Gaucher disease type 1 is a rare, progressive, inherited condition that causes many different symptoms; however, it can be managed appropriately.
Gaucher (pronounced go-shay) disease is a rare, progressive, inherited, genetic condition that causes a fatty substance, called glucosylceramide (gloo-ko-sil-saramide, also called GL-1), to build up in various tissues including liver, spleen, and bone marrow.
As GL-1 builds up, people with Gaucher disease type 1 may experience excessive bruising and bleeding, as well as a protruding abdomen caused by swelling of the liver and/or spleen.
More than 90% of Gaucher disease patients are type 1.
Gaucher disease type 1 can be managed effectively once a diagnosis is made.
The build up of GL-1 affects different systems throughout your body—primarily blood and organs like the liver, spleen, and bones.
A deficiency in red blood cells (called anemia), which may cause fatigue; reduced blood clotting cells (called platelets), which makes it harder for your blood to clot and may cause
bruising or bleeding
Enlarged liver and spleen, causing a distended or enlarged abdomen
Bone problems such as delayed growth (in children), bone weakness, bone pain, bone erosion, and the possibility of the eventual collapse of the bone itself
It is the accumulation of GL-1 that causes the signs and symptoms of Gaucher disease type 1.
However, some patients may not show any symptoms at all despite their disease progressing.
Cerezyme® (imiglucerase) for injection is indicated for treatment of adults and pediatric patients 2 years of age and older with Type 1 Gaucher disease that results in one or more of the following conditions:
Hypersensitivity and Infusion-Associated Reactions: Serious allergic reactions (anaphylaxis) have been reported in patients treated with Cerezyme. Symptoms suggestive of an allergic reaction have been reported during or shortly after an infusion and include itching, flushing, hives, swelling, chest discomfort, shortness of breath, coughing, cyanosis (a bluish discoloration of the skin due to diminished oxygen), rapid heart rate, and low blood pressure. Inform your doctor and seek medical care if you experience any of these symptoms. If you have had an allergic reaction to Cerezyme, you and your doctor should use caution if you continue to receive treatment with Cerezyme.
Immune Responses: 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.
Adverse reactions reported in adults include back pain, chills, dizziness, fatigue, headache, hypersensitivity reactions, nausea, pyrexia, and vomiting.
Adverse reactions reported in pediatric patients 2 years of age and older are similar to adults.
Please see the Full Prescribing Information.
To report SUSPECTED ADVERSE REACTIONS, contact Sanofi Medical Information at 1-800-745-4447, Option 2.