伴有INSR外显子14纯合子缺失的Donohue综合征1例。

IF 0.4 Q3 MEDICINE, GENERAL & INTERNAL
Oxford Medical Case Reports Pub Date : 2025-09-28 eCollection Date: 2025-09-01 DOI:10.1093/omcr/omaf183
José Daniel Almazán Monroy, Arodys Julianny Valle Martinez, Sara Elizabeth Milla Salguero, Eduardo Smelin Perdomo Domínguez
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引用次数: 0

摘要

Donohue综合征(DS)是一种罕见的常染色体隐性遗传病,由胰岛素受体(INSR)的纯合或复合杂合突变引起,导致严重的胰岛素抵抗。其特点是极度高胰岛素血症、空腹低血糖、餐后高血糖、严重生长受限和畸形特征,由于代谢不稳定和感染,第一年死亡率高。我们报告了一名3个月大的洪都拉斯女孩,其INSR外显子14纯合缺失,表现为严重的胰岛素抵抗,代谢失调和面部畸形。尽管用奥曲肽、二甲双胍和麦芽糖糊精治疗,患者病情仍恶化,导致感染性休克、弥散性血管内凝血和多器官衰竭。该病例强调了将DS的基因型与表型相关联的挑战,并强调了了解特定INSR突变如何影响治疗反应和临床结果的重要性。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Donohue syndrome with a homozygous INSR exon 14 deletion: a case report.

Donohue syndrome (DS) is a rare autosomal recessive disorder caused by homozygous or compound heterozygous mutations in insulin receptor (INSR), leading to severe insulin resistance. It is characterized by extreme hyperinsulinemia, fasting hypoglycemia, postprandial hyperglycemia, severe growth restriction, and dysmorphic features, with a high mortality rate in the first year due to metabolic instability and infections. We report the case of a 3-month-old Honduran girl with a homozygous exon 14 deletion in INSR who presented with severe insulin resistance, metabolic dysregulation, and dysmorphic facial features. Despite treatment with octreotide, metformin, and maltodextrin, the patient's condition worsened, leading to septic shock, disseminated intravascular coagulation, and multiple organ failure. This case highlights the challenges in correlating genotype with phenotype in DS and emphasizes the importance of understanding how specific INSR mutations influence the treatment response and clinical outcomes.

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来源期刊
Oxford Medical Case Reports
Oxford Medical Case Reports MEDICINE, GENERAL & INTERNAL-
CiteScore
0.90
自引率
0.00%
发文量
125
审稿时长
19 weeks
期刊介绍: Oxford Medical Case Reports (OMCR) is an open access, peer-reviewed online journal publishing original and educationally valuable case reports that expand the field of medicine. The journal covers all medical specialities including cardiology, rheumatology, nephrology, oncology, neurology, and reproduction, comprising a comprehensive resource for physicians in all fields and at all stages of training. Oxford Medical Case Reports deposits all articles in PubMed Central (PMC). Physicians and researchers can find your work through PubMed , helping you reach the widest possible audience. The journal is also indexed in the Web of Science Core Collection . Oxford Medical Case Reports publishes case reports under the following categories: Allergy Audiovestibular medicine Cardiology and cardiovascular systems Critical care medicine Dermatology Emergency medicine Endocrinology and metabolism Gastroenterology and hepatology Geriatrics and gerontology Haematology Immunology Infectious diseases and tropical medicine Medical disorders in pregnancy Medical ophthalmology Nephrology Neurology Oncology Paediatrics Pain Palliative medicine Pharmacology and pharmacy Psychiatry Radiology, nuclear medicine, and medical imaging Respiratory disorders Rheumatology Sexual and reproductive health Sports Medicine Substance abuse.
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