An ongoing problem: Rhesus hemolytic disease of the newborn - A decade of experience in a single centre.

BACKGROUND: The objectives were to evaluate the descriptive features of newborns with a diagnosis of Rhesus (Rh) hemolytic disease, to determine the morbidity and mortality rates, to evaluate the treatment methods and the factors affecting treatment requirements and clinical outcomes during a ten-year period at a tertiary center. METHODS: Newborn infants who had a positive direct Coombs test and/or had a history of intrauterine transfusion (IUT) due to Rh hemolytic disease were included. The data regarding the prenatal, natal and postnatal periods were collected from hospital records. RESULTS: A total of 260 neonates were included of which 51.2% were female. The mean ± standard deviation gestational age was 36.9 ± 2.7 weeks. The rate of preterm birth was 41.2%. Of 257 mothers whose obstetric medical history could be accessed, 87.2% were multigravida, whereas 76.3% were multiparous. Among mothers who had a reliable history of anti-D immunoglobulin prophylaxis (n=191), 51.3% had not received anti-D immunoglobulin prophylaxis in their previous pregnancies. The antenatal transfusion rate was 31.7% and the frequency of hydrops fetalis was 8.8%. While combined exchange transfusion (ET) and phototherapy (PT) was performed in 15.4% of the babies, the majority either needed phototherapy only (51.1%) or no treatment (33.5%). The mortality rate was 3.8 % (n = 10), and nine babies out of these 10 were those with severe hydrops fetalis. CONCLUSION: This study showed that Rh hemolytic disease is still a major problem in developing countries. Multiple comorbidities may occur in addition to life threatening complications, including hydrops fetalis, anemia and severe hyperbilirubinemia. High rates of multiparity and low rates of anti-D immunoglobulin prophylaxis are potential barriers for the eradication of the disease. It should be remembered that Rh hemolytic disease is a preventable disease in the presence of appropriate antenatal follow-up and care facilities.

Electronic home monitoring of children with cystic fibrosis to detect and treat acute pulmonary exacerbations and its effect on 1-year FEV1.

BACKGROUND: We aimed to investigate the effect of the use of electronic home spirometry in children with cystic fibrosis (CF) on 1-year FEV1 (% predicted, pp) change. METHODS: This is a randomised, one-year prospective study including children with CF between 6 and 18 years of age. Subjects were randomised into home spirometry group (HSG) and usual care group (UCG). Children in HSG performed two pulmonary function tests (PFT) per week. Data regarding acute pulmonary exacerbations (PEx) was obtained from patients' records. At baseline and 12th month, health related quality of life questionnaire for CF patients (CFQ-R) and lung clearance index (LCI) were performed. RESULTS: Sixty children were recruited with a median (IQR) age of 13.3 (11.4-15.4) years. Absolute change in FEV1pp from baseline to 12th month as median (IQR) was +1% (-6.75-9.75) in HSG and -2.50% (-7.50-3.25) in UCG (p = 0.10). Sensitivity analysis including only adherent children in HSG (n = 22), yielded an increase of 5% (-3.50-12) in HSG and a decrease of 2.50% (-7.50-3.25) in UCG (p = 0.009). A total of 29 (96.7%) subjects in HSG and 23 (76.7%) in UCG had PEx (p = 0.05). Absolute change in median (IQR) LCI2.5 from baseline to the 12th month was -1.6 [-2.9-0] (p<0.001) in HSG and -1.5 [-2.8-(-0.6)] (p<0.001) in UCG (p = 0.94). There was a significant increase in the social domain of the CFQ-R in HSG (from 59.1 to 76.2, p = 0.01). CONCLUSIONS: Electronic home monitoring of children with CF by spirometry may result in improvement in lung function.

Implementation of standardized cystic fibrosis care algorithm to improve the center data-quality improvement project international collaboration.

