Clonal Selection and Evolution after Treatment of Severe Aplastic Anemia

NEJM Evidence 2026

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Authors: Fernanda Gutierrez‐Rodrigues, Emma M. Groarke, Rui Miao, Ruba Shalhoub, Lemlem Alemu, Hiroki Mizumaki, Olga Rios, Joshua Glass, Jibran Durrani, Luiz Fernando Bazzo Catto, Luca Arcuri, Sachiko Kajigaya
+11 more Shouguo Gao, Subrata Paul, Justin Lack, Masanori Yoshida, Diego Quinones Raffo, Jennifer Lotter, Colin O. Wu, Cynthia E. Dunbar, Marcin Włodarski, Neal S. Young, Bhavisha A. Patel
DOI: 10.1056/evidoa2500171
Cited by: 4
References: 36

Abstract

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 was defined as the presence of somatic mutations at a variant allele frequency of 0.1% or greater. RESULTS: clones usually present before treatment. CONCLUSIONS: mutated clones.

Fields of Study & Subjects

Hematopoietic Stem Cell Transplantation Complement system in diseases Platelet Disorders and Treatments

Publication details

Journal / source
NEJM Evidence
Publisher
Massachusetts Medical Society
Volume
5
Issue
5
Pages
EVIDoa2500171–EVIDoa2500171
Publication date
28 April 2026
Language
EN