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Page 177 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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Page 178 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. Jennison, C., and B. W. Turnbull. 1983. Confidence intervals for a binomial parameter following a multistage test with application to MIL-STD 105D and medical trials. Technometrics 25:49–58. Khoury, M. J., T. H. Beaty, and B. H. Cohen. 1993. Fundamentals of Genetic
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Page 179 Epidemiology. Monographs in Epidemiology and Biostatistics, Vol. 22. New York: Oxford University Press. Knapp, R. G., and M. C. Miller. 1992. Clinical Epidemiology and Biostatistics (The National Medical Series for Independent Study). Baltimore: Williams & Wilkins. Kramer, M. S. 1988. Clinical Epidemiology and Biostatistics: A Primer for Clinical Investigators and Decision-Makers. New York: Springer-Verlag. Last, J. ed. 1995. A Dictionary of Epidemilogy (Edited for the International Epidemiological Association). New York: Oxford University Press. Lazaridis, E., and R. Gonin. 1997. Continuously monitored stopping boundary methodologies: The issues of sensitivity, association and trial suspension. Statistics in Medicine 16:1925–1941. Levin, B. 1986. Empirical Bayes' estimation in heterogeneous matched binary samples with systematic aging effects. In: Adaptive Statistical Procedure and Related Topics. J. van Ryzin, ed. Institute of Mathematics and Statistics Lecture Notes—Monograph Series 8:179–194. 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. O'Quigley, J., and L. Z. Shen. 1996. Continual reassessment method: a likelihood approach. Biometrics 52:673–684. 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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Page 180 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. Zeger, S. L., and S. D. Harlow. 1987. Mathematical models from laws of growth to tools for biologic analysis: fifty years of “Growth”. Growth 51:1–21. 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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Page 181 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. Concato, J., N. Shah, and R. I. Horwitz. 2000. Randomized, controlled trials,
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Page 182 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. Fleming, T. R. 1990. Evaluation of active control trials in AIDS. Journal of Acquired Immune Deficiency Syndrome 3(Suppl. 2):S82–S87. 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. Fleming, T. R., V. DeGruttola, and D. L. DeMets. 1998. Surrogate endpoints. In: AIDS Clinical Review 1997/1998. P. A. Volberding and M. A. Jacobson, eds. New York: Marcel Dekker.
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Page 183 Fleming, T. R., and D. L. DeMets. 1993. Monitoring of clinical trials: issues and recommendations. Controlled Clinical Trials 14:183–197. Fleming, T. R., and D. L. DeMets. 1996. Surrogate endpoints in clinical trials: are we being misled? Annals of Internal Medicine 125:605–613. Fleming, T. R., and S. J. Green. 1988. Guidelines for the reporting of clinical trials. Seminars in Oncology 15:455–461. 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. Fleming, T. R., and L. F. Watelet. 1989. Approaches to monitoring clinical trials. Journal of the National Cancer Institute 81:188–193. 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. Gehan, E. A. 1978. Comparative clinical trials with historical controls: a statistician's view. Biomedicine 28:13–19. Gehan, E. A. 1982. Design of controlled clinical trials: use of historical controls. Cancer Treatment Reports 66:1089–1093. Gehan, E. A. 1986. Update on planning of Phase II clinical trials. Drugs Under Experimental and Clinical Research 12:43–50. Gehan, E. A. 1988. Methodological issues in cancer clinical trials: the comparison of therapies. Biomedicine and Pharmacotherapy 42:161–165. Gehan, E. A. 1997. The scientific basis of clinical trials: statistical aspects. Clinical Cancer Research 3(12 Pt 2):2587–2590. Gehan, E. A., and E. J. Freireich. 1981. Cancer clinical trials. A rational basis for use of historical controls. Seminars in Oncology 8:430–436. Gehan, E. A., and M. A. Schneiderman. 1990. Historical and methodological developments in clinical trials at the National Cancer Institute. Statistics in Medicine 9:871–880. (Discussion, 903–906.) Gehan, E. A., L. G. Ensign, D. S. Kamen, and P. F. Thall. 1994. An optimal three-stage design for phase II clinical trials. Statistics in Medicine 13:1727–1736. 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. Green, L. A., F. S. Rhame, R. W. Price, D. C. Perlman, L. G. Capps, J. H. Sampson, L. R. Deyton, S. M. Schnittman, E. J. Fisher, G. E. Bartsch, E. A. Krum, and J. D. Neaton.
