Inborn errors of metabolism:
Indications for TEGSEDI:
Treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Give as SC inj in abdomen, upper thigh, or outer area of upper arm; rotate inj sites. 284mg once weekly. Monitoring and treatment recommendations: see full labeling.
Platelet count <100x109/L. History of acute glomerulonephritis due to Tegsedi.
Risk of thrombocytopenia, glomerulonephritis. Do not initiate if platelets <100x109/L or UPCR ≥1000mg/g; discontinue and/or give corticosteroids (based on platelet count) or immunosuppressants; see full labeling. Avoid Tegsedi therapy if glucocorticoids or immunosuppressants are not advised. Monitor platelets, serum creatinine, eGFR, urine protein to creatinine ratio (UPCR), and urinalysis prior to treatment, every 2 weeks during, and for 8 weeks after discontinuation. Perform repeat platelet count if EDTA-mediated platelet clumping is suspected. Withhold if UPCR ≥1000mg/g or eGFR <45mL/min/1.73m2 develops; may resume treatment once resolved. Permanently discontinue if acute glomerulonephritis is confirmed. Nephrotic syndrome. Monitor ALT/AST, and total bilirubin prior to treatment, every 4 months during (monthly in liver transplant recipients), and for 8 weeks after discontinuation. Interrupt or discontinue if hepatic dysfunction or signs of liver transplant rejection develop. Risk of stroke and CNS arterial dissection. Discontinue if hypersensitivity reaction occurs. Give recommended Vit. A supplementation; refer for eye exam if symptoms of Vit. A deficiency (eg, night blindness). Moderate or severe hepatic impairment, severe renal impairment or ESRD: not studied. Elderly. Pregnancy. Nursing mothers.
Thrombocytopenia risk with concomitant antiplatelets (eg, adenosine, clopidogrel, prasugrel, ticagrelor, ticlopidine, aspirin, NSAIDs) or anticoagulants (eg, heparin, warfarin); consider discontinuing these agents if platelets <50x109/L. Caution with concomitant nephrotoxic drugs or drugs that may impair renal function.
Inj site reactions, nausea, headache, fatigue, thrombocytopenia, fever; glomerulonephritis, renal toxicity, inflammatory/immune effects, hepatic effects, hypersensitivity, reduced serum Vit. A.
Generic Drug Availability:
Single-dose prefilled syringes—1, 4