Credit: Logan Tuttle
MILAN—Eltrombopag can elicit consistent platelet responses in children with immune thrombocytopenia (ITP), according to research presented at the 19th Congress of the European Hematology Association (EHA).
Results of the phase 3 PETIT2 study showed that eltrombopag can significantly improve platelet counts in pediatric ITP patients, when compared to placebo.
In fact, the drug enabled 61% of children to stop taking or reduce the dose of their other ITP medication.
John D. Grainger, MD, of the Royal Manchester Children’s Hospital in the UK, reported these results at the EHA Congress as abstract S732. The research was sponsored by GlaxoSmithKline, the makers of eltrombopag.
The PETIT2 trial, the largest study to date of pediatric patients with ITP, was conducted to establish eltrombopag’s efficacy, safety, and tolerability in children. The drug is not yet approved anywhere in the world for use in children.
The study included 92 children from 38 centers in 14 countries. All patients were 18 years old or younger, had chronic ITP for at least 12 months, had platelet counts less than 30 Gi/L, and had failed at least 1 prior treatment.
The researchers conducted the trial in 2 parts. The first lasted 13 weeks and randomized participants to receive either eltrombopag or placebo. The second phase lasted through week 24, and all participants received eltrombopag.
The primary endpoint was an increase in platelet count to 50 Gi/L or more. And eltrombopag met this endpoint, with a statistically significant improvement in platelet counts.
Almost 40% of patients maintained a consistent platelet count for 6 of 8 weeks, compared to 3% of patients who received placebo (P<0.001).
Fifty percent of eltrombopag-treated patients experienced reduced bleeding by week 12, and 66% did so by the end of the study.
Sixty-one percent of eltrombopag-treated patients were able to stop or reduce the other ITP medications they were taking.
These results were consistent across the ages enrolled.
The investigators did not observe any new safety concerns related to eltrombopag. The most common adverse events, which occurred more frequently in the eltrombopag-treated patients, were nasopharyngitis, rhinitis, cough, and respiratory tract infection.
Almost 13% of the eltrombopag-treated patients and 10% of the placebo-treated patients experienced grade 3/4 adverse events. Eight percent of eltrombopag-treated patients and 14% of placebo-treated patients had serious adverse events.
Four children discontinued the study due to a lack of response, and 5 children had abnormal liver tests that returned to normal after stopping the drug.
Given these results, the investigators concluded that eltrombopag has the potential to treat childhood ITP.
Eltrombopag is marketed as Promacta in the United States and Revolade in the European Union.