BACKGROUND: A collaboration between the University of Michigan (U of M) Cystic Fibrosis Center (CFC) and Marmara University (MU) CFC was initiated to improve the health status of people with cystic fibrosis (pwCF) at MU through implementing Quality Improvement (QI) initiatives. The main aim was to improve lung function in children with FEV1pp <80. The secondary aim was to assess the changes in health related quality of life. METHODS: Included in the project were pwCF who received cystic fibrosis (CF) care at the MU CFC and were 6-18 years of age with an FEV1pp <80. Flow charts were created and a standardized CF care algorithm was implemented. Weekly case review were done to develop individualized treatment plans. Appropriate intervention was applied and patient data were assessed at baseline, 3, 6, 9 and 12 months. The Cystic Fibrosis Revised Questionnaire (CFQ-R) was completed. RESULTS: 55 pwCF were included (mean age:11.8 ± 3.3 years). Mean FEV1pp (SD) at baseline, 6 and 12 month was 63.7 (14.6), 66.9 (16.6), 70.4 (19.2), respectively, with a relative increase of 5.0% in 6 months (p:0.002) and 10.5% in 12 months compared to baseline (p<0.001). Physical functioning, eating problems and respiratory symptoms domains of the CFQ-R questionnaire were improved at the end of the one year for 6-13 (p = 0.024, p = 0.009, p = 0.002) and 13-18 year olds (p = 0.013, p = 0.002, p = 0.038). CONCLUSION: There was significant improvement in pwCF with FEV1<80%pp after implementing this QI project. The processes and assessments used can be adopted by other low-middle income countries to improve similar measures.

Night-to-night variability of polygraphy in children with sleep disordered breathing symptoms.

INTRODUCTION: Polygraphy (PG) can be used as an alternative test for the diagnosis of obstructive sleep apnea syndrome (OSAS) in children. Night-to-night variability of PG in children is not known. Our aim was to determine whether a single night PG was reliable for OSAS diagnosis in children with symptoms of sleep-disordered breathing (SDB). MATERIALS AND METHODS: Otherwise healthy children who had been evaluated for symptoms of SDB were included. Two nocturnal PGs were performed 2-7 days apart. Demographic and clinical characteristics, Pediatric Sleep Questionnaire, and modified Epworth Sleepiness Scale were recorded. OSAS was diagnosed if obstructive apnea-hypopnea index was (oAHI) ⩾ 1/h and classified as mild (oAHI: 1-4.9/h), moderate (oAHI: 5-9.9/h), and severe (oAHI ⩾ 10/h). RESULTS: Forty-eight patients were included (37.5% female, age 10.8 ± 3.9 years) to the study. There were no significant differences in oAHI values and other respiratory parameters between the two PGs (p > 0.05). Thirty-nine children were diagnosed with OSAS if the highest oAHI over any single night was used for diagnosis. Thirty-three of the 39 children (84.6%) were diagnosed with OSAS with the first PG while 35 of 39 (89.7%) children were diagnosed with OSAS with the second PG. There was an agreement for identifying OSAS and its severity between the two PGs in our study even though there were few individual intra-subject differences in oAHI. CONCLUSION: There was no significant first-night effect for PG in this study which suggests that a single night PG is adequate for diagnosis of OSAS in children with SDB- related symptoms.

The utility of risk assessment tools for acute pulmonary embolism in children.

