Hengqu (hetrombopag) / Jiangsu Hengrui Pharma - LARVOL DELTA

A Multicenter, Single-Arm Exploratory Clinical Study on the Efficacy and Safety of Herombopag Olamine Tablets in Patients with Breast Cancer Experiencing Thrombocytopenia Induced by T-DM1 (ChiCTR) - P2 | N=56 | Recruiting | Sponsor: Sun Yat-sen Memorial Hospital, Sun Yat-sen University; Sun Yat-sen Memorial Hospital, Sun Yat-sen University

New P2 trial • Breast Cancer • Oncology • Solid Tumor • Thrombocytopenia

Hetrombopag for the treatment of lower-risk myelodysplastic syndromes with thrombocytopenia: A prospective, single-arm, multicenter study (ASH 2025) - P=N/A | "Hetrombopag is effective in increasing platelet counts and demonstrates an acceptabletoxicity profile in LR-MDS patients with thrombocytopenia, suggesting that hetrombopag may be apromising new therapeutic option for this population. Nevertheless, further larger, prospective andcontrolled studies are warranted to better define the role of hetrombopag in the treatment of LR-MDS."

Clinical • Acute Myelogenous Leukemia • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Immune Thrombocytopenic Purpura • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Thrombocytopenia • Thrombocytopenic Purpura • ASXL1 • BCOR • DNMT3A • U2AF1

A multicenter, prospective, phase II clinical study of hytrombopag ethanolamine tablets combined with immunosuppressive therapy in newly diagnosed patients with non-severe aplastic anemia (ChiCTR) - P=N/A | N=76 | Not yet recruiting | Sponsor: Tianjin Medical University General Hospital; Tianjin Medical University General Hospital

New trial • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies

Hetrombopag for the treatment of chemotherapy-induced thrombocytopenia in patients with solid tumors. (PubMed, Res Pract Thromb Haemost) - "Safety profile showed good tolerability of hetrombopag (monotherapy or combined with rhTPO/rhIL-11) in patients. Hetrombopag may be an effective and well-tolerated treatment option for CIT in patients with solid tumors."

Journal • Review • Hematological Disorders • Oncology • Solid Tumor • Thrombocytopenia

Efficacy and Safety of Herombopag Ethanolamine Tablets in Combination with Cyclosporine and Androgen for the Treatment of Transfusion-Dependent Non-Severe Aplastic Anemia (TD-NSAA) Patients—A Single-Arm, Prospective, Multicenter Phase II Clinical Study (ChiCTR) - P4 | N=42 | Recruiting | Sponsor: West China Hospital of Sichuan University; West China Hospital of Sichuan University

New P4 trial • Anemia • Aplastic Anemia • Hematological Disorders

COMPARATIVE STUDY OF HETROMBOPAG VS. ELTROMBOPAG IN PROMOTING PLATELET ENGRAFTMENT POST-AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION: A MULTICENTER CLINICAL TRIAL (EBMT 2026) - "This study is the first to demonstrate that hetrombopag significantly accelerates neutrophil and platelet engraftment in auto-HSCT patients compared to eltrombopag."

Clinical • Acute Myelogenous Leukemia • B Cell Lymphoma • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Leukemia • Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Nephrology • Non-Hodgkin’s Lymphoma • T Cell Non-Hodgkin Lymphoma • Thrombocytopenia • Transplantation

No increased risk of hepatic enzyme abnormalities with non-peptidic thrombopoietin receptor agonists in immune thrombocytopenia: A meta-analysis of randomized controlled trials. (PubMed, Exp Ther Med) - "Non-peptidic thrombopoietin receptor agonists (TPO-RAs), including eltrombopag, avatrombopag and hetrombopag, are used as second-line therapies for immune thrombocytopenia (ITP). The present meta-analysis was registered in the International Prospective Register of Systematic Reviews (registration no. CRD420251084782)."

Journal • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Long-term efficacy and safety of hetrombopag in paediatric patients with immune thrombocytopenia. (PubMed, Br J Haematol) - "Furthermore, among the nine patients who switched from other TPO-RAs, seven patients achieved OR during initial treatment, including three patients who achieved CR and four patients who achieved R. The overall incidence of adverse events was 37.6%, with no serious adverse events reported. Our findings highlight that hetrombopag is both safe and effective in paediatric patients and may serve as a viable option for patients for whom first-line therapy fails."

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

A Study of Hetrombopag Combined With rhTPO in the Treatment of Cancer Therapy-induced Thrombocytopenia (CTIT) in Solid Tumor Patients. (clinicaltrials.gov) - P2 | N=204 | Not yet recruiting | Sponsor: Sun Yat-sen University

New P2 trial • Oncology • Solid Tumor • Thrombocytopenia

Hetrombopag Added to Cyclosporine as the First-Line Treatment for Patients With Non-Severe Aplastic Anemia: A Phase 2 Multicenter Trial. (PubMed, Am J Hematol) - P2 | "Cyclosporine A (CsA) monotherapy often yields suboptimal responses, highlighting an unmet clinical need for more effective therapies. Importantly, no clonal progression to myelodysplastic syndrome or leukemia was observed. These findings support hetrombopag plus CsA as a potential first-line therapeutic intervention for NSAA, especially in TD-NSAA patients."

