Clinical characteristics of children with asthma exacerbations: a cross-sectional descriptive study.

AIM: In this cross-sectional descriptive study, we aimed to determine the clinical characteristics of children admitted to a tertiary hospital with asthma exacerbations in a city in southern Turkey where aeroallergens are common and to determine how these characteristics affect the severity of exacerbations. METHODS: Data from a cross-sectional analysis of children with asthma exacerbations who were followed up at the Cukurova University Medical Faculty Pediatric Emergency Department (ED) and Pediatric Allergy & Immunology inpatient clinic were retrospectively analyzed. The study included 106 children who were diagnosed with asthma and did not have any additional comorbidities. In a comparative analysis, the clinical characteristics and laboratory parameters of children with mild/moderate and severe exacerbations were examined. RESULTS: While 81.1% of the patients had mild/moderate exacerbation, 18.8% had severe exacerbation. Additional atopic disease, Alternaria positivity in the skin prick test, the frequency of exacerbations in the previous year, Streptococcus pneumoniae infection, and the rate of noncompliance with treatment were significantly higher in children with severe asthma exacerbations. PEF, FEV1, and FEV1/FVC values were considerably lower in patients with severe exacerbations. CONCLUSIONS: Bacterial infections, presence of atopic disease, Alternaria exposure, low spirometric measures, number of exacerbations in the previous year, and low rate of treatment adherence may be relevant in predicting the severity of asthma exacerbations.

Therapeutic modalities and clinical outcomes in a large cohort with LRBA deficiency and CTLA4 insufficiency.

BACKGROUND: LPS-responsive beige-like anchor (LRBA) deficiency (LRBA-/-) and cytotoxic T-lymphocyte-associated antigen-4 (CTLA4) insufficiency (CTLA4+/-) are mechanistically overlapped diseases presenting with recurrent infections and autoimmunity. The effectiveness of different treatment regimens remains unknown. OBJECTIVE: Our aim was to determine the comparative efficacy and long-term outcome of therapy with immunosuppressants, CTLA4-immunoglobulin (abatacept), and hematopoietic stem cell transplantation (HSCT) in a single-country multicenter cohort of 98 patients with a 5-year median follow-up. METHODS: The 98 patients (63 LRBA-/- and 35 CTLA4+/-) were followed and evaluated at baseline and every 6 months for clinical manifestations and response to the respective therapies. RESULTS: The LRBA-/- patients exhibited a more severe disease course than did the CTLA4+/- patients, requiring more immunosuppressants, abatacept, and HSCT to control their symptoms. Among the 58 patients who received abatacept as either a primary or rescue therapy, sustained complete control was achieved in 46 (79.3%) without severe side effects. In contrast, most patients who received immunosuppressants as primary therapy (n = 61) showed either partial or no disease control (72.1%), necessitating additional immunosuppressants, abatacept, or transplantation. Patients with partial or no response to abatacept (n = 12) had longer disease activity before abatacept therapy, with higher organ involvement and poorer disease outcomes than those with a complete response. HSCT was performed in 14 LRBA-/- patients; 9 patients (64.2%) showed complete remission, and 3 (21.3%) continued to receive immunosuppressants after transplantation. HSCT and abatacept therapy gave rise to similar probabilities of survival. CONCLUSIONS: Abatacept is superior to immunosuppressants in controlling disease manifestations over the long term, especially when started early, and it may provide a safe and effective therapeutic alternative to transplantation.

Clinical findings of patients with cystic fibrosis according to newborn screening results.

BACKGROUND: Cystic fibrosis (CF) is a lethal recessive genetic disease caused by loss of function associated with mutations in the CF trans-membrane conductance regulator. It is highly prevalent (approximately 1 in 3,500) in Caucasians. The aim of this study was to compare demographic and clinical features, diagnostic tests, treatments, and complications of patients with CF whose newborn screening (NBS) with twice-repeated immune reactive trypsinogen testing was positive, normal, and not performed. METHODS: In this study, 359 of all 1,488 CF patients recorded in the CF Registry of Turkey in 2018, who had been born through the process of NBS, were evaluated. Demographic and clinical features were compared in patients diagnosed with positive NBS (Group 1), normal (Group 2), or without NBS (Group 3). RESULTS: In Group 1, there were 299 patients, in Group 2, there were 40 patients, and in Group 3, there were 20 patients. Among all patients, the median age at diagnosis was 0.17 years. The median age at diagnosis was higher in Groups 2 and 3 than in Group 1 (P = 0.001). Fecal elastase results were higher in Group 2 (P = 0.033). The weight z-score was lower and chronic Staphylococcus aureus infection was more common in Group 3 (P = 0.017, P = 0.004, respectively). CONCLUSIONS: Frequency of growth retardation and chronic S. aureus infection can be reduced with an early diagnosis using NBS. In the presence of clinical suspicion in patients with normal NBS, further analyses such as genetic testing should be performed, especially to prevent missing patients with severe mutations.

