The efficacy and safety of RNA interference for the treatment of primary hyperoxaluria: a systematic review and meta-analysis.

Primary hyperoxaluria (PH) are rare inherited disorders of liver glyoxylate metabolism. The main symptoms are related to the precipitation of calcium oxalate crystals in the urinary tract with progressive renal damage. The severity of disease can result in kidney failure and systemic oxalosis. Until recently, RNA interference (RNAi) has been demonstrated as a therapeutic avenue for PH. We conducted a systematic review and meta-analysis to assessed the efficacy and safety of RNAi in the treatment of PH patients. The present systemic review systematically and comprehensively summarizes the pathophysiological mechanisms by which hyperoxalemia leads to kidney failure. Furthermore, we provide a detailed summary of the mechanisms of RNAi drug action in the pharmacological treatment of PH. The enrolled studies indicated that early RNAi intervention is beneficial for patients, especially in maintaining stable kidney function and reversing the effects of hyperoxaluria. Furthermore, high-dose and long time-duration RNAi therapy may have a better clinical effect. The efficacy of RNAi combined with hemodialysis seems to be promising, and it deserves more well-designed trials with large sample sizes in the future. RNAi therapy plays an important role in the treatment of PH. Early RNAi intervention is beneficial for patients, especially in maintaining stable kidney function and reversing the effects of hyperoxaluria. Furthermore, high-dose and long time-duration RNAi therapy may have a better clinical effect, and acceptable safety. The efficacy of RNAi combined with hemodialysis seems to be promising in PH treatment.