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Page 184 1998. Experience with a cross-study endpoint review committee for AIDS clinical trials. AIDS 12:1983–1990. Heitjan, D. F. 1999. Ignorability and bias in clinical trials. Statistics in Medicine 18:2421– 2434. Herson, J. 1979. Predictive probability early termination plans for phase II clinical trials. Biometrics 35:775–783. Herson, J., and S. K. Carter. 1986. Calibrated phase II clinical trials in oncology. Statistics in Medicine 5:441–447. Institute of Medicine. 1990. Modern Methods of Clinical Investigation. Washington, DC: National Academy Press. Kessler, D. A., J. P. Siegel, P. D. Noguchi, K. C. Zoon, K. L. Feiden, and J. Woodcock. 1993. Regulation of somatic-cell therapy and gene therapy by the Food and Drug Administration. New England Journal of Medicine 329:1169–1173. 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. Martenson, J. A., Jr., G. Hyland, C. G. Moertel, J. A. Mailliard, J. R. O'Fallon, R. T. Collins, R. F. Morton, H. H. Tewfik, R. L. Moore, A. R. Frank, R. E. Urias, and R. L. Deming. 1996. Olsalazine is contraindicated during pelvic radiation therapy: results of a double-blind, randomized clinical trial. International Journal of Radiation Oncology, Biology and Physics 35:299–303. Matthews, J. N. S. 1995. Small clinical trials: are they all bad? Statistics in Medicine 14:115-126. May, G. S., D. L. DeMets, L. M. Friedman, C. Furberg, and E. Passamani. 1981. The randomized clinical trial: bias in analysis. Circulation 64:669–673. Moher, D., A. R. Jadad, and P. Tugwell. 1996. Assessing the quality of randomized
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Page 185 controlled trials. Current issues and future directions. International Journal of Technology Assessment and Health Care 12:195–208. 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. Mosteller, F. 1996. The promise of risk-based allocation trials in assessing new treatments. American Journal of Public Health 86:622–623. 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. Neaton, J. D., D. N. Wentworth, F. Rhame, C. Hogan, D. I. Abrams, and L. Deyton. 1994. Considerations in choice of a clinical endpoint for AIDS clinical trials. Statistics in Medicine 13:2107–2125. Pocock, S. J., and D. R. Elbourne. 2000. Randomized trials or observational tribulations? New England Journal of Medicine 342:1907–1909. Pocock, S. J., N. L. Geller, and A. A. Tsiatis. 1987. The analysis of multiple endpoints in clinical trials. Biometrics 43:487–498. Schaper, C., T. R. Fleming, S. G. Self, and W. N. Rida. 1995. Statistical issues in the design of HIV vaccine trials. Annual Review of Public Health 16:1–22. 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. Siegel, J. P. 2000. Equivalence and noninferiority trials. American Heart Journal 139:S166– S170. Sloan, J. A., C. L. Loprinzi, S. A. Kuross, A. W. Miser, J. R. O'Fallon, M. R. Mahoney, I. M. Heid, M. E. Bretscher, and N. L. Vaught. 1998. Randomized comparison of four tools measuring overall quality of life in patients with advanced cancer. Journal of Clinical Oncology 16:3662–3673. Sposto, R., and D. O. Stram. 1999. A strategic view of randomized trial design in low-incidence pediatric cancer. Statistics in Medicine 18:1183–1197. Thall, P. F., and R. M. Simon. 1995. Recent developments in the design of phase II clinical trials. In: Recent Advances in Clinical Trial Design and Analysis. P. F. Thall, ed. Boston: Kluwer. Thall, P. F., R. M. Simon, and E. H. Estey. 1996. New statistical strategy for monitoring
OCR for page 186
Page 186 safety and efficacy in single-arm clinical trials. Journal of Clinical Oncology 14:296– 303. Tsai, J. F., L.M. Li, and J. K. Chen. 2000. Reconstruction of damaged cornea by transplantation of autologous limbal epithelial cells. New England Journal of Medicine 343:86–93. Vico, L., P. Collet, A. Guignandon, M. H. Lafage-Proust, T. Thomas, M. Rehailia, and C. Alexandre. 2000. Effects of long-term microgravity exposure on cancellous and cortical weight-bearing bones of cosmonauts. The Lancet 355:1607–1611. Whitehead, J. 1993. The case for frequentism in clinical trials. Statistics in Medicine 12:1405–1413. (Discussion, 12:1415–1419.) Whitehead, J. 1999. Stopping a clinical trial properly. The Lancet 353:2164. (Comments, 353:943–944.) Whitehead, J. 2000. Monitoring and evaluating clinical trials data. Intensive Care Medicine 26 (Suppl. 1):S84–S88. Whitehead, J., and M. Mittlbock. 1998. The interpretation of clinical trials of immediate versus delayed therapy. Lifetime Data Analysis 4:253–263. Whitehead, J., D. G. Altman, M.K. Parmar, S.P. Stenning, P.M. Fayers, and D. Machin. 1995. Randomised consent designs in cancer clinical trials. European Journal of Cancer 31A:1934–1944. PHASE I CLINICAL TRIALS Ahn, C. 1998. An evaluation of phase I cancer clinical trial designs. Statistics in Medicine 17:1537–1549. Babb, J., A. Rogatko, and S. Zacks. 1998. Cancer phase I clinical trials: efficient dose escalation with overdose control. Statistics in Medicine 17:1103–1120. Berlin, J., J. A. Stewart, B. Storer, K. D. Tusch, R. Z. Arzoomanian, D. Alberti, C. Feierabend, K. Simon, and G. Wilding. 1998. Phase I clinical and pharmacokinetic trial of penclomedine using a novel, two-stage trial design for patients with advanced malignancy. Journal of Clinical Oncology 16:1142–1149. Durham, S. D., N. Flournoy, and W. F. Rosenberger. 1997. A random walk rule for phase I clinical trials. Bioemetrics 53:745–760. Geller, N. L. 1984. Design of phase I and II clinical trials in cancer: a statistician's view. Cancer Investigation 2:483–491. Goodman, S. N., M. L. Zahurak, and S. Piantadosi. 1995. Some practical improvements in the continual reassessment method for phase I studies. Statistics in Medicine 14:1149–1161. Gordon, N. H., and J. K. Willson. 1992. Using toxicity grades in the design and analysis of cancer phase I clinical trials. Statistics in Medicine 11:2063–2075. Hageboutros, A., A. Rogatko, E. M. Newman, C. McAleer, J. Brennan, F. P. LaCreta, G. R. Hudes, R. F. Ozols, and P. J. O'Dwyer. 1995. Phase I study of phosphonacetyl-l-aspartate, 5-fluorouracil, and leucovorin patients with advanced cancer. Cancer Chemotherapy and Pharmacology 35:205–212. Heyd, J. M., and B. P. Carlin. 1999. Adaptive design improvements in the continual reassessment method for phase I studies. Statistics in Medicine 18:1307–1321.
