Malnutrition (MN) is common in children with acute and chronic illnesses. The use of questionnaires to assess nutritional risk in children admitted to hospital is a simple and quick way to identify patients who need nutritional advice and nutritional support. Various English-language screening questionnaires, such as STRONGkids nutritional risk screening tool for children, in particular, have been developed and validated. The purpose of the study is to adapt the STRONGkids nutritional risk screening tool for the Russian language speaking audience and to determine the reliability and validity of its new language version. Materials and methods of research: linguistic ratification of the STRONGkids questionnaire was carried out and the Russian language version was created. This single-center prospective study included 419 children admitted from October, 2019 to March, 2021 for treatment in Therapeutic Pediatric (cardiac, nephrological, gastroenterological, pulmonological etc.) and Surgical Pediatric (abdominal, thoracic, otorhinolaryngological, cardiosurgical etc.) Departments of the National Medical Research Center for Children’s Health (Moscow, Russia) with a hospital stay longer than a single day. The reliability of the questionnaire was assessed in 50 children. The anthropometric indicators were used to assess the current validity; the duration of hospitalization was used to assess the predictive validity. Body mass index-for-age (BMI-for-age) <-2SD and Length/height-for-age <-2SD z-scores were criteria for acute and chronic MN, respectively. Results: almost complete (κ=0.84) inter-expert agreement was found during the evaluation of reliability of the STRONGkids questionnaire. The validation study included 419 patients (51% boys/49% girls) aged from 1 month till 17 years and 11 months old (8 years old as median) with hospitalization duration from 1 to 100 days (10 days as median), of which 314 (75%) and 105 (25%) therapeutic pediatric and surgical pediatric patients, respectively. The frequency of acute and chronic MN at admission was 9.1% and 7.4%, respectively, and the percentage of children at low, moderate and high risk (assessed by the questionnaire) was 22.7%, 66.6% and 10.7%, respectively. Children at nutritional risk had statistically significantly (p<0.05) lower BMI-for-age and Length/height-for-age z-scores, greater incidence of MN and longer hospital stays than patients without nutritional risk. A chronic disease addendum, complications and high nutritional risk according to the questionnaire are independent unfavorable factors for an increase in the duration of hospitalization. Conclusion: the Russian language version of the STRONGkids nutritional risk screening tool for children was adapted and validated to assess nutritional risk in children admitted to hospital for treatment. Since right now it can be of use for identifying patients at high risk of MN for the purpose of their additional examination and timely administration of nutritional support and can be used both in routine pediatric clinical practice and in purpose of further scientific research.

Background. Health Related Quality of Life (HRQL) is a person’s perception of the effect of a disease on physical, social and psychological functioning and wealth. Questionnaires that help assess the HRQL can give a concept of child’s state, identify arcane issues and they are of great importance for a full understanding of how the state of health affects the child. Universal HRQL tools allow to compare results with general parameters, indicators with specific symptoms better reflect clinically significant moments. The most widespread in the Russian Federation was the general PedsQL^TM 4.0 questionnaire, widely used in practice to assess HRQL in children with various pathologies. Objective.The aim of the study is to describe the analysis of linguistic accuracy and authenticity of the content of the Russian version of the module of gastrointestinal symptoms of the PedsQL^TM questionnaire, measuring HRQL, in children with gastrointestinal disorders (GIDs).Materials and methods. The establishment of linguistic accuracy and authenticity of the content was carried out according to international standards. The process included forward translation, scientific assessment and coordination, reverse translation, verification of the reverse translation and interviews with 17 children aged 5–18 years with symptoms of GIDs and 20 parents of children with symptoms of GIDs aged 2–18 years.Results. The Russian version of PedsQL^TM module of gastrointestinal symptoms (report from children 5-18 years old, report from parents for children 2–18 years old) was developed without significant difficulties. Eight questions required discussion after the forward translation, one change was made after the reverse translation, and three changes were made after the study of the symptom’s module by patients and parents.Conclusion. A conceptually equivalent version of the of PedsQL^TM module of gastrointestinal symptoms in Russian has been developed for children aged 2–18 years. It allows to improve the assessment of HRQL in children with GIDs in the Russian Federation. To assess authenticity and reliability of the Russian version of the module, it’s recommended to conduct further research using a larger sample

