Management and outcomes of refractory immune thrombocytopenia in pediatric patients: A retrospective analysis from Ospedale Pediatrico Bambino Gesù

Immune thrombocytopenia (ITP) in pediatrics is typically self-limiting, yet a subset develops refractory ITP (rITP), which persists despite standard first-line treatments. Clinical evidence on the efficacy of second-line therapies in this condition, such as mycophenolate mofetil (MMF), rituximab (RTX) and romiplostim, remains limited. This study aims to evaluate the efficacy of second-line therapies for the management of rITP in a cohort of 33 pediatric patients treated at Ospedale Pediatrico Bambino Gesù over a 16-year period. Some of these patients switched second-line therapies due to inadequate clinical responses, thus receiving more than one treatment over the study period. Collectively, patients in our cohort received MMF (n = 20), RTX (n = 16), romiplostim (n = 15), dapsone (n = 3) and sirolimus (n = 2). Response rates were 50% (10/20) for mycophenolate mofetil (MMF), 18.8% (3/16) for rituximab, and 66.7% (10/15) for romiplostim. None of the 3 patients treated with dapsone showed clinical benefits. One of the two patient undergoing sirolimus achieved a sustained clinical response. Combination therapies showed promising results, particularly the association of MMF and romiplostim. Our results provide new insights into the management of pediatric rITP, while highlighting the need for further studies to establish evidence-based treatment guidelines and improve outcomes of this condition.