Luchesa Smith Aphra, Benetou Christina, Bullock Hayley, Kuczynski Adam, Rudebeck Sarah, Hanson Katie, Crichton Sarah, Mankad Kshitij, Siddiqui Ata, Byrne Susan, Lim Ming, Hemingway Cheryl
University College London Medical School, London WC1E 6DE, UK.
Children's Neurosciences, Evelina London Children's Hospital, Guy's and St Thomas' NHS Foundation Trust, London SE1 7EH, UK.
Children (Basel). 2020 Nov 9;7(11):222. doi: 10.3390/children7110222.
Considerable progress has been made in the understanding and treatment of paediatric-onset multiple sclerosis (POMS); how this has translated into more effective care is less well understood. Here, we evaluate how recent advances have affected patient management and outcomes with a retrospective review of POMS patients managed at two paediatric neuroimmunology centres. Two cohorts, seen within a decade, were compared to investigate associations between management approaches and outcomes. Demographic, clinical and neurocognitive data were extracted from case notes and analysed. Of 51 patients, 24 were seen during the period 2007-2010 and 27 during the period 2015-2016. Median age at onset was 13.7 years; time from symptom onset to diagnosis was 9 months. Disease-modifying therapies were commenced in 19 earlier-cohort and 24 later-cohort patients. Median time from diagnosis to treatment was 9 months for earlier vs. 3.5 months in later patients ( 0.013). A wider variety of treatments were used in the later cohort (four medications earlier vs. seven in the later and two clinical trials), with increased quality of life and neurocognitive monitoring (8% vs. 48% completed PedsQL quality of life inventory; 58% vs. 89% completed neurocognitive assessment). In both cohorts, patients were responsive to disease-modifying therapy (mean annualised relapse rate pre-treatment 2.7 vs. 1.7, mean post-treatment 0.74 vs. 0.37 in earlier vs. later cohorts). In conclusion, over the years, POMS patients were treated sooner with a wider variety of medications and monitored more comprehensively. However, this hugely uncontrolled cohort did not allow us to identify key determinants for the improvements observed.
在儿童多发性硬化症（POMS）的理解和治疗方面已经取得了相当大的进展；然而，这些进展如何转化为更有效的护理却鲜为人知。在此，我们通过对在两个儿科神经免疫学中心接受治疗的POMS患者进行回顾性研究，评估近期进展如何影响患者管理和治疗结果。比较了十年内观察到的两个队列，以研究管理方法与治疗结果之间的关联。从病例记录中提取人口统计学、临床和神经认知数据并进行分析。51例患者中，24例在2007 - 2010年期间就诊，27例在2015 - 2016年期间就诊。发病时的中位年龄为13.7岁；从症状出现到诊断的时间为9个月。19例早期队列患者和24例晚期队列患者开始使用疾病修正疗法。早期患者从诊断到治疗的中位时间为9个月，而晚期患者为3.5个月（P = 0.013）。晚期队列使用了更多种类的治疗方法（早期使用四种药物，晚期使用七种药物以及两项临床试验），生活质量和神经认知监测有所增加（完成PedsQL生活质量量表的比例分别为8%对48%；完成神经认知评估的比例分别为58%对89%）。在两个队列中，患者对疾病修正疗法均有反应（早期队列治疗前平均年化复发率为2.7，治疗后为0.74；晚期队列治疗前为1.7，治疗后为0.37）。总之，多年来，POMS患者接受治疗的时间更早，使用的药物种类更多，监测也更全面。然而，这个完全不受控制的队列无法让我们确定所观察到的改善的关键决定因素。
