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

Rare Intersections: Two Cases Illustrating the Immune Complexity of Chronic Lymphocytic Leukemia Associated With Paraneoplastic Pemphigus With Ocular Involvement and Temozolomide-Induced Autoimmune Cytopenias in Concurrent Lymphoproliferative Disease (SOHO 2026) - "Despite aggressive multimodal immunosuppression (prednisone, cyclosporine, upadacitinib, intravenous methylprednisolone, IVIG, and cyclophosphamide) alongside venetoclax-obinutuzumab, he achieved near-complete MRD-negative remission but developed fulminant corneal melt with uveal prolapse requiring left eye evisceration. Venetoclax was subsequently discontinued due to severe cytopenias, and therapy was transitioned to acalabrutinib to leverage BTK inhibition for both sustained CLL control and its favorable T-cell immunomodulatory properties relevant to PNP pathogenesis. Escalation with a higher dose of upadacitinib and ruxolitinib is under consideration for refractory mucosal disease...Romiplostim was initiated, with rituximab under consideration for refractory thrombocytopenia... Together, these cases demonstrate that CLL is not a passive bystander but an active immunologic substrate that amplifies the risk of severe paraneoplastic syndromes and unmasks autoimmune..."

Clinical • Brain Cancer • Chronic Lymphocytic Leukemia • Glioblastoma • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • IGH • MGMT • RUNX1

Retrospective evaluation of romiplostim use for breast cancer patients with chemotherapy-induced thrombocytopenia (ESMO 2026) - No abstract available

Retrospective data • Breast Cancer • Oncology • Solid Tumor

Synergistic Effects of Romiplostim and Avatrombopag in Severe Chemotherapy-Induced Thrombocytopenia: A Serendipitous Finding. (PubMed, OMICS) - "Romiplostim combined with avatrombopag yields rapid platelet recovery with favorable safety for severe refractory CIT, enabling chemotherapy resumption. It is a promising salvage strategy for single-agent TPO-RA nonresponders, warranting prospective randomized controlled trials for validation."

Journal • Cardiovascular • Thrombocytopenia • Venous Thromboembolism

Resolution of Clonal Cytopenia of Undetermined Significance Following Autologous Stem Cell Boost in a Patient with Multiple Myeloma: A Case Report (IMS 2026) - "He received bortezomib, lenalidomide, and dexamethasone induction and autologous stem cell transplant consolidation with melphalan prep followed by stem cell rescue utilizing 6.32 x 10^6 CD34+ cells/kg...He remained transfusion dependent despite supportive care including epoetin alfa and romiplostim... This case suggests that autologous stem cell boost may represent a potential treatment option for select patients with CCUS and refractory cytopenias, a population for whom no established disease ‑ modifying therapies currently exist. These findings support consideration of early collection and storage of additional autologous stem cells when available. Stem cell boost may provide a valuable salvage strategy in certain clinical settings."

Case report • Clinical • IO biomarker • Hematological Malignancies • Multiple Myeloma • Myelodysplastic Syndrome • CD34

Preemptive Tocilizumab in Patients with Relapsed-Refractory Multiple Myeloma Receiving Bispecific: A Case Series (IMS 2026) - "Teclistamab (Tec), talquetamab (Tal), and elranatamab (Elra) are bispecific antibodies (BsAbs) approved for relapsed-refractory multiple myeloma (RRMM)...All patients received intravenous immunoglobulin to maintain functional IgG ≥500 mg/dL, filgrastim to maintain absolute neutrophil 1.00 K/mcL, epoetin alfa to maintain hemoglobin above 10 g/dL, and romiplostim to maintain platelets above 50 K/mcL...The only incident of CRS occurred with a single patient who developed grade 1 CRS on C1D3 which resolved after one dose of dexamethasone 10 mg... Preemptive Toci may decrease the incidence and severity of CRS in patients with RRMM receiving BsAbs, without compromising response. In this small observational analysis, no patients experienced prolonged hospitalization for management of CRS."

