Talk to Your Doctor

Getting involved in your care can help you take control of your life. Getting involved means having a good partnership with your doctor. This section suggests some steps you may take to build this partnership and increase your understanding of Gaucher disease.

Be prepared

To make the most of doctor visits for you or your child, consider planning ahead. Whether it’s your first meeting or you’ve been seeing the doctor for years, you may find it helpful to prepare a list of your questions and concerns beforehand. If it is your first visit to a new doctor, here are some things you may want to include:

  • All of your symptoms, when they began, how often you or your child experience them, and how they’ve changed over time
  • The medications you or your child take, including any over-the-counter medications or dietary supplements (vitamins)
  • You or your child’s medical history
  • Any problems you or your child may have with daily activities

Get educated

The more you know about Type 1 Gaucher disease, the greater the role you can play in maintaining good health. There are a lot of places to get information about Type 1 Gaucher disease, management, and treatment. Your doctor is usually your primary source of information. It may help to write down any new information he or she gives you, or ask your doctor to provide written advice and instructions before you leave the office. The doctor also may have other materials to give you about Gaucher disease.

The internet may also be a valuable resource. Additional websites can be found here. You may want to print out any useful pages you find for later use or to show to your doctor.

Any time you take in new information from any source, it may assist you to take notes. You can then add any questions to your list for your next doctor’s visit.

Ask questions

Remember, you may want to ask your doctor questions if you need more information or clarification. Don’t be embarrassed to ask him or her to define unfamiliar words or to explain any recommendations differently. It’s also a good idea to repeat what your doctor has told you in your own words, just to make sure you have understood everything. You might even want to bring a friend or family member with you to hear what the doctor has to say.

Be honest

Being honest will help you to develop a good partnership with your doctor. This may mean telling him or her about symptoms that may be difficult for you to talk about, or admitting that you have not understood something he or she has told you. However, it may be helpful for the doctor to know these things. A change or a new symptom may mean that the treatment needs to be adjusted. Clarifying any misunderstandings may help your doctor better understand your needs.

Understand the treatment

Here are a few questions you may want to ask about your therapy and medications:

  • How and when should it be taken?
  • What are the side effects?
  • How will it interact with other medications?
  • Is it covered by my insurance?

The more you understand, the more you can be an active participant in your treatment.

Indication & Usage

Cerezyme® (imiglucerase for injection) is indicated for long-term enzyme replacement therapy for pediatric and adult patients with a confirmed diagnosis of Type 1 Gaucher disease that results in one or more of the following conditions:

  1. anemia (low red blood cell count)
  2. thrombocytopenia (low blood platelet count)
  3. bone disease
  4. hepatomegaly or splenomegaly (enlarged liver or spleen)

Important Safety Information

Approximately 15% of patients have developed immune responses (antibodies) to Cerezyme during the first year of therapy. These patients have a higher risk of an allergic reaction (hypersensitivity). Your doctor may periodically test for the presence of antibodies. Serious allergic reactions (anaphylaxis) have been reported in less than 1% of patients. Symptoms suggestive of allergic reaction happened in approximately 7% of patients, and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), and low blood pressure. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.

High blood pressure in the arteries of the lungs (pulmonary hypertension) and pneumonia have been observed in less than 1% of patients during treatment with Cerezyme. These are also known complications of Gaucher disease regardless of treatment. If you experience symptoms such as shortness of breath or chest pain, with or without fever, contact your doctor.

Approximately 14% of patients have experienced side effects related to treatment with Cerezyme. Some of these reactions occur at the site of injection such as discomfort, itching, burning, swelling or uninfected abscess. Other side effects, each of which was reported by less than 2% of patients, include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and rapid heart rate. Temporary swelling in the legs has also been observed with drugs like Cerezyme.

Please see Full Prescribing Information (PDF).

You may report side effects to the FDA at 1-800-FDA-1088.

Reference 1. Data on file. Genzyme Corporation; Cambridge, MA.