国产骨化三醇治疗甲状旁腺功能减退症或假性甲状旁腺功能减退症所致低钙血症：一项前瞻性自身对照临床试验。

Treatment of hypocalcemia caused by hypoparathyroidism or pseudohypoparathyroidism with domestic-made calcitriol: a prospective and self-controlled clinical trial.

作者信息

Wang Ou, Xing Xiao-ping, Meng Xun-wu, Xia Wei-bo, Li Mei, Jiang Yan, Hu Ying-ying, Liu Huai-cheng

机构信息

Department of Endocrinology, Key Lab of Endocrinology Ministry of Health, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences, Beijing 100730, China.

出版信息

Chin Med J (Engl). 2009 Feb 5;122(3):279-83.

PMID:19236804

Abstract

BACKGROUND

Parathyroid hormone deficiency or resistance may cause hypocalcemia with related symptoms and signs. Lifelong treatment of calcium combined with vitamin D or its metabolites is always necessary for these patients. Here we reported a prospective and open-label trial to investigate the efficacy and safety of domestic-made calcitriol in treatment of hypocalcemia caused by hypoparathyroidism or pseudohypoparathyroidism.

METHODS

Twenty-four patients with confirmed hypoparathyroidism or pseudohypoparathyroidism aged (36.5 +/- 11.0) years old were studied. Among them, 16 patients had idiopathic hypoparathyroidism, 2 had pseudohypoparathyroidism and 6 had hypoparathyroidism secondary to cervical surgery. Serum calcium levels were lower than 1.88 mmol/L. Oral calcitriol was administered twice or three times with elemental calcium 1.2 g per day. All patients were followed every 4 weeks throughout the 12-week period. Dose adjustments of calcitriol were based on serum and urinary calcium levels and symptoms of hypocalcemia.

RESULTS

Twenty patients were included by the end of this study. Muscular weakness, cramps, extremity paresthesia, Chovestek's sign and Trousseau's sign were relieved in 76.9%, 100%, 94.4%, 93.3% and 78.9% of patients, respectively. Serum calcium, plasma ionized calcium and serum phosphorus levels were (1.54+/-0.25) mmol/L, (0.64+/-0.10) mmol/L and (2.00+/-0.46) mmol/L at baseline, and reached (2.20+/-0.20) mmol/L, (0.95+/-0.06) mmol/L and (1.68+/-0.25) mmol/L (P<0.01) at the 12th week of treatment, respectively. Eighty percent of patients were assessed as effective and 20% as partly effective. Three, four and eight patients had hypercalciuria at the 4th, 8th and 12th week of treatment, respectively, which were reduced by thiazide diuretics. The final dose of calcitriol was (1.09+/-0.50) microg/d.

CONCLUSIONS

Calcitriol combined with calcium can be used in treatment of hypocalcemia caused by hypoparathyroidism or pseudohypoparathyroidism effectively and safely. Serum and urinary calcium levels should be monitored during the course of the therapy.

摘要

背景

甲状旁腺激素缺乏或抵抗可能导致低钙血症及相关症状和体征。这些患者始终需要终身补充钙剂并联合维生素D或其代谢产物进行治疗。在此，我们报告一项前瞻性开放标签试验，以研究国产骨化三醇治疗甲状旁腺功能减退或假性甲状旁腺功能减退所致低钙血症的疗效和安全性。

方法

对24例确诊为甲状旁腺功能减退或假性甲状旁腺功能减退的患者进行研究，患者年龄为（36.5±11.0）岁。其中，16例为特发性甲状旁腺功能减退，2例为假性甲状旁腺功能减退，6例为颈部手术后甲状旁腺功能减退。血清钙水平低于1.88 mmol/L。口服骨化三醇，每日2次或3次，同时每日补充元素钙1.2 g。在整个12周期间，每4周对所有患者进行随访。根据血清和尿钙水平以及低钙血症症状调整骨化三醇剂量。

结果

本研究结束时纳入20例患者。肌肉无力、痉挛、肢体感觉异常、Chovestek征和Trousseau征分别在76.9%、100%、94.4%、93.3%和78.9%的患者中得到缓解。基线时血清钙、血浆离子钙和血清磷水平分别为（1.54±0.25）mmol/L、（0.64±0.10）mmol/L和（2.00±0.46）mmol/L，治疗第12周时分别达到（2.20±0.20）mmol/L、（0.95±0.06）mmol/L和（1.68±0.25）mmol/L（P<0.01）。80%的患者评估为有效，20%为部分有效。治疗第4、8和12周时分别有3、4和8例患者出现高钙尿症，使用噻嗪类利尿剂后高钙尿症减轻。骨化三醇的最终剂量为（1.09±0.50）μg/d。