口服克拉德滨治疗多发性硬化症——香港临床经验

Chun-Ho Choi , Jason Ka-Yeung Fong , Siu-Hung Li , Kary Ka-Wai Chan , Alexander Yuk-Lun Lau
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摘要

背景:口服克拉德滨是一种选择性免疫重建疗法,为多发性硬化症(MS)的疾病改善治疗(DMT)提供了一种短期选择。多发性硬化症(MS)是一种慢性神经退行性疾病,以炎症和脱髓鞘为特征,影响全球280万人。尽管在2期和3期试验中证明了其安全性和有效性,但亚洲人群口服氯德里滨治疗的数据仍然有限。本病例系列旨在通过介绍在香港接受治疗的亚洲多发性硬化症患者,为疾病的临床谱提供真实的经验。方法:本回顾性病例系列包括2019年至2021年在香港诊断为复发-缓解型多发性硬化症并接受口服克拉德里滨治疗的9例患者。这些患者在公立和私立医院接受治疗,随访以评估口服克拉德里滨治疗的效果。从患者病历中提取相关数据。结果总体上是积极的,9例患者中有6例在治疗开始2-4年后显示稳定或改善的扩展残疾状态量表(EDSS)评分,7例在治疗开始2-5年内无复发。仅有2例患者出现轻度复发。后续MRI扫描未发现任何患者出现新的病变。1例短暂性淋巴细胞减少被注意到,这并不需要停止治疗。结论我们对这些患者的临床经验与临床试验报告的安全性和有效性结果一致。这些观察结果支持短期治疗方案在减轻患者总体治疗负担和提高治疗依从性方面的有效性。为了证实和建立这些发现,并在更大的队列中探索长期结果,有必要进一步研究和持续监测患者。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Oral cladribine for management of multiple sclerosis–Clinical experience from Hong Kong

Background

Oral cladribine, a selective immune reconstitution therapy, offers a short-course option for disease-modifying therapy (DMT) in multiple sclerosis (MS), a chronic neurodegenerative disease characterized by inflammation and demyelination affecting 2.8 million people worldwide. Despite its proven safety and efficacy in phase 2 and 3 trials, data for oral cladribine treatment in Asian populations remain limited. This case series aims to contribute real-world experience across the clinical spectrum of disease by presenting Asian MS patients treated in Hong Kong.

Methods

This retrospective case series comprises 9 patients diagnosed with relapsing-remitting MS and received oral cladribine treatment in Hong Kong between 2019 and 2021. These patients, treated in both public and private hospitals, were followed up to assess the outcomes of oral cladribine therapy. Relevant data were extracted from patient's medical records.

Results

Outcomes were generally positive, with 6 out of 9 patients showing stable or improved Expanded Disability Status Scale (EDSS) scores 2–4 years after treatment initiation and 7 remaining relapse-free within 2–5 years of treatment initiation. Only 2 patients experienced mild relapses. Follow-up MRI scans revealed no new lesion development in any patient. One case of transient lymphopenia was noted, which did not require discontinuation of treatment.

Conclusions

Our clinical experience with these patients aligned with the safety and efficacy outcomes reported in clinical trials. These observations support the efficacy of a short-course regimen in reducing overall treatment burden for patients and potentially improving adherence to therapy. To substantiate and build upon these findings and explore long-term outcomes in larger cohorts, further research and continued monitoring of patients is necessary.
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