Multiplicity Considerations in Clinical Trials

Cardiovascular morbidity and mortality in the losartan intervention for endpoint reduction in hypertension study (LIFE): A randomised trial against atenolol

Dahlöf, B., Devereux, R. B., Kjeldsen, S. E., Julius, S., Beevers, G., de Faire, U., Fyhrquist, F., Ibsen, H., Kristiansson, K., Lederballe-Pedersen, O., et al. (2002) · The Lancet · doi:10.1016/s0140-6736(02)08089-3 · Cited by 5,407

Enzalutamide in Metastatic Prostate Cancer before Chemotherapy

Beer, T. M., Armstrong, A. J., Rathkopf, D. E., Loriot, Y., Sternberg, C. N., Higano, C. S., Iversen, P., Bhattacharya, S., Carles, J., Chowdhury, S., et al. (2014) · New England Journal of Medicine · doi:10.1056/nejmoa1405095 · Cited by 3,028

Abiraterone in Metastatic Prostate Cancer without Previous Chemotherapy

Ryan, C. J., Smith, M. R., de Bono, J. S., Molina, A., Logothetis, C. J., de Souza, P., Fizazi, K., Mainwaring, P., Piulats, J. M., Ng, S., et al. (2013) · New England Journal of Medicine · doi:10.1056/nejmoa1209096 · Cited by 2,796

Increased MET Gene Copy Number Negatively Affects Survival of Surgically Resected Non–Small-Cell Lung Cancer Patients

Cappuzzo, F., Marchetti, A., Skokan, M. C., Rossi, E., Gajapathy, S., Felicioni, L., Grammastro, M. D., Sciarrotta, M., Buttitta, F., Incarbone, M., et al. (2009) · Journal of Clinical Oncology · doi:10.1200/jco.2008.19.1635 · Cited by 563

Extended Thromboprophylaxis with Betrixaban in Acutely Ill Medical Patients

Cohen, A. T., Harrington, R. A., Goldhaber, S. Z., Hull, R. D., Wiens, B. L., Gold, A., Hernandez, A. F., & Gibson, C. M. (2016) · New England Journal of Medicine · doi:10.1056/nejmoa1601747 · Cited by 439

Combining Multiple Comparisons and Modeling Techniques in Dose‐Response Studies

Bretz, F., Pinheiro, J. C., & Branson, M. (2005) · Biometrics · doi:10.1111/j.1541-0420.2005.00344.x · Cited by 399

Tutorial in biostatistics: data‐driven subgroup identification and analysis in clinical trials

Lipkovich, I., Dmitrienko, A., & B., R. (2017) · Statistics in Medicine · doi:10.1002/sim.7064 · Cited by 291

Multiple Testing Problems in Pharmaceutical Statistics

(2009) · Chapman & Hall/CRC eBooks · doi:10.1201/9781584889854 · Cited by 274

Lurasidone in the Treatment of Schizophrenia: A Randomized, Double-Blind, Placebo- and Olanzapine-Controlled Study

Meltzer, H. Y., Cucchiaro, J. B., Silva, R., Ogasa, M., Phillips, D., Xu, J., Kalali, A. H., Schweizer, E. E., Pikalov, A., & Loebel, A. D. (2011) · American Journal of Psychiatry · doi:10.1176/appi.ajp.2011.10060907 · Cited by 251

Multiple testing in clinical trials

Bauer, P. (1991) · Statistics in Medicine · doi:10.1002/sim.4780100609 · Cited by 241

How to find a meta-analysis you can trust

Nelson, R. L. (2019) · Techniques in Coloproctology · doi:10.1007/s10151-019-02069-9

Controlling for Multiplicity, Eligibility, and Exclusions

Salter, A. R., & Miller, J. P. (2019) · Springer eBooks · doi:10.1007/978-3-319-52677-5_210-1

Immuntherapie im Plattenepithelkarzinom des Ösophagus - ein neuer Standard?

