Eltrombopag shown to be effective in children with ITP

9 October 2015  •  Author: Victoria White

A University of Manchester-led team of researchers have demonstrated the relative safety and effectiveness of eltrombopag (Promacta, Novartis) in children with persistent or chronic immune thrombocytopenia (ITP).


ITP is an autoimmune disorder where the immune system attacks platelets and blood fails to clot as it should. Four in every 100,000 children develop the disorder each year globally. The symptoms of ITP include bleeding and bruising more easily. Frequent nose bleeds and bleeding from the gums can be common, and bruising often appears as purple patches or tiny red spots on the skin. On rare occasions bleeding can be life threatening. The condition may resolve by itself, but for one in every four of the affected children, the condition becomes chronic persisting after primary intervention and lasting for more than twelve months.

Eltrombopag consistently stabilised the platelet count for 40% of children receiving the treatment

Historically, second-line treatment options for children with ITP have been scarce and, one of the earliest options, surgical removal of the spleen (splenectomy) was associated with a high risk of sepsis and thrombosis. A better understanding about the underlying cause of ITP led to the development of the use of newer immunosuppressant agents, including rituximab. More recently, thrombopoietin receptor agonists, like eltrombopag, have been approved for the treatment of thrombocytopenia in adult patients with chronic ITP who have had an inadequate response or are intolerant to other treatments.

Chief investigator Dr John Grainger, Consultant Paediatric Haematologist and Honorary Lecturer at The University of Manchester, explained, “The studies, funded by GlaxoSmithKline, provide clinicians with much needed evidence to help decide when eltrombopag would benefit paediatric patients and provide dosage regimens suitable for paediatric patients.

“The studies demonstrate that eltrombopag is well tolerated and effective, consistently stabilising the platelet count to over 50 X 109 per litre within 2-6 weeks for 40% of children receiving the treatment, compared with 0 per cent on the placebo arm.”

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