Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling

Eliane Gluckman, Hal E. Broxmeyer, Arleen D. Auerbach, Henry S. Friedman, Gordon W. Douglas, Agnes Devergie, Helene Esperou, Dominique Thierry, Gerard Socie, Pierre Lehn, Scott Cooper, Denis English, Joanne Kurtzberg, Judith Bard, Edward A. Boyse

Research output: Contribution to journalArticle

Abstract

The clinical manifestations of Fanconi's anemia, an autosomal recessive disorder, include progressive pancyto-penia, a predisposition to neoplasia, and nonhematopoietic developmental anomalies [1-3]. Hypersensitivity to the clastogenic effect of DNA-cross-linking agents such as diepoxybutane acts as a diagnostic indicator of the genotype of Fanconi's anemia, both prenatally and postnatally [3-6]. Prenatal HLA typing has made it possible to ascertain whether a fetus is HLA-identical to an affected sibling [7]. We report here on hematopoietic reconstitution in a boy with severe Fanconi's anemia who received cryo-pre-served umbilical-cord blood from a sister shown by prenatal testing to be unaffected by the disorder, to have a normal karyotype, and to be HLA-identical to the patient. We used a pretransplantation conditioning procedure developed specifcally for the treatment of such patients [8]; this technique makes use of the hypersensitivity of the abnormal cells to alkylating agents that cross-link DNA [9,10] and to irradiation [11] In this case, the availability of cord blood obviated the need for obtaining bone marrow from the infant sibling. This use of cord blood followed the suggestion of one of us that blood retrieved from umbilical cord at delivery, usually discarded, might restore hematopoiesis - a proposal supported by preparatory studies by some of us [12] and consistent with reports on the presence of hematopoietic stem and multipotential (CFU-GEMM), erythroid (BFU-E), and granulocyte-macrophage (CFU-GM) progenitor cells in human umbilical-cord blood (see the references cited by Broxmeyer et al. [12]).

Original languageEnglish (US)
JournalCellular Therapy and Transplantation
Volume2
Issue number7
DOIs
StatePublished - Dec 1 2010

Fingerprint

Fanconi Anemia
Fetal Blood
Siblings
Hypersensitivity
Myeloid Progenitor Cells
Histocompatibility Testing
Granulocyte-Macrophage Progenitor Cells
Erythroid Precursor Cells
Alkylating Agents
DNA
Hematopoiesis
Karyotype
Granulocytes
Fetus
Stem Cells
Bone Marrow
Macrophages
Genotype
Neoplasms

Keywords

  • Anemia
  • Aplastic therapy
  • Blood preservation
  • Fanconi anemia
  • Female
  • Fetal blood
  • Hematopoietic stem cell transplantation
  • Histocompatibility testing
  • HLA antigens
  • Humans
  • Male
  • Pregnancy
  • Prenatal diagnosis
  • Preschool child

ASJC Scopus subject areas

  • Molecular Medicine
  • Transplantation

Cite this

Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. / Gluckman, Eliane; Broxmeyer, Hal E.; Auerbach, Arleen D.; Friedman, Henry S.; Douglas, Gordon W.; Devergie, Agnes; Esperou, Helene; Thierry, Dominique; Socie, Gerard; Lehn, Pierre; Cooper, Scott; English, Denis; Kurtzberg, Joanne; Bard, Judith; Boyse, Edward A.

In: Cellular Therapy and Transplantation, Vol. 2, No. 7, 01.12.2010.

Research output: Contribution to journalArticle

Gluckman, E, Broxmeyer, HE, Auerbach, AD, Friedman, HS, Douglas, GW, Devergie, A, Esperou, H, Thierry, D, Socie, G, Lehn, P, Cooper, S, English, D, Kurtzberg, J, Bard, J & Boyse, EA 2010, 'Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling', Cellular Therapy and Transplantation, vol. 2, no. 7. https://doi.org/10.3205/ctt-2010-en-000079.01
Gluckman, Eliane ; Broxmeyer, Hal E. ; Auerbach, Arleen D. ; Friedman, Henry S. ; Douglas, Gordon W. ; Devergie, Agnes ; Esperou, Helene ; Thierry, Dominique ; Socie, Gerard ; Lehn, Pierre ; Cooper, Scott ; English, Denis ; Kurtzberg, Joanne ; Bard, Judith ; Boyse, Edward A. / Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling. In: Cellular Therapy and Transplantation. 2010 ; Vol. 2, No. 7.
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AU - Socie, Gerard

AU - Lehn, Pierre

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