Skip to main content
Journal cover image

Long-term survival and cure after marrow transplantation for congenital hypoplastic anaemia (Diamond-Blackfan syndrome).

Publication ,  Journal Article
Greinix, HT; Storb, R; Sanders, JE; Deeg, HJ; Doney, KC; Sullivan, KM; Witherspoon, RP
Published in: Br J Haematol
July 1993

Four patients with Diamond-Blackfan syndrome (congenital hypoplastic anaemia) whose disease was resistant to corticosteroid treatment and who were red blood cell transfusion-dependent, were given marrow grafts from allogeneic human-leucocyte-antigen (HLA)-identical siblings. The patients were conditioned with regimens including cyclophosphamide and busulfan. Three of four patients had sustained and complete marrow engraftment. One patient showed early signs of haematopoietic recovery but died on day 35 of pulmonary toxicity. The three surviving patients are well with normal haematopoiesis and Karnofsky performance scores of 100%, 3.0, 7.4 and 10.6 years after transplantation. Congenital hypoplastic anaemia can be treated successfully by allogeneic marrow grafts.

Duke Scholars

Published In

Br J Haematol

DOI

ISSN

0007-1048

Publication Date

July 1993

Volume

84

Issue

3

Start / End Page

515 / 520

Location

England

Related Subject Headings

  • Male
  • Immunology
  • Humans
  • Hematopoiesis
  • Graft vs Host Disease
  • Graft Survival
  • Follow-Up Studies
  • Female
  • Fanconi Anemia
  • Chronic Disease
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Greinix, H. T., Storb, R., Sanders, J. E., Deeg, H. J., Doney, K. C., Sullivan, K. M., & Witherspoon, R. P. (1993). Long-term survival and cure after marrow transplantation for congenital hypoplastic anaemia (Diamond-Blackfan syndrome). Br J Haematol, 84(3), 515–520. https://doi.org/10.1111/j.1365-2141.1993.tb03109.x
Greinix, H. T., R. Storb, J. E. Sanders, H. J. Deeg, K. C. Doney, K. M. Sullivan, and R. P. Witherspoon. “Long-term survival and cure after marrow transplantation for congenital hypoplastic anaemia (Diamond-Blackfan syndrome).Br J Haematol 84, no. 3 (July 1993): 515–20. https://doi.org/10.1111/j.1365-2141.1993.tb03109.x.
Greinix HT, Storb R, Sanders JE, Deeg HJ, Doney KC, Sullivan KM, et al. Long-term survival and cure after marrow transplantation for congenital hypoplastic anaemia (Diamond-Blackfan syndrome). Br J Haematol. 1993 Jul;84(3):515–20.
Greinix, H. T., et al. “Long-term survival and cure after marrow transplantation for congenital hypoplastic anaemia (Diamond-Blackfan syndrome).Br J Haematol, vol. 84, no. 3, July 1993, pp. 515–20. Pubmed, doi:10.1111/j.1365-2141.1993.tb03109.x.
Greinix HT, Storb R, Sanders JE, Deeg HJ, Doney KC, Sullivan KM, Witherspoon RP. Long-term survival and cure after marrow transplantation for congenital hypoplastic anaemia (Diamond-Blackfan syndrome). Br J Haematol. 1993 Jul;84(3):515–520.
Journal cover image

Published In

Br J Haematol

DOI

ISSN

0007-1048

Publication Date

July 1993

Volume

84

Issue

3

Start / End Page

515 / 520

Location

England

Related Subject Headings

  • Male
  • Immunology
  • Humans
  • Hematopoiesis
  • Graft vs Host Disease
  • Graft Survival
  • Follow-Up Studies
  • Female
  • Fanconi Anemia
  • Chronic Disease