Akıncı Ayşehan, Dündar İsmail, Kıvılcım Meltem
İnönü University Faculty of Medicine, Department of Pediatric Endocrinology, Malatya, Turkey, E-mail:
J Clin Res Pediatr Endocrinol. 2017 Jun 1;9(2):172-178. doi: 10.4274/jcrpe.3486. Epub 2016 Oct 31.
High doses of oral calcium or long-term calcium infusions are recommended to correct the hypocalcemia and secondary hyperparathyroidism in patients with hereditary 1,25 dihydroxyvitamin D3-resistant rickets (HVDRR). Preliminary studies revealed that calcimimetics may be a safe and effective therapeutic choice in children with secondary hyperparathyroidism. Our aim was to observe the efficacy of cinacalcet in the normalization of secondary hyperparathyroidism and hypophosphatemia in two siblings aged 2.5 years and 6 months with HVDRR who did not respond to traditional treatment regimes. Both patients were admitted to the hospital with severe hypocalcemia. They were treated with high doses of calcitriol and calcium infusions intravenously. Secondary hyperparathyroidism was normalized temporarily, but did not improve completely. Cinacalcet (0.25 mg/kg) once a day along with the high doses of oral calcium and calcitriol was added to the treatment schedule. After 3 months, biochemical and radiologic findings reverted to normal. Our findings indicate that cinacalcet is effective in normalizing the hyperparathyroidism and hypophosphatemia in these cases and in improving the bone pathology.
对于患有遗传性1,25 - 二羟维生素D3抵抗性佝偻病（HVDRR）的患者，建议使用高剂量口服钙或长期钙输注来纠正低钙血症和继发性甲状旁腺功能亢进。初步研究表明，拟钙剂可能是治疗继发性甲状旁腺功能亢进儿童的一种安全有效的治疗选择。我们的目的是观察西那卡塞对两名分别为2.5岁和6个月大的HVDRR患儿继发性甲状旁腺功能亢进和低磷血症恢复正常的疗效，这两名患儿对传统治疗方案无反应。两名患者均因严重低钙血症入院。他们接受了高剂量骨化三醇和静脉补钙治疗。继发性甲状旁腺功能亢进暂时恢复正常，但未完全改善。治疗方案中加入了西那卡塞（0.25 mg/kg），每日一次，同时给予高剂量口服钙和骨化三醇。3个月后，生化和影像学检查结果恢复正常。我们的研究结果表明，西那卡塞在使这些病例的甲状旁腺功能亢进和低磷血症恢复正常以及改善骨骼病理方面是有效的。
