Nplate (romiplostim) / Amgen, Kyowa Kirin - LARVOL DELTA

CT-PLATE: Preoperative Use of Romiplostim in Thrombocytopenic Patients Undergoing Cardiac Surgery. (clinicaltrials.gov) - P2 | N=136 | Not yet recruiting | Sponsor: Nantes University Hospital

New P2 trial • Cardiovascular • Hematological Disorders

A randomized phase 2 Trial of romiplostim N01 versus recombinant human thrombopoietin for promoting platelet engraftment after allogeneic hematopoietic stem cell transplantation (ASH 2025) - P4 | "Optimal management of delayed platelet engraftment post-allo-HSCT remains clinically challenging. While prior studies have been limited to single-arm, single-center designs, this first randomized trial comparing romiplostim N01 versus rhTPO will generate pivotal evidence to optimize thrombopoietic support strategies and inform clinical guidelines."

Clinical • P2 data • Bone Marrow Transplantation • Graft versus Host Disease • Hematological Malignancies • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

A prospective, single-arm, multicenter clinical study on the efficacy and safety of romiplostim N01 in acclerating platelet engraftment after autologous hematopoietic stem cell transplantation (ASH 2025) - P4 | "As of July 15, 2025, 23 of 50 planned patients have been enrolled. Recruitment and follow-up are ongoing."

Clinical • Bone Marrow Transplantation • Hematological Malignancies • Immune Thrombocytopenic Purpura • Infectious Disease • Leukemia • Lymphoma • Multiple Myeloma • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation

Comprehensive cost analysis of 4th line + therapies for relapsed/refractory multiple myeloma in Germany: Drug, co-medication, and office-based treatment perspective (ASH 2025) - "Whilst there is no official myeloma registry in Germany, treatments we considered were reimbursable combination therapies frequently used in the 4 th line treatment of RRMM in Germany in 2023, containing: carfilzomib, daratumumab, elotuzumab, melflufen, selinexor, talquetamab and teclistamab, and newly approved therapeutic options like elranatamab, along with evidence-based recommendations regarding premedication, comedication, and mandatory prophylaxis of treatment-related adverse events, as outlined in the Summary of Product Characteristics (SmPC) and published literature... Costs for myeloma drugs and combinations show a broad variation, from 88.863€ for Elotuzumab/Revlimid/Dexamethasone (ERd), to 178.850€ for talquetamab treatment. The second lowest in terms of annual costs was melflufen with 106.839€, followed by Elotuzumab/Pomalidomide/Dexamethasone (EPd):119.301€, teclistamab: 124.626€, Selinexor/Dexamethasone (Sd): 129.976€, elranatamab: 146.706€ and..."

Cost-analysis • HEOR • Hematological Malignancies • Multiple Myeloma

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

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 real-world Study of romiplostim and short-course prednisone as first-line therapy for elderly immune thrombocytopenia (≥60 Years) (ASH 2025) - "Prednisone was administered at a low dose of 0.5 mg/kg for 1-2 weeks, and the effective dose was gradually reduced to discontinuation. Disclosures: No relevant conflicts of interest to declare."

Clinical • Real-world • Real-world evidence • Aplastic Anemia • Autoimmune Hemolytic Anemia • CNS Disorders • Diabetes • Gastrointestinal Disorder • Hematological Malignancies • Hepatitis B • Hepatology • Human Immunodeficiency Virus • Hypertension • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Leukemia • Metabolic Disorders • Multiple Myeloma • Musculoskeletal Diseases • Myelodysplastic Syndrome • Myeloproliferative Neoplasm • Orthopedics • Osteoporosis • Rheumatology • Sleep Disorder • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura

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

Real-world effectiveness of romiplostim N01 with or without TPO-RA for cancer therapy-induced thrombocytopenia in cancer patients (ASH 2025) - P2 | "These real-world results suggest romiplostim N01, with or without TPO-RA, is an effective treatment for CTIT, demonstrating high response rates and rapid platelet recovery in a mixed cancer patient population."

Clinical • Real-world • Real-world effectiveness • Real-world evidence • Esophageal Cancer • Gastric Cancer • Gastroesophageal Cancer • Gynecologic Cancers • Lung Cancer • Oncology • Pancreatic Cancer • Solid Tumor • Thrombocytopenia

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

A diagnostic pivot: Paroxysmal nocturnal hemoglobinuria mimicking refractory immune thrombocytopenia in an elderly woman with autoimmune comorbidities (ASH 2025) - "Case Description: An 82-year-old woman with a history of Sjögren's syndrome and autoimmune hypothyroidism presented with progressive fatigue, mucosal bleeding, and worsening thrombocytopenia (platelets 9 x 10⁹/L) unresponsive to corticosteroids, IVIG, and weekly romiplostim over two months...Complement inhibition with ravulizumab or eculizumab can transform the disease course by reducing hemolysis, thrombosis, and transfusion dependence. In conclusion, this case illustrates that classic hemolytic PNH may present as isolated thrombocytopenia in patients with autoimmune backgrounds. Early recognition and targeted therapy can significantly improve patient outcomes and quality of life, even in the elderly."

