Broadly effective metabolic and immune recovery with C5 inhibition in CHAPLE disease.

Complement hyperactivation, angiopathic thrombosis and protein-losing enteropathy (CHAPLE disease) is a lethal disease caused by genetic loss of the complement regulatory protein CD55, leading to overactivation of complement and innate immunity together with immunodeficiency due to immunoglobulin wasting in the intestine. We report in vivo human data accumulated using the complement C5 inhibitor eculizumab for the medical treatment of patients with CHAPLE disease. We observed cessation of gastrointestinal pathology together with restoration of normal immunity and metabolism. We found that patients rapidly renormalized immunoglobulin concentrations and other serum proteins as revealed by aptamer profiling, re-established a healthy gut microbiome, discontinued immunoglobulin replacement and other treatments and exhibited catch-up growth. Thus, we show that blockade of C5 by eculizumab effectively re-establishes regulation of the innate immune complement system to substantially reduce the pathophysiological manifestations of CD55 deficiency in humans.

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.

Experiences and attitudes of parents of children with cow's milk and other food-allergy.

INTRODUCTION AND OBJECTIVE: The attitude and behaviors of parents are important in the management of children with food allergy (FA). The aim of this study is to evaluate the experiences and attitudes of parents of children with allergy to cow's milk and other FA. MATERIALS AND METHODS: The parents of children with FA were asked to complete an 18-item questionnaire to evaluate the FA history and experiences during diagnosis, treatment, and follow up. RESULTS: The data from 558 (91.2%) survey questionnaire that were filled completely were analyzed. The mean age of the parents was 33.4+4.9. It was found that most common food allergen was cow's milk (85.3%). The mean time to diagnosis from the onset of symptoms was 10.9±18.4 months. Around 229 parents (41.6%) admitted to at least four different physicians and 68 (12.3%) parents admitted to at least five different physicians before diagnosis. The median time to diagnosis from the onset of symptoms was five (1-108) months in the patients admitted to four or more physicians, but it was one (1-48) month in the patients that admitted to less physicians (p˂0.001). The most common symptoms were dermatitis and mucus-bloody stool, the least common ones were cardiovascular symptoms. Only 21.1% of the patients were able to use hypoallergenic formulas (HAF) in accordance with the recommendation of the physician. CONCLUSIONS: Delayed diagnosis of FA is a major concern, and during this period the patients admit many physicians. A majority of the patients with CMPA experience difficulties while using HAFs, and only one-fifth of them is able to use formula regularly.

Proven Food-Induced Acute Urticaria and Predictive Factors for Definitive Diagnosis in Childhood.

BACKGROUND: Urticaria can be the only sign of a food allergy or can be seen together with other signs and symptoms of a food allergy. OBJECTIVE: To determine the demographic, etiologic, and clinical features of food-induced acute urticaria in childhood. METHODS: Patients suspected of food-induced acute urticaria were included in this prospective cross-sectional multicenter study. RESULTS: Two hundred twenty-nine urticaria cases were included in this study. Seventeen patients who did not meet the inclusion criteria of the study were excluded. Of the 212 included cases, 179 (84.4%) were diagnosed with definitive food-induced acute urticaria. The most common foods causing acute urticaria were cow's milk, hen's eggs, and nuts in 56.4, 35.2, and 19% of cases, respectively. The positive predictive value of a history of milk-induced acute urticaria together with a milk-specific IgE >5 kU/L for cow's milk-induced acute urticaria was 92% (95% CI: 81-96%). A history of cow's milk-induced and/or hen's egg-induced acute urticaria was consistent with a definitive diagnosis of food-induced urticaria (Chen's kappa: 0.664 and 0.627 for milk and eggs, respectively). Urticaria activity scores were higher in patients with food-induced acute urticaria (p = 0.002). CONCLUSION: Cow's milk, hen's eggs, and nuts were the most common allergens in the etiology of childhood food-induced acute urticaria. Although the urticaria activity score provides guidance for diagnosis, an oral food challenge is often essential for the definitive diagnosis of a patient with a history of food-induced acute urticaria.

The pharmacists' ability to use pressurized metered-dose inhalers with a spacer device and factors affecting it.