Büttner, N., & Thimme, R. (2020) · Gastro-News · doi:10.1007/s15036-020-0270-8

Multiplicity and statistical significance

Berdine, G. G. (2020) · The Southwest Respiratory and Critical Care Chronicles · doi:10.12746/swrccc.v8i34.681

Interpreting Multiple Outcomes of Pupillary Light Reflex in Sport-Related Concussion in Adolescents

Beaulieu, W. T., & Glassman, A. R. (2020) · JAMA Ophthalmology · doi:10.1001/jamaophthalmol.2020.3461

Association of Obesity with Breast Cancer

Lohmann, A. E. (2019) · TSpace (University of Toronto)

Controlling for Multiplicity, Eligibility, and Exclusions

Salter, A. R., & Miller, J. P. (2022) · Springer eBooks · doi:10.1007/978-3-319-52636-2_210

Can subgroup analyses in randomized trials guide individualized patient care?

Aronson, D. (2022) · European Journal of Heart Failure · doi:10.1002/ejhf.2710

Prevalência de relato seletivo de desfechos em ensaios clínicos randomizados que utilizam o modelo de cirurgia de terceiros molares

Santos, V. P. D. A. (2023) · Digital Library of Theses and Dissertations (Universidade de São Paulo) · doi:10.11606/d.23.2023.tde-26062023-101328

Pièges et mésusages en analyse de données

Supplisson, O., & Sofonea, M. T. (2023) · Anesthésie & Réanimation · doi:10.1016/j.anrea.2023.08.002

Randomised trials – additional aspects of analysis and interpretation

Hackshaw, A. K. (2024) · Wiley eBooks · doi:10.1002/9781119502678.ch7

Effectiveness of a Father-Inclusive Parenting Intervention on a Child’s Diet, Early Stimulation, and Father Involvement in Childcare in Rural Malawi: A Study Protocol for a Four-Arm, Cluster-Randomized Controlled TrialPreprint

Choi, S., Shibanuma, A., Kiriya, J., Kazembe, N., & Jimba, M. (2024) · Qeios · doi:10.32388/3xfllw

Adaptive Designs

Ye, J., & Nie, L. (2024) · ICSA book series in statistics · doi:10.1007/978-3-031-60843-8_9

An efficient vaccine clinical trial: ACTG A5379 hepatitis B vaccine trial in persons with HIV

Kang, M., Marks, K., Cox, A. L., & Sherman, K. E. (2025) · Vaccine · doi:10.1016/j.vaccine.2025.127028

On multiple comparisons and competitive variables

Ciocca, M. E., Mastaï, R., & Cagide, A. M. (2025) · Archivos Argentinos de Pediatria · doi:10.5546/aap.2025-10696.eng

Considerations for results of the patient-reported outcome analyses of the TALAPRO-2 trial

de Boissieu, P., & Chevret, S. (2025) · The Lancet Oncology · doi:10.1016/s1470-2045(25)00228-1

A prospective single-masked, non-inferiority, parallel-group randomized controlled trial of the efficacy of a ChatGPT-based AI chatbot to improve Boston bowel preparation scores for colonoscopy preparation: A trial protocol

Azmi, N. M., Jalal, M. I. A., Ashar, S. H. M., Nazri, M. I. M., Jie, Y., Ganeson, N., Augustine, J. K., & Yew, S. Q. (2025) · PLoS ONE · doi:10.1371/journal.pone.0334349

Additional file 1 of Age-based targeting of biannual azithromycin distribution for child survival in Niger: an adaptive cluster-randomized trial protocol (AVENIR)

O’Brien, K. S., Arzika, A. M., Amza, A., Maliki, R., Ousmane, S., Kadri, B., Nassirou, B., Mankara, A. K., Harouna, A. N., Colby, E., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14516236

Additional file 1 of Optimizing a Bayesian hierarchical adaptive platform trial design for stroke patients

Gao, G., Gajewski, B. J., Wick, J., Beall, J., Saver, J. L., & Meinzer, C. (2022) · Figshare · doi:10.6084/m9.figshare.21009582

Additional file 1 of Effectiveness of an expert assessment and individualised treatment compared with a minimal home-based exercise program in women with late-term shoulder impairments after primary breast cancer surgery: study protocol for a randomised controlled trial

