Dose-adapted post-transplant cyclophosphamide for HLA-haploidentical transplantation in Fanconi anemia

作者：Thakar M S^*; Bonfim C; Walters M C; Storb R; Pasquini R; Burroughs L; Sandmaier B M; Woolfrey A; Kiem H P

来源：Bone Marrow Transplantation, 2017, 52(4): 570-573.

DOI：10.1038/bmt.2016.301

We developed a haploidentical transplantation protocol with post-transplant cyclophosphamide (CY) for in vivo T-cell depletion (TCD) using a novel adapted-dosing schedule (25 mg/kg on days +3 and +4) for Fanconi anemia (FA). With median follow-up of 3 years (range, 37 days to 6.2 years), all six patients engrafted. Two patients with multiple pre-transplant comorbidities died, one from sepsis and one from sepsis with associated chronic GVHD. Four patients without preexisting comorbidities and early transplant referrals are alive with 100% donor chimerism and excellent performance status. We conclude that adjusted-dosing post-transplant CY is effective in in vivo TCD to promote full donor engraftment in patients with FA.

• 出版日期2017-4