Patient characteristics
Characteristic . | All patients (N = 54) . | Feasibility cohort (n = 21) . | ||||||
---|---|---|---|---|---|---|---|---|
No. . | % . | Median . | Range . | No. . | % . | Median . | Range . | |
Age, y | 75 | 23-90 | 76 | 55-87 | ||||
Sex | ||||||||
Male | 41 | 76 | 15 | 71 | ||||
Female | 13 | 24 | 6 | 29 | ||||
Diagnosis | ||||||||
AML | 10 | 19 | 1 | 5 | ||||
De novo AML | 4 | 7 | 0 | 0 | ||||
Secondary AML* | 6 | 11 | 1 | 5 | ||||
MDS | 33 | 61 | 17 | 81 | ||||
Lower-risk MDS (IPSS-R <4) | 12 | 22 | 5 | 24 | ||||
Higher-risk MDS (IPSS-R ≥4) | 21 | 39 | 12 | 57 | ||||
MDS/MPN | 11 | 20 | 3 | 14 | ||||
CMML | 4 | 7 | 1 | 5 | ||||
Other MDS/MPN | 7 | 13 | 2 | 9 | ||||
Prognostic factor | ||||||||
Adverse cytogenetics or mutations† | 28 | 52 | 11 | 52 | ||||
Bone marrow blast percent | 8 | 1-90 | 5 | 1-31 | ||||
No. of mutations‡ | 2 | 0-6 | 2 | 0-6 | ||||
Frequent mutations | ||||||||
TET2 | 12 | 22 | 5 | 24 | ||||
ASXL1 | 11 | 20 | 5 | 24 | ||||
DNMT3A | 10 | 19 | 4 | 19 | ||||
STAG2 | 9 | 17 | 5 | 24 | ||||
SF3B1 | 9 | 17 | 2 | 10 | ||||
RUNX1 | 8 | 15 | 4 | 19 | ||||
BCOR | 8 | 15 | 2 | 10 | ||||
NRAS | 8 | 15 | 1 | 5 | ||||
SRSF2 | 7 | 13 | 4 | 19 | ||||
KRAS | 7 | 13 | 2 | 10 | ||||
TP53 | 6 | 11 | 1 | 5 | ||||
EZH2 | 5 | 9 | 2 | 10 | ||||
CEBPA | 5 | 9 | 2 | 10 | ||||
U2AF1 | 4 | 7 | 2 | 10 | ||||
JAK2 V617F | 3 | 6 | 1 | 5 | ||||
FLT3-ITD | 3 | 6 | 0 | 0 | ||||
Previous HMA therapy | ||||||||
Yes | 32 | 59 | 21 | 100 | ||||
No | 22 | 41 | 0 | 0 |
Characteristic . | All patients (N = 54) . | Feasibility cohort (n = 21) . | ||||||
---|---|---|---|---|---|---|---|---|
No. . | % . | Median . | Range . | No. . | % . | Median . | Range . | |
Age, y | 75 | 23-90 | 76 | 55-87 | ||||
Sex | ||||||||
Male | 41 | 76 | 15 | 71 | ||||
Female | 13 | 24 | 6 | 29 | ||||
Diagnosis | ||||||||
AML | 10 | 19 | 1 | 5 | ||||
De novo AML | 4 | 7 | 0 | 0 | ||||
Secondary AML* | 6 | 11 | 1 | 5 | ||||
MDS | 33 | 61 | 17 | 81 | ||||
Lower-risk MDS (IPSS-R <4) | 12 | 22 | 5 | 24 | ||||
Higher-risk MDS (IPSS-R ≥4) | 21 | 39 | 12 | 57 | ||||
MDS/MPN | 11 | 20 | 3 | 14 | ||||
CMML | 4 | 7 | 1 | 5 | ||||
Other MDS/MPN | 7 | 13 | 2 | 9 | ||||
Prognostic factor | ||||||||
Adverse cytogenetics or mutations† | 28 | 52 | 11 | 52 | ||||
Bone marrow blast percent | 8 | 1-90 | 5 | 1-31 | ||||
No. of mutations‡ | 2 | 0-6 | 2 | 0-6 | ||||
Frequent mutations | ||||||||
TET2 | 12 | 22 | 5 | 24 | ||||
ASXL1 | 11 | 20 | 5 | 24 | ||||
DNMT3A | 10 | 19 | 4 | 19 | ||||
STAG2 | 9 | 17 | 5 | 24 | ||||
SF3B1 | 9 | 17 | 2 | 10 | ||||
RUNX1 | 8 | 15 | 4 | 19 | ||||
BCOR | 8 | 15 | 2 | 10 | ||||
NRAS | 8 | 15 | 1 | 5 | ||||
SRSF2 | 7 | 13 | 4 | 19 | ||||
KRAS | 7 | 13 | 2 | 10 | ||||
TP53 | 6 | 11 | 1 | 5 | ||||
EZH2 | 5 | 9 | 2 | 10 | ||||
CEBPA | 5 | 9 | 2 | 10 | ||||
U2AF1 | 4 | 7 | 2 | 10 | ||||
JAK2 V617F | 3 | 6 | 1 | 5 | ||||
FLT3-ITD | 3 | 6 | 0 | 0 | ||||
Previous HMA therapy | ||||||||
Yes | 32 | 59 | 21 | 100 | ||||
No | 22 | 41 | 0 | 0 |
CMML, chronic myelomonocytic leukemia.
Includes patients with an antecedent myeloid neoplasm or therapy-related AML.
For MDS and MDS/MPNs, adverse cytogenetics were defined by IPSS-R and adverse mutations included TP53, EZH2, ETV6, RUNX1, and ASXL1; for AML, adverse cytogenetics and mutations were defined by the 2017 European LeukemiaNet criteria.
Includes pathogenic and likely pathogenic mutations and excludes variants of unknown significance.