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Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.

Publication ,  Journal Article
Lund, TC; Cathey, SS; Miller, WP; Eapen, M; Andreansky, M; Dvorak, CC; Davis, JH; Dalal, JD; Devine, SM; Eames, GM; Ferguson, WS; Giller, RH ...
Published in: Biol Blood Marrow Transplant
November 2014

Mucolipidosis type II (MLII), or I-cell disease, is a rare but severe disorder affecting localization of enzymes to the lysosome, generally resulting in death before the 10th birthday. Although hematopoietic stem cell transplantation (HSCT) has been used to successfully treat some lysosomal storage diseases, only 2 cases have been reported on the use of HSCT to treat MLII. For the first time, we describe the combined international experience in the use of HSCT for MLII in 22 patients. Although 95% of the patients engrafted, overall survival was low, with only 6 patients (27%) alive at last follow-up. The most common cause of death post-transplant was cardiovascular complications, most likely due to disease progression. Survivors were globally delayed in development and often required complex medical support, such as gastrostomy tubes for nutrition and tracheostomy with mechanical ventilation. Although HSCT has demonstrated efficacy in treating some lysosomal storage disorders, the neurologic outcome and survival for patents with MLII were poor. Therefore, new medical and cellular therapies should be sought for these patients.

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Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

November 2014

Volume

20

Issue

11

Start / End Page

1847 / 1851

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • Surveys and Questionnaires
  • Mucolipidoses
  • Infant
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Data Collection
  • Child, Preschool
 

Citation

APA
Chicago
ICMJE
MLA
NLM
Lund, T. C., Cathey, S. S., Miller, W. P., Eapen, M., Andreansky, M., Dvorak, C. C., … Orchard, P. J. (2014). Outcomes after hematopoietic stem cell transplantation for children with I-cell disease. Biol Blood Marrow Transplant, 20(11), 1847–1851. https://doi.org/10.1016/j.bbmt.2014.06.019
Lund, Troy C., Sara S. Cathey, Weston P. Miller, Mary Eapen, Martin Andreansky, Christopher C. Dvorak, Jeffrey H. Davis, et al. “Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.Biol Blood Marrow Transplant 20, no. 11 (November 2014): 1847–51. https://doi.org/10.1016/j.bbmt.2014.06.019.
Lund TC, Cathey SS, Miller WP, Eapen M, Andreansky M, Dvorak CC, et al. Outcomes after hematopoietic stem cell transplantation for children with I-cell disease. Biol Blood Marrow Transplant. 2014 Nov;20(11):1847–51.
Lund, Troy C., et al. “Outcomes after hematopoietic stem cell transplantation for children with I-cell disease.Biol Blood Marrow Transplant, vol. 20, no. 11, Nov. 2014, pp. 1847–51. Pubmed, doi:10.1016/j.bbmt.2014.06.019.
Lund TC, Cathey SS, Miller WP, Eapen M, Andreansky M, Dvorak CC, Davis JH, Dalal JD, Devine SM, Eames GM, Ferguson WS, Giller RH, He W, Kurtzberg J, Krance R, Katsanis E, Lewis VA, Sahdev I, Orchard PJ. Outcomes after hematopoietic stem cell transplantation for children with I-cell disease. Biol Blood Marrow Transplant. 2014 Nov;20(11):1847–1851.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

November 2014

Volume

20

Issue

11

Start / End Page

1847 / 1851

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • Surveys and Questionnaires
  • Mucolipidoses
  • Infant
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Data Collection
  • Child, Preschool