Advances in chemical pharmacotherapy for the treatment of pediatric immune thrombocytopenia

ABSTRACT

Introduction: Immune thrombocytopenia (ITP) is an autoimmune bleeding disorder of heterogeneous pathophysiological mechanisms. Treatment endpoints include elevation of platelets and reduction of bleeding risk, elevation of quality of life, reduction of concomitant therapies and prevention from bleeding. Persistent and chronic ITP is more common in adults but occurs in children. Standard therapies include corticosteroids and immunoglobulins, both associated with side effects. There are new treatments, such as thrombopoietin-receptor agonists and promising investigational drugs.

Areas covered: Experience from the management of adults is valuable for children with persistent and chronic symptomatic ITP. In this review first- and second-line therapies, but also investigational drugs for children with ITP are discussed.

Expert opinion: Although time-consuming and based on experience, children with no or mild bleeding can be safely managed with a watch and wait strategy. Chronic symptomatic immune thrombocytopenia is an area of second-line treatments based on a highly individualized approach. Furthermore, there are investigational drugs, which may also be of benefit for children with chronic symptomatic ITP.

KEYWORDS:

• Drugs
• platelets
• immune thrombocytopenia
• thrombopoietin-receptor agonists
• investigational drugs

Article highlights

• Primary ITP is a diagnosis of exclusion without availability of specific tests. Continuous differential diagnostic considerations characterize an adequate management of patients with ITP.

• The heterogeneous background of ITP explains the response variety to treatments, making standardization of diagnostic procedures and therapeutic strategies difficult. The approach to a patient with ITP remains individual, in spite of practice guidelines and algorithms.

• ITP of children is often self-limited and does not need treatment. The outcome is similar, whether corticosteroids or immunoglobulins or no drugs are given at first presentation, particularly in children younger than 10 years and in those presenting with an abrupt onset of bleeding lasting less than 2 weeks.

• A small subgroup of children with chronic symptomatic ITP needs treatment. For these children second-line treatments are available, most of them with experience in adults, however, there is growing pediatric information for thrombopoietin-receptor agonists and for rituximab.

• There are new drugs investigated in adult patients with chronic ITP with high potential to achieve licensure. These drugs together with second-line treatments have the potential to defer or avoid splenectomy.

• There are discussions and attempts to avoid corticosteroids and to revisit first-line treatments.

This box summarizes key points contained in the article.

Declaration of Interest

T Kühne has received research funds from Amgen Inc and is a member of an advisory board of UCB Celltech. He 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. Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This manuscript has not been funded.