Ataxia-Telangiectasia in Resource-Limited Settings: A Diagnostic Challenge

资源匮乏环境下的毛细血管扩张性共济失调:诊断挑战

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Abstract

Ataxia-telangiectasia (A-T) is a rare autosomal recessive disorder characterized by cerebellar ataxia and oculocutaneous telangiectasias, with increased malignancy risk from radiation-induced DNA double-strand breaks. Its multisystem involvement makes diagnosis challenging, and confirmation requires identifying biallelic pathogenic variants in the ATM gene. We report an eight-year-old boy with unsteady gait and bilateral ocular telangiectasia. He had no recurrent infections, and immunoglobulin (Ig) levels were normal, except for low IgA. Although serum alpha-fetoprotein was elevated and MRI demonstrated cerebellar atrophy, findings suggestive of A-T, definitive genetic testing for ATM mutations was not available.

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