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 |
| . | 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.