• Rémy Duléry, Juliana Bastos, Annalisa Paviglianiti, Florent Malard, Eolia Brissot, Giorgia Battipaglia, Clémence Médiavilla, Federica Giannotti, Anne Banet, Zoé Van de Wyngaert, Tounes Ledraa, Ramdane Belhocine, Simona Sestili, Rosa Adaeva, Simona Lapusan, Françoise Isnard, Ollivier Legrand, Anne Vekhoff, Marie-Thérèse Rubio, Annalisa Ruggeri, and Mohamad Mohty.
• Department of Hematology and Cellular Therapy, Saint Antoine Hospital, AP-HP, Paris, France; INSERM, UMR 938, Paris, France; Sorbonne Universités, Université Pierre et Marie Curie Paris 6, Paris, France.
• Biol. Blood Marrow Transplant. 2019 Jul 1; 25 (7): 1407-1415.

AbstractWe report the outcomes of 51 patients who underwent unmanipulated haploidentical hematopoietic stem cell transplantation (haplo-HSCT) with post-transplantation cyclophosphamide (PT-Cy) and antithymocyte globulin (ATG), from peripheral blood stem cells (PBSCs) or bone marrow, after receipt of a TBF (thiotepa, busulfan, and fludarabine) conditioning regimen. Their median age was 55 years (range, 16 to 72 years). Hematologic diagnoses included acute leukemias (n = 31), lymphoid neoplasm (n = 12), myeloproliferative neoplasm (n = 5), and myelodysplastic syndromes (n = 3). Thirty-seven patients (73%) were in complete remission. Graft-versus-host disease (GVHD) prophylaxis consisted of cyclosporine and mycophenolate for all patients, associated with ATG in 39 patients (76.5%). The median time to neutrophil engraftment was 17 days (range, 12 to 34 days). The cumulative incidences of grade II-IV and grade III-IV acute GVHD were 27.5% and 14%, respectively. In patients receiving a PBSC graft and ATG prophylaxis, grade II-IV aGVHD occurred in 16% of patients. The use of ATG and a lower thiotepa dose (5 mg/kg versus 10 mg/kg) were associated with a reduced cumulative incidence of grade II-IV acute GVHD (P = .03 and .005, respectively). The 2-year cumulative incidence of chronic GVHD was 29% and was significantly reduced to 13% with the lower thiotepa dose (P = .002). After a median follow-up of 25 months (range, 12 to 62 months), the cumulative incidences of nonrelapse mortality, relapse, overall survival (OS), disease-free survival (DFS), and GVHD-free, relapse-free survival (GFRFS) were 20%, 22.5%, 67%, 58%, and 51%, respectively. Pretransplantation disease status (complete remission versus others) was the main factor associated with OS, DFS, and GFRFS. In conclusion, the TBF conditioning regimen is an appealing platform in the haplo-HSCT setting with PT-Cy in terms of engraftment rate, toxicity, and disease control. We found no benefit of a thiotepa dose of 10 mg/kg compared with a dose of 5 mg/kg. ATG reduced the risk of acute GVHD without comprising outcomes.Copyright © 2019. Published by Elsevier Inc.

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