Romiplostim for the Treatment of Immune Thrombocytopenia: Spotlight on Patient Acceptability and Ease of Use

Megan M Gilbert, Amanda B Grimes, Taylor Olmsted Kim, Jenny M Despotovic Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Houston, TX, USACorrespondence: Megan M GilbertDepartment of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Texas Children’s Hospital, 6701 Fannin Suite 1510, Houston, TX 77030, USATel +1 (832) 824-4736Fax +1 (832) 825-4846Email megan.gilbert@bcm.eduAbstract: 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.Keywords: romiplostim, immune thrombocytopenia, quality of life, patient preference