Effect of Ingested Interferon-α on β-Cell Function in Children With New-Onset Type 1 Diabetes

  1. Staley A. Brod, MD2
  1. 1National Institute of Diabetes and Digestive and Kidney Diseases, Bethesda, Maryland;
  2. 2University of Texas Medical School, Department of Neurology, Houston, Texas;
  3. 3University of Texas Southwestern, Dallas, Texas;
  4. 4Children's Mercy Hospital & Clinics, Kansas City, Missouri;
  5. 5St. Paul Children's Hospital, St. Paul, Minnesota;
  6. 6University of Texas Medical School, Department of Internal Medicine, Houston, Texas.
  1. Corresponding author: Kristina I. Rother, kr58q{at}nih.gov.

Abstract

OBJECTIVE To evaluate the safety and efficacy of ingested human recombinant interferon-α (hrIFN-α) for preservation of β-cell function in young patients with recent-onset type 1 diabetes.

RESEARCH DESIGN AND METHODS Subjects aged 3–25 years in whom type 1 diabetes was diagnosed within 6 weeks of enrollment were randomly assigned to receive ingested hrIFN-α at 5,000 or 30,000 units or placebo once daily for 1 year. The primary outcome was change in C-peptide secretion after a mixed meal.

RESULTS Individuals in the placebo group (n = 30) lost 56 ± 29% of their C-peptide secretion from 0 to 12 months, expressed as area under the curve (AUC) in response to a mixed meal. In contrast, children treated with hrIFN-α lost 29 ± 54 and 48 ± 35% (for 5,000 [n = 27] and 30,000 units [n = 31], respectively, P = 0.028, ANOVA adjusted for age, baseline C-peptide AUC, and study site). Bonferroni post hoc analyses for placebo versus 5,000 units and placebo versus 30,000 units demonstrated that the overall trend was determined by the 5,000-unit treatment group. Adverse events occurred at similar rates in all treatment groups.

CONCLUSIONS Ingested hrIFN-α was safe at the doses used. Patients in the 5,000-unit hrIFN-α treatment group maintained more β-cell function 1 year after study enrollment than individuals in the placebo group, whereas this effect was not observed in patients who received 30,000 units hrIFN-α. Further studies of low-dose ingested hrIFN-α in new-onset type 1 diabetes are needed to confirm this effect.

Footnotes

  • A full list of investigators can be found in an online appendix.

  • Clinical trial reg. no. NCT00024518, clinicaltrials.gov.

  • The costs of publication of this article were defrayed in part by the payment of page charges. This article must therefore be hereby marked “advertisement” in accordance with 18 U.S.C. Section 1734 solely to indicate this fact.

  • Received November 11, 2008.
  • Accepted March 25, 2009.

Readers may use this article as long as the work is properly cited, the use is educational and not for profit, and the work is not altered. See http://creativecommons.org/licenses/by-nc-nd/3.0/ for details.

| Table of Contents