In the present study, we investigated the long-term efficacy of eltrombopag in pediatric patients with ITP. Our results suggest that the OR rate, CR rate, R rate, DR rate, NR rate, TFR, and relapse rate were 67.0%, 55.3%, 11.7%, 56.3%, 33.0%, 60% and 36.2%, respectively. Furthermore, newly diagnosed ITP patients showed a higher DR rate, R, TFR rate and lower relapse rate compared to those with persistent and chronic ITP patients. Of note, the CR rate, DR rate, and TFR rate of 5 patients under six months were all 100% without suffering from relapse. The most common adverse event was an elevation in transaminase levels occurring in approximately 7.77% of cases.
Eltrombopag is currently widely adopted as a second-line therapy for ITP. Eltrombopag stimulates thrombopoiesis and is considered suitable for use in in pediatric patients [21]. In our study, 67.0% of patients achieved OR, 55.3% of patients achieved CR, 11.7% of patients achieved R, which is consistent with previously reported for children with ITP [22].Although, our OR rate is lower than what has been reported in the children with chronic ITP [18].During the follow-up period, we observed a considerable number of parents had high expectations for the platelet count, leading to the poor compliance. Three children discontinued the treatment at 1 week and four children at 2 weeks. Therefore, it is important to enhance the parents’ awareness of the purpose of ITP treatment in the future. Additionally, it may be related with the sample size, as well as some parents discontinuing eltrombopag treatment due to its high cost.
Previous studies indicated that eltrombopag plays a disease-modifying role rather than serving as a temporary measure to transiently increase the platelet count[18]. It has been reported that eltrombopag can obtain TRF in adult patients without relapse after discontinuation of therapy [23]. In our study, TFR in pediatric patients with ITP was higher than the reported in adults[23]. These results indicated that long-term treatment with eltrombopag had good effective on pediatric patients. Furthermore, our results demonstrated that newly diagnosed patients with ITP had higher DR rate, R, TFR rate, while displaying a lower relapse rate compared to those with persistent and chronic ITP. Notably, children under the age of 3 demonstrated higher ORR, DR rate, and TFR rate along with a lower relapse rate, but there were no significant. Our results showed baseline platelet counts were not associated with response.
Due to limited data regarding the application of eltrombopag in the youngest children, we pay more attention to its efficacy and safety on them. Interestingly, the efficacy of eltrombopag in the treatment of youngest children is encouraging. the CR rate, DR rate, and TFR rate of five patients under six months old were all 100%. They weren’t suffered relapse and had fewer adverse events. Therefore, the youngest children may benefit the most from the eltrombopag treatment.
Previous study has indicated that the adverse effects of eltrombopag were headache, hepatotoxicity, vomiting, hair loss, rash, stomachache, bone marrow fibrosis, and thrombosis [24]. It is reported the most common Adverse Events were headache (7%) and thrombocytosis (6%)[25]. However, our results indicated that the most frequently adverse effect was hepatotoxicity, but it wasn’t severe. Additionally, some patients were suffered from headache, vomiting, hair loss, rash and stomachache (Table 4). However, bone marrow fibrosis and thrombosis weren’t observed during eltrombopag therapy.