COmparisoN of TastES of Available NutriTional integrated or non-integrated formulas for infants older than 1 year of age with cow's milk allergy: A multicenter, prospective, single blind, cross-sectional observational clinical study (CONTEST Study).

Palatability of the infant formulas lacking cow milk protein formulas is reported by parents to be an important drawback. The purpose of this study is to examine decisions made by mothers of infants having cow milk protein allergy, and physicians concerning the palatability of unflavored extensively hydrolyzed formulas and amino acid-based formulas. We conducted a multi-center, randomized, single-blinded, observational taste study involving 149 pediatricians from gastroenterology and allergy subspecialties at 14 tertiary healthcare units from different regions of Turkey and involving 94 mothers of infants with cow milk protein allergy. Blinding was performed for seven formulas available in the market, which were the most commonly prescribed for feeding: four AAFs (Neocate-Numil®, Aptamil Pregomin AS-Numil®, Alfamino-Nestle®, Comidagen-Mamma®), one AAF specifically designed to address the growing nutritional and lifestyle needs of children >1 year (Neocate Junior-Numil®), 2 eHFs (Bebelac Pepti Junior-Numil®, Similac Alimentum-Abott®). Considering all three formula characteristics, Neocate junior-Numil® ranked as the number 1 product among seven products by mothers (63.8%) and physicians (69.8%). The ratings of mothers were significantly higher than the physicians (8.1 points and 6.1 points, respectively; p < 0.001). No difference was found in terms of taste, smell, and appearance for Neocate junior-Numil® between the mothers' and physicians' ratings. Since caregivers have responsibility for careful selection of replacement products for infants with cow milk protein allergy, it is noteworthy that increased awareness and confidence in the palatability characteristics of these products should motivate mothers and physicians to comply with replacement treatment in the long term.

Clinical phenotypes of childhood food allergies based on immune mechanisms: A multicenter study.

Background: Food allergies (FA) are an important public health concern that place a major burden on the lives of children and their families. The complex pathogenesis of FAs results in multisystemic and heterogenous clinical presentations. Objective: To evaluate, according to immune mechanisms, the characteristics and risk factors of childhood FA in Turkey. Methods: This descriptive multicenter study included 1248 children with FA, aged < 18 years,, who were evaluated by pediatric allergists in 26 different centers. Results: Immune mechanisms of FA were immunoglobulin E (IgE) mediated in 71.8%, non-IgE mediated in 15.5%, and mixed IgE/non-IgE mediated in 12.7% of the patients. An episode of anaphylaxis had occurred in 17.6% of IgE-mediated FA. The most common food allergens were classified into five categories (in order of decreasing frequency): cow's milk, egg, tree nuts and/or peanut, wheat, and seafood. Allergies to cow's milk and egg declined significantly with age, whereas tree nuts and/or peanut allergies increased with age. The 0-2 year age group accounted for 62.5% of the cases. The most frequent cause of FA and food anaphylaxis was cow's milk before age 13 years and tree nuts and/or peanut during adolescence (ages 13-18 years). Compared with other phenotypes, male sex (odds ratio [OR] 1.486; p = 0.032), sibling(s) (OR 1.581; p = 0.021), and maternal atopy (OR 1.531; p = 0.045) increased the likelihood of IgE-mediated FA, whereas high household income (OR 1.862; p = 0.026) increased the likelihood of non-IgE-mediated FA in multivariate regression analysis. Conclusion: This study showed that the clinical findings of FA were highly variable, depending on age and underlying immune mechanism. Knowing the population characteristics will enable better management of FA in children.

Experience of Autoimmune and autoinflammatory diseases in a Turkish pediatric cohort with primary immunodeficiencies.

