Abstract
Allogeneic hematopoietic stem cell transplantation (HSCT) is the only treatment option to cure chronic granulomatous disease (CGD), although the selection of an alternative donor for patients with mismatched sibling donors and the role of conditioning regimens in patients with severe complications remain controversial. Here we report the clinical course of four patients with CGD who underwent HSCT in our hospital. Patient 1 underwent transplantation from an HLA-locus mismatched related donor, with a myeloablative conditioning regimen including total body irradiation (TBI). Engraftment was rapid, but he developed a second malignancy 5 years after HSCT and succumbed. Patients 2 and 3 underwent transplantation from HLA-matched sibling donors (MSD), with reduced-intensity conditioning (RIC) including low-dose TBI; engraftment was rapid. Patient 4 had severe complications and underwent transplantation from an alternative donor, with RIC including low-dose TBI. However, he died of sepsis before achieving engraftment. If matched sibling donors are available, RIC including low-dose TBI can provide stable engraftment. However, an optimal conditioning regimen for patients without MSD or those with severe complications remains unclear. Further studies are necessary to clarify these issues.
