TY - JOUR
T1 - Allogeneic hematopoietic cell transplantation using fludarabine plus myeloablative busulfan and melphalan confers promising survival in high-risk hematopoietic neoplasms
T2 - a single-center retrospective analysis
AU - Edahiro, Taro
AU - Kawase, Takakazu
AU - Nagoshi, Hisao
AU - Fujino, Keita
AU - Toishigawa, Kayo
AU - Miyama, Takahiko
AU - Mino, Tatsuji
AU - Yoshida, Tetsumi
AU - Morioka, Takehiko
AU - Hirata, Yuji
AU - Noma, Mitsunori
AU - Fujii, Teruhisa
AU - Nishizawa, Masatoshi
AU - Fukushima, Noriyasu
AU - Ichinohe, Tatsuo
N1 - Funding Information:
This study was supported in part by Grants-in-Aid from the Japan Agency for Medical Research and Development (AMED) [grant number #19ek0510022h0002, #19ek0510023h0003, and #19ek0510027h0001 to TI), and the Program of the network-type Joint Usage/Research Center for Radiation Disaster Medical Science of Hiroshima University, Nagasaki University, and Fukushima Medical University (to TI). We thank all of the staff in our department and members of Hiroshima University Hospital who dedicatedly provided the best care for patients in this study and their donors, with a special gratitude to compassionate hematopoietic cell transplant coordinators: Yuka Asano, Akihiro Osawa, and Kaho Ogawa. We also thank Sachiko Fukumoto, Nanae Nakaju, Sajeda Chowdhury, Emi Nakai, Sanae Furuya, and Masako Ninomiya for their excellent technical and secretarial assistance.
Funding Information:
The Next Generation Development of Genome and Cellular Therapy Program at RIRBM is cooperatively funded by Repertoire Genesis Inc. and Hiroshima University. TI has received speaker honoraria from Bristol-Myers Squibb, Celgene, Janssen Pharmaceutical K.K., and Kyowa Kirin Co. and research funding from Astellas Pharma, Chugai Pharmaceutical Co., CSL Behring, Eisai Co., FUJIFILM Wako Chemicals., Kyowa Kirin Co., Ono Pharmaceutical Co., Pfizer, Nippon Shinyaku Co., MSD, Otsuka Pharmaceutical Co., Repertoire Genesis Inc., Sumitomo Dainippon Pharma Co., Taiho Pharmaceutical Co., Takara Bio Inc., Takeda Pharmaceutical Co., Zenyaku Kogyo Co. No other authors have relevant conflict-of-interest to declare.
Publisher Copyright:
© 2021 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2021
Y1 - 2021
N2 - Objectives: Optimal selection of pretransplant conditioning is crucially vital for improving survival and quality-of-life of patients who receive allogeneic hematopoietic cell transplantation (allo-HCT), particularly in those with high-risk diseases. In this study, we evaluated the efficacy and safety of recently-developed reduced-toxicity myeloablative regimen that combines fludarabine, intravenous busulfan, and melphalan (FBM). Methods: We conducted a single-center retrospective analysis of 39 patients (23 with myeloid neoplasms and 16 with lymphoid neoplasms), with a median age of 50 (range, 17–68) years, who underwent their first allo-HCT using the FBM regimen. Graft types were bone marrow in 11, peripheral blood in 11, and cord blood in 17 patients. Cyclosporine- or tacrolimus-based graft-versus-host disease (GVHD) prophylaxis was administered. The primary end point of the study was the overall survival rate at 2-year after transplantation. Results: After a median follow-up of 910 days for the surviving patients, 2-year overall survival was 62% for the entire cohort; 73% in the low-to-intermediate-risk group and 44% in the high-to-very high-risk group classified by the refined CIBMTR Disease Risk Index. Cumulative incidences of engraftment, grade II-IV acute GVHD, chronic GVHD, relapse, and non-relapse mortality were 95%, 56%, 56%, 31%, and 17%, respectively. Conclusion: These results suggest that our FBM regimen can be applied to allo-HCT using various graft types and yields acceptable outcomes with relatively low non-relapse mortality in both myeloid and lymphoid neoplasms. Also, we observed a promising survival in the group of patients with high-risk diseases, warranting more accumulation of patients and longer follow-up.
AB - Objectives: Optimal selection of pretransplant conditioning is crucially vital for improving survival and quality-of-life of patients who receive allogeneic hematopoietic cell transplantation (allo-HCT), particularly in those with high-risk diseases. In this study, we evaluated the efficacy and safety of recently-developed reduced-toxicity myeloablative regimen that combines fludarabine, intravenous busulfan, and melphalan (FBM). Methods: We conducted a single-center retrospective analysis of 39 patients (23 with myeloid neoplasms and 16 with lymphoid neoplasms), with a median age of 50 (range, 17–68) years, who underwent their first allo-HCT using the FBM regimen. Graft types were bone marrow in 11, peripheral blood in 11, and cord blood in 17 patients. Cyclosporine- or tacrolimus-based graft-versus-host disease (GVHD) prophylaxis was administered. The primary end point of the study was the overall survival rate at 2-year after transplantation. Results: After a median follow-up of 910 days for the surviving patients, 2-year overall survival was 62% for the entire cohort; 73% in the low-to-intermediate-risk group and 44% in the high-to-very high-risk group classified by the refined CIBMTR Disease Risk Index. Cumulative incidences of engraftment, grade II-IV acute GVHD, chronic GVHD, relapse, and non-relapse mortality were 95%, 56%, 56%, 31%, and 17%, respectively. Conclusion: These results suggest that our FBM regimen can be applied to allo-HCT using various graft types and yields acceptable outcomes with relatively low non-relapse mortality in both myeloid and lymphoid neoplasms. Also, we observed a promising survival in the group of patients with high-risk diseases, warranting more accumulation of patients and longer follow-up.
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U2 - 10.1080/16078454.2021.1881228
DO - 10.1080/16078454.2021.1881228
M3 - Article
C2 - 33594942
AN - SCOPUS:85101093733
VL - 26
SP - 186
EP - 198
JO - Hematology
JF - Hematology
SN - 1024-5332
IS - 1
ER -