Long-term outcomes of 176 patients with X-linked hyper-IgM syndrome treated with or without hematopoietic cell transplantation

MT de la Morena, D Leonard, TR Torgerson… - Journal of allergy and …, 2017 - Elsevier
Background X-linked hyper-IgM syndrome (XHIGM) is a primary immunodeficiency with high
morbidity and mortality compared with those seen in healthy subjects. Hematopoietic cell
transplantation (HCT) has been considered a curative therapy, but the procedure has
inherent complications and might not be available for all patients. Objectives We sought to
collect data on the clinical presentation, treatment, and follow-up of a large sample of
patients with XHIGM to (1) compare long-term overall survival and general well-being of …