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Appendix C
Selected Bibliography on Small Clinical Trials
This bibliography is a selection of published references dealing with analytical approaches and statistical methods applicable to clinical trials, particularly small clinical trials. The citations span the field broadly and are the result of searches of the Medline database and suggestions from the committee and from the experts in the field who made presentations at the committee's invitational workshop. The references are organized into specific categories to aid the reader. Although comprehensive, the list is selective, and the committee believes that it is an up-to-date bibliography that will assist researchers in learning more about design and analytical methods applicable to small clinical trials.
GENERAL
Bohning, D. 1999. Computer-Assisted Analysis of Mixtures and Applications: MetaAnalysis, Disease Mapping, and Others. Boca Raton, FL: Chapman & Hall/CRC.
Cai, J., H. Zhou, and C. E. Davis. 1997. Estimating the mean hazard ratio parameters for clustered survival data with random clusters. Statistics in Medicine 16:2009–2020.
D'Agostino, R. B., and H. Kwan. 1995. Measuring effectiveness. What to expect without a randomized control group. Medical Care 33 (Suppl. 4):AS95–AS105.
Davis, C. E. 1997. Secondary endpoints can be validly analyzed, even if the primary endpoint does not provide clear statistical significance. Controlled Clinical Trials 18:557–560.
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Davis, C. E., S. Hunsberger, D. M. Murray, R. R. Fabsitz, J. H. Himes, L. K. Stephenson, B. Caballero, and B. Skipper. 1999. Design and statistical analysis for the Pathways study. American Journal of Clinical Nutrition 69(Suppl. 4):S760–S763.
Dieterich, M., S. N. Goodman, R. R. Rojas-Corona, A. B. Emralino, J, D, Jimenez, and M. E. Sherman. 1994. Multivariate analysis of prognostic features in malignant pleural effusions from breast cancer patients. Acta Cytologica 38:945–952.
Everitt, B. S. 1994. Statistical Methods in Medical Investigations. New York: Halsted Press.
Everitt, B. S. 1995. The Cambridge Dictionary of Statistics in the Medical Sciences. New York: Cambridge University Press.
Gehan, E. A. 1965. A generalized two-sample Wilcoxon test for doubly censored data. Biometrika 52:650–653.
Gehan, E. A., and N. A. Lemak. 1994. Statistics in Medical Research: Developments in Clinical Trials. New York: Plenum Medical Book Co.
Gibbons, J. D., I. Olkin, and M. Sobel. 1999. Selecting and Ordering Populations: A New Statistical Methodology. Philadelphia: SIAM.
Gilbert, P. B., S. G. Self, and M. A. Ashby. 1998. Statistical methods for assessing differential vaccine protection against human immunodeficiency virus types. Biometrics 54:799–814.
Goodman, S. N. 1992. A comment on replication, p-values and evidence. Statistics in Medicine 11:875–879.
Goodman, S. N. 1993. P values, hypothesis tests, and likelihood: implications for epidemiology of a neglected historical debate. American Journal of Epidemiology 137:485–496; (Discussion, 137:497–501).
Goodman, S. N. 1994. Confidence limits vs power, calculations. Epidemiology 5:266– 268. (Discussion, 5:268–269.
Goodman, S. N. 1999. Toward evidence-based medical statistics. 1. The P value fallacy. Annals of Internal Medicine 130:995–1004.
Goodman, S. N. 1999. Toward evidence-based medical statistics. 2. The Bayes factor. Annals of Internal Medicine 130:1005–1013.
Goodman, S. N., and J. A. Berlin. 1994. The use of predicted confidence intervals when planning experiments and the misuse of power when interpreting results. Annals of Internal Medicine 121:200–206.
Goodman, S. N., D. G. Altman, and S. L. George. 1998. Statistical reviewing policies of medical journals: caveat lector? Journal of General Internal Medicine 13:753–756.
Halloran, M. E., and D. Berry, eds. 2000. Statistical Models in Epidemiology, the Environment, and Clinical Trials. The Ima Volumes in Mathematics and Its Applications, Vol. 116. New York: Springer-Verlag.
Hoppensteadt, F. C., and C. S. Peskin. 1992. Mathematics in Medicine and the Life Sciences. New York: Springer-Verlag.
Ildstad, S. T., D. J. Tollerud, M. E. Bigelow, and J. P. Remensnyder. 1989. A multivariate analysis of determinants of survival for patients with squamous cell carcinoma of the head and neck. Annals of Surgery 209:237–41.