Objective: The aim of this study is to evaluate the pharmacists' ability to use pMDIs with a spacer device and the factors that affect this ability.Method: Face to face interviews were conducted with the pharmacists. A nine item questionnaire was completed and the checklist for how to use pMDIs with a spacer device was filled out.Results: A total of 307 pharmacists voluntarily participated in this study. Fifty-six (18.2%) of the pharmacists stated that they did not know how to use pMDIs with a spacer device. These pharmacists were excluded and remaining 251 pharmacists included in the study. Only 100 (39.8%) pharmacists demonstrated all of the inhaler spacer device usage steps correctly. The step in which pharmacists made the most mistakes was "take 5-6 deep and slow breaths, hold for 10 s and slow breaths." Those pharmacists who were more likely to correctly use pMDIs with a spacer device were younger (p = 0.023), had dispensed more asthma medications per day (p < 0.001), had dispensed more asthma medications per day for patients younger than six years of age (p = 0.016), and sold inhaler spacer devices at their pharmacy (p = 0.042).Conclusion: Approximately one third of the pharmacists in the current study were able to correctly demonstrate all of the steps for proper usage of pMDIs with a spacer device, which indicates that pharmacists should be included in the training program and be provided continuous training on the use of pMDIs with a spacer device.

Food-induced anaphylaxis in early childhood and factors associated with its severity.

Background: Several factors that increase the risk of severe food-induced anaphylaxis have been identified. Objective: We aimed to determine the demographic, etiologic, and clinical features of food-induced anaphylaxis in early childhood and also any other factors associated with severe anaphylaxis. Methods: We carried out a medical chart review of anaphylaxis cases from 16 pediatric allergy and immunology centers in Turkey. Results: The data of 227 patients with 266 food-induced anaphylaxis episodes were included in the study. The median (interquartile range) age of the first anaphylaxis episode was 9 months (6-18 months); 160 of these patients were boys (70.5%). The anaphylaxis episodes were mild in 75 cases (28.2%), moderate in 154 cases (57.9%), and severe in 37 cases (13.9%). The most frequent food allergens involved were cow's milk (47.4%), nuts (16.7%), and hen's egg (15.8%). Epinephrine was administered in only 98 (36.8%) of these anaphylaxis episodes. A logistic regression analysis revealed two statistically significant factors that were independently associated with severe anaphylaxis: the presence of angioedema and hoarseness during the anaphylactic episode. Urticaria was observed less frequently in patients who developed hypotension. In addition, confusion and syncope were associated with 25.9- and 44.6-fold increases, respectively, in the risk of concomitant hypotension. Conclusion: Cow's milk, nuts, and hen's egg caused the majority of mild and moderate-to-severe anaphylaxis episodes. The presence of angioedema and hoarseness in any patient who presents with a history of food-induced anaphylaxis should alert clinicians that the reaction may be severe. In addition, the presence of confusion, syncope, or stridor probably indicates concomitant hypotension.

Acute generalized exanthematous pustulosis due to Epstein-Barr virus infection in a neonate.

Acute generalized exanthematous pustulosis (AGEP) is an uncommon inflammatory subcorneal pustular dermatosis typically caused by exposure to a medication. Several viral infections have also been implicated in its development. We describe herein a rare case of AGEP associated with acute Epstein-Barr virus (EBV) infection in a neonate.

Prevalence of atopic diseases in children with papular urticaria.

BACKGROUND: Papular urticaria is a hypersensitivity reaction characterized by chronic and recurrent papular erythema. It occurs as a result of the bites of mosquitoes, fleas, bed bugs, and other insects; and it is generally seen in children. This study examines the prevalence of atopic diseases in patients with papular urticaria. METHODS: The medical records of 130 pediatric patients with the diagnosis of papular urticaria between August 2017 and August 2019, whose disease progression was followed in two tertiary care centers, were reviewed retrospectively. The patients were divided into two groups: under 5 and above 5 years old. The prevalence of the atopic disease in children with papular urticaria was compared with those in age-matched controls without papular urticaria. RESULTS: The study included 130 patients who were diagnosed with papular urticaria (64 males, 66 females, median age: 60 months). The prevalences of atopic disease, recurrent wheezing, and atopic dermatitis were higher in the group under 5 years old with papular urticaria than in the same-age control group (p=0.001, 0.002, and 0.001, respectively). The prevalences of atopic disease, asthma, allergic rhinitis, and atopic dermatitis were higher in the group above 5 years old with papular urticaria than in the same-age control group (p=0.001, 0.001, 0.001, and 0.007, respectively). CONCLUSIONS: Many children with papular urticaria are atopic children. These patients should be assessed not only in terms of papular urticaria but also in terms of comorbid atopic diseases.

ILC3 deficiency and generalized ILC abnormalities in DOCK8-deficient patients.

