The clinical presentation and genetic diagnosis of Tangier disease in the pediatric age group.

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Tác giả: Muhammet Ensar Doğan, Ayten Güleç, Hakan Gümüş, Banu Kadıoğlu Yılmaz, Fatih Kardaş, Selcan Öztürk, Hüseyin Per, Pembe Soylu Üstkoyuncu

Ngôn ngữ: eng

Ký hiệu phân loại: 809.008 History and description with respect to kinds of persons

Thông tin xuất bản: Germany : Journal of pediatric endocrinology & metabolism : JPEM , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 678811

 OBJECTIVES: Tangier disease (TD) is a rare autosomal recessive condition characterized by high-density lipoprotein (HDL) deficiency
  involving symptoms of polyneuropathy, hyperplastic orange-yellow tonsils, vision disorder, and sudden cardiac death. The major clinical symptoms of TD may not all be co-present. This study evaluates patients diagnosed with TD in childhood to improve the possibility of early diagnosis of asymptomatic cases by reporting our patients' clinical characteristics in order to minimize delayed diagnosis and emphasize the importance of TD, easily detected by HDL measurement. METHODS: This retrospective and cross-sectional study investigated seven patients from three different families diagnosed with TD. RESULTS: Four of seven patients were girls. Median age was 5.7 years at symptom onset and 6.5 years at diagnosis. The index case presented with neuropathy findings, and TD was diagnosed based on genetic analysis. Low lipid levels were determined in a sibling and cousins with cardiac death and gait disturbance in the family. TD was confirmed by genetic investigation. Our other patients were evaluated due to anemia, thrombocytopenia, yellow-orange hypertrophy in the tonsils, and organomegaly. Diagnosis was established with genetic analysis and low HDL. No coronary artery disease or ocular involvement was observed in any case. CONCLUSIONS: All patients presenting with neuropathy and gait disorders should undergo detailed tonsil examinations and HDL tests. Genetic analysis should be carried out if necessary. Family screening should be recommended to patients with consanguineous marriages after diagnosis of TD.
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