OCR for page 187
Page 187 Korn, E. L., D. Midthune, T. T. Chen, L. V. Rubinstein, M. C. Christian, and R. M. Simon. 1994. A comparison of two phase I trial designs. Statistics in Medicine 13:1799–1806. Kramar, A., A. Lebecq, and E. Candalh. 1999. Continual reassessment methods in phase I trials of the combination of the two drugs in oncology. Statistics in Medicine 18:1849–1864. Lin, H. M., and M. D. Hughes. 1995. Use of historical marker data for assessing treatment effects in phase I/II trials when subject selection is determined by baseline marker level. Biometrics 51:1053–1063. O'Dwyer, P. J., T. C. Hamilton, F. P. LaCreta, J. M. Gallo, D. Kilpatrick, T. Halbherr, J. Brennan, M. A. Bookman, J. Hoffman, R. C. Young, R. L. Comis, and R. F. Ozols. 1996. Phase I trial of buthionine sulfoximine in combination with melphalan in patients with cancer. Journal of Clinical Oncology 14:249–256. O'Quigley, J. 1992. Estimating the probability of toxicity at the recommended dose following a phase I clinical trial in cancer. Biometrics 48:853–862. O'Quigley, J., L. Z. Shen, and A. Gamst. 1999. Two-sample continual reassessment method. Journal of Biopharmaceutical Statistics 9:17–44. O'Quigley, J., M. Pepe, and L. Fisher. 1990. Continual reassessment method: a practical design for phase I clinical trials in cancer. Biometrics 46:33–48. Savarese, D. M., A. M. Denicoff, S. L. Berg, M. Hillig, S. P. Baker, J. A. O'Shauhnessy, C. Chow, G. A. Otterson, F. M. Balis, and D. G. Poplack. 1993. Phase I study of highdose piroxantrone with granulocyte colony-stimulating factor. Journal of Clinical Oncology 11:1795–1803. Simon, R., B. Freidlin, L. Rubinstein, S. G. Arbuck, J. Collins, and M. C. Christian. 1997. Accelerated titration designs for phase I clinical trials in oncology. Journal of the National Cancer Institute 89:1138–1147. Smith, T. L., J. J. Lee, H. M. Kantarjian, S. S. Legha, and M. N. Raber. 1996. Design and results of phase I cancer clinical trials: three-year experience at M. D. Anderson Cancer Center. Journal of Clinical Oncology 14:287–295. Storer, B. E. 1993. Small sample confidence sets for the MTD in phase I clinical trial. Biometrics 49:1117–1125. Thall, P. F., and K. T. Russell. 1998. A strategy for dose-finding and safety monitoring based on efficacy and adverse outcomes in phase I/II clinical trials. Biometrics 54:251–264. Thall, P. F., E. H. Estey, and H. G. Sung. 1999. A new statistical method for dose-finding based on efficacy and toxicity in early phase clinical trials. Investigational New Drugs 17:155–167. Thall, P. F., J. J. Lee, C. H. Cheng, and E. H. Estey. 1999. Accrual strategies for phase I trials with delayed patient outcome. Statistics in Medicine 18:1155–1169. PHASE III CLINICAL TRIALS Bellamy, N., J. Kirwan, M. Boers, P. Brooks, V. Strand, P. Tugwell, R. Altman, K. Brandt, M. Dougados, and M. Lequesne. 1997. Recommendations for a core set of outcome measures for future phase III clinical trials in knee, hip, and hand osteoarthritis. Consensus development at OMERACT III. Journal of Rheumatology 24:799–802.
OCR for page 190
Page 190 pharmacokinetic/pharmacodynamic models to dose determination. Statistics in Medicine 14:971–986. Whitehead, J., and H. Brunier. 1995. Bayesian decision procedures for dose determining experiments. Statistics in Medicine 14:885–893. Whitehead, J., S. Patterson, S. Francis, M. Ireson, and D. Webber. 1999. A novel Bayesian decision procedure for early-phase dose-finding studies. Journal of Biopharmaceutical Statistics 9:583–597. DECISION ANALYSIS Anis, A. H., P. Tugwell, G. A. Wells, and D. G. Stewart. 1996. A cost-effectiveness analysis of cyclosporine in rheumatoid arthritis. Journal of Rheumatology 23:609–616. Backhouse, M. E. 1998. An investment appraisal approach to clinical trial design. Health Economics 7:605–619. Balas, E. A., R. A. Kretschmer, W. Gnann, D. A. West, S. A. Boren, R. M. Centor, M. Nerlich, M. Gupta, T. D. West, and N. S. Soderstrom. 1998. Interpreting cost analyses of clinical interventions. Journal of the American Medical Association 279:54–57. Berry, D. A., M. C. Wolff, and D. Sack. 1994. Decision making during a phase III randomized controlled trial. Controlled Clinical Trials 15:360–378. Centor, R. M., and J. M. Witherspoon. 1982. Treating sore throats in the emergency room. The importance of follow-up in decision making. Medical Decision Making 2:463–469. Claxton, H. 1999. The irrelevance of inference: a decision-making approach to the stochastic evaluation of health care technologies. Journal of Health Economics 18:341–364. Heitjan, D. F., P. S. Houts, and H. A. Harvey. 1992. A decision-theoretic evaluation of early stopping rules. Statistics in Medicine 11:673–683. Heitjan, D. F., A. J. Moskowitz, and W. Whang. 1999. Bayesian estimation of cost-effectiveness ratios from clinical trials. Health Economics 8:191–201. Heudebert, G. R., R. Marks, C. M. Wilcox, and R. M. Centor. 1997. Choice of long term strategy for the management of patients with severe esophagitis: a cost-utility analysis. Gastroenterology 112:1078–1086. Heudebert, G. R., R. M. Centor, J. C. Klapow, R. Marks, L. Johnson, and C. M. Wilcox. 2000. What is heartburn worth? A cost-utility analysis of management strategies. Journal of General Internal Medicine 15:175–182. Hillner, B. E. and R. M. Centor. 1987. What a difference a day makes: a decision analysis of adult streptococcal pharyngitis. Journal of General Internal Medicine 2:244–250. Huster, W. J., and G. G. Enas. 1998. A framework establishing clear decision criteria for the assessment of drug efficacy. Statistics in Medicine 17:1829–1838. Jonsson, B., and P. E. Bebbington. 1994. What price depression? The cost of depression and the cost-effectiveness of pharmacological treatment. British Journal of Psychiatry 164:665–673. (applied decision analysis). Kattan, M. W., M. E. Cowen, and B. J. Miles. 1997. A decision analysis for treatment of clinically localized prostate cancer. Journal of General Internal Medicine 12:299– 305.
