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
Guidelines for the development and validation of new potency assays for the evaluation of umbilical cord blood.
Stephen R. Spellman,Carolyn Katovich Hurley,Colleen Brady,Lisa Phillips-Johnson,Robert Chow,Mary J. Laughlin,John McMannis,Jo Anna Reems,Donna Regan,Pablo Rubinstein,Joanne Kurtzberg +10 more
TL;DR: The desired qualities of these assays are described and how the industry proposes to co-operate with developers to bring relevant assays to market are proposed.
Journal ArticleDOI
Past, present, and future efforts to enhance the efficacy of cord blood hematopoietic cell transplantation.
TL;DR: Cord blood has been used as a viable source of hematopoietic stem cells (HSCs) in over 35,000 clinical HCT efforts to treat the same variety of malignant and non-malignant disorders treated by bone marrow (BM) and mobilized peripheral blood (mPB) using HLA-matched or partially HLA disparate related or unrelated donor cells for adult and children recipients as discussed by the authors.
Journal ArticleDOI
Hematopoietic stem and progenitor cell harvesting: technical advances and clinical utility.
TL;DR: This review describes the methods used for harvesting based on recent studies or developments around these methods, and more particularly, the means developed to increase the numbers of HSPCs harvested in each method.
Journal ArticleDOI
Umbilical cord cell banking--implications for the future.
TL;DR: The first successful cord cell transplant to a sibling with Fanconi's anaemia took place 15 years ago and there are now nearly 100 cord blood banks worldwide as mentioned in this paper. But more general ethical questions also arise regarding ownership, consent, confidentiality, costs and quality standards and patenting.
Journal ArticleDOI
Mislabeled units of umbilical cord blood detected by a quality assurance program at the transplantation center.
Jeffrey McCullough,David H. McKenna,Diane Kadidlo,David H. Maurer,Harriett J. Noreen,Kathy French,Claudio G. Brunstein,John E. Wagner +7 more
TL;DR: In a 6-year period, 2 of 871 cord blood units sent to us for transplantation were mislabeled and potentially would have been transplanted incorrectly and this error rate of 1 per 249 (0.4%) patients could have potentially devastating consequences.
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.
Journal ArticleDOI
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.