Objective To retrospectively analyze the engraftment, transplant-related complications and survival after unrelated cord blood transplantation (UCBT) in patients with hematologic malignancies. Methods Fifty consecutive patients with hematological malignancies (median age, 19 years; median weight, 53 kg) were treated with UCBT in single center from April 2000 to August 2009. Thirty-nine patients were high-risk or refractory. Double UCB grafts were used for 26 patients, while single UCB graft for 24 patients. Myeloablative conditioning was given to 45 cases and non-myeloablative regimens to 5 cases. All patients were given a combination of cyclosporin A (CsA) and mycophenolate mofetil (MMF) for graft-versus-host disease (GVHD) prophylaxis. Results The median total nucleated cell (TNC) dose was 4.0 (range, 1.95-16.24)×10~7 TNC/ kginfused, and CD34~+ cell dose was 2.74(range, 0.67-29.28)×10~5/kginfused. Forty-two of 50 patients acquired engraftment with implantation rate being 86%. The median time to engraftment (absolute neutrophil count>500/mm~3 and platelets 20 000/L) was 19 and 34 days. The cumulative incidence of neutrophil engraftment by day 42 was 86.3%(95% confidence interval [CI] 0.769-0.957); the cumulative incidence of platelets engraftment by day 120 was 72.3% (95% CI 0.620-0.821). Twenty cases developed acute GVHD, and the incidence of acute GVHD of grades Ⅲ/Ⅳ by day 100 was 7.1%. The incidence of chronic GVHD within 2 years was 17.4%. During a median follow-up period of 22 months (range 4-116), Overall 6-month, 1-year and 2-year survival rate was 66.2%(95% CI 0.590-0.734), 57.4%(95% CI 0.496-0.652), 54.2%(95% CI 0.462-0.622), respectively. For the patients with non-advanced hemotologic malignancies, 6-month, 1-year and 2-year survival rate was 73.2% (95% CI 0.659-0.805), 66.1% (95% CI 0.579-0.743), and 62.2% (95% CI 0.542-0.682) respectively. Five cases relapsed. The cumulative incidence of relapse within 2 years was 16.2% (95% CI 0.099-0.225). Twenty-one cases died mainly due to infection. Conclusion UCBT could be safely and effectively used for adult patients with hematologic malignancies.

Objective To evaluate the effect of pretransplant iron overload on the clinical efficacy of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with severe aplastic anemia (SAA). Methods Clinical data of 80 SAA recipients who underwent allo-HSCT for the first time were retrospectively analyzed. According to the incidence of iron overload, all recipients were divided into the iron overload group (n=20) and non-iron overload group (n=60). The engraftment rate, incidence of postoperative complications and clinical prognosis of the recipients afterallo-HSCT were statistically compared between two groups. The influencing factors of 2-year overall survival (OS) and 180 d transplantation related mortality (TRM) were analyzed by Cox proportional hazards regression model. Results The engraftment rate of neutrophils in the non-iron overload group was 98% (59/60), significantly higher than 75% (15/20) in the iron overload group (P < 0.05). The engraftment rate of platelet in the non-iron overload group was 90% (54/60), significantly higher than 65% (13/20) in the iron overload group (P < 0.05). The incidence rate of bloodstream infection in the non-iron overload group was 23% (14/60), remarkably lower than 40% (8/20) in the iron overload group (P < 0.05). The 180 d TRM of the recipients in the non-iron overload group was 17%, significantly lower than 45% in the iron overload group (P < 0.05). The 1- and 2-year OS of the recipients in the non-iron overload group were 82% and 80%, significantly higher than 50% and 44% in the iron overload group (both P < 0.05). Iron overload or not was an independent risk factor of the OS and TRM of the recipients (both P < 0.05). Conclusions Iron overload can affect the OS and TRM of SAA patients after allo-HSCT.

