Transfusion-dependent non-severe aplastic anemia: characteristics and outcomes in the clinic

Front Immunol. 2023 Jul 11:14:1197982. doi: 10.3389/fimmu.2023.1197982. eCollection 2023.

Abstract

Transfusion-dependent non-severe aplastic anemia (TD-NSAA) is a rare condition of bone marrow failure that can persist for a long time or develop into severe aplastic anemia (SAA). Little is known about the clinical and laboratory characteristics, and disease prognosis and outcomes in TD-NSAA patients. The clinical and laboratory data of 124 consecutive TD-NSAA patients in the Chinese Eastern Collaboration Group of Anemia from December 2013 and January 2017 were analyzed retrospectively. In 124 TD-NSAA patients, the median age was 32 years (range: 3-80) and the median disease course was 38 months (range: 3-363). Common complications were iron overload (53/101, 52.5%), liver and kidney dysfunction (42/124, 33.9%), diabetes mellitus/impaired glucose tolerance (24/124, 19.4%), and severe infection (29 cases, 23.4%). 58% of patients (57/124) developed severe aplastic anemia with a median progression time of 24 months (range: 3-216). Patients with absolute neutrophil count (ANC) <0.5×109/L, severe infection, or iron overload had a higher probability of progression to SAA (P=0.022, P=0.025, P=0.001). Patients receiving antithymocyte globulin (ATG) plus Cyclosporin A (CsA) had a higher overall response rate compared to those receiving CsA alone (56.7% vs 19.3%, P < 0.001). The addition of ATG was the favorable factor for efficacy (P=0.003). Fourteen patients developed secondary clonal hematologic disease: eleven patients with paroxysmal nocturnal hemoglobinuria, two patients with myelodysplastic syndromes, and one patient with acute myeloid leukemia, respectively. Ten patients (8.1%) died with a median follow-up of 12 months (range: 3- 36 months). Patients with TD-NSAA usually have a prolonged course of disease, and are prone to be complicated with important organ damage and disease progression to SAA. Intensive immunosuppressive therapy based on ATG might be an appropriate approach for TD-NSAA. Clinical trial registration: http://www.chictr.org.cn/edit.aspx?pid=125480&htm=4, identifier ChiCTR2100045895.

Keywords: antithymocyte globulin; aplastic anemia; cyclosporin A; immunosuppressive therapy; non-severe; transfusion-dependent.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Anemia, Aplastic* / therapy
  • Antilymphocyte Serum / therapeutic use
  • Cyclosporine / therapeutic use
  • Humans
  • Iron Overload* / drug therapy
  • Iron Overload* / etiology
  • Retrospective Studies

Substances

  • Cyclosporine
  • Antilymphocyte Serum

Grants and funding

This study was supported by the National Natural Science Foundation of China (Grant No. 81900109), Jiangsu Province Capability Improvement Project through Science, Technology, and Education (ZDXK202209), Young Scholars Fostering Fund of the First Affiliated Hospital of Nanjing Medical University (PY2022054), the Open Project of Immune Cell Transformation Research Center in the Affiliated Jiangning Hospital of Nanjing Medical University (JNYYZXKY202214) and the Research and Development Fund of Kangda College of Nanjing Medical University.