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Am J Hematol. 2017 Oct;92(10):997-1003. doi: 10.1002/ajh.24823. Epub 2017 Jul 19.

Thiotepa-based conditioning versus total body irradiation as myeloablative conditioning prior to allogeneic stem cell transplantation for acute lymphoblastic leukemia: A matched-pair analysis from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.

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EBMT Office Paris, Hôpital Saint-Antoine, Paris, France.
Hematology Division, Chaim Sheba Medical Center, Tel Aviv University, Tel-Hashomer, Israel.
Hospital Universitario La Fe, Servicio de Hematologia, Valencia, Spain.
Stem Cell Transplant Unit, Tor Vergata University of Rome, Policlinico Universitario Tor Vergata, Rome, Italy.
Department of Bone Marrow Transplantation, Hadassah University Hospital, Jerusalem, Israel.
Department of Medicine, Hematology, Oncology, University of Freiburg, Freiburg, Germany.
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico IRCCS, Milano, Italy.
Department of Bone Marrow Transplantation, University Hospital, Essen, Germany.
Department of Hematology, University Hospital, Hematology, Basel, Switzerland.
Department of Hematology-BMT, Hopital St. Louis, Paris, France.
Transplantation Unit, Department of Hematology Adult Stem Cell, Ankara University Faculty of Medicine, Ankara, Turkey.
King Faisal Specialist Hospital & Research Centre, Oncology (Section of Adult Haematolgy/BMT), Riyadh, Saudi Arabia.
Department of Hematology/Oncology, University of Münster, Münster, Germany.
Maria Sklodowska-Curie Memorial Cancer Center and Institute of Oncology, Gliwice, Poland.


The optimal conditioning regimen to employ before hematopoietic stem cell transplantation in acute lymphoblastic leukemia (ALL) is still undecided, and while cyclophosphamide/total body irradiation (Cy/TBI) is the most commonly used myeloablative regimen, there are concerns regarding long-term toxicity for patients conditioned with this regimen. Thiotepa-based conditioning is an emerging radiation-free regimen with recent publications indicative of comparable clinical outcomes to TBI-based conditioning. In this analysis of the acute leukemia working party of the EBMT, we performed a retrospective matched-pair analysis, evaluating the outcome of adult patients with ALL who received thiotepa-based conditioning (n = 180) with those receiving Cy/TBI conditioning (n = 540). The 2-year leukemia-free survival and overall survival (OS) rates of both conditioning regimens were comparable, 33% for thiotepa [95% confidence interval (CI): 26.4-42.8] versus 39% for Cy/TBI (95% CI: 34.8-44.5] (P = .33) and 46.5% [95% CI: 38.6-56.1] versus 48.8% [95% CI: 44.2-54] (P = .9), respectively. There was no significant difference between the two regimens in the incidence of either acute graft versus host disease (GVHD) or chronic GVHD. Multivariate analysis demonstrated increased relapse incidence for thiotepa conditioning compared to Cy/TBI (HR = 1.78, 95% CI, 1.07-2.95; P = .03) which did not affect OS. Our results indicate that thiotepa-based conditioning may not be inferior to Cy/TBI for adult patients with ALL.

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