Best of 2023: Primary Hyperoxaluria
High morbidity and mortality rates found in patients with advanced PH1
Advanced primary hyperoxaluria 1 (PH1) is a severe genetic disease characterized by excessive oxalate production, leading to progressive kidney damage and systemic oxalosis, according to a study that examined the natural history of the disease. The study, presented during Kidney Week 2023, highlights the high morbidity and mortality associated with advanced PH1, with patients facing significant challenges in managing the condition. Liver and/or kidney transplantation are frequently pursued but come with substantial risks. Early detection and improved therapeutic interventions are urgently needed to enhance the prognosis and treatment options for individuals affected by PH1.
New study reveals higher prevalence of primary hyperoxaluria worldwide
A recent study has revealed a higher global prevalence of primary hyperoxaluria (PH) than previously reported, according to a presentation at the Kidney Week 2023 Annual Meeting. The results found that the estimated global prevalence of PH is at approximately 1 in 58,823 individuals, significantly higher than previously reported figures. This equates to roughly 136,000 affected individuals worldwide, indicating a potential underdiagnosis or reduced disease penetrance.
Promising results for nedosiran in primary hyperoxaluria presented during Kidney Week 2023
Nedosiran, an RNA interference therapy, has shown promising results in treating primary hyperoxaluria (PH), a rare genetic disorder, according to data presented at Kidney Week 2023 Annual Meeting.The 30-month interim analysis of the PHYOX3 study indicates that nedosiran was well-tolerated in patients with PH1 and resulted in a sustained reduction in urinary oxalate excretion, a key factor in preventing kidney damage.
Combined liver-kidney transplantation may offer hope for pediatric patients
Combined liver-kidney transplantation (CLKT) is a significant and successful surgical procedure, particularly in pediatric cases where congenital diseases drive the need for transplantation, according to a recent review that emphasizes the importance of developing decision-making algorithms that consider region-specific organ allocation systems and medical environments to ensure judicious use and equitable distribution of this life-saving intervention.