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

Synergistic Effects of Romiplostim and Avatrombopag in Severe Chemotherapy-Induced Thrombocytopenia: A Serendipitous Finding. (PubMed, OMICS) - "Romiplostim combined with avatrombopag yields rapid platelet recovery with favorable safety for severe refractory CIT, enabling chemotherapy resumption. It is a promising salvage strategy for single-agent TPO-RA nonresponders, warranting prospective randomized controlled trials for validation."

Journal • Cardiovascular • Thrombocytopenia • Venous Thromboembolism

VAY2EXPLORE: A Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's Choice Thrombopoietin Receptor Agonist (IC TPO-RA) in Patients With Primary Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P2 | N=164 | Recruiting | Sponsor: Novartis Pharmaceuticals | Trial primary completion date: May 2028 ➔ Jan 2029

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

Efficacy and safety of different TPO-RAs combined with cyclosporine in the treatment of NSAA. (PubMed, Ther Adv Hematol) - "Currently, three small-molecule TPO-RAs (eltrombopag, hetrombopag, and avatrombopag) are available for clinical use and work well...A retrospective analysis was performed to assess the efficacy and side effects of cyclosporine A (CsA) combined with different TPO - RAs in 67 NSAA patients...Similar to SAA, NSAA patients should also be initiated with an adequate dose of TPO-RAs therapy. Elderly patients and those with concurrent infections are at higher risk of mortality, prolonged hospitalizations, and require more clinical attention."

Journal • Aplastic Anemia • Hematological Disorders • Hepatology • Infectious Disease • Liver Failure

Avatrombopag as a novel salvage therapy for refractory thrombocytopenia in THPO-mutated congenital amegakaryocytic thrombocytopenia after allogeneic hematopoietic stem cell transplantation. (PubMed, Haematologica) - "Not available."

Journal • Bone Marrow Transplantation • Hematological Disorders • Thrombocytopenia • Transplantation

Neurologic complications of Evans syndrome. (PubMed, BMJ Case Rep) - "Despite limited improvement with first-line therapies, the patient experienced haematologic recovery and clinical stabilisation following initiation of avatrombopag, a thrombopoietin receptor agonist. This case highlights the importance of early recognition of treatment refractoriness and supports the emerging role of avatrombopag as a valuable therapeutic option for persistent thrombocytopenia in Evans syndrome."

Journal • Autoimmune Hemolytic Anemia • Cerebral Hemorrhage • CNS Disorders • Critical care • Epilepsy • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Thrombocytopenia • Thrombocytopenic Purpura

ACT-GI: Avatrombopag vs. Placebo for CIT in GI Malignancies (clinicaltrials.gov) - P2 | N=49 | Completed | Sponsor: Hanny Al-Samkari, MD | Active, not recruiting ➔ Completed | Trial completion date: Jul 2026 ➔ Apr 2026

Trial completion • Trial completion date • Gastrointestinal Cancer • Oncology • Solid Tumor • Thrombocytopenia

Avatrombopag in thrombopoietin receptor agonist-naïve patients with immune thrombocytopenia: real-world treatment patterns and clinical outcomes from the REAL-AVA 3.0 study. (PubMed, Res Pract Thromb Haemost) - "Most living patients who discontinued AVA (59.6%) received no further ITP treatment. AVA is an effective treatment option for TPO-RA-naïve patients with ITP, demonstrating a high real-world rate of sustained PC response."

Clinical data • HEOR • Journal • Real-world evidence • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

A Study of Avatrombopag in Adults With Aplastic Anemia Who Did Not Respond to, Cannot Receive, or Had Return of Disease After Standard Treatment (clinicaltrials.gov) - P2/3 | N=26 | Not yet recruiting | Sponsor: Swedish Orphan Biovitrum

New P2/3 trial • Anemia • Aplastic Anemia • Hematological Disorders

Outcomes, Treatment Patterns, and Resource Use in European Adults with Immune Thrombocytopenia Treated with Avatrombopag. (PubMed, TH Open) - "The incidence rate (95% confidence interval) of ITP-related outpatient visits and inpatient admissions was 8.9 (8.2-9.5) and 0.1 (0.1-0.2) per person-year. Conclusion This study supports clinical trials' findings and other postmarketing evidence demonstrating the effectiveness of avatrombopag."

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

Treatment of CCHF with supportive care, favipiravir, and avatrombopag. (PubMed, Clin Infect Dis) - "This multimodal approach was associated with rapid platelet recovery, reduced transfusion requirements, and viral clearance. Combining targeted antivirals with thrombopoietin receptor agonists represents a promising strategy for managing severe CCHF coagulopathy."

Journal • Hematological Disorders

Managing Acute STEMI in the Setting of Immune Thrombocytopenic Purpura: A Therapeutic Dilemma. (PubMed, JACC Case Rep) - "Thrombocytopenia in ITP does not protect against acute coronary syndrome. Navigating the balance between bleeding and clotting following a STEMI event requires a tailored, flexible approach."

