Skip to main content
SpringerLink
Log in

Cord Blood Transplantation for Pediatric Non-Malignant Conditions

  • Chapter
  • First Online:
Frontiers of Cord Blood Science

  • 525 Accesses

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Log in via an institution
eBook
USD 16.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 119.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 169.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

References

  1. Kurtzberg J, Cord blood transplantation in genetic disorders. Biol Blood Marrow Transplant 2004; 10(10):735–736.

    Google Scholar 

  2. Gatti RA, Meuwissen HJ, Allen HD et al., Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968; 2(7583):1366–1369.

    PubMed  CAS  Google Scholar 

  3. Speck B, Zwaan FE, van Rood JJ et al., Allogeneic bone marrow transplantation in a patient with aplastic anemia using a phenotypically HLA-identical unrelated donor. Transplantation 1973; 16(1):24–28.

    PubMed  CAS  Google Scholar 

  4. Reisner Y, Kapoor N, Kirkpatrick D et al., Transplantation for severe combined immunodeficiency with HLA-A,B,D,DR incompatible parental marrow cells fractionated by soybean agglutinin and sheep red blood cells. Blood 1983; 61(2):341–348.

    PubMed  CAS  Google Scholar 

  5. Hobbs JR, Hugh-Jones K, Barrett AJ et al., Reversal of clinical features of Hurler’s disease and biochemical improvement after treatment by bone-marrow transplantation. Lancet 1981; 2(8249):709–712.

    PubMed  CAS  Google Scholar 

  6. Gluckman E, Broxmeyer HA, Auerbach AD et al., Hematopoietic reconstitution in a patient with Fanconi’s anemia by means of umbilical-cord blood from an HLA-identical sibling. N Engl J Med 1989; 321(17):1174–1178.

    PubMed  CAS  Google Scholar 

  7. Grewal SS, Kahn JP, Macmillan ML et al., Successful hematopoietic stem cell transplantation for Fanconi anemia from an unaffected HLA-genotype-identical sibling selected using preimplantation genetic diagnosis. Blood 2004; 103(3):1147–1151.

    PubMed  CAS  Google Scholar 

  8. Bielorai B, Hughes MR, Auerbach AD et al., Successful umbilical cord blood transplantation for Fanconi anemia using preimplantation genetic diagnosis for HLA-matched donor. Am J Hematol 2004; 77(4):397–399.

    PubMed  Google Scholar 

  9. Kurtzberg J, Laughlin M, Graham ML et al., Placental blood as a source of hematopoietic stem cells for transplantation into unrelated recipients. N Engl J Med 1996; 335(3):157–166.

    PubMed  CAS  Google Scholar 

  10. Wagner JE, Kernan NA, Steinbuch M et al., Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease. Lancet 1995; 346(8969):214–219.

    PubMed  CAS  Google Scholar 

  11. http://www.BMDW.org.(accessed 9/9/2006).

    Google Scholar 

  12. Wagner JE, Kahn JP, Wolf SM et al., Preimplantation testing to produce an HLA-matched donor infant. JAMA 2004; 292(7):803–804.

    PubMed  CAS  Google Scholar 

  13. Wolf SM, Kahn JP, Wagner JE, Using preimplantation genetic diagnosis to create a stem cell donor: issues, guidelines & limits. J Law Med Ethics 2003; 31(3):327–339.

    PubMed  Google Scholar 

  14. Spriggs M, Savulescu J, “Saviour siblings”. J Med Ethics 2002; 28(5):289.

    PubMed  CAS  Google Scholar 

  15. Pennings G, Schots R, Liebaers I, Ethical considerations on preimplantation genetic diagnosis for HLA typing to match a future child as a donor of haematopoietic stem cells to a sibling. Hum Reprod 2002; 17(3):534–538.

    PubMed  CAS  Google Scholar 

  16. Gaipa G, Dassi M, Perseghin P et al., Allogeneic bone marrow stem cell transplantation following CD34\(+\) immunomagnetic enrichment in patients with inherited metabolic storage diseases. Bone Marrow Transplant 2003; 31(10):857–860.

    PubMed  CAS  Google Scholar 

  17. Peters C, Shapiro EG, Anderson J et al., Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 1998; 91(7):2601–2608.

    PubMed  CAS  Google Scholar 

  18. Staba SL, Escolar ML, Poe M et al., Cord-blood transplants from unrelated donors in patients with Hurler’s syndrome. N Engl J Med 2004; 350(19):1960–1969.

    PubMed  CAS  Google Scholar 

  19. Yabe H, Inoue H, Matsumoto M et al., Unmanipulated HLA-haploidentical bone marrow transplantation for the treatment of fatal, nonmalignant diseases in children and adolescents. Int J Hematol 2004; 80(1):78–82.

