Effects and Predictive Factors of Immunosuppressive Therapy Combined with Umbilical Cord Blood Infusion in Patients with Severe Aplastic Anemia

PURPOSE: To investigate the efficacy and safety of umbilical cord blood (UCB) infusion (UCBI) plus immunosuppressive therapy (IST) treatment in comparison to IST treatment, as well as predictive factors for clinical responses, in severe aplastic anemia (SAA) patients. MATERIALS AND METHODS: Totally, 93 patients with SAA were enrolled in this cohort study. In the IST group, rabbit antithymocyte globulin (r-ATG) combined with cyclosporine A (CsA) was administered, while in the IST+UBCI group, r-ATG, CsA, and UCB were used. RESULTS: After 6 months of treatment, UCBI+IST achieved a higher complete response (CR) rate (p=0.002) and an elevated overall response rate (ORR) (p=0.004), compared to IST. Regarding hematopoietic recovery at month 6, platelet responses in the UCBI+IST group were better than those in the IST group (p=0.002), and UCBI+IST treatment facilitated increasing trends in absolute neutrophil count (ANC) response (p=0.056). Kaplan-Meier curves illuminated UCBI+IST achieved faster ANC response (p < 0.001) and platelet response (p < 0.001), compared with IST therapy. There was no difference in overall survival (OS) between the two groups (p=0.620). Furthermore, logistic regression analysis demonstrated that UCBI+IST was an independent predicting factor for both CR (p=0.001) and ORR (p < 0.001), compared to IST; meanwhile, very severe aplastic anemia (VSAA) and ANC could predict clinical responses as well. However, Cox proportional hazard regression indicated that VSAA (p=0.003), but not UCBI+IST, affected OS. Safety profiles showed that UCBI+IST therapy did not elevate adverse events, compared with IST treatment. CONCLUSION: UCBI+IST achieved better clinical responses and hematopoietic recovery than IST, and was well tolerated in SAA patients.

Long-term remission of leukemia after failure of cord blood stem cell transplantation for high-risk childhood acute lymphoblastic leukemia:a report of one case and literature review / 器官移植

Objective To analyze the reasons for long-term remission of leukemia after failure of cord blood stem cell transplantation for high-risk childhood acute lymphoblastic leukemia.Methods Clinical data of 1 child with high-risk acute lymphoblastic leukemia undergoing cord blood stem cell transplantation in the Department of Hematology of the Third Affiliated Hospital of Sun Yat-sen University in September 201 3 were collected.The treatment course and prognosis of the child were analyzed.In combination with literatures,the possible reasons for the good treatment effect on leukemia after transplantation failure were analyzed.Results The hematopoietic recovery of the child after the first cord blood stem cell transplantation was poor.The child underwent cord blood infusion in November 201 3 and did not undergo further treatment for the primary disease due to economic reasons. However, the hematopoietic function recovered 1 5 months after cord blood transplantation.Till September 201 5,2-year disease free survival after cord blood transplantation was obtained.Conclusions Cord blood transplantation or infusion may repair the function of hematopoietic system and immune system,and may take effect with no need of successful transplantation.The failure of cord blood transplantation for childhood high-risk leukemia does not mean treatment failure.Children patients may obtain long-term disease free survival.