Relationship between exercise capacity and glucose tolerance in cystic fibrosis.

Pediatr Pulmonol. 2017 Nov 08;:

Authors: Foster K, Huang G, Zhang N, Crisalli J, Chini B, Amin R, Elder D

Abstract
BACKGROUND: Improved exercise capacity (EC) and normal glucose tolerance (NGT) are independently associated with favorable outcomes in CF, however, little information on this relationship exists in patients with CF.
METHODS: Cardiopulmonary exercise tests, oral glucose tolerance tests (OGTT), and HbA1c values measured within a 12-month period were evaluated on 83 pediatric patients diagnosed with CF. Patients were categorized as having NGT, abnormal glucose tolerance (AGT), or CF-related diabetes (CFRD).
RESULTS: EC decreased as severity of glucose intolerance increased across NGT, AGT, and CFRD groups (P = 0.02). Compared to patients with NGT, patients with CFRD had lower peak VO2  mL/kg/min (33.0 ± 7.3 vs 41.3 ± 9.4, P = 0.01), lower VO2 % (81 ± 20 vs 93 ± 17, P = 0.03), and higher HbA1c (6.9 ± 1.7 vs 5.4 ± 0.4, P < 0.01). There was a positive association with age and FEV1 % with EC in the 17 patients with CFRD. In the 66 patients without diabetes, peak EC was positively associated with FEV1 % and negatively associated with age, fasting insulin, and insulin 120 min. After accounting for age and FEV1 %, multivariate analyses indicated that insulin and glucose values at 120 min predicted EC.
CONCLUSIONS: These data provide evidence that poor glucose tolerance is associated with lower EC in pediatric patients with CF. There was a significant relationship between glucose and insulin values obtained by OGTT with EC in a sample of non-diabetic patients with preserved lung function. Future studies are warranted to confirm these findings and investigate the potential role of exercise in the management or prevention of CFRD.

PMID: 29115018 [PubMed - as supplied by publisher]

Cystic fibrosis newborn screening: outcome of infants with normal sweat tests.

Arch Dis Child. 2017 Nov 07;:

Authors: Edmondson C, Grime C, Prasad A, Cowlard J, Nwokoro CEC, Ruiz G, Wallis C, Balfour-Lynn IM

Abstract
Newborn babies positively screened for cystic fibrosis (CF) (high serum immunoreactive trypsin (IRT) with DNA analysis) are referred for a diagnostic sweat test, which may be normal (sweat chloride <30 mmol/L). Unless two gene mutations are identified during Newborn screening (NBS), the babies are discharged from follow-up. We wished to check that none had subsequently developed symptoms suggestive of CF. We retrospectively reviewed patient notes and contacted general practitioners of all babies with a negative sweat test, conducted in one of the four paediatric specialist CF centres in London, over the first 6 years of screening in South East England.Of 511 babies referred, 95 (19%) had a normal sweat test. Five (5%) had CF diagnosed genetically, two of them on extended genome sequencing after clinical suspicion. Eleven (12%) were designated as CF screen positive inconclusive diagnosis (CFSPID); one of the five CF children was originally designated as CFSPID. Seventy-nine (83%) were assumed to be false-positive cases and discharged; follow-up data were available for 51/79 (65%); 32/51 (63%) had no health issues, 19/51 (37%) had other significant non-CF pathology.These results are reassuring in that within the limitations of those lost to follow-up, CF symptoms have not emerged in the discharged children. The high non-CF morbidity in these children may relate to known causes of high IRT at birth. Clinicians need to be aware that a child can have CF despite a normal sweat test following NBS, and if symptoms suggest the diagnosis, further testing, including extended genome sequencing, is required.

PMID: 29113966 [PubMed - as supplied by publisher]

Impact of extensive antibiotic treatment on faecal carriage of antibiotic-resistant enterobacteria in children in a low resistance prevalence setting.

PLoS One. 2017;12(11):e0187618

Authors: Knudsen PK, Brandtzaeg P, Høiby EA, Bohlin J, Samuelsen Ø, Steinbakk M, Abrahamsen TG, Müller F, Gammelsrud KW

