Enzyme replacement therapy in pediatric patients with Gaucher disease: What should we use as maintenance dosage?

IntroductionNo consensus exists on the minimal dose of enzyme replacement therapy (ERT) effective to maintain therapeutic goals in pediatric Gaucher patients.ObjectiveEvaluate the efficacy of low dosage ERT to maintain treatment goals.ResultsSix patients had a maintenance dose of 30–35 U/kg/month. All patients, with the exception of one L444P/L444P homozygote, maintained therapeutic goals.DiscussionA low maintenance dose may be adequate in most pediatric patients. L444P homozygotes may require a higher maintenance dosage.