Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Case Report
  • Published:

Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia

Bone Marrow Transplantation volume 25, pages 1093–1097 (2000)Cite this article

Abstract

This report describes unrelated umbilical cord blood transplantation for a 10-month-old infant boy with mucopolysaccharidosis IIB (Hunter syndrome), an X-linked metabolic storage disorder due to deficiency of iduronate sulfatase. Two years after transplant ∼55% normal plasma enzyme activity has been restored and abnormal urinary excretion of glycosaminoglycans has nearly completely resolved. The boy has exhibited normal growth and development after transplant. Nine months after transplant he developed severe autoimmune hemolytic anemia and required 14 months of corticosteroid treatment to prevent clinically significant anemia. Bone marrow transplantation for Hunter syndrome and post-transplant hemolytic anemia are reviewed. Bone Marrow Transplantation (2000) 25, 1093–1097.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

Similar content being viewed by others

References

  1. Neufeld EF, Muenzer J . The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The Metabolic Basis of Inherited Disease 6th edn. McGraw-Hill: New York 1995 pp2465–2494

    Google Scholar 

  2. Young ID, Harper PS, Newcombe RG, Archer IM . A clinical and genetic study of Hunter's syndrome. 2. Differences between the mild and severe forms J Med Genet 1982 19: 408–411

    Article  CAS  Google Scholar 

  3. Young ID, Harper PS . Incidence of Hunter's syndrome Hum Genet 1982 60: 391–392

    Article  CAS  Google Scholar 

  4. Young ID, Harper PS, Archer IM, Newcombe RG . A clinical and genetic study of Hunter's syndrome. 1. Heterogeneity J Med Genet 1982 19: 401–407

    Article  CAS  Google Scholar 

  5. Li P, Bellows AB, Thompson JN . Molecular basis of iduronate-2-sulphatase gene mutations in patients with mucopolysaccharidosis type II (Hunter syndrome) J Med Genet 1999 36: 21–27

    CAS  PubMed  PubMed Central  Google Scholar 

  6. Gort L, Chabas A, Coll MJ . Hunter disease in the Spanish population: molecular analysis in 31 families J Inher Metabol Dis 1998 21: 655–661

    Article  CAS  Google Scholar 

  7. Young ID, Harper PS . The natural history of the severe form of Hunter's syndrome: a study based on 52 cases Dev Med Child Neurol 1983 25: 481–489

    Article  CAS  Google Scholar 

  8. Young ID, Harper PS . Mild form of Hunter's syndrome: clinical delineation based on 31 cases Arch Dis Child 1982 57: 828–836

    Article  CAS  Google Scholar 

  9. McKinnis EJ, Sulzbacher S, Rutledge JC et al. Bone marrow transplantation in Hunter syndrome J Pediatr 1996 129: 145–148

    Article  CAS  Google Scholar 

  10. Li P, Thompson JN, Hug G et al. Biochemical and molecular analysis in a patient with the severe form of Hunter syndrome after bone marrow transplantation Am J Med Genet 1996 64: 531–535

    Article  CAS  Google Scholar 

  11. Vellodi A, Young E, Cooper A et al. Long-term follow-up following bone marrow transplantation for Hunter disease J Inher Metabol Dis 1999 22: 638–648

    Article  CAS  Google Scholar 

  12. 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

    Article  CAS  Google Scholar 

  13. Bergstrom SK, Quinn JJ, Greenstein R, Ascensao J . Long-term follow-up of a patient transplanted for Hunter's disease type IIB: a case report and literature review Bone Marrow Transplant 1994 14: 653–658

    CAS  PubMed  Google Scholar 

  14. Rubinstein P, Carrier C, Scaradavou A et al. Outcomes among 562 recipients of placental-blood transplants from unrelated donors New Engl J Med 1998 339: 1565–1577

    Article  CAS  Google Scholar 

  15. Chen FE, Owen I, Savage D et al. Late onset haemolysis and red cell autoimmunisation after allogeneic bone marrow transplant Bone Marrow Transplant 1997 19: 491–495

    Article  CAS  Google Scholar 

  16. Sherer Y, Shoenfeld Y . Autoimmune diseases and autoimmunity post-bone marrow transplantation Bone Marrow Transplant 1998 22: 873–881

