Role of Allogeneic Hematopoietic Stem Cell Transplant for Chronic Granulomatous Disease (CGD): a Report of the United States Immunodeficiency Network.

摘要: Chronic granulomatous disease (CGD) is a primary immunodeficiency for which allogeneic hematopoietic stem cell transplant (HSCT) offers potential cure. Direct comparison of HSCT to non-HSCT management in the North American population was performed identify clinical factors associated with overall survival (OS) and transplant-related (TRS). Retrospective review CGD subjects enrolled United States Immunodeficiency Network. Survival estimated by Kaplan-Meier method modeled proportional hazards regression. We identified 507 patients (66% CYBB mutants) diagnosed 1953–2016. Fifty underwent HSCT. Median follow-up 9.1 years after diagnosis (0–45.8 years). OS negatively mutation (HR = 6.25; p = 0.034) not (88% v. 85% ± HCT) (HR = 1.26; p = 0.65). Transplant at ≤ 14 years old improved TRS (93% 82% T + 60 months) (HR = − 4.51; p = 0.035). Patients transplanted before 15 years had fewer severe infections pre-HSCT (mean 0.95 2.13; p = 0.047). No mortality reported receiving cells from matched siblings. Infection incidence declined post-HSCT greater than or equal four (p = 0.0010). Compared ≥ 15 years old, post-transplant survivors higher mean performance score (93.2 85.9; p = 0.0039) lower frequency disability (11% 52%; p = 0.014). Allogeneic reduced infection functional performance, but change survival. Transplant-related elevated undergoing old. Consider prior late adolescence severely diminished reactive oxygen intermediate synthesis, particularly if sibling available.