Singhal B S, Geeta Sheth, Hundalani Shilpa G, Menon Suresh
Department of Neurology, Bombay Hospital Institute of Medical Sciences, Mumbai, India.
Neurol India. 2009 Jul-Aug;57(4):418-23. doi: 10.4103/0028-3886.55611.
Mitoxantrone is an approved disease modifying agent for treatment of multiple sclerosis (MS). The aim of the study was to assess its efficacy and safety in Indian MS patients.
A total of 23 patients with clinically definite MS (Poser criteria) were enrolled in an open label study. Of which, 21 satisfied the McDonald's criteria for MS and two satisfied the diagnostic criteria of neuromyelitis optica (NMO). The numbers of relapses and expanded disability status scale (EDSS) score were used as primary and secondary outcome measures. The patients were monitored for the adverse effects.
In 17 (15 MS and two NMO) patients who completed one year of therapy, there was significant difference in the mean annual relapse rates [before 0.879+/-0.58; on mitoxantrone 0.091+/-0.17, (P=0.003)]. Of the 17 patients, ten (MS 9 and NMO 1) completed therapy for two years. Annual relapse rates [before (1.024+/-0.59), on therapy (0.155+/-0.21), (P=0.0054)] and EDSS score [before start of therapy 5.3, at the end of therapy 2.4, (P=0.001)] showed significant benefit in the ten patients who completed two years therapy. This benefit persisted during the mean follow-up period of two and a half years after completion of therapy. The adverse events noted in the entire cohort were leucopenia in four patients and asymptomatic reversible decrease in cardiac ejection fraction in one patient. Leucopenia was severe in two patients requiring discontinuation of the therapy and mitoxantrone was also discontinued in the patient with cardiotoxicity.
Mitoxantrone, as an initial therapy, decreases clinical exacerbations and disability progression, and has a reasonable safety profile in Indian patients with MS and NMO.
米托蒽醌是一种已获批准用于治疗多发性硬化症（MS）的疾病修饰药物。本研究的目的是评估其对印度MS患者的疗效和安全性。
共有23例临床确诊的MS患者（符合波泽标准）纳入一项开放标签研究。其中，21例符合MS的麦克唐纳标准，2例符合视神经脊髓炎（NMO）的诊断标准。复发次数和扩展残疾状态量表（EDSS）评分用作主要和次要结局指标。对患者进行不良反应监测。
17例（15例MS和2例NMO）完成一年治疗的患者，年平均复发率有显著差异[治疗前0.879±0.58；使用米托蒽醌时0.091±0.17，（P = 0.003）]。17例患者中，10例（9例MS和1例NMO）完成了两年治疗。年复发率[治疗前（1.024±0.59），治疗时（0.155±0.21），（P = 0.0054）]和EDSS评分[治疗开始前5.3，治疗结束时2.4，（P = 0.001）]在完成两年治疗的10例患者中显示出显著益处。这种益处持续到治疗完成后的平均两年半随访期。整个队列中观察到的不良事件为4例患者出现白细胞减少，1例患者出现无症状可逆性心脏射血分数降低。2例患者白细胞减少严重，需要停止治疗，出现心脏毒性的患者也停用了米托蒽醌。
米托蒽醌作为初始治疗药物，可减少临床病情加重和残疾进展，在印度MS和NMO患者中具有合理的安全性。
