Monitoring Patients

Because of the multisystemic aspects of Fabry disease, coordination of care among the treating physician and specialists is essential for comprehensive treatment and monitoring of patients on Fabrazyme^® (agalsidase beta).

To connect with physicians with experience in treating Fabry disease, please contact Genzyme at 617-768-9000 or toll-free 800-745-4447, option 2.

Comprehensive Annual Evaluations ^[1]

In a 2003 publication, a consensus panel of physicians with experience in treating Fabry disease recommended the following:

• Annual assessments, including physical examination and routine hematology, chemistry, and urinalysis
• Annual urinary protein (including protein to creatinine ratio) and creatinine clearance assessments to determine renal function
• Echocardiogram and EKG at least every 2 years to detect and monitor cardiac abnormalities
• For female carriers, a complete baseline examination as described above. Symptomatic carriers may be followed annually.
• Baseline MRIs of kidney, heart, and brain to document disease progression and assess the effect of treatment.
• Abnormal findings may be monitored more frequently as indicated.

^{Fabry Registry}

While physicians determine the actual frequency of necessary assessments according to a patient's individualized need for medical care and routine follow-up, Genzyme’s Fabry Registry provides a recommended Schedule of Assessments, which may be useful as a guideline. These guidelines have been developed based on the input of physicians from the international medical community with experience in the care of patients with Fabry disease.

In order to better understand the variability and progression of Fabry disease in the population as a whole and in women, and to monitor and evaluate long-term treatment effects of Fabrazyme, the Fabry Registry has been established. The Registry will also monitor the effect of Fabrazyme on pregnant women and their offspring, and determine if Fabrazyme is excreted in breast milk. Patients should be informed of the Fabry Registry and encouraged to participate. They should be advised that their participation is voluntary and may involve long-term follow-up.

Physicians can monitor their patients’ disease status by utilizing Fabry Registry’s patient-specific reports, by exchanging clinical data among physicians to facilitate clinical decision-making, and accessing information on current treatment guidelines and practice patterns. Patient and physician identifiable information submitted to the Fabry Registry is maintained as confidential.

For more information visit Genzyme’s www.fabryregistry.com website or call 617-591-5500 or toll-free 800-745-4447, ext. 15500.

Important Safety Information Fabrazyme (agalsidase beta) is indicated for use in patients with Fabry disease.  Fabrazyme reduces globotriaosylceramide (GL-3) deposition in capillary endothelium of the kidney and certain other cell types. The reduction of GL-3 inclusions suggests that Fabrazyme may ameliorate disease expression; however, the relationship of GL-3 inclusion reduction to specific clinical manifestations of Fabry disease has not been established. The most serious and most common adverse reactions reported with Fabrazyme are infusion reactions.  Serious and/or frequently occurring related adverse reactions consisted of one or more of the following events: chills, pyrexia, feeling hot or cold, dyspnea, nausea, flushing, headache, vomiting, paresthesia, fatigue, pruritus, pain in extremity, hypertension, chest pain, throat tightness, abdominal pain, dizziness, tachycardia, nasal congestion, diarrhea, edema peripheral, myalgia, back pain, pallor, bradycardia, urticaria, hypotension, face edema, rash, and somnolence. The occurrence of somnolence can be attributed to clinical trial specified pre-treatment with antihistamines.  Other reported serious adverse events included stroke, pain, ataxia, bradycardia, cardiac arrhythmia, cardiac arrest, decreased cardiac output, vertigo, hypoacousia, and nephrotic syndrome.  These adverse events also occur as manifestations of Fabry disease; an alteration in frequency or severity cannot be determined from the small numbers of patients studied. Infusion reactions occurred in many patients treated with Fabrazyme and some of the reactions were severe.  Patients should be given antipyretics prior to infusion.  Infusion reactions occurred in some patients after receiving pretreatment with antipyretics, antihistamines, and oral steroids.  Infusion reactions declined in frequency with continued use of Fabrazyme.  However, infusion reactions may still occur despite extended duration of Fabrazyme treatment.  Because of the potential for severe infusion reactions, appropriate medical support measures should be readily available when Fabrazyme is administered.  Patients with compromised cardiac function should be monitored closely if the decision is made to administer Fabrazyme. Most patients develop IgG antibodies to Fabrazyme.  A few patients developed IgE or skin test reactivity specific to Fabrazyme.  Physicians should consider testing for IgE in patients who experienced suspected allergic reactions and consider the risks and benefits of continued treatment in patients with anti- Fabrazyme IgE.   Patients with Fabrazyme- specific IgE antibody have been treated using a rechallenge protocol.  Rechallenge of these patients should only occur under the direct supervision of qualified personnel, with appropriate medical support measures readily available. The safety and efficacy in patients younger than 8 years of age have not been evaluated.  IgE immunologic responses in pediatric patients may differ from those in adults, as IgG seroconversion was associated with prolonged half-life concentrations of Fabrazyme, which is rarely observed in adult patients. Fabrazyme is available by prescription only. Side effects should be reported promptly to Genzyme Medical Information at 800-745-4447, option 2. To learn more, please see the full prescribing information (PDF) or contact Genzyme at 1-800-745-4447.

^References

1. Desnick RJ, Brady R, Barranger J, Collins AJ, Germain DP, Goldman M, Grabowski G, Packman S, Wilcox WR. Fabry disease, an under-recognized multisystemic disorder: expert recommendations for diagnosis, management, and enzyme replacement therapy. Ann Intern Med 2003;138:338-346.