Transplant of hematopoietic stem cells in childhood: Where we are and where we are going

Haematologica

Volumn 83, Issue 6, 1998, Pages 550-563

  Locatelli, Franco ^a   Burgio, Giuseppe Roberto ^a  

^a UNIVERSITY OF PAVIA   (Italy)

Author keywords

Bioethics and transplantation; Bone marrow transplantation; Hematopoietic stem cell transplantation; Peripheral blood transplantation; Placental blood transplantation; Transplant of hematological disorders in childhood

Indexed keywords

BONE MARROW TRANSPLANTATION; CANCER CHEMOTHERAPY; CHILD; CHILDHOOD DISEASE; GRAFT VERSUS HOST REACTION; HEMATOLOGIC DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HLA TYPING; HUMAN; MEDICAL ETHICS; REVIEW; SECOND CANCER; TREATMENT INDICATION; UMBILICAL CORD BLOOD;

CHILD; CHILD, PRESCHOOL; GRAFT SURVIVAL; HEMATOPOIETIC STEM CELL MOBILIZATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; NEOPLASMS;

EID: 0031820549     PISSN: 03906078     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (100)

1. Gatti RA, Meuwissen HJ, Allen HD, Hong R, Good RA. Immunological reconstitution of sex-linked lymphopenic immunological deficiency. Lancet 1968; ii:1366-9.
2. Bach FH, Albertini RJ, Joo P, AndersonJL, Bortin MM. Bone-marrow transplantation in a patient with the Wiskott-Aldrich syndrome. Lancet 1968; ii:1364-6.
3. Burnet M. Self and not-self. Melbourne: University Press; 1970.
4. Horowitz SD, Bach FH, Groshong T, Hong R, Yunis EJ. Treatment of severe combined immunodeficiency with bone-marrow from an unrelated, mixed-leucocyte-culture-non-reactive donor. Lancet 1975; ii:431-3.
5. Speck B, Zwaan FE, van Rood JJ, Eernisse JG. Allogeneic bone marrow transplantation in a patient with aplastic anemia using a phenotypically HLA-identical unrelated donor. Transplantation 1973; 16:24-8.
6. Hansen JA, Clift RA, Thomas ED, Buckner CD, Storb R, Giblett ER. Transplantation of marrow from an unrelated donor to a patient with acute leukemia. N Engl J Med 1980; 303:565-7.
7. Hows JM, Yin JL, Marsh J, et al. Histocompatible unrelated volunteer donors compared with HLA nonidentical family donors in marrow transplantation for aplastic anemia and leukemia. Blood 1986; 68:1322-8.
8. Anasetti C, Amos D, Beatty PG, et al. Effect of HLA compatibility on engraftment of bone marrow transplants in patients with leukemia or lymphoma. N Engl J Med 1989; 320:197-204.
9. Anasetti C, Hansen J. Effect of HLA incompatibility in marrow transplantation from unrelated and HLA-mismatched related donors. Transf Sci 1994; 14:221-30.
10. Madrigal JA, Scott I, Arguello R, Szydlo R, Little AM, Goldman JM. Factors influencing the outcome of bone marrow transplants using unrelated donors. Immunol Rev 1997; 157:153-66.
11. Cleaver S. The Anthony Nolan Research Centre and other matching registries. In: Treleaven J, Barrett J, eds. Bone marrow transplantation in practice. Edinburgh: Churchill Livingstone; 1992. p. 361-6.
12. Hansen JA, McCullough J. Alternative donor-unrelated donor BMT. In: Champlin RE, Gale RP, eds. New strategies in bone marrow transplantation. New York: Wiley-Liss; 1991. p. 453-7.
13. Dupont B. Immunology of hematopoietic stem cell transplantation: a brief review of its history. Immunol Rev 1997; 157. p. 5-12.
14. Hansen JA, Petersdorf E, Martin PJ, Anasetti C. Hematopoietic stem cell transplants from unrelated donors. Immunol Rev 1997; 157:141-51.
15. Oudshoorn M, van Leeuwen A, Zanden HG, van Rood JJ. Bone Marrow Donors Worldwide: a successful exercise in international cooperation. Bone Marrow Transplant 1994; 14:3-8.
16. Kernan NA, Bartsch G, Ash RC, et al. Analysis of 462 transplantations from unrelated donors facilitated by the National Marrow Donor Program [see comments]. N Engl J Med 1993; 328:593-602.
17. Oakhill A, Pamphilon DH, Potter MN, et al. Unrelated donor bone marrow transplantation for children with relapsed acute lymphoblastic leukaemia in second complete remission. Br J Haematol 1996; 94:574-8.
18. Balduzzi A, Gooley T, Anasetti C, et al. Unrelated donor marrow transplantation in children. Blood 1995; 86:3247-56.
