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
Reads0
Chats0
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
Citations
More filters
Journal ArticleDOI
Optimizing engraftment--source and dose of stem cells.
Norbert Schmitz,John Barrett +1 more
TL;DR: An understanding of the impact of stem cell source and dose is essential to obtain optimum conditions for a successful outcome after transplant, determining engraftment, transplant-related mortality and risk of leukemic relapse.
Journal ArticleDOI
Allogeneic bone marrow transplantation
Theresa Franco,D. Allen Gould +1 more
TL;DR: From highly investigational to standardized therapy, allogeneic BMT has established its role in the treatment of selected diseases and will bring new challenges and opportunities for patients, families, and health care providers.
Journal ArticleDOI
Prenatal identification of potential donors for umbilical cord blood transplantation for Fanconi anemia
TL;DR: Results suggest that the hematopoietic disorder in FA is related to an underlying problem with cell proliferation, and the possibility that umbilical cord blood from a single individual can be used as an alternative to bone marrow for hematopolietic reconstitution has now been demonstrated by the successful engraftment of two patients with FA.
Journal ArticleDOI
Ontogenic emergence of the hematon, a morphogenetic stromal unit that supports multipotential hematopoietic progenitors in mouse bone marrow.
TL;DR: Unexpectedly, bone marrow taken between embryonic day 17 and day 5 was unable to support myeloid cell production in long-term cultures or to support day 35 LTC-IC growth, but a gain in stromal cell competence occurred between days 7 and 10, which was correlated with the emergence of hematon in the BM.
Journal ArticleDOI
Family-directed umbilical cord blood banking.
Eliane Gluckman,Annalisa Ruggeri,Vanderson Rocha,Etienne Baudoux,Michael Boo,Joanne Kurtzberg,K. Welte,Cristina Navarrete,Suzanna M. van Walraven +8 more
TL;DR: Targeted efforts to bank family cord blood units that can be used for a sibling diagnosed with a disease which can be cured by allogeneic hematopoietic stem cell transplantation or for research that investigates the use of allogenicic or autologous cord blood cells are supported.
References
More filters
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.