Vera Ghali: Mitapivat Opens a New Chapter in Thalassemia Treatment
Vera Ghali, Member of the Lebanese Order of Physicians and Lebanese Pediatric Society, shared The Lancet‘s post on X, adding:
“A global, double-blind randomized ENERGIZE-T trial, conducted across 19 countries in North and South America, Europe, Asia-Pacific, and the Middle East, found that the orally administered drug, mitapivat, showed efficacy and well-tolerated adverse events (AE) in patients with transfusion-dependent alpha- and beta- thalassemia (TDT) who are more than 18 years.
Mitapivat is an oral allosteric pyruvate kinase (PK) activator that causes an increase in ATP production, reduces oxidative damage, and helps RBCs live longer.
It is to be administered as 100mg dose taken twice daily.
It is the first oral disease-modifying therapy for adults with TDT alpha-and beta- thalassemia that significantly reduced the transfusion burden for these patients and the associated risks and provides new treatment options.
However, patients taking mitapivat must be followed-up monthly for liver enzyme elevations due to risks of hepatotoxicity, and it is contraindicated in patients with liver cirrhosis and other liver diseases.
Liver toxicities are reversible upon discontinuation of mitapivat.”
The Lancet shared a post on X about a recent article by Maria Domenica Cappellini et al., adding:
“A new study assessed the efficacy and safety of mitapivat in adults with transfusion-dependent thalassaemia.
The study found that mitapivat significantly reduced the transfusion burden and was generally well tolerated, showing a favourable benefit–risk profile.”
Title: Efficacy and safety of mitapivat in adults with transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE-T): a double-blind, randomised, multicentre, placebo-controlled, phase 3 trial
Authors: Maria Domenica Cappellini, Sujit Sheth, Ali Taher, Hanny Al-Samkari, Ali Bülent Antmen, David Beneitez
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