Remission

In most national registries, approximately 40% of patients with DBA are transfusion-dependent, having failed to respond or having become refractory to steroids, while 40% are steroid-dependent, and 20% are transfusion-independent on no medication (‘‘in remission’’) (Willig et al, 1999a; Lipton et al, 2006). Remission occurs in some patients who are initially steroid responsive when steroids can be stopped completely with continued maintenance of adequate hemoglobin levels. A small number of steroid non-responders may also enter remission even after prolonged transfusion dependence. The DBAR defines remission as a stable, physiologically acceptable hemoglobin, maintained for at least six months independent of corticosteroids, transfusions or other therapy. Seventy percent of the remitters in the DBAR did so within the first decade of life. Most patients have a sustained remission, but some can go in and out of remission. Hormonal stress in pregnancy appears to be an important factor contributing to ‘‘relapse’’; this may be transient. Remission in DBA patients is not uncommon, but it is still unclear what “triggers” a remission or causes someone to relapse.