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在粘多糖糖症中药物脱敏化后的两年随访

Federico Spataro¹, Roberto Ria², Nada Chaoul³

¹Post Graduate School in Allergology and Internal Medicine "Guido Baccelli", Department of Precision and Regenerative Medicine and Ionian Area-(DiMePRe-J), School of Medicine, Aldo Moro University of Bari, Bari, 70124, Italy. federico.spataro@uniba.it.

Orphanet journal of rare diseases

|December 28, 2024

概括

患有酶替代疗法 (ERT) 过敏反应的粘多糖症 (MPS) 患者可以从联合脱敏方法中受益. 这种方法有时与奥马利祖马布 (omalizumab) 增强,有助于安全地恢复ERT输液.

科学领域:

罕见的遗传疾病罕见的遗传疾病
lysosomal 储存疾病 lysosomal 储存疾病
免疫学和过敏学

背景情况:

粘多糖症 (MPS) 类型1和2是罕见的遗传性疾病,由酶缺乏引起,导致有害的葡萄糖氨基酸积累.
使用laronidase和idursulfase的酶替代疗法 (ERT) 是标准治疗方法,但可以引发过敏反应 (HR).
过敏反应往往需要停止ERT,突出需要有效的管理策略,如脱敏.

研究的目的:

评估对MPS患者的综合脱敏方案,这些患者对ERT有过敏反应.
在为期两年的随访期间评估这种脱敏方法的长期疗效和安全性.
探索辅助疗法来管理反复出现的过敏反应.

主要方法:

采用了一种联合脱敏感化策略,包括静脉输入快速脱敏感化和用重组酶进行皮下免疫治疗.
第一个患有MPS型I的患者接受了拉罗尼达斯,经历了复发的HR,用添加的奥马利祖马布进行管理.
第二个患有MPS类型2的患者接受了idursulfase,通过皮肤检测证实了完全脱敏.

主要成果:

联合脱敏方法成功地在MPS患者中恢复了ERT,至少持续了一年.
该MPS型I患者需要额外的奥马利祖马布 (抗IgE) 治疗,以在初始HR复发后保持ERT耐受性.
这位MPS II型患者在一年的联合脱敏感化后,通过皮肤检测呈阴性结果,获得了对idursulfase ERT的持续耐受性.

结论:

联合脱敏感化策略有效地在MPS患者中建立短期免疫耐受性.
在失去了免疫耐受性的情况下,奥马利祖马布是恢复ERT的可行的治疗选择.
这种方法为患有MPS和过敏反应的患者提供了持续基本ERT的途径.

关键词:

脱敏感化脱敏感化药物过敏药物过敏药物过敏酶替代疗法是一种酶替代疗法.亨特综合征是一种"猎人综合征".溶酶体储存障碍溶酶体储存障碍粘多糖糖症是什么粘多糖症是什么这就是Omalizumab.谢伊综合征 (Scheie综合征) 是一种

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