Observational studyOrphanet journal of rare diseases2025
Serum creatinine to cystatin C ratio as monitoring biomarker in Chinese adult spinal muscular atrophy: a prospective cohort study.
Observational study in Orphanet journal of rare diseases, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 4 papers.
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Who cites it
4 citing papers in PubMed.
- Serum creatinine to cystatin C ratio as a biomarker for monitoring motor-function in children with spinal muscular atrophy treated with nusinersen: a retrospective cohort study.BMC neurology · 2026Article
- Serum creatinine outperforms the creatinine to cystatin C ratio and creatinine muscle index as biomarkers in Chinese pediatric spinal muscular atrophy.Frontiers in human neuroscience · 2026Article
- Motor function and safety of nusinersen for spinal muscular atrophy: a systematic review and meta-analysis among Chinese patients.Frontiers in pediatrics · 2026Review
- Application of Biomarkers in Spinal Muscular Atrophy.International journal of molecular sciences · 2025Review
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Authors and funding
8 authors.
Funding
Abstract
backgroundThe creatinine to cystatin C ratio (CCR) can be used as a biomarker of muscle mass and strength, but no studies have evaluated whether it can be used as a biomarker to monitor the efficacy of treatment with nusinersen in Chinese adults with 5q-associated spinal muscular atrophy (SMA).
methodsIn this prospective observational study, 28 adult SMA patients were followed for 18 months. Data on motor function and daily activities were collected using the Hammersmith Functional Motor Scale Expanded (HFMSE), Revised Upper Limb Module (RULM), 6-Minute Walking Test (6WMT), and Barthel Index (BI). Serum levels of creatinine (Cr), creatine kinase (CK), and CCR were measured. Spearman correlation and linear mixed models analyzed the relationships between functional scores and laboratory indicators.
resultsHFMSE scores showed significant improvement at Visit 5 (V5) [+ 1.04 points, p = 0.016), V6 (+ 1.61, p = 0.012), V7 (+ 1.93, p < 0.001), and V8 (+ 1.89, p < 0.001)], while RULM scores improved significantly at V5 (+ 2.00 points, p = 0.024), V7 (+ 2.00, p = 0.032), and V8 (+ 2.00, p < 0.001). The BI also exhibited significant improvement at V7 (+ 5.00, p < 0.001) and V8 (+ 2.50, p < 0.001). The 6MWT did not show significant improvement (p > 0.05). Serum CCR levels were significantly higher than baseline at V5 (+ 4.35 points, p < 0.001), V7 (+ 5.12, p < 0.001), and V8 (+ 6.59, p < 0.001). Cr levels were significantly higher than baseline at V5 (+ 2.39 points, p < 0.001) and V7 (+ 0.90, p < 0.001), while log10 CK levels remained unchanged (p > 0.05). Spearman correlation analysis revealed CCR showed the strongest correlation with functional scores, followed by Cr and log10 CK. Further linear mixed-effect model indicated that after adjusting for covariates, only CCR showed a dynamic positive correlation with HFMSE scores (β = 0.280; 95% CI 0.023-0.537, p = 0.033), while other serum indicators had no correlation with functional scores.
conclusionLong-term use of nusinersen effectively improves motor function and quality of life in adult SMA patients, and CCR may be a better indicator to reflect changes in motor function during treatment compared to other blood biomarkers.
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