Table 1.
Disposition reason, n (%) | Ponatinib pretreated n = 29 |
Ponatinib naive n = 19 |
All patients N = 48 |
---|---|---|---|
Patients treated | |||
Treatment ongoinga | 15 (51.7) | 12 (63.2) | 27 (56.3) |
End of treatment | 14 (48.3) | 7 (36.8) | 21 (43.8) |
Primary reason for end of treatment | |||
Physician decisionb | 8 (27.6) | 3 (15.8) | 11 (22.9) |
Adverse event | 2 (6.9) | 2 (10.5) | 4 (8.3) |
Patient/guardian decision | 0 | 0 | 0 |
Progressive disease | 2 (6.9)c | 0 | 2 (4.2)c |
Death | 0 | 2 (10.5) | 2 (4.2) |
Technical problems | 1 (3.4)d | 0 | 1 (2.1)d |
Protocol deviation | 1 (3.4)e | 0 | 1 (2.1)e |
aPatients ongoing at the time of the cutoff (January 6, 2021).
bReasons for physician decision included loss of response (n = 3), loss or lack of response with transfer to stem cell transplant (n = 3), lack of efficacy (n = 2), to receive stem cell transplant (n = 2), and transfer to investigator-initiated trial (n = 1).
cPatients with disease progression to accelerated phase.
dPatient travel to trial site restricted due to COVID-19. Moved to a managed access program.
ePatient required prohibited medication (hydroxyurea). Continued treatment under a managed access program.