Background Children with chronic refractory Immune Thrombocytopenia are a small, but demanding group, representing 5% of the total cases of Immune thrombocytopenia. Changing the nomenclature itself, from idiopathic to immune, is evidence of the increasing awareness and improvements in the management of immune thrombocytopenia. Treatment for this disease is always a challenge taking into consideration that the few known therapies have significant side effects and toxicities.
Aim To evaluate treatment strategies and the effect on heterogenous evolution of children with chronic Immune Thrombocytopenia.
Methods Chronic refractory Immune Thrombocytopenia is defined as disease that does not respond to or relapses after first 2 lines of treatment, with over one year of evolution. Based on 8 cases within our clinic, we discuss treatment options available for children with chronic refractory Immune Thrombocytopenia. In addition to detailed history and physical examination, there were analized full blood count, review of the peripheral blood smear, routine tests of hemostasis and bone marrow examination was performed to search for myelodysplastic syndromes. At the same time, infections (CMV,EBV, HIV, HP) and autoimmune disorders (LES, APS) were ruled out. All patients who failed prior treatment, meaning first 2 lines of treatment (multiple steroids, intravenous immunoglobulins, splenectomy) underwent treatment with molecule targeting therapy (Rituximab), nonspecific immunosuppresive agents (Cyclosporin, Mycophenolat Mofetil, Sirolimus) or thrombopoietin receptor agonists.
Results A total of 8 patients with chronic refractory Immune Thrombocytopenia were analised. The median age at diagnosis was 6,5 and sex ratio 1:1. Disease evolution is between 3 and 10 years. Treatment for the studied cases consisted of multiple courses of steroids and intravenous immunoglobulins (all patients), splenectomy (4 patient), Rituximab (4 patients), Cyclosporin (6 patients), Mycophenolat Mofetil (6 patients) and Sirolimus (3 patients). 2 patients underwent therapy with thrombopoietin receptor agonist (Romiplostin), with only one favourable response. At present time, 6 patients are receiving combined immunosupressive therapy and steroids, while 2 are stable with one immunosupressive agent. In spite of abnormal platelet count, for these patients is most significant to have no risk factors nor haemorrhagic events. Among immunosupressive agents, early relapse was frequently seen after administration of Cyclosporin, compared to other immunosupressive agents.
Conclusions The goal of long-term immunosupressive treatment strategies for Immune Thrombocytopenia is to achieve a platelet count that is associated with adequate hemostasis, rather than normal platelet count. As outcomes vary from one patient to another, aspects regarding age, clinical features, bleeding risks, likely side-effects, economic considerations, life quality as well as the physicians concerns are essential.
- chronic refractory immune thrombocytopenia
- immunosupressive treatment
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