<p>Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use</p>

Abstract

Immune thrombocytopenia (ITP) is an immune-mediated disorder resulting in platelet destruction and subsequent thrombocytopenia. Bleeding symptoms range from mild cutaneous bleeding to life-threatening hemorrhage. Romiplostim, a peptide-antibody fusion product, is a thrombopoietin receptor agonist (TPO-RA) indicated for use in patients with ITP. Romiplostim is US Food and Drug Administration (FDA) approved in children ≥1 year of age with ITP of >6 months' duration who have had an inadequate response to first-line therapies or splenectomy. FDA approval in adults with chronic ITP was expanded in October 2019 to include adults with newly diagnosed (<3 months' duration) and persistent (3-12 months' duration) ITP who demonstrated an inadequate response to first-line therapies, including corticosteroids and immunoglobulins, or splenectomy. The newly published 2019 American Society of Hematology ITP Guidelines place TPO-RAs, including romiplostim, as second-line therapies in both children and adults. Here, we review the use of romiplostim as second-line therapy with a spotlight on health-related quality of life, ease of use, and patient preference.

Real-world impact of primary immune thrombocytopenia and treatment with thrombopoietin receptor agonists on quality of life based on patient-reported experience: Results from a questionnaire conducted in Switzerland, Austria, and Belgium

“…In studies of patients with ITP, patients’ perspectives on how their disease and treatments affect their lives are typically assessed using patient-reported outcome instruments, which give a score for HRQoL (including sub-scores for individual domains of life) or the severity of symptoms [ 5 , 17 , 21 – 23 , 25 , 27 , 31 – 33 ]. However, the scores derived from these instruments may not give the full picture, because they do not capture patients’ feelings and opinions about their lives or ability to engage in specific daily activities (e.g., sport, work, holiday), or effects on relationships [ 23 , 34 ].…”

Immune Thrombocytopenia Associated with Hepatitis B Virus and Autoimmune Hepatitis and Recovery of Platelet Count following Liver Transplantation

“…In 2019, the US Food and Drug Administration (FDA) expanded the use criteria for romiplostim, a TPO receptor agonist, to adults with refractory ITP [ 6 ]. Romiplostim is a peptide-antibody fusion product that is administered via intravenous infusion and is reported to be well-tolerated with high patient satisfaction and quality of life [ 9 ].…”

Therapeutic Outcomes of High Dose-Dexamethasone versus Prednisolone + Azathioprine, Rituximab, Eltrombopag, and Romiplostim Strategies in Persistent, Chronic, Refractory, and Relapsed Immune Thrombocytopenia Patients

“…Eltrombopag increases platelet production by stimulating the proliferation and differentiation of megakaryocytes in the bone marrow via the Janus kinase/signal transducer and activator of the transcription (JAK/STAT) signaling pathway [ 24 , 25 ]. Romiplostim is a peptide-antibody fusion composed of two dimerized peptides conjugated to the heavy chain of IgG1 [ 26 ]. These peptides contain a 14-amino acid sequence distinct from the endogenous thrombopoietin sequence.…”