Multidrug-Refractory Immune Thrombocytopenia in a Pediatric: A First-in-Literature Case Report and Therapeutic Challenge

Immune thrombocytopenia (ITP) is a common autoimmune disorder in children characterized by isolated thrombocytopenia. While most pediatric cases resolve spontaneously, a subset progresses to refractory ITP, defined by the failure to respond to at least two lines of standard therapy. We report the case of a 15-month-old male with severe, treatment-refractory ITP. Despite sequential administration of six major therapeutic agents – intravenous immunoglobulin, high-dose corticosteroids, mycophenolate mofetil, rituximab, vincristine, and eltrombopag – the patient exhibited persistent thrombocytopenia without durable platelet recovery. A comprehensive evaluation excluded secondary causes, and all treatments were well tolerated, with confirmed adherence. The patient remains under close monitoring with consideration of novel immunosuppressive agents, including sirolimus and daratumumab. This case underscores the limitations of current therapeutic approaches in multidrug-refractory pediatric ITP and highlights key learning points, the value of early genetic testing to exclude inherited thrombocytopenias, and the need for biomarker-driven, individualized treatment strategies to optimize outcomes in severe refractory cases.