Complete recovery of hemopoiesis following bone marrow transplant in a
patient with unresponsive congenital hypoplastic anemia (Blackfan- Diamond
syndrome)
A Iriondo, J Garijo, J Baro, E Conde, JM Pastor, A Sabanes, V Hermosa, MC Sainz, L Perez de la Lastra and A Zubizarreta
Allogeneic bone marrow transplantation (BMT) was carried out on a 5-
year-old boy with congenital hypoplastic anemia (CHA), who did not respond
to corticosteroids and who was displaying signs of progressive
hemosiderosis. Pretransplant preparation had to be modified because
respiratory failure and cerebral edema supervened. This preparatory regimen
consisted of busulfan (2 mg/kg for four days), cyclophosphamide (50 mg/kg
for one day), and total body irradiation (750 rad). Hemopoiesis was
completely restored and is still maintained 650 days after transplantation.
This is the second published report on the use of BMT to treat a patient
with CHA, and it is the first time it has resulted in long-term survival.
BMT should be considered for patients with CHA who do not respond to
corticosteroids.
Volume 64,
Issue 2,
pp. 348-351,
08/01/1984
Copyright © 1984 by The American Society of Hematology
