Improved Metabolic Correction in Patients with LSD Treated with Hematopoietic Stem Cell Transplant

2 Apr by Mommy

Improved Metabolic Correction in Patients with Lysosomal Storage Disease Treated with Hematopoietic Stem Cell Transplant Compared with Enzyme Replacement Therapy
Robert F. Wynn, MD, J. Ed Wraith, FRCPCH, Jean Mercer, Anne O’Meara, FRCPI, Karen Tylee, BSc, Margaret Thornley, Heather J. Church, PhD, Brian W. Bigger, PhD

 

Received 22 July 2008; received in revised form 12 September 2008; accepted 3 November 2008.

We compared substrate reduction in patients with lysosomal storage disorder treated with hematopoietic stem cell transplant and found that it was significantly reduced compared with patients treated with pharmacological enzyme replacement therapy. These data might support the wider application of hematopoietic stem cell transplant in the treatment of lysosomal storage disorders.

~ Filed Under: Gaucher's Disease

Comments

  1. Cristina says:
    04/02/2009 at 2:10 pm

    Were you able to get a full copy of that text? I can probably get it for you if you don’t have it. It sounds interesting.

    As I imagine you’ve been doing, we’ve also been looking at all the available LSD treatments for Bertrand. Since there are so few for which an enzyme therapy exists (Gaucher’s is lucky in that regard) we’re doing the evaluation for stem cell transplant at Duke next week. Just in case.

    There are so many cases in which we wouldn’t pursue such a drastic treatment and only a few in which we would. For us it feels good to be ready. As parents, we’re all just trying to do for our kids as we would like done to us (if we were in their shoes). 🙂

    Cristina’s last blog post..Bertrand Gets Adopted

  2. heather says:
    04/02/2009 at 3:11 pm

    Could be promising?