Islet cell transplantation in type 1 diabetes: an analysis of efficacy outcomes and considerations for trial designs.
2012 Jul
Journal Article
Authors:
Tiwari, J.L.;
Schneider, B.;
Barton, F.;
Anderson, S.A.
Secondary:
Am J Transplant
Volume:
12
Pagination:
1898-907
Issue:
7
PMID:
22486926
DOI:
10.1111/j.1600-6143.2012.04038.x
Keywords:
Adult; Diabetes Mellitus, Type 1; Female; Glycated Hemoglobin A; Humans; Islets of Langerhans Transplantation; Male; Registries; Survival Analysis; Treatment Outcome
Abstract:
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 Kaplan-Meier (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.
