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Open Access 25-04-2024 | Methotrexate | Review Article

Advances in Immune Tolerance Induction in Enzyme Replacement Therapy

Authors: Aslı İnci, Fatih Süheyl Ezgü, Leyla Tümer

Published in: Pediatric Drugs | Issue 3/2024

Abstract

Inborn errors of metabolism (IEMs) are a group of genetic diseases that occur due to the either deficiency of an enzyme involved in a metabolic/biochemical pathway or other disturbances in the metabolic pathway including transport protein or activator protein deficiencies, cofactor deficiencies, organelle biogenesis, maturation or trafficking problems. These disorders are collectively significant due to their substantial impact on both the well-being and survival of affected individuals. In the quest for effective treatments, enzyme replacement therapy (ERT) has emerged as a viable strategy for patients with many of the lysosomal storage disorders (LSD) and enzyme substitution therapy in the rare form of the other inborn errors of metabolism including phenylketonuria and hypophosphatasia. However, a major challenge associated with enzyme infusion in patients with these disorders, mainly LSD, is the development of high antibody titres. Strategies focusing on immunomodulation have shown promise in inducing immune tolerance to ERT, leading to improved overall survival rates. The implementation of immunomodulation concurrent with ERT administration has also resulted in a decreased occurrence of IgG antibody development compared with cases treated solely with ERT. By incorporating the knowledge gained from current approaches and analysing the outcomes of immune tolerance induction (ITI) modalities from clinical and preclinical trials have demonstrated significant improvement in the efficacy of ERT. In this comprehensive review, the progress in ITI modalities is assessed, drawing insights from both clinical and preclinical trials. The focus is on evaluating the advancements in ITI within the context of IEM, specifically addressing LSDs managed through ERT.

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Title: Advances in Immune Tolerance Induction in Enzyme Replacement Therapy
Authors: Aslı İnci
Fatih Süheyl Ezgü
Leyla Tümer
Publication date: 25-04-2024
Publisher: Springer International Publishing
Keywords: Methotrexate
Methotrexate
Rituximab
Rituximab
Published in: Pediatric Drugs / Issue 3/2024
Print ISSN: 1174-5878
Electronic ISSN: 1179-2019
DOI: https://doi.org/10.1007/s40272-024-00627-9

Keynote webinar | Spotlight on medication adherence

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Advances in Immune Tolerance Induction in Enzyme Replacement Therapy

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Keynote webinar | Spotlight on medication adherence

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Other articles of this Issue 3/2024

Improving Methylphenidate Titration in Children with Attention-Deficit/Hyperactivity Disorder (ADHD): A Randomized Controlled Trial Using Placebo-Controlled Titration Implemented in Clinical Practice

Treatment Options for Alopecia Areata in Children and Adolescents

Perforated appendicitis: room for improvement?

Real-World Data on the Use of Sirolimus in Asian Children with Vascular Malformations

The Effect of Polymorphisms and Other Biomarkers on Infliximab Exposure in Paediatric Inflammatory Bowel Disease: Development of a Population Pharmacokinetic Model

Neonatal Necrotizing Enterocolitis: An Update on Pathophysiology, Treatment, and Prevention