Abstract
Background
Currently, there is no consensus on the treatment of persistent and chronic primary immune thrombocytopenia (ITP) in children. This systematic review evaluates the safety and efficacy of current therapeutic options.
Methods
A comprehensive literature search was performed across MEDLINE/PubMed, Elsevier databases (EMBASE, Scopus), Web of Science (WoS; core collection), and Cochrane (CENTRAL). We included articles published between 1995 and 2026, reporting on safety and/or efficacy of therapies in children and adolescents (<18 years) with persistent or chronic primary ITP.
Results
Ninety-four studies were included. Watchful waiting resulted in a complete remission rate of 14-76%. Of note, thrombopoietin receptor agonists demonstrated the best efficacy/safety balance: acceptable and durable response rates (40-78%) with favourable safety profiles. Splenectomy also yields high complete response rates, but carries a risk of life-threatening complications. Besides, rituximab, sirolimus and mycophenolate mofetil show good efficacy but safety data are scarce. In contrast, first-line therapies (corticosteroids, IVIG and anti-D) provide only short-term benefit whereas cyclosporine A, dapsone and interferon-alpha demonstrate inconsistent efficacy in addition to relevant side effects.
Conclusion
The choice of therapeutic options in paediatric persistent and chronic ITP is extensive. While watchful waiting may be appropriate for many patients, thrombopoietin receptor agonists emerge as the preferred option when pharmacological treatment is indicated. Long-term safety evaluation and comparative studies focusing on patient-centred outcomes remain warranted.