Interpretation of guidelines for reporting outcomes in trial reports: the CONSORT-Outcomes 2022 Extension_Chinese Journal of Evidence-Based Medicine

Authors：

PANG Bo ^1,2 , ZHANG Chenyao ^1,2 , MA Yucong ^1,2 , DU Liang ³ , ZHANG Yonggang ³ , TIAN Jinhui ⁴ ,  ZHANG Junhua ^1,2

1. NMPA Key Laboratory for Evidence-Based Evaluation of Traditional Chinese Medicine, Tianjin 301617, P. R. China;
2. Evidence-based Medicine Center, Tianjin University of Traditional Chinese Medicine, Tianjin 301617, P. R. China;
3. Department of Evidence-Based Medicine and Clinical Epidemiology, West China Hospital, Sichuan University, Chengdu 610041, P. R. China;
4. Evidence-based Medicine Center, Lanzhou University, Lanzhou 730000, P. R. China;

Corresponding author：

ZHANG Junhua, Email: zjhtcm@foxmail.com

Keywords：

CONSORT-Outcomes; Clinical trials; Outcome; Reporting standard; Interpretation

DOI：

10.7507/1672-2531.202312088

Video：

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Abstract Full text Figures/Tables Video References Cited by

The complete, transparent, and standardized reporting of the outcome of a clinical trial is a key factor in ensuring the practicality, reproducibility, and transparency of the trial, and reducing bias in selective reporting. The Consolidated Standards of Reporting Trials (CONSORT) 2010 statement provides normative guidelines for the reporting of clinical trials. In December 2022, JAMA released the Guidelines for Reporting Outcomes in Trial Reports (CONSORT-Outcomes) 2022 Extension, aiming to explain the entries related to trial outcomes, sample size, statistical methods, and auxiliary analysis in the CONSORT 2010 statement, to further improve the standards for outcome reporting in clinical trial reports. This article combines research examples to interpret the CONSORT-Outcomes 2022 Extension, in order to provide normative references for domestic scholars to report clinical research results.

