Nplate
Evidence Grade A — Regulatory approved. 847 published studies. 109 registered clinical trials.
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Romiplostim (sold as Nplate) stimulates the bone marrow to produce more platelets — the blood cells essential for clotting. It is approved for immune thrombocytopenia (ITP), a condition where the immune system destroys platelets faster than the body can replace them, leaving patients at risk of dangerous bleeding. Given as a weekly injection with dose adjusted based on platelet counts.
847 published studies: 626 human, 30 animal, 22 in-vitro, 217 reviews
Romiplostim is marketed as Nplate (approved August 2008) for chronic immune thrombocytopenia in adults and children who have not responded adequately to other treatments. Administered as a weekly subcutaneous injection with dose adjusted to maintain platelet counts.
In clinical trials, 38–56% of patients achieved durable platelet responses, and long-term extension data over five years showed sustained effectiveness. Romiplostim transformed ITP management by offering an alternative to immunosuppression and splenectomy (surgical removal of the spleen). Potential risks include bone marrow fibrosis with long-term use (generally mild and reversible) and rebound thrombocytopenia if treatment is stopped abruptly.
The body normally regulates platelet production through thrombopoietin (TPO), a hormone that tells bone marrow cells to make more platelets. In ITP, platelet destruction outpaces production. Romiplostim activates the same TPO receptor on bone marrow megakaryocytes (the cells that produce platelets), stimulating them to multiply, mature, and release platelets into the blood. Despite activating the TPO receptor, romiplostim has no structural resemblance to natural TPO — it was discovered through random peptide library screening — which means it does not trigger the antibody responses that plagued earlier TPO-like drugs.
Clinical trials showed durable platelet responses in 38–56% of patients, and long-term data extending over ten years confirm sustained effectiveness. Romiplostim transformed ITP management by offering an alternative to immune-suppressing drugs and splenectomy (surgical removal of the spleen, which was previously a common approach for resistant ITP). Potential risks include mild bone marrow fibrosis with long-term use (generally reversible after stopping), blood clotting events, and rebound low platelets if treatment is stopped abruptly. A risk management programme (NEXUS) is required. There is a small background rate of blood cancers (MDS/AML) in ITP patients, and whether romiplostim contributes to this risk remains unclear from current data.
Preoperative Use of Romiplostim in Thrombocytopenic Patients Undergoing Cardiac Surgery.
UI-Romi-02; Romiplostim Added to Standard of Care for Treatment Naive and Relapsed or Refractory Severe Aplastic Anemia
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)
Phase II Study of Orelabrutinib in Combination With Romiplostim N01 in Patients With Primary Immune Thrombocytopenia (ITP) Who Have Received At Least One Prior Line of Therapy
Romiplostim Versus rhTPO for Platelet Engraftment After Transplant in MDS and AA
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