Abstract
We prospectively studied the consequences of extensive antibiotic treatment on faecal carriage of antibiotic-resistant enterobacteria in a cohort of children with cystic fibrosis (CF) and a cohort of children with cancer compared to healthy children with no or low antibiotic exposure. The study was conducted in Norway in a low resistance prevalence setting. Sixty longitudinally collected faecal samples from children with CF (n = 32), 88 samples from children with cancer (n = 45) and 127 samples from healthy children (n = 70) were examined. A direct MIC-gradient strip method was used to detect resistant Enterobacteriaceae by applying Etest strips directly onto agar-plates swabbed with faecal samples. Whole genome sequencing (WGS) data were analysed to identify resistance mechanisms in 28 multidrug-resistant Escherichia coli isolates. The prevalence of resistance to third-generation cephalosporins, gentamicin and ciprofloxacin was low in all the study groups. At inclusion the prevalence of ampicillin-resistant E. coli and trimethoprim-sulfamethoxazole-resistant E. coli in the CF group compared to healthy controls was 58.6% vs. 28.4% (p = 0.005) and 48.3% vs. 14.9% (p = 0.001), respectively, with a similar prevalence at the end of the study. The prevalence of resistant enterobacteria was not significantly different in the children with cancer compared to the healthy children, not even at the end of the study when the children with cancer had been treated with repeated courses of broad-spectrum antibiotics. Children with cancer were mainly treated with intravenous antibiotics, while the CF group mainly received peroral treatment. Our observations indicate that the mode of administration of antibiotics and the general level of antimicrobial resistance in the community may have an impact on emergence of resistance in intestinal enterobacteria during antibiotic treatment. The WGS analyses detected acquired resistance genes and/or chromosomal mutations that explained the observed phenotypic resistance in all 28 multidrug-resistant E. coli isolates examined.

PMID: 29112974 [PubMed - in process]

Splenectomy versus conservative management for acute sequestration crises in people with sickle cell disease.

Cochrane Database Syst Rev. 2017 Nov 07;11:CD003425

Authors: Owusu-Ofori S, Remmington T

Abstract
BACKGROUND: Acute splenic sequestration crises are a complication of sickle cell disease, with high mortality rates and frequent recurrence in survivors of first attacks. Splenectomy and blood transfusion, with their consequences, are the mainstay of long-term management used in different parts of the world. This is a 2017 update of a Cochrane Review first published in 2002, and previously updated, most recently in 2015.
OBJECTIVES: To assess whether splenectomy (total or partial), to prevent acute splenic sequestration crises in people with sickle cell disease, improved survival and decreased morbidity in people with sickle cell disease, as compared with regular blood transfusions.
SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Haemoglobinopathies Trials Register, which comprises of references identified from comprehensive electronic database searches and handsearching relevant journals and abstract books of conference proceedings. We also searched clinical trial registries. Additional trials were sought from the reference lists of the trials and reviews identified by the search strategy.Date of the most recent search: 14 August 2017.
SELECTION CRITERIA: All randomized or quasi-randomized controlled trials comparing splenectomy (total or partial) to prevent recurrence of acute splenic sequestration crises with no treatment or blood transfusions in people with sickle cell disease.
DATA COLLECTION AND ANALYSIS: No trials of splenectomy for acute splenic sequestration were found.
MAIN RESULTS: No trials of splenectomy for acute splenic sequestration were found.
AUTHORS' CONCLUSIONS: Splenectomy, if full, will prevent further sequestration and if partial, may reduce the recurrence of acute splenic sequestration crises. However, there is a lack of evidence from trials showing that splenectomy improves survival and decreases morbidity in people with sickle cell disease. There is a need for a well-designed, adequately-powered, randomized controlled trial to assess the benefits and risks of splenectomy compared to transfusion programmes, as a means of improving survival and decreasing mortality from acute splenic sequestration in people with sickle cell disease.There are no trials included in the review and we have not identified any relevant trials up to August 2017. We will continue to run searches to identify any potentially relevant trials; however, we do not plan to update other sections of the review until new trials are published.

PMID: 29112240 [PubMed - as supplied by publisher]

Vitamin D supplementation in respiratory diseases - evidence from RCT.

Pol Arch Intern Med. 2017 Nov 02;:

Authors: Mathyssen C, Gayan-Ramirez G, Bouillon R, Janssens W

Abstract
Pulmonary diseases are one of the most important causes for morbidity and mortality. Although vitamin D is best known for its role in calcium, phosphorus and bone homeostasis, in the recent years vitamin D has gained attention because of a wide range of extra-skeletal effects including its immunomodulatory and antibacterial potential. Vitamin D deficiency is highly prevalent in chronic pulmonary diseases such as Chronic Obstructive Pulmonary Disease (COPD), cystic fibrosis, tuberculosis and asthma and several clinical studies have been conducted investigating the effect of vitamin D supplementation on disease outcomes. In this review we searched for positive evidence on vitamin D supplementation from randomized controlled trials and elaborate on the optimal vitamin D serum levels and dosing regimens for an effective intervention. While vitamin D supplementation seems to be beneficial as an add-on treatment for adult asthma patients and a potent intervention to reduce exacerbations in COPD patients, there is little evidence for its therapeutic use in cystic fibrosis, pneumonia and tuberculosis.

