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40例重型β地中海贫血青少年的钙稳态:大剂量胆钙化醇肌肉注射效果的病例对照研究

Calcium homeostasis in 40 adolescents with beta-thalassemia major: a case-control study of the effects of intramuscular injection of a megadose of cholecalciferol.

作者信息

Soliman Ashraf, Adel Ashraf, Wagdy Magda, Al Ali Maryam, ElMulla Naima

机构信息

Department of Pediatrics, Hamad Medical Center, Doha, Qatar.

出版信息

Pediatr Endocrinol Rev. 2008 Oct;6 Suppl 1:149-54.

Abstract

UNLABELLED

The prevalence of disturbed calcium homeostasis in adolescents with beta thalassemia major (T) varies among different populations. Moreover, the cholecalciferol uptake required to achieve or maintain any given serum 25-hydroxycholecalciferol level is not well known, particularly within ranges of the probable physiologic supply of the vitamin.

OBJECTIVES

The objectives of the study were to estimate the prevalence of abnormal calcium homeostasis in 40 adolescent thalassemic patients, and to investigate the effect of an IM injection of a mega dose of vitamin D3 on serum 25-hydroxycholecalciferol (25-OH D) concentration and other calcium homeostasis parameters in vitamin D deficient (VDD) thalassemic adolescents, and to compare these results with those for non-thalassemic adolescents with VDD.

DESIGN

In this prospective study we measured parameters of calcium homeostasis in 40 adolescents with T and 40 matched non-thalassemic (NT) controls. An IM dose (10,000 IU/kg, max 600,000 IU) of cholecalciferol was injected in those with VDD (38 adolescents with thalassemia and 26 non-thalassemic adolescents). Parameters of calcium homeostasis were measured at intervals of 3 months during the course of the study.

RESULTS

Of the 40 adolescents with T, 2 had hypoparathyroidism and low 25-OH D, and 2 had hypocalcemia with hypophosphatemia, high alkaline phosphatase (ALP), high PTH and serum 25-OH D below ng/ml. The rest of the patients (n=36) had low circulating 25-OH D concentrations with normal serum Ca and PO4 concentrations. Of the 40 non-thalassemic adolescents, 26 had 25-OH D levels below 20 ng/ml (65%). Patients with T and VDD had lower circulating PTH and ALP concentrations compared to non-thalassemic patients with VDD. Significant improvement of symptoms related to vitamin D deficiency was reported in 18 out of 26 of symptomatic T and 12 out of 16 of NT adolescents at 1 to 3 months after the injection. Three months after injecting vitamin D the mean serum 25-OH D concentration was lower in the T group as compared to the NT group but the majority of patients had 25-OH D levels equal to or greater than 20 ng/ml.

CONCLUSION

Vitamin D deficiency was detected in 100 % of our thalassemic adolescents. An IM injection of a mega dose of cholecalciferol is an effective therapy for treatment of hypovitaminosis D in thalassemic and non-thalassemic adolescents for 3 months but its effects do not persist for 6 months.

摘要

未标注

重型β地中海贫血(T)青少年中钙稳态紊乱的患病率在不同人群中有所不同。此外,达到或维持任何给定血清25-羟胆钙化醇水平所需的胆钙化醇摄入量尚不清楚,尤其是在维生素可能的生理供应范围内。

目的

本研究的目的是估计40例青少年地中海贫血患者钙稳态异常的患病率,研究肌肉注射大剂量维生素D3对对维生素D缺乏(VDD)的地中海贫血青少年血清25-羟胆钙化醇(25-OH D)浓度及其他钙稳态参数的影响,并将这些结果与非地中海贫血VDD青少年的结果进行比较。

设计

在这项前瞻性研究中,我们测量了40例T青少年和40例匹配的非地中海贫血(NT)对照的钙稳态参数。对VDD患者(38例地中海贫血青少年和26例非地中海贫血青少年)肌肉注射剂量为10,000 IU/kg(最大600,000 IU)的胆钙化醇。在研究过程中,每隔3个月测量一次钙稳态参数。

结果

40例T青少年中,2例甲状旁腺功能减退且25-OH D水平低,2例低钙血症伴低磷血症、高碱性磷酸酶(ALP)、高甲状旁腺激素(PTH)且血清25-OH D低于ng/ml。其余患者(n = 36)循环25-OH D浓度低,但血清钙和磷酸浓度正常。40例非地中海贫血青少年中,26例25-OH D水平低于20 ng/ml(65%)。与非地中海贫血VDD患者相比,T和VDD患者的循环PTH和ALP浓度较低。注射后1至3个月,26例有症状的T青少年中有18例、16例NT青少年中有12例报告维生素D缺乏相关症状有显著改善。注射维生素D三个月后,T组的平均血清25-OH D浓度低于NT组,但大多数患者的25-OH D水平等于或高于20 ng/ml。

结论

我们所有的地中海贫血青少年均检测到维生素D缺乏。肌肉注射大剂量胆钙化醇对治疗地中海贫血和非地中海贫血青少年的维生素D缺乏症有效3个月,但效果不会持续6个月。

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