The use of D-penicillamine in cystinuria: efficacy and untoward reactions.

A retrospective study was conducted to assess the efficacy of D-penicillamine in the management of cystinuria, as well as to define the frequency and nature of untoward reactions to this drug. Fifty-six individuals were identified who, by stone analysis and/or biochemical studies, met the accepted diagnostic criteria for phenotypic cystinuria. The majority of these patients presented in the second decade of life with evidence of stone formation: renal colic, hematuria, and/or stone passage. Thirty-five individuals were considered to have clinically advanced cystinuria because they had required at least one urinary tract lithotomy. In these advanced cases, frequency of subsequent lithotomies and episodes of renal colic per 100 patient-years of observation were used as indices to measure the efficacy of D-penicillamine treatment. By both measurements, D-penicillamine significantly improved the clinical course of patients. The incidence of acute drug sensitivity reactions (rash, fever, and/or arthropathy) was in excess of 40 percent. Delayed drug-induced proteinuria occurred in 34 percent of treated patients. We conclude that D-penicillamine is useful in the treatment of cystinuria. Because of the significant number of untoward drug reactions, however, we believe the drug should be instituted only in selected, high-risk patients.