Long-term chimerism and B-cell function after bone marrow transplantation in patients with severe combined immunodeficiency with B cells: A single-center study of 22 patients

Blood

Volumn 94, Issue 8, 1999, Pages 2923-2930

  Haddad, Elie ^a   Le Deist, Françoise ^b   Aucouturier, Pierre ^b   Cavazzana Calvo, Marina ^b   Blanche, Stephane ^b   De Saint Basile, Geneviève ^b   Fischer, Alain ^b  

^a HÔPITAL NECKER ENFANTS MALADES   (France)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

CD27 ANTIGEN; IMMUNOGLOBULIN ALLOTYPE;

ALLOTYPE; ARTICLE; B LYMPHOCYTE; BONE MARROW TRANSPLANTATION; CELL FUNCTION; CHIMERA; CONTROLLED STUDY; HUMAN; HUMAN CELL; IMMUNE DEFICIENCY; MONOCYTE; PRIORITY JOURNAL;

EID: 0032833976     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v94.8.2923.420k44_2923_2930     Document Type: Article

References (55)

1. Fischer A, Cavazzana-Calvo M, De Saint Basile G, De Villartay JP, Di Santo JP, Hivroz C, Rieux-Laucat F, Le Deist F: Naturally occurring primary deficiencies of the immune system. Annu Rev Immunol 15:93, 1997
2. Dooren LJ, Vossen JM: Severe combined immunodeficiency: Reconstitution of the immune system following bone marrow transplantation, in van Bekkum DW, Löwenberg B (eds): Bone Marrow Transplantation. Biological Mechanisms and Clinical Practice. New York, NY, Dekker, 1985, p 351
3. Primary Immunodeficiency Diseases: Report of a WHO Scientific Group. Clin Exp Immunol 109:1, 1997 (suppl 1)
4. Rosen FS, Cooper MD, Wedgewood RJP: The primary immunodeficiencies. N Engl J Med 333:431, 1995
5. Nogushi M, Yi H, Rosenblatt HM, Filipovich AH, Adelstein S, Modi WS, McBride OW, Leonard WJ: Interleukin-2 receptor y chain mutation results in X-linked severe combined immunodeficiency in humans. Cell 73:147, 1993
6. Puck JM, Deschenes SM, Porter JC, Dutra AS, Brown CJ, Willard HF, Henthorn PS: The interleukin-2 receptor gamma chain maps to Xq13.1 and is mutated in X-linked severe combined immunodeficiency, SCIDX1. Hum Mol Genet 2:1099, 1993
7. Macchi P, Villa A, Giliani S, Sacco MG, Frattini A, Porta F, Ugazio AG, Johnston JA, Candotti F, O'Shea JJ, Vezzoni P, Notarangelo LD: Mutations of JAK-3 gene in patients with autosomal recessive severe combined immunodeficiency. Nature 377:65, 1995
8. Russell SM, Tayebi N, Nakajima H, Riedy MC, Roberts JL, Aman MJ, Migone TS, Noguchi M, Markert ML, Buckley RH, O'Shea J, Leonard WJ: Mutation of Jak3 in a patient with SCID: Essential role of JAK3 in lymphoid development. Science 270:797, 1995
9. Puel A, Ziegler SF, Buckley RH, Leonard WJ: Defective IL7R expression in T(-)B(+)NK(+) severe combined immunodeficiency. Nat Genet 20:394, 1998
10. Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA: Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 2:1366, 1968
11. Kenny AB, Hitzig WH: Bone marrow transplantation for severe combined immunodeficiency disease. Eur J Paediatr 131:155, 1979
12. O'Reilly RJ, Brochstein J, Dinsmore L, Kirkpatrick D: Marrow transplantation for congenital disorders. Semin Hematol 21:188, 1984
13. Fischer A, Friedrich W, Levinsky R, Vossen J, Griscelli C, Kubanek B, Morgan G, Wagemaker G, Landais P: Bone marrow transplantation for immunodeficiency and osteopetrosis. European survey 1968-1985. Lancet 1:1080, 1986
14. Buckley RH, Schiff SE, Schiff RI, Markert ML, Williams LW, Roberts JL, Myers LA, Ward FE: Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 340:508, 1999
15. Reisner Y, Kapoor N, Kirkpatrick D, Pollack MS, Cunningham-Rundles S, Dupont B, Hodes MZ, Good RA, O'Reilly RJ: Transplantation for severe combined immunodeficiency with HLA A, B, D, DR incompatible paternal marrow fractionated by soybean agglutinin and sheep red blood cells. Blood 61:341, 1983
16. Friedrich W, Goldmann SF, Ebell W, Blutters-Sawatzki R, Gaedicke G, Raghavachar A, Peter HH, Belohradsky B, Kreth W, Kubanek B, Kleihauer E: Severe combined immunodeficiency: Treatment by bone marrow transplantation in 15 infants using HLA-haploidentical donors. Eur J Pediatr 144:125, 1985
17. Buckley RH, Schiff SE, Sampson HA, Schiff RI, Markert ML, Knutsen AP, Hershfield MS, Huang AT, Mickey GH, Ward FE: Development of immunity in human severe primary T-cell deficiency following haploidentical bone marrow stem cell transplantation. J Immunol 136:2398, 1986
