Article
Clonal Selection and Evolution after Treatment of Severe Aplastic Anemia.
NEJM evidence - 1 May 2026
Gutierrez-Rodrigues Fernanda, Groarke Emma M, Miao Rui, Shalhoub Ruba, Alemu Lemlem, Mizumaki Hiroki, Rios Olga, Glass Joshua, Durrani Jibran, Bazzo Catto Luiz Fernando, Arcuri Luca, Kajigaya Sachiko, Gao Shouguo, Paul Subrata, Lack Justin, Yoshida Masanori, Quinones Raffo Diego, Lotter Jennifer, Wu Colin O, Dunbar Cynthia E, Wlodarski Marcin, Young Neal S, Patel Bhavisha A
Abstract excerpt
BACKGROUND: Clonal hematopoiesis (CH) is a feature of severe aplastic anemia (SAA), but its clinical significance is debated. METHODS: We integrated longitudinal clinical and CH data from patients with SAA treated with immunosuppression plus eltrombopag (IST-EPAG) in a phase 2 trial to characterize clonal dynamics during recovery and progression to myeloid cancer or paroxysmal nocturnal hemoglobinuria (PNH); CH...
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