Why the study?
To extend molecular findings and evaluate the clinical and histopathological relationship in a previously published case of unusual hypokalemic periodic paralysis.
The pathogenic variant p.Arg1239His in the CACNA1S gene can present with an unusual phenotype of late-onset limb-girdle myopathy and vacuolar myopathy on muscle biopsy.
Does not yet alter diagnostic algorithms for hypokalemic periodic paralysis; extends CACNA1S phenotypic associations but leaves open confirmatory studies.
Several clinical phenotypes have been described related to the CACNA1S gene (calcium channel voltage-dependent L-type alpha-1S subunit), such as autosomal dominant hypokalemic periodic paralysis 1 and autosomal dominant malignant hyperthermia susceptibility and are associated with autosomal dominant and recessive congenital myopathy. Recently, an interesting case of a 58-year-old male patient was published describing an unusual clinical presentation of hypokalemic periodic paralysis where a late-onset limb-girdle myopathy had developed 41 years after paralysis occurred when the patient was 11 years old. Muscle biopsy results were consistent with myopathic changes and revealed the presence of vacuoles, without inflammatory reaction. Later, molecular analysis revealed a pathogenic variant c.3716G>A (p.Arg1239His) in exon 30 of the CACNA1S gene. This technical report provides an extension of the molecular findings and evaluates the clinical and histopathological relationship previously published regarding this case.
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Benitez-Alonso et al. (2022) studied this question.
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