TREATMENT OF SYSTEMIC-SCLEROSIS WITH EXTRACORPOREAL PHOTOCHEMOTHERAPY - RESULTS OF A MULTICENTER TRIAL
TREATMENT OF SYSTEMIC-SCLEROSIS WITH EXTRACORPOREAL PHOTOCHEMOTHERAPY - RESULTS OF A MULTICENTER TRIAL
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DOI：
10.1001/archderm.128.3.337
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发表时间：
1992-03-01
影响因子：
--
通讯作者：
EDELSON, RL
中科院分区：
文献类型：
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作者：
ROOK, AH;FREUNDLICH, B;EDELSON, RL
背景和设计。在体外光化学疗法的试点研究中，两名接受这种疗法的系统性硬化症患者经历了显着的临床改善。这些结果促使了一项多中心试验的发展，以检查体外光化学疗法治疗系统性硬化症的益处。79例近期发作的系统性硬化症患者（平均症状持续时间为1.83年），在过去6个月内进行性皮肤受累，进入一项随机、平行组、单盲临床试验，比较每月连续2天给予的体外光化学治疗与最大剂量为750 mg/d的D-青霉胺治疗。设盲的临床检查者评估皮肤严重程度评分（厚度）、受累表面积百分比、口孔和手闭合。同时进行一系列皮肤活检和肺功能检查。- 治疗6个月后，31名接受光化学疗法的患者中有21名（68%）和25名接受D-青霉胺治疗的患者中有8名（32%）的皮肤严重程度评分出现显著改善，而31名接受光化学疗法的患者中有3名（10%）和25名接受青霉胺治疗的患者中有8名（32%）出现显著恶化，因此表明接受光化学疗法的个体的反应率显著更高（P = 0.02）。在6个月和10个月的评估点，接受光化疗的患者的平均皮肤严重程度评分、平均皮肤受累百分比和平均口腔孔径测量值均较基线显著改善。平均右手和左手闭合测量值在治疗10个月时也有显著改善。相比之下，在接受D-青霉胺治疗的患者中，在治疗6个月后，皮肤疾病的参数均无显著改善，尽管对于那些继续治疗的患者，平均皮肤严重程度评分和平均皮肤受累百分比改善了10个月。皮肤活检研究揭示了临床改善与真皮层厚度降低之间的相关性。体外光化学疗法的不良反应是最小的，并不需要停止治疗的任何患者接受这种疗法; 6例患者永久停止使用D-去甲肾上腺素治疗由于不良反应。- 对于近期发作的系统性硬化症患者，体外光化学疗法是一种耐受性良好的治疗方法，可能部分逆转导致皮肤硬化的过程。
Background and Design. -In a pilot study of extracorporeal photochemotherapy, two patients with systemic sclerosis who received this therapy experienced significant clinical improvement. These results prompted the development of a multicenter trial to examine the benefit of extracorporeal photochemotherapy in the treatment of systemic sclerosis. Seventy-nine patients with systemic sclerosis of recent onset (mean symptom duration, 1.83 years) and progressive skin involvement during the preceding 6 months entered a randomized, parallel-group, single-blinded clinical trial comparing extracorporeal photochemotherapy treatments given on 2 consecutive days monthly with treatment with D-penicillamine at a maximum dose of 750 mg/d. Blinded clinical examiners evaluated skin severity score (thickness), percent surface area involvement, oral aperture, and hand closure. Serial skin biopsies and pulmonary function studies were also performed.Results. -Following 6 months of treatment, significant improvement in skin severity score occurred in 21 (68%) of 31 patients receiving photochemotherapy and in eight (32%) of 25 receiving D-penicillamine treatment, while significant worsening occurred in three (10%) of 31 receiving photochemotherapy and in eight (32%) of 25 receiving penicillamine treatment, thus indicating a significantly higher response rate for individuals who received photochemotherapy (P = .02). At both the 6- and 10-month evaluation points, the mean skin severity score, mean percent skin involvement, and mean oral aperture measurements were significantly improved from baseline among those who received photochemotherapy. Mean right and left hand closure measurements had also improved significantly by 10 months of therapy. By comparison, among the patients treated with D-penicillamine, none of the parameters of cutaneous disease had improved significantly after 6 months of therapy, although for those individuals in whom treatment was continued, the mean skin severity score and mean percent skin involvement had improved by 10 months. Skin biopsy studies revealed a correlation between clinical improvement and decreased thickness of the dermal layer. Adverse effects of extracorporeal photochemotherapy were minimal and did not require discontinuation of treatment in any of the patients receiving this therapy; six patients permanently discontinued the use of D-pencillamine treatment due to adverse effects.Conclusions. -For patients with systemic sclerosis of recent onset, extracorporeal photochemotherapy is a well-tolerated treatment that may partially reverse the process that results in cutaneous sclerosis.