Fagius J, Lundgren J, Oberg G
Department of Neurology, University Hospital, Uppsala, Sweden.
Mult Scler. 2009 Feb;15(2):229-37. doi: 10.1177/1352458508096875. Epub 2008 Sep 19.
During the last 15 years, high-dose chemotherapy with autologous hematopoietic stem cell transplantation (HSCT) has globally been performed for severe multiple sclerosis (MS). Most patients have been in progressive phase with long disease duration. As a rule, treatment effect has been minor or moderate.
Since 2004, we have performed HSCT in nine young patients with "malignant" relapsing-remitting MS. Criteria for treatment were short duration of disease; very frequent, severe relapses; recent improvement periods indicating potential for recovery after strong immunosuppression.
Median age at treatment was 27 (range 9-34) years, MS duration 26 (4-100) months, and annualized relapse rate 10 (4-12). Median Disability Status Scale (extended disability status scale, EDSS) at HSCT was 7.0 (3.5-8.0). Median follow-up time April 2008 is 29 (23-47) months. Median EDSS improvement is 3.5 (1.0-7.0), clearly surpassing most previous reports. One patient relapsed mildly with rapid recovery 7 months after HSCT. All patients are otherwise stable, median EDSS being 2.0 (0-6.0). Before HSCT, 61 relapses occurred in 82 patient months; during follow-up, one relapse in 289 patient months.
This small series of patients with "malignant" relapsing-remitting MS suggests HSCT to be an effective treatment option for this relatively rare disease course. It further suggests that future criteria for HSCT in MS should be close to the present ones.
在过去15年中，全球范围内已对重症多发性硬化症（MS）患者进行了大剂量化疗联合自体造血干细胞移植（HSCT）。大多数患者处于疾病进展期，病程较长。通常，治疗效果轻微或中等。
自2004年以来，我们对9例患有“恶性”复发缓解型MS的年轻患者进行了HSCT。治疗标准为病程短；复发频繁、严重；近期有改善期，表明在强力免疫抑制后有恢复潜力。
治疗时的中位年龄为27岁（范围9 - 34岁），MS病程为26个月（4 - 100个月），年化复发率为10次（4 - 12次）。HSCT时的中位残疾状态量表（扩展残疾状态量表，EDSS）为7.0（3.5 - 8.0）。截至2008年4月的中位随访时间为29个月（23 - 47个月）。EDSS的中位改善值为3.5（1.0 - 7.0），明显超过大多数先前的报告。1例患者在HSCT后7个月轻度复发，但恢复迅速。其他所有患者病情稳定，中位EDSS为2.0（0 - 6.0）。HSCT前，82个患者月内发生61次复发；随访期间，289个患者月内发生1次复发。
这一小系列患有“恶性”复发缓解型MS的患者表明，HSCT是这种相对罕见病程的有效治疗选择。这还表明，未来MS患者HSCT的标准应与目前的标准相近。
