Unrelated cord blood transplantation for severe combined immunodeficiency and other primary immunodeficiencies

Bone Marrow Transplantation

Volumn 41, Issue 7, 2008, Pages 627-633

  Diaz de Heredia C ^a   Ortega, J J ^a   Diaz M A ^b   Olive T ^a   Badell, I ^c   Gonzalez Vicent M ^b   Sanchez de Toledo J ^a  

^a HOSPITAL UNIVERSITARI VALL D HEBRON   (Spain)

^b HOSPITAL INFANTIL UNIVERSITARIO NIÑO JESÚS   (Spain)

^c HOSPITAL SANT PAU   (Spain)

Author keywords

[No Author keywords available]

Indexed keywords

ACICLOVIR; BUSULFAN; COTRIMOXAZOLE; CYCLOPHOSPHAMIDE; CYCLOSPORIN; CYTARABINE; ETOPOSIDE; FLUCONAZOLE; FLUDARABINE; IMMUNOGLOBULIN; MELPHALAN; METHOTREXATE; METHYLPREDNISOLONE; STEROID; THYMOCYTE ANTIBODY;

ACUTE GRAFT VERSUS HOST DISEASE; AGE DISTRIBUTION; ARTICLE; AUTOIMMUNE HEMOLYTIC ANEMIA; BODY WEIGHT; CHRONIC GRAFT VERSUS HOST DISEASE; CLINICAL ARTICLE; CONTROLLED STUDY; CORD BLOOD STEM CELL TRANSPLANTATION; DRUG RESPONSE; FEMALE; HLA MATCHING; HUMAN; INFANT; INTERSTITIAL LUNG DISEASE; MALE; MORTALITY; MYCOSIS; NEUTROPHIL COUNT; OMENN SYNDROME; PRESCHOOL CHILD; PRIORITY JOURNAL; SEVERE COMBINED IMMUNODEFICIENCY; SINGLE DRUG DOSE; SURVIVAL RATE; SURVIVAL TIME; VALIDATION PROCESS; VIRUS INFECTION; WISKOTT ALDRICH SYNDROME; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

CHILD, PRESCHOOL; COHORT STUDIES; CORD BLOOD STEM CELL TRANSPLANTATION; FEMALE; HUMANS; INFANT; MALE; RETROSPECTIVE STUDIES; SEVERE COMBINED IMMUNODEFICIENCY; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; TRANSPLANTATION, HOMOLOGOUS; WISKOTT-ALDRICH SYNDROME;

EID: 42149141140     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1705946     Document Type: Article

References (44)

