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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Current thawing and infusion practice of cryopreserved cord blood: the impact on graft quality, recipient safety, and transplantation outcomes
TL;DR: Methods of handling, thawing, and infusion of cord blood (CB) products vary substantially among thaw/transplant centers (TCs) especially when the TC is unable to follow recommendations due to foreseeable technical, quality, and/or clinical factors.
Journal ArticleDOI
Bone marrow transplantation from the personal viewpoint
TL;DR: A rather personal account of the early work with bone marrow transplantation that was to lead eventually to the award of the Nobel Prize in 1990 is written.
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Red blood cell depletion with a semiautomated system or hydroxyethyl starch sedimentation for routine cord blood banking: a comparative study
Pilar Solves,Vicente Mirabet,Dolores Planelles,I. Blasco,Alfredo Perales,Francisco Carbonell-Uberos,M Angeles Soler,Roberto Roig +7 more
TL;DR: A large number of studies have been performed to establish techniques for volume reduction of CB units, and the results suggest that volume reduction techniques should be considered for long-term cord blood banking.
Journal ArticleDOI
Improving outcomes in umbilical cord blood transplantation: State of the art
Corey Cutler,Karen K. Ballen +1 more
TL;DR: Strategies to improve umbilical cord blood transplant outcomes are discussed, focusing on cord blood unit selection, expansion, and homing efficiency.
Journal ArticleDOI
Immune reconstitution in children after unrelated cord blood transplantation
Paul Szabolcs,Donna Niedzwiecki +1 more
TL;DR: There is a great need to learn more about the biology of immune recovery after UCBT and there is realistic hope that clinical translation of new immunotherapy strategies could enhance immune competence afterUCBT either by having an impact on the thymic-independent early period or by fosteringThymic recovery.
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.
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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.