Romiplostim

Summary

Romiplostim (Nplate) is an FDA-approved thrombopoietin receptor agonist used to treat chronic immune thrombocytopenia (ITP) in adults and pediatric patients (≥1 year). It stimulates platelet production by mimicking the activity of endogenous thrombopoietin, reducing the risk of bleeding in patients with insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Mechanism of Action

Romiplostim is a fusion protein (peptibody) consisting of an IgG1 Fc domain linked to thrombopoietin (TPO) receptor-binding peptides. It binds to and activates the TPO receptor (c-Mpl) on megakaryocytes and their precursors, stimulating proliferation and differentiation to increase platelet production.

Routes of Administration

Goals & Uses

• Chemotherapy-induced thrombocytopenia (investigational)Oncology / Supportive CareLow
• Pediatric ITP managementPediatric HematologyHigh
• Increase platelet count in chronic ITPHematology / ThrombocytopeniaHigh
• Reduce bleeding risk in ITPHematology / HemostasisHigh
• Myelodysplastic syndrome-associated thrombocytopenia (investigational)Hematology / OncologyLow

Contraindications

• Hematologic malignancies other than ITPOncologicHigh
• Myelodysplastic syndrome (MDS)OncologicHigh
• Hypersensitivity to romiplostim or product componentsImmunologicHigh

Adverse Effects

• Rebound thrombocytopeniaHematologicalCommon
• HeadacheNeurologicCommonPain in the head or upper neck
• Bone marrow reticulin formationHematologicalUncommon
• Arthralgia / myalgiaMusculoskeletalCommon
• Progression to AML / MDS exacerbationOncologicRare
• ThromboembolismCardiovascularUncommon

Drug Interactions

• Immunosuppressants (e.g., corticosteroids, azathioprine)Low
• Antiplatelet agents (e.g., aspirin, clopidogrel)Moderate
• Anticoagulants (e.g., warfarin, heparin)Moderate

Population Constraints

• PregnancyReproductive SafetyRelative
• Hepatic impairmentOrgan FunctionRelative
• Renal impairmentOrgan ImpairmentRelative
• LactationReproductiveRelative
• Pediatric patients <1 yearAgeAbsolute

Regulatory Status

• European UnionApprovedApproved: Chronic immune thrombocytopenic purpura (ITP) in adults, Chronic ITP in pediatric patients ≥1 yearEMA approved 2009 (Nplate). Conditions and monitoring requirements apply under EU label.
• United StatesApprovedApproved: Chronic immune thrombocytopenia (ITP) in adults, Chronic immune thrombocytopenia (ITP) in pediatric patients ≥1 yearFDA approved August 2008. Available under REMS program. Boxed warning for risk of progression to AML/MDS.
• United KingdomApprovedApproved: Chronic immune thrombocytopenia (ITP) in adults, Chronic ITP in pediatric patients ≥1 yearApproved by MHRA post-Brexit with similar indications to EU label.

Approved by the FDA in August 2008 for chronic ITP. Approved by EMA in 2009. Available under a Risk Evaluation and Mitigation Strategy (REMS) program in the US due to concerns about bone marrow reticulin formation and progression to MDS/AML risk with prolonged use.

Evidence & Sources

No sources recorded yet.