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Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.

Publication ,  Journal Article
Staba, SL; Escolar, ML; Poe, M; Kim, Y; Martin, PL; Szabolcs, P; Allison-Thacker, J; Wood, S; Wenger, DA; Rubinstein, P; Hopwood, JJ ...
Published in: N Engl J Med
May 6, 2004

BACKGROUND: Hurler's syndrome (the most severe form of mucopolysaccharidosis type I) causes progressive deterioration of the central nervous system and death in childhood. Allogeneic bone marrow transplantation before the age of two years halts disease progression and prolongs life, but many children lack a bone marrow donor. We investigated the feasibility of using cord-blood transplants from unrelated donors and a myeloablative preparative regimen that did not involve total-body irradiation in young children with Hurler's syndrome. METHODS: Between December 1995 and October 2002, 20 consecutive children with Hurler's syndrome received busulfan, cyclophosphamide, and antithymocyte globulin before receiving cord-blood transplants from unrelated donors. The children were subsequently evaluated for engraftment, adverse effects, and effects on disease symptoms. RESULTS: Cord-blood donors had normal alpha-L-iduronidase activity (mean number of cells, 10.53x10(7) per kilogram of body weight) and were discordant for up to three of six HLA markers. Neutrophil engraftment occurred a median of 24 days after transplantation. Five patients had grade II or grade III acute graft-versus-host disease; none had extensive chronic graft-versus-host disease. Seventeen of the 20 children were alive a median of 905 days after transplantation, with complete donor chimerism and normal peripheral-blood alpha-L-iduronidase activity (event-free survival rate, 85 percent). Transplantation improved neurocognitive performance and decreased somatic features of Hurler's syndrome. CONCLUSIONS: Cord blood from unrelated donors appears to be an excellent source of stem cells for transplantation in patients with Hurler's syndrome. Sustained engraftment can be achieved without total-body irradiation. Cord-blood transplantation favorably altered the natural history of Hurler's syndrome and thus may be important to consider in young children with this form of the disease.

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Published In

N Engl J Med

DOI

EISSN

1533-4406

Publication Date

May 6, 2004

Volume

350

Issue

19

Start / End Page

1960 / 1969

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Neutrophils
  • Mucopolysaccharidosis I
  • Methylprednisolone
  • Male
  • Infant, Newborn
  • Infant
  • Immunosuppressive Agents
  • Iduronidase
  • Humans
 

Citation

APA
Chicago
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MLA
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Staba, S. L., Escolar, M. L., Poe, M., Kim, Y., Martin, P. L., Szabolcs, P., … Kurtzberg, J. (2004). Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med, 350(19), 1960–1969. https://doi.org/10.1056/NEJMoa032613
Staba, Susan L., Maria L. Escolar, Michele Poe, Young Kim, Paul L. Martin, Paul Szabolcs, June Allison-Thacker, et al. “Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.N Engl J Med 350, no. 19 (May 6, 2004): 1960–69. https://doi.org/10.1056/NEJMoa032613.
Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, et al. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004 May 6;350(19):1960–9.
Staba, Susan L., et al. “Cord-blood transplants from unrelated donors in patients with Hurler's syndrome.N Engl J Med, vol. 350, no. 19, May 2004, pp. 1960–69. Pubmed, doi:10.1056/NEJMoa032613.
Staba SL, Escolar ML, Poe M, Kim Y, Martin PL, Szabolcs P, Allison-Thacker J, Wood S, Wenger DA, Rubinstein P, Hopwood JJ, Krivit W, Kurtzberg J. Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med. 2004 May 6;350(19):1960–1969.

Published In

N Engl J Med

DOI

EISSN

1533-4406

Publication Date

May 6, 2004

Volume

350

Issue

19

Start / End Page

1960 / 1969

Location

United States

Related Subject Headings

  • Transplantation Conditioning
  • Neutrophils
  • Mucopolysaccharidosis I
  • Methylprednisolone
  • Male
  • Infant, Newborn
  • Infant
  • Immunosuppressive Agents
  • Iduronidase
  • Humans