Table 1.
Demographics and Disease Characteristics for HSCT Recipients with TMA
| Treated with Eculizumab (n = 39) | Not Treated (n = 30) | P | |
|---|---|---|---|
| Median age, yr (IQR) | 5.3 (3.6–11.1) | 8.3 (3.3–13.8) | .56 |
| Male | 17 (56.7%) | 26 (66.7%) | .46 |
| Race | 1.00 | ||
| White | 22 (73.3%) | 29 (74.4%) | |
| Non-white | 8 (26.7%) | 10 (25.6%) | |
| Diagnosis | .41 | ||
| Bone marrow failure | 9 (30%) | 15 (38.5%) | |
| Immune deficiency | 13 (43.3%) | 17 (43.5%) | |
| Malignancy | 4 (13.3%) | 6 (15.4%) | |
| Benign hematology, genetic/metabolic | 4 (13.3%) | 1 (2.6%) | |
| Donor type | .031 | ||
| Related | 5 (16.7%) | 10 (25.6%) | |
| Unrelated | 20 (66.7%) | 29 (74.4%) | |
| Autologous | 5 (16.7%) | 0 (0%) | |
| Stem cell source | .52 | ||
| Bone marrow | 24 (80%) | 26 (66.7%) | |
| Peripheral blood | 5 (16.7%) | 10 (25.6%) | |
| Cord blood | 1 (3.3%) | 3 (7.7%) | |
| HLA match | .59 | ||
| Matched | 16/25 (64%) | 28 (72%) | |
| Mismatched | 9/25 (36%) | 11 (28%) | |
| Conditioning regimen | .63 | ||
| Myeloablative | 18 (60%) | 20 (51.3%) | |
| Reduced intensity | 12 (40%) | 19 (48.7%) | |
| Calcineurin inhibitor for GVDH prophylaxis | 25/25 (100%) | 37/39 (95%) | .52 |
| GVHD, grades III–IV | 15/25 (60%) | 10/39 (26%) | .1 |
| TMA diagnosis, median days after HSCT (IQR) | 28 (13.8–48.5) | 32 (17–43) | .41 |
| Meningococcal meningitis | 0 (0%) | 0 (0%) | 1.00 |
| Bacteremia | 10 (33%) | 11 (28%) | .79 |
| Fungemia | 2 (3.3%) | 0 (0%) | .19 |
| Cytomegalovirus viremia | 7 (23%) | 7 (18%) | .76 |
| Epstein-Barr virus viremia | 11 (36.6%) | 4 (10.3%) | .02 |
IQR indicates interquartile range.