Low-dose anti-thymocyte globulin (ATG) preserves β-cell function and improves HbA1c in new-onset type 1 diabetes

Michael J. Haller, Desmond A. Schatz, Jay S. Skyler, Jeffrey P. Krischer, Brian N. Bundy, Jessica L. Miller, Mark A. Atkinson, Dorothy J. Becker, David Baidal, Linda A. DiMeglio, Stephen E. Gitelman, Robin Goland, Peter A. Gottlieb, Kevan C. Herold, Jennifer B. Marks, Antoinette Moran, Henry Rodriguez, William Russell, Darrell M. Wilson, Carla J. Greenbaum

Research output: Contribution to journalArticlepeer-review

47 Scopus citations


OBJECTIVE: A pilot study suggested that combination therapy with low-dose anti-thymocyte globulin (ATG) and pegylated granulocyte colony-stimulating factor (GCSF) preserves C-peptide in established type 1 diabetes (T1D) (duration 4 months to 2 years). We hypothesized that 1) low-dose ATG/GCSF or 2) low-dose ATG alone would slow the decline of β-cell function in patients with new-onset T1D (duration <100 days). RESEARCH DESIGN AND METHODS: A three-arm, randomized, double-masked, placebo-controlled trial was performed by the Type 1 Diabetes TrialNet Study Group in89 subjects: 29 subjects randomized to ATG (2.5 mg/kg intravenously) followed by pegylated GCSF (6 mg subcutaneously every 2 weeks for 6 doses), 29 to ATG alone (2.5 mg/kg), and 31 to placebo. The primary end point was mean area under the curve (AUC) C-peptide during a 2-h mixed-meal tolerance test 1 year after initiation of therapy. Significance was defined as one-sided P value < 0.025. RESULTS: The 1-year mean AUC C-peptide was significantly higher in subjects treated with ATG (0.646 nmol/L) versus placebo (0.406 nmol/L) (P = 0.0003) but not in those treated with ATG/GCSF (0.528 nmol/L) versus placebo (P = 0.031). HbA1c was significantly reduced at 1 year in subjects treated with ATG and ATG/GCSF, P = 0.002 and 0.011, respectively. CONCLUSIONS: Low-dose ATG slowed decline of C-peptide and reduced HbA1c in new-onset T1D. Addition of GCSF did not enhance C-peptide preservation afforded by low-dose ATG. Futurestudies should be considered to determine wheth erlow-dose ATG aloneorin combination with other agents may prevent or delay the onset of the disease.

Original languageEnglish (US)
Pages (from-to)1917-1925
Number of pages9
JournalDiabetes care
Issue number9
StatePublished - Sep 1 2018

ASJC Scopus subject areas

  • Internal Medicine
  • Endocrinology, Diabetes and Metabolism
  • Advanced and Specialized Nursing


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