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

Thrombopoietin receptor agonists in immune thrombocytopenia: a comparative review of mechanisms, efficacy, and the future of personalized management. (PubMed, Expert Opin Pharmacother) - "The three approved agents - romiplostim, eltrombopag, and avatrombopag - all stimulate the c-Mpl receptor but differ in their molecular interactions, pharmacokinetics, and potential immunomodulatory properties...TPO-RAs now represent a cornerstone of chronic ITP management and are expected to see broader use as growing evidence supports earlier and more individualized treatment strategies. Future progress will rely on personalized therapeutic strategies, informed monitoring, and AI-driven predictive tools that together may enhance remission durability and long-term clinical outcomes."

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

Real-World Safety and Efficacy of Avatrombopag in Adults with Immune Thrombocytopenia: A Systematic Review and Meta-Analysis. (PubMed, Eur J Haematol) - "In real-world practice, avatrombopag demonstrated high platelet response rates and a low pooled incidence of thrombotic events. These findings add real-world evidence on avatrombopag outcomes in adult immune thrombocytopenia."

Journal • Real-world evidence • Retrospective data • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • 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

REDUCTION IN CONCOMITANT THERAPY USE WITH RILZABRUTINIB AND SUSTAINED RESPONSE IN ADULTS WITH PERSISTENT/CHRONIC IMMUNE THROMBOCYTOPENIA (ITP) IN THE PHASE 3 LUNA3 LONG-TERM EXTENSION (LTE) PERIOD (THSNA 2026) - P3 | "Ten LTE patients discontinued corticosteroids (switched to rilzabrutinib monotherapy), 2 reduced corticosteroids ≥50% from DB baseline, and 6 reduced corticosteroid dose to <5 mg (prednisone qd)...Of 11 patients on concomitant TPO-RA or corticosteroid/TPO-RA at DB baseline, 5 discontinued TPO-RA before entering LTE (romiplostim: n=1; avatrombopag: n=2; eltrombopag: n=2) and 1 during the LTE (eltrombopag)... Rilzabrutinib continued to demonstrate favorable safety, sustained platelet responses and improved ITP-related symptoms during the LTE period. Most LTE patients receiving concomitant corticosteroids and/or TPO-RA were able to discontinue/reduce use while maintaining platelet counts. These results further underscore the disease-modifying potential of multi-immune modulation with rilzabrutinib in patients with ITP."

Clinical • P3 data • Hematological Disorders • Immune Modulation • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura

UMBILICAL-CORD MESENCHYMAL STROMAL CELLS PLUS AVATROMBOPAG FOR PEDIATRIC POOR GRAFT FUNCTION/PROLONGED ISOLATED THROMBOCYTOPENIA AFTER ALLOGENEIC HEMATOPOIETIC STEM-CELL TRANSPLANTATION: A SINGLE-CENTER EXPERIENCE (EBMT 2026) - "Umbilical-cord MSC combined with avatrombopag is a well-tolerated, rapidly active front-line regimen for pediatric PGF/PIT after allo-HSCT, with a CR rate exceeding 80%. Prospective multicentre studies are warranted to confirm these findings."

Clinical • Stroma • Acute Lymphocytic Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Pediatrics • Thrombocytopenia • Transplantation

MINI-ATG/TBI CONDITIONING REGIMEN FACILITATES CURE OF ACQUIRED APLASTIC ANEMIA IN CHILDREN VIA UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (EBMT 2026) - P4 | "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)...However, CMV viremia incidence differed significantly (6.25% vs. 66.67%, P=0.001), attributed to the widespread use of letermovir for CMV prophylaxis after 2024... These data support the mini-ATG/TBI regimen as an effective conditioning approach for UCBT in acquired aplastic anemia, demonstrating particular utility in pediatric patients or low body weight adults."

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

Avatrombopag Reduces Platelet Transfusion Requirement in Thrombocytopenia Subsequent to Antineoplastic Therapies in Haematological Patients: The Experience of a Tertiary Centre. (PubMed, J Clin Med) - "No safety concerns were reported. AVA shows promise in safely reducing thrombocytopenia-associated transfusion needs in haematological malignancy."

