Table 2.

Prophylactic therapies for CGD

CategoryMedicationSuggested dosingPotential adverse eventsNotes
Antibiotics Trimethoprim/sulfamethoxazole 5 mg/kg/day TMP, Max 160 mg TMP daily Hematologic: agranulocytosis, hemolysis, and thrombocytopenia
Renal: interstitial nephritis and hyperkalemia
GI: abdominal pain, diarrhea, and pancreatitis
Dermatologic: photosensitivity and Stevens-Johnson syndrome 
Monitor CBC, Cr, and K at baseline and 1 mo
Assess G6PD status given potential risk 
Second line: ​ ​ ​ 
Levofloxacin 10 mg/kg/dose BID, max 750 mg/dose MSK: Risk of tendinopathy
CV: QTc prolongation
Neuro: Peripheral neuropathy and lowering of seizure threshold 
Monitor CBC, hepatic panel, and Cr at baseline and 1 mo 
Antifungals Itraconazole 5 mg/kg/day GI: transaminitis; abdominal pain and diarrhea (in oral suspension)
CV: hepatotoxicity (black box warning for ventricular dysfunction)
Neuro: peripheral neuropathy
Drug–drug interactions due to CYP3A4 inhibitors 
Goal drug trough level: ≥1 mcg/ml (incl. metabolites); monitor trough after 7–14 days (if no loading dose given) of dose changes or initiation of any interacting medications, and at 1–3 mo intervals
Monitor hepatic panel at baseline and q6 mo
Oral formulation and pills are not interchangeable 
Posaconazole DR tablet: GI: Transaminitis, potent CYP3A4 inhibitor
CV: QTc prolongation 
Tablets and suspension formulations are not interchangeable (see Data S1).
Goal drug trough level: ≥0.7 mcg/ml 
​ 10 to <20 kg: 100 mg BID × 2 doses, then 100 mg daily 
​ 20 to <40 kg: 200 mg BID × 2 doses, then 200 mg daily 
​ ≥40 kg: 300 mg BID × 2 doses, then 300 mg daily 
Voriconazole 9 mg/kg BID, max 200 mg/dose GI: transaminitis
Neuro: visual disturbance, optic neuritis, vision color changes, hallucination, and peripheral neuropathy
CV: QTc prolongation
Derm: photosensitivity/dermatologic cancer 
Risk of photosensitivity and skin cancers
Goal drug trough level: 0.5–5 mcg/ml 
Immunomodulators IFN-γ-1b 50 mcg/m2 BSA, or 1.5 mcg/kg/dose (<0.5 m2) three times weekly Fevers, cytopenias, and transaminitis May induce low-grade fevers with initial medication start, which may be treated or prevented with NSAIDs 
CategoryMedicationSuggested dosingPotential adverse eventsNotes
Antibiotics Trimethoprim/sulfamethoxazole 5 mg/kg/day TMP, Max 160 mg TMP daily Hematologic: agranulocytosis, hemolysis, and thrombocytopenia
Renal: interstitial nephritis and hyperkalemia
GI: abdominal pain, diarrhea, and pancreatitis
Dermatologic: photosensitivity and Stevens-Johnson syndrome 
Monitor CBC, Cr, and K at baseline and 1 mo
Assess G6PD status given potential risk 
Second line: ​ ​ ​ 
Levofloxacin 10 mg/kg/dose BID, max 750 mg/dose MSK: Risk of tendinopathy
CV: QTc prolongation
Neuro: Peripheral neuropathy and lowering of seizure threshold 
Monitor CBC, hepatic panel, and Cr at baseline and 1 mo 
Antifungals Itraconazole 5 mg/kg/day GI: transaminitis; abdominal pain and diarrhea (in oral suspension)
CV: hepatotoxicity (black box warning for ventricular dysfunction)
Neuro: peripheral neuropathy
Drug–drug interactions due to CYP3A4 inhibitors 
Goal drug trough level: ≥1 mcg/ml (incl. metabolites); monitor trough after 7–14 days (if no loading dose given) of dose changes or initiation of any interacting medications, and at 1–3 mo intervals
Monitor hepatic panel at baseline and q6 mo
Oral formulation and pills are not interchangeable 
Posaconazole DR tablet: GI: Transaminitis, potent CYP3A4 inhibitor
CV: QTc prolongation 
Tablets and suspension formulations are not interchangeable (see Data S1).
Goal drug trough level: ≥0.7 mcg/ml 
​ 10 to <20 kg: 100 mg BID × 2 doses, then 100 mg daily 
​ 20 to <40 kg: 200 mg BID × 2 doses, then 200 mg daily 
​ ≥40 kg: 300 mg BID × 2 doses, then 300 mg daily 
Voriconazole 9 mg/kg BID, max 200 mg/dose GI: transaminitis
Neuro: visual disturbance, optic neuritis, vision color changes, hallucination, and peripheral neuropathy
CV: QTc prolongation
Derm: photosensitivity/dermatologic cancer 
Risk of photosensitivity and skin cancers
Goal drug trough level: 0.5–5 mcg/ml 
Immunomodulators IFN-γ-1b 50 mcg/m2 BSA, or 1.5 mcg/kg/dose (<0.5 m2) three times weekly Fevers, cytopenias, and transaminitis May induce low-grade fevers with initial medication start, which may be treated or prevented with NSAIDs 

G6PD, glucose-6-phosphate dehydrogenase. Cr: creatinine, K: potassium; Gl: glucose; BID: twice daily; q6: every 6 hours; CV: cardiovascular; QTc: corrected QT interval; DR: delayed release

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