JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY, cilt.145, sa.5, ss.1452-1463, 2020 (SCI-Expanded)
Background: Recent findings strongly support hematopoietic stem cell transplantation (HSCT) in patients with severe presentation of LPS-responsive beige-like anchor protein (LRBA) deficiency, but long-term follow-up and survival data beyond previous patient reports or meta-reviews are scarce for those patients who do not receive a transplant.