BACKGROUND: Dedicator of cytokinesis 8 (DOCK8) deficiency is the main cause of the autosomal recessive hyper-IgE syndrome (HIES). We previously reported the selective loss of group 3 innate lymphoid cell (ILC) number and function in a Dock8-deficient mouse model. In this study, we sought to test whether DOCK8 is required for the function and maintenance of ILC subsets in humans. METHODS: Peripheral blood ILC1-3 subsets of 16 DOCK8-deficient patients recruited at the pretransplant stage, and seven patients with autosomal dominant (AD) HIES due to STAT3 mutations, were compared with those of healthy controls or post-transplant DOCK8-deficient patients (n = 12) by flow cytometry and real-time qPCR. Sorted total ILCs from DOCK8- or STAT3-mutant patients and healthy controls were assayed for survival, apoptosis, proliferation, and activation by IL-7, IL-23, and IL-12 by cell culture, flow cytometry, and phospho-flow assays. RESULTS: DOCK8-deficient but not STAT3-mutant patients exhibited a profound depletion of ILC3s, and to a lesser extent ILC2s, in their peripheral blood. DOCK8-deficient ILC1-3 subsets had defective proliferation, expressed lower levels of IL-7R, responded less to IL-7, IL-12, or IL-23 cytokines, and were more prone to apoptosis compared with those of healthy controls. CONCLUSION: DOCK8 regulates human ILC3 expansion and survival, and more globally ILC cytokine signaling and proliferation. DOCK8 deficiency leads to loss of ILC3 from peripheral blood. ILC3 deficiency may contribute to the susceptibility of DOCK8-deficient patients to infections.

Characteristics of persistent diaper dermatitis in children with food allergy.

BACKGROUND/OBJECTIVES: Diaper dermatitis is often caused by irritant contact occurring beneath the diaper of an infant, and it is aggravated by factors such as dampness, friction, urea, and feces. Food-allergic patients are known to exhibit various skin lesions ranging from urticaria to eczema. This study aims to determine the relationship between persistent diaper dermatitis and food allergy. METHODS: A retrospective chart review was conducted of pediatric patients with a diagnosis of persistent diaper dermatitis between August 2015 and November 2017. RESULTS: The study included 157 patients diagnosed with persistent diaper dermatitis (67 male, 72 female; median age: 13 months). Diaper dermatitis was more common and included the whole perineum in children who had multiple food allergies (P = 0.001). In children with multiple food allergies, the course of diaper dermatitis was more severe, and the condition did not respond to topical treatment (P = 0.025). A longer elimination diet was required for patients with Type I reactions and persistent diaper dermatitis (P = 0.018). In patients with Type II and mixed reactions, diaper dermatitis was more diffuse and covered the whole perineum (P = 0.025). In patients with Type II and mixed reactions, diaper dermatitis was more severe and did not respond to topical treatment (P = 0.025). CONCLUSIONS: Persistent diaper dermatitis lasting longer than a month may be associated with food allergy. The diaper rash may also be the only indicator of the food allergy. Elimination of the responsible food may allow these patients to recover from persistent diaper dermatitis.

Sleep patterns of young children with newly diagnosed atopic dermatitis.

INTRODUCTION: Even though atopic dermatitis (AD) most often begins in the first year of life, it is not well known whether sleep disturbances occur following the onset of the disease or whether they develop later. AIM: To determine the sleep patterns of young children already diagnosed with AD in comparison to a control group by using a validated questionnaire, the Brief Infant Sleep Questionnaire (BISQ). MATERIAL AND METHODS: Forty-six children with a new diagnosis of AD and 60 healthy children, aged 3-36 months, participated in the study. Their sleep behaviors were assessed using the BISQ along with a structured sociodemographic data form. RESULTS: It was found that when compared with healthy children, children with AD did not have decreased daily total sleep duration (p = 0.1); however, it was found that they woke up more frequently at night (52.2% vs. 40%, p = 0.4) and they stayed awake significantly longer than 60 min when they woke up (41.3% vs. 11.7%, p < 0.05). In addition, mothers of children with AD reported that their children had three times as many sleep problems compared to the reports of mothers of healthy children. CONCLUSIONS: The findings of this study showed that sleep disturbance was more common in young children with already diagnosed AD, and the BISQ provided a practical way to assess the sleep patterns. The use of a screening tool to enable early identification and treatment of childhood sleep problems among patients with AD should be encouraged.

