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

U.S. mortality trends in acquired aplastic anemia, 1999–2021: A joinpoint analysis using CDC wonder data (ASH 2025) - "Survival has improved dramatically since immunosuppressive therapy (IST) and allogeneic hematopoietic stem cell transplantation (HSCT) became standard, and after eltrombopag was added to therapy...This coincides with widespread adoption of horse antithymocyte globulin plus cyclosporine regimens, leading to reported 70–80 % hematologic response rates and improved survival...The study underscores the translational impact of clinical innovations on population-level outcomes. Continued surveillance is crucial as newer agents, biomarkers, and transplantation strategies emerge."

Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome

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

Golidocitinib, a selective JAK1 kinase inhibitor, in treatment of late thrombocytopenia after CAR-T cell therapy: Case report (ASH 2025) - P | "The patient was subsequently treated with romiplostim (250 μg, once weekly) for 2 weeks, but this did not lead to a significant improvement in platelet counts...Eltrombopag (2.5 mg orally, once daily) was administered for 2 weeks to manage thrombocytopenia...Golidocitinib, a highly selective JAK1 inhibitor, has shown promising activity in treating this adverse event. However, more clinical cases and further studies are needed to confirm this hypothesis."

CAR T-Cell Therapy • Case report • Clinical • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Epstein-Barr Virus Infections • Follicular Lymphoma • Gene Therapies • Hematological Disorders • Hematological Malignancies • Lymphoma • Movement Disorders • Non-Hodgkin’s Lymphoma • T Cell Non-Hodgkin Lymphoma • Thrombocytopenia • GZMK • JAK1

Efficacy of combination immunotherapy of eltrombopag, ATG and cyclosporine in aplastic anaemia (ASH 2025) - "Triple immunotherapy with Eltrombopag, ATG, and Cyclosporine shows a promising and well-tolerated first-line treatment for severe AA as it has faster, deeper, and more sustained hematologic responses with minimal side effects. These findings support the incorporation of Eltrombopag into frontline management for patients with AA. Large scale clinical trials are required to assess durability of response and late clonal risks."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders

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

Optimizing PNH treatment with the complement inhibitor pegcetacoplan: A case report (ASH 2025) - "The current treatment landscape includes 6 approved complement cascade inhibitors: 3 C5 inhibitors (eculizumab, ravulizumab, crovalimab), 1 C3/C3b inhibitor (pegcetacoplan), 1 factor B inhibitor (iptacopan), and 1 factor D inhibitor used as add-on treatment (danicopan)...Concomitant medications included apixaban, penicillin, and folic acid. In November 2020, her platelets count declined, and a bone marrow evaluation was diagnostic for moderate aplastic anemia (55-65% cellularity for age) and she was started on eltrombopag and cyclosporin. Despite ravulizumab and eltrombopag treatments, the patient developed significant anemia related to extravascular hemolysis (hemoglobin, 5.7 g/dL; LDH, 495 U/L; C5, 26.1 mg/dL [high]; complement hemolytic activity 50 [CH50], 7 U/mL [low])...After receiving both pegcetacoplan and iptacopan for 1 week and rivaroxaban 10 mg once daily for 48 hours, pegcetacoplan treatment ended on May 16, 2024... For this patient with PNH, the..."

Case report • Clinical • Anorexia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Infectious Disease • Meningococcal Infections • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

Initiating Eltrombopag (EPAG) Early for aplastic Anemia (AA) may Lead to greater clinical benefits: A systematic review and meta-analysis (ASH 2025) - "Concurrent therapy of EPAG should be performed and extending EPAG treatment beyond 12 months may not yield significantly enhanced benefits. Systematic Review Registration https://www.crd.york.ac.uk/prospero/, identifier CRD42024604778 KEY WORDS Aplastic anemia (AA), Bone marrow failure (BMF), Eltrombopag(EPAG), Immunosuppressive therapy (IST), Thrombopoietin receptor agonists (TPO-RA)"

Retrospective data • Review • Anemia • Aplastic Anemia • Hematological Disorders

Qualitative assessment of predictors of response to immunosuppressive therapy in aplastic anemia: A systematic literature review (ASH 2025) - "Recommended treatments include hematopoietic stem cell transplantation or immunosuppressive therapy (IST) with horse antithymocyte globulin and cyclosporine (double therapy) ± eltrombopag (triple therapy). This SLR combines all findings to date and adds genetic and molecular predictors to general AA knowledge. Further, more in-depth analyses are planned to elucidate details regarding the identified predictors, drill down on conflicting findings such as those related to PNH clones and explore additional predictors."

