Knobler Robert M, French Lars E, Kim Youn, Bisaccia Emil, Graninger Winfried, Nahavandi Hesam, Strobl Frank J, Keystone Edward, Mehlmauer Marilyn, Rook Alain H, Braverman Irwin
Department of Dermatology, Medical University of Vienna, Vienna, Austria.
J Am Acad Dermatol. 2006 May;54(5):793-9. doi: 10.1016/j.jaad.2005.11.1091.
Systemic sclerosis is a multisystemic connective tissue disease with marked involvement of the skin and joints for which few effective evidence based therapies are available. To further investigate the efficacy of extracorporeal photochemotherapy on early aggressive cutaneous disease, a randomized, double-blind, placebo-controlled trial was performed.
Our aim was to evaluate the efficacy of photopheresis in the treatment of patients with systemic sclerosis (scleroderma).
This randomized, double-blind, placebo-controlled clinical trial was conducted at 16 investigational sites in the United States, Canada, and Europe. Sixty-four patients with typical clinical and histologic findings of scleroderma, of less than 2 years' duration, were studied. Patients did not receive any other concomitant treatment for scleroderma. Patients were randomized to receive either active or sham photopheresis treatment on two consecutive days monthly for 12 months. Severity of skin (skin scores assessed in 22 body regions) and joint involvement (60 joints examined for contractures) were assessed on a monthly basis.
A statistically significant improvement in skin scores as compared with baseline was observed at 6 months (P = .0024) and 12 months (P = .008) among those who received active photopheresis, but not among those who received sham photopheresis. Comparison of skin scores between the two study arms did not achieve statistical significance because of the small sample size of the study arms. Joint involvement was also significantly improved after 6 months (P = .002) and 12 months (P = .001) of active photopheresis when compared with baseline.
The study lacks sufficient statistical power to reveal a significant difference in skin and joint manifestations between the active and sham photopheresis arms.
Photopheresis induced significant improvement of skin and joint involvement in patients with scleroderma of recent onset; however, any effect when compared with sham treatment and a possible placebo effect may be modest.
系统性硬化症是一种多系统结缔组织疾病，皮肤和关节受累明显，目前几乎没有有效的循证治疗方法。为进一步研究体外光化学疗法对早期侵袭性皮肤疾病的疗效，开展了一项随机、双盲、安慰剂对照试验。
我们的目的是评估光分离置换法治疗系统性硬化症（硬皮病）患者的疗效。
这项随机、双盲、安慰剂对照临床试验在美国、加拿大和欧洲的16个研究地点进行。研究了64例硬皮病典型临床和组织学表现、病程少于2年的患者。患者未接受硬皮病的任何其他联合治疗。患者被随机分为两组，每月连续两天接受活性或假光分离置换法治疗，共12个月。每月评估皮肤严重程度（在22个身体部位评估皮肤评分）和关节受累情况（检查60个关节是否有挛缩）。
接受活性光分离置换法的患者在6个月（P = 0.0024）和12个月（P = 0.008）时，与基线相比皮肤评分有统计学显著改善，而接受假光分离置换法的患者则没有。由于研究组样本量小，两组之间的皮肤评分比较未达到统计学显著性。与基线相比，活性光分离置换法治疗6个月（P = 0.002）和12个月（P = 0.001）后，关节受累情况也有显著改善。
该研究缺乏足够的统计效力来揭示活性和假光分离置换法两组在皮肤和关节表现上的显著差异。
光分离置换法使近期发病的硬皮病患者的皮肤和关节受累情况有显著改善；然而，与假治疗相比的任何效果以及可能的安慰剂效应可能都较小。
