Cited by 426Statistical Methods in Clinical TrialsStatistical Methods and Bayesian InferenceStatistical Methods in Epidemiology

The DOI matches its Crossref record; 31 of the 31 references it cites were found in a registry or an index.

The statistical interpretation of pilot trials: should significance thresholds be reconsidered?

Open access

Lee, Ellen C, Whitehead, Amy L, Jacques, Richard M, Julious, Steven A·2014·BMC Medical Research Methodology

DOI 10.1186/1471-2288-14-41PMC PMC3994566

Open this paper in CoChat — free account Open PDF

Abstract

BACKGROUND: In an evaluation of a new health technology, a pilot trial may be undertaken prior to a trial that makes a definitive assessment of benefit. The objective of pilot studies is to provide sufficient evidence that a larger definitive trial can be undertaken and, at times, to provide a preliminary assessment of benefit. METHODS: We describe significance thresholds, confidence intervals and surrogate markers in the context of pilot studies and how Bayesian methods can be used in pilot trials. We use a worked example to illustrate the issues raised. RESULTS: We show how significance levels other than the traditional 5% should be considered to provide preliminary evidence for efficacy and how estimation and confidence intervals should be the focus to provide an estimated range of possible treatment effects. We also illustrate how Bayesian methods could also assist in the early assessment of a health technology. CONCLUSIONS: We recommend that in pilot trials the focus should be on descriptive statistics and estimation, using confidence intervals, rather than formal hypothesis testing and that confidence intervals other than 95% confidence intervals, such as 85% or 75%, be used for the estimation. The confidence interval should then be interpreted with regards to the minimum clinically important difference. We also recommend that Bayesian methods be used to assist in the interpretation of pilot trials. Surrogate endpoints can also be used in pilot trials but they must reliably predict the overall effect on the clinical outcome.

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Integrity signals

Registry

Crossref · 10.1186/1471-2288-14-41

Cites

31 of 31 references verified0 citation errors · 0 not checkable

Statistics

Figure 1 Mean difference in SF-36 GH dimension scores between treatment and control with confidence intervals (based on n = 31 patients).CC BY · PubMed Central

Figure 2 Prior, observed and posterior distributions for non-informative, pessimistic and optimistic priors.CC BY · PubMed Central

Claims the paper makes

The claims and statistics of this paper haven’t been checked yet.

Run the check — free, no account needed. It reads the open-access text and checks the sources it cites.

Claims other papers make about this one

Each is checked against this paper’s own text: does it say what the citing paper says it does?

Self-check

Percentages, sums and reported differences re-computed from the paper’s own numbers.

The self-check needs the paper’s full text.

Biomarkers and surrogate endpoints: Preferred definitions and conceptual framework
Biomarkers Definitions Working Group (2001) · Clinical Pharmacology & Therapeutics · doi:10.1067/mcp.2001.113989 · Cited by 6,771
A tutorial on pilot studies: the what, why and how
Thabane, L., Ma, J., Chu, R., Cheng, J., Ismaila, A., Rios, L. P., Robson, R., Thabane, M., Giangregorio, L., & Goldsmith, C. H. (2010) · BMC Medical Research Methodology · doi:10.1186/1471-2288-10-1 · Cited by 3,384
Design and analysis of pilot studies: recommendations for good practice
Lancaster, G. A., Dodd, S., & Williamson, P. R. (2004) · Journal of Evaluation in Clinical Practice · doi:10.1111/j..2002.384.doc.x · Cited by 2,572
Surrogate endpoints in clinical trials: Definition and operational criteria
Prentice, R. L. (1989) · Statistics in Medicine · doi:10.1002/sim.4780080407 · Cited by 2,057
Bayesian Approaches to Clinical Trials and Health‐Care Evaluation
Spiegelhalter, D. J., Abrams, K. R., & Myles, J. P. (2003) · Wiley eBooks · doi:10.1002/0470092602 · Cited by 1,687
Surrogate End Points in Clinical Trials: Are We Being Misled?
Fleming, T. R., & DeMets, D. L. (1996) · Annals of Internal Medicine · doi:10.7326/0003-4819-125-7-199610010-00011 · Cited by 1,651
What is a pilot or feasibility study? A review of current practice and editorial policy
Arain, M., Campbell, M. J., Cooper, C. L., & Lancaster, G. A. (2010) · BMC Medical Research Methodology · doi:10.1186/1471-2288-10-67 · Cited by 1,483
Bayesian Statistics: An Introduction
Ziegel, E. R., & Lee, P. M. (1998) · Technometrics · doi:10.2307/1270551 · Cited by 1,191
Multiple Imputation and its Application
Carpenter, J. R., & Kenward, M. G. (2013) · doi:10.1002/9781119942283 · Cited by 913
Considerations in the Evaluation of Surrogate Endpoints in Clinical Trials
Gruttola, V. D., Clax, P. A., DeMets, D. L., Downing, G. J., Ellenberg, S. S., Friedman, L., Gail, M. H., Prentice, R. L., Wittes, J., & Zeger, S. L. (2001) · Controlled Clinical Trials · doi:10.1016/s0197-2456(01)00153-2 · Cited by 428

Show all 31 references

Research works printed but not linked: 1

International Conference on Harmonisation: ICH E9 statistical principals for clinical trials. 1998, http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf,

