Diagnosis and treatment of paroxysmal nocturnal hemoglobinuria in children: clinical cases
https://doi.org/10.17650/2311-1267-2016-3-4-77-80
Abstract
Paroxysmal nocturnal hemoglobinuria (PNH) at children presents at children rarely in comparison with adults, but clinical presentation characterized by the same symptoms and complications like at the adult population of patients. Children present with chronic hemolysis, thrombosis, abdominal pain, infection complications, fatigue, decreasing of quality of life. In this case, the feature of PNH at children is predominant combination of diseases with bone marrow failure (most common, aplastic anemia) and relatively rare hemoglobinuria, what can complicate in-time diagnosing. Blood testing with the help of flow cytometry allows establishing diagnosis and risking groups for PNH determination presented in the international guidelines. In-time diagnostics of diseases is very important for treatment tactics chosen and prognosis, and gain the high importance with the appearance of possibility of pathogenetic therapy by eculizumab medication. Eculizumab is the humanized monoclonal antibody which inhibits the activity of complement system and prevents development of chronic intravascular hemolysis in case of PNH. Effectiveness and safety of eculizumab were proven in clinical studies both for children and adults. This article presents own experience of treatment of 2 children with PNH on the base of Uliyanovsk Regional Clinical Hospital.
About the Author
V. N. Timofeeva
Ulyanovsk Regional Children's Clinical Hospital named after a public and political figure of the Ulyanovsk region Yu.F. Goryachev;
Russian Federation
42 Radishcheva St., Ulyanovsk, 432011
Russian Federation
42 Radishcheva St., Ulyanovsk, 432011
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Review
For citations:
Timofeeva V.N. Diagnosis and treatment of paroxysmal nocturnal hemoglobinuria in children: clinical cases. Russian Journal of Pediatric Hematology and Oncology. 2016;3(4):77-80. (In Russ.) https://doi.org/10.17650/2311-1267-2016-3-4-77-80
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