Abstract: PO1998
Temporal Relationship of Transplant, Dialysis, and Medications for Children with Primary Hyperoxaluria
Session Information
- Pediatric Nephrology: Genetics, Kidney Stones, Quality Improvement, and Case Reports
November 04, 2021 | Location: On-Demand, Virtual Only
Abstract Time: 10:00 AM - 12:00 PM
Category: Pediatric Nephrology
- 1700 Pediatric Nephrology
Authors
- Tasian, Gregory Edward, Children's Hospital of Philadelphia Division of Urology, Philadelphia, Pennsylvania, United States
- Dickinson, Kimberley, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Razzaghi, Hanieh, Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Utidjian, Levon H., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Karafilidis, John, Dicerna Pharmaceuticals Inc, Cambridge, Massachusetts, United States
- Mucha, Lisa, Dicerna Pharmaceuticals Inc, Cambridge, Massachusetts, United States
- Forrest, Christopher B., Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
- Ching, Christina B., Nationwide Children's Hospital, Columbus, Ohio, United States
Background
Primary hyperoxaluria (PH), a rare inborn error of metabolism resulting in hepatic overproduction of oxalate, causes kidney stones and, for some, end-stage renal disease and systemic oxalosis. Our objective was to determine the timing of dialysis, renal transplant, and use of medications to treat PH manifestations in children relative to their diagnosis.
Methods
A retrospective cohort study was conducted in PEDSnet, a clinical research network of 7 US pediatric health systems. Data from PEDSnet were queried to identify patients <18 years old with a diagnostic code for or related to PH between 2009 – 2020. Outcomes queried were renal transplant, initiation of dialysis, first prescription for medications used to treat end-organ manifestations of PH, and specialty care visits. Outcomes were evaluated relative to cohort entrance date (CED), defined as date of first PH-related diagnostic code.
Results
341 patients were identified. Median age at CED was 9.4 years (IQR 5.0, 13.0). Median follow-up was 2.9 years (IQR 1.1, 6.0). Median eGFR was 117.33 ml/min (IQR 95.75, 139.79). Most patients with renal transplant were transplanted prior to CED. Similarly, dialysis (n=14) was initiated in the majority before CED and at a younger age than those starting dialysis after CED. Prescription drug therapy before CED was high (29% patients with B6 use; Table). Nephrology was the specialty most commonly responsible for initial PH diagnosis (69% on CED).
Conclusion
In one of the largest cohorts of children in the US with PH, dialysis and renal transplant occurred before diagnosis, suggesting significant morbidity when diagnosis is delayed. Medications for the organs affected by PH were consistently prescribed before diagnosis, suggesting an opportunity for earlier PH identification to enable tailored therapy to potentially delay or prevent need for dialysis and transplant.
| Patients (n=341) | Number Pre-CED | Number On or After CED | |
| Medications (*Denotes results where PEDsnet governance prevents reporting numbers <11 to maintain patient privacy) | |||
| Pyridoxine (Vitamin B6) | 58 (17%) | 17 | 41 |
| Citrate | 146 (43%) | 71 | 75 |
| Thiazides | 79 (23%) | 38 | 41 |
| Erythrocyte-stimulating agents | 15 (4%) | <11* | <11* |
| Age in years at first dialysis, median (IQR) | 2.31 [0.71, 5.92] | 1.15 [0.54, 4.92] | 17.45 [11.25, 20.36] |
Funding
- Commercial Support – Dicerna Pharmaceuticals