A European network facilitates access to precision medicine and clinical trials for children and adolescents with relapsed leukemia or lymphoma

Relapsed or refractory pediatric leukemias and lymphomas continue to represent one of the major challenges in childhood cancer. Despite the improvements in survival achieved over recent decades, these cases often have limited therapeutic options, and only a small proportion of patients gain access to clinical trials involving innovative treatments.

To improve patient access to targeted therapies, a group of European experts led by the Princess Máxima Center in Utrecht (the Netherlands) established the International Leukemia/Lymphoma Target Board (iLTB), a virtual platform for discussing therapeutic options for children and adolescents with high-risk leukemia or lymphoma. The results of this initiative have been published in the journal  Haematologica. Dr Pablo Velasco, attending physician in the Department of Pediatric Oncology and Hematology at Vall d’Hebron University Hospital and researcher in the Childhood Cancer and Blood Disorders group at the Vall d’Hebron Research Institute (VHIR), participated in this initiative as coordinator of the Spanish ReALLNet network together with Dr José Luis Fuster, Head of the Pediatric Hemato-Oncology Department at Virgen de la Arrixaca Hospital (Murcia).

The iLTB brings together clinical experts and researchers in leukemia and lymphoma, immunotherapy, hematopoietic stem cell transplantation, and clinical trials to review each case on an individual basis. Using patients’ clinical, molecular, and genetic data, the board identifies potential therapeutic targets and provides treatment recommendations to the medical teams responsible for each patient.

During its first year of operation, between early 2023 and early 2024, the board reviewed 42 cases from 18 European countries. All involved high-risk patients who had previously received treatment. After assessing each clinical situation, the panel recommended therapeutic approaches that differed from those initially proposed in many cases, primarily immunotherapies. The recommendations were implemented in 81% of cases.

Overall, 36% of patients were able to access either a clinical trial or a compassionate use programme, representing a threefold increase compared with previous figures. This finding highlights the importance of international networks in expanding the range of available therapeutic options. In nearly 80% of these cases, the pediatric patients participated in clinical trials or programmes outside their country of residence.

“Thanks to this international expert panel, we have improved access to innovative treatments for children and adolescents with high-risk leukemia and lymphoma, as well as their survival”, says Dr Velasco. “Precision medicine and international cooperation are essential to providing more opportunities for children experiencing leukemia or lymphoma relapse, both within and beyond their own country. Access to international clinical trials offers new therapeutic opportunities for patients who often have very few alternatives”, he adds.

The initiative has also highlighted the need to continue working to ensure that families have equitable access to international clinical trials, particularly when the available treatments are located in other European countries.

In this context, Vall d’Hebron currently has 16 phase I-II clinical trials for relapsed leukemia/lymphoma and benefits from the collaboration between VHIR and the Vall d’Hebron Institute of Oncology (VHIO), enabling access to adult clinical trials that also include pediatric patients.

Vall d’Hebron coordinates the ReALLNet network in Spain, which focuses on research into relapsed acute lymphoblastic leukemia. Within this network, genetic and functional studies of cases are carried out and discussed by experts to identify the best therapeutic options for each patient. In addition, the network helps identify the most complex patients who may subsequently be evaluated by the iLTB and potentially enrolled in international clinical trials. Of the approximately 80 cases reviewed by ReALLNet, around ten have already been discussed by the European committee.