Clinical • Aplastic Anemia • Cardiovascular • Complement-mediated Rare Disorders • Endocrine Disorders • Fibrosis • Hematological Disorders • Hepatology • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Leukopenia • Meningococcal Infections • Myelodysplastic Syndrome • Paroxysmal Nocturnal Hemoglobinuria • Pneumococcal Infections • Rare Diseases • Sjogren's Syndrome • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • CD55 • CD59 • HP

Romiplostim N01 enhances platelet engraftment in ASCT using non-cryopreserved pbscs for plasma cell neoplasms: A single-center phase II study in China (ASH 2025) - "Other baseline characteristics were also comparable, such as Durie-Salmon stage, R-ISS stage, HCT-CI scpre, disease response prior to HSCT, Plerixafor use, single dayapheresis and Melphalan dose.All patients achieved hematologic engraftment. At a median follow-up of 10.8 months, 2-year overall survival did not differsignificantly (85.7% ± 13.2% vs 84.4% ± 8.3%, P=0.717).In patients with plasma cell neoplasms undergoing ASCT with non-cryopreserved PBSCs, RomiplostimN01 significantly accelerated platelet engraftment and reduced hospitalization costs withoutcompromising safety. These findings support its use as an effective alternative to traditionalcryopreserved PBSCs with rhTPO support."

Clinical • P2 data • Amyloidosis • Bone Marrow Transplantation • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Mucositis • Multiple Myeloma • Neutropenia • Plasmacytoma

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

Retrospective analysis of efficacy and safety of romiplostim N01 in the treatment of chemotherapy-induced thrombocytopenia (CIT) in solid tumors (ASH 2025) - "The results of this study demonstrate that subcutaneous injection of romiplostim N01 is aneffective treatment option for CIT, showing high response rates and short recovery time in mixed patientpopulations."

Retrospective data • Colorectal Cancer • Esophageal Cancer • Gastric Cancer • Genito-urinary Cancer • Hepatocellular Cancer • Lung Cancer • Oncology • Pancreatic Cancer • Solid Tumor • Thrombocytopenia

Efficacy and safety of romiplostim N01 in cancer treatment–induced thrombocytopenia: Results from a phase II off-label study in China (ASH 2025) - "Itachieved rapid platelet recovery, improved bleeding symptoms, reduced fatigue, and enhanced patient-reported quality of life, without serious safety concerns. Larger, randomized trials with longer follow-upare warranted to confirm these preliminary results and to further define its clinical role in CTITmanagement."

Clinical • P2 data • Colorectal Cancer • Hematological Malignancies • Immune Thrombocytopenic Purpura • Oncology • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

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

End-of-study results from the ICON3 pines trial, a phase 3, randomized trial of eltrombopag vs. standard first-line treatment for newly diagnosed immune thrombocytopenia in children (ASH 2025) - P3 | "Patients (pts) ages 1-<18 with primary ITP,≤3 months from diagnosis, with platelet count <30 x109/L who required pharmacologic treatment per thetreating clinician were randomized 2:1 to receive the experimental treatment, eltrombopag (epag), orinvestigator's choice of one of 3 standard first-line therapies (SOC): prednisone, IVIg, or anti-D globulin atspecified doses...The most commonly used subsequent agents were romiplostim, rituximab, and mycophenolatemofetil in pts randomized to epag, and epag and romiplostim for pts randomized to SOC.106 pts completed the full 1-year study (73 [94%] for epag; 33 [83%] for SOC).18 pts (25%) in the epag armremained on study drug at 1 year... In this population of pediatric pts with newly diagnosed ITP, 47% overall had diseaseresolution by 1 year, with no difference between treatment arms in 1-year response rates. SROT at 12months was 56% in epag and 61% in SOC. Many pts on the epag arm were able to discontinuemedication..."

Clinical • P3 data • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

A multicenter, randomized, open-label study of dexamethasone versus romiplostim plus dexamethasone as first line treatment in patients with newly diagnosed immune thrombocytopenia: Interim results of rodex study (ASH 2025) - "These interim results suggest that ROM+DEX SROT and reduces treatment failure compared to DEX asmonotherapy as first line treatment in newly diagnosed ITP. The combination regimen appears safe andbetter tolerated, potentially due to early platelet stabilization and reduced corticosteroid exposure. Finalresults will clarify long-term."

