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Bone Marrow Transplantation

  • P. M. Hoogerbrugge
  • J. M. J. J. Vossen

Abstract

Allogeneic bone marrow transplantation (BMT) has been used successfully during recent decades for the treatment of patients suffering from hematologic malignancies and other diseases of cells of the hemopoietic system, e.g., aplastic anemia and severe combined immune deficiency (SCID) disease. Following appropriate conditioning of the recipient of an allogeneic bone marrow graft, his or her hemopoietic system is replaced by that of the bone marrow donor and a chimeric state is achieved. Engraftment of hemopoietic stem cells from an allogeneic donor requires intensive cytoreduction and immunosuppression of the immunologically competent recipient in order to create space, achieve engraftment, and prevent rejection of the graft. Pretreatment of children for BMT for inherited metabolic disorders consisted mostly of intensive pretreatment, e.g., by cyclophosphamide (50mg/kg per day, ×4) and busulfan (4 mg/kg per day, ×4) [1]. Alternatively, total body irradiation or total lymph node irradiation was used. After allogeneic BMT a reverse rejection of the recipient tissue by the donor bone marrow graft, the so-called graft-versus-host disease (GvHD), must be prevented. Matching of donor and recipient for the major histocompatibility complex antigens (HLA antigens), T cell depletion of the graft, and total gastrointestinal decontamination and administration (post-transplantation) of methotrexate and/or cyclosporin A are effective in preventing or mitigating GvHD [2].

Keywords

Bone Marrow Transplantation Gauche Disease Allogeneic Bone Marrow Lysosomal Storage Disease Severe Combine Immune Deficiency 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

