1. Low-Dose ATG/GCSF in Established Type 1 Diabetes: A Five-Year Follow-up Report.
- Author
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Lin, Andrea, Mack, Jasmine A, Bruggeman, Brittany, Jacobsen, Laura M, Posgai, Amanda L, Wasserfall, Clive H, Brusko, Todd M, Atkinson, Mark A, Gitelman, Stephen E, Gottlieb, Peter A, Gurka, Matthew J, Mathews, Clayton E, Schatz, Desmond A, and Haller, Michael J
- Subjects
Diabetes ,Clinical Research ,Clinical Trials and Supportive Activities ,Metabolic and endocrine ,Antilymphocyte Serum ,Area Under Curve ,C-Peptide ,Diabetes Mellitus ,Type 1 ,Granulocyte Colony-Stimulating Factor ,Humans ,Pilot Projects ,Medical and Health Sciences ,Endocrinology & Metabolism - 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.
- Published
- 2021