Fiche publication
Date publication
juillet 2025
Journal
Blood advances
Auteurs
Membres identifiés du Cancéropôle Est :
Dr LIOURE Bruno
Tous les auteurs :
Chanut M, Cabannes-Hamy A, Balsat M, Sahki N, Raffoux E, Hicheri Y, Lioure B, Laemmel L, Leguay TT, Cacheux V, Gallego-Hernanz MP, Pieragostini A, Roth-Guepin G, Gabellier L, Denizon N, Willems L, Cabrera Q, Chantepie SP, Coiteux V, Capdupuy C, Carre M, Machet A, Lambert J, Roux S, Nicolini FE, Boissel N, Clappier E, Kim R, Rousselot P
Lien Pubmed
Résumé
Asciminib (ASC) is an allosteric inhibitor of BCR::ABL1 that binds the myristoylation site of the ABL1 protein. We report the use of ASC in relapsed Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL) and lymphoid blast crisis of chronic myeloid leukemia (LBC-CML) in 41 patients retrospectively collected in France. Median age was 56 years (range: 19-84). Most patients (78%) received ASC as a third-line or later treatment, with 93% previously treated with ponatinib. Twenty-seven out of 35 analyzed patients had BCR::ABL1 mutations, including T315I (n=18) or compound mutations (n=8). ASC was prescribed at 200 mg BID in 83% of the patients, alone (n=20) or in combination (n=21). Overall, 30/36 evaluable patients achieved a composite complete response (CR/CRi), including measurable residual disease negativity (defined by BCR::ABL1/ABL1 <0.01% in bone marrow) in 13/23 patients (57%). The median overall survival and event-free survival (EFS) were 9.8 and 4.9 months respectively. EFS was improved when ASC was administered in combination rather than as monotherapy (7.93 vs 4.23 months). Post-ASC mutations were analyzed in 7 relapsing patients, identifying a new Q252H mutation in 2 cases. In conclusion, our findings suggest that ASC is an effective salvage therapy for Ph+ ALL and LBC-CML.
Référence
Blood Adv. 2025 07 2;:
