Pilot Project «All-Russian Registry of Patients with Severe Bronchial Asthma.» The First Results in a Children’s Group of Patients (Moscow’s Observation Program)

The article presents the first results of the pilot observation program «The All-Russian Severe Asthma Patient Registry» which was started in Moscow. Objectives. The objective of this initiative is the accumulation of clinical data on patients with severe bronchial asthma in the Russian Federation. Methods. The chosen method was a registry, the rights holders of which were 3 professional associations — Interregional Public Organization «Russian Respiratory Society», Public Organization «Union of Pediatricians of Russia», All-Russian Public Organization «Association of Allergologists and Clinical Immunologists». Results. The results of the pilot project are presented by the data analysis of electronic case report forms of 36 children (totally, the project included 100 patients, 64 adults and 36 children) enrolled in the program from June to December 2016. Using the registry, a detailed analysis of the clinical characteristics of a group of children with severe atopic bronchial asthma was carried out, the results of a pharmacoepidemiological evaluation of therapy were given. Obtained by the researchers, a high frequency of the assignment of a genetically engineered biologic drug of omalizumab meets the international recommendations but does not reflect the all-Russian reality and can be explained by inclusion in the registry of pediatric patients observed only in the Federal State Autonomous Institution «National Scientific and Practical Center of Children’s Health» of the Ministry of Health of the Russian Federation. Conclusion. To determine true indicators that characterize bronchial asthma in children in the Russian Federation it is necessary, undoubtedly, to expand the geography of project participants

Пилотный проект «Общероссийский регистр пациентов с тяжелой бронхиальной астмой». Первые результаты в детской группе пациентов (наблюдательная программа г. Москвы)

The article presents the first results of the pilot observation program «The All-Russian Severe Asthma Patient Registry» which was started in Moscow. Objectives. The objective of this initiative is the accumulation of clinical data on patients with severe bronchial asthma in the Russian Federation. Methods. The chosen method was a registry, the rights holders of which were 3 professional associations — Interregional Public Organization «Russian Respiratory Society», Public Organization «Union of Pediatricians of Russia», All-Russian Public Organization «Association of Allergologists and Clinical Immunologists». Results. The results of the pilot project are presented by the data analysis of electronic case report forms of 36 children (totally, the project included 100 patients, 64 adults and 36 children) enrolled in the program from June to December 2016. Using the registry, a detailed analysis of the clinical characteristics of a group of children with severe atopic bronchial asthma was carried out, the results of a pharmacoepidemiological evaluation of therapy were given. Obtained by the researchers, a high frequency of the assignment of a genetically engineered biologic drug of omalizumab meets the international recommendations but does not reflect the all-Russian reality and can be explained by inclusion in the registry of pediatric patients observed only in the Federal State Autonomous Institution «National Scientific and Practical Center of Children’s Health» of the Ministry of Health of the Russian Federation. Conclusion. To determine true indicators that characterize bronchial asthma in children in the Russian Federation it is necessary, undoubtedly, to expand the geography of project participants.В статье представлены первые результаты пилотной наблюдательной программы «Общероссийский регистр пациентов с тяжелой бронхиальной астмой», стартовавшей в Москве. Цель данной инициативы — аккумуляция клинических сведений о больных тяжелой бронхиальной астмой в Российской Федерации. Методы. Методом был выбран регистр, правообладателями которого стали 3 профессиональные ассоциации — Межрегиональная общественная организация «Российское респираторное общество», Общественная организация «Союз педиатров России», Всероссийская общественная организация «Ассоциация аллергологов и клинических иммунологов». Результаты пилотного проекта, представлены анализом данных электронных индивидуальных регистрационных карт 36 пациентов детского возраста (всего в проект было включено 100 больных, из них 64 взрослых и 36 детей), прошедших регистрацию в программе с июня по декабрь 2016 г. С использованием регистра проведен подробный анализ клинической характеристики группы детей с тяжелой атопической бронхиальной астмой, даны результаты фармакоэпидемиологической оценки терапии. Полученная исследователями высокая частота назначения генно- инженерного биологического препарата омализумаба соответствует международным рекомендациям, однако не отражает общероссийской действительности и может быть объяснена включением в регистр пациентов детского возраста, наблюдающихся только в ФГАУ «ННПЦЗД» Минздрава России. Заключение. Для определения истинных показателей, характеризующих бронхиальную астму у детей в Российской Федерации, несомненно, необходимо расширение географии участников проекта.

