The PedAL/EuPAL Project: A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia

Valeria Ceolin, Sae Ishimaru, Seth E. Karol, Francisco Bautista, Bianca Frederika Goemans, Gwenaëlle Gueguen, Marieke Willemse, Laura Di Laurenzio, Jennifer Lukin, Harm van Tinteren, Franco Locatelli, Arnaud Petit, Daisuke Tomizawa, Alice Norton, Gertjan Kaspers, Dirk Reinhardt, Sarah K. Tasian, Gwen Nichols, Edward Anders Kolb, Christian Michel Zwaan*Todd Michael Cooper

*Corresponding author for this work

Research output: Contribution to journalArticleAcademicpeer-review

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Abstract

The prognosis of children with acute myeloid leukemia (AML) has improved incrementally over the last few decades. However, at relapse, overall survival (OS) is approximately 40–50% and is even lower for patients with chemo-refractory disease. Effective and less toxic therapies are urgently needed for these children. The Pediatric Acute Leukemia (PedAL) program is a strategic global initiative that aims to overcome the obstacles in treating children with relapsed/refractory acute leukemia and is supported by the Leukemia and Lymphoma Society in collaboration with the Children’s Oncology Group, the Innovative Therapies for Children with Cancer consortium, and the European Pediatric Acute Leukemia (EuPAL) foundation, amongst others. In Europe, the study is set up as a complex clinical trial with a stratification approach to allocate patients to sub-trials of targeted inhibitors at relapse and employing harmonized response and safety definitions across sub-trials. The PedAL/EuPAL international collaboration aims to determine new standards of care for AML in a first and second relapse, using biology-based selection markers for treatment stratification, and deliver essential data to move drugs to front-line pediatric AML studies. An overview of potential treatment targets in pediatric AML, focused on drugs that are planned to be included in the PedAL/EuPAL project, is provided in this manuscript.

Original languageEnglish
Article number78
JournalCancers
Volume16
Issue number1
DOIs
Publication statusPublished - 1 Jan 2024

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