Journal
AMERICAN JOURNAL OF TRANSPLANTATION
Volume 12, Issue 7, Pages 1898-1907Publisher
WILEY-BLACKWELL
DOI: 10.1111/j.1600-6143.2012.04038.x
Keywords
Clinical efficacy; clinical trials; islet transplantation; Kaplan-Meier survival analysis; sample size; type 1 diabetes
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Funding
- FDA Office of Critical Path Programs Grant
- National Institute of Diabetes, Digestive and Kidney Diseases
- Juvenile Diabetes Research Foundation
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To estimate treatment effect size and other parameters required for planning the designs and analyses of future phase 3 islet transplant trials, we analyzed key clinical and laboratory outcomes of 347 allogeneic islet transplant recipients, using data from the Collaborative Islet Transplant Registry (CITR). At 1 year, approximately 59% of all transplant recipients were free of severe hypoglycemic events and maintained hemoglobin A1c (HbA1c) level of =6.5%. The KaplanMeier (KM) survival analyses showed that 69%, 54% and 44% of these 1-year responders maintained this composite endpoint at 2, 3 and 4 years, respectively. Ninety-one percent of all recipients were free of severe hypoglycemic episodes at 1 year. Furthermore, the KM survival estimates showed that 91%, 85% and 80% of these subjects maintained this clinical benefit at 2, 3 and 4 years, respectively. These results can be very useful in developing framework for study designs, sample size estimates, and statistical analysis plans for future pivotal trials of islet cell transplantation in type 1 diabetes.
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