Abstract
We report 2 cases of infants with acute and persistent Immune Thrombocytopenic Purpura (ITP) treated with Eltrombopag. Since ITP is rare in infants, robust evidence about how to treat these patients is not available. Both children underwent multiple lines of treatment without success and were successfully managed with off-label use of Eltrombopag. We did not observe any of the reported adverse effects of the drug and complete remission was achieved in both cases. In one child, we were able to discontinue treatment without any ITP relapse. This is the first report of an off-label use of Eltrombopag in infants.
IntroductionImmune Thrombocytopenia Purpura (ITP) is an immune-mediated disorder characterized by isolated thrombocytopenia (platelet count <100.000/microL), with or without bleeding signs in an otherwise healthy patient [1]. It is one of the most common acquired bleeding disorders (incidence: 5-10/100.000 children/year) [2]. ITP can be classified as acute, when it is within three months from the onset; persistent, when the disease was diagnosed in the time interval between the previous 3 months and 1 year; and chronic, when it persists longer than one year. A large case series of children with ITP from the registry of the Intercontinental Childhood ITP Study Group showed that the vast majority of patients (69.1%) had 1 to 10 years of age, only a small percentage of patients (7.6%) were younger than 1 year [3]. Since ITP rarely presents in children younger than 1-year, robust evidence about how infants should be managed is scarce. In the pediatric setting, multiple factors besides the mere platelet count must be evaluated to decide whether to treat the patient. Thus, scoring the clinical severity through objective bleeding risk scales helps decide between a “watch and wait” approach or a pharmacological intervention. Regarding children with persistent/chronic ITP, a “watch-and-wait” approach is frequently chosen, and rescue therapies can be administered. Nevertheless, living with a low platelet count can severely impact the quality of life (QoL) of children and their families[4]; in these cases an alternative strategy should be considered, such as Thrombopoietin-Receptor Agonists (TPO-RAs)[5].
TPO-RAs stimulate platelet production and can influence platelet antibody production and T regulatory cell count [6–8]. Currently two TPO-RAs, Eltrombopag and Romiplostim, have been licensed for children > 1-year-old with ITP lasting longer than 6 months with unsatisfactory response to standard therapies [7].
Here we report two infants presenting, respectively, with acute and persistent ITP and who were both managed with off-label use of Eltrombopag.