Posology : מינונים

4.2 Posology and method of administration
Therapy should be directed by physicians who are knowledgeable in the management of Gaucher disease or Niemann-Pick type C disease, as appropriate.

Posology

Dosage in type 1 Gaucher disease
Adults
The recommended starting dose for the treatment of patients with type 1 Gaucher disease is 100 mg three times a day.
Temporary dose reduction to 100 mg once or twice a day may be necessary in some patients because of diarrhoea.

Paediatric population
The efficacy of Zavesca in children and adolescents aged 0-17 years with type 1 Gaucher disease has not been established. No data are available.

Dosage in Niemann-Pick type C disease
Adult
The recommended dose for the treatment of adult patients with Niemann-Pick type C disease is 200 mg three times a day.

Paediatric population
The recommended dose for the treatment of adolescent patients (12 years of age and above) with Niemann-Pick type C disease is 200 mg three times a day.


Dosing in patients under the age of 12 years should be adjusted on the basis of body surface area as illustrated below:
Body surface area (m2)                     Recommended dose
> 1.25                                    200 mg three times a day
> 0.88 - 1.25                             200 mg twice a day
> 0.73 - 0.88                             100 mg three times a day
> 0.47 - 0.73                             100 mg twice a day
≤ 0.47                                    100 mg once a day

Temporary dose reduction may be necessary in some patients because of diarrhoea.

The benefit to the patient of treatment with Zavesca should be evaluated on a regular basis (see section 4.4).

There is limited experience with the use of Zavesca in Niemann-Pick type C disease patients under the age of 4 years.

Special populations
Elderly
There is no experience with the use of Zavesca in patients over the age of 70.

Renal impairment
Pharmacokinetic data indicate increased systemic exposure to miglustat in patients with renal impairment. In patients with an adjusted creatinine clearance of 50-70 mL/min/1.73 m2, administration should commence at a dose of 100 mg twice daily in patients with type 1 Gaucher disease and at a dose of 200 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease.

In patients with an adjusted creatinine clearance of 30-50 mL/min/1.73 m2, administration should commence at a dose of 100 mg once daily in patients with type 1 Gaucher disease and at a dose of 100 mg twice daily (adjusted for body surface area in patients below the age of 12) in patients with Niemann-Pick type C disease. Use in patients with severe renal impairment (creatinine clearance < 30 mL/min/1.73 m2) is not recommended (see sections 4.4 and 5.2).

Hepatic impairment
Zavesca has not been evaluated in patients with hepatic impairment.

Method of administration
Zavesca can be taken with or without food.