Most frequently reported AEs (any grade, ≥10% of patients in either arm) by system organ class and MedDRA preferred term
| System organ class/abnormality preferred term . | Defibrotide treatment group (n = 102), n (%) . | Historical-control cohort (n = 32), n (%) . |
|---|---|---|
| Blood and lymphatic system disorders | ||
| Coagulopathy | 2 (2) | 5 (15.6) |
| Cardiac disorders | ||
| Bradycardia | 10 (9.8) | 6 (18.8) |
| Tachycardia | 10 (9.8) | 14 (43.8) |
| GI disorders | ||
| Diarrhea | 24 (23.5) | 12 (37.5) |
| Nausea | 13 (12.7) | 10 (31.3) |
| Vomiting | 20 (19.6) | 8 (25) |
| Abdominal pain | 4 (3.9) | 7 (21.9) |
| Constipation | 6 (5.9) | 5 (15.6) |
| Lip hemorrhage | 2 (2) | 4 (12.5) |
| General disorders and administration site conditions | ||
| MOF* | 15 (14.7) | 3 (9.4) |
| Pyrexia | 14 (13.7) | 9 (28.1) |
| Peripheral edema | 13 (12.7) | 4 (12.5) |
| Hypothermia | 5 (4.9) | 5 (15.6) |
| Generalized edema | 8 (7.8) | 8 (25) |
| Edema | 3 (2.9) | 7 (21.9) |
| Immune system disorders | ||
| GVHD in skin | 5 (4.9) | 5 (15.6) |
| Metabolism and nutrition disorders | ||
| Hyperglycemia | 5 (4.9) | 4 (12.5) |
| Fluid overload | 0 | 5 (15.6) |
| Metabolic acidosis | 1 (1) | 4 (12.5) |
| Nervous system disorders | ||
| Tremor | 2 (2) | 4 (12.5) |
| Psychiatric disorders | ||
| Agitation | 11 (10.8) | 9 (28.1) |
| Confusional state | 4 (3.9) | 5 (15.6) |
| Anxiety | 4 (3.9) | 4 (12.5) |
| Insomnia | 1 (1) | 4 (12.5) |
| Disorientation | 0 | 4 (12.5) |
| Renal and urinary disorders | ||
| Hematuria | 10 (9.8) | 5 (15.6) |
| Respiratory, thoracic, and mediastinal disorders | ||
| Epistaxis | 13 (12.7) | 5 (15.6) |
| Respiratory failure | 12 (11.8) | 4 (12.5) |
| Pulmonary alveolar hemorrhage | 12 (11.8) | 5 (15.6) |
| Pleural effusion | 5 (4.9) | 6 (18.8) |
| Rales | 1 (1) | 6 (18.8) |
| Dyspnea | 0 | 5 (15.6) |
| Skin and subcutaneous tissue disorders | ||
| Blister | 6 (5.9) | 8 (25) |
| Petechiae | 6 (5.9) | 9 (28.1) |
| Rash | 8 (7.8) | 7 (21.9) |
| Skin disorder | 3 (2.9) | 5 (15.6) |
| Alopecia | 2 (2) | 5 (15.6) |
| Vascular disorders | ||
| Hypotension | 40 (39.2) | 16 (50) |
| System organ class/abnormality preferred term . | Defibrotide treatment group (n = 102), n (%) . | Historical-control cohort (n = 32), n (%) . |
|---|---|---|
| Blood and lymphatic system disorders | ||
| Coagulopathy | 2 (2) | 5 (15.6) |
| Cardiac disorders | ||
| Bradycardia | 10 (9.8) | 6 (18.8) |
| Tachycardia | 10 (9.8) | 14 (43.8) |
| GI disorders | ||
| Diarrhea | 24 (23.5) | 12 (37.5) |
| Nausea | 13 (12.7) | 10 (31.3) |
| Vomiting | 20 (19.6) | 8 (25) |
| Abdominal pain | 4 (3.9) | 7 (21.9) |
| Constipation | 6 (5.9) | 5 (15.6) |
| Lip hemorrhage | 2 (2) | 4 (12.5) |
| General disorders and administration site conditions | ||
| MOF* | 15 (14.7) | 3 (9.4) |
| Pyrexia | 14 (13.7) | 9 (28.1) |
| Peripheral edema | 13 (12.7) | 4 (12.5) |
| Hypothermia | 5 (4.9) | 5 (15.6) |
| Generalized edema | 8 (7.8) | 8 (25) |
| Edema | 3 (2.9) | 7 (21.9) |
| Immune system disorders | ||
| GVHD in skin | 5 (4.9) | 5 (15.6) |
| Metabolism and nutrition disorders | ||
| Hyperglycemia | 5 (4.9) | 4 (12.5) |
| Fluid overload | 0 | 5 (15.6) |
| Metabolic acidosis | 1 (1) | 4 (12.5) |
| Nervous system disorders | ||
| Tremor | 2 (2) | 4 (12.5) |
| Psychiatric disorders | ||
| Agitation | 11 (10.8) | 9 (28.1) |
| Confusional state | 4 (3.9) | 5 (15.6) |
| Anxiety | 4 (3.9) | 4 (12.5) |
| Insomnia | 1 (1) | 4 (12.5) |
| Disorientation | 0 | 4 (12.5) |
| Renal and urinary disorders | ||
| Hematuria | 10 (9.8) | 5 (15.6) |
| Respiratory, thoracic, and mediastinal disorders | ||
| Epistaxis | 13 (12.7) | 5 (15.6) |
| Respiratory failure | 12 (11.8) | 4 (12.5) |
| Pulmonary alveolar hemorrhage | 12 (11.8) | 5 (15.6) |
| Pleural effusion | 5 (4.9) | 6 (18.8) |
| Rales | 1 (1) | 6 (18.8) |
| Dyspnea | 0 | 5 (15.6) |
| Skin and subcutaneous tissue disorders | ||
| Blister | 6 (5.9) | 8 (25) |
| Petechiae | 6 (5.9) | 9 (28.1) |
| Rash | 8 (7.8) | 7 (21.9) |
| Skin disorder | 3 (2.9) | 5 (15.6) |
| Alopecia | 2 (2) | 5 (15.6) |
| Vascular disorders | ||
| Hypotension | 40 (39.2) | 16 (50) |
GI, gastrointestinal; MedDRA, Medical Dictionary for Regulatory Activities.
Neither symptoms indicative of underlying disease (VOD/SOS with MOF) nor events and laboratory values expected following HSCT were recorded as AEs unless the event was considered serious. For the defibrotide group, the protocol instructed study sites to capture symptoms of VOD/SOS and/or MOF as an AE, if the event also met criteria for seriousness.