Iron chelation of hetrombopag in aplastic anemia: a post hoc analysis of a phase II study.

Ann Hematol

State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Chinese Academy of Medical Sciences & Peking Union Medical College, 288 Nanjing Road, Heping District, Tianjin, 300020, China.

Published: December 2022

AI Article Synopsis

  • Hetrombopag is a medicine approved in China that helps patients with a serious blood condition called severe aplastic anemia (SAA).
  • A study with 35 patients showed that more than half of them had lower levels of a substance called serum ferritin, indicating less iron in their blood, after using hetrombopag for 18 weeks.
  • The medicine was especially effective for patients who responded well to treatment, making it a promising option for reducing excess iron in the body.

Article Abstract

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 ΔSF 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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Source
http://dx.doi.org/10.1007/s00277-022-04968-8DOI Listing

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