Psychometric Properties of the Persian Version of Cystic Fibrosis Questionnaire: Revised in Children with Cystic Fibrosis.

Background: Cystic fibrosis is a progressive, fatal disease affecting the quality of life. The cystic fibrosis questionnaire-revised (CFQ-R) is an efficient tool to monitor health-related quality of life in patients. The aim of this study was to explore the psychometric properties of the child and parent versions of the Persian version of the CFQ-R in the Iranian population. Methods: Fifty children with cystic fibrosis (6-11 years) and their parents were allocated in this methodological study to examine convergent validity, discriminant validity, test-retest reliability (n = 30), internal consistency, ceiling and floor effects, and agreement between two versions of the CFQ-R. Results: Convergent validity was confirmed for parent proxy (P < 0.05). CFQ-R discriminated patients among stages of disease severity based on lung function, age, and BMI (P < 0.05). Test-retest analysis revealed good to excellent reliability (inter-class correlation coefficient (ICC) = 0.78-0.97). In most domains, lower quality of life scores was obtained in the parent proxy compared to the child version (P < 0.05). Domain-specific correlations were found between the child version and parent proxy (P < 0.05). Internal consistency was generally confirmed (α = 0.13-0.83 in child version and α = 0.25-0.87 in parent proxy). There were no floor effects. Ceiling effects were mostly seen for physical, digestion, and body image domains in the child version and for eating, weight, and school domains in the parent proxy. Conclusions: The child version and parent proxy of the Persian CFQ-R are valid and reliable measures and can be applied in clinical trials to monitor the quality of life in children with cystic fibrosis. It is recommended to use both versions in conjunction to better interpret the quality of life aspects of children with cystic fibrosis.

Evaluation of safety and efficacy of allogeneic adipose tissue-derived mesenchymal stem cells in pediatric bronchiolitis obliterans syndrome (BoS) after allogeneic hematopoietic stem cell transplantation (allo-HSCT).

BACKGROUND: Allo-HSCT is a definite approach for the management of a wide variety of lethal and debilitating malignant and non-malignant disorders. However, its two main complications, acute and chronic graft-versus-host disease (GVHD), exert significant morbidities and mortalities. BoS, as a manifestation of chronic lung GVHD, is a gruesome complication of allo-HSCT, and for those with steroid-refractory disease, no approved second-line therapies exist. Mesenchymal stem cells (MSCs) exert anti-inflammatory and growth-promoting effects, and their administration against a wide range of inflammatory and neurologic disorders, as well as GVHD, has been associated with promising outcomes. However, literature on the safety and effectiveness of MSC therapy for BoS and pediatric cGVHD is scarce. METHODS: We designed a single-arm trial to administer adipose tissue (AT)-derived MSCs to pediatric patients with refractory BoS after allo-HSCT. AT-MSCs from obese, otherwise healthy donors were cultured in an ISO class 1 clean room and injected into the antecubital vein of eligible patients with a dose of 1 × 106/kg. The primary endpoints included a complete or partial response to therapy [in terms of increased forced expiratory volume in one second (FEV1) values and steroid dose reduction] and its safety profile. RESULTS: Four eligible patients with a median age of 6.5 years were enrolled in the study. Steroid-induced osteoporosis and myopathy were present in three cases. A partial response was evident in three cases after a single injection of AT-MSCs. The treatment was safe and tolerable, and no treatment-related adverse events were noted. Two patients developed manageable COVID-19 infections one and 4 months after AT-MSC injection. After a median follow-up duration of 19 months, all cases are still alive and have had no indications for lung transplantation. CONCLUSIONS: AT-MSCs could be safely administered to our pediatric cases with BoS post-allo-HSCT. Considering their advanced stage of disease, their sub-optimal functional capacity due to steroid-induced complications, and COVID-19 infection post-treatment, we believe that AT-MSC therapy can have possible efficacy in the management of pediatric BoS. The conduction of further studies with larger sample sizes and more frequent injections is prudent for further optimization of AT-MSC therapy against BoS. Trial registration Iranian Registry of Clinical Trials (IRCT), IRCT20201202049568N2. Registered 22 February 2021, https://en.irct.ir/trial/53143 .

Distribution and Characteristics of Bacteria Isolated from Cystic Fibrosis Patients with Pulmonary Exacerbation.

Background: Cystic fibrosis (CF) is an inherited recessive disorder characterized by recurrent and persistent pulmonary infections, resulting in lung function deterioration and early mortality. Methods: A cross-sectional study was conducted on the bacterial profile and antibiotic resistance pattern of 103 respiratory specimens from CF patients with signs of pulmonary exacerbation. Antibiotic susceptibility testing and biofilm formation of Staphylococcus aureus and Pseudomonas aeruginosa isolates were performed by the Kirby-Bauer disc diffusion method and microtiter plate assay, respectively. Molecular typing of S. aureus and P. aeruginosa isolates was carried out by spa typing and repetitive extragenic palindromic element PCR. Results: In a total of 129 isolates, the most prevalent organisms were S. aureus (55.3%) and P. aeruginosa (41.7%). Other less prevalent bacterial isolates include coagulase-negative staphylococci, Escherichia coli, klebsiella spp., Enterobacter spp., and Achromobacter xylosoxidans. The highest rate of resistance for S. aureus was observed to azithromycin and erythromycin (80%), ciprofloxacin (52.3%), clindamycin (44.6%) and tetracycline (43%). Twenty percent of S. aureus isolates were methicillin-resistant S. aureus (MRSA) and 47.6% were MDR S. aureus. For P. aeruginosa isolates the highest resistance was to cefepime (38.3%) and levofloxacin (33.3%) and 20% showed MDR phenotype. Conclusion: Our study demonstrated a significant decline in the prevalence of P. aeruginosa infections in comparison to previous studies. We found S. aureus to be more prevalent in younger patients, whereas mucoid P. aeruginosa showed a shift in prevalence toward older ages. Molecular typing methods showed great diversity between isolates.