BACKGROUND AND AIM: Pulmonary embolism (PE) is a potentially life-threatening disease in children. The objective of the study is to evaluate the utility of adult-based pulmonary embolism rule-out criteria (PERC), Pediatric PE Model, and D-dimer in the diagnosis of PE in children. MATERIAL AND METHODS: The study consisted of patients under 18 years of age who were consulted to the Pediatric Pulmonology Clinic for the evaluation of PE. Patients were divided into two groups based on the confirmation of PE. The group with the presence of PE (n = 20) consisted of children who were diagnosed with PE. The group with the absence of PE (n = 28) consisted of children with clinically suspected PE but negative diagnostic imaging. Adult validated clinical decision PERC rule and Pediatric PE Model were retrospectively applied to the patients. RESULTS: In the study, PERC demonstrated a sensitivity of 60% and a specificity of 46% for the diagnosis of PE in children. When PE Model was evaluated for the children, it was found a 50% sensitivity and 75% specificity. Combining PE Model and PERC rule with D-dimer did not increase the specificity and sensitivity. Smoking was found to be relevant for PE in the childhood. Twenty-five percent of the patients had a genetic tendency for PE. All of the patients had an underlying disease as well. CONCLUSION: None of the current risk assessment tools (PE Model, PERC, D-dimer) were found to be accurate in predicting PE. Further larger population studies are still required to develop a better diagnostic approach.

Respiratory outcome of spinal muscular atrophy type 1 patients treated with nusinersen.

BACKGROUND: Respiratory failure is the leading cause of mortality in spinal muscular atrophy type 1 (SMA1) children. The current study aims to evaluate the effect of nusinersen treatment on respiratory outcome of the patients with SMA1. METHODS: In this retrospective, single-center study, 52 SMA1 patients treated with nusinersen were included in the analysis. Patients were divided into two groups based on their age at the time of their first nusinersen treatment (Group 1: ≤6 months, Group 2: >6 months). Respiratory outcome on the 180th day of treatment is defined as the type of ventilation support (spontaneous breathing, noninvasive ventilation (NIV), and tracheostomized or intubated on invasive mechanical ventilation). Demographic data, respiratory outcome, and Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores were obtained from medical records. RESULTS: On the 180th day of treatment, 46 of the 52 (88.4%) children were alive. Prevalence of the mortality was similar in both groups (P = 0.65). The comparison of respiratory outcome in patients between group 1 and group 2 was as follows: spontaneous breathing, 7 (43.7%) versus 4 (13.3%) (P = 0.03); NIV <16 h/day, 3 (18.7%) versus 4 (13.3%) (P = 0.68); invasive mechanical ventilation, 6 (37.5%) versus 22 (73.3%) (P = 0.01). There were no patients using NIV ≥16 h/day. There were significant improvements in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders scores of the patients at day 180 in comparison with the baseline (P < 0.001). CONCLUSIONS: Early initiation of nusinersen treatment in SMA1 patients may alter the disease's natural course.

The effect of coronavirus disease 2019 on anxiety levels of children with cystic fibrosis and healthy peers.

BACKGROUND: Cystic fibrosis (CF) is a chronic disease causing recurrent respiratory tract infections. Viral respiratory tract infections are more severe in CF. The first case of coronavirus disease 2019 (COVID-19) was seen in Turkey on March 11, 2020, and nationwide school closure and lockdown were implemented. School closure and home confinement might have adverse effects on children's physical and mental health. In this study, we aimed to compare the effect of the COVID-19 pandemic on psychological reactions of CF patients and healthy controls. METHODS: This is a controlled cross-sectional study including 7-18-year-old children with CF. The survey included questions regarding family environment and peer relations, self-care, and psychological reactions to the COVID-19 pandemic. The questionnaire was administered to children via telephone calls under parental supervision. RESULTS: We evaluated 132 CF patients and 135 of their healthy peers. Mean age was 11.5 ± 2.9 years in the CF group and 11.8 ± 3.2 years in the control group (P = 0.98). There were 55 girls (41.7%) in the CF group and 81 girls (60%) in the control group (P = 0.027). The socioeconomic status of their families was similar. The CF patients were found to be less anxious for family members at risk of COVID-19, less upset about school closure, and less anxious about the COVID-19 pandemic (P < 0.001, 0.02, 0.01, respectively). CONCLUSIONS: Cystic fibrosis patients seem to show more resilience in coping with the pandemic. Appropriate psychological support should be provided to them and resilience strategies in coping with the pandemic should be nurtured.

