Pharmacological Interventions in the Management of Alport Syndrome: A Systematic Review

Background: Alport syndrome is a genetic disorder primarily affecting the kidney, with significant renal and extrarenal complications. Despite progress in the management of Alport syndrome, effective treatment options are limited. This systematic review aimed to synthesize current evidence regarding potential therapeutic interventions for Alport syndrome.

Methods: A comprehensive literature search was conducted across databases including PubMed, Embase, and Cochrane Library. The eligibility criteria included studies involving patients with Alport syndrome undergoing interventions such as bardoxolone methyl, ramipril, or losartan. Both randomized control trials and non-randomized clinical trials were considered. Key outcomes were changes in renal function parameters and disease progression. Study selection, data extraction, and synthesis were conducted according to standard systematic review guidelines.

Results: A total of 137 studies were identified initially, with four studies meeting the eligibility criteria. These studies collectively included 313 patients with a mean age range of 8.8 to 39.2 years. Three pharmacological interventions were evaluated - bardoxolone methyl, ramipril, and losartan. All demonstrated beneficial impacts on kidney function parameters. Bardoxolone methyl and ramipril showed potential in slowing disease progression, while losartan significantly lowered proteinuria. An additional finding was that the severity of the genetic variant of the disease might impact disease progression and treatment outcomes.

Conclusion: The findings of this systematic review suggest that bardoxolone methyl, ramipril, and losartan may offer promising interventions for managing Alport syndrome, potentially slowing disease progression and improving kidney function. However, the need for larger, more rigorous trials is evident to substantiate these findings and to explore the impact of genetic variant severity on disease progression and treatment response. This review underscores the importance of continued research efforts in improving therapeutic strategies and personalizing treatment for patients with Alport syndrome.