Chronic leukemia in children: focus on targeted therapies
On World Chronic Leukemia Day for children, we take a look at a major scientific breakthrough from the 2000s: targeted therapies.
These treatments have revolutionized the care of children with leukemia. While chronic leukemia is extremely rare in Belgium, accounting for just 3 to 5% of pediatric leukemia cases, it presents a significant medical challenge. Thanks to targeted therapies, the prognosis for young patients at the Children’s Hospital (HUDERF) has improved dramatically, further establishing the hospital as a key reference center in pediatric oncology.
These treatments work by precisely blocking the protein responsible for the uncontrolled growth of leukemia cells, while sparing healthy cells
A rare and complex disease
Unlike acute leukemia, which progresses quickly, chronic leukemia develops more slowly. However, without the proper treatment, it can be just as serious. Chronic myeloid leukemia (CML), the most common form in children, is linked to a genetic abnormality known as the Philadelphia chromosome. Only a handful of cases are diagnosed each year in Belgium, but the complexity of the disease requires highly specialized care.
From bone marrow transplants to targeted therapies
For many years, bone marrow transplants were the only treatment option for children with CML. However, in the early 2000s, the introduction of targeted therapies revolutionized the way CML is treated. These innovative drugs, especially tyrosine kinase inhibitors, provide a less invasive alternative in the form of oral medication, with outstanding results for young patients treated in the pediatric hemato-oncology department at HUDERF.
Personalized follow-up
While targeted therapies are highly effective, they require close and regular monitoring to adjust the dosage for each patient. Since 2016, children treated at HUDERF have been invited to participate in the International Pediatric CML Registry. The goal of this registry is to deepen our understanding of the disease in young patients, identify prognostic factors that influence treatment outcomes, and track the long-term side effects of these therapies. This initiative helps refine treatments, ensuring they are more precisely tailored to each child’s needs.
Targeted therapies: a revolution in treating chronic leukemia
The arrival of targeted therapies has truly transformed how chronic leukemias are managed. Unlike traditional chemotherapy, these treatments work by precisely blocking the protein responsible for the uncontrolled growth of leukemia cells, while sparing healthy cells. Their oral administration makes them far less invasive and much better tolerated, significantly reducing side effects. These therapies not only improve the prognosis for patients but also enhance the overall quality of life for children with chronic leukemia.
Support research!
Ongoing research promises to further improve cancer treatments, developing therapies that are even more precise and effective. Supporting organizations like Kick Cancer, which play a crucial role in driving these advances, is essential. This year, you can contribute to research by joining the Run to Kick event, taking place in Bois de la Cambre on September 29. Don't forget to cheer on the HUDERF team, Wakili, who are running to support this vital cause.