The ISPAT project: Implementation of a standardized training program for caregivers of children with tracheostomy.

BACKGROUND: Tracheostomy-related morbidity and mortality mainly occur due to decannulation, misplacement, or obstruction of the tube. A standardized training can improve the skills and confidence of the caregivers in tracheostomy care (TC). OBJECTIVE: Our primary aim was to evaluate the efficiency of standardized training program on the knowledge and skills (changing-suctioning the tracheostomy tube) of the participants regarding TC. MATERIALS AND METHODS: Sixty-five caregivers of children with tracheostomy were included. First, participants were evaluated with written test about TC and participated in the practical tests. Then, they were asked to participate in a standardized training session, including theoretical and practical parts. Baseline and postintervention assessments were compared through written and practical tests conducted on the same day. RESULTS: A significant improvement was observed in the written test score after the training. The median number of correct answers of the written test including 23 questions increased 26%, from 12 to 18 (p < .001). The median number of correct steps in tracheostomy tube change (from 9 to 16 correct steps out of 16 steps, 44% increase) and suctioning the tracheostomy tube (from 9 to 17 correct steps out of 18 steps, 44% increase) also improved significantly after the training (p < .001, for both). CONCLUSION: Theoretical courses and practical hands-on-training (HOT) courses are highly effective in improving the practices in TC. A standardized training program including HOT should be implemented before discharge from the hospital. Still there is a need to assess the impact of the program on tracheostomy-related complications, morbidity, and mortality in the long term.

Comparison of intravenous and non-intravenous antibiotic regimens in eradication of P. aeruginosa and MRSA in cystic fibrosis.

BACKGROUND: Chronic pulmonary infection is the leading cause of mortality and morbidity in patients with cystic fibrosis (CF). The most common pathogens isolated in CF are Staphylococcus aureus (SA) and Pseudomonas aeruginosa (PA). Chronic infection of PA and methicillin-resistant S. aureus (MRSA) are associated with worse survival and antibiotic eradication treatment is recommended for both. This study compared the outcomes between intravenous (IV) and non-IV antibiotics in eradication of PA and MRSA. METHODS: This was a single-center retrospective study. All respiratory specimen cultures of 309 CF patients and eradication regimens between 2015 and 2019 were reviewed. Patients received eradication treatment in case of first ever isolation or new isolation after being infection-free ≥1 year. The primary analysis was the comparison of the percentage of successful eradication after receiving IV and non-IV eradication regimens. Demographic and clinical risk factors for eradication failure were also analyzed. RESULTS: One hundred and two patients with PA isolations and 48 patients with MRSA were analyzed. At 1 year, 21.6% in PA group and 35.4% in MRSA group were successfully eradicated. There was not any statistically significant difference between IV versus non-IV antibiotic regimens on eradication in either group. Additionally, none of the clinical risk factors was significantly associated with eradication failure in PA and MRSA groups. CONCLUSION: In the eradication of PA and MRSA, IV and non-IV treatment regimens did not show any superiority to one another. Non-parenteral eradication could be a better option considering the cost-effectiveness and the treatment burden of IV treatments due to hospitalization and the need for IV access.

The effects of nebulizer hygiene training on the practices of cystic fibrosis patients and caregivers.

BACKGROUND: Nebulizers can be contaminated with microorganisms and may be a source of infection in the lower airways in patients with cystic fibrosis (CF). OBJECTIVE: Primary aim of this study was to determine the level of knowledge regarding nebulizer hygiene and adherence to CF foundation infection prevention and control (IPC) measures of CF patients in our center. We also evaluated the effect of a standardized training program on nebulizer cleaning and disinfection practises with pre and posttest. METHODS: Caregivers of 173 CF patients followed at Marmara University CF Center filled a questionnaire (pretest) regarding nebulizer hygiene and received didactic education including pictures and videos based on the cystic fibrosis foundation (CFF) IPC guidelines, patients were also provided educational materials. Posttest was performed 1-3 months after the education session. RESULTS: Following standardized training, usage of appropriate methods according to CFF IPC guidelines improved significantly. Frequency of nebulizer cleaning after each use increased from 58.4% to 78% (p < .01) and disinfection frequency after each/daily usage increased from 33.6% to 75.7% (p < .01). Additionally, methods of cleaning and storage of the nebulizer, also improved significantly (p < .01, p < .01). CONCLUSION: Education was highly effective to increase the rate of proper practices for nebulizer hygiene. The necessity of cleaning, disinfection, careful drying, correct storage of the nebulizer parts, and changing the nebulizer equipment within recommended time should be emphasized to CF families regularly.