PMID: 29112181 [PubMed - as supplied by publisher]

Assessment of Liver Disease Progression in cystic Fibrosis Using Transient Elastography.

J Pediatr Gastroenterol Nutr. 2017 Nov 03;:

Authors: Gominon AL, Frison E, Hiriart JB, Vergniol J, Clouzeau H, Enaud R, Bui S, Fayon M, de Ledinghen V, Lamireau T

Abstract
OBJECTIVES: Cystic fibrosis related liver disease (CFLD) can develop silently in early life and approximately 10% of children with cystic fibrosis (CF) become cirrhotic before adulthood. Clinical, biological and ultrasound criteria used to define CFLD often reveal liver involvement at an advanced stage. The aim of this retrospective study was to assess the progression of liver stiffness measurement (LSM) in pediatric CF patients.
METHODS: The change of LSM, expressed as kPa/year and %/year, was measured using transient elastography (TE, Fibroscan) in 82 CF children (median age: 6.8 years, IQR: 5.8). Mean time interval between the two LSM was 3.5 years.
RESULTS: Median initial liver stiffness was 3.7 kPa (IQR: 1.3)., and then progressed by 0.23 kPa/year, i.e. 6%/year. The 7 patients who developed CFLD had a higher initial level of alanine aminotransferase (50 [IQR:15] vs 30 [IQR:18], p = 0.0001) and presented a more rapid progression of LSM (0.94 vs 0.23 kPa/year, p = 0.02).
CONCLUSIONS: This study shows that the slope of worsening of liver stiffness is greater in patients who will develop CFLD, suggesting that annual TE may be useful to detect risk of severe liver disease at an earlier stage.

PMID: 29112089 [PubMed - as supplied by publisher]

Experience of using non-invasive ventilation as an adjunct to airway clearance techniques in adults with cystic fibrosis-A qualitative study.

Physiother Theory Pract. 2017 Nov 07;:1-12

Authors: Rodriguez Hortal MC, Hedborg A, Biguet G, Nygren-Bonnier M

Abstract
BACKGROUND: Adults with cystic fibrosis (CF) suffer from abnormally thick mucus that is difficult to clear from the airways. Different airway clearance techniques (ACTs) can be used to clear secretions and non-invasive ventilation (NIV) can be used as an adjunct to these techniques. ACTs are ideally introduced at the time of diagnosis and thereafter modified throughout the patient's lifespan and disease progress.
PURPOSE: The research aim was to describe adult patients' views and experiences with using NIV as an adjunct to ACT.
METHOD: Eighteen adults with CF were interviewed about their experiences with using NIV during ACT. Semi-structured interviews were conducted and analyzed in accordance with qualitative content analysis.
RESULTS: The results gave rise to the overall theme 'Becoming Friends with NIV' and six associated categories: 1) getting a sense of control and feedback; 2) getting support; 3) dealing with doubt; 4) finding the rhythm; 5) feeling the effects; and 6) finding their own motivation. The findings represent a learning process for adults during the implementation stages of NIV; the physiotherapist was found to play a key role in this process.
CONCLUSION: 'Becoming Friends with NIV' involves a learning process for adults with CF. To facilitate this learning process, different aspects should be taken into account so as to promote independence and self-management, which in turn allows the patient to experience the treatment as meaningful. The findings are relevant to physiotherapists working with adults and NIV, as improved insight into and understanding of the relationship may have a positive influence on the outcome and success of NIV usage.

PMID: 29111843 [PubMed - as supplied by publisher]

Nano into micro formulations of tobramycin for the treatment of Pseudomonas aeruginosa infections in cystic fibrosis.

Biomacromolecules. 2017 Nov 07;:

Authors: Porsio B, Cusimano MG, Schillaci D, Craparo EF, Giammona G, Cavallaro G

Abstract
Here, nano into micro formulations (NiMs) of tobramycin for the treatment of Pseudomonas aeruginosa airway infections in cystic fibrosis (CF) are described. NiMs were produced by spray drying a solution containing polymers or sugars and a nanometric Polyanion-Tobramcyin Complex (PTC), able to achieve a prolonged antibiotic release. NiMs properties were compared to TOBI®Podhaler®(Novartis), the only one commercially available dry powder inhalatory formulation based on porous microparticles. Produced NiMs showed adequate characteristics for pulmonary administration, as spherical shape, micrometric size and high cytocompatibility towards human bronchial epithelial cells. Contrarily to TOBI®Podhaler®, some of produced NiMs, thanks to their specific chemical composition, are able to facilitate the drug diffusion through the mucus secretion, achieving, at the same time, a sustained tobramycin delivery. Moreover, NiMs showed pronounced antimicrobial activity against P.aeruginosa pathogens and their biofilm, if compared to free tobramycin and TOBI®Podhaler®, demonstrating the potential of obtained formulations as drug delivery systems for the treatment of pulmonary infections in CF patients.

