Article
Targeted therapy with the mutant IDH2 inhibitor enasidenib for high-risk IDH2-mutant myelodysplastic syndrome.
Blood advances - 13 Jun 2023
DiNardo Courtney D, Venugopal Sangeetha, Lachowiez Curtis, Takahashi Koichi, Loghavi Sanam, Montalban-Bravo Guillermo, Wang Xuemei, Carraway Hetty, Sekeres Mikkael, Sukkur Ameenah, Hammond Danielle, Chien Kelly, Maiti Abhishek, Masarova Lucia, Sasaki Koji, Alvarado Yesid, Kadia Tapan, Short Nicholas J, Daver Naval, Borthakur Gautam, Ravandi Farhad, Kantarjian Hagop M, Patel Bhumika, Dezern Amy, Roboz Gail, Garcia-Manero Guillermo
Abstract excerpt
The isocitrate dehydrogenase enzyme 2 (IDH2) gene is mutated in ∼5% of patients with myelodysplastic syndrome (MDS). Enasidenib is an oral, selective, mutant IDH2 inhibitor approved for IDH2-mutated (mIDH2) relapsed/refractory acute myeloid leukemia. We designed a 2-arm multicenter study to evaluate safety and efficacy of (A) the combination of enasidenib with azacitidine for newly diagnosed mIDH2 MDS, and (B)...
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