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Review
. 2024 Sep:67:101222.
doi: 10.1016/j.blre.2024.101222. Epub 2024 Jun 20.

Immune thrombocytopenia: Pathophysiology and impacts of Romiplostim treatment

John W Semple  1 , Alexandra Schifferli  2 , Nichola Cooper  3 , Hossam Saad  4 , Daniel T Mytych  4 , Lynette S Chea  5 , Adrian Newland  6
Affiliations
Free article
Review

Immune thrombocytopenia: Pathophysiology and impacts of Romiplostim treatment

John W Semple et al. Blood Rev. 2024 Sep.
Free article

Abstract

Immune thrombocytopenia (ITP) is an autoimmune bleeding disease caused by immune-mediated platelet destruction and decreased platelet production. ITP is characterized by an isolated thrombocytopenia (<100 × 109/L) and increased risk of bleeding. The disease has a complex pathophysiology wherein immune tolerance breakdown leads to platelet and megakaryocyte destruction. Therapeutics such as corticosteroids, intravenous immunoglobulins (IVIg), rituximab, and thrombopoietin receptor agonists (TPO-RAs) aim to increase platelet counts to prevent hemorrhage and increase quality of life. TPO-RAs act via stimulation of TPO receptors on megakaryocytes to directly stimulate platelet production. Romiplostim is a TPO-RA that has become a mainstay in the treatment of ITP. Treatment significantly increases megakaryocyte maturation and growth leading to improved platelet production and it has recently been shown to have additional immunomodulatory effects in treated patients. This review will highlight the complex pathophysiology of ITP and discuss the usage of Romiplostim in ITP and its ability to potentially immunomodulate autoimmunity.

Keywords: Immune thrombocytopenia (ITP); Immunomodulation; Pathophysiology; Remission; Romiplostim; Therapy free remission.

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Conflict of interest statement

Declaration of competing interest H Saad, DT Mytych, and LS Chea are employees and stockholders of Amgen. JW Semple has received and participated in Amgen Honouraria and Ad boards; Argenx Honouraria; CellPhire Therapeutics Research funding; Ionis Research funding; Novartis Grants, Honouraria, Ad boards; Sobi Honouraria; Takeda Honouraria; UCB Honouraria and Ad boards. A Schifferli has received and participated in Novartis Honoraria, Research Funding and Grant; Sobi Honoraria; Platelet Disorder Support Association (PDSA) grant recipient. N Cooper has received honoraria for speaking engagements and participated in advisory boards with Amgen, Novartis, Principia, and Rigel; and has received support for clinical trials from Amgen, Novartis, Rigel, Principia, and UCB. A Newland has acted as a consultant for Amgen, Angle, Argenx, Grifols, GSK, Novartis, and UCB Biosciences; he has also participated in advisory boards and/or as a speaker at medical education events sponsored by Amgen, Argenx, Grifols, GSK, Novartis and Roche; and, finally, he has received research support from Amgen, BMS, GSK, Novartis, and Octapharma.

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