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[一名患有亨特综合征(黏多糖贮积症II型)儿童的艾度硫酸酯酶脱敏治疗]

[Idursulfase desensitization in a child with Hunter syndrome (mucopolysaccharidosis II)].

作者信息

Bustamante Lucrecia L, Garavaglia Luciano, Garramone Esteban I, Amartino Hernán, Parisi Claudio As

机构信息

Sección Alergia Adultos, Servicio de Clínica Médica, Consultorio de Alergia a Fármacos, Hospital Italiano de Buenos Aires.

Clínica de Mucopolisacaridosis y Enfermedades Neurometabólicas, Servicio de Neurología Infantil, Hospital Universitario Austral.

出版信息

Arch Argent Pediatr. 2021 Feb;119(1):e41-e44. doi: 10.5546/aap.2021.e41.

Abstract

Enzyme replacement therapy with idursulfase decreases morbidity and improves quality of life of patients with mucopolysaccharidosis ii. Immediate hypersensitivity reactions to this drug have been described. Desensitization is a treatment that induces temporary tolerance to a culprit drug, allowing the allergic patient to receive the medication. We present the case of a 7-year-old patient diagnosed with Hunter syndrome who presented, after 4 years of treatment, two episodes of anaphylaxis during the infusion of idursulfase. Detection of specific immunoglobulin E was carried out using skin tests, with intradermal reaction at a 1/10 dilution (0.2 mg/ml) being positive. A 12-step desensitization protocol was performed without presenting adverse events. The allergological evaluation and the possibility of desensitization were useful tools in the management of our patient.

摘要

用艾度硫酸酯酶进行酶替代疗法可降低黏多糖贮积症II型患者的发病率并改善其生活质量。已报道过对该药物的速发型超敏反应。脱敏是一种诱导对致病药物产生暂时耐受性的治疗方法,使过敏患者能够接受该药物治疗。我们报告一例7岁被诊断为亨特综合征的患者,在接受4年治疗后,在输注艾度硫酸酯酶期间出现了两次过敏反应。通过皮肤试验进行特异性免疫球蛋白E检测,皮内反应在1/10稀释度(0.2mg/ml)时呈阳性。实施了一个12步脱敏方案,未出现不良事件。过敏学评估和脱敏可能性是管理我们这位患者的有用工具。

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