    PubMed  Google Scholar 

  20. Wagner JE, 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(3):795–802.

    PubMed  CAS  Google Scholar 

  21. Rocha V, Cornish J, Sievers EL et al., Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood 2001; 97(10):2962–2971.

    PubMed  CAS  Google Scholar 

  22. Shaw PJ, Bleakley M, Lau L, Unrelated cord blood transplant as salvage following non-engraftment of unrelated marrow transplant? Bone Marrow Transplant 2004; 34(3): 275–276.

    PubMed  CAS  Google Scholar 

  23. Motwani J, Lawson SE, Darbyshire PJ, Successful HSCT using nonradiotherapy-based conditioning regimens and alternative donors in patients with Fanconi anaemia – experience in a single UK centre. Bone Marrow Transplant 2005; 36(5):405–410.

    PubMed  CAS  Google Scholar 

  24. Martin PL, Carter SL, Kernan NA et al., Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant 2006; 12(2):184–194.

    PubMed  Google Scholar 

  25. Gluckman E, Rocha V, Chastang C, Cord blood banking and transplant in Europe. Eurocord. Bone Marrow Transplant 1998; 22 Suppl 1:S68–S74.

    PubMed  Google Scholar 

  26. Fraser CJ, Hirsch BA, Dayton V et al., First report of donor cell-derived acute leukemia as a complication of umbilical cord blood transplantation. Blood 2005; 106(13):4377–4380.

    PubMed  CAS  Google Scholar 

  27. Sevilla J, Querol S, Molines A et al., Transient donor cell-derived myelodysplastic syndrome with monosomy 7 after unrelated cord blood transplantation. Eur J Haematol 2006; 77(3): 259–263.

    PubMed  Google Scholar 

  28. Gluckman E, Rocha V, Chevret S, Results of unrelated umbilical cord blood hematopoietic stem cell transplant. Transfus Clin Biol 2001; 8(3):146–154.

    PubMed  CAS  Google Scholar 

  29. Frassoni F, Podesta M, Maccario R et al., Cord blood transplantation provides better reconstitution of hematopoietic reservoir compared with bone marrow transplantation. Blood 2003; 102(3):1138–1141.

    PubMed  CAS  Google Scholar 

  30. Locatelli F, Maccario R, Comoli P et al., Hematopoietic and immune recovery after transplantation of cord blood progenitor cells in children. Bone Marrow Transplant 1996; 18(6): 1095–1101.

    PubMed  CAS  Google Scholar 

  31. Thomson BG, Robertson KA, Gowan D et al., Analysis of engraftment, graft-versus-host disease, and immune recovery following unrelated donor cord blood transplantation. Blood 2000; 96(8):2703–2711.

    PubMed  CAS  Google Scholar 

  32. Abu-Ghosh A, Goldman S, Slone V et al., Immunological reconstitution and correlation of circulating serum inflammatory mediators/cytokines with the incidence of acute graft-versus-host disease during the first 100 days following unrelated umbilical cord blood transplantation. Bone Marrow Transplant 1999; 24(5):535–544.

    PubMed  CAS  Google Scholar 

  33. Gluckman E, Rocha V, Boyer-Chammard A et al., Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group. N Engl J Med 1997; 337(6):373–381.

    PubMed  CAS  Google Scholar 

  34. Bradley MB, Satwani P, Baldinger L et al., Reduced intensity allogeneic umbilical cord blood transplantation in children and adolescent recipients with malignant and non-malignant diseases. Bone Marrow Transplant 2007; 40(7): 621–631.

    Google Scholar 

  35. Lucarelli G, Galimberti M, Polchi P et al., Bone marrow transplantation in patients with thalassemia. N Engl J Med 1990; 322(7):417–421.

    PubMed  CAS  Google Scholar 

  36. Lucarelli G, Clift RA, Galimberti M et al., Marrow transplantation for patients with thalassemia: results in class 3 patients. Blood 1996; 87(5):2082–2088.

    PubMed  CAS  Google Scholar 

  37. Storb RF, Lucarelli G, McSweeney PA et al., Hematopoietic cell transplantation for benign hematological disorders and solid tumors. Hematol Am Soc Hematol Educ Program 2003;372–397.

    Google Scholar 

  38. Lucarelli G, Galimberti M, Polchi P et al., Marrow transplantation in patients with thalassemia responsive to iron chelation therapy. N Engl J Med 1993; 329(12):840–844.

    PubMed  CAS  Google Scholar 

  39. Gaziev D, Polchi P, Galimberti M et al., Graft-versus-host disease after bone marrow transplantation for thalassemia: an analysis of incidence and risk factors. Transplantation 1997; 63(6):854–860.