Journal • Hematological Disorders • Hematological Malignancies • Oncology • Thrombocytopenia

UMBILICAL-CORD MESENCHYMAL STROMAL CELLS PLUS AVATROMBOPAG FOR PEDIATRIC POOR GRAFT FUNCTION/PROLONGED ISOLATED THROMBOCYTOPENIA AFTER ALLOGENEIC HEMATOPOIETIC STEM-CELL TRANSPLANTATION: A SINGLE-CENTER EXPERIENCE (EBMT 2026) - "Umbilical-cord MSC combined with avatrombopag is a well-tolerated, rapidly active front-line regimen for pediatric PGF/PIT after allo-HSCT, with a CR rate exceeding 80%. Prospective multicentre studies are warranted to confirm these findings."

Clinical • Stroma • Acute Lymphocytic Leukemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Pediatrics • Thrombocytopenia • Transplantation

MINI-ATG/TBI CONDITIONING REGIMEN FACILITATES CURE OF ACQUIRED APLASTIC ANEMIA IN CHILDREN VIA UNRELATED UMBILICAL CORD BLOOD TRANSPLANTATION (EBMT 2026) - P4 | "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)...However, CMV viremia incidence differed significantly (6.25% vs. 66.67%, P=0.001), attributed to the widespread use of letermovir for CMV prophylaxis after 2024... These data support the mini-ATG/TBI regimen as an effective conditioning approach for UCBT in acquired aplastic anemia, demonstrating particular utility in pediatric patients or low body weight adults."

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

Avatrombopag and Platelet Recovery in Sepsis-Associated Thrombocytopenia: A Retrospective Observational Cohort Study. (PubMed, Clin Appl Thromb Hemost) - "Subgroup analyses suggested greater platelet recovery benefit in patients with higher platelet transfusion requirements and greater illness severity.ConclusionsIn critically ill patients with SAT, avatrombopag administration was associated with improved delayed platelet recovery without increased bleeding or thrombotic risk. This hematologic benefit did not translate into improved short-term survival and was accompanied by prolonged ICU stay."

Journal • Observational data • Retrospective data • Critical care • Hematological Disorders • Infectious Disease • Septic Shock • Thrombocytopenia

Real World Long Term TPO-RA: Sustaining Remission or Time to Stop? (ICKSH 2026) - "Thrombopoietin receptor agonists (TPO -RAs), including romiplostim , eltrombopag , and avatrombopag , stimu late megakaryocyte proliferation through activation of the thrombopoietin receptor (c -MPL). N Engl J Med. 2008."

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

Thromboembolic adverse events associated with TPO-RA in ITP treatment: a pharmacovigilance analysis of the FDA Adverse Event Reporting System. (PubMed, Front Immunol) - "Avatrombopag, Eltrombopag, and Romiplostim yielded 105, 1,044, and 943 thromboembolic AE reports, respectively. Real-world pharmacovigilance evidence indicates that thromboembolic event reporting for TPO-RAs shows drug-specific patterns, with venous events predominating and a notable fraction occurring early after treatment initiation. Patients of very advanced age, those with higher body weight, and those receiving prolonged therapy appear particularly vulnerable, supporting proactive baseline risk stratification and sustained thrombosis surveillance throughout treatment."

Adverse events • Journal • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Myocardial Infarction • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Cost-effectiveness of hetrombopag, eltrombopag, and avatrombopag for chronic immune thrombocytopenia in China: a cost-utility analysis. (PubMed, Front Public Health) - "It dominates eltrombopag and offers a favorable economic profile compared with avatrombopag. These findings support the use of hetrombopag as a preferred option in resource-limited settings."

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

Integrated Assessment of Three NDSRIs Through CPCA Prioritization, In Silico Toxicity Assessment, and Stability Indicating LC-MS/MS Method for Avatrombopag Maleate. (PubMed, Biomed Chromatogr) - "The source of the NDSRI formation has also been demonstrated; finally, their selective, sensitive, and stability indicating LC-MS/MS-based analytical method has been implemented. This wholistic lifecycle approach eliminates the risk-assessment gaps and ratifies the regulatory requirements, thereby endures the product quality and patients safety."