Bispecific • Clinical • Hematological Malignancies • Infectious Disease • Multiple Myeloma

Weekly Metronomic Venetoclax and Decitabine/Cedazuridine in Acute Myeloid Leukemia Patient Declining Blood Transfusion. (PubMed, Case Rep Oncol) - "Due to refusal of transfusions, he was treated with a metronomic, all-oral regimen of VEN 400 mg weekly and decitabine/cedazuridine (DEC-C) 35/100 mg weekly, supported by erythropoiesis-stimulating agent (darbepoetin alfa) and thrombopoietin agonist (romiplostim) as needed. This case demonstrates that a metronomic, all-oral VEN and DEC-C regimen can achieve hematologic improvement and molecular response in AML while maintaining transfusion independence. It highlights a feasible and safe therapeutic option for patients declining blood products, achieving disease control with minimal toxicity."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • ASXL1 • DDX41 • PPM1D

Efficacy and Safety of Romiplostim N01 for Injection in Promoting Platelet Reconstitution After Allogeneic Hematopoietic Stem Cell Transplantation (clinicaltrials.gov) - P2 | N=87 | Not yet recruiting | Sponsor: The First Affiliated Hospital of Henan University of Science and Technology

New P2 trial • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Thrombocytopenia • Transplantation

Romiplostim for cancer treatment-induced thrombocytopenia in acute promyelocytic leukemia: a case report. (PubMed, Front Oncol) - "All-trans retinoic acid (ATRA) combined with arsenic trioxide (ATO) is a first-line treatment regimen for acute promyelocytic leukemia (APL), with generally milder myelosuppressive effects compared with conventional chemotherapy...A 60-year-old female patient with APL developed refractory grade 3-4 thrombocytopenia during ATO maintenance therapy, with no significant response to recombinant human thrombopoietin or hetrombopag treatment (platelet nadir: 8 × 109/L; duration of platelet count <50 × 109/L: 8 weeks)...Romiplostim may promote the recovery of residual megakaryopoietic capacity and provide a potential therapeutic option for such patients. However, its precise mechanism of action and the patient populations most likely to benefit require further investigation."

Journal • Acute Promyelocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Thrombocytopenia

Chemotherapy-induced thrombocytopenia: from "platelet counting" to ecological repair of the bone marrow. (PubMed, Support Care Cancer) - "The "seed and soil" model organizes previously published evidence and should be regarded as hypothesis-generating in human CIT. Prospective studies are needed before microenvironment-directed combinations or CIT-specific biomarker thresholds can be recommended."

Journal • Review • Hematological Disorders • Thrombocytopenia

Temozolomide-Induced Immune Dysregulation in a Patient With Concurrent Glioblastoma and Chronic Lymphocytic Leukemia: A Case of Warm Autoimmune Hemolytic Anemia and Refractory Thrombocytopenia (SOHO 2026) - "Avatrombopag was ineffective, and romiplostim was subsequently initiated. This case highlights the immune complexity of TMZ therapy in patients with concurrent CLL and T-LGL expansion. TMZ may precipitate or unmask autoimmune cytopenias in immunologically predisposed patients. Vigilance for immune-mediated hematologic toxicity beyond expected myelosuppression is warranted in patients with underlying lymphoproliferative disease."

Clinical • Brain Cancer • Chronic Lymphocytic Leukemia • Glioblastoma • Hematological Malignancies • Leukemia • Oncology • Solid Tumor • DNMT3A • HP • MGMT • RUNX1 • TET2

Early Safety and Efficacy of CAR-T Cell Therapy in Precursor Myeloma: Results of the CAR-PRISM Study Using Ciltacabtagene Autoleucel in High-Risk Smoldering Myeloma (ASH 2024) - "Patients receive lymphodepletion (LD) with fludarabine and cyclophosphamide followed by cilta-cel infusion...Tocilizumab was administered to 4 patients and 2 patients received dexamethasone...One patient experienced grade 4 immune related thrombocytopenia secondary to fludarabine, which resolved within 2 weeks after treatment with dexamethasone, IVIG and romiplostim...All patients achieved MRD negative (10-6 ) disease and long-term follow up is required to determine whether these responses will be sustained. The benefit of no induction therapy and potential long-term remissions may be disruptive for future therapeutic algorithms in MM."

