Nplate (romiplostim)

P2, N=36, Not yet recruiting, Wuhan Union Hospital, China

P=N/A, N=36, Not yet recruiting, Union Hospital, Tongji Medical College, Huazhong University of Science and Technology; Union Hospital, Tongji Medical College, Huazhong University of

P2, N=129, Recruiting, Institute of Hematology & Blood Diseases Hospital, China

P3, N=162, Recruiting, Amgen | Trial completion date: Feb 2027 --> Mar 2028 | Trial primary completion date: Feb 2026 --> Mar 2027

P2, N=30, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting

P2, N=48, Not yet recruiting, Institute of Hematology & Blood Diseases Hospital, China

P=N/A, N=36, Active, not recruiting, Kyowa Kirin Co., Ltd. | Enrolling by invitation --> Active, not recruiting | Trial completion date: Jun 2024 --> Jun 2026

P1, N=15, Completed, Anjali Sharathkumar | Active, not recruiting --> Completed | Trial completion date: Jun 2024 --> Jan 2024

P4, N=40, Not yet recruiting, Peking Union Medical College Hospital

P3, N=160, Active, not recruiting, Geropharm

P1, N=15, Active, not recruiting, Anjali Sharathkumar | Trial primary completion date: Jun 2024 --> Jan 2024

P3, N=60, Not yet recruiting, Shandong University

P2, N=30, Not yet recruiting, M.D. Anderson Cancer Center

P4, N=60, Completed, Sobi, Inc. | Active, not recruiting --> Completed

P3, N=30, Recruiting, Ain Shams University

3%) each received red cells, platelets, G-CSF and romiplostim; majority did not need supportive treatment...mEASIX score may be used as an early predictor of CRS for permissive use of Tocilizumab before onset of severe symptoms. This is the first-in-India industry study evaluating mEASIX and ICAHT in patients treated with CAR-T cell therapy. mEASIX score 1 at D+4 was mildly predictive of CRS. All patients developed ICAHT but only a small number of patients needed supportive treatment.

Enoxaparin was started for thromboses and levofloxacin for neutropenia...Darbepoetin alfa and romiplostim were added...Ravulizumab is non-inferior to eculizumab, with the added advantage of less frequent dosing (every 8 weeks versus 2 weeks)...Heparin is preferred in acute thrombotic states. Most hematologists recommend anticoagulation till a few months after starting complement inhibition.

Case Presentation: We present a 14-year-old Hispanic male with Philadelphia chromosome positive acute lymphoblastic leukemia (Ph+ ALL) who received a maternal haploidentical HSCT with myeloablative conditioning and post-transplant cyclophosphamide...Multiple treatments including romiplostim; eltrombopag; high dose immune globulin (IVIG); rituximab; methylprednisolone; and decitabine were not successful in improving platelet count (Figure 1)...Patient continues to maintain platelets above 100 x10 3/uL with no medication toxicities or adverse events noted. Patient has been able to return to his pre-alloHSCT lifestyle including contact sports and re-immunizations.

Bone marrow biopsy showed partial CLL involvement; bendamustine+rituximab therapy was augmented with granulocyte colony-stimulating factor (g-CSF) and romiplostim to mitigate worsening pancytopenia, without response. Laboratory evaluation revealed a pattern supportive of the clinical impression of HLH, while bone marrow biopsy showed persistent CLL, new reticulin fibrosis, megakaryocytic proliferation, and 32% mutated TET2, but no compelling morphologic evidence of hemophagocytosis. The patient recovered with dexamethasone and g-CSF support.

P2, N=60, Completed, Memorial Sloan Kettering Cancer Center | Active, not recruiting --> Completed

Patients with ITP lasting for ≥3 months (persistent or chronic primary ITP), and who do not respond adequately or maintain a response to initial therapy, are usually treated with advanced therapies such as thrombopoietin receptor agonists (TPO-RAs [avatrombopag/romiplostim/eltrombopag]), rituximab, and fostamatinib. All-cause hospitalization was high in this population. Further studies are needed to understand long-term clinical and economic outcomes among ITP patients.

Patients who fail to respond or maintain a response to initial therapy (e.g., corticosteroids, intravenous immunoglobulins), move to advanced therapies with robust evidence such as thrombopoietin receptor agonists (TPO-RAs [eltrombopag/ romiplostim/ avatrombopag]), rituximab, and fostamatinib, as per the international consensus guidelines. Approximately 5% of patients underwent splenectomy (incidence rate: 27 per 1000 person-years [95% CI: 15–40]) with mean (SD) time of 245 (186) days post initiation of advanced ITP therapy (Table 2). Conclusion This study demonstrated high clinical burden and need for subsequent additional therapies among patients with primary ITP lasting ≥3 months, which is evident from high rates of switching between advanced therapies, bleeding events requiring medical attention, TEs, and ITP-related hospitalizations.

P3, N=162, Active, not recruiting, Amgen | Recruiting --> Active, not recruiting

P4, N=60, Active, not recruiting, Sobi, Inc. | Recruiting --> Active, not recruiting | N=100 --> 60

We report a case series of 6 individuals with breast cancer that experienced dose-reductions and therapy delays due to thrombocytopenia secondary to trastuzumab emtansine or trastuzumab deruxtecan therapy and received intervention with TPO-RA. All 6 were able to resume therapy with TPO-RA support.

