Table 1.
Patient Demographics and Baseline Characteristics
| Number Patients | Percent | |
|---|---|---|
| Total Patient Enrolled | 33* | 100 |
| Enrollment Time | 11/28/16 – 10/21/20 | |
| Age at Study Entry | ||
| >60 | 31 | 94 |
| Median (Range) | 73 (54 – 85) | |
| Gender | ||
| Female | 11 | 33 |
| Male | 22 | 67 |
| ECOG Performance Status at Study Entry | ||
| 0 | 8 | 24 |
| 1 | 24 | 73 |
| 2 | 1 | 3 |
| Diagnosis | ||
| MDS | 30 | 91 |
| CMML-1 | 2 | 6 |
| CMML-2 | 1 | 3 |
| IPSS-R Prognostic Score for MDS at Study Entry (n=30 MDS) | ||
| Intermediate (Risk Score > 3 and <=4.5) | 11 | 37 |
| High (Risk Score > 4.5 and <=6) | 17 | 57 |
| Very High (Risk Score >6) | 2 | 7 |
| Disease Status at Study Entry | ||
| Refractory Disease | 23 | 70 |
| Relapsed Disease | 10 | 30 |
| Baseline Bone Marrow Blasts (IHC) | ||
| ≤ 2.0% | 6 | 19 |
| 2.1% – 5.0% | 10 | 31 |
| >5% | 16 | 50 |
| Median (Range) | 5.5% (0% – 20%) | |
| Inadequate Specimen | 1 | |
| ASXL1 Mutation | ||
| Shared Mutation | 5 | 15 |
| Myeloid Mutation Only | 7 | 21 |
| No Mutation | 21 | 64 |
| Number of Prior Chemotherapy Regimens | ||
| 1 | 18 | 55 |
| 2 | 13 | 39 |
| 3 | 2 | 6 |
| Median (Range) | 1 (1 – 3) | |
| Received Prior Hypomethylating Agent (HMAs) | ||
| Yes | 33 | 100 |
| Both AZA and Dacogen (Sequentially) | 6 | 18 |
| Only AZA | 25 | 76 |
| Only Dacogen | 2 | 6 |
| Median (Range) Cycles of Prior AZA | 9 (3 – 61) | |
| Other Prior Treatment | ||
| Lenalidomide | 6 | 18 |
| Guadecitabine | 2 | 6 |
| Low-dose Cytarabine | 2 | 6 |
| 7+3 (ARA-C with Idarubicin) | 1 | 3 |
| Adrimaycin, Cytoxan, Taxotere | 1 | 3 |
| Cisplatin | 1 | 3 |
| Cyclophosphamide | 1 | 3 |
| Doxorubicin | 1 | 3 |
| Vincristine Sulfate | 1 | 3 |
| ASXL1 Mutation Status | ||
| Mutation | 12 | 36 |
| No Mutation | 15 | 45 |
| Not Available | 6 | 18 |
| RUNX1 Mutation Status | ||
| Mutation | 8 | 24 |
| No Mutation | 19 | 58 |
| Not Available | 6 | 18 |
| EZH2 Mutation Status | ||
| Mutation | 7 | 21 |
| No Mutation | 20 | 61 |
| Not Available | 6 | 18 |
| TET2 Mutation Status | ||
| Mutation | 6 | 18 |
| No Mutation | 21 | 64 |
| Not Available | 6 | 18 |
| DNMT3A Mutation Status | ||
| Mutation | 12 | 36 |
| No Mutation | 15 | 45 |
| Not Available | 6 | 18 |