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Penicillamine Cuprimine®
Wilson disease: 0.75 and 1.5 g in 2-4 divided doses that results in an initial 24-hour cupriuresis of over 2 mg should be continued for about three months, by which time the most reliable method of monitoring maintenance treatment is the determination of free copper in the serum. This equals the difference between quantitatively determined total copper and ceruloplasmin copper.
Adequately treated patients will usually have less than 10 mcg free copper/dL of serum. It is seldom necessary to exceed a dosage of 2 g/day (144-146).
Cystinuria: 2 g/day for adults, with a range of 1 to 4 g/day. For pediatric patients, dosage can be based on 30 mg/kg/day divided in 4 doses (146).
Note: Adjust dose to limit cystine excretion to 100-200 mg/day.
Capsules and tablets 250 mg
PO
Dermatologic: Rash (5% )
Gastrointestinal: Diarrhea, epigastric pain, loss of appetite, loss of taste (12% ), nausea, ulcer of mouth (7% to 13% ), vomiting
Hematologic: myelosuppression
Renal: Proteinuria (6% )
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144. Mori E, Yamadori A, Tsutsumi A, Kyotani Y. [Adult-onset Hartnup disease presenting with neuropsychiatric symptoms but without skin lesions]. Rinsho shinkeigaku = Clinical neurology 1989;29(6):687-92
145. Brown BG, Zhao XQ, Chait A, et al. Simvastatin and niacin, antioxidant vitamins, or the combination for the prevention of coronary disease. N Engl J Med 2001;345(22):1583-92 doi: 10.1056/NEJMoa011090[published Online First: Epub Date]|.
146. Masurel-Paulet A, Poggi-Bach J, Rolland MO, et al. NTBC treatment in tyrosinaemia type I: long-term outcome in French patients. J Inherit Metab Dis 2008;31(1):81-7 doi: 10.1007/s10545-008-0793-1[published Online First: Epub Date]|.
