The predictability of results of pediatric clinical trials is often limited for a number of reasons. Among the main ones is the imperfect functioning of organ due to immature ontogeny of enzyme and organ systems in children, and the presence of special subpopulations of full-term newborns and preterm neonates sometimes being in a critical condition. The main task of a present-day pediatric investigational plan is to develop drugs and to elaborate doses that are «specifically designed», not simply «suitable» for neonates. Other reasons for limited predictability are as follows: adult data extrapolation constraint due to children’s anatomic and physiological features, the lack of clinical trial subjects resulting in inability to select an optimal dose by its escalation, the absence of consensus on the ethical aspect of pediatric clinical trials, the etiopathogenetic difference of some diseases and conditions depending on subject’s age, and prevalence of placebo-effect in children. Nowadays it is supposed to be very important to publish all, even failed, pediatric trials to improve the accuracy of pharmacological effects modeling in different subpopulations.

The deficiency of lysosomic acid lipase is a rare hereditary enzymopathy. The focus of this article is the present condition of this issue. The authors demonstrate epidemiological data and etiopathogenetic features of two phenotypic forms of lysosomic acid lipase deficiency — Wolman disease and cholesterol ester storage disease. Clinical features of both — the rapidly progressing form and the slowly developing cholesterol ester storage disease — are described in detail in this article. The algorithm and crucial steps of differential diagnosis are described in detail. Also, indications to pathogenetic therapy are carefully formulated, and the tactic of enzyme replacing therapy is given. The modern approaches to the management of child patients are described.

The focus of this article is Gaucher disease — a rare enough hereditary pathology. The authors present the most up-to-date epidemiological data and features of Gaucher disease etiopathogenesis. They offer clinical characteristics for the various types of this disease. The algorithm and crucial steps of differential diagnosis are described in detail. Also, the tactic and algorithms of enzymereplacing therapy (pathogenetic treatement of this hereditary enzymopathy) are carefully presented, together with the modern scheme of managing patients according to the corresponding health care delivery stages.

Background: Specialized foods, based on aminoacids without phenilalanin, are the main source of protein for patients with phenilketonuria of all ages. Based on modern technologies, new Russian-made foods were created. They have an optimized aminoacid and micronutrient composition, which increases their bioavailability.

Objective: Our aim was to investigate the clinical efficiency of the new Russian-made specialized foods based on aminoacids without phenylalanine, in patients with phenylketonuria.

Methods: Evaluating physical, somatic and neuropsychic development as well as measuring phenylalanine blood level were carried out twice: before the prescription and after 1 month of using the new Russian-made specialized foods. The chemical composition of the ration was controlled using dietology methods.

Results: 57 children at the age of 14 days to 15 years with phenylketonuria (which was detected in neonatal screening) were picked for this study. A hypophenylalanine diet has been prescribed for all children no later than at the age of 3 months of life. In children of the first year (I group) (among which there were children with high phenylalanine blood levels) phenylalanine concentration decreased from 5,5 (4,0; 21,0) to 4,4 (3,7; 4,7) mg/dl (p = 0,014) while using Russian-made specialized foods. In this group of children the psychomotor and physical indices improved. In early childhood-, preschool- and school-aged patients phenylalanine blood level remained steady. The chemical composition of the ration with Russian-made foods, based on aminoacids without phenylalanine, corresponded to the reference intake of main nutrients and energy.

Conclusion: Hypophenylalanine diet with new Russian-made specialized foods without phenylalanine (which is designed for phenylketonuria patients of various age groups) showed high clinical efficiency of these foods.

Background: The contemporary application of Botulinum toxin A (BTA) in cerebral palsy (CP) implies multilevel injections both in on-label and off-label muscles. However, there is no single international opinion on the effective and safe dosages, target muscles, and intervals between the injections.

Objective: Our aim was to analyze the Russian multicenter independent experience of single and repeated multilevel injections of Abobotulinum toxin А in patients with spastic forms of CP.

Methods: 8 independent referral CP-centers (10 hospitals) in different regions of Russia. Authors evaluated intervals between the injections, dosages of the BTA for the whole procedure, for the body mass, for the each muscle, and functional segment of the extremities.