1.	Schulz KF, Altman DG, Moher D, et al. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ, 2010, 340: c332.
2.	Clarke M. Standardising outcomes for clinical trials and systematic reviews. Trials, 2007, 8: 39.
3.	Butcher NJ, Monsour A, Mew EJ, et al. Guidelines for reporting outcomes in trial reports: the CONSORT-outcomes 2022 extension. JAMA, 2022, 328(22): 2252-2264.
4.	Cuschieri S. The CONSORT statement. Saudi J Anaesth, 2019, 13(Suppl 1): S27-S30.
5.	Moher D, Hopewell S, Schulz KF, et al. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. Int J Surg, 2012, 10(1): 28-55.
6.	Bhaloo Z, Adams D, Liu Y, et al. Primary outcomes reporting in trials (PORTal): a systematic review of inadequate reporting in pediatric randomized controlled trials. J Clin Epidemiol, 2017, 81: 33-41.
7.	Mayo-Wilson E, Fusco N, Li T, et al. Multiple outcomes and analyses in clinical trials create challenges for interpretation and research synthesis. J Clin Epidemiol, 2017, 86: 39-50.
8.	Dwan K, Altman DG, Arnaiz JA, et al. Systematic review of the empirical evidence of study publication bias and outcome reporting bias. PLoS One, 2008, 3(8): e3081.
9.	Ioannidis JP, Evans SJ, Gøtzsche PC, et al. Better reporting of harms in randomized trials: an extension of the CONSORT statement. Ann Intern Med, 2004, 141(10): 781-788.
10.	Calvert M, Blazeby J, Altman DG, et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA, 2013, 309(8): 814-822.
11.	Butcher NJ, Mew EJ, Monsour A, et al. Outcome reporting recommendations for clinical trial protocols and reports: a scoping review. Trials, 2020, 21(1): 620.
12.	Butcher NJ, Monsour A, Mew EJ, et al. Improving outcome reporting in clinical trial reports and protocols: study protocol for the Instrument for reporting Planned Endpoints in Clinical Trials (InsPECT). Trials, 2019, 20(1): 161.
13.	Moher D, Schulz KF, Simera I, et al. Guidance for developers of health research reporting guidelines. PLoS Med, 2010, 7(2): e1000217.
14.	Butcher NJ, Mew EJ, Saeed L, et al. Guidance for reporting outcomes in clinical trials: scoping review protocol. BMJ Open, 2019, 9(2): e023001.
15.	Butcher NJ, Monsour A, Mew EJ, et al. Guidelines for reporting outcomes in trial protocols: the SPIRIT-outcomes 2022 extension. JAMA, 2022, 328(23): 2345-2356.
16.	OMERACT. OMERACT (outcome measures in rheumatology): domain resources.
17.	Williamson P, Altman D, Blazeby J, et al. Driving up the quality and relevance of research through the use of agreed core outcomes. J Health Serv Res Policy, 2012, 17(1): 1-2.
18.	Carli F, Bousquet-Dion G, Awasthi R, et al. Effect of multimodal prehabilitation vs postoperative rehabilitation on 30-day postoperative complications for frail patients undergoing resection of colorectal cancer: a randomized clinical trial. JAMA Surg, 2020, 155(3): 233-242.
19.	Page MJ, McKenzie JE, Forbes A. Many scenarios exist for selective inclusion and reporting of results in randomized trials and systematic reviews. J Clin Epidemiol, 2013, 66(5): 524-537.
20.	Mayo-Wilson E, Li T, Fusco N, et al. Cherry-picking by trialists and meta-analysts can drive conclusions about intervention efficacy. J Clin Epidemiol, 2017, 91: 95-110.
21.	Chan AW, Tetzlaff JM, Gøtzsche PC, et al. SPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trials. BMJ, 2013, 346: e7586.
22.	Zarin DA, Tse T, Williams RJ, et al. The ClinicalTrials. gov results database-update and key issues. N Engl J Med, 2011, 364(9): 852-860.
23.	Persell SD, Peprah YA, Lipiszko D, et al. Effect of home blood pressure monitoring via a smartphone hypertension coaching application or tracking application on adults with uncontrolled hypertension: a randomized clinical trial. JAMA Netw Open, 2020, 3(3): e200255.
24.	Terwee CB, Peipert JD, Chapman R, et al. Minimal important change (MIC): a conceptual clarification and systematic review of MIC estimates of PROMIS measures. Qual Life Res, 2021, 30(10): 2729-2754.
25.	Carrasco-Labra A, Devji T, Qasim A, et al. Minimal important difference estimates for patient-reported outcomes: a systematic survey. J Clin Epidemiol, 2021, 133: 61-71.
26.	Devji T, Carrasco-Labra A, Guyatt G. Mind the methods of determining minimal important differences: three critical issues to consider. Evid Based Ment Health, 2021, 24(2): 77-81.
27.	Okereke OI, Vyas CM, Mischoulon D, et al. Effect of long-term supplementation with marine omega-3 fatty acids vs placebo on risk of depression or clinically relevant depressive symptoms and on change in mood scores: a randomized clinical trial. JAMA, 2021, 326(23): 2385-2394.
28.	Hochberg MC, Guermazi A, Guehring H, et al. Effect of intra-articular sprifermin vs placebo on femorotibial joint cartilage thickness in patients with osteoarthritis: the FORWARD randomized clinical trial. JAMA, 2019, 322(14): 1360-1370.
