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Pregnancy Category: B
ClassificationTherapeutic: replacement enzyme
Mucopolysaccharridosis VI (MPS IV).
Replaces a deficient enzyme in MPS IV. Without replacement, glycoaminoglycans accumulate resulting in cell, organ and tissue dysfunction.
Improved walking and stair climbing.
Absorption: IV administration results in complete bioavailability.
Distribution: Widely distributed.
Metabolism and Excretion: Unknown.
Half-life: 9 min (after one week of treatment, 26 min (after 24 weeks of treatment).
Time/action profile (inprove exercise parameters)
Contraindicated in: None.
Use Cautiously in: Febrile or respiratory illness; Pediatric: Children <5 yr (safety not established); Obstetric / Lactation: Safety not established.
Adverse Reactions/Side Effects
- malaise (most frequent)
Ear, Eye, Nose, Throat
- conjunctivitis (most frequent)
- cornela opacification (most frequent)
- ear pain
- dyspnea (most frequent)
- chest pain (most frequent)
- ↑ BP
- facial edema (most frequent)
- gastroenteritis (most frequent)
- abdominal pain
- areflexia (most frequent)
- spinal/cervical cord compression (life-threatening)
- infusion reactions
- rigors (most frequent)
Drug-Drug interactionNone noted.
Intravenous (Adults and Children >5 yr) 1 mg/kg once weekly.
Solution for IV administration (diluted prior to use): 5 mg/5 mL
- Assess for infusion reactions (fever, chills/rigors, headache, rash, mild to moderate urticaria); may occur in over half of patients. May also cause nausea, vomiting, elevated BP, retrosternal pain, abdominal pain, malaise, and joint pain. Severe reactions include angioneurotic edema, hypotension, dyspnea, bronchospasm, respiratory distress, apnea. May occur as late as week 55 of treatment. Patients should receive antihistamines and antipyretics prior to administration to minimize occurrence. Slowing or temporary interruption of infusion and administration of additional antihistamines, antipyretics, and occasionally corticosteroids usually stop symptoms. Most patients are able to complete infusion. Administer subsequent infusions at a slower rate additional prophylactic antihistamines, and, if reaction is severe, prophylactic corticosteroids. If severe reaction occurs, discontinue infusion immediately and treat symptoms. Consider risk versus benefit before re-administering galsulfase.
- Assess for sleep apnea, common in patients with MPS VI, prior to therapy. Administration of pretreatment antihistamines may increase risk. Supplemental oxygen and continuous positive airway pressure (CPAP) should be available during infusion.
- Assess vital signs and respiratory status prior to administration. Administration should be delayed in patients with an acute febrile or respiratory illness.
- Monitor for signs and symptoms of spinal/cervical cord compression (back pain, paralysis of limbs below level of compression, urinary and fecal incontinence). Manage symptomatically.
Potential Nursing DiagnosesActivity intolerance (Indications)
Deficient knowledge, related to medication regimen (Patient/Family Teaching)
- Pretreat with antihistamines with or without antipyretics 30–60 min prior to start of infusion.
- Intermittent Infusion: Diluent: 250 mL bag of 0.9% NaClDetermine number of vials to be diluted based on patient weight; round to nearest whole vial. Remove vials from refrigerator and allow to reach room temperature. Do not use after expiration date. Do not allow vials to remain at room temperature for longer than 24 hrs prior to dilution. Do not heat or microwave vials. Solution is clear to slightly opalescent and colorless to pale yellow; a few translucent particles may be present. Do not administer solutions that are discolored or contain particulate matter. Withdraw and discard amount equal to galsulfase from a 250 mL bag of 0.9% NaCl (not necessary if using 100 mL bag in patients susceptible to fluid volume overload). Slowly withdraw calculated volume from galsulfase vials using caution to avoid excessive agitation, may denature and render inactive. Do not use a filter needle. Slowly add galsulfase to 0.9% NaCl; avoid agitation. Gently rotate infusion bag to ensure distribution; do not shake. Discard unused portions. Use diluted solution immediately; storage should not exceed 48 hr from time of preparation to completion of administration. Use PVC containers and administer via PVC infusion set with an in-line low protein binding 0.2 micrometer filter.
- Rate: Administer at a rate of 6 mL/hr for first hour. If infusion is well tolerated, rate may be increased to 80 mL/hr for next 3 hr. Administer total volume of infusion over no less than 4 hr. If 100 mL bag is used, decrease infusion rate so total volume is infused over at least 4 hrs. Infusion time can be extended for up to 20 hr if infusion reactions occur.
- Additive Incompatibility: Do not admix or administer with other products.
- Inform patient that a Clinical Surveillance Program has been established to better understand the variability and progression of MPS VI and to evaluate long term effects of galsulfase. Encourage patients to participate; participation is voluntary and may be long term. For information visit www.MPSVI.com/CSP or call 866–906–6100.
- Improved walking and stair climbing capacity in patients with MPS VI.
a rarely used miscellaneous drug.
indications This drug is used to treat mucopolysaccharidosis VI and Maroteaux-Lamy syndrome.