Sistema FMUSP-HC: Faculdade de Medicina da Universidade de São Paulo (FMUSP) e Hospital das Clínicas da FMUSPHOCHHAUS, AndreasREA, DelphineBOQUIMPANI, CarlaMINAMI, YosukeCORTES, Jorge E.HUGHES, Timothy P.APPERLEY, Jane F.LOMAIA, ElzaVOLOSHIN, SergeyTURKINA, AnnaKIM, Dong-WookABDO, AndreFOGLIATTO, Laura MariaCOUTRE, Philipp leSASAKI, KojiKIM, Dennis Dong HwanSAUSSELE, SusanneANNUNZIATA, MarioCHAUDHRI, NaeemCHEE, LynetteGARCIA-GUTIERREZ, ValentinKAPOOR, ShrutiALLEPUZ, AlexQUENET, SaraBEDOUCHA, VeroniqueMAURO, Michael J.2023-04-142023-04-142023LEUKEMIA, v.37, n.3, p.617-626, 20230887-6924https://observatorio.fm.usp.br/handle/OPI/53226Asciminib, the first BCR::ABL1 inhibitor that Specifically Targets the ABL Myristoyl Pocket (STAMP), is approved worldwide for the treatment of adults with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (CML-CP) treated with >= 2 prior tyrosine kinase inhibitors (TKIs). In ASCEMBL, patients with CML-CP treated with >= 2 prior TKIs were randomized (stratified by baseline major cytogenetic response [MCyR]) 2:1 to asciminib 40 mg twice daily or bosutinib 500 mg once daily. Consistent with previously published primary analysis results, after a median follow-up of 2.3 years, asciminib continued to demonstrate superior efficacy and better safety and tolerability than bosutinib. The major molecular response (MMR) rate at week 96 (key secondary endpoint) was 37.6% with asciminib vs 15.8% with bosutinib; the MMR rate difference between the arms, after adjusting for baseline MCyR, was 21.7% (95% CI, 10.53-32.95; two-sided p = 0.001). Fewer grade >= 3 adverse events (AEs) (56.4% vs 68.4%) and AEs leading to treatment discontinuation (7.7% vs 26.3%) occurred with asciminib than with bosutinib. A higher proportion of patients on asciminib than bosutinib remained on treatment and continued to derive benefit over time, supporting asciminib as a standard of care for patients with CML-CP previously treated with >= 2 TKIs.engrestrictedAccessadverse eventsmanagementrecommendationstherapyarticleCopyright SPRINGERNATURE10.1038/s41375-023-01829-9OncologyHematology1476-5551