29.	International Conference on Harmonisation. E9: statistical principles for clinical trials: current step 4 version.
30.	Mignard C, Maho-Vaillant M, Golinski ML, et al. Factors associated with short-term relapse in patients with pemphigus who receive rituximab as first-line therapy: a post hoc analysis of a randomized clinical trial. JAMA Dermatol, 2020, 156(5): 545-552.
31.	Yu L, Luo L, Long X, et al. High-dose PPI-amoxicillin dual therapy with or without bismuth for first-line Helicobacter pylori therapy: a randomized trial. Helicobacter, 2019, 24(4): e12596.
32.	Kitt J, Fox R, Frost A, et al. Long-term blood pressure control after hypertensive pregnancy following physician-optimized self-management: the POP-HT randomized clinical trial. JAMA, 2023, 330(20): 1991-1999.
33.	Ferreira-González I, Permanyer-Miralda G, Busse JW, et al. Methodologic discussions for using and interpreting composite endpoints are limited, but still identify major concerns. J Clin Epidemiol, 2007, 60(7): 651-657.
34.	Cordoba G, Schwartz L, Woloshin S, et al. Definition, reporting, and interpretation of composite outcomes in clinical trials: systematic review. BMJ, 2010, 341: c3920.
35.	Eunethta (European Network for Health Technology Assessment). Guideline: endpoints used for relative effectiveness assessment of pharmaceuticals: composite endpoints.
36.	Chappell LC, Bell JL, Smith A, et al. Ursodeoxycholic acid versus placebo in women with intrahepatic cholestasis of pregnancy (PITCHES): a randomised controlled trial. Lancet, 2019, 394(10201): 849-860.
37.	Sholzberg M, Tang GH, Rahhal H, et al. Effectiveness of therapeutic heparin versus prophylactic heparin on death, mechanical ventilation, or intensive care unit admission in moderately ill patients with covid-19 admitted to hospital: RAPID randomised clinical trial. BMJ, 2021, 375: n2400.
38.	Dyrbye LN, Shanafelt TD, Gill PR, et al. Effect of a professional coaching intervention on the well-being and distress of physicians: a pilot randomized clinical trial. JAMA Intern Med, 2019, 179(10): 1406-1414.
39.	Dankiewicz J, Cronberg T, Lilja G, et al. Targeted hypothermia versus targeted Normothermia after out-of-hospital cardiac arrest (TTM2): a randomized clinical trial-Rationale and design. Am Heart J, 2019, 217: 23-31.
40.	De Los Reyes A, Augenstein TM, Wang M, et al. The validity of the multi-informant approach to assessing child and adolescent mental health. Psychol Bull, 2015, 141(4): 858-900.
41.	FDA-NIH BiomarkerWorking Group. BEST (biomarkers, endpoints, and other tools) resource.
42.	Hossain SJ, Tofail F, Mehrin SF, et al. Six-year follow-up of childhood stimulation on development of children with and without anemia. Pediatrics, 2023, 151(Suppl 2): e2023060221E.
43.	Cook JA, Julious SA, Sones W, et al. Practical help for specifying the target difference in sample size calculations for RCTs: the DELTA2 five-stage study, including a workshop. Health Technol Assess, 2019, 23(60): 1-88.
44.	Ferreira ML, Herbert RD, Ferreira PH, et al. A critical review of methods used to determine the smallest worthwhile effect of interventions for low back pain. J Clin Epidemiol, 2012, 65(3): 253-261.
45.	Mouelhi Y, Jouve E, Castelli C, et al. How is the minimal clinically important difference established in health-related quality of life instruments. Review of anchors and methods. Health Qual Life Outcomes, 2020, 18(1): 136.
46.	Treasure T, Farewell V, Macbeth F, et al. Pulmonary metastasectomy versus continued active monitoring in colorectal cancer (PulMiCC): a multicentre randomised clinical trial. Trials, 2019, 20(1): 718.
47.	European Medicines Agency. Guideline on multiplicity issues in clinical trials.
48.	Akl EA, Shawwa K, Kahale LA, et al. Reporting missing participant data in randomised trials: systematic survey of the methodological literature and a proposed guide. BMJ Open, 2015, 5(12): e008431.
49.	National Research Council (US) Panel on Handling Missing Data in Clinical Trials. The prevention and treatment of missing data in clinical trials. Washington (DC): National Academies Press (US), 2010.
50.	Rodgers H, Bosomworth H, Krebs HI, et al. Robot assisted training for the upper limb after stroke (RATULS): a multicentre randomised controlled trial. Lancet, 2019, 394(10192): 51-62.
51.	Kahan BC, Ahmad T, Forbes G, et al. Public availability and adherence to prespecified statistical analysis approaches was low in published randomized trials. J Clin Epidemiol, 2020, 128: 29-34.
52.	Bardy AH. Bias in reporting clinical trials. Br J Clin Pharmacol, 1998, 46(2): 147-150.
53.	Kirkham JJ, Dwan KM, Altman DG, et al. The impact of outcome reporting bias in randomised controlled trials on a cohort of systematic reviews. BMJ, 2010, 340: c365.