Journal • P2 data • Anemia • Aplastic Anemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

A Study to Evaluate the Efficacy and Safety of Hetrombopag Olamine Tablets Vs Placebo in Patients With Chemotherapy-Induced Thrombocytopenia (clinicaltrials.gov) - P3 | N=150 | Not yet recruiting | Sponsor: Jiangsu HengRui Medicine Co., Ltd.

New P3 trial • Hematological Disorders • Neutropenia • Thrombocytopenia

Hetrombopag for enhancing platelet engraftment after haploidentical allogeneic hematopoietic stem cell transplantation in patients with severe thalassemia: An observational study (ASH 2025) - P | "Graft-versus-host disease prophylaxis included cyclophosphamide at a total dose of 100 mg/kg (divided into two doses on day +3 and +4), methotrexate at 10 mg/m² (administered on days +1, +2, +5, and +6), and cyclosporine A at 4 mg/kg/day starting from day +6. A significant difference in transfusion volume between groups may have introduced bias in post-transplant outcome analysis. These findings suggest that relying solely on platelet engraftment time as a measure of engraftment efficacy may be inadequate and potentially limiting."

Clinical • Observational data • Beta-Thalassemia • Bone Marrow Transplantation • Genetic Disorders • Graft versus Host Disease • Hematological Disorders • Immunology • Liver Failure • Musculoskeletal Pain • Nephrology • Transplantation

Comparative study of hetrombopag vs. eltrombopag in promoting platelet engraftment post-autologous hematopoietic stem cell transplantation: A multicenter clinical trial (ASH 2025) - "This study is the first to demonstrate that hetrombopag significantly accelerates neutrophil and platelet engraftment in auto-HSCT patients compared to eltrombopag."

Clinical • Acute Myelogenous Leukemia • Aplastic Anemia • B Cell Lymphoma • Bone Marrow Transplantation • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Hodgkin Lymphoma • Immune Thrombocytopenic Purpura • Leukemia • Lymphoma • Mantle Cell Lymphoma • Multiple Myeloma • Nephrology • Non-Hodgkin’s Lymphoma • T Cell Non-Hodgkin Lymphoma • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

Trial in progress: Romiplostim N01 combined with immunosuppressive therapy in patients diagnosed with non-severe aplastic anemia, a Phase II study (ASH 2025) - P2 | "However, clinical data on romiplostim in NSAA remain limited, and no studies to date have evaluated the efficacy and safety of Romiplostim N01 in this population.Study Design and Methods We initiated a prospective, open-label, multi-centre, single-arm phase II trial (ChiCTR2500096280), conducted across multiple regions in China, to evaluate the efficacy and safety of Romiplostim N01 combined with cyclosporine or tacrolimus in patients diagnosed with NSAA and severe thrombocytopenia (platelet counts <30×10⁹/L)...Major eligibility criteria are: age ≥ 16, confirmed NSAA diagnosis, ECOG performance status of 0–2, QT interval <460 ms on electrocardiogram, adequate hepatic and renal function, prior TPO-RA recipients (including eltrombopag, hetrombopag, or avatrombopag) must complete a 1-month washout period before enrollment.Endpoints The primary endpoint is the overall hematologic response rate at weeks 12 and 24...A total of 40 subjects are..."

Clinical • P2 data • Anemia • Aplastic Anemia • Thrombocytopenia

First report: A novel deep intronic mutation and treatment using hetrombopag olamine of a patient with MYH9 syndrome and immune thrombocytopenia (ASH 2025) - "He hadbeen misdiagnosed with ITP for over a decade, showing poor response to steroids, eltrombopag, andrhTPO. This is the first report of MYH9-RD caused by a deep intronic c.2977-75C>T mutation, whichinduces exonization and protein truncation. The positive clinical response to hetrombopag olaminedemonstrates its therapeutic potential in MYH9-RD. Our findings support the inclusion of deep intronicvariants in diagnostic pipelines and suggest TPO-RAs as a promising therapeutic approach in such cases."

Clinical • Cataract • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Ophthalmology • Otorhinolaryngology • Rare Diseases • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura • MYH9 • RAS

Response to Hetrombopag Among patients with acute and persistent primary immune thrombocytopenia: A real-world study in China (ASH 2025) - "Most patients with acute and persistent ITP achieved a treatment response withhetrombopag therapy. Acute ITP patients showed better therapeutic outcomes when treated withhetrombopag. The average platelet count increased with prolonged treatment duration."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Liver Failure • Thrombocytopenia • Thrombocytopenic Purpura

Short-term hetrombopag for the management of preoperative thrombocytopenia: A multicenter clinical trial (ASH 2025) - P4 | "Hetrombopag 7.5 mg daily demonstrated a high overall achievement rate (85.7%) forincrement of platelet count to the desired threshold within 14 days before surgery. Success wasunaffected by baseline-target difference or prior TPO-RA exposure in this cohort."

