Rani P Radha, Maheshwari Rushikesh, Prasad N Rajendra, Karthik Reddy T S, Reddy P Amaresh
Department of Endocrinology and Metabolism, Narayana Medical College and Hospital, Nellore, Andhra Pradesh, India.
Indian J Endocrinol Metab. 2013 Dec;17(Suppl 3):S665-6. doi: 10.4103/2230-8210.123562.
There are two types of vitamin D dependent rickets (VDDR) that cause rickets in children. VDDR type 1 (VDDR-I) is caused by an inborn error of vitamin D metabolism, which interferes with renal conversion of calcidiol (25OHD) to calcitriol (1,25(OH)2D) by the enzyme 1-α-hydroxylase. Patients with VDDR-I have mutations of chromosome 12 that affect the gene for the enzyme 1-α-hydroxylase, resulting in decreased levels of 1,25(OH) vitamin D. Clinical features include growth failure, hypotonia, weakness, rachitic rosary, convulsions, tetany, open fontanels and pathologic fractures. We report a case of VDDR-I in 14-month-old male child. Establishing an early diagnosis of these genetic forms of rickets is challenging, especially in developing countries where nutritional rickets is the most common variety of the disease where genetic diagnosis is not always possible because of financial constraints. A prompt diagnosis is necessary to initiate adequate treatment, resolve biochemical features and prevent complications, such as severe deformities that may require surgical intervention.
有两种类型的维生素D依赖性佝偻病(VDDR)可导致儿童患佝偻病。1型维生素D依赖性佝偻病(VDDR - I)是由维生素D代谢的先天性缺陷引起的,它会干扰肾脏中由1-α-羟化酶将骨化二醇(25OHD)转化为骨化三醇(1,25(OH)2D)的过程。VDDR - I患者的12号染色体发生突变,影响1-α-羟化酶基因,导致1,25(OH)维生素D水平降低。临床特征包括生长发育迟缓、肌张力减退、虚弱、佝偻病串珠、惊厥、手足搐搦、囟门未闭和病理性骨折。我们报告一例14个月大男童患VDDR - I的病例。对这些遗传性佝偻病进行早期诊断具有挑战性,尤其是在发展中国家,营养性佝偻病是最常见的佝偻病类型,由于经济限制,往往无法进行基因诊断。及时诊断对于启动适当治疗、解决生化特征以及预防并发症(如可能需要手术干预的严重畸形)是必要的。