1. Taylor N, Weinberg K. Congenital defects of T lymphocyte function. Intern J Ped Hematol Oncol 1996; 3: 415-424.
2. Cavazzana-Calvo M, Lagresle C, Hacein-Bey AS, Fisher A. Gene therapy for severe combined immunodeficiency. Annu Rev Med 2005; 56: 585-602.
3. Gaspar HB, Parsley K, Howe S, King P, Kimberley CG, Sinclair J et al Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gamma retroviral vector. Lancet 2004; 364: 2181-2187.
4. Fisher A, Landais P, Friedrich W, Morgan G, Gerritsen B, Fasth A et al. European experience of bone marrow transplantation for severe combined immunodeficiency. Lancet 1990; 336: 850-854.
5. Antoine C, Muller S, Cant A, Cavazzano-Calvo M, Veys P, Vosen J et al Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: Report of the European experience 1968-1999. Lancet 2003; 361: 553-560.
6. Buckley RH, Schiff SE, Schiff RI, Market ML, Williams LW, Roberts JL et al. Hematopoietic stem cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999; 340: 508-516.
7. Fisher A, Landais P, Friedrich W, Geritsen B, Fasth A, Porta F et al Bone marrow transplantation in Europe for primary immunodeficiencies other than severe combined immunodeficiency: A report from the European Group for BMT and the European Group for immunodeficiency. Blood 1994; 83: 1149-1154.
8. Ozsahin H, Le Deist F, Benkerrou M, Cavazzama-Calvo M, Gomez L, Griscelli C et al. Bone marrow transplantation in 26 patients with Wiskott-Aldrich syndrome from a single center. J Pediatr 1996; 129: 238-244.
9. Gatti RA, Meewissen HJ, Allen HD, Hong R, Good R. Immunological reconstitution of sex-linked lymphogenic immunological deficiency. Lancet 1968; 2: 1366-1369.
10. Parkman R, Rappeport J, Geha R, Belli J, Cassady R, Levey R et al. Complete correction of the Wiskott-Aldrich syndrome by allogeneic bone marrow transplantation. N. Engl J Med 1978; 298: 921-927.
11. Reisner Y, Kapoor N, Kirpatrick D, Pollack MS, Cunningham-Rudies D, Dupont B et al. Transplantation for severe combined immunodeficiency with HL-A, B, DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells. Blood 1983; 61: 341-348.
12. Myers LA, Patel DD, Puck JM, Buckley RH. Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 2002; 99: 872-878.
13. Haddad E, Landais P, Friedrich N, Gerritsen B, Cavazzana-Calvo M, Morgan G et al. Long-term immune reconstitution and outcome after HLA non-identical T-cell depleted bone marrow transplantation for severe combined immunodeficiency: A European retrospective study of 116 patients. Blood 1998, 91: 3646-3653.
14. Filipovich AH, Shapiro RS, Ramsay NKC, Kim T, Blazar B, Kersey J et al: Unrelated donor bone marrow transplantation for correction of lethal congenital immunodeficiencies. Blood 1992; 80: 270-276.
15. Dalal J, Reid B, Doyle J, Freedman M, Calderword S, Saunders F et al Matched unrelated bone marrow transplantation for combined immunodeficiency. Bone Marrow Transplant 2005; 25: 613-626.
16. Filipovich AH, Stone JV, Tomany SC, Ireland M, Kollman C, Pelz CJ et al. Impact of donor-type on outcome of bone marrow transplantation for Wiskott-Aldrich syndrome: Collaborative study of the International Bone Marrow Transplant Registry and the National Marrow Donor Program. Blood 2001; 97: 1598-1603.
17. Wagner JE, Rosenthal J, Sweetman R, Shu XO, Davies S, Ramsay NKC et al. Successful transplantation of HLA-matched and HLA-mismatched umbilical cord blood from unrelated donor; analysis of engraftment and acute graft-versus host disease. Blood 1996; 88: 795-802.
18. Gluckman E, Rocha V, Boyer-Chammard A, Locatelli F, Arcese W, Pasquini R et al. Outcome of cord blood transplantation from related and unrelated donors. N Eng J Med 1997; 337: 373-382.
19. Barker JN, Davies SM, de For T, Ramsay NKC, Weisdof DJ, Wagner JE. Survival after transplantation of unrelated umbilical cord blood is comparable to that of leukocyte antigen-matched unrelated donor bone marrow: Results of a matched-pair analysis. Blood 2001; 97: 2957-2961.
20. Rocha V, Cornish J, Sievers EL, Filipovich A, Locatelli F, Peters C et al. Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood 2001; 97: 2962-2971.
21. Ziegner UHM, Ochs HD, Schanen C, Feig SA, Seyama K, Futatoni T et al Unrelated umbilical cord stem cell transplantation for X-linked immunodeficiencies. J Pediatr 2001; 138: 570-573.
22. Benito A, Diaz MA, Alonso F, Fontan G, Madero L. Successful unrelated umbilical cord blood transplantation in a child with Omenn's syndrome. Pediatr Hematol Oncol 1999; 16: 361-366.
