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

LB03: Infusion pharmacist-driven romiplostim (NPlate®) dosing protocol to optimize efficiency in an outpatient hematology & oncology cancer center (HOPA 2026) - "From May to November 2025, a total of 370 doses of romiplostim were given, and of these, 131 (35.4%) were given per the dosing protocol. No significant reduction in time to order verification was observed between doses adjusted on and off protocol (13.3 versus 13 minutes). However, we did note a reduction in doses adjusted by the provider for patients on protocol, with 55.2% and 33.3% of doses adjusted by the provider for patients off and on protocol, respectively."

Clinical • Immune Thrombocytopenic Purpura • Oncology • Thrombocytopenia • Thrombocytopenic Purpura

Evaluation of Romiplostim Use for the Management of Thrombocytopenia After CAR T-cell Therapy (HOPA 2026) - "Results pending."

CAR T-Cell Therapy • Hematological Malignancies • Thrombocytopenia

Institutional Review of Romiplostim Dosing in Patients with Hematologic Malignancies after Treatment-Related Thrombocytopenia (Chemotherapy, Bispecific Therapy and/or Chimeric Antigen Receptor-T cells) (HOPA 2026) - "Research in progress"

Bispecific • CAR T-Cell Therapy • Clinical • Review • Hematological Malignancies • Immune Thrombocytopenic Purpura • Oncology • Solid Tumor • Thrombocytopenia • Thrombocytopenic Purpura • Thrombocytosis

Infusion Pharmacist–Driven Romiplostim Dosing Protocol to Optimize Efficiency in an Outpatient Hematology and Oncology Cancer Center (HOPA 2026) - "The implementation of a pharmacist-driven romiplostim dosing protocol improved efficiency for our cancer center because it effectively shifted the workload of dose adjustment from the provider to the pharmacist. In addition, we identified numerous patients on long-term romiplostim initiated prior to the protocol implementation, representing possible missed opportunities. Although no difference in time to order verification was noted, we expect a potential reduction in chair time as more patients are transitioned to the protocol."

Clinical • Late-breaking abstract • Bone Marrow Transplantation • Immune Thrombocytopenic Purpura • Oncology • Thrombocytopenia • Thrombocytopenic Purpura

A Comparative Analysis of the Efficacy, Safety and Mechanism of Action of Flebogamma DIF, Fostamatinib and Romiplostim in Immune Thrombocytopenia. (PubMed, Life (Basel)) - "Overall, these therapies offer distinct advantages depending on disease chronicity, patient age, comorbidities, and treatment history. This review underscores the importance of personalized treatment strategies and highlights the need for further long-term, comparative studies to guide evidence-based ITP management."

Clinical • Journal • Review • Cardiovascular • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis

FIRST REPORTED ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ALLO-HSCT) FOR RAD50 DEFICIENCY: INITIAL CLINICAL EXPERIENCE IN TWO PEDIATRIC PATIENTS (EBMT 2026) - "Within one year, bone marrow dysplasia with a del(5q) clone and blasts up to 6–7% evolved, consistent with MDS on the background of inborn error of immunity.Unrelated-donor allo-HSCT (conditioning regimen: fludarabine 150 mg/m², busulfan 4 mg/kg, ATG 5 mg/kg and rituximab 375 mg/m², with PTCy-based GVHD prophylaxis) was complicated by HHV-6 infection, severe infusion-associated rash, and subsequent graft rejection by day +60. Six months later a second haploidentical HSCT from the mother (conditioning regimen: fludarabine 150 mg/m², treosulfan 21 g/m², rituximab 375 mg/m² and alemtuzumab 0.4 mg/kg with PTCy-based GVHD prophylaxis) resulted in timely engraftment and was complicated by megakaryocytic lineage hypofunction, viral reactivation, COVID-19 infection, and mild skin GVHD.Case #2...The course was marked by transfusion dependence, recurrent infections and refractory thrombocytopenia despite romiplostim and eltrombopag therapy.At the age of..."

Clinical • Acute Kidney Injury • Aplastic Anemia • Bone Marrow Transplantation • CNS Disorders • Graft versus Host Disease • Hematological Disorders • Herpes Simplex • Immunology • Infectious Disease • Metabolic Disorders • Nephrology • Novel Coronavirus Disease • Pediatrics • Thrombocytopenia • Transplant Rejection • Transplantation • RAD50

ROMIPLOSTIM IN PEDIATRIC PATIENTS POST HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR EARLY PLATELET ENGRAFTMENT- AN OPEN LABEL RANDOMISED CONTROLLED TRIAL (EBMT 2026) - "Romiplostim was safe and well tolerated following pediatric HSCT. While it did not significantly shorten platelet engraftment time, the intervention group demonstrated higher platelet and neutrophil counts by day +28, particularly among allogeneic and malignant subgroups. These findings support the feasibility of romiplostim use post-HSCT and warrant larger multicenter trials to establish its efficacy in the pediatric population."

