title: [Long- term outcome of thalidomide and cyclosporine in patients with IPSS low/intermediate- 1 myelodysplastic syndromes].
abstract: OBJECTIVE
  To investigate the long- term outcome of cyclosporin A (CsA) combined with thalidomide regime for Chinese patients with IPSS low/intermediate- 1 myelodysplastic syndromes (MDS) without del（5q）and the predictive variables which could impact the response to the therapy.
  
  
  METHODS
  Seventy-six MDS patients who were treated with these drugs at a single institute in China were retrospectively analyzed. The polymorphism of cereblon gene, rs1672753, was detected in patients of this cohort by PCR and direct sequencing.
  
  
  RESULTS
  A total of 53% of patients showed hematological improvement（HI）to the therapy. Thirty-one patients（31/73, 43%）achieved erythrocyte response（HI-E）; 15 patients（15/50, 30%）achieved neutrophil response（HI-N); 18 patients（18/58, 31%）achieved platelet response（HI-P). Twenty-seven of the 50 patients（46%）who were dependent on red blood cell transfusion achieved HI- E and became independent of transfusion. The median duration of response among the responders was 22 months (range, 1- 131 + months). Bone marrow blasts ≤2% was the only factor associated with longer response duration in univariate analysis （P=0.010）. There was no significant difference between the two groups of celeblon gene rs1672753 polymorphism either on the response rate or the response duration. The median survival of 67 patients without stem cell transplantation was 82 months. In multivariate analyses, factors significantly correlated with survival were IPSS-R（HR=3.461, 95%CI 1.126-10.639, P=0.030), age ≥ 60 y（HR=4.120, 95%CI 1.070-15.867, P=0.040）and HI-N（HR=7.733, 95%CI 1.007-59.396, P=0.049).
  
  
  CONCLUSIONS
  CsA combined with thalidomide regime could improve the anemia symptom in low/int-1 risk MDS patients without del（5q）. The predictive value of cereblon gene polymorphism, rs1672753, could not be verified in this study.
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Jingya
authors.initials: J
authors.lastname: Wang
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Tiejun
authors.initials: T
authors.lastname: Qin
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Zefeng
authors.initials: Z
authors.lastname: Xu
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Yue
authors.initials: Y
authors.lastname: Zhang
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Hongli
authors.initials: H
authors.lastname: Zhang
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Liwei
authors.initials: L
authors.lastname: Fang
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Lijuan
authors.initials: L
authors.lastname: Pan
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Naibo
authors.initials: N
authors.lastname: Hu
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Shiqiang
authors.initials: S
authors.lastname: Qu
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Bing
authors.initials: B
authors.lastname: Li
authors.affiliation: Institute of Hematology and Blood Disease Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, Tianjin 300020, China.
authors.forename: Zhijian
authors.initials: Z
authors.lastname: Xiao
mesh_terms: D048868:Adaptor Proteins, Signal Transducing; D000740:Anemia; D001792:Blood Platelets; D001803:Blood Transfusion; D001853:Bone Marrow; D002681:China; D016572:Cyclosporine / Q000627:therapeutic use*; D004912:Erythrocytes; D006801:Humans; D009190:Myelodysplastic Syndromes / Q000145:classification / Q000188:drug therapy*; D009504:Neutrophils; D010447:Peptide Hydrolases / Q000378:metabolism; D012074:Remission Induction; D012189:Retrospective Studies; D013792:Thalidomide / Q000627:therapeutic use*; D016896:Treatment Outcome; D044767:Ubiquitin-Protein Ligases
chemical_list: D048868:Adaptor Proteins, Signal Transducing; C502543:CRBN protein, human; D013792:Thalidomide; D016572:Cyclosporine; D044767:Ubiquitin-Protein Ligases; D010447:Peptide Hydrolases