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Epidemiology. Monographs in Epidemiology and Biostatistics,
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Last, J. ed. 1995. A Dictionary of Epidemilogy (Edited for the International Epidemiological Association). New York: Oxford University Press.
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Levin, B. 1987. Conditional likelihood analysis in stratum-matched retrospective studies with polytomous disease states. Communications in Statistics 16:699–718.
Levin, B. 1990. The saddlepoint correction in conditional logistic likelihood analysis. Biometrika 77:275–285.
Levin, B., and F. Kong. 1990. Barlett's bias correction to the profile score function is a saddlepoint correction. Biometrika 77:219–221.
Levin, B., and H. Robbins. 1981. Selecting the highest probability in binomial or multinomial trials. Proceedings of the National Academy of Sciences USA 78:4663– 4666.
Liang, K. Y., S. G. Self, K. J. Bandeen-Roche, and S. L. Zeger. 1995. Some recent developments for regression analysis of multivariate failure time data. Lifetime Data Analysis 1:403–415.
McPherson, G. 1990. Statistical analysis: the statistician's view. Pp. 154–180 In: Statistics in Scientific Investigation. Its Basis, Application, and Interpretation. New York: Springer-Verlag.
McPherson, G. 1990. Studying association and correlation. Pp. 475–508 In: Statistics in Scientific Investigation. Its Basis, Application, and Interpretation. New York: Springer-Verlag.
Meinert, C. L., 1986. Clinical Trials: Design, Conduct, and Analysis. New York: Oxford University Press.
Murray, D. M. 1998. Design and Analysis of Group-Randomized Trials. Monographs in Epidemiology and Biostatistics, Vol. 29. New York: Oxford University Press.
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Petitti, D. B. 1994. Meta-Analysis, Decision Analysis, and Cost-Effectiveness Analysis: Methods for Quantitative Synthesis in Medicine. New York: Oxford University Press.
Pham, B., A. Cranney, M. Boers, A. C. Verhoeven, G. Wells, and P. Tugwell. 1999. Validity of area-under-the-curve analysis to summarize effect in rheumatoid arthritis clinical trials. Journal of Rheumatology 26:712–716.
Racine-Poon, A., and J. Wakefield. 1998. Statistical methods for population pharmacokinetic modelling. Statistical Methods in Medical Research 7:63–84.
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Sevin, S. 1996. Statistical Analysis of Epidemiologic Data. Monographs in Epidemiology and Biostatistics, Vol. 25. New York: Oxford University Press.
Sevin, S. 1998. Modern Applied Biostatistical Methods: Using S-Plus. Monographs in Epidemiology and Biostatistics, Vol. 28. New York: Oxford University Press.
Stroup, D. F. 1999. Symposium on Statistical Bases for Public Health Decision Making: From Exploration to Modelling. Closing remarks. Statistics in Medicine 18:3373– 3375.
Tersmette, A. C., S. N. Goodman, G. J. Offerhaus, K. W. Tersmette, F. M. Giardello, J. P. Vandenbroucke, and G. N. Tytgat. 1991. Multivariate analysis of the risk of stomach cancer after ulcer surgery in an Amsterdam cohort of postgasterectomy patients. American Journal of Epidemiology 134:14–21.
Weinstein, G. S., and B. Levin. 1989. The effect of cross-over on the statistical power of randomized studies. Annals of Thoracic Surgery 48:490–495.
Weiss, N. S. 1996. Clinical Epidemiology: The Study of the Outcome of Illness. Monographs in Epidemiology and Biostatistics, Vol. 27. New York: Oxford University Press.
Zeger, S. L. 1991. Statistical reasoning in epidemiology. American Journal of Epidemiology 134:1062–1066.
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Zeger, S. L. and J. Katz. 1994. Estimation of design effects in cluster surveys. Annals of Epidemiology 4:295–301.
Zeger, S. L., and K.Y. Liang. 1993. Regression analysis for correlated data. Annual Review of Public Health 14:43–68.
Zeger, S. L., and A. Sommer. 1991. On estimating efficacy from clinical trials. Statistics in Medicine 10:45–52.
Zeger, S. L., L. C. See, and P. J. Diggle. 1989. Statistical methods for monitoring the AIDS epidemic. Statistics in Medicine 8:3–21.
Zeger, S. L., C. B. Hall, and K. J. Bandeen-Roche. 1996. Adjusted variable plots for Cox's proportional hazards regression model. Lifetime Data Analysis 2:73–90.
Zeger, S. L., D. Thomas, F. Dominici, J. M. Samet, J. Schwartz, D. Dockery, and A. Cohen. 2000. Exposure measurement error in time-series studies of air pollution: concepts and consequences. Environmental Health Perspective 108:419–426.
SAMPLE SIZE
Brunier, H. C., and J. Whitehead. 1994. Sample sizes for phase II clinical trials derived from Bayesian decision theory. Statistics in Medicine 13:2493–2502.
Flournoy, N., and I. Olkin. 1995. Do small trials square with large ones? The Lancet 345:741–742.
Gehan, E. A. 1961. The determination of the number of patients required in a preliminary and a follow-up trial of a new chemotherapeutic agent. Journal of Chronic Disease 13:346–353.
Gould, A. L. 1995. Planning and revising the sample size for a trial. Statistics in Medicine 14:1039–1051.
Hanfelt, J. J., R. S. Slack, and E. A. Gehan. 1999. A modification of Simon's optimal design for phase II trials when the criterion is median sample size. Controlled Clinical Trials 20:555–566.
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Kim, K., and D. L. DeMets. 1992. Sample size determination for group sequential clinical trials with immediate response. Statistics in Medicine 11:1391–1399.
McMahon, R. P., M. Proschan, N. L. Geller, P. H. Stone, and G. Sopko. 1994. Sample size calculation for clinical trials in which entry criteria and outcomes are counts of events. Statistics in Medicine 13:859–870.
Mehta, C. R., N. R. Patel, and P. Senchaudhuri. 1998. Exact power and sample-size computations for the Cochran-Armitage trend test. Biometrics 54:1615–1621.
Rochon, J. 1998. Application of GEE procedures for sample size calculations in repeated measures experiments. Statistics in Medicine 17:1643–1658.
Simon, R. M., and R. W. Makuch. 1982. Sample size requirements for comparing time-to-failure among k treatment groups. Journal of Chronic Disease 35:861–867.
Stallard, N. 1998. Sample size determination for phase II clinical trials based on Bayesian decision theory. Biometrics 54:279–294.
Wels, G., A. Cranney, B. Shea, and P. Tugwell. 1997. Responsiveness of endpoints in osteoporosis clinical trials. Journal of Rheumatology 24:1230–1233.
Whitehead, J. 1985. Designing phase II studies in the context of a programme of clinical research. Biometrics 41:373–383.
Whitehead, J. 1986. Sample sizes for phase II and phase III clinical trials: an integrated approach. Statistics in Medicine 5(5):459-464.
Whitehead, J. 1996. Sample sizes calculations for ordered categorical data. Statistics in Medicine 15:1065–1066.
Whitehead, J., K. Bolland, and M.R. Sooriyarachchi. 1998. Sample size review in a head injury trial with ordered categorical responses. Statistics in Medicine 17:2835–2847.
Wolfe, R., and J. B. Carlin. 1999. Sample-size calculation for log-transformed outcome measure. Controlled Clinical Trials 20:547–554.
CLINICAL TRIALS
Begg, C., M. Cho, S. Eastwood, R. Horton, D. Moher, I. Olkin, R. Pitkin, D. Rennie, K. F. Schulz, D. Simel, and D. F. Stroup. 1996. Improving the quality of reporting of randomized controlled trials. The CONSORT statement. Journal of the American Medical Association 276:637–639.
Benson, K., and A. J. Hartz. 2000. A comparison of observational studies and randomized, controlled trials. New England Journal of Medicine 342:1878–1886.
Black, H. R., W. J. Elliott, J. D. Neaton, G. Grandits, P. Grambsch, R. H. Grimm, Jr., L. Hansson, Y. Lacouciere, J. Muller, P. Sleight, M. A. Weber, W. B. White, G. Williams, J. Wittes, A. Zanchetti, and T. D. Fakouhi. 1998. Rationale and design for the Controlled Onset Verapamil Investigation of Cardiovascular Endpoints (CONVINCE) trial. Controlled Clinical Trials 19:370–390.
Bombardier, C., and P. Tugwell. 1985. Controversies in the analysis of long term clinical trials of slow acting drugs. Journal of Rheumatology 12:403–405.
Buyse, M., S. L. George, S. Evans, N. L. Geller, J. Ranstam, B. Scherrer, E. Lesaffre, G. Murray, L. Edler, J. Hutton, T. Colton, P. Lachenbruch, and B. L. Verma. 1999. The role of biostatistics in the prevention, detection and treatment of fraud in clinical trials. Statistics in Medicine 18:3435–3451.
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observational studies, and the hierarchy of research designs. New England Journal of Medicine 342:1887–1892.
