Demographic and HCT characteristics of recipients of allogeneic HCT with HR TA-TMA, treated with eculizumab vs supportive care only
| . | Eculizumab (N = 19) . | Supportive care only (N = 36) . | P . |
|---|---|---|---|
| n (%) . | n (%) . | ||
| Organ dysfunction∗ | |||
| Pericardial effusion | 2 (11) | 6 (17) | .700 |
| Pulmonary HTN | 2 (11) | 2 (6) | .602 |
| DAH | 0 (0) | 1 (3) | >.999 |
| Respiratory support | 10 (53) | 21 (58) | .685 |
| ECMO | 0 (0) | 1 (3) | >.999 |
| Double of serum Cr | 14 (74) | 27 (75) | >.999 |
| Renal replacement | 3 (16) | 9 (25) | .511 |
| HTN requiring ggt | 10 (53) | 14 (39) | .328 |
| AMS or PRES | 2 (11) | 7 (19) | .473 |
| GI bleeding | 6 (32) | 0 (0) | <.001 |
| Concurrent morbidities∗ | |||
| Severe grade 3/4 acute GVHD | 5 (26) | 9 (25) | >.999 |
| VOD | 2 (11) | 4 (11) | >.999 |
| Infection (bacterial or viral) | 10 (53) | 25 (69) | .218 |
| . | Eculizumab (N = 19) . | Supportive care only (N = 36) . | P . |
|---|---|---|---|
| n (%) . | n (%) . | ||
| Organ dysfunction∗ | |||
| Pericardial effusion | 2 (11) | 6 (17) | .700 |
| Pulmonary HTN | 2 (11) | 2 (6) | .602 |
| DAH | 0 (0) | 1 (3) | >.999 |
| Respiratory support | 10 (53) | 21 (58) | .685 |
| ECMO | 0 (0) | 1 (3) | >.999 |
| Double of serum Cr | 14 (74) | 27 (75) | >.999 |
| Renal replacement | 3 (16) | 9 (25) | .511 |
| HTN requiring ggt | 10 (53) | 14 (39) | .328 |
| AMS or PRES | 2 (11) | 7 (19) | .473 |
| GI bleeding | 6 (32) | 0 (0) | <.001 |
| Concurrent morbidities∗ | |||
| Severe grade 3/4 acute GVHD | 5 (26) | 9 (25) | >.999 |
| VOD | 2 (11) | 4 (11) | >.999 |
| Infection (bacterial or viral) | 10 (53) | 25 (69) | .218 |
| . | Estimate ± SE (%) . | Estimate ± SE (%) . | P value . |
|---|---|---|---|
| OS | .613 | ||
| 100-d | 84.2 ± 8.4 | 80.6 ± 6.6 | |
| 180-d | 73.7 ± 10.1 | 69.4 ± 7.7 | |
| 1-y | 52.6 ± 11.4 | 66.6 ± 7.9 | |
| NRM | .590 | ||
| 100-d | 26.3 ± 10.4 | 27.8 ± 7.6 | |
| 180-d | 36.8 ± 11.4 | 33.6 ± 8.0 | |
| 1-y | 47.4 ± 11.9 | 33.6 ± 8.0 |
| . | Estimate ± SE (%) . | Estimate ± SE (%) . | P value . |
|---|---|---|---|
| OS | .613 | ||
| 100-d | 84.2 ± 8.4 | 80.6 ± 6.6 | |
| 180-d | 73.7 ± 10.1 | 69.4 ± 7.7 | |
| 1-y | 52.6 ± 11.4 | 66.6 ± 7.9 | |
| NRM | .590 | ||
| 100-d | 26.3 ± 10.4 | 27.8 ± 7.6 | |
| 180-d | 36.8 ± 11.4 | 33.6 ± 8.0 | |
| 1-y | 47.4 ± 11.9 | 33.6 ± 8.0 |
| . | Median (range) . | Median (range) . | P value . |
|---|---|---|---|
| Time from transplant until patient met TA-TMA criteria (d) | 14 (0-85) | 29 (6-97) | .021 |
| Number of inpatient days | 156 (29-267) | 132.5 (47-331) | .405 |
| Duration of ICU stay (d) | 17 (0-61)† | 20 (1-162)† | .405 |
| Day 15 to day 365 after HCT | |||
| Inpatient cost per day ($) | 8 593 (4 148-31 108) | 4 609 (2 939-21 258) | <.001 |
| Total cost ($) | 1 419 823 (286 175-7 217 095) | 672 578 (156 379-3 911 402) | .001 |
| . | Median (range) . | Median (range) . | P value . |
|---|---|---|---|
| Time from transplant until patient met TA-TMA criteria (d) | 14 (0-85) | 29 (6-97) | .021 |
| Number of inpatient days | 156 (29-267) | 132.5 (47-331) | .405 |
| Duration of ICU stay (d) | 17 (0-61)† | 20 (1-162)† | .405 |
| Day 15 to day 365 after HCT | |||
| Inpatient cost per day ($) | 8 593 (4 148-31 108) | 4 609 (2 939-21 258) | <.001 |
| Total cost ($) | 1 419 823 (286 175-7 217 095) | 672 578 (156 379-3 911 402) | .001 |
Fisher exact test was used to calculate P values for binary data.
Mann-Whitney test was used to calculate P values for continuous data.
Log-rank test was used to calculate P values for time-to-event data.
Patients were stratified as being at high risk if they had sCb-9 level > the upper limit of normal and proteinuria > 2 mg/mg or either elevated sC5b-9 level or proteinuria and multiorgan dysfunction.
Treatment of eculizumab vs supportive care was at the discretion of the attending provider.
AMS, altered mental status; Cr, creatinine; DAH, diffuse alveolar hemorrhage; ECMO, extra corporal membrane oxygenation; ggt, continuous medication; HTN, hypertension; PRES, posterior reversible encephalopathy syndrome; VOD, veno-occlusive disease.
The bolded values are statistically significant.
Not mutually exclusive. A given patient could have more than 1 type of organ dysfunction or concurrent morbidity.
n = 17 eculizumab-treated and n = 26 supportive care patients were in the ICU.