Objective: To compare the clinical efficacy of umbilical cord blood transplantation (UCBT) and hematopoietic stem cell transplantation from HLA-matched sibling donors (MSD-HSCT) in the treatment of myelodysplastic syndrome-EB (MDS-EB) or acute myeloid leukemia with myelodysplasia-related changes (AML-MRC) . Methods: A cohort of 64 patients (including 38 cases of MDS-EB and 26 cases of AML-MRC) who received UCBT/MSD-HSCT from February 2011 to December 2017 were retrospectively analyzed. Results: ①Compared with MSD-HSCT group, UCBT group had a higher proportion of AML-MRC patients [52.8% (19/36) vs 25.0% (7/28) , P=0.025], and a lower median age [13 (1.5-52) years vs 32 (10-57) years, P=0.001]. ②The engraftment of neutrophils both in UCBT and MSD-HSCT groups on +42 d was 100%, and the median engraftment time was 17.5 (11-31) d and 11.5 (10-20) d, respectively. The engraftment of platelet at +100 d in UCBT group was 91.4%, the median engraftment time was 40 (15-96) d; The engraftment of platelet at +100 d in MSD-HSCT group was 100%, and the median engraftment time was 15 (11-43) d. ③There were no statistically significant differences in terms of the cumulative incidence of Ⅱ-Ⅳ and Ⅲ/Ⅳ aGVHD of 100 d and transplant related mortality (TRM) of 180 d, relapse rate, overall survival (OS) , disease-free survival (DFS) between UCBT and MSD-HSCT groups (P>0.05) . ④The 3-year cumulative incidence of chronic GVHD (cGVHD) and severe chronic GVHD in UCBT group were lower than of MSD-HSCT group [28.3% (95%CI 13.4%-45.3%) vs 67.9% (95%CI 46.1%-82.4%) , P=0.002; 10.3% (95%CI 2.5%-24.8%) vs 50.0% (95%CI 30.0%-67.1%) , respectively, P<0.001]. The cumulative 3-year incidence of GVHD-free and relapse-free survival (GRFS) of UCBT group was significantly higher than of MSD-HSCT group [55.0% (95%CI 36.0%-70.6%) vs 28.6% (95%CI 13.5%-45.6%) , P=0.038]. Conclusion UCBT could obtain better quality of life after transplantation than MSD-HSCT in treatment of MDS-EB/AML-MRC.

Objective: To explore the clinical efficacy and safety of unrelated umbilical cord blood transplantation (UCBT) in the treatment of refractory and relapsed acute leukemia (AL) patients. Methods: The clinical data of 22 refractory and relapsed AL patients who were treated with UCBT as salvage therapy from November 2009 to May 2017 were retrospectively analyzed. All patients received a myeloablative conditioning regimen for prevention of graft-versus-host disease (GVHD) with cyclosporine A (CSA)/short course of mycophenolate mofetil (MMF). Results: ①Of 22 patients, 9 cases were male and 13 female. The median age was 23 (15-44) years and median weight of 52.5 (43-82) kg. All patients were transplanted with a median umbilical cord blood nucleated cells of 3.07 (1.71-5.30)×10⁷/kg (by weight), the median CD34⁺ cells was 1.60 (0.63-3.04)×10⁵/kg (by weight). ②The myeloid cumulative implantation rate was 95.5% (95%CI 45.2-99.7%) after transplantation of 42 d, with the median implantation time of 19 (13-27) d. The platelet cumulative implantation rate after transplantation of 120 d was 81.8% (95%CI 54.2-93.6%), the median implantation time of 42 (20-164) d. ③The incidence of Ⅱ-Ⅳ, Ⅲ-Ⅳ aGVHD and the 2 year cumulative incidence of cGVHD were 36.4%, 13.6% and 40.3% respectively. ④ The transplant related mortality (TRM) after transplantation of 180d was 22.7%, 2 year cumulative rate of relapse was 18.7% (95%CI 3.6-42.5%), 2 year disease-free survival rate (DFS) and overall survival rate (OS) were 53.7% and 58.1%, respectively. Conclusion The preliminary results show that the use of UCBT is safe and effective for refractory and relapsed AL patients who fail to respond to conventional chemotherapy.

