Dialysis and renal transplantation often occur prior to the diagnosis of primary hyperoxaluria (PH) in children, suggesting the presence of significant morbidity associated with delayed PH diagnosis, investigators reported during the American Society of Nephrology’s Kidney Week 2021.
“Medications for the organs affected by calcium oxalate deposition were consistently prescribed before diagnosis, suggesting an opportunity for early PH identification to be initiated that may enable tailored therapy to potentially delay or prevent need for dialysis and transplant,” Gregory E. Tasian, MD, MSc, MSCE of Children’s Hospital of Philadelphia, and colleagues concluded in a poster presentation.
PH is a rare hereditary metabolic derangement that results in overproduction of oxalate in the liver that can cause kidney stones and end-stage kidney disease.
Dr Tasian and colleagues studied 341 pediatric patients with presumed PH who had a mean age of 9.4 years at their cohort entry date (CED), defined as the date of a first PH-related diagnostic code. The median follow-up period was 2.9 years.
Most patients started dialysis before CED and at a younger age than those starting dialysis after CED. Of those who received a kidney transplant, the majority received a kidney prior to CED. Nephrology was the specialty most commonly responsible for the initial PH diagnosis (69% on CED). Some patients received prescription drug therapy for end-organ manifestations of PH prior to CED.
Of the 341 patients, 58, 146, and 79 patients received vitamin B6, citrates, and thiazides, respectively. Of these, 17, 71, and 38 received the medications prior to CED, respectively.
Disclosure: The study was supported by Dicerna Pharmaceuticals. Please see the original references for a full list of disclosures.
Tasian GE, Dickinson K, Razzaghi H, et al. Temporal relationship of transplant, dialysis, and medications for children with primary hyperoxaluria. Presented at: Kidney Week 2021, November 2-7, 2021. Poster PO1998.