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
Department of Endocrinology №1, First Moscow State Medical University (Sechenov University), Moscow.
Republican Endocrinological Dispensary, Grozny, Chechen Republic.
Eur J Transl Myol. 2024 Oct 30;34(4):12897. doi: 10.4081/ejtm.2024.12897.
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.
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。