OCR for page 191
Page 191 Mennemeyer, S. T., and L. P. Cyr. 1997. A bootstrap approach to medical decision analysis. Journal of Health Economics 16:741–747. Midgette, A. S., J. B. Wong, J. R. Beshansky, A. Porath, C. Fleming, and S. G. Pauker. 1994. Cost-effectiveness of streptokinase for acute myocardial infarction: a combined meta-analysis and decision analysis of the effects of infarct location and of likelihood of infarction. Medical Decision Making 14:108–117. O'Connor, A. M., P. Tugwell, G. A. Wells, T. Elmslie, E. Jolly, G. Hollingworth, R. McPherson, H. Bunn, I. Graham, and E. Drake. 1998. A decision aid for women considering hormone therapy after menopause: decision support framework and evaluation. Patient Education Counseling 33:267–279. Pliskin, J. S. 1999. Towards better decision making in growth hormone therapy. Hormone Research 51 (Suppl. 1):30–35. Samsa, G. P., R. A. Reutter, G. Parmigiani, M. Ancukiewicz, P. Abrahamse, J. Lipscomb, and D. B. Matchar. 1999. Performing cost-effectiveness analysis by integrating randomized trial data with a comprehensive decision model: application to treatment of acute ischemic stroke. Journal of Clinical Epidemilogy 52:259–271. Siegel, J. E., M. C. Weinstein, L. B. Russell, and M. R. Gold. 1996. Recommendations for reporting cost-effectiveness analyses. Journal of the American Medical Association 276:1339–1341. Stallard, N., P. F. Thall, and J. Whitehead. 1999. Decision theoretic designs for phase II clinical trials with multiple outcomes. Biometrics 55:971–977. Stangl, D. K. 1995. Prediction and decision making using Bayesian hierarchical models. Statistics in Medicine 14:2173–2190. Tugwell, P. 1996. Economic evaluation of the management of pain in osteoarthritis. Drugs 52(Suppl. 3):48–58. Tugwell, P., K. J. Bennett, D. L. Sackett, and R. B. Haynes. 1985. The measurement iterative loop: a framework for the critical appraisal of need, benefits and costs of health interventions. Journal of Chronic Disease 38:339–351. Whitehead, J., and H. Brunier. 1995. Bayesian decision procedures for dose determining experiments. Statistics in Medicine 14:885-893. (Discussion, 895–899.) Whitehead, J., and D. Williamson. 1998. Bayesian decision procedures based on logistic regression models for dose-finding studies. Journal of Biopharmaceutical Statistics 8: 445–467. META-ANALYSIS Aitkin, M. 1999. Meta-analysis by random effect modelling in generalized linear models. Statistics in Medicine 18:2343–2351. Bass, E. B., N. R. Powe, S. N. Goodman, S. L. Graziano, R. I. Griffiths, T. S. Kickler, and J. R. Wingard. 1993. Efficacy of immune globulin in preventing complications of bone marrow transplantation: a meta-analysis. Bone Marrow Transplant 12:273–282. Berkeg C. S., A. Antczak-Bouckoms, D. C. Hoaglin, F. Mosteller, and B. L. Pihlstrom. 1995. Multiple-outcomes meta-analysis of treatments for periodontal disease. Journal of Dental Research 74:1030–1039. Berkey, C. S., D. C. Hoaglin, F. Mosteller, and G. A. Colditz. 1995. A random effects regression model for meta-analysis. Statistics in Medicine 14:395–411.
OCR for page 192
Page 192 Berkey, C. S., Hoaglin, D. C., A. Antczak-Bouckoms, F. Mosteller, and G. A. Colditz. 1998. Meta-analysis of multiple outcomes by regression with random effects. Statistics in Medicine 17:2537–2550. Boissel, J. P., J. Blanchard, E. Panak, J. C. Peyrieux, and H. Sacks. 1989. Considerations for the meta-analysis of randomized clinical trials. Controlled Clinical Trials 10:254– 281. Boutitie, F., F. Gueyffier, S. J. Pocock, and J. P. Boissel.1998. Assessing treatment-time interaction in clinical trials with time to event data: a meta-analysis of hypertension trials. Statistics in Medicine 17:2883–2903. Bravata, D. M., I. Olkin, A. E. Barnato, E. B. Keeffe, and D. K. Owens. 1999. Health related quality of life after liver transplantation: a meta-analysis. Liver Transplant and Surgery 5:318–331. Brown, S. A., and L. V. Hedges. 1994. Predicting metabolic control in diabetes: a pilot study using meta-analysis to estimate a linear model. Nursing Research 43:362–368. Brumback, B. A., L. B. Holmes, and L. M. Ryan. 1999. Adverse effects of chorionic villus sampling: a meta-analysis. Statistics in Medicine 18:2163–2175. Bucher, H. C., G. H. Guyatt, L. E. Griffith, and S. D. Walter. 1997. The results of direct and indirect treatment comparisons in meta-analysis of randomized controlled clinical trials. Journal of Clinical Epidemiology 50:683–691. Bucher, H. C., R. J. Cook, G. H. Guyatt, J. D. Lang, D. J. Cook, R. Hatala, and D. L. Hunt. 1996. Effects of dietary calcium supplementation on blood pressure. A meta-analysis of randomized controlled trials. Journal of the American Medical Association 275:1016–1022. Cappelleri, J. C., J. P. Ioannidis, C. H. Schmid, S. D. de Ferranti, M. Aubert, T. C. Chalmers, and J. Lau. 1996. Large trials vs meta-analysis of smaller trials: how do their results compare? Journal of the American Medical Association 276:1332–1338. Christensen, E., and C. Gluud. 1995. Glucocorticoids are ineffective in alcoholic hepatitis: a meta-analysis adjusting for confounding variables. Gut 37:113–118. Cook, R. J., and S. D. Walter 1997. A logistic model for trend in 2 x 2 kappa tables with applications to meta-analysis. Biometrics 53:352–357. Daniels, M. J., and M. D. Hughes. 1997. Meta-analysis for the evaluation of potential surrogate markers. Statistics in Medicine 16:1965–1982. DerSimonian, R. 1996. Meta-analysis in the design and monitoring of clinical trials. Statistics in Medicine 15:1237–1248. Dickersin, K., K. Higgins, and C. L. Meinert. 1990. Identification of meta-analyses. The need for standard terminology. Controlled Clinical Trials 11:52–66. Earle, C. C., B. Pham, and G. A. Wells. 2000. An assessment of methods to combine published survival curves. Medical Decision Making 20:104–111. Egger, M., G. Davey-Smith, C. Stettler, and P. Diem. 1997. Risk of adverse effects of intensified treatment in insulin-dependent diabetes mellitus: a meta-analysis. Diabetic Medicine 14:919–928. Farraz, M. B., P. Tugwell, C. H. Goldsmith, and E. Atra. 1990. Meta-analysis of sulfasalazine in ankolysing spondylitis. Journal of Rheumatology 17:1482–1486. Frost, C., R. Clarke, and H. Beacon. 1999. Use of hierarchical models for meta-analysis: experience in the metabolic ward studies of diet and blood cholesterol. Statistics in Medicine 18:1657–1676.