Влияние ферментозаместительной терапии на обструкцию верхних дыхательных путей у детей с мукополисахаридозами: ретроспективное когортное исследование

Background. Data on the efficacy of enzyme replacement therapy (ERT) in relation to the pathology of ENT organs and respiratory performance in sleep in children with mucopolysaccharidosis (MPS) is poorly presented in the literature. Our aim was to assess the effect of ERT on the upper respiratory tract in children with MPS. Methods. According to the case histories, we studied treatment results of children with MPS type I and II who received ERT in the Research Center of Children’s Health from January 2007 to November 2016. The severity of upper airway obstruction and its change during ERT was assessed according to indices of apnea-hypopnea and desaturation (SpO2), average/minimal SpO2, duration of SpO2 episodes < 90%, and hypertrophy degree of palatine tonsils and adenoids. Results. The severity of the obstructive sleep apnea syndrome did not progress in children (n = 15) with MPS against the background of ERT with a median duration of 38 (23; 48) months: initially, the apnea-hypopnea index was 3 (1.3; 7.7), while the repeated study — 2.6 (0.9; 13.5) (p = 0.507). There was also no statistically significant change in cardiorespiratory monitoring values. Conclusion. Long-term ERT in children with MPS type I and II interferes with the progression of airway obstruction.Обоснование. Данные об эффективности ферментозаместительной терапии (ФЗТ) в отношении патологии ЛОР- органов и показателей дыхания во сне у детей с мукополисахаридозом (МПС) в литературе представлены скудно.Цель исследования — оценить влияние ФЗТ на состояние верхних дыхательных путей у детей с МПС.Методы. По историям болезни изучали результаты лечения детей с МПС I и II типов, получавших ФЗТ в Научном центре здоровья детей (ныне ФГАУ «НМИЦ здоровья детей» Минздрава России) с января 2007 по ноябрь 2016 г. Выраженность обструкции верхних дыхательных путей и ее изменение на фоне ФЗТ оценивали по индексам апноэ-гипопноэ и десатурации (SpO2), средней/минимальной SpO2, продолжительности эпизодов SpO2< 90% и степени гипертрофии небных миндалин и аденоидов.Результаты. У детей (n=15) с МПС на фоне ФЗТ продолжительностью (медиана) 38 (23; 48) мес тяжесть синдрома обструктивного апноэ сна не прогрессировала: исходно индекс апноэ-гипопноэ составил 3 (1,3; 7,7), при повторном исследовании — 2,6 (0,9; 13,5) (р=0,507). Также не установлено статистически значимого изменения показателей кардиореспираторного мониторинга.Заключение. Длительная ФЗТ у детей с МПС I и II типов препятствует прогрессированию обструкции дыхательных путей.КОНФЛИКТ ИНТЕРЕСОВ Л.С. Намазова-Баранова — получение исследовательских грантов от фармацевтических компаний Пьер Фабр, Genzyme Europe B. V., ООО «Астра зенека Фармасьютикалз», Gilead / PRA «Фармасьютикал Рисерч Ассошиэйтс СиАйЭс», Bionorica, Teva Branded Pharmaceutical products R&D, Inc / ООО «ППД Девелопмент (Смоленск)», «Сталлержен С. А.» / «Квинтайлс ГезмбХ» (Австрия). Л.М. Кузенкова, А.К. Геворкян, Т.В. Подклетнова, Н.Д. Вашакмадзе читают лекции для компаний «Санофи Джензайм», «Шайер», «Биомарин». Остальные авторы подтвердили отсутствие конфликта интересов, о котором необходимо сообщить

