A Dutch survey of people with Pompe disease has assessed the safety of administering enzyme replacement therapy at home, a strategy that has been offered in the Netherlands since 2008. The results were published in May 2023:
- the study analysed data from 116 patients (including 82 with the adult form) receiving a home infusion of alglucosidase alfa (myozyme®), administered by a nurse once every fortnight;
- the data covered a period of one year and were collected every three months by means of questionnaires on side-effects occurring within 48 hours of the infusion;
- a few mild symptoms were reported in 17 participants, mainly fatigue;
- reactions associated with the infusion (anaphylaxis, allergy, etc.) were reported by four participants but did not require emergency treatment.
In a second paper published in June 2023, the same team compared adverse events during hospital and home infusions in 121 people with the adult form of the disease, who had initially received treatment in hospital for at least a year without experiencing side effects during several consecutive infusions:
- reactions to more than 18,000 infusions, a quarter of which were carried out in hospital over a period from 1999 to 2018, were analysed;
- the risk of an adverse reaction was found to be equivalent between the two approaches (2.9% in hospital and 0.8% at home).
Given the low risks observed, the authors believe that this study could serve as a basis for adopting this approach in other countries.
A third study assessed the safety of this approach and satisfaction among 32 patients in 14 Italian care centres:
- home treatment significantly reduced the number of missed injections from an average of 1.3 to 0.7;
- the vast majority of patients (97%) said they preferred home infusion, finding it more convenient, less restrictive and less stressful;
- adverse events were rare and mild.
Voir aussi « Maladie de Pompe : vers une adoption plus large de l’enzymothérapie à domicile ? »
