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A Model-based Approach to Sample Size Estimation in Recent Onset Type 1 Diabetes

Overview
Journal Diabetes Metab Res Rev
Specialty Endocrinology
Date 2016 Mar 19
PMID 26991448
Citations 18
Authors
Brian N Bundy
Jeffrey P Krischer
Affiliations
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Abstract

Background: The area under the curve C-peptide following a 2-h mixed meal tolerance test from 498 individuals enrolled on five prior TrialNet studies of recent onset type 1 diabetes from baseline to 12 months after enrolment were modelled to produce estimates of its rate of loss and variance.

Results: Age at diagnosis and baseline C-peptide were found to be significant predictors, and adjusting for these in an ANCOVA resulted in estimates with lower variance.

Conclusions: Using these results as planning parameters for new studies results in a nearly 50% reduction in the target sample size. The modelling also produces an expected C-peptide that can be used in observed versus expected calculations to estimate the presumption of benefit in ongoing trials. Copyright © 2016 John Wiley & Sons, Ltd.

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