Improved diagnostics and survival for all children with Acute Myeloid Leukemia treated within the NOPHO-DB-SHIP consortium; a cross-European collaboration

Call
EU4H-2021-PJ-02
Investigador principal
Jose Maria Fernandez Navarro
Role
Participant
Year
2022

We hereby apply for funding for the practical implementation of front-line genetic diagnostics and targeted treatment for children with acute myeloid leukemia (AML) within our consortium. AML is a severe form of blood cancer and despite intensive chemotherapy, treatment results are dissatisfactory. Through recent advances in risk-adapted treatment, based on 1) the cytogenetic basis of the disease and 2) treatment response as measured by MRD (minimal residual disease), survival rates are typically around 70%.

AML in children is rare and necessitates international collaborations to improve outcome. NOPHO countries (Nordic Society Pediatric Oncology Hematology) Sweden, Denmark, Finland, Norway, Iceland, Estonia, Lithuania, Latvia, together with the Netherlands, Belgium and Hong Kong, started the AML12 treatment protocol year 2012. Since 2012, Spain, Israel and Portugal have joined and currently our consortium is named NOPHO-DB-SHIP. Central in AML12 was cytogenetic risk-group allocation and advanced MRDbased treatment adaptation, resulting in an unprecedented survival of 80% for patients with access to the full diagnostic workup. Herein, we present the successor of AML12; the CHildhood International Protocol - Acute Myeloid Leukemia 2022 (CHIP-AML22). CHIP-AML22 aims to consolidate cytogenetic and flow MRD risk-adaptation and make this available across all participating countries.

In addition, based on the lessons learned from AML12, more advanced cytogenetic profiling of disease will be implemented with the aim to improved risk-stratification. Further, we aim that next generation sequencing (NGS) will be used to guide novel tailored and targeted treatment for a subset of patients, as well as to further optimize MRD-analysis. In many participating countries NGS techniques are not performed as standard of care. We aim for a cross-consortium implementation of these techniques and treatments to allow equal access to optimal care for all disease children with AML.