Objective To investigate the effect of dysfunction of T lymphocytes on clonal haematogenesis in patients with myelodysplastic syndrome (MDS). Methods The cytogentics, the subsets of lymphocytes and their activation in 76 patients with MDS were analyzed. Results There were 36 patients with normal karyotype and 40 patients with abnormal karyotype. The incidence of abnormal karyotype were 52.6 %. There were 24 cases (60.0 %) with trisomy 8 (+8) in 40 cases of abnormal karyotype. The expression rates of CD+3 CD-19 cells, CD+3 CD-4 CD+8 cells and CD+3 HLA-DR+ cells in MDS were significantly increased, and CD-3 (CD16CD56)+ cells were significantly lower than that in control group. The expression rates of CD+3 (CD16CD56)+ cells in MDS with abnormal karyotype were significantly higher than that in control group. The expression rates of CD+3 CD+4 CD-8 cells in +8 MDS were significantly lower than that in MDS patients with normal karyotype and with other abnormal karyotype. The ratio of CD4/CD8 in +8 MDS were significantly lower than that in control group. Conclusion The abnormalities of T cell subsets and functions in patients with MDS were observed and the proliferation of malignant clone was prevalent which indicated a poor prognosis in MDS with abnormal karyotype. Dysfunction of immunosurveillance was more aggravated in +8 MDS, which led to excess proliferation of malignant clone and over inhibition of remaining haematogenesis.

Objective To investigate the survival of oral arsenic-containing Qinghuang Powder (QHP) and low intensive chemotherapy (LIC) in the treatment of elderly patients with acute myeloid leukemia (AML).Methods Forty-two AML patients older than 60 years in Xiyuan Hospital from January 2015 to December 2017 were retrospectively analyzed.Of them,20 cases were treated with QHP (QHP group),22 cases were treated with LIC (LIC group).The survivals of the two groups were compared.Results There was no significant difference of median survival time (13 months vs.13.5 months,x2 =0.096,P =0.757),1-year survival rates (59.1％ vs.70.0 ％,x2 =0.543,P =0.461),2-year survival rates (13.6 ％ vs.15.0 ％,x2 =0.016,P ＞ 0.05),and 3-year survival rates (4.6 ％ vs.5.0 ％,x2 =0.005,P ＞ 0.05) between LIC and QHP groups.There was no significant difference of median survival time in age ≥75 year (12 months vs.12.5 months,x2 =1.317,P =0.251),performance status scores ＞ 2 (12 months vs.12 months,x2 =0.834,P =0.361),hematopoietic stem cell transplantation with combined disease index ＞ 2 (12 months vs.13 months,x2 =1.726,P =0.189),secondary AML (10 months vs.14 months,x2 =1.552,P =0.213),and poor cytogenetics (12 months vs.8 months,x2 =0.479,P =0.489) between LIC and QHP group.Conclusion The survival of elderly AML patients is considerable in patients treated with oral QHP and LIC,which suggests that oral QHP may be an equivalent alternative treatment since elderly AML (especially more than 75 years) patients refused to LIC therapy.

OBJECTIVETo explore the efficacy and safety of deferasirox in aplastic anemia (AA)patients with iron overload.

METHODSA single arm, multi- center, prospective, open- label study was conducted to evaluate absolute change in serum ferritin (SF)from baseline to 12 months of deferasirox administration, initially at a dose of 20 mg·kg(-1)·d(-1), and the safety in 64 AA patients with iron overload.

RESULTSAll patients started their deferasirox treatment with a daily dose of 20 mg · kg(-1) ·d(-1). The mean actual dose was (18.6±3.60) mg · kg(-1)·d(-1). The median SF decreased from 4 924 (2 718- 6 765)μg/L at baseline (n=64) to 3 036 (1 474- 5 551)μg/L at 12 months (n=23) with the percentage change from baseline as 38%. A median SF decrease of 651 (126-2 125)μg/L was observed at the end of study in 23 patients who completed 12 months' treatment, the median SF level decreased by 1 167(580-4 806)μg/L [5 271(3 420-8 278)μg/L at baseline; 3 036(1 474-5 551)μg/L after 12 months' treatment; the percentage change from baseline as 42% ] after 12 months of deferasirox treatment. The most common adverse events (AEs) were increased serum creatinine levels (40.98%), gastrointestinal discomfort (40.98%), elevated liver transaminase (ALT: 21.31%; AST: 13.11%)and proteinuria (24.59%). The increased serum creatinine levels were reversible and non-progressive. Of 38 patients with concomitant cyclosporine use, 12(31.8%)patients had two consecutive values >ULN, 10(26.3%)patients had two consecutive values >1.33 baseline values, but only 1(2.6%)patient's serum creatinine increased more than 1.33 baseline values and exceeded ULN. For both AST and ALT, no patients experienced two post- baseline values >5 ×ULN or >10 × ULN during the whole study. In AA patients with low baseline PLT count (less than 50 × 10(9)/L), there was no decrease for median PLT level during 12 months' treatment period.