The value of the clinical history for the diagnosis of immediate nonsteroidal anti-inflammatory drug hypersensitivity and safe alternative drugs in children.

BACKGROUND: Diagnosing hypersensitivity reactions that develop as a result of nonsteroidal anti-inflammatory drugs (NSAID) with a history is mostly misleading, and skin tests and/or provocation tests are needed for a definitive diagnosis. OBJECTIVE: To determine the frequency of actual NSAID hypersensitivity and whether there are any parameters in the history to predict NSAID hypersensitivity. In addition, to determine safe alternative drugs for children who are diagnosed with actual NSAID hypersensitivity. METHODS: Children with a history of NSAID hypersensitivity were evaluated by an allergist. Safe alternatives in children with a confirmed NSAID hypersensitivity were found by oral provocation tests. RESULTS: Sixty-four patients who were admitted with a suspicion of immediate-type reaction to NSAIDs were included in the study. The median age of the patients was 6 years old (range, 1-17 years), and 37 of the patients (57.8%) were boys. We performed skin tests for suspected NSAID in 35 patients (54.7%). Of these, two had positive results. Provocation tests were performed with 62 patients whose skin test results were negative or for whom skin tests were not available. During the provocation tests, 16 patients (25.8%) developed reactions. Low- and high-dose acetaminophen, nimesulide, and tolmetin sodium were used to find safe alternative drugs. Two patients developed reactions to high-dose acetaminophen but no reaction to nimesulide and tolmetin sodium. When statistically significant parameters were analyzed in a logistic regression model, the presence of multiple NSAIDs hypersensitivity in the patient history (odds ratio 26.6 [95% confidence interval, 1.47-481.63]; p = 0.026) and the emergence of a reaction within an hour (odds ratio 26.4 [95% confidence interval, 1.73-403.11]; p = 0.019) were found as the independent factors to predicted actual NSAID hypersensitivity. CONCLUSION: The emergence of a reaction within an hour of taking the drug and the presence of multiple NSAIDs hypersensitivity history increased the possibility of actual NSAID hypersensitivity. Nimesulide, low-dose acetaminophen, and tolmetin sodium could be used as safe alternative drugs in patients with multiple NSAIDs hypersensitivity.

Long-term follow-up of re-sting reactions in children with moderate to severe venom hypersensitivity.

UNLABELLED: Few data exists about re-sting reactions and their prognosis in children with moderate to severe venom hypersensitivity. The reasons behind not consenting to or prematurely ending venom immunotherapy (VIT) and the preparedness of children who refused or quit VIT for future moderate-severe systemic reaction (SR) to re-stings have not been studied. Data on children with moderate to severe SR after Hymenoptera stings was collected for a 17-year period using our database. A standardized questionnaire was administered to patients who accepted to be interviewed at the clinic. These patients were evaluated in terms of their preparedness for future moderate-severe SR to re-stings. A total of 55 children, 75 % of whom commenced on VIT, were included in the analysis. Different reasons exist for not consenting to VIT; the most common of which is living at a distance from the allergy center. There were no differences in terms of the number of re-stung patients (27.7 and 27.2 %, respectively) and moderate-severe SR (60 and 16.6 %, respectively) between children who prematurely ended or who did not consent to VIT and children who completed VIT. Sixty-four percent of the children who refused or discontinued VIT were not prepared for future moderate-severe SR to re-stings. CONCLUSION: Long-term prognosis for re-sting reactions is good in children with moderate to severe SR to venoms. Some of the reasons behind refusing or discontinuing VIT may be related to quality of life issues. Preparedness of children who refused or discontinue VIT in emergencies is very low.

The Platelet Parameters as Inflammatory Markers in Preschool Children with Atopic Eczema.

BACKGROUND: Mean platelet volume has been frequently used as an inflammatory marker in various diseases associated with inflammation. In this study, we compared platelet parameter levels between preschool children with and without atopic eczema (AE). METHODS: Preschool children with AE and age-matched healthy children were included in the study. Complete blood count was assessed in children with AE while platelet parameters were compared between children with and without AE. RESULTS: One hundred twenty eight pediatric patients (78 boys, mean age: 14 months) diagnosed with AE and 128 healthy patients (71 boys, mean age: 12 months ) were included in the study. There were no statistically significant differences between the genders (p = 0.375) and ages (p = 0.273) of both groups. WBC (p = 0.952), Hb (p = 0.370), MCV (p = 0.314) and RDW values (p = 0.124), and platelet counts (p = 0.198) of both groups were similar. In the AE group, while the MPV value was found to be higher (p = 0.003), mean PDW value (p = 0.025) and PLT/MPV ratio were found to be lower (p = 0.021). In addition, there was no correlation between the severity of AE and MPV (rho; 0.1, p = 0.257), PDW (rho; -0.1, p = 0.269) and PLT/MPV (rho; 0.07, p = 0.432) ratio. CONCLUSIONS: In patients with AE, as a sign of inflammation, PDW value and PLT/MPV ratio decrease while MPV value increases. This study has also shown that there is no association between the severity of AE and platelet parameters.

