Home infusion therapy for Pompe disease
Background: Pompe disease is a lysosomal multisystem disorder with predominant proximal myopathy. Treatment with enzyme replacement therapy (ERT) is available requiring life-long biweekly infusions of recombinant α-glucosidase. To minimize the burden of ERT patients ask for home infusion therapy.
Aims and methods: Pompe disease experts from Germany, Austria, and Switzerland discussed in two consensus meetings in 2019 and 2020 requirements for home infusion therapy, adequate execution of treatment, and the legal situation for delegating physicians.
Results and discussion: Home infusion therapy is principally feasible for patients with Pompe disease if certain preconditions are fulfilled, but the decision to implement has to be made on an individual basis. The treating physician delegates the execution of ERT ad personam to nursing staff but retains full legal responsibility. Home infusion therapy has to be carried out by specially trained and qualified staff. Infusion-related risks comprise mainly allergic reactions, and adequate medical treatment must be warranted. In German-speaking countries, clear rules for conducting home infusion therapy are needed to reduce psychosocial stress for patients with Pompe disease, and providing legal certainty for delegating physicians.
Hahn A, Lampe C, Boentert M, Hundsberger T, Löscher W, Wenninger S, Ziegler A, Lagler F, Ballhausen D, Schlegel T, Schoser B. Heiminfusionstherapie bei Morbus Pompe: Konsensusempfehlungen für den deutschsprachigen Raum . Fortschr Neurol Psychiatr. 2021 Apr 27. German. doi: 10.1055/a-1482-6041. Epub ahead of print. Erratum for: Fortschr Neurol Psychiatr. 2021 Feb 09;: PMID: 33906241.