CAR T-Cell Therapy • Clinical • Anemia • CNS Disorders • Dyslipidemia • Hypertriglyceridemia • Infectious Disease • Movement Disorders • Multiple Myeloma • Neutropenia • Parkinson's Disease • Smoldering Multiple Myeloma • Thrombocytopenia • CD4

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

ROMISTER: A Study of Romiplostim Plus Predniso(lo)ne vs. Predniso(lo)ne Alone for the Treatment of Previously Untreated Primary Immune Thrombocytopenia (ITP) (clinicaltrials.gov) - P3 | N=126 | Recruiting | Sponsor: Amgen | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Immune Thrombocytopenic Purpura • Thrombocytopenia • Thrombocytopenic Purpura

An unsual case of late onset mycophenolate mofetil induced pancytopenia with sterile oral ulcers in a kidney transplant recipient: A case report (TTS 2026) - "Meropenem and Caspofungin started empirically...G-CSF(300 mcg OD) initiated,teicoplanin (Day 5 for neutropenic fever), 2 units PRBCs, 2 SDP platelets (for gross hematuria), and romiplostim 50 mcg SC...KTRs surviving febrile neutropenia face 31% acute rejection and 15% graft loss risk from persistent dose reduction. Our patient requires vigilant immunological monitoring on steroid-tacrolimus regimen with plan to reintroduce MMF in a lower dose later."

Case report • Clinical • Febrile Neutropenia • Hematological Disorders • Hemophagocytic lymphohistiocytosis • Infectious Disease • Leukopenia • Neutropenia • Thrombocytopenia • Transplant Rejection • Transplantation

Recurrent Infection-Triggered Immune Thrombocytopenia Flares in Medullary Sponge Kidney: A Case Report. (PubMed, Cureus) - "Over several months, she experienced recurrent urological events, including obstructive nephropathy, extended-spectrum beta-lactamase (ESBL) urosepsis, pyelonephritis, ureteritis, and repeated ureteral stenting. During a March 2026 admission for right pyelonephritis with bilateral stents, her platelet count fell abruptly from approximately 120 × 10³/µL to 7 × 10³/µL, accompanied by gross hematuria, and improved after intravenous immunoglobulin and dexamethasone...She was treated with meropenem, fluconazole, hydration, analgesia, and intravenous immunoglobulin, while corticosteroids were deferred because of active infection and romiplostim was held...This case emphasizes that recurrent MSK-related urinary infections may precipitate clinically significant ITP flares. Early platelet monitoring and coordinated hematology, infectious disease, and urology input are essential when infection control, bleeding risk, and procedural decisions..."

Journal • Hematological Disorders • Immune Thrombocytopenic Purpura • Immunology • Infectious Disease • Metabolic Disorders • Nephrology • Pain • Renal Calculi • Renal Disease • Thrombocytopenia • Thrombocytopenic Purpura • Urology

Chromosome 17p deletion in aplastic anaemia with paroxysmal nocturnal haemoglobinuria clone: a diagnostic challenge. (PubMed, J Hematop) - "Low-level chromosome 17p deletion may occur in AA-PNH overlap syndrome without morphologic or molecular evidence of myeloid neoplasm. Careful clinicopathologic correlation and long-term surveillance are essential before attributing such abnormalities to clonal evolution."

Journal • Anemia • Aplastic Anemia • Complement-mediated Rare Disorders • Hematological Disorders • Hematological Malignancies • Oncology • Paroxysmal Nocturnal Hemoglobinuria • TP53

Transient ST-segment elevation following coronary artery bypass grafting in a patient with immune thrombocytopenia treated with romiplostim: a case report. (PubMed, Egypt Heart J) - "Transient ST-segment elevation after CABG requires systematic evaluation for both ischemic and non-ischemic causes. In this patient, the combination of inferior ST-segment elevation, PR-segment depression, a documented pericardial friction rub, preserved ventricular function, absence of regional wall motion abnormalities, declining hs-cTnI, elevated inflammatory markers, and spontaneous ECG resolution favored an early postoperative inflammatory process. Romiplostim should be regarded as part of the patient's overall thrombotic-risk profile rather than as a proven direct cause of the transient ECG abnormality."

