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Umbilical Cord Blood Mononuclear Cells for Ex-Vivo Gene Therapy
This study was supported by the grant of Russian Science Foundation No 16-15-00010. Kazan Federal University was supported by the Russian Government Program of Competitive Growth.DisclosuresNo relevant conflicts of interest to declare.
Source: Blood - November 21, 2018 Category: Hematology Authors: Bashirov, F. V., Salafutdinov, I. I., Sokolov, M. E., Izmailov, A. A., Markosyan, V. A., Fadeev, F. O., Rizvanov, A., Islamov, R. I. Tags: 801. Gene Therapy and Transfer Source Type: research

Benefits and Limitations of Long-Term Eculizumab Treatment for Paroxysmal Nocturnal Hemoglobinuria (PNH): Real-World Data from Large Cohort Study in Russia
Conclusions: The results of the study show both the high efficacy and limitations of treatment with eculizumab for PNH in real-world practice. Prospectively confirmed significant improvement of the overall survival on eculizumab stress the need for faster and wider access to costly therapy. Nevertherless, a number of limitations, including BTH and extravascular hemolysis, lack of control of bone marrow failure and further clonal evolution, determine the relevance of next-generation complement inhibitors and risk-adjusted allogeneic HSCT as a curative option.DisclosuresKulagin: Alexion Pharmaceuticals, Inc: Consultancy, Honoraria.
Source: Blood - November 21, 2018 Category: Hematology Authors: Kulagin, A., Klimova, O., Rudakova, T., Golubovskaya, I., Ivanova, M., Smirnova, A., Lapina, A., Bykova, T., Babenko, E., Popova, M., Dobronravov, V., Afanasyev, B. Tags: 508. Bone Marrow Failure: Poster II Source Type: research