BACKGROUND: Primary immunodeficiency diseases (PID) are the diseases characterized by a dysfunction of the immune system. Affected patients share a different phenotype such as chronic infections, allergy, autoimmunity, and autoinflammation. METHODS: In all, 433 children with PID were enrolled in this study. Clinical, laboratory, and demographic data of patients were reviewed retrospectively to investigate autoimmune and autoinflammatory complications. Autoinflammation in all patients with inflammation was confirmed by genetic analysis after excluding infectious etiology. RESULTS: Clinical features of 433 PID patients were evaluated retrospectively with long-term follow-up. Autoimmune disorders were identified in 69 (15.9%) patients with PID; 31 (45%) patients had a history of autoimmune disease before diagnosis of PID. The frequency of autoimmunity in immune dysregulation subgroup (76.6%) was higher than other forms of PID. The most common autoimmune manifestations were reported to be Addison's disease, hypoparathyroidism, and autoimmune hemolytic anemia. Autoinflammation were identified in 22 of the 433 (5.1%) patients with PID, including hyper immunoglobulin D syndrome (n = 9), Aicardi-Goutieres syndrome 1 (n = 6), adenosine deaminase 2 deficiency (n = 3), Blau syndrome (n = 2), tumor necrosis factor (TNF) receptor-associated periodic syndrome (n = 1), and auto-inflammation and phospholipase Cγ2-associated antibody deficiency and immune dysregulation syndrome (n = 1). CONCLUSIONS: It is important to recognize association between autoimmunity, autoinflammation, and PID, which in the future could be useful for increased awareness and early diagnosis for these diseases.

Twenty-Year Experience of a Single Referral Center on Pediatric Familial Mediterranean Fever: What Has Changed Over the Last Decade?

BACKGROUND/OBJECTIVE: Familial Mediterranean fever (FMF) is the most common autoinflammatory disease and is characterized by recurrent fever and serositis episodes. We aimed to share our 20-year FMF experience, clarify a phenotype-genotype correlation, and compare the characteristics and outcomes of pediatric FMF patients over the last 2 decades in this study. METHODS: This medical record review study included 714 pediatric FMF patients (340 females, 374 males), diagnosed by Tel Hashomer diagnostic criteria between January 2009 and January 2019 and followed up in our department. Demographic and disease characteristics, obtained from medical records of the patients, were compared between patients with M694V homozygosity and other genotypes and showed whether they were diagnosed before (n = 137) or after January 2010 (n = 577). χ2, Student t, and Mann-Whitney U tests were used to compare categorical and continuous variables between these groups. RESULTS: The most common symptoms were abdominal pain (92%), fever (89.5%), and arthralgia (64.5%). Mean ages at symptom onset and diagnosis were 5.16 ± 3.73 and 7.71 ± 3.87 years, respectively. M694V homozygosity was recorded in 111 patients (15.5%). Fever, arthralgia, arthritis, myalgia, erysipela-like erythema, colchicine resistance, and subclinical inflammation were more frequent, and mean disease severity score was higher in patients with M694V homozygosity. Fever, chest pain, and proteinuria were statistically more frequent in patients diagnosed before January 2010. Although M694V homozygosity rate was similar, patients diagnosed in the last decade had lower mean disease severity score. CONCLUSIONS: With this study, we speculate that although genotype and delay in diagnosis were similar, patients diagnosed in the last decade have a milder disease severity.

Clinical manifestations and outcomes of 420 children with Henoch Schönlein Purpura from a single referral center from Turkey: A three-year experience.

Objectives: Henoch Schönlein Purpura (HSP) is the most common systemic vasculitis in childhood. We aimed to evaluate the clinical features, seasonal variation, treatment outcomes and the possible predicting factors related to outcome among a large cohort of pediatric HSP patients.Methods: We conducted a medical record review study between July 2016 and January 2019 and evaluated the clinical manifestations and potential risk factors for severe gastrointestinal (GI) involvement, biopsy-proven nephritis and relapses.Results: The study included 420 HSP patients, of which the mean age at diagnosis was 7.68 ± 3.15 years. Clinical manifestations were arthralgia and/or arthritis (n = 244, 58.1%), abdominal pain (n = 235, 56%), subcutaneous edema (n = 163, 38.8%), and renal involvement (n = 125, 29.8%). Disease recurred for at least once, in 69 (16.4%) patients and colchicine treatment yielded a favorable response in 11 of 12 relapsing patients, who did not respond to ibuprofen or steroids. Frequencies of renal involvement and biopsy-proven nephritis were higher in patients with severe GI involvement. Besides, patients with biopsy-proven nephritis had higher rates of abdominal pain, intussusception, severe GI involvement, and systemic steroid administration.Conclusion: We speculate that renal involvement, biopsy-proven nephritis and severe GI involvement can be related to each other. Colchicine may be effective in patients with relapsing disease.

