Baseline information for each treated off-trial patient and an explanation for why each was not enrolled in a venetoclax clinical trial
| Pt . | Age, y . | Prior HMA . | AHD . | Cytogenetic risk group . | Baseline mutational profile . | Best response . | Predicted CR% based on AML SCORE . | Death within 60 d . | Predicted early death % based on AML SCORE13 . | Reason not enrolled in a clinical trial . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 75 | No | Yes | Intermediate | IDH2, SRSF2, TET2 | CRi | 54.8 | No | 19.6 | Study unavailable |
| 2 | 72 | No | No | Intermediate | NPM1, TET2, GATA2 | CR | 73.8 | No | 26.1 | Study unavailable |
| 3 | 82 | No | No | Unfavorable | None | Resistant disease | 13.1 | No | 55.9 | Other malignancy |
| 4 | 59 | No | No | Intermediate | FLT3 TKD, NPM1 | CR | 70.5 | No | 14 | Age <65 y |
| 5 | 85 | No | No | Unknown | IDH2, NRAS, SRSF2, SMC1A | CR | 41.7 | No | 34.2 | Renal insufficiency |
| 6 | 68 | No | No | Unfavorable | ASXL1, RUNX1, TET2 | Resistant disease | 20.7 | No | 38.1 | Other malignancy |
| 7 | 73 | No | No | Intermediate | RUNX1, PHF6 | CR | 58.9 | No | 26.1 | Study unavailable |
| 8 | 79 | No | Yes | Unfavorable | JAK2, TP53, BCORL1 | CR | 21.2 | No | 39 | Evolved from MPN |
| 9 | 79 | No | No | Intermediate | IDH2, ASXL1, RUNX1, SRSF2, FLT3, RAD21, PHF6 | CR | 48.7 | No | 26.9 | Study unavailable |
| 10 | 70 | No | No | Intermediate | NPM1, FLT3 TKD, DNMT3A | CR | 58.6 | No | 17.4 | Study unavailable |
| 11 | 63 | No | No | Unfavorable | ASXL1, NRAS, TET2 | Resistant disease | 38 | Yes | 25.1 | Chronic respiratory disease requiring continuous oxygen use |
| 12 | 61 | No | No | Intermediate | ASXL1, SRSF2, STAG2, ZRSR2, RUNX1 | CRi | 78.5 | No | 8.9 | Active hepatitis C |
| 13 | 75 | No | No | Unfavorable | ASXL1, DNMT3A, RUNX1, U2AF1, BCORL1, PHF6, CBL, TET2 | Resistant disease | 30.5 | Yes | 39 | Study unavailable |
| 14 | 66 | No | No | Intermediate | TP53, CEBPA, ASXL1, SRSF2, STAG2 | Uninterpretable | 57.9 | No | 16.8 | Other malignancy |
| 15 | 68 | No | No | Intermediate | NPM1, DNMT3A, FLT3 ITD, IDH2, RUNX1 | CRi | 37 | No | 47.1 | Chronic respiratory disease requiring continuous oxygen use |
| 16 | 48 | No | No | Intermediate | NPM1, NRAS, SMC1A | N/A | N/A | Yes | N/A | Age <65 y |
| 17 | 73 | No | No | Intermediate | ASXL1, RUNX1, WT1, CEBPA, EZH2, TET2 | CR | 52.6 | No | 34.2 | Unable to provide informed consent |
| 18 | 68 | No | No | Intermediate | FTL3 ITD, DNMT3A, NPM1, | CRi | 51.7 | No | 33.1 | Unable to provide informed consent |
| 19 | 72 | No | No | Intermediate | NPM1, FLT3-ITD, DNMT3A | CR | 51.7 | No | 33.1 | Study unavailable |
| 20 | 83 | No | No | Unfavorable | TET2, PTPN11 | CR | 71.5 | No | 30.4 | Core-binding factor |
| 21 | 81 | No | No | Intermediate | FLT3 ITD, CEBPA, TET2, NPM1 | CRi | 59.6 | No | 26.9 | Logistics of travel |
| 22 | 72 | No | No | Intermediate | ASXL1, SETBP1, PHF6, MLL | MLFS | 58.9 | No | 26.1 | Active infection |
| 23 | 68 | No | No | Unfavorable | TP53 | CRi | 30.5 | No | 39 | Logistics of travel |
| 24 | 79 | No | No | Unfavorable | DNMT3A, IDH1 | CR | 16.8 | No | 47.5 | Other malignancy |
| 25 | 74 | No | No | Unfavorable | None | CR | 71.5 | No | 30.4 | Core-binding factor |
| 26 | 33 | No | No | Unfavorable | ASXL1 | CR | N/A | No | 19.6 | Age <65 y |
| 27 | 78 | Yes | Yes | Unknown | SRSF2, STAG2, CEBPA, ETV6 | Resistant disease | 30.4 | No | 34.2 | Prior hypomethylator |
| 28 | 71 | Yes | Yes | Unknown | ASXL1, SRSF2, STAG2, TET2 | MLFS | 36.1 | No | 26.1 | Prior hypomethylator |
| 29 | 68 | Yes | Yes | Unknown | JAK2, TP53, ASXL1, EZH2, KDM6A | N/A | 40.5 | Yes | 22.6 | Prior hypomethylator |
| 30 | 71 | Yes | Yes | Unfavorable | TP53, EZH2 | MLFS | 23.1 | No | 34.1 | Prior hypomethylator |
