Combination treatment for resistant and refractory immune thrombocytopenia in children: a literature review and seven clinical cases

Introduction. Immune thrombocytopenia (ITP) is characterized by increased platelet destruction and suboptimal platelet production, which results in reduced platelet counts in peripheral blood and bleeding of varying severity. In children with ITP, remission is often achieved with first-line therapy or even spontaneously. Thrombopoietin receptor agonists and rituximab are considered modern therapies of choice in severe and/or chronic course of the disease, splenectomy is performed less frequently. Despite the favorable prognosis, some patients may exhibit resistance to initial therapy and a small proportion of patients fail to respond to several courses of first- and/or second-line ITP-directed therapies, thus developing refractory ITP. The management of patients with resistant and refractory ITP is a clinical challenge. Clinical cases. The article presents seven clinical cases of severe ITP of varying duration. The patients aged 1.5 to 17 years received combination therapy due to unsatisfactory therapeutic response in order to prevent severe hemorrhagic complications. The article describes the clinical presentation and different therapeutic approaches, discusses modern possibilities and combined treatment regimens for resistant and refractory ITP in children. Conclusion. Overall, current studies of pathogenesis and the development of guidelines for managing ITP patients aim to optimize treatment, particularly for resistant, refractory, severe persistent and chronic forms of the disease, reduce the risk of bleeding, increase the likelihood of achieving long-term remission, and improve the quality of life of patients and their families.