Sorted by citations

Items 201–300 of 426

Protocol of a feasibility randomised controlled trial of Empowered Conversations: training family carers to enhance their relationships and communication with people living with dementia.
Eastham, C., Mansell, W., Sutton, C., Prior, Y., Keady, J. D., Shields, G., Riley, C., Bowker, G., Sylvestre, Y., & Morris, L. (2023) · NIHR Open Research · doi:10.3310/nihropenres.13441.1 · Cited by 7
Identification of blood lipid markers of docetaxel treatment in prostate cancer patients
Finnerty, M. C., Leach, F. E., Zakharia, Y., Nepple, K. G., Bartlett, M. G., Henry, M. D., & Cummings, B. S. (2024) · Scientific Reports · doi:10.1038/s41598-024-73074-8 · Cited by 7
Feasibility and outcomes of a trauma-informed model of care in residential treatment for substance use
Walter, Z. C., Carlyle, M., Mefodeva, V., Glasgow, S., Newland, G., Nixon, R. D. V., Cobham, V. E., & Hides, L. M. (2024) · Journal of Substance Use and Addiction Treatment · doi:10.1016/j.josat.2024.209571 · Cited by 8
Video-delivered emotion-focused mindfulness therapy for late- life anxiety: study protocol for a feasibility randomized controlled trial
Hatch, S., Kessler, D., Finlayson, M. L., & Rej, S. (2021) · Pilot and Feasibility Studies · doi:10.1186/s40814-021-00905-0 · Cited by 7
Seizure first aid training for people with epilepsy attending emergency departments and their significant others: the SAFE intervention and feasibility RCT
Noble, A. J., Nevitt, S. J., Holmes, E. A. F., Ridsdale, L., Morgan, M., Smith, C. T., Hughes, D. A., Goodacre, S. W., Marson, A. G., & Snape, D. (2020) · Health Services and Delivery Research · doi:10.3310/hsdr08390 · Cited by 7
Feasibility and pilot studies pave the way for definitive trials
Harvey, L. A. (2018) · Spinal Cord · doi:10.1038/s41393-018-0184-x · Cited by 12
Feasibility and clinical potential of exercise interventions during neoadjuvant chemoradiotherapy in patients with esophageal and rectal cancer
Mast, I. H., Gootjes, E. C., Rütten, H., den Hartogh, M. D., Brouwer, C. G., Nagtegaal, I. D., van der Post, R. S., Hopman, M. T. E., van den Heuvel, B., Rosman, C., et al. (2025) · Journal of sport and health science/Journal of Sport and Health Science · doi:10.1016/j.jshs.2025.101060 · Cited by 6
The relational approach to treating self-harm (RELATE): A feasibility randomised controlled trial of cognitive analytic therapy for adults who self-harm versus treatment at usual
Taylor, P. J., Adeyemi, I., Marlow, K., Cottam, S., Airnes, Z., Howells, V., Dunn, B. D., Elliott, R. A., Hann, M., Latham, C., et al. (2025) · Psychiatry Research · doi:10.1016/j.psychres.2025.116859 · Cited by 6
Analysing Gender Effect on the Speech Act of Suggestion: A Pilot Study
Sharqawi, M. A., & Anthony, E. M. (2020) · Academic Journal of Interdisciplinary Studies · doi:10.36941/ajis-2020-0062 · Cited by 6
A Validation Pilot Study Comparing Telemedicine Images to a Face-to-Face Airway Exam for Conducting the Anesthesia Preoperative Airway Evaluation
Reddy, K., Awuku, K., Shah, Y. M., Tewfik, G., Xiong, M., Spivack, E., Bekker, A., & Grech, D. (2021) · Open Journal of Anesthesiology · doi:10.4236/ojanes.2021.117020 · Cited by 6
A study of elite sport-inspired coaching for patients after allogeneic hematopoietic stem cell transplantation
Cuvelier, S., Blaise, D., Boher, J., Villaron, C., Justafré, S., Pakradouni, J., Granata, A., Fürst, S., Dantin, P., Viens, P., et al. (2021) · Bone Marrow Transplantation · doi:10.1038/s41409-021-01401-y · Cited by 6
Comprehensive Assessment of Spatial Ability in Children: A Computerized Tasks Battery
Soluki, S., Yazdani, S., Arjmandnia, A. A., Fathabadi, J., Hassanzadeh, S., & Nejati, V. (2021) · Advances in Cognitive Psychology · doi:10.5709/acp-0315-0 · Cited by 6
Managing mobility outcomes in vulnerable seniors ( MMOVeS ): a randomized controlled pilot study
Figueiredo, S. M., Morais, J. A., & Mayo, N. E. (2017) · Clinical Rehabilitation · doi:10.1177/0269215517705941 · Cited by 6
Enhancing student competency in risky clinical environments: Evaluating an online education program
Graj, E., Sheen, J. A., Dudley, A. L., Sutherland‐Smith, W., & McGillivray, J. (2018) · Australian Psychologist · doi:10.1111/ap.12364 · Cited by 6
Effects of Education Savings Accounts on Student Engagement: Instrumental Variable Analysis
Ansong, D., Okumu, M., Kim, Y. K., Despard, M., Darfo-Oduro, R. K. A., & Small, E. (2019) · Global Social Welfare · doi:10.1007/s40609-019-00142-7 · Cited by 6
Effectiveness of a tailored rehabilitation versus standard strengthening programme for patients with shoulder pain: a protocol for a feasibility randomised controlled trial (the Otago MASTER trial)
Ribeiro, D. C., Tangrood, Z. J., Sole, G., & Abbott, J. H. (2019) · BMJ Open · doi:10.1136/bmjopen-2018-028261 · Cited by 7
Individual health trainers to support health and well-being for people under community supervision in the criminal justice system: the STRENGTHEN pilot RCT
Callaghan, L., Thompson, T., Creanor, S., Quinn, C., Senior, J., Green, C., Hawton, A., Byng, R., Wallace, G., Sinclair, J., et al. (2019) · Public Health Research · doi:10.3310/phr07200 · Cited by 6
Seizure First Aid Training For people with Epilepsy (SAFE) frequently attending emergency departments and their significant others: results of a UK multi-centre randomised controlled pilot trial
