Short summary

This study aims to optimise dexamethasone treatment in childhood Acute Lymphoblastic Leukaemia (ALL) by investigating its pharmacokinetics, including penetration into cerebrospinal fluid (CSF). By analysing blood and CSF samples, and monitoring bone mineral density, the study will provide data to personalise dosing, reduce toxicities, and improve outcomes. The research involves collaboration across Danish paediatric oncology departments.

Rationale

The clinical relevance of this project lies in its potential to significantly improve the treatment outcomes for children with Acute Lymphoblastic Leukaemia (ALL), the most common type of childhood cancer. While survival rates for ALL have improved markedly in recent decades, the use of dexamethasone-a key component of ALL treatment protocols-presents significant challenges due to its associated toxicities and long-term side effects. These include bone mineral density loss and other severe complications, which can adversely affect the quality of life for survivors. Currently, the optimal administration of dexamethasone in children with ALL is not well understood, particularly its penetration into the cerebrospinal fluid (CSF), which is critical for effectively treating central nervous system (CNS) leukaemia. Given that 25% of ALL patients present with CNS involvement at diagnosis, understanding dexamethasone's pharmacokinetics, including its CSF penetration, is essential for ensuring that treatment is both effective and safe. This study seeks to address these gaps by conducting a population-based prospective cohort study involving children aged 1-17.9 years who are diagnosed and treated for ALL at one of the four Danish paediatric oncology departments. By systematically monitoring dexamethasone levels in blood and CSF, as well as assessing bone mineral density and other toxicities, this research will generate valuable data that can be used to personalise treatment. The goal is to tailor dexamethasone dosages to each patient's unique needs, minimising toxicities while maintaining therapeutic efficacy. The findings from this study have the potential to directly influence clinical practice, leading to more individualised treatment protocols that reduce the risk of relapse and the burden of long-term side effects.

Description of the cohort

Children aged 1-17.9 years diagnosed and treated for ALL at one of the four Danish paediatric oncology departments at Copenhagen University Hospital, Aarhus University Hospital, Odense University Hospital and Aalborg University Hospital.

Data and biological material

Blood samples Cerebrospinal fluid DXA-scan Questionare data Data from the patient journal

Collaborating researchers and departments

Department of Paediatrics and Adolescent Medicine, Aarhus University Hospital

• Karen Schow Jensen
• Birgitte Klug Albertsen