Allogeneic cellular gene therapy in hemoglobinopathies--evaluation of @entity1583 in XXXX .

@entity1 @entity12497 @entity64 @entity3458 @entity5837 @entity1583 @entity432 @entity2395 @entity2530
Many @entity1 with @entity2395 have been cured with BMT since the first successful transplant in 1981. Allogeneic stem cell gene therapy is the only treatment option for @entity1 with @entity3458 ( @entity3458 ). A total of 11 @entity1 with a median age of 12 years (range, 2-16), affected by @entity3458 , received @entity1583 from HLA-identical, related donors following a myeloablative-conditioning regimen. Indications for transplantation were @entity432 , @entity5837 , @entity2530 , chronic RBC transfusions, or @entity64 . All @entity1 had sustained engraftment. One @entity1 became a stable mixed chimera with 25% of donor cells at 4 years after transplantation. One @entity1 died at 1 year after transplantation. The probability of survival, @entity3458 -free survival and TRM at 5 years after transplant were 90, 90 and 10%, respectively. All 10 surviving @entity1 remained free of any @entity3458 -related events after transplantation. In conclusion, these data confirm @entity12497 from a suitable HLA-matched, related donor should become the primary option for curing @entity1 with @entity3458 . There is an excellent survival rate and a return to normal life, free of @entity3458 -related events.