Noble, A. J., Snape, D., Nevitt, S. J., Holmes, E. A. F., Morgan, M., Smith, C. T., Hughes, D. A., Buchanan, M., McVicar, J., MacCallum, E., et al. (2020) · BMJ Open · doi:10.1136/bmjopen-2019-035516 · Cited by 5
A Pre–Post, Mixed-Methods Study to Pilot Test a Gamified Heart Failure Self-Care Education Intervention
Lukey, A., Mackay, M., Hasan, K., & Rush, K. L. (2023) · Games for Health Journal · doi:10.1089/g4h.2022.0132 · Cited by 5
Exoskeleton-assisted training to enhance lower limb motor recovery in subacute stroke: does timing matter? A pilot randomized trial
Schröder, J., Yperzeele, L., Embrechts, E., Loureiro-Chaves, R. F., Hallemans, A., Lafosse, C., Truijen, S., Kwakkel, G., & Saeys, W. (2024) · Frontiers in Stroke · doi:10.3389/fstro.2024.1379083 · Cited by 5
A mental imagery intervention targeting suicidal ideation in university students: An assessor-blind, randomised controlled feasibility trial
Palmier‐Claus, J. E., Duxbury, P. J., Pratt, D., Parker, S., Sutton, C., Lobban, F. A., Moorhouse, J., Kerry, E., Russell, C., Nyakutsikwa, B., et al. (2025) · Behaviour Research and Therapy · doi:10.1016/j.brat.2025.104780 · Cited by 5
A randomised controlled feasibility trial of a clinical protocol to manage hypotension during major non‐cardiac surgery
Wijeysundera, D. N., Duncan, D., Garijo, J. M., Jerath, A., Meineri, M., Parotto, M., Wąsowicz, M., McCluskey, S. A., & AMIGO‐1 Study Investigators (2022) · Anaesthesia · doi:10.1111/anae.15715 · Cited by 5
Towards precision home visiting: results at six months postpartum from a randomized pilot implementation trial to assess the feasibility of a precision approach to Family Spirit
Ingalls, A., Rebman, P., Martin, L., Kushman, E., Leonard, A., Cisler, A., Gschwind, I., Brayak, A., Amsler, A. M., & Haroz, E. E. (2022) · BMC Pregnancy and Childbirth · doi:10.1186/s12884-022-05057-4 · Cited by 5
Positive psychological interventions on alcohol use and consequences: Pilot randomized trial in a young adult cannabis-using sample
Trinh, C. D., Girard, R., Schick, M. R., & Spillane, N. S. (2023) · Journal of Substance Use and Addiction Treatment · doi:10.1016/j.josat.2023.209241 · Cited by 5
An integrated mental health video consultations model for patients with somatic symptom disorder in primary care: The randomized VISION pilot trial
Haun, M. W., van Eickels, D., Tönnies, J., Graue, L., Ayoub-Schreifeldt, M., Wensing, M., Hartmann, M., Szécsényi, J., Wild, B., & Friederich, H. (2024) · Journal of Psychosomatic Research · doi:10.1016/j.jpsychores.2024.111801 · Cited by 5
Feasibility and acceptability of a tailored health coaching intervention to improve type 2 diabetes self-management in Saudi Arabia: a mixed-methods randomised feasibility trial
Almulhim, A. N., Alhowaish, A., Madani, A., AlQaddan, M., Altuwalah, A. S., & Goyder, E. (2024) · BMJ Open · doi:10.1136/bmjopen-2023-078631 · Cited by 5
Inter-organizational alignment and implementation outcomes in integrated mental healthcare for children and adolescents: a cross-sectional observational study
Zhang, Y., Larson, M. F., Ehrhart, M. G., King, K. M., Locke, J. J., Cook, C. R., & Lyon, A. R. (2024) · Implementation Science · doi:10.1186/s13012-024-01364-w · Cited by 6
Comparing five‐factor personality traits and psychological distress between dysphonic patients and individuals with vocally healthy voices
Aghajanzadeh, M., Dabirmoghaddam, P., Soleimani, M., & Saeedi, S. (2024) · Brain and Behavior · doi:10.1002/brb3.3641 · Cited by 7
Temperature‐controlled laminar airflow ( TLA ) device in the treatment of children with severe atopic eczema: Open‐label, proof‐of‐concept study
Gore, C., Gore, R. B., Fontanella, S., Haider, S., & Čustović, A. (2018) · Clinical & Experimental Allergy · doi:10.1111/cea.13105 · Cited by 5
Value beyond the P : The Case for Higher-Quality and Better-publicized Pilot and Feasibility Trials
Brown, S. M., Paine, R., Lanspa, M. J., & Gong, M. N. (2019) · Annals of the American Thoracic Society · doi:10.1513/annalsats.201901-059ps · Cited by 5
Characteristics of piloting longitudinal birth cohort surveys: a systematic review
Blažev, M., Babarović, T., & Serracant, P. (2020) · Quality & Quantity · doi:10.1007/s11135-020-01042-1 · Cited by 5
Subjective Assessment of Occupational Exoskeletons: Feasibility Study for a Custom Survey for Braces
Sposito, M., Caldwell, D. G., Momi, E. D., & Ortiz, J. (2021) · Biosystems & biorobotics · doi:10.1007/978-3-030-69547-7_32 · Cited by 5
A Mixed-Methods Feasibility Study of Breastfeeding Attitudes among Southern African Americans
Felder, T. M., Çayır, E., Nkwonta, C. A., Tucker, C. M., Harris, E. H., & Jackson, J. (2021) · Western Journal of Nursing Research · doi:10.1177/01939459211045431 · Cited by 5
Exploring the usability and feasibility of a mobile music listening application for people living in the community with post-stroke aphasia
Best, B., Campbell, J., Roxbury, T., Worthy, P., & Copland, D. A. (2023) · Disability and Rehabilitation · doi:10.1080/09638288.2022.2161646 · Cited by 5
Feasibility and Effectiveness of a Personalized Home-Based Motor-Cognitive Training Program in Community-Dwelling Older Adults: Protocol for a Pragmatic Pilot Randomized Controlled Trial
