Background

Intravenous recombinant enzyme replacement therapy (ERT) is currently available for 8 lysosomal diseases. Hypersensitivity reactions (HSRs) may be observed during this long-term treatment.

Objective

To evaluate the frequency and clinical treatment features of ERT HSRs and the management of desensitizations in children.

Methods

Medical records were reviewed retrospectively for patients who received ERT. Those who had experienced HSRs to ERT were included in the study. The demographic characteristics of the patients, culprit enzyme, signs and symptoms, diagnostic tests, management of the reaction, and the protocol employed for the maintenance of ERT were recorded.

Results

During the study period, 54 patients received ERT in our institution. A total of 11 patients (20.4%) experienced HSR to ERT. All reactions were of immediate type. The most common symptoms were cutaneous manifestations. A total of 9 patients experienced urticaria, and 2 had anaphylaxis as initial reaction. Patients who had isolated cutaneous symptoms continued their treatments with antihistamines, corticosteroid premedication, slower infusion rate or both. Patients who had recurrent urticaria with these modalities or those who had anaphylaxis continued their ERT with desensitization (n = 8). A total of 3 patients required revisions in desensitization protocols because of recurrent anaphylaxis.

Conclusion

The reactions that develop during this long-term treatment may be treated by premedication-prolonged infusion, but in some patients, desensitization protocols are necessary for the continuation of therapy. Revisions in desensitization protocols may be required.

中文翻译：

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溶酶体贮积病患儿对酶替代疗法的超敏反应的管理。

背景

静脉重组酶替代疗法（ERT）目前可用于8种溶酶体疾病。在此长期治疗过程中可能会出现超敏反应（HSR）。

目的

评估ERT HSR的频率和临床治疗特征以及儿童脱敏的管理。

方法

对接受ERT的患者的病历进行回顾性审查。那些对ERT有过高铁经历的人也包括在研究中。记录患者的人口统计学特征，罪魁祸首，症状和体征，诊断测试，反应管理以及用于维持ERT的方案。

结果

在研究期间，我们机构有54位患者接受了ERT。共有11名患者（20.4％）经历了ERT的HSR。所有反应均为即时反应。最常见的症状是皮肤表现。共有9例患者出现荨麻疹，其中2例作为初始反应出现过敏反应。皮肤症状较轻的患者继续使用抗组胺药，糖皮质激素类药物，输注速度较慢或两者同时进行治疗。患有这些形式的荨麻疹复发或过敏性反应的患者继续接受脱敏治疗（n = 8）。由于反复过敏反应，共有3例患者需要修改脱敏方案。

结论

在长期治疗期间发生的反应可以通过服药前长时间输注来治疗，但在某些患者中，脱敏方案对于继续治疗是必需的。可能需要修改脱敏协议。