Table 2.
Disease characteristics, responses, and outcomes of all patients.
| Dosing cohort/accrual number | Prior treatment/CR1 duration | Salvage status | Cytogenetics | Mutations | Dose limiting toxicities | Response | Duration of response | Reason for withdrawal | Comments |
|---|---|---|---|---|---|---|---|---|---|
| “0”/#1 | IA + transplant/12 yrs | 1 | Diploid | None | Two G3 | CRi | 1.5 mo | death | Course 2 delayed due to myelosuppression |
| “0”/#2 | 7 + 3/1.5 yrs | 1 | t(8, 21) | JAK2 | None | CR, MRD+ | 3.5 mo+ | ASCT | Died post-transplant from MI |
| “0”/#3 | 7 + 3/0.5 yrs | 1 | Diploid | IDH2 | None | NR | – | NR | Transitioned to IDH inhibitor |
| “0”/#4 | 7 + 3, FIA + ASCT + azacitidine/0.25 yrs | 2 | Complex | DNMT3A | None | NR | – | NR | No response to reinduction. Died of progressive disease a month later |
| “0”/#5 | CIA, FLAG-IDA + ASCT, decitabine/0.75 yrs | 3 | del(9q) | ND | None | NR | – | NR | Died of sepsis 3 months post study withdrawal |
| “0”/#6 | FIA/3 yrs | 1 | Diploid | RUNX1 | None | CRi | 1 mo+ | ASCT | 10% blasts after first cycle. On re-induction, developed MRD- Cri |
| “1”/#7 | Azacitidine/0.25 yrs | 1 | Diploid | IDH1, NPM1 | None | CR | 6 mo | loss of response | Relapsed at the end of 6 consolidation cycles |
| “1”/#8 | 7 + 3/0.3 yrs | 1 | Trisomy11 | NRAS | None | NR | – | NR | Transitioned to subsequent salvage chemotherapy and bridged to transplant |
| “1”/#9 | CLAD/LDAC/0.3 yrs | 1 | Diploid | ND | None | NR | – | NR | Transitioned to subsequent salvage chemotherapy |
| “1”/#10 | Azacitidine/refractory | 1 | Diploid | TET2,RUNX1, SF3B1 | None | NR | – | NR | Found to have lymphoma. |
| “1”/#11 | DAC, 7+3, DAC-venetoclax/refractory | 4 | Complex | TP53 | None | NR | – | NR | Failed other therapies. Transitioned to comfort care |
ASCT, allogeneic stem cell transplant; CIA, clofarabine, idarubicin, and cytarabine; CLAD/LDAC, cladribine, low dose cytarabine; CR, complete remission; Cri, CR with incomplete blood count recovery; DAC, decitabine; FLAG-IDA, fludarabine, idarubicin, cytarabine, G-CSF; FIA, fludarabine, idarubicin, and cytarabine;IA, idarubicin, high dose cytarabine; G- grade; MRD, minimal residual disease; NR, no response; MI, myocardial infarction; mo– months; mo+: in remission and alive; yrs, years; 7 + 3: idarubicin and intermediate dose cytarabine.