Seinsche, J., de Bruin, E. D., Saibene, E., Rizzo, F., Carpinella, I., Ferrarin, M., Ifanger, S., Moza, S., & Giannouli, E. (2023) · JMIR Research Protocols · doi:10.2196/49377 · Cited by 5
Everyday discrimination and telomere length in a multiethnic cohort of breast cancer survivors
Aghaee, S., Allen, A. M., Ramírez, J. A., Shariff‐Marco, S., Allen, L., DeRouen, M. C., Elmofty, M., Márquez-Magaña, L. M., & Gomez, S. L. (2020) · Ethnicity and Health · doi:10.1080/13557858.2020.1739231 · Cited by 5
Measuring Memory for Treatment Using Patient Conceptualizations of Clinical Vignettes: A Pilot Psychometric Study in the Context of Cognitive Therapy for Depression
Zieve, G. G., Armstrong, C. C., Richardson, I. M., Garcia, S. B., & Harvey, A. G. (2022) · Cognitive Therapy and Research · doi:10.1007/s10608-022-10293-1 · Cited by 5
Evaluation of a Circadian Rhythm and Sleep-Focused Mobile Health Intervention for the Prevention of Accelerated Summer Weight Gain Among Elementary School–Age Children: Protocol for a Randomized Controlled Feasibility Study
Moreno, J. P., Dadabhoy, H., Musaad, S. M. A., Baranowski, T., Thompson, D. I., Alfano, C. A., & Crowley, S. J. (2022) · JMIR Research Protocols · doi:10.2196/37002 · Cited by 5
Assessment of the Effect of Rehmannia glutinosa Leaf Extract in Maintaining Skin Health: A Proof-of-Concept, Double-Blind, Randomized, Placebo-Controlled Clinical Trial
Srivastava, S., Huang, S. F., & Jagtap, M. (2024) · Clinical Cosmetic and Investigational Dermatology · doi:10.2147/ccid.s448928 · Cited by 4
Building social-emotional skills in a college career and life planning course: A preliminary investigation of the impact of skills practice on wellbeing
Conley, C. S., Nowakowski, L. M., Hareli, M., & Gonzales, C. H. (2025) · Social and Emotional Learning Research Practice and Policy · doi:10.1016/j.sel.2025.100107 · Cited by 4
Effects of a 5-Day Back Squat Overreaching Protocol on Strength Performance, Perceived Recovery and Wellness Responses: A Pilot Trial
Bell, L., Ruddock, A. D., Boriel, J., Maden‐Wilkinson, T., Thompson, S. W., Wright, K. J., Burke, K., & Rogerson, D. (2025) · Journal of Functional Morphology and Kinesiology · doi:10.3390/jfmk10020227 · Cited by 4
Structural brain improvements following individually tailored serious exergame-based training in mild neurocognitive disorders: exploratory randomized controlled trial
Manser, P., Rosio, M., Schmidt, A., Michels, L., & de Bruin, E. D. (2025) · Alzheimer s Research & Therapy · doi:10.1186/s13195-025-01835-2 · Cited by 4
Effects of manual diaphragm release on pain, disability and diaphragm function in patients with chronic neck pain: a pilot randomized controlled trial
Cheng, K.-C., Hii, E. Y. X., Lin, Y.-N., Kuo, Y., & Tsai, Y. (2025) · BMC Complementary Medicine and Therapies · doi:10.1186/s12906-025-05090-8 · Cited by 4
Exercise and Reduced Nicotine Content Cigarettes in Adult Female Smokers: A Pilot Trial
Li, C., Yao, N. A., Miller, S. L., Macpherson, C., Hassinger, T. E., Love, K. M., & Malin, S. K. (2022) · International Journal of Environmental Research and Public Health · doi:10.3390/ijerph19116647 · Cited by 4
Embedded Specialist Palliative Care in Cystic Fibrosis: Results of a Randomized Feasibility Clinical Trial
Kavalieratos, D., Lowers, J., Moreines, L. T., Hoydich, Z., Arnold, R. M., Yabes, J. G., Richless, C., Ikejiani, D. Z., Teuteberg, W., & Pilewski, J. M. (2022) · Journal of Palliative Medicine · doi:10.1089/jpm.2022.0349 · Cited by 4
Mixed Reality for Engineering Design Review Using Finite Element Analysis
Poh, E., Liong, K., & Lee, J. S. A. (2022) · IEEE International Symposium on Mixed and Augmented Reality Adjunct (ISMAR-Adjunct) · doi:10.1109/ismar-adjunct57072.2022.00136 · Cited by 4
Assessing the relationship between cardiovascular and small airway disease and acute events in COPD: The ARCADIA study protocol
Rogliani, P., Radovanovic, D., Ora, J., Starc, N., Verri, S., Pistocchini, E., & Calzetta, L. (2023) · Pulmonary Pharmacology & Therapeutics · doi:10.1016/j.pupt.2023.102231 · Cited by 4
Healing rate of hospital‐acquired skin tears using adhesive silicone foam versus meshed silicone interface dressings: A prospective, randomized, non‐inferiority pilot study
Fulbrook, P., Miles, S., & Williams, D. M. (2023) · International Journal of Nursing Practice · doi:10.1111/ijn.13229 · Cited by 4
Palmitoylethanolamide Does Not Affect Recovery from Exercise-Induced Muscle Damage in Healthy Males
Schouten, M., Dalle, S., Costamagna, D., Ramaekers, M., Bogaerts, S., Thienen, R. V., Peers, K. H. E., Thomis, M. A., & Koppo, K. (2024) · Medicine & Science in Sports & Exercise · doi:10.1249/mss.0000000000003517 · Cited by 7
A Specialised Antenatal Music Therapy (SAMT) programme for pregnant women with mental health conditions: A mixed-methods proof-of-concept study in Ireland
Cheung, P. S., MOHAMAD, M. R., Fitzpatrick, K., Tighe, S. M., Clifford, A. M., & McCaffrey, T. (2024) · Nordic Journal of Music Therapy · doi:10.1080/08098131.2024.2396099 · Cited by 4
Direct Oral Anticoagulants Are Comparable to Low Molecular Weight Heparin at Sustaining the Circulating Extracellular Vesicle and Inflammatory Profiles of Cancer Associated Thrombosis Patients: An Observational Pilot Study
