Abstract
This study evaluated the safety and efficacy of thiotepa-based regimens before allogeneic stem cell transplantation in 310 adult patients with AML. Disease status at the time of transplantation was CR1 in 50%, CR2+ in 23.5% and advanced disease in 26.5%. Transplantation was performed from haploidentical (35%), matched sibling (27%), unrelated (20%) or cord blood (18%) donors. As for safety: mucositis occurred in 46.8% of the patients and the cumulative incidence (CI) of sinusoidal obstruction syndrome was 4.0%. With a median follow-up of 37 months, the CI of acute GvHD grade>II was 26.5%, whereas CI of chronic GvHD was 28.1% at 3 years. CI for non-relapse mortality at 3 years was 38.4%, 49.7% and 45.4% for patients in CR1, CR2+ and advanced disease, respectively (P=0.10). Relapse incidence at 3 years was 20.2, 30.7 and 40.6% in these three respective groups (P=0.002). CI for 3-year leukemia-free survival and overall survival were 41.4% and 45.6% (CR1), 19.6% and 27.7% (CR2+), and 13.9% and 13.6% (advanced disease), respectively (P<10−4 for both). Our data suggest that thiotepa-based conditioning therapy in AML is feasible, effective and safe, as investigated for sinusoidal obstruction syndrome and mucositis.
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Acknowledgements
We thank all national registries, all EBMT centers and all data managers for their great work and help. See Supplementary Appendix for list of institutions reporting data included in this study.
Author contributions
SE, MM, AN and ML designed the study. ML collected the data from the database of the ALWP of the EBMT (chaired by MM and AN). JF, DB, AO, SS, AR, LK and GM were the principal investigators of the seven highest recruiting centers of the study. ML, SE, MM and AN analyzed and interpreted the data. SE, ML, MM and AN wrote or revised the manuscript, and all authors reviewed its final version.
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MM received lectures honoraria and research support from Riemser whose product is discussed in this work. The remaining authors declare no conflict of interest.
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Eder, S., Labopin, M., Finke, J. et al. Safety and efficacy of thiotepa-based conditioning for allogeneic transplantation in AML: a survey from the ALWP of the EBMT. Bone Marrow Transplant 52, 238–244 (2017). https://doi.org/10.1038/bmt.2016.239
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DOI: https://doi.org/10.1038/bmt.2016.239
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