Регистры больных муковисцидозом: отечественный и зарубежный опыт

Cystic fibrosis is a multi-organ genetic disease that reduces life expectancy and requires costly complex therapy. The economic burden of cystic fibrosis is determined by the cost of treatment, the conditions for optimizing medical technologies based on epidemiological knowledge of the disease course nature and the therapy effectiveness. In many countries cystic fibrosis patient registries have been created that allow to assess the quality, efficiency and optimality of medical care. Registries are formed at the regional, national, and international levels, which facilitates the collection of complete and reliable patient information for subsequent analysis, comparison and synthesis of clinical and epidemiological indicators. The review is devoted to the actual principles of organization of domestic and some foreign cystic fibrosis patient registries. The article selectively provides examples of information from registries. The application of positive experience of the work of foreign registries in Russia can improve the quality of medical care for patients with cystic fibrosis.Муковисцидоз — полиорганное генетическое заболевание, сокращающее продолжительность жизни и требующее затратной комплексной терапии. Экономическое бремя муковисцидоза определяется стоимостью лечения, условиями оптимизации медицинских технологий на основании эпидемиологических знаний о характере течения заболевания и результативности проводимой терапии. Во многих странах созданы регистры больных муковисцидозом, позволяющие оценивать качество, эффективность и оптимальность медицинской помощи. Регистры сформированы на региональном, национальном и международном уровнях, что способствует сбору полных и достоверных сведений о больных для последующего анализа, сравнения и обобщения клинико-эпидемиологических показателей. Обзор посвящен актуальным на сегодняшний день принципам организации отечественных и некоторых зарубежных регистров больных муковисцидозом. В статье выборочно приводятся примеры сведений, содержащихся в регистрах. Применение положительного опыта работы зарубежных регистров в России может улучшить качество медицинской помощи больным муковисцидозом.

Cystic Fibrosis Patient Registries: Domestic and Foreign Experience

Cystic fibrosis is a multi-organ genetic disease that reduces life expectancy and requires costly complex therapy. The economic burden of cystic fibrosis is determined by the cost of treatment, the conditions for optimizing medical technologies based on epidemiological knowledge of the disease course nature and the therapy effectiveness. In many countries cystic fibrosis patient registries have been created that allow to assess the quality, efficiency and optimality of medical care. Registries are formed at the regional, national, and international levels, which facilitates the collection of complete and reliable patient information for subsequent analysis, comparison and synthesis of clinical and epidemiological indicators. The review is devoted to the actual principles of organization of domestic and some foreign cystic fibrosis patient registries. The article selectively provides examples of information from registries. The application of positive experience of the work of foreign registries in Russia can improve the quality of medical care for patients with cystic fibrosis

Real-world study of efficacy, risk management and reasons for discontinuation of natalizumab for treatment of multiple sclerosis in Russia.

BackgroundNTZ is approved in Russia for the treatment of highly active relapsing remitting multiple sclerosis and is reimbursed via federal budget program. However, no data about NTZ treatment in Russia and the effect of federal reimbursement have been performed so far.ObjectiveTo characterize the population of patients receiving natalizumab and assess the efficacy and risk-management plan (RMP) implementation of NTZ therapy in routine clinical practice in Russia.MethodsWe analyzed data for 334 patients, who received at least one infusion of NTZ. Relapse rate, MRI activity, NEDA-3 status after 2 years were assessed. Anti-JC virus antibodies status and RMP implementation were evaluated. Drop-out rate and reasons for therapy discontinuation were analyzed.ResultsPatients switched to natalizumab in Russia are mainly female (63%), with median EDSS score of 3.5 and high disease activity: 93% had at least 1 relapse and 58% had both T1Gd+ and new T2 lesion a year before therapy initiation. Introduction of federal reimbursement allowed patients with less relapses to start therapy with natalizumab. The only predictor of 6-month progression was EDSS score at the baseline of therapy (HR = 2.1375, 95%CI 1.0026-4.5570, p = 0.0492). 82% patients reached NEDA-3 at 24 month of therapy. 25% of patients discontinued NTZ for reasons: tolerability (14.5%), JCV antibody status (61%), and patient's decision (17%). RMP was implemented in only 36% patients.ConclusionNatalizumab appeared to have high efficacy in Russian clinical practice. Federal reimbursement allowed less active patients to start natalizumab. More efforts should be done to improve RMP implementation