Summary of AEs during years 2 and 3 of deferiprone treatment in the FIRST-EXT study (safety population)
| . | Deferiprone (N = 134), n (%) . |
|---|---|
| Overall summary of AEs | |
| AEs | 104 (77.6) |
| Severe AEs | 19 (14.2) |
| SAEs | 35 (26.1) |
| AEs at least possibly related to study treatment∗ | 41 (30.6) |
| SAEs at least possibly related to study treatment∗ | 13 (9.7) |
| AEs leading to withdrawal† | 2 (1.5) |
| SAEs by preferred term seen in >1 patient∗ | |
| Sickle cell crisis | 19 (14.2) |
| Neutropenia‡,§ | 12 (9.0) |
| Pyrexia | 5 (3.7) |
| Cholecystectomy | 3 (2.2) |
| Agranulocytosis‡,§ | 2 (1.5) |
| Pneumonia | 2 (1.5) |
| Arthralgia | 2 (1.5) |
| Splenectomy | 2 (1.5) |
| Hypotension | 2 (1.5) |
| Treatment-related AEs by preferred term seen in ≥5% of patients‖ | |
| Neutropenia§ | 12 (9.0) |
| Neutrophil count decreased§ | 12 (9.0) |
| Abdominal pain¶ | 10 (7.5) |
| . | Deferiprone (N = 134), n (%) . |
|---|---|
| Overall summary of AEs | |
| AEs | 104 (77.6) |
| Severe AEs | 19 (14.2) |
| SAEs | 35 (26.1) |
| AEs at least possibly related to study treatment∗ | 41 (30.6) |
| SAEs at least possibly related to study treatment∗ | 13 (9.7) |
| AEs leading to withdrawal† | 2 (1.5) |
| SAEs by preferred term seen in >1 patient∗ | |
| Sickle cell crisis | 19 (14.2) |
| Neutropenia‡,§ | 12 (9.0) |
| Pyrexia | 5 (3.7) |
| Cholecystectomy | 3 (2.2) |
| Agranulocytosis‡,§ | 2 (1.5) |
| Pneumonia | 2 (1.5) |
| Arthralgia | 2 (1.5) |
| Splenectomy | 2 (1.5) |
| Hypotension | 2 (1.5) |
| Treatment-related AEs by preferred term seen in ≥5% of patients‖ | |
| Neutropenia§ | 12 (9.0) |
| Neutrophil count decreased§ | 12 (9.0) |
| Abdominal pain¶ | 10 (7.5) |
AE, adverse event; ALT, alanine aminotransferase; AST, aspartate aminotransferase; SAE, serious adverse event.
The causal relationship of an AE to study drug was assessed according to the World Health Organization criteria13 and defined as not related, possibly related, probably related, and definitely related. SAEs were classified by the principal investigator and represent the data as collected.
Of the 2 patients (1.5%) who withdrew from the study owing to AEs, 1 experienced AEs of thrombocytopenia and neutropenia, and 1 was hospitalized for generalized edema.
Neutropenia and agranulocytosis were serious AEs that were deemed to be at least possibly related to study treatment.
Neutropenia and agranulocytosis were confirmed if the ANC was within the specified range on 2 consecutive measurements a maximum of 3 days apart, whereby neutropenia was an ANC ≥ 0.5 × 109/L and < 1.5 × 109/L and agranulocytosis was an ANC < 0.5 × 109/L. Decreased neutrophil count was defined as a single occurrence of ANC < 1.5 × 109/L with no confirmatory second value below this threshold within 3 days.
AEs at least possibly related to deferiprone.
Includes the preferred terms of abdominal pain and abdominal pain (upper).