Table 1.
Baseline demographics and clinical characteristics
| mITT population (N=21) | PP population (N=15) | |
|---|---|---|
| Sex, n (%) | ||
| Male | 11 (52.4) | 6 (40.0) |
| Female | 10 (47.6) | 9 (60.0) |
| Age, years, median (range) | 59.0 (22–86) | 52.0 (22–84) |
| Weight, kg, median (range) | 62.9 (37.9–103.5) | 65.8 (43.6–103.5) |
| BMI, kg/m2, median (range) | 24.7 (17.3–36.2) | 24.7 (18.4–36.2) |
| Time since TTP diagnosis, days, median (range) | 2.0 (1–299) | 1 (1–299) |
| Initial TTP episode, n (%) | 16 (76.2) | 12 (80.0) |
| Number of previous TTP episodes, n (%) | ||
| 0 | 16 (76.2) | 12 (80.0) |
| 1 | 5 (23.8) | 3 (20.0) |
| Platelet count, x109/L, median (range) | 21.5 (8–78) | 23.0 (8–70) |
| ADAMTS13 activity level, n (%) | ||
| <10% | 18 (85.7) | 15 (100.0) |
| ≥10% | 3 (14.3) | 0 |
| LDH, U/L median (range) | 581 (227–1929) | 575 (227–1794) |
| Cardiac troponin I level, μg/L, median (range) | 0.12 (0.03–7.47) | 0.12 (0.03–3.52) |
| Serum creatinine level, μmol/L, median (range) | 79.5 (49.0–294.0) | 67.0 (49.0–226.0) |
| GCS score, n (%) | ||
| ≤12 | 3 (14.3) | 2 (13.3) |
| 13–15 | 18 (85.7) | 13 (86.7) |
| SMMSE score, mean (range) | 17.3 (0–30) | 17.7 (0–30) |
| Severity of disease, n (%) | ||
| Very severea | 13 (61.9) | 9 (60.0) |
| Less severe | 8 (38.1) | 6 (40.0) |
ADAMTS13 a disintegrin and metalloproteinase with a thrombospondin type 1 motif, member 13; BMI body mass index, GCS Glasgow coma scale, LDH lactate dehydrogenase, mITT modified intention-to-treat, PP per protocol, SMMSE standardized Mini-Mental State Examination; TTP thrombotic thrombocytopenia purpura
aFrench severity score ≥3, severe neurologic involvement (e.g., coma, seizures, focal deficit), or cardiac involvement (cardiac troponin I level > 2.5 x upper limit of normal).