Clinical • Bone Marrow Transplantation • Pediatrics • Transplantation

LESS INFLAMMATION, SAME SURVIVAL: PLERIXAFOR MOBILIZATION REDUCES ENGRAFTMENT CRS IN PAEDIATRIC HSCT COMPARED TO GCSF ALONE (EBMT 2026) - "Recipient outcomes evaluated included engraftment kinetics, CRS, engraftment syndrome, acute GVHD, mortality, relapse, TATMA, secondary graft dysfunction requiring romiplostim, SOS, day +15/+30 chimerism, viral reactivation, and immune reconstitution across CD4 subsets, NK and B cells at days +30, +60 and +90. The CD34:CD3 ratio differed significantly between mobilization strategies. G-CSF–only mobilization yielded grafts richer in CD34⁺ stem cells, whereas plerixafor-mobilized grafts were more T-cell enriched. These differences in graft biology did not translate into inferior clinical outcomes in the plerixafor group.Engraftment CRS was significantly lower in the plerixafor cohort and anakinra requirement for cytokine control was numerically reduced, indicating a lower inflammatory burden despite graft compositional differences.Immune recovery, survival, and complication profiles remained equivalent between mobilization strategies.Overall, plerixafor is a safe and..."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Inflammation • Pediatrics • CD34

ROMIPLOSTIM IN PEDIATRIC PATIENTS POST HEMATOPOIETIC STEM CELL TRANSPLANTATION FOR EARLY PLATELET ENGRAFTMENT- AN OPEN LABEL RANDOMISED CONTROLLED TRIAL (EBMT 2026) - "Romiplostim was safe and well tolerated following pediatric HSCT. While it did not significantly shorten platelet engraftment time, the intervention group demonstrated higher platelet and neutrophil counts by day +28, particularly among allogeneic and malignant subgroups. These findings support the feasibility of romiplostim use post-HSCT and warrant larger multicenter trials to establish its efficacy in the pediatric population."

Clinical • Bone Marrow Transplantation • Pediatrics • Transplantation

A CASE OF RESOLVED SEVERE HEMATOLOGICAL TOXICITY AFTER ANTI-BCMA CAR-T THERAPY FOR MULTIPLE MYELOMA (EBMT 2026) - "Lymphodepleting therapy consisted of fludarabine 30 mg x m2 and cyclophosphamide 300 mg x m2 for three consecutive days.The CAR-T cell dose was 1.27 cells x 106/kg of patient body weight, the ratio of CD4+ to CD8+ cells was 1.15...Grade 3 CRS was detected on day 7, which required 2-fold administration of Tocilizumab at a dose of 8 mg/kg, vasopressor support, and respiratory support... Despite achieving deep durable responses in multiple myeloma, CAR-T therapy may be associated with severe and prolonged hematological toxicity requiring complex and long-term therapy."

CAR T-Cell Therapy • Clinical • Conjunctivitis • Cytomegalovirus Infection • Hematological Malignancies • Hemophagocytic lymphohistiocytosis • Immunology • Infectious Disease • Multiple Myeloma • Neutropenia • Ocular Infections • Ocular Inflammation • Ophthalmology • Rare Diseases • Respiratory Diseases • Thrombocytopenia • CD4

EFFICACY OF ROMIPLOSTIM ON HASTENING PLATELET COUNT RECOVERY IN POST-TRANSPLANT PATIENTS: A SINGLE-CENTER EXPERIENCE (EBMT 2026) - "Romiplostim administration (2-3 mcg/kg, D+5 to D+10) did not significantly hasten platelet engraftment in this cohort. None of the patients experienced any adverse effects from romiplostim. However, amongst patients who had a later engraftment, there was a trend toward benefit from romiplostim."

