Demographics and patient characteristics for phase 1 dose-finding cohort and expansion cohort
| Characteristics . | Phase 1 dose-finding cohort Number/median (range) . | Expansion cohort Number/median (range) . |
|---|---|---|
| No. of patients | 12 | 18 |
| Age, y | 7 (3-21) | 7 (2-14) |
| Sex, M/F | 7/5 | 7/11 |
| Race | ||
| White | 12 | 17 |
| Asian | - | 1 |
| Ethnicity | ||
| Non-Hispanic | 12 | 17 |
| Hispanic | - | 1 |
| FA genotype | ||
| FANCA | 10 | 10 |
| FANCC | 1 | 4 |
| FANCD1 | 1 | 1 |
| FANCG | - | 2 |
| FANCI | - | 1 |
| Marrow failure status | ||
| None | 3 | 4 |
| Mild | 4 | 8 |
| Moderate | 3 | 4 |
| Severe | 2 | 2 |
| Characteristics . | Phase 1 dose-finding cohort Number/median (range) . | Expansion cohort Number/median (range) . |
|---|---|---|
| No. of patients | 12 | 18 |
| Age, y | 7 (3-21) | 7 (2-14) |
| Sex, M/F | 7/5 | 7/11 |
| Race | ||
| White | 12 | 17 |
| Asian | - | 1 |
| Ethnicity | ||
| Non-Hispanic | 12 | 17 |
| Hispanic | - | 1 |
| FA genotype | ||
| FANCA | 10 | 10 |
| FANCC | 1 | 4 |
| FANCD1 | 1 | 1 |
| FANCG | - | 2 |
| FANCI | - | 1 |
| Marrow failure status | ||
| None | 3 | 4 |
| Mild | 4 | 8 |
| Moderate | 3 | 4 |
| Severe | 2 | 2 |
Marrow failure status was defined using standard clinical criteria. Mild: ANC <1500/μL, platelet count <150 to 50x109/L, or Hb ≥8 to <11 g/dL. Moderate: ANC <1000/μL, platelet count <50x109/L, or Hb <8 g/dL. Severe: ANC <500/μL, platelet count <30x109/L, or Hb <8 g/dL.
Hb, hemoglobin; M/F, male/female.