Improved outcomes for leukemia in young patients (ages 0–45) through personalized therapies that offer greater safety and fewer side effects

Acute lymphoblastic leukemia (ALL) is a malignant disease of the lymphocytes, a subtype of white blood cells. It involves the uncontrolled production of malignant blasts in the bone marrow and blood. The blasts may be B lymphocytes or T lymphocytes. Blasts are an immature and abnormal form of white blood cells. The overproduction of blasts disrupts normal blood formation and the immune system. Many patients have swollen lymph nodes and an enlarged spleen. The exact cause of ALL is not fully understood, but genetic mutations and environmental factors play a role. For example, errors in cell division can cause parts of one chromosome to be transferred to another. Treatment typically consists of several drugs with very different mechanisms of action. The goal is to cure the disease. Ideally, a so-called molecular remission is achieved, in which no abnormal cells are detectable even at the molecular level.

The goal of the ALLTogether1 study is to evaluate the results of previous treatment protocols and, using an adapted protocol, to improve survival rates and reduce treatment side effects by tailoring therapy to each patient’s individual risk of relapse. The study will assess how patients are doing after 5 years, whether and what side effects have occurred in the meantime, and how effective the therapy has been. The study does not fundamentally investigate any specific, novel, or as-yet-unapproved medications; however, it does examine, for example, how the use of a specific class of drugs—tyrosine kinase inhibitors (TKIs), such as imatinib—can be optimized. Building on this study, standardized diagnostic procedures, registration systems, and risk-based treatments will be established. In the long term, this study aims to facilitate the planning of new experimental therapy trials. By participating in the study, relevant data regarding treatment will be integrated into an international database. This includes, among other things, recording patients’ risk profiles, which are used to select a specific treatment. Subsequently, the effectiveness of the treatment and the occurrence of side effects will be monitored over the long term.

Women and men between the ages of 0 and 45 with newly diagnosed ALL are eligible to participate in this study; in the case of B-ALL, this refers to the so-called precursor B-ALL variant. Patients with so-called mature B-ALL or a positive Philadelphia chromosome, as well as pregnant or breastfeeding women, are not eligible to participate.