    PubMed  CAS  Google Scholar 

  40. Karnon J, Zeuner D, Brown J et al., Lifetime treatment costs of beta-thalassemia major. Clin Lab Haematol 1999; 21(6):377–385.

    PubMed  CAS  Google Scholar 

  41. Issaragrisil S, Visuthisakchai S, Suvatte V et al., Brief report: transplantation of cord-blood stem cells into a patient with severe thalassemia. N Engl J Med 1995; 332(6):367–369.

    PubMed  CAS  Google Scholar 

  42. Issaragrisil S, Umbilical cord blood transplantation for thalassemia. Curr Hematol Rep 2005; 4(6):415–416.

    PubMed  Google Scholar 

  43. Issaragrisil S, Cord blood transplantation in thalassemia. Blood Cells 1994; 20(2–3): 259–262.

    PubMed  CAS  Google Scholar 

  44. Miniero R, Rocha V, Saracco P et al., Cord blood transplantation (CBT) in haemoglobinopathies. Eurocord. Bone Marrow Transplant 1998; 22 Suppl 1:S78–S79.

    PubMed  Google Scholar 

  45. Walters MC, Quirolo L, Trachtenberg ET et al., Sibling donor cord blood transplantation for thalassemia major: Experience of the Sibling Donor Cord Blood Program. Ann N Y Acad Sci 2005; 1054:206–213.

    PubMed  Google Scholar 

  46. Locatelli F, Rocha V, Reed W et al., Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood 2003; 101(6):2137–2143.

    PubMed  CAS  Google Scholar 

  47. Locatelli F, Rocha V, Chastang C et al., Factors associated with outcome after cord blood transplantation in children with acute leukemia. Eurocord-Cord Blood Transplant Group. Blood 1999; 93(11):3662–3671.

    PubMed  CAS  Google Scholar 

  48. Krishnamurti L, Abel S, Maiers M et al., Availability of unrelated donors for hematopoietic stem cell transplantation for haemoglobinopathies. Bone Marrow Transplant 2003; 31(7):547–550.

    PubMed  CAS  Google Scholar 

  49. Hall JG, Martin PL, Wood S et al., Unrelated umbilical cord blood transplantation for an infant with beta-thalassemia major. J Pediatr Hematol Oncol 2004; 26(6):382–385.

    PubMed  Google Scholar 

  50. Tan PH, Hwang WY, Goh YT et al., Unrelated peripheral blood and cord blood hematopoietic stem cell transplants for thalassemia major. Am J Hematol 2004; 75(4):209–212.

    PubMed  Google Scholar 

  51. Jaing TH, Hung IJ, Yang CP et al., Successful unrelated cord blood transplantation in a child with beta-thalassemia major. J Trop Pediatr 2005; 51(2):122–124.

    PubMed  Google Scholar 

  52. Fang J, Huang S, Chen C et al., Unrelated umbilical cord blood transplant for beta-thalassemia major. J Trop Pediatr 2003; 49(2):71–73.

    PubMed  Google Scholar 

  53. Jaing TH, Yang CP, Hung IJ et al., Transplantation of unrelated donor umbilical cord blood utilizing double-unit grafts for five teenagers with transfusion-dependent thalassemia. Bone Marrow Transplant 2007;40(4):307–311.

    PubMed  Google Scholar 

  54. Rocha V, Chastang C, Souillet G et al., Related cord blood transplants: the Eurocord experience from 78 transplants. Eurocord Transplant group. Bone Marrow Transplant 1998; 21 Suppl 3:S59-S62.

    PubMed  Google Scholar 

  55. Styczynski J, Cheung YK, Garvin J et al., Outcomes of unrelated cord blood transplantation in pediatric recipients. Bone Marrow Transplant 2004; 34(2):129–136.

    PubMed  CAS  Google Scholar 

  56. Rocha V, Wagner JE Jr, Sobocinski KA et al., Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources. N Engl J Med 2000; 342(25):1846–1854.

    PubMed  CAS  Google Scholar 

  57. Johnson FL, Look AT, Gockerman J et al., Bone-marrow transplantation in a patient with sickle-cell anemia. N Engl J Med 1984; 311(12):780–783.

    PubMed  CAS  Google Scholar 

  58. Walters MC, Patience M, Leisenring W et al., Bone marrow transplantation for sickle cell disease. N Engl J Med 1996; 335(6):369–376.

    PubMed  CAS  Google Scholar 

  59. Sullivan KM, Parkman R, Walters MC, Bone marrow transplantation for non-malignant disease. Hematology (Am Soc Hematol Educ Program) Volume 2000; 319–338.

