Almost a third of patients with Pompe disease treated with recombinant alglucosidase alfa (rhGAA) developed anaphylaxis in a study published in the International Archives of Allergy and Immunology. When rapid drug desensitization (RDD) with rhGAA was applied no serious breakthrough reactions occurred.

“Anaphylaxis with rhGAA is not rare,” the study authors concluded. They also said that RDD with rhGAA “is safe and effective in the long term.”

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rhGAA is the only disease-specific treatment in the form of enzyme replacement therapy for the treatment of Pompe disease. However, it can cause anaphylaxis, which can be life threatening.

This is the first study investigating anaphylaxis and its management in the long term in patients with Pompe disease treated with rhGAA.

The team, led by Arzu Bakirtas, MD, from the department of pediatric allergy, Gazi University Faculty of Medicine in Ankara, Turkey, assessed all children diagnosed with Pompe disease and followed up at their institution from January 2009 to September 2021. The researchers analyzed the medical records of the children for the development of anaphylaxis and RDD with rhGAA.

In this time period, 14 patients were diagnosed with Pompe disease, and 13 were treated with rhGAA. The children were followed up for a median of 20 months. Of these 13 patients, 4 (30.8%) developed moderate to severe anaphylaxis and received RDD with rhGAA. 

“A total of 390 RDDs have been performed so far without any serious breakthrough reactions,” the researchers noted. They concluded that the procedure is safe and efficient in the long term. 

RDD is a procedure where the anaphylaxis-causing drug is started at a very low dose and the dose is increased rapidly by doubling the previous dose every 15 to 20 minutes until the desired dose is reached with the aim to establish a temporary tolerance to the drug.

Reference

Ertoy Karagol HI, Inci A, Terece SP, et al. Long-term experience with anaphylaxis and desensitization to alglucosidase alfa in Pompe disease. Int Arch Allergy Immunol. Published online January 9, 2023. doi:10.1159/000528343