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Mucopolysaccharidosis type I (MPS I) Monitoring


Continuous monitoring of MPS I progression is an essential part of disease management.1-5

Minimum Recommended Schedule of Assessments for MPS I Patients1-5

The Recommended Schedule of Assessments represents the core MPS I disease-related assessments that allow evaluations of a patient's disease progression over time. Physicians will determine the actual frequency of necessary assessments according to a patient's individualized need for medical care and routine follow-up.

 Initital assessmentsEvery 6 monthsEvery 12 monthsEvery other year
General
Demographics   
Patient Diagnosisa   
Medical History  
Physical Examination  
General Appearance  
MPS I Disease Clinical Assessment
Neurologic/CNS
MRI of Brain  
MRI of Spine  
Medical Nerve Conduction Velocity  
Cognitive Testing (DQ/IQ)  
Opthalmologic
Visual Acuity  
Retinal Examination  
Corneal Examination  
Auditory
Audiometry  
Cardiac
Echocardiogram  
ECG  
Respiratoryb
FVC/FEV₁  
Sleep Study  
Gastrointestinal
Spleen Volumec  
Liver Volumec  
Musculoskeletal

Skeletal Survey by X-rayd

  
Vitals and Laboratory Tests
Height/Weight  
Head Circumferenced  
Blood Pressure  
Enzyme Activity Level   
Urinary GAG Level  
Urinalysis  
Functional Outcome Measurements
MPS Health Assessment Questionnaire, or other toolsb  

All tests requiring sedation are recommended only if sedation is considered to be safe for the patient.

aDiagnosis would include genotype, if available.
bMay not be possible in noncooperative patients or patients <5 to 6 years of age.
cThe recommended method for obtaining organ volumes is MRI or CT to enable quantitative analysis; however, if in the opinion of the investigator it is unsafe to sedate the patient, then ultrasound may be substituted.
dThese assessments will be measured in pediatric patients only, unless determined otherwise by the treating physician.

CNS=central nervous system; DQ/IQ=developmental quotient/intellectual quotient; ECG=electrocardiogram; FEV₁=forced expiratory volume in one second; FVC=forced vital capacity; GAG=glycosaminoglycan; MPS I=mucopolysaccharidosis type I; MRI=magnetic resonance imaging.

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Indication

ALDURAZYME® (laronidase) is indicated for the treatment of adult and pediatric patients with Hurler and Hurler-Scheie forms of Mucopolysaccharidosis I (MPS I) and for patients with the Scheie form who have moderate to severe symptoms.

Limitations of Use:

  • The safety and effectiveness of treating mildly affected patients with the Scheie form have not been established.
  • The effect of ALDURAZYME on central nervous system manifestations of the disorder has not been determined.

Important Safety Information

WARNING: HYPERSENTIVITIY REACTIONS INCLUDING ANAPHYLAXIS, and ACUTE RESPIRATORY COMPLICATIONS ASSOCIATED WITH ADMINISTRATION

Hypersensitivity Reactions Including Anaphylaxis
Patients treated with ALDURAZYME  have experienced life-threatening hypersensitivity reactions, including anaphylaxis. Appropriate medical monitoring and support measures, including cardiopulmonary resuscitation equipment, should be readily available during ALDURAZYME administration. If a severe hypersensitivity reaction (e.g., anaphylaxis) occurs, discontinue ALDURAZYME immediately and initiate appropriate medical treatment. In patients with severe hypersensitivity reactions, a desensitization procedure to ALDURAZYME may be considered.

Acute Respiratory Complications Associated with Administration
Patients with compromised respiratory function or acute respiratory disease may be at risk of serious acute exacerbation of their respiratory compromise due to infusion reactions and require additional monitoring.

Warnings and Precautions
Hypersensitivity Reactions Including Anaphylaxis: See Boxed WARNING.

  • Pre-existing upper airway obstruction may contribute to the severity of some reactions. Consider premedicating patients with antihistamines, with or without antipyretics. Because of the potential for recurrent reactions, some patients who experience initial severe reactions may require prolonged observation.
  • Consider risks and benefits of re-administering ALDURAZYME following severe hypersensitivity reactions. Patients may be rechallenged using slower infusion rates which may be increased if tolerated to reach the recommended rate. If a mild or moderate hypersensitivity reaction occurs, consider temporarily holding the infusion or slowing the infusion rate.

Acute Respiratory Complications Associated with Administration: See Boxed WARNING.

  • Patients with an acute febrile or respiratory illness may be at greater risk for infusion reactions. Consider the patient’s clinical status prior to administration of ALDURAZYME and consider delaying the infusion.
  • Evaluation of airway patency should be considered prior to initiating ALDURAZYME. Patients using supplemental oxygen or continuous positive airway pressure (CPAP) during sleep should have these treatments readily available during infusion in the event of an infusion reaction or extreme drowsiness/sleep induced by antihistamine use.

Acute Cardiorespiratory Failure:

  • Use caution when administering ALDURAZYME to patients susceptible to fluid overload, or with an acute underlying respiratory illness or compromised cardiac and/or respiratory function for whom fluid restriction is indicated. Consider a decreased total infusion volume and infusion rate when administering ALDURAZYME to these patients.

Infusion-Associated Reactions:

  • ALDURAZYME may cause infusion-associated reactions (IARs). Consider pre-medicating with antihistamines, with or without antipyretics, however IARs may still occur in patients after receiving pre-medication. Discontinue immediately or adjust the infusion rate based on the severity of the reaction.

Adverse Reactions
Patients 6 months of age and older

  • The most common adverse reactions reported in ≥10% of patients were infusion reactions, which included pyrexia, chills, blood pressure increased, tachycardia, and oxygen saturation decreased.

Patients 6 years of age and older

  • The most common adverse reactions reported in ≥10% of patients were rash, upper respiratory tract infection, injection site reaction, hyperreflexia, paresthesia, flushing, and poor venous access.

Please see Full Prescribing Information including Boxed WARNING for Aldurazyme.

Indication

Important Safety Information

Reference: 1. Berger KI et al. J Inherit Metab Dis. 2012;36(2):201-210. 2. Braunlin EA et al. J Inherit Metab Dis. 2011;34(6):1183-1197. 3. White KK. Rheumatology. 2011; 50 (Suppl 5):v26-v33. https://doi.org/10.1093/rheumatology/ker393 4. Muenzer J et al. Pediatrics. 2009; 123:19-29. 5. Pastores GM et al. Mol Genet Metab. 2007;91(1):37-47.

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