1. Schulz KF, Altman DG, Moher D, et al. CONSORT 2010 statement: updated guidelines for reporting parallel group randomised trials. BMJ, 2010, 340: c332.
2. Clarke M. Standardising outcomes for clinical trials and systematic reviews. Trials, 2007, 8: 39.
3. Butcher NJ, Monsour A, Mew EJ, et al. Guidelines for reporting outcomes in trial reports: the CONSORT-outcomes 2022 extension. JAMA, 2022, 328(22): 2252-2264.
4. Cuschieri S. The CONSORT statement. Saudi J Anaesth, 2019, 13(Suppl 1): S27-S30.
5. Moher D, Hopewell S, Schulz KF, et al. CONSORT 2010 explanation and elaboration: updated guidelines for reporting parallel group randomised trials. Int J Surg, 2012, 10(1): 28-55.
6. Bhaloo Z, Adams D, Liu Y, et al. Primary outcomes reporting in trials (PORTal): a systematic review of inadequate reporting in pediatric randomized controlled trials. J Clin Epidemiol, 2017, 81: 33-41.
7. Mayo-Wilson E, Fusco N, Li T, et al. Multiple outcomes and analyses in clinical trials create challenges for interpretation and research synthesis. J Clin Epidemiol, 2017, 86: 39-50.
8. Dwan K, Altman DG, Arnaiz JA, et al. Systematic review of the empirical evidence of study publication bias and outcome reporting bias. PLoS One, 2008, 3(8): e3081.
9. Ioannidis JP, Evans SJ, Gøtzsche PC, et al. Better reporting of harms in randomized trials: an extension of the CONSORT statement. Ann Intern Med, 2004, 141(10): 781-788.
10. Calvert M, Blazeby J, Altman DG, et al. Reporting of patient-reported outcomes in randomized trials: the CONSORT PRO extension. JAMA, 2013, 309(8): 814-822.
11. Butcher NJ, Mew EJ, Monsour A, et al. Outcome reporting recommendations for clinical trial protocols and reports: a scoping review. Trials, 2020, 21(1): 620.
12. Butcher NJ, Monsour A, Mew EJ, et al. Improving outcome reporting in clinical trial reports and protocols: study protocol for the Instrument for reporting Planned Endpoints in Clinical Trials (InsPECT). Trials, 2019, 20(1): 161.
13. Moher D, Schulz KF, Simera I, et al. Guidance for developers of health research reporting guidelines. PLoS Med, 2010, 7(2): e1000217.
14. Butcher NJ, Mew EJ, Saeed L, et al. Guidance for reporting outcomes in clinical trials: scoping review protocol. BMJ Open, 2019, 9(2): e023001.
15. Butcher NJ, Monsour A, Mew EJ, et al. Guidelines for reporting outcomes in trial protocols: the SPIRIT-outcomes 2022 extension. JAMA, 2022, 328(23): 2345-2356.
16. OMERACT. OMERACT (outcome measures in rheumatology): domain resources.
17. Williamson P, Altman D, Blazeby J, et al. Driving up the quality and relevance of research through the use of agreed core outcomes. J Health Serv Res Policy, 2012, 17(1): 1-2.
18. Carli F, Bousquet-Dion G, Awasthi R, et al. Effect of multimodal prehabilitation vs postoperative rehabilitation on 30-day postoperative complications for frail patients undergoing resection of colorectal cancer: a randomized clinical trial. JAMA Surg, 2020, 155(3): 233-242.
19. Page MJ, McKenzie JE, Forbes A. Many scenarios exist for selective inclusion and reporting of results in randomized trials and systematic reviews. J Clin Epidemiol, 2013, 66(5): 524-537.
20. Mayo-Wilson E, Li T, Fusco N, et al. Cherry-picking by trialists and meta-analysts can drive conclusions about intervention efficacy. J Clin Epidemiol, 2017, 91: 95-110.
