Experience of protein C administration in children with acquired deficiency

Abstract

Тhere is increasing experience of protein C concentrate administration in world practice, but despite that, information of this drug administration in patients with oncohematological diseases and primary immunodeficiency syndromes is lacking. Objective: to study the effectiveness of protein C concentrate administration in pediatric patients with acquired protein C deficiency during the treatment of oncological, hematological or immunological diseases. Medical charts of 12 patients who received inpatient treatment and protein C concentrate administration in the Dmitry Rogachev National Clinical Research Center from 01/01/2012–12/31/18 were analyzed. Depending on the presence or absence of thrombosis, the patients were divided into two groups. Single and daily doses, the number of injections per day, the duration of therapy and the percentage of activity of protein C activity were studied in both groups. Вoth groups included 6 patients, median of a single administrated dose of protein C was lower in the group of patients with thrombosis than in patients without them (20 and 71.4 IU/kg, p < 0.0001), while there were obtained no differences between treatment efficacy (p = 0.45). When comparing the administered dose of the drug in children with unresolved and resolved thrombosis, it was found that the median single dose in patients with ineffective treatment was lower than in those who had effective treatment (8.78 and 71.4 IU/kg, respectively, p < 0.0001); the median daily dose was also lower in the group with ineffective treatment (20 and 71.4 IU/kg, respectively, p < 0.005). Рrotein C administration in children with acquired deficiency for the purpose of antithrombotic prophylaxis can be potentially effective, especially in those patients who already have a thrombosis at the moment of administration. The effectiveness of such prophylaxis may depend on the dose of the injected concentrate. To determine the appropriate dose and mode of administration of the drug in children a prospective study is required. The study was approved by the Independent Ethics Committee of the Dmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology, and Immunology.