کد مقاله | کد نشریه | سال انتشار | مقاله انگلیسی | نسخه تمام متن |
---|---|---|---|---|
5524251 | 1546245 | 2017 | 8 صفحه PDF | دانلود رایگان |
- Haploidentical transplantation using PT-CY expands access for FA patients without matched related or unrelated donors.
- High rates of hemorrhagic cystitis and viral reactivation were found.
- Haploidentical transplant using PT-CY results in acceptable overall survival comparable with other donor sources.
We describe haploidentical bone marrow transplantation with post-transplant cyclophosphamide (PT-CY) for 30 patients with Fanconi anemia (FA). Twenty-six patients were transplanted upfront, and the preparatory regimens included fludarabine 150âmg/m2â+âtotal body irradiation 200 to 300 cGy ± CY 10âmg/kg without (nâ=â12) or with rabbit antithymocyte globulin (r-ATG) 4 to 5âmg/kg (nâ=â14). Four patients were rescued after primary or secondary graft failure after related or unrelated donor transplantation with the above regimen with (nâ=â2) or without r-ATG (nâ=â2). PT-CY at 25âmg/kg/day (total dose, 50âmg/kg) followed by cyclosporine and mycophenolate mofetil was given to all patients. All patients engrafted in the subgroup of patients who did not receive r-ATG (nâ=â14), but their transplant course was complicated by high rates of acute and chronic graft-versus-host disease (GVHD), and only 8 patients are alive. In the subgroup that received r-ATG (nâ=â16), 14 patients had sustained engraftment, severe GVHD rates were lower, and 13 patients are alive. Hemorrhagic cystitis occurred in 50% of patients, whereas cytomegalovirus reactivation occurred in 75%. One-year overall survival for the entire cohort was 73% (95% CI, 64% to 81%), and all surviving patients achieved full donor chimerism. In conclusion, haploidentical donor transplantation with PT-CY is a suitable option for FA patients without a matched related or unrelated donor.
Journal: Biology of Blood and Marrow Transplantation - Volume 23, Issue 2, February 2017, Pages 310-317