دورية أكاديمية
Recommendations for the definition of clinical responder in insulin preservation studies.
| العنوان: | Recommendations for the definition of clinical responder in insulin preservation studies. |
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| المؤلفون: | Beam, Craig A, Gitelman, Stephen E, Palmer, Jerry P |
| المصدر: | Diabetes ; ISSN:1939-327X ; Volume:63 ; Issue:9 |
| بيانات النشر: | Silverchair Information Systems |
| سنة النشر: | 2014 |
| المجموعة: | PubMed Central (PMC) |
| الوصف: | Clinical responder studies should contribute to the translation of effective treatments and interventions to the clinic. Since ultimately this translation will involve regulatory approval, we recommend that clinical trials prespecify a responder definition that can be assessed against the requirements and suggestions of regulatory agencies. In this article, we propose a clinical responder definition to specifically assist researchers and regulatory agencies in interpreting the clinical importance of statistically significant findings for studies of interventions intended to preserve β-cell function in newly diagnosed type 1 diabetes. We focus on studies of 6-month β-cell preservation in type 1 diabetes as measured by 2-h-stimulated C-peptide. We introduce criteria (bias, reliability, and external validity) for the assessment of responder definitions to ensure they meet U.S. Food and Drug Administration and European Medicines Agency guidelines. Using data from several published TrialNet studies, we evaluate our definition (no decrease in C-peptide) against published alternatives and determine that our definition has minimum bias with external validity. We observe that reliability could be improved by using changes in C-peptide later than 6 months beyond baseline. In sum, to support efficacy claims of β-cell preservation therapies in type 1 diabetes submitted to U.S. and European regulatory agencies, we recommend use of our definition. |
| نوع الوثيقة: | article in journal/newspaper |
| اللغة: | English |
| العلاقة: | https://doi.org/10.2337/db14-0095Test; https://pubmed.ncbi.nlm.nih.gov/24722251Test; https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4141373Test/ |
| DOI: | 10.2337/db14-0095 |
| الإتاحة: | https://doi.org/10.2337/db14-0095Test https://pubmed.ncbi.nlm.nih.gov/24722251Test https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4141373Test/ |
| حقوق: | © 2014 by the American Diabetes Association. Readers may use this article as long as the work is properly cited, the use is educational and not for profit, and the work is not altered. |
| رقم الانضمام: | edsbas.3343F584 |
| قاعدة البيانات: | BASE |
| DOI: | 10.2337/db14-0095 |
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