Table 5.
MODS and resolution in patients treated with eculizumab
| During study | 6 mo after hrTMA diagnosis | 1 y after HCT | |
|---|---|---|---|
| Study participants, n | 21 | 15 | 13 |
| Number of participants with MODS, n (%) | 21 (100) | 4 (26.7) | 2 (15.4) |
| Number of organs affected, median (range) | 4 (2-7) | 1 (1-3) | 1 (1-1) |
| Participants requiring PICU care, n (%) | 13 (62) | 1 (6)∗ | 1 (7)∗ |
| ICU days, median (IQR) | 35 (6-48) | ||
| Hospitalization days, median (IQR) | 71 (41-112) | ||
| Renal | |||
| Participants with AKI, n (%) | 21 (100) | 7 (40) | 3 (23) |
| Participants not requiring RRT, n (%) | 15/21 (71) | 4 (26.7) | 2 (15.4) |
| Participants requiring RRT, n (%) | 6/21 (29) | 3† (20) | 1 (7.6) |
| Lowest cystatin-C GFR, mL/min per 1.73 m2 for participants not on dialysis, median (IQR) | 38 (29-54.5) | 77 (59-95) | 79 (70-95) |
| Highest serum creatinine (mg/dL) for patients not on RRT, median (IQR) | 0.99 (0.7-1.6) | 0.38 (0.45-0.62) | 0.56 (0.53-0.6) |
| Serum creatinine elevation × (times) pre-HCT baseline | 1.1× (1-1.8) | 1.27 (1.2-1.5) | 1.14 (1-1.35) |
| Max rUPCR (mg/mg) for participants not on dialysis, median (IQR) | 7 (5-15) | 0.43 (0.2-0.5) | 0.3 (0.2-0.4) |
| Hypertension | |||
| Severe hypertension, n (%) | 17 (81) | 3 (20) | 0 |
| Any hypertension, n (%) | 19 (90) | 5 (33) | 5 (38) |
| Pulmonary | |||
| Participants requiring oxygen, n (%) | 16 (76) | 1 (6)∗ | 1 (7)∗ |
| Days on oxygen support, median (IQR) | 17 (6-34) | ||
| Participants requiring positive pressure ventilation, n (%) | 9 (43) | 1 (6)∗ | 1 (7)∗ |
| Days on positive pressure ventilation, median (IQR) | 22 (9-35) | ||
| Cardiovascular | |||
| Pulmonary hypertension, n (%) | 6 (29) | 0 | 0 |
| Left ventricular dysfunction, n (%) | 4 (19) | 0 | 0 |
| Serositis | |||
| Pericardial effusion, n (%) | 13 (62) | 0 | 0 |
| Cardiac tamponade requiring pericardiocentesis, n (%) | 3 (14) | 0 | 0 |
| Pleural effusions, n (%) | 4 (19) | 0 | 0 |
| Ascites, n (%)‡ | 3 (14) | 0 | 0 |
| CNS, n (%) | |||
| Mental status changes | 8 (38) | 0 | 0 |
| Stroke/PRES | 1 (5) | 0 | 0 |
| GI system, n (%) | |||
| Intestinal TA-TMA with or without bleeding | 9 (43) | 1 (6)† | 0 |
| Intestinal bleeding | 8 (38) | 1 (6)† | 0 |
RRT includes continues RRT and/or dialysis.
PICU, pediatric intensive care unit; PRES, posterior reversible encephalopathy syndrome.
∗
Same patient with diffuse alveolar damage with resolved hrTMA.
†
Same patient with intestinal TMA, intestinal GVHD, mental status changes, and RRT for hyperamonemia clearance.
‡
Participants with VOD of liver.