19. Burgio GR, Nespoli L, Porta F, Bonetti F. Bone marrow transplantation in children: 20 years after its first successful application. Haematologica 1988; 73:437-43.
20. Fischer A, Landais P, Friedrich W, et al. European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet 1990; 336:850-4.
21. Locasciulli A, van't Veer L, Bacigalupo A, et al. Treatment with marrow transplantation or immunosuppression of childhood acquired severe aplastic anemia: a report from the EBMT SAA Working Party. Bone Marrow Transplant 1990; 6:211-7.
22. Wengler GS, Lanfranchi A, Frusca T, et al. In-utero transplantation of parental CD34 haematopoietic progenitor cells in a patient with X-linked severe combined immunodeficiency (SCIDXI). Lancet 1996; 348: 1484-7.
23. 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: 1174-8.
24. Richman CM, Weiner RS, Yankee RA. Increase in circulating stem cells following chemotherapy in man. Blood 1976; 47:1031-9.
25. Duhrsen U, Villeval JL, Boyd J, Kannourakis G, Morstyn G, Metcalf D. Effects of recombinant human granulocyte colony-stimulating factor on hematopoietic progenitor cells in cancer patients. Blood 1988; 72:2074-81.
26. Demeocq F, Kanold J, Chassagne J, et al. Successful blood stem cell collection and transplant in children weighing less than 25 kg. Bone Marrow Transplant 1994; 13:43-50.
27. Takaue Y, Kawano Y, Abe T, et al. Collection and transplantation of peripheral blood stem cells in very small children weighing 20 kg or less. Blood 1995; 86:372-80.
28. Fukuda M, Kojima S, Matsumoto K, Matsuyama T. Autotransplantation of peripheral blood stem cells mobilized by chemotherapy and recombinant human granulocyte colony-stimulating factor in childhood neuroblastoma and non-Hodgkin's lymphoma. Br J Haematol 1992; 80:327-31.
29. Hartmann O, Le Corroller AG, Blaise D, et al. Peripheral blood stem cell and bone marrow transplantation for solid tumors and lymphomas: hematologic recovery and costs. A randomized, controlled trial. Ann Intern Med 1997; 126:600-7.
30. Gianni AM. Where do we stand with respect to the use of peripheral blood progenitor cells? [editorial; comment]. Ann Oncol 1994; 5:781-4.
31. Bensinger WI, Clift R, Martin P, et al. Allogeneic peripheral blood stem cell transplantation in patients with advanced hematologic malignancies: a retrospective comparison with marrow transplantation. Blood 1996; 88:2794-800.
32. Goldman J. Peripheral blood stem cells for allografting [editorial] [see comments]. Blood 1995; 85:1413-5.
33. Zecca M, Perotti C, Marradi P, et al. Recombinant human G-CSF-mobilized peripheral blood stem cells for second allogeneic transplant after bone marrow graft rejection in children. Br J Haematol 1996; 92:432-4.
34. 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; 337:373-81.
35. Wagner JE, Kernan NA, Steinbuch M, Broxmeyer HE, Gluckman E. Allogeneic sibling umbilical-cord-blood transplantation in children with malignant and non-malignant disease [see comments]. Lancet 1995; 346:214-9.
36. 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; 335:157-66.
37. Gluckman E, WagnerJ, Hows J, Kernan N, Bradley B, Broxmeyer HE. Cord blood banking for hematopoietic stem cell transplantation: an international cord blood transplant registry. Bone Marrow Transplant 1993; 11:199-200.
38. Sugarman J, Reisner EG, Kurtzberg J. Ethical aspects of banking placental blood for transplantation [see comments]. JAMA 1995; 274:1783-5.
39. Silberstein LE, Jefferies LC. Placental-blood banking-a new frontier in transfusion medicine [editorial; comment]. N Engl J Med 1996; 335:199-201.
40. Burgio GR, Locatelli F. Transplant of bone marrow and cord blood hematopoietic stem cells in pediatric practice, revisited according to the fundamental principles of bioethics. Bone Marrow Transplant 1997; 19:1163-8.
41. Bertolini F, Battaglia M, De Iulio C, Sirchia G, Rosti L. Placental blood collection: effects on newborns [letter] [see comments]. Blood 1995; 85:3361-2.
42. Aversa F, Tabilio A, Terenzi A, et al. Successful engraftment of T-cell-depleted haploidentical "three-loci" incompatible transplants in leukemia patients by addition of recombinant human granulocyte colony-stimulating factor-mobilized peripheral blood progenitor cells to bone marrow inoculum. Blood 1994; 84:3948-55.
