Identification of autoimmune polyendocrine syndrome type 1 in patients with isolated hypoparathyroidism

Clin Endocrinol (Oxf). 2016 Oct;85(4):544-50. doi: 10.1111/cen.13111. Epub 2016 Jun 28.

Abstract

Objective: The prevalence of autoimmune polyendocrine syndrome type 1 (APS1) among isolated hypoparathyroidism (HP) or primary adrenal insufficiency (PAI) is not well established. We studied the frequency of APS1 in patients with HP or PAI by measuring interferon-α (IFN-α) antibody levels, a highly sensitive and specific marker for APS1.

Design, patients and measurements: In a single-centre cross-sectional study, 37 Indian patients with isolated HP and 40 patients with PAI were tested for IFN-α antibody using an indirect ELISA. In patients with elevated IFN-α antibody, the autoimmune regulator (AIRE) gene was bidirectionally sequenced.

Results: Three (8·1%) patients with isolated HP had elevated IFN-α antibody levels (range: 367-17382 units; positive titre >56 units). Homozygous or compound heterozygous AIRE mutations were detected in all three patients, including a novel mutation (p.T68P). All three APS1 patients had atypical features. The first patient, diagnosed at 7 years of age, died suddenly 5 months later. The second patient had late-onset HP (at the age of 34 years) and a solitary episode of transient mucocutaneous candidiasis 5 years later. The final patient developed HP at the age of 14 years and premature ovarian insufficiency 14 years later. Interleukin-22 antibodies, as well as most other organ-specific antibodies, were absent in the 3 APS1 patients. All patients with PAI were negative for IFN-α antibody.

Conclusion: Eight percentage of patients with isolated HP had elevated IFN-α antibody levels and AIRE mutation-positive APS1. All APS1 patients had atypical clinical features. Testing for IFN-α antibody should be considered in patients with idiopathic HP.

MeSH terms

  • AIRE Protein
  • Addison Disease / complications*
  • Adolescent
  • Adult
  • Antibodies / analysis
  • Child
  • Cross-Sectional Studies
  • Female
  • Humans
  • Hypoparathyroidism / complications
  • Hypoparathyroidism / etiology
  • Hypoparathyroidism / immunology*
  • Interferon-alpha / immunology
  • Male
  • Middle Aged
  • Mutation
  • Polyendocrinopathies, Autoimmune / diagnosis*
  • Transcription Factors / genetics
  • Young Adult

Substances

  • Antibodies
  • Interferon-alpha
  • Transcription Factors

Associated data

  • GENBANK/NM_000383·3