Relapsed pediatric acute myeloid leukaemia: state-of-the-art in 2023

Abstract

Although outcomes for children and adolescents with newly-diagnosed acute myeloid leukaemia (AML) have improved significantly over the past two decades, more than one-third of patients nonetheless continue to relapse and experience suboptimal long-term outcomes. Given the small numbers of patients with relapsed AML and historic logistical barriers to international collaboration including poor trial funding and drug availability, management of AML relapse has varied amongst paediatric oncology cooperative groups with several utilised salvage regimens and a lack of universally-defined response criteria. The landscape of relapsed paediatric AML treatment is rapidly changing, however, as the international AML community harnesses collective knowledge and resources to characterize the genetic and immunophenotypic heterogeneity of relapsed disease, identify biologic targets of interest within specific AML subtypes, develop new precision medicine approaches for collaborative investigation in earlyphase clinical trials, and tackle challenges of universal drug access across the globe. This review provides a comprehensive overview of progress achieved to date in the treatment of paediatric patients with relapsed AML and highlights modern, state-of-the-art therapeutic approaches under active and emerging clinical investigation that have been facilitated by international collaboration amongst academic paediatric oncologists, laboratory scientists, regulatory agencies, pharmaceutical partners, cancer research sponsors, and patient advocates.