Retrospective analyzes of adverse events during biologic agents in children with juvenile idiopathic arthritis from a single center in Turkey.

OBJECTIVES: Juvenile idiopathic arthritis is the most common rheumatic disease in childhood. Biologic agents have changed the course of juvenile idiopathic arthritis. However, there are concerns regarding the occurrence of serious adverse events in patients receiving biologic agents. The aim of this study was to evaluate adverse events in children with juvenile idiopathic arthritis receiving biologic agents. MATERIAL AND METHODS: This retrospective study includes juvenile idiopathic arthritis patients receiving biologic agents. Demographic features and adverse events during biologic agents were collected from medical files. Adverse events that either resulted in death, were life-threatening, required inpatient hospitalization, or resulted in persistent or significant disability/incapacity were considered as serious adverse events. RESULTS: In total, 162 juvenile idiopathic arthritis patients (55.6% female) receiving biologic agents were enrolled: 101 (62.3%) patients treated with etanercept, 27 (16.7) with tocilizumab, 14 (8.6%) with adalimumab, 15 (9.2%) with anti-interleukin 1 agents (13 canakinumab, 2 anakinra), and 5 (3.1%) with infliximab. 75.9% of the patients received concomitantly disease-modifying anti-rheumatic drugs, and 20.4% received disease-modifying anti-rheumatic drugs plus corticosteroid. The mean age at initiation of the biologic agent was 10.5 ±4.3 years. The mean age at the study enrolment was 12.1 ±4.5 years. The mean follow-up duration was 19.7 ±2.1 months. The most frequent adverse event was upper respiratory tract infections (54.3%) followed by urinary tract infections (21%). Anaphylaxis occurred in 3 patients (1.9%): 2 with tocilizumab and one with infliximab. Macrophage activation syndrome occurred in 1 patient (0.6%) receiving tocilizumab. Lung tuberculosis developed in 2 patients (1.2%) receiving canakinumab. The frequency of serious adverse events in total was 6.7%. CONCLUSIONS: While the most frequent adverse events during biologic agents was upper respiratory tract infections, the frequency of serious adverse events was 6.7%; therefore, juvenile idiopathic arthritis patients receiving biologic agents should be carefully evaluated for these adverse events in clinical practice.

Is Henoch-Schönlein purpura a susceptibility factor for functional gastrointestinal disorders in children?

Henoch-Schönlein purpura (HSP), the most common childhood vasculitis is characterized by non-thrombocytopenic palpable purpura, arthritis/arthralgia, abdominal pain and renal involvement. Functional gastrointestinal disorders (FGIDs) are heterogeneous disease spectrum with unclear etiology and include the most common subtypes: functional dyspepsia, irritable bowel syndrome (IBS), functional abdominal pain and functional constipation. Formerly, FGIDs were known as non-organic disorders; however, recent advances revealed that low-grade inflammation may also play a role. We aimed to clarify whether HSP predisposes to FGIDs in pediatric population. Seventy-four children with HSP, diagnosed at least 6 months before the study and 78 healthy controls were enrolled to the study. Patients with red flag signs for organic GI disorders were excluded. Rome IV criteria were utilized for FGIDs diagnosis. We compared the frequencies of FGIDs between HSP patients and healthy subjects. We also examined the parameters including age, abdominal pain, arthralgia, bloody stool, renal involvement and treatment with corticosteroids and laboratory results at HSP diagnosis such as erythrocyte sedimentation rate, C-reactive protein, hemoglobin, leukocytes and platelet counts among patients with and without FGIDs. Overall FGIDs and IBS frequency were 35.1% (n = 26) and 10.8% (n = 8) in HSP patients, 19.2% (n = 15) and 2.6% (n = 2) in healthy controls, respectively. Disease characteristics and laboratory parameters at disease onset were similar between HSP patients with and without FGIDs. Overall FGIDs rate, particularly IBS were statistically higher in HSP patients. We speculate that children with preceding HSP may be predisposed to FGIDs. Since the FGIDs pathogenesis is still remains unclear, further studies are needed to confirm this hypothesis and clarify the etiology. Physicians also should pay attention to FGIDs in HSP patients with ongoing abdominal pain and thus prevent this comorbidity with dietary and psychologic measures.

