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两名多发性硬化症儿科患者的长期芬戈莫德治疗。

Long-term fingolimod treatment in two pediatric patients with multiple sclerosis.

机构信息

Sant'Andrea Hospital, Rome, Italy.

Department of Neurology and Psychiatry, La Sapienza University of Rome, Rome, Italy.

出版信息

Neurol Sci. 2021 May;42(Suppl 1):29-36. doi: 10.1007/s10072-021-05116-2. Epub 2021 Mar 10.

Abstract

Data suggest that patients with pediatric-onset multiple sclerosis (POMS) should initiate treatment with a disease-modifying therapy early to slow progression. The PARADIGMS trial demonstrated that oral fingolimod reduced the annual rate of relapse by 82% compared with intramuscular interferon beta-1a in children with POMS. The PARADIGMS study had a follow-up of 2 years, but no data are available about the safety and efficacy of fingolimod for longer periods in children with POMS. Here we present two cases of children with POMS who achieved sustained clinical benefit from treatment with fingolimod for more than 2 years. The first patient, an 11-year-old male, who participate in the PARADIGMS study, was treatment naïve at the time of fingolimod initiation. His clinical condition remained stable over 5 years of treatment, with no relapses and no radiological lesion progression. The second patient was a female who initiated fingolimod at the age of 12 years, 2 years after her POMS diagnosis and after an 8-month trial of interferon beta-1a. The patient had experienced two relapses during interferon beta-1a but had no relapses in more than 2 years of treatment with fingolimod, and her MRI scans showed no new or active lesions. These data show that prolonged treatment with fingolimod can be safe and effective during long-term treatment as first- or second-line therapy in children with POMS.

摘要

数据表明,儿科发病多发性硬化症(POMS)患者应尽早开始使用疾病修正疗法,以减缓疾病进展。PARADIGMS 试验表明,与肌内干扰素β-1a 相比,口服 fingolimod 可使儿科 POMS 患者的年复发率降低 82%。PARADIGMS 研究的随访时间为 2 年,但尚无儿童 POMS 患者使用 fingolimod 更长时间的安全性和疗效数据。在此,我们报告两例儿科 POMS 患者,他们在接受 fingolimod 治疗超过 2 年后获得了持续的临床获益。第一例患者为 11 岁男性,参加了 PARADIGMS 研究,在开始使用 fingolimod 时处于治疗初治期。他的临床状况在 5 年的治疗期间保持稳定,无复发且无影像学病变进展。第二位患者是一名女性,在 POMS 诊断后 2 年,在干扰素β-1a 试验 8 个月后开始使用 fingolimod。该患者在干扰素β-1a 治疗期间经历了 2 次复发,但在使用 fingolimod 治疗超过 2 年后无复发,其 MRI 扫描显示无新发或活动性病变。这些数据表明,在儿童 POMS 患者中,作为一线或二线治疗药物,延长 fingolimod 的治疗时间可以是安全且有效的。

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