By: Helmut Kiene, M.D. Original title: Eine Methode zur Wirksamkeitsbeurteilung bei kleinen Patientenzahlen. Der Merkurstab 1996; 49:277-9. DOI: https://doi.org/10.14271/DMS-16907-DE English by A. R. Meuss, FIL, MTA. This translation is published with the kind permission of the journal Der Merkurstab.
JAM Vol. 14(1), Spring 1997
The method presented in this paper will, in the author's opinion, serve to document clinical results when numbers are small. There is no need for controls, and documentation may also be retrospective.
Essentially the method bases on a criterion often used in everyday clinical practice - the ratio of times for which symptoms persisted before and after treatment. If the time for which symptoms persisted after starting treatment is relatively short compared to the time before treatment, this may be taken to indicate that the treatment was successful. Results are most impressive if the post-treatment period comes close to zero. This is the kind of "instant cure" known from neural therapy, for instance. But when symptoms have persisted for five years, for example, and have disappeared four weeks after starting treatment, this, too, is a powerful indication of efficacy.
Such an indication may actually gain power of evidence if a similar situation can be shown for several patients and if this is also the total number of patients who have had the treatment in question. (This is the crux of the matter. There must be no selection of successful cases!) A highly convincing documentation of the efficacy of a treatment would be the following (unselected) complete set of six case records.
Citation: Kiene, H. (1997). A Method of Assessing Efficacy with Small Patient Numbers (A. R. Meuss, Trans.). Journal of Anthroposophic Medicine, 14(1), 47–49.
(2017) Riley, David S.; Barber, Melissa S.; Kienle, Gunver S.; Aronson, Jeffrey K.; von Schoen-Angerer, Tido; Tugwell, Peter; Kiene, Helmut; Helfand, Mark; Altman, Douglas G.; Sox, Harold; Werthmann, Paul G.; Moher, David; Rison, Richard A.
Background: Well-written and transparent case reports (1) reveal early signals of potential benefits, harms, and information on the use of resources; (2) provide information for clinical research and clinical practice guidelines, and (3) inform medical education. High-quality case reports are more likely when authors follow reporting guidelines. During 2011e2012, a group of clinicians, researchers, and journal editors developed recommendations for the accurate reporting of information in case reports that resulted in the CARE (CAse REport)
Statement and Checklist. They were presented at the 2013 International Congress on Peer Review and Biomedical Publication, have been endorsed by multiple medical journals, and translated into nine languages.
Objectives: This explanation and elaboration document has the objective to increase the use and dissemination of the CARE Checklist in writing and publishing case reports.
Article Design and Setting: Each item from the CARE Checklist is explained and accompanied by published examples. The explanations and examples in this document are designed to support the writing of high-quality case reports by authors and their critical appraisal by editors, peer reviewers, and readers.
Results and Conclusion: This article and the 2013 CARE Statement and Checklist, available from the CARE website [www.care-statement.org] and the EQUATOR Network [www.equator-network.org], are resources for improving the completeness and transparency of case reports.
Keywords: Case report; Case study; EQUATOR network; Health research reporting guidelines; CARE guideline; Timelines; N-of-1
Citation: Riley, D. S., Barber, M. S., Kienle, G. S., Aronson, J. K., von Schoen-Angerer, T., Tugwell, P., Kiene, H., Helfand, M., Altman, D. G., Sox, H., Werthmann, P. G., Moher, D., Rison, R. A., Shamseer, L., Koch, C. A., Sun, G. H., Hanaway, P., Sudak, N. L., Kaszkin-Bettag, M., … Gagnier, J. J. (2017). CARE guidelines for case reports: Explanation and elaboration document. Journal of Clinical Epidemiology, 89, 218–235. https://doi.org/10.1016/j.jclinepi.2017.04.026
[Input to repository: Riley, David S.; Barber, Melissa S., Kienle, Gunver S.; Aronson, Jeffrey K.; von Schoen-Angerer, Tido; Tugwell, Peter; Kiene, Helmut; Helfand, Mark; Altman, Douglas G.; Sox, Harold; Werthmann, Paul G.; Moher, David; Rison, Richard A.; ]
