Cheson B D, Cassileth P A, Head D R, Schiffer C A, Bennett J M, Bloomfield C D, Brunning R, Gale R P, Grever M R, Keating M J
Clinical Investigations Branch, National Cancer Institute, Bethesda, MD 20892.
J Clin Oncol. 1990 May;8(5):813-9. doi: 10.1200/JCO.1990.8.5.813.
The National Cancer Institute (NCI) sponsored a workshop to develop a set of standardized diagnostic and response criteria for acute myeloid leukemia (AML) clinical trials. The French-American-British (FAB) classification was retained for diagnosing AML, with the addition of patients with bone marrow morphologic features of a myelodysplastic syndrome and less than 30% bone marrow blasts, but with greater than or equal to 30% blasts in the peripheral blood. In this report, there are four important subgroups of AML not defined in the FAB classification that are discussed: undifferentiated acute leukemia, MO (AML lacking definitive myeloid differentiation by morphology or conventional cytochemistry but with ultrastructural or immunophenotypic evidence for AML), mixed lineage leukemia, and hypocellular AML. Definitions of response for clinical trials are presented to facilitate comparisons among different studies. Complete remission is considered the only response worth reporting in phase III trials, since lesser responses do not improve survival. Partial remissions may be of interest to identify active new agents in phase I and II studies. Monoclonal antibodies and cytogenetic studies are not part of the routine assessment of remission or reassessment at relapse, and their role in the evaluation of patients with AML is currently being evaluated in clinical trials. Although we recognize that some of the definitions in this report are arbitrary, generalized use of these guidelines will make results of clinical trials more comparable and interpretable.
美国国立癌症研究所（NCI）主办了一次研讨会，旨在为急性髓系白血病（AML）临床试验制定一套标准化的诊断和反应标准。AML的诊断保留了法美英（FAB）分类法，新增了具有骨髓增生异常综合征骨髓形态学特征且骨髓原始细胞小于30%，但外周血原始细胞大于或等于30%的患者。在本报告中，讨论了FAB分类法未定义的AML的四个重要亚组：未分化急性白血病、MO（形态学或传统细胞化学缺乏明确髓系分化但有AML超微结构或免疫表型证据的AML）、混合谱系白血病和低细胞性AML。给出了临床试验反应的定义，以方便不同研究之间的比较。完全缓解被认为是III期试验中唯一值得报告的反应，因为较轻的反应并不能提高生存率。部分缓解可能有助于在I期和II期研究中识别有活性的新药。单克隆抗体和细胞遗传学研究不是缓解常规评估或复发时重新评估的一部分，它们在AML患者评估中的作用目前正在临床试验中进行评估。尽管我们认识到本报告中的一些定义是任意的，但广泛使用这些指南将使临床试验结果更具可比性和可解释性。
