The effects of nusinersen treatment on respiratory status of children with spinal muscular atrophy.

BACKGROUND: Respiratory involvement is the main factor predicting the prognosis of spinal muscular atrophy (SMA). Significant responses in motor functions have been demonstrated with nusinersen, but pulmonary outcomes are still varied. We aimed to explore the effects of nusinersen on the respiratory functions of patients with SMA. METHODS: Patients with SMA who were receiving regular nusinersen treatment in our tertiary care hospital were enrolled in this study. We evaluated the patients in terms of the necessity to ventilatory or nutritional support, presence of motor involvement and other comorbidities related with prognosis at three consecutive assessments. RESULTS: The study group consisted of 43 patients (18 type 1, 12 type 2, and 13 type 3) with SMA with a mean age of 27.8 months at diagnosis and 60.8 months at the beginning of nusinersen treatment. The respiratory function improvements were noted in six patients at third assessment. Early initiation of nusinersen was significantly correlated with reduced hospital admissions (P = 0.026). Nutritional support and weight gain were remarkable in the ventilatory-supported group. Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores were significantly higher in the non-tracheostomized group in patients with SMA type 1 (P < 0.005). CONCLUSIONS: We posit that nusinersen may change the natural prognosis of SMA and improve care of children with SMA. Following up children with SMA for longer periods under nusinersen may be beneficial for understanding the effects of treatment. Results of our study need to be supported by future long-term studies to reach a consensus on nusinersen, considering the overall genetic and environmental status as well as the cost-effectiveness of the treatment.

Comparisons of Clinical Features and Outcomes of COVID-19 between Patients with Pediatric Onset Inflammatory Rheumatic Diseases and Healthy Children.

(1) Background: We aimed to describe the clinical features and outcomes of coronavirus disease-2019 (COVID-19) in children and late adolescents with inflammatory rheumatic diseases (IRD) and to measure their severity risks by comparing them with healthy children. (2) Methods: Among children and late adolescents found to be severe acute respiratory syndrome-coronavirus-2 (SARS-CoV-2) positive via polymerase chain reaction (PCR) test, IRD patients with an at least six-months follow-up duration, and healthy children were included in the study. Data were obtained retrospectively. (3) Results: A total of 658 (339 (51.5%) females) (healthy children: 506, IRD patients: 152) subjects were included in the study. While 570 of 658 (86.6%) experienced COVID-19-related symptoms, only 21 (3.19%) required hospitalization with a median duration of 5 (1-30) days. Fever, dry cough, and fatigue were the most common symptoms. None of evaluated subjects died, and all recovered without any significant sequelae. The presence of any IRD was found to increase the risk of both hospitalization (OR: 5.205; 95% CI: 2.003-13.524) and symptomatic infection (OR: 2.579; 95% CI: 1.068-6.228). Furthermore, increasing age was significantly associated with symptomatic infection (OR: 1.051; 95% CI: 1.009-1.095). (4) Conclusions: Our study emphasizes that pediatric rheumatologists should monitor their patients closely for relatively poor COVID-19 outcomes.

The Role of Telehealth Services in Children with Cystic Fibrosis During Coronavirus Disease 2019 Outbreak.

Objectives:This study aimed to monitor the health and nutritional status of pediatric cystic fibrosis (CF) patients via telehealth services during the novel coronavirus disease 2019 (COVID-19). Additional aims were to determine the level of anxiety in the patients and their caregivers and to determine the COVID-19 transmission status among CF patients.Materials and Methods:The CF team supported the patients via remote contact. During telehealth services interviews, in addition to obtaining information about the patients' anthropometric measurements, health status, and CF-related complaints, the State-Trait Anxiety Inventory (STAI) was administered to the patients and controls. The Hospital Anxiety and Depression Scale (HAD) was administered to their caregivers.Results:The study included 144 pediatric CF patients (74 males and 70 females). Mean age of the patients was 8.9 years. In all, 42 (29.2%) of the patients were tested for COVID-19, of which 4 were positive. The mean STAI score was significantly lower in the patient group than in the control group (p < 0.001). The mean HAD anxiety score was significantly higher in the caregivers of the CF patients, compared to the caregivers of the controls (p = 0.005). In addition, the mean HAD depression score was significantly higher in the caregivers of the CF patients (p < 0.001).Conclusions:Telehealth is an innovative method for providing health care services while maintaining social distance and avoiding the risk of exposure and spread of COVID-19. Telehealth services reduce patient and parental anxiety and increase the level of confidence in managing CF-related complications.

Efficacy of standardized tracheostomy training with a simulation model for healthcare providers: A study by ISPAT team.

