Miscellaneous musculoskeletal disorders:
Indications for ZOLGENSMA:
Treatment of pediatrics <2yrs of age with spinal muscular atrophy (SMA) with bi-allelic mutations in the survival motor neuron 1 (SMN1) gene.
Limitations of Use:
Safety and efficacy of repeat administration has not been evaluated. Use in patients with advanced SMA (eg, complete paralysis of limbs, permanent ventilator dependence) has not been evaluated.
≥2yrs: not applicable.
Give as a slow IV infusion over 60mins. <2yrs: 1.1×1014 vg/kg (see full labeling). Starting one day prior to Zolgensma infusion: give systemic corticosteroids equivalent to oral prednisolone 1mg/kg/day for 30 days, then taper dose for the next 28 days if LFTs are unremarkable.
Acute serious liver injury.
Risk of acute serious liver injury. Monitor liver function prior to infusion, weekly for the 1st month, then every other week for the 2nd/3rd months until unremarkable results. Monitor platelets, troponin-I prior to infusion, weekly for the 1st month, then every other week (platelets) and monthly (troponin-I) for the 2nd/3rd months until levels return to baseline. Perform baseline anti-AAV9 antibody testing prior to infusion; may retest if titers are >1:50. Premature neonates: delay infusion until full-term gestational age is reached.
Adeno-associated virus vector-based gene therapy.
Adjust vaccination schedule to accommodate concomitant corticosteroid use before and after infusion.
Elevated aminotransferases, vomiting; thrombocytopenia, elevated troponin-I.
Customized kit—1 (2–9 vials + alcohol wipes)