Acquired somatic mutations in relation to EPAG treatment. (A) Targeted deep sequencing for a panel of genes previously reported as associated with myeloid cancers (MC) or aplastic anemia (AA) was performed on bone marrow cells from all patients at baseline and at the primary end point. Each row indicates a specific MC/AA gene found mutated in at least 1 patient, and each column represents each patient’s samples at baseline and at the primary end point. The panel of MC/AA genes is given in the supplemental Methods, and the mutations detected are listed in supplemental Table 4. Patients are grouped according to response (green at primary end point designates response) and the presence of abnormal cytogenetics (pink box). (B) The percentage of variant alleles is shown on the y-axis at baseline and the primary end point for the 9 individual mutations detected in 6 patients, with a P value (paired Student t test) comparing VAF at baseline and primary end point. (C) The percentage of glycosylphosphatidylinositol-negative (GPI^neg) PNH granulocytes in 11 patients with detectable clones at baseline and/or the primary end point are plotted, with a P value (paired Student t test) comparing baseline and primary end point. The 3 patients with germline mutations (UPN10, UPN30, and UPN34) had no somatic mutations detected.