Related Experiment Videos
Replacement therapy with imiglucerase for type 1 Gaucher's disease
A Zimran1, D Elstein, E Levy-Lahad
1Gaucher Clinic, Jerusalem, Israel.
Lancet (London, England)
|June 10, 1995
Summary
Low-dose imiglucerase effectively treats Gaucher's disease, reducing organ size and improving blood counts. This cost-effective approach offers a satisfactory clinical response without compromising patient quality of life.
Area of Science:
- Biochemistry
- Genetics
- Pharmacology
Background:
- Gaucher's disease is the most common sphingolipidosis, stemming from glucocerebrosidase deficiency.
- Alglucerase, a placental enzyme therapy, is established as safe and effective for Gaucher's disease.
- Imiglucerase is a recombinant enzyme used for Gaucher's disease treatment.
Purpose of the Study:
- To evaluate the efficacy and safety of low-dose imiglucerase in treating Gaucher's disease.
- To compare two distinct low-dose imiglucerase dosing schedules: 15 u/kg fortnightly versus 2.5 u/kg thrice weekly.
Main Methods:
- Ten patients with Gaucher's disease received imiglucerase therapy.
- Treatment involved two dosing schedules: 15 u/kg once every two weeks or 2.5 u/kg three times per week.
- Clinical outcomes were assessed over 12 months.
Main Results:
- Significant reductions in spleen (36.4%) and liver (14.5%) volumes were observed after 12 months.
- Mean increases in hemoglobin (13.4%) and platelet counts (25.7%) were recorded.
- No serious adverse events were reported, and no significant differences were found between the two dosing schedules.
Conclusions:
- Low-dose imiglucerase demonstrates satisfactory clinical efficacy in Gaucher's disease treatment.
- Both tested low-dose imiglucerase schedules are effective and well-tolerated.
- Low-dose, low-frequency imiglucerase presents a potentially cost-effective alternative with maintained quality of life.