Gaucher Registry

The Gaucher Registry (sponsored by Sanofi Genzyme) is the world’s largest cooperative observational study on Gaucher disease. The International Collaborative Gaucher Group (ICGG) established the registry in 1991 as a longitudinal database tracking outcomes of routine clinical practice. 

All patients with a confirmed diagnosis of Gaucher disease are eligible for inclusion. By enrolling patients in the registry, participating physicians will receive patient-specific reports to monitor disease status.

The Gaucher Registry maintains its integrity by a commitment to patient privacy and confidentiality, as well as a commitment to the quality of the data. To ensure the most comprehensive data, the Gaucher Registry is open to all patients with Gaucher disease, regardless of treatment modality.

To learn more visit the Gaucher Registry.

The clinical data in this database are voluntarily submitted by physicians worldwide and have been collected and assessed according to local practice guidelines. The specific data collected on each patient may vary depending on differences in management approaches by participating physicians and/or availability of data. Patients with Gaucher disease type 1 may have received Ceredase® (alglucerase injection) and/or Cerezyme at some point in their care, treated at various doses and schedules and for variable durations; the majority of patients have been receiving Cerezyme since 1997.

References.

1. Weinreb NJ, Charrow J, Andersson HC, et al. Effectiveness of enzyme replacement therapy in 1028 patients with type 1 Gaucher disease after 2 to 5 years of treatment: a report from the Gaucher Registry. Am J Med. 2002;113(2):112-119.

2. Grabowski GA, Barton NW, Pastores G, et al. Enzyme therapy in Type 1 Gaucher disease: comparative efficacy of mannoseterminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med. 1995;122(1):33-39.

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 to Cerezyme during the first year of therapy. Approximately 46% of patients with detectable IgG antibodies experienced symptoms of hypersensitivity, and these patients have a higher risk of hypersensitivity. It is suggested that patients be monitored periodically for IgG antibody formation during the first year of treatment.

Hypersensitivity has also been observed in patients without detectable IgG antibodies. Symptoms suggestive of hypersensitivity have been noted in approximately 6.6% of all patients, and anaphylactoid reactions in less than 1%. Treatment with Cerezyme should be approached with caution in patients who have exhibited hypersensitivity symptoms such as pruritus, flushing, urticarial, angioedema, chest discomfort, dyspnea, coughing, cyanosis, and hypotension. Pre-treatment with antihistamines and/or corticosteroids and a reduced rate of infusion may allow continued treatment in most patients.

In less than 1% of patients, pulmonary hypertension and pneumonia have been observed during treatment with Cerezyme. These are known complications of Gaucher disease regardless of treatment. Patients with respiratory symptoms in the absence of fever should be evaluated for the presence of pulmonary hypertension.

Approximately 13.8% of patients have experienced adverse events related to treatment with Cerezyme. Some of these are injection site reactions such as discomfort, pruritus, burning, swelling or sterile abscess at the site at the site of venipuncture. Additional adverse reactions that have been reported include nausea, abdominal pain, vomiting, diarrhea, rash, fatigue, headache, fever, dizziness, chills, backache, and tachycardia. Transient peripheral edema has also been reported for this therapeutic class of drug.

To report suspected adverse reactions, contact Sanofi Genzyme at 800-745-4447, option 2

Please see Full Prescribing Information (PDF).