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. 2023 Sep;37(5):685-698.
doi: 10.1007/s40259-023-00609-2. Epub 2023 Jun 16.

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

Imke A M Ditters  1 , Harmke A van Kooten  2 , Nadine A M E van der Beek  2 , Jacqueline F Hardon  1 , Gamida Ismailova  2 , Esther Brusse  2 , Michelle E Kruijshaar  1 , Ans T van der Ploeg  3 , Johanna M P van den Hout #  1 , Hidde H Huidekoper #  1
Affiliations

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

Imke A M Ditters et al. BioDrugs. 2023 Sep.

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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Conflict of interest statement

Imke A. M. Ditters, Harmke A. van Kooten, Jacqueline F. Hardon, Gamida Ismailova, Esther Brusse, Michelle E. Kruijshaar and Hidde H. Huidekoper declare no conflicts of interest. Ans T. van der Ploeg received funding for research, clinical trials, and advisory fees from Sanofi, Amicus Therapeutics, Spark Therapeutics, Denali Therapeutics and Takeda working on ERT or next­generation therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under agreements with Erasmus MC University Medical Center and the relevant industry. Nadine A. M. E. van der Beek received funding for research, clinical trials, and advisory fees from Sanofi working on ERT or next­generation therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under an agreement between this company and Erasmus MC University Medical Center and the relevant industry. She also received a Veni grant from ZonMW (project no. 09150161910230). Johanna M. P. van den Hout received funding for research, clinical trials, and advisory fees from Sanofi, Denali Therapeutics and Takeda working on ERT or next­generation therapies in the field of Pompe disease, other lysosomal storage diseases or neuromuscular disorders, under agreements with Erasmus MC University Medical Center and the relevant industry.

Figures

Fig. 1
Fig. 1
Flow chart depicting when to start with enzyme replacement therapy in the home situation. IAR infusion-associated reaction
Fig. 2
Fig. 2
Protocol regarding IARs and subsequent infusions. a The course of action after an IAR in the acute situation. b Course of action the next infusion after a (possible) IAR has occurred. IAR infusion-associated reaction, ALS advanced life support, CTCAE Common Terminology Criteria for Adverse Events, ERT enzyme replacement therapy
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