Clinical • Cardiovascular • Diabetes • Hematological Disorders • Hypertension • Immune Thrombocytopenic Purpura • Infectious Disease • Metabolic Disorders • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

Rituximab beats the odds in ITP? a 5-year national showdown against TPO-ras (ASH 2025) - "Background The two commonly used second-line strategies for Immune thrombocytopenia (ITP) include rituximab, ananti-CD20 monoclonal antibody, and thrombopoietin receptor agonists (TPO-RAs), such as eltrombopagand romiplostim. Despite a higher overall hospitalization rate, the hematologic and survival advantages ofrituximab suggest it is a favorable second-line option for many patients with ITP. Further prospectivestudies and randomized controlled trials are needed to confirm these real-world observations and informclinical guidelines"

Cardiovascular • Cerebral Hemorrhage • CNS Disorders • Gastroenterology • Hematological Disorders • Immune Thrombocytopenic Purpura • Infectious Disease • Pulmonary Embolism • Respiratory Diseases • Septic Shock • Thrombocytopenia • Thrombocytopenic Purpura

Rapid decrease in age-adjusted mortality rates associated with ITP following eltrombopag aand romiplostin approvals, but not in TMA following eculizumab approval, 1999-2023 (ASH 2025) - "Introduction: Idiopathic Thrombocytopenic Purpura (ITP), Thrombotic Microangiopathy (TMA) andDisseminated Intravascular Coagulation (DIC) are hematologic disorders characterized by disruption incoagulation pathways. We found a consistent decrease in AAMR for DIC, TMA and ITP (31.5% for DIC through 2007,37.7% for TMA through 2007, 16.7% for ITP through 2008). Interestingly, the decrease in AAMR for TMAand ITP correlated with the approvals for Eculizumab (2007, then 2011 specifically for cm-HUS) andEltrombopag and Romiplostim (2008 for ITP). Following these approvals, there was a rapid decrease inthe AAMR for ITP among all groups (73% from 2007-2023), the AAMR from TMA continued its downwardtrajectory at a similar pre-Eculizumab rate and there was only a slight decrease in the AAMR for DIC."

Atypical Hemolytic Uremic Syndrome • Cardiovascular • Complement-mediated Rare Disorders • Hypertension • Immune Thrombocytopenic Purpura • Infectious Disease • Nephrology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases • Septic Shock • Thrombocytopenic Purpura

Efficacy and safety of romiplostim N01 for second-line treatment of pediatric immune thrombocytopenia: A multicenter prospective observational study (ASH 2025) - P4 | "Romiplostim N01 showed rapid efficacy in pediatric ITP, providing early and effectivebleeding control with a well-tolerated safety. By facilitating rapid attainment of safer platelet thresholds,romiplostim N01 may mitigate bleeding risks during physical activities and improve overall quality of life.These findings support romiplostim N01 as a viable second-line option for children with refractory ITP.Further larger-scale studies with longer follow-up periods are needed to confirm long-term outcomesand optimize dosing strategies."

Clinical • Observational data • Hematological Disorders • Hematological Malignancies • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Myelodysplastic Syndrome • Pediatrics • Respiratory Diseases • Thrombocytopenia • Thrombocytopenic Purpura

Long-term remissions in refractory ITP patients by daratumumab (ASH 2025) - "All patients were previously treated with TPO agonists and rituximab and had at least four prior ITPtherapy lines with a median disease duration of six years (range: 1-35 years)...It should be noted that the time until response can take up to 10 weeks and therefore therapyshould not be discontinued too early if an adequate increase in platelet count has not yet been reached.Another important observation is that in one relapsed case after initial response to daratumumab, wewere able to achieve a second remission with a combination therapy of daratumumab and romiplostim,which indicates that daratumumab re-exposure is a considerable option.In recent years, another CD38 antibody (CM313, Lumrotatug) has been developed for ITP, which alsoshowed an excellent response with an overall response rate of 95% in a phase 2 study for patients withrefractory/chronic ITP (Chen et al., NEJM, 2024)...Looking ahead, we are also exploring the possibility of combining daratumumab with..."

Clinical • Hematological Disorders • Hematological Malignancies • Immune Thrombocytopenic Purpura • Multiple Myeloma • Thrombocytopenic Purpura • SYK

Complete donor chimerism following deceased-donor liver transplant in an adolescent with hepatitis-associated severe aplastic anemia (ASH 2025) - "Filgrastim was initiated on POD19, with her absoluteneutrophil count (ANC) rising from 60/µL to > 2,000/µL within 5 d, and discontinued on POD40...Romiplostim was held for periods of 2–4 mo (e.g., 20–24 mo pLT); however, theplt cts progressively dropped to the 140,000s and responded to intermittent low-dose administration (3mcg/kg)...Although this may reflectthe incidence of HA-SAA in children versus adults, other age-related factors may contribute, e.g., numberof donor-derived HSCs transferred and size of the recipient. Additional factors may include conditionswith extramedullary hematopoiesis or increased circulating CD34+ cells at time of liver donation."

Anemia • Aplastic Anemia • Dermatitis • Dermatology • Graft versus Host Disease • Hepatology • Immunology • Inflammation • Liver Failure • Thrombocytopenia • Transplantation • CD34