Chronic granulomatous disease - conventional treatment vs. hematopoietic stem cell transplantation: an update

Purpose of review We update and summarize the recent findings in conventional treatment and hematopoietic stem cell transplantation in chronic granulomatous disease (CGD). We also summarize the contemporary view on when hematopoietic stem cell transplantation should be the preferred treatment of choice in CGD. Recent findings Azole antifungal treatment in CGD has improved survival. With prolonged survival, inflammatory complications are an emerging problem in CGD. Several studies now present excellent results with stem cell transplantation in severe CGD, also with reduced intensity conditioning. Summary Several lines of evidence now suggest that stem cell transplantation should be the preferred treatment of choice in severe CGD, if there is an available donor. This should be performed as soon as possible to avoid severe sequelae from infection and inflammation.