P1, N=15, Active, not recruiting, Anjali Sharathkumar | Recruiting --> Active, not recruiting | N=25 --> 15 | Trial completion date: Dec 2023 --> Jun 2024 | Trial primary completion date: Jun 2023 --> Jun 2024

P2, N=62, Completed, Memorial Sloan Kettering Cancer Center | Active, not recruiting --> Completed

Accordingly, Srsf2 delays myelofibrosis induced by the thrombopoietin receptor agonist Romiplostim in Jak2 wild-type animals. These results unveil JAK2 exon 14 skipping promotion as a strategy to reduce JAK/STAT signaling in pathological conditions.

P2, N=0, Withdrawn, European Organisation for Research and Treatment of Cancer - EORTC | N=100 --> 0 | Trial completion date: Apr 2025 --> Dec 2022 | Recruiting --> Withdrawn | Trial primary completion date: Sep 2024 --> Dec 2022

In alignment with NCCN guidelines, we recommend that patients with CIT participate in a clinical trial for consideration of TPO-RA treatment or consider off-label use of romiplostim when participation in clinical trials is not possible. The literature to date supports the use of TPO-RAs for treatment of persistent CIT. Further data is needed to describe the long-term efficacy, safety, and prescribing practices of TPO-RAs in a diverse patient population with a variety of tumor types and chemotherapy regimens in addition to exploring the underlying biology of CIT.

In terms of the management of prolonged thrombocytopenia, most survey participants favored TPO agonists such as eltrombopag or romiplostim (86%), followed by autologous stem cell boost, if available (43%). These findings underline the current heterogeneity of practice patterns regarding ICAHT and invite the development of consensus guidelines, which may harmonize grading, establish standard operating procedures for diagnosis, and set management guidelines. For this purpose, an international expert panel will meet in Lille in March 2023. The resulting consensus guidelines will be presented together with the survey results at the EHA meeting.

Background: Romiplostim (Rom), a thrombopoietin receptor agonist (TPO-RA), has been shown to promote tri-lineage hematopoiesis in patients with acquired aplastic anemia (AA) refractory to immunosuppressive therapy (IST) or eltrombopag, another TPO-RA. This is the first study of Rom combined with CsA in patients with acquired AA requiring transfusions who were previously untreated with IST. This ATG-free doublet regimen produced high HRR at Week 27 (41.7%), with manageable AEs, and could therefore serve as a new first-line treatment option in patients with AA depending on the severity and tolerability of ATG. Thrombopoietin (TPO), Aplastic anemia, Bone marrow failure

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.

This group included one cephalexin induced ITP and one patient subsequently diagnosed with myelodysplasia...The other three patients started romiplostim at 3, 5 and 5 months...Regardless, sustained remissions occurred in both groups showing both doses can be effective. It was also a well-tolerated treatment with just 1 infective complication.

P3, N=162, Recruiting, Amgen | Trial completion date: Jul 2024 --> Mar 2025 | Trial primary completion date: Aug 2023 --> Apr 2024

A retrospective study was conducted on patients who received TPO-RAs eltrombopag or romiplostim because of severe persistent thrombocytopenia after alloHSCT at the University Hospital Centre Zagreb, Croatia, from 1/2018 until 11/2022...Nine (52.9%) patients received post-transplant cyclophosphamide... Results of this single-centre retrospective analysis confirmed successful usage of TPO-RAs with 76.5% patients who achieved platelet response with well-known safety profile in the setting of demanding persistent severe thrombocytopenia after alloHSCT, that needs to be further evaluated in larger prospective studies.

P3, N=162, Recruiting, Amgen | Trial completion date: Mar 2025 --> Jan 2027 | Trial primary completion date: Apr 2024 --> Feb 2026

Persistent cytopenias, including thrombocytopenia, are a frequent side effect and can lead to substantial clinical complications (bleeding, infections,...).We report the case of a 47-year-old Caucasian man who received in November 2021 anti-CD19 CAR-T (Brexucabtagene autoleucel) for relapsed Philadelphia + B-ALL diagnosed in 2008. He previously received intensive chemotherapy, tyrosine kinase inhibitors (including asciminib), and two allogenic stem cell transplantations...After 6 weeks, the patient became transfusion independent and thrombocytosis increased until >50G/L. There was no evidence of adverse effects.Romiplostim seems to be a safe and efficient treatment, and should be considered for persistent transfusion-dependent thrombocytopenia following CAR-T cell therapy.

Patients received prophylactic vitamin B12, folic acid, oral iron and vitamin K supplements, as well as tranexamic acid and romiplostim for platelets < 10 thousand/microliter and erythropoetin support if hemoglobin (Hb) < 10 g/dL. In this retrospective cohort of real-world patients receiving ASCT under a bloodless therapy protocol, we have shown that our observed transplant-related morbidity and mortality outcomes warrants consideration of high-dose chemotherapy and ASCT for patients who cannot receive blood products. Further investigations are ongoing.

Three patients were treated with thrombopoietin receptor agonists (2 eltrombopag, 1 romiplostim) and had minimal transient platelet count increases not meeting the IWG MDS criteria for response. Three patients have received allogeneic hematopoietic stem cell transplant.Conclusion :This series describes a subset of CCUS patients with isolated thrombocytopenia associated with SRSF2 mutations. We also describe atypical megakaryocytic findings which do not meet current WHO diagnostic criteria for dysmegakaryopoiesis; however, the uniform presence of these findings in this cohort suggests an association with thrombocytopenia and CCUS.