Cystic fibrosis prevalence among a group of high-risk children in the main referral children hospital in Iran.

BACKGROUND: Knowledge about cystic fibrosis (CF) in Iran is very limited. The objective of this study was to determine the prevalence of CF among a group of high-risk children with suggestive clinical features in the main referral hospital in Iran. MATERIALS AND METHODS: This study children consisted of 505 patients who had presented with one or more of the following symptoms: chronic or recurrent respiratory symptoms, gastrointestinal symptoms as rectal prolapse, steatorrhea, hepatobiliary disease as prolonged jaundice, failure to thrive, hyperglycemia and glycosuria, hypochloremic metabolic alkalosis, hypoprothrombinemia, anemia or edema, and positive family history of CF. Patients were screened using pilocarpine iontophoresis to collect sweat and chemical analysis of its chloride content with classic Gibson and Cooke technique. RESULTS: Of 505 patients, 89 (17.6%) had positive sweat chloride screening test. Five (1%) patients had required cystic fibrosis transmembrane conductive regulator protein mutation analysis to confirm CF. CONCLUSION: Our findings suggest that in Iran, CF is more common than what previously anticipated. Larger studies are warranted to identify the incidence, molecular basis, and clinical pattern of CF in the Iranian population.

Erratum to: Nebulized Magnesium Sulfate in Acute Bronchiolitis: A Randomized Controlled Trial.

In the original article, the authors failed to cite the study by Kose M et al. entitled "The efficacy of nebulized salbutamol, magnesium sulfate and salbutamol/magnesium sulfate combination in moderate bronchiolitis. Eur J Pediatr 2014;173: 1157-1160" as the first in literature to assess the efficacy of magnesium sulfate in the treatment of acute bronchiolitis. The authors apologize for this unintentional omission.

Effect of Metronidazole on Halitosis of 2 to 10 Years Old Children.

BACKGROUND: Regarding the fact that halitosis has social and personal aspects which can lead to social embarrassment and consequently low self-esteem and self-confidence in subjects suffering from the problem, especially children, its proper treatment is an important issue. OBJECTIVES: The aim of this study was to evaluate the effect of metronidazole as a nonspecific antimicrobial agent in the treatment of halitosis in children. MATERIALS AND METHODS: In this study, 2-10 years old children with oral halitosis were enrolled. Children without H. pylori infection and parasitic infection were randomized in two interventional and control groups. Metronidazole was given 5mg/kg/day for one week. Information regarding the demographic characteristics of studied population and halitosis (duration and time of day with more halitosis and its severity) before and after intervention was recorded using a questionnaire. RESULTS: 77 children with halitosis were studied in two interventional (40 children) and control (37 children) groups. There was no significant difference between two groups before intervention. After intervention, halitosis improvement rate - according to the reports of mothers of studied children - was higher significantly in intervention group (P < 0.05). CONCLUSIONS: The results support the effectiveness of metronidazole in the treatment of halitosis. Moreover, it supports recent findings regarding the participation of specific bacteria specially unculturable ones in the pathogenesis of the disease.

Nebulized Magnesium Sulfate in Acute Bronchiolitis: A Randomized Controlled Trial.

OBJECTIVE: To assess the efficacy of nebulized magnesium sulfate as a bronchodilator in infants hospitalized with acute bronchiolitis. METHODS: This three-center double masked randomized clinical trial comprised 120 children with moderate to severe bronchiolitis. They were randomly assigned into two groups: the first group was treated with nebulized magnesium sulfate (40 mg/kg) and nebulized epinephrine (0.1 ml/kg) and the second group (control) was treated with nebulized epinephrine (0.1 ml/kg). The primary outcome was the length of hospital stay. The use of oxygen, temperature, oxygen saturation (SPO2), pulse rate (PR), respiratory rate (RR) and respiratory distress assessment instrument (RDAI) score were measured in the beginning of the study and during hospitalization. RESULTS: The mean (SD) age of 120 infants was 5.1(± 2.6) mo and 60% were boys. The length of hospital stay was not different between the two groups (P > 0.01). Use of oxygen supplementation, SPO2 and vital signs were similar in the two groups. Improvement in RDAI score was significantly better in infants treated with nebulized magnesium sulfate than in the other group (P 0.01). CONCLUSIONS: Thus, in infants with acute bronchiolitis, the effect of nebulized magnesium sulfate is comparable to nebulized epinephrine. However nebulized magnesium sulfate can improve the clinical score so it may have additive effect to reduce symptoms during hospitalization.