Screening of depression and anxiety in adolescents with cystic fibrosis and caregivers in Turkey by PHQ-9 and GAD-7 questionnaires.

BACKGROUND: Depression and anxiety symptoms in patients with cystic fibrosis (CF) and their caregivers are 2-3 times higher than in the normal population. This study aims to evaluate the frequency and severity of depression and anxiety symptoms and to determine possible risk factors in CF patients and their mother and/or fathers at Marmara University CF center. METHODS: The study included 132 CF patients who were followed up at our CF center. Patient Health Questionnaire (PHQ-9) and the Generalized Anxiety Disorder Questionnaire (GAD-7) were used to screen depression and anxiety. The questionnaires were completed by 50 CF patients (aged 12-17 years) and 132 parents of patients (aged 0-17 years). RESULTS: While moderate to severe depressive symptoms were seen in 26% of patients, 33.7% of mothers and 14.6% of fathers; moderate to severe anxiety were present in 18%, 21.8% and 8.5%, respectively. None of the demographic characteristics was identified as a predictor of depression or anxiety. GAD-7 scores have shown a higher prevalence of anxiety in mothers of patients with chronic methicillin-resistant Staphylococcus aureus (p = .034). Additionally, hospitalization in the last 12 months was significantly correlated with higher PHQ-9 scores in fathers (p = .043). Analysis of patients' adherence to medical treatment and airway clearance showed higher depression and anxiety in mothers of the nonadherent group (p = .002). CONCLUSION: Depression and anxiety were common in CF patients and their parents. These results illustrate the importance of depression/anxiety screening and psychosocial support for the CF patient and their parents.

Long-term respiratory outcomes of post-op congenital lung malformations.

BACKGROUND: Congenital lung malformations (CLM) are rare disorders and surgical intervention is the definitive treatment. Our aim is to evaluate the long-term lung function of patients with CLM after surgery compared to healthy children. METHODS: Sixteen children with CLM (M/F: 9/7) and 30 age-matched, healthy controls (M/F: 13/17) were included in the study. Demographic data were recorded and both groups were compared by spirometry and the nitrogen-based Lung Clearance Index (LCI). RESULTS: Mean ± SD age of the patients was 12.0 ± 5.4 years. The mean forced expiratory volume in 1 s (FEV1 ), forced vital capacity (FVC), FEV1 /FVC, and forced expiratory flow between 25% and 75% of force expiration (FEF25-75 ) predicted was, 86.68 ± 16.65, 88.00 ± 14.58, 97.44 ± 9.89, and 79.00 ± 26.41, respectively in the patient group. Patients with CLM had significantly lower values in FEV1 , FVC, FEF25-75 than healthy controls (P = 0.002, P 0.007, P 0.045). While the mean LCI2,5% value in patients' group was 8.33 ± 1.52, it was 7.28 ± 0.80 in healthy controls (P = 0.023). Strong inverse correlation between LCI and FEV1 , FEV1 /FVC was detected in the patient group (P = 0.023; r: -0.581, P 0.017; r: -0.606 respectively). CONCLUSION: This study revealed that, in long-term follow-up, patients who had surgery because of CLM have impairment in the pulmonary function compared to healthy children and LCI may be more accurate in detecting airway diseases early than spirometry.