    Google Scholar 

  60. Vermylen C, Cornu G, Ferster A et al., Bone marrow transplantation in sickle cell disease: the Belgian experience. Bone Marrow Transplant 1993; 12 Suppl 1:116–117.

    PubMed  Google Scholar 

  61. Walters MC, Storb R, Patience M et al., Impact of bone marrow transplantation for symptomatic sickle cell disease: an interim report. Multicenter investigation of bone marrow transplantation for sickle cell disease. Blood 2000; 95(6):1918–1924.

    PubMed  CAS  Google Scholar 

  62. Adamkiewicz TV, Mehta PS, Boyer MW et al., Transplantation of unrelated placental blood cells in children with high-risk sickle cell disease. Bone Marrow Transplant 2004; 34(5):405–411.

    PubMed  CAS  Google Scholar 

  63. Hill RS, Petersen FB, Storb R et al., Mixed hematologic chimerism after allogeneic marrow transplantation for severe aplastic anemia is associated with a higher risk of graft rejection and a lessened incidence of acute graft-versus-host disease. Blood 1986; 67(3):811–816.

    PubMed  CAS  Google Scholar 

  64. Andreani M, Manna M, Lucarelli G et al., Persistence of mixed chimerism in patients transplanted for the treatment of thalassemia. Blood 1996; 87(8):3494–3499.

    PubMed  CAS  Google Scholar 

  65. Slavin S, Nagler A, Naparstek E et al., Nonmyeloablative stem cell transplantation and cell therapy as an alternative to conventional bone marrow transplantation with lethal cytoreduction for the treatment of malignant and nonmalignant hematologic diseases. Blood 1998; 91(3):756–763.

    PubMed  CAS  Google Scholar 

  66. Iannone R, Casella JF, Fuchs EJ et al., Results of minimally toxic nonmyeloablative transplantation in patients with sickle cell anemia and beta-thalassemia. Biol Blood Marrow Transplant 2003; 9(8):519–528.

    PubMed  Google Scholar 

  67. Shaw PH, Haut PR, Olszewski M et al., Hematopoietic stem-cell transplantation using unrelated cord-blood versus matched sibling marrow in pediatric bone marrow failure syndrome: one center’s experience. Pediatr Transplant 1999; 3(4):315–321.

    PubMed  CAS  Google Scholar 

  68. Guardiola P, Socie G, Pasquini R et al., Allogeneic stem cell transplantation for Fanconi anaemia. Severe Aplastic Anaemia Working Party of the EBMT and EUFAR. European Group for Blood and Marrow Transplantation. Bone Marrow Transplant 1998; 21 Suppl 2:S24–S27.

    PubMed  Google Scholar 

  69. Guardiola P, Pasquini R, Dokal I et al., Outcome of 69 allogeneic stem cell transplantations for Fanconi anemia using HLA-matched unrelated donors: a study on behalf of the European Group for Blood and Marrow Transplantation. Blood 2000; 95(2):422–429.

    PubMed  CAS  Google Scholar 

  70. Kuliev A, Rechitsky S, Tur-Kaspa I et al., Preimplantation genetics: Improving access to stem cell therapy. Ann N Y Acad Sci 2005; 1054:223–227.

    PubMed  Google Scholar 

  71. Kuliev A, Rechitsky S, Verlinsky O et al., Preimplantation diagnosis and HLA typing for haemoglobin disorders. Reprod Biomed Online 2005; 11(3):362–370.

    PubMed  CAS  Google Scholar 

  72. Gluckman E, Bone marrow transplantation in Fanconi’s anemia. Stem Cells 1993; 11 Suppl 2:180–183.

    PubMed  Google Scholar 

  73. Gluckman E, Auerbach AD, Horowitz MM et al., Bone marrow transplantation for Fanconi anemia. Blood 1995; 86(7):2856–2862.

    PubMed  CAS  Google Scholar 

  74. Barker JN, Davies SM, Defor T et al., Survival after transplantation of unrelated donor umbilical cord blood is comparable to that of human leukocyte antigen-matched unrelated donor bone marrow: results of a matched-pair analysis. Blood 2001; 97(10):2957–2961.

    PubMed  CAS  Google Scholar 

  75. de la Fuente J., Reiss S, McCloy M et al., Non-TBI stem cell transplantation protocol for Fanconi anaemia using HLA-compatible sibling and unrelated donors. Bone Marrow Transplant 2003; 32(7):653–656.

    PubMed  Google Scholar 

  76. Tischkowitz M, Dokal I, Fanconi anaemia and leukaemia – clinical and molecular aspects. Br J Haematol 2004; 126(2):176–191.