Association between thyroid autoimmunity and recurrent angioedema in children.

BACKGROUND: The association between thyroid autoimmunity (TA) and idiopathic isolated angioedema (or angioedema without urticaria) has not been evaluated in either children or in adults up until now. We, therefore, aimed to investigate underlying or concomitant TA and/or autoimmune thyroid disease in children diagnosed with recurrent idiopathic angioedema. METHODS: Children who were consecutively diagnosed with recurrent idiopathic histaminergic acquired angioedema (IH-AAE) between January 2011 and January 2014 constituted the case group. A standard diagnostic and therapeutic algorithm was applied to all the patients with recurrent IH-AAE. Thyroid autoantibodies and thyroid function tests were measured in all the patients with recurrent IH-AAE and in healthy control groups. Prophylaxis with an antihistamine was started for patients with frequently recurrent IH-AAE. RESULTS: Eighty consecutive children with recurrent IH-AAE (49 boys; median age, 8.3 years) and 80 healthy children (39 boys; median, 8 years) were enrolled in this prospective, case-control study (p > 0.05 for age and sex). The IH-AAE group was significantly different than the control group with respect to TA (13.7% versus 2.5%, respectively; p = 0.009) but was similar with respect to autoimmune thyroid disease (3.7% versus 0%, respectively; p = 0.2). The median follow-up of the recurrent IH-AAE group was 34 months (range, 12-45 months). Patients with recurrent IH-AAE with and those without TA were not different with respect to either the need or the duration of antihistamine prophylaxis (p > 0.05 for both). CONCLUSIONS: Recurrent IH-AAE may be related to or associated with TA and/or autoimmune thyroid diseases in some children. However, exploring to see whether this association is a causal link or just an epiphenomenon deserves further investigation and longer follow-ups.

Relationship between hypogammaglobulinemia and severity of atopic dermatitis.

BACKGROUND: Atopic dermatitis is an itchy, inflammatory, chronic, or chronically relapsing skin disease. The disease occurs in people who have an "atopic tendency" or may appear as a clinical sign of primary immunodeficiency. OBJECTIVES: To determine the relation between severity of atopic dermatitis and hypogammaglobulinemia. METHODS: One hundred sixty pediatric patients with atopic dermatitis (98 boys and 62 girls, 1-60 months old, median age 14.5 months) and 95 healthy children (57 boys and 38 girls, median age 16 months; control group) were included in the study. In patients with atopic dermatitis, the severity of disease was determined by the SCORing Atopic Dermatitis index. Serum immunoglobulin levels of all patients and children in the control group were measured by nephelometry on admission. RESULTS: The incidence of hypogammaglobulinemia was higher in patients with atopic dermatitis than in the control group (P = .009). The main reason for this difference was the low level of IgG in the atopic dermatitis group (P = .024). Analysis of the relation between hypogammaglobulinemia and the severity of atopic dermatitis showed no statistically significant difference between the group with mild to moderate atopic dermatitis and the group with severe atopic dermatitis with respect to hypogammaglobulinemia (P = .859), IgG (P = .068), IgA (P = .410), and IgM (P = .776) values. CONCLUSION: Hypogammaglobulinemia was more frequent in patients with atopic dermatitis compared with the control group, mostly owing to the low IgG level. Hypogammaglobulinemia is not associated with the severity of atopic dermatitis.

Potential predictors of relapse after treatment of asthma exacerbations in children.