Symptomatic multifocal avascular necrosis in an adolescent with neuropsychiatric systemic lupus erythematosus.

Systemic lupus erythematosus (SLE) is an autoimmune disorder characterized by malar rash, oral ulcers, arthralgia, photosensitivity and nephritis. Herein, we report a rare comorbidity, multiple avascular necrosis (AVN), in an adolescent SLE patient and also highlight the importance of risk factors for this comorbidity with a brief literature review. A 13-year-old female patient was admitted with severe headache, visual plus auditory hallucinations, polyarthritis and a history of recurrent oral ulcers. Acneiform malar rash, arthritis, cytopenia, low complement levels and autoantibody positivity yielded SLE diagnosis. We diagnosed her as having multifocal AVN after the 4th dose of cyclophosphamide, with bilateral knee pain and swelling and typical geographical lesions on magnetic resonance imaging. Avascular necrosis is a rare comorbidity of SLE and neuropsychiatric involvement, cyclophosphamide administration and severe disease may be the possible risk factors in addition to corticosteroid use. Further multicenter studies investigating the possible risk factors of AVN with a large number of patients are needed.

Decreased serum vitamin B12 and vitamin D levels affect sleep quality in children with familial Mediterranean fever.

Familial Mediterranean fever is an autoinflammatory disorder characterized by recurrent fever and serositis. We aimed to describe serum vitamin B12 and vitamin D levels and their correlation with self-reported sleep quality of pediatric FMF patients. Sixty-three children with FMF were enrolled to the study. Information on sleep quality was obtained using self-administration of Pittsburg Sleep Quality Index (PSQI). The patients were divided into subgroups depending on vitamin D serum concentrations: ≥ 20 and < 20 ng/ml. We also grouped patients according to their serum B12 concentration: ≥ 200, < 200 pg/ml. B12 levels were not correlated with PSQI scores, whereas significant correlation was found between vitamin D and total PSQI scores and daytime sleepiness. Total PSQI score, sleep disorders and daytime sleepiness sub-scores were statistically high in patients with serum D vitamin levels below 20 ng/ml. Sleep efficiency was found lower in patients with serum B12 levels below 200 pg/ml. B12 may have a positive role on effective sleep. More importantly, we suggest that vitamin D is protective against sleep disorders and poor sleep, it may also improve daytime activities.

The Trend of Change of Allergic Diseases over the Years: Three Repeated Surveys from 1994 to 2014.

BACKGROUND: Although it is known that there has been an increase in asthma and allergic diseases among school-aged children, results vary between countries. The aim of this study was to examine trends in the prevalence of these diseases in the city of Adana (south Turkey) over the last 20 years based on the results of 3 cross-sectional studies. METHODS: Three cross-sectional surveys were performed 10 years apart in south Turkey. Here, we compare the surveys conducted among children between the ages of 6 and 14 years from 1994, 2004, and 2014. The participants were randomly chosen among children attending participating primary schools in Adana. The questionnaire in the first survey contained almost the same questions as the ISAAC. In the 2 latter surveys, we used the ISAAC questionnaire to investigate the prevalence of asthma and other allergic diseases. RESULTS: A total of 2,334 children in 1994, 3,728 children in 2004, and 3,209 children in 2014 were included in the final analysis. The results showed a statistically significant increase in the prevalence of physician-diagnosed allergic diseases across the years, respectively: asthma (5, 4.6, and 8.9%), allergic rhinitis (8.8, 11.4, and 15.6%), and atopic dermatitis (5, 9.9, and 7%). In contrast, the prevalence of parent-reported current wheeze (16.2, 13.2%) decreased in the last 10 years. CONCLUSIONS: Our findings were consistent with the increased prevalence of childhood asthma and other allergic diseases. However, this pattern did not occur in respiratory symptoms.

The ADAPAR Birth Cohort Study: Food Allergy Results at Five Years and New Insights.

