Weening R S, Leitz G J, Seger R A
Emma Children's Hospital, Department of Paediatrics, Amsterdam, The Netherlands.
Eur J Pediatr. 1995 Apr;154(4):295-8. doi: 10.1007/BF01957365.
This was an uncontrolled, open-label follow up study of a previous 12-month, randomized, double-blind, placebo-controlled trial performed to assess the long-term efficacy and safety of Recombinant Human Interferon Gamma (rIFN-gamma) in patients with chronic granulomatous disease (CGD). In two centres, 28 patients (24 male, 4 female) with a mean age of 16 years (range 3-37) entered the open-label phase. The patients were treated for a mean of 880 days (range 97-1375 days). Visits were scheduled every 180 days and patients completed one to six visits. rIFN-gamma was administered subcutaneously three times weekly at a dose of 0.05 mg per m2. During the open-label phase of the study 12 patients experienced a serious infection requiring hospitalization within 880 days. The median infection-free time was 993 days. No obvious increase of infections over time was seen. Phagocyte superoxide anion production and phagocyte staphylococcal killing were not influenced by therapy. Seven patients were withdrawn from the study, one because of an adverse reaction, three on their own wish and the other three because they changed to another trial. No patient died during the study. Conclusion. Treatment of patients with CGD with intracellular active antibiotics and additional interferon gamma as infection prophylaxis is safe and justified.
这是一项非对照、开放标签的随访研究，其基于之前一项为期12个月的随机、双盲、安慰剂对照试验开展，该试验旨在评估重组人干扰素γ（rIFN-γ）对慢性肉芽肿病（CGD）患者的长期疗效和安全性。在两个中心，28名患者（24名男性，4名女性）进入开放标签阶段，平均年龄为16岁（范围3 - 37岁）。患者平均接受治疗880天（范围97 - 1375天）。每180天安排一次随访，患者完成了1至6次随访。rIFN-γ每周皮下注射三次，剂量为每平方米0.05毫克。在研究的开放标签阶段，12名患者在880天内发生严重感染需要住院治疗。无感染的中位时间为993天。未观察到感染随时间明显增加。吞噬细胞超氧阴离子产生和吞噬细胞对葡萄球菌的杀伤不受治疗影响。7名患者退出研究，1名因不良反应退出，3名自行退出，另外3名因转至另一项试验而退出。研究期间无患者死亡。结论。用细胞内活性抗生素及额外的干扰素γ治疗CGD患者以预防感染是安全且合理的。
