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Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease.

Publication ,  Journal Article
Allewelt, H; Taskindoust, M; Troy, J; Page, K; Wood, S; Parikh, S; Prasad, VK; Kurtzberg, J
Published in: Biol Blood Marrow Transplant
November 2018

Allogeneic hematopoietic stem cell transplantation (HSCT) can retard the progression of early infantile Krabbe disease (EIKD). Superior outcomes are achieved if HSCT is performed before the onset of symptoms; however, little information is available about the long-term outcomes in surviving patients. We now describe functional outcomes in presymptomatic infants who underwent HSCT for EIKD at ≤ 2 months of age. Records of the 19 patients who underwent HSCT for EIKD at ≤ 2 months of age from 1996 to 2010 were reviewed. Long-term functional outcomes were compared between those transplanted at < 30 days and ≥ 30 days of life. Median age at transplant was 27 days (range, 19 to 61). Median follow-up of the cohort was 12.6 years. Overall survival at 5 and 10 years post-transplant was 84.2% (95% confidence interval, 58.7% to 94.6%) and 78.6% (95% confidence interval, 52.5% to 91.4%), respectively. More favorable outcomes were seen in patients who underwent HSCT at < 30 days of age, particularly in domains of mobility (P = .01), communication (P = .02), and feeding (P = .008). Improved functional outcomes were observed when HSCT was performed in the first month of life, defining a critical period for intervention. These results support the implementation of newborn screening to enable rapid diagnosis and early treatment of infants with EIKD.

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Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

November 2018

Volume

24

Issue

11

Start / End Page

2233 / 2238

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • Survival Analysis
  • Male
  • Leukodystrophy, Globoid Cell
  • Infant, Newborn
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Female
 

Citation

APA
Chicago
ICMJE
MLA
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Allewelt, H., Taskindoust, M., Troy, J., Page, K., Wood, S., Parikh, S., … Kurtzberg, J. (2018). Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease. Biol Blood Marrow Transplant, 24(11), 2233–2238. https://doi.org/10.1016/j.bbmt.2018.06.020
Allewelt, Heather, Mahsa Taskindoust, Jesse Troy, Kristin Page, Susan Wood, Suhag Parikh, Vinod K. Prasad, and Joanne Kurtzberg. “Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease.Biol Blood Marrow Transplant 24, no. 11 (November 2018): 2233–38. https://doi.org/10.1016/j.bbmt.2018.06.020.
Allewelt H, Taskindoust M, Troy J, Page K, Wood S, Parikh S, et al. Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease. Biol Blood Marrow Transplant. 2018 Nov;24(11):2233–8.
Allewelt, Heather, et al. “Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease.Biol Blood Marrow Transplant, vol. 24, no. 11, Nov. 2018, pp. 2233–38. Pubmed, doi:10.1016/j.bbmt.2018.06.020.
Allewelt H, Taskindoust M, Troy J, Page K, Wood S, Parikh S, Prasad VK, Kurtzberg J. Long-Term Functional Outcomes after Hematopoietic Stem Cell Transplant for Early Infantile Krabbe Disease. Biol Blood Marrow Transplant. 2018 Nov;24(11):2233–2238.
Journal cover image

Published In

Biol Blood Marrow Transplant

DOI

EISSN

1523-6536

Publication Date

November 2018

Volume

24

Issue

11

Start / End Page

2233 / 2238

Location

United States

Related Subject Headings

  • Treatment Outcome
  • Transplantation Conditioning
  • Survival Analysis
  • Male
  • Leukodystrophy, Globoid Cell
  • Infant, Newborn
  • Immunology
  • Humans
  • Hematopoietic Stem Cell Transplantation
  • Female