ESPN 50th Annual Meeting

ESPN 2017

Chronic Kidney Disease and Renal Transplant Outcomes in Patients with Cystinosis
Elenberg Ewa 1

1- Texas Children’s Hospital, Baylor College of Medicine

North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) was stablished in 1987 with the goal to study transplant patients (pts). In 1992 the database was expanded to include dialysis pts and since 1994, data collection began on pts with CKD.  As of 2015, the NAPRTCS has data on over 19,000 children with CKD, on dialysis and with renal transplants. Cystinosis is a rare genetic disease. There are little data on outcomes of CKD, dialysis and kidney transplantation in cystinosis pts. 


Material and methods:

Retrospective analysis of NAPRTCS database of registered cystinosis pts with CKD, dialysis and renal transplant. Data on cystinosis pts are compared to other pts in NAPRTCS registry who have non-recurrent, ie non-immunologic forms of potential graft loss. Specific aims were to describe the natural history of cystinosis, to investigate potential differences in CKD, dialysis and transplant pts.  


CKD data from 117 cystinosis pts were compared to 2109 pts with CKD from other causes. There was a statistical significance in growth delay in cystinosis pts and use of growth hormone (rGH). Cystinosis pts have significantly lower blood pressure (BP), poorer height Z-score, lower calcium, phosphate and higher alkaline phosphatase levels. Cystinosis pts progressed to ESRD faster than other pts with CKD.  There were no statistically significant differences in gender, serum creatinine, bicarbonate, PTH level, anemia and erythropoietin use. Dialysis data from 95 cystinosis pts were compared to 1472 dialysis pts from other causes. There was statistical significance in BP, height and weight Z-score, growth deficit, the use of rGH, calcium and PTH levels. Dialysis survival was better in cystinosis pts. There is no statistical difference in gender, dialysis modality, anemia, erythropoietin use, albumin, phosphorus level and time to transplant while on dialysis. Data obtained from 231 transplant pts with cystinosis vs. 2844 other causes, revealed statistical significance in height and weight Z-scores. There was no statistical difference in gender, graft survival, donor source (LRD vs. DD), preemptive transplant, time to first rejection, number of transplants.


CKD and dialysis cystinosis pts differ from other CKD and dialysis pts. Cystinosis pts progress to ESRD faster than other pts, growth deficit in cystinosis pts is significantly worse and rGH is more commonly used. Calcium and PTH levels are lower and alkaline phosphatase is higher in CKD cystinosis pts. Dialysis survival in cystinosis pts is better. Transplant cystinosis patients have no difference in donor source, preemptive transplant, graft survival and time to first rejection