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醋酸格拉替雷治疗儿童和青少年期多发性硬化症患者。

Glatiramer acetate treatment in patients with childhood and juvenile onset multiple sclerosis.

作者信息

Kornek B, Bernert G, Balassy C, Geldner J, Prayer D, Feucht M

机构信息

Department of Neuropsychiatry of Childhood and Adolescence, University of Vienna, Vienna, Austria.

出版信息

Neuropediatrics. 2003 Jun;34(3):120-6. doi: 10.1055/s-2003-41274.

Abstract

BACKGROUND

Recent data indicate that in multiple sclerosis disease onset before the age of 16 is more common than previously assumed. However, current therapeutic options are limited to the treatment of acute attacks in these patients. Glatiramer acetate has been successfully applied in adults with relapsing-remitting multiple sclerosis, but there are no data available about the use of glatiramer acetate in childhood and juvenile onset multiple sclerosis.

METHODS

Seven patients with relapsing-remitting multiple sclerosis and disease onset between 9 and 16 years of age were treated with daily subcutaneous injection of 20 mg glatiramer acetate (Copaxone). Patients were followed for 24 months. Treatment was initiated in all patients before the age of 18.

RESULTS

The use of glatiramer acetate in our cohort of early onset multiple sclerosis patients was safe and well tolerated. Two out of seven patients remained relapse-free during the two year period. Clinical disability as measured by the EDSS remained stable in three out of seven patients.

CONCLUSION

Due to the small number of patients the efficacy of the treatment has to be interpreted with caution. However, there might be a more pronounced treatment benefit in patients with low disability at treatment initiation and early treatment onset.

摘要

背景

最近的数据表明,16岁之前发病的多发性硬化症比之前认为的更为常见。然而,目前的治疗选择仅限于治疗这些患者的急性发作。醋酸格拉替雷已成功应用于复发缓解型多发性硬化症的成人患者,但尚无关于醋酸格拉替雷在儿童和青少年期发病的多发性硬化症中应用的数据。

方法

7例复发缓解型多发性硬化症患者,发病年龄在9至16岁之间,每日皮下注射20mg醋酸格拉替雷(考帕松)进行治疗。对患者进行了24个月的随访。所有患者均在18岁之前开始治疗。

结果

在我们的早发性多发性硬化症患者队列中,使用醋酸格拉替雷是安全的,耐受性良好。7例患者中有2例在两年期间无复发。用扩展残疾状态量表(EDSS)衡量的临床残疾在7例患者中有3例保持稳定。

结论

由于患者数量较少,治疗效果必须谨慎解读。然而,在治疗开始时残疾程度较低且治疗开始较早的患者中,可能会有更显著的治疗益处。

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