Long-Term Experience with Anaphylaxis and Desensitization to Alglucosidase Alfa in Pompe Disease


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Karagol H. I. E., İnci A., Terece S. P., Kılıç A., Demir F., Yapar D., ...More

International Archives of Allergy and Immunology, vol.184, no.4, pp.370-375, 2023 (SCI-Expanded) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 184 Issue: 4
  • Publication Date: 2023
  • Doi Number: 10.1159/000528343
  • Journal Name: International Archives of Allergy and Immunology
  • Journal Indexes: Science Citation Index Expanded (SCI-EXPANDED), Scopus, Academic Search Premier, BIOSIS, CAB Abstracts, EMBASE, Food Science & Technology Abstracts, MEDLINE, Veterinary Science Database
  • Page Numbers: pp.370-375
  • Keywords: Anaphylaxis, Pompe disease, Rapid drug desensitization, Recombinant alglucosidase alfa
  • Gazi University Affiliated: Yes

Abstract

Background: and Objective: Pompe disease (PD) is an inherited lysosomal storage disease that progresses with glycogen accumulation in many tissues, due to the deficiency of the acid-alpha glucosidase enzyme. Recombinant alglucosidase alfa (rhGAA) is the only disease-specific treatment option, in the form of enzyme replacement therapy (ERT). Anaphylaxis can develop with rhGAA. There is no study evaluating anaphylaxis and its management in PD in the long term. We aimed to evaluate the development of anaphylaxis and rapid drug desensitization (RDD) with rhGAA in children with PD. Materials and Methods: All children diagnosed and followed up in our institution with PD over 12 years between January 2009 and September 2021 were evaluated for development of anaphylaxis and RDD with rhGAA from medical records. Results: Fourteen patients, 64% of whom were female and diagnosed with PD (1 juvenile, 13 infantile types) during the study period included in the study. The median age at diagnosis was 3.2 months (1-40 months). The median follow-up time of the patients was 20 months (1-129 months). Thirteen patients were given rh- GAA, one died before ERT. Four (30.8%) patients developed moderate to severe anaphylaxis, and RDD was applied with rhGAA. A total of 390 RDDs have been performed so far without any serious breakthrough reactions during all RDDs. Conclusions: Anaphylaxis with rhGAA is not rare and RDD with rhGAA is safe and effective in the long term.