Table 2:
Patient disposition
| Cohort 1 |
Cohort 2 |
|||
|---|---|---|---|---|
|
FLT3-ITD positive (n=112) |
FLT3-ITD negative (n=44) |
FLT3-ITD positive (n=136) |
FLT3-ITD negative (n=40) |
|
| Total number of patients who discontinued study treatment | 110 (98%)* | 44 (100%) | 134 (99%)* | 40 (100%) |
| Disease relapse† | 44 (39%) | 11 (25%) | 29 (21%) | 2 (5%) |
| Lack of response or disease progression† | 21 (19%) | 19 (43%) | 28 (21%) | 15 (38%) |
| Adverse events | 25 (22%) | 7 (16%) | 20 (15%) | 3 (8%) |
| QTcF prolongation | 3 (3%) | 0 | 2 (1%) | 1 (3%) |
| Elective haemopoietic stem cell transplantation | 11 (10%) | 1 (2%) | 47 (35%) | 14 (35%) |
| Deaths‡ | 6 (5%) | 4 (9%) | 5 (4%) | 3 (8%) |
| Patient withdrew consent | 2 (2%) | 0 | 0 | 0 |
| Patient non-compliant with dosing schedule | 1 (1%) | 0 | 2 (1%) | 0 |
| Other reasons | 0 | 2 (5%) | 3 (2%) | 3 (8%) |
Data are n (%). One patient with unknown FLT3-ITD status was enrolled in cohort 1, but was not included in the mutation level-specific analyses and therefore is not included in this table. Patients with more than one reason for treatment discontinuation were only counted once and included in the most relevant category. FLT3-ITD=FMS-like tyrosine kinase 3 internal tandem duplication. QTcF=QT interval corrected using Fridericia′s correction formula.
At the time of data cutoff, four patients were alive and receiving quizartinib treatment (two in cohort 1 and two in cohort 2).
These numbers were based on investigator assessment and do not reflect the definition of relapse from quizartinib as defined in the appendix (p 1). Disease relapse was a predefined category with no further text specifications. Conversely, patients included in the no response or disease progression category were based on specific text descriptions provided by the investigators if the adverse event or other category was selected at the time of study treatment discontinuation.
Cause of death in cohort 1: pneumonia (n=2), bacterial sepsis (n=1), sepsis (n=1), subdural haematoma (n=1), systemic inflammatory response syndrome (n=1), cerebral haemorrhage (n=1), myocardial infarction (n=1), haemorrhagic stroke (n=1), and acute hepatic failure (n=1). Cause of death in cohort 2: acute myeloid leukaemia progression (n=3), bacterial sepsis (n=1), haemorrhage (n=1), cardiac arrest (n=1), lung infection (n=1), and sepsis (n=1).