| Pt . | Age, y . | Prior HMA . | AHD . | Cytogenetic risk group . | Baseline mutational profile . | Best response . | Predicted CR% based on AML SCORE . | Death within 60 d . | Predicted early death % based on AML SCORE13 . | Reason not enrolled in a clinical trial . |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | 75 | No | Yes | Intermediate | IDH2, SRSF2, TET2 | CRi | 54.8 | No | 19.6 | Study unavailable |
| 2 | 72 | No | No | Intermediate | NPM1, TET2, GATA2 | CR | 73.8 | No | 26.1 | Study unavailable |
| 3 | 82 | No | No | Unfavorable | None | Resistant disease | 13.1 | No | 55.9 | Other malignancy |
| 4 | 59 | No | No | Intermediate | FLT3 TKD, NPM1 | CR | 70.5 | No | 14 | Age <65 y |
| 5 | 85 | No | No | Unknown | IDH2, NRAS, SRSF2, SMC1A | CR | 41.7 | No | 34.2 | Renal insufficiency |
| 6 | 68 | No | No | Unfavorable | ASXL1, RUNX1, TET2 | Resistant disease | 20.7 | No | 38.1 | Other malignancy |
| 7 | 73 | No | No | Intermediate | RUNX1, PHF6 | CR | 58.9 | No | 26.1 | Study unavailable |
| 8 | 79 | No | Yes | Unfavorable | JAK2, TP53, BCORL1 | CR | 21.2 | No | 39 | Evolved from MPN |
| 9 | 79 | No | No | Intermediate | IDH2, ASXL1, RUNX1, SRSF2, FLT3, RAD21, PHF6 | CR | 48.7 | No | 26.9 | Study unavailable |
| 10 | 70 | No | No | Intermediate | NPM1, FLT3 TKD, DNMT3A | CR | 58.6 | No | 17.4 | Study unavailable |
| 11 | 63 | No | No | Unfavorable | ASXL1, NRAS, TET2 | Resistant disease | 38 | Yes | 25.1 | Chronic respiratory disease requiring continuous oxygen use |
| 12 | 61 | No | No | Intermediate | ASXL1, SRSF2, STAG2, ZRSR2, RUNX1 | CRi | 78.5 | No | 8.9 | Active hepatitis C |
| 13 | 75 | No | No | Unfavorable | ASXL1, DNMT3A, RUNX1, U2AF1, BCORL1, PHF6, CBL, TET2 | Resistant disease | 30.5 | Yes | 39 | Study unavailable |
| 14 | 66 | No | No | Intermediate | TP53, CEBPA, ASXL1, SRSF2, STAG2 | Uninterpretable | 57.9 | No | 16.8 | Other malignancy |
| 15 | 68 | No | No | Intermediate | NPM1, DNMT3A, FLT3 ITD, IDH2, RUNX1 | CRi | 37 | No | 47.1 | Chronic respiratory disease requiring continuous oxygen use |
| 16 | 48 | No | No | Intermediate | NPM1, NRAS, SMC1A | N/A | N/A | Yes | N/A | Age <65 y |
| 17 | 73 | No | No | Intermediate | ASXL1, RUNX1, WT1, CEBPA, EZH2, TET2 | CR | 52.6 | No | 34.2 | Unable to provide informed consent |
| 18 | 68 | No | No | Intermediate | FTL3 ITD, DNMT3A, NPM1, | CRi | 51.7 | No | 33.1 | Unable to provide informed consent |
| 19 | 72 | No | No | Intermediate | NPM1, FLT3-ITD, DNMT3A | CR | 51.7 | No | 33.1 | Study unavailable |
| 20 | 83 | No | No | Unfavorable | TET2, PTPN11 | CR | 71.5 | No | 30.4 | Core-binding factor |
| 21 | 81 | No | No | Intermediate | FLT3 ITD, CEBPA, TET2, NPM1 | CRi | 59.6 | No | 26.9 | Logistics of travel |
| 22 | 72 | No | No | Intermediate | ASXL1, SETBP1, PHF6, MLL | MLFS | 58.9 | No | 26.1 | Active infection |
| 23 | 68 | No | No | Unfavorable | TP53 | CRi | 30.5 | No | 39 | Logistics of travel |
| 24 | 79 | No | No | Unfavorable | DNMT3A, IDH1 | CR | 16.8 | No | 47.5 | Other malignancy |
| 25 | 74 | No | No | Unfavorable | None | CR | 71.5 | No | 30.4 | Core-binding factor |
| 26 | 33 | No | No | Unfavorable | ASXL1 | CR | N/A | No | 19.6 | Age <65 y |
| 27 | 78 | Yes | Yes | Unknown | SRSF2, STAG2, CEBPA, ETV6 | Resistant disease | 30.4 | No | 34.2 | Prior hypomethylator |
| 28 | 71 | Yes | Yes | Unknown | ASXL1, SRSF2, STAG2, TET2 | MLFS | 36.1 | No | 26.1 | Prior hypomethylator |
| 29 | 68 | Yes | Yes | Unknown | JAK2, TP53, ASXL1, EZH2, KDM6A | N/A | 40.5 | Yes | 22.6 | Prior hypomethylator |
| 30 | 71 | Yes | Yes | Unfavorable | TP53, EZH2 | MLFS | 23.1 | No | 34.1 | Prior hypomethylator |
AHD, antecedent hematological disorder; MPN, myeloproliferative neoplasm; PHA, prior hypomethylating agent; Pt, patient.