BACKGROUND: Although food allergy (FA) is often a transient condition during childhood, when and in whom FA will resolve can be affected by many factors. In this study, we analyzed the data at 5 years on 33 children diagnosed with FA in the ADAPAR (Adana Pediatric Allergy Research) birth cohort study in southern Turkey. METHODS: Thirty-three infants detected as having FA at the end of their first year in the ADAPAR study were assessed every 6 months until the age of 5 years. Each follow-up included a clinical examination, questionnaire, blood sampling and a skin-prick test. RESULTS: Culprit allergens were cow's milk (n = 20), eggs (n = 17), chicken meat (n = 1) and bananas (n = 1). Of the 17 patients with egg allergy, 14 developed complete tolerance and 1 developed partial tolerance (i.e. tolerance to baked food). Of the 20 patients with milk allergy, complete tolerance was observed in 16 and partial tolerance in 1. The mean age of tolerance to egg was 22.4 ± 7.5 months and to cow's milk, it was 20.9 ± 1.1 months. Complete tolerance developed in 1 case allergic to chicken meat and in 1 case allergic to banana. Other allergic conditions were also determined: allergic rhinitis in 27.2%, atopic dermatitis in 21.2%, asthma in 9%, urticaria in 9% and drugs in 9%. CONCLUSIONS: Our results confirm early and high tolerance rates before school age in children with food allergies that started in infancy. This will help pediatricians to give more informed advice to parents of infants with cow's milk or hen's egg allergy.

Clinical Features of Food Allergy during the 1st Year of Life: The ADAPAR Birth Cohort Study.

BACKGROUND: Although food allergies (FAs) occur most commonly during the 1st year of life, there is limited information available regarding the epidemiology of FAs. In this study, we investigated the incidence of FA and the associated risk factors during the 1st year of life in southeast Turkey. METHODS: This study is a prospective evaluation of 1,377 infants born at the Balcali Hospital (Çukurova University) and includes four routine follow-up assessments until the age of 1 year. At birth, a physical examination was performed, cord blood samples were taken, and parents completed a baseline questionnaire. Follow-up visits were scheduled at 3, 6 and 12 months and included the infants' physical examination and a follow-up questionnaire. A skin prick test (SPT) was performed and food-specific IgE levels were measured at 6 and 12 months. Telephone interviews were conducted when the infants were 9 months of age, and the questionnaire was administered. The diagnosis of FA was based on food-specific IgE levels, positive SPT results, associated clinical findings and an oral food challenge (OFC) test. RESULTS: Patient histories, physical examinations and laboratory results indicated a possible FA in 90 infants (6.5%) during the 1st year of life. All of them underwent OFC testing with the suspected foods, and FA was confirmed in 33 cases (2.4%). Cow's milk allergy was the major cause of FA. Skin reactions were major clinical findings in FA. A family history of atopy was identified as the major risk factor for FA. CONCLUSIONS: The prevalence and risk factors of FA in our region are consistent with those reported in the literature.

Correlation between nasal eosinophils and nasal airflows in children with asthma and/or rhinitis monosensitised to house dust mites.

BACKGROUND: Allergic rhinitis and asthma due to mite sensitisation are diseases which are frequently associated and characterised by persistent inflammation. In the present study, we aimed to investigate the relationship between nasal airflows and nasal eosinophils in patients with asthma and/or rhinitis due to house dust mite sensitisation. METHODS: Twenty-four children with both rhinitis and asthma (R+A), 13 children with rhinitis and no asthma (R) and 10 non-allergic healthy children were evaluated prospectively. The patients belonging to the first two groups had moderate-severe grade of nasal obstruction. Total nasal symptom scores, peak nasal inspiratory flows (PNIFs) obtained by anterior rhinomanometry, skin prick tests, nasal eosinophils and FEV1 values were all assessed. RESULTS: Percentages of nasal eosinophils and PNIFs in patients with R+A and R (r=-0.415, p=0.04) were found to be statistically significant and to have an inverse correlation. Skin prick tests were also significantly correlated with nasal eosinophils and PNIFs (r=0.372, p=0.01 and r=-0.306, p=0.04, respectively). Both PNIFs and nasal eosinophils of patients with R+A were significantly correlated with FEV1 values (r=-0.641, p=0.001 and r=0.548, p=0.007, respectively). CONCLUSION: In this study, a close relationship was demonstrated between eosinophil infiltration and nasal airflows in children having asthma and/or rhinitis monosensitised to mites. Additionally, the significant association found between FEV1 values and nasal eosinophils or PNIFs supported the close link of upper and lower airways.

Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators.

