The Journal of Pediatrics
Volume 119, Issue 6 , Pages 907-912 , December 1991

Marrow transplantation from human leukocyte antigen-identical or haploidentical donors for correction of Wiskott-Aldrich syndrome

Received 25 February 1991 ,Accepted 20 June 1991.

References 

  1. Wiskott A. Familiarer, Angeborener Morbus Werlhofii?. Monatsschr Kinderheilkd. 1937;68:212–216
  2. Aldrich R, Steinberg A, Campbell D. Pedigree demonstrating a sex-linked recessive condition characterized by draining ears, eczematoid dermatitis and bloody diarrhea. Pediatrics. 1954;13:133–138
  3. Perry GS, Spector BD, Schuman LM, et al. The Wiskott-Aldrich syndrome in the United States and Canada (1892–1979). J Pediatr. 1980;97:72–78
  4. Parkman R, Remold-O'Donnell E, Kenney DM, et al. Surface protein abnormalities in the lymphocytes and platelets from patients with Wiskott-Aldrich syndrome. Lancet. 1981;2:1387–1389
  5. Remold-O'Donnell E, Kenny DM, Parkman R, et al. Characterization of a human lymphocyte surface sialoglycoprotein that in defective in Wiskott-Aldrich syndrome. J Exp Med. 1984;159:1705–1723
  6. Remold-O'Donnell E, Zimmerman C, Kenney D, et al. Expression on blood cells of sialophorin, the surface glycoprotein that is defective in Wiskott-Aldrich syndrome. Blood. 1987;70:104–109
  7. Silverman LB, Wong RCK, Remold-O'Donnell E, et al. Mechanism of mononuclear cell activation by an anti-CD43 (sialophorin) agonistic antibody. J Immunol. 1989;142:4194–4200
  8. Shelley CS, Remold-O'Donnell E, Davis AE, et al. Molecular characterization of sialophorin (CD 43), the lymphocyte surface sialoglycoprotein defective in Wiskott-Aldrich syndrome. Proc Natl Acad Sci USA. 1989;86:2819–2823
  9. Bach FH, Albertini RJ, Anderson JL, Joo P, Bortin MM. Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet. 1968;2:1364–1366
  10. Parkman R, Rappeport J, Geha R, et al. Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone marrow transplantation. N Engl J Med. 1978;298:921–927
  11. Kapoor N, Kirkpatrick D, Blaese RM, et al. Reconstitution of normal megakaryocytopoiesis and immunologic functions in Wiskott-Aldrich syndrome by marrow transplantation following myeloablation and immunosuppression with busulfan and cyclophosphamide. Blood. 1981;57:692–696
  12. Meuwissen HJ, Bortin MM, Bach FH, et al. Long-term survival after bone marrow transplantation: a fifteen-year follow-up report of a patient with Wiskott-Aldrich syndrome. J Pediatr. 1984;105:365–369
  13. Watson RD, Gershwin ME, Smithwick E, Castles JJ, Rurbner G. Cutaneous T-cell lymphoma and leukocytoclastic vasculitis in a long-term survivor of Wiskott-Aldrich syndrome. Ann Allergy. 1985;55:654–657Watson RD, Gershwin ME, Smithwick E, Castles JJ, Rurbner G. Cutaneous T-cell lymphoma and leukocytoclastic vasculitis in a long-term survivor of Wiskott-Aldrich syndrome. Ann Allergy. 1985;55:703–705
  14. Dinsmore R, Reich LM, Kapoor N, et al. ABH incompatible bone marrow transplantation: removal of erythrocytes by starch sedimentation. Br J Haematol. 1983;54:441–449
  15. Reisner Y, Kapoor N, Kirkpatrick D, et al. Transplantation for severe combined immunodeficiency with HLA-A, B, D, Dr incompatible parental marrow fractionated by soybean agglutinin and sheep red blood cells. Blood. 1983;61:341–348
  16. 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;297:1311–1318
  17. Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18:295–304
  18. Slavin RE, Woodruff JM. The pathology of bone marrow transplantation. Pathol Annu. 1974;9:291–344
  19. Shulman HM, Sullivan KM, Weiden PL, et al. Chronic graftversus-host syndrome in man: a long-term clinicopathologic study of twenty Seattle patients. Am J Med. 1980;69:204–217
  20. Emanuel D, Cunningham I, Jules-Elysee K, et al. Cytomegalovirus pneumonia after bone marrow transplantation success-fully treated with the combination of ganciclovir and high-dose intravenous immune globulin. Ann Intern Med. 1988;109:777–782