Macleod, H., Copty, N., Doherty, D. G., Weiss, L., Fouhy, E., Power, R. F., Ryan, N., Saeed, K., O’Rourke, E., Faryal, R., et al. (2025) · Cancer Medicine · doi:10.1002/cam4.70920 · Cited by 4
Acceptability and feasibility of a nursing intervention to promote sensitive mother-infant interactions in the NICU
Lavallée, A., Côté, J., Luu, T. M., Bell, L., Grou, B., Blondin, S. E., & Aita, M. (2022) · Journal of Neonatal Nursing · doi:10.1016/j.jnn.2022.07.011 · Cited by 4
Effect of Tacrolimus Levels on Cerebral Blood Flow and Cognitive Function: A Pilot Study
Tariq, H., Young, K. J., Montgomery, R. N., Mukherjee, R., Lepping, R. J., Brooks, W. M., & Gupta, A. (2024) · Kidney360 · doi:10.34067/kid.0000000000000473 · Cited by 4
Feasibility of allied health assistant management of people with acute hip fracture: protocol for a feasibility randomised controlled trial
Snowdon, D. A., Vincent, P., Callisaya, M. L., Collyer, T. A., Wang, Y. T., & Taylor, N. F. (2021) · BMJ Open · doi:10.1136/bmjopen-2021-054298 · Cited by 4
A 16-week progressive exercise training intervention in treatment-naïve chronic lymphocytic leukaemia: a randomised-controlled pilot study
Brown, F. F., Oliver, R., Eddy, R., Causer, A. J., Emery, A., Collier‐Bain, H. D., Dutton, D. A., Crowe, J., Augustine, D. X., Graby, J., et al. (2024) · Frontiers in Oncology · doi:10.3389/fonc.2024.1472551 · Cited by 3
The Brain in Cross-Cultural Adjustment: A Pilot Study of Japanese Expatriates Living in the United States
Kokubun, K., Nemoto, K., & Yamakawa, Y. (2025) · Brain Sciences · doi:10.3390/brainsci15060617 · Cited by 3
Balancing best practice and reality in behavioral intervention development: A survey of principal investigators funded by the National Institutes of Health
von Klinggraeff, L., Burkart, S. A., Pfledderer, C. D., McLain, A. C., Armstrong, B., Weaver, R. G., & Beets, M. W. (2024) · Translational Behavioral Medicine · doi:10.1093/tbm/ibae009 · Cited by 3
Memory dependent modulation of hippocampal theta power through frontoparietal phase synchronous brain stimulation
Ehrhardt, N. M., Hanna, J., Hayek, D., Fleischmann, R., Grittner, U., Thielscher, A., Flöel, A., & Antonenko, D. (2025) · Scientific Reports · doi:10.1038/s41598-025-09841-y · Cited by 3
Inorganic nitrate supplementation may improve diastolic function and the O2 cost of exercise in cancer survivors: a pilot study
Turpin, V. G., Lovoy, G. M., Parr, S. K., Hammond, S. T., Post, H. K., Caldwell, J. T., Banister, H. R., Scheuermann, B., Colburn, T. D., & Ade, C. J. (2022) · Supportive Care in Cancer · doi:10.1007/s00520-022-07520-6 · Cited by 3
A Pilot Evaluation of an Educational Video to Support Consent to a Pediatric Malignancy Biobank
Robertson, E. G., Komesaroff, R., Eisenstat, D. D., Robertson, A., & Ludlow, L. E. A. (2024) · Biopreservation and Biobanking · doi:10.1089/bio.2024.0011 · Cited by 3
Rationale and Design of a Study to Assess the Engagement and Usefulness of the Care4Today Connect Digital Health Application for Disease Management in Coronary Artery Disease and Peripheral Artery Disease (iPACE‐CVD Study)
Lakkireddy, D., Angiolillo, D. J., Charlton-Ouw, K. M., Jefferson, B. K., Peeran, S. M., Bisharat, M. B., Ortega‐Paz, L., Harxhi, A., Kaul, S., Michaud, E., et al. (2024) · Clinical Cardiology · doi:10.1002/clc.70039 · Cited by 3
Early initiation of fast‐track care for persons living with HIV initiating dolutegravir‐based regimens during a period of severe civil unrest in Port‐au‐Prince, Haiti: a pilot randomized trial
Marc, J. B., Pierre, S., Ducatel, O., Homeus, F., Zion, A., Rivera, V. R., Dorvil, N., Sévère, P., Guiteau, C., Rouzier, V., et al. (2025) · Journal of the International AIDS Society · doi:10.1002/jia2.26419 · Cited by 3
Conducting Psychological Intervention Research in the Information Age: Reconsidering the “State of the Field”
Jones, D. J., Anton, M. T., Zachary, C., & Loiselle, R. (2018) · Journal of Technology in Behavioral Science · doi:10.1007/s41347-018-0072-4 · Cited by 3
The Development and Feasibility of a Manualised Therapeutic Playgroup for Children with Developmental Delay
Armstrong, J., Pieterse, B., Elliott, C. M., Wray, J. A., Davidson, E., Mizen, J., & Girdler, S. J. (2020) · Journal of Child and Family Studies · doi:10.1007/s10826-020-01789-z · Cited by 3
Pilot randomized controlled trial Protocol: Life context-informed pre-visit planning to improve care plans for primary care patients with multiple chronic conditions including diabetes
Magnan, E., Gosdin, M., Tancredi, D. J., & Jerant, A. F. (2021) · Journal of Multimorbidity and Comorbidity · doi:10.1177/26335565211062387 · Cited by 3
An Extended Commentary on Post-publication Peer Review in Organizational Neuroscience
Prochilo, G. A., Louis, W. R., Bode, S., Zacher, H., & Molenberghs, P. (2019) · Meta-Psychology · doi:10.15626/mp.2018.935 · Cited by 3
Mental Imagery to Reduce Alcohol-related harm in patients with alcohol dependence and alcohol-related liver damaGE: the MIRAGE pilot trial protocol
Dhanda, A., Allende, H., Allgar, V. L., Andrade, J., Bailey, M. P., Callaghan, L., Cocking, L., Goodwin, E., Hawton, A., Hayward, C. J., et al. (2022) · BMJ Open · doi:10.1136/bmjopen-2021-060498 · Cited by 3
Set up and assessment of progression criteria for internal pilots: the Brushing RemInder 4 Good oral HealTh (BRIGHT) trial example
