Treatment of patients with Mucopolysaccharidosis type IVA (MPS IVA, Morquio A Syndrome).
Consists of an enzyme produced by recombinant DNA technology that hydrolyzes sulfate from galactose-6–sulfate or N-acetyl-galactosamine-6–sulfate. Lack of this enzyme in Mucoplysaccharidosis results in intracellular accumulation of glycosaminoglycans (GAG) producing widespread cellular, tissue and organ dysfunction.
Decreased accumulation of GAG, decreased end organ dysfunction and improved musculoskeletal performance MPS IVA.
Absorption: IV administration results in complete bioavailability.
Distribution: Taken up by cells.
Metabolism and Excretion: Unknown.
Half-life: Initially–7 min; after prolonged treatment–35.9 min (due to neutralizing antibodies).
TIME/ACTION PROFILE (blood levels)
|IV||unknown†||end of infusion||unknown|
- None noted.
Use Cautiously in:
- Concurrent acute febrile/respiratory illness (↑ risk of life threatening complications of hypersensitivity reaction)
- Sleep apnea (supportive measures should be available during infusion)
- OB: Use during pregnancy only if potential benefit justifies potential risk to fetus
- Consider maternal need for elosulfase alfa and potential adverse effects to infant
- Pedi: Safe and effective use in children <5 yr has not been established.
Adverse Reactions/Side Effects
CNS: fatigue, headache
GI: abdominal pain, nausea, vomiting
Misc: chills, fever, HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS
* CAPITALS indicate life-threatening.
Underline indicate most frequent.
IV: (Adults and Children) ≥5 yr): 2 mg/kg once weekly; pretreatment with antihistamines with/without antipyretics recommended.
Solution for intravenous use (requires further dilution): 5 mg/5mL single-use vial
- Monitor for signs and symptoms of anaphylaxis (cough, erythema, throat tightness, urticaria, flushing, cyanosis, hypotension, rash, dyspnea, chest discomfort, nausea, abdominal pain, retching, vomiting). Discontinue infusion and manage symptomatically. May occur 30 min to 3 hr after start of infusion and as late as 47th infusion.
- Evaluate airway patency and risk of sleep apnea. Provide supplemental oxygen or continuous positive airway pressure (CPAP) during sleep and infusion in case of acute reaction or extreme sleepiness induced by antihistamine.
- Deficient knowledge, related to medication regimen (Indications)
- Pre-treat with antihistamines, with or without antipyretics, 30–60 min prior to start of infusion.
- Intermittent Infusion: Determine number of vials required. Diluent: Dilute with 0.9% NaCl to a final volume of 100 mL for patients weighing less than 25 kg or 250 mL for patients weighing 25 kg or more. Solution is clear to slightly opalescent and colorless to pale yellow; do not administer solutions that are discolored or contain particulate matter. Gently rotate to mix; do not shake or agitate. Use diluted solution immediately or refrigerate up to 24 hr followed by up to 24 hr at room temperature during infusion.
- Rate:Infuse over at least 3.5 (100 mL) or 4.5 hr (250 mL) through a low-protein binding infusion set with a low-protein binding 0.2 micron in-line filter.
- Explain the purpose of elosulfate alfa to patient.
- Advise patient to notify health care professional immediately if signs and symptoms of anaphylaxis occur.
- Advise female patients to notify health care professional if pregnancy is planned or suspected or if breastfeeding. Encourage patient to enroll in Morquio A Registry to collect data on pregnant women with MPS IVA treated with Vimizim by contacting MARS@bmrn.com or call 1-800-983-4587.
Decreased end organ dysfunction and improved musculoskeletal performance.
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