Registry Study on Systematic Data Collection for Children and Adolescents with Acute Myeloid Leukemia

Acute leukemias are rapidly progressing, aggressive forms of cancer that arise from immature white blood cells or bone marrow cells and usually spread very quickly. The malignant leukemia cells (blasts) develop in the bone marrow and, among other things, displace the healthy bone marrow, preventing the production of sufficient numbers of functional blood cells. In cases of the disease, patients frequently experience infections, bleeding, reduced physical performance, and shortness of breath. In acute myeloid leukemia (AML), the precursors of the so-called myeloid cell line of leukocytes are abnormal. Overall, AML is a rare disease—though it is the second most common type of blood cancer (leukemia) in children.

There are various subtypes of AML that differ based on genetic characteristics—that is, depending on the specific changes in the cancer cells’ genetic material. By determining these characteristics precisely, doctors aim to tailor the treatment and address the individual disease as specifically as possible. Treatment for AML typically involves intensive chemotherapy, which is divided into several phases: induction, consolidation, and sometimes maintenance therapy. Various chemotherapy drugs are used in this process. Chemotherapy drugs act as cytotoxins. They destroy not only cancer cells but also healthy cells in the body. This can lead to severe side effects—such as organ damage or an increased risk of infections.

The goal of this study is to record all cases of acute myeloid leukemia (AML) in children and adolescents treated at the participating hospitals and in the participating countries. The aim is to gain an accurate overview of how frequently the disease occurs and how it is treated. In addition, the study examines the specific characteristics of the leukemia cells in patients. These include changes in the genetic material (genetic) and in gene expression (molecular/epigenetic). This information helps to better understand the disease and improve treatment in the long term. Another goal of the study is to determine whether tiny amounts of leukemia cells remain in the body after treatment. This is called minimal residual disease (MRD). MRD testing is performed once a month on blood samples to identify whether there is a risk of relapse. Overall, the study aims to help better understand the disease, detect relapses earlier, and further improve treatment for children and adolescents with AML in the future. The study is purely observational; only patient data is collected. It is important to continue researching these genetic and molecular characteristics in order to further improve the chances of a cure and to make therapies even more personalized and less invasive.

Children and adolescents up to age 18 who have a form of AML are eligible to participate in the study.