Lahey M E, Heyn R M, Newton W A, Shore N, Smith W B, Leikin S, Hammond D
Med Pediatr Oncol. 1979;7(3):197-203. doi: 10.1002/mpo.2950070302.
A prospective study for histiocytosis X was designed to determine whether "good risk" patients, ie, those without evidence of dysfunction of liver, lung, or hemopoietic system, would respond to single agent therapy; in this case chlorambucil (CMB) used in a dose of 5 mgm/m2/day. If there was no response after an adequate trial period, treatment was initiated with four drugs using a combination of prednisone, vinblastine, cyclophosphamide and methotrexate. There were 26 evaluable patients, 57% of whom were less than two years of age at onset of therapy. There were three complete and four partial responses to CMB for a response rate of 26.9%. Sixteen patients received an adequate trial of four-drug therapy with three complete and two partial responses for a response rate of 33%. These responses were inferior to those previously reported for either single agents or combined therapy in histiocytosis X.
一项关于朗格汉斯细胞组织细胞增多症X的前瞻性研究旨在确定“低风险”患者，即那些没有肝脏、肺部或造血系统功能障碍证据的患者，是否会对单药治疗产生反应；在本研究中使用的单药是苯丁酸氮芥（CMB），剂量为5毫克/平方米/天。如果在足够的试验期后没有反应，则开始使用泼尼松、长春新碱、环磷酰胺和甲氨蝶呤联合的四种药物进行治疗。共有26例可评估患者，其中57%在开始治疗时年龄小于两岁。CMB治疗有3例完全缓解和4例部分缓解，缓解率为26.9%。16例患者接受了四种药物治疗的充分试验，有3例完全缓解和2例部分缓解，缓解率为33%。这些缓解率低于先前报道的朗格汉斯细胞组织细胞增多症X单药治疗或联合治疗的缓解率。
