Patient demographic and clinical characteristics
| . | Defibrotide treatment group* (n = 102) . | Historical-control cohort (n = 32) . | P† . |
|---|---|---|---|
| Age at HSCT (y) | .836 | ||
| Mean (SD) | 26 (21.37) | 25.1 (20.23) | — |
| Median (range) | 21 (0-72) | 18 (1-57) | — |
| Age category (y), n (%) | .951 | ||
| ≤16 | 44 (43.1) | 14 (43.8) | — |
| >16 | 58 (56.9) | 18 (56.3) | — |
| Gender (n [%]) | .511 | ||
| Male | 64 (62.7) | 18 (56.3) | — |
| Female | 38 (37.3) | 14 (43.8) | — |
| Weight at baseline (kg) | .880 | ||
| Mean (SD) | 53.7 (33.7) | 52.6 (30.6) | — |
| Median (range) | 60.4 (4-135) | 58.4 (6-111) | — |
| Height (cm)‡ | .853 | ||
| Mean (SD) | 140.1 (42.8) | 141.7 (41) | — |
| Race (n [%]) | .418 | ||
| White | 77 (75.5) | 23 (71.9) | — |
| Black or African American | 6 (5.9) | 2 (6.3) | — |
| Hispanic or Latino | 10 (9.8) | 1 (3.1) | — |
| Asian | 4 (3.9) | 2 (6.3) | — |
| Native Hawaiian/other Pacific Islander | 1 (1.0) | 0 | — |
| Other§ | 4 (3.9) | 4 (12.5) | — |
| Use of ursodeoxycholic acid | 71 (69.6%) | 21 (65.6%) | — |
| Use of low-dose dopamine/dopamine HCl | 34 (33.3%) | 18 (56.3%) | — |
| Stratification factors,||n (%) | |||
| ≤16-years-old | 44 (43.1) | 14 (43.8) | .951 |
| Allogeneic transplant | 90 (88.2) | 27 (84.4) | .567 |
| Prior HSCT | 13 (12.7) | 3 (9.4) | .608 |
| Ventilator/dialysis-dependent at study entry | 34 (33.3) | 7 (21.9) | .220 |
| . | Defibrotide treatment group* (n = 102) . | Historical-control cohort (n = 32) . | P† . |
|---|---|---|---|
| Age at HSCT (y) | .836 | ||
| Mean (SD) | 26 (21.37) | 25.1 (20.23) | — |
| Median (range) | 21 (0-72) | 18 (1-57) | — |
| Age category (y), n (%) | .951 | ||
| ≤16 | 44 (43.1) | 14 (43.8) | — |
| >16 | 58 (56.9) | 18 (56.3) | — |
| Gender (n [%]) | .511 | ||
| Male | 64 (62.7) | 18 (56.3) | — |
| Female | 38 (37.3) | 14 (43.8) | — |
| Weight at baseline (kg) | .880 | ||
| Mean (SD) | 53.7 (33.7) | 52.6 (30.6) | — |
| Median (range) | 60.4 (4-135) | 58.4 (6-111) | — |
| Height (cm)‡ | .853 | ||
| Mean (SD) | 140.1 (42.8) | 141.7 (41) | — |
| Race (n [%]) | .418 | ||
| White | 77 (75.5) | 23 (71.9) | — |
| Black or African American | 6 (5.9) | 2 (6.3) | — |
| Hispanic or Latino | 10 (9.8) | 1 (3.1) | — |
| Asian | 4 (3.9) | 2 (6.3) | — |
| Native Hawaiian/other Pacific Islander | 1 (1.0) | 0 | — |
| Other§ | 4 (3.9) | 4 (12.5) | — |
| Use of ursodeoxycholic acid | 71 (69.6%) | 21 (65.6%) | — |
| Use of low-dose dopamine/dopamine HCl | 34 (33.3%) | 18 (56.3%) | — |
| Stratification factors,||n (%) | |||
| ≤16-years-old | 44 (43.1) | 14 (43.8) | .951 |
| Allogeneic transplant | 90 (88.2) | 27 (84.4) | .567 |
| Prior HSCT | 13 (12.7) | 3 (9.4) | .608 |
| Ventilator/dialysis-dependent at study entry | 34 (33.3) | 7 (21.9) | .220 |
SD, standard deviation.
Three patients in the defibrotide group had an inclusion criteria violation (VOD/SOS diagnosis by day +24, day +24, and day +25, respectively); 1 had an exclusion criteria violation (requirement for multiple pressors at study entry). None of these 4 patients achieved a CR by day +100.
P value from a two-sample Student t test for continuous variables and from a χ2 test for categorical or dichotomous variables; excludes no applicable, missing, and unknown values.
Height available for only 101 defibrotide-treated patients and 31 historical-control patients.
Race = “Other” includes “Unknown,” “UNK,” “Unavailable,” mixed race, “Arab,” and “UK.”
Stratification data were examined using Fisher’s exact test; no statistically significant differences between the 4 stratification variables were observed between the 2 groups.