Journal ArticleDOI
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,Agnès Devergie,Helene Esperou,Dominique Thierry,Gérard Socié,Pierre Lehn,Scott Cooper,Denis English,Joanne Kurtzberg,Judith Bard,Edward A. Boyse +14 more
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TLDR
It is necessary to select patients suitable for vaginal or laparoscopic mesh placement for Fanconi's anemia preoperatively on the basis of prior history and once they provide informed consent for surgery.Abstract:
The clinical manifestations of Fanconi’s anemia, an autosomal recessive disorder, include progressive pancytopenia, 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-preserved 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 specifically 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]).read more
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Journal ArticleDOI
Multilineage differentiation activity by cells isolated from umbilical cord blood: expression of bone, fat, and neural markers.
H. S. Goodwin,Alma R. Bicknese,S. N. Chien,B. D. Bogucki,D. A. Oliver,C. O. Quinn,Donna A. Wall +6 more
TL;DR: Cord blood contains a population of cells that can be expanded in culture and are able to express the phenotype of multiple lineages, similar to what has been previously reported with bone marrow.
Journal ArticleDOI
Placental and/or umbilical cord blood: an alternative source of hematopoietic stem cells for transplantation
Mitchell S. Cairo,John E. Wagner +1 more
TL;DR: HLA-MATCHED RELATED allogeneic stem cell transplantation has been successfully used as the treatment of choice in selected high-risk or recurrent hematologic malignancies, marrow failure syndromes, severe congenital immunodeficiency states, and selected metabolic disorders.
Journal ArticleDOI
Factors associated with outcomes of unrelated cord blood transplant: Guidelines for donor choice
Eliane Gluckman,Vanderson Rocha,William Arcese,Gérard Michel,Guillermo Sanz,Kawah Chan,Tsuneo A. Takahashi,Juan Ortega,Alexandra H. Filipovich,Franco Locatelli,Shigetaka Asano,Franca Fagioli,Marcus R. Vowels,Anne Sirvent,J. P. Laporte,Karin Tiedemann,Sergio Amadori,Manuel Abecassis,Pierre Bordigoni,Blanca Diez,Peter J. Shaw,Ajay Vora,Maurizio Caniglia,Federico Garnier,Irina Ionescu,Joan Garcia,Gesine Koegler,Paolo Rebulla,Sylvie Chevret +28 more
TL;DR: Optizing cord blood donor selection based mainly on cell dose and human leukocyte antigen (HLA) disparities may further improve results of unrelated cord blood transplants (UCBT) and provide indications for a better choice of cord blood units according to cord blood cell content and HLA.
Journal ArticleDOI
Preimplantation diagnosis for Fanconi anemia combined with HLA matching.
TL;DR: To the authors' knowledge, this is the first PGD with HLA matching, demonstrating feasibility of preselecting unaffected embryos that can also be an HLA-compatible source for stem cell transplantation for a sibling.
Journal ArticleDOI
Signaling pathways governing stem-cell fate
TL;DR: The focus of this review is to highlight some of the most recent developments concerning signaling pathways governing HSC fate, including Notch, Wingless-type (Wnt), Sonic hedgehog (Shh), and Smad pathways.
References
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Progress in Clinical and Biological Research
TL;DR: All with an interest in tumours will find something to stimulate them in this book but even these items are to some extent outweighed by the insight given into the thoughts and policies of this fascinating country.
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Human umbilical cord blood as a potential source of transplantable hematopoietic stem/progenitor cells
Hal E. Broxmeyer,Gordon W. Douglas,Giao Hangoc,Scott Cooper,Judith Bard,Denis English,Margaret Arny,Lewis Thomas,Edward A. Boyse +8 more
TL;DR: It was determined that granulocyte-macrophage, erythroid, and multipotential progenitor cells remained functionally viable in cord blood untreated except for addition of anticoagulant for at least 3 days at 4 degrees C or 25 degrees C (room temperature), though not at 37 degrees C, implying that these cells could be satisfactorily studied and used or cryopreserved for therapy.
Journal ArticleDOI
Susceptibility of Fanconi's anaemia fibroblasts to chromosome damage by carcinogens
TL;DR: Experiments in which viable FA fibroblasts were exposed to a direct-acting mutagen or carcinogen for a period of 6 d, ensuring chronic exposure of the cells during one or more cell cycles, until increased cell density inhibited further cell division.
Journal Article
Familial constitutional panmyelocytopathy, Fanconi's anemia (F.A.). I. Clinical aspects.
Journal ArticleDOI
International Fanconi Anemia Registry: relation of clinical symptoms to diepoxybutane sensitivity.
TL;DR: It is concluded that hypersensitivity to the clastogenic effect of DEB is a useful discriminator for FA and a simplified scoring method for classifying patients on the basis of eight clinical manifestations that are the best predictors for FA is presented.