Effectiveness and safety of eltrombopag in the first-line therapy of
severe aplastic anemia in children
Abstract
Background: Eltrombopag (E-PAG) is being investigated for the treatment
of aplastic anemia (AA) by stimulating hematopoietic stem cell (HSC)
proliferation. Objective: To evaluate the effectiveness and safety of
E-PAG in first-line therapy for pediatric AA. Methods: The present
retrospective study reviewed the pediatric patients with newly diagnosed
AA in immunosuppressive therapy (IST) therapy with E-PAG at the single
center from March to September in 2017. All patients were followed up
for >2 years. Results: A total of 14 patients (8 males),
aged 86 months, were enrolled in this study. E-PAG was administered with
a median time to initiation of 19.5 days after IST, and the median
course of treatment was 253 days. The rate of complete response and
overall response at 6 months were 64.3% (9/14 case) and 78.6% (11/14
cases) respectively. The survival rate was 100%, and no relapses
occurred in responders. E-PAG was well-tolerated; however, the most
common adverse events included indirect bilirubin elevation, jaundice,
and transient liver-enzyme elevation. By the end of follow-up, bone
marrow chromosomes were normal, and no abnormal myelodysplastic
syndromes (MDS) -related clones appeared. Conclusions: The addition of
E-PAG to IST was associated with the markedly increased complete
response with respect to hematology in pediatric patients with SAA than
in a historical cohort without the unacceptable side effects.