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<p>Lysosomal Storage Diseases (LSDs) encompass a range of genetic disorders characterized by enzyme deficiencies that lead to substrate accumulation and progressive tissue damage. Enzyme Replacement Therapy (ERT) is the primary treatment for LSDs, yet it is often associated with hypersensitivity reactions (HSRs), ranging from mild rashes to severe anaphylaxis. These reactions, frequently driven by anti-drug antibodies, pose significant challenges in treatment adherence and patient outcomes. This paper outlines a stepwise approach to managing HSRs during ERT, focusing on three escalating strategies. The first-line approach involves premedication using antihistamines, corticosteroids, antileukotrienes, and bronchodilators to prevent or reduce the severity of HSRs.</p><p>For patients who continue to experience HSRs despite premedication, desensitization protocols are recommended, involving the gradual reintroduction of ERT in controlled, increasing doses.</p><p>In cases of refractory HSRs, omalizumab, a monoclonal antibody targeting IgE, has been successfully used as a third-line intervention.</p><p>This comprehensive, stepwise strategy aims to provide clinicians a guide to manage these challenges, offering practical steps for optimizing treatment while ensuring patient safety. Future research is needed to further validate these management techniques and explore new therapeutic options for optimizing care in this rare but critical patient population.</p>

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How to manage hypersensitivity reactions to enzyme replacement therapy in lysosomal storage diseases?

  • 期刊论文

  • 出版模式：

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  • 发表日期：

    2025年6月6日

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  • Federico Spataro,
  • Antonio Giovanni Solimando,
  • Vanessa Desantis,
  • Angelo Vacca,
  • Attilio Di Girolamo,
  • Roberto Ria

摘要

Lysosomal Storage Diseases (LSDs) encompass a range of genetic disorders characterized by enzyme deficiencies that lead to substrate accumulation and progressive tissue damage. Enzyme Replacement Therapy (ERT) is the primary treatment for LSDs, yet it is often associated with hypersensitivity reactions (HSRs), ranging from mild rashes to severe anaphylaxis. These reactions, frequently driven by anti-drug antibodies, pose significant challenges in treatment adherence and patient outcomes. This paper outlines a stepwise approach to managing HSRs during ERT, focusing on three escalating strategies. The first-line approach involves premedication using antihistamines, corticosteroids, antileukotrienes, and bronchodilators to prevent or reduce the severity of HSRs.

For patients who continue to experience HSRs despite premedication, desensitization protocols are recommended, involving the gradual reintroduction of ERT in controlled, increasing doses.

In cases of refractory HSRs, omalizumab, a monoclonal antibody targeting IgE, has been successfully used as a third-line intervention.

This comprehensive, stepwise strategy aims to provide clinicians a guide to manage these challenges, offering practical steps for optimizing treatment while ensuring patient safety. Future research is needed to further validate these management techniques and explore new therapeutic options for optimizing care in this rare but critical patient population.