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 study
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
- MMR by Week 24 was achieved by 58% (n=11/19) in ponatinib-naive patients vs 31% (n=8/26) for patients pretreated with ponatinib11
- 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
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 is defined as 0% to 35% Ph+ metaphases.12
MR4 is defined as BCR-ABL1IS ≤0.01%.2
MR4.5 is defined as BCR-ABL1IS ≤0.0032%.2
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.
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.
dCCyR analysis based on patients who were not in CCyR at baseline.
eEstimated using a common risk difference stratified by baseline MCyR status.
fEstimated using a Cochran-Mantel-Haenszel 2-sided test stratified by baseline MCyR status.
gNonresponders were censored at their last molecular assessment date.
hDiscontinuation from treatment for any reason, without prior achievement of MMR, is considered a competing event.
References: 1. Scemblix [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals Corp; 2021. 2. Rea D, Mauro M, Boquimpani C, et al. Blood. 2021;[in press]. 3. Kantarjian HM, Giles FJ, Bhalla KN, et al. Blood. 2011;117(4):1141-1145. 4. O’Brien SG, Guilhot F, Larson RA, et al. N Engl J Med. 2003;348(11):994-1004. 5. Saglio G, Kim D-W, Issaragrisil S, et al. N Engl J Med. 2010;362(24):2251-2259. 6. Kantarjian H, Shah NP, Hochhaus A, et al. N Engl J Med. 2010;362(24):2260-2270. 7. Cortes JE, Gambacorti-Passerini C, Deininger MW, et al. J Clin Oncol. 2018;36(3):231-237. 8. Cortes JE, Kantarjian HM, Brümmendorf TH, et al. Blood. 2011;118(17):4567-4576. 9. Shah NP, Kantarjian HM, Kim DW, et al. J Clin Oncol. 2008;26(19):3204-3212. 10. Soverini S, Gnani A, Colarossi S, et al. Blood. 2009;114(10):2168-2171. 11. Data on file. ABL001 Investigator’s Brochure. Novartis Pharmaceuticals Corp; 2021. 12. Data on file. CABL001A2301 clinical study report. Novartis Pharmaceuticals Corp; 2021.