The clinical efficacy and safety of SCEMBLIX for the treatment of patients with Ph+ CML-CP who have the T315I mutation were evaluated in a multicenter, open-label study1
MMR was achieved by 96 weeks in 49% (95% CI, 34-64) of the patients (n=45) treated with SCEMBLIX.1
The median duration of treatment was 108 weeks (range: 2 to 215 weeks).1
- By Week 24, MMR was achieved in 58% (n=11/19) in ponatinib-naive patients vs 31% (n=8/26) of patients pretreated with ponatinib12
- Percentages of patients who had previously received 1, 2, 3, 4, or 5 or more TKIs were 18%, 31%, 36%, 13%, and 2.2%, respectively1
Efficacy was based on 45 patients with Ph+ CML-CP who have the T315I mutation who received SCEMBLIX at a dose of 200 mg bid. Patients continued treatment until unacceptable toxicity or treatment failure occurred.1
bid, twice daily; CCyR, complete cytogenetic response; CML-CP, chronic myeloid leukemia in chronic phase; IS, International Scale; MCyR, major cytogenetic response; MMR, major molecular response; MR, molecular response; qd, once daily; TKI, tyrosine kinase inhibitor.
MMR was defined as BCR::ABL1IS ≤0.1%.1
CCyR was defined as 0% of Philadelphia chromosome–positive metaphases in bone marrow aspirate with at least 20 examined.1
MCyR was defined as 0% to 35% Ph+ metaphases.11
MR4 was defined as BCR::ABL1IS ≤0.01%.2
MR4.5 was defined as BCR::ABL1IS ≤0.0032%.2
aMust meet the definition of treatment failure per the 2013 European LeukemiaNet recommendations. Patients meeting treatment failure criteria on bosutinib could be switched to SCEMBLIX.
bDefined as nonhematologic grade 3 or 4 toxicity while on therapy; persistent grade 2 toxicity, unresponsive to optimal management, including dose adjustments; or hematologic grade 3 or 4 toxicity while on therapy, recurrent after dose reduction to the lowest recommended dose.
cPatients will continue to receive study treatment for up to 96 weeks after the last patient’s first dose or 48 weeks after the last patient switches to SCEMBLIX, whichever is longer.
dEstimated using a common risk difference stratified by baseline MCyR status.
eEstimated using a Cochran-Mantel-Haenszel 2-sided test stratified by baseline MCyR status.
fCCyR analysis based on patients who were not in CCyR at baseline.
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