PEDIATRICS

G.Y. Zhang, R. Spicer, S. Mctaggart, L.P. Roy, S.I. Alexander
Centre For Kidney Research, The Children's Hospital at Weastmead, Weastmead/NSW/AUSTRALIA

Body: INTRODUCTION: Cystinosis is an autosomal recessive lysosomal storage disease resulting from intracellular cystine accumulation. This severe progressive multi-system disease causes renal complications in childhood, including progressive renal failure with subsequent need for renal replacement therapy. Early, continual and consistent oral cysteamine treatment has resulted in improved outcomes with demonstrated slower deterioration in renal glomerular function, although paediatric patients continue to need transplantation. This study investigates outcomes following transplantation of such patients. METHOD: Demographic and transplant-related data obtained from ANZDATA registry for patients with cystinosis between 1981 and 2008. Results dichotomised in to early group (EG) pre-cysteamine (1981-1994) and later group (LG) post-cysteamine availability (1995-2008). RESULTS: Since 1981, 44 renal transplants have been performed in 36 children with cystinosis in Australia and New Zealand. 20 (56%) were male. 14 (39%) were pre-emptively transplanted, 2 received HD and 21 peritoneal dialysis prior to transplantation. RRT commenced at 10.6years (EG) 12.3years (LG). Transplantation occurred at mean age of 11.8 yrs (EG) and 12.7years (LG) with 11/17 (65%)(EG) were transplanted pre-emptively; with 2/19 (10%)(LG) pre-emptively. 7 (19%) of recipients had two transplants. 21 (58%) were alive with a functioning graft, 16 lost their graft, 11 died of whom 6 had a functioning graft. 5 children (14%) experienced rejection (9 episodes). CONCLUSIONS: More recent paediatric transplant recipients with cystinosis have delayed RRT and transplantation following cysteamine introduction. Rejection was limited in keeping with reduced immune function found in cystinosis patients.

Disclosure: All authors have declared no conflicts of interest.