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Could eltrombopag and romiplostim play a role in treatment of pediatric ITP?
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Yes, potentially in some pediatric patients
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The answer is yes. There has been a pediatric trial of romiplostim, and we were one of the participating institutions. However, the study was very small and will not lead to an application for pediatric licensing.
Theoretically, there would be value in using these two new agents in children with ITP. First, they could have potential in those pediatric patients with chronic refractory ITP who are similar to adults for whom the drugs have been licensed; there are definitely children in that same category.
But these agents could also potentially be used in patients whose ITP is not chronic. For example, they could be employed in a child who was recently diagnosed, but two or three months later continues to bleed a great deal. To be able to have an oral agent or a subcutaneous injection that might temporarily increase their platelet count and prevent bleeding until their ITP resolved could be beneficial in certain patients, assuming there is continued scrutiny of the agents as far as their safety.
Despite this, there is some downside to these drugs. They are outrageously expensive and there still is not much known about long-term side effects.
George Buchanan, MD, is a Professor of Pediatrics at UT Southwestern Medical Center.
Yes, especially in adolescents
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There is potential to use these drugs in pediatric patients. Older adolescents are really just like young adults; they are biologically the same. Really severe ITP in a 16- or 17-year-old probably is not any different from ITP in a 19-, 20- or 21-year-old. So if other modalities aren’t working in these patients, those are ‘pediatric’ patients who are probably going to get these TPO-receptor mimetics first.
There has been at least one pediatric study for romiplostim and studies for eltrombopag are either in the planning stages or already open. As more studies come along, it will be easier to think about treating younger kids.
The drugs will be very expensive, and long-term experience is limited, so at least at first they should be used only in the most refractory patients or in patients who have unacceptable toxicities to other treatment regimens. I’m almost sure that is the right niche for these drugs.
In fact, that is what we have done here. We have some patients either with ITP or Evans syndrome who have either had toxicities to other therapies or bleeding despite other therapies and those are the people getting treated with these drugs.
However, cost is one of the concerns with these new drugs. The drugs only work as long as they are given and many patients have rebound thrombocytopenia when the drugs are stopped. It is not clear how to pick a duration of therapy. There are also other disadvantages: Eltrombopag has to be given with no food intake for a couple hours before and after taking the drug, so that would limit its use in young children. Romiplostim is parenteral, which at the very least, makes it a nuisance.
Ellis Neufeld, MD, PhD, is Associate Chief, Division of Hematology/Oncology and Associate Professor of Pediatrics at the Children’s Hospital of Boston and an Associate Professor at Harvard Medical School.