Long-term outcome following cyclosporine-related neurotoxicity in paediatric allogeneic haematopoietic stem cell transplantation

With increasing numbers of children and adults undergoing allogeneic haematopoietic stem cell transplantation (HSCT), neurotoxicity is emerging as an important cause of transplant-related morbidity. 1 , 2 , 3 The calcineurin inhibitor cyclosporine A (CI CSA) is the most frequently used agent for prevention of GvHD in both adult and paediatric patients. CSA is an established cause of post-transplantation central nervous system (CNS) toxicity, typically characterised by posterior reversible encephalopathy syndrome (PRES). 4 Even though neurological complications of CSA seldom result in mortality, the necessitated withdrawal of this potent anti-GvHD agent can have major implications on clinical outcome particularly in the face of on-going GvHD. The spectrum of CSA-related neurological complications has been well characterised in several case series of paediatric HSCT recipients, 5 , 6 , 7 however, long-term follow-up data of those children who experience neurotoxicity are lacking. Here, we present the outcome of 26 children who developed CSA-neurotoxicity following allogeneic HSCT.