TY - JOUR
T1 - The PedAL/EuPAL Project
T2 - A Global Initiative to Address the Unmet Medical Needs of Pediatric Patients with Relapsed or Refractory Acute Myeloid Leukemia
AU - Ceolin, Valeria
AU - Ishimaru, Sae
AU - Karol, Seth E.
AU - Bautista, Francisco
AU - Goemans, Bianca Frederika
AU - Gueguen, Gwenaëlle
AU - Willemse, Marieke
AU - di Laurenzio, Laura
AU - Lukin, Jennifer
AU - van Tinteren, Harm
AU - Locatelli, Franco
AU - Petit, Arnaud
AU - Tomizawa, Daisuke
AU - Norton, Alice
AU - Kaspers, Gertjan
AU - Reinhardt, Dirk
AU - Tasian, Sarah K.
AU - Nichols, Gwen
AU - Kolb, Edward Anders
AU - Zwaan, Christian Michel
AU - Cooper, Todd Michael
N1 - Funding Information: We thank the Leukemia and Lymphoma Society, the Gateway Foundation, the Lisa Dean Moseley Foundation, and AbbVie/Genentech/Roche for their financial support in the process of realizing and opening new trials, as well as the Princess Máxima Center, German Pediatric Oncology and Hematology (GPOH), and PRN. S.K.T. is a Scholar of the Leukemia and Lymphoma Society and holds the Joshua Kahan Endowed Chair in Pediatric Leukemia Research at the Children’s Hospital of Philadelphia. Publisher Copyright: © 2023 by the authors.
PY - 2024/1/1
Y1 - 2024/1/1
N2 - 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.
AB - 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.
KW - EuPAL
KW - PedAL
KW - acute myeloid leukemia
KW - clinical trials
KW - pediatric
KW - refractory/relapsed
UR - http://www.scopus.com/inward/record.url?scp=85182172854&partnerID=8YFLogxK
U2 - https://doi.org/10.3390/cancers16010078
DO - https://doi.org/10.3390/cancers16010078
M3 - Article
C2 - 38201506
SN - 2072-6694
VL - 16
JO - Cancers
JF - Cancers
IS - 1
M1 - 78
ER -