Study summary · research use only
Myostatin and follistatin as monitoring and prognostic biomarkers in dysferlinopathy
Plain-language summary
Paraphrased from the published abstract below — not a verdict on whether anything works.
This human study assessed serum myostatin and follistatin as monitoring or prognostic biomarkers in dysferlinopathy, a muscular dystrophy, measuring myostatin twice three years apart in 76 patients and 38 controls, and follistatin in 62 of these patients and 31 controls. Baseline myostatin, but not follistatin, correlated with baseline motor function and MRI measures, but three-year change in myostatin did not correlate with functional or MRI changes in individual patients. Function, serum creatine kinase, and C-reactive protein, but not age, were independently related to myostatin concentration. Baseline myostatin predicted loss of ambulation but not rate of change of functional or MRI measures. The authors describe myostatin as a possible surrogate for function or muscle mass, but not a promising treatment target in dysferlinopathy.
Abstract
Myostatin is a myokine which acts upon skeletal muscle to inhibit growth and regeneration. Myostatin is endogenously antagonised by follistatin. This study assessed serum myostatin and follistatin concentrations as monitoring or prognostic biomarkers in dysferlinopathy, an autosomal recessively inherited muscular dystrophy. Myostatin was quantified twice with a three-year interval in 76 patients with dysferlinopathy and 38 controls. Follistatin was quantified in 62 of these patients at the same timepoints, and in 31 controls. Correlations with motor function, muscle fat fraction and contractile cross-sectional area were performed. A regression model was used to account for confounding variables. Baseline myostatin, but not follistatin, correlated with baseline function and MRI measures. However, in individual patients, three-year change in myostatin did not correlate with functional or MRI changes. Linear modelling demonstrated that function, serum creatine kinase and C-reactive protein, but not age, were independently related to myostatin concentration. Baseline myostatin concentration predicted loss of ambulation but not rate of change of functional or MRI measures, even when relative inhibition with follistatin was considered. With adjustment for extra-muscular causes of variation, myostatin could form a surrogate measure of functional ability or muscle mass, however myostatin inhibition does not form a promising treatment target in dysferlinopathy.
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