    PubMed  CAS  Google Scholar 

  77. Gluckman E, Results of unrelated cord blood transplant in patients with bone marrow failure syndromes. Biol Blood Marrow Transplant 2004; 10(10):736–737. Ref Type: Abstract.

    Google Scholar 

  78. Buckley RH, Schiff SE, Schiff RI et al., Hematopoietic stem-cell transplantation for the treatment of severe combined immunodeficiency. N Engl J Med 1999; 340(7):508–516.

    PubMed  CAS  Google Scholar 

  79. Buckley RH, Schiff SE, Sampson HA et al., Development of immunity in human severe primary T cell deficiency following haploidentical bone marrow stem cell transplantation. J Immunol 1986; 136(7):2398–2407.

    PubMed  CAS  Google Scholar 

  80. Tan PL, Wagner JE, Auerbach AD et al., Successful engraftment without radiation after fludarabine-based regimen in Fanconi anemia patients undergoing genotypically identical donor hematopoietic cell transplantation. Pediatr Blood Cancer 2006; 46(5):630–636.

    PubMed  Google Scholar 

  81. de Medeiros CR, Silva LM, Pasquini R, Unrelated cord blood transplantation in a Fanconi anemia patient using fludarabine-based conditioning. Bone Marrow Transplant 2001; 28(1):110–112.

    PubMed  Google Scholar 

  82. Aker M, Varadi G, Slavin S et al., Fludarabine-based protocol for human umbilical cord blood transplantation in children with Fanconi anemia. J Pediatr Hematol Oncol 1999; 21(3):237–239.

    PubMed  CAS  Google Scholar 

  83. Yoshimasu T, Tanaka R, Suenobu S et al., Prompt and durable hematopoietic reconstitution by unrelated cord blood transplantation in a child with Fanconi anemia. Bone Marrow Transplant 2001; 27(7):767–769.

    PubMed  CAS  Google Scholar 

  84. Wagner JE, Barker JN, Defor TE et al., Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and nonmalignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood 2002; 100(5): 1611–1618.

    PubMed  CAS  Google Scholar 

  85. Rubinstein P, Carrier C, Scaradavou A et al., Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 1998; 339(22):1565–1577.

    PubMed  CAS  Google Scholar 

  86. Macmillan ML, Davies SM, Wagner JE et al., Engraftment of unrelated donor stem cells in children with familial amegakaryocytic thrombocytopenia. Bone Marrow Transplant 1998; 21(7):735–737.

    PubMed  CAS  Google Scholar 

  87. Ohga S, Ichino K, Goto K et al., Unrelated donor cord blood transplantation for childhood severe aplastic anemia after a modified conditioning. Pediatr Transplant 2006; 10(4): 497–500.

    PubMed  Google Scholar 

  88. Dianzani I, Garelli E, Ramenghi U, Diamond-Blackfan Anaemia: an overview. Paediatr Drugs 2000; 2(5):345–355.

    PubMed  CAS  Google Scholar 

  89. Bonno M, Azuma E, Nakano T et al., Successful hematopoietic reconstitution by transplantation of umbilical cord blood cells in a transfusion-dependent child with Diamond-Blackfan anemia. Bone Marrow Transplant 1997; 19(1):83–85.

    PubMed  CAS  Google Scholar 

  90. Nobili B, Rossi G, De SP et al., Successful umbilical cord blood transplantation in a child with dyskeratosis congenita after a fludarabine-based reduced-intensity conditioning regimen. Br J Haematol 2002; 119(2):573–574.

    Google Scholar 

  91. Mino E, Kobayashi R, Yoshida M et al., Umbilical cord blood stem cell transplantation from unrelated HLA-matched donor in an infant with severe congenital neutropenia. Bone Marrow Transplant 2004; 33(9):969–971.

    PubMed  CAS  Google Scholar 

  92. Nakazawa Y, Sakashita K, Kinoshita M et al., Successful unrelated cord blood transplantation using a reduced-intensity conditioning regimen in a 6-month-old infant with congenital neutropenia complicated by severe pneumonia. Int J Hematol 2004; 80(3):287–290.

    PubMed  Google Scholar 

  93. Vibhakar R, Radhi M, Rumelhart S et al., Successful unrelated umbilical cord blood transplantation in children with Shwachman-Diamond syndrome. Bone Marrow Transplant 2005; 36(10):855–861.

    PubMed  CAS  Google Scholar 

  94. Fischer A, Severe combined immunodeficiencies (SCID). Clin Exp Immunol 2000; 122(2):143–149.

    PubMed  CAS  Google Scholar 

  95. Myers LA, Patel DD, Puck JM et al., Hematopoietic stem cell transplantation for severe combined immunodeficiency in the neonatal period leads to superior thymic output and improved survival. Blood 2002; 99(3):872–878.