Introduction. The advent of pathogenetic therapy has significantly changed the prognosis for spinal muscular atrophy (SMA). However, our knowledge of the pathogenetic methods of treating SMA is mostly based on the results of clinical trials, which are largely limited due to the narrow criteria for selecting patients and the short duration of the following-up. Objective. To evaluate the efficacy of onasemnogene abeparvovec gene therapy in patients with SMA type I with clinical manifestations of the disease and in children at the presymptomatic stage of the disease in real clinical practice. Materials and methods. The study included seventy nine SMA children including 42 boys (53.2%). The diagnosis was verified during DNA diagnostics. The children were divided into two subgroups, depending on whether they had symptoms of SMA at the time of inclusion in the study or not. 44 children had SMA type I with the onset of clinical symptoms before the age of 6 months of life. 35 children were at the presymptomatic stage of the disease. All patients received gene therapy with onasemnogene abeparvovec, the average age at the moment of conduction of therapy was 2.90 ± 1.74 months (95% CI: 2.51–3.29 months), min — 1.00 month, max — 7.00 months. A comprehensive assessment of clinical parameters (stages of motor development milestones according to WHO recommendations, HINE-2 and CHOP-INTEND scores) was carried out before the initiation of gene therapy and 6, 12, 18 and 24 months after its implementation. Results. Children with SMA type I showed favour trend in motor development milestones after gene therapy. By the end of the second year of the follow-up, 88.6% of patients held their head erect and rolled from back to sides; 60.5% could sit without support, but only 10.3% were able to achieve all motor skills, but none of these children achieved them in accordance with WHO criteria. The HINE-2 score increased from Me of 2.0 (1.00–2.25) points at the initiation of therapy to Me of 20.00 (16.50-24.50) points by the end of the follow-up period. Only two children (4.5%) of this subgroup reached the maximum score of 26 points. The CHOP-INTEND score increased from Me of 30.0 (22.00–37.25) points before starting treatment to Me of 60.0 (58.0-64.0) points by 24 months of follow-up. Only 11.4% of patients with SMA type I reached the maximum score of 64 points. The majority of the presymptomatic patients in our study achieved all motor development milestones according to WHO criteria according to their age on the background of gene therapy. All children from this subgroup who were under sufficient supervision and reached the age of walking alone (23 children) had a maximum HINE-2 score of 26 points by the age of 18 months. All children from this subgroup reached the maximum CHOP-INTEND score of 64 points by the age of 6 months. Conclusion. The usage of onasemnogene abeparvovec in children with SMA type I both significantly modifies the course of the disease and improves outcomes compared with the natural history of SMA course with early onset, and the usage of gene therapy in most of the presymptomatic patients leads to the achievement of motor development milestones in accordance with WHO criteria.

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The aim is to identify parents’ (legal representatives’) satisfaction with the quality of medical care provided to their disabled children to improve this type of service further. Materials and methods. A sociological survey was conducted of 506 legal representatives of minors (aged from birth to 17 years) with the status of a disabled child. The study design is single-center, non-randomized, uncontrolled. Results. An analysis of the living conditions of a disabled child in the family, the parents’ assessment of his health status, the problems arising in the registration of disability, in the provision of medical and rehabilitation assistance, and issues of medical and social support made it possible to determine the position of this part of the child population in current legal and medical and social conditions. The main problems were the collection of a large number of documents when registering a disability, a long wait for the day of examination, the remoteness of the medical and social examination bureau, the lack of specialist doctors, problems with obtaining subsidized drugs, the lack of consideration of the individual needs of the child when conducting unique rehabilitation programs, the need to apply at the same time to various organizations and departments, violation of rights in the provision of medical services to a disabled child. Conclusion. The obtained information is very significant for further improving the provision of medical and social assistance to disabled children and children with disabilities. The main task today is to develop mechanisms for implementing the declared rights and freedoms of persons with disabilities, the obligations assumed by the State concerning them.

Spastic muscles in the pathological motor patterns may change at different ages that leads to the changes in anti-spastic treatment. To study the specific patterns of spasticity in CP patients with level III according to the Gross Motor Function Classification System (GMFCS) in different age periods. A retrospective analysis of injection protocols of Abobotulinum toxin A for 99 patients with bilateral spastic CP GMFCS III at the age of 2 to 12 years was performed. Spasticity patterns were evaluated according to the frequency of target muscle selection for the botulinum toxin A (Abobotulinum toxin A) injections. The equinus foot deformity (89.7%, 95%CI 72.8-97.2) and its combination with internal hip rotation (79.3%, 95% CI 61.3-90.5) and/or hip adduction (65.5%, 95% CI 47.3-80.2) were the main lower extremity spasticity pattern for the patients aged 2 to 4 years. Between 4 and 6 years, there was the decrease in injections to the gastrocnemius muscle (75%, 95% CI 52.8-89.2), and less to the m. gracilis (70%, 95% CI 47.9-85.7) and hip adductors (55% 95 CI 34.2-74.2). In the 6-12 year old patients, the predominant spasticity patterns were internal hip rotation (80%, 95% CI 66.8-88.9), adduction (54%, 95% CI 40.4-67.0) and knee flexion (66%, 95% CI 52.1-77.6). The leading upper extremity patterns at all observed ages were forearm pronation (70.7%, 95% CI 61.1-78.8) and elbow flexion (47.5%, 95% CI 37.9-57.2). The frequency of injections to these muscles slightly decreased in elder groups. CP GMFCS III patients demonstrate age-related changes of the spasticity patterns both in lower and upper extremities. These changes and typical spastic muscles should be taken into account during the rehabilitation and botulinum toxin treatment planning, as well as in the detection and prevention of the orthopaedic complications.