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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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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Placental/umbilical cord blood transplantation
Girolamo Sirchia,Paolo Rebulla +1 more
TL;DR: The data reported by the investigators of the New York Placental/Umbilical Cord Blood Program and of the Eurocord group indicate that the clinical outcome of allogeneic unrelated CB transplantation is significantly related to cell dose, being more effective in children than in adults, and is highly dependent on disease stage at transplantation.
Featured Article Stereotactic brain injection of human umbilical cord blood mesenchymal stem cells in patients with Alzheimer's disease dementia: A phase 1 clinical trial
Hee Jin Kim,Sang Won Seo,Jong Wook Chang,Jung Il Lee,Chi Hun Kim,Juhee Chin,Soo Jin Choi,Hunki Kwon,Hyuk Jin Yun,Jongmin Lee,Sung Tae Kim,Yearn Seong Choe,Kyung Han Lee,Duk L. Na +13 more
TL;DR: A phase 1 clinical trial in nine patients with mild‐to‐moderate Alzheimer's disease to evaluate the safety and dose‐limiting toxicity of stereotactic brain injection of human umbilical cord blood–derived mesenchymal stem cells (hUCB‐MSCs).
Journal ArticleDOI
Umbilical cord blood transplantation.
TL;DR: In the unrelated donor transplant setting, shorter time to transplant and tolerance of 1-2 human leukocyte antigen mismatch, which increases the chance of finding a suitable donor, are evident advantages over bone marrow transplantation as mentioned in this paper.
Journal ArticleDOI
Hematopoietic potential of cryopreserved and ex vivo manipulated umbilical cord blood progenitor cells evaluated in vitro and in vivo.
David DiGiusto,Robert J. Lee,James J. Moon,Katherine Moss,Tom O'Toole,Amy Voytovich,Donna Webster,James J. Mulé +7 more
TL;DR: Human UCB is a rich source of multipotent hematopoietic progenitors that can be cryopreserved, enriched by physical methods, and expanded in a limited fashion without measurable loss of long-term culture or in vivo engrafting potential as measured in these assays.
Journal ArticleDOI
Results of the Cord Blood Transplantation (COBLT) Study unrelated donor banking program
Joanne Kurtzberg,Mitchell S. Cairo,John K. Fraser,L.A. Baxter-Lowe,Geoff Cohen,Shelly L. Carter,Nancy A. Kernan +6 more
TL;DR: The goals of the Cord Blood Transplantation Study banking program were to develop standard operating procedures for cord blood donor recruitment and banking and to build an ethnically diverse unrelated CB bank to support a transplantation protocol.
References
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Journal ArticleDOI
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.