A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease
A multicenter prospective phase 2 randomized study of extracorporeal photopheresis for treatment of chronic graft-versus-host disease
复制标题
DOI：
10.1182/blood-2008-03-141481
复制
发表时间：
2008-10-01
期刊：
影响因子：
20.3
通讯作者：
Greinix, Hildegard T.
中科院分区：
文献类型：
--
作者：
Flowers, Mary E. D.;Apperley, Jane F.;Greinix, Hildegard T.
慢性移植物抗宿主病（cGVHD）是成功的造血细胞移植的主要限制。在皮质类固醇治疗不能充分控制的cGVHD皮肤表现患者中，比较了体外循环血液分离术（ECP）12至24周联合标准治疗与单独标准治疗的安全性和有效性。主要疗效终点是第12周时10个身体区域的总皮肤评分（TSS）较基线的百分比变化的盲态定量比较。95名患者被随机分配至ECP和标准治疗组（n = 48）或单独标准治疗组（n = 47）.第12周时，ECP组TSS改善的中位百分比为14.5%，对照组为8.5%（P = 0.48）。第12周时，ECP组类固醇剂量至少降低50%且TSS较基线至少降低25%的患者比例为8.3%，对照组为0%（P = 0.04）。皮肤完全或部分反应的非盲研究者评估显示，支持ECP的显著改善（P < .001）。ECP通常耐受良好。这些结果表明，ECP可能有类固醇节约的作用，在治疗cGVHD。临床试验在www.临床试验gov as NCT 00054613.
Chronic graft-versus-host disease (cGVHD) is a major limitation of successful hematopoietic cell transplantation. The safety and efficacy of extracorporeal photopheresis (ECP) for 12 to 24 weeks together with standard therapy was compared with standard therapy alone in patients with cutaneous manifestations of cGVHD that could not be adequately controlled by corticosteroid treatment. The primary efficacy end point was a blinded quantitative comparison of percent change from baseline in Total Skin Score (TSS) of 10 body regions at week 12. Ninety- five patients were randomized to either ECP and standard therapy (n = 48) or standard therapy alone (n = 47). The median percentage improvement in TSS at week 12 was 14.5% for the ECP arm and 8.5% for the control arm (P = .48). The proportion of patients who had at least a 50% reduction in steroid dose and at least a 25% decrease from baseline in TSS was 8.3% in the ECP arm at week 12 and 0% in the control arm (P = .04). The nonblinded investigator assessment of skin complete or partial responses revealed a significant improvement in favor of ECP (P < .001). ECP was generally well tolerated. These results suggest that ECP may have a steroid-sparing effect in the treatment of cGVHD. Clinical trials registered at www. Clinical Trials. gov as NCT00054613.