Overall summary of AEs and treatment-related AEs in the safety population
| Preferred term . | DFP (N = 152) n (%) . | DFO (N = 76) n (%) . | P value (DFP vs DFO) (Fisher exact) . |
|---|---|---|---|
| Overall summary of AEs | |||
| AE | 134 (88.2) | 67 (88.2) | 1.0000 |
| Severe AE | 25 (16.4) | 5 (6.6) | 0.0393 |
| SAE | 40 (26.3) | 14 (18.4) | 0.2472 |
| Treatment-related AE* | 78 (51.3) | 30 (39.5) | 0.1215 |
| Treatment-related SAE* | 9 (5.9) | 3 (3.9) | 0.7550 |
| Withdrew due to an AE† | 7 (4.6) | 3 (3.9) | 1.0000 |
| Treatment-related AEs by preferred term occurring in ≥ 5% of patients | |||
| Abdominal pain‡ | 26 (17.1) | 3 (3.9) | 0.0053 |
| Vomiting | 22 (14.5) | 1 (1.3) | 0.0009 |
| Pyrexia | 14 (9.2) | 7 (9.2) | 1.0000 |
| ALT level increased above normal | 14 (9.2) | 0 (0.0) | 0.0059 |
| AST level increased above normal | 14 (9.2) | 0 (0.0) | 0.0059 |
| Neutrophil count decreased | 9 (5.9) | 2 (2.6) | 0.3441 |
| Nausea | 8 (5.3) | 2 (2.6) | 0.5024 |
| Chromaturia | 8 (5.3) | 1 (1.3) | 0.2781 |
| Injection-site pain | 0 (0.0) | 5 (6.6) | 0.0038 |
| Preferred term . | DFP (N = 152) n (%) . | DFO (N = 76) n (%) . | P value (DFP vs DFO) (Fisher exact) . |
|---|---|---|---|
| Overall summary of AEs | |||
| AE | 134 (88.2) | 67 (88.2) | 1.0000 |
| Severe AE | 25 (16.4) | 5 (6.6) | 0.0393 |
| SAE | 40 (26.3) | 14 (18.4) | 0.2472 |
| Treatment-related AE* | 78 (51.3) | 30 (39.5) | 0.1215 |
| Treatment-related SAE* | 9 (5.9) | 3 (3.9) | 0.7550 |
| Withdrew due to an AE† | 7 (4.6) | 3 (3.9) | 1.0000 |
| Treatment-related AEs by preferred term occurring in ≥ 5% of patients | |||
| Abdominal pain‡ | 26 (17.1) | 3 (3.9) | 0.0053 |
| Vomiting | 22 (14.5) | 1 (1.3) | 0.0009 |
| Pyrexia | 14 (9.2) | 7 (9.2) | 1.0000 |
| ALT level increased above normal | 14 (9.2) | 0 (0.0) | 0.0059 |
| AST level increased above normal | 14 (9.2) | 0 (0.0) | 0.0059 |
| Neutrophil count decreased | 9 (5.9) | 2 (2.6) | 0.3441 |
| Nausea | 8 (5.3) | 2 (2.6) | 0.5024 |
| Chromaturia | 8 (5.3) | 1 (1.3) | 0.2781 |
| Injection-site pain | 0 (0.0) | 5 (6.6) | 0.0038 |
DFO, deferoxamine; DFP, deferiprone.
The causal relationship to study medication was based on the investigator’s and sponsor’s assessments.
AEs leading to withdrawal from the study included abdominal pain and vomiting (DFP n = 2), nausea (DFO n = 1), acute chest syndrome (DFP = 1), pneumonia (DFO = 1), motor car accident (DFP n = 1, DFO n = 1), hepatic and splenic complications (DFP n = 1), neutropenia (DFP n = 1), and agranulocytosis (DFP n = 1).
Includes the preferred terms of abdominal pain and abdominal pain upper.