23. Fagioli F, Biaxin E, Berger M, Nesi F, Saroglia EH, Minero R et al Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency. Bone Marrow Transplant 2003; 31: 133-136.
24. Knutsen AP, Steffen M, Wassmer K, Wall DA. Umbilical cord blood transplantation in Wiskott-Aldrich syndrome. J Pediatr 2003; 142: 519-523.
25. Vowels MR, Reg LPT, Berdukas V, Ford D, Thierry D, Purtilo D et al Correction of X-linked lymphoproliferative disease by transplantation of cord-blood stem cells. N Engl J Med 1993; 329: 1623-1625.
26. Mino E, Kobayashi R, Yoshida M, Suzuki Y, Yamada M, Kobayashi K. Umbilical cord blood stem cell transplantation from unrelated HLA-matched donor in an infant with severe congenital neutropenia. Bone Marrow Transplant 2004; 33: 969-971.
27. Schwinger N, Urban CH, Lackner CH, Benesch M, Kerbl R, Dornbusch HJ et al. Unrelated 5/6-locus matched umbilical cord blood transplantation in a 23-month-old child with hemophagocytic lymphohistiocytosis. Bone Marrow Transplant 1998; 22: 393-396.
28. Ortega JJ, Yaniv I, Rocha V, Garnier F, Ebell W, Souillet G, et al. on behalf of Eurocord Transplant Group. Cord blood transplantation for inborn errors. Blood 1998; 10 (Suppl 1): 92 (abstract no. 291).
29. Ortega JJ, Rocha V, Wall D, Filipovich A, Porcher R, Pasquini R et al Unrelated cord blood transplant in children with severe primary immunodeficiencies. A Eurocord analysis. Blood 2001, 11 (Suppl 1): 98 (abstract no. 667).
30. Knutsen AP, Wall DA. Umbilical cord blood transplantation in severe T cell immunodeficiency disorders: Two-year experience. J Clin Immunol 2000; 20: 466-476.
31. Tsuji Y, Imai K, Kajiwara A, Aoki Y, Isoda T, Tomizawa D et al. Hematopoietic stem cell transplantation for 30 patients with primary immunodeficiency diseases: 20 years' experience of a single team. Bone Marrow Transplant 2006; 37: 469-477.
32. Bhattacharya A, Slatter MA, Chapman CE, Barge D, Jackson A, Flood TJ et al. Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplant 2005; 36: 295-299.
33. Comans-Bitter WM, de Groot R, van den BR. Immunophenotyping of blood lymphocytes in childhood: Reference values for lymphocyte subpopulations. J Pediatr 1997; 130: 388-393.
34. Thomson BG, Robertson KA, Gowan D, Heilman D, Broxmeyer HE, Emmanuel D et al. Analysis of engraftment, graft-versus-host disease and immune recovery following unrelated cord blood transplantation. Blood 2000; 96: 2703-2711.
35. Giraud P, Thuret I, Revison D, Chambost H, Brunet C, Novakovitch G et al. Immune reconstitution and outcome after unrelated cord blood transplantation: A single paediatric institution experience. Bone Marrow Transplant 2000; 25: 53-57.
36. Niehues T, Rocha V, Filipovich AH, Chan KV, Porcher R, Michel G et al Factors affecting lymphocyte subset reconstitution after either related or unrelated cord blood transplantation in children - a Eurocord analysis. Br J Hematol 2001; 114: 42-48.
37. Moretta A, Maccario R, Fagioli F, Giraldi E, Busca A, Montagna D et al. Analysis of immune reconstitution in children undergoing cord blood transplantation. Exp Hematol 2001; 29: 371-379.
38. Lankester AC, Visser LFA, Hartwig NG, Bredius RGM, Gaspar HB, van der Burg M et al. Allogeneic lymphoproliferative disease: Two cases in one family and review of the literature. Bone Marrow Transplant 2005; 36: 99-106.
39. Loechelt BJ, Shapiro RS, Jyonouchi H, Filipovich AH. Mismatched bone marrow transplantation for Omenn syndrome a variant of severe combined immunodeficiency. Bone Marrow Transplant 1995; 16: 381-385.
40. Rossi G, Zecca M, Giorgiani G, Bonetti F, De Stefano P, Locatelli F. Non-myeloablative stem cell transplantation for severe combined immunodeficiency-Omenn syndrome. Br J Haematol 2004; 125: 405-417.
41. Rao K, Amnolia PJ, Jones A, Cale CM, Naik P, King D et al. Improved survival after unrelated bone marrow transplantation in children with primary immunodeficiency using a reduced-intensity conditioning regimen. Blood 2005; 105: 879-885.
42. Veys P, Rao K, Amrolia P. Stem cell transplantation for congenital immunodeficiencies using reduced-intensity conditioning. Bone Marrow Transplant 2005; 35: S45-S47.
43. Toren A, Nagler A, Amariglio N, Neumann Y, Golan H, Bilori B et al Successful human umbilical cord blood stem cell transplantation without conditioning in severe combined immunodeficiency, Bone Marrow Transplant 1999; 23: 405-408.
44. Barker JN, Scaradavou A, Stevens CE, Rubinstein P. Analysis of 608 umbilical cord blood transplants: HLA is a critical determinant of transplant-related mortality in the post-engraftment period even in the absence of acute graft-vs-host disease. Blood 2005; 11 (Suppl 1): 102 (abstract no. 92).