Acute myeloid leukaemia (AML) is the most common acute leukaemia in adults, and approximately 25% of all newly diagnosed cases carry the FLT3-internal tandem duplication (ITD) gene mutation.
FLT3- ITD is a driver for disease progression and high leukaemic burden and is associated with an unfavourable prognosis including shorter overall survival and increased risk of relapse.
Quizartinib is a next-generation highly potent and specific type II FLT3 inhibitor being developed specifically for FLT3-ITD positive AML and has previously shown single-agent activity in patients with relapsed/refractory disease.
The QuANTUM-First international, randomized, double-blind, placebo controlled phase 3 study sought to determine whether the addition of quizartinib to standard induction and post-remission consolidation therapy followed by single-agent continuation of quizartinib for up to three years improved overall survival in patients with newly diagnosed FLT3-ITD positive AML.
The results showed that the addition of quizartinib significantly improved overall survival compared with standard induction and consolidation therapy alone.
The median overall survival was 31.9 months for patients receiving quizartinib and 15.1 months for patients receiving standard treatment alone.
Quizartinib showed a safety profile consistent with what has previously been observed.
Rates of grade ≥3 neutropenia, grade 3/4 prolonged QT on electrocardiogram, and discontinuations due to adverse events were higher in quizartinib-treated patients.
In summary, quizartinib added to standard induction and consolidation therapy and continued as monotherapy for up to three years resulted in statistically significant and clinically meaningful improvement of overall survival in adult patients with newly diagnosed FLT3-ITD positive AML.
Watch Dr Erba's press conference here
Watch Dr Erba's interview on the study here