Makhani N, Gorman M P, Branson H M, Stazzone L, Banwell B L, Chitnis T
The Hospital for Sick Children, University of Toronto, Canada.
Neurology. 2009 Jun 16;72(24):2076-82. doi: 10.1212/WNL.0b013e3181a8164c. Epub 2009 May 13.
To review our multicenter experience with cyclophosphamide in the treatment of children with multiple sclerosis (MS).
Retrospective chart review of children with MS treated with cyclophosphamide. Demographic, clinical, treatment, and MRI parameters were collected.
We identified 17 children with MS treated with cyclophosphamide. All but one had worsening of Expanded Disability Status Scale scores or multiple relapses prior to treatment initiation. Children were treated with one of three regimens: 1) induction therapy alone; 2) induction therapy with pulse maintenance therapy; or 3) pulse maintenance therapy alone. Treatment resulted in a reduction in relapse rate and stabilization of disability scores assessed 1 year after treatment initiation in the majority of patients. Longer follow-up was available for most cases. Cyclophosphamide was well tolerated in most patients. However, side effects included vomiting, transient alopecia, osteoporosis, and amenorrhea. One patient developed bladder carcinoma that was successfully treated.
Cyclophosphamide is an option for the treatment of children with aggressive multiple sclerosis refractory to first-line therapies. Recommendations regarding patient selection, treatment administration, and monitoring are discussed.
回顾我们使用环磷酰胺治疗儿童多发性硬化症（MS）的多中心经验。
对接受环磷酰胺治疗的MS儿童进行回顾性病历审查。收集人口统计学、临床、治疗和MRI参数。
我们确定了17例接受环磷酰胺治疗的MS儿童。除1例之外，所有患儿在开始治疗前扩展残疾状态量表评分均恶化或出现多次复发。患儿接受三种治疗方案之一：1）单纯诱导治疗；2）诱导治疗加脉冲维持治疗；或3）单纯脉冲维持治疗。治疗使大多数患者在开始治疗1年后复发率降低且残疾评分稳定。大多数病例有更长时间的随访。大多数患者对环磷酰胺耐受性良好。然而，副作用包括呕吐、短暂脱发、骨质疏松和闭经。1例患者发生膀胱癌，经成功治疗。
环磷酰胺是治疗一线疗法难治的侵袭性儿童多发性硬化症的一种选择。讨论了关于患者选择、治疗给药和监测的建议。
