SUMMARY
Despite improvements in the outcome of newly diagnosed paediatric haematological malignancies, the prognosis of refractory and high-risk relapsed patients remains poor, and their treatment is challenging. The increased use of molecular and functional profiling technologies and the growing number of targeted therapies provide opportunities to alter the treatment landscape of these patients and offer the potential to improve patient outcomes. However, this approach comes with many challenges. First, there is a need for accessibility to the analysis and interpretation of sequencing technologies. Second, the prioritisation of targetable events and matching treatments needs to be established. Third, there is a need to facilitate access to early-phase clinical trials. Recently, the international Leukaemia/Lymphoma Target Board (iLTB, NCT05270096, ITCC107) was initiated by the Prinses Máxima Centre and developed in collaboration with the international community to address these barriers. The iLTB provides a forum for an international panel of experts to advise the treating physicians on the best possible treatment options for their patients diagnosed with relapsed or refractory haematological malignancies, for which there is no standard treatment option. In addition, the iLTB aims to facilitate enrolment in clinical trials and collect structured real-world data on patients treated with compassionate use programs. The purpose of this article is to discuss the current challenges in relapsed and refractory haematological malignancies and to introduce the use of international tumour boards to help physicians when confronted with difficult cases.
(BELG J HEMATOL 2024;15(7):263–8)