PMID: 29111673 [PubMed - as supplied by publisher]

Longitudinal development of initial, chronic and mucoid Pseudomonas aeruginosa infection in young children with cystic fibrosis.

J Cyst Fibros. 2017 Oct 27;:

Authors: Heltshe SL, Khan U, Beckett V, Baines A, Emerson J, Sanders DB, Gibson RL, Morgan W, Rosenfeld M

Abstract
BACKGROUND: While the emergence of chronic and mucoid Pseudomonas aeruginosa (Pa) infection are both associated with poorer outcomes among CF patients, their relationship is poorly understood. We examined the longitudinal relationship of incident, chronic and mucoid Pa in a contemporary, young CF cohort in the current era of Pa eradication therapy.
METHODS: This retrospective cohort was comprised of patients in the U.S. CF Foundation Patient Registry born 2006-2015, diagnosed before age 2, and with at least 3 respiratory cultures annually. Incidence and age-specific prevalence of Pa infection stages (initial and chronic [≥ 3Pa+cultures in prior year]) and of mucoid Pa were summarized. Transition times and the interaction between Pa stage and acquisition of mucoid Pa were examined via Cox models.
RESULTS: Among the 5592 CF patients in the cohort followed to a mean age of 5.5years, 64% (n=3580) acquired Pa. Of those, 13% (n=455) developed chronic Pa and 17% (n=594) cultured mucoid Pa. Among those with mucoid Pa, 36% (211/594) had it on their first recorded Pa+culture, while mucoid Pa emerged at or after entering the chronic stage in 12% (73/594). Mucoidy was associated with significantly increased risk of transition to chronic Pa infection (HR=2.59, 95% CI 2.11, 3.19).
CONCLUSIONS: Two-thirds of early-diagnosed young children with CF acquired Pa during a median 5.6years of follow up, among whom 13% developed chronic Pa and 17% acquired mucoid Pa. Contrary to our hypothesis, 87% of young children who developed mucoid Pa did so before becoming chronically infected.

PMID: 29110966 [PubMed - as supplied by publisher]

Long-term management of patients with end-stage lung diseases.

Best Pract Res Clin Anaesthesiol. 2017 Jun;31(2):167-178

Authors: Briganti DF, D'Ovidio F

Abstract
Long-term management of end-stage lung disease differs from interstitial lung disease to chronic obstructive pulmonary disease to cystic fibrosis to pulmonary vascular disease. The management includes pharmacological therapy that is disease specific such as antibiotic therapy for cystic fibrosis, antifibrotic drugs in idiopathic pulmonary fibrosis; long-acting beta-agonists, long-acting muscarinic antagonist, and inhaled corticosteroids in chronic obstructive pulmonary disease; and vasodilators in pulmonary arterial hypertension. Moreover, non-pharmacological therapy is essential in the treatment of these diseases, in particular, rehabilitation and supportive therapy, which are necessary in all end-stage lung diseases and specific intervention such as non-invasive ventilation in chronic obstructive pulmonary disease and cystic fibrosis, surgical therapy in chronic obstructive pulmonary disease, and airway clearance in cystic fibrosis. The goal is not only to prolong survival, but it is fundamental to keep patients in good general conditions for transplantation. Transplantation, indeed, remains the only therapeutic option that could prolong survival in patients with terminal lung disease when medical or surgical therapies are not available or not effective anymore.

PMID: 29110790 [PubMed - in process]

Advance care planning for patients with chronic respiratory diseases: a systematic review of preferences and practices.

Thorax. 2017 Nov 06;:

Authors: Jabbarian LJ, Zwakman M, van der Heide A, Kars MC, Janssen DJA, van Delden JJ, Rietjens JAC, Korfage IJ

Abstract
BACKGROUND: Advance care planning (ACP) supports patients in identifying and documenting their preferences and timely discussing them with their relatives and healthcare professionals (HCPs). Since the British Thoracic Society encourages ACP in chronic respiratory disease, the objective was to systematically review ACP practice in chronic respiratory disease, attitudes of patients and HCPs and barