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

Abstract

BACKGROUND AND AIMS:

This study aimed at comparing the efficacy and safety of severe aplastic anemia (SAA) cases that had met the criteria for SAA at the time of diagnosis (group A) with SAA that had progressed from non-SAA (NSAA) (group B), both undergoing first-line immunosuppressive therapy (IST). Additionally, group B was compared with SAA that had progressed from NSAA and who had been treated by allogeneic hematopoietic stem cell transplantation (allo-HSCT) (group C).

METHODS:

We retrospectively compared 608 consecutive patients in group A (n = 232), group B (n = 229) and group C (n = 147) 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, treatment-related mortality (TRM), secondary clonal disease, 5-year overall survival (OS) and failure-free survival (FFS) were indirectly compared between group A and group B, group B and group C.

RESULTS:

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 TRM and secondary clonal disease (P > 0.05). There were no differences in estimated 5-year OS 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 FFS 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% vs. 39.7% ± 3.4%, P < 0.0001).

CONCLUSION:

These results indicate that IST is less effective in SAA progressing from non-SAA but allo-HSCT can improve outcomes.

• Authors: Limin Liu, Xin Zhao, Miao Miao, Yanming Zhang, Wenjing Jiao, Meiqing Lei, Huifen Zhou, Qingyuan Wang, Yifeng Cai, Liyun Zhao, Xiaohui Shangguan, Zefa Liu, Jinge Xu, Fengkui Zhang, Depei Wu
• Journal: Frontiers in oncology (Front Oncol) Vol. 11 Pg. 739561 ( 2021) ISSN: 2234-943X [Print] Switzerland
• PMID: 34621679 (Publication Type: Journal Article)
• Copyright: Copyright © 2021 Liu, Zhao, Miao, Zhang, Jiao, Lei, Zhou, Wang, Cai, Zhao, Shangguan, Liu, Xu, Zhang and Wu.