INTRODUCTION: Tracheostomy care in children may be challenging, due to lack of knowledge of healthcare providers (HCPs). The aim of this study was to determine the level of knowledge of HCP who follow patients with tracheostomy and to increase this level with theoretical training and training in a simulation laboratory. MATERIALS AND METHODS: ISPAT (IStanbul PAediatric Tracheostomy), a multidisciplinary team for tracheostomy care was established and a training program was prepared. Participants were subjected to theoretical and practical pretests which evaluated their knowledge levels and skills for care, follow-up, and treatment of a patient with tracheostomy. After the theoretical and practical training given to the participants with a simulation model, theoretical and practical posttests were applied. RESULTS: Fifty-one HCP from nine tertiary pediatric clinics in Istanbul were enrolled in the training program. Only six (11.8%) of them had received standardized training programs previously. Regarding the theoretical tests, seven of the 33 questions were indicated as essential. The knowledge level of the participants based on the essential questions significantly increased after the training (p < 0.05 for all of the essential questions). The total number of correct answers and correct answers of three subheadings also significantly increased after the practical training (p < 0.001 for all). Ninety-five percent of the participants assessed the course as good or excellent in general. CONCLUSION: Training in a simulation laboratory in combination with theoretical education can improve the knowledge and skills of the HCP enabling improved care of children with a tracheostomy.

General health status of children with asthma during the COVID-19 pandemic.

BACKGROUND: Since the COVID-19 pandemic became a serious health concern globally, patients with chronic diseases have required close attention with regard to general risks and individual treatment. We aimed to reveal the general health status of pediatric asthmatic patients during the pandemic, considering the role of household factors in parental attitudes. METHODS: We asked 60 asthmatic patients and their parents to respond to a questionnaire, with the aim of revealing the current health status of the patients and the general approach of the family to asthma management during the pandemic. RESULTS: A total of eight patients had had an asthma attack during the outbreak, but there was no confirmed correlation with COVID-19 infection. Most of the parents had never considered stopping their children's current medications. However, the majority of them reported concerns about the failure of the ambulatory care services and almost all saw their children as being at high risk for COVID-19 infection. There was no significant relationship between these concerns and their psychological status (P > 0.05). CONCLUSIONS: The crucial point regarding asthma management is to control patients' medical and psychological status to minimize the effects of the pandemic. Healthcare professionals should also pay attention to members of the patients' households because their adaptation to the "new normal" of pandemic may directly affect the patients' state of health.

Immunomodulator effect of topical zinc oxide application in tuberculin skin test.

OBJECTIVE: We aimed to evaluate the sensitivity of tuberculin skintest (purified protein derivative-PPD) by topical zinc application on test site to improve diagnostic reliability. METHODS: We performed this study in 100 children aged 6-14 years, and plasma zinc levels were analyzed after 10-12 hours fasting. After PPD, we applied 40% zinc oxide cream on one forearm and placebo on the other forearm. PPD indurations were measured 72 hours later. RESULTS: In this study, 26% of the children showed increases in PPD induration following local zinc applications. There was no correlation between indurations size and serum zinc levels. CONCLUSION: We concluded that topical zinc cream application can enhance sensitivity of tuberculin reactivityin the diagnosis of tuberculosis.

Association of glycosylated hemoglobin (HbA1c) levels with Iinsulin resistance in obese children.

OBJECTIVES: We investigated the relationship between insulin resistance reflected by homeostasis model assessment (HOMA-IR) index and serum HbA1c levels of obese children. MATERIAL AND METHODS: This study included 70 obese and 60 normal weight healthy children between the ages of 3 and 15. Anthropometric measures and biochemical tests (fasting glucose, fasting insulin, HbA1c) were performed on all subjects. Plasma glucose levels were measured by the glucose oxidase method. Plasma insulin concentrations were measured by radioimmunoassay (RIA). HOMA-IR index was used to estimate insulin resistance. A cut-off HOMA-IR level of ≥2.5 was accepted. The HbA1c analysis was performed using high-pressure liquid chromatography. The statistical analysis was performed using SPSS 5. Student's unpaired t-test and the Mann-Whitney U test were used to determine statistical significance. RESULTS: Gender distribution did not reveal significant difference among the obese (F: 48.6%, M: 51.4%) and the non-obese (F: 46.7%, M: 53.3%) groups. The mean age value was significantly higher in the obese group (10.09 ± 3.09) (p > 0.005) than the non-obese group (8.31 ± 3.14) (p < 0.05). The mean value of body mass index (BMI) was 25.55 ± 4.3 in the obese group and 16.63 ± 2.3 in the non-obese group. The mean HOMA-IR values of obese group (2.84 ± 1.77) was significantly higher than the non-obese group (1.50 ± 0.95) (p < 0.005). Insulin resistance was significantly higher in the obese group. Subjects with HOMA-IR ≥2.5 levels in the obese group had significantly higher HbA1c values than those with HOMA-IR <2.5 levels. CONCLUSIONS: High HbA1c levels in obese children can be used as a screening tool to detect insulin sensitivity and resistance at an early stage.