43. Jecker NS. Conceiving a child to save a child: reproductive and filial ethics. J Clin Ethics 1990; 1:99-103.
44. Burgio GR, Nespoli L, Locatelli F. Bone marrow transplantation in children: between primum non nocere (above all, do not harm) and primum adiuvare (above all, help). In: Burgio GR, Lantos JD, eds. Primum non nocere today. A symposium on pediatric bioethics. Amsterdam: Elsevier; 1994. p. 77-93.
45. Burgio GR, Nespoli L, Porta F, Locatelli F. Programmed bone-marrow donor for a leukaemic sibling, 10 years on [letter]. Lancet 1997; 349:1482.
46. Horowitz MM, Gale RP, Sondel PM, et al. Graft-versus-leukemia reactions after bone marrow transplantation. Blood 1990; 75:555-62.
47. Spencer A, Szydlo RM, Brookes PA, et al. Bone marrow transplantation for chronic myeloid leukemia with volunteer unrelated donors using ex vivo or in vivo T-cell depletion: major prognostic impact of HLA class I identity between donor and recipient. Blood 1995; 86:3590-7.
48. Petersdorf EW, Longton GM, Anasetti C, et al. Definition of HLA-DQ as a transplantation antigen. Proc Natl Acad Sci USA 1996; 93:15358-63.
49. Kaminski E, Hows J, Man S, et al. Prediction of graft versus host disease by frequency analysis of cytotoxic T cells after unrelated donor bone marrow transplantation. Transplantation 1989; 48:608-13.
50. Theobald M, Nierle T, Bunjes D, Arnold R, Heimpel H. Host-specific interleukin-2-secreting donor T-cell precursors as predictors of acute graft-versus-host disease in bone marrow transplantation between HLA-identical siblings [see comments]. N Engl J Med 1992; 327:1613-7.
51. Schwarer AP, Jiang YZ, Brookes PA, et al. Frequency of anti-recipient alloreactive helper T-cell precursors in donor blood and graft-versus-host disease after HLA-identical sibling bone-marrow transplantation. Lancet 1993; 341:203-5.
52. Dini G, Lanino E, Lamparelli T, et al. Unrelated donor marrow transplantation: initial experience of the Italian bone marrow transplant group (GITMO). Bone Marrow Transplant 1996; 17:55-62.
53. Deeg HJ, Socie G, Schoch G, et al. Malignancies after marrow transplantation for aplastic anemia and Fanconi anemia: a joint Seattle and Paris analysis of results in 700 patients. Blood 1996; 87:386-92.
54. Socie G, Henry Amar M, Bacigalupo A, et al. Malignant tumors occurring after treatment of aplastic anemia. European Bone Marrow Transplantation-Severe Aplastic Anaemia Working Party. N Engl J Med 1993; 329: 1152-7.
55. 131I-labeled anti-CD45 antibody, combined with cyclophosphamide and total body irradiation. Blood 1995; 85:1122-31.
56. Storb R, Deeg HJ, Farewell V, et al. Marrow transplantation for severe aplastic anemia: methotrexate alone compared with a combination of methotrexate and cyclosporine for prevention of acute graft-versus-host disease. Blood 1986; 68:119-25.
57. Storb R, Deeg HJ, Whitehead J, et al. Methotrexate and cyclosporine compared with cyclosporine alone for prophylaxis of acute graft versus host disease after marrow transplantation for leukemia. N Engl J Med 1986; 314:729-35.
58. Nash RA, Etzioni R, Storb R, et al. Tacrolimus (FK506) alone or in combination with methotrexate or methyl-prednisolone for the prevention of acute graft-versus-host disease after marrow transplantation from HLA-matched siblings: a single-center study. Blood 1995; 85:3746-53.
59. Nash RA, Pineiro LA, Storb R, et al. FK506 in combination with methotrexate for the prevention of graft-versus-host disease after marrow transplantation from matched unrelated donors. Blood 1996; 88:3634-41.
60. Gisselbrecht C, Prentice HG, Bacigalupo A, et al. Placebo-controlled phase III trial of lenograstim in bone-marrow transplantation [published erratum appears in Lancet 1994 Mar 26; 343:804]. Lancet 1994; 343:696-700.
61. Locatelli F, Pession A, Zecca M, et al. Use of recombinant human granulocyte colony-stimulating factor in children given allogeneic bone marrow transplantation for acute or chronic leukemia. Bone Marrow Transplant 1996; 17:31-7.
