Elst E F, Van Suijlekom-Smit L W, Oranje A P
Department of Dermatology and Venereology, University Hospital Rotterdam, The Netherlands.
Pediatr Dermatol. 1999 Jan-Feb;16(1):53-8. doi: 10.1046/j.1525-1470.1999.99016.x.
Linear scleroderma is a connective tissue disorder that characteristically involves the skin. Skin induration and pigmentary changes present in a linear distribution. Severe functional and cosmetic disability may occur, especially in growing children. No effective therapy for the fibrotic stage of scleroderma is available at present. Recently a beneficial effect of oral 1, 25-dihydroxyvitamin D3 (calcitriol) treatment was reported in adults. Calcitriol has a dose-dependent inhibition on fibroblast proliferation and collagen synthesis and has immunoregulatory activities. We assessed the efficacy of oral calcitriol treatment in seven pediatric patients with linear scleroderma. During the treatment dietary calcium intake was restricted. Calcium, inorganic phosphate, creatinine, and urea in the serum and urine was monitored. The urinary calcium:creatinine ratio was measured. The effects of the treatment were evaluated using a clinical scoring system. No side effects were observed. Five of the seven patients showed a good to excellent improvement of their lesions. One of them partly relapsed after 19 months, but showed an excellent response to a second therapy session with calcitriol. One patient with rapidly progressive disease failed to respond to therapy. Our results indicate that calcitriol can be an effective agent for treating localized scleroderma in children.
线状硬皮病是一种典型累及皮肤的结缔组织疾病。皮肤硬化和色素改变呈线状分布。严重的功能和外观残疾可能会出现，尤其是在正在成长的儿童中。目前对于硬皮病纤维化阶段尚无有效的治疗方法。最近有报道称，口服1,25 - 二羟维生素D3（骨化三醇）治疗对成人有效。骨化三醇对成纤维细胞增殖和胶原蛋白合成具有剂量依赖性抑制作用，并具有免疫调节活性。我们评估了口服骨化三醇治疗7例儿童线状硬皮病患者的疗效。治疗期间限制饮食中的钙摄入量。监测血清和尿液中的钙、无机磷、肌酐和尿素。测量尿钙：肌酐比值。使用临床评分系统评估治疗效果。未观察到副作用。7例患者中有5例病变有良好至极佳的改善。其中1例在19个月后部分复发，但对第二次骨化三醇治疗疗程反应良好。1例疾病快速进展的患者对治疗无反应。我们的结果表明，骨化三醇可能是治疗儿童局限性硬皮病的有效药物。
