Finding “intermediate” ground in transplant and HLH

Abstract

The Bone Marrow Transplant Clinical Trials Network (BMT CTN) study 1204 of stem cell transplantation for hemophagocytic lymphohistiocytosis (HLH) and primary immunodeficiencies (PID) presented in this issue of Blood by Allen et al emphasizes how far treatment and outcomes in these rare diseases have come but also how important multicenter trials are to find a “sweet spot” in the design of curative regimens.1.