Doptelet (avatrombopag) / SOBI, Fosun Pharma - LARVOL DELTA

Pulmonary Embolism and Myocardial Infarction With Non-obstructive Coronary Arteries in Immune Thrombocytopenia: Unmasking Underlying Antiphospholipid Syndrome. (PubMed, Cureus) - "This case report presents the clinical scenario of a 35-year-old male patient who experienced chest pain due to a combination of pulmonary embolism (PE) and myocardial infarction with non-obstructive coronary arteries (MINOCA), concurrently while undergoing treatment with avatrombopag for immune thrombocytopenia (ITP). After establishing the diagnosis, our patient was commenced on warfarin, and his treatment protocol for ITP was changed to a different drug. He remains under haematology follow-up."

Journal • Cardiovascular • Genetic Disorders • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Myocardial Infarction • Pain • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Successful treatment with favipiravir, steroids and avatrombopag of a patient with Crimean-Congo haemorrhagic fever in a high-level isolation unit (ESCMID Global 2026) - No abstract available

Clinical

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

Efficacy of Avatrombopag in Primary/Secondary Prevention of ADC Drug-Induced Thrombocytopenia in Breast Cancer (ChiCTR) - P=N/A | N=72 | Not yet recruiting | Sponsor: Fujian Cancer Hospital; Fujian Cancer Hospital

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

Comment on "The application of avatrombopag in chronic liver disease patients with severe thrombocytopenia undergoing procedures in China: a prospective real-world cohort study". (PubMed, Int J Surg) - No abstract available

Journal • Real-world evidence • Hematological Disorders • Hepatology • Thrombocytopenia

AVA-PED 301: Avatrombopag for the Treatment of Thrombocytopenia in Pediatric Subjects With Immune Thrombocytopenia for ≥6 Months (clinicaltrials.gov) - P3 | N=75 | Completed | Sponsor: Sobi, Inc. | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

Evaluation of Avatrombopag for the Treatment of Thrombocytopenia in Japanese Adults With Chronic ITP (clinicaltrials.gov) - P3 | N=19 | Completed | Sponsor: Sobi, Inc. | Active, not recruiting ➔ Completed

Trial completion • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

Efficacy and safety of avatrombopag in aplastic anemia: a comprehensive review of clinical evidence. (PubMed, Ther Adv Hematol) - "Eltrombopag improved outcomes when added to immunosuppressive therapy, but its association with hepatotoxicity limits its suitability for patients with underlying liver disease or elevated baseline liver enzymes. Its tolerability in patients with liver dysfunction distinguishes it from earlier agents in this drug class. Ongoing trials will clarify optimal dosing strategies and define its future role within first-line therapy and salvage treatment pathways for aplastic anemia."

Journal • Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hepatology • Liver Failure • Transplantation

Combining an immunomodulatory drug with a TPO-RA to treat multirefractory ITP patients: The Spanish ITP Group experience. (PubMed, Br J Haematol) - "The IS/IMs were steroids, purine synthesis inhibitors and fostamatinib. The TPO-RAs were romiplostim, eltrombopag and avatrombopag...Three thromboembolic events and 14 infections that required hospitalization were reported, none fatal. The combination of IS/IMs and TPO-RAs arises as an alternative option of treatment in multirefractory ITP."

Journal • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura

Avatrombopag in immune thrombocytopenia and beyond: Current evidence and emerging perspectives. (PubMed, Blood Rev) - "Real-world studies further reinforce these findings, supporting avatrombopag's profile in routine clinical practice. Beyond ITP, emerging evidence suggests potential roles for avatrombopag in other thrombocytopenic settings, including chemotherapy-induced thrombocytopenia, aplastic anemia, and post-hematopoietic stem-cell transplantation, where early data appear promising."