Objective To study the curative efficacy and safety of single-unit umbilical cord blood transplantation (sUCBT) for malignant hematologic diseases,which is provided by China's public cord blood bank.Methods We retrospectively analyzed 409 cases of malignant hematologic diseases who accepted myeloablative single-unit unrelated donor UCBT without ATG at our center between May 2008 and December 2016.A comparative analysis was made on the total nuclear cells (TNC) of the umbilical cord blood before freezing and after thawing,the cells of CD34+,the recovery rate of cells and the clinical effect of UCBT.Result 409 units of umbilical cord blood used in UCBT respectively came from eight China's public cord blood banks.The average TNC of 409 units of umbilical cord blood before freezing and after the tubular recovery were respectively 18.5 × 108 and 16.34 × 108 (p =0.000).The average recovery rate of the tubular recovery was 88.5％,and there was significant difference among cord blood banks (P =0.000).The average TNC of umbilical cord blood before freezing and transfusion were respectively 18.5 × 108 and 15.86 × 108 (p =0.000).The average recovery rate of umbilical cord blood transfusion was 85.9％,with the difference being significant among cord blood banks (P =0.000).The average number of CD34+ cells before freezing and after the tubular recovery was 11.18 × 106and 8.68 × 106 (p =0.000).The average recovery rate of CD34+ cells after the tubular recovery was 80.75 ％,with the difference being significant among the cord blood banks (P =0.000).At 42nd day after UCBT,the cumulative incidence of neutrophil engraftment was 95.4％,and the median time of the engraftment was 17 days (11-38 days).The cumulative incidence of platelet engraftment at 120th day was 84.6％,and the median time of the engraftment was 36 days (14-93 days).The cumulative incidence of erythrocyte engraftment at 60th day was 92％,and the median time of engraftment was 22 days (9d-60 days).After the umbilical cord blood provided by each bank was used in UCBT,it got the difference in cumulative incidence of engraftment.The P values for cumulative incidence of neutrophil,platelet and erythrocyte engraftment were respectively 0.004,0.01 and 0.000 2,with the differences being statistically significant.At 100th day after UCBT,the cumulative incidence of Ⅱ-Ⅳ and Ⅲ-Ⅳ degrees of acute graft-versus-host disease (aGVHD) was respectively 28.63％ and 15.7％.After umbilical cord blood provided by each bank was used in UCBT,it got the difference in cumulative incidence of aGVHD.There was no significant difference between Ⅱ-Ⅳ and Ⅲ-Ⅳ degrees (P =0.809 and 0.68 respectively).At 3rd year after UCBT,the cumulative incidence of relapse was 15.89％.After umbilical cord blood provided by each bank was used in UCBT,there was no significant difference in the cumulative incidence of relapse (P =0.898).At 3rd year after UCBT,the overall survival (OS) rate and disease free survival (DFS) rate were respectively 66.7％ and 59％.After umbilical cord blood provided by each bank was used in UCBT,it got the difference in OS and DFS.There was no significant difference in OS and DFS (P =0.566 and 0.703 respectively).At 3rd year after sUCBT,the rate of graft-versus-host diseases/relapse-free survival (GRFS) was 54.3％.After umbilical cord blood provided by each bank was used in UCBT,there was no significant difference in the rate of GRFS (P =0.449).Conclusion The umbilical cord blood provided by China's public cord blood bank was used in UCBT.It has a high safety and good efficacy in treating malignant hematologic diseases.But it needs to set up the standardized and normalized quality-control system of umbilical cord blood for China's public cord blood bank.

BACKGROUND: Umbilical cord blood hematopoietic stem cell transplantation is more and more widely used as a radical treatment for acute leukemia, but its therapeutic effect in different leukemias has not been compared. By comparing the efficacy of diseases, it can guide different patients to choose the transplantation method.OBJECTIVE: To compare and analyze the therapeutic effect of umbilical cord blood hematopoietic stem cell transplantation on acute myeloid leukemia and acute lymphocytic leukemia. METHODS: Clinical data of 306 cases of acute leukemia treated by unrelated umbilical cord blood hematopoietic stem cell transplantation were retrospectively analyzed, including 112 patients with acute myeloid leukemia and 194 with acute lymphoblastic leukemia. All patients received myeloablative conditioning without antithymocyte, and the prevention of graft-versus-host disease was cyclosporine combined with mycophenolate mofetil. RESULTS AND CONCLUSION: (1) Except that the relapse rate after acute lymphoblastic leukemia transplantation was slightly higher than acute myeloid leukemia, the efficacy of the two groups of patients after receiving unrelated umbilical cord blood hematopoietic stem cell transplantation was basically the same. (2) In the group of adolescents and young adults (aged 15-39 years), the rate of neutrophil and platelet implantation in acute myeloid leukemia was faster than in acute lymphoblastic leukemia. Among them, CD34+ cell number and pretreatment program were independent influencing factors for neutrophil implantation, while CD34+ cell number was also an independent influencing factor for platelet implantation. In this age group, the recurrence rate of acute lymphoblastic leukemia patients after transplantation was still higher than that of acute myeloid leukemia, in which chronic graft-versus-host disease was an independent influencing factor. (3) Immune reconstruction testing after transplantation suggests that cord blood CD8+ T cell reconstruction in patients with acute myeloid leukemia was better than in acute lymphoblastic leukemia patients 4 months after transplantation. (4) The above data show that pre-treatment of unrelated cord blood transplantation without antithymocyte globulin has a good effect on acute lymphocytic leukemia and acute myeloid leukemia. Department of Hematology of The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China is qualified for stem cell transplantation.

