Autoimmune polyendocrine syndrome medical therapy

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1]; Associate Editor(s)-in-Chief: Akshun Kalia M.B.B.S.[2]

Medical therapy for autoimmune polyendocrine syndrome (APS) depends upon the sub-type and the organ system involved. In APS, the focus is to treat the presenting disorder which can either be mucocutaneous candidiasis, hypoparathyroidism, adrenal insufficiency or autoimmune thyroiditis. The goal of treatment is to correct hormone deficiencies, prevent complications, and reduce morbidity. Treatment includes monitoring of glandular functions for early detection of glandular failure, lifelong hormone replacement therapy for established glandular failure, and familial screening.

Medical therapy for autoimmune polyendocrine syndrome (APS) depends upon the sub-type and the organ system involved.

• In APS the focus is to treat the presenting disorder which can either be mucocutaneous candidiasis, hypoparathyroidism, adrenal insufficiency or autoimmune thyroiditis.
• Patient education is an important part of treatment in APS. Patients with APS who present with mucocutaneous candidiasis may take upto 5-10 years to develop hypoparathyroidism and another 10 years to show manifestations of adrenal insufficiency. Therefore, patients with family history of APS or an early age onset of APS should be advised to undergo regular surveillance.

• Treatment of APS includes:^{[1][2][3][4][5][6][7][8][9][10]}
  • Mucocutaneous candidiasis:
    • Preferred regimen (1): Fluconazole 800 mg (12 mg/kg) on day 1, then 400 mg daily (6 mg/kg/day) for 14 days after first negative blood culture and resolution of signs/symptoms
    • Alternative regimen (1): Ketoconazole 200-400 mg/day PO may increase to 400 mg once daily if response is insufficient. Continue until active fungal infection is resolved; some infections may require a treatment duration of up to 6 months.
  • Hypoparathyroidism: Conventional therapy for hypoparathyroidism:
    • Oral calcium: Preferred regimen (1): Calcium carbonate (40% elemental calcium) (better absorption with meals)

      • Alternative regimen (1): Calcium citrate (21% elemental calcium) (more effective in patients with achlorhydria and proton pump inhibitors‘ use, worsening constipation)

    • Vitamin D supplementation

      • Preferred regimen (1): Calcitriol 0.25 to 2 μg q24h (>.75 μg administered in divided doses)
      • Preferred regimen (2): Cholecalciferol (parent vitamin D3)
      • Preferred regimen (3): Ergocalciferol (parent vitamin D2)
      • Alternative regimen (1): 1α-Hydroxyvitamin D (alfacalcidol) (used outside the United States)
      • Alternative regimen (2): Dihydrotachysterol (used outside the United States)

      Note(1): Serum calcium (corrected for albumin), phosphorus, and creatinine concentrations should be measured weekly to monthly during dose adjustments, and twice annually once a stable regimen has been reached.

      Note(2): 24-hour urinary calcium and creatinine should be considered during dose adjustments and should be measured twice annually on a stable regimen to evaluate for renal toxicity.

  • Adrenal insufficiency (Addison’s disease):
    • Glucocorticosteroid: Preferred regimen (1): Cortisone 10 to 37.5 mg q12h orally given in 2 divided doses with two-thirds of the total dose given in the morning and one third in the afternoon

      • Preferred regimen (2): Hydrocortisone : 15-30 mg/day orally given in 2 divided doses with two-thirds of the total dose given in the morning and one third in the afternoon
      • Preferred regimen (3): Dexamethasone : 0.25 to 0.75 mg orally once daily
      • Preferred regimen (4): Prednisone : 2.5 to 5 mg orally once daily

    • Mineralocorticosteroid: Preferred regimen (1): Fludrocortisone : 0.1 to 0.2 mg orally once daily
    • Patients of Addison’s disease with mild-to-moderate stress:

      • Alternative regimen (1): Cortisone 50-100 mg/day orally given in 2 divided doses with two-thirds of the total dose given in the morning (around 8 a.m.) and one third in the afternoon (noon to 4 p.m.) for 3 days
      • Alternative regimen (2): Hydrocortisone 30-90 mg/day orally given in 2 divided doses with two-thirds of the total dose given in the morning (around 8 a.m.) and one third in the afternoon (noon to 4 p.m.) for 3 days
      • Alternative regimen (3): Dexamethasone 0.50 to 2.25 mg orally once daily for 3 days
      • Alternative regimen (4): Prednisone 5-15 mg orally once daily for 3 days

    • Patients of Addison’s disease with severe stress:

      • Alternative regimen (5): Hydrocortisone sodium succinate 100 mg intravenously every 6-8 hours

  • Autoimmune thyroiditis:

    • • Preferred regimen (1) Synthetic levothyroxine (L-T4) 1.6–1.8 μg/kg of body weight per day orally.

• For complete therapy for hypopituitarism please click here.

• For complete therapy for diabetes mellitus type 1 please click here.

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