title: Better transplant outcome with pre-transplant marrow response after hypomethylating treatment in higher-risk MDS with excess blasts.
abstract: Hypomethylating treatment (HMT) has been suggested as a feasible bridge to hematopoietic stem cell transplantation (HSCT), but controversies exist around influences of HMT response on transplant outcomes. To assess the safety and influences of pre-transplant HMT focusing on debulking effects and transplant outcomes, we retrospectively analyzed consecutive HSCT-eligible patients who received HMT for higher-risk MDS with excess blasts. Of all 98 patients, 11 patients failed to proceed to HSCT and HMT-related mortality occurred in 8 patients. When excluding 9 patients who refused HSCT, 87% of scheduled HSCT (77 of 89) was performed after a median of 3 cycles (range, 1-8) of HMT. The 4-year overall survival after HMT (n = 98) and HSCT (n = 77) was 44.0% and 53.6%, respectively. Transplant outcomes were significantly different by the final response at HSCT; marrow response group (complete remission, marrow complete remission with or without hematologic improvement) showed significantly better 4-year disease-free survival compared to no marrow response group (n = 36, 87.3% vs. n = 41, 10.7%, P < 0.001). This difference between the groups was also evident in overall survival (90.9% vs. 8.6%, P < 0.001) and cumulative incidences of relapse (6.5% vs. 45.4%, P < 0.001) and treatment-related mortality (6.2% vs. 43.9%, P < 0.001). These observations indicate that pre-transplant HMT is a feasible bridging treatment in patients with excess blasts regarding high success rate of proceeding to transplantation and good survival rate. Marrow response at HSCT regardless of concomitant hematological improvement is an independent predictor of better survival, suggesting that immediate HSCT rather than continuing HMT should be performed once marrow response is achieved.
authors.affiliation: Incheon St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Incheon, Korea.
authors.forename: Seung-Ah
authors.initials: SA
authors.lastname: Yahng
authors.affiliation: Department of Laboratory Medicine, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Myungshin
authors.initials: M
authors.lastname: Kim
authors.affiliation: Department of Medical Informatics, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Tae-Min
authors.initials: TM
authors.lastname: Kim
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Young-Woo
authors.initials: YW
authors.lastname: Jeon
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Jae-Ho
authors.initials: JH
authors.lastname: Yoon
authors.affiliation: Yeoido St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Seung-Hwan
authors.initials: SH
authors.lastname: Shin
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Sung-Eun
authors.initials: SE
authors.lastname: Lee
authors.affiliation: Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Ki-Seong
authors.initials: KS
authors.lastname: Eom
authors.affiliation: Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Seok
authors.initials: S
authors.lastname: Lee
authors.affiliation: Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Chang-Ki
authors.initials: CK
authors.lastname: Min
authors.affiliation: Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Hee-Je
authors.initials: HJ
authors.lastname: Kim
authors.affiliation: Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Dong-Wook
authors.initials: DW
authors.lastname: Kim
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Jong-Wook
authors.initials: JW
authors.lastname: Lee
authors.affiliation: Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Woo-Sung
authors.initials: WS
authors.lastname: Min
authors.affiliation: Leukemia Research Institute, College of Medicine, The Catholic University of Korea, Seoul, Korea.|Catholic Blood and Marrow Transplantation Center, Seoul St. Mary's Hospital, College of Medicine, The Catholic University of Korea, Seoul, Korea.
authors.forename: Yoo-Jin
authors.initials: YJ
authors.lastname: Kim
mesh_terms: D001374:Azacitidine / Q000031:analogs & derivatives / Q000627:therapeutic use; D001853:Bone Marrow / Q000187:drug effects / Q000473:pathology; D016026:Bone Marrow Transplantation / Q000379:methods*; D003131:Combined Modality Therapy; D019175:DNA Methylation / Q000187:drug effects / Q000235:genetics*; D000077209:Decitabine; D004791:Enzyme Inhibitors / Q000627:therapeutic use; D005260:Female; D018380:Hematopoietic Stem Cell Transplantation / Q000379:methods*; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D015999:Multivariate Analysis; D009190:Myelodysplastic Syndromes / Q000235:genetics / Q000628:therapy*; D012189:Retrospective Studies; D012307:Risk Factors; D014184:Transplantation, Homologous; D016896:Treatment Outcome
chemical_list: D004791:Enzyme Inhibitors; D000077209:Decitabine; D001374:Azacitidine
keywords: allogeneic hematopoietic stem cell transplantation; higher-risk myelodysplastic syndrome; hypomethylating treatment; marrow response