Journal • Review • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

Avatrombopag for treating children and adolescents with immune thrombocytopenia: a plain language summary. (PubMed, Expert Rev Hematol) - No abstract available

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

Avatrombopag Combined With All-trans Retinoic Acid in the Treatment of Primary Immune Thrombocytopenia (clinicaltrials.gov) - P2/3 | N=248 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China

New P2/3 trial • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

The holistic trial: A comprehensive evaluation of two second-line therapeutic approaches for immune thrombocytopenia (ITP) – a pragmatic randomized controlled trial (ASH 2025) - P3 | "This trial aims to determine which of the two most widely used second-line therapies, rituximab or TPO-RA (avatrombopag) offers the better outcome and should be selected first, and whether specific patient subgroups may benefit more from one approach than the other. Furthermore, we seek to acquire knowledge on patient's perspectives, incorporating their preferences and experiences in treatment decisions, especially in relation to HR QoL . Funding statement: The study is sponsored by Østfold Hospital, Norway and supported by a grant from the Norwegian Regional Health Authority and SOBI."

Clinical • Hematological Disorders • Hepatitis B • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammation • Thrombocytopenia • Thrombocytopenic Purpura

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

A case for CD38 targeted therapy in multi-refractory immune thrombocytopenia (ASH 2025) - "Initial treatment with high-dose dexamethasone and IVIG was ineffective. He subsequently received rituximab, eltrombopag and avatrombopag...During weeks 8–14, he was treated with romiplostim, a second steroid pulse, mycophenolate, cyclophosphamide, and cyclosporine without response...While this study is ongoing, we present this case to highlight that daratumumab can be associated with a rapid platelet recovery in multi-refractory ITP, was safe and well tolerated with fostamatinib and danazol, and produced a durable response after a finite treatment course (12 weekly doses). In conclusion, for patients with prolonged, severe ITP unresponsive to conventional treatments, daratumumab offers a rational, mechanism-based intervention. Our case contributes to growing evidence supporting anti-CD38 immunotherapy in ITP and underscores the need for clinical trials to more broadly evaluate other plasma cell directed therapies for the treatment of ITP."

Clinical • Autoimmune Hemolytic Anemia • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Hodgkin Lymphoma • Immune Thrombocytopenic Purpura • Immunology • Lymphoma • Non-Hodgkin’s Lymphoma • Thrombocytopenia • Thrombocytopenic Purpura

Avatrombopag: Beyond the technical sheet - a single-center experience (ASH 2025) - "Avatrombopag was initiated at 20 mg/day, increasing platelet counts to 40–90000/ml, enabling safe initiation of Dabigatran Etexilato. These encouraging observations underscore the need for prospective, controlled studies to validate the efficacy and safety of Avatrombopag in these off-label settings and to better define its role in personalized thrombocytopenia management. Ongoing real-world evidence is essential to inform the evolution of treatment guidelines"

Clinical • Atrial Fibrillation • Cardiovascular • Diabetes • Fibrosis • Hematological Disorders • Hemophilia • Hemophilia A • Hepatology • Hypertension • Immune Thrombocytopenic Purpura • Immunology • Metabolic Disorders • Portal Hypertension • Rare Diseases • Thrombocytopenia • Thrombocytopenic Purpura • Type 2 Diabetes Mellitus

Comparative effectiveness of second-line therapies for chronic immune thrombocytopenia: A network meta-analysis of RCTs (ASH 2025) - "The treatment groups were: Avatrombopag (96), Eltrombopag (283), Fostamatinib (101), Rilzabrutinib (133), Romiplostim (2116), and Rozanolixizumab (41)...Rituximab was analyzed indirectly and showed moderate efficacy... This NMA provides a comparative analysis of second-line therapies for chronic ITP, with Rozanolixizumab identified as the most effective treatment. Despite variability in safety profiles, all therapies demonstrated significant clinical benefits, supporting the adoption of personalized treatment strategies."