Journal

Suspected doxycycline-induced thrombocytopenia: Two case reports and a review of the literature. (PubMed, Medicine (Baltimore)) - "These cases suggest that clinicians should be aware of this rare but serious adverse effect. Monitoring platelet counts during doxycycline therapy, particularly in complex patients - especially those receiving multiple medications or with underlying coagulopathy, may help with early detection and timely intervention."

Journal • Review • Gastrointestinal Disorder • Hematological Disorders • Thrombocytopenia

The many faces of CMML: Paraneoplastic systemic autoinflammatory disorders (SAID). A case series (DKK 2026) - "Low-dose prednisolone and hydroxyurea led to full recovery. Case 2: A 68-year-old female with concomitant polymyalgia rheumatica and idiopathic thrombocytopenic purpura initially responded to steroids; after relapse and avatrombopag failure, bone marrow showed CMML-0, two years later progressing to CMML-1 with newly detected liver fibrosis consistent with IgG4-associated autoimmune hepatitis required immunosuppression.... High suspicion index may prompt earlier bone marrow evaluation, guide immunosuppressive therapy, and improve outcomes."

Clinical • Autoimmune Hepatitis • Chronic Myeloid Leukemia • Chronic Myelomonocytic Leukemia • Dermatitis • Fibrosis • Giant Cell Arteritis • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Interstitial Lung Disease • Liver Cirrhosis • Musculoskeletal Diseases • Musculoskeletal Pain • Myelodysplastic Syndrome • Palliative care • Pulmonary Disease • Respiratory Diseases • Scleroderma • Systemic Sclerosis • Thrombocytopenic Purpura • IDH1

Avatrombopag for post-transplant thrombocytopenia following allogeneic haematopoietic stem cell transplantation: A single-centre retrospective study. (PubMed, Br J Haematol) - "Thrombopoietin receptor agonists (TPO-RAs) like eltrombopag or romiplostim are effective but have pharmacologic and practical limitations. No severe adverse events or complications were reported. Notably, these findings suggest that avatrombopag is a promising alternative for PGF, offering practical advantages over other TPO-RAs, although prospective studies are needed with a larger sample size of patients to confirm these results."

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

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 | Not yet recruiting | Sponsor: Novartis Pharmaceuticals

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

Refractory Primary Immune Thrombocytopenia With Bleeding and Thrombosis: A Case Report. (PubMed, Am J Case Rep) - "He was nonresponsive to initial prednisone treatment and had petechial gastritis...Still, the patient was refractory to other therapeutic options, including vincristine and anti-D immunoglobulins, showing only transient platelet count response while also showing cerebral hemorrhage. Hence, he was treated with splenectomy and rituximab...A course of immunosuppressant therapy with azathioprine and mycophenolate mofetil was started with no response. Alternative therapies including eltrombopag, bortezomib, and dapsone resulted in no response, and romiplostim was re-started, causing acute coronary syndrome and unstable angina pectoris. Therefore, fostamatinib was started, but then was stopped due to melena. Finally, avatrombopag was started at a personalized dose of 20 mg daily in association with dual antiplatelet therapy, and this achieved good platelet count response. CONCLUSIONS This report shows that ITP is characterized by complexity, requiring management of..."

Journal • Acute Coronary Syndrome • Cardiovascular • Cerebral Hemorrhage • Gastrointestinal Disorder • Hematological Disorders • Immune Thrombocytopenic Purpura • Myocardial Ischemia • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Successful Management of Relapsed Severe Immune Thrombocytopenia Using Avatrombopag: A Case Report. (PubMed, Case Rep Hematol) - "We report the case of a 37-year-old woman with relapsed severe ITP, unresponsive to corticosteroids, IVIG, rituximab, romiplostim, eltrombopag, vincristine, cyclosporine, and splenectomy. This case demonstrates the efficacy of avatrombopag in a patient unresponsive to multiple prior therapies, including other TPO-RAs and splenectomy. Further studies are warranted to determine optimal treatment sequencing and long-term outcomes for patients in this challenging subgroup."

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

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

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

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