The DBAR was established in 1993. There are 268 registered Diamond Blackfan Anemia (DBA) patients (pts) in the U.S. and Canada; 5 have been excluded due to misdiagnoses. Of these 263 DBA pts, 203 are alive, 11 are dead, and information has not been obtained from 49 pts; the male:female ratio is 1:0.93. The current median age is 11yr 1mo (range, 1mo to 45yr 5mo). The median ages at presentation and diagnosis respectively, were 8 weeks (range, birth to 8yr 4mo) and 12 weeks (range, birth to 24yr). Ninety-one percent of the pts presented during the first year of life. Multiple cases were found in 16 of 199 families (8.0%, 12 presumed dominant inheritance and 4 recessive). Physical abnormalities, excluding short stature, were found in 48.1%; 22.6% had more than one anomaly. Of these anomalies 41% were of the face and neck, 36% upper limb and hand, 33% genitourinary, and 27% cardiac. Corticosteroids(CS) and red cell transfusions have been the mainstays of treatment. Of 203 pts, 81.3% were initially responsive to CS while 16.7% were CS non-responsive. Currently 40.5% are on daily or alternate day CS therapy. Significant CS related side effects are reported in the majority of pts (e.g. pathologic fractures 17.7%, cataracts 9.2%). Thirty-nine percent are receiving red cell transfusions: 30.8% were never CS responsive, 19.2% became refractory, and 44.9% could not be weaned to an acceptable CS dose. Thirty-two of 165 responders entered spontaneous remission (SR); 29 remain in SR. Only 2 of 34 of CS non-responders ever entered SR (p=.05). The median age of onset of first SR was 4yr 3mo (range, 7mo to 37yr 11mo). The median duration of SR was 5yr 6mo (range, 8mo to 36yr 8mo). There are 11 deaths reported in the DBAR; 3 due to iron overload, 2 of PCP, 1 from severe aplastic anemia and 5 from transplant related complications. Of 12 pts with stem cell transplants, all 4 matched related transplants are alive, only 2 of 6 unrelated and 0 of 1 mismatched related transplants are living; one is too early to evaluate. These data suggest the need for a reevaluation of current practices related to CS treatment, perhaps in favor of a more liberal use of red cell transfusion therapy. The role of alternative donor stem cell transplantation in DBA must be carefully considered, especially in pts with a higher likelihood of SR.