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

Bone Marrow Transplantation

Volumn 25, Issue 10, 2000, Pages 1093-1097

  Mullen, C A ^a,b   Richard, L A ^a   Chan, K W ^a   Thompson, J N ^c  

^a UNIVERSITY OF TEXAS   (United States)

^b Department of Immunology ^*  (United States)

^c UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

Author keywords

Autoimmune hemolytic anemia; Bone marrow transplantation; Hunter syndrome; Mucopolysaccharidosis

Indexed keywords

BUSULFAN; CORTICOSTEROID; CREATININE; CYCLOPHOSPHAMIDE; GLYCOSAMINOGLYCAN; HEXOSAMINE; IDURONATE 2 SULFATASE; MEPREDNISONE; METHOTREXATE; PREDNISONE; TACROLIMUS; THYMOCYTE ANTIBODY; URONIC ACID;

ARTICLE; AUTOIMMUNE HEMOLYTIC ANEMIA; CASE REPORT; CHILD GROWTH; ENZYME BLOOD LEVEL; GRAFT VERSUS HOST REACTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; HUNTER SYNDROME; INFANT; MALE; PRIORITY JOURNAL; UMBILICAL CORD BLOOD; URINARY EXCRETION;

ADRENAL CORTEX HORMONES; ANEMIA, HEMOLYTIC, AUTOIMMUNE; ANTIBODY SPECIFICITY; AUTOIMMUNE DISEASES; COOMBS' TEST; ETHICS, MEDICAL; FETAL BLOOD; GLYCOSAMINOGLYCANS; GRAFT VS HOST DISEASE; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; IDURONATE SULFATASE; IMMUNOGLOBULIN G; IMMUNOSUPPRESSIVE AGENTS; INFANT, NEWBORN; INFORMED CONSENT; MALE; MUCOPOLYSACCHARIDOSIS II; POINT MUTATION; TRANSPLANTATION, HOMOLOGOUS;

EID: 0034024729     PISSN: 02683369     EISSN: 14765365     Source Type: Journal    
DOI: 10.1038/sj.bmt.1702397     Document Type: Article

References (26)

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, pp 2465–2494.
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.
3. Young ID, Harper PS. Incidence of Hunter’s syndrome. Hum Genet 1982; 60: 391–392.
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.
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.
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.
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.
8. Young ID, Harper PS. Mild form of Hunter’s syndrome: clinical delineation based on 31 cases. Arch Dis Child 1982; 57: 828–836.
9. McKinnis EJ, Sulzbacher S, Rutledge JC et al. Bone marrow transplantation in Hunter syndrome. J Pediatr 1996; 129: 145–148.
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.
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.
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.
13. Bergstrom SK, Quinn JJ, Greenstein R, Ascensao J. Longterm 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.
14. Rubinstein P, Carrier C, Scaradavou A et al. Outcomes among recipients of placental-blood transplants from unrelated donors. New Engl J Med 1998; 339: 1565–1577.
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.
16. Sherer Y, Shoenfeld Y. Autoimmune diseases and autoimmunity post-bone marrow transplantation. Bone Marrow Transplant 1998; 22: 873–881.
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.
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, pp 447–477.
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.
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.
21. Wang WC. Evans syndrome in childhood: pathophysiology, clinical course, and treatment. Am J Pediatr Hematol Oncol 1988; 10: 330–338.
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.
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, pp 101–120.
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.
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.
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, pp 567–586.