Management of hypersensitivity reactions to enzyme replacement therapy in children with lysosomal storage diseases.


Journal

Annals of allergy, asthma & immunology : official publication of the American College of Allergy, Asthma, & Immunology
ISSN: 1534-4436
Titre abrégé: Ann Allergy Asthma Immunol
Pays: United States
ID NLM: 9503580

Informations de publication

Date de publication:
10 2020
Historique:
received: 30 04 2020
revised: 16 06 2020
accepted: 13 07 2020
pubmed: 21 7 2020
medline: 20 11 2020
entrez: 21 7 2020
Statut: ppublish

Résumé

Intravenous recombinant enzyme replacement therapy (ERT) is currently available for 8 lysosomal diseases. Hypersensitivity reactions (HSRs) may be observed during this long-term treatment. To evaluate the frequency and clinical treatment features of ERT HSRs and the management of desensitizations in children. Medical records were reviewed retrospectively for patients who received ERT. Those who had experienced HSRs to ERT were included in the study. The demographic characteristics of the patients, culprit enzyme, signs and symptoms, diagnostic tests, management of the reaction, and the protocol employed for the maintenance of ERT were recorded. During the study period, 54 patients received ERT in our institution. A total of 11 patients (20.4%) experienced HSR to ERT. All reactions were of immediate type. The most common symptoms were cutaneous manifestations. A total of 9 patients experienced urticaria, and 2 had anaphylaxis as initial reaction. Patients who had isolated cutaneous symptoms continued their treatments with antihistamines, corticosteroid premedication, slower infusion rate or both. Patients who had recurrent urticaria with these modalities or those who had anaphylaxis continued their ERT with desensitization (n = 8). A total of 3 patients required revisions in desensitization protocols because of recurrent anaphylaxis. The reactions that develop during this long-term treatment may be treated by premedication-prolonged infusion, but in some patients, desensitization protocols are necessary for the continuation of therapy. Revisions in desensitization protocols may be required.

Sections du résumé

BACKGROUND
Intravenous recombinant enzyme replacement therapy (ERT) is currently available for 8 lysosomal diseases. Hypersensitivity reactions (HSRs) may be observed during this long-term treatment.
OBJECTIVE
To evaluate the frequency and clinical treatment features of ERT HSRs and the management of desensitizations in children.
METHODS
Medical records were reviewed retrospectively for patients who received ERT. Those who had experienced HSRs to ERT were included in the study. The demographic characteristics of the patients, culprit enzyme, signs and symptoms, diagnostic tests, management of the reaction, and the protocol employed for the maintenance of ERT were recorded.
RESULTS
During the study period, 54 patients received ERT in our institution. A total of 11 patients (20.4%) experienced HSR to ERT. All reactions were of immediate type. The most common symptoms were cutaneous manifestations. A total of 9 patients experienced urticaria, and 2 had anaphylaxis as initial reaction. Patients who had isolated cutaneous symptoms continued their treatments with antihistamines, corticosteroid premedication, slower infusion rate or both. Patients who had recurrent urticaria with these modalities or those who had anaphylaxis continued their ERT with desensitization (n = 8). A total of 3 patients required revisions in desensitization protocols because of recurrent anaphylaxis.
CONCLUSION
The reactions that develop during this long-term treatment may be treated by premedication-prolonged infusion, but in some patients, desensitization protocols are necessary for the continuation of therapy. Revisions in desensitization protocols may be required.

Identifiants

pubmed: 32687987
pii: S1081-1206(20)30487-7
doi: 10.1016/j.anai.2020.07.010
pii:
doi:

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

460-467

Informations de copyright

Copyright © 2020 American College of Allergy, Asthma & Immunology. Published by Elsevier Inc. All rights reserved.

Auteurs

Irem Turgay Yagmur (I)

Department of Pediatric Allergy and Immunology, Ankara City Hospital, Ankara, Turkey. Electronic address: iremtrgy@gmail.com.

Ozlem Unal Uzun (O)

Department of Pediatric Metabolic Disorders, Ankara City Hospital, Ankara, Turkey.

Aynur Kucukcongar Yavas (A)

Department of Pediatric Metabolic Disorders, Ankara City Hospital, Ankara, Turkey.

Ilknur Kulhas Celik (I)

Department of Pediatric Allergy and Immunology, Ankara City Hospital, Ankara, Turkey.

Muge Toyran (M)

Department of Pediatric Allergy and Immunology, Ankara City Hospital, University of Health Sciences, Ankara, Turkey.

Mehmet Gunduz (M)

Department of Pediatric Metabolic Disorders, Ankara City Hospital, University of Health Sciences, Ankara, Turkey.

Ersoy Civelek (E)

Department of Pediatric Allergy and Immunology, Ankara City Hospital, University of Health Sciences, Ankara, Turkey.

Emine Dibek Misirlioglu (E)

Department of Pediatric Allergy and Immunology, Ankara City Hospital, University of Health Sciences, Ankara, Turkey.

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Classifications MeSH