Dismukes W E, Bennett J E, Drutz D J, Graybill J R, Remington J S, Stevens D A
Rev Infect Dis. 1980 Jul-Aug;2(4):535-45. doi: 10.1093/clinids/2.4.535.
A therapeutic trial of antifungal therapy in humans requires not only well-defined objectives and a sufficient number of patients, but also standardized criteria for the diagnosis and evaluation of response, including the efficacy, failure, and toxicity of any new agent or treatment modality. Generally, the retention of a patient in a study should require, as a minimum, confirmation of the etiologic diagnosis by culture of the organism from the infected site. Two different systems for evaluation of therapy are proposed. The first is a set of definitions of response to therapy; these are adaptable for use in any type of prospective clinical trial and emphasize cure as the best possible outcome. The second system of evaluation employs scored criteria and thereby enables quantitative comparison of the status of a patient before therapy with his or her status at intervals during therapy, at the end of therapy, and at intervals after therapy. This system provides an objective means of measuring improvement in a given patient; thus it is most useful in those diseases in which significant improvement rather than cure is a more realistic goal of therapy, e.g., coccidioidomycosis.
针对人类的抗真菌治疗试验不仅需要明确的目标和足够数量的患者，还需要用于诊断和评估反应的标准化标准，包括任何新药物或治疗方式的疗效、失败情况和毒性。一般来说，患者至少应通过从感染部位培养出病原体来确认病因诊断，才能继续留在研究中。本文提出了两种不同的治疗评估系统。第一种是一组治疗反应的定义；这些定义适用于任何类型的前瞻性临床试验，并强调治愈是最佳的可能结果。第二种评估系统采用评分标准，从而能够对患者治疗前的状态与治疗期间、治疗结束时以及治疗后的不同时间点的状态进行定量比较。该系统提供了一种衡量特定患者病情改善的客观方法；因此，它在那些以显著改善而非治愈作为更现实治疗目标的疾病中最为有用，例如球孢子菌病。
