imetelstat (Rytelo)
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Indications
- relapsed or refractory low- to intermediate-risk myelodysplastic syndrome with transfusion-dependent anemia requiring >= 4 RBC units over 8 weeks & not or no longer candidates for erythropoiesis-stimulating agents
Dosage
- 7.1 mg/kg administered as IV infusion over 2 hours every 28 days
Adverse effects
- >= 10%
- thrombocytopenia, leukopenia, neutropenia, prolonged PTT
- elevated serum ALT, serum AST, serum alkaline phosphatase
- fatigue, arthralgia/myalgia, COVID-19, headache
Mechanism of action
- oligonucleotide telomerase inhibitor
- provides transfusion independence (up to 1 year)
- disease-modifying activity for heavily transfused patients [2]
More general terms
References
- ↑ Worcester S FDA Approves Imetelstat for Lower-Risk Myelodysplastic Syndromes. Medscape. June 7, 2024 https://www.medscape.com/viewarticle/fda-approves-imetelstat-lower-risk-myelodysplastic-syndromes-2024a1000as3
- ↑ 2.0 2.1 Platzbecker U, Santini V, Fenaux P et al Imetelstat in patients with lower-risk myelodysplastic syndromes who have relapsed or are refractory to erythropoiesis-stimulating agents (IMerge): a multinational, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2024 Jan 20;403(10423):249-260. PMID: https://www.ncbi.nlm.nih.gov/pubmed/38048786 Clinical Trial.