Objective: To compare the efficacy of unrelated cord blood transplantation (UCBT) and HLA-identical sibling peripheral blood stem cell transplantation (PBSCT) for the treatment of adult hematological malignancies. Methods: From April 2011 to December 2015, a total of 81 patients receiving single-unit UCBT and 57 patients receiving HLA-identical sibling PBSCT were enrolled in this study. All of the patients received myelablative conditioning. Cyclosporine combined with mycophenolate mofetil was adopted for GVHD prophylaxis. Results: The cumulative incidence of neutropil engraftment at day-42 was 95.0% and 100% in UCBT and sibling PBSCT groups, respectively (P=0.863) . Platelet engraftment at day 100 was 87.3% (95%CI 76.8%-93.1%) in UCBT group, which was significantly lower than that of sibling PBSCT group[98.2% (95%CI 87.3%-99.7%) ] (P=0.005) . There were no significant differences in terms of Ⅱ-Ⅳ acute GVHD or Ⅲ-Ⅳ acute GVHD in two groups (P=0.142, 0.521) . The 3-year chronic GVHD and extensive chronic GVHD were 14.9% (95%CI 5.2%-23.5%) and 11.2% (95%CI 2.9%-18.7%) , respectively in UCBT group, which was significantly lower than that of sibling PBSCT group[35.2% (95%CI 19.4%-47.8%) , 31.4% (95%CI 16.2%-43.9%) ] (P=0.008, 0.009) . The 3-year transplant-related mortality (TRM) was similar between two groups (30.1% vs 23.2%, P=0.464) . The relapse rate at 3-year in UCBT group[12.9% (95%CI 6.6%-21.5%) ]was significantly lower than that in sibling PBSCT group[24.3% (95%CI 13.5%-36.8%) ] (P=0.039) . There were no significant differences in terms of overall survival (OS) and disease-free survival (DFS) between two groups (58.6% vs 54.8%, P=0.634; 57.0% vs 52.4%, P=0.563) . But GVHD-free and relapse-free survival (GRFS) in UCBT group [55.7% (95%CI 44.1%-65.8%) ]was significantly higher than that of sibling PBSCT group[42.9% (95%CI 29.8%-55.3%) ] (P=0.047) . Conclusions For adult hematological malignancies, the incidences of acute GVHD and TRM were similar between UCBT and sibling PBSCT recipients, and the incidences of chronic GVHD and relapse were lower in UCBT recipients. UCBT recipients had higher GRFS rate although OS and DFS were similar between two groups, which may reflect the real recovery and better quality of life following UCBT.

Background::With an increasing number of patients with hematological malignancies being treated with umbilical cord blood transplantation (UCBT), the correlation between immune reconstitution (IR) after UCBT and graft-versus-host disease (GVHD) has been reported successively, but reports on double-negative T (DNT) cell reconstitution and its association with acute GVHD (aGVHD) after UCBT are lacking.Methods::A population-based observational study was conducted among 131 patients with hematological malignancies who underwent single-unit UCBT as their first transplant at the Department of Hematology, the First Affiliated Hospital of USTC, between August 2018 and June 2021. IR differences were compared between the patients with and without aGVHD.Results::The absolute number of DNT cells in the healthy Chinese population was 109 (70-157)/μL, accounting for 5.82 (3.98-8.19)% of lymphocytes. DNT cells showed delayed recovery and could not reach their normal levels even one year after transplantation. Importantly, the absolute number and percentage of DNT cells were significantly higher in UCBT patients without aGVHD than in those with aGVHD within one year ( F = 4.684, P = 0.039 and F = 5.583, P = 0.026, respectively). In addition, the number of DNT cells in the first month after transplantation decreased significantly with the degree of aGVHD increased, and faster DNT cell reconstitution in the first month after UCBT was an independent protective factor for aGVHD (HR = 0.46, 95% confidence interval [CI]: 0.23-0.93; P = 0.031). Conclusions::Compared to the number of DNT cells in Chinese healthy people, the reconstitution of DNT cells in adults with hematological malignancies after UCBT was slow. In addition, the faster reconstitution of DNT cells in the early stage after transplantation was associated with a lower incidence of aGVHD.

