Unrelated donor marrow transplantation for congenital immunodeficiency and metabolic disease: An update of the experience of the Japan Marrow Donor Program

International Journal of Hematology

Volumn 80, Issue 2, 2004, Pages 174-182

  Sakata, Naoki ^a   Kawa, Keisei ^a   Kato, Koji ^b   Yabe, Hiromasa ^c   Yabe, Miharu ^c   Nagasawa, Masayuki ^d   Mugishima, Hideo ^e   Kigasawa, Hisato ^f   Tsuchida, Masahiro ^g   Akiyama, Yuichi ^h   Morisima, Yasuo ⁱ   Kodera, Yoshihisa ^b   Kato, Shunichi ^c  

^a RESEARCH INSTITUTE FOR MATERNAL AND CHILD HEALTH   (Japan)

^b JAPANESE RED CROSS NAGOYA FIRST HOSPITAL   (Japan)

^c TOKAI UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^d TOKYO MEDICAL AND DENTAL UNIVERSITY   (Japan)

^e NIHON UNIVERSITY SCHOOL OF MEDICINE   (Japan)

^f KANAGAWA CHILDREN'S MEDICAL CENTER   (Japan)

^g Ibaraki Children's Hospital   (Japan)

^h NATIONAL HOSPITAL ORGANIZATION KYOTO MEDICAL CENTER   (Japan)

ⁱ AICHI CANCER CENTER HOSPITAL   (Japan)

Author keywords

Congenital immunodeficiency; Congenital metabolic disease; Hemophagocytic syndrome; Japan Marrow Donor Program (JMDP); Unrelated bone marrow transplantation

Indexed keywords

HLA A ANTIGEN; HLA B ANTIGEN; HLA DR ANTIGEN;

ADOLESCENT; ADRENOLEUKODYSTROPHY; ADULT; AGED; BONE MARROW TRANSPLANTATION; CHILD; DONOR; FEMALE; GRAFT VERSUS HOST REACTION; HLA MATCHING; HUMAN; IMMUNE DEFICIENCY; INBORN ERROR OF METABOLISM; INFANT; JAPAN; MAJOR CLINICAL STUDY; MALE; MUCOPOLYSACCHARIDOSIS; REVIEW;

ADOLESCENT; ADULT; BONE MARROW TRANSPLANTATION; CHILD; CHILD, PRESCHOOL; FOLLOW-UP STUDIES; GENETIC DISEASES, INBORN; GRAFT VS HOST DISEASE; HISTOCOMPATIBILITY TESTING; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; INFANT; METABOLISM, INBORN ERRORS; MIDDLE AGED; RETROSPECTIVE STUDIES; SURVIVAL ANALYSIS; TRANSPLANTATION CONDITIONING; TREATMENT FAILURE;

EID: 4444226925     PISSN: 09255710     EISSN: None     Source Type: Journal    
DOI: 10.1532/IJH97.04055     Document Type: Review

References (28)

1. Gordon-Smith EC, Fairhead SM, Chipping PM, et al. Bone-marrow transplantation for severe aplastic anaemia using histocompatible unrelated volunteer donors. Br Med J (Clin Res Ed). 1982;285:835-837.
2. McCullough J, Rogers G, Dahl R, et al. Development and operation of a program to obtain volunteer bone marrow donors unrelated to the patient. Transfusion. 1986;26:315-323.
3. McElligott MC, Menitove JE, Aster RH. Recruitment of unrelated persons as bone marrow donors: a preliminary experience. Transfusion. 1986;26:309-314.
4. Glucksberg H, Storb R, Fefer A, et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation. 1974;18:295-304.
5. Kaplan EL, Meier P. Nonparametric estimation from incomplete observation. J Am Stat Assoc. 1958;53:457-481.
6. Kernan NA, Bartsch G, Ash RC, et al. Analysis of 462 transplantations from unrelated donors facilitated by the National Marrow Donor Program. N Engl J Med. 1993;328:593-602.
7. Miano M, Porta F, Locatelli F, et al. Unrelated donor marrow transplantation for inborn errors. Bone Marrow Transplant. 1998;21(suppl 2):S37-S41.
8. Gustafsson A, Remberger M, Winiarski J, Ringden O. Unrelated bone marrow transplantation in children: outcome and a comparison with sibling donor grafting. Bone Marrow Transplant. 2000;25:1059-1065.
9. Dini G, Cancedda R, Locatelli F, et al. Unrelated donor marrow transplantation: an update of the experience of the Italian Bone Marrow Group (GITMO). Haematologica. 2001;86:451-456.
10. Mugishima H, Takahashi T, Nagamura T, Asano S, Saito H. Umbilical cord blood for unrelated bone marrow replacement: Asia bank and Japan cord blood bank network update. Int J Hematol. 2002;76(suppl 2):284-286.
11. 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;100:1611-1618.
12. 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;91:2601-2608.
13. + augmented elutriated allografts for nonmalignant diseases. Bone Marrow Transplant. 2003;31:1073-1080.
14. Ogawa H, Soma T, Hosen N, et al. Combination of tacrolimus, methotrexate and methylprednisolone prevents acute but not chronic graft-versus-host disease in unrelated bone marrow transplantation. Transplantation. 2002;74:236-243.
15. Morishima Y, Sasazuki T, Inoko H, et al. The clinical significance of human leukocyte antigen (HLA) allele compatibility in patients receiving a marrow transplant from serologically HLA-A, HLA-B, and HLA-DR matched unrelated donors. Blood. 2002;99:4200-4206.
16. Sullivan KM, Parkman R, Walters MC. Bone marrow transplantation for non-malignant disease. Hematology (Am Soc Hematol Educ Program). 2000:319-338.
17. Filipovich AH, Shapiro RS, Ramsay NK, et al. Unrelated donor bone marrow transplantation for correction of lethal congenital immunodeficiencies. Blood. 1992;80:270-276.
18. Dalal I, Reid B, Doyle J, et al. Matched unrelated bone marrow transplantation for combined immunodeficiency. Bone Marrow Transplant. 2000;25:613-621.
19. 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;361:553-560.
20. Giralt S, Estey E, Albitar M, et al. Engraftment of allogeneic hematopoietic progenitor cells with purine analog-containing chemotherapy: harnessing graft-versus-leukemia without myeloablative therapy. Blood. 1997;89:4531-4536.
21. 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;91:756-763.
22. Amrolia P, Caspar HB, Hassan A, et al. Nonmyeloablative stem cell transplantation for congenital immunodeficiencies. Blood. 2000;96:1239-1246.
23. Gaspar HB, Amrolia P, Hassan A, et al. Non-myeloablative stem cell transplantation for congenital immunodeficiencies. Recent Results Cancer Res. 2002;159:134-142.
24. 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;2:709-712.
25. Whitley CB, Belani KG, Chang PN, et al. Long-term outcome of Hurler syndrome following bone marrow transplantation. Am J Med Genet. 1993;46:209-218.
26. Hoogerbrugge PM, Brouwer OF, Bordigoni P, et al. Allogeneic bone marrow transplantation for lysosomal storage diseases: the European Group for Bone Marrow Transplantation. Lancet. 1995;345:1398-1402.
27. Vellodi A, Young EP, Cooper A, et al. Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. Arch Dis Child. 1997;76:92-99.
28. Peters C, Balthazor M, Shapiro EG, et al. Outcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome. Blood. 1996;87:4894-4902.