Penicillamine Therapy For Pediatric Cystinuria: Experience From A Cohort Of American Children

January 26, 2009 at 11:00 am Leave a comment – A study from The Children’s Hospital of Philadelphia, Metabolic Disease Section evaluated the use of penicillamine for therapy for pediatric cystinuria. The group reviewed the charts of 11 children with cystinuria treated at their clinic using a gradual dose escalation of penicillamine. They found that all of these children tolerated the penicillamine well with no toxicity. They all had improved urinary cystine concentrations in their urine. Only 2 of the patients experienced any significant side effects from the therapy. None of the patients had stones when they were compliant with their treatment.

The group found that the medicine is tolerated well after gradual initiation. They also stated that providing patients stay on their therapy, the stones would remain in remission.

I agree that penicillamine is the best therapy, even in the pediatric population for cystinuria. However, it has been my experience that having these children take such a large amount of medication a few times per day is quite daunting. In my practice, I can tell which children are on their penicillamine and when they are off it, even if they are telling me they are taking it everyday. Some will present with a multitude of stones and then once we clear them, they will be stone free while they are on their therapy, then they come back admitting that they stopped their therapy.

The true interesting point of cystinuria is that, in the past, cystinuria was the main cause of urinary lithiasis in the pediatric population. Now, it has been largely overtaken by calcium oxalate stone formers that are more citrate, hydration, and diet related.

Deberardinis RJ, Coughlin CR 2nd, Kaplan P

J Urol. 2008 Oct 23. Epub ahead of print.


Written by Medical Editor Pasquale Casale, MD

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