Comparison of two corticosteroid regimens on brain volumetrics in patients with Duchenne muscular dystrophy

Congenital Adrenal Hyperplasia (CAH) has been reported to manifest with white matter aberrations. However, many previous studies included only small samples, restricted their analyses to females, lacked a control group, and/or did not correct for brain size. Here we examined the largest sample to date, comprising 53 male and female participants with CAH, who were matched with 53 male and female controls in terms of sex, age, education, and verbal intelligence. The four groups were compared with respect to their total white matter as well as white matter hyperintensities while applying brain size corrections. For both measures, total white matter and white matter hyperintensities, there were no significant sex differences or group-by-sex interactions. However, individuals with CAH had significantly smaller total white matter volumes compared to controls. Our findings align with previous reports of white matter variations in CAH. The absence of a group-by-sex interaction suggests that white matter variations in CAH may not be attributable to prenatal androgens. Instead, they may be a result of the condition itself and/or its treatment with glucocorticoids. The latter aspect warrants follow-up, particularly given that glucocorticoids are employed not only in CAH but also in other medical conditions.

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Investigating the effects of prednisolone on behavior in mouse models of Duchenne muscular dystrophy

Verhaeg,

van de Vijver,

Tanganyika-de Winter

et al. 2024

Preprint

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Background: Next to progressive muscle loss, Duchenne muscular dystrophy patients suffer from behavioral and cognitive problems. This is due to mutations in the DMD gene, that result in the lack of dystrophin in both the muscles and brain. As part of the standards of care, patients receive corticosteroids (prednisolone or deflazacort) to slow down muscle degeneration. The precise consequences of chronic corticosteroid usage on the behavior of DMD patients remain unclear, mainly due to challenges of recruiting corticosteroid naïve patients into clinical studies. Objective: This study used DMD mouse models, representing mutations resulting in lack of one or more dystrophin isoforms, to analyze the effects of corticosteroid treatment on different behavioral domains. Methods: Prednisolone (PDN) or placebo was administered via a subcutaneous 60-day slow release pellet (66 μg/day) and mice were subjected to several behavioral tests. Results: Unfortunately, the pellet only exposed mice to PDN for half of the intended duration. During the time of PDN exposure, we found a small amelioration in anxiety but were unable to find any differences in social interaction and spatial learning and memory. Conclusions: Short term exposure to PDN via a slow release pellet does not seem to negatively affect anxiety, social interaction or spatial learning and memory. We cannot rule out that a longer treatment period than 4 weeks would affect behavior in DMD mice.

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Comparison of two corticosteroid regimens on brain volumetrics in patients with Duchenne muscular dystrophy

Cited by 3 publications

References 55 publications

Genotype and corticosteroid treatment are distinctively associated with gray matter characteristics in patients with Duchenne muscular dystrophy

Genotype and corticosteroid treatment are distinctively associated with gray matter characteristics in patients with Duchenne muscular dystrophy

White matter variations in congenital adrenal hyperplasia: possible implications for glucocorticoid treatment

Investigating the effects of prednisolone on behavior in mouse models of Duchenne muscular dystrophy

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