HEOR • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura • SYK

Avatrombopag: Beyond efficacy (ASH 2025) - "Prior to switch, 36.36% of patients received concomitant medication for ITP (preferably Prednisone in doses < 5-10 mg/day)...2, Cooper et al, The Cost-Effectiveness of Avatrombopag Versus Eltrombopag and Romiplostim in the Treatment of Patients with Immune Thrombocytopenia in the UK, K. J. Mark...Pascual C. et al, Avespa Study: Effectiveness and Safety of Avatrombopag in Immune Thrombocytopenia (ITP). a Real-World Study of the Spanish ITP Group (GEPTI).Blood (2024)144, Supplement 1, 713"

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

Patient-reported outcomes of avatrombopag for chronic immune thrombocytopenia: Interim analysis of the Phase 4 adopt study (ASH 2025) - "This interim analysis of the ADOPT study supports the real-world effectiveness and safety ofAVA for treating people with ITP. Improvements in PROs, including health status, HRQoL, and ITP-relatedactivity impairment, in addition to clinical outcomes, underscore the potential of AVA to benefit patientswith ITP."

Clinical • P4 data • Patient reported outcomes • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Mood Disorders • Psychiatry • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

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

Real world data on the use of thrombopoietin receptor agonists in patients after allogeneic hematopoietic stem cell transplantation (ASH 2025) - "Eltrombopag was the most frequently used TPO-RA followed by Romiplostim andAvatrombopag in 20 (74%), 4 (15%) and 3 (11%) patients, respectively. Given the observed data we conclude that TPO-RA treatment after allogeneic HSCT might be avaluable option for patients with prolonged or secondary thrombocytopenia. However, prospectiveanalyses are necessary to determine a potential difference between TPO-RA effectiveness and plateletrise in the course of delayed graft function."

Clinical • Real-world • Real-world evidence • Acute Graft versus Host Disease • Bone Marrow Transplantation • Cardiovascular • Chronic Graft versus Host Disease • Gastroenterology • Graft versus Host Disease • Hematological Disorders • Immunology • Thrombocytopenia • Transplantation • CD34

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

Mini-ATG/TBI conditioning regimen facilitates cure of acquired aplastic anemia in children via unrelated umbilical cord blood transplantation (ASH 2025) - P4 | "The mini-ATG/TBI regimen comprised: rabbit ATG (rATG, 2.5 mg/kg)on day -7, total body irradiation (TBI, 3 Gy) on day -7, fludarabine (FLU, 40 mg/m²) on days -6 to -2, andcyclophosphamide (CTX, 40 mg/m²) on days -4 and -2...In the mini-ATG/TBI group,odd-numbered patients received avatrombopag starting on day +7 to promote platelet engraftment,while even-numbered patients received thrombopoietin (TPO)... These data support the mini-ATG/TBI regimen as an effective conditioning approach forUCBT in acquired aplastic anemia, demonstrating particular utility in pediatric patients or low bodyweight adults.Key words: Aplastic Anemia; Unrelated Umbilical Cord Blood Transplantation; mini ATG"

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Epstein-Barr Virus Infections • Graft versus Host Disease • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Septic Shock • Transplantation

Clinical characteristics, treatment strategies, and response patterns in evans syndrome: Initial analysis from the spanish resti cohort (ASH 2025) - "Cyclosporine was used in two patients with dual-lineage cytopenias,resulting in AIHA response in 41 days and neutrophil recovery in 15 days.Fostamatinib was used in four patients with concurrent ITP and AIHA...The median time from diagnosis to fostamatinib initiation was 531 days.All patients were on concomitant therapy (corticosteroids or avatrombopag)...This analysis from RESTI highlights the clinical heterogeneity and treatment challenges in Evanssyndrome. Corticosteroids remain first line of therapy but theyt are associated with incomplete ortransient responses in many patients. Rituximab demonstrated moderate efficacy across lineages, whilefostamatinib showed rapid, durable responses in both ITP and AIHA without observed toxicity, evenallowing treatment withdrawal in some cases."

Clinical • Autoimmune Hemolytic Anemia • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Neutropenia • Thrombocytopenia • Thrombocytopenic Purpura