Compliance
In Fabry disease, GL-3 accumulation may begin at birth or before, and continues progressively. GL-3 accumulation in renal endothelial cells may play a role in renal failure.
The goal of enzyme replacement therapy with Fabrazyme® (agalsidase beta) is to provide the body with a regular supply of enzyme to reduce the GL-3 substrate and prevent re-accumulation. Treatment with Fabrazyme is expected to be lifelong because in most patients with Fabry disease the body produces insufficient quantities of the α-GAL enzyme.
The reduction of GL-3 inclusions suggests that Fabrazyme may ameliorate disease expression; however the relationship between GL-3 reduction and specific clinical manifestations of Fabry disease has not been established.
Points you may wish to discuss with your patients
Regular treatment: Compliance with long-term infusion therapy can be a challenge. You may refer patients to the Infusion Scheduler tool to help them keep track of their infusion schedule.
Health Insurance coverage information: Genzyme Treatment Support can provide patients with answers on health insurance questions and concerns. Go to Health Insurance and Reimbursement Information for more details.
Patients are not alone: Although Fabry disease is rare, a large patient information and support network exists. The Patient Support Networks section has links to groups all over the world. Patients can also go to the Living with Fabry Disease section to read about other patients living with Fabry disease.
“Talk to Your Doc” Worksheet: To help patients keep track of the ways in which treatment is affecting their lives and how they feel, a Talk to your Doc (PDF) worksheet is available. Patients can be encouraged to share information from this worksheet with their physicians.
To download this PDF, you must have Adobe® Acrobat® Reader, available here.
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.
|
|
 |
|
Genzyme Corporate
