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<p>Chimeric antigen receptor (CAR) T-cell therapy targeting B cells has shown promising results, including drug-free remission, in adult-onset autoimmune diseases. Extending this therapeutic approach to the pediatric population, particularly for juvenile autoimmune diseases, presents an exciting opportunity. However, challenges specific to juvenile-onset autoimmune conditions, such as long-term adverse events, heightened disease activity, and the imperative to reduce steroid exposure, must be considered. While this strategy appears viable for these severe conditions, the limited data available for this population and the absence of evidence on cases with a high genetic component, such as monogenic lupus, represent significant challenges. Most monogenic lupus cases are associated with innate immune defects, and the involvement of B cells in these genetic anomalies remains poorly understood. In this review, we examine the potential indications, current knowledge, and limitations of CAR-T cell therapy in juvenile-onset autoimmune diseases, extending the discussion beyond early-onset lupus.</p>

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CAR-T cell therapy for juvenile-onset autoimmune diseases: a promising future?

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年5月10日

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  • Maurine Jouret,
  • Sebastien Viel,
  • Benjamin Fournier,
  • Sarah Benezech,
  • Jérome Avouac,
  • Marc Scherlinger,
  • Alexandre Belot,
  • Gaetane Nocturne,
  • David Launay,
  • Benjamin Terrier,
  • Camille Bigenwald,
  • Christophe Richez,
  • Noémie Jourde-Chiche,
  • Sylvain Choquet,
  • Divi Cornec,
  • Jean Sibilia,
  • Thierry Martin,
  • Samuel Bitoun,
  • Zahir Amoura,
  • Olivier Hermine,
  • Xavier Mariette,
  • Emmanuel Donnadieu,
  • Philippe Bousso,
  • Grégory Pugnet,
  • Justine Decroocq,
  • Edouard Forcade,
  • Alain Meyer,
  • Christian Jorgensen,
  • Laurent Peyrin-Biroulet

摘要

Chimeric antigen receptor (CAR) T-cell therapy targeting B cells has shown promising results, including drug-free remission, in adult-onset autoimmune diseases. Extending this therapeutic approach to the pediatric population, particularly for juvenile autoimmune diseases, presents an exciting opportunity. However, challenges specific to juvenile-onset autoimmune conditions, such as long-term adverse events, heightened disease activity, and the imperative to reduce steroid exposure, must be considered. While this strategy appears viable for these severe conditions, the limited data available for this population and the absence of evidence on cases with a high genetic component, such as monogenic lupus, represent significant challenges. Most monogenic lupus cases are associated with innate immune defects, and the involvement of B cells in these genetic anomalies remains poorly understood. In this review, we examine the potential indications, current knowledge, and limitations of CAR-T cell therapy in juvenile-onset autoimmune diseases, extending the discussion beyond early-onset lupus.