Childhood Leukemias
Leukemia in children is similar to that in adults but may have some particularities. Acute leukemia is a type of cancer characterized by the dissemination of cancerous cells originating from the bone marrow stem cells. They multiply chaotically, invade the bone marrow, enter the bloodstream, and can thus invade the entire body. This proliferation hampers the production of all blood cells (white and red blood cells, platelets).
In 2023, this disease was the most common cancer in children (approximately 30% of pediatric cancer cases).
The two main types in children are acute lymphoblastic leukemia (80% of cases) and acute myeloid leukemia. The exact causes of leukemia in children are not always clearly defined, but it is thought that they may be related to genetic, environmental factors, or a combination of certain events occurring at the level of the bone marrow stem cells.
Learn more about leukemias: link
About the Project
Acute promyelocytic leukemia (APL) is a subtype of acute myeloid leukemia (AML) with specific clinical, morphological, and cytogenetic characteristics. They are designated as “acute leukemia M3,” associated with specific genetic alterations and coagulation abnormalities. They are characterized by the accumulation of myeloid cells blocked at the promyelocytic stage in the bone marrow and blood. The median age at diagnosis is similar to that of other AML subtypes (9-10 years), with very rare cases of APL diagnosed in the first two years of life.
While until the late 1980s, chemotherapy using anthracyclines and cytarabine was the only therapeutic option for patients with APL, the introduction of all-trans retinoic acid (ATRA) for the treatment of adults and children has led to increased remission rates and reduced morbidity and mortality. Despite the dramatic improvements achieved in first-line treatment of APL with ATRA and anthracycline-based treatments, relapses still occur in about 20% of patients, and these treatment regimens are associated with significant toxicities and late effects, particularly cardiac. In a recent randomized clinical trial conducted in adults with this disease, it was demonstrated that a combination of arsenic trioxide (ATO) and ATRA yielded better survival rates with significantly lower toxicity compared to standard treatment. These data indicate that patients with standard-risk APL can be cured without chemotherapy (with ATO/ATRA only).
Current treatment strategies for childhood APL aim to reduce the incidence of relapse and chemotherapy-induced toxicity. The introduction of ATRA has been crucial both for anti-leukemic efficacy and for reducing early mortality rates. Furthermore, the results obtained with the combination of ATRA and ATO in adults and reported cases in pediatric literature have led to the establishment of this European study.
The aim of this study is to demonstrate equivalent efficacy and safety of this chemotherapy-free treatment compared to standard protocols combining ATRA and chemotherapy. The trial will be open to all patients diagnosed with acute promyelocytic leukemia and aged under 18 years. It will be an international study, including the largest European pediatric groups, among which France. The study recruitment duration will be 36 months with a minimum patient follow-up of 2 years.
Project Progress:
- Start in September 2020
- 08/01/2022, 19 standard-risk patients and 12 high-risk patients had been enrolled in Italy (14) and France (5).
Project summary
- Promoter: CHU Bordeaux
- Principal Investigator: Dr. Stéphane Ducassou
- Program Duration: September 2020 – September 2024
- Number of Patients: 35 patients in 27 SFCE centers
- Countries Involved: French part of a trial coordinated by the Associazione Italiana di Ematologia ed Oncologia Pediatrica (A.I.E.O.P)
- Imagine for Margo Funding: €109,500.
This trial was co-financed by the Paris Heart Rally 2020 and selected by the SFCE.
