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Acute Lymphoblastic Leukemia

ALL: Acute Lymphoblastic Leukemia


Acute Lymphoblastic Leukemia (ALL) is a life-threatening disease if not treated immediately. It arises from hematopoietic stem cells in the bone marrow that have acquired genomic lesions. The non-functional malignant cells proliferative at the expense of healthy cells, resulting in reduced numbers of platelets and red & white blood cells (pancytopenia).

The disease has a rapid onset and easily spreads to other organs. The treatment of ALL has dramatically improved in the past few decades, largely due to improvements in protocol design, supportive care, and risk stratification. The five-year event-free survival currently is more than 80% for children and 30-70% adults largely depending on age.  ALL treatment protocols are intensive, typically involving multiple chemotherapeutic drugs administered over several months or years. The drugs are toxic and may cause substantial long-term morbidity particularly in pediatric patients. Stem cell transplantation is reserved for fit high-risk patients. In contrast to the other hematologic malignancies that are studied in HARMONY, ALL occurs more frequently in children than adults.

Key questions

As ALL constitutes a rare disease, it is crucial to perform multicenter research. By pooling data from multiple clinical trials, HARMONY researchers aim to create a dataset of ~20,000 ALL patients spanning the entire age range from infants to older patients. We aim to identify 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 ALL include:

Can we identify risk profiles that predict clinical course and drug response?

What is the disease pathophysiology, disease course, and treatment outcome in subsets of ALL?

Can we harmonize the measurement of outcomes in ALL trials?

ALL Features

HARMONY Leadership: ALL