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Enzyme replacement therapy for Gaucher's disease
1Department of Molecular and Experimental Medicine, Scripps Research Institute, La Jolla, CA 92037, USA.
Bailliere'S Clinical Haematology
|March 14, 1998
Summary
Modified placental human glucocerebrosidase (alglucerase) and recombinant glucocerebrosidase (imiglucerase) effectively treat Gaucher disease. Optimal dosing for Gaucher disease treatment is 15-30 U/kg/month, with frequent administration yielding the best results.
Area of Science:
- Enzyme Replacement Therapy
- Metabolic Disorders
- Genetic Diseases
Background:
- Type 1 Gaucher disease is a lysosomal storage disorder.
- Enzyme replacement therapy (ERT) with alglucerase or imiglucerase is a standard treatment.
- ERT aims to ameliorate disease manifestations.
Purpose of the Study:
- To evaluate the dose-response relationship of alglucerase and imiglucerase in treating Type 1 Gaucher disease.
- To determine the optimal dosage and administration frequency for ERT in Gaucher disease.
Main Methods:
- Analysis of a large dataset on patient responses to varying doses of alglucerase and imiglucerase.
- Assessment of treatment efficacy based on amelioration of hepatosplenomegaly, hematological, and bone manifestations.
- Comparison of treatment outcomes across different dosage ranges (15-260 U/kg/month).
Main Results:
- Treatment response rates for Gaucher disease manifestations are independent of dose from 30 to 260 U/kg/month.
- A dose of 15 U/kg/month shows comparable efficacy to higher doses in most cases.
- Severe Gaucher disease manifestations respond faster than mild cases, irrespective of dose (except at 15 U/kg/month).
Conclusions:
- The recommended dosage for alglucerase or imiglucerase in Gaucher disease is 15-30 U/kg/month.
- Frequent administration (three times weekly) appears to be the most effective method for ERT.
- Dose optimization can improve treatment outcomes for Gaucher disease.