Post-transplantation Cyclophosphamide and Sirolimus after Haploidentical Hematopoietic Stem Cell Transplantation Using a Treosulfan-based Myeloablative Conditioning and Peripheral Blood Stem Cells

Nicoletta Cieri, Raffaella Greco, Lara Crucitti, Mara Morelli, Fabio Giglio, Giorgia Levati, Andrea Assanelli, Matteo G. Carrabba, Laura Bellio, Raffaella Milani, Francesca Lorentino, Maria Teresa Lupo Stanghellini, Tiago De Freitas, Sarah Marktel, Massimo Bernardi, Consuelo Corti, Luca Vago, Chiara Bonini, Fabio Ciceri, Jacopo Peccatori

Research output: Contribution to journalArticle


Haploidentical hematopoietic stem cell transplantation (HSCT) performed using bone marrow (BM) grafts and post-transplantation cyclophosphamide (PTCy) has gained much interest for the excellent toxicity profile after both reduced-intensity and myeloablative conditioning. We investigated, in a cohort of 40 high-risk hematological patients, the feasibility of peripheral blood stem cells grafts after a treosulfan-melphalan myeloablative conditioning, followed by a PTCy and sirolimus-based graft-versus-host disease (GVHD) prophylaxis (Sir-PTCy). Donor engraftment occurred in all patients, with full donor chimerism achieved by day 30. Post-HSCT recovery of lymphocyte subsets was broad and fast, with a median time to CD4 > 200/μL of 41 days. Cumulative incidences of grade II to IV and III-IV acute GVHD were 15% and 7.5%, respectively, and were associated with a significant early increase in circulating regulatory T cells at day 15 after HSCT, with values <5% being predictive of subsequent GVHD occurrence. The 1-year cumulative incidence of chronic GVHD was 20%. Nonrelapse mortality (NRM) at 100 days and 1 year were 12% and 17%, respectively. With a median follow-up for living patients of 15 months, the estimated 1-year overall and disease-free survival (DFS) was 56% and 48%, respectively. Outcomes were more favorable in patients who underwent transplantation in complete remission (1-year DFS 71%) versus patients who underwent transplantation with active disease (DFS, 34%; P=.01). Overall, myeloablative haploidentical HSCT with peripheral blood stem cells (PBSC) and Sir-PTCy is a feasible treatment option: the low rates of GVHD and NRM as well as the favorable immune reconstitution profile pave the way for a prospective comparative trial comparing BM and PBSC in this specific transplantation setting.

Original languageEnglish
Pages (from-to)1506-1514
Number of pages9
JournalBiology of Blood and Marrow Transplantation
Issue number8
Publication statusPublished - Aug 1 2015



  • Allogeneic stem cell transplantation
  • Haploidentical transplantation
  • Peripheral blood stem cell transplantation
  • Post-transplantation cyclophosphamide
  • Sirolimus

ASJC Scopus subject areas

  • Transplantation
  • Hematology

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