期刊
ANNALS OF HEMATOLOGY
卷 101, 期 12, 页码 2611-2616出版社
SPRINGER
DOI: 10.1007/s00277-022-04968-8
关键词
Aplastic anemia; Hetrombopag; Iron chelation
类别
资金
- National Nature Science Foundation of China [81900127]
- Non-profit Central Research Institute Fund of Chinese Academy of Medical Science [2020-RW310-002]
Hetrombopag is the only CFDA-approved thrombopoietin receptor agonist for severe aplastic anemia in China. A post hoc analysis of a phase II clinical trial showed that hetrombopag had a powerful and rapid iron chelation effect.
Hetrombopag is the only CFDA-approved thrombopoietin (TPO) receptor agonist for severe aplastic anemia (SAA) in China. Its chemical structure has an iron chelation domain. To explore the iron chelation effect of hetrombopag, we performed a post hoc analysis of the phase II clinical trial (NCT03557099). Thirty-five immunosuppressive therapy (IST)-refractory SAA patients were enrolled in the study, and the longitudinal changes of serum ferritin (SF) were assessed. At 18 weeks post-hetrombopag initiation, 51.4% of patients showed decreased SF levels by a median of 49.0 (18.1-95.5) % from baseline (median Delta SF decrease value, 917.2 ng/ml, range from 104.0 to 7030.0 ng/ml). A decrease in SF was found in 75.0% of hematologic responders and 31.6% of non-responders. Among the 24 patients with iron overload, 12 had decreased SF levels by up to 51% of the baseline. Patients with normal SF levels also showed decreased SF levels, and iron deficiency occurred in two patients. In conclusion, hetrombopag showed a powerful and rapid iron chelation effect.
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