18. Fischer A, Durandy A, de Villartay JP, Vilmer E, Le Deist F, Gerota I, Griscelli C: HLA-haploidentical bone marrow transplantation for severe combined immunodeficiency using E rosette fractionation and cyclosporin. Blood 67:444, 1986
19. 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 1:273, 1989
20. Fischer A, Landais P, Friedrich W, Morgan G, Gerritsen B, Fasth A, Porta F, Griscelli C, Goldman SF, Levinsky R, Vossen J: European experience of bone marrow transplantation for severe combined immunodeficiency. Lancet 2:850, 1990
21. Buckley RH, Schiff SE, Schiff RI, Roberts JL, Markert ML, Peters W, Williams LW, Ward FE: Haploidentical bone marrow stem cell transplantation in human severe combined immunodeficiency. Semin Hematol 30:92, 1993 (suppl 4)
22. Parkman R: The biology of bone marrow transplantation for severe combined immune deficiency. Adv Immunol 49:381, 1991
23. van Leeuwen JEM, van Tol MJD, Joosten AM, Schellekens PTA, Langlois van den Bergh R, Waaijer JLM, Oudeman-Gruber NJ, van der Weijden-Ragas CPM, Roos MTL, Gerritsen EJA, van den Berg H, Haraldsson A, Meera Khan P, Vossen JM: Relationship between patterns of engraftmenl in peripheral blood and immune reconstitution after allogenic bone marrow transplantation for (severe) combined immunodeficiency. Blood 84:3936, 1994
24. Bertrand Y, Landais P, Friedrich W, Gerritsen B, Morgan G, Fasth A, Cavazzana-Calvo M, Porta F, Cant A, Espanol T, Müller S, Veys P, Vossen J, Haddad E, Fischer A: Influence of SCID phenotype on the outcome of HLA non identical T-cell depleted bone marrow transplantation. A retrospective European survey from the European group for BMT (EBMT) and the European Society for Immunodeficiency (ESID). J Pediatr 136:740, 1999
25. Haddad E, Landais P, Friedrich W, Gerritsen B, Cavazzana-Calvo M, Morgan G, Bertrand Y, Fasth A, Porta F, Cant A, Espanol T, Muller S, Veys P, Vossen J, Fischer A: 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 91:2646, 1998
26. Dror Y, Gallagher R, Wara DW, Colombe BW, Merino A, Benkerrou M, Cowan MJ: Immune reconstitution in severe combined immunodeficiency disease after Lectin-treated, T-cell-depleted haplocompatible bone marrow transplantation. Blood 81:2021, 1993
27. Wijnaendts L, Le Deist F, Griscelli C, Fischer A: Development of immunologic functions after bone marrow transplantation in 33 patients with severe combined immunodeficiency. Blood 74:2212, 1989
28. Buckley RH: Bone marrow reconstitution in primary immunodeficiency, in Rich RR (ed): Clinical Immunology. St Louis, MO, Mosby, 1996, p 1813
29. O'Reilly RJ, Keever C, Kernan N, Flomenberg N, Dupont B, Collins N, Brochstein J, Kirkpatrik D, Kapoor N, Burns J, Reisner Y: Investigation of humoral immune deficiencies following T-cell depleted, HLA-haplotype mismatched parental marrow transplants for the treatment of severe combined immunodeficiency disease, in Eibl MM, Rosen FS (eds): Primary Immunodeficiency Diseases. Amsterdam, The Netherlands, Elsevier, 1986, p 301
30. Vossen JM: Bone marrow transplantation in the treatment of primary immunodeficiencies. Ann Clin Res 19:285, 1987
31. Himelstein BP, Puck JM, August C, Pierson G, Bunin N: Earlier bone marrow transplantation is better: Two siblings with X-linked severe combined immunodeficiency treated with the same T-cell depleted maternal marrow. J Pediatr 122:289, 1993
32. Matthews DJ, Clark PA, Herbert J, Morgan G, Armitage RJ, Kinnon C, Minty A, Grabstein KH, Caput D, Ferrara P, Callard R: Function of the interleukin-2 (IL-2) receptor γ-chain in biologic responses of X-linked severe combined immunodeficient B cells to IL-2, IL-4, IL-13, and IL-15. Blood 85:38, 1995
33. Izuhara K, Heike T, Otsuka T, Yamaoka K, Mayumi M, Imamura T, Niho Y, Harada N: Signal transduction pathway of interleukin-4 and interleukin-13 in human B cells derived from X-linked severe combined immunodeficiency patients. J Biol Chem 271:619, 1996
34. Morelon E, Dautry-Varsat A, Le Deist F, Hacein-Bey S, Fischer A, De Saint Basile G: T lymphocyte differentiation and proliferation in the absence of the cytoplasmic tail of the common receptor γ chain in a SCID-Xl patient. Blood 88:1708, 1995
