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Review

Imetelstat

No authors listed
In: LiverTox: Clinical and Research Information on Drug-Induced Liver Injury [Internet]. Bethesda (MD): National Institute of Diabetes and Digestive and Kidney Diseases; 2012.
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Review

Imetelstat

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Excerpt

Imetelstat is an oligonucleotide inhibitor of telomerase used to treat patients with myelodysplastic syndromes with transfusion dependent anemia. Imetelstat is associated with frequent serum aminotransferase elevations during therapy but has not been implicated in cases of clinically apparent liver injury with jaundice.

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References

    1. FDA. Multi-disciplinary Review. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2024/217779Orig1s000M...(FDA website including product label and multidiscipline review of data submitted in support of imetelstat approval includes discussion of hepatotoxicity, therapy being associated with increase in ALT elevations [43% vs 37% with placebo] and bilirubin [39% vs 39%], with several patients having concurrent elevations in both, but most abnormalities resolved spontaneously suggesting that most elevations were due to the underlying condition; in preregistration studies, there were no instances of clinically apparent liver injury attributed to therapy).
    1. Tefferi A, Lasho TL, Begna KH, Patnaik MM, Zblewski DL, Finke CM, Laborde RR, et al. A pilot study of the telomerase inhibitor imetelstat for myelofibrosis. N Engl J Med. 2015;373:908-19.(Among 33 patients with high or intermediate risk myelofibrosis treated with imetelstat [9.4 mg/kg iv] every 1 to 3 weeks, remission was induced in 7 [21%] and adverse events were frequent, especially thrombocytopenia and anemia; ALT elevations arose in 27%, AST in 55%, alkaline phosphatase in 36% and bilirubin in 49% of subjects, but most abnormalities were mild and self-limited and there were no instances of clinically apparent liver injury attributable to imetelstat). - PubMed
    1. Baerlocher GM, Oppliger Leibundgut E, Ottmann OG, Spitzer G, Odenike O, McDevitt MA, Röth A, et al. Telomerase inhibitor imetelstat in patients with essential thrombocythemia. N Engl J Med. 2015;373: 920-8.(Among 18 patients with essential thrombocytosis treated with imetelstat iv once weekly to lower platelet counts to less than 300,000/μL, a hematologic response occurred in all 18, common adverse events were neutropenia, anemia, headache, and syncope; while ALT elevations arose in 72%, they were above 5 times ULN in only 2 [11%]). - PubMed
    1. Mascarenhas J, Komrokji RS, Palandri F, Martino B, Niederwieser D, Reiter A, Scott BL, et al. Randomized, single-blind, multicenter phase II study of two doses of imetelstat in relapsed or refractory myelofibrosis. J Clin Oncol. 2021;39:2881-2892.(Among 107 patients with relapsed or refractory myelofibrosis treated with 2 doses of imetelstat, symptom responses were achieved in 32%, and one year objective responses occurred in 79% and 84%; liver test adverse events occurred in 13%, but there were no instances of clinically apparent drug induced liver injury). - PubMed
    1. Santini V. Advances in myelodysplastic syndrome. Curr Opin Oncol. 2021;33:681-6.(Review of the pathogenesis, clinical features, complications, diagnosis, cytogenetics, and therapy of MDS mentions the use of luspatercept as well as telomerase inhibitors and hypomethylating agents). - PubMed

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