2005, Number 2
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Rev Invest Clin 2005; 57 (2)
Pediatric hematopoietic stem-cell transplantation. Primary immunodeficit treatment alternative
Olaya-Vargas A
Language: Spanish
References: 52
Page: 324-332
PDF size: 74.32 Kb.
ABSTRACT
The congenital immunodeficiency disorders in which the defect has been clearly traced to the stem cell can be cured with allogeneic stem-cell transplantation (SCT) from an unaffected donor. Widespread application of this treatment modality has been tempered by the fact that risk-benefit considerations do not always favor a procedure that carries a significant risk for morbidity and mortality. Some malignant disorders of childhood eventually have to be treated by an autologous or allogeneic SCT, however nonmalignant disorders can also be treated with this approach. This article reviews the current status of SCT for nonmalignant inherited immunodeficiency disorders.
REFERENCES
Gatti RA, Meewissenn HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968; 2: 1366-9.
Bach FH, Albertini RJ, Anderson JL, et al. Bone marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet 1968; 2: 1364-6.
Copenhagen Study Group of Immunodeficiencies. Bone-marrow transplantation from an HLA-non-identical but mixed-lymphocyte-culture identical donor. Lancet 1973; 2: 1146-50.
O’Reilly RJ, Dupont B, Pahwa S, et al. Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor. N Engl J Med 1977; 297: 1311-18.
Rosen FS, Cooper MD, Wedgewoods RJP. The primary immunodeficiency disease. N Engl J Med 1995; 333: 431-40.
Rosen FS, Aiuti F, Hitzig W, et al. Meeting report: Primary immunodeficiency disease. Clin Immunol Inmunophathol 1983; 28: 450-75.
Gibllet ER, Anderson JE, Cohen F, Pallara B, Meeuwisen HG. Adenosine-deaminase deficiency in two patients with severely impaired cellular immunitary. Lancet 1972; 2: 1067-9.
Carson DA, Kaye J, Seegmiller JE. Lymphospecific toxicity in adenosine deaminase deficiency and urine nucleoside phosphorylase deficiency: possible role of the nucleoside kinase(s). Proc Natl Acad Sci USA 1997; 74: 5677-82.
DeVaal OM, Seynhave V. Reticular dysgenesis. Lancet 1959; 2: 1123-5.
Ommen G. Familial reticuloendotheliosis. N Engl J Med 1965; 273: 427-32.
Ruco LP, Stoppacciaro A, Pezzella F, et al. The Omenn syndrome: Histological, immunohistochemical and ultraestructural evidence for a partial T cell deficiency evolvyng in an abnormal proliferation of T lymphocites and s-100+/T6+ Langerhans-like cells. Virchows Arch 1985; 407: 69-82.
Gatti RA, Platt N, Pomerance HH, et al. Hereditary lymphopenic agammaglobulinemia associated with a distinctive form of short-limbed dwarfsm and ectodermal dysplasia. J Pediatr 1969; 75: 675-84.
Gelfand EW, Oliver JM, Schuurman RK, Matherson DS, Dosch HM. Abnormal lymphocyte capping in a patient with severe combined immunodeficiency. N Engl Med 1979; 301: 1245-9.
Jung LKL, Fu SM, Hara T, Kappor N, Good RA. Defective expression of T cell associated glycoprotein in severe combined immunodeficiency. J Clin Invest 1986; 77: 940-6.
Alarcon B, Rigueiro JR, Arnaiz-Villena A, Terhorst C. Familial defect in the surface expression of the T-cell receptor-CD3 complex. N Engl J Med 1988; 319: 1203-8.
Roifman CM, Hummel D, Martinez-Valdez H, et al. Depletion of CD8+ cells in human thymic medulla results in selective immune deficiency. J Exp Med 1989; 170: 2177-82.