35. Fischer A, Friedrich W, Fasth A, Blanche S, Le Deist F, Girault D, Veber F, Vossen J, Lopez M, Griscelli C, Hirn M: 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. Blood 77:249, 1991
36. Hale G, Cobbold S, Waldmann H: T cell depletion with Campath-1 in allogenic bone marrow transplantation. Transplantation 45:753, 1988
37. Przepiorka D, Weisdorf D, Martin P, Klingemann HG, Beatly P, Hows J, Thomas ED: Meeting report: Consensus conference on acute GVHD grading. Bone Marrow Transplant 15:825, 1995
38. Dib C, Fauré S, Fizames C, Samson D, Drouot N, Vignal A, Millasseau P, Marc S, Hazan J, Seboun E, Lathrop M, Gyapay G, Morissette J, Weissenbach J: A comprehensive map of the human genome based on 5,264 microsatellites. Nature 380:152, 1996
39. Allen RC, Belmont JW: Trinucleotide repeat polymorphism at DXS101. Hum Mol Genet 2:1508, 1993
40. Hearn CM, Todd JA: Tetranucleotide repeat polymorphism at the HPRT locus. Nucleic Acids Res 19:5450, 1991
41. Berthet F, Le Deist F, Duliege AM, Griscelli C, Fischer A: Clinical consequences and treatment of primary immunodeficiency syndromes characterized by functional T and B lymphocyte anomalies (combined immune deficiency). Pediatrics 93:265, 1994
42. Klein U, Rajewsky K, Kuppers R: Human immunoglobulin (Ig)M+IgD+ peripheral blood B cells expressing the CD27 cell surface antigen carry somatically mutated variable region genes: CD27 as a general marker for somatically mutated (memory) B cells. J Exp Med 188:1679, 1998
43. Friedrich W, Knobloch C, Greher J, Hartmann W, Peter HH, Goldmann SF, Kleihauer E: Bone marrow transplantation in severe combined immunodeficiency: Potential and current limitations. Immunodef Rev 4:315, 1993
44. Moen RC, Horwitz S, Sondel PM, Borcherding WR, Trigg ME, Billing R, Hong R: Immunologic reconstitution after haploidentical bone marrow transplantation for immune deficiency disorders: Treatment of bone marrow cells with monoclonal antibody CT-2 and complement. Blood 70:664, 1987
45. Storek J, Witherspoon RP, Storb R: T cell reconstitution after bone marrow transplantation into adult patients does not resemble T cell development in early life. Bone Marrow Transplant 16:413, 1995
46. Weinberg K, Annet G, Kashyap A, Lenarsky C, Forman SJ, Parkman R: The effect of thymic function on immunocompetence following bone marrow transplantation. Biol Blood Bone Marrow Transplant 1:18, 1995
47. Felsburg PJ, Somberg RL, Hartnett BJ, Suter SF, Henthom PS, Moore PF, Weinberg KI, Ochs HD: Full immunological reconstitution following nonconditioned bone marrow transplantation for canine X-linked severe combined immunodeficiency. Blood 90:3214, 1997
48. Chu E, Umetsu D, Rosen F, Geha RS: Major histocompatibility restriction of antigen recognition by T cells in a recipient of haplotype mismatched human bone marrow transplantation. J Clin Invest 72:1124, 1983
49. Roncarolo MG, Touraine JL, Banchereau J: Cooperation between major histocompatibility complex mismatched mononuclear cells from a human chimera in the production of antigen-specific antibody. J Clin Invest 77:673, 1986
50. Roncarolo MG, Yssel H, Touraine JL, Bachetta R, Gebuhrer L, de Vries JE, Spits H: Antigen recognition by MHC-incompatible cells of a human mismatched chimera. J Exp Med 158:2139, 1988
51. Roberts JL, Volkman DJ, Buckley RH: Modified MHC restriction of donor-origin T cells in humans with severe combined immunodeficiency transplanted with haploidentical bone marrow stem cells. J Immunol 143:1575, 1989
52. Geha RS, Rosen FS: The evolution of MHC restrictions in antigen recognition by T cells in a haploidentical bone marrow transplant recipient. J Immunol 143:84, 1989
53. Gougeon ML, Drean G, Le Deist F, Dousseau M, Fevrier M, Diu A, Theze J, Griscelli C, Fischer A: Human severe combined immunodeficiency disease. Phenotypical and functional characteristics of peripheral B lymphocytes. J Immunol 145:2873, 1990
54. Conley ME, Lavoie A, Briggs C, Brown P, Guerra C, Puck JM: Nonrandom X chromosome inactivation in B cells from carriers of X chromosome-linked severe combined immunodeficiency. Proc Natl Acad Sci USA 85:3090, 1988
55. Mebius RE, Rennert P, Weissman IL: Developing lymph nodes collect CD4+CD3- Ltbeta+ cells that can differentiate to APC, NK cells, and follicular cells but not T or B cells. Immunity 7:493, 1997