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

Autor: Giorgia Levati, Matteo Carrabba, Chiara Bonini, Luca Vago, Fabio Giglio, Fabio Ciceri, Lara Crucitti, Francesca Lorentino, Andrea Assanelli, Laura Bellio, Raffaella Milani, Sarah Marktel, Massimo Bernardi, Maria Teresa Lupo Stanghellini, Consuelo Corti, Raffaella Greco, Tiago De Freitas, Nicoletta Cieri, Jacopo Peccatori, Mara Morelli
Přispěvatelé: Cieri, N, Greco, R, Crucitti, L, Morelli, M, Giglio, F, Levati, G, Assanelli, A, Carrabba, Mg, Bellio, L, Milani, R, Lorentino, F, Lupo Stanghellini, Mt, De Freitas, T, Marktel, S, Bernardi, M, Corti, C, Vago, L, Bonini, MARIA CHIARA, Ciceri, Fabio, Peccatori, J.
Rok vydání: 2015
Předmět:
Adult
Male
Oncology
medicine.medical_specialty
Transplantation Conditioning
Cyclophosphamide
Haploidentical transplantation
Post transplantation cyclophosphamide
medicine.medical_treatment
Hematopoietic stem cell transplantation
Treosulfan
Peripheral Blood Stem Cells
Disease-Free Survival
Cohort Studies
Young Adult
Internal medicine
Antineoplastic Combined Chemotherapy Protocols
medicine
Humans
Transplantation
Homologous

Post-transplantation cyclophosphamide
Busulfan
Aged
Aged
80 and over

Sirolimus
Peripheral Blood Stem Cell Transplantation
Transplantation
Antibiotics
Antineoplastic

business.industry
Myeloablative conditioning
Hematopoietic Stem Cell Transplantation
Hematology
Middle Aged
Myeloablative Agonists
Allogeneic stem cell transplantation
Treatment Outcome
surgical procedures
operative

medicine.anatomical_structure
Immunology
Female
Bone marrow
business
medicine.drug
Popis: 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/mu L 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. (C) 2015 American Society for Blood and Marrow Transplantation.
Databáze: OpenAIRE