Alglucerase Brand Name– Ceredase
What is Alglucerase
Alglucerase is used in the treatment of patients with Gaucher’s disease. It is a parenteral, semisynthetic replacement for the endogenous enzyme glucocerebrosidase.
Alglucerase differs from the endogenous enzyme by its terminal mannose residue.
Alglucerase is produced from pooled human placental tissue from selected donors and was approved by the FDA in April 1991. A related product, imiglucerase (Cerezyme(TM)), produced by recombinant DNA technics, was approved March 1994 (see Imiglucerase monograph).
Aglucerase (Ceredase®) is only available to patients who were treated with the product prior to the introduction of imiglucerase and who cannot tolerate imiglucerase therapy.
Mechanism of Action
Alglucerase substitutes for the deficient enzyme glucosylceramidase in patients with Gaucher’s disease.
Gaucher’s disease is a congenital disorder of lipid metabolism. It results from a deficiency of glucosylceramidase, a necessary catalyst for the hydrolysis of glucosylceramide, an endogenous, very insoluble glycolipid. Patients with this condition have a build-up of this glycolipid in lysosomes of phagocytic cells, which are found in storage cells of the liver, spleen, and bone marrow as well as in the lung, kidney, and intestine.
Clinically, this build-up is associated with splenomegaly, hepatomegaly, increased skin pigmentation, and lesions of bone.
Alglucerase is designed to be preferentially taken up by macrophages, the drug’s site of action.
Treatment with alglucerase results in hydrolysis of the accumulated glycolipid within macrophages and improvement in hemoglobin, hematocrit, and platelet counts, and a decrease in hepatomegaly and splenomegaly.
Reductions in size of an enlarged liver and spleen correspond to hematological improvement and patients’ subjective improvement.
Case series studies using alglucerase enzyme replacement therapy have shown improvement in growth rate for those children and adolescents experiencing growth retardation related to Gaucher disease.
Treatment with alglucerase does not cure the underlying condition, but it does provide improvement.
Continued use is required to maintain suppression of symptoms.
Indications
- Gaucher disease
For replacement therapy of glucosylceramidase in confirmed (type 1) Gaucher disease
Side Effects
- abdominal pain
- chills
- diarrhea
- fever
- infection
- injection site reaction
- nausea
- precocious puberty
- pruritus
- vomiting
Monitoring Parameters
- laboratory monitoring not necessary
Contraindications
- breast-feeding
- children
- infants
- infection
- neonates
- precocious puberty
- pregnancy
- prostate cancer
- requires an experienced clinician
- viral infection
Interactions
There are no drug interactions associated with Alglucerase products.