Cranney, A., P. Tugwell, S. Cummings, P. Sambrook, J. Adachi, A. J. Silman, W. J. Gillespie, D. T. Felson, B. Shea, and G. Wells. 1997. Osteoporosis clinical trials endpoints: candidate variables and clinimetric properties. Journal of Rheumatology 24:1222–1229.
DeMets, D. L. 1987. Practical aspects in data monitoring: a brief overview. Statistics in Medicine 6:753–760.
DeMets, D. L. 1990. Methodological issues in AIDS clinical trials. Data monitoring and sequential analysis—an academic perspective. Journal of Acquired Immune Deficiency Syndrome 3(Suppl. 2):S124–S133.
DeMets, D. L. 1997. Distinction between fraud, bias, errors, misunderstanding, and incompetence. Controlled Clinical Trials 18:637–650. (Discussion, 661–666.)
DeMets, D. L., and M. Halperin. 1982. Early stopping in the two-sample problem for bounded random variables. Controlled Clinical Trials 3:1–11.
DeMets, D. L., and C. L. Meinert. 1991. Data integrity. Controlled Clinical Trials 12:727– 730.
DeMets, D. L., S. J. Pocock, and D. G. Julian. 1999. The agonizing negative trend in monitoring clinical trials. The Lancet 354:1983–1988.
Elliott, T. E., R. P. Dinapoli, J. R. O'Fallon, J. E. Krook, J. D. Earle, R. F. Morton, R. Levitt, L. K. Tschetter, B. W. Scheithauer, D. M. Pfeifle, D. I. Twito, and R. A. Nelimark. 1997. Randomized trial of radiation therapy (RT) plus dibromodulcitol (DBD) versus RT plus BCNU in high grade astrocytoma. Journal of Neurooncology 33:239–250.
Estey, E. 1997. Prognostic factors in clinical cancer trials. Clinical Cancer Research 3:2591–2593.
Fine, J., J. Duff, R. Chen, W. Hutchison, A. M. Lozano, and A. E. Lang. 2000. Long-term follow-up of unilateral pallidotomy in advanced Parkinson's disease. New England Journal of Medicine 342:1708–1714.
Finkelstein, M. O., B. Levin, and H. Robbins. 1996. Clinical and prophylactic trials with assured new treatment for those at greater risk. I. A design proposal. American Journal of Public Health 86:691–695.
Finkelstein, M. O., B. Levin, and H. Robbins. 1996. Clinical and prophylactic trials with assured new treatment for those at greater risk: II. Examples. American Journal of Public Health 86:696–705.
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Fleming, T. R. 1994. Surrogate markers in AIDS and cancer trials. Statistics in Medicine 13:1423–1435. (Discussion 13:1437–1440).
Fleming, T. R. 1999. Issues in the design of clinical trials: insights from the trastuzumab (Herceptin) experience. Seminar in Oncology 26(4 Suppl. 12):102–107.
Fleming, T. R. 2000. Design and interpretation of equivalence trials. American Heart Journal 139:171–176.
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Fleming, T. R., and D. L. DeMets. 1993. Monitoring of clinical trials: issues and recommendations. Controlled Clinical Trials 14:183–197.
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Fleming, T. R., S. J. Green, and D. P. Harrington. 1984. Considerations for monitoring and evaluating treatment effects in clinical trials. Controlled Clinical Trials 5:55–66.
Fleming, T. R., R. L. Prentice, M. S. Pepe, and D. Glidden. 1994. Surrogate and auxiliary endpoints in clinical trials, with potential applications in cancer and AIDS research. Statistics in Medicine 13:955–968.
Fleming, T. R., J. D. Neaton, A. Goldman, D. L. DeMets, C. Launer, J. Korvick, and D. Abrams. 1995. Insights from monitoring the CPCRA didanosine/xalcitabine trial. Journal of Acquired Immune Deficieney Syndrome. 10 (Suppl. 2):S9–S18.
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Foote, L. R., C. L. Loprinzi, A. R. Frank, J. R. O'Fallon, S. Gulavita, H. H. Tewfik, M. A. Ryan, J. M. Earle, and P. Novotny. 1994. Randomized trial of a chlorhexidine mouthwash for alleviation of radiation-induced mucositis. Journal of Clinical Oncology 12:2630–2633.
Gazarian, M., P. Tugwell, M. Boers, C. Bombardier, P. Brooks, R. Day, V. Strand, and G. Wells. 1999. Patient based methods for assessing adverse events in clinical trials in rheumatology. Progress Report for OMERACT Drug Toxicity Working Party Outcome Measures in Rheumatology. Journal of Rheumatology 26:207–209.
