Goodkin D E, Plencner S, Palmer-Saxerud J, Teetzen M, Hertsgaard D
Arch Neurol. 1987 Aug;44(8):823-7. doi: 10.1001/archneur.1987.00520200027013.
Twenty-seven patients with chronic progressive multiple sclerosis were treated with high-dose intravenous cyclophosphamide induction on either an impatient or outpatient basis. Following induction, patients were randomized to alternate-month outpatient "maintenance" or "no maintenance" therapy. These groups, as well as 24 nonrandomized control patients, were compared with each other after 12, 18, and 24 months of follow-up. All groups were similar in age, sex, duration of disease, and degree of disability before treatment. Fifty-nine percent of all cyclophosphamide-treated patients were stable at 12 months compared with 17% of all patients in the nonrandomized control group at 12 months. A statistically significant difference persisted at 18 and 24 months. A trend favoring maintenance therapy when compared with no maintenance therapy was evident at 12, 18, and 24 months, but was not statistically significant. Inpatient vs outpatient induction therapy failed to influence treatment outcome. Toxic side effects of nausea and vomiting presented a serious obstacle to maintenance therapy as administered in this protocol.
27例慢性进行性多发性硬化症患者接受了大剂量静脉注射环磷酰胺诱导治疗，治疗方式为住院或门诊治疗。诱导治疗后，患者被随机分为接受每月一次门诊“维持”治疗或“不进行维持”治疗的两组。在随访12、18和24个月后，对这些组以及24例未随机分组的对照患者进行了相互比较。所有组在年龄、性别、病程和治疗前残疾程度方面相似。在12个月时，所有接受环磷酰胺治疗的患者中有59%病情稳定，而未随机分组的对照组患者在12个月时这一比例为17%。在18个月和24个月时，这种差异仍具有统计学意义。在12、18和24个月时，与不进行维持治疗相比，维持治疗有明显的优势趋势，但无统计学意义。住院诱导治疗与门诊诱导治疗对治疗结果没有影响。恶心和呕吐等毒性副作用对本方案中实施的维持治疗构成了严重障碍。
