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Pediatric Hematologic Malignancies

Pediatric Hematologic Malignancies


Pediatric Malignancies are the second leading cause of mortality in children in developed countries and hematologic malignancies (HMs) account for 40% of pediatric malignancies. Typical symptoms of pediatric HMs are fatigue and frequent infections. Acute lymphoblastic leukemia (ALL) is the most common HM in children; the other HMs that are studied in HARMONY are rare in children.

The treatment of childhood HMs has dramatically improved in the past few decades, largely as a result of improvements in protocol design, supportive care, and risk stratification. The contemporary five-year event-free survival is ~80% for ALL and ~60% for AML in children. Current treatment protocols are intensive, typically involving multiple chemotherapeutic drugs administered over several months or years. Stem cell transplantation is reserved for high-risk patients. Although many pediatric HM patients can be cured, the treatments are highly toxic and may cause substantial long-term morbidity.

Key questions

Many prognostic factors have already been identified for pediatric HMs, e.g., age of onset, gender, genetic constitution of the malignant cells, and minimal residual disease after chemotherapy. To a certain extent, these factors can predict outcomes such as the risk of relapse, but they have largely been studied in isolation so far.

By pooling data of thousands of patients, HARMONY researchers will be able to study combinations of prognostic factors, which may constitute risk profiles that can reliably predict clinical course and drug response, enabling physicians to rapidly select the most promising and least toxic treatment strategy for a particular patient.

Examples of key questions that HARMONY will address for pediatric HMs are:

Can we de-intensify chemotherapy in certain subgroups of patients?

Can we find risk profiles that identify non-responders?

What is the optimal cut-off age for treating a patient as a child or an adult?

Pediatric HM features

HARMONY Leadership: Pediatric HM