Promacta (eltrombopag) / GSK, Novartis, Royalty - LARVOL DELTA

A Paradoxical Case of Bleeding and Clots: The Hidden Thrombotic Threat in Immune Thrombocytopenic Purpura. (PubMed, Clin Case Rep) - "We describe a young woman with established ITP who developed deep vein thrombosis and pulmonary embolism shortly after commencing norethisterone for menstrual regulation while also using eltrombopag, creating a combination of therapeutic and physiological prothrombotic risks. This case underscores the complex interplay between ITP, platelet-stimulating agents, and hormonal therapy, illustrating how thrombosis may occur even in the context of thrombocytopenia. It highlights the importance of maintaining a high index of suspicion for thromboembolic complications in ITP patients receiving TPO-RA therapy, particularly when additional risk factors coexist, and reinforces the need for careful risk-benefit assessment when initiating or continuing agents that may contribute to thrombogenesis."

Journal • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Pain • Pulmonary Disease • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Lenalidomide and Eltrombopag for Treatment of Low or Intermediate Risk Myelodysplastic Syndrome: Result of a Phase 2 Clinical Trial. (PubMed, Clin Cancer Res) - P2 | "ELT and LEN are well tolerated and effective in achieving hematological improvement in patients with low/intermediate-risk MDS."

Journal • P2 data • Fibrosis • Hematological Malignancies • Immunology • Myelodysplastic Syndrome • Thrombocytopenia

Osimertinib Induced Bone Marrow Aplasia Case Report (IASLC-WCLC 2026) - "Initially, she began treatment with carboplatin and pemetrexed while awaiting next-generation sequencing results...She was treated empirically with cefuroxime due to concurrent respiratory illness within her household...Therapy was switched to erlotinib (Tarceva), and supportive agents including eltrombopag and darbepoetin were initiated...Despite these interventions, her cytopenias persisted, and her blood counts did not recover. Conclusions : This case highlights that osimertinib induced bone marrow aplasia is a rare adverse event but it is important to monitor patients for hematological toxicities closely while on osimertinib therapy."

Case report • Clinical • Anorexia • Aplastic Anemia • Cardiovascular • Cough • Infectious Disease • Interstitial Lung Disease • Lung Adenocarcinoma • Lung Cancer • Non Small Cell Lung Cancer • Pneumonia • Pulmonary Disease • Respiratory Diseases • Solid Tumor

Severe aplastic anemia as a rare autoimmune complication of ankylosing spondylitis: A rare case report. (PubMed, Medicine (Baltimore)) - "This case suggests a possible immunological link between AA and AS, given their shared pathways of T-cell-mediated cytotoxicity and cytokine activation. However, this single observation should be interpreted with caution and does not establish causation. It may nonetheless contribute to the emerging literature on potential pathophysiological overlaps between rheumatologic disorders and bone marrow failure syndromes."

Journal • Anemia • Ankylosing Spondylitis • Aplastic Anemia • Fatigue • Hematological Disorders • Immunology • Inflammatory Arthritis • Oncology • Rheumatology • Seronegative Spondyloarthropathies • Spondylarthritis • TNFA

Eltrombopag for chemotherapy-induced thrombocytopenia (CIT) in advanced gastrointestinal (GI) cancers: a retrospective analysis (ESMO 2026) - No abstract available

Metastases • Retrospective data • Gastrointestinal Cancer • Oncology

Response to: citation clarification regarding feline eltrombopag in the ACVIM consensus statement on the treatment of immune thrombocytopenia in dogs and cats. (PubMed, J Vet Intern Med) - No abstract available

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

Temozolomide-induced aplastic anemia - retrospective analysis and management (EANO 2026) - "Background: Concurrent chemoradiotherapy (cCRT) with temozolomide (TMZ) and its adjuvant administration has been the standard of care for high grade gliomas (HGGs) including glioblastoma for 20 years. GM-CSF daily application led to partial or complete recovery in all patients. The most limiting adverse event for patients with TIAA seems to be thrombocytopenia. Eltrombopag lead to limited efficacy with ongoing need of platelets substitution."

Retrospective data • Astrocytoma • Brain Cancer • Glioblastoma • Glioma • High Grade Glioma • Oligodendroglioma • Oncology • Solid Tumor • CHEK2 • CSF2 • MGMT

Longitudinal Divergence Between CONUT and GNRI Following Decreased Food Intake Temporally Associated With Trimethoprim-Sulfamethoxazole Administration in an Older Patient With Immune Thrombocytopenia and Dementia: A Case Report. (PubMed, Cureus) - "After treatment with corticosteroids, intravenous immunoglobulin, and eltrombopag, his platelet count recovered. This case indicates that, in older patients with dementia, improvement in laboratory-based nutritional indices does not necessarily reflect recovery from chronic malnutrition. Longitudinal evaluation combining the CONUT score and the GNRI may provide complementary information in complex clinical settings."

Journal • Alzheimer's Disease • Anorexia • CNS Disorders • Dementia • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Inflammation • Pneumonia • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

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

Observational Study on Eltrombopag in Hypocellular Myelodysplastic Neoplasms (clinicaltrials.gov) - P=N/A | N=100 | Active, not recruiting | Sponsor: Fondazione Italiana Sindromi Mielodisplastiche-ETS

New trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology

Efficacy and Safety of rhTPO Combined with TPO-RA in the Prophylaxis of Chemotherapy-Induced Thrombocytopenia (CTIT) in High-Risk Patients with Solid Tumors (ChiCTR) - P=N/A | N=90 | Not yet recruiting | Sponsor: Ningbo Medical Centre Lihuili Hospital; Ningbo Medical Centre Lihuili Hospital

New trial • Hematological Disorders • Hepatocellular Cancer • Oncology • Solid Tumor • Thrombocytopenia

Efficacy and Safety of Eltrombopag for the Prevention of Thrombocytopenia Following the First Consolidation Chemotherapy in Patients with Acute Myeloid Leukemia: A Multicenter, Randomized, Placebo-Controlled Clinical Trial (ChiCTR) - P3 | N=256 | Not yet recruiting | Sponsor: Peking University Shenzhen Hospital; Peking University Shenzhen Hospital

New P3 trial • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Thrombocytopenia

Clinical study on the efficacy and safety of eltrombopag olamine tablets in secondary prevention of chemotherapy-induced thrombocytopenia in rare tumors (ChiCTR) - P2 | N=44 | Not yet recruiting | Sponsor: The Afliated Cancer Hospital of Shandong First Medical University; The Afliated Cancer Hospital of Shandong First Medical University

New P2 trial • Hematological Disorders • Oncology • Thrombocytopenia

BILATERAL PULMONARY INFARCTION AS A COMPLICATION OF ELTROMBOPAG IN AN OLDER PATIENT WITH CHRONIC ITP AND NORMAL PLATELET COUNT (CHEST 2026) - No abstract available

Clinical • Cardiovascular

Successful renal transplant in a MYH9 gene related disorder with planned preoperative care (TTS 2026) - "He received Eltrombopag 50 mg once a day for 6 days prior to surgery with which his platelet counts improved.3 He received methylprednisolone and Basiliximab induction before renal transplant on 5 August 2025 along with 1 single donor platelet (SDP) transfusion of 200 mL preoperatively.4 Transplant surgery was initiated with recipient graft bed preparation well ahead of donor nephrectomy to assess the bleeding risk. Renal transplantation in MYH9 gene related disorders pose challenges requiring detailed evaluation and careful planning. A bleeding risk can be assessed well ahead by preparing the renal bed for graft before graft nephrectomy and an addition of Eltrombopag and SDP before surgery can be helpful in mitigating the risk of bleeding. Reference: 1."

Cardiovascular • Cataract • Gastrointestinal Disorder • Genetic Disorders • Glomerulonephritis • Hematological Disorders • Immune Thrombocytopenic Purpura • Nephrology • Ophthalmology • Otorhinolaryngology • Retinal Disorders • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation • MUTYH • MYH9 • PKHD1

Citation clarification regarding feline eltrombopag in the "ACVIM consensus statement on the treatment of immune thrombocytopenia in dogs and cats". (PubMed, J Vet Intern Med) - No abstract available

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

Mechanism of PLA2G15 in Bone Invasion of Recurrent Skull Base Chordoma (EANO 2026) - "Nilotinib and eltrombopag were identified as potential PLA2G15 inhibitors. A PLA2G15-high lipid-associated macrophage subset drives bone invasion in recurrent chordoma. Mechanistically, PLA2G15 promotes LPC-LPA metabolic reprogramming and activates LPAR5-JAK2-STAT3 signaling, thereby enhancing macrophage osteolytic activity and tumor invasiveness. These findings identify a microenvironmental mechanism of osteolysis in recurrent chordoma and nominate PLA2G15 as a potential therapeutic target."

Chordoma • Oncology • APOE • CD8 • LPAR5

Cost-effectiveness analysis of eltrombopag, hetrombopag and romiplostim for second-line treatment of primary immune thrombocytopenia in China. (PubMed, Front Med (Lausanne)) - "Eltrombopag was likely to be the most cost-effective second-line treatment option for ITP compared with hetrombopag and romiplostim. Our findings provide economic evidence to support decision-making regarding the use of TPO-RAs in China."

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

Efficacy and safety of eltrombopag in the treatment of cancer therapy-induced thrombocytopenia: a large-scale, multicenter, retrospective study in China. (PubMed, Blood Sci) - No abstract available

Journal • Retrospective data • Hematological Disorders • Oncology • Thrombocytopenia

Eltrombopag for Low-Risk Myelodysplastic Syndromes With Thrombocytopenia: Interim Results of a Phase-II, Randomized, Placebo-Controlled Clinical Trial (EQOL-MDS). (PubMed, J Clin Oncol) - P2 | "Eltrombopag was effective and relatively safe in low-risk MDS with severe thrombocytopenia. This trial is registered with ClinicalTrials.gov identifier: NCT02912208 and EU Clinical Trials Register: EudraCT No. 2010-022890-33."

Clinical • Journal • P2 data • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Thrombocytopenia

Structure prediction and drug screening targeting monkeypox virus polymerase and surface proteins. (PubMed, J Comput Aided Mol Des) - "The global outbreak and ongoing spread of the monkeypox virus (MPXV) have highlighted the urgent need for effective antiviral therapeutics...Three compounds, including cepharanthine, eltrombopag, and simeprevir, exhibited measurable A35R‑associated binding signals with equilibrium dissociation constants (KD) in the micromolar range...However, no antiviral activity has been demonstrated for these compounds; therefore, the three hit compounds warrant further optimization and biological evaluation. Our integrated approach provides a framework for rapid drug screening against emerging viral threats."

Journal • Preclinical • Infectious Disease

Efficacy and safety comparison of hetrombopag and eltrombopag in children with cancer therapy-induced thrombocytopenia: a single-center retrospective study. (PubMed, Front Med (Lausanne)) - "In pediatric CTIT patients, eltrombopag was linked to improved platelet response and less transfusion dependence, accompanied by a tolerable safety profile. As the first study focusing on eltrombopag for pediatric CTIT, these findings provide critical evidence supporting its clinical application as a promising therapeutic option for this understudied population."

Journal • Retrospective data • Hematological Disorders • Leukopenia • Oncology • Pediatrics • Thrombocytopenia • Thrombocytosis

Leukemic Transformation and Clinical Outcomes in Thrombocytopenic Myelodysplastic Syndrome Patients Treated With Thrombopoietin Receptor Agonists: A Real-World TriNetX Cohort Study (SOHO 2026) - "The exposed cohort had MDS treated with eltrombopag/romiplostim; the comparator cohort had MDS without exposure to either TPO-RA. In this real-world propensity-matched analysis, TPORA exposure was not associated with an increased risk of AML transformation at 1 year. However, TPO-RA–treated patients had higher all-cause mortality, thromboembolic events, and major bleeding. These findings are potentially limited by unknown residual confounding factors."

Clinical • Clinical data • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

A Phase 2 Open-Label Study to Assess the Tolerability of Ianalumab (VAY736) With Investigator's-Choice Thrombopoietin Receptor Agonist in Patients With Primary Immune Thrombocytopenia: VAY2EXPLORE Study Design (SOHO 2026) - P2, P3 | " The phase 2 multicenter, open-label study (VAY2EXPLORE; NCT07421167) will enroll approximately 149 patients with primary ITP, with 25–60 patients each on IC eltrombopag, avatrombopag, or romiplostim per approved prescribing information for ≤6 months. The VAY2EXPLORE study will expand upon previous findings and assess the tolerability and benefit of ianalumab with IC TPO-RAs in patients with ITP. Funding: This study was funded by Novartis Pharmaceuticals Corporation."

Clinical • P2 data • Oncology