Results: 1872 protocols of effective BTA injections (1–14 repeated injections) for 724 patients with spastic CP were included. The age of the patients was between 8 months to 17 years 4 months at the beginning of the treatment (with a mean of 3 years 10 months). Multilevel BTA injections were indicated for the majority (n = 634, 87.6%) of the patients in all the centers. The medians of the dosages for the first BTA injection were between 30–31 U/kg (500 U), the repeated injections doses up to 45 U/kg (1000 U) (in most centers). The median intervals between the repeated injections were 180–200 days in 484 (66.9%) patients and 140–180 days in 157 (24.7%) patients. In 2 centers, children with GMFCS IV–V were injected more often than others.

Conclusion: Multilevel BTA injections were indicated for the most patients. The initial dose of Abobotulinum toxin A was 30–31 U/kg. The repeated injections dose could increase up to 40 U/kg. The repeated injections were done in 140–200 days after the previous injection.

Thanks to modern molecular-genetic microbiota analysis, numerous previously unstudied bacteria were identified. As a result, the conception of human microbiocenosis was significally changed, particularly the conception of intestinal microbiota, which substantially determines human health. The ways the bacteria affect the metagenome, endocrine regulation and function of the nervous system have been described in recent years. At its early stages, the formation of intestinal microbiota depends on such factors as a complicated gestation course, prematurity, surgical delivery, late breast latching, artificial feeding since birth or antibiotic therapy. However, the results of latest studies indicate that the microbial factor affects children long before their birth. Present scientific hypothesis regarding torpid microbial inflammation in uterine cavity as a cause of disturbanses in the gestation course or of preterm labour was also formed in connection with investigating placenta and amniotic fluid microbiocenoses. This survey presents analysis of the latest data concerning the affection of both antenatal and postnatal factors at early stages of intestinal microbiota conditioning in children.

Despite the huge and seamingly undisputable success of vaccinal prevention, a critical situation is developing today in the context of immunization-controlled infections control. Increasing antivaccination propahanda leads to a decrease in the collective immunity and the occurance of high-contagenous infectious diseases in various places of the world. It is a disturbing tendency — the usage of antivaccinal ideas for populist purposes. This article contains several examples of how such tactics lead to severe consequences for public health: pertussis and morbilli epidemia in Europe, poliomyelitis epidemia in African and Asian countries.

In 2013 WHO re-evaluated its main goals of the polio eradication program. A modernization program was accepted with regard to the National vaccination calendars worldwide which includes a step-by-step refusal from the living polio vaccine (OPV) and a total transition to the inactivated polio vaccine (IPV) starting in 2019. Because of the total eradication of the polio type 2 virus, as an intermediate step the 3-valence OPV was substituted with the 2-valence OPV, which does not contain the type 2 polio virus, in April 2016. The aim of the article is to present the history of polio prevention and to state the reasons for the adoption of 3rd edition of the Global Polio Eradication Initiative. The new approaches were defined for eradication of wild polio virus type 1 and vaccine related strains. A new strategy for global switch to inactivated polio vaccine by 2019 was suggested.

Background: Vitamin D-deficient states remain highly prevalent in children, according to Russian and foreign studies. Because of this, it is important to conduct timely prevention of rachitis in children, taking into account the commitment to therapy from the parents’ side.

Objective: Our aim was to study the preventive efficacy of the Russian-manufactured vitamin D3 solutio oleosa with regard to preventing rachitis.

Methods: A short-term prospective comparative study has been conducted. Vitamin D3 was used in the dosages of 400 and 800 ME or 500 and 1000 ME, in the form of an oil or water solution of cholecalciferol in mature and premature (according to the gestation term) children, correspondingly. The control group was comprised of mature children of a comparable age, who received no vitaminoprophilaxis (parental refusal). We evaluate clinical symptoms of rachitis before and after 1 month after the beginning of the study.

Results: 111 children aged 4–6 months, of them 66 mature and 45 premature were included into the study. None of the children developed rachitis symptoms while taking vitamin D3 while 8 out of 15 controls (53%) developed signs of rachitis.

Conclusion: We demonstrated a comparative efficacy of oil and water solutions of vitamin D3 with regard to preventing rachitis in mature and premature children.