Table 1.
Patient demographics
| Cohort 1 | Cohort 2 | ||
|---|---|---|---|
| Characteristics | SAA | SAA | MDS |
| No. | 88 | 39 | 31 |
| Median age, years (range) | 33 (2–75) | 31 (6–82) | 60 (23–86) |
| Sex, male/female | 48/40 | 20/19 | 17/14 |
| Disease status | RCUD (7), RCMD (15) | ||
| RARS (4), RAEB-1 (5) | |||
| Treatment | |||
| IST | 41 | 15 | |
| IST+Eltrombopag | 47 | 24 | |
| Time analysis | |||
| Samples at diagnosis | 88 | 39 | 31 |
| Samples 6m after | 23 | 0 | 0 |
| treatment | |||
| Responses to treatment | |||
| Responders | 46 | 29 | |
| Complete response | 21 | 15 | |
| Partial response | 25 | 14 | |
| Non-responders | 37 | 10 | |
| Blood counts, median (range) | |||
| ANC (K/uL) | 0.43 (0.01–9.23) | 0.37 (0.01–3.15) | 1.18 (0.13–4.19) |
| HGB (g/dL) | 8.7 (5.1–13.7) | 8.2 (6.4–11.3) | 9.7 (4.9–12.7) |
| ARC (K/uL) | 24.95 (0–95.7) | 25.7 (5–78.7) | 59.8 (2.3–115.7) |
| PLT (K/uL) | 20 (3–229) | 25 (2–377) | 34 (9–240) |
Abbreviations: SAA, sever aplastic anemia; MDS, myelodysplastic anemia; IST, immunosuppressive therapy; 6m, 6 months; ANC, absolute neutrophil counts; HGB, hemoglobin; ARC, absolute reticulocyte counts; PLT, platelet. RCUD, refractory cytopenia with unilineage dysplasia; RCMD, Refractory cytopenia with multilineage dysplasia; RARS, Refractory anemia with ringed sideroblasts; RAEB-1, Refractory anemia with excess blasts-1.