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Data of (young) children is generally underrepresented in research, with dosing regimens often extrapolated from adults, showing high pharmacokinetic (PK) variability with body weight-based or body surface area-based dosing regimens. This potentially result in suboptimal treatment, such as the occurrence of adverse effects, insufficient efficacy and adherence issues. For the immunosuppressive treatment with gold-standard drugs in children, there remains considerable room for improvement. Refinement of current dosing strategies could directly or indirectly reduce toxicity, optimise adherence and improve efficacy in children with autoimmune diseases and in the setting of paediatric haematopoietic cell transplantation. 

This thesis focused on treatment refinement and enhancing the use of gold-standard immunosuppressive therapies in children with immune diseases and in paediatric haematopoietic cell transplantation (HCT) and concluded that the current dosing strategy of gold-standard immunosuppressants in children needs to be evaluated and refined, since the standard dosing regimens result in suboptimal treatment in a part of paediatric patients. Pharmacokinetic-pharmacodynamic research in children is still highly needed, as paediatric data is generally underrepresented in studies. This will contribute to the ultimate goal: reduce toxicity, improve efficacy and optimise adherence in children with immunosuppressive therapies.