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

Authors Gilbert MM, Grimes AB, Kim TO, Despotovic JM

Received 28 December 2019

Accepted for publication 28 April 2020

Published 22 July 2020 Volume 2020:14 Pages 1237—1250


Checked for plagiarism Yes

Review by Single anonymous peer review

Peer reviewer comments 2

Editor who approved publication: Dr Johnny Chen

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, USA

Correspondence: Megan M Gilbert
Department of Pediatrics, Section of Hematology/Oncology, Baylor College of Medicine, Texas Children’s Hospital, 6701 Fannin Suite 1510, Houston, TX 77030, USA
Tel +1 (832) 824-4736
Fax +1 (832) 825-4846

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.

Keywords: romiplostim, immune thrombocytopenia, quality of life, patient preference

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