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Table 1 Medical therapy for Cushing’s disease

From: Update on medical treatment for Cushing’s disease

Classification Drug name Dose Possible adverse events; Close monitoring is necessary for all drugs
Steroidogenesis inhibitors Ketoconazole Oral 200–1200 mg/day
2–3 times a day
Hepatitis, gastrointestinal disturbances, gynecomastia, skin rash, adrenal insufficiency.
Fluconazole Oral 100–200 mg/day
2 times a day
AEs similar to ketoconazole, not well studied
Metyrapone Oral 0.5–6 g/day
4 times a day
Hirsutism, acne, hypertension, hypokalemia, edema, gastritis, nausea, adrenal insufficiency.
Etomidate IV bolus of 0.03 mg/kg followed by 0.02–0.08 mg/kg/h Somnolence, myoclonus, nausea, vomiting, dystonic reactions, adrenal insufficiency.
Mitotane Oral 2–5 g/day
3 times a day
Gastrointestinal disturbances, hepatitis, neurologic manifestations, gynecomastia, neutropenia, lipid disorders, adrenal insufficiency; teratogenic
Osilodrostat Oral 10–60 mg/day; phase III clinical trial: NCT 02180217 Nausea, asthenia, diarrhea, adrenal insufficiency. Testosterone increase in women.
Levoketoconazole Oral 400 mg /day; phase III clinical trial: NCT 01838551 N/A.
Dopamine D2R agonists Cabergoline Oral 0.5–7 mg/week Headache, nausea, dizziness, nasal congestion, hypotension, depression
SRLs Pasireotidea SC 300–1800 µg/day
2 times a day
Hyperglycemia, diabetes, diarrhea, nausea, abdominal pain, cholelithiasis, QT prolongation
Pasireotide LAR IM 30–60 mg/month; phase III clinical trial
GR antagonist Mifepristonea Oral 300–1200 mg/day
Once daily
Nausea, fatigue, headache, hypokalemia, arthralgia, vomiting, peripheral edema, hypertension, dizziness, adrenal insufficiency, PAECs, endometrial thickening, vaginal bleeding; termination of pregnancy.
CDK2/Cyclin E inhibitor R-Roscovitine Oral Phase II clinical trial; clinicaltrials.gov identifier: NCT02160730  
Nuclear receptor ligand Retinoic acid Oral Phase II clinical trial; clinicaltrialsregister.eu number: 2008-006379-65  
  1. D2R dopamine D2 receptor, SRL somatostatin receptor ligand, SC subcutaneous, IM intramuscular, LAR long-acting repeatable, PAECs- progesterone-receptor modulator-associated endometrial changes
  2. aFDA approved