Ainsworth, H. R., Marshman, Z., Whiteside, K., Sykes, D., Fairhurst, C. M., Turner, E., Chestnutt, I. G., Day, P. F., Dey, D., Elliott, L., et al. (2023) · Pilot and Feasibility Studies · doi:10.1186/s40814-023-01243-z · Cited by 3
Relationship Between Aerodynamic Measurement of Maximum Phonation Time With Acoustic Analysis and the Effects of Sex and Dysphonia Type
Saeedi, S., Khoddami, S. M., Dabirmoghaddam, P., Jalaie, S., & Aghajanzadeh, M. (2023) · Journal of Voice · doi:10.1016/j.jvoice.2023.02.026 · Cited by 3
Pilot study of the effectiveness of a telehealth group for improving peer relationships for adolescents with neurofibromatosis type 1
Glad, D. M., Pardej, S. K., Olszewski, E., & Klein-Tasman, B. (2024) · Orphanet Journal of Rare Diseases · doi:10.1186/s13023-024-03093-1 · Cited by 4
A prospective non-randomized feasibility study of an online membership-based fitness program for promoting physical activity in people with mobility impairments
Malone, L. A., Mehta, T. S., Mendonca, C. J., Mohanraj, S., & Thirumalai, M. (2024) · Pilot and Feasibility Studies · doi:10.1186/s40814-024-01528-x · Cited by 3
Small numbers, big decisions: guidance for sample size in pilot trials
Lewis, M., Bromley, K., & Joseph, R. (2025) · BMJ · doi:10.1136/bmj.r1885 · Cited by 3
Gamified optimized diabetes management with artificial intelligence-powered rural telehealth intervention (GODART): protocol for an optimization pilot and feasibility trial
Mehta, T., John, T., El Zein, A., Faught, V., Nawshin, T., Chilke, T. S., Cohen, C. W., Cherrington, A., & Thirumalai, M. (2025) · JMIR Research Protocols · doi:10.2196/70271 · Cited by 2
Feasibility and preliminary effects of an app-based physical activity intervention for individuals with depression (MoodMover): A protocol for a single-arm, pre-post intervention study
Tang, Y., Gierc, M., La, H., Liu, S., Lam, R. W., Puterman, E., & Faulkner, G. (2025) · PLOS One · doi:10.1371/journal.pone.0321958 · Cited by 2
Response inhibition training as an intervention to modify liking and wanting for foods based on energy density: a proof of concept study
Moore, H., White, M. J., Finlayson, G., & King, N. A. (2023) · Journal of Behavioral Medicine · doi:10.1007/s10865-023-00453-3 · Cited by 2
Evaluating the Impact of Seated Pilates on Functional Outcomes Among Those With Mild, Moderate, and Severe Multiple Sclerosis Impairment: A Pilot Feasibility Trial
Adamson, B., Lindsay, K. G., Almasri, L., Duffy, M. K., Kirkpatrick, S., & Hernandez, M. E. (2024) · Adapted Physical Activity Quarterly · doi:10.1123/apaq.2023-0159 · Cited by 3
An mHealth Intervention With Financial Incentives to Promote Smoking Cessation and Physical Activity Among Black Adults: Protocol for a Feasibility Randomized Controlled Trial
Alexander, A. C., Businelle, M. S., Cheney, M. K., Cohn, A. M., McNeill, L. H., Short, K. R., Frank-Pearce, S. G., Bradley, D., Estrada, K., Flores, I., et al. (2024) · JMIR Research Protocols · doi:10.2196/69771 · Cited by 2
Using the Q-method to explore pregnant and breastfeeding women’s perceptions of antibiotic use in Mahajanga, Northwest Madagascar
Rakotosamimanana, S., Harimanana, A. N., Razafindrabesa, T., Ravololomihanta, V., Kapesa, L., Mattern, C., & Kassié, D. (2025) · BMC Public Health · doi:10.1186/s12889-025-23140-1 · Cited by 2
Association Between Brain Substructure Dose and Scholastic Performance in Pediatric Brain Tumor Survivors Who Received Radiation Therapy
Vega, R. B. M., Indelicato, D. J., Bradley, J. A., Mobley, E. M., Viviers, E., Morris, C. G., Markatia, A., Ramahi, Y., Delgado, C. F., Fisher, C. L., et al. (2025) · International Journal of Radiation Oncology*Biology*Physics · doi:10.1016/j.ijrobp.2025.07.1418 · Cited by 2
The ‘ Reducing Psychosis Risk by Targeting Trauma’ Trial: Protocol of a Feasibility Randomised Controlled Trial of Trauma‐Focused Cognitive Behavioural Therapy and Eye Movement Desensitisation and Reprocessing Therapy for People With At‐Risk Mental States
Varese, F., Cartwright, K. L., Larkin, A., Sandys, M., Flinn, A., Newton, A., Lamonby, J., Samji, M., Holden, C., Bowe, S. E., et al. (2025) · Early Intervention in Psychiatry · doi:10.1111/eip.70095 · Cited by 2
Feasibility of a cluster randomised trial on the effect of trauma life support training: a pilot study in India
Wärnberg, M. G., Basak, D., Berg, J., Chatterjee, S., Felländer‐Tsai, L., Ghag, G. S., Juillard, C. J., Khajanchi, M. U., Khan, T., Mishra, A. K., et al. (2025) · BMJ Open · doi:10.1136/bmjopen-2025-099020 · Cited by 2
Evaluating the feasibility and potential effectiveness of a mobile-based intervention to promote the mental health of informal caregivers of persons with dementia in Singapore: Results from a mixed-methods two-arm pilot randomized controlled trial
Yuan, Q., Lee, Y. T., Samari, E., Zhang, Y., Goveas, R. R., Ng, L. L., & Subramaniam, M. (2025) · Journal of Affective Disorders · doi:10.1016/j.jad.2025.121059 · Cited by 2
Outcomes of a Pilot Mobile App Just-in-Time-Adaptive-Intervention Supporting Coping Skills in Trauma Affected People with HIV
Gasik, R. E., Skeen, S. J., Tokarz, S., Theall, K. P., & Clum, G. A. (2026) · AIDS and Behavior · doi:10.1007/s10461-026-05032-6 · Cited by 2