Clinical • Post-transplantation • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Hepatology • Lymphoma • Thrombocytopenia • Transplantation

FIRST REPORTED ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION (ALLO-HSCT) FOR RAD50 DEFICIENCY: INITIAL CLINICAL EXPERIENCE IN TWO PEDIATRIC PATIENTS (EBMT 2026) - "Within one year, bone marrow dysplasia with a del(5q) clone and blasts up to 6–7% evolved, consistent with MDS on the background of inborn error of immunity.Unrelated-donor allo-HSCT (conditioning regimen: fludarabine 150 mg/m², busulfan 4 mg/kg, ATG 5 mg/kg and rituximab 375 mg/m², with PTCy-based GVHD prophylaxis) was complicated by HHV-6 infection, severe infusion-associated rash, and subsequent graft rejection by day +60. Six months later a second haploidentical HSCT from the mother (conditioning regimen: fludarabine 150 mg/m², treosulfan 21 g/m², rituximab 375 mg/m² and alemtuzumab 0.4 mg/kg with PTCy-based GVHD prophylaxis) resulted in timely engraftment and was complicated by megakaryocytic lineage hypofunction, viral reactivation, COVID-19 infection, and mild skin GVHD.Case #2...The course was marked by transfusion dependence, recurrent infections and refractory thrombocytopenia despite romiplostim and eltrombopag therapy.At the age of..."

Clinical • Acute Kidney Injury • Aplastic Anemia • Bone Marrow Transplantation • CNS Disorders • Graft versus Host Disease • Hematological Disorders • Herpes Simplex • Immunology • Infectious Disease • Metabolic Disorders • Nephrology • Novel Coronavirus Disease • Pediatrics • Thrombocytopenia • Transplant Rejection • Transplantation • RAD50

LESS INFLAMMATION, SAME SURVIVAL: PLERIXAFOR MOBILIZATION REDUCES ENGRAFTMENT CRS IN PAEDIATRIC HSCT COMPARED TO GCSF ALONE (EBMT 2026) - "Recipient outcomes evaluated included engraftment kinetics, CRS, engraftment syndrome, acute GVHD, mortality, relapse, TATMA, secondary graft dysfunction requiring romiplostim, SOS, day +15/+30 chimerism, viral reactivation, and immune reconstitution across CD4 subsets, NK and B cells at days +30, +60 and +90. The CD34:CD3 ratio differed significantly between mobilization strategies. G-CSF–only mobilization yielded grafts richer in CD34⁺ stem cells, whereas plerixafor-mobilized grafts were more T-cell enriched. These differences in graft biology did not translate into inferior clinical outcomes in the plerixafor group.Engraftment CRS was significantly lower in the plerixafor cohort and anakinra requirement for cytokine control was numerically reduced, indicating a lower inflammatory burden despite graft compositional differences.Immune recovery, survival, and complication profiles remained equivalent between mobilization strategies.Overall, plerixafor is a safe and..."

Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Immunology • Inflammation • Pediatrics • CD34

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

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

Efficacy and safety of romiplostim N01 for refractory aplastic anemia: a retrospective single-center study (PubMed, Zhonghua Xue Ye Xue Za Zhi) - "No new or expanding paroxysmal nocturnal hemoglobinuria (PNH) clones were detected. These findings suggest that romiplostim N01 can induce rapid and high hematologic response rates with a favorable safety profile in patients with refractory AA who have failed full-dose cyclosporine A and multiple oral TPO-RA; no obvious short-term signals of clonal evolution were observed."

Journal • Retrospective data • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Hepatology • Liver Failure • Myelodysplastic Syndrome • Oncology • Paroxysmal Nocturnal Hemoglobinuria • Rare Diseases

ACQUIRED CHYLOMICRONEMIA SYNDROME WITH SEVERE HYPERTRIGLYCERIDEMIC PANCREATITIS AND CONCURRENT IMMUNE THROMBOCYTOPENIA IN A RENAL TRANSPLANT RECIPIENT (ACC 2026) - "Decision-Making: Triglycerides improved with insulin infusion, but thrombocytopenia was refractory to dexamethasone, IVIG, and romiplostim during the index admission...Lipid therapy was intensified with ezetimibe, icosapent ethyl, and a switch from gemfibrozil to fenofibrate...Olezarsen was initiated, resulting in rapid, sustained triglyceride reduction... We report an extremely rare autoimmune overlap syndrome involving acquired chylomicronemia and ITP. While APOC3 inhibitors have shown efficacy in familial and multifactorial forms, their use in acquired chylomicronemia has not been previously described. This successful response supports the need for further investigation of APOC3 inhibition in this population."

Clinical • Chronic Kidney Disease • Diabetes • Dyslipidemia • Focal Segmental Glomerulosclerosis • Genetic Disorders • Glomerulonephritis • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Obesity • Pancreatitis • Rare Diseases • Renal Disease • Severe Hypertriglyceridemia • Thrombocytopenia • Thrombocytopenic Purpura • Transplantation • Type 2 Diabetes Mellitus

ROMIPLOSTIM N01 FOR PROMOTING PLATELET ENGRAFTMENT AFTER ALLO-HSCT: INTERIM RESULTS OF A PHASE II EXPLORATORY TRIAL FROM NICHE COHORT (EBMT 2026) - "In summary, this interim analysis indicates that romiplostim N01 may accelerate platelet engraftment after allo-HSCT and demonstrated favorable tolerability and safety during the observation period; however, its clinical value requires confirmation in larger studies."

