Home-Based Infusion of Alglucosidase Alfa Can Safely be Implemented in Adults with Late-Onset Pompe Disease: Lessons Learned from 18,380 Infusions.

IF 5.4 2区医学 Q1 IMMUNOLOGY BioDrugs Pub Date : 2023-09-01 DOI:10.1007/s40259-023-00609-2

Imke A M Ditters, Harmke A van Kooten, Nadine A M E van der Beek, Jacqueline F Hardon, Gamida Ismailova, Esther Brusse, Michelle E Kruijshaar, Ans T van der Ploeg, Johanna M P van den Hout, Hidde H Huidekoper

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引用次数: 1

Abstract

Background: Enzyme replacement therapy (ERT) with alglucosidase alfa is the treatment for patients with Pompe disease, a hereditary metabolic myopathy. Home-based ERT is unavailable in many countries because of the boxed warning alglucosidase alfa received due to the risk of infusion-associated reactions (IARs). Since 2008, home infusions have been provided in The Netherlands.

Objectives: This study aimed to provide an overview of our experience with home-based infusions with alglucosidase alfa in adult Pompe patients, focusing on safety, including management of IARs.

Method: We analysed infusion data and IARs from adult patients starting ERT between 1999 and 2018. ERT was initially given in the hospital during the first year. Patients were eligible for home treatment if they were without IARs for multiple consecutive infusions and if a trained home nurse, with on-call back-up by a doctor, was available. The healthcare providers graded IARs.

Results: We analysed data on 18,380 infusions with alglucosidase alfa in 121 adult patients; 4961 infusions (27.0%) were given in hospital and 13,419 (73.0%) were given at home. IARs occurred in 144 (2.9%) hospital infusions and 113 (0.8%) home infusions; 115 (79.9% of 144) IARs in hospital and 104 (92.0% of 113) IARs at home were mild, 25 IARs (17.4%) in hospital and 8 IARs (7.1%) at home were moderate, and very few severe IARs occurred (4 IARs in hospital [2.8%] and 1 IAR at home [0.9%]). Only one IAR in the home situation required immediate clinical evaluation in the hospital.

Conclusion: Given the small numbers of IARs that occurred with the home infusions, of which only one was severe, we conclude that alglucosidase alfa can be administered safely in the home situation, provided the appropriate infrastructure is present.

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家庭输注Alfa糖苷酶可以安全地用于成人迟发性庞贝病:从18380次输注中获得的经验教训

背景:alfa糖苷酶替代疗法(ERT)是一种遗传性代谢性肌病Pompe病患者的治疗方法。由于存在输注相关反应(IARs)的风险，许多国家无法提供家庭ERT。自2008年起，荷兰开始提供家庭注射。目的:本研究旨在概述我们在成人Pompe患者中以家庭为基础输注alfa的经验，重点关注安全性，包括iar的管理。方法:我们分析1999年至2018年开始ERT治疗的成年患者的输注数据和iar。ERT最初是在第一年在医院进行的。如果患者没有连续多次输液的IARs，并且有一名训练有素的家庭护士，并有医生随叫随到的支持，他们就有资格接受家庭治疗。医疗保健提供者对iar进行分级。结果:我们分析了121例成人患者18380例输注alfa糖苷酶的数据;在医院输液4961例(27.0%)，在家输液13419例(73.0%)。医院输液144例(2.9%)，家庭输液113例(0.8%);144例中有115例(79.9%)住院，104例(92.0%)家中有轻度IAR, 25例(17.4%)家中有中度IAR, 8例(7.1%)家中有中度IAR，极少发生重度IAR(医院4例(2.8%)家中1例(0.9%))。只有一个家庭病例需要立即到医院进行临床评估。结论:鉴于家庭输注发生的iar数量很少，其中只有一例是严重的，我们得出结论，只要有适当的基础设施，al葡糖苷酶可以在家庭情况下安全给药。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

BioDrugs 医学-免疫学

CiteScore

12.60

自引率

2.90%

发文量

审稿时长

>12 weeks

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