OCR for page 193
Page 193 Geller, N. L., and M. Proschan. 1996. Meta-analysis of clinical trials: a consumer's guide. Journal of Biopharmaceutical Statistics 6:377–394. Gloaguen, V., J. Cottraux, M. Cucherat, and I. M. Blackburn. 1998. A meta-analysis of the effects of cognitive therapy in depressed patients. Journal of Affective Disorder 49:59–72. Goodman, S. N. 1989. Meta-analysis and evidence. Controlled Clinical Trials 10:188– 204. Goodman, S. N. 1991. Have you ever meta-analysis you didn't like? Annals of Internal Medicine 114:244–246. Hasselblad, V., and D. C. McCrory. 1995. Meta-analytic tools for decision making: a practical guide. Medical Decision Making 15:81–96. Hasselblad, V., and L. V. Hedges. 1995. Meta-analysis of screening and diagnostic tests. Psychology Bulletin 117:167–178. Hauck, W. W., S. Anderson, and S. M. Marcus. 1998. Should we adjust for covariates in nonlinear regression analyses of randomized trials? Controlled Clinical Trials 19:249– 256. Hedges, L. V. 1984. Research synthesis: the state of the art. International Journal of Aging and Human Development 19:85–93. Hedges, L. V. 1997. Improving meta-analysis for policy purposes. NIDA Research Monograph 170:202–215. Hendrick, R. E., R. A. Smith, J. H. Rutledge III, and C. R. Smart. 1997. Benefit of screening mammography in women aged 40-49: a new meta-analysis of randomized controlled trials. Journal of the National Cancer Institute Monograph 22:87–92. Higgins, J. P., and A. Whitehead. 1996. Borrowing strength from external trials in a meta-analysis. Statistics in Medicine 15:2733–2749. Homik, J. E., A. Cranney, B. Shea, P. Tugwell, G. Wells, J. D. Adachi, and M. E. Suarez-Almazor. 1999. A meta-analysis on the use of biophosphonates in corticosteroid induced osteoporosis. Journal of Rheumatology 26:1148–1157. Hughes, E. G. 1997. The effectiveness of ovulation induction and intrauterine insemination in the treatment of persistent infertility: a meta-analysis. Human Reproduction 12:1865–1872. Ioannidis, J P., and J. Lau. 1997. The impact of high-risk patients on the results of clinical trials. Journal of Clinical Epidemiology 50:1089–1098. Ioannidis, J. P., and J. Lau. 1998. Heterogeneity of the baseline risk within patient populations of clinical trials: a proposed evaluation algorithm. American Journal of Epidemiology 148:1117–1126. Ioannidis, J. P., J. C. Cappelleri, P. R. Skolnik, J. Lau, and H. S. Sacks. 1996. A meta-analysis of the relative efficacy and toxicity of Pneumocystis carinii prophylactic regimens. Archives of Internal Medicine 156:177–188. Jadad, A. R., D. J. Cook, A. Jones, T. P. Klassen, P. Tugwell, M. Moher, and D. Moher. 1998. Methodology and reports of systematic reviews and meta-analyses: a comparison of Cochrane reviews with articles published in paper-based journals. Journal of the American Medical Association 280:278–280. Janicak, P. G., J. M. Davis, R. D. Gibbons, S. Ericksen, S. Chang, and P. Gallagher. 1985. Efficacy of ECT: a meta-analysis. American Journal of Psychiatry 142:297–302. Jones, D. R. 1995. Meta-analysis: weighing the evidence. Statistics in Medicine 14:137– 149.
OCR for page 194
Page 194 Kahn, J. G., B. J. Becker, L. MacIsaa, J. K. Amory, J. Neuhaus, I. Olkin, and M. D. Creinin. 2000. The efficacy of medical abortion: a meta-analysis. Contraception 61:29–40. Karpf, D. B., D. R. Shapiro, E. Seema, K. E. Ensrud, C. C. Jonhston, Jr., S. Adai, S. T. Harris, A. C. Santora II, L. J. Hirsch, L. Oppenheimer, and D. Thompson. 1997. Prevention of nonvertebral fractures by alendronate. A meta-analysis. Journal of the American Medical Association 277:1159–1164. Lau, J., J. P. Ioannidis, and C. H. Schmid.1998. Summing up evidence: one answer is not always enough. The Lancet 351:123–127. Linde, K., N. Clausius, G. Ramirez, D. Melchart, F. Eitel, L. V. Hedges, and W. B. Jonas. 1997. Are the clinical effects of homeopathy placebo effects? A meta-analysis of placebo-controlled trials. The Lancet 350:834–843. Lumley, T., and A. Keech. 1995. Meta-meta-analysis with confidence. The Lancet 346:576–577. Meinert, C. L. 1989. Meta-analysis: science or religion? Controlled Clinical Trials 10(4 Suppl.):257S–263S. Meynaud-Kraemer, L., C. Colin, P. Vergnon, and X. Barth. 1999. Wound infection in open versus laparocopic appendectomy. A meta-analysis. International Journal of Technology Assessment in Health Care 15:380–391. Moher, D., and I. Olkin. 1995. Meta-analysis of randomized controlled trials. A concern for standards. Journal of the American Medical Association 274:1962–1964. Moher, D., B. Pham, A. Jones, D. J. Cook, A. R. Jadad, M. Moher, P. Tugwell, T. P. Klassen. 1998. Does quality of reports of randomised trials affect estimates of intervention efficacy reported in meta-analyses? The Lancet 352:609–613. Moher, D., D. J. Cook, A. R. Jadad, and P. Tugwell, M. Moher, A. Jones, B. Pham, and T. P. Klassen. 1999. Assessing the quality of reports of randomized trials: implications for the conduct of meta-analyses. Health Technology Assessment 3:i–iv, 1-98. Moher, D., D. J. Cook, S. Eastwood, I. Olkin, D. Rennie, and D. F. Stroupe. 1999. Improving the quality of reports of meta-analyses of randomised controlled trials in the QUOROM statement. Quality of reporting of meta-analyses. The Lancet 354:1896–1900. Mushlin, A. I., R. W. Kouides, and D. E. Shapiro. 1998. Estimating the accuracy of screening mammography: a meta-analysis. American Journal of Preventive Medicine 14:143–153. Normand, S. L. 1999. Meta-analysis: formulating, evaluating, combining, and reporting. Statistics in Medicine 18:321–359. Olkin, I. 1994. Re: “A critical look at some popular meta-analytic methods. American Journal of Epidemiology 140:297–299. (Discussion, 140:300–301. Olkin, I. 1995. Meta-analysis: reconciling the results of independent studies. Statistics in Medicine 14:457–472. Olkin, I. 1995. Statistical and theoretical considerations in meta-analysis. Journal of Clinical Epidemiology 48:133–146. Olkin, I. 1996. Meta-analysis: current issues in research synthesis. Statistics in Medicine 15:1253–1257. Olkin, I. 1999. Diagnostic statistical procedures in medical meta-analyses. Statistics in Medicine 18:2331–2341.
