Table 1.
Study demographics and disease characteristics
| Treated with eculizumab n=18 |
Untreated subjects n=11 |
p-value | |
|---|---|---|---|
| Transplant characteristics | |||
| Age (years)# | 4.6 (2 – 15.2) | 8.2 (1.8 – 11.8) | 0.9 |
| Actual weight (kg) | 17 (13.5 – 44.5) | 15.4 (12.6 – 44) | 0.77 |
| Male gender | 11 (61.1%) | 7 (63.6%) | 1 |
| Race | 0.4 | ||
| Caucasian | 13 (72.2%) | 6 (54.5%) | |
| Non-Caucasian | 5 (27.8%) | 5 (45.5%) | |
| Underlying diagnosis | 0.03 | ||
| Bone marrow failure | 0 (0%) | 3 (27.3%) | |
| Immune Deficiency | 9 (50%) | 6 (54.5%) | |
| Malignancy | 8 (44.4%) | 1 (9.1%) | |
| Other | 1 (5.6%) | 1 (9.1%) | |
| Donor type | 0.04 | ||
| Related | 1 (5.6%) | 4 (36.4%) | |
| Unrelated | 13 (72.2%) | 7 (63.6%) | |
| Autologous | 4 (22.2%) | 0 (0%) | |
| Stem cell source | 1 | ||
| Bone Marrow | 13 (72.2%) | 7 (63.6%) | |
| PBSCs | 4 (22.2%) | 3 (27.3%) | |
| Cord Blood | 1 (5.6%) | 1 (9.1%) | |
| HLA Match | 0.11 | ||
| Matched | 4 /14 (22.2%) | 7/11 (63.6%) | |
| Mismatched | 10 /14 (55.6%) | 4/11 (36.4%) | |
| Conditioning Regimen | 0.44 | ||
| Myeloablative | 12 (66.7%) | 5 (45.5%) | |
| Reduced intensity | 6 (33.3%) | 6 (54.5%) | |
| Cyclosporine GVHD prophylaxis | 14/14 (100%) | 11/11(100%) | 1 |
| GVHD (grade 3–4) | 9 (50%) | 6 (54.5%) | 0.70 |
| Disease features at TMA diagnosis | |||
| TMA diagnosis (day after HSCT) | 30 (18 – 55.5) | 26 (17 – 38.5) | 0.79 |
| Blood sC5b-9 (normal <244ng/ml) | 373.5 (301 – 744) | 458.5 (324.2 – 708.4) | 0.24 |
| Cystatin C estimated GFR (mg/ml) | 29.5 (17.8 – 46) | 45 (34.5 – 56) | 0.77 |
| Urine random protein/creatinine ratio | 10.3 (6.5 – 20.8) | 2.6 (2.5 – 3.3) | <0.0001 |
| Renal replacement therapy | 7 (38.9%) | 4 (36.4%) | 1 |
| Hypertension | 18 (100%) | 11(100%) | 1 |
| Pericardial effusion | 15 (83.3%)* | 9 (81.8%)** | 1 |
| PRES | 2 (11%) | 1(9.1%) | 1 |
| CNS bleed | 1(5.6%) | 0(0%) | 1 |
| Pulmonary hypertension | 3 (16.7%) | 2 (18%) | 1 |
| Gastrointestinal bleeding | 11(61%) | 7(63.6%) | 1 |
| Respiratory failure | 2 (11%) | 1(9.1%) | 1 |
Data shown as median (interquartile range) or n(%).
#
three subjects in treated group and two in untreated group were young adults 19–29 years of age.
* 4/15 (26.7%) and **1/9 (11%) had cardiac tamponade.
GVHD, graft versus host disease; TMA, thrombotic microangiopathy; GFR, glomerular filtration rate; PRES: posterior reversible encephalopathy syndrome; CNS, central nervous system.