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1496 European pediatric Acute Leukemia (EuPAL) Registry

Program: Oral and Poster Abstracts
Session: 613. Acute Myeloid Leukemias: Clinical and Epidemiological: Poster I
Hematology Disease Topics & Pathways:
Research, Clinical Research, pediatric, Diseases, therapy sequence, Therapies, registries, Myeloid Malignancies, Human, Study Population
Saturday, December 9, 2023, 5:30 PM-7:30 PM

Katarzyna Majstrowicz, PhD1,2*, Katharina Waack1*, Evangelia Antoniou, MD3*, Naghmeh Niktoreh, MD4*, Gwen Nichols, MD5, Laura Di Laurenzio, PhD6*, Michele S. Redell, MD7, Todd Cooper, DO8, E. Anders Kolb, MD9, Sarah K Tasian, MD10, Franco Locatelli, MD, PhD11, C. Michel Zwaan, MD12,13* and Dirk Reinhardt, MD2,4

1Pediatric Research Network, Essen, Germany
2AML-BFM Study Group, Essen, Germany
3University Children’s Hospital Essen. Department of Pediatric Hematology and Oncology, Essen, Germany
4Department of Pediatrics III, University Children’s Hospital Essen, University of Duisburg-Essen, Essen, Germany
5The Leukemia & Lymphoma Society, Rye Brook, NY
6Leukemia & Lymphoma Society, Rye Brook, NY
7Texas Children's Cancer Center, Baylor College of Medicine, Houston, TX
8Division of Pediatric Hematology, Oncology, Bone Marrow Transplant & Cellular Therapy, Seattle Children's Hospital, Seattle, WA
9Leukemia & Lymphoma Society, Bronx, NY
10Children's Hospital of Philadelphia, Philadelphia, PA
11Department of Pediatric Hematology/Oncology, Cell and Gene Therapy, IRCCS Bambino Gesù Children’s Hospital, Catholic University of the Sacred Heart, Rome, Italy
12Prinses Máxima Center for Pediatric Oncology Research, Utrecht, Netherlands
13Erasmus University Medical Center, Rotterdam, Netherlands

The Leukemia Lymphoma Society (LLS) Pediatric Acute Leukemia (PedAL) Initiative is a collaborative international effort that aims to improve the cure of children with relapsed/refractory (R/R) and other high-risk leukemias. Its comprehensive approach included preclinical and clinical research, standardization and improvement of diagnostics, quality management, and global data integration and analysis. The European Pediatric Acute Leukemia (EuPAL) Foundation was established as the PedAL Initiative partner to support European stakeholders in these efforts.

A major focus of the EuPAL Registry and PedAL Initiative is to increase access to and test new drugs targeting leukemia-specific biologic and genetic characteristics in pediatric patients with R/R acute myeloid leukemia (AML). However, identification and recruitment of patients remains a challenge. For this purpose, the APAL2020SC screening trial (NCT04726241) in North America, Australia, and New Zealand and the EuPAL registry in Europe have been launched. In collaboration with national and international study groups, pediatric leukemia reference laboratories, and participating clinical trial sites, each child with R/R AML should be registered. In Europe, the EuPAL registry office identifies the best available trial for the patient based upon leukemia-associated genetic and immunophenotypic data and closest trial site(s) and coordinates contact between the treating physician/site and the trial site as needed.


The EuPAL registry has been approved by the leading ethic committee in Germany with extension to several other European countries ongoing. To date, 35 children and adolescents with R/R AML have been enrolled since June 2022.

The median age at initial diagnosis was 7.3 (range 0.2 to 17.7) years with median duration of remission 1.3 (0.3 to 5.3) years. Most patients were registered at first relapse (n=29) with 3 patients in second relapse and 1 patient in third relapse. Two children were diagnosed with a molecular relapse (n=2). Fourteen patients have been transplanted in 1st CR (n=9) /2nd CR (n=5) prior to registration

Regarding the initial risk groups at diagnosis, most relapses occurred in the intermediate risk group (IR) n=19) and the high-rik group (HR; n=12). The others were standard risk, one isolated CNS relapse in APL and two in children with trisomy 21.

Patients were treated at relapse on the AML-BFM 2017 registry (treatment recommendation: Idarubicin, fludarabine, cytarabine (IDA-FLA), FLA and HSCT), the AIEOP-BFM-AML 2020 trial (FLA- gemtuzumab ozogamicin (GO) or IDA-FLA t(8;21)), the AMORE 2017 trial (azacytidine and HSCT; n=2) or received off-label therapies (cytarabine/GO, venetoclax/cytarabine, or azacytidine).


The EuPAL registry has been successfully established and is beginning to generate relevant knowledge regarding the frequency and timing of pediatric AML relapse, leukemia-associated genetics, and immunophenotypic characteristics. In collaboration with the LLS PedAL Initiative, the EuPAL registry will continue to facilitate recruitment of children with R/R AML and other leukemias to clinical trials testing innovative therapies, as well as improve understanding of potential bias in clinical trial referrals. Current challenges include integration of more European countries into the EuPAL Registry and increasing the number of open early-phase PedAL/EuPAL clinical trials.

Disclosures: Tasian: Kura Oncology: Membership on an entity's Board of Directors or advisory committees, Research Funding; Aleta Biotherapeutics: Membership on an entity's Board of Directors or advisory committees; Amgen: Other: travel support ; Beam Therapeutics: Research Funding; Incyte Corporation: Research Funding; Syndax Pharmaceuticals: Membership on an entity's Board of Directors or advisory committees. Zwaan: Novartis: Consultancy, Other: Advisory role; Incyte: Consultancy; Jazz Pharmaceutical: Research Funding; Takeda: Research Funding; Pfizer: Consultancy, Research Funding; Abbvie: Research Funding; Kura Oncology: Consultancy; Abbvie: Research Funding; Syndax: Research Funding; Gilead: Consultancy; BMS: Consultancy; Sanofi: Other: Advisory role; ITCC Hem Malignancies Committee: Membership on an entity's Board of Directors or advisory committees. Reinhardt: Medac: Consultancy, Research Funding, Speakers Bureau; Eusa: Speakers Bureau; Cerus: Membership on an entity's Board of Directors or advisory committees, Speakers Bureau; BluebirdBio: Research Funding, Speakers Bureau; MSD: Speakers Bureau; Immedica: Consultancy, Membership on an entity's Board of Directors or advisory committees; Novartis: Honoraria; JAZZ Pharmaceutical: Research Funding.

OffLabel Disclosure: venetoclax azacitidin Gemtuzumab Ozogamicin ==> treatment recommendation in ped AML

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