Feder, K. M., Rahr, H. B., Lautrup, M. D., Egebæk, H. K., Christensen, R., & Ingwersen, K. G. (2022) · Figshare · doi:10.6084/m9.figshare.20522589

Additional file 7 of The effects of different doses of exercise on pancreatic β-cell function in patients with newly diagnosed type 2 diabetes: study protocol for and rationale behind the “DOSE-EX” multi-arm parallel-group randomised clinical trial

Lyngbæk, M. P. P., Legaard, G. E., Bennetsen, S. L., Feineis, C. S., Rasmussen, V., Moegelberg, N., Brinkløv, C. F., Nielsen, A. B., Kofoed, K. S., Lauridsen, C. A., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14361947

Additional file 3 of The effects of different doses of exercise on pancreatic β-cell function in patients with newly diagnosed type 2 diabetes: study protocol for and rationale behind the “DOSE-EX” multi-arm parallel-group randomised clinical trial

Lyngbæk, M. P. P., Legaard, G. E., Bennetsen, S. L., Feineis, C. S., Rasmussen, V., Moegelberg, N., Brinkløv, C. F., Nielsen, A. B., Kofoed, K. S., Lauridsen, C. A., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14361935

Additional file 2 of Effectiveness of an expert assessment and individualised treatment compared with a minimal home-based exercise program in women with late-term shoulder impairments after primary breast cancer surgery: study protocol for a randomised controlled trial

Feder, K. M., Rahr, H. B., Lautrup, M. D., Egebæk, H. K., Christensen, R., & Ingwersen, K. G. (2022) · Figshare · doi:10.6084/m9.figshare.20522592

Additional file 2 of The effects of different doses of exercise on pancreatic β-cell function in patients with newly diagnosed type 2 diabetes: study protocol for and rationale behind the “DOSE-EX” multi-arm parallel-group randomised clinical trial

Lyngbæk, M. P. P., Legaard, G. E., Bennetsen, S. L., Feineis, C. S., Rasmussen, V., Moegelberg, N., Brinkløv, C. F., Nielsen, A. B., Kofoed, K. S., Lauridsen, C. A., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14361932

Additional file 6 of The effects of different doses of exercise on pancreatic β-cell function in patients with newly diagnosed type 2 diabetes: study protocol for and rationale behind the “DOSE-EX” multi-arm parallel-group randomised clinical trial

Lyngbæk, M. P. P., Legaard, G. E., Bennetsen, S. L., Feineis, C. S., Rasmussen, V., Moegelberg, N., Brinkløv, C. F., Nielsen, A. B., Kofoed, K. S., Lauridsen, C. A., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14361944

Additional file 1 of The effects of different doses of exercise on pancreatic β-cell function in patients with newly diagnosed type 2 diabetes: study protocol for and rationale behind the “DOSE-EX” multi-arm parallel-group randomised clinical trial

Lyngbæk, M. P. P., Legaard, G. E., Bennetsen, S. L., Feineis, C. S., Rasmussen, V., Moegelberg, N., Brinkløv, C. F., Nielsen, A. B., Kofoed, K. S., Lauridsen, C. A., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14361929

Additional file 5 of The effects of different doses of exercise on pancreatic β-cell function in patients with newly diagnosed type 2 diabetes: study protocol for and rationale behind the “DOSE-EX” multi-arm parallel-group randomised clinical trial

Lyngbæk, M. P. P., Legaard, G. E., Bennetsen, S. L., Feineis, C. S., Rasmussen, V., Moegelberg, N., Brinkløv, C. F., Nielsen, A. B., Kofoed, K. S., Lauridsen, C. A., et al. (2021) · Figshare · doi:10.6084/m9.figshare.14361941

Efficacy of Racecadotril in acute diarrhea in children: systematic review of double blind randomized clinical trials

Bittar, I., GUYENARD, L., Blanchard, C., & Boussageon, R. (2026) · European Journal of Clinical Pharmacology · doi:10.1007/s00228-025-03927-2 · Cited by 2

Lifestyle, sleep quality, and cognitive function in resistant hypertension: One-year follow-up from the TRIUMPH trial

