Suppr超能文献

不同症状严重程度的卡恩斯-塞尔综合征中的内分泌紊乱:两例病例报告及文献综述

Endocrine disorders in Kearns-Sayre syndrome with different severity of symptoms: two case reports and a literature review.

作者信息

Amergoolov Ilya I, Khruleva Yulia I, Pavlova Maria G, Likhodey Natalia V, Sulaev Abu M, Surkova Elena V, Sych Yulia P, Kalashnikova Marina F, Arustamyan Anna S, Martirosyan Gratsinna A, Lew-Gor Simione T

机构信息

Department of Endocrinology №1, First Moscow State Medical University (Sechenov University), Moscow.

Republican Endocrinological Dispensary, Grozny, Chechen Republic.

出版信息

Eur J Transl Myol. 2024 Oct 30;34(4):12897. doi: 10.4081/ejtm.2024.12897.

Abstract

Kearns-Sayre Syndrome (KSS) is a variant of mitochondrial disorder caused by a Mitochondrial Deoxyribonucleic Acid (mtDNA) deletion. Clinical manifestations of KSS can include different organ and system involvement. Different organ malfunctions, more often cardiac dysfunction, can lead to death. No effective treatment of this condition exists to date. Here, we report two patients with KSS. Female patient with a large-scale deletion of 7,020 base pairs (bp) suffered from hypogonadism, diabetes mellitus with fluctuating glucose levels, and had poor general health. A male patient with a common 4,977 bp deletion did not have diabetes mellitus but had impaired glucose tolerance. He also had a higher level of general health than our female patient. Both patients had reduced Bone Mineral Density (BMD). In female patients, calcium and vitamin D supplementation combined with metabolic therapy and nutritional drink supplements helped increase BMD (up to 32% in L1-L4). Comparing these two patients suggests that the larger the mtDNA deletion is, the more severe the course of the disease is. Not only does the size of the mtDNA deletion probably determine the severity of the disease, but also such factors as mtDNA heteroplasmy level, presence of mtDNA duplications, and pleioplasmy. Moreover, continuous nonconsecutive metabolic therapy and nutritional supplements are helpful in the prevention of deterioration of symptoms and general health.

摘要

卡恩斯-塞尔综合征(KSS)是一种由线粒体脱氧核糖核酸(mtDNA)缺失引起的线粒体疾病变体。KSS的临床表现可包括不同器官和系统受累。不同器官功能障碍,更常见的是心脏功能障碍,可导致死亡。迄今为止,尚无针对这种疾病的有效治疗方法。在此,我们报告两名KSS患者。一名女性患者有7020个碱基对(bp)的大规模缺失,患有性腺功能减退、血糖水平波动的糖尿病,总体健康状况较差。一名男性患者有常见的4977 bp缺失,没有患糖尿病,但糖耐量受损。他的总体健康状况也比我们的女性患者好。两名患者的骨密度(BMD)均降低。对于女性患者,补充钙和维生素D并结合代谢疗法和营养饮料补充剂有助于提高骨密度(L1-L4处提高多达32%)。比较这两名患者表明,mtDNA缺失越大,疾病进程越严重。mtDNA缺失的大小可能不仅决定疾病的严重程度,mtDNA异质性水平、mtDNA重复的存在以及多效性等因素也是如此。此外,持续的非连续代谢疗法和营养补充剂有助于预防症状恶化和总体健康状况下降。

相似文献

3
Kearns-Sayre syndrome case. Novel 5,9 kb mtDNA deletion.
Mol Genet Genomic Med. 2023 Jan;11(1):e2059. doi: 10.1002/mgg3.2059. Epub 2022 Oct 1.
4
Mitochondrial DNA deletion with Kearns Sayre syndrome in a child with Addison disease.
Eur J Pediatr. 1998 Aug;157(8):643-7. doi: 10.1007/s004310050902.
5
Polyendocrinopathy and multisystem involvement are common phenotypic features of Kearns-Sayre syndrome.
Eur J Transl Myol. 2025 Jun 27;35(2). doi: 10.4081/ejtm.2025.13634. Epub 2025 Apr 14.
6
Are duplications of mitochondrial DNA characteristic of Kearns-Sayre syndrome?
Hum Mol Genet. 1994 Jun;3(6):947-51. doi: 10.1093/hmg/3.6.947.
7
Kearns Sayre Syndrome--case report with review of literature.
Indian J Pediatr. 2012 May;79(5):650-4. doi: 10.1007/s12098-011-0618-3. Epub 2012 Jan 10.
8
High proportions of mtDNA duplications in patients with Kearns-Sayre syndrome occur in the heart.
Am J Med Genet. 1997 Sep 5;71(4):443-52. doi: 10.1002/(sici)1096-8628(19970905)71:4<443::aid-ajmg14>3.0.co;2-g.
10
Clinical Phenotype and Genetic Features of a Pair of Chinese Twins with Kearns-Sayre Syndrome.
DNA Cell Biol. 2020 Aug;39(8):1449-1457. doi: 10.1089/dna.2019.5010. Epub 2020 Jun 29.

引用本文的文献

1
Polyendocrinopathy and multisystem involvement are common phenotypic features of Kearns-Sayre syndrome.
Eur J Transl Myol. 2025 Jun 27;35(2). doi: 10.4081/ejtm.2025.13634. Epub 2025 Apr 14.

本文引用的文献

1
Endocrine features of primary mitochondrial diseases.
Curr Opin Endocrinol Diabetes Obes. 2024 Feb 1;31(1):34-42. doi: 10.1097/MED.0000000000000848. Epub 2023 Dec 4.
2
Endocrine Manifestations and New Developments in Mitochondrial Disease.
Endocr Rev. 2022 May 12;43(3):583-609. doi: 10.1210/endrev/bnab036.
4
Psychosocial Aspects of Diabetes Technology Use: The Child and Family Perspective.
Endocrinol Metab Clin North Am. 2020 Mar;49(1):127-141. doi: 10.1016/j.ecl.2019.10.004. Epub 2019 Nov 29.
5
Risk factors for poor bone health in primary mitochondrial disease.
J Inherit Metab Dis. 2017 Sep;40(5):673-683. doi: 10.1007/s10545-017-0046-2. Epub 2017 Apr 27.
6
Efficacy of growth hormone therapy in Kearns-Sayre syndrome: the KIGS experience.
J Pediatr Endocrinol Metab. 2016 Nov 1;29(11):1319-1324. doi: 10.1515/jpem-2016-0172.
7
Low prevalence of patients with mitochondrial disease in the German/Austrian DPV diabetes registry.
Eur J Pediatr. 2016 May;175(5):613-22. doi: 10.1007/s00431-015-2675-5. Epub 2015 Dec 15.
8
Kearns-Sayre syndrome: a case series of 35 adults and children.
Int J Gen Med. 2014 Jul 3;7:325-32. doi: 10.2147/IJGM.S65560. eCollection 2014.

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验