Alternatives for managing patients with newly diagnosed immune thrombocytopenia: a narrative review

ABSTRACT

Introduction

Primary immune thrombocytopenia (ITP) is an acquired bleeding disorder. Conventionally, first-line ITP therapy aims to obtain a rapid response and stop or decrease the risk of bleeding by increasing the platelet count. At this point, the duration of the response, the tolerability, and the long-term safety of pharmacologic interventions are considered less of a priority. Combination treatments that simultaneously address multiple disease mechanisms are an attractive strategy to increase efficacy in acute ITP therapy. In this review, we discuss the treatment of newly diagnosed ITP patients, emphasizing the use of new combinations to benefit from their synergy.

Areas covered

This article summarizes conventional treatment, recent and novel combinations, and COVID-19 management recommendations of newly diagnosed ITP patients.

Expert opinion

The key areas for improvement consider the long-term effects of conventional first-line therapy, reducing relapse rates, and extending responses to achieve long-term remission. Although corticosteroids remain a first-line therapy, restricting their use to avoid toxicity and the increasing use of rituximab and TPO-RAs in the first three months after diagnosis open the landscape for future interventions in frontline therapy for ITP. First-line therapy intensification or synergistic drug combination offers a potential and realistic shift in future treatment guidelines.

KEYWORDS:

• ITP
• management
• first-line therapy
• combination therapies
• newly diagnosed
• narrative review

Article highlights

• Most adults with immune thrombocytopenia who receive current standard therapy will relapse or develop chronic disease.

• Guidelines still suggest corticosteroids as a first-line treatment with intravenous immunoglobulin when a rapid response is needed.

• Combination therapy is appealing in newly diagnosed patients with drugs efficacious in chronic or relapsed ITP.

• Single-arm, phase 2 and phase 3 randomized studies have shown that treatment efficacy can improve with first-line therapy combinations, combining corticosteroids with TPO-RAs, rituximab, ATRA, MMF, and oseltamivir.

Acknowledgments

We thank Sergio Lozano-Rodríguez for his critical review and editor assistance of the article.

Declaration of Interest

D Gómez-Almaguer declares speaker and/or advisory (Honoraria) for Celgene, Amgen, Novartis, Takeda, Abbvie, Pfizer, Roché, Teva, and BMS. A Gómez-De León declares advisory for BMS (Honoraria) and Abbvie. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the 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 paper was not funded.