BACKGROUND: Knowledge of factors that affect relapse will allow close monitoring of patients at risk, resulting in a decreased rate of readmission to the emergency department. OBJECTIVE: To determine risk factors associated with relapse within 7 days after treatment of asthma exacerbations in children. METHODS: This was a multicenter, prospective study of children with asthma attacks. Patients between the ages of 6 months and 17 years who met the criteria between June 2009 and September 2012 were considered. RESULTS: The study included 1177 patients (775 males [65.8%]) with a mean (SD) age of 70.72 (48.24) months. Of them, 199 (16.9%) had a relapse within 1 week after being discharged from the hospital. Factors independently associated with relapse identified by a logistic regression model for the 1,177 study visits were having taken a short-acting inhaled ß2-agonist within 6 hours before admission (odds ratio [OR], 2.43; 95% confidence interval [CI], 1.728-3.426; P = .001), presence of retraction on physical examination (OR, 1.76; 95% CI, 1.123-2.774; P = .01), no prescription for high-dose inhaled steroids on release (OR, 2.02; 95% CI, 1.370-3.002; P < .001), and not being given a written instructional plan (OR, 1.55; 95% CI, 1.080-2.226; P = .02). CONCLUSION: Whereas having taken short-acting ß2-agonists within 6 hours before admission and the presence of retractions on physical examination increased the risk of relapse after treatment of the acute attack, being given high-dose inhaled steroids and a written instructional plan when being sent home reduced the risk.

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.

Factors associated with pulmonary function decline of patients in the cystic fibrosis registry of Turkey: A retrospective cohort study.

BACKGROUND: The decline in pulmonary function is a predictor of disease progression in patients with cystic fibrosis (CF). This study aimed to determine the decline rate of percent predicted forced expiratory volume in 1 s (ppFEV1) based on the data of the CF Registry of Turkey. The secondary aim was to investigate the risk factors related to the decline in ppFEV1. METHODS: A retrospective cohort study of CF patients over 6 years old, with pulmonary function data over at least 2 years of follow-up was extracted from the national CF registry for years 2017-2019. Patients were classified according to disease severity and age groups. Multivariate analysis was used to predict the decline in ppFEV1 and to investigate the associated risk factors. RESULTS: A total of 1722 pulmonary function test results were available from 574 patients over the study period. Mean diagnostic age was older and weight for age, height for age, and body mass index z scores were significantly lower in the group of ppFEV1 < 40, while chronic Pseudomonas aeruginosa (p < .001) and mucoid P. aeruginosa colonization (p < .001) were significantly higher in this group (p < .001). Overall mean annual ppFEV1 decline was -0.97% (95% confidence interval [CI] = -0.02 to -1.92%). The mean change of ppFEV1 was significantly higher in the group with ppFEV1 ≥ 70 compared with the other (ppFEV1 < 40 and ppFEV1: 40-69) two groups (p = .004). Chronic P. aeruginosa colonization (odds ratio [OR] = 1.79 95% CI = 1.26-2.54; p = .01) and initial ppFEV1 ≥ 70 (OR = 2.98 95% CI = 1.06-8.36), p = .038) were associated with significant ppFEV1 decline in the whole cohort. CONCLUSIONS: This data analysis recommends close follow-up of patients with normal initial ppFEV1 levels at baseline; advocates for early interventions for P. aeruginosa; and underlines the importance of nutritional interventions to slow down lung disease progression.

A real-life study on acquired skills from using an adrenaline autoinjector.

BACKGROUND: Training programs performed by allergists have increased the ability of patients' recognition and management of anaphylaxis. We aim to investigate the permanence of effect of an anaphylaxis training program and to determine the factors affecting it beyond training given by allergists. METHODS: Children and/or their caregivers who had been prescribed an adrenaline autoinjector at least 1 year before were invited to take part in the study. The knowledge about anaphylaxis was assessed using a questionnaire and the skills were practically tested. RESULTS: Sixty-four (50 caregivers/14 children >12 years of age) of 80 patients who accepted the invitation were included in the study. Fifty-nine patients obtained the autoinjector after initial prescription. Among them, 42 (71%) still had the device at the time of the study. The most common reason for not having the autoinjector was no longer feeling it was necessary (54.6%). Of the cases, 39.4% were competent in autoinjector use. There was a significant relation between adrenaline autoinjector competency and regular allergy visits (p = 0.010), believing that it is necessary (p = 0.04), having an adrenaline autoinjector (p = 0.003), and previous history of severe anaphylaxis (p = 0.010). Autoinjector competency score decreased as time elapsed from the last visit (rho = -0.382; p = 0.002) and the first instruction (rho = -0.317; p = 0.01). Regular visits (p = 0.009) and history of severe anaphylaxis (p = 0.007) were found as independent factors having an effect on adrenaline autoinjector competency. CONCLUSIONS: Training of patients/caregivers by allergists does not guarantee the permanence of acquired skills on anaphylaxis in the long run. Regular follow-up visits should be fostered.