  21. Novogrodsky A, Katachalski E. Induction of lymphocyte transformation by sequential treatment with neuraminidase and galactose oxidase. Proc Natl Acad Sci USA. 1973;70:1824–1827
  22. Greer WL, Higgins E, Sutherland DR, et al. Altered expression of leukocyte sialoglycoprotein in Wiskott-Aldrich syndrome is associated with a specific defect in O-glycosylation. Biochem Cell Biol. 1989;67:503–509
  23. Kernan NA, Byers V, Scannon PJ, et al. Treatment of steroidresistant acute graft-vs-host disease by in vivo administration of an anti-T-cell ricin A chain immunotoxin. JAMA. 1988;259:3154–3157
  24. Shapiro RS, Chauvenet A, McGuire W, et al. Treatment of B-cell lymphoproliferative disorders with interferon alfa and intravenous gamma globulin (letter). N Engl J Med. 1988;318:1334
  25. Shapiro RS, McClain K, Frizzera G, et al. Epstein-Barr virus-associated B-cell lymphoproliferative disorders following bone marrow transplantation. Blood. 1988;71:1234–1243
  26. Blanche S, Le Deist F, Veber F, et al. Treatment of severe Epstein-Barr virus-induced polyclonal B-lymphocyte proliferation by anti-B-cell monoclonal antibodies: two cases after HLA-mismatched bone marrow transplantation. Ann Intern Med. 1988;108:199–203
  27. Fischer A, Griscelli C, Friedrich W, et al. Bone-marrow transplantation for immunodeficiencies and osteopetrosis: European survey, 1968–1985. Lancet. 1986;2:1080–1084
  28. Rimm IJ, Rappeport JM. Bone marrow transplantation for the Wiskott-Aldrich syndrome: long-term follow-up. Transplantation. 1990;50:617–620
  29. Lum LG, Tubergen DG, Corash L, Blaese RM. Splenectomy in the management of the thrombocytopenia of the Wiskott-Aldrich syndrome. N Engl J Med. 1980;302:892–896
  30. O'Reilly RJ, Keever CA, Small TN, Brochstein J. The use of HLA-non-identical T-cell-depleted marrow transplants for correction of severe combined immunodeficiency disease. Immunodefic Rev. 1989;1:273–309
  31. Gassmann W, Uharek L, Wottge H-U, Schmitz N, Loffler H, Mueller-Ruchholtz W. Comparison of cyclophosphamide, cytarabine, and etoposide as immunosuppressive agents before allogeneic bone marrow transplantation. Blood. 1988;72:1574–1579
  32. Frizzera G, Hanto DW, Gajl-Peczalska KJ, et al. Polymorphic diffuse B-cell hyperplasias and lymphomas in renal transplant recipients. Cancer Res. 1981;41:4262–4279
  33. Skinner JC, Gilbert EF, Hong R, et al. B cell lymphoproliferative disorders following T cell depleted allogeneic, bone marrow transplantation. Am J Pediatr Hematol Oncol. 1988;10:112–119
  34. Martin PJ, Shulman HM, Schubach WH, et al. Fatal Epstein-Barr virus-associated proliferation of donor B cells after treatment of acute graft-versus-host disease with a murine anti-T-cell antibody. Ann Intern Med. 1984;101:310–315
  35. Rumelhart SL, Trigg ME, Horowitz SD, Hong R. Monoclonal antibody T-cell-depleted HLA-haploidentical bone marrow transplantation for Wiskott-Aldrich syndrome. Blood. 1990;75:1031–1035
  36. Fischer A, Friedrich W, Fasth A, et al. Reduction of graft failure by a monoclonal antibody (anti-LFA-1 CD11a) after HLA nonidentical bone marrow transplantation in children with immunodeficiencies, osteopetrosis and Fanconi's anemia: a European Group for Immunodeficiency/European Group for Bone Marrow Transplantation report. Blood. 1991;77:249–256
  37. Ash RC, Casper JT, Chitambar CR, et al. Successful allogeneic transplantation of T-cell-depleted bone marrow from closely HLA-matched unrelated donors. N Engl J Med. 1990;322:485–494

1 Supported in part by a grant (CA 33050) from the National Institutes of Health, the Charles A. Dana Foundation, the Zelda Weintraub Cancer Foundation, the Robert J. Kleberg and Helen C. Kleberg Foundation, the Vincent Astor Foundation, and the Frito-Lay/New York Yankees Challenge Fund.

PII: S0022-3476(05)83041-0

The Journal of Pediatrics
Volume 119, Issue 6 , Pages 907-912 , December 1991