Emerging trends in transplantation of inherited metabolic diseases.
Publication
, Journal Article
Prasad, VK; Kurtzberg, J
Published in: Bone Marrow Transplant
January 2008
Allogeneic hematopoietic stem cell transplantation (HSCT) can prolong life and improve its quality in patients with inherited metabolic diseases. HSCT offers a permanent source of enzyme replacement therapy and also might mediate nonhematopoietic cell regeneration or repair. Unrelated cord blood is an exciting newer graft source for treatment of patients with these fatal disorders, providing increased access to donors and significant clinical efficacy, particularly when transplantation is performed in early stages. Pre-transplant performance status is highly predictive of overall survival.
Duke Scholars
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Published In
Bone Marrow Transplant
DOI
ISSN
0268-3369
Publication Date
January 2008
Volume
41
Issue
2
Start / End Page
99 / 108
Location
England
Related Subject Headings
- Treatment Outcome
- Metabolism, Inborn Errors
- Kaplan-Meier Estimate
- Infant, Newborn
- Infant
- Immunology
- Humans
- Hematopoietic Stem Cell Transplantation
- Graft Survival
- Cord Blood Stem Cell Transplantation
Citation
APA
Chicago
ICMJE
MLA
NLM
Prasad, V. K., & Kurtzberg, J. (2008). Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant, 41(2), 99–108. https://doi.org/10.1038/sj.bmt.1705970
Prasad, V. K., and J. Kurtzberg. “Emerging trends in transplantation of inherited metabolic diseases.” Bone Marrow Transplant 41, no. 2 (January 2008): 99–108. https://doi.org/10.1038/sj.bmt.1705970.
Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99–108.
Prasad, V. K., and J. Kurtzberg. “Emerging trends in transplantation of inherited metabolic diseases.” Bone Marrow Transplant, vol. 41, no. 2, Jan. 2008, pp. 99–108. Pubmed, doi:10.1038/sj.bmt.1705970.
Prasad VK, Kurtzberg J. Emerging trends in transplantation of inherited metabolic diseases. Bone Marrow Transplant. 2008 Jan;41(2):99–108.
Published In
Bone Marrow Transplant
DOI
ISSN
0268-3369
Publication Date
January 2008
Volume
41
Issue
2
Start / End Page
99 / 108
Location
England
Related Subject Headings
- Treatment Outcome
- Metabolism, Inborn Errors
- Kaplan-Meier Estimate
- Infant, Newborn
- Infant
- Immunology
- Humans
- Hematopoietic Stem Cell Transplantation
- Graft Survival
- Cord Blood Stem Cell Transplantation