    PubMed  CAS  Google Scholar 

  96. Antoine C, Muller S, Cant A et al., Long-term survival and transplantation of haemopoietic stem cells for immunodeficiencies: report of the European experience 1968-99. Lancet 2003; 361(9357):553–560.

    PubMed  Google Scholar 

  97. Dalal I, Reid B, Doyle J et al., Matched unrelated bone marrow transplantation for combined immunodeficiency. Bone Marrow Transplant 2000; 25(6):613–621.

    PubMed  CAS  Google Scholar 

  98. Knutsen AP, Wall DA, Kinetics of T-cell development of umbilical cord blood transplantation in severe T-cell immunodeficiency disorders. J Allergy Clin Immunol 1999; 103(5 Pt 1): 823–832.

    PubMed  CAS  Google Scholar 

  99. Fischer A, Landais P, Friedrich W et al., European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet 1990; 336(8719):850–854.

    PubMed  CAS  Google Scholar 

  100. Bhattacharya A, Slatter MA, Chapman CE et al., Single centre experience of umbilical cord stem cell transplantation for primary immunodeficiency. Bone Marrow Transplant 2005; 36(4):295–299.

    PubMed  CAS  Google Scholar 

  101. Muller SM, Kohn T, Schulz AS et al., Similar pattern of thymic-dependent T-cell reconstitution in infants with severe combined immunodeficiency after human leukocyte antigen (HLA)-identical and HLA-nonidentical stem cell transplantation. Blood 2000; 96(13): 4344–4349.

    PubMed  CAS  Google Scholar 

  102. Gennery AR, Dickinson AM, Brigham K et al., CAMPATH-1 M T-cell depleted BMT for SCID: long-term follow-up of 19 children treated 1987-98 in a single center. Cytotherapy 2001; 3(3):221–232.

    PubMed  CAS  Google Scholar 

  103. Knutsen AP, Wall DA, Umbilical cord blood transplantation in severe T-cell immunodeficiency disorders: two-year experience. J Clin Immunol 2000; 20(6):466–476.

    PubMed  CAS  Google Scholar 

  104. Tsuji Y, Imai K, Kajiwara M 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(5):469–477.

    PubMed  CAS  Google Scholar 

  105. Ziegner UH, Ochs HD, Schanen C et al., Unrelated umbilical cord stem cell transplantation for X-linked immunodeficiencies. J Pediatr 2001; 138(4):570–573.

    PubMed  CAS  Google Scholar 

  106. Goebel WS, Nelson RP, Jr., Brahmi Z et al., Serial transplantation resulting in tolerance to an unrelated cord blood graft. Transplantation 2006; 81(11):1596–1599.

    PubMed  Google Scholar 

  107. Fagioli F, Biasin E, Berger M et al., Successful unrelated cord blood transplantation in two children with severe combined immunodeficiency syndrome. Bone Marrow Transplant 2003; 31(2):133–136.

    PubMed  CAS  Google Scholar 

  108. Filipovich AH, Stone JV, Tomany SC 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(6):1598–1603.

    PubMed  CAS  Google Scholar 

  109. Rumelhart SL, Trigg ME, Horowitz SD et al., Monoclonal antibody T-cell-depleted HLA-haploidentical bone marrow transplantation for Wiskott-Aldrich syndrome. Blood 1990; 75(4):1031–1035.

    PubMed  CAS  Google Scholar 

  110. Knutsen AP, Steffen M, Wassmer K et al., Umbilical cord blood transplantation in Wiskott Aldrich syndrome. J Pediatr 2003; 142(5):519–523.

    PubMed  Google Scholar 

  111. Kaneko M, Watanabe T, Watanabe H et al., Successful unrelated cord blood transplantation in an infant with Wiskott-Aldrich syndrome following recurrent cytomegalovirus disease. Int J Hematol 2003; 78(5):457–460.

    PubMed  Google Scholar 

  112. Bhattacharya A, Slatter M, Curtis A et al., Successful umbilical cord blood stem cell transplantation for chronic granulomatous disease. Bone Marrow Transplant 2003; 31(5): 403–405.

    PubMed  CAS  Google Scholar 

  113. Broom MA, Wang LL, Otta SK et al., Successful umbilical cord blood stem cell transplantation in a patient with Rothmund-Thomson syndrome and combined immunodeficiency. Clin Genet 2006; 69(4):337–343.

    PubMed  CAS  Google Scholar 

  114. Slatter MA, Bhattacharya A, Flood TJ et al., Use of two unrelated umbilical cord stem cell units in stem cell transplantation for Wiskott-Aldrich syndrome. Pediatr Blood Cancer 2006; 47(3):332–334.

