Avatrombopag for treating children and adolescents with immune thrombocytopenia: a plain language summary

Plain Language Summary: What is this summary about? This plain language summary describes the results of a phase 3 study that explored how a drug called avatrombopag worked in children and adolescents aged 1 to 17 years who had a bleeding disorder called primary immune thrombocytopenia (ITP) and whose platelet levels did not increase enough after receiving a previous treatment for ITP. What were the key takeaways? A total of 75 children and adolescents with ITP participated in this study. Overall, 28% of participants who were treated with avatrombopag had platelet levels that reached the platelet response threshold in at least 6 of 8 weeks without taking other medications for ITP, compared with 0% of participants treated with placebo. Additionally, 81% of participants reached the platelet response threshold in at least 2 consecutive weeks, compared with 0% of participants treated with placebo. Most adverse events that occurred during the study were mild or moderate, with the most common events being petechiae, nosebleed, bruising, and headache. What were the main conclusions reported by the researchers? Avatrombopag effectively and safely increased platelet count in many children and adolescents with ITP who previously had not responded well to other treatments. With administration by mouth, no restrictions on the types of food that it can be taken with, and no need for additional liver monitoring, avatrombopag is a new treatment option that overcomes some of the challenges of older therapies available for children and adolescents with ITP.