Shih-Hsiang Chen, Taiwan
Chang Gung Memorial Hospital Pediatric Hematology/OncologyPresenter of 1 Presentation
SUCCESSFUL OUTCOME FOLLOWING ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION IN TAIWANESE CHILDREN WITH PRIMARY IMMUNODEFICIENCY
Abstract
Background and Aims
Children with primary immunodeficiencies (PIDs) constitute life-threatening medical emergencies. Allogeneic hematopoietic stem cell transplantation (Allo-HSCT) has been shown to be curative for children with PIDs. We analyzed the outcome of allo-HSCT in Taiwanese children with PIDs at a single pediatric transplantation center using both related and unrelated donors.
Methods
Eighteen children with the diagnosis of PIDs treated at Chang Gung Memorial Hospital between 2004 and 2018 were identified and analyzed. The medical records including the diagnosis of PIDs, transplant protocol, engraftment, adverse events, and treatment outcome retrospectively reviewed.
Results
There were 8 patients with severe combined immunodeficiency, 6 with chronic granulomatous disease, 3 with Wiskott-Aldrich syndrome, and 1 with congenital neutropenia. The median age of transplant was 8 months (range: 2 – 143 months). Conditioning regimens were myeloablative conditioning in 13 (72.2%) patients and reduced-intensity conditioning in 3 (16.7%). The stem cell sources were unrelated cord blood in 15 (83.3%), and peripheral blood progenitor cells in 3 (16.7%). Three patients required a second HSCT (cord blood transplantation) owing to graft failure or rejection. Grade III acute graft-versus-host disease occurred in 2 patients. Three patients had limited chronic GVHD involving the skin. The overall survival at 5 years for the entire cohort was 76.4%. In all surviving patients, either stable mixed chimerism or full donor chimerism were observed.
Conclusions
Allo-HSCT is safe and effective in Taiwanese children with PID. Unrelated cord blood can serve as an ideal donor source for children with PID if a matched sibling donor is not available.