Clonal Selection and Evolution after Treatment of Severe Aplastic Anemia

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.