Abstract: SA-PO041
Outcomes of Dialysis and Transplantation in Cystinosis from NAPRTCS Database
Session Information
- Transplantation: Clinical Outcomes
October 27, 2018 | Location: Exhibit Hall, San Diego Convention Center
Abstract Time: 10:00 AM - 12:00 PM
Category: Transplantation
- 1802 Transplantation: Clinical
Author
- Elenberg, Ewa, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, United States
Background
Cystinosis (Cys) is a rare genetic disease. There are no sufficient published data on outcomes of dialysis and kidney transplantation in Cys patients (pts). North American Pediatric Renal Trials and Collaborative Studies (NAPRTCS) was established in 1987 with the goal to study transplant pts. The database was expanded in 1992 to include dialysis pts. As of 2015, the NAPRTCS has data on over 7039 dialysis and 18225 transplant pts.
Methods
Retrospective analysis of NAPRTCS database of registered dialysis and renal transplant cystinosis pts. Data on Cys pts are compared to other registry pts with non-recurrent, i.e. non-immunologic causes (NIC) of potential graft loss. Specific aims were to investigate potential differences among studied dialysis and transplant pts.
Results
Dialysis data from 95 Cys pts were compared to 1472 NIC dialysis pts. There was statistical significance in blood pressure (BP), height and weight Z-Score, growth deficit, the use of rGH, calcium and PTH levels. Dialysis survival was better in Cys pts (p-value 0.046). There is no statistical difference in gender, dialysis modality, anemia, erythropoietin use, albumin, phosphorus level and time to transplant while on dialysis. Transplant data from 231 transplant Cys pts were compared to 2844 transplant NIC pts. Data was collected in 6 months (mo) interval from baseline at 30 days post transplant (p/Tx) to 60 mo p/Tx. Baseline height Z-Score was low in both groups: with mean Z-Score in NIC -2 and in Cys – 3.39 (p-value<0.005). Though improved height Z-Score at 60 mo p/TX to mean -1.84 in NIC, vs.-2.92 in Cys, the difference remains statistically significant. Weight Z-Score between 2 groups remains statistically significant with baseline mean at -1.22 for NCI vs. -2.59 for Cys. Although weight Z-score is gradually improving over time in both groups, at 60mo p/Tx mean Z-Score for NIC is -0.33 vs Cys -1.68 (p-value <0.005). There was no statistical difference in gender, creatinine levels, systolic and diastolic blood pressure Z-Scores, graft survival, donor source (LRD vs. DD), preemptive transplant, time to first rejection, number of transplants.
Conclusion
Height and weight Z-Scores in Cys dialysis and transplant pts were poorer when compared NIC pts. Cys pts have improved dialysis survival. Cys transplant pts did not differ from NIC pts in BP Z-Scores, donor source, preemptive transplant, graft survival, time to first rejection.
Funding
- NIDDK Support – This is investigator initiated study, supported by Horizon