Safety and efficacy of hetrombopag in patients with chronic immune thrombocytopenia: a single-arm, open-label, multi-center phase 1 study.

Autor:	Mei H; Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China., Chen X; Hematology Department, The First Affiliated Hospital of Air Force Medical University, Xi'an, China., Zhou J; Hematology Department, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China., Luo J; Hematology Department, The Second Hospital of Hebei Medical University, Shijiazhuang, China., Shi Q; Hematology Department, The Second Affiliated Hospital of Nanchang University, Nanchang, China., Liu J; Hematology Department, The Third Xiangya Hospital of Central South University, Changsha, China., Wu D; Hematology Department, The First Affiliated Hospital of Soochow University, Suzhou, China., Chen G; Hematology Department, The First Affiliated Hospital of Nanchang University, Nanchang, China., Tai Y; Department of Clinical Development, Jiangsu Hengrui Pharmaceuticals Co., Ltd., Shanghai, China., Xiong J; Department of Clinical Development, Jiangsu Hengrui Pharmaceuticals Co., Ltd., Shanghai, China., Zou J; Department of Clinical Development, Jiangsu Hengrui Pharmaceuticals Co., Ltd., Shanghai, China., Hu Y; Department of Hematology, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology, Wuhan, China.
Jazyk:	angličtina
Zdroj:	Annals of translational medicine [Ann Transl Med] 2022 Jan; Vol. 10 (2), pp. 30.
DOI:	10.21037/atm-21-4361
Abstrakt:	Background: Thrombopoietin receptor agonists (TPO-RAs) are promising therapeutic strategy for patients with immune thrombocytopenia (ITP). We conducted this phase 1 trial (NCT02614846) to evaluate the preliminary efficacy and safety of hetrombopag (a TPO-RA) in patients with ITP. Methods: Patients with ITP who had an insufficient response or had progressed on at least one standard treatment for ITP were given hetrombopag orally at an initial dose of 5 mg once daily for up to 6 weeks. The primary endpoint was the proportion of patients who achieved platelet counts of ≥50×10 9 /L at week 6. Results: A total of 37 eligible patients received hetrombopag treatment. This study met its primary endpoint, 22 (59.5%, 95% CI: 42.1-75.3) patients responded to hetrombopag, achieving platelet counts ≥50×10 9 /L at week 6. Of the 29 (78.4%, 95% CI: 61.8-90.2%) patients who responded at least once during the study, the median time from treatment initiation to first response was 2.1 weeks (95% CI: 1.3-4.1 weeks). The median accumulative response duration was 3.1 weeks [interquartile range (IQR), 2.1-4.1 weeks]. The incidence of bleeding was reduced with hetrombopag treatment compared to the baseline. Adverse events (AEs) occurred in 32 (86.5%) patients and treatment-related AEs occurred in 13 (35.1%) patients. Two (5.4%) serious AEs were reported, but neither were treatment related. The dose was modified in one (2.7%) patient due to an AE. There were no incidences of treatment discontinuation/interruption or death. Conclusions: Hetrombopag showed preliminary activity in elevating platelet counts and reducing bleeding in patients with chronic ITP who had received at least one standard therapy. It was well-tolerated. Competing Interests: Conflicts of Interest: All authors have completed the ICMJE uniform disclosure form (available at https://atm.amegroups.com/article/view/10.21037/atm-21-4361/coif). YT, JX, and JZ report that they are employees of Jiangsu Hengrui Pharmaceuticals Co., Ltd. All other authors report that this study was funded by Jiangsu Hengrui Pharmaceuticals Co., Ltd. The authors have no other conflicts of interest to declare. (2022 Annals of Translational Medicine. All rights reserved.)
Databáze:	MEDLINE
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