nodgoРоссийский журнал детской гематологии и онкологии (РЖДГиО)Russian Journal of Pediatric Hematology and Oncology2311-12672413-5496LTD “Graphica”10.17650/2311-1267-2018-5-3-89-94nodgo-411Research ArticleОБЗОРЫ ЛИТЕРАТУРЫLITERATURE REVIEWSИндивидуализированный подход к профилактической терапии пациентов с тяжелой гемофилией АIndividualized approach to profilactic treatment in patients with severe hemophilia AЗозуляН. И.ZozulyaN. I.

Надежда Ивановна Зозуля.

125167, Москва, Новый Зыковский проезд, 4

4 Novyi Zykovskiy proezd, Moscow, 125167

zozulya.n@blood.ruРумянцевА. Г.RumyantsevA. G.

117997, Москва, ул. Саморы Машела, 1

1 Samory Mashela St., Moscow, 117997

ФГБУ «НМИЦ гематологии» Минздрава РоссииРоссияNational Medical Research Center for Hematology, Ministry of Health of RussiaRussian FederationФГБУ «НМИЦ ДГОИ им. Дмитрия Рогачева» Минздрава РоссииРоссияDmitry Rogachev National Medical Research Center of Pediatric Hematology, Oncology and Immunology, Ministry of Health of RussiaRussian Federation201816092018538894Copyright © Зозуля Н.И., Румянцев А.Г., 20182018Зозуля Н.И., Румянцев А.Г.Zozulya N.I., Rumyantsev A.G.Данная работа распространяется под лицензией Creative Commons Attribution 4.0.This work is licensed under a Creative Commons Attribution 4.0 License.https://journal.nodgo.org/jour/article/view/411

Пациентам с тяжелой и среднетяжелой формой гемофилии А традиционно назначается стандартная профилактика препаратом фактора свертывания VIII (FVIII), целью которой является достижение ноля кровотечений в год и остаточной активности FVIII не менее 1 %. У многих пациентов стандартный подход не позволяет достичь этих целей, что связано с целым рядом факторов: возраст больного, образ жизни, уровень физической активности, состояние суставов, мышечный тонус, комплаентность пациента, индивидуальный фармакокинетический (ФК) ответ на введение FVIII. В зависимости от уровня физической активности целевая остаточная активность FVIII может составлять 2, 3 или даже 5 %. В настоящее время активно изучается и внедряется в клиническую практику индивидуализированный подход к лечению пациентов с тяжелой формой гемофилии А, основанный на ФК-профиле пациента. Индивидуализация профилактики у пациентов с тяжелой формой гемофилии – реальная потребность врача и пациента. Отмечается высокая вариабельность значений периода полувыведения FVIII у разных больных. Необходим контроль длительности периода времени, когда остаточная активность FVIII составляет менее 1 %, т. е. периода, напрямую связанного с риском возникновения спонтанных кровотечений. Для пациентов, получающих лечение препаратом октоког альфа, разработано программное обеспечение (ПО) myPKFiT* на основе web-приложения, позволяющее моделировать режим дозирования с учетом ФК-профиля пациента на основании определения активности FVIII в 2 образцах крови. ПО позволяет изменять (повышать) целевой уровень остаточной активности FVIII, учитывая образ жизни и уровень физической активности пациента. Возможности ПО позволяют наглядно продемонстрировать больному активность FVIII при различных дозах и интервалах введения препарата, а также обозначить риски, возникающие при пропуске приема препарата. Таким образом, myPKFiT решает важную задачу индивидуализированного подхода к подбору и коррекции терапии, способствует улучшению сотрудничества и взаимопонимания врача и пациента, повышению приверженности больного к терапии и достижению оптимальных результатов.

Patients with a severe and moderate form of hemophilia A have traditionally been prescribed standard prevention with a coagulation factor VIII (FVIII), the goal of which is to achieve zero bleedings per year and a remaining activity of FVIII no fewer than 1 %. The standard approach does not allow achieving these goals in many patients due to a variety of factors: age of the patient, lifestyle, level of physical activity, condition of joints, muscle tone, patient compliance, individual pharmacokinetic (PK) response to FVIII administration. The target remaining activity of FVIII may be 2, 3 or even 5 % depending on the level of physical activity. Nowadays an individualized approach to the treatment of patients with severe hemophilia A based on the patient's PK profile is actively being explored and implemented in clinical practice. Individualization of prevention in patients with severe hemophilia is a real need for the physician and patient. There is a high variability in the values of the half-life period of FVIII in different patients. It is necessary to monitor the duration of the time period when the remaining activity of FVIII is less than 1 %, i.e., the period which is directly linked to the risk of spontaneous hemorrhage. For the patients getting treatment of Octocog alfa there has been developed the software (SW) myPKFiT* on the basis of web application which allows to simulate a dosage regimen taking into account the patient's PC profile based on the determination of FVIII activity in 2 blood sample. The SW allows changing (increase) the target level of the remaining activity of FVIII considering the lifestyle and the level of physical activity of the patient. The ability of SW allows the patient to demonstrate the activity of FVIII at various doses and intervals of drugs, as well as identify the risks that arise when a drug is missed. Therefore, myPKFiT solves an important task of the individualized approach to selection and correction of therapy, improves the collaboration and mutual understanding between the physician and the patient, up regulates of the patient adherence to the therapy and achieves optimal results.

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The authors declare that there are no conflicts of interest present.