    Article  CAS  Google Scholar 

  17. Sullivan KM, Witherspoon RP, Storb R et al. Prednisone and azathioprine compared with prednisone and placebo for treatment of chronic graft-v-host disease: prognostic influence of prolonged thrombocytopenia after allogeneic marrow transplantation Blood 1988 72: 546–554

    CAS  PubMed  Google Scholar 

  18. Hart DNJ, Fearnley DB . The effect of GvHD on the hematopoietic system. In: Ferrara JLM, Deeg HJ, Burakoff SJ (eds) Graft-vs.-Host Disease 2nd edn. Marcel Dekker: New York 1997 pp447–477

    Google Scholar 

  19. Drobyski WR, Potluri J, Sauer D, Gottschall JL . Autoimmune hemolytic anemia following T cell-depleted allogeneic bone marrow transplantation Bone Marrow Transplant 1996 17: 1093–1099

    CAS  PubMed  Google Scholar 

  20. De Lord C, Marsh JC, Smith JG et al. Fatal autoimmune pancytopenia following bone marrow transplantation for aplastic anaemia Bone Marrow Transplant 1996 18: 237–239

    CAS  PubMed  Google Scholar 

  21. Wang WC . Evans syndrome in childhood: pathophysiology, clinical course, and treatment Am J Pediatr Hematol Oncol 1988 10: 330–338

    Article  CAS  Google Scholar 

  22. Kottaridis PD, Rees H, Smith G et al. A fatal case of autoimmune thrombocytopenia with an IgM anti-GPIb/IX following one antigen mismatched unrelated donor bone marrow transplantation Bone Marrow Transplant 1999 23: 739–741

    Article  CAS  Google Scholar 

  23. Gilliland BC . Autoimmune hemolytic anemia. In: Rossi EC, Simon TL, Moss GS, Gould SA (eds) Principles of Transfusion Medicine 2nd edn: Williams & Wilkins: Baltimore 1996 pp101–120

    Google Scholar 

  24. Braun SE, Aronovich EL, Anderson RA et al. Metabolic correction and cross-correction of mucopolysaccharidosis type II (Hunter syndrome) by retroviral-mediated gene transfer and expression of human iduronate-2-sulfatase Proc Natl Acad Sci USA 1993 90: 11830–11834

    Article  CAS  Google Scholar 

  25. Stroncek DF, Hubel A, Shankar RA et al. Retroviral transduction and expansion of peripheral blood lymphocytes for the treatment of mucopolysaccharidosis type II, Hunter's syndrome Transfusion 1999 39: 343–350

    Article  CAS  Google Scholar 

  26. Thompson JN, Nowakowski RW . Enzymatic diagnosis of selected mucopolysaccharisoses: Hunter, Morquio, Type A, and Sanfilippo types A, B, C, and D and procedures for measurement of 35SO4-glycosaminoglycans. In: Hommes F (ed) Techniques in Diagnostic Human Biochemical Genetics: A Laboratory Manual John Wiley & Sons: New York 1991 567–586

    Google Scholar 

Download references

Acknowledgements

This work was supported in part by Clinical Oncology Career Development Award CDA-96-61 from the American Cancer Society (CAM).

Author information

Authors and Affiliations

  1. Department of Pediatrics, University of Texas MD Anderson Cancer Center, Houston, Texas, USA

    CA Mullen & KW Chan

  2. Department of Immunology, University of Texas MD Anderson Cancer Center, Houston, Texas, USA

    CA Mullen

  3. Laboratory of Medical Genetics, University of Alabama at Birmingham, Birmingham, Alabama, USA

    JN Thompson

Authors
  1. CA Mullen
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. JN Thompson
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. LA Richard
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. KW Chan
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Mullen, C., Thompson, J., Richard, L. et al. Unrelated umbilical cord blood transplantation in infancy for mucopolysaccharidosis type IIB (Hunter syndrome) complicated by autoimmune hemolytic anemia. Bone Marrow Transplant 25, 1093–1097 (2000). https://doi.org/10.1038/sj.bmt.1702397

Download citation

  • Received:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/sj.bmt.1702397

Keywords

This article is cited by

Search

Advanced search

Quick links