Table 1.

Patient demographics and baseline characteristics

CharacteristicN = 32
Median age (range), years71 (19–84)
Sex, n (%)
 Female16 (50)
 Male16 (50)
Diagnosis, n (%)
 Relapsed/refractory30 (94)
 Newly diagnosed2 (6)
Ethnicity, n (%)
 White25 (78)
 Black4 (13)
 Asian3 (9)
ECOG performance score, n (%)a
 03 (9)
 114 (44)
 214 (44)
 Missing1 (3)
Any prior therapy, n (%)30 (94)
 Prior regimens ≥313 (41)
 Prior standard induction (3+7) therapy17 (53)
 Prior hypomethylating agents24 (75)
 Prior allogeneic stem cell transplant4 (13)
 Treatment naïve2 (6)
Prior myeloid disorder, n (%)
 Prior myelodysplastic syndromeb11 (35)
 Prior myeloproliferative neoplasm2 (6)
Molecular markersc, n (%)
IDH mutationsd12 (38)
FLT3-ITDe4 (13)
 BCR–ABL1 (3)
 JAK21 (3)
 KRAS1 (3)
 MLL1 (3)
 NPM14 (13)
 CEBPα2 (6)
Cytogenetics, n (%)
 del(7q)10 (31)
 Complex10 (31)
 None2 (6)
  • Abbreviation: ECOG, Eastern Cooperative Oncology Group.

  • aPercentage based on known values.

  • bIncludes n = 4 with therapy-related myelodysplastic syndrome with transformation to AML.

  • cCytogenetics were evaluated for all patients at the investigator sites. Not all patients had molecular marker analysis; 8 of 32 did not have IDH mutational analysis performed at the sites. Data included patients with expression of more than one marker.

  • dTwo were IDH1 mutations and 10 were IDH2 mutations. Of the 12, 11 were in a known site that leads to (R)-2-hydroxyglutarate, and 1 mutation was not in the putative hotspot (exon 3; D76 frameshift).

  • eThree with FLT3-ITD and a concomitant IDH mutation were confirmed at study entry, and one site reported FLT3-ITD mutation was not detected at study entry (assay sensitivity was 0.1%).