|STUDIES INCLUDED A BROAD RANGE OF ADULT AND PEDIATRIC PATIENTS WITH VOD WITH RENAL OR PULMONARY DYSFUNCTION FOLLOWING HSCT1|
|Study 11,2||Study 21,3||Study 31,4,5|
|Study design||Phase 3
|Expanded access study|
|Number of patients||102||75||512|
|VOD associated with:||Multi-organ dysfunction (pulmonary, renal, or both)a||Multi-organ dysfunction||Renal or pulmonary dysfunction|
|Median age (years) (range)||21 (<1, 72)||32 (<1, 61)||14 (<1, 69)|
|Type of transplant, n (%) allogeneic||90 (88)||67 (89)||450 (88)|
|Type of transplant, n (%) autologous||12 (12)||8 (11)||61 (12)|
|Ventilator or dialysis dependent at study entry, n (%)||34 (33)||8 (11)||225 (42)|
|Median number of days on treatment (days) (range)||21.5 (1, 58)||19.5 (3, 83)||21.0b (1, 91)|
|Treatment dose of Defitelio||6.25 mg/kg infusion every 6 hours|
aA diagnosis of VOD was made using the following criteria: bilirubin ≥2 mg/dL and at least 2 of the following—hepatomegaly, ascites, and weight gain >5% by Day +21 post HSCT. The multi-organ dysfunction was to have occurred by Day +28 post HSCT. Patients were not permitted to receive concomitant medications such as heparin, warfarin, or alteplase because of an increased risk of bleeding.
bDuration of treatment from first dose to last dose is presented because days without treatment were not captured for the expanded access study.
HSCT=hematopoietic stem-cell transplantation; VOD=veno-occlusive disease.