Usual Adult Dose for Gaucher Disease

Usual dose: 100 mg orally 3 times a day at regular intervals

Comments:

• Decreasing consumption of foods high in disaccharides (e.g., lactose or sucrose) and not taking this drug with food has been shown to reduce the risk and/or intensity of gastrointestinal adverse events.
• Dose reduction to 100 mg orally once or twice a day may be necessary due to adverse reactions such as tremor or diarrhea.

Renal Dose Adjustments

CrCl 50 to 70 mL/min/1.73 m2: Initial dose: 100 mg orally twice a day.
CrCl 30 to 50 mL/min/1.73 m2: Initial dose: 100 mg orally once a day.
CrCl less than 30 mL/min/1.73 m2: Not recommended.

Liver Dose Adjustments

Data not available

Dose Adjustments

Dose Reductions to 100 mg once or twice a day may be desired in patients experiencing side effects, such as diarrhea or tremor.

Loperamide has been shown to be effective in patients experiencing diarrhea.

Precautions

Safety and efficacy have not been established in patients younger than 18 years.

Consult WARNINGS section for dosing related precautions.

Dialysis

Data not available

Other Comments

Administration advice:

• Take orally at regular intervals
• A missed dose should be taken at the next scheduled time

• Therapy should be guided by physicians experienced in managing Gaucher disease patients.

• Assess platelet count at baseline and as clinically indicated.
• Neurological evaluation at baseline and every 6 months
• Vitamin B12 levels in patients with type 1 Gaucher disease

• Patients should be instructed to report: numbness, tingling, pain, or burning in hands or feet.
• Patients should be instructed to report new or worsening tremor.
• Patients should be advised to adhere to dietary instructions and report diarrhea and/or excessive weight loss.
• Advise patient to speak to physician or health care professional if pregnant, intend to become pregnant, or are breastfeeding.