AUTHOR=Liu Limin , Zhao Xin , Miao Miao , Zhang Yanming , Jiao Wenjing , Lei Meiqing , Zhou Huifen , Wang Qingyuan , Cai Yifeng , Zhao Liyun , Shangguan Xiaohui , Liu Zefa , Xu Jinge , Zhang Fengkui , Wu Depei 

TITLE=Inefficacy of Immunosuppressive Therapy for Severe Aplastic Anemia Progressing From Non-SAA: Improved Outcome After Allogeneic Hematopoietic Stem Cell Transplantation

JOURNAL=Frontiers in Oncology

VOLUME=Volume 11 - 2021

YEAR=2021

URL=https://www.frontiersin.org/journals/oncology/articles/10.3389/fonc.2021.739561

DOI=10.3389/fonc.2021.739561

ISSN=2234-943X

ABSTRACT=We retrospectively compared 232 severe aplastic anemia (SAA) cases that had met the criteria for SAA at the time of diagnosis (group A) with 229 SAA that had progressed from non-SAA (NSAA) (group B), both undergoing first-line immunosuppressive therapy (IST). Additionally, group B was compared with 147 SAA that had progressed from NSAA and who had been treated by allogeneic haematopoietic stem cell transplantation (allo-HSCT) (group C) between June 2002 and December 2019. Six months after treatment, the rate of overall response and the fraction of patients who had achieved normal blood values in group A was higher than in group B (65.24% vs. 40.54%, P < 0.0001; 23.33% vs. 2.25%, P < 0.0001); the same was true for group C (92.50% vs. 2.25%, P < 0.0001). The rate of relapse in group B was higher than in group C (P < 0.0001), but there were no differences in treatment-related mortality and secondary clonal disease (P > 0.05). There were no differences in estimated 5-year overall survival between groups A and B (83.8% ± 2.6% vs. 85.8% ± 2.6%, P = 0.837), or between B and C (85.8% ± 2.6% vs. 77.9% ± 3.4%, P = 0.051). The estimated 5-year failure-free survival in groups A and C was higher than for group B (57.1% ± 3.3% vs. 39.7% ± 3.4%, P < 0.001; 76.7% ± 3.5%, 39.7% ± 3.4%, P < 0.0001). These results indicate that IST is less effective in SAA progressing from non-SAA but allo-HSCT can improve outcomes.