Review • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Hematological Disorders • B3GAT1 • CD8 • IFNG • TGFB1

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

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

Long-term outcomes of intensive immunosuppressive therapy combined with eltrombopag in patients with severe aplastic anemia (ASH 2025) - "EPAG could promote the recovery of ANC and BPC. The risk of relapse increases with patient age. EPAG does not increase the risk of progression to myeloid neoplasms in SAA patients treated by IST."

Clinical • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • ASXL1 • BCOR • BCORL1 • DNMT3A • KRAS • RUNX1 • TET2 • TP53

Primary results from VAYHIT2, a randomized, double-blind, phase 3 trial of ianalumab plus eltrombopag versus placebo plus eltrombopag in patients with primary immune thrombocytopenia (ITP) who failed first-line corticosteroid treatment (ASH 2025) - P3 | "Ianalumab in combination with eltrombopag prolonged TTF, improved SR6, reduced fatigue, facilitated tapering off eltrombopag, and delayed need for subsequent therapy in pts with primary ITP previously treated with corticosteroids. Ianalumab was well tolerated, with no observed increase in infection risk relative to placebo. Ianalumab may be disease-modifying when used early in the course of ITP."

Clinical • Late-breaking abstract • P3 data • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Neutropenia • Thrombocytopenia • Thrombocytopenic Purpura

Eltrombopag plus G-CSF for peripheral blood stem cell mobilization: Final analysis of a single arm, Phase 2 trial (ASH 2025) - "However, 9% of patients in the plerixafor era still could not harvestadequate peripheral blood stem cells (PBSC) for auto-HSCT1. Interestingly, ahigher peak eltrombopag serum concentration on ESHAP D8 correlated with a higher quantity ofharvested CD34+ cells (Spearman's rank correlation, rho = 0.41, p = 0.04119), which is compatible with theproposed mechanism of action of this intervention.ConclusionsAdding eltrombopag to stem cell mobilization for autologous PBSC harvest is safe and promising inimproving PBSC harvest efficiency. The efficacy and long-term safety warrant further exploration withconfirmatory studies."

P2 data • Bone Marrow Transplantation • Cardiovascular • Diffuse Large B Cell Lymphoma • Hematological Disorders • Hematological Malignancies • Lymphoma • Venous Thromboembolism • CD34

Outcomes of omidubicel-expanded umbilical cord blood transplantation in patients with severe aplastic anemia (ASH 2025) - P1/2 | "Theconditioning regimen included horse anti-thymocyte globulin (ATG 40 mg/kg, D-11 to -8),cyclophosphamide (60 mg/kg, D-7 and -6), fludarabine (25 mg/m2, D-5 to -1) and 2 Gy total bodyirradiation (TBI) on D-1. Graft versus host disease (GVHD) prophylaxis consisted oftacrolimus/mycophenolate mofetil (MMF)...ResultsFrom August 2017 to June 2025, 18 SAA patients (3 in Cohort 1 and 15 in Cohort 2) who failedATG/cyclosporine/eltrombopag were transplanted...Despite high-risk features, patients experienced extremely rapidand sustained engraftment, early immune recovery, low rates of GVHD, and encouraging survivaloutcomes. These promising interim results warrant further investigation."

