Characteristics and clinical outcomes of patients treated on the phase 1 clinical trial
Patient . | Age, y . | Sex . | Diagnosis . | Disease characteristics . | Disease status at SCT . | MRD . | Relapse risk . | Donor . | Engraftment . | Time to ANC 500 . | Chimerism at day 28 . | Grade 2-4 aGVHD . | Grade 3-4 aGVHD . | Systemic steroids . | cGVHD . | Outcome of SCT . | Last follow-up . |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | 40 | M | CML | Refractory to TKIs | CP1 | − | Int | Son | Yes | 20 | Mixed | No | No | No | No | Died (TRM) | Day +85 |
2 | 52 | F | AML | FLT3+ | Relapse pretransplant | + | High | Son | Yes | 17 | Donor | Yes | No | Yes | No | Relapsed | Day +582 |
3 | 60 | M | CML, MDS | Monosomy 7 | CP1 | − | High | Daughter | Yes | 27 | Donor | Yes | No | No | No | Remission | Day +755 |
4 | 38 | F | AML | PIF, cx. cyto, MLL+ | CR1 | + | High | Sister | Yes | 16 | Donor | No | No | No | No | Remission | Day +410 |
5 | 43 | M | AML | PIF, diploid cyto | CR1 | − | High | Brother | Yes | 18 | Donor | Yes | No | Yes | No | Remission | Day +419 |
6 | 34 | M | CML | BP CNS+ | CP2 | − | High | Sister | Yes | 21 | Donor | No | No | No | No | Remission | Day +481 |
7 | 23 | M | AML | FLT3+ | CR1 | − | High | Sister | Yes | 15 | Donor | No | No | No | No | Remission | Day +417 |
8 | 18 | F | AML | ASXL1+ | CR1 | + | High | Father | Yes | 21 | Donor | Yes | No | Yes | No | Remission | Day +316 |
9 | 58 | F | AML | MLL+ | CR1 | − | High | Daughter | Yes | 19 | Donor | Yes | No | No | No | Remission | Day +412 |
10 | 45 | F | CML | BP CNS+ | CP2 | − | High | Brother | Yes | 19 | Donor | Yes | No | Yes | No | Remission | Day +477 |
11 | 55 | M | AML | MLL+ | CR1 | + | High | Son | Yes | 14 | Donor | No | No | No | No | Remission | Day +448 |
12 | 25 | F | AML | PIF, diploid cyto | CR1 | − | High | Sister | Yes | 17 | Donor | Yes | No | Yes | No | Remission | Day +363 |
13 | 48 | F | CML | Refractory to TKIs | CP1 | + | Int | Daughter | Yes | 19 | Donor | No | No | No | No | Remission | Day +236 |
Patient . | Age, y . | Sex . | Diagnosis . | Disease characteristics . | Disease status at SCT . | MRD . | Relapse risk . | Donor . | Engraftment . | Time to ANC 500 . | Chimerism at day 28 . | Grade 2-4 aGVHD . | Grade 3-4 aGVHD . | Systemic steroids . | cGVHD . | Outcome of SCT . | Last follow-up . |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | 40 | M | CML | Refractory to TKIs | CP1 | − | Int | Son | Yes | 20 | Mixed | No | No | No | No | Died (TRM) | Day +85 |
2 | 52 | F | AML | FLT3+ | Relapse pretransplant | + | High | Son | Yes | 17 | Donor | Yes | No | Yes | No | Relapsed | Day +582 |
3 | 60 | M | CML, MDS | Monosomy 7 | CP1 | − | High | Daughter | Yes | 27 | Donor | Yes | No | No | No | Remission | Day +755 |
4 | 38 | F | AML | PIF, cx. cyto, MLL+ | CR1 | + | High | Sister | Yes | 16 | Donor | No | No | No | No | Remission | Day +410 |
5 | 43 | M | AML | PIF, diploid cyto | CR1 | − | High | Brother | Yes | 18 | Donor | Yes | No | Yes | No | Remission | Day +419 |
6 | 34 | M | CML | BP CNS+ | CP2 | − | High | Sister | Yes | 21 | Donor | No | No | No | No | Remission | Day +481 |
7 | 23 | M | AML | FLT3+ | CR1 | − | High | Sister | Yes | 15 | Donor | No | No | No | No | Remission | Day +417 |
8 | 18 | F | AML | ASXL1+ | CR1 | + | High | Father | Yes | 21 | Donor | Yes | No | Yes | No | Remission | Day +316 |
9 | 58 | F | AML | MLL+ | CR1 | − | High | Daughter | Yes | 19 | Donor | Yes | No | No | No | Remission | Day +412 |
10 | 45 | F | CML | BP CNS+ | CP2 | − | High | Brother | Yes | 19 | Donor | Yes | No | Yes | No | Remission | Day +477 |
11 | 55 | M | AML | MLL+ | CR1 | + | High | Son | Yes | 14 | Donor | No | No | No | No | Remission | Day +448 |
12 | 25 | F | AML | PIF, diploid cyto | CR1 | − | High | Sister | Yes | 17 | Donor | Yes | No | Yes | No | Remission | Day +363 |
13 | 48 | F | CML | Refractory to TKIs | CP1 | + | Int | Daughter | Yes | 19 | Donor | No | No | No | No | Remission | Day +236 |
AML, acute myeloid leukemia; ANC, absolute neutrophil count; BP, blast phase; CML, chronic myeloid leukemia; CNS, central nervous system; CP1, chronic phase 1; CP2, chronic phase 2; CR1, first complete remission; CR2, second complete remission; cx., complex; cyto, complex cytogenetics; F, female; FLT3, FMS-like tyrosine kinase 3; GVHD, graft-versus-host disease; Int, intermediate; M, male; MDS, myelodysplastic syndrome; MLL, mixed lineage leukemia gene; MRD, minimal residual disease; PIF, primary induction failure; SCT, stem cell transplant; TKI, tyrosine kinase inhibitor.