Systematic Review of Management Strategies for Alport Syndrome: Implications for Male Patients
Bibliographic record
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. Protocol Registration: The protocol for this systematic review is registered in the Open Science Framework (OSF): osf. io/k86ms.
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How this classification was reachedexpand
Full frame machine prediction
Teacher imitationNot calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.
Distilled classifier scores by category (both heads)
| Category | Codex | Gemma |
|---|---|---|
| Metaresearch | 0.020 | 0.098 |
| Meta-epidemiology (narrow) | 0.002 | 0.001 |
| Meta-epidemiology (broad) | 0.011 | 0.009 |
| Bibliometrics | 0.009 | 0.008 |
| Science and technology studies | 0.001 | 0.001 |
| Scholarly communication | 0.003 | 0.003 |
| Open science | 0.002 | 0.002 |
| Research integrity | 0.002 | 0.002 |
| Insufficient payload (model declined to judge) | 0.008 | 0.000 |
Machine scores (provisional)
The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.
Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.
score_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from itClassification
machine, unvalidatedMachine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.
How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".