Who can participate
- Children, adolescents, and young adults diagnosed with relapsed or refractory hematological malignancies who are below the age of 25
- No standard of care treatment is available for them
Goal
To discuss the tumor profile with an international group of experts in the fields of biology and new drugs called the "international Leukemia/Lymphoma Target Board" (iLTB).
The tumor profile provides insight into the characteristics of the child's leukemia/lymphoma cells. This may include abnormalities of the leukemia cells (inside and/or at the cell surface) or sensitivity to a drug in laboratory tests. These characteristics may offer the possibility to treat the malignant cells differently with new drugs now that the child's disease is not responding to standard treatment options.
To this end, the platform evaluates the characteristics of the leukemia/lymphoma cells and treatments already given, and then advises on possible appropriate clinical trials or alternatives if a clinical trial is not available.
Background
The origin of many forms of (childhood) cancer lies in the biological properties of the leukemia/lymphoma cells. The biological characteristics of the leukemia/lymphoma cells are determined as part of standard diagnostic procedures (called 'tumor profile'). These biological features are unique to each patient and may explain why the disease progresses differently in each patient and why patients may respond differently to treatment.
The choice of treatment can be very difficult when the disease has become unresponsive (refractory) or has come back (relapse) and standard treatment options are lacking. Therefore, we want to discuss the child's leukemia/lymphoma profile with an international group of experts in the fields of biology, clinical trials, new drugs, and specific disease experts, called the international Leukemia/Lymphoma Target Board (iLTB).
Discussing the tumor profile offers the opportunity for some children to be treated with a drug that more closely targets their tumor-specific features. In recent years, more targeted therapies and new drugs have been developed. However, this is not yet accepted as standard of care treatment, and it is not always clear whether such a drug is effective to treat relapsing or refractory leukemia/lymphoma. This is because of the rarity of these diseases and the variation in biological features in each child's leukemia/lymphoma, or because the new drug has not yet been extensively tested in childhood leukemia/lymphoma.