2024/10/21 by Adi Shacham Abulafia, Adi Shacham‐Abulafia, Yulia Volcheck +9 · 1 citation
Medicine · #Acute Myeloid Leukemia Research #Chronic Myeloid Leukemia Treatments #Lung Cancer Treatments and Mutations
paper · pdf · doi:10.1111/ejh.14330
openalex publication_date 2024/10/21 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/30
OBJECTIVES: Asciminib, a novel allosteric BCR::ABL1 inhibitor, targets the ABL1 myristoyl pocket to potentially reduce toxicity and enhance efficacy. It is approved for Philadelphia chromosome-positive chronic-phase chronic myeloid leukemia (CML-CP) in patients with resistance or intolerance to two or more tyrosine kinase inhibitors (TKIs) or those with the T315I mutation. METHODS: This retrospective analysis evaluated patients with CML treated with asciminib under a managed-access program across eight Israeli centers from July 2019 to August 2022. We assessed treatment responses, toxicities, event-free survival (EFS), and overall survival (OS) using Kaplan-Meier methods. RESULTS: The study included 30 patients who had received a median of three prior TKIs, with 73% starting asciminib due to intolerance. After a median follow-up of 7.1 months, 85% of those without prior complete cytogenetic response (CCyR) achieved CCyR, and 60% previously not in major molecular response (MMR) attained MMR. Resistance was rare (10%), with no cardiovascular events reported despite high baseline comorbidity (73%). Median EFS was 47 months; median OS was not reached. CONCLUSION: Asciminib demonstrates significant efficacy and tolerability in heavily pretreated patients with CML-CP, with no new cardiovascular events observed. Further long-term studies are necessary to explore its full cardiovascular impact.