Systematic Review of Management Strategies for Alport Syndrome: Implications for Male Patients

IF 2.1 Q2 MEDICINE, GENERAL & INTERNAL

Health Science Reports Pub Date : 2025-03-30 DOI:10.1002/hsr2.70595

Zouina Sarfraz, Ayesha Khan, Maryyam Liaqat, Aden Khan, Faheem Javad, Meher Saleem, Azza Sarfraz, Musfira Khalid, Muzna Sarfraz, Manish Kc, Omar Irfan

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Abstract

Background and Aims

Alport Syndrome (AS) is a rare genetic disorder characterized by progressive kidney disease, hearing loss, and ocular abnormalities, with an incidence of approximately 1 in 50,000 newborns. Due to the severity of the disease, particularly in males with X-linked inheritance, this systematic review consolidates current management strategies, highlighting advancements and existing gaps in treatment options.

Methods

This systematic review followed a protocol registered on the OSF platform (osf. io/k86ms). A comprehensive search of PubMed, Web of Science, Scopus, Cochrane Library, Embase, ClinicalTrials.gov, and the WHO ICTRP was completed by December 24, 2023. Studies eligible for inclusion were clinical trials or observational studies evaluating AS management. Four clinical trials from six publications and two observational studies met the inclusion criteria. The risk of bias was assessed using the Cochrane ROB 2 tool for clinical trials and the Newcastle–Ottawa Scale (NOS) for observational studies. Key interventions examined included bardoxolone methyl, ramipril, and losartan.

Results

Bardoxolone methyl, ramipril, and losartan demonstrated potential benefits in slowing renal disease progression in AS. Observational studies indicated that early intervention might delay the need for dialysis and improve life expectancy. However, significant heterogeneity among studies precluded quantitative synthesis. Ongoing studies on AS management encompass 25 trials involving 52,135 participants, reflecting an active area of research.

Conclusion

Bardoxolone methyl, ramipril, and losartan show promise in delaying renal failure in AS. Nonetheless, the findings highlight the critical need for larger, more diverse trials to validate these therapies and explore additional treatment strategies. Future research must aim to address these evidence gaps, improving treatment efficacy and patient quality of life, particularly for males disproportionately affected by the disease.