Just as Pompe disease affects people differently, responses to Lumizyme® (alglucosidase alfa) treatment will also vary from person to person. Talk to your healthcare provider about what you might expect from treatment with Lumizyme. Some people find it useful to keep notes of any changes they experience over the course of their treatment, so they can keep track of important information to share with their healthcare team.
Regular visits with your healthcare provider are important to check how you’re responding to Lumizyme and to assess your overall health. You can assist with the monitoring process by keeping a journal to record changes in your health and condition, any new symptoms that have developed between visits. Record improvements as well as any declines in function that you observe. Taking photos and videos over time can be a good way to document changes and share them with your medical team. Open communication is important so that your healthcare providers are aware of all changes and can provide you with appropriate care.
In addition to regular check-ups with your healthcare provider, it is recommended that most people with Pompe disease have comprehensive medical evaluations that allow a variety of specialists to monitor how Pompe disease and treatment are affecting different parts of the body.
Antibodies are proteins produced naturally by the body to “attack” foreign substances. In some patients, the body may react to Lumizyme as a foreign substance and form antibodies. There are two types of antibodies that may develop:
It is important to follow up with your healthcare provider for regular monitoring when you are receiving Lumizyme treatment to find out if antibodies have formed and to follow the level (titers) of these antibodies over time. As a general guideline:
For more information on antibodies and monitoring, see the Full Prescribing Information or talk to your healthcare provider.
LUMIZYME® (alglucosidase alfa) is an enzyme replacement therapy for patients with Pompe disease (acid α-glucosidase (GAA) deficiency).
WARNING: RISK OF ANAPHYLAXIS, HYPERSENSITIVITY AND IMMUNE-MEDIATED REACTIONS, AND RISK OF CARDIORESPIRATORY FAILURE
Anaphylaxis and Hypersensitivity Reactions: Life-threatening anaphylaxis and hypersensitivity reactions have been observed in some patients during and after treatment with alglucosidase alfa. If such a reaction is severe enough, your doctor may decide to immediately discontinue the infusion and provide you with immediate medical care. Appropriate medical support and monitoring measures should be available during infusion.
Immune-Mediated Reactions: You or your child may be monitored for the development of systemic immune-mediated reactions while receiving Lumizyme. If these reactions occur, your doctor may discontinue the infusion and initiate appropriate medical treatment.
Risk of Acute Cardiorespiratory Failure: Infant Pompe patients with heart or breathing problems who are experiencing an acute respiratory condition may be at risk for increasing the seriousness of these problems as a result of Lumizyme administration due to the infusion fluid, and your child’s doctor may require additional monitoring for these infants.
Risk of Cardiac Arrhythmia and Sudden Cardiac Death during General Anesthesia for Central Venous Catheter Placement: Caution should be used when administering general anesthesia for the placement of a central venous catheter intended for Lumizyme infusion. Ventricular arrhythmias and slow heart rate resulting in cardiac arrest or death have been observed in infant Pompe patients with cardiac hypertrophy during general anesthesia for central venous catheter placement. Appropriate medical support and monitoring measures should be available during infusion.
Risk of Antibody Development: Patients with infantile-onset Pompe disease (IOPD) should be managed by a clinical specialist knowledgeable in immune tolerance induction in Pompe disease to optimize treatment. Some patients who develop high and sustained IgG antibody levels, including certain IOPD patients, may have a reduced response to Lumizyme.
Monitoring: Laboratory Tests: Patients should be monitored for IgG antibody formation every 3 months for 2 years and then annually thereafter.
The most frequently reported adverse reactions during Lumizyme studies in patients were allergy reactions and included: anaphylaxis, rash, fever, flushing/feeling hot, hives, headache, excessive sweating, nausea, cough, less oxygen in the blood, fast heart rate, rapid breathing, chest discomfort, dizziness, muscle twitching, agitation, bluish or purple skin, redness of skin, high blood pressure/increased blood pressure, facial paleness, chills, tremor, vomiting, fatigue, and muscle pain.
Please see the Full Prescribing Information for complete details, including boxed WARNING.