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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Kinetics of chimerism during the early post-transplant period in pediatric patients with malignant and non-malignant hematologic disorders: implications for timely detection of engraftment, graft failure and rejection.
J Dubovsky,H Daxberger,Gerhard Fritsch,Dieter Printz,Christoph Peters,S. Matthes,Helmut Gadner,Thomas Lion +7 more
TL;DR: Close monitoring by STR-PCR showed that graft rejection and autologous recovery can occur early and with very rapid dynamics, providing support for the concept of initiating genotype analyses early after SCT and monitoring at rather short intervals to permit timely evaluation of clinically relevant processes.
Journal ArticleDOI
Endothelial cell precursors are normal components of human umbilical cord blood
TL;DR: It is concluded that endothelial cell precursors circulate in UCB, and may be derived from the CD34+ cell fraction.
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High efficiency retroviral mediated gene transduction into single isolated immature and replatable CD34(3+) hematopoietic stem/progenitor cells from human umbilical cord blood.
TL;DR: Stable expression of the transduced gene into single purified stem/progenitor cells with replating capacity is demonstrated, results that should be applicable for future clinical studies that may utilize selected subsets of stem and progenitor cells for gene therapy.
Journal ArticleDOI
Toward cell therapy using placenta-derived cells: disease mechanisms, cell biology, preclinical studies, and regulatory aspects at the round table.
Ornella Parolini,Francesco Alviano,Irene Bergwerf,Diana Boraschi,Cosimo De Bari,Peter De Waele,Massimo Dominici,Marco Evangelista,Werner Falk,Simone Hennerbichler,David C. Hess,Giacomo Lanzoni,Bing Liu,Fabio Marongiu,Colin McGuckin,Stefan Mohr,Maria Luisa Nolli,Racheli Ofir,Peter Ponsaerts,Luca Romagnoli,Abraham Solomon,Maddalena Soncini,Stephen C. Strom,Daniel Surbek,Sankar Venkatachalam,Sankar Venkatachalam,Susanne Wolbank,Steffen M. Zeisberger,Andy Zeitlin,Andreas H. Zisch,Cesar V. Borlongan +30 more
TL;DR: The international workshop held in Brescia, Italy, in March 2009 aimed to harness an understanding of basic inflammatory mechanisms inherent in human diseases with updated findings regarding biological and therapeutic properties of human placenta-derived cells, with particular emphasis on their potential for treating inflammatory diseases.
Journal ArticleDOI
Selection of optimal alternative graft source: Mismatched unrelated donor, umbilical cord blood, or haploidentical transplant
TL;DR: The rationale for donor choice and the need of phase 3 studies to help answer the question of suitably matched unrelated donor choice are discussed.
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.