PMID- 26838271 OWN - NLM STAT- MEDLINE DCOM- 20160606 LR - 20220410 IS - 2157-6564 (Print) IS - 2157-6580 (Electronic) IS - 2157-6564 (Linking) VI - 5 IP - 4 DP - 2016 Apr TI - A Study of Human Leukocyte Antigen Mismatched Cellular Therapy (Stem Cell Microtransplantation) in High-Risk Myelodysplastic Syndrome or Transformed Acute Myelogenous Leukemia. PG - 524-9 LID - 10.5966/sctm.2015-0196 [doi] AB - The treatment outcomes of myelodysplastic syndrome (MDS) and transformed acute myelogenous leukemia (tAML) remain very unsatisfactory. We designed a combination of human leukocyte antigen (HLA)-mismatched hematopoietic stem cell microtransplantation (MST) with chemotherapy for patients with MDS and tAML and evaluated its effects and toxicity. Patients were between 13 and 79 years old. Patients with MDS (n=21) were given HLA-mismatched MST combined with decitabine and cytarabine; patients with tAML (n=22) were given HLA-mismatched MST combined with decitabine and cytarabine, and also mitoxantrone. Patients in complete remission (CR) also received MST plus decitabine and medium-dose cytarabine chemotherapy without graft-versus-host disease (GVHD) prophylaxis. The overall response rate of the patients with MDS was significantly higher than that of those with tAML (81% vs. 50%; p=.03). The CR rates were 52.4% and 36.4% in the two groups, respectively. There was no difference in the cytogenetic CR rate between the MDS and tAML groups (85.7% vs. 70%, respectively; p=.7). The 24-month overall survival of the patients with MDS was significantly higher than that of the patients with tAML (84.7% and 34.1%, respectively; p=.003). The median recovery times of neutrophils and platelets were, respectively, 14 and 17 days in the patients with MDS, and 16 and 19 days in those with tAML. The treatment-related mortality rates were 4.8% and 18.2%, respectively, in the MDS and tAML groups (p=.34). No GVHD was observed in any patient. Microtransplantation combined with decitabine and chemotherapy may provide a novel, effective, and safe treatment for high-risk MDS and tAML. SIGNIFICANCE: Microtransplantation (MST) refers to regular chemotherapy combined with granulocyte colony-stimulating factor-mobilized peripheral blood stem cell infusion of human leukocyte antigen-mismatched donor cells without using immunosuppressive agents. It aims to support hematopoietic recovery and perform graft-versus-leukemia (GVL) effects but differs from traditional allogeneic stem cell transplantation because the rate of donor cell chimerism is low and there is and no graft-versus-host disease (GVHD) risk. Thus, a trial was designed to evaluate the safety and efficacy of MST in patients with myelodysplastic syndrome and those with transformed acute myelogenous leukemia. Higher complete remission and cytogenetic complete response rates were observed, and the treatment improved disease progress-free survival, sped hematopoietic recovery, and avoided GVHD. CI - (c)AlphaMed Press. FAU - Hu, Kai-Xun AU - Hu KX AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Sun, Qi-Yun AU - Sun QY AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Guo, Mei AU - Guo M AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Qiao, Jun-Xiao AU - Qiao JX AD - Department of Hematology, Second Artillery General Hospital, Beijing, People's Republic of China. FAU - Yu, Chang-Lin AU - Yu CL AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Qiao, Jian-Hui AU - Qiao JH AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Dong, Zheng AU - Dong Z AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Sun, Wan-Jun AU - Sun WJ AD - Department of Hematology, Second Artillery General Hospital, Beijing, People's Republic of China. FAU - Zuo, Hong-Li AU - Zuo HL AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Huang, Ya-Jing AU - Huang YJ AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Cai, Bo AU - Cai B AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China. FAU - Ai, Hui-Sheng AU - Ai HS AD - Department of Hematology and Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, Beijing, People's Republic of China huishengai@163.com. LA - eng PT - Clinical Trial PT - Journal Article PT - Multicenter Study DEP - 20160202 PL - England TA - Stem Cells Transl Med JT - Stem cells translational medicine JID - 101578022 RN - 0 (HLA Antigens) SB - IM MH - Adolescent MH - Adult MH - Aged MH - Cell Transformation, Neoplastic MH - Chimerism/statistics & numerical data MH - Female MH - Graft vs Host Disease/epidemiology/immunology MH - HLA Antigens/*immunology MH - Hematopoietic Stem Cell Transplantation/adverse effects/*methods/statistics & numerical data MH - Histocompatibility Testing MH - Humans MH - Leukemia, Myeloid, Acute/epidemiology/immunology/pathology/*therapy MH - Male MH - Middle Aged MH - Myelodysplastic Syndromes/epidemiology/immunology/pathology/*therapy MH - Remission Induction MH - Survival Analysis MH - Transplantation Chimera/*immunology MH - Young Adult PMC - PMC4798738 OTO - NOTNLM OT - Decitabine OT - Microtransplantation OT - Myelodysplastic syndrome OT - Transformed acute myeloid leukemia EDAT- 2016/02/04 06:00 MHDA- 2016/06/09 06:00 PMCR- 2017/04/01 CRDT- 2016/02/04 06:00 PHST- 2015/08/11 00:00 [received] PHST- 2015/11/02 00:00 [accepted] PHST- 2016/02/04 06:00 [entrez] PHST- 2016/02/04 06:00 [pubmed] PHST- 2016/06/09 06:00 [medline] PHST- 2017/04/01 00:00 [pmc-release] AID - sctm.2015-0196 [pii] AID - 20150196 [pii] AID - 10.5966/sctm.2015-0196 [doi] PST - ppublish SO - Stem Cells Transl Med. 2016 Apr;5(4):524-9. doi: 10.5966/sctm.2015-0196. Epub 2016 Feb 2.