denotes an abstract that is clinically relevant.
denotes that this is a recommended PHD Trainee Session.
denotes that this is a ticketed session.Session: 908. Outcomes Research: Myeloid Malignancies: Poster II
Hematology Disease Topics & Pathways:
Research, Adult, Clinical Research, Health outcomes research, CML, Chronic Myeloid Malignancies, Diseases, Real-world evidence, Myeloid Malignancies, Study Population, Human
Methods: In this retrospective panel-based chart review study, eligible oncologists/hematologists from the US with experience treating patients with CML reported de-identified data via an online case report form from February to April 2024. Eligible adult patients with CML-CP did not have the T315I mutation and initiated asciminib (index) between January 2022 and June 2023 after one prior TKI. Time to treatment discontinuation, and time to BCR::ABL1 ≤0.1% (MR3 or better; major molecular response) and BCR::ABL1 ≤1% (MR2 or better) were measured using Kaplan-Meier analyses. BCR::ABL ≤0.01% (MR4 or better, including undetectable; deep molecular response) as best response, progression to accelerated phase/blast crisis (AP/BC), and adverse events (AEs; any grades) were also assessed. Subgroup analyses were conducted in patients who discontinued the first TKI due to resistance/suboptimal response, and by first- or second-generation (1G/2G) TKI as first TKI.
Results: A total of 149 eligible patient charts reported by 51 physicians (49.0% community, 51.0% academic; 82.4% ≥10 years of practice) were reviewed. The 149 patients had median age 63.0 years and were 45.0% female, 56.4% White, 24.2% Black/African American, and 15.4% Hispanic/Latino. At CML diagnosis, 65.8% had an intermediate-risk and 12.8% a high-risk Sokal score, and 11.4% had ECOG performance score ≥2. Patients received imatinib (48.3%), dasatinib (38.9%), nilotinib (9.4%), or bosutinib (3.4%) as first TKI, with a median treatment duration of 52.1 weeks. The primary reason for first TKI discontinuation included resistance (43.6%), suboptimal response (34.9%), and intolerance/management of adverse events (4.7%). Asciminib was initiated at 40 mg twice daily by 42.3% and 80 mg once daily by 43.6% of patients.
By 48 weeks post index, 93.3% of patients remained on asciminib (resistance/suboptimal response to first TKI: 93.7%; 1G TKI as first TKI: 92.9%; 2G TKI as first TKI: 93.7%). Median time to MR3 or better was 35.0 weeks and the median time to MR2 or better was 21.4 weeks. By 48 weeks post index, 68.2% of patients achieved or maintained MR3 or better (resistance/suboptimal response to first TKI: 70.2%; 1G TKI as first TKI: 61.6%; 2G TKI as first TKI: 73.1%) and 87.8% of patients achieved or maintained MR2 or better (resistance/suboptimal response to first TKI: 92.6%; 1G TKI as first TKI: 86.0%; 2G TKI as first TKI: 88.9%).
Over a median follow-up of 50.8 weeks, MR4 or better was achieved by 45.0% of patients as best response on asciminib (resistance/suboptimal response to first TKI: 49.5%; 1G TKI as first TKI: 40.3%; 2G TKI as first TKI: 49.4%), and no patients were observed to progress to AP/BC post index. Constitutional AEs including fatigue (8.7%), headache (4.7%), rash (3.4%) and abdominal pain (2.0%); gastrointestinal AEs including nausea (8.7%), diarrhea (5.4%) and vomiting (5.4%); and cytopenia (4.0%) were observed post index.
Conclusions: This real-world study of asciminib demonstrated that over two-thirds of patients achieved or maintained MR3, and nine out of ten patients reached the key response milestone goal of MR2 by 48 weeks, despite inclusion of a majority with resistance/suboptimal response to a prior TKI. Deep molecular response was reported as the best response for nearly half of patients during the first year of treatment with asciminib. Low discontinuation rates, regardless of type of prior TKI used, and low frequency of AEs were observed post asciminib initiation. These results suggest that asciminib is well-tolerated and rapidly effective among patients with CML who received one prior TKI in the US clinical practice.
Disclosures: Atallah: Novartis Pharmaceuticals Corporation: Honoraria. Sadek: Novartis Pharmaceuticals Corporation: Current Employment. Wei: Novartis Pharmaceuticals Corporation: Current Employment, Current equity holder in publicly-traded company, Divested equity in a private or publicly-traded company in the past 24 months. Latremouille-Viau: Analysis Group: Other: Dominick Latremouille-Viau is an employee of Analysis Group, Inc. (AG), an economic consulting firm. AG receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis. Rossi: Novartis Pharmaceuticals Corporation: Other: Carmine Rossi is an employee of Analysis Group, Inc, an economic consulting firm that receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis Pharmaceuticals Corporation. Damon: Novartis Pharmaceuticals Corporation: Current Employment, Current equity holder in publicly-traded company. Yang: Novartis Pharmaceuticals: Current Employment. Bellefleur: Novartis Pharmaceuticals Corporation: Other: Remi Bellefleur is an employee of Analysis Group, Inc, an economic consulting firm that receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis Pharmaceuticals Corporation. Guérin: Novartis Pharmaceuticals Corporation: Other: Annie Guerin is an employee of Analysis Group, Inc, an economic consulting firm that receives consulting fees from pharmaceutical companies and other ineligible companies, including Novartis Pharmaceuticals Corporation. Jadhav: Novartis Pharmaceuticals: Current Employment, Current equity holder in publicly-traded company.
OffLabel Disclosure: Asciminib is FDA-approved for patients with Ph+ CML-CP previously treated with ≥2 TKIs or with the T315I mutation, and is currently being assessed in clinical trials in newly diagnosed patients (ASC4FIRST, NCT04971226) or those previously treated with one TKI (ASC2ESCALATE, NCT05384587). The FDA has granted Breakthrough Therapy designation to asciminib in 2024, for the treatment of adult patients with newly diagnosed Ph+ CML-CP. Asciminib was also recently granted FDA Priority Review for the treatment of adults with newly diagnosed CML. Asciminib for the treatment of CML-CP after one prior TKI is already observed in the US clinical practice and this study describes real-world clinical outcomes in these patients treated with asciminib after one prior TKI in the US. This is a retrospective observational study with secondary data use. The study received an IRB exemption.
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