Therapeutic options for CTLA-4 insufficiency.

Egg D., Rump I. C. , Mitsuiki N., Rojas-Restrepo J., Maccari M., Schwab C., ...More

The Journal of allergy and clinical immunology, vol.149, pp.736-746, 2022 (Peer-Reviewed Journal) identifier identifier identifier

  • Publication Type: Article / Article
  • Volume: 149
  • Publication Date: 2022
  • Doi Number: 10.1016/j.jaci.2021.04.039
  • Journal Name: The Journal of allergy and clinical immunology
  • Journal Indexes: Science Citation Index Expanded, Scopus, PASCAL, BIOSIS, CAB Abstracts, EMBASE, Food Science & Technology Abstracts, International Pharmaceutical Abstracts, MEDLINE, Veterinary Science Database
  • Page Numbers: pp.736-746
  • Keywords: CTLA-4, common variable immunodeficiency, primary immunodeficiency, diagnosis, treatment, abatacept, sirolimus, LRBA, rituximab, HSCT, IMMUNE DYSREGULATION, MANAGEMENT, RITUXIMAB, DEFICIENCY, MUTATIONS, DISEASE


Background: Heterozygous germline mutations in cytotoxic T lymphocyte-associated antigen-4 (CTLA4) impair the immunomodulatory function of regulatory T cells. Affected individuals are prone to life-threatening autoimmune and lymphoproliferative complications. A number of therapeutic options are currently being used with variable effectiveness.