By: Kerri Fitzgerald
An Italian real-world study assessed the use of asciminib in a compassionate use setting in heavily pretreated patients with chronic-phase chronic myeloid leukemia (CP-CML) and demonstrated the tyrosine kinase inhibitor’s (TKI) tolerability in this patient subgroup. Massimo Breccia, MD, of Sapienza University in Rome, and colleagues published the results in Hematological Oncology.
This retrospective study included 77 patients (50.6% male; median age, 63 years) with CP-CML who were heavily pretreated; they had received a median of three previous TKIs and 55.8% were treated with ponatinib. In this cohort, 57.1% and 42.9% patients switched to asciminib because of resistance or intolerance, respectively. More than half of patients (51.7%) reported having three or more comorbidities.
Asciminib maintained or improved molecular responses (MRs) in most patients; more than half (n=41; 53%) achieved a major molecular response (MMR) or better, with 25 patients (32.5%) reaching deep molecular response.
Patients with T315I mutation also responded to asciminib, while ponatinib pretreated patients showed lower MR improvements compared to ponatinib-naïve patients. Previously ponatinib-treated patients also had a lower probability of achieving MMR versus ponatinib-naïve patients (P=0.0262).
Patients who were intolerant to prior TKIs were more likely to achieve MR compared to those who were resistant to previous TKI use, though this difference was not statistically significant (P=0.0262), and the authors reported that these patients responded “positively” to asciminib.
Most adverse events (84.4%) were non-hematologic in nature, the most common of which were gastrointestinal disorders (8.9%) and increased lipase/amylase (4.4%). Among hematologic adverse events, thrombocytopenia (11.2%) was most common.
The study is limited by its retrospective nature and the size of the patient population.
“Asciminib is a compelling therapeutic option for CML-CP patients, even outside of clinical trials, demonstrating a remarkable effectiveness and tolerability in patients treated with ≥2 TKIs and with multiple comorbidities,” the authors concluded.
Reference
Breccia M, Rossi AR, Giai V, et al. Real-world efficacy profile of compassionate use of asciminib in an Italian, multi-resistant chronic-phase chronic myeloid leukemia (CML-CP) patient population. Hematol Oncol. 2025;43(3):e70101. doi:10.1002/hon.70101
