Abatacept as a Long-Term Targeted Therapy for LRBA Deficiency.

ABSTRACT:

Background

LPS-responsive beige-like anchor (LRBA) deficiency presents with susceptibility to infections, autoimmunity, and lymphoproliferation. The long-term efficacy of cytotoxic T-lymphocyte-associated antigen 4-immunoglobulin (abatacept) as targeted therapy for its immune dysregulatory features remains to be established.

Objective

To determine the clinical and immunologic features of LRBA deficiency and long-term efficacy of abatacept treatment in controlling the different disease manifestations.

Methods

Twenty-two LRBA-deficient patients were recruited from different immunology centers and followed prospectively. Eighteen patients on abatacept were evaluated every 3 months for long-term clinical and immunologic responses. LRBA expression, lymphocyte subpopulation

SUBMITTER: Kiykim A 

PROVIDER: S-EPMC6842687 | biostudies-literature | 2019 Nov - Dec

REPOSITORIES: biostudies-literature