Treatment of Severe Forms of LPS-Responsive Beige-like Anchor Protein (LRBA) Deficiency by Allogeneic Hematopoietic Stem Cell Transplantation

Publication date: Available online 10 May 2017
Source:Journal of Allergy and Clinical Immunology
Author(s): Markus G. Seidel, Katrin Böhm, Figen Dogu, Austen Worth, Adrian Thrasher, Benoit Florkin, Aydan İkincioğulları, Anke Peters, Shahrzad Bakhtiar, Marie Meeths, Polina Stepensky, Isabelle Meyts, Svetlana O. Sharapova, Laura Gámez-Díaz, Lennart Hammarström, Stephan Ehl, Bodo Grimbacher, Andrew R. Gennery

Teaser

Capsule summary: LRBA deficiency, a life-threatening immunodeficiency/autoimmunity disorder, was treated by HSCT in 12 patients, four of whom died from transplant-related causes. Six patients were cured without further need of immunosuppression, and two are in partial remission.


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