This study aims to discuss the therapeutic effect of magnesium isoglycyrrhizinate on hepatitis B virus(HBV)transgenic mouse and its effect on cellular immunity and liver inflammation. The changes of serum aspartate aminotransferase(AST)and alanine aminotransferase(ALT)activity, the difference of serum hepatitis B surface antigen(HBsAg), liver tissue HBsAg mRNA, and the pathological morphological changes of liver tissue were detected to investigate the hepatic inflammatory lesions and the efficacy of magnesium isoglycyrrhizinate in HBV transgenic mouse. Peripheral blood lymphocytes were classified by flow cytometry, and serum cytokines were detected by cytometric bead array(CBA)to explore the mechanism of magnesium isoglycyrrhizinate to reduce hepatic inflammatory lesions in HBV transgenic mouse. After grouping HBV transgenic mouse with serum transaminase activity and 35 days of continuous administration, serum transaminases level in magnesium isoglycyrrhizinate ［15 mg/(kg ·d)］ group was significantly lower than that in control group(P< 0. 05), serum HBsAg protein and liver tissue HBsAg mRNA increased with time, but there was no significant difference between the two groups. The main pathological changes of liver were liver cell swelling, necrosis and focal inflammatory cell infiltration, and the pathological changes of liver in magnesium isoglycyrrhizinate group were lighter than those in control group. The number of CD8+ cells in the blood of magnesium isoglycyrrhizinate group was significantly less than that in the control group(P< 0. 05)and the CD4+/CD8+ cell ratio was significantly higher than that in the control group(P< 0. 05). The content of inflammatory cytokines in serum of magnesium isoglycyrrhizinate group decreased significantly(P< 0. 05). Magnesium isoglycyrrhizinate can regulate the immune function of HBV transgenic mouse, decrease the infiltration of inflammatory cells in hepatic tissue and hepatocyte injury, but do not affect the expression of hepatocyte HBsAg.

Objective:To explore the feasibility and efficacy of umbilical cord blood transplantation (UCBT) in the treatment of paroxysmal nocturnal hemoglobinuria (PNH).Methods:From May 2014 to December 2019, clinical data were retrospectively reviewed for 7 PNH patients undergoing UCBT. The grades were severe ( n=6) and extremely severe ( n=1). The causes were primary PNH ( n=4) and PNH-aplastic anemia (AA) syndrome ( n=3). There were 5 males and 2 females with a median age of 29 (20-47) years, a median weight of 60(50-71) kg and a median time from diagnosis to transplantation of 62.5(7.7-171) months. All of them were accompanied by transfusion dependence. Myeloablative ( n=6) and reduced-intensity ( n=1) pretreatment was offered. The regimen of preventing GVHD was cyclosporine A plus short-term mycophenolate mofetil without ATG. The median number of input nucleated cells was 2.4(1.71-4.28)×10 7/kg and the median number of CD34+ cells 1.58(0.88-3.02)×10 5/kg. Results:Neutrophil and erythroid engraftment was obtained with a median neutrophil engraftment time of 17(15-21) days and a median erythroid engraftment time of 27. Engraftment time of 37(25-101) days for platelets >20×10 9/L and 62(27-157) days for platelets >50×10 9/L. The incidence of 100-day acute GVHD was 28.6%(95%CI 0-55.3%). The severity of GVHD was grade Ⅱ° acute ( n=2) and mild ( n=1). The median follow-up period was 13.5(3-71.4) months. Six patients survived while another with PNH-AA syndrome with iron overload died of gastrointestinal hemorrhage. The 2-year overall survival rate was 83.3%(95%CI 27.3-97.5%). Conclusions:With excellent engraftment and survival in the treatment of PNH, UCBT is indicated for patients without HLA full-match donor. PNH-AA syndrome with iron overload may be one of the important prognostic factors.