Congenital Marrow Failure Syndrome


CBMFS are a unique group of bone marrow failure disorders that present with pancytopenia and bone marrow failure. With regards to HSCT, they are different because

a) They need different conditioning regimens because of their unique sensitivity to drugs
b) Their prognosis with HSCT is different from patients with idiopathic aplastic anemia
c) They are at a higher risk of developing malignancies later unlike aplastic anemia
d) HSCT does not reduce the risk of these malignancies

Hence it is important to study this group separately from aplastic anemia to understand the effect of HSCT on these patients

a) Study the different conditioning regimens, donor source and GVHD prophylaxis in HSCT
b) Study the toxicity and outcome of HSCT in this group
c) Study the long term toxicity and morbidity in long term survivors in this group.




Chairman: Biju George (India)

WG Members

Name Country Institute