Journal • Acute Coronary Syndrome • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Myocardial Infarction • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Avatrombopag in heavily pretreated chronic immune thrombocytopenia: a single center experience from Oman. (PubMed, Front Med (Lausanne)) - "ADRs occurred in 33.3% (primarily body pain and rash); no hepatotoxicity or treatment discontinuation due to ADRs was documented. In chronic, heavily pretreated ITP, avatrombopag was associated with clinically meaningful platelet responses, substantial steroid-sparing, and an acceptable tolerability profile."

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

Second Quarter Business Highlights:…Commercial Products (GlobeNewswire) - "ETUARY (pirfenidone), the Company's primary product approved in China for idiopathic pulmonary fibrosis (IPF), generated $28.0 million in sales for the quarter ended June 30, 2026, compared to $23.5 million for the same period in 2025....Contiva (avatrombopag maleate tablets), launched in March 2025 and indicated for thrombocytopenia in adults with chronic liver disease and immune thrombocytopenic purpura, generated $0.9 million in sales for the quarter ended June 30, 2026, compared to $1.5 million for the same period in 2025."

Sales • Idiopathic Pulmonary Fibrosis • Immune Thrombocytopenic Purpura

Avatrombopag switching from other thrombopoietic agents in severe and persistent cancer treatment-induced thrombocytopenia: a multicentre real-world study. (PubMed, Front Med (Lausanne)) - "No grade ≥3 adverse events were observed. Switching to avatrombopag was associated with platelet recovery, reduced transfusion dependence, fewer interruptions to antineoplastic therapy, and an acceptable safety profile for severe and persistent CTIT."

Journal • Real-world evidence • Hematological Disorders • Hematological Malignancies • Oncology • Solid Tumor • Thrombocytopenia

Real-world efficacy and safety of avatrombopag in Japanese patients with chronic liver disease and severe thrombocytopenia. (PubMed, J Gastroenterol) - "While interpretation requires caution due to the small sample size, in Japanese real-world practice, AVA was relatively safe and effective in preventing perioperative bleeding and avoiding platelet transfusions."

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

Efficacy and Safety of Avatrombopag in Patients with Chronic Liver Disease and Thrombocytopenia Undergoing Elective Surgery. (PubMed, J Clin Med) - "No thrombotic or bleeding events were recorded in this small cohort; however, the limited sample size precludes definitive conclusions on thrombosis risk. Avatrombopag demonstrated high efficacy and favorable safety for elevating PLT in patients with CLD, with a higher baseline PLT predicting a better response."

Journal • Cardiovascular • Hematological Disorders • Hepatology • Thrombocytopenia • Thrombosis

Thrombopoietin Receptor Agonists Versus Rituximab for Second-Line Treatment of Immune Thrombocytopenia in Patients With Active Solid Malignancies: A Systematic Review and Meta-Analysis (ISTH 2026) - "After failure of first-line corticosteroids, both thrombopoietin receptor agonists (TPO-RAs: romiplostim, eltrombopag, avatrombopag) and rituximab are widely used second-line therapies. No funding was received for this study. DOI*10.1016/j.rpth.2026.105541"

Retrospective data • Review • Immune Thrombocytopenic Purpura • Infectious Disease • Oncology • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Eltrombopag Response in MYH9-related diseases is Genotype-Dependent: A Systematic Evaluation (ISTH 2026) - "One patient with a p.G1517V tail-domain mutation remained NR but achieved CR after switching to avatrombopag 40 mg/day for 2 weeks. To achieve faster platelet elevation, a higher initial dose may be administered. Table or Figure Upload (1) Page 2 Table or Figure Upload (2) Page 3 DOI*10.1016/j.rpth.2026.106166"

Hematological Disorders

REAL-WORLD OUTCOMES OF AVATROMBOPAG AFTER PRIOR ELTROMBOPAG OR ROMIPLOSTIM THERAPY IN IMMUNE THROMBOCYTOPENIA: A STUDY FROM THE SPANISH GEPTI GROUP (GEPTI) (ISTH 2026) - "These findings support AVA as an effective and safe therapeutic option for patients who have failed ELT or ROMI due to Page 2 inadequate efficacy, convenience or AEs. Table or Figure Upload (1) DOI*10.1016/j.rpth.2026.103648"

Clinical • Real-world • Real-world evidence • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Ischemic stroke • Thrombocytopenia • Thrombocytopenic Purpura

INDIRECT COMPARISON OF ORAL THROMBOPOIETIN RECEPTOR AGONISTS IN PAEDIATRIC INMUNE THROMBOCYTOPENIA: EFFICACY, SAFETY AND ADHERENCE ANALYSIS (ISTH 2026) - "Background Chronic paediatric Immune Thrombocytopenia (ITP) requires second-line therapy, with oral Thrombopoietin Receptor Agonists (TPO-RAs)—Eltrombopag (ELT) and Avatrombopag (AVA)—being key options. The removal of dietary restrictions and monitoring requirements makes AVA the preferred TPO-RA in paediatric ITP, particularly for patients with poor adherence risk or concomitant hepatopathy, pending confirmation via a head-to-head non-inferiority study. Table or Figure Upload (1) Table 1 Page 2 Table or Figure Upload (2) Any DOI*10.1016/j.rpth.2026.104443"