CONCLUSIONSAA patients with iron overload could achieve satisfactory efficacy of iron chelation by deferasirox treatment. The drug was well tolerated with a clinically manageable safety profile and no major adverse events.

Anemia, Aplastic ; drug therapy ; Benzoates ; therapeutic use ; Blood Transfusion ; China ; Ferritins ; blood ; Humans ; Iron ; blood ; Iron Chelating Agents ; therapeutic use ; Iron Overload ; drug therapy ; Liver ; Prospective Studies ; Triazoles ; therapeutic use

Objective: To investigate the clinical effects of Qinghuang powder, low-intensity chemotherapy and Qinghuang powder alternated with low-intensity chemotherapy in the treatment of elderly acute myeloid leukemia patients progressed from myelodysplastic syndromes (MDS-AML). Methods: A total of 32 elderly patients with MDS-AML treated in Xiyuan Hospital, China Academy of Chinese Medical Sciences from January 2014 to December 2017 were retrospectively analyzed. Of them, 12 patients received Qinghuang powder (Qinghuang powder group), 6 patients received Qinghuang powder alternated with low-intensity chemotherapy (alternating group), and 14 patients received low-intensity chemotherapy (low-intensity chemotherapy group), based on the real world of patient's voluntary choice of treatment. The efficacy and adverse reactions in the 3 groups were observed. Results: The overall response number of Qinghuang powder, alternating, and low-intensity chemotherapy groups were 2, 2 and 4 cases, respectively, there was no significant difference among the 3 groups (P > 0.05). The median overall survival time of 3 groups were 14, 12 and 8 months, respectively, there was no significant difference among the 3 groups (P > 0.05). Two cases in Qinghuang powder group presented with gastrointestinal reactions; 3 cases in alternating group with myelosuppression, 2 cases with liver function damage, and 2 cases with gastrointestinal reaction; 11 cases in low-intensity chemotherapy group presented with bone marrow suppression, 3 cases with liver function damage, 7 cases with gastrointestinal reactions, and 2 cases with myocardial enzyme changes. The incidences of myelosuppression, liver function damage and myocardial enzyme changes in Qinghuang powder group were lower than those in the alternating group and low-intensity chemotherapy group. Conclusion The efficacy of Qinghuang powder or Qinghuang powder alternated with low-intensity chemotherapy for the treatment of elderly MDS-AML is not worse than the low-intensity chemotherapy, furthermore Qinghuang powder or Qinghuang powder alternated with low-intensity chemotherapy has fewer adverse reactions and better tolerance.

Objective: To analyze the gene mutations in the patients with myelodysplastic syndromes (MDS). Methods: Forty-seven patients with MDS newly diagnosed in Xiyuan Hospital, China Academy of Chinese Medical Sciences from January 2016 to July 2017 were enrolled. NGS 127-gene panel was used to detect the gene mutations, and the relationship between the gene mutations and the clinicopathological features was also analyzed. Results: Thirty-one (66.0 %) cases had gene mutations in 47 patients with MDS, and 23 gene mutations were detected with clinical significances. There were 7 mutant genes with a mutation frequency over 5 % in the population, including U2AF1 (23.4 %), SF3B1 (12.8 %), ASXL1 (10.6 %), TET2 (8.5 %), BCOR (8.5 %), TP53 (8.5 %) and DNMT3A (6.4 %) in turn. Among 31 patients with gene mutations, 16 (51.6 %) patients had ≥ 2 synergistic mutations, and 12 cases had synergistic mutations in different genetic functional groups, which was higher than that in same genetic functional groups (4 cases). There was a tendency of coexistence in IDH2-KRAS, IDH2-SRSF2, IDH2-STAG2, KRAS-SRSF2, KRAS-STAG2, RUNX1-PHF6, EZH2-ASXL1, EZH2-ZRSR2, and NPM1-NRAS (all P < 0.05). The variant allele frequency (VAF) of signaling pathway related genes including JAK2, KRAS, NRAS, SH2B3 was low in general and in a sub-clone status. JAK2 gene mutation was observed in 1 case with MDS-U. SH2B3 gene mutation was observed in a patient with very poor prognosis of karyotype. SETPB1 and EZH1 gene mutations were observed in two patients with high-risk revised international prognostic scoring system (IPSS-R). Conclusions The common mutated genes include U2AF1, SF3B1, ASXL1 and TET2. The genes in different genetic functional groups tend to synergistic mutations. Gene mutations can be used to predict the prognosis of diseases and become the target in the treatment of MDS.