Boson Peak and Superstructural Groups in Na2O-B2O3 Glasses

Low-frequency Raman spectra of the sodium borate glasses with Na2O content ranging from 0 to 30 mol% were measured and analyzed from the point of view of their structure in the intermediate range order. Our results show that there is a simple linear correlation between correlation length, lc, and an average size, , of the area of ordered arrangement of atoms, obtained via the distribution of the (super)structural units and their representative size. Six types of the areas of ordered arrangement of atoms were determined, being related to the presence of the B3O3⌀3 boroxol rings, B3O3⌀4- triborate rings, B5O6⌀4- pentaborate groups, B8O10⌀62- tetraborate groups, B4O5⌀42- diborate groups, and B3O63- cyclic metaborate anions in the sodium borate glasses. It was shown that the size of the areas of ordered arrangement of atoms can be determined from the simple geometric analysis of the crystallographic data on the superstructural units

Termodynamický model a vysokoteplotní Ramanova spektra Na2O-B2O3 sklotvorných tavenin

The set of 33 baseline subtracted and thermally corrected Raman spectra of xNa(2)O center dot(1-x)B2O3 (x = 0.10, 0.15, 0.20, 0.25, 0.30, 0.40, and 0.50) glassforming melts measured at temperatures ranging from 501 degrees C to 1145 degrees C was analyzed. The real error estimated by Principal Component Analysis indicated 3-4 independent components. The Multivariate Curve Analysis (MCR) performed for three components resulted in the Raman spectra (so called loadings) and relative abundances (so called scores) of each component. The thermodynamic model of Shakhmatkin and Vedishcheva (SVTDM) was evaluated for each studied melt. Ten following system components were considered: Na2O, B2O3, 3Na(2)O center dot B2O3 (N3B), 2Na(2)O center dot B2O3 (N2B), Na2O center dot B2O3 (NB), Na2O center dot 2B(2)O(3) (NB2), Na2O center dot 3B(2)O(3) (NB3), Na2O center dot 4B(2)O(3) (NB4), Na2O center dot 5B(2)O(3) (NB5), and Na2O center dot 9B(2)O(3) (NB9). The Malfait's spectral decomposition was performed considering the equilibrium molar amounts of the components with highest abundance, i.e. B, NB, and NB4. The obtained partial Raman spectra were compared with the loadings obtained by MCR. The acceptable coincidence was found. The MCR adjusted scores were close to the SVTDM equilibrium molar amounts of system components considered in the Malfait's decomposition. The obtained results validate the correctness of the SVTDM. (C) 2019 Elsevier B.V. All rights reserved.Vysokoteplotní Ramanova spektra pro sklotvorné taveniny Na2O-B2O3 s obsahem Na2O 10 až 50 molárních procent byla změřena a analyzována ve spojení s termodynamickým modelem SVTDM

Termokinetické chování Al2O3-PBO-B2O3 skel

Thermokinetic behavior of the Al2O3-PbO-B2O3 glassy system was investigated by means of differential scanning calorimetry, X-ray diffraction analysis and Raman spectroscopy. The glass transition kinetics was described in terms of the Tool-Narayanaswamy-Moynihan model. The compositional evolution of the relaxation parameters was explained in terms of the structural changes and movements of the characteristic structural units detected by Raman spectroscopy. Crystal formation in the studied glassy matrices was investigated in dependence on composition and particle size. The crystal growth was suppressed by increasing particle size as well as by increasing Al2O3 content; formation of crystalline boron oxides was replaced by formation of mixed Al2O3/PbO oxides and Pb4B2O7 phase. The nucleation-growth Johnson-Mehl-Avrami model and the empirical autocatalytic model of.Sestak and Berggren were used to describe the complex crystallization kinetics. The Avramov-.Sestak concept of temperature dependent activation energy was successfully applied and tested on the real-life experimental data. The corresponding methodology was critically reviewed.Termokinetické chování skel na bázi Al2O3-PBO-B2O3 bylo studováno technikami DSC, XRD a Ramanovou spektroskopií. Kinetika skelného přechodu byla popsána modelem TNM. Pro popis krystalizační kinetiky byly použity modely JMA a Sestak-Bergrren. Kompoziční trendy termokinetického chování byly popsány. Koncept Avramov-Šesták pro teplotní závislost aktivační energie byl úspěšně aplikován a testován

