(mi-gloo-stat) ,


(trade name)


Therapeutic: none assigned
Pharmacologic: enzyme inhibitors
Pregnancy Category: X


Mild to moderate type 1 Gaucher's disease, when enzyme replacement therapy is not an option.


Competitively and reversibly inhibits glucosylceramide synthase which is the initial step in the production of glycosphingolipids. The glycosphingolipid glucosylceramide accumulates in tissue in Gaucher's disease.

Therapeutic effects

Decreased production/accumulation of glycosphingolipid glucosylceramide with decreased tissue damage.


Absorption: Well absorbed following oral administration.
Distribution: Distributes into extravascular tissues.
Metabolism and Excretion: Not metabolized; excreted mostly unchanged in urine.
Half-life: 6–7 hr.

Time/action profile (blood levels)

POunknown2–2.5 hr8 hr


Contraindicated in: Hypersensitivity; Obstetric / Lactation: Pregnancy or lactation; Severe renal impairment (<30 mL/min).
Use Cautiously in: Mild to moderate renal impairment (dose alteration recommended if CCr <70 mL/min); Geriatric: Consider age related decrease in body mass, cardiac, renal and hepatic function, other chronic illnesses and concurrent drug therapies; Pediatric: Children <18 yr (safety not established).

Adverse Reactions/Side Effects

Central nervous system

  • headache (most frequent)


  • abdominal pain (most frequent)
  • diarrhea (most frequent)
  • flatulence (most frequent)
  • nausea (most frequent)
  • weight loss (most frequent)
  • anorexia
  • dyspepsia


  • ↓ male fertility


  • thrombocytopenia


  • weight loss (most frequent)


  • paresthesia
  • peripheral neuropathy
  • tremor


Drug-Drug interaction

↑ clearance of imiglucerase (should not be used concurrently).


Oral (Adults) 100 mg three time daily at regular intervals.

Renal Impairment

Oral (Adults) CCr 50–70 mL/min- 100 mg twice daily; CCr 30–50 mL/min- 100 mg once daily.


Capsules: 100 mg

Nursing implications

Nursing assessment

  • Neurological evaluations should be performed at baseline and every 6 mo during therapy. If symptoms of peripheral neuropathy (numbness, tingling) occur, discontinuation of therapy may be considered.
  • Assess for tremor. May begin during first month of therapy and may resolve between 1–3 mo of therapy; may require discontinuation of therapy.
  • Assess for diarrhea and weight loss. Advise patients with diarrhea to avoid high carbohydrate foods.
  • Lab Test Considerations: May cause thrombocytopenia.

Potential Nursing Diagnoses

Deficient knowledge, related to medication regimen (Patient/Family Teaching)


  • Oral: Administer 3 times daily at regular intervals. Capsules should be swallowed whole with water. May be administered without regard to food.

Patient/Family Teaching

  • Instruct patient to take miglustat as directed at the same time each day. If a dose is missed, skip the dose and take the next capsule at the usual time. Advise patient that sharing of this medication may be dangerous.
  • Advise patient to notify healthcare professional immediately if pregnancy is planned or suspected or if breastfeeding. Instruct male patients to maintain a reliable form of contraception during and for at least 3 mo after therapy.
  • Advise patient to consult health care professional prior to taking and Rx or OTC mediations or herbal products concurrently with miglustat.
  • Instruct patient to notify healthcare professional if numbness, pain, or burning in the hands and feet occur or if tremor develops or existing tremor worsens.

Evaluation/Desired Outcomes

  • Decreases in spleen and liver volumes in patients with Gaucher's disease.


a trademark for miglustat.
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