62. Sheridan WP, Morstyn G, Wolf M, et al. Granulocyte colony-stimulating factor and neutrophil recovery after high-dose chemotherapy and autologous bone marrow transplantation. Lancet 1989; 2:891-5.
63. De Witte T, Gratwohl A, Van Der Lely N, et al. Recombinant human granulocyte-macrophage colony-stimulating factor accelerates neutrophil and monocyte recovery after allogeneic T-cell-depleted bone marrow transplantation. Blood 1992; 79:1359-65.
64. Gerna G, Furione M, Baldanti F, Percivalle E, Comoli P, Locatelli F. Quantitation of human cytomegalovirus DNA in bone marrow transplant recipients. Br J Haematol 1995; 91:674-83.
65. Locatelli F, Percivalle E, Comoli P, et al. Human cytomegalovirus (HCMV) infection in paediatric patients given allogeneic bone marrow transplantation: role of early antiviral treatment for HCMV antigenaemia on patients' outcome. Br J Haematol 1994; 88:64-71.
66. Goodrich JM, Mori M, Gleaves CA, et al. Early treatment with ganciclovir to prevent cytomegalovirus disease after allogeneic bone marrow transplantation. N Engl J Med 1991; 325:1601-7.
67. Richardson PG, Elias AD, Krishnan A, et al. Treatment of severe veno-occlusive disease with defibrotide: compassionate use results in efficacy without significant toxicity in a high risk population [abstract]. Blood 1997; 90:252a.
68. Bearman SI, Shuhart MC, Hinds MS, McDonald GB. Recombinant human tissue plasminogen activator for the treatment of established severe venocclusive disease of the liver after bone marrow transplantation. Blood 1992; 80:2458-62.
69. Bearman SI. The syndrome of hepatic veno-occlusive disease after marrow transplantation. Blood 1995; 85:3005-20.
70. Walter EA, Greenberg PD, Gilbert MJ, et al. Reconstitution of cellular immunity against cytomegalovirus in recipients of allogeneic bone marrow by transfer of T-cell clones from the donor [see comments]. N Engl J Med 1995; 333:1038-44.
71. Enright H, Haake R, Weisdorf D, et al. Cytomegalovirus pneumonia after bone marrow transplantation. Risk factors and response to therapy. Transplantation 1993; 55:1339-46.
72. Rooney CM, Smith CA, Ng CY, et al. Use of gene-modified virus-specific T lymphocytes to control Epstein-Barr-virus-related lymphoproliferation. Lancet 1995; 345:9-13.
73. Kolb HJ, Mittermuller J, Clemm C, et al. Donor leukocyte transfusions for treatment of recurrent chronic myelogenous leukemia in marrow transplant patients. Blood 1990; 76:2462-5.
74. van Rhee F, Lin F, Cullis JO, et al. Relapse of chronic myeloid leukemia after allogeneic bone marrow transplant: the case for giving donor leukocyte transfusions before the onset of hematologic relapse. Blood 1994; 83:3377-83.
75. Mackinnon S, Papadopoulos EB, Carabasi MH, et al. Adoptive immunotherapy evaluating escalating doses of donor leukocytes for relapse of chronic myeloid leukemia after bone marrow transplantation: separation of graft-versus-leukemia responses from graft-versus-host disease. Blood 1995; 86:1261-8.
76. Falkenburg JH, Smit WM, Willemze R. Cytotoxic T-lymphocyte (CTL) responses against acute or chronic myeloid leukemia. Immunol Rev 1997; 157:223-30.
77. Bonilla MA, Dale D, Zeidler C, et al. Long-term safety of treatment with recombinant human granulocyte colony-stimulating factor (r-metHuG-CSF) in patients with severe congenital neutropenias. Br J Haematol 1994; 88:723-30.
78. Weening RS, Leitz GJ, Seger RA. Recombinant human interferon-gamma in patients with chronic granulomatous disease-European follow up study. Eur J Pediatr 1995; 154:295-8.
79. Hershfield MS, Buckley RH, Greenberg ML, et al. Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase. N Engl J Med 1987; 316:589-96.
80. Gluckman E, Horowitz MM, Champlin RE, et al. Bone marrow transplantation for severe aplastic anemia: influence of conditioning and graft-versus-host disease prophylaxis regimens on outcome. Blood 1992; 79:269-75.
81. Bacigalupo A, Broccia G, Corda G, et al. Antilymphocyte globulin, cyclosporin, and granulocyte colony-stimulating factor in patients with acquired severe aplastic anemia (SAA): a pilot study of the EBMT SAA Working Party. Blood 1995; 85:1348-53.
