PMID- 26284307 OWN - NLM STAT- MEDLINE DCOM- 20160208 LR - 20151102 IS - 1873-2399 (Electronic) IS - 0301-472X (Linking) VI - 43 IP - 11 DP - 2015 Nov TI - HLA haploidentical peripheral blood stem cell transplantation using reduced dose of posttransplantation cyclophosphamide for poor-prognosis or refractory leukemia and myelodysplastic syndrome. PG - 921-929.e1 LID - S0301-472X(15)00568-8 [pii] LID - 10.1016/j.exphem.2015.07.006 [doi] AB - Nonmyeloablative, human leukocyte antigen (HLA) haploidentical, T-cell-replete bone marrow transplantation followed by high-dose posttransplantation cyclophosphamide (PT/Cy) has recently been developed. This transplantation milieu has resulted in favorable outcomes with low transplantation-related mortality, owing to a low incidence of graft-versus-host disease (GVHD), without increased infectious complications. However, the high relapse rate remains a major concern. We therefore performed a prospective pilot study of HLA haploidentical peripheral blood stem cell transplantation (PBSCT) with intensified conditioning, followed by two lower doses of PT/Cy. A total of 20 patients with refractory or poor-prognosis myelodysplastic syndrome (MDS) and leukemia were enrolled in the study. A trend toward a lower incidence of grade III-IV acute GVHD at day 100 in the group receiving 25 mg/kg x 2 doses of PT/Cy, compared with the group receiving 25 mg/kg of PT/Cy (9.1% vs. 33%, p = 0.20), was noted. However, the cumulative incidence of chronic GVHD was low, at 10% irrespective of PT/Cy dose. The number of infused CD34(+) cells significantly correlated with the grade of acute GVHD (p = 0.004). In addition, the occurrence of BK virus hemorrhagic cystitis was significantly more common in the double-dose PT/Cy group (25% vs. 0%, p = 0.043), especially when combined with busulfan. The probability of overall survival at 1 year in the double-dose group tended to be better compared with that in the single-dose group (64% vs. 44%, respectively; p = 0.20). In conclusion, HLA haploidentical, T-cell-replete PBSCT with 25 mg/kg x 2 doses of PT/Cy might be a feasible option for treating high-risk leukemia and MDS. CI - Copyright (c) 2015 ISEH - International Society for Experimental Hematology. Published by Elsevier Inc. All rights reserved. FAU - Nakamae, Hirohisa AU - Nakamae H AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. Electronic address: hirohisa@msic.med.osaka-cu.ac.jp. FAU - Koh, Hideo AU - Koh H AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Katayama, Takako AU - Katayama T AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Nishimoto, Mitsutaka AU - Nishimoto M AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Hayashi, Yoshiki AU - Hayashi Y AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Nakashima, Yasuhiro AU - Nakashima Y AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Nakane, Takahiko AU - Nakane T AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Nakamae, Mika AU - Nakamae M AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Hirose, Asao AU - Hirose A AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. FAU - Hino, Masayuki AU - Hino M AD - Department of Hematology, Graduate School of Medicine, Osaka City University, Osaka, Japan. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't DEP - 20150815 PL - Netherlands TA - Exp Hematol JT - Experimental hematology JID - 0402313 RN - 8N3DW7272P (Cyclophosphamide) RN - G1LN9045DK (Busulfan) SB - IM MH - Acute Disease MH - Adolescent MH - Adult MH - Aged MH - Busulfan/administration & dosage MH - Cyclophosphamide/administration & dosage MH - Disease-Free Survival MH - Female MH - Graft vs Host Disease/mortality/prevention & control MH - Humans MH - Incidence MH - Leukemia/*mortality/*therapy MH - Male MH - Middle Aged MH - Myelodysplastic Syndromes/*mortality/*therapy MH - *Peripheral Blood Stem Cell Transplantation MH - Pilot Projects MH - Prospective Studies MH - Survival Rate EDAT- 2015/08/19 06:00 MHDA- 2016/02/09 06:00 CRDT- 2015/08/19 06:00 PHST- 2015/04/18 00:00 [received] PHST- 2015/07/06 00:00 [revised] PHST- 2015/07/31 00:00 [accepted] PHST- 2015/08/19 06:00 [entrez] PHST- 2015/08/19 06:00 [pubmed] PHST- 2016/02/09 06:00 [medline] AID - S0301-472X(15)00568-8 [pii] AID - 10.1016/j.exphem.2015.07.006 [doi] PST - ppublish SO - Exp Hematol. 2015 Nov;43(11):921-929.e1. doi: 10.1016/j.exphem.2015.07.006. Epub 2015 Aug 15.