Establishment of erythropoiesis following bone marrow transplantation in a
patient with congenital hypoplastic anemia (Diamond-Blackfan syndrome)
CS August, E King, JH Githens, K McIntosh, JR Humbert, A Greensheer and RB Johnson
Marrow transplantation was attempted in a 13-yr-old boy with congenital
hypoplastic anemia who had never responded to corticosteroid therapy. Prior
to the transplant, he had received 238 transfusions, at least 12 of which
were from his father. He was prepared for grafting with antilymphocyte
globulin, procarbazine, and total body irradiation (1000 rads). The
patient, whose red cells were Group B, then received marrow cells from his
Group O, histocompatible, sister. Thereafter, reticulocytes, Group O
erythrocytes, and female leukocytes appeared in the peripheral blood.
Erythroid precursors were seen in the patient's marrow for the first time
in his life, and all lacked fluorescent Y chromosomes. Dividing cells were
all female. After initially progressing well, the patient developed
interstitial pneumonia and died 55 days after the transplant. The
successful erythroid graft suggested that this patient's failure to produce
red blood cells was due to a defective stem cell rather than to a humoral
defect, plasma inhibitor, or abnormal marrow microenvironment. It suggested
further that sibling marrow may be engrafted in patients who have received
multiple transfusions, even from a parent.
Volume 48,
Issue 4,
pp. 491-498,
10/01/1976
Copyright © 1976 by The American Society of Hematology