Monafo WJ, Polmar SH, Neudof S, Mather A, Filipovich AH. A hereditary immunodeficiency characterized by CD8+ T lymphocytes deficiency and impaired lymphocyte activation. Clin Exp Immunol 1992; 90: 390-3.
Cooper MD, Chase HP, Lowman JT, Krivit W, Good RA. The Wiskott-Aldrich syndrome. An immunologyc deficiency disease involving the afferent limb of immunity. Am J Med 1968; 44: 499-513.
Blasé RM, Stoober W, Brown RS, Waldmann RA. The Wiscott-Aldrich syndrome. A disorder with possible defect antigen processing or recognition. Lancet 1968; 1: 1056-61.
Parkman R, Remold-O’Donnell E, Kenney DM, Perrine S, Rosen FS. Surfase protein abnormalities in lymphocytes and plateles from patients with Wilscott-Aldrich syndrome. Lancet 1981; 2: 1387-8.
Shelly CS, Remold-O’Donnell E, Davis III, AE, et al. Molecular characterization of sialophorin (CD43), the lymphocyte surface sialoglycoprotein defective in Wiskott-Aldrich syndrome. Proc Natl Acade Sci USA 1989; 86: 819-22.
Buckley RH, Schiff S, Schiff R, et al. Haploidentical bone marrow transplantation in human severe combined immunodeficiency. Semin Hematol 1993; 30(Supl. 4): 92-104.
Fischer A, Landais P, Friedrich W, et al. European experience of bone marrow transplantation for severe combined immunodeficiency disease. Lancet 1990; 336: 830-4.
Stephan JL, Viekova V, Le Deist F, et al. Severe combined immunodeficiency. A retrospective single center study of clinical presentation and outcome in 117 patients. J Pediatr 1993; 123: 564-72.
Buckley RH. Bone marrow reconstitution in primary immunodeficiency. Clin Immunol Principles Practice 1995; 2: 1813-30.
Gricelli C, Dorandy A, Villerizier JL, Bauses JJ, Daguiland E. Selective defect of precursor T cells associated with apparently normal B lymphocytes in severe combined immunodeficiency disease. J Pediatr 1978; 93: 404-11.
Korver K, Delange GG, Van Der Verg ER, et al. Lymphoid chimerism after allogeneic bone marrow transplantation. Transplantation 1987; 44: 643-50.
Kenny AB, Hitzig WH. Bone marrow transplantation for severe combined immunodeficiency. Eur J Pediatr 1979; 131: 155-77.
Leggiardo RJ, Winkenstein JA, Hughes WT. Prevalence of pneumocystis carini pneumonitis in severe combined immunodeficiency. J Pediatr 1981; 99: 96-8.
Hall CB, McBride JT, Walsh EE, et al. Aerosolised ribavirin treatmen of infants with respiratory syncitial viral infections. A randomized doble-blind study. N Engl J Med 1983; 308: 1443-7.
Emmanuel D, Caminham I, Jules-Elysea K, et al. Cytomegalovirus pneumonia after bone marrow transplantation succerfully treated with the combination of ganciclovir and high dose intravenous immune globulin. Ann Intern Med 1988; 109: 777-82.
Kurtzberg J, Laughlin M, Graham M, et al. Placental blood as a source of hematopoyetic stem cell for transplantation into unrelated recipients. N Engl J Med 1996; 335: 157-165.
Wagner J, Rosenthal J, Sweetman R, et al. Successful transplantation of HLA-matched and HLA mismatched umbilical cord blood from unrelated donors: Analysis of engraftment and acute graft-versus-host disease. Blood 1996; 88: 795-802.
Gluckman E, Rocha V, Boyer-Chammard A, et al. Outcome of cord blood transplantation from related and unrelated donors. N Engl J Med 1997; 337: 373-82.
Von Boehmer H, Sprent J, Nabholz M. Tolerance to histocompatibility determinants in tetraparental bone marrow chimeras. J Exp Med 1975; 141: 322-34.
