Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

Barzaghi F., Hernandez L. C. A. , Neven B., Ricci S., Kucuk Z. Y. , Bleesing J. J. , ...Daha Fazla

The Journal of allergy and clinical immunology, cilt.141, sa.3, 2018 (SCI İndekslerine Giren Dergi) identifier identifier identifier

  • Yayın Türü: Makale / Tam Makale
  • Cilt numarası: 141 Konu: 3
  • Basım Tarihi: 2018
  • Doi Numarası: 10.1016/j.jaci.2017.10.041
  • Dergi Adı: The Journal of allergy and clinical immunology


Background: Immunodysregulation polyendocrinopathy enteropathy x-linked(IPEX) syndrome is a monogenic autoimmune disease caused by FOXP3 mutations. Because it is a rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) and immunosuppression (IS), have not been thoroughly examined.