Conference on the Treatment of Early Stage Breast Cancer. Journal of the National Cancer Institute Monograph 11: Bethesda, MD.
13. DURRLEMAN, S. & SIMON, R. (1989). Flexible regression models with cubic splines. Stat. Med., 8, 551-561.
14. EELES, R.A., FISHER, C., AHERN, R.P., ROBINSON, M., RHYS-EVANS, P., HENK, J.M., ARCHER, D. & HARMAN, C.L. (1993). Head and neck sarcomas: prognostic factors and implications for treatment. Br. J. Cancer, 68, 201-207.
15. FAYERS, P.M. & MACHIN, D. (1994). How many patients are necessary? Br. J Cancer (in press).
16. FAYERS, P.M., RUSTIN, G., WOOD, R., NELSTROP, A., LEONARD, R.C.F., WILKINSON, P., CRUICKSHANK, D., MCALLISTER, E.J., REDMAN, C.W.E., PARKER, D., SCOTT, I.V., SLEVIN, M.L. & ROULSTON, J.E. (1993). The prognostic value of serum CA 125 in patients with advanced ovarian carcinoma: an analysis of 573 patients by the Medical Research Council Working Party on Gynaecological Cancer. Int. J. Gynecol. Cancer, 3, 285-292.
17. FISHER, B., WOLMARK, N., ROCKETTE, H., REDMOND, C., DEUTSCH, M., WICKERMAN, D.R., FISHER, E.R., CAPLAN, R., JONES, J., LERNER, H., GORDON, P., FELDMAN, P., CRUZ, A., LEGAULT POISSON, S., WEXLER, M., LAWRENCE, W., ROBDIOUX, R. & OTHER NSABP INVESTIGATORS (1988). Postoperative adjuvant chemotherapy or radiation therapy for rectal cancer. Results from NSABP Protocol R-01. J. Natl Cancer Inst., 80, 21-29.
18. FREIMAN, J.A., CHALMERS, T.C., SMITH, H. & KUEBLER, R.R. (1992). The importance of beta, the type II error, and sample size in the design and interpretation of the randomized controlled trial: survey of two sets of ‘negative’ trials. In Medical Uses of Statistics, 2nd edn. Bailar, J.C. & Mosteller, F. (eds) pp. 357-373. Books: Boston.
19. GAIL, M.H. (1981). Evaluating serial cancer marker studies in patients at risk of recurrent disease. Biometrics, 37, 67-78.
20. GAIL, M. & SIMON, R. (1985). Testing for qualitative interactions between treatment effects and patient subsets. Biometrics, 41, 361-372.
21. GARDNER, M.J. & ALTMAN, D.G. (eds) (1989). Statistics with Confidence. BMJ Books: London.
22. GASPARINI, G., POZZA, F. & HARRIS, A L. (1993). Evaluating the potential usefulness of new prognostic and predictive indicators in node-negative breast cancer patients. J. Natl Cancer Inst., 85, 1206-1219.
23. GEORGE, S.L. (1988). Identification and assessment of prognostic factors. Semin. Oncol., 15, 462-471.
24. GRAY-DONALD, K. & KRAMER, M.S. (1988). Causality inference in observational vs. experimental studies. An empirical comparison. Am. J. Epidemiol., 127, 885-892.
25. GREGORY, W.M. (1988). Adjusting survival curves for imbalances in prognostic factors. Br. J. Cancer, 58, 202-204.
26. GUSTERSON, B.A., GELBER, R.D., GOLDHIRSCH, A., PRICE, K.N., SAVE-SODERBERGH, J., ANBAZHAGAN, R., STYLES, J., RUDENSTAM, C.-M., GOLUH, R., REED, R., MARTINEZ-TELLO, F., TILTMAN, A., TORHORST, J., GRIGOLATO, P., BETTLEHEIM, R., NEVILLE, A.M., BURKI, K., CASTIGLIONE, M., COLLINS, J., LINDTNER, J. & SENN, H.-J. FOR THE INTERNATIONAL LUDWIG BREAST CANCER STUDY GROUP (1992). Prognostics importance of c-erb 0-2 expression in breast cancer. J. Clin. Oncol., 10, 1049-1056.
27. HARRELL, F.E., LEE, K.L., MATCHAR, D.B. & REICHERT, T.A. (1985). Regression models for prognostic prediction: advantages, problems, and suggested solutions. Cancer Treat. Rep., 69, 1071-1077.