Reisner Y, Itsicovitch L, Meshorer A, Sharon N. Hematopoietic stem cell transplantation using mouse bone marrow and spleen cells fractionated by lectins. Proc Natl Acad Sci USA 1978; 75: 2933-6.
Reisner Y, Kapoor N, Kirkpatrick D, et al. Transplantation for acute leukemia with HLA-A and B non-identicalparental marrow cells fractionated with soybean agglutinin and sheep red cells. Lancet 1981; 2: 327-31.
Reisner Y, Kapoor N, Kirkpatric D, et al. Transplantation for severe combined immunodeficiency HLA-A,B,D,Dr incompatible parental marrow fractionate by soybean agglutinin and sheep red blood cells. Blood 1983; 61: 341-8.
O’Reilly RJ, Kapoor N, Kirkpatric D, et al. Transplantation for severe combined immunodeficiency using histoincompatible parental marrow fractionated by soybean agglutinin and sheep red bloos cells: Experience in six consecutive cases. Transplant Proc 1983; 17: 455-9.
Reinherz E, Geha R, Rappeport JM, et al. Reconstitution after transplantation with T-lymphocyte-depleted HLA haplotype-mismatched bone marrow for severe combined immunodeficiency. Proc Natl Acad Sci USA 1982; 79: 6047-51.
Kapoor N, Jung LKL, Engelhard D, et al. Lymphoma in a patient with severe combined immunodeficiency with adenosine-diaminase deficiency, following un-sustained engrafment of histoincompatible TCD bone marrow. J Pediatr 1986; 100: 435-8.
Fischer A, Durandy A, De Villaraty JP, et al. HLA-haploidentical bone marrow transplantation for severe combined immunodeficiency using E-rosete fractionation and cyclosporine. Blood 1986; 67: 444-9.
Ritz J. Use of monoclonal antibodies in autologous and allogeneic bone marrow transplantation. Clin Hematol 1983; 12: 813-32.
Moen RC, Horowitz SD, Sondel PM, et al. Immunologic reconstitution after haploidentical bone marrow transplantation for immune deficiency disorders: Treatment of bone marrow cells with monoclonal antibody CT-2 and complement. Blood 1987; 70: 664-9.
Morgan G, Linen DC, Knott LT, et al. Succesful haploidentical mismatched bone marrow trasplantation in severe combined immunodeficiency: T cell removal using CAMPATH-1 monoclonal antibody and E-roseting. Br J Hematol 1986; 62: 421-30.
Dickinson AM, Reid MM, Abunum M, et al. In vitro T cells depletions using Compath 1M for mis-match BMT for severe combined immunodeficiency(SCID). Bone Marrow Transplant 1997; 19: 323-9.
Flake A, Ronkarolo M, Puck J, et al. Treatment of X-lilinked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. N Engl J Med 1996; 335: 1806-10.
Frame J, Collins NJ, Cartagena T, et al. T-cell depletion of human bone marrow: Comparison of COMPATH-1ª plus complement, anti T cell ricin a chain immonotoxin and soybean aglutinin alone or in combination with sheep erithrocytes or immunomagnetic beads. Transplantations 1989; 47: 984-8.
Dror Y, Gallagher RM, Wara DW, et al. Immune reconstitution in severe combined immunodeficiency disease after lectin-treated, TCD haplo-identical bone marrow transplantation. Blood 1993; 81: 2021-30.
Touraine J. In utero transplantation of fetal liver stem cell transplantation. J Am Med Assoc 1997; 278: 932-7.
Flake AW, Roncarolo MG, Puck JM, et al. Treatment of X-linked severe combined immunodeficiency by in utero transplantation of paternal bone marrow. N Engl J Med 1996; 335: 1806-10.
Wengler G, Lanfranchi A, Frusca T, et al. In-utero transplantation of paternal CD34 hematopoietic progenitor cells in a patient with x-linked severe combined immunodeficiency (SCIDXI). Lancet 1996; 348: 1484-7.