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硫代硫酸钠治疗儿童和青年钙化性尿毒症小动脉病

Sodium thiosulfate treatment for calcific uremic arteriolopathy in children and young adults.

作者信息

Araya Carlos E, Fennell Robert S, Neiberger Richard E, Dharnidharka Vikas R

机构信息

Division of Pediatric Nephrology, Department of Pediatrics, University of Florida Health Science Center, Gainesville, FL 32610-0296, USA.

出版信息

Clin J Am Soc Nephrol. 2006 Nov;1(6):1161-6. doi: 10.2215/CJN.01520506. Epub 2006 Aug 30.

Abstract

In adult patients with ESRD, calcific uremic arteriolopathy (CUA) is an uncommon but life-threatening complication. No effective therapy exists, although anecdotal case reports highlight the use of sodium thiosulfate (STS), a calcium-chelating agent with antioxidant properties. CUA is rare in children, and STS use has not been reported. The objective of this study was to determine the influence of STS treatment on three patients with CUA in a pediatric chronic dialysis unit. The patients were between 12 and 21 yr of age; two were male; and primary diagnoses were obstructive uropathy, renal dysplasia, and calcineurin nephrotoxicity. Time from ESRD to CUA diagnosis was 1, 9, and 20 yr. Diagnosis was made by tissue biopsy and three-phase bone scan. Pain was the presenting symptom. Initial treatment included discontinuation of calcitriol and use of non-calcium-based phosphate binders and low-calcium dialysate concentration. STS dosage was 25 g/1.73 m(2) per dose intravenously after each hemodialysis session. For optimization of removal of calcium deposits, patient three received a combination of STS and continuous venovenous hemofiltration for the first 10 d. All patients demonstrated rapid pain relief. Within weeks, skin induration and joint mobility of the extremities improved. Radiographic evidence of reduction in the calcium deposits occurred within 3 mo of initiation of STS. The only complication was prolonged QT interval in one patient as a result of hypocalcemia, who was resolved by use of a higher dialysate calcium concentration. STS seems well tolerated in children and young adults with CUA and has mild adverse effects. For determination of its efficacy, optimum dosage, duration of therapy, and dialysis modality, controlled trials are needed.

摘要

在成年终末期肾病(ESRD)患者中,钙化性尿毒症小动脉病(CUA)是一种罕见但危及生命的并发症。尽管有个别病例报告强调使用具有抗氧化特性的钙螯合剂硫代硫酸钠(STS),但目前尚无有效的治疗方法。CUA在儿童中罕见,且尚未有使用STS的报道。本研究的目的是确定STS治疗对一家儿科慢性透析单位的3例CUA患者的影响。患者年龄在12至21岁之间;2例为男性;主要诊断为梗阻性尿路病、肾发育不良和钙调神经磷酸酶肾毒性。从ESRD到CUA诊断的时间分别为1年、9年和20年。通过组织活检和三相骨扫描进行诊断。疼痛是主要症状。初始治疗包括停用骨化三醇,使用非钙基磷结合剂和低钙透析液浓度。每次血液透析后静脉注射STS剂量为25 g/1.73 m²。为了优化钙沉积物的清除,患者3在开始的10天内接受了STS与持续静静脉血液滤过联合治疗。所有患者均表现出疼痛迅速缓解。数周内,皮肤硬结和四肢关节活动度改善。在开始使用STS的3个月内出现了钙沉积物减少的影像学证据。唯一的并发症是1例患者因低钙血症出现QT间期延长,通过使用较高的透析液钙浓度得以解决。STS在患有CUA的儿童和年轻人中似乎耐受性良好,且不良反应轻微。为了确定其疗效、最佳剂量、治疗持续时间和透析方式,需要进行对照试验。

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