From the Guidelines
Romiplostim (Nplate) is not recommended for use in patients with Acute Myeloid Leukemia (AML). This is based on the most recent and highest quality evidence available, which suggests that the use of thrombopoietin receptor agonists (TPO-RAs) like romiplostim in patients with myelodysplastic syndromes (MDS) and thrombocytopenia has shown promise, but its use in AML is not supported due to concerns about potentially promoting leukemic cell growth and disease progression 1.
Key Considerations
- The primary concern with using romiplostim in AML is its potential to stimulate thrombopoietin receptors on leukemic blasts, which could lead to increased leukemic cell growth and worsen the disease outcome.
- Thrombocytopenia in AML is typically a result of bone marrow infiltration by leukemic cells and the effects of chemotherapy, rather than an immune-mediated process as seen in immune thrombocytopenia (ITP), for which romiplostim is approved.
- The appropriate management of thrombocytopenia in AML involves treating the underlying leukemia with chemotherapy and using platelet transfusions based on clinical guidelines and bleeding risk assessment.
Clinical Guidance
- For patients with AML and thrombocytopenia, consultation with a hematologist-oncologist is crucial to determine the best treatment approach, focusing on managing the leukemia and supporting the patient through the treatment process.
- The use of TPO-RAs like romiplostim in AML patients is not currently recommended outside of a clinical trial setting due to the potential risks and the lack of strong evidence supporting its safety and efficacy in this context 1.
From the FDA Drug Label
Nplate is not indicated for the treatment of thrombocytopenia due to MDS or any cause of thrombocytopenia other than ITP. Nplate stimulates certain bone marrow cells to make platelets and increases the risk of progression to acute myelogenous leukemia in patients with myelodysplastic syndromes
No, you should not use Nplate in Acute Myeloid Leukemia (AML) as it is not indicated for this condition and may increase the risk of progression of myelodysplastic syndromes to AML 2.
From the Research
Use of Nplate in Acute Myeloid Leukemia (AML)
- Nplate (romiplostim) is a thrombopoietic agent that has been studied in patients with thrombocytopenia, including those with AML.
- A study published in 2012 3 reported on the efficacy of romiplostim in a patient with AML and chemotherapy-induced transfusion-refractory thrombocytopenia, suggesting that romiplostim could be helpful in the management of AML patients with transfusion-refractory thrombocytopenia.
- However, other studies have raised concerns about the potential risk of progression to AML in patients with myelodysplastic syndrome (MDS) treated with romiplostim 4, 5, 6.
- A randomized controlled trial published in 2013 7 evaluated the efficacy and safety of romiplostim in patients with low- or intermediate-risk MDS receiving decitabine, and found that adding romiplostim to decitabine treatment was well tolerated and may be beneficial, with trends toward higher platelet counts and lower platelet transfusion rates and bleeding events.
- Overall, the evidence suggests that romiplostim may be effective in increasing platelet counts and reducing bleeding events in patients with AML and thrombocytopenia, but its use should be carefully considered in the context of potential risks and benefits, particularly with regard to the risk of progression to AML.
Key Findings
- Romiplostim can increase platelet counts and reduce bleeding events in patients with AML and thrombocytopenia 3, 7.
- The risk of progression to AML in patients with MDS treated with romiplostim is a concern, but the evidence is not consistent across studies 4, 5, 6.
- Romiplostim is generally well tolerated, but treatment-related adverse events can occur 5, 6, 7.