Eltrombopag w leczeniu przewlekłej małopłytkowości immunologicznej u pacjentów pediatrycznych – doświadczenia ośrodka

*Katarzyna Koch, Paweł Łaguna, Anna Adamowicz-Salach, Marek Karwacki, Anna Klukowska, Izabela Romanowska, Ewa Celi, Michał Matysiak

Summary
Introduction. Immune thrombocytopenia (ITP) is an autoimmune disease that results in a reduction of blood platelets <100 X 109/l. Immune thrombocytopenia for over 12 months is defined as chronic ITP. The introduction of a drug program to chronic thrombocytopenia with the use of thrombopoietin receptor agonists (eltrombopag) is associated with high hopes of achieving lasting remission of the disease and increasing the chances of avoiding splenectomy.
Aim of the study. Evaluation of the effectiveness of eltrombopag is increasing the platelet count in patients with cITP treated in the Department of Pediatric Hematology and Oncology of the Medical University of Warsaw.
Materials and methods. The study group consisted of 6 patients (6 girls and 4 boys) aged 4 to 12 years, with cITP. The patients' qualification for the examination and further treatment were realized in accordance with the criteria of the B.98 drug program „Eltrombopag in the treatment of pediatric patients with cITP”. The first of the reported patients was treated with eltrombopag from September 2016 to December 2016, before the drug program was created.
Results. All patients responded to eltrombopag treatment. One of the patients has already completed therapy and the remaining patients are still treated according to the B.98 drug program. Two patients receive eltrombopag in monotherapy, three patients require additional immunosuppressive drugs. During treatment, three patients experienced a decrease in the number of platelets requiring the use of immunoglobulin infusions or pulses from a methylprednisolone. During the therapy no hepatotoxicity or development of cataract was observed.

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