Hematopoietic stem cell transplantation for XXXX .

@entity1 @entity8 @entity17053 @entity9764 @entity1141
UNASSIGNED: @entity17053 features @entity1141 . To date, there has been no curative therapy for this @entity1141 . No systemic report on a long-term effect of @entity8 ( @entity8 ) for Morquio A has been described. We conducted @entity8 for 4 cases with Morquio A (age at @entity8 : 4-15years, mean 10.5years) and followed them at least 10years (range 11-28years; mean 19years). Current age ranged between 25 and 36years of age (mean 29.5years). All cases had a successful full engraftment of allogeneic bone marrow transplantation without serious GVHD. Transplanted bone marrow derived from HLA-identical siblings (three cases) or HLA-identical unrelated donor. The levels of the enzyme activity in the recipient's lymphocytes reached the levels of donors' enzyme activities within two years after @entity8 . For the successive over 10years post-BMT, @entity9764 activity in lymphocytes was maintained at the same level as the donors. Except one case who had osteotomy in both legs one year later post BMT, other three cases had no orthopedic surgical intervention. All cases remained ambulatory, and three of them could walk over 400m. Activity of daily living (ADL) in @entity1 with @entity8 was better than untreated @entity1 . The @entity1 who underwent @entity8 at four years of age showed the best ADL score. In conclusion, the long-term study of @entity8 has demonstrated therapeutic effect in amelioration of progression of the disease in respiratory function, ADL, and biochemical findings, suggesting that @entity8 is a therapeutic option for @entity1 with Morquio A.
