MIGLUSTAT (Systemic)?
Some commonly used brand names are:
In the U.S.?
? Not commercially available in Canada.
Category
- Substrate reductiontherapy
Description
Miglustat ((MIH-glue-stat)) is used to treatadults with mild to moderate type 1 Gaucher disease. Miglustatis only used in people who cannot be treated with enzyme replacement therapy.Type 1 Gaucher disease is a disease you get from both your parents. Peoplewith type 1 Gaucher disease are missing an enzyme (naturally occurring substancein your body) that breaks down a chemical in your body called glucosylceramide.Too much glucosylceramide causes liver and spleen enlargement, changes inthe blood, and bone disease. Miglustat works by stopping the body from makingglucosylceramide.
This medicine is available only with your doctor's prescription, in thefollowing dosage forms:
Before Using This Medicine
In deciding to use a medicine,the risks of taking the medicine must be weighed against the good it willdo. This is a decision you and your doctor will make. For miglustat, the followingshould be considered:
Allergies?Tell your doctor if you have ever had any unusualor allergic reaction to miglustat. Also tell your doctor and pharmacist ifyou are allergic to any other substances, such as foods, preservatives, ordyes.
Pregnancy?Miglustat is not recommended during pregnancy.It has been shown to cause serious birth defects or other problems in animals.Before taking this medicine be sure your doctor knows if you are pregnantor are planning to become pregnant. Be sure you have discussed this with yourdoctor. Males who are taking this medicine should use effective birth controland should continue using birth control for at least three months after stoppingthe medicine.
Breast-feeding?It is not known whether miglustat passes intobreast milk. It is not recommended for use during breast feeding because itmay cause unwanted effects in nursing babies. You should decide to breastfeed or take miglustat, but not both. Be sure you have discussed this withyour doctor.
Children?This medicine is not used in children under 18years of age.
Older adults?Many medicines have not been specifically studiedin older people. Therefore, it may not be known whether they work the sameway the do in younger adults. This medicine is not expected to cause differentside effects or problems in older people than it does in younger adults.
Other medical problems?The presence of other medical problems mayaffect the use of miglustat. Make sure you tell your doctor if you have anyother medical problems, especially:
- Gaucher disease, Type 1, severe?this medicine is not currentlybeing used in patients with severe Type 1 Gaucher disease
- Kidney disease?this condition may cause you to have more miglustatin your body; your doctor may want to change the amount of miglustat thatyou take
Proper Use of This Medicine
It is importantto take miglustat exactly as your doctor prescribed. You should take yourmedicine at the same time or at the same times each day.
The capsules should be swallowed whole with water and may be taken withor without food. Check with your doctor or pharmacist if you have any questions.
Your doctor may recommend changes to your diet to help with some side effects.It is important that you follow these changes.
Dosing?The dose of miglustat will be different fordifferent patients. Follow your doctor's orders or thedirections on the label. The following information includes only theaverage doses of miglustat. If your dose is different,do not change it unless your doctor tells you to do so.
- For oral dosage form (capsules):
- For Mild to Moderate Type 1 Gaucher disease
- Adults?One 100 milligram (mg) capsule given three times a day;your doctor may change this dose as needed
- Children?Use is not recommended in children under the age of18
Missed dose?If you miss a dose of this medicine, take itas soon as possible. However, if it is almost time for your next dose, skipthe missed dose and go back to your regular dosing schedule. Do not doubledoses.
Storage?To store this medicine:
- Keep out of the reach of children.
- Keep the medicine from freezing. Do not refrigerate.
- Do not keep outdated medicine or medicine no longer needed. Ask yourhealth care professional how you should dispose of any medicine you do notuse. Be sure that any discarded medicine is out of the reach of children.
Precautions While Using This Medicine
Miglustatcan cause problems affecting your nerves. If you have hand tremors (shakymovements) or if miglustat worsens a hand tremor you already have call yourdoctor. Your doctor might want to change your dose of miglustat.
If you experience numbness and tingling in your hands,arms, legs, or feet (peripheral neuropathy) call your doctor rightaway.
It is very important that your doctor check you atregular visits. Your doctor will also want to test your nerves (neurologicalexam) before you start taking miglustat and may repeat this test at a latertime.
Diarrhea is the most common side effect for people taking miglustat. Yourdoctor may give you another medicine (anti-diarrheal) to help treat diarrheaif it is a problem for you. Your doctor may also recommend changes to yourdiet. You may also lose weight when you start treatment with miglustat.
It is very important to discuss with your doctor if you are pregnant orplan to become pregnant before starting miglustat. You should use effectivebirth control while taking miglustat. Miglustat may also harm a man's sperm.All men should use effective birth control during treatment and for threemonths after stopping treatment.
Side Effects of This Medicine
Along with its needed effects, a medicinemay cause some unwanted effects. Although not all of these side effects mayoccur, if they do occur they may need medical attention.
Other side effects mayoccur that usually do not need medical attention. These side effects may goaway during treatment as your body adjusts to the medicine. However, checkwith your doctor if any of the following side effects continue or are bothersome.
More Common
Acid or sour stomach; back pain; belching; bloated, full feeling; change in vision; cramps; diarrhea; difficulty having a bowelmovement (stool); dizziness; dry mouth; excess air or gas in stomach or intestines; full or bloated feeling or pressure in the stomach; headache; heartburn; heaviness in limbs; indigestion; leg cramps; loss of appetite; memory loss; menstrual changes; nausea; pain or discomfort in chest,upper stomach, or throat; passing gas; stomach discomfort, upset or pain; swelling; swelling of abdominal or stomach area; unsteady walk; vomiting; weakness; weight loss
Other side effectsnot listed above may also occur in some patients. If you notice any othereffects, check with your doctor.
Developed: 03/01/2004