82. Kojima S, Fukuda M, Miyajima Y, Matsuyama T, Horibe K. Treatment of aplastic anemia in children with recombinant human granulocyte colony-stimulating factor. Blood 1991; 77:937-41.
83. Hows J, Bacigalupo A, Downie T, Bradley B. Alternative donor BMT (ALT-BMT) for severe aplastic anaemia (SAA) and Fanconi anaemia (FA) in Europe [abstract]. Bone Marrow Transplant 1996; 17 (suppl. 1):S56.
84. Ohara A, Kojima S, Hamajima N, et al. Myelodysplastic syndrome and acute myelogenous leukemia as a late clonal complication in children with acquired aplastic anemia. Blood 1997; 90:1009-13.
85. Fenaux P, Le Deley MC, Castaigne S, et al. Effect of all transretinoic acid in newly diagnosed acute promyelocytic leukemia. Results of a multicenter randomized trial. European APL 91 Group. Blood 1993; 82:3241-9.
86. Mandelli F, Diverio D, Avvisati G, et al. Molecular remission in PML/RAR alpha-positive acute promyelocytic leukemia by combined all-trans retinoic acid and idarubicin (AIDA) therapy. Gruppo Italiano-Malattie Ematologiche Maligne dell'Adulto and Associazione Italiana di Ematologia ed Oncologia Pediatrica Cooperative Groups. Blood 1997; 90:1014-21.
87. Miller-WHJ, Levine K, De Blasio A, Frankel SR, Dmitrovsky E, Warrell-RPJ. Detection of minimal residual disease in acute promyelocytic leukemia by a reverse transcription polymerase chain reaction assay for the PML/RAR-α fusion mRNA. Blood 1993; 82:1689-94.
88. Meloni G, Diverio D, Vignetti M, et al. Autologous bone marrow transplantation for acute promyelocytic leukemia in second remission: prognostic relevance of pretransplant minimal residual disease assessment by reverse-transcription polymerase chain reaction of the PML/RAR-α fusion gene. Blood 1997; 90:1321-5.
89. Majolino I, Pearce R, Taghipour G, Goldstone AH. Peripheral-blood stem-cell transplantation versus autologous bone marrow transplantation in Hodgkin's and non-Hodgkin's lymphomas: a new matched-pair analysis of the European Group for Blood and Marrow Transplantation Registry Data. Lymphoma Working Party of the European Group for Blood and Marrow Transplantation. J Clin Oncol 1997; 15:509-17.
90. Locatelli F, Ravelli A, Maccario R, et al. Autologous peripheral blood selected CD34+ cell transplantation for the treatment of severe progressive systemic sclerosis [abstract]. Blood 1996; 88 (suppl. 1):284b.
91. Marmont AM, Tyndall A, Gratwohl A, Vischer T. Haemopoietic precursor-cell transplants for autoimmune diseases. Lancet 1995; 345:978.
92. Burt RG, Traynor A, Ramsey-Goldman R. Hematopoietic stem-cell transplantation for systemic lupus erythematosus [letter]. N Engl J Med 1997; 337:1777-8.
93. Kolb HJ, Bender Gotze C. Late complications after allogeneic bone marrow transplantation for leukaemia. Bone Marrow Transplant 1990; 6:61-72.
94. Locatelli F, Giorgiani G, Pession A, Bozzola M. Late effects in children after bone marrow transplantation: a review. Haematologica 1993; 78:319-28.
95. Curtis RE, Rowlings PA, Deeg HJ, et al. Solid cancers after bone marrow transplantation [see comments]. N Engl J Med 1997; 336:897-904.
96. Witherspoon RP, Fisher LD, Schoch G, et al. Secondary cancers after bone marrow transplantation for leukemia or aplastic anemia [see comments]. N Engl J Med 1989; 321:784-9.
97. Neglia JP, Meadows AT, Robison LL, et al. Second neoplasms after acute lymphoblastic leukemia in childhood. N Engl J Med 1991; 325:1330-6.
98. Hinterberger Fischer M, Kier P, Kalhs P, et al. Fertility, pregnancies and offspring complications after bone marrow transplantation. Bone Marrow Transplant 1991; 7:5-9.
99. Giorgiani G, Bozzola M, Locatelli F, et al. Role of busulfan and total body irradiation on growth of prepuberal children receiving bone marrow transplantation and results of treatment with recombinant human growth hormone. Blood 1995; 86:825-31.
100. Lantos JD. Children as organ donors: an argument for involuntary altruism. In: Burgio GR, Lantos JD, eds. Primum non nocere today. A symposium on pediatric bioethics. Amsterdam: Elsevier; 1994. p. 67-75.