Evaluation of airway patency should be considered prior to initiation of Careful consideration should be given to the patient’s clinical status prior to administration ofĪLDURAZYME and consider delaying ALDURAZYME infusion. Patients with an acute febrile or respiratory illness at the time of ALDURAZYME infusion may be at greater risk for Extreme care should be exercised, with appropriate resuscitation measures available, if theĭecision is made to re-administer the product. The risks and benefits of re-administering ALDURAZYME following an anaphylactic or severe allergic reaction Recurrent reactions, some patients who experience initial severe reactions may require prolonged observation. Medical support should be readily available when ALDURAZYME is administered. Due to the potential for severe allergic reactions, appropriate Have contributed to the severity of some reactions. In patients with MPS I, pre-existing upper airway obstruction may In clinical studies and postmarketing safety experience with ALDURAZYME, approximately 1% of patientsĮxperienced severe or serious allergic reactions. Interventions have included resuscitation, mechanical ventilatory support, emergency tracheotomy, hospitalization,Īnd treatment with inhaled beta-adrenergic agonists, epinephrine, and IV corticosteroids. Caution should be exercised if epinephrine is being consideredįor use in patients with MPS I due to the increased prevalence of coronary artery disease in these patients. If anaphylactic or other severe allergic reactions occur, immediately discontinue the infusion ofĪLDURAZYME and initiate appropriate treatment. Some of these reactions were life-threatening and included respiratory failure, respiratoryĭistress, stridor, tachypnea, bronchospasm, obstructive airways disorder, hypoxia, hypotension, bradycardia, and
Īnaphylaxis and severe allergic reactions have been observed in patients during or up to 3 hours afterĪLDURAZYME infusions. MPS I is historically classified as Hurler, Hurler-Scheie, and Scheie syndromes, but due to overlap in presentation is now commonly referred to severe (Hurler) or attenuated (Huler-Scheie, Scheie) disease. Individuals with the attenuated form of the disease can have some of the same physical symptoms, but they generally have limited cognitive impairment and may survive into adulthood. Their life span is typically less than 10 years. Individuals with the most severe form of MPS I (Hurler) typically suffer from a number of progressively debilitating symptoms, including cognitive impairment. The disease is highly heterogeneous, spanning a spectrum of severity. Recurrent Ear, Nose, and Throat InfectionsĬlinical manifestations of MPS I show a chronic and progressive course that is multisystemic in nature. MPS I is considered to be the prototypical lysosomal storage disorder with progressive multi-systemic disease and presenting features that vary depending on where a patient is on the disease continuum. Ultimately the accumulation causes cell, tissue, and organ dysfunction by largely unknown pathophysiological mechanisms. These glycosaminoglycans, which are important constituents of the extra cellular matrix, joint fluid, and connective tissue throughout the body, progressively accumulate in the lysosome. This process is essential for normal growth and homeostasis of tissues. This results in an inability of the lysosome to act in the stepwise degradation of certain glycosaminoglycans (GAG) - namely dermatan sulfate and heparan sulfate.
As a result of this defect, the cells of people with MPS I are either unable to produce the enzyme or produce it in low amounts.
The disease is caused by a defect in the gene coding for the lysosomal enzyme alpha-L-iduronidase. Mucopolysaccharidosis I (MPS I) is a rare, autosomal recessive genetic disease that affects multiple organ systems and tissues.