OCR for page 195
Page 195 Olkin, I., and A. Sampson. 1998. Comparison of meta-analysis of variance of individual patient data. Biometrics 54:317–322. Parmar, M. K., L. A. Stewart, and D. G. Altman. 1996. Meta-analyses of randomised trials: when the whole is more than just the sum of the parts. British Journal of Cancer 74:496–501. Parmar, M. K., V. Torri, and L. Stewart. 1998. Extracting summary statistics to perform meta-analyses of the published literature for survival endpoints. Statistics in Medicine 17:2815–2834. Psaty, B. M., N. L. Smith, D. S. Siscovick, T. D. Koepsell, N. S. Weiss, S. R. Heckbert, R. N. Lemaitre, E. H. Wagner, and C. D. Furberg. 1997. Health outcomes associated with anti-hypertensive therapies used as first-line agents. A systematic review and analysis. Journal of the American Medical Association 277:739–745. Rosenthal, R. 1991. Meta-Analytic Procedures for Social Research. Applied Social Research Methods Series, Vol. 6. Newbury Park, CA: Sage Publications. Rothwell, P. M. 1995. Can overall results of clinical trials be applied to all patients? The Lancet 345: 1616–1619. Sankoh, A. J., M. Al-Osh, and M. F. Huque. 1999. On the utility of the Dirichlet distribution for meta-analysis of clinical studies. Journal of Biopharmaceutical Statistics 9:289–306. Schmid, C. H., J. Lau, M. W. McIntosh, and J. C. Cappelleri. 1998. An empirical study of the effect of the control rate as a predictor of treatment efficacy in meta-analysis of clinical trials. Statistics in Medicine 17:1923–1942. Schulz, K. F. 1995. Meta-analyses of interventional trials done in populations with different risks. The Lancet 3451304–1305. Segal, J. B., R. L. McNamara, M. R. Miller, N. Kim, S. N. Goodman, N. R. Powe, K. A. Robinson, and E. B. Bass. 2000. Prevention of thromboembolism in atrial fibrillation. A meta-analysis of trials of anticoagulants and antiplatelet drugs. Journal of General Internal Medicine 15:56–67. Shannon, W. D., and D. Banks. 1999. Combining classification trees using MLE. Statistics in Medicine 18:727–740. Song, F. 1999. Exploring heterogeneity in meta-analysis: is the L'Abbe plot useful? Journal of Clinical Epidemiology 52:725–730. Steinberg, K. K., S. B. Thacker, S. J. Smith, D. F. Stroup, M. M. Zack, W. D. Flanders, and R. L. Berkelman. 1991. A meta-analysis of the effect of estrogen replacement therapy on the risk of breast cancer. Journal of the American Medical Association 265:1985–1990. Steinberg, K. K., S. J. Smith, D. F. Stroup, I. Olkin, N. C. Lee, G. D. Williamson, and S. B. Thacker. 1997. Comparison of effect estimates from a meta-analysis of summary data from published studies and from a meta-analysis using individual patient data for ovarian cancer studies. American Journal of Epidemiology 145:917–925. Stijnen, T. 2000. Tutorial in biostatistics. Meta-analysis: formulating, evaluating, combining, and reporting by S. L. Normand (letter). Statistics in Medicine 19:759– 761. Stram, D. O. 1996. Meta-analysis of published data using a linear mixed-effects model. Biometrics 52:536–544. Stroup, D. F., J. A. Berlin, S. C. Morton, I. Olkin, G. D. Williamson, D. Rennie, D. Moher, B. J. Becker, T. A. Sipe, and S. B. Thacker. 2000. Meta-analysis of
OCR for page 196
Page 196 observational studies in epidemiology: a proposal for reporting. Meta-analysis of Observational Studies in Epidemiology (MOOSE) Group. Journal of the American Medical Association 283:2008–2012. Szczech, L. A., J. A. Berlin, S. Aradhye, R. A. Grosman, and H. I. Feldman. 1997. Effect of anti-lymphocyte induction therapy on renal allograft survival: a meta-analysis. Journal of the American Society of Nephrology 8:1771–1777. Thacker, S. B., D. F. Stroup, and H. B. Peterson. 1998. Meta-analysis for the practicing obstetrician-gynecologist. Clinical Obstetrics and Gynecology 41:275–281. Thompson, S. G., and S. J. Sharp. 1999. Explaining heterogeneity in meta-analysis: a comparison of methods. Statistics in Medicine 18:2693–2708. Thompson, S. G., T. C. Smith, and S. J. Sharp. 1997. Investigating underlying risk as a source of heterogeneity in meta-analysis. Statistics in Medicine 16:2741–2758. Tugwell, P. 1996. Combination therapy in rheumatoid arthritis: meta-analysis. Journal of Rheumatology 44(Suppl.):43-46. Whitehead, A. 1997. A prospectively planned cumulative meta-analysis applied to a series of concurrent clinical trials. Statistics in Medicine 16:2901–2913. Whitehead, A., A. J. Bailey, and D. Elbourne. 1999. Combining summaries of binary outcomes with those of continuous outcomes in a meta-analysis. Journal of Biopharmaceutical Statistics 9:1–16. Whitehead, J., and A. Whitehead. 1991. A general parametric approach to the meta-analysis of randomized clinical trials. Statistics in Medicine 10:1665–1677. META-ANALYSIS, CONFIDENCE PROFILE METHOD Ananth, C. V., and J. S. Preisser. 1999. Bivariate logistic regression: modelling the association of small for gestational age births in twin gestations. Statistics in Medicine 18: 2011–2023. Eddy, D. M., V. Hasselblad, and R. Schacter. 1990. An introduction to a Bayesian method for meta-analysis: the confidence profile method. Medical Decision Making 10:15– 23. Eddy, D. M., V. Hasselblad, and R. Shachter. 1992. Meta-Analysis by the Confidence Profile Method. The Statistical Synthesis of Evidence. Boston: Academic Press, Inc./ Harcourt Brace Jovanovich, Publishers. Hardy, R. J., and S. G. Thompson. 1996. A likelihood approach to meta-analysis with random effects. Statistics in Medicine 15:619–629. Hardy, R. J., and S. G. Thompson. 1998. Detecting and describing heterogeneity in meta-analysis. Statistics in Medicine 17:841–856. N-OF-1 CLINICAL TRIALS Cook, D. J. 1996. Randomized trials in single subjects: the n-of-1 study. Psychopharmacology Bulletin 32:363–367. Edgington, E. S. 1996. Randomized single-subject experimental designs. Behaviour Research and Therapy 34:567–574. Fleming, T. R. 1982. One-sample multiple testing procedure for phase II clinical trials. Biometrics 38:143–151.
