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Cysteamine bitartrate (Cystagon®)

Cystinosis
Begin with 10mg/kg/day and increase weekly until reaching the maintenance dose (60 to 90 mg of free base/kg/day) or (1.3 to 1.95 g/m2 per day).

The recommended adult dose is 500 mg free base q6 hours; however, for both children and adults, the dose is titrated to reduce, if possible, leukocyte cystine concentration (measured 5-6 hours after a dose) to below 1 nmol half-cystine/mg protein (50-52).

50 mg and 150 mg capsules

PO

Dermatologic: Rash (7% )

Gastrointestinal: Diarrhea (16% ), Loss of appetite (31% ), Vomiting (35% )

Neurologic: Lethargy (11% ), Somnolence

Other: Fever (22% )

4

50. Sprecher DL, Daniels SR. Rational approach to pharmacologic reduction of cholesterol levels in children. J Pediatr 1996;129(1):4-7

51. Tonstad S, Knudtzon J, Sivertsen M, Refsum H, Ose L. Efficacy and safety of cholestyramine therapy in peripubertal and prepubertal children with familial hypercholesterolemia. J Pediatr 1996;129(1):42-9

52. Gonzales E, Gerhardt MF, Fabre M, et al. Oral cholic acid for hereditary defects of primary bile acid synthesis: a safe and effective long-term therapy. Gastroenterology 2009;137(4):1310-20 e1-3 doi: 10.1053/j.gastro.2009.07.043[published Online First: Epub Date]|.

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