Steroid-sensitive nephrotic syndrome The road towards better understanding and outcomes

Steroid-sensitive nephrotic syndrome (SSNS) is a rare disease of childhood characterised by the urinary loss of proteins, low serum albumin, and oedema. Yet, the exact aetiology remains unknown. Corticosteroids have been the cornerstone of initial treatment, still relapses are common (80%). Levamisole is considered a perfect candidate to prevent relapses of first onset SSNS. This anthelminthic drug has relatively few side-effects and has already shown its efficacy in relapsing SSNS.
This thesis focuses on improving our understanding and the outcomes of first onset SSNS in children, with a special focus on the steroid-sparing drug levamisole. This was achieved by taking a closer look at the epidemiology, assessing the psychological impact of the diagnosis and initial treatment of SSNS on children and their parents, and increasing our knowledge in terms of mechanism of action and pharmacokinetics of two common drugs used the treatment of SSNS (prednisolone and levamisole). Last, the study protocol of an ongoing large randomised controlled trial to evaluate the efficacy and safety of levamisole in the prevention of relapses of first onset SSNS is presented.