Intravenous immunoglobulins (IVIg) in childhood immune thrombocytopenia: towards personalized medicine—a narrative review

Abstract

: In childhood immune thrombocytopenia (ITP), the morbidity is significant due to the risk of bleeding and a reduced health-related quality of life (HRQoL). Current management guidelines indicate that treatment is considered for children with moderate to severe bleeding, or a reduced HRQoL. Intravenous immunoglobulin (IVIg) treatment has been introduced in childhood ITP in 1981. IVIg treatment speeds up the remission of thrombocytopenia in newly diagnosed ITP and reduces bleeding symptoms, but this has the disadvantage of side effects and costs. Some data indicated that IVIg may modulate the late disease outcomes of ITP, but our recent randomized controlled trial (RCT) showed that IVIg does not affect the development of chronic ITP. Overall, 60% of children with newly diagnosed ITP show a sustained response to IVIg. Such a sustained response after IVIg treatment is strongly associated with long-term remission from ITP. Recent molecular and clinical data show that treatment responders could be identified before IVIg is administered. The same molecular and clinical characteristics that identify treatment responders are otherwise associated with a transient, self-limiting ITP disease course, suggesting the identification of a distinct subgroup of ITP patients. Thus, the development of multivariate clinical and molecular prediction scores could allow to individualize treatment decisions, i.e., target IVIg to those who benefit the most. These prediction scores may also be used to design studies aimed at identifying children who benefit from adjunctive or alternative treatments, such as thrombopoietin receptor agonists (TPO-Ra).