nical trial included patients from the following races: 60% White, 27% Asian, 7% Black or African American, 1%Native Hawaiian or Other Pacific Islander, 1% other and 3% unknown. XOSPATA was given orally at a starting dose of120 mg daily until unacceptable toxicity or lack of clinical benefit. Dose reductions were allowed, to manage adverseevents, and dose increases were allowed, to increase clinical benefit.
The other baseline demographic and disease characteristics are shown in Table 4.
Table 4: Baseline Demographic and Disease Characteristics in Patients with Relapsed
or Refractory AML
Demographic and Disease Characteristics
ADMIRAL
XOSPATA (120 mg daily)
N=138
Demographics
Median Age (Years) (Range) 60 (20, 84)
Age Categories, n (%)
<65 years 85 (62)
≥65 years 53 (38)
Sex, n (%)
Male 64 (46)
Female 74 (54)
Baseline ECOG, n (%)
0-1 113 (82)
≥2 25 (18)
Disease Characteristics
Untreated relapse AML, n (%) 82 (59)
Primary refractory AML, n (%) 56 (41)
Refractory relapse AML, n (%) 0
Median number of relapses (Range) 1 (0, 2)
Number of relapses, n (%)
0 56 (41)
1 80 (58)
2 or more 2 (1)
Prior Stem Cell Transplantation, n (%) 27 (20)
Transfusion Dependent at Baseline, n (%)* 106 (77)
FLT3 Mutation Status, n (%)
ITD alone 121 (88)
TKD alone 12 (9)
ITD and TKD 5 (4)
AML: acute myeloid leukemia; FLT3: FMS-related tyrosine kinase 3; ITD: internal tandem
duplication; TKD: D835/I836 tyrosine kinase domain point mutation; ECOG PS: Eastern Cooperative
Oncology Group performance status
*Patients were defined as transfusion dependent at baseline if they received any red blood cell or
platelet transfusions within the 56-day baseline period.
The determination of efficacy was established on the basis of the rate of complete remission (CR)/complete remissionwith partial hematologic recovery (CRh), the duration of CR/CRh (DOR), and the rate of conversion from transfusiondependence to transfusion independence in the ADMIRAL trial. The median follow-up was 4.6 months (95% CI: 2.8,15.8). Fourteen patients were still in remission at the time of the first interim DOR analysis. The efficacy results areshown in Table 5.
For patients who achieved a CR/CRh, the median time to first response was 3.6 months (range, 0.9 to 9.6 months). TheCR/CRh rate was 29 of 126 in patients with FLT3-ITD or FLT3-ITD/TKD and 0 of 12 in patients with FLT3-TKD only.
Among the 106 patients who were dependent on red blood cell (RBC) and/or platelet transfusions at baseline, 33 (31.1%)became independent of RBC and platelet transfusions during any 56-day post-baseline period. For the 32 patients whowere independent of both RBC and platelet transfusions at baseline, 17 (53.1%) remained transfusion-independent duringany 56-day post-baseline period。
Table 5: Efficacy Results in Patients with Relapsed or Refractory AML
ADMIRAL
XOSPATA (120 mg daily)
Remission Rate N=138
CR*/CRh† n/N (%) 29/138 (21)
95% CI‡ 14.5, 28.8
Median DOR§ (months) 4.6
Range (months) 0.1 to 15.8¶
CR* n/N (%) 16/138 (11.6)
95% CI‡ 6.8, 18.1
Median DOR§ (months) 8.6
Range (months) 1 to 13.8
CRh† n/N (%) 13/138 (9.4)
95% CI‡ 5.1, 15.6
Median DOR§ (months) 2.9
Range (months) 0.1 to 15.8¶
CI: confidence interval; NE: not estimable; NR: not reached; Only responses prior to HSCT were included in resp