28. HASTIE, T., SLEEPERS, S. & TIBSHIRANI, R. (1992). Flexible co-variate effects in the proportional hazards model. Breast Cancer Res. Treat., 22, 241-250.
29. HAUCK, W.W. & MIIKE, R. (1991). A proposal for examining and reporting stepwise regressions. Stat. Med., 10, 711-715.
30. HILSENBECK, S.G., CLARK, G.M. & MCGUIRE, W.L. (1992). Why do so many prognostic factors fail to pan out? Breast Cancer Res. Treat., 22, 197-206.
31. KORN, E.L. & SIMON, R. (1990). Measures of explained variations for surival data. Stat. Med., 9, 487-503.
32. LEVINE, M.N., BROWMAN, G.P., GENT, M., ROBERTS, R. & GOODYEAR, M. (1991). When is a prognostic factor useful? A guide for the perplexed. J. Clin. Oncol, 9, 348-356.
33. LIPPMAN, M E., ALLEGRA, J.C., THOMPSON, E.B. & 7 others (1978). Lack of estrogen receptor is associated with an increased response rate to cytotoxic chemotherapy in metastatic breast cancer. N. Engl. J. Med., 298, 1223-1228.
34. MCGUIRE, W.L. (1991). Breast cancer prognostic factors: evaluation guidelines. J. Natl Cancer Inst., 83, 154-155.
35. O’REILLY, S.M. & RICHARDS, M.A. (1992). Is DNA flow cytometry a useful investigation in breast cancer? Eur. J. Cancer, 28, 504-507.
36. PALMER, M.K., HANN, I.M., JONES, P.M. & EVANS, D.I.K. (1980). A score at diagnosis for predicting length of remission in childhood acute lymphoblastic leukemia. Br. J. Cancer, 42, 841-849.
37. PETO, R. (1982). Statistical aspects of cancer trials. In Treatment of Cancer, Halnam, K.E. (ed.) Chapman & Hall: London.
38. RAWSON, N.S.B. & PETO, J. (1990). An overview of prognostic factors in small cell lung cancer. Br. J. Cancer, 61, 597-604.
39. ROYSTON, P. & ALTMAN, D.G. (1994). Regression using fractional polynomials of continuous covariates: parsimonious parametric modelling. Appl. Stat. (in press).
40. SCHMOOR, C., ULM, K. & SCHUMACHER, M. (1993). Comparison of the Cox model and the regression tree procedure in analyzing a randomized clinical trial. Statistics in Medicine, 12, 2351-2366.
41. SCHOENFELD, D. (1981). The asymptotic properties of nonparametric tests for comparing survival distributions. Biometrika, 68, 316-319.
42. SHIPP, M.A., HARRINGTON, D., ANDERSON, J., ARMITAGE, J., BONNADONNA, G., BRTTINGER, G., CABANILLAS, F., CANNELLOS, G., COIFFIER, B., CONNORS, J., COWAN, R., CROWTHER, D., ENGELHARD, M., FISHER, R., GISSELBRECHT, C., HORNING, S., LEPAGE, E., LISTER, A., NEERWALDT, J., MONTSERRAT, E., NISSEN, N., OKEN, N., PETERSON, B., TONDINI, C., VELASQUEZ, W. & YEAP, B. (1992). Development of a predictive model for aggressive lymphoma: the international non-Hogkin’s lymphoma prognostic factors project. Proc. Am. Soc. Clin. Oncol., 11, 319.
43. SIMON, R. (1982). Patient subsets and variation in therapeutic efficacy. Br. J. Clin. Pharmacol., 14, 473-482.
44. SIMON, R. (1986). Confidence limits for reporting results of clinical trials. Ann. Intern. Med., 105, 429-435.
45. STEYERBERG, E.W., KEIZER, H.J., ZWARTENDIJK, J., VAN RIJK, G.L., VAN GROENINGEN, C.J., HABBEMA, J.D.F. & STOTER, G. (1993). Prognosis after resection of residual masses following chemotherapy for metastatic nonseminomatous testicular cancer: a multivariate analysis. Br. J. Cancer, 68, 195-200.
46. TUKEY, J.W. (1977). Some thoughts on clinical trials, especially problems of multiplicity. Science, 198, 679-684.