Table 15 Response in CML Studies  Chronic Phase IFN Failure
(n=532) Accelerated Phase
(n=235) Myeloid Blast Crisis
(n=260)
  600 mg n=158 600 mg n=223
 400 mg 400 mg n=77 400 mg n=37
 % of patients [CI 95%]
Hematologic Response1 95% [92.3−96.3] 71%[64.8-76.8] 31% [25.2−36.8]
Complete Hematologic
Response (CHR) 95% 38% 7%
No Evidence of Leukemia (NEL) Not applicable 13% 5%
Return to Chronic Phase (RTC)  Not applicable 20% 18%
Major Cytogenetic Response2 60% [55.3−63.8] 21% [16.2−27.1] 7% [4.5−11.2]
(Unconfirmed3) (65%) (27%) (15%)
Complete4 (Unconfirmed3) 39% (47%) 16% (20%) 2% (7%)
1Hematologic response criteria (all responses to be confirmed after ≥4 weeks):
CHR:Chronic phase study [WBC <10 x 109/L, platelet <450 x 109/L, myelocytes + metamyelocytes <5% in blood, no blasts and promyelocytes in blood, basophils <20%, no extramedullary involvement] and in the accelerated and blast crisis studies [ANC ≥1.5 x 109/L, platelets ≥100 x 109/L, no blood blasts, BM blasts <5% and no extramedullary disease]
NEL: Same criteria as for CHR but ANC ≥1 x 109/L and platelets ≥20 x 109/L (accelerated and blast crisis studies)
RTC: <15% blasts BM and PB, <30% blasts + promyelocytes in BM and PB, <20% basophils in PB, no extramedullary disease other than spleen and liver (accelerated and blast crisis studies).
BM=bone marrow, PB=peripheral blood
2Cytogenetic response criteria (confirmed after ≥4 weeks): complete (0% Ph+ metaphases) or partial (1%-35%). A major response (0%-35%) combines both complete and partial responses.
3 Unconfirmed cytogenetic response is based on a single bone marrow cytogenetic eva luation, therefore unconfirmed complete or partial cytogenetic responses might have had a lesser cytogenetic response on a subsequent bone marrow eva luation.
4 Complete cytogenetic response confirmed by a second bone marrow cytogenetic eva luation performed at least 1 month after the initial bone marrow study.

The median time to hematologic response was 1 month. In late chronic phase CML, with a median time from diagnosis of 32 months, an estimated 87.8% of patients who achieved MCyR maintained their response 2 years after achieving their initial response. After 2 years of treatment, an estimated 85.4% of patients were free of progression to AP or BC, and estimated overall survival was 90.8% [88.3, 93.2]. In accelerated phase, median duration of hematologic response was 28.8 months for patients with an initial dose of 600 mg (16.5 months for 400 mg). An estimated 63.8% of patients who achieved MCyR were still in response 2 years after achieving initial response. The median survival was 20.9 [13.1, 34.4] months for the 400 mg group and was not yet reached for the 600 mg group (p=0.0097). An estimated 46.2% [34.7, 57.7] vs. 65.8% [58.4, 73.3] of patients were still alive after 2 years of treatment in the 400 mg vs. 600 mg dose groups, respectively. In blast crisis, the estimated median duration of hematologic response is 10 months. An estimated 27.2% [16.8, 37.7] of hematologic responders maintained their response 2 years after achieving their initial response. Median survival was 6.9 [5.8, 8.6] months, and an estimated 18.3% [13.4, 23.3] of all patients with blast crisis were alive 2 years after start of study.

Efficacy results were similar in men and women and in patients younger and older