P2 data • Bone Marrow Transplantation • Hematological Malignancies • Thrombocytopenia

EFFICACY AND SAFETY OF ROMIPLOSTIM VERSUS RECOMBINANT HUMAN THROMBOPOIETIN (RHTPO) FOR HEMATOPOIETIC RECONSTRUCTION IN PATIENTS UNDERGOING AUTOLOGOUS HEMATOPOIETIC STEM CELL TRANSPLANTATION (ASCT) (EBMT 2026) - "In ASCT patients, Romiplostim is non-inferior to rhTPO regarding primary efficacy endpoints for hematopoietic reconstruction. Although the difference in engraftment time was not statistically significant, Romiplostim offers potential advantages in safety, convenience, and cost, and demonstrates a significant promoting effect on lymphocyte recovery, suggesting a more active role in accelerating immune reconstitution."

Clinical • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Thrombocytopenia • Transplantation

ROMIPLOSTIM N01 FOR PROMOTING PLATELET ENGRAFTMENT AFTER ALLO-HSCT: INTERIM RESULTS OF A PHASE II EXPLORATORY TRIAL FROM NICHE COHORT (EBMT 2026) - "In summary, this interim analysis indicates that romiplostim N01 may accelerate platelet engraftment after allo-HSCT and demonstrated favorable tolerability and safety during the observation period; however, its clinical value requires confirmation in larger studies."

P2 data • Bone Marrow Transplantation • Hematological Malignancies • Thrombocytopenia

Romiplostim versus Placebo for Chemotherapy-Induced Thrombocytopenia. (PubMed, N Engl J Med) - P3 | "In this phase 3, placebo-controlled trial, romiplostim was efficacious in treating CIT. (Funded by Amgen and the Biomedical Advanced Research and Development Authority; RECITE ClinicalTrials.gov number, NCT03362177.)."

Journal • Cardiovascular • Colorectal Cancer • Gastric Cancer • Gastroesophageal Cancer • Gastrointestinal Cancer • Hematological Disorders • Oncology • Pain • Pancreatic Cancer • Solid Tumor • Thrombocytopenia

Efficacy and safety of romiplostim with horse anti-thymocyte globulin and cyclosporine in acquired aplastic anemia. (PubMed, Int J Hematol) - "This study evaluated the efficacy and safety of high-dose romiplostim (ROMI) combined with horse anti-thymocyte globulin (hATG) and cyclosporine A (CyA) as first-line immunosuppressive therapy for aplastic anemia (AA). High-dose ROMI combined with hATG and CyA demonstrated favorable efficacy and tolerability as a first-line treatment for transfusion-dependent AA. However, larger prospective studies are required to confirm these findings."

Journal • Anemia • Aplastic Anemia • Hematological Disorders

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

Clinical Evidence of Romiplostim in Aplastic Anemia: Korean Expert Recommendations (ICKSH 2026) - "Horse antithymocyte globulin is not available, and rabbit ATG is exclusively used in clinical practice, which is associated with deepe r and more prolonged lymphocyte depletion. Furthermore, regulatory restrictions limit the maximum approved dose of eltrombopag in Korea compared with Western countries, potentially affecting treatment efficacy in some patients...Accumulating clinical evidence suggests that sequential use or switching between TPO -RAs may provide meaningful therapeutic benefit in selected patients with AA. This lecture will review the key clinical trials of TPO -RAs in AA, discuss mechanistic and clinical differences between available agents, and highlight practical treatment strategies with particular attention to the clinical and regulatory context in Korea ."

Clinical • Anemia • Aplastic Anemia • Hematological Disorders

A New Chapter in ITP Management - Updated Trials (ICKSH 2026) - "Current treatments include rituximab, a monoclonal antibody that targets CD20 on B cells; thrombopoietin receptor agonists (TPO -RA), such as eltrombopag and romiplostim, which stimulate platelet production in the bone marrow; and splenectomy, a surgical intervention often effective in cases involving splenic platelet destruction. This overview aims to elucidate the currently available treatment options for refractory ITP, discuss the emerging rationale behind novel therapeutic approaches, and highlight best practices derived from both published studies and real -world clinical experiences. By optimizing management strategies, healthcare providers can enhance patient safety and efficacy in treating this complex disorder, ultimately improving the quality of life for indiv iduals affected by refractory ITP."

Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura