Asciminib monotherapy as frontline treatment of chronic-phase chronic myeloid leukemia: results from the ASCEND study
Details
Publication Year 2024-11-07,Volume 144,Issue #19,Page 1993-2001
Journal Title
Blood
Publication Type
Research article
Abstract
Asciminib is a myristoyl site BCR::ABL1 inhibitor approved for patients with chronic-phase chronic myeloid leukemia (CP-CML) failing ≥2 prior lines of therapy. The Australasian Leukaemia and Lymphoma Group conducted the Asciminib Evaluation in Newly Diagnosed CML study to assess efficacy of asciminib for newly diagnosed CP-CML. Patients commenced asciminib 40 mg twice daily. Patients with treatment failure, defined as BCR::ABL1 of >10% at 3 or 6 months, or >1% at 12 or 18 months, received either imatinib, nilotinib, or dasatinib in addition to asciminib. In patients with suboptimal response, defined as levels of 1% to 10% at 6 months, >0.1% to 1% at 12 months, or >0.01% to 1% at 18 months, the asciminib dose was increased to 80 mg twice daily. With a median follow-up of 21 months (range, 0-36), 82 of 101 patients continue asciminib. Most common reasons for treatment discontinuation were adverse events (6%), loss of response (4%), and withdrawn consent (5%). There were no deaths; 1 patient developed lymphoid blast crisis. The coprimary end points were early molecular response (BCR::ABL1 of ≤10% at 3 months), achieved in 93% (96% confidence interval [CI], 86-97%), and major molecular response by 12 months achieved in 79%; (95% CI, 70-87%), respectively. Cumulative incidence of molecular response 4.5 was 53% by 24 months. One patient had 2 cerebrovascular events; no other arterial occlusive events were reported. Asciminib as frontline CP-CML therapy leads to high rates of molecular response with excellent tolerance and a low rate of discontinuation for toxicity. This trial was registered at https://www.anzctr.org.au/ as #ACTRN12620000851965.
Publisher
American Society of Hematology
Keywords
Humans; Male; Middle Aged; Female; Aged; Adult; Aged, 80 and over; *Leukemia, Myeloid, Chronic-Phase/drug therapy; Fusion Proteins, bcr-abl/antagonists & inhibitors/genetics; Young Adult; Antineoplastic Agents/therapeutic use/adverse effects; Protein Kinase Inhibitors/therapeutic use/adverse effects/administration & dosage; Pyrimidines/therapeutic use/adverse effects/administration & dosage; Treatment Outcome; Imatinib Mesylate/therapeutic use/adverse effects/administration & dosage; Follow-Up Studies; Niacinamide/analogs & derivatives; Pyrazoles
Department(s)
Haematology
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