Novel recurrent mutations and genetic diversity in Sudanese children with Adrenal Insufficiency.

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Tác giả: Mohamed A Abdullah, Amna I Ahmed, Omer O Babiker, Li F Chan, Luqman S Fauzi, Charlotte L Hall, Samar S Hassan, Claire Hutchison, Areej A Ibrahim, Avinaash V Maharaj, Saptarshi Maitra, Lucia M Marroquin Ramirez, Louise A Metherell, Ghassan Mohamadsalih, Marwa Mohammedali, Salwa A Musa, Younus Qamar, Jordan Read, Chris J Smith

Ngôn ngữ: eng

Ký hiệu phân loại: 978.02 1800–1899

Thông tin xuất bản: England : European journal of endocrinology , 2025

Mô tả vật lý:

Bộ sưu tập: NCBI

ID: 685012

BACKGROUND: Studies of Primary Adrenal Insufficiency (PAI) in African children are rare but in Sudan, congenital adrenal hyperplasia (CAH) and Triple A syndrome are the most common genetic causes. Differential diagnosis is challenging, especially in resource-limited settings, where presentation can mimic common childhood diseases and facilities for biochemical and genetic testing may be restricted. PATIENTS & METHODS: Forty-eight (48) patients from 43 families (31M:17F) with PAI were included (CAH/Triple A excluded). Additional features seen included white matter changes on MRI, auto-immune features and/or obesity. Sanger and whole exome sequencing (WES) were employed for diagnosis, confirmation and segregation with in vitro assays to investigate potential splice defects. RESULTS: In 21/43 families a genetic aetiology consistent with non-autoimmune PAI was discovered, and in 3 families AIRE mutations were found, indicating an autoimmune origin. In Sudan, ABCD1/NNT/AIRE mutations were commonest, including recurrent NNT splice and AIRE deletion mutations. In 2 families we identified ARSA mutations fitting a diagnosis of Metachromatic Leukodystrophy (MLD), in which adrenal insufficiency has not previously been described. In the remaining 17 families, no causative gene mutations were found. Putative causal variants for co-morbidities were concomitantly detected. CONCLUSION: In this population WES revealed itself as a useful frontline tool for the differential diagnosis of individuals presenting with adrenal insufficiency, including discrimination between MLD and adrenoleukodystrophy, and giving plausible gene defects for additional co-morbidities such as obesity. Such genetic diagnoses are crucial to design optimal treatment plans and for genetic counselling in affected individuals and their families.
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