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资源有限国家新生儿甲状旁腺功能亢进症的诊断和管理挑战:来自苏丹一个家庭的病例系列。

Challenges in diagnosis and management of neonatal hyperparathyroidism in a resource-limited country: a case series from a Sudanese family.

机构信息

Department of Pediatric Endocrinology, Gaafar Ibn Auf Pediatric Tertiary Hospital, Khartoum, Sudan.

Radbound University Medical Center, Nijmegen, Netherlands.

出版信息

Pan Afr Med J. 2021 Oct 15;40:105. doi: 10.11604/pamj.2021.40.105.29527. eCollection 2021.

Abstract

Neonatal hyperparathyroidism is a rare disease caused by a homozygous inactivating mutation in the calcium sensing receptor gene. It presents early in life with life threatening manifestations of hypercalcemia, if left untreated the condition may be lethal. This is the first case series reported from Sudan. Three Sudanese siblings presented with severe symptoms of hypercalcemia in the form of polyuria, failure to thrive and multiple bone fractures. Serum calcium and parathyroid hormone levels were very high with low phosphate and normal alkaline phosphatase levels. Ultrasonography and sestamibi scan were normal and did not assist in diagnosing their condition. Medical management was a great challenge due to unavailability of medications such as parentral bisphosphonates and calcimimetics. Parathyroidectomy was inevitable. Tissue biopsies revealed parathyroid hyperplasia and no adenoma. Gene sequencing revealed a homozygous missense mutation: c 2038 C T p (Arg680Cys) in two siblings, both parents were heterozygous for the same missense mutation. Our report reflects the challenges in diagnosis and management of neonatal hyperparathyroidism in resource limited countries. We also highlight the importance of genetic testing in the diagnosis and management of such cases in countries with high rates of consanguineous marriage.

摘要

新生儿甲状旁腺功能亢进症是一种罕见疾病,由钙敏感受体基因的纯合失活突变引起。如果不治疗,它会在生命早期出现危及生命的高钙血症表现,这种疾病可能是致命的。这是来自苏丹的首例病例系列报告。三名苏丹裔兄弟姐妹表现出严重的高钙血症症状,包括多尿、生长不良和多处骨折。血清钙和甲状旁腺激素水平非常高,而磷酸盐水平低,碱性磷酸酶水平正常。超声和 sestamibi 扫描正常,无法协助诊断他们的病情。由于缺乏静脉注射双膦酸盐和钙敏感受体激动剂等药物,医疗管理极具挑战。甲状旁腺切除术是不可避免的。组织活检显示甲状旁腺增生,无腺瘤。基因测序显示,两个兄弟姐妹均存在纯合错义突变:c.2038C>T p.(Arg680Cys),父母双方均为同一错义突变的杂合子。我们的报告反映了资源有限国家新生儿甲状旁腺功能亢进症的诊断和管理方面的挑战。我们还强调了在高近亲结婚率国家,基因检测在这类病例的诊断和管理中的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/4952/8627147/f3fbc2ec1cb1/PAMJ-40-105-g001.jpg

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