Feasibility and pilot efficacy of cognitive remediation for people in residential substance use treatment
Allan, J., Thompson, A., Carlyle, M., Thomas, M., & Medalia, A. A. (2021) · Drug and Alcohol Review · doi:10.1111/dar.13288 · Cited by 2
Targeted Teacher Education to Improve Primary Preservice Teachers’ Knowledge and Understanding of UV and Effective Sun Teachers’ Knowledge and Understanding of UV and Effective Sun Protection Measures for Children Protection Measures for Children
Scott, J. J., Johnston, R., Bear, N., Bear, N., Gregory, S., Bear, N., Blane, S., Strickland, M., Darby, J., & Gray, E. S. (2021) · The Australian journal of teacher education · doi:10.14221/ajte.2021v46n3.4 · Cited by 2
Evaluation of a Brief Attachment-Based Parenting Program: Aware Parenting
Baltrotsky, K., Bradbury, J., Rose, M., Grace, S., & Doran, F. M. (2022) · Child & Family Behavior Therapy · doi:10.1080/07317107.2022.2141600 · Cited by 2
A trauma-informed intervention for foster caregivers in Central Massachusetts: A mixed methods pilot evaluation
Campbell, J. K., Jiménez, S. P. S., Ahola, S. B., Hempstead, M., Shei, A., & Rothman, E. F. (2023) · Children and Youth Services Review · doi:10.1016/j.childyouth.2023.107086 · Cited by 2
A personalised nutrition intervention for adolescent depression: a mixed-methods feasibility pilot study
Campisi, S. C., Liang, M., Anthony, S. J., Dettmer, E. L., & Korczak, D. J. (2024) · British Journal Of Nutrition · doi:10.1017/s0007114524001338 · Cited by 2
Online Intervention for Muscle Tension Dysphonia
Nguyen‐Feng, V. N., Frazier, P. A., Liu, Y., Feddema, E., Wils, B., Nikcevich, E., Stockness, A., Lim, K. O., Hu, A. C., Butcher, L., et al. (2025) · JAMA Otolaryngology–Head & Neck Surgery · doi:10.1001/jamaoto.2024.5255 · Cited by 3
Saving lives together in sport: − A pilot study attempting to increase the number and availability of automated external defibrillators and to initiate voluntary training in cardiopulmonary resuscitation with the use of automated external defibrillators in Norwegian amateur football clubs
Steenstrup, S. E., Kramer‐Johansen, J., & Berge, H. M. (2025) · Resuscitation · doi:10.1016/j.resuscitation.2025.110586 · Cited by 2
Improving facial prosthesis construction with a contactless scanning and digital workflow: A feasibility crossover randomized controlled trial of digital versus conventional manufacture of orbital and nasal prostheses
Jablonski, R. Y., Coward, T. J., Reed, C. A., Bartlett, P., Keeling, A. J., Bojke, C., Nattress, B. R., & Pavitt, S. H. (2025) · Journal of Prosthetic Dentistry · doi:10.1016/j.prosdent.2025.03.002 · Cited by 2
A pilot study to evaluate the feasibility and acceptability of a mobile phone counselling intervention for perinatal women living with HIV and depression in India
Duggal, M., Sarna, A., Dahiya, N., Singh, R. J., Jeon, S., Subramanian, A., Sood, M., Singh, P., Kaur, G., & Reynolds, N. R. (2025) · Oxford Open Digital Health · doi:10.1093/oodh/oqaf012 · Cited by 2
Feasibility, acceptability, and exploratory outcomes of a virtual cognitive behavioural therapy‐based group intervention for persistent fatigue in endometriosis: The Managing Fatigue in Endometriosis (MEND) programme
Spyrelis, A., Loades, M. E., & Roomaney, R. (2025) · British Journal of Health Psychology · doi:10.1111/bjhp.70014 · Cited by 2
Accounting for Pilot Study Uncertainty in Sample Size Determination of Randomized Controlled Trials
Shi, Y., Liu, F., Li, S., & Chen, J. (2020) · Statistics in Biopharmaceutical Research · doi:10.1080/19466315.2020.1831951 · Cited by 3
Are new words predictable?
Zaefferer, D. (2021) · Studies in language companion series · doi:10.1075/slcs.220.05zae · Cited by 2
Staying Active with Multimorbidity In Acute hospital settings (StAMInA) trial: protocol for a feasibility randomised controlled trial of allied health assistant mobility rehabilitation for patients with multimorbidity
Snowdon, D. A., Wang, Y. T., Callisaya, M. L., Collyer, T. A., Jolliffe, L. J., Johns, N. A., Vincent, P., Pragash, N., & Taylor, N. F. (2024) · BMJ Open · doi:10.1136/bmjopen-2023-078843 · Cited by 2
Circulating miRNA profiles and the risk of hemorrhagic transformation after thrombolytic treatment of acute ischemic stroke: a pilot study
Stańczak, M., Wyszomirski, A., Słonimska, P., Kołodziej, B., Jabłoński, B., Stanisławska‐Sachadyn, A., & Karaszewski, B. (2024) · Frontiers in Neurology · doi:10.3389/fneur.2024.1399345 · Cited by 2
Three-outcome designs for external pilot trials with progression criteria
Wilson, D. T., Hudson, E. M., & Brown, S. (2024) · BMC Medical Research Methodology · doi:10.1186/s12874-024-02351-x · Cited by 2
Feasibility of an exergaming training program in type 2 diabetes mellitus: A mixed method study
Savić, N., Petry, H., de Bruin, E. D., Lehmann, R., Eggenberger, P., Adcock, M., Hirschmann, R., & Knols, R. H. (2024) · Digital Health · doi:10.1177/20552076241285090 · Cited by 2
E-Cigarette switching and financial incentives to promote combustible cigarette cessation among adults accessing shelter services: A pilot study
Sifat, M. S., Alexander, A. C., Businelle, M. S., Frank-Pearce, S. G., Boozary, L. K., Wagener, T. L., Ahluwalia, J. S., & Kendzor, D. E. (2024) · Drug and Alcohol Dependence Reports · doi:10.1016/j.dadr.2024.100295 · Cited by 2

