Symptom Management
Both symptom management and enzyme replacement therapy can play a role in the treatment of people with Fabry disease. Since Fabry disease affects many organ systems it is possible that you may see several types of doctors.
For some people, managing the symptoms of Fabry disease can mean making changes in your lifestyle. This may include avoiding or preparing for certain activities that may make symptoms worsen. Some people may require medications on a daily basis for pain or gastrointestinal symptoms. The table below lists how physicians may choose to manage the symptoms of Fabry disease.
Emotional Support is Important
Emotional support and family counseling can be an important part of your care. Depression and/or denial are common, as is guilt about passing on the defective gene to children. Family and individual counseling can be important for people living with Fabry disease and their families.
Contact with other people and families struggling with similar issues may help with feelings of isolation, loneliness, and despair. Several organizations and medical centers offer websites, newsletters, support groups, and information on Fabry disease. Visit the Support and Resources section for more.
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.
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