Symptom Management

In addition to treatment with Cerezyme, there are a variety of therapies and types of support available to help manage the different symptoms of Type 1 Gaucher disease. Treatment will vary depending on the course of your patient’s illness. Below are some of the commonly used techniques to manage the symptoms of Type 1 Gaucher disease.

Skeletal and joint pain

Gaucher patients may experience severe bone pain, called “bone crisis,” thought to be caused, in part, by insufficient blood circulation to the bone due to interference from Gaucher cells. To alleviate discomfort in this population, a variety of techniques and treatments have been used.  Analgesics may be used to control acute and chronic bone pain. During bone crises, common analgesics are often insufficient to control pain and opioids may need to be used for a short period of time.Extended bed rest and/or hospitalization may be required. Longstanding bone pathology usually leads to joint destruction as well, which may cause chronic pain. In addition to analgesics, orthopedic intervention may be necessary to control joint pain. These procedures include orthopedic surgical techniques to relieve pressure from damaged bony areas and/or the insertion of prosthetic devices, such as hip replacements, in joints that have been destroyed by the disease process. Gaucher patients may also need to use a cane or wheelchair or undergo physiotherapy exercises to assist with mobility.^1,2,3

Osteopenia

Osteopenia can be a debilitating complication of Gaucher disease. Certain drugs used to treat osteopenia may increase bone density.

Hematological complications

In the past, Gaucher patients with severe low red blood cell counts, severe thrombocytopenia and/or hypersplenism were frequently considered for splenectomy. Today, however, splenectomy is generally not recommended. Splenectomy increases the susceptibility to severe bacterial infections,¹ and may lead to increased liver and skeletal symptoms for patients with Gaucher disease. When splenectomy is considered today, it is usually delayed as long as possible and partial splenectomy (which may be more difficult to perform) may be recommended over total splenectomy.²

Nutritional deficiencies

Mineral or vitamin supplements may be recommended for nutritional deficiencies that affect Gaucher patients. Talk to your doctor to learn more.

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
2. thrombocytopenia
3. bone disease
4. hepatomegaly or splenomegaly

Important Safety Information

Approximately 15% of patients have developed IgG antibodies, and these patients have a higher risk of hypersensitivity reaction. Therefore periodic monitoring is suggested; caution should be exercised in patients with antibodies or prior symptoms of hypersensitivity. Symptoms suggestive of hypersensitivity occurred in 6.6% of patients, and include anaphylactoid reaction, pruritus, flushing, urticaria, angioedema, chest discomfort, dyspnea, coughing, cyanosis and hypotension.

Reactions related to Cerezyme administration have been reported in less than 15% of patients. Each of the following events occurred in less than 2% of the total patient population. Reported adverse events include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Adverse events associated with the route of administration include discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of injection.

To report suspected adverse reactions, contact Genzyme at 800-745-4447, option 2 or FDA at 800-FDA-1088 or http://www.fda.gov/Safety/MedWatch

Please see Full Prescribing Information (PDF).