ANALYSIS OF CONTROLLED TRIAL OF CHLORAMBUCIL VS. PLACEBO

硬皮病（PSS）不是一种良性疾病， 死亡率和发病率。 直到最近， 这种疾病的治疗进展甚微。 一些药物有 但没有一种被证明是有效的。 在过去的9年里，我们参与了一项双盲、平行、 苯丁酸氮芥与安慰剂的研究，其中每名患者都参加了研究 三年的药。 目前，我们已经招募了70名患者。 的 本研究产生的数据质量导致 发表了18篇描述性文章，并编写了几份 更多. 这些出版物证明了我们有能力保持 数据。 此外，我们能够保持双盲性质， 的研究。 在本申请中，我们建议分析本研究的结果。 使用统计技术，如卡方分析，t统计， 方差分析、协方差分析、多变量技术和功效分析， 将检查结果指标，如死亡率，失败率或辍学率， 功能结局的差异以及个体和组合 随着时间的推移内脏功能的测量。 在2年结束时 根据本分析的要求，我们将：1）评估以下药物的疗效： 苯丁酸氮芥稳定和/或改善PSS; 2）评价了 一组客观上 测量PSS活性的变化; 3）希望有发展的预后 预测哪些患者最能从这种药物中获益 疗法

Scleroderma (PSS) is not a benign disease and is associated with high mortality and morbidity. Until very recently there has been remarkably little progress in the treatment of this disease. A number of drugs have been studied but none have been shown to be efficacious. Over the last 9 years we have been involved in a double-blind, parallel, study of chlorambucil versus placebo in which each patient is on the study drug for 3 years. At the present time we have enrolled 70 patients. The quality of the data generated from this study has resulted in the publication of 18 descriptive articles and the preparation of several more. These publications attest to our ability to maintain the quality of the data. Further, we have been able to maintain the double-blind nature of the study. We propose, in this application, to analyze the results of this study. Using statistical techniques such as chi-square analysis, t statistics, ANOVA, covariance analysis, multi-variate techniques and power analysis, we will examine outcome measures such as mortality, failure or drop out rates, differences in functional outcome and changes in individual and combined measures of visceral function over time. At the end of the 2 years required for this analysis we will: 1) have evaluated the efficacy of Chlorambucil in stablizing and/or improving PSS; 2) have evaluated the reliability and sensitivity of a set of measurements which objectively measure changes in PSS activity; 3) hope to have developed prognostic indicies to predict which patients could most benefit from this drug therapy.