Low-Dose ATG/GCSF in Established Type 1 Diabetes: A Five-Year Follow-up Report
- 25 February 2021
- journal article
- research article
- Published by American Diabetes Association in Diabetes
- Vol. 70 (5), 1123-1129
- https://doi.org/10.2337/db20-1103
Abstract
Previously, we demonstrated low-dose antithymocyte globulin (ATG) and granulocyte colony-stimulating factor (GCSF) immunotherapy preserved C-peptide for 2 years in a pilot study of patients with established type 1 diabetes (n = 25). Here, we evaluated the long-term outcomes of ATG/GCSF in study participants with 5 years of available follow-up data (n = 15). The primary end point was area under the curve (AUC) C-peptide during a 2-h mixed-meal tolerance test. After 5 years, there were no statistically significant differences in AUC C-peptide when comparing those who received ATG/GCSF versus placebo (P = 0.41). A modeling framework based on mean trajectories in C-peptide AUC over 5 years, accounting for differing trends between groups, was applied to recategorize responders (n = 9) and nonresponders (n = 7). ATG/GCSF reponders demonstrated nearly unchanged HbA1c over 5 years (mean [95% CI] adjusted change 0.29% [–0.69%, 1.27%]), but the study was not powered for comparisons against nonresponders 1.75% (–0.57%, 4.06%) or placebo recipients 1.44% (0.21%, 2.66%). These data underscore the importance of long-term follow-up in previous and ongoing phase 2 trials of low-dose ATG in recent-onset type 1 diabetes.Keywords
Funding Information
- Leona M. and Harry B. Helmsley Charitable Trust (09PG-T1D022)
- National Institutes of Health National Institute of Allergy and Infectious Diseases (P01-AI042288)
- National Institute of Diabetes and Digestive and Kidney Diseases (T32 DK108736, 1UL1TR000064, UL1TR000004, UL1TR001082)
- McJunkin Family Charitable Foundation
- Sanofi-Genzyme
- Amgen, Inc.
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