INTRODUCTION: Cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs target the underlying defect and improve CFTR function. They are a part of standard care in many countries, but not all patients are eligible for these drugs due to age and genotype. Here, we aimed to determine the characteristics of non-eligible patients for CFTR modulators in the CF registry of Turkey (CFRT) to highlight their clinical needs. METHODS: This retrospective cohort study included CF patient data from the CFRT in 2021. The decision of eligibility for the CFTR modulator was determined according to the 'Vertex treatment-Finder' on the Vertex® website. Demographic and clinical characteristics of patients were compared between eligible (group 1) and ineligible (group 2) groups for CFTR modulators. RESULTS: Among the study population (N = 1527), 873 (57.2%) were in group 1 and 654 (42.8%) were in group 2. There was no statistical difference between groups regarding sex, meconium ileus history, diagnoses via newborn screening, FEV1 z-score, CF-associated complications, organ transplant history, and death. Patients in group 2 had a higher incidence of pancreatic insufficiency (87.7% vs. 83.2%, p = .010), lower median height z-scores (-0.87 vs. -0.55, p < .001), lower median body mass index z-scores (-0.65 vs. -0.50, p < .001), longer days receiving antibiotics due to pulmonary exacerbation (0 [interquartile range, IQR: 0-2] vs. 0 [IQR: 0-7], p = 0.001), and more non-invasive ventilation support (2.6% vs. 0.9%, p = 0.008) than patients in group 1. CONCLUSION: The ineligible group had worse clinical outcomes than the eligible group. This highlights their need for life-changing drugs to improve clinical outcomes.

Two year follow-up of clinical and inflammation parameters in children monosensitized to mites undergoing subcutaneous and sublingual immunotherapy.

BACKGROUND: Both SCIT (subcutaneous immunotherapy) and SLIT (sublingual immunotherapy) have clinical and immunologic efficacy in children with rhinitis and asthma but comparative studies are scarce. OBJECTIVE: To investigate the clinical and immunological efficacy of mite-specific SLIT and SCIT in children with rhinitis and asthma. METHOD: Thirty children monosensitized to house dust mite were randomized to receive either active SCIT or SLIT or placebo for 1 yr in a double-blind double-dummy placebo controlled design (Yukselen A et al., Int Arch Allergy Immunol 2012; 157:288-298). Thereafter, the placebo group was randomized to receive SCIT or SLIT, and for 1 yr all patients received active treatment with SCIT or SLIT. Symptom scores, drug usage, titrated skin prick tests, nasal and bronchial allergen provocation doses, serum house dust mite-specific immunglobulin E, sIgG4, IL-10 and IFN- g levels were evaluated. RESULTS: The reduction of clinical scores with SLIT was more evident after 2 years of treatment in comparison to both the baseline and DBPC phase of the study. The change in titrated skin prick tests and nasal provocative doses was more prominent with both SCIT and SLIT at the end of the open phase. Although the increase inbronchial provocative doses was not significant at the end of the first year of treatment with SLIT, it reached a statistically significant difference after two years of treatment. CONCLUSION: The clinical efficacy of SLIT is more prominent at the end of the second year, although this improvement is observed from the first year of treatment with SCIT in mite-sensitive children.

Clinical characteristics of patients requiring lung transplantation referral in national cystic fibrosis registry data.

BACKGROUND: We aimed to determine the number of cystic fibrosis (CF) patients recorded in the Cystic Fibrosis Registry of Türkiye (CFRT) who were in need of lung transplantation (LT) referral and examine clinical differences between patients who were LT candidates due to rapid forced expiratory volume in one second (FEV1) decline and LT candidates without rapid FEV1 decline in the last year to identify a preventable cause in patients with such rapid FEV1 decline. METHODS: All CF patients recorded in the CFRT in 2018 were evaluated in terms of LT. Patients were divided into those with FEV1 below 50% and in need of LT due to a decrease of 20% or more in the previous year (Group 1) and those who did not have FEV1 decline of more than 20% in the previous year but had other indications for LT (Group 2). Demographic and clinical features were compared between the two groups. RESULTS: Of 1488 patients registered in CFRT, 58 had a need for LT. Twenty patients were included in Group 1 and others in Group 2. Our findings did not reveal any significant variations in treatment, chronic infection status, or complications between the two groups. The average weight z-score was significantly higher in Group 1. Positive correlations were detected between weight z-score and FEV1 in 2017 in Group 1 and between FEV1 values in 2017 and 2018 in Group 2. CONCLUSIONS: There appears to be a relationship between the nutritional status and weight z-scores of CF patients and pulmonary function, which may indirectly affect the need for lung transplantation referral.