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• W2039401478 abstract "See related article, p 570 . The first report of hematologic correction of a genetic disorder with the use of umbilical cord blood transplantation, in 1989,1Gluckman E Broxmeyer HA Auerbach AD Friedman HS Douglas GW Devergie A et al.Hematopoietic reconstitution in a patient with Fanconi’s anemia by means of umbilical-cord blood from an HLA-identical sibling.N Engl J Med. 1989; 321: 1174-1178Crossref PubMed Scopus (1762) Google Scholar described the recovery of a child with Fanconi’s anemia who received a transplant of placental blood from a fully histocompatible sibling donor. Subsequently, cord blood transplants from siblings with less complete HLA matching were also reported to be beneficial.2Wagner JE Kernan NA Steinbuch M Broxmeyer HE Gluckman E. Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease.Lancet. 1995; 346: 214-219Abstract PubMed Google Scholar The instances of graft-versus-host disease observed were remarkably fewer and milder than expected for the degree of mismatching, based on historical experience with bone marrow sources. It was hypothesized that the relative naivete of the immune cells in cord blood products permitted a greater degree of HLA disparity between donor and recipient, and furthermore, that partially mismatched unrelated cord blood units could prove a useful alternative source of hematopoietic stem cells for patients lacking a closely matched related or unrelated bone marrow donor. Fueled initially by enthusiasm for what was hailed as a “safer” and more rapidly available form of unrelated donor transplantation, many pediatric transplant centers experimented with cord blood transplants during the following decade. Because collection of and access to cord blood were unregulated in the early era, even small clinical programs that did not meet the certification requirements for participation in the National Marrow Donor Program engaged in the initial investigational treatments with this approach. The first composite publication of results of 44 cases of cord blood transplantation, compiled informally by an international group of investigators in 1995, reflected a highly heterogeneous group of malignant and non-malignant diagnoses and various degrees of donor-recipient matching.2Wagner JE Kernan NA Steinbuch M Broxmeyer HE Gluckman E. Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease.Lancet. 1995; 346: 214-219Abstract PubMed Google Scholar That report identified 3 patients with X-linked immunodeficiencies (X-linked severe combined immunodeficiency syndrome, Wiskott-Aldrich syndrome, and X-linked lymphoproliferative disorder)—all of whom experienced favorable outcomes. In this issue of The Journal, Ziegner et al3Ziegner UHM Ochs HD Schanen C Feig SA Seyama K Futatani T et al.Unrelated umbilical cord stem cell transplantation for X-linked immunodeficiencies.J Pediatr. 2001; 138: 570-573Abstract Full Text Full Text PDF PubMed Scopus (49) Google Scholar report on 3 additional boys with prematurely lethal X-linked immunodeficiencies: 2 brothers with X-linked lymphoproliferative disorder and another with X-linked CD40 ligand deficiency. All 3 of these patients were treated with 5/6 antigen matched cord blood transplants, experienced only mild GvHD (although one boy had a prolonged episode of immune-mediated thrombocytopenia), and have demonstrated full immunologic recovery at 2 years after their treatment. These case studies argue for a role for cord blood transplantation in the treatment of children with life-threatening genetic immunodeficiencies. The ledger of pros and cons for consideration of cord blood transplantation as an alternative to marrow from an unrelated adult includes the following arguments. In favor of the use of unrelated cryopreserved cord blood are: (1) a shorter interval between identification of the suitable cord blood and transplantation, compared with waiting for identification and medical clearance of a marrow donor; (2) decreased incidence of classical GvHD; (3) increased likelihood of finding a suitable match, if 4/6 matching is accepted; (4) low likelihood of transmitting cytomegalovirus or Epstein-Barr virus infection to a seronegative recipient. Limitations of the use of cord blood transplants include: (1) limited cell and stem cell dose; (2) because of the anonymity of the donor, no subsequent stem cell or immune cell infusions are possible (eg, may not be adequate in the setting of “mini” or less myeloablative conditioning protocols); (3) lack of medical history for the donor (possible transmission of another genetic disease that is not routinely monitored at the time of cord blood collection). In favor of the use of marrow are: (1) a larger donor pool, with potential to find a suitably closely matched donor for >80% of most recipients (<60% for African Americans, however); (2) fewer limitations on administered cell dose and possibility of future stem cell and lymphocyte collections, if needed; (3) potential to select cytomegalovirus- or Epstein-Barr–immune donor, if recipient is seropositive. However, 12 years after the initial successes were achieved, and now that an estimated 1500 cord blood transplantations have been performed, we are still without a clear understanding of how this technology can be optimally applied now or in the future. Collection of outcome data after cord blood transplantations, including those performed in the United States, is incomplete, especially in the critical details regarding resolution of underlying genetic disorders. No prospective studies comparing the efficacy of cord blood transplantation with other strategies, in particular unrelated bone marrow transplantation, have been developed. Retrospective analyses of outcomes from the major registries in the world, Eurocord and the New York Blood Center,4Gluckman E Rocha V Boyer-Chammard A Locatelli F Arcese W Pasquini R et al.Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group.N Engl J Med. 1997; 337: 373-381Crossref PubMed Scopus (1182) Google Scholar, 5Rubinstein P Carrier C Scaradavou A Kurtzberg J Adamson J Migliaccio AR et al.Outcomes among 562 recipients of placental-blood transplants from unrelated donors.N Engl J Med. 1998; 339: 1565-1577Crossref PubMed Scopus (1185) Google Scholar reflect overall survival rates for highly heterogeneous groups of recipients of around 30% at 2 or 3 years after transplantation of unrelated cord blood—no better than for heterogeneous pediatric cohorts treated with unrelated bone marrow transplants.6Hongeng S Krance RA Bowman LC Srivastava DK Cunningham JM Horwitz EM et al.Outcomes of transplantation with matched-sibling and unrelated-donor bone marrow in children with leukaemia.Lancet. 1997; 350: 767-771Abstract Full Text Full Text PDF PubMed Scopus (147) Google Scholar, 7Woolfrey A Frangoul H Anasetti C. Unrelated bone marrow transplants for children with acute lymphoblastic leukemia [abstract].Blood. 1999; 94: 712aGoogle Scholar A retrospective analysis of children receiving unrelated bone marrow versus cord blood transplants for acute leukemia performed by the Eurocord consortium also showed no disease-free survival advantage to either approach.8Locatelli F Rocha V Chastang C Arcese W Michel G Abecasis M et al.Factors associated with outcome after cord blood transplantation in children with acute leukemia. Eurocord-Cord Blood Transplant Group.Blood. 1999; 93: 3662-3671PubMed Google Scholar The long-term durability of cord blood grafts will not be known for years. Although laboratory studies of the ex vivo expansion of cord blood stem cells with cytokine combinations continue, clinical benefit remains to be shown. What have we learned? Studies in basic hematopoiesis of cord blood reveal that it is a rich source of stem/progenitor cells, which are in a slow, non-cycling state at the time of collection and cryopreservation. Cord blood stem cells have extensive capacity for proliferation and contain a high proportion of cells apparently capable of self-renewal and long-term repopulation of the marrow compartments.9Broxmeyer H. Cord blood stem and progenitor cell therapy for immunodeficiency and other disorders.in: Immunology and allergy clinics of North America.vol 16. WB Saunders, Philadelphia1996: 439-452Google Scholar Engraftment of white blood cells and platelets after cord blood transplantation, however, is delayed compared with bone marrow grafts.10Kurtzberg J Laughlin M Graham ML Smith C Olson JF Halperin EC et al.Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients.N Engl J Med. 1996; 335: 157-166Crossref PubMed Scopus (994) Google Scholar Immunologic studies indicate that T and natural killer cells in the cord blood have reduced effector cell capacity, including cytotoxicity and cytokine production.11Roncarolo MG Bigler M Ciuti E Martino S Tovo PA. Immune responses by cord blood cells.Blood Cells. 1994; 20: 573-585PubMed Google Scholar, 12Chalmers IM Janossy G Contreras M Navarrete C. Intracellular cytokine profile of cord and adult blood lymphocytes.Blood. 1998; 92: 11-18PubMed Google Scholar However, cord blood immune cells can be readily activated by exposure to alloantigen or proinflammatory cytokines to mediate many of the mature effector mechanisms, especially when transferred to an antigenic milieu (eg, transplant recipient) for which evolution has not prepared them. To wit, the relapse rate after unrelated cord blood transplantation versus bone marrow transplantation is similarly low when compared with matched marrow transplantation. Cord blood may also differ from matched marrow harvests and mobilized peripheral blood collections by a lack of preformed or precursor immune regulatory cells that can lower the risk of late “immunoregulatory dysfunction” after mismatched transplantation. Although the incidence of classical GvHD is reportedly lower after transplantation of unrelated cord blood versus marrow, anecdotally there appears to be a substantial incidence of late autoimmune phenomena. Risk of late infections as a cause of morbidity and death is similar among recipients of unrelated cord and bone marrow transplants, irrespective of the rate of GvHD.13Nuckols JD. Autopsy findings in umbilical cord blood transplant recipients.Am J Clin Pathol. 1999; 112: 335-342PubMed Google Scholar Factors favoring improved outcome with cord blood transplants across all recipient diagnostic groups include higher cell dose with respect to recipient weight and recipient cytomegalovirus seronegativity.4Gluckman E Rocha V Boyer-Chammard A Locatelli F Arcese W Pasquini R et al.Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group.N Engl J Med. 1997; 337: 373-381Crossref PubMed Scopus (1182) Google Scholar Currently, young children with immunodeficiencies who are treated with 5/6 or 6/6 matched unrelated marrow transplants enjoy response rates comparable to those achieved with matched sibling donor transplantation.14Filipovich A. Stem cell transplantation from unrelated donors for correction of primary immunodeficiencies.in: Immunology and allergy clinics of North America.vol. 16. WB Saunders, Philadelphia1996: 337-392Google Scholar Suitably matched (6/6 or 5/6) unrelated bone marrow donors can be identified for the majority of children without sibling donors who require transplantation. Although the number of cord blood transplantations performed for immunodeficiencies is still small, it is probably time to attempt a retrospective comparison with results of unrelated marrow transplantations to test for any potential advantage of either modality. If the ongoing study of feasibility of cord blood transplantation supported by the National Heart, Lung, and Blood Institute shows benefit to recipients of 4/6 or even 3/6 matched cord blood grafts, this could establish use of cord blood as a valuable addition to the armamentarium of hematopoietic stem cell sources for patients lacking other options and provide the rationale for funding future cord blood banking." @default.
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