Table 3.
Baseline characteristics and responses of the MDS cohort (N = 8)
| Patient | Age (years) | Disease subtype | Number of prior lines of therapy | Baseline bone marrow blasts | IPSS-R risk | Mutations | Best response | Duration of response (months) |
|---|---|---|---|---|---|---|---|---|
| #1 | 59 | MDS | 1 | 15% | Very high | TP53, BRINP3 | Not evaluable | N/A |
| #2 | 71 | MDS | 2 | 4% | High | ASXL1, NF1, RUNX1, SF3B1, PHF6 | Stable disease | 5.1 |
| #3 | 83 | CMML | 1 | 6% | Intermediate | SF3B1, SRSF2, ASXL1, RUNX1, SETBP1, PRPF40B | mCR | 4.4 |
| #4 | 74 | MDS | 1 | 3% | Very high | TP53 | CR | 14.8 (ongoing) |
| #5 | 49 | CMML | 2 | 11% | Very high | ASXL1, PTPN11, U2AF1 | mCR | 2.8 |
| #6 | 72 | MDS | 1 | 2% | High | EZH2, SF3B1, TET2, RAD21 | HI-P | 10.9 |
| #7 | 76 | MDS | 1 | 9% | High | TP53, ASXL2, ETV6, SETBP1, ZRSR2 | mCR + HI-E + HI-N | 8.0 |
| #8 | 78 | MDS | 3 | 14% | Very high | IDH2, SRSF2, FLT3-D835, RUNX1 | mCR + HI-N | 3.9 |
Abbreviations: MDS, myelodysplastic syndrome; CMML, chronic myelomonocytic leukemia; IPSS-R, Revised International Prognostic Scoring System; CR, complete response; mCR, marrow complete response; HI, hematologic improvement; HI-E, HI with erythroid response; HI-N, HI with neutrophil response; HI-P, HI with platelet response