Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study
Abstract Background Clinical data on hetrombopag, a novel thrombopoietin receptor agonist (TPO-RA) developed in China, remain limited for the treatment of immune thrombocytopenia (ITP). This retrospective study aimed to investigate the efficacy and safety of hetrombopag for ITP patients in the rea...
Saved in:
| Main Authors: | , , , , , |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
BMC
2025-07-01
|
| Series: | European Journal of Medical Research |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s40001-025-02850-7 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
| _version_ | 1849238735573483520 |
|---|---|
| author | Dan Wang Cheng Chang Zhongmin Zou Yao Quan Xi Zhang Yimei Feng |
| author_facet | Dan Wang Cheng Chang Zhongmin Zou Yao Quan Xi Zhang Yimei Feng |
| author_sort | Dan Wang |
| collection | DOAJ |
| description | Abstract Background Clinical data on hetrombopag, a novel thrombopoietin receptor agonist (TPO-RA) developed in China, remain limited for the treatment of immune thrombocytopenia (ITP). This retrospective study aimed to investigate the efficacy and safety of hetrombopag for ITP patients in the real-world setting. Methods Patients with ITP received hetrombopag monotherapy at an initial dose of 2.5 mg or 5.0 mg once daily. The primary endpoint was the proportion of responders whose platelet count ≥ 50 × 109 /L, which were assessed at week 4, 8 and 12 after treatment. The secondary endpoint was the safety profile of hetrombopag. Results A total of 50 ITP patients were enrolled. The median time to response was 7 days; the mean platelet count increased from 16 × 10⁹/L at baseline to 104 × 10⁹/L at week 12; the response rate was 71% at week 12. The 5 mg/d group showed significantly better efficacy as early as week 2 compared with the 2.5 mg/d group (P < 0.05). Among 15 nonresponders in the 2.5 mg group, 11 patients were switched to the 5 mg group, and 6 of them then achieved response. The clinical response was reached in 7 of 8 ITP patients switching from eltrombopag to hetrombopag. There was no difference in the overall response rate between patients receiving hetrombopag as a second-line therapy and later-line therapies (P = 0.951). No serious adverse events were observed. In addition, the total number of megakaryocytes in the bone marrow was positively associated with treatment efficacy at week 4 (P = 0.001). Conclusions Hetrombopag was effective and safe in the real-world treatment of ITP. An initial dosage of hetrombopag 5 mg/d and a higher megakaryocyte count in bone marrow may predict better therapeutic efficacy. |
| format | Article |
| id | doaj-art-efc2dedc50e9462b80366e98143689f9 |
| institution | Kabale University |
| issn | 2047-783X |
| language | English |
| publishDate | 2025-07-01 |
| publisher | BMC |
| record_format | Article |
| series | European Journal of Medical Research |
| spelling | doaj-art-efc2dedc50e9462b80366e98143689f92025-08-20T04:01:25ZengBMCEuropean Journal of Medical Research2047-783X2025-07-013011810.1186/s40001-025-02850-7Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort studyDan Wang0Cheng Chang1Zhongmin Zou2Yao Quan3Xi Zhang4Yimei Feng5Department of Hematology, The Second Affiliated Hospital of Army Medical University (Third Military Medical University)Department of Hematology, The Fifth People’s Hospital of ChongqingDepartment of Chemical Defense Medicine, School of Military Preventive Medicine, Army Medical UniversityDepartment of Hematology, The Second Affiliated Hospital of Army Medical University (Third Military Medical University)Department of Hematology, The Second Affiliated Hospital of Army Medical University (Third Military Medical University)Department of Hematology, The Second Affiliated Hospital of Army Medical University (Third Military Medical University)Abstract Background Clinical data on hetrombopag, a novel thrombopoietin receptor agonist (TPO-RA) developed in China, remain limited for the treatment of immune thrombocytopenia (ITP). This retrospective study aimed to investigate the efficacy and safety of hetrombopag for ITP patients in the real-world setting. Methods Patients with ITP received hetrombopag monotherapy at an initial dose of 2.5 mg or 5.0 mg once daily. The primary endpoint was the proportion of responders whose platelet count ≥ 50 × 109 /L, which were assessed at week 4, 8 and 12 after treatment. The secondary endpoint was the safety profile of hetrombopag. Results A total of 50 ITP patients were enrolled. The median time to response was 7 days; the mean platelet count increased from 16 × 10⁹/L at baseline to 104 × 10⁹/L at week 12; the response rate was 71% at week 12. The 5 mg/d group showed significantly better efficacy as early as week 2 compared with the 2.5 mg/d group (P < 0.05). Among 15 nonresponders in the 2.5 mg group, 11 patients were switched to the 5 mg group, and 6 of them then achieved response. The clinical response was reached in 7 of 8 ITP patients switching from eltrombopag to hetrombopag. There was no difference in the overall response rate between patients receiving hetrombopag as a second-line therapy and later-line therapies (P = 0.951). No serious adverse events were observed. In addition, the total number of megakaryocytes in the bone marrow was positively associated with treatment efficacy at week 4 (P = 0.001). Conclusions Hetrombopag was effective and safe in the real-world treatment of ITP. An initial dosage of hetrombopag 5 mg/d and a higher megakaryocyte count in bone marrow may predict better therapeutic efficacy.https://doi.org/10.1186/s40001-025-02850-7Relapsed/refractory immune thrombocytopenia (R/R ITP)HetrombopagReal-worldOverall response rateThrombopoietin receptor agonist (TPO-RA) |
| spellingShingle | Dan Wang Cheng Chang Zhongmin Zou Yao Quan Xi Zhang Yimei Feng Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study European Journal of Medical Research Relapsed/refractory immune thrombocytopenia (R/R ITP) Hetrombopag Real-world Overall response rate Thrombopoietin receptor agonist (TPO-RA) |
| title | Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study |
| title_full | Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study |
| title_fullStr | Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study |
| title_full_unstemmed | Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study |
| title_short | Real-world experience of hetrombopag in immune thrombocytopenia treatment: a retrospective cohort study |
| title_sort | real world experience of hetrombopag in immune thrombocytopenia treatment a retrospective cohort study |
| topic | Relapsed/refractory immune thrombocytopenia (R/R ITP) Hetrombopag Real-world Overall response rate Thrombopoietin receptor agonist (TPO-RA) |
| url | https://doi.org/10.1186/s40001-025-02850-7 |
| work_keys_str_mv | AT danwang realworldexperienceofhetrombopaginimmunethrombocytopeniatreatmentaretrospectivecohortstudy AT chengchang realworldexperienceofhetrombopaginimmunethrombocytopeniatreatmentaretrospectivecohortstudy AT zhongminzou realworldexperienceofhetrombopaginimmunethrombocytopeniatreatmentaretrospectivecohortstudy AT yaoquan realworldexperienceofhetrombopaginimmunethrombocytopeniatreatmentaretrospectivecohortstudy AT xizhang realworldexperienceofhetrombopaginimmunethrombocytopeniatreatmentaretrospectivecohortstudy AT yimeifeng realworldexperienceofhetrombopaginimmunethrombocytopeniatreatmentaretrospectivecohortstudy |