Figure 1.
Enrollment flow diagram. A total of 236 patients were screened to enroll 180 in this phase 2 trial between 2012 and 2020. Most patients not enrolled in the study did not meet eligibility criteria or declined enrollment to pursue alternative therapy, primarily bone marrow transplantation. In Cohorts 1-3, one subject was found to have abnormal baseline cytogenetics, which precluded treatment on this protocol and therefore was taken off the study to pursue appropriate treatment. One patient enrolled on the extension cohort, who had ANC <200 x 109/L, was initiated on treatment prior to the results of pretreatment cytogenetics as allowed per protocol but was taken off the study to pursue appropriate treatment after chromosome 7 abnormalities were identified (marked by * in the diagram). Both patients were not included in the reported secondary analysis. A total of 178 patients were included for hematologic response at 6 months and clonal evolution analysis. A total of 145 patients who had achieved a response at 6 months were included for relapse analysis. Of note, the Clinical Center at NIH performs screening at multiple levels, making the application of a CONSORT analysis complicated.