Table 1. Characteristics of the Patients at Baseline*.
| Characteristic | Cohort 1 (N = 30) | Cohort 2 (N = 31) | Cohort 3 (N = 31) | All Patients (N = 92) |
|---|---|---|---|---|
| Age — yr | ||||
| Median | 39 | 28 | 29 | 32 |
| Range | 12–72 | 3–68 | 11–82 | 3–82 |
| Age distribution — no. (%) | ||||
| <18 yr | 5 (17) | 6 (19) | 8 (26) | 19 (21) |
| 18–64 yr | 20 (67) | 23 (74) | 18 (58) | 61 (66) |
| ≥65 yr | 5 (17) | 2 (6) | 5 (16) | 12 (13) |
| Sex — no. (%) | ||||
| Male | 16 (53) | 17 (55) | 17 (55) | 50 (54) |
| Female | 14 (47) | 14 (45) | 14 (45) | 42 (46) |
| GPI-deficient neutrophils† | ||||
| Range — % | <1–99 | <1–99 | <1–79 | <1–99 |
| Distribution — no./total no. (%) | ||||
| <1% | 20/28 (71) | 18/30 (60) | 15/26 (58) | 53/84 (63) |
| ≥1% | 8/28 (29) | 12/30 (40) | 11/26 (42) | 31/84 (37) |
| Neutrophil count — per cubic millimeter | ||||
| Median | 275 | 330 | 300 | 310 |
| Range | 0–1380 | 0–900 | 0–1810 | 0–1810 |
| Reticulocyte count — per cubic millimeter | ||||
| Median | 22,550 | 14,600 | 24,300 | 19,950 |
| Range | 2400–51,600 | 1600–52,900 | 2000–60,400 | 1600–60,400 |
| Platelet count — per cubic millimeter | ||||
| Median | 9500 | 8000 | 8000 | 9000 |
| Range | 2000–37,000 | 1000–14,000 | 0–27,000 | 0–37,000 |
| Thrombopoietin — pg/ml | ||||
| Median | 3133 | 3255 | 3111 | 3163 |
| Range | 1945–4443 | 1806–4955 | 2003–4793 | 1806–4955 |
The baseline characteristics of the enrolled patients are shown according to cohort and in the total population of patients. All patients received horse antithymocyte globulin on days 1 to 4 and cyclosporine from day 1 to 6 months. All the patients received eltrombopag at an age-dependent dose, with cohort 1 receiving eltrombopag from day 14 to 6 months, cohort 2 from day 14 to 3 months, and cohort 3 from day 1 to 6 months. Patient 11 in cohort 1 was excluded from the analysis owing to ineligibility (Fig. S1A in the Supplementary Appendix). Patient 17 in cohort 1 had fluctuating blood counts at the 6-month time point owing to a viral infection and was categorized as having a complete response.
Glycosylphosphatidylinositol (GPI)–deficient neutrophils could not be measured in eight patients owing to severe neutropenia.