• Klinische Pädiatrie · May 2004

    Review

    Hematopoietic stem cell transplantation for mucopolysaccharidoses and leukodystrophies.

    • M Sauer, S Grewal, and C Peters.
    • Division of Pediatric Hematology/Oncology, Blood and Marrow Transplantation, University of Minnesota, Minneapolis, Minnesota, USA. sauer.martin@mh-hannover.de
    • Klin Padiatr. 2004 May 1; 216 (3): 163-8.

    AbstractThe only effective treatment for selected metabolic diseases is a successful allogeneic hematopoietic stem cell transplantation (HCT). Best results with HCT are obtained when performed early in the course of the disease. Hence, timely identification and referral are critical. Also, early identification of affected patients during the newborn period via screening may be invaluable, particularly for the infantile onset rapidly progressive forms of diseases. Rapid availability of a donor graft is often crucial for these patients. Preliminary experience suggests that results after umbilical cord blood (UCB) transplant may be comparable to those after marrow transplants. UCB grafts have certain benefits of rapid availability and even reduced risk of GvHD. Hence, UCB transplant represents an alternative to marrow HCT. Related haploidentical HCT, possibly with very high doses of CD34+ cells, may also represent an option. However, expertise has been developed in very few transplant centers and no large reports are available of its use for patients with inherited metabolic diseases.

      Pubmed     Full text   Copy Citation     Plaintext  

      Add institutional full text...

    Notes

     
    Knowledge, pearl, summary or comment to share?
    300 characters remaining
    help        
    You can also include formatting, links, images and footnotes in your notes
    • Simple formatting can be added to notes, such as *italics*, _underline_ or **bold**.
    • Superscript can be denoted by <sup>text</sup> and subscript <sub>text</sub>.
    • Numbered or bulleted lists can be created using either numbered lines 1. 2. 3., hyphens - or asterisks *.
    • Links can be included with: [my link to pubmed](http://pubmed.com)
    • Images can be included with: ![alt text](https://bestmedicaljournal.com/study_graph.jpg "Image Title Text")
    • For footnotes use [^1](This is a footnote.) inline.
    • Or use an inline reference [^1] to refer to a longer footnote elseweher in the document [^1]: This is a long footnote..

    hide…