Inborn errors of metabolism:
Indications for ONPATTRO:
Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
See full labeling. Premedicate with IV corticosteroid, oral acetaminophen, IV H1- and H2-blocker on the day of Onpattro infusion at least 60mins prior to initiation; may reduce corticosteroid as needed, if related adverse reactions occur. Give as IV infusion over ~80mins at an initial rate of 1mL/min for first 15mins, then increase to ~3mL/min for the remainder. <100kg: 0.3mg/kg once every 3 weeks. ≥100kg: 30mg once every 3 weeks.
Monitor for infusion-related reactions during infusion; consider slowing or interrupting infusion as clinically indicated; discontinue and do not resume if serious or life-threatening reaction occurs. Monitor infusion site for possible infiltration during infusion; manage suspected extravasation accordingly. Reduced serum Vit. A levels may develop; give supplementation at the recommended daily allowance of Vit. A. Refer patients for eye exam if ocular symptoms suggestive of Vit. A deficiency (eg, night blindness). Moderate or severe hepatic impairment: not studied. Severe renal impairment or ESRD: not studied. Pregnancy. Nursing mothers.
Small interfering RNA (siRNA).
Upper respiratory tract infections, infusion-related reactions, dyspepsia, dyspnea, muscle spasms, arthralgia, erythema, bronchitis, vertigo.