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References

  1. 1.
    Hobbs JR, Hugh-Jones K, Byrom N, Chambers DCO, Henry K, James JCO, Lucas CF, Rogers TR (1981) Reversal of clinical features of Hurler’s disease and biochemical improvement after treatment by bone marrow transplantation. Lancet ii: 709–712Google Scholar
  2. 2.
    Wagemaker G (1985) Hemopoietic cell differentiation. In: Van Bekkum DW, Lowenberg B (eds) Bone marrow transplantation: biological mechanisms and clinical practice. Dekker, New York, pp 1–72Google Scholar
  3. 3.
    Fischer A, Landais P, Friedrich W et al (1990) European experience of bone-marrow transplantation for severe combined immunodeficiency. Lancet 336: 850–854PubMedCrossRefGoogle Scholar
  4. 4.
    Frederickson DS, Sloan HR (1972) gluco-csylceramide lipidoses: Gaucher disease. In: Stanbury JB, Wyngaarden JB, Fredrickson DS, et al (eds) The metabolic basis of inherited diseases, 3rd edn. McGraw-Hill, New York, pp. 730–759Google Scholar
  5. 5.
    Olsen I, Muir H, Smith R, Fensom A, Watt DJ (1983) Direct enzyme transfer from lymphocytes is specific. Nature 306: 75–77PubMedCrossRefGoogle Scholar
  6. 6.
    Neufeld EF (1980) The uptake of enzymes into ly-sosomes. In: Birth defects: original article series, vol XVI. March of Dimes Birth Defects Foundation, pp 77–84Google Scholar
  7. 7.
    Hoogerbrugge PM, Suzuki K, Suzuki K, Poorthuis BJHM, Kobayashi T, Wagemaker G, van Bekkum DW (1988) Donor-derived cells in the central nervous system of twitcher mice after bone marrow transplantation. Science 239: 1035–1038PubMedCrossRefGoogle Scholar
  8. 8.
    Shull RM, Hastings NE, Selcer RR, Jones JB, Smith JR, Cullen WC, Constantopoulos G (1987) Bone marrow transplantation in canine mucopolysaccharidosis I: effects within the central nervous system. J Clin Invest 79: 435–443PubMedCrossRefGoogle Scholar
  9. 9.
    Hoogerbrugge PM, Brouwer OF, Fischer A (1993) Allogeneic bone marrow transplantation ( BMT) in metabolic diseases. Blood 82: 344aGoogle Scholar
  10. 10.
    Downie C, Hanckock MR, Hobbs JR (1992) Long term outcome after BMT for Hurler’s syndrome. In: Hobbs JR, Riches PG (eds) Correction of certain genetic diseases by transplantation. Cogent Trust, Middlesex, pp 1–13Google Scholar
  11. 11.
    Krivit W, Whitley CB, Chang P, Balzar B, Filipovich A, Kim T, Shapiro E, Ramsay N, Kersey J (1989) Lysosomal storage diseases treated by bone marrow transplantation. In: Gale RP, Champlin R (eds) Bone marrow transplantation: current controversions. Liss, New York, pp 367–378Google Scholar
  12. 12.
    Krivit W, Shapiro E (1994) Bone marrow transplantation for storage diseases: the plan and need for a control study. In: Forman S, Thomas ED, Blume K (eds) Bone marrow transplantation. Blackwell Scientific, LondonGoogle Scholar
  13. 13.
    Krivit W, Shapiro E (1991) Bone marrow transplantation for storage diseases. In: Desnick RJ (ed) Treatment of genetic diseases. Livingstone, New York, pp 203–221Google Scholar
  14. 14.
    Krivit W, Pierpont ME, Ayaz K, Tsai M, Ramsay NKC, Kersey JH, Weisdorf S, Sibley R, Snover D, McGovern MM, Schwartz MF, Desnick RJ (1984) Bone marrow transplantation in the Marateaux- Lamy syndrome (mucopolysaccharidosis VI). N Engl J Med 311: 1606–1611PubMedCrossRefGoogle Scholar
  15. 15.
    Ringden O, Groth CG, Erikson A, Bäckman L, Granquist S, Mänsson J, Svennerholm L (1988) Long term follow up of the first successful bone marrow transplantation in Gaucher disease. Transplantation 46: 66–70PubMedCrossRefGoogle Scholar
  16. 16.
    Erikson A, Groth CG, Mänsson JE, Percy A, Ringden, O, Svennerholm L (1990) Clinical and biochemical outcome of marrow transplantation forGoogle Scholar
  17. Gaucher disease of the Norrbottnian type. Acta Pediatr Scand 79: 680–685Google Scholar
  18. 17.
    Hobbs JR, Hugh-Jones K, Shaw PJ, Lindsay I, Hancock M (1987) Beneficial effect of pre-trans-plant splenectomy on displacement bone marrow transplantation for Gaucher’s syndrome. Lancet i: 1111–1115Google Scholar
  19. 18.
    Barton NW, Furbish FS, Myrray GJ, Garfield M, Brady RO (1990) Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease. Proc Natl Acad Sci U S A 87: 1913–1916PubMedCrossRefGoogle Scholar
  20. 19.
    Tsai P, Lipton JM, Sahdev I, Najfeld V, Rankin LR, Slyper AH, Ludman M, Grabowski GA (1992) Allogenic bone marrow transplantation in severe Gaucher disease. Pediatr Res 31: 503–507PubMedCrossRefGoogle Scholar
  21. 20.
    Krivit W, Shapiro E, Kennedy E, Lipton ME, Lockman LA, Smith S, Summers G, Wenger DA, Tsai M, Ramsay N, Kersey J, Yao J, Kaye E (1990) Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation. N Engl J Med 322: 28–32PubMedCrossRefGoogle Scholar
  22. 21.
    Bayever E, Ladisch S, Phillipart M, Brill N, Nuwer M, Sparkes RS, Feig SA (1985) Bone marrow transplantation for metachromatic leukodystrophy. Lancet ii: 471–473.Google Scholar
  23. 22.
    Ladisch S, Bayever E, Phillipart M, Feig SA (1986) Biochemical findings after bone marrow transplantation for metachromatic leukodystrophy: a preliminary report. In: Krivit W, Paul NW (eds) Bone marrow transplantation for treatment of lysosomal storage diseases. Liss, New York, pp 69–76 (March of Dimes Birth Defects Foundation original article series, vol 22, 1 )Google Scholar
  24. 23.
    Vellodi A, Hobbs JR, O’Donnell NM, Coulter BS, Hugh-Jones K (1987) Treatment of Niemann-Pick disease type B by allogeneic bone marrow transplantation. Br Med J 295: 1375–1376CrossRefGoogle Scholar
  25. 24.
    Bayever E, Kamani N, Machin GA et al (1992) Bone marrow transplantation for Niemann-Pick type IA disease. J Inherited Metab Dis 15: 919–928PubMedCrossRefGoogle Scholar
  26. 25.
    Shapiro EG, Lockman L, Kennedy W et al (1991) Bone marrow transplantation as treatment for globoid cell leukodystrophy. In: Desnick RJ (ed) Treatment of genetic diseases. Livingstone, New York, pp 223–238Google Scholar
  27. 26.
    Hobbs JR (1988) Displacement bone marrow transplantation and immunoprophylaxis for genetic disease. Adv Intern Med 33: 81–118PubMedGoogle Scholar
  28. 27.
    Watson JG, Medwin DG, Goldfinch ME, Pearson AD J (1986) Bone marrow transplantation for Pompe’s disease. N Engl J Med 314: 385PubMedGoogle Scholar
  29. 28.
    Harris RE, Hannon D, Vogler C, Hug G (1986) Bone marrow transplantation in type II glycogen storage disease. Birth Defects 22 (1): 119–132.PubMedGoogle Scholar
  30. 29.
    Hoogerbrugge PM, Poorthuis BJHM, Wagemaker G, van Bekkum DW, Suzuki K (1989) Bone marrow transplantation in twitcher mice: effects in the central nervous system. In: Gale RP, Champlin R (eds) Bone marrow transplantation: current controversions. Liss, New York, pp 331–336Google Scholar
  31. 30.
    Taylor RM, Farrow BRH, Stewart GJ, Healy PJ, Tiver K (1987) Lysosomal enzyme replacement in neural tissue by allogeneic bone marrow trans- plantation following total lymphoid irradiation in canine fucosidosis. Transplant Proc 19: 2730–2734Google Scholar
  32. 31.
    Sakiyama T, Tsuda M, Owada M, Joh K, Miyawaki S, Kitagawa T (1983) Bone marrow transplantation for Niemann-Pick mice. Biochem Biophys Res428Commun 113: 605–610Google Scholar

Copyright information

© Springer-Verlag Berlin Heidelberg 1995

Authors and Affiliations

  • P. M. Hoogerbrugge
  • J. M. J. J. Vossen

There are no affiliations available

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