Samples from 34 adult patients newly diagnosed with core binding factor leukemia (CBFL) were collected both at the time of diagnosis and at relapse and were centrally analyzed. Eligible patients received either standard induction CT known as “3 + 7” or an equivalent regimen, according to the recruiting center's policy. Patients who achieved CR or CRi received 3 courses of high-dose ARA-C (Cytarabine) 3000 mg/m2 every 12 h on days 1, 3, and 5, along with midostaurin at the dose of 50 mg b.i.d from Day 8 to Day 21 as part of consolidation therapy. Following the completion of the consolidation phase, patients received midostaurin as a monotherapy at the dose of 50 mg b.i.d. for 1 year as continuation therapy. The CR rate was 97%; we recorded an OS rate of 73.52% and a DFS rate of 48.4% for the entire cohort. The RI was 38.8% in the CBFB::MYH11 and 66.6% in the RUNX1::RUNX1T1 group. MRD (Measurable Residual Disease) was assessed by RQ-PCR at 10 time points throughout the study, as indicated by arrows.

Efficacy of Midostaurin Combined with Intensive Chemotherapy in Core Binding Factor Leukemia: A Phase II Clinical Trial / R. Cairoli, A. Gatti, G. Grillo, M.R. Stefanucci, B. Di Camillo, M. Fumagalli, M. Krampera, G. Nadali, I.P. Zappasod, E. Borlenghi, E. Todisco, M. Ubezio, M. Bernardi, A. Molteni, C. Basilico, M. Turrini, R. Greco, V. Mancini, M. Riva, D.P. Bernasconi, B. Brando, S.M. Veronese, A. Beghini. - In: AMERICAN JOURNAL OF HEMATOLOGY. - ISSN 0361-8609. - 100:2(2025 Feb), pp. 27547.346-27547.349. [10.1002/ajh.27547]

Efficacy of Midostaurin Combined with Intensive Chemotherapy in Core Binding Factor Leukemia: A Phase II Clinical Trial

A. Beghini
Ultimo
2025

Abstract

Samples from 34 adult patients newly diagnosed with core binding factor leukemia (CBFL) were collected both at the time of diagnosis and at relapse and were centrally analyzed. Eligible patients received either standard induction CT known as “3 + 7” or an equivalent regimen, according to the recruiting center's policy. Patients who achieved CR or CRi received 3 courses of high-dose ARA-C (Cytarabine) 3000 mg/m2 every 12 h on days 1, 3, and 5, along with midostaurin at the dose of 50 mg b.i.d from Day 8 to Day 21 as part of consolidation therapy. Following the completion of the consolidation phase, patients received midostaurin as a monotherapy at the dose of 50 mg b.i.d. for 1 year as continuation therapy. The CR rate was 97%; we recorded an OS rate of 73.52% and a DFS rate of 48.4% for the entire cohort. The RI was 38.8% in the CBFB::MYH11 and 66.6% in the RUNX1::RUNX1T1 group. MRD (Measurable Residual Disease) was assessed by RQ-PCR at 10 time points throughout the study, as indicated by arrows.
Core-Binding Factor Leukemia; AML; Midostaurin; KIT; FLT3
Settore BIOS-08/A - Biologia molecolare
Settore MEDS-09/B - Malattie del sangue
feb-2025
10-dic-2024
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/2434/1119290
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