21. Chan AW, Tetzlaff JM, Gøtzsche PC, et al. SPIRIT 2013 explanation and elaboration: guidance for protocols of clinical trials. BMJ, 2013, 346: e7586.
22. Zarin DA, Tse T, Williams RJ, et al. The ClinicalTrials. gov results database-update and key issues. N Engl J Med, 2011, 364(9): 852-860.
23. Persell SD, Peprah YA, Lipiszko D, et al. Effect of home blood pressure monitoring via a smartphone hypertension coaching application or tracking application on adults with uncontrolled hypertension: a randomized clinical trial. JAMA Netw Open, 2020, 3(3): e200255.
24. Terwee CB, Peipert JD, Chapman R, et al. Minimal important change (MIC): a conceptual clarification and systematic review of MIC estimates of PROMIS measures. Qual Life Res, 2021, 30(10): 2729-2754.
25. Carrasco-Labra A, Devji T, Qasim A, et al. Minimal important difference estimates for patient-reported outcomes: a systematic survey. J Clin Epidemiol, 2021, 133: 61-71.
26. Devji T, Carrasco-Labra A, Guyatt G. Mind the methods of determining minimal important differences: three critical issues to consider. Evid Based Ment Health, 2021, 24(2): 77-81.
27. Okereke OI, Vyas CM, Mischoulon D, et al. Effect of long-term supplementation with marine omega-3 fatty acids vs placebo on risk of depression or clinically relevant depressive symptoms and on change in mood scores: a randomized clinical trial. JAMA, 2021, 326(23): 2385-2394.
28. Hochberg MC, Guermazi A, Guehring H, et al. Effect of intra-articular sprifermin vs placebo on femorotibial joint cartilage thickness in patients with osteoarthritis: the FORWARD randomized clinical trial. JAMA, 2019, 322(14): 1360-1370.
29. International Conference on Harmonisation. E9: statistical principles for clinical trials: current step 4 version.
30. Mignard C, Maho-Vaillant M, Golinski ML, et al. Factors associated with short-term relapse in patients with pemphigus who receive rituximab as first-line therapy: a post hoc analysis of a randomized clinical trial. JAMA Dermatol, 2020, 156(5): 545-552.
31. Yu L, Luo L, Long X, et al. High-dose PPI-amoxicillin dual therapy with or without bismuth for first-line Helicobacter pylori therapy: a randomized trial. Helicobacter, 2019, 24(4): e12596.
32. Kitt J, Fox R, Frost A, et al. Long-term blood pressure control after hypertensive pregnancy following physician-optimized self-management: the POP-HT randomized clinical trial. JAMA, 2023, 330(20): 1991-1999.
33. Ferreira-González I, Permanyer-Miralda G, Busse JW, et al. Methodologic discussions for using and interpreting composite endpoints are limited, but still identify major concerns. J Clin Epidemiol, 2007, 60(7): 651-657.
34. Cordoba G, Schwartz L, Woloshin S, et al. Definition, reporting, and interpretation of composite outcomes in clinical trials: systematic review. BMJ, 2010, 341: c3920.
35. Eunethta (European Network for Health Technology Assessment). Guideline: endpoints used for relative effectiveness assessment of pharmaceuticals: composite endpoints.
36. Chappell LC, Bell JL, Smith A, et al. Ursodeoxycholic acid versus placebo in women with intrahepatic cholestasis of pregnancy (PITCHES): a randomised controlled trial. Lancet, 2019, 394(10201): 849-860.