Depression and anxiety in mothers of home ventilated children before and during COVID-19 pandemic.

BACKGROUND: Due to advances in technology, home ventilation in children has increased in recent years. The provision of proper care for a home-ventilated (HV) child can have a strong impact on the lifestyle of caregivers. The aim of this study was to evaluate the depression and anxiety levels of the mothers of HV children during the current COVID-19 pandemic and compare them to those of mothers of healthy peers. METHODS: This cross-sectional study was conducted on HV children (n = 21) and a control group of healthy peers (n = 32) by means of a questionnaire completed by the mothers of the children of both groups. Psychometric scales, such as the Beck Depression Inventory (BDI), the State-Trait Anxiety Inventory-State (STAI-S), and the State-Trait Anxiety Inventory-Trait (STAI-T), were used to assess participants. RESULTS: During the pandemic signs of depression were present in 8 (38.1%) of the case group and 8 (25%) of the healthy control group. Comparison of the BDI scores from before and during the pandemic showed no difference between mothers of the HV children (p = .09). Scores for BDI and STAI-T were higher in the case group than in the control group, whereas there was no significant difference in STAI-S scores. CONCLUSION: Depression and anxiety levels of mothers of HV children were found to be higher during the COVID-19 pandemic. Economic, medical, and social support resources are needed to reduce levels of depression and anxiety and help mothers of those children dependent on technology.

Comparison of conventional chest physiotherapy and oscillatory positive expiratory pressure therapy in primary ciliary dyskinesia.

BACKGROUND: Chest physiotherapy (CP) is a recommended treatment modality in primary ciliary dyskinesia (PCD). OBJECTIVE: Primary aim was to compare the efficacy and safety of the conventional chest physiotherapy (CCP) and oscillatory positive expiratory pressure therapy (OPEPT). Secondary aims were to compare the exacerbation rate, time until the first exacerbation, patient compliance and comfort between the two CP methods. METHODS: This is a 6 month randomized, controlled crossover trial. Patients >6 years of age with PCD were randomized into two groups, first group was assigned to OPEPT (Acapella®) for 3 months while second group was assigned to CCP. Groups were crossed over to the other modality after a 15-day washout period. Pulmonary function tests (PFTs) and compliance were monitored by monthly clinic visits. RESULTS: There was a significant increase in FEV1 , FEF25-75 , and PEF values (p = .018, p = .020, and p = .016, respectively) in the OPEPT group and in FVC values (p = .007) in CCP group compared to baseline. However PFT increase at 3rd month was not superior to each other with both physiotherapy methods. Median acute pulmonary exacerbation rate and time period until the first exacerbation were similar in both groups (p = .821, p = .092, respectively). Comfort and effectiveness of OPEPT was higher than CCP according to patients (p = .029 and p = .042, respectively). There were no adverse effects with either therapy. CONCLUSIONS: OPEPT was as effective as CCP in PCD patients. OPEPT was more comfortable and effective than CCP according to patients. OPEPT might be an efficient alternative method for airway cleareance in PCD patients.

An Adolescent Presented With Hemoptysis: Pulmonary Artery Aneurysm in Pediatric Behçet's Disease.

Behçet's disease (BD) is a rare autoimmune and chronic inflammatory vasculitis, characterized by relapsing episodes of oral aphthous and genital ulcers, skin lesions, ocular lesions and vascular involvement. Pulmonary artery involvement is rare in BD but it carries a high mortality risk. In this article, we report a 15-year-old male patient presented with a two-month history of hemoptysis, cough, fewer and weight loss. On physical examination, auscultation revealed decreased breath sounds at left lung base. Bronchoscopy showed narrowed left lower lobe bronchus due to the external compression. Computed tomography angiography revealed multiple bilateral pulmonary artery aneurysms. Pathergy test was positive and he was diagnosed with BD. BD should be considered in the differential diagnosis of childhood hemoptysis.