Table 10 Treatment of Chronic Granulomatous Disease

Prophylaxis of Infection

Antibacterial therapy

Trimethoprim-sulfamethoxazole (TMP-SMX) 5 mg/kg/day (based upon the TMP component, maximum dose 320 mg P.O in two divided daily doses) [187]

Antifungal therapy

Itraconazole 5 mg/kg [85] (maximum dose 200 mg orally daily)

Immunomodulatory therapy

Interferon-gamma (IFN-γ) [85, 137] 50 μg/m² (subcutaneous) three times a week 1.5 μg/Kg (subcutaneous) three times a week for children <0.5 m²

Management of Infection

Empirical treatment

TMP-SMX/Fluoroquinolone/Antifungal (Voriconazole)

   • Burkholderia, Serratia species: TMP-SMX

   • Nocardia species: TMP-SMX and/or Cabapenem

   • Staphylococcus aureus:TMP-SMX or Vancomycin

   • Fungal infection: Antifungal agent ±Steroid

Liver abscess

Surgical excision [111]; IFN γ [108, 120]

Granulocyte Transfusion

Unirradiated white blood cells [183, 184]

Definitive treatment

Stem cell transplant

HLA identical sibling umbilical cord stem cell transplantation (UCSCT) after myeloablative conditioning (Stem cell transplantation from a HLA-identical donor may, at present, be the only proven curative approach to CGD) [185–187]

Gene therapy

Still experimental [188–192]