    PubMed  CAS  Google Scholar 

  115. Ohtsuka Y, Shimizu T, Nishizawa K et al., Successful engraftment and decrease of cytomegalovirus load after cord blood stem cell transplantation in a patient with DiGeorge syndrome. Eur J Pediatr 2004; 163(12):747–748.

    PubMed  Google Scholar 

  116. Myers LA, Hershfield MS, Neale WT et al., Purine nucleoside phosphorylase deficiency (PNP-def) presenting with lymphopenia and developmental delay: successful correction with umbilical cord blood transplantation. J Pediatr 2004; 145(5):710–712.

    PubMed  CAS  Google Scholar 

  117. Tono C, Takahashi Y, Terui K et al., Correction of immunodeficiency associated with NEMO mutation by umbilical cord blood transplantation using a reduced-intensity conditioning regimen. Bone Marrow Transplant 2007;39(12):801–804.

    PubMed  CAS  Google Scholar 

  118. Howard V, Myers LA, Williams DA et al., Stem cell transplants for patients with X-linked agammaglobulinemia. Clin Immunol 2003; 107(2):98–102.

    PubMed  CAS  Google Scholar 

  119. Vowels MR, Tang RL, Berdoukas V et al., Brief report: correction of X-linked lymphoproliferative disease by transplantation of cord-blood stem cells. N Engl J Med 1993; 329(22):1623–1625.

    PubMed  CAS  Google Scholar 

  120. Krivit W, Sung JH, Shapiro EG et al., Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases. Cell Transplant 1995; 4(4):385–392.

    PubMed  CAS  Google Scholar 

  121. Boelens JJ, Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis 2006; 29(2–3):413–420.

    PubMed  Google Scholar 

  122. Peters C, Steward CG, Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines. Bone Marrow Transplant 2003; 31(4): 229–239.

    PubMed  CAS  Google Scholar 

  123. Wall DA, Carter SL, Kernan NA et al., Busulfan/melphalan/antithymocyte globulin followed by unrelated donor cord blood transplantation for treatment of infant leukemia and leukemia in young children: the Cord Blood Transplantation study (COBLT) experience. Biology of Blood and Marrow Transplantation 2005; 11(8):637–646.

    PubMed  CAS  Google Scholar 

  124. Peters C, Balthazor M, Shapiro EG et al., Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood 1996; 87(11):4894–4902.

    PubMed  CAS  Google Scholar 

  125. Krivit W, Aubourg P, Shapiro E et al., Bone marrow transplantation for globoid cell leukodystrophy, adrenoleukodystrophy, metachromatic leukodystrophy, and Hurler syndrome. Curr Opin Hematol 1999; 6(6):377–382.

    PubMed  CAS  Google Scholar 

  126. Krivit W, Peters C, Shapiro EG, Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III. Curr Opin Neurol 1999; 12(2):167–176.

    PubMed  CAS  Google Scholar 

  127. Boelens JJ, Wynn RF, O’Meara A et al., Outcomes of hematopoietic stem cell transplantation for Hurler’s syndrome in Europe: a risk factor analysis for graft failure. Bone Marrow Transplant 2007; 40(3):225–233.

    PubMed  CAS  Google Scholar 

  128. Church H, Tylee K, Copper A et al., Biochemical monitoring after haematopoietic stem cell transplant for Hurler syndrome (MPSIH): implications for functional outcome after transplant in metabolic disease. Bone Marrow Transplant 2007; 39(4) :207–210.

    PubMed  CAS  Google Scholar 

  129. Escolar ML, Poe MD, Provenzale JM et al., Transplantation of umbilical-cord blood in babies with infantile Krabbe’s disease. N Engl J Med 2005; 352(20):2069–2081.

    PubMed  CAS  Google Scholar 

  130. Escolar ML, Poe MD, Martin HR et al., A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation. Pediatrics 2006; 118(3):e879–e889.

    PubMed  Google Scholar 

  131. Shapiro E, Krivit W, Lockman L et al., Long-term effect of bone-marrow transplantation for childhood-onset cerebral X-linked adrenoleukodystrophy. Lancet 2000; 356(9231): 713–718.

    PubMed  CAS  Google Scholar 

  132. Shapiro EG, Lockman LA, Balthazor M et al., Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis 1995; 18(4):413–429.

    PubMed  CAS  Google Scholar 

  133. Peters C, Charnas LR, Tan Y et al., Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood 2004; 104(3):881–888.

    PubMed  CAS  Google Scholar 

  134. Beam D, Poe M, Provenzale J et al., Outcomes of Unrelated Umbilical Cord Blood Transplantation for X-Linked Adrenoleukodystrophy. Biol Blood Marrow Transplant 2007; 13(6):665–674.

