Эффективность и безопасность длительной терапии пегилированным ин-терфероном α-2а истинной полицитемии у ребенка

Data on the clinical course and treatment of polycythemia vera (PV) in children are extremely limited; the results of any treatment are currently unknown. Authors represent a clinical case report of a PV pediatric patient who has been receiving long-term cytoreductive therapy with pegylated interferon α-2a (PEG-IFN-α2a): 14 y/o male, first visit to the National Scientific and Practical Center for Pediatric Hematology, Oncology and Immunology named after Dmitry Rogachev (Moscow, Russia) in Apr., 2017 at the age of 8 y/o with pronounced changes in the hemogram. In the general blood test: an increase in hemoglobin - 175 g/l, hematocrit - 50.3%, erythrocytosis - up to 6.99x1012/l, other indicators were normal. Molecular genetic examination of bone marrow using high-throughput sequencing revealed a somatic mutation of the JAK2 gene c.1849G>T, p.Val617Phe with an allelic load of 30.23%. Based on the examination results, the patient was diagnosed with PV. PEG-IFN-α2a therapy was initiated. In total, the patient has been receiving the therapy for 7 years; according to the results of control examinations, a persistent partial clinical, hematological and molecular response is maintained throughout the treatment period. Mild adverse events were observed against the background of the therapy: grade 2 neutropenia, grade 1 increase in liver transaminases according to CTCAE v5.0. Conclusion: the use of the PEG-IFN-α2a effectively normalizes the clinical and hematological manifestations of PV in children with no significant adverse events of its use recorded so far.