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Penicillamine therapy for pediatric cystinuria: experience from a cohort of American children.

AbstractPURPOSE:
Cystinuria, an inherited defect of dibasic amino acid transport, causes accumulation of urinary cystine and cystine urolithiasis. In adults penicillamine reduces stone formation but has a high incidence of dose limiting toxicity. We evaluated the effects of penicillamine in preventing stone formation and with respect to toxicity in our cystinuria cohort at a pediatric biochemical genetics clinic.
MATERIALS AND METHODS:
We reviewed the charts of all 11 children with cystinuria treated at our clinic using a gradual dose escalation of penicillamine. Mean +/- SD patient age at diagnosis was 5.8 +/- 4.3 years (range 1.2 to 12). We tracked urinary cystine concentration before and after initiation of treatment, penicillamine side effects and incidence of new stones during maintenance therapy.
RESULTS:
During the gradual escalation of penicillamine to the target dose none of the 11 patients experienced toxicity and all had improved urinary cystine concentration (mean +/- SD percent reduction 54% +/- 25%, range 5% to 81%). We followed the patients for a total of 1,203 months (mean +/- SD 109 +/- 73 months, range 41 to 221), periodically assessing urinary cystine concentration, urine protein content, complete blood count, blood urea nitrogen, creatinine and liver function. During this time only 2 patients experienced significant side effects and no patient had stones or stone crises while compliant with treatment.
CONCLUSIONS:
In our cohort penicillamine was well tolerated after gradual initiation and it reduced urinary cystine concentration. Long-term compliance with the medication appeared to protect patients from acute stone crises.
AuthorsRalph J DeBerardinis, Curtis R Coughlin 2nd, Paige Kaplan
JournalThe Journal of urology (J Urol) Vol. 180 Issue 6 Pg. 2620-3 (Dec 2008) ISSN: 1527-3792 [Electronic] United States
PMID18951580 (Publication Type: Journal Article)
Chemical References
  • Penicillamine
Topics
  • Adolescent
  • Child
  • Child, Preschool
  • Cystinuria (complications, drug therapy)
  • Female
  • Humans
  • Infant
  • Male
  • Penicillamine (therapeutic use)
  • Retrospective Studies
  • United States
  • Urinary Calculi (etiology, prevention & control)

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