Clinical • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Real-world clinical trial of hetrombopag (TPO-RA) in the treatment of primary immune thrombocytopenia (ITP) (ASH 2025) - "This real-world study further validates the overall efficacy and favorable safety profile of Hetrombopag inITP treatment. Consistent therapeutic benefits were observed across all ITP stages, indicating comparableefficacy in newly diagnosed, persistent, and chronic ITP patients."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Liver Failure • Thrombocytopenia • Thrombocytopenic Purpura

Hetrombopag for patients with persistent primary immune thrombocytopenia: A post-hoc analysis of a multicenter, randomized phase Ⅲ Trial (ASH 2025) - P3 | "Hetrombopag indicated favorable safe and efficacy performance in patients with persistentITP, aligning with results observed in chronic ITP and overall ITP patients."

Clinical • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and safety of hetrombopag, a novel thrombopoietin receptor agonist, in children and adolescents with immune thrombocytopenia: Results from a randomized, multicenter, placebo-controlled phase 3 trial (ASH 2025) - P3 | "Once-daily oral hetrombopag produced durable platelet responses and demonstrated afavorable safety profile in children and adolescents with chronic ITP, supporting its potential as a noveloral second-line treatment option in this population."

Clinical • P3 data • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

TPO-ras for persistent thrombocytopenia following CAR-T therapy in multiple myeloma: A multicenter clinical experience (ASH 2025) - "Univariate andmultivariate analyses were conducted between patients with PT (cohort 1) and without PT (cohort 2).Patients with PT were treated with thrombopoietin receptor agonists (TPO-RAs), including: hetrombopag(initial dose: 2.5 mg/day), avatrombopag (initial dose: 20 mg/day), eltrombopag (initial dose: 50 mg/day).Response was defined as transfusion independency along with resolution of platelets > 50×10⁹/L forthree consecutive values on different days. Hyperferritinemia post-CAR-T independently predicted PT, while TPO-RAs demonstrated both platelet-boosting effects and marrow recovery. Further prospective studies are warranted to validate thesefindings and optimize therapeutic protocols."

Clinical • IO biomarker • Hematological Disorders • Hematological Malignancies • Multiple Myeloma • Thrombocytopenia

Real-world study on thrombopoietin receptor agonists combined with rituximab in the treatment of Relapsed/Refractory primary immune thrombocytopenia (ASH 2025) - "Combination regimens utilized avatrombopag (n=23), eltrombopag (n=32), or hetrombopag (n=33).Rituximab was administered as standard-dose (375 mg/m² weekly ×4; n=41) or reduced-dose regimens(n=47). Of 42 patients who discontinued TPO-RAs from thecombination regimen, cessation reasons included: drug-related hepatotoxicity (n=2), inadequatetherapeutic response (n=27), and 13 patients achieving sustained remission after complete treatmentwithdrawal. After combination therapy, only 4 cases (4.5%) experienced grade ≥3 adverse events (2 cases ofpulmonary infection and 2 cases of abnormal liver function).ConclusionThis multicenter study demonstrates that combination therapy with TPO-RAs and rituximab appears tobe a favorable option for relapsed/refractory ITP patients, including patients with prior TPO-RAs failure."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and safety of hetrombopag in the treatment of Pediatric immune thrombocytopenia: A Real-World Observational Study (ASH 2025) - P | "No death or serious adverse events were reported.Conclusion Hetrombopag showed efficacy in elevating and sustaining platelet counts in pediatric ITPpatients, with a favorable safety profile. Notably, it remained effective even in patients who had showninadequate response to prior eltrombopag therapy."

Clinical • Observational data • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

Real-world safety and effectiveness of hetrombopag in patients with primary immune thrombocytopenia (ASH 2025) - P | "Management of ITP remains challenging, particularly forpatients who are refractory or intolerant to first-line treatments such as corticosteroids or intravenousimmunoglobulin (IVIg). Hetrombopag demonstrated favorable real-world safety and effectiveness in patients withITP, leading to sustained improvements in platelet counts and reduced reliance on concomitanttherapies. These findings provide meaningful evidence supporting the use of hetrombopag in routineclinical practice and underscore its potential as a core component in the long-term management of ITP."

Clinical • Real-world • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and safety of romiplostim N01 combined with cyclosporine in refractory aplastic anemia patients:a multicenter phase II study (ASH 2025) - P4 | "This prospective, open-label, multicenterphase II trial (ChiCTR2400085301) aims to evaluate the efficacy and safety of romiplostim N01 incombination with cyclosporine in patients with refractory AA.Eligible patients had been previously treated with standard IST (cyclosporine and/or anti-thymocyteglobulin) plus TPO-RA (eltrombopag or hetrombopag) for at least 3 months without response. Romiplostim N01 in combination with cyclosporine demonstrated promising efficacy and a favorablesafety profile in patients with AA refractory to multiple TPO-RAs plus IST. These preliminary resultssupport further investigation of this combination as a therapeutic option for refractory AA."

Clinical • P2 data • Anemia • Aplastic Anemia • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura