Efficacy
ELN, deaths
DISEASE PROGRESSION
The best overall response was CR in 2 (3.9% [95% CI 0.5–13.5]) patients and CRi in 3 (5.9% [95% CI 1.2–16.2]) patients (see Supplementary Table 3 in the Online Resource), with an overall rate of 9.8% (95% CI 3.3–21.4). CR + CRi was achieved by 3 (6.0%) of the 50 patients in Cycle 1. Nine of the 50 patients receiving Cycle 1 proceeded to Cycle 2, of whom 3 (33.3%) patients achieved CR + CRi in Cycle 2. Among the 5 patients who achieved CR or CRi (see Supplementary Table 3 in the Online Resource), there were 2 and 3 patients stratified with ELN favorable or intermediate risk, respectively. One patient who achieved CR/CRi had previously received 2 HSCTs, 1 had previously been treated with 3 induction regimens, and the remaining patients had received 1 or 2 prior induction regimens.The median OS was 2.8 (95% CI 1.7–4.2) months, with 45 deaths reported (88.2%; see Supplementary Fig. 1 in the Online Resource). Of the 45 deaths, disease progression was the most common cause in 35 (77.8%) patients.