    PubMed  Google Scholar 

  135. Lee V, Li CK, Shing MM et al., Umbilical cord blood transplantation for Maroteaux-Lamy syndrome (mucopolysaccharidosis type VI). Bone Marrow Transplant 2000; 26(4):455–458.

    PubMed  CAS  Google Scholar 

  136. Meyer-Wentrup F, Foell J, Wawer A et al., Unrelated cord blood transplantation in an infant with severe multisystem Langerhans cell histiocytosis: clinical outcome, engraftment and culture of monocyte-derived dendritic cells. Bone Marrow Transplant 2004; 33(8):875–876.

    PubMed  CAS  Google Scholar 

  137. Suminoe A, Matsuzaki A, Hattori H et al., Unrelated cord blood transplantation for an infant with chemotherapy-resistant progressive Langerhans cell histiocytosis. J Pediatr Hematol Oncol 2001; 23(9):633–636.

    PubMed  CAS  Google Scholar 

  138. Nagarajan R, Neglia J, Ramsay N et al., Successful treatment of refractory Langerhans cell histiocytosis with unrelated cord blood transplantation. J Pediatr Hematol Oncol 2001; 23(9):629–632.

    PubMed  CAS  Google Scholar 

  139. Urban C, Lackner H, Sovinz P et al., Successful unrelated cord blood transplantation in a 7-year-old boy with Evans syndrome refractory to immunosuppression and double autologous stem cell transplantation. Eur J Haematol 2006; 76(6):526–530.

    PubMed  Google Scholar 

  140. Mizumoto H, Hata D, Yamamoto K et al., Familial hemophagocytic lymphohistiocytosis with the MUNC13-4 mutation: a case report. Eur J Pediatr 2006; 165(6):384–388.

    PubMed  Google Scholar 

  141. Iguchi A, Kobayashi R, Sato TZ et al., Successful report of reduced-intensity stem cell transplantation from unrelated umbilical cord blood in a girl with chronic active Epstein-Barr virus infection. J Pediatr Hematol Oncol 2006; 28(4):254–256.

    PubMed  Google Scholar 

  142. Ishimura M, Ohga S, Nomura A et al., Successful umbilical cord blood transplantation for severe chronic active Epstein-Barr virus infection after the double failure of hematopoietic stem cell transplantation. Am J Hematol 2005; 80(3):207–212.

    PubMed  Google Scholar 

  143. Toubo T, Suga N, Ohga S et al., Successful unrelated cord blood transplantation for Epstein-Barr virus-associated lymphoproliferative disease with hemophagocytic syndrome. Int J Hematol 2004; 80(5):458–462.

    PubMed  Google Scholar 

  144. Zhang C, Feng HY, Huang SL et al., Therapy of Duchenne muscular dystrophy with umbilical cord blood stem cell transplantation. Zhonghua Yi Xue Yi Chuan Xue Za Zhi 2005; 22(4):399–405.

    PubMed  CAS  Google Scholar 

  145. Zhang C, Chen W, Xiao LL et al., Allogeneic umbilical cord blood stem cell transplantation in Duchenne muscular dystrophy. Zhonghua Yi Xue Za Zhi 2005; 85(8):522–525.

    PubMed  Google Scholar 

  146. Andolina M, Maximova N, Rabusin M et al., Failure of a sibling umbilical cord blood transplantation to correct hemophilia A. Haematologica 2004; 89(7):ECR22.

    PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Head, BMT Service, Senior Staff Oncologist, Oncology Unit, Clinical Associate Professor, Discipline of Paediatrics & Child Health, Children’s Hospital at Westmead, Sydney, NSW 2145, Australia

    Peter J. Shaw

Authors
  1. Tatjana Kilo
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Peter J. Shaw
    View author publications

    You can also search for this author in PubMed Google Scholar

Editor information

Phillip Stubblefield Niranjan Bhattacharya

Rights and permissions

Reprints and permissions

Copyright information

© 2009 Springer-Verlag London Limited

About this chapter

Cite this chapter

Kilo, T., Shaw, P.J. (2009). Cord Blood Transplantation for Pediatric Non-Malignant Conditions. In: Stubblefield, P., Bhattacharya, N. (eds) Frontiers of Cord Blood Science. Springer, London. https://doi.org/10.1007/978-1-84800-167-1_7

Download citation

  • DOI: https://doi.org/10.1007/978-1-84800-167-1_7

  • Published:

  • Publisher Name: Springer, London

  • Print ISBN: 978-1-84800-166-4

  • Online ISBN: 978-1-84800-167-1

  • eBook Packages: MedicineMedicine (R0)

Publish with us

Policies and ethics

Search

Navigation