EFFICACY AND SAFETY OF ENZYME REPLACEMENT THERAPY IN CHILDREN WITH MUCOPOLYSACCHARIDOSIS TYPE I, II, AND VI: A SINGLE-CENTER COHORT STUDY

Background. There are limited data on the efficacy of long-term enzyme replacement therapy (ERT) in children with mucopolysaccharidosis (MPS).Objective. Our aim was to study the efficacy and safety of long-term ERT in children with MPS type I, II, and VI.Methods. We analyzed the results of ERT with laronidase, idursulfase, and galsulfase in children with MPS type I, II, and VI admitted to the federal research center from January 2007 to November 2016. The response rate was assessed by the level of normalized urinary excretion of glycosaminoglycans (GAGs) (the ratio of GAGs concentration to urine creatinine) recalculated in percent (%) exceedance of the upper limit of normal for the corresponding age. Data on the administered therapy and its results, including adverse events, is extracted from the medical records of in-patients.Results. The results of treatment (intravenous infusions, intervals between administrations from 4 to 10 days) were studied in 33 children (5 of them were girls) with MPS type I (n = 4; laronidase at a dose of 0.58 mg/kg), II (n = 26; idursulfase at a dose of 0.5 mg/kg), and VI (n = 3; galsulfase at a dose of 1 mg/kg). A decrease in the normalized urinary excretion of GAGs from 376% (172; 791) to 54% (0; 146) exceedance of the upper limit of normal for the age (p < 0.001) was noted in the course of ERT lasting (median) 27 (14; 41) months. A decrease in the normalized GAGs excretion below the upper limit of normal for the age was established in 12/33 (36%) patients. ERT-associated adverse events were identified in 12 patients; one case required a two-fold therapy interruption. The development of nephrotic syndrome in the course of ERT in patients with severe MPS II was first described.Conclusion. Long-term ERT in children with MPS type I, II, and VI is characterized by acceptable efficacy and safety. Key words: children, mucopolysaccharidosis, enzyme replacement therapy, laronidase, idursulfase, galsulfase, glycosaminoglycans

The Impact of Enzyme-Replacement Therapy on Upper Airway Obstruction in Children with Mucopolysaccharidoses: Retrospective Cohort Study

Background. Data on the efficacy of enzyme replacement therapy (ERT) in relation to the pathology of ENT organs and respiratory performance in sleep in children with mucopolysaccharidosis (MPS) is poorly presented in the literature. Our aim was to assess the effect of ERT on the upper respiratory tract in children with MPS. Methods. According to the case histories, we studied treatment results of children with MPS type I and II who received ERT in the Research Center of Children’s Health from January 2007 to November 2016. The severity of upper airway obstruction and its change during ERT was assessed according to indices of apnea-hypopnea and desaturation (SpO2), average/minimal SpO2, duration of SpO2 episodes < 90%, and hypertrophy degree of palatine tonsils and adenoids. Results. The severity of the obstructive sleep apnea syndrome did not progress in children (n = 15) with MPS against the background of ERT with a median duration of 38 (23; 48) months: initially, the apnea-hypopnea index was 3 (1.3; 7.7), while the repeated study — 2.6 (0.9; 13.5) (p = 0.507). There was also no statistically significant change in cardiorespiratory monitoring values. Conclusion. Long-term ERT in children with MPS type I and II interferes with the progression of airway obstruction