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

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机构： [1]State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital,NationalClinicalResearch Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Chinese Academyof Medical Sciences &Peking Union Medical College, 288 Nanjing Road, Heping District, Tianjin 300020, China [2]Jiangsu Province Hospital, Nanjing, China [3]Institute of Hematology, West China Hospital of SichuanUniversity, Chengdu, China [4]Peking Union Medical College Hospital, Beijing, China [5]The First Hospital of Bethune, Jilin University, Changchun,China [6]Guangzhou First People’s Hospital, Guangzhou, China [7]Huashan Hospital Afliated to Fudan University, Shanghai,China [8]The First Afliated Hospital of Nanchang University,Nanchang, China [9]Shaanxi Provincial People’s Hospital,Xi’an, China [10]The Second Hospital of Shanxi Medical University, Taiyuan,China [11]Tianjin Medical University General Hospital, Tianjin, China [12]Zhongda Hospital Afliated to Southeast University,Nanjing, China [13]The First Afliated Hospital of Zhengzhou University,Zhengzhou, China [14]Afliated Hospital of Nantong University, Nantong, China [15]Henan Cancer Hospital, Zhengzhou, China [16]The First Afliated Hospital of Suzhou University, Suzhou,China [17]Lanzhou University Second Hospital, Lanzhou, China

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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.© 2022. The Author(s), under exclusive licence to Springer-Verlag GmbH Germany, part of Springer Nature.

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大类 | 3 区医学

小类 | 3 区血液学

最新[2023]版：

大类 | 3 区医学

小类 | 3 区血液学

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第一作者机构： [1]State Key Laboratory of Experimental Hematology, Institute of Hematology & Blood Diseases Hospital,NationalClinicalResearch Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Chinese Academyof Medical Sciences &Peking Union Medical College, 288 Nanjing Road, Heping District, Tianjin 300020, China

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