Current and emerging treatments for immune thrombocytopenia

ABSTRACT

Introduction: Immune thrombocytopenia (ITP) is an autoimmune disease. Even though there are many treatments available, some patients remain resistant to multiple treatments. Therefore, it is very important to develop new treatment options.

Areas covered: Here, the authors summarize several current and emerging treatments developed for ITP in recent years. They include a summary of their mechanisms of action and clinical trial results.

Expert opinion: At present, the first-line treatment of ITP is glucocorticoid and intravenous immunoglobulin (IVIg). Other traditional therapies include splenectomy, thrombopoietin (TPO), rituximab and other immunosuppressive agents. The several emerging treatments developed recently for ITP may change the treatment pattern in the future.

KEYWORDS:

• Immune thrombocytopenia
• TPO-RA
• eltrombopag
• romiplostim
• avatrombopag
• Syk inhibitor
• fostamatinib
• FcRn
• monoclonal antibody
• platelet desialization
• atorvastatin

Article highlights

• The current and emerging therapeutics for ITP mainly include suppressing immune response-mediated platelet destruction, promoting platelet production, and regulating the microenvironment of bone marrow.

• TPO-RA, including eltrombopag, romiplostim, and avatrombopag, has a good efficiency and tolerable AEs in ITP patients. Avatrombopag is a newly developed TPO-RA, which is taken orally with no dietary limits and has a similar biological activity to eltrombopag and romiplostim.

• Fostamatinib, as the Syk inhibitor, has been approved by the FDA for the treatment of adult chronic ITP and may become a new option for chronic, refractory ITP patients.

• Blockage of co-stimulatory molecules, including anti‐CD154 monoclonal antibody, CD44 antibody, and CTLA4‐Ig, may play the role of immunomodulation to treat ITP as well as other autoimmune diseases.

• Oseltamivir phosphate may inhibit platelet desialization to reduce clear of platelets in liver.

• Atorvastatin may have the potential to treat ITP because of effects on BM EPCs.

Acknowledgments

The authors would like to thank Professor. Man-Chiu Poon (University of Calgary, Canada) for critical review the manuscript.

Declaration of interest

The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or conflict with the subject matter or materials discussed in this manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This project was supported in part by grants of National Natural Science Foundation of China (81670118, 81470286), CAMS Innovation Fund for Medical Sciences (CIFMS) (2016-I2M-1-002) and Tianjin Key Project for Basic Research (18JCZDJC35000).