Adherence • Clinical • Cardiovascular • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

EMPATHI Study: Real-World Evidence on Adult ITP Management, Treatment Response, and Patient Burden in Spain (ISTH 2026) - "TPO ‑ RAs remain the preferred option in second ‑ line treatment, while rituximab plays a residual role. The most frequently used average weekly doses are: eltrombopag 350mg (72%), avatrombopag 140mg (72%), fostamatinib 1400mg (52%), and romiplostim 3-4 μg/kg (40% each)...Current Treatment Patterns by Line of Therapy Page 2 Table or Figure Upload (2) Figure 1. Response rate of ITP patients per product and line according to the GEPTI 2023 guidelines DOI*10.1016/j.rpth.2026.104431"

Clinical • HEOR • Real-world • Real-world evidence • Cardiovascular • Dyslipidemia • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Metabolic Disorders • Pulmonary Arterial Hypertension • Thrombocytopenia • Thrombocytopenic Purpura

CHARACTERIZATION OF PATIENTS WITH IMMUNE THROMBOCYTOPENIA IN SPAIN. PRELIMINARY RESULTS FROM RESTI: SPANISH REGISTRY OF PRIMARY IMMUNE THROMBOCYTOPENIA (ISTH 2026) - "Corticosteroids were the most frequently used first-line therapy (95%), with prednisone (67.3%) and dexamethasone (39.7%) being the most commonly prescribed agents. Methylprednisolone (13.2%) and deflazacort (0.9%) were used less frequently...Second-line and subsequent therapies were widely used, particularly thrombopoietin receptor agonists, including eltrombopag (45.4%), avatrombopag (37.9%), and romiplostim (28.8%). Fostamatinib was prescribed in 12.1% of patients, and rituximab in 10%...Ultimately, RESTI aims to support harmonization of clinical practice and to serve as a reference framework for future national and international collaborative studies. Table or Figure Upload (1) Page 3 Table or Figure Upload (2) DOI*10.1016/j.rpth.2026.104420"

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

LONG-TERM OUTCOMES OF THROMBOPOIETIN RECEPTOR AGONISTS IN CHILDREN WITH IMMUNE THROMBOCYTOPENIA, WITH SPECIAL REFERENCE TO THE OFF-LABEL USE OF AVATROMBOPAG (EHA 2026) - "Among the 19 patients with cITP, two adolescents failed to respond to multiple therapies (including IVIG, corticosteroids, eltrombopag/romiplostim and rituximab) and presented with bleeding diathesis, so it was decided to try a newer TPO-RA, avatrombopag (approved for cITP treatment in adults). Summary/Conclusion In our study, TPO-RAs were effective with favorable safety profile for treating pediatric ITP demonstrating good long-term outcome. In two refractory cases, with significant impairment of quality of life and high risk of severe bleeding, the newer TPO-RA avatrombopag proved an effective and well-tolerated alternative therapeutic option."

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

Preliminary experience with oral thrombopoietin-receptor agonists, primarily hetrombopag, in pediatric immune thrombocytopenia: a pilot single-center retrospective report. (PubMed, Transl Pediatr) - "We conducted a single-center retrospective study of 18 children with ITP treated with oral TPO-RAs (hetrombopag, eltrombopag, or avatrombopag) at Qingdao Women and Children's Hospital from January 2023 to June 2024. In this preliminary, pilot-scale hetrombopag-predominant cohort, oral TPO-RA use was associated with increased platelet counts; however, the limited sample size and lack of statistically significant improvement in bleeding scores preclude definitive conclusions regarding the overall clinical utility of these agents beyond their ability to increase platelet counts. Given the retrospective design, heterogeneity of exposure, and limited sample size, the findings are hypothesis-generating and should be confirmed in prospective studies."

Journal • Retrospective data • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

A Retrospective Analysis of Low-Dose Decitabine combined with thrombopoietin receptor agonists for Refractory Severe Cancer therapy-induced thrombocytopenia: Efficacy, Safety, and Prognostic Factors (ISTH 2026) - "Methods This study retrospectively collected 96 CTIT patients receiving low-dose Decitabine (3.5 mg/m²/day for 3 consecutive days) combined with TPO-RAs (including eltrombopag, hetrombopag, avatrombopag, romiplostim, or rhTPO) from January 2019 to April 2025. Baseline Characteristics of the Enrolled Patients Page 2 Page 3 Table or Figure Upload (2) Table 2. Summary of Treatment Efficacy for the Combination Regimen DOI*10.1016/j.rpth.2026.106073"

Biomarker • Retrospective data • Leukopenia • Neutropenia • Oncology • Thrombocytopenia • Venous Thromboembolism