Authors: Lee, E. C., Whitehead, A. L., Jacques, R. M., & Julious, S. A.
Affiliations: University of Sheffield (GB)
Corresponding author: Julious, Steven A
ORCID: Lee, Ellen C
Whitehead, Amy L
Jacques, Richard M
Julious, Steven A
Venue: BMC Medical Research Methodology
Publisher: Springer Science and Business Media LLC
Published: 2014-03-20
Volume: 14
Issue: 1
Pages: 41-41
ISSN: 1471-2288 (ISSN-L)
DOI: 10.1186/1471-2288-14-41
PMC: PMC3994566
OpenAlex: W2108052506
Cited by: 423
References: 31
Citations by year: 2026: 26 · 2025: 60 · 2024: 50 · 2023: 59 · 2022: 52 · 2021: 40 · 2020: 34 · 2019: 30 · 2018: 31 · 2017: 16 · 2016: 14 · 2015: 9 · 2014: 2
FWCI: 14.45
Type: journal article
Language: en
Open access: gold
doi.org
Licence: cc-by
www.springer.com/tdm (unspecified)
Locations: BMC Medical Research Methodology (publishedVersion, cc-by)
Europe PMC (PubMed Central) (publishedVersion, cc-by)
PubMed Central (publishedVersion, cc-by)
PubMed (publishedVersion)
White Rose Research Online (University of Leeds, The University of Sheffield, University of York) (acceptedVersion)
ePrints Soton (University of Southampton) (acceptedVersion)
Topics: Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Clinical Trials
Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods and Bayesian Inference
Physical Sciences › Mathematics › Statistics and Probability › Statistical Methods in Epidemiology
Keywords: pilot trial, significance threshold, confidence intervals, Bayesian analysis, surrogate endpoints, minimal clinically important difference, health technology assessment, treatment effect estimation, hypothesis testing
MeSH terms: Bayes Theorem, Data Interpretation, Statistical, Humans, Leg Ulcer, Pilot Projects, Research Design, Biomarkers, Confidence Intervals, Treatment Outcome

About these checks

Registry, retraction, Cites and statistics results are computed, not judged, with one exception: a printed reference that no index lists is read once by a model before it counts as a citation error. Claims are extracted by a model and kept only when their quote appears verbatim in the text; each cited source is then checked, with a model’s help, for whether it says what the claim relies on. Those verdicts can be wrong — read the claim’s quote and the cited source before relying on one.

Statistics and arithmetic are recomputed, not judged.

Confidence bands are shown only for fields calibrated on our annotated test set. None is calibrated yet, so no band is shown.

What counts as a claim ›How we measure confidence ›How we check this ›

Are you an author, or do you see a problem with this page? Request a review

The statistical interpretation of pilot trials: should significance thresholds be reconsidered?

Abstract

Overview

Integrity signals

Figures

Verification

Claims the paper makes

Claims other papers make about this one

Self-check

Citations

Cites

Cited by

Related

Metadata

Record

About these checks