Hengqu (hetrombopag) / Jiangsu Hengrui Pharma - LARVOL DELTA

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

Prolonged thrombocytopenia following diverse CAR-T cell therapies: High incidence, inflammatory risk factors, and a comparative efficacy analysis of TPO-receptor agonist strategies in a real-world cohort (ASH 2025) - "The efficacy of TPO-RA interventions—includingmonotherapy (Eltrombopag, Avatrombopag, Hetrombopag, Lusutrombopag, Romiplostim, or rhTPO) andcombination strategies—was evaluated. Prolonged, severe thrombocytopenia is a frequent, inflammation-driven complication of CAR-T therapy. Our real-world data demonstrate a clear efficacy hierarchy among TPO-RAs, with romiplostimbeing the most effective single agent. Crucially, combination TPO-RA strategies, particularly thoseinvolving romiplostim or sequential rhTPO, significantly accelerate platelet recovery in severe caseswithout increasing toxicity."

CAR T-Cell Therapy • Clinical • Real-world • Real-world evidence • B Cell Non-Hodgkin Lymphoma • Cardiovascular • Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • IFNG

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

Hetrombopag for the treatment of chemotherapy-induced thrombocytopenia in advanced solid tumors: A multicenter, randomized, double-blind, placebo-controlled phase III trial (ASH 2025) - P3 | "In patients with CIT from solid tumors, continuous hetrombopag administration throughoutchemotherapy cycles maintained stable platelet counts and thereby facilitated on-schedule full-dosechemotherapy with a favorable safety profile, addressing an unmet clinical need and offering a viableoral strategy for CIT management."

Clinical • Metastases • P3 data • Colorectal Cancer • Gastric Cancer • Oncology • Solid Tumor • Thrombocytopenia

Thrombopoietin receptor agonists combined with intensive immunosuppressive therapy improve outcomes in elderly patients with severe aplastic anemia: A single-center retrospective study (ASH 2025) - "Of the cohort, 25received IST plus Hetrombopag (HPAG), including 13 with SAA and 12 with VSAA... The addition of TPO-RA to IST demonstrated superior efficacy compared to IST alone inelderly patients with SAA or VSAA, yielding higher remission rates and improved clinical outcomes whilemaintaining favorable tolerability. These results underscore the potential of TPO-RA combination therapyin this patient population and warrant further investigation."

Retrospective data • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Infectious Disease • Myelodysplastic Syndrome • Neutropenia • Novel Coronavirus Disease • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Hetrombopag for patients with persistent primary immune thrombocytopenia: a post hoc analysis of a multicenter, randomized phase Ⅲ trial. (PubMed, Res Pract Thromb Haemost) - P3 | "Furthermore, hetrombopag manifested favorable safety profile in patients with persistent ITP, and safety outcomes were comparable with those in chronic and overall ITP populations. Hetrombopag showed favorable safe and efficacy profile in patients with persistent ITP, aligning with results observed in patients with chronic ITP and overall ITP."

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

Combination of anti-CD20 and hetrombopag in relapsed/refractory immune thrombocytopenia: a case series. (PubMed, Front Med (Lausanne)) - "Accordingly, the patient was treated with avatrombopag monotherapy, which maintained the PLT count at normal levels...After treatment with ripertamab-hetrombopag, the patient received combination therapy with hetrombopag, glucocorticoids, tacrolimus, and hydroxychloroquine, with the PLT count being maintained within the normal range. The other two patients remained in sustained CR throughout the follow-up period. Combining anti-CD20 monoclonal antibody with hetrombopag may offer therapeutic benefits in patients with relapsed/refractory ITP."

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

Hetrombopag for thrombocytopenia induced by concurrent or sequential chemoradiotherapy in patients with solid tumors: a double-cohort trial. (PubMed, BMC Cancer) - No abstract available

Journal • Hematological Disorders • Oncology • Solid Tumor • Thrombocytopenia

Herombopag Treated T-DM1 Induced Platelet Reduction (clinicaltrials.gov) - P2 | N=56 | Recruiting | Sponsor: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University

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

Effect of Hetrombopag Combined with Rhtpo in Patients with Severe Immune Thrombocytopenia (ASH 2023) - "Patients receiving other ITP drugs as maintenance therapy were eligible if previous glucocorticoid therapy doses were stable, and rituximab was used for at least 2 months and other immunosuppressants were stable for at least 4 weeks. Patients who had received IVIG, avatrombopag, eltrombopag, or romiplostim within 2 weeks prior to enrollment were excluded... This study innovatively explores the efficacy and safety of combination therapy with hetrombopag and rhTPO in the treatment of severe ITP. It is preliminarily confirmed that this combination therapy can effectively and quickly improve the early platelet response of ITP patients, avoid bleeding damage, and is expected to become a new treatment choice for severe ITP patients. We also look forward to the overall efficacy evaluation results of all patients after completing treatment in the future."

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

Lusutrombopag or hetrombopag supports in vitro megakaryopoiesis better than other thrombopoietin receptor agonists. (PubMed, Br J Haematol) - No abstract available

Journal • Preclinical

A Clinical Study of Hetrombopag for Prevention of Thrombocytopenia Induced by Gemcitabine Plus Cisplatin in the Treatment of Nasopharyngeal Carcinoma (clinicaltrials.gov) - P2 | N=35 | Not yet recruiting | Sponsor: Fujian Cancer Hospital

New P2 trial • Head and Neck Cancer • Hematological Disorders • Nasopharyngeal Carcinoma • Oncology • Solid Tumor • Thrombocytopenia