Treatments and drugs

To treat Gaucher's disease, your doctor may recommend:

  • Enzyme replacement therapy. This approach replaces the deficient enzyme with synthetic enzymes. These replacement enzymes are administered in an outpatient procedure through a vein (intravenously), typically at two-week intervals at high doses.

    Although effectiveness can vary, it's frequently effective in people with type 1 Gaucher's disease and, in some cases, type 3. In many people, enzyme replacement therapy can reduce the enlargement of the liver and spleen, and help to resolve blood abnormalities. Children treated with enzyme therapy often experience a growth spurt and weight gain as skeletal abnormalities are moderated.

    This treatment is most effective when started prior to development of significant organ enlargement or bone impairment. It's unclear whether this therapy is effective for the neurological problems of Gaucher's disease. Occasionally, people experience an allergic or hypersensitivity reaction to enzyme treatment, triggering a rash, nausea, diarrhea or abdominal pain.

  • Bone marrow transplantation. This surgical procedure has been used for severe cases of Gaucher's disease. In this technique, blood-forming cells that have been damaged by Gaucher's are removed and replaced, which can reverse many of Gaucher's signs and symptoms. Because this approach is high-risk, it's performed much less often than is enzyme replacement therapy.
  • The medication miglustat (Zavesca). The effectiveness of this oral medication in people with Gaucher's disease is still being studied, but it may be capable of interfering with the production of glucocerebrosides in some people with type 1 disease. Diarrhea and weight loss are common side effects.

Prognosis
People with mild cases of Gaucher's disease, particularly those who develop it in adulthood, have normal life expectancies. Children whose illness begins during infancy generally don't live beyond the age of 2 years old.

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