Smith, P. J., Blumenthal, J. A., Ingle, K., Watkins, L. L., Avorgbedor, F., Mabe, S., Kraus, W. E., Tyson, C. C., Hinderliter, A. L., & Sherwood, A. (2026) · Journal of Alzheimer s Disease · doi:10.1177/13872877251409716 · Cited by 1

Vector‐Based Comparison and Average Slope Can Refine Bioequivalence Claims: A Machine and Deep Learning Approach

Kokkali, M., & Karalis, V. D. (2026) · Biopharmaceutics & Drug Disposition · doi:10.1002/bdd.70023

Refining Normoxemia Targets in Acute Burn Care: Opportunities Beyond Supplemental Oxygen-Free Days

Tang, F., Han, C., & Liu, S. (2026) · The Journal of Trauma: Injury, Infection, and Critical Care · doi:10.1097/ta.0000000000004950

Analyse de l'efficacité et de la toxicité de l’irinotecan : Méta-analyses sur données individuelles dans le cancer gastrique avancé ou métastatique. Développements cliniques et méthodologiques

Chamseddine, A. N. (2021) · theses.fr (ABES) · doi:10.70675/68ea9779zddf2z4d17z9e21z2100d9fbb840

Power calculation and sample size

Acevedo, D. C. A., & Valderrama-Molina, C. O. (2026) · Elsevier eBooks · doi:10.1016/b978-0-443-34264-6.00003-4

Efficacy of a WeChat mini-program-based self-management intervention for bipolar disorder: A randomized controlled trial

Lin, X., Ba, Z., Lu, D., Ma, R., Chen, M., Zhu, C., Liao, Y., Liu, Y., & Luo, X. (2026) · Journal of Affective Disorders · doi:10.1016/j.jad.2026.121947

Vector‐based comparison as a novel framework for assessing multi‐endpoint clinical trials: Applications to actual data

Kokkali, M., & Karalis, V. D. (2026) · Clinical and Translational Discovery · doi:10.1002/ctd2.70180

From Signal to Certainty: Why COPD Needs More Than One Trial

Cazzola, M., Stolz, D., Sin, D. D., Matera, M. G., & Rogliani, P. (2026) · Lung · doi:10.1007/s00408-026-00914-x

Effect of a digital intervention on mental health symptoms in adults with chronic conditions: A three-arm randomized controlled trial

Johnson, E., Hyde, A. M., Corrick, S., Isley, S., Wright, G., Ezekowitz, J. A., Zheng, Y., Lee‐Baggley, D., Spiers, J. A., Spence, J. C., et al. (2026) · PLoS Medicine · doi:10.1371/journal.pmed.1005198

Effect of Kangaroo Mother Care during the first 72 hours of life on early growth and breastfeeding in normal birthweight newborns: protocol for a randomised controlled trial

Kumar, A., Mishra, M., Tiwari, M., Singh, V. P., Mukhopadhyay, R., Srivastava, A., Kumar, M., Agarwal, A. T. D., Singh, S. N., Tripathi, S., et al. (2026) · BMJ Open · doi:10.1136/bmjopen-2026-116601

Comparison of multiple testing procedures for clinical trials with two doses and multiple ranked endpoints

Minini, P., Paux, G., & Dmitrienko, A. (2026) · Journal of Biopharmaceutical Statistics · doi:10.1080/10543406.2026.2741424

Multiplicity Issues in Clinical Trials

Sozu, T. (2015) · Japanese Journal of Biometrics · doi:10.5691/jjb.36.s87

- Multiple Comparisons

Rothmann, M. D., Wiens, B. L., & Chan, I. S. F. (2011) · Chapman & Hall/CRC eBooks · doi:10.1201/b11039-9

Multiple Testing in Clinical Trials

Dmitrienko, A. A., & Hsu, J. C. (2005) · Encyclopedia of Statistical Sciences · doi:10.1002/0471667196.ess7006 · Cited by 1

Clinical trials must cope better with multiplicity

Wittes, J. T. (2012) · Nature Medicine · doi:10.1038/nm1112-1607 · Cited by 6

21 The Multiple Comparison Issue in Health Care Research

Moyé, L. A. (2007) · Handbook of statistics · doi:10.1016/s0169-7161(07)27021-x · Cited by 2