Romiplostim for the treatment of primary immune thrombocytopenia

Abstract

Romiplostim is a peptibody designed to evade the immune response to recombinant thrombopoietin and to stimulate the production of new platelets. The pathophysiology underlying primary immune thrombocytopenia (ITP) has shown that not only accelerated peripheral platelet destruction but also suppression of the production of new platelets can be responsible for the persistence of thrombocytopenia. Several prospective and well-designed trials have shown that weekly subcutaneous administration of romiplostim induces more durable platelet responses and less treatment failure compared with placebo or standard of care, not only in adult but also pediatric chronic ITP patients regardless of splenectomy status. Treatment with romiplostim also resulted in less use of rescue medication and permitted the majority of patients receiving concurrent ITP drugs to reduce or discontinue these therapies, avoiding further immunosuppression. Romiplostim has been shown to have overall a very favorable safety profile and to be well tolerated relative to other ITP treatments.

Keywords::

• efficacy
• guidelines
• immune thrombocytopenia
• primary immune thrombocytopenia
• recommendations
• romiplostim
• safety
• thrombopoietic receptor agonist
• treatment

Finanacial & competing interests disclosure

A Janssens has received consultancy fees and honoraria from Amgen and GlaxoSmithKline. The author has 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.

No writing assistance was utilized in the production of this manuscript.