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Goldberg, R. M., C. L. Loprinzi, J. A. Mailliard, J. R. O'Fallon, J. E. Krook, C. Ghosh, R. D. Hestorff, S. F. Chong, N. F. Reuter, and T. G. Shanahan. 1995. Pentoxifylline for treatment of cancer anorexia and cachexia? A randomized, double-blind, placebo-controlled trial. Journal of Clinical Oncology 13:2856–2859.
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Keyserling, T. C., A. S. Ammerman, C. E. Davis, M. C. Mok, J. Garrett, and R. Simpson, Jr. 1997. A randomized controlled trial of a physician-directed treatment program for low-income patients with high blood cholesterol: the Southeast Cholesterol Project. Archives of Family Medicine 6:135–145.
Knatterud, G. L., F. W. Rockhold, S. L. George, F. B. Barton, C. E. Davis, W. R. Fairweather, T. Honohan, R. Mowery, and R. O'Neill. 1998. Guidelines for quality assurance in multicenter trials: a position paper. Controlled Clinical Trials 19:477– 493.
Lazaridis, E. N. and R. Gonin. 1997. Continuously monitored stopping boundary methodologies: the issues of sensitivity, association and trial suspension. Statistics in Medicine 16:1925–1941.
Lin, D. Y., T. R. Fleming, and V. De Grutolla. 1997. Estimating the proportion of treatment effect explained by a surrogate marker. Statistics in Medicine 16:1515– 1527.
Loprinzi, C. L., L. M. Athman, C. G. Kardinal, J. R. O'Fallon, J. A. See, B. K. Bruce, A. M. Dose, A. W. Miser, P. S. Kern, L. K. Tschetter, and S. Rayson. 1996. Randomized trial of dietician counseling to try to prevent weight gain associated with breast cancer adjuvant chemotherapy. Oncology 53:228–232.
Loprinzi, C. L., S. A. Kuross, J. T. O'Fallon, D. H. Gesme, Jr., J. B. Gerstner, R. M. Rospond, C. D. Cobau, and R. M. Goldberg. 1994. Randomized placebo-controlled trial evaluation of hydrazine sulfate in patients with colorectal cancer. Journal of Clinical Oncology 12:1121–1125.
Mahe, C., and S. Chevret. 1999. Estimation of the treatment effect in a clinical trial when recurrent events define the endpoint. Statistics in Medicine 18:1821–1829.
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Morgan, J. M., D. M. Capuzzi, J. R. Guyton, R. M. Centor, R. Goldberg, D. C. Robbins, D. DiPette, S. Jenkins, and S. Marcovina. 1996. Treatment effect of Niaspan, a controlled-release niacin, in patients with hypercholesterolemia: a placebo-controlled trial. Journal of Cardiovascular Pharmacology and Therapeutics 1:195–202.
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Neaton, J. D., and G. E. Bartsch. 1992. Impact of measurement error and temporal variability on the estimation of event probabilities for risk factor intervention trials. Statistics in Medicine 11:1719–1729.
Neaton, J. D., A. G. Duchene, K. H. Svendsen, and D. Wentworth. 1990. An examination of the efficiency of some quality assurance methods commonly employed in clinical trials. Statistics in Medicine 9:115–123. (Discussion, 124.)
Neaton, J. D., G. E. Bartsch, S. K. Broste, J. D. Cohen, and N. M. Simon. 1991. A case of data alteration in the Multiple Risk Factor Intervention Trial (MRFIT). Controlled Clinical Trials 12:731–740.
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Shapiro, A. M. J., J. R. T. Lakey, E. A. Ryan, G. S. Korbutt, E. Toth, G. L. Warnock, N. M. Kneteman, and R. V. Rajotte. 2000. Islet transplantation in seven patients with type I diabetes mellitus using a glucocorticoid-free immunosuppressive regimen. New England Journal of Medicine 343:230–238
Sharples, K., T. R. Fleming, S. MacMahon, A. Moore, I. Reid, and B. Scoggins. 1998. Monitoring clinical trials. New Zealand Medical Journal 111:322–325.
Shen, Y., and T. R. Fleming. 1999. Assessing effects on long-term survival after early termination of randomized trials. Lifetime Data Analysis 5:55–66.
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safety and efficacy in single-arm clinical trials. Journal of Clinical Oncology 14:296– 303.
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Whitehead, J. 1993. The case for frequentism in clinical trials. Statistics in Medicine 12:1405–1413. (Discussion, 12:1415–1419.)
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PHASE III CLINICAL TRIALS
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BAYESIAN APPROACH
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