In partnership with your geneticist and healthcare team, you can take charge of your Morquio A. Your healthcare team can help you determine which assessments are most important for you and how often you should have them done.
Your geneticist may take the lead in coordinating many aspects of your treatment plan, such as tests and visits, as well as arranging for VIMIZIM® (elosulfase alfa) treatment.
Because Morquio A is progressive, it is important to keep track of your symptoms. There are a number of assessments that will help you and your healthcare team track your symptoms and progress. The Assessments Overview Chart provides a convenient summary of the various types of assessments and when they are recommended. You can use this with your healthcare team to help manage your Morquio A.
| Specialist | Assessment to Consider | Suggested Frequency |
|---|---|---|
| Geneticist / Pediatrician | Endurance (6-minute walk test or timed 25 foot walk test) | At baseline and then annually |
| Growth | Every visit | |
| Urine test (urinary glycosaminoglycan or uGAG levels) | At baseline and then every 6 months | |
| Disease burden (pain quality of life, activities of daily living, fatigue, wheelchair/walking aid use) | Annually | |
| Radiologist | Brain (MRI or CT scan) | At diagnosis and as clinically indicated |
| Hips lower extremities, and skeletal abnormalities | At diagnosis and as clinically indicated | |
| Whole spine (MRI or CT scan for injury and changes to spinal canal/cord) | Annually, as clinically indicated | |
| Standing or sitting plain radiography of the cervical and thoracolumbar spine | At diagnosis and then every 2-3 years, as clinically indicated | |
| Cardiologist | Assessments of vital signs including oxygen saturation, right arm and leg blood pressure measurements, carful ausculation, and ECG | At diagnosis and then annually, as clinically indicated |
| Pulmonologist | Respiratory function tests by spirometry in children ages 5 and up. Respiratory rate and arterial oxygen saturation before and after annual endurance testing | At baseline and then annually until child stops growing, then every 2-3 years |
| Overnight sleep study | At diagnosis and then every 3 years | |
| Neurologist | Neurological function and spinal exams to detect any issues | Every visit (minimally every 6 months) |
| Ophthalmologist | Eye exam including visual acuity, refraction, slit-lamp examination of cornea, funduscopic evaluation including optic nerve, and measurement of intraocular pressure | At baseline and every 6 months or at least annually |
| ENT (ear, nose, throat) | Hearing evaluation including tympanometry | Every 3-6 months (children) Every 6-12 months(adults) |
| Visualisation of the upper airway with fiberoptic examination | At diagnosis and then annually | |
| Dentist | Dental evaluation with consideration of subacute endocarditis prophylaxis for certain procedures | Annually |
| Physical therapist / Occupational therapist / Speech therapist | Assesment and support of limb function and speech problems as needed | As clinically indicated |
What is the most important information I should know about VIMIZIM?
Severe and life-threatening hypersensitivity reactions, including anaphylaxis, can occur during any VIMIZIM infusion, as early as 30 minutes from the start and up to 6 days after infusion, and as late into treatment as the 47th infusion. These reactions can occur in people receiving VIMIZIM for the first time or in people who have previously received VIMZIM without having an allergic reaction.
Your doctor will tell you about the symptoms of life-threatening hypersensitivity reactions, including anaphylaxis and when to seek immediate medical care. Typical signs of anaphylaxis include cough, rash, throat tightness, hives, flushing, changes in skin color, low blood pressure, shortness of breath, chest pain, and gastrointestinal symptoms such as nausea, abdominal pain, retching, and vomiting. If a severe allergic reaction (e.g., anaphylaxis) occurs during infusion, the infusion should be stopped immediately, and you should receive medical attention. Contact your doctor or get medical help right away if these symptoms occur after VIMIZIM infusions.
If you have a respiratory illness, you may be at risk for a sudden worsening of your condition, and you may require additional monitoring.
What else should I know about Vimizim?
VIMIZIM is a prescription medicine. Before treatment with VIMIZIM, it is important to discuss your medical history with your doctor. Tell your doctor if you are sick or taking any medication and if you are allergic to any medicines. Also tell your doctor if you are pregnant, planning to become pregnant, or are a nursing mother. Your doctor will decide if VIMIZIM is right for you. If you have questions or would like more information about VIMIZIM, contact your doctor.
You should receive medication such as antihistamines before VIMIZIM infusions to reduce the risk of reactions. If a reaction occurs, the infusion should be slowed or stopped and you may be given additional medication. If a severe allergic reaction (e.g., anaphylaxis) occurs during infusion, the infusion should be stopped immediately, and you should receive medical treatment.
If you have acute febrile or respiratory illness at the time of VIMIZIM infusion, you may be at higher risk of life-threatening complications from hypersensitivity reactions. If you use supplemental oxygen or continuous positive airway pressure (CPAP), you should have it available during your infusion in the event of a sudden reaction, or extreme drowsiness/sleep from antihistamines.
Spinal cord damage may occur due to the natural MPS IVA disease process. Signs of spinal cord injury include back pain, numbness and paralysis of limbs, and loss of bladder and bowel control. Contact your doctor immediately if you develop any of these symptoms.
What are the most common side effects of VIMIZIM?
The most common side effects reported during VIMIZIM infusions included fever, vomiting, headache, nausea, abdominal pain, chills, and fatigue. These are not all the possible side effects with VIMIZIM. Talk to your doctor if you have any symptoms that bother you or that do not go away.
Call your doctor for medical advice about side effects. You may report side effects to BioMarin at 1-866-906-6100 and the FDA by visiting www.fda.gov/medwatch or calling 1-800-FDA-1088.
Please see full Prescribing Information, with important warning for risk of anaphylaxis.
VIMIZIM® (elosulfase alfa) is indicated for patients with mucopolysaccharidosis type IVA (MPS IVA; Morquio A syndrome).