OCR for page 197
Page 197 Frasca, M. A., and J. C. Aldag. 1988. The single-patient clinical trial. American Family Physician 37:195–199. Guyatt, G. H., D. Sackett, J. Adachi, R. Roberts, J. Chong, D. Rosenbloom, and J. Keller. 1988. A clinician's guide for conducting randomized trials in individual patients. Canadian Medical Association Journal 139:497–503. Hodgson, M. 1993. n-of-1 clinical trials. The practice of environmental and occupational medicine. Journal of Occupational Medicine 35:375–380. Johannessen, T. 1991. Controlled trials in single subjects. 1. Value in clinical medicine. British Medical Journal 303:173–174. Johannessen, T., D. Fosstvedt, and H. Petersen. 1988. Experience with multi-crossover model in dyspepsia. Scandinavian Journal of Gastroenterology Supplement 147:33– 37. Johannessen, T., D. Fosstvedt, and H. Petersen. 1990. Statistical aspects of controlled single subject trials. Family Practice 7:325–328. Johannessen, T., D. Fosstvedt, and H. Petersen. 1991. Combined single subject trials. Scandinavian Journal of Primary Health Care 9:23–27. Johannessen, T., H. Petersen, P. Kristensen, and D. Fosstvedt. 1991. The controlled single subject trial. Scandinavian Journal of Primary Health Care 9:17–21. Johannessen, T., P. Kirsten, H. Petersen, D. Fosstvedt, I. Loge, P. M. Kleveland, and J. Dybdahl. 1991. The symptomatic effect of 1-day treatment periods with cimetidine in dyspepsia. Combined results from randomized, controlled, single-subject trials. Scandinavian Journal of Gastroenterology 26:974–980. Keller, J. L., G. H. Guyatt, R. S. Robarts, J. D. Adachi, and D. Rosenbloom. 1988. An n-of-1 service: applying the scientific method in clinical practice. Scandinavian Journal of Gastroenterology Supplement 147:22–29. Lewis, J. A. 1991. Controlled trials in single subjects. 2. Limitations of use. British Medical Journal 303:175–176. Lindberg, G. 1988. Single case studies in clinical trials. Scandinavian Journal of Gastroenterology Supplement 147:30–32. Lukoff, D., D. Edwards, and M. Miller. 1998. The case study as a scientific method for researching alternative therapies. Alternative Therapies in Health Medicine 4:44–52. Menard, J., M. Bellet, and D. Serrurier. 1990. From the parallel group design to the crossover design, and from the group approach to the individual approach. American Journal of Hypertension 3:815–819. Morley, S., and M. Adams. 1989. Some simple statistical tests for exploring single-case time-series data. British Journal of Clinical Psychology 28 (Pt 1):1–18. O'Brien, P. C. 1997. The use and misuse of n-of-1 studies (editorial). International Journal of Oral and Maxillofacial Implants 12:293. Sandvik, L. 1988. Single case studies from a statistician's point of view. Scandinavian Journal of Gastroenterology Supplement 147:38–39. Sjoden, P. O. 1988. Single case studies in psychology and psychiatry. Scandinavian Journal of Gastroenterology Supplement 147:11–21. Spiegelhalter, D. J. 1988. Statistical issues in studies of individual response. Scandinavian Journal of Gastroenterology Supplement 147:40–45. Treasure, W. 1996. N-of-1 trials. Placebos should be abandoned. British Medical Journal 313: 427–428. Whitehead, J., W. M. Gregory, K. Bolland, and R. L. Souhami. 1997. Cautionary tales of
OCR for page 198
Page 198 survival analysis: conflicting analyses from a clinical trial in breast cancer. British Journal of Cancer 76:551–558. Wulff, H. R. 1988. Single case studies: an introduction. Scandinavian Journal of Gastroenterology Supplement 147:7–10. Zucker, D. R., C. H. Schmid, M. W. McIntosh, R. B. D'Agostino, H. P. Selker, and J. Lau. 1997. Combining single patient trials to estimate population treatment effects and to evaluate individual patient responses to treatment. Journal of Clinical Epidemiology 50:401–410. SEQUENTIAL ANALYSIS Armitage, P. 1975. Sequential Medical Trials. New York: Wiley. Atkinson, A. C. 1999. Optimum biased-coin designs for sequential treatment allocation with covariate information. Statistics in Medicine 18:1741–1752. (Discussion, 18:1753–1755.) Bellissant, E., J. Benichou, and C. Chastang. 1996. The group sequential triangular test for phase II cancer clinical trials. American Journal of Clinical Oncology 19:422–430. Betensky, R. A. 1997. Conditional power calculations for early acceptance of Ho embedded in sequential tests. Statistics in Medicine 16:465–477. Brooks, M. M., A. Hallstrom, and M. Peckova. 1995. A simulation study used to design the sequential monitoring plan for a clinical trial. Statistics in Medicine 14:2227– 2237. Carlin, B. P., J. B. Kadane, and A. E. Gelfand. 1998. Approaches for optimal sequential decision analysis in clinical trials. Biometrics 54:964–975. Conoway, M. R. and G. R. Petroni. 1996. Designs for phase II trials allowing for a tradeoff between response and toxicity. Biometrics 52:1375–1386. (applied sequential methods) DeMets, D. L. 1989. Group sequential procedures: calendar versus information time. Statistics in Medicine 8:1191–1198. DeMets, D. L. and G. Lan. 1995. The alpha spending function approach to interim data analyses. Cancer Treatment and Research 75:1–27. DeMets, D. L., D. Y. Lin, and L. J. Wei. 1991. Exact statistical inference for group sequential trials. Biometrics 47:1399–1408. Emerson, S. S. 1995. Stopping a clinical trial very early based on unplanned interim analyses: a group sequential approach. Biometrics 51:1152–1162. Emerson, S. S., and T. R. Fleming. 1989. Symmetric group sequential test designs. Biometrics 45:905–923. Emerson, S. S., and T. R. Fleming. 1990. Interim analyses in clinical trials. Oncology 4:126–133. (Discussion, 4:134, 136.) Fleming, T. R., D. P. Harrington, and P. C. O'Brien. 1984. Designs for group sequential tests. Controlled Clinical Trials 5:348–361. Geller, N. L., and Z. H. Li. 1991. On the choice of times for data analysis in group sequential clinical trials. Biometrics 47:745–750. Hallstrom, A., R. McBride, and R. Moore. 1995. Toward vital status sweeps: a case history in sequential monitoring. Statistics in Medicine 14:1927–1931.
