Successful correction of severe combined immune deficiency following transplantation with bone marrow from HLA-matched unrelated donors

Italian Journal of Pediatrics

Volumn 33, Issue 1, 2007, Pages 24-26

  Grunebaum, E ^a   Notarangelo, L D ^b   Roifman, Chaim M ^a,c  

^a HOSPITAL FOR SICK CHILDREN   (Canada)

^b UNIVERSITY OF BRESCIA   (Italy)

^c Canadian Centre for Primary Immunodeficiency   (Canada)

Author keywords

Bone marrow transplantation; HLA donor; Severe combined immunedeficiency

Indexed keywords

BONE MARROW TRANSPLANTATION; COMBINED IMMUNODEFICIENCY; DONOR; HLA SYSTEM; HUMAN; SHORT SURVEY; TREATMENT OUTCOME;

EID: 34547138926     PISSN: 17208424     EISSN: 18247288     Source Type: Journal    
DOI: None     Document Type: Short Survey

References (22)

1. Huang H, Manton KG. Newborn screening for severe combined immunodeficiency (SCID): a review. Front Biosci 2005;10:1024-39.
2. Fischer 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;336:850-4.
3. Haddad E, Landais P, Friedrich W, Gerritsen B, Cavazzana-Calvo M, Morgan G, et al. Long-term immune reconstitution and outcome after HLA-nonidentical T-cell-depleted bone marrow transplantation for severe combined immunodeficiency: a European retrospective study of 116 patients. Blood 1998;91:3646-53.
4. Cavazzana-Calvo M, Andre-Schmutz I, Hacein-Bey-Abina S, Bensoussan D, Le Deist F, Fischer A. Improving immune reconstitution while preventing graft-versus-host disease in allogeneic stem cell transplantation. Semin Hematol 2002;39:32-40.
5. Amrolia PJ, Muccioli-Casadei G, Yvon E, Huls H, Sili U, Wieder ED, et al. Selective depletion of donor alloreactive T cells without loss of antiviral or antileukemic responses. Blood 2003;102:2292-9.
6. Ball LM, Lankester AC, Bredius RG, Fibbe WE, van Tol MJ, Egeler RM. Graft dysfunction and delayed immune reconstitution following haploidentical peripheral blood hematopoietic stem cell transplantation. Bone Marrow Transplant 2005;35(Suppl 1):S35-8.
7. Patel DD, Gooding ME, Parrott RE, Curtis KM, Haynes BF, Buckley RH. Thymic function after hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 2000;342:1325-32.
8. Fischer A, Le Deist F, Hacein-Bey-Abina S, Andre-Schmutz I, Basile Gde S, de Villartay JP. Severe combined immunodeficiency. A model disease for molecular immunology and therapy. Immunol Rev 2005;203:98-109.
9. Slatter MA, Bhattacharya A, Flood TJ, Spickett GP, Cant AJ, Abinun M, et al. Polysaccharide antibody responses are impaired post bone marrow transplantation for severe combined immunodeficiency, but not other primary immunodeficiencies. Bone Marrow Transplant 2003;32:225-9.
10. Veys P, Amrolia P, Rao K. The role of haploidentical stem cell transplantation in the management of children with haematological disorders. Br J Haematol 2003;123:193-206.
11. Antoine C, Muller S, Cant A, Cavazzana-Calvo M, Veys P, Vossen J, et al. Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet 2003;361:553-60.
12. Buckley RH, Schiff SE, Schiff RI, Markert L, Williams LW, Roberts JL, et al. Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999;340:508-6.
13. Smogorzewska EM, Brooks J, Annett G, Kapoor N, Crooks GM, Kohn DB, et al. T cell depleted haploidentical bone marrow transplantation for the treatment of children with severe combined immunodeficiency. Arch Immunol Ther Exp (Warsz) 2000;48:111-8.
14. O'Reilly RJ, Dupont B, Pahwa S, Grimes E, Smithwick EM, Pahwa R, et al. Reconstitution in severe combined immunodeficiency by transplantation of marrow from an unrelated donor. N Engl J Med 1977;297:1311-8.
15. Anasetti C, Petersdorf EW, Martin PJ, Woolfrey A, Hansen JA. Trends in transplantation of hematopoietic stem cells from unrelated donors. Curr Opin Hematol 2001;8:337-41.
16. Caillat-Zucman S, Le Deist F, Haddad E, Gannage M, Dal Cortivo L, Jabado N, et al. Impact of HLA matching on outcome of hematopoietic stem cell transplantation in children with inherited diseases: a single-center comparative analysis of genoidentical, haploidentical or unrelated donors. Bone Marrow Transplant 2004;33:1089-95.
17. Dalal I, Reid B, Doyle J, Freedman M, Calderwood S, Saunders F, et al. Matched unrelated bone marrow transplantation for combined immunodeficiency. Bone Marrow Transplant 2000;25:613-21.
18. Filipovich AH, Shapiro RS, Ramsay NK, Kim T, Blazar B, Kersey J, et al. Unrelated donor bone marrow transplantation for correction of lethal congenital immunodeficiencies. Blood 1992;80:270-6.
19. Rao K, Amrolia PJ, Jones A, Cale CM, Naik P, King D, et al. Improved survival after unrelated donor bone marrow transplant in children with primary immunodeficiency using a reduced intensity conditioning regimen. Blood 2005;105:879-85.
20. Grunebaum E, Mazzolari E, DalleraD, Atkinson, A, Redi B, Notarangelo LD, et al. Bne marrow transplantation for severe combined immune deficiency. JAMA 2006;295:508-18.
21. Tsuji Y, Imai K, Kajiwara M, 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;37:469-77.
22. Borghans JA, Bredius RG, Hazenberg MD, Roelofs H, Jol-van der Zijde EC, Heidt J, et al. Early determinants of long-term T-cell reconstitution after hematopoietic stem cell transplantation for severe combined immunodeficiency. Blood 2006;108:763-9.