Clinical and genetic information about CMML patients enrolled in the KB003 clinical trial
| Patient . | Age, y . | Sex . | CMML Classification . | Cytogenetics . | Molecular abnormalities . | Prior therapies . | Maximum KB003 dose, mg* . | Cycles given, n . | Best response . | Duration of best response, mo . | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| WHO . | FAB . | ||||||||||
| 1 | 73 | M | CMML-1 | Proliferative | 45,X,-Y | ASXL1, CBL, IDH2, SRSF2 | Hydroxyurea | 200 | 4 | None | — |
| 2 | 77 | M | CMML-0 | Proliferative | 46,XY,t(3;12;6)(p21;q21;q23)[8]/46,XY[12] | ASXL1, EZH2, TET2 | Hydroxyurea | 200 | 2 | None | — |
| 3 | 52 | M | CMML-0 | Dysplastic | 46,XY | SRSF2, TET2 | None | 600 (initially enrolled in 200-mg cohort) | 25 | Clinical benefit (platelet response) | 24 |
| 4 | 80 | M | CMML-0 | Proliferative | 46,XY | SRSF2, TET2 | Darbepoietin alfa, investigational agent (clinical trial), decitabine, investigational agent (clinical trial) | 400 | 6 | Stable disease | 6 |
| 5 | 74 | F | CMML-0 | Proliferative | 46,XX,del(20)(q11.2q13.1) [17]/46,XX [3] | ASXL1, NRAS, TET2 | Azacitidine, lenalidomide, hydroxyurea, decitabine, investigational agent (clinical trial) | 600 (initially enrolled in 400-mg cohort) | 29 | Stable disease | 26 |
| 6 | 68 | M | CMML-0 | Dysplastic | 46,XY | ASXL1, BCOR, EZH2, SF3B1 | Azacitidine | 600 | 2 | None | — |
| 7 | 66 | M | CMML-0 | Proliferative | 46,XY | ASXL1, NRAS, SRSF2, TET2 | Investigational agent (clinical trial) | 600 | 9 | Clinical benefit (platelet response) | 7 |
| 8 | 71 | M | CMML-0 | Proliferative | 45,X,-Y | ASXL1, NRAS, RUNX1 | None | 600 | 11 | Clinical benefit (platelet response) | 10 |
| 9 | 79 | M | CMML-2 | Proliferative | 46,XY | Not available | Hydroxyurea | 600 | 1 | None | — |
| 10 | 62 | M | CMML-2 | Dysplastic | 46,XY | CBL, TET2 | Decitabine | 600 | 1 | None | — |
| 11 | 68 | M | CMML-1 | Proliferative | 46,XY | ASXL1, NRAS, SRSF2, TET2 | None | 400 | 4 | Partial marrow response | 3 |
| 12 | 85 | M | CMML-0 | Dysplastic | 46,XY | TET2, SRSF2 | Decitabine | 600 | 4 | Stable disease | 3 |
| 13 | 76 | M | CMML-2 | Proliferative | 45,-7,XY | ASXL1, SRSF2 | Azacitidine, decitabine | 600 | 2 | Stable disease | 1 |
| 14 | 77 | M | CMML-1 | Dysplastic | 46, XX | TET2, EZH2, SETBP1, RUNX1 | Azacitidine | 600 | 4 | Clinical benefit (neutrophil response) | 6 |
| 15 | 71 | F | CMML-0 | Proliferative | 46, XX | TET2, SF3B1, JAK2 | Ruxolitinib, decitabine, azacitidine | 600 | 14 | Clinical benefit (spleen response) | 14 |
| Patient . | Age, y . | Sex . | CMML Classification . | Cytogenetics . | Molecular abnormalities . | Prior therapies . | Maximum KB003 dose, mg* . | Cycles given, n . | Best response . | Duration of best response, mo . | |
|---|---|---|---|---|---|---|---|---|---|---|---|
| WHO . | FAB . | ||||||||||
| 1 | 73 | M | CMML-1 | Proliferative | 45,X,-Y | ASXL1, CBL, IDH2, SRSF2 | Hydroxyurea | 200 | 4 | None | — |
| 2 | 77 | M | CMML-0 | Proliferative | 46,XY,t(3;12;6)(p21;q21;q23)[8]/46,XY[12] | ASXL1, EZH2, TET2 | Hydroxyurea | 200 | 2 | None | — |
| 3 | 52 | M | CMML-0 | Dysplastic | 46,XY | SRSF2, TET2 | None | 600 (initially enrolled in 200-mg cohort) | 25 | Clinical benefit (platelet response) | 24 |
| 4 | 80 | M | CMML-0 | Proliferative | 46,XY | SRSF2, TET2 | Darbepoietin alfa, investigational agent (clinical trial), decitabine, investigational agent (clinical trial) | 400 | 6 | Stable disease | 6 |
| 5 | 74 | F | CMML-0 | Proliferative | 46,XX,del(20)(q11.2q13.1) [17]/46,XX [3] | ASXL1, NRAS, TET2 | Azacitidine, lenalidomide, hydroxyurea, decitabine, investigational agent (clinical trial) | 600 (initially enrolled in 400-mg cohort) | 29 | Stable disease | 26 |
| 6 | 68 | M | CMML-0 | Dysplastic | 46,XY | ASXL1, BCOR, EZH2, SF3B1 | Azacitidine | 600 | 2 | None | — |
| 7 | 66 | M | CMML-0 | Proliferative | 46,XY | ASXL1, NRAS, SRSF2, TET2 | Investigational agent (clinical trial) | 600 | 9 | Clinical benefit (platelet response) | 7 |
| 8 | 71 | M | CMML-0 | Proliferative | 45,X,-Y | ASXL1, NRAS, RUNX1 | None | 600 | 11 | Clinical benefit (platelet response) | 10 |
| 9 | 79 | M | CMML-2 | Proliferative | 46,XY | Not available | Hydroxyurea | 600 | 1 | None | — |
| 10 | 62 | M | CMML-2 | Dysplastic | 46,XY | CBL, TET2 | Decitabine | 600 | 1 | None | — |
| 11 | 68 | M | CMML-1 | Proliferative | 46,XY | ASXL1, NRAS, SRSF2, TET2 | None | 400 | 4 | Partial marrow response | 3 |
| 12 | 85 | M | CMML-0 | Dysplastic | 46,XY | TET2, SRSF2 | Decitabine | 600 | 4 | Stable disease | 3 |
| 13 | 76 | M | CMML-2 | Proliferative | 45,-7,XY | ASXL1, SRSF2 | Azacitidine, decitabine | 600 | 2 | Stable disease | 1 |
| 14 | 77 | M | CMML-1 | Dysplastic | 46, XX | TET2, EZH2, SETBP1, RUNX1 | Azacitidine | 600 | 4 | Clinical benefit (neutrophil response) | 6 |
| 15 | 71 | F | CMML-0 | Proliferative | 46, XX | TET2, SF3B1, JAK2 | Ruxolitinib, decitabine, azacitidine | 600 | 14 | Clinical benefit (spleen response) | 14 |
F, female; FAB, French-American-British classification; M, male.
Given every 28 days.