Clinical practice recommendations for the management of Alport syndrome: a joint statement of the Korean Society of Nephrology and the Korean Society of Pediatric Nephrology.

BACKGROUND

Alport syndrome (AS) is a hereditary kidney disorder characterized by progressive hematuria, proteinuria, and kidney failure, frequently accompanied by hearing loss and ocular abnormalities. Although blockade of the renin-angiotensin system (RAS) is established as the first-line therapy, additional pharmacological interventions have been explored. To formulate evidence-based recommendations tailored to the Korean healthcare setting, the Genetic Kidney Disease Study Group of the Korean Society of Nephrology conducted a systematic review evaluating four therapeutic strategies: RAS blockade, cyclosporine, mineralocorticoid receptor antagonists (MRAs), and sodium-glucose cotransporter 2 inhibitors (SGLT2i).

METHODS

The quality of evidence and strength of recommendations were assessed using the Grading of Recommendations Assessment, Development and Evaluation framework.

RESULTS

The review confirmed that early initiation of RAS blockade delays progression to kidney failure and improves long-term survival, with the greatest benefit when initiated at the stage of microscopic hematuria or microalbuminuria. In contrast, evidence supporting cyclosporine , MRAs, and SGLT2i remains limited, primarily derived from small observational studies with short follow-up and heterogeneous populations. Although cyclosporine and MRAs have demonstrated short-term reductions in proteinuria, neither has shown sustained renoprotective effects, and cyclosporine is associated with a substantial risk of nephrotoxicity. Evidence regarding SGLT2i is inconsistent, with insufficient data on long-term efficacy in AS.

CONCLUSION

Accordingly, these guidelines strongly recommend early RAS blockade as the cornerstone of AS, while no recommendations are made for routine use of cyclosporine, MRAs, or SGLT2i. Well-designed clinical trials are needed to expand therapeutic options and improve long-term outcomes in AS.