37. Sholzberg M, Tang GH, Rahhal H, et al. Effectiveness of therapeutic heparin versus prophylactic heparin on death, mechanical ventilation, or intensive care unit admission in moderately ill patients with covid-19 admitted to hospital: RAPID randomised clinical trial. BMJ, 2021, 375: n2400.
38. Dyrbye LN, Shanafelt TD, Gill PR, et al. Effect of a professional coaching intervention on the well-being and distress of physicians: a pilot randomized clinical trial. JAMA Intern Med, 2019, 179(10): 1406-1414.
39. Dankiewicz J, Cronberg T, Lilja G, et al. Targeted hypothermia versus targeted Normothermia after out-of-hospital cardiac arrest (TTM2): a randomized clinical trial-Rationale and design. Am Heart J, 2019, 217: 23-31.
40. De Los Reyes A, Augenstein TM, Wang M, et al. The validity of the multi-informant approach to assessing child and adolescent mental health. Psychol Bull, 2015, 141(4): 858-900.
41. FDA-NIH BiomarkerWorking Group. BEST (biomarkers, endpoints, and other tools) resource.
42. Hossain SJ, Tofail F, Mehrin SF, et al. Six-year follow-up of childhood stimulation on development of children with and without anemia. Pediatrics, 2023, 151(Suppl 2): e2023060221E.
43. Cook JA, Julious SA, Sones W, et al. Practical help for specifying the target difference in sample size calculations for RCTs: the DELTA2 five-stage study, including a workshop. Health Technol Assess, 2019, 23(60): 1-88.
44. Ferreira ML, Herbert RD, Ferreira PH, et al. A critical review of methods used to determine the smallest worthwhile effect of interventions for low back pain. J Clin Epidemiol, 2012, 65(3): 253-261.
45. Mouelhi Y, Jouve E, Castelli C, et al. How is the minimal clinically important difference established in health-related quality of life instruments. Review of anchors and methods. Health Qual Life Outcomes, 2020, 18(1): 136.
46. Treasure T, Farewell V, Macbeth F, et al. Pulmonary metastasectomy versus continued active monitoring in colorectal cancer (PulMiCC): a multicentre randomised clinical trial. Trials, 2019, 20(1): 718.
47. European Medicines Agency. Guideline on multiplicity issues in clinical trials.
48. Akl EA, Shawwa K, Kahale LA, et al. Reporting missing participant data in randomised trials: systematic survey of the methodological literature and a proposed guide. BMJ Open, 2015, 5(12): e008431.
49. National Research Council (US) Panel on Handling Missing Data in Clinical Trials. The prevention and treatment of missing data in clinical trials. Washington (DC): National Academies Press (US), 2010.
50. Rodgers H, Bosomworth H, Krebs HI, et al. Robot assisted training for the upper limb after stroke (RATULS): a multicentre randomised controlled trial. Lancet, 2019, 394(10192): 51-62.
51. Kahan BC, Ahmad T, Forbes G, et al. Public availability and adherence to prespecified statistical analysis approaches was low in published randomized trials. J Clin Epidemiol, 2020, 128: 29-34.
52. Bardy AH. Bias in reporting clinical trials. Br J Clin Pharmacol, 1998, 46(2): 147-150.
53. Kirkham JJ, Dwan KM, Altman DG, et al. The impact of outcome reporting bias in randomised controlled trials on a cohort of systematic reviews. BMJ, 2010, 340: c365.

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