Journal • Cardiovascular • CNS Disorders • Coronary Artery Disease • Depression • Hematological Disorders • Immune Thrombocytopenic Purpura • Inflammation • Myocardial Infarction • Myocardial Ischemia • Psychiatry • Thrombocytopenia • Thrombocytopenic Purpura • Thrombosis • CRP • TNNI3

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

Real-world romiplostim dosing, response and home administration in paediatric immune thrombocytopenia. (PubMed, Br J Haematol) - No abstract available

Journal • Real-world evidence • Hematological Disorders • Immune Thrombocytopenic Purpura • Pediatrics • Thrombocytopenia • Thrombocytopenic Purpura

CLINICAL AND MOLECULAR MARKERS FOR PREDICTING RESPONSE TO ROMIPLOSTIM TREATMENT IN LOWER-RISK MYELODYSPLASTIC SYNDROMES (EHA 2022) - P2 | "To avoid overfitting of variables and to confirm our results, the here presented response prediction model needs to be validated in an external independent cohort. * U.P. and L.A. contributed equally to this study as senior authors"

Biomarker • Clinical • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Thrombocytopenia • SRSF2

Prospective validation of a biomarker-driven response prediction model to romiplostim in lower-risk myelodysplastic neoplasms - results of the EUROPE trial by EMSCO. (PubMed, Leukemia) - "Sequential analysis of variant allelic frequency of mutations like SRSF2 did not reveal an impact of ROM on clonal evolution in both responders and non-responders. In summary, our study confirms the safety and efficacy of ROM in LR-MDS patients and may allow to better define subgroups of patients with a high likelihood of response."

Biomarker • Journal • Hematological Disorders • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • Thrombocytopenia • SRSF2

Open-Label, Pilot Study of Romiplostim for Thrombocytopenia After Autologous Hematopoietic Cell Transplantation. (PubMed, Blood Adv) - P2 | "In conclusion, romiplostim showed promising activity and safety after autoHCT, but the improvement in platelet counts occurred later than the goal of shortening the duration and depth of the platelet nadir. This trial was registered at www.clinicaltrials.gov (NCT04478123)."

Journal • Cardiovascular • Hematological Disorders • Hematological Malignancies • Lymphoma • Multiple Myeloma • Oncology • Pulmonary Embolism • Respiratory Diseases • Thrombocytopenia • Transplantation

A PILOT STUDY OF THE ANTI-SLAMF7 MONOCLONAL ANTIBODY, ELOTUZUMAB, IN PATIENTS WITH MYELOFIBROSIS. (EHA 2023) - P2 | "Elotuzumab (Elo), a SLAMF7-targeting monoclonal antibody, inhibited the differentiation of MF patient-derived fibrocytes in vitro and romiplostim-induced bone marrow (BM) fibrosis and splenomegaly in vivo (Maekawa, Blood 2019). Elo is active in the treatment of MF and has an excellent safety profile. A total of 15 pts are planned to be accrued to this pilot study. Future studies in combination with JAKi therapy appear warranted."

Clinical • Acute Myelogenous Leukemia • Diabetes • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Pain • Pneumonia • Polycythemia Vera • Respiratory Diseases • Thrombocytosis • SLAMF7

Beyond Platelet Transfusions: Romiplostim Reduces Chemotherapy Dose Modifications in Chemotherapy-Induced Thrombocytopenia: A Meta-Analysis of Randomized Controlled Trials (SOHO 2026) - "In patients with CIT, romiplostim is associated with a higher likelihood of maintaining planned chemotherapy dosing without a clear increase in serious adverse events. These findings support its potential role in preserving chemotherapy dose intensity. Given the limited number of trials and imprecision in secondary outcomes, larger randomized studies across tumor types and treatment regimens are warranted before routine clinical adoption."

Retrospective data • Oncology

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