Clinical • Acute Graft versus Host Disease • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Graft versus Host Disease • Hematological Disorders • Immunology • Infectious Disease • Neutropenia • Transplantation • CD34

Real-world data on cardiovascular disease incidence in adults with acquired immune aplastic anemia. (ASH 2025) - "Patients had a median of 1 CVD risk factor (former/current smoker, BMI ≥30, hypertension,chronic kidney disease, hyperlipidemia, diabetes, hypo/hyperthyroidism) pre-AA.Treatment exposure included 81% receiving horse (n=60) or rabbit (n=2) anti-thymocyte globulin (ATG),83% cyclosporine (CSA), 58% eltrombopag (Epag), and 25% hematopoietic stem cell transplant (HSCT).Median times from AA to first ATG, CSA, and Epag were 6, 4, and 11 weeks, respectively. Arrhythmias appeared temporally linked to ATG and CSA exposure. The impactof CH, CE, and individual somatic mutations on IS, CAD, and PAD incidence remains unclear and warrantsfurther studies in larger cohorts."

Clinical • Real-world • Real-world evidence • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Cardiovascular • Chronic Kidney Disease • Complement-mediated Rare Disorders • Congestive Heart Failure • Coronary Artery Disease • Diabetes • Dyslipidemia • Endocrine Disorders • Heart Failure • Hematological Disorders • Hematological Malignancies • Hypertension • Ischemic stroke • Metabolic Disorders • Myelodysplastic Syndrome • Myocardial Infarction • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Peripheral Arterial Disease • Rare Diseases • Renal Disease • BCOR • BCORL1 • CHEK2 • U2AF1

Real-world outcomes of eltrombopag-containing regimens as frontline treatment for aplastic anemia: A multi-center retrospective study (ASH 2025) - "Treatment regimens included EPAG(N=8), EPAG+cyclosporin A (CsA) (N=58) and EPAG+CsA+antithymocyte globulin (ATG) (N=52).For MAA patients, most were treated with EPAG+CsA (N=14). Early treatment initiation might benefitAA patients. These findings highlight the need for prospective trials to optimise EPAG-based strategies.Finally, prospective comparison with frontline allogeneic HSCT is also warranted."

Real-world • Real-world evidence • Retrospective data • Anemia • Aplastic Anemia • Bone Marrow Transplantation • Complement-mediated Rare Disorders • Hematological Disorders • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

The CSMD1 gene is involved in hematopoietic stem cell HSC pruning in patients with acquired immune-mediated aplastic anemia (ASH 2025) - P3 | "Here we present a deep analysis of CSMD1 mutations in IAA patientsenrolled in the RACE trial, looking for the frequency of mutations, its association with clinical variablesand other somatic mutations, the dynamics of the mutated clones across the 2-year study period and ain silico structural analysis predicting functional consequences.MethodsThe RACE trial enrolled 197 IAA patients randomized to receive standard IST (anti-thymocyte globulin[ATG] and cyclosporine A) with or without eltrombopag. Although CSMD1is a known tumor suppressor gene, in the context of IAA clones carrying specific CSMD1 mutation mighthave a transient survival advantage in the context of hematopoietic toxicity during ATG treatment. Thus,CSMD1-mutated HSC/HCP seem to be an example of pruning selection, where some intrinsic advantagetransiently emerging in specific micro-environmental setting may eventually become evident at the timeof hematological recovery."

Clinical • Anemia • Aplastic Anemia • CSMD1 • PHF6 • PPM1D • PTPN11 • RAD21 • STAG2

Luspatercept combined with immunosuppressive therapy reduces inflammation in aplastic anemia by inhibiting monocyte pyroptosis (ASH 2025) - "CD8+ T cells were further co-cultured with monocytes to assess their functional activity andcytokine secretion profile. The triple therapy (luspatercept + cyclosporine + eltrombopag) significantly restoredhematopoietic function in patients with SAA. This study demonstrates that luspatercept achieves multi-target immunomodulation inpatients with SAA by inhibiting the NF-κB/NLRP3 crosstalk in monocytes. This action mitigatesinflammation and pyroptosis, disrupting the pathogenic cycle and promoting hematopoietic recovery."

Anemia • Aplastic Anemia • Hematological Disorders • Immunology • Inflammation • CD14 • CD8 • GLI2 • NLRP3

Clinico-pathological profile and treatment patterns of immune mediated aplastic anemia in resource constrained settings – an analysis from the aplastic anemia registry of India (ASH 2025) - "About 15% of these patients were on combination therapy – Cyclosporine with Danazol orCyclosporine with Eltrombopag. Majority of patients [>65%] in India present to the hospital with Severe or Very Severeaplastic anemia. Androgens and Cyclosporine continue to remain the mainstay of treatment thoughincreasingly TPO agonists are being used in the relapsed refractory setting. Strategies to improve accessto curative treatment options such as ATG and BMT need to be explored."

Anemia • Aplastic Anemia • Gynecology • Hematological Disorders • Hepatology

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

Daratumumab can lead to long-lasting remissions in patients with refractory immune thrombocytopenia but with a high incidence of severe infections (ASH 2025) - "In addition, 2 patients hadantibodies against GPIIb-IIIa (acquired Glanzmann syndrome, n=1) and GPVI (n=1) responsible for chronicbleeding symptoms.Median ITP duration was 78 months [range 4-594], and patients had previously received a mediannumber of 8 [range, 3-12] treatment lines for ITP, including corticosteroids (100%), rituximab (100%),intravenous immunoglobulin (95%), thrombopoietin receptor agonists (95%; including eltrombopag[90%] and romiplostim [86%]), mycophenolate mofetil (81%), splenectomy (71%), fostamatinib (48%), andone or more other immunosuppressive drug (43%). However, thiscame at the cost of a high rate of severe infections in this particular group of heavily treated, frequentlysplenectomized, immunocompromised and fragile patients. Careful assessment of benefit/risk balance istherefore warranted before daratumumab administration."

Clinical • Congestive Heart Failure • Genetic Disorders • Heart Failure • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Inflammatory Arthritis • Multiple Myeloma • Neutropenia • Otorhinolaryngology • Pneumonia • Respiratory Diseases • Rheumatoid Arthritis • Rheumatology • Septic Shock • Thrombocytopenia • Thrombocytopenic Purpura

Adverse events reported with eltrombopag and romiplostim: Faers database (ASH 2025) - "This FAERS analysis confirms known safety profiles of both thrombopoietin receptoragonists. Eltrombopag demonstrates a clear hepatic safety signal requiring regular liver functionmonitoring, while romiplostim shows expected injection site reactions. Both drugs exhibit similar rates ofthrombotic complications."

Adverse events • Dermatology • Hematological Disorders • Hepatology • Liver Failure

Real-world effectiveness and safety of fostamatinib in difficult-to-treat patients: Results of a three-year registry in France. (ASH 2025) - "Seventy-height percent ofpatients had been exposed to eltrombopag, 74.4% to romiplostim, 86.0% to rituximab, 48.8% tomycopenolate or azathioprine, and 28.0% were splenectomized. Fostamatinib was used in previously very heavily treated patients, with response rates of44.0% at M3 and of 20.0% at M24. Combination therapy with TPO-RA is an option to consider in thispopulation. No new safety signal was observed."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Hematological Malignancies • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Lymphoma • Musculoskeletal Diseases • Myocardial Infarction • Neutropenia • Pulmonary Arterial Hypertension • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Adding eltrombopag to immunosuppressive treatment with cyclosporine as front-line therapy for moderate aplastic anemia improves trilineage hematologic response: One-year analysis of the randomised, placebo-controlled, double-blind Phase III trial eltrombopag for moderate aplastic anemia (EMAA Trial) (ASH 2025) - P2/3 | "Introduction:Eltrombopag (EPAG) combined with horse antithymocyte globulin and cyclosporine A (CSA) when givenas first-line treatment of patients (pts) with severe aplastic anemia (SAA) improves rate, rapidity, andstrength of hematologic response. Adding EPAG to CSA treatment for first-line treatment of MAA significantly improves trilineagehematologic response at wk 24. Addition of EPAG in pts without CR after 24 wks of single-agent CSAtreatment also improves the trilineage hematologic response rate. The cumulative incidence of trilineageresponse was significantly better in pts randomized into the EPAG arm."

Clinical • P3 data • Anemia • Aplastic Anemia • Cardiovascular • Hematological Disorders • Pulmonary Embolism • Respiratory Diseases