OCR for page 199
Page 199 Heitjan, D. F. 1997. Bayesian interim analysis of phase II cancer clinical trials. Statistics in Medicine 16:1791–1802. Jiang, W. 1999. Group sequential procedures for repeated events data with frailty. Journal of Biopharmaceutical Statistics 9:379–399. Kim, K., and D. L. DeMets.1987. Confidence intervals following group sequential tests in clinical trials. Biometrics 43:857–864. Lai, T. L., B. Levin, H. Robbins, and D. Siegmund. 1980. Sequential medical trials. Proceedings of the National Academy of Sciences USA 77:3135–3138. Lan, K. K., and D. L. DeMets. 1989. Changing frequency of interim analysis in sequential monitoring. Biometrics 45:1017–1020. Lan, K. K., W. F. Rosenberger, and J. M. Lachin. 1995. Sequential monitoring of survival data with the Wilcoxon statistic. Biometrics 51:1175–1183. Lee, J. W., and D. L. DeMets. 1995. Group sequential comparison of changes: ad-hoc versus more exact method. Biometrics 51:21–30. Lee, J. W., and H. N. Sather. 1995. Group sequential methods for comparison of cure rates in clinical trials. Biometrics 51:756–763. Liu, W. 1995. A group sequential procedure for all pairwise comparisons of k treatments based on the range statistic. Biometrics 51:946–955. O'Fallon, J. R. 1985. Policies for interim analysis and interim reporting of results. Cancer Treatment Reports 69:1101–1116. O'Quigley, J., and L. Z. Shen. 1996. Continual reassessment method: a likelihood approach. Biometrics 52:673–684. Pocock, S. J., and M. D. Hughes. 1989. Practical problems in interim analyses, with particular regard to estimation. Controlled Clinical Trials 10(Suppl.):209S–221S. Reboussin D. M., D. L. DeMets, K. Kim, and K. K. Lan. 2000. Computations for group sequential boundaries using the Lan-DeMets spending function method. Controlled Clinical Trials 21:190–207. Tan, M., and X. Xiong. 1996. Continuous and group sequential conditional probability ratio tests for phase II clinical trials. Statistics in Medicine 15:2037–2051. Tan, M., X. Xiong, and M. H. Kutner. 1998. Clinical trial designs based on sequential conditional probability ratio tests and reverse stochastic curtailing. Biometrics 54(2):682-695. Whitehead, J. 1986. Supplementary analysis at the conclusion of a sequential clinical trial. Biometrics 42:461–471. Whitehead, J. 1991. Four problems with group sequential methods. Controlled Clinical Trials 12:340–344. Whitehead, J. 1992. Overrunning and underrunning in sequential clinical trials. Controlled Clinical Trials 13:106–121. Whitehead, J. 1993. Interim analyses and stopping rules in cancer clinical trials. British Journal of Cancer 68:1179–1185. Whitehead, J. 1994. Sequential methods based on the boundaries approach for the clinical comparison of survival times. Statistics in Medicine 13:1357–1368. (Discussion 1369–1370.) Whitehead, J. 1997. The Design and Analysis of Sequential Clinical Trials. New York: Ellis Horwood. Whitehead, J. 1998. Sequential designs for equivalence studies. Statistics in Medicine 15:2703–2715.
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Page 200 Whitehead, J. 1999. A unified theory for sequential clinical trials. Statistics in Medicine 18: 2271–2286. Whitehead, J., and K. M. Facey. 1990. An improved approximation for calculation of confidence intervals after a sequential clinical trial. Statistics in Medicine 9:1277– 1285. Whitehead, J., and K. M. Facey. 1990. The impact that group sequential tests would have made on ECOG clinical trials. Statistics in Medicine 9:853–854. Whitehead, J., and D. R. Jones. 1979. Group sequential methods. British Journal of Cancer 40:171–172. Whitehead, J., and P. Marek. 1985. A Fortran program for the design and analysis of sequential clinical trials. Computers and Biomedical Research 18:176–183. Whitehead, J., and M. R. Sooriyarachchi. 1998. A method for sequential analysis of survival data with non-proportional hazards. Biometrics 54:1072–1084. Whitehead, J., and I. Stratton. 1983. Group sequential clinical trials with triangular continuation regions. Biometrics 39:227–236. Whitehead, J., and P. Thomas. 1997. A sequential trial of painkillers in arthritis: issues of multiple comparisons with control and of interval-censored survival data. Journal of Biopharmaceutical Statistics 7:333–353. Whitehead, J., D. R. Jones, and C. E. Newman. 1982. The design of a sequential clinical trial for the comparison of two lung cancer treatments. Statistics in Medicine 1:73– 82. Whitehead, J., D. R. Jones, and S. H. Ellis. 1983. The analysis of a sequential clinical trial for the comparison of two lung cancer treatments. Statistics in Medicine 2:183–190. Whitehead, J., A. N. Donaldson, R. Stephens, and D. Machin. 1993. A simulated sequential analysis based on data from two MRC trials. British Journal of Cancer 68:1171–1178. Williams, P. L. 1996. Sequential monitoring of clinical trials with multiple survival endpoints. Statistics in Medicine 15:2341–2357.
Representative terms from entire chapter: