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帕米膦酸盐是治疗β地中海贫血患者骨质疏松症的有效药物。

Pamidronate is an effective treatment for osteoporosis in patients with beta-thalassaemia.

作者信息

Voskaridou Ersi, Terpos Evangelos, Spina Giovanna, Palermos John, Rahemtulla Amin, Loutradi Aphroditi, Loukopoulos Dimitris

机构信息

Thalassaemia Centre, Laikon General Hospital, Athens, Greece.

出版信息

Br J Haematol. 2003 Nov;123(4):730-7. doi: 10.1046/j.1365-2141.2003.04657.x.

DOI:10.1046/j.1365-2141.2003.04657.x
PMID:14616979
Abstract

Osteoporosis in beta-thalassaemia is multifactorial; increased osteoclast function seems to play an important role in its pathogenesis. The aim of this study was to evaluate the effect of pamidronate on the osteoporosis of thalassaemia. To this effect we studied 26 patients who received this drug in doses of 30 or 60 mg i.v. once a month over 12 months. The effects were monitored by measuring bone mineral density (BMD) in association with markers of osteoclast function [soluble receptor activator of nuclear factor-kappa B ligand (sRANKL), osteoprotegerin (OPG)] and of bone remodelling [N-telopeptide of collagen type-I (NTX), tartrate-resistant acid phosphatase isoform-5b (TRACP-5b), bone-alkaline phosphatase (bALP), and osteocalcin (OC)]. Thirty healthy individuals were also studied, as controls. NTX, TRACP-5b, bALP and OC levels were significantly higher in thalassaemic patients compared with controls; in contrast, OPG levels were significantly lower, while the levels of sRANKL varied within normal limits. Administration of pamidronate was followed by a clear decrease of NTX, TRACP-5b, OPG, and OC, and by a significant increase in the BMD of the lumbar spine, which was similar in patients of both treatment groups. These data suggest that pamidronate, at a monthly dose of 30 mg, is an effective treatment for thalassaemic osteoporosis.

摘要

β地中海贫血中的骨质疏松是多因素导致的;破骨细胞功能增强似乎在其发病机制中起重要作用。本研究的目的是评估帕米膦酸盐对地中海贫血骨质疏松的影响。为此,我们研究了26例患者,他们每月静脉注射一次该药物,剂量为30或60毫克,持续12个月。通过测量骨密度(BMD)并结合破骨细胞功能标志物[核因子κB受体活化因子配体(sRANKL)、骨保护素(OPG)]以及骨重塑标志物[Ⅰ型胶原N-端肽(NTX)、抗酒石酸酸性磷酸酶同工酶5b(TRACP-5b)、骨碱性磷酸酶(bALP)和骨钙素(OC)]来监测效果。还研究了30名健康个体作为对照。与对照组相比,地中海贫血患者的NTX、TRACP-5b、bALP和OC水平显著更高;相反,OPG水平显著更低,而sRANKL水平在正常范围内变化。给予帕米膦酸盐后,NTX、TRACP-5b、OPG和OC明显降低,腰椎骨密度显著增加,两个治疗组的患者情况相似。这些数据表明,每月30毫克剂量的帕米膦酸盐是治疗地中海贫血骨质疏松的有效方法。

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