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

        Xia Zhang,Zhangzhi Li,Wei Geng,Bin Song,Chucheng Wan 연세대학교의과대학 2018 Yonsei medical journal Vol.59 No.5

        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 overallresponse rate (ORR) (p=0.004), compared to IST. Regarding hematopoietic recovery at month 6, platelet responses in theUCBI+IST group were better than those in the IST group (p=0.002), and UCBI+IST treatment facilitated increasing trends in absoluteneutrophil 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) betweenthe two groups (p=0.620). Furthermore, logistic regression analysis demonstrated that UCBI+IST was an independent predictingfactor for both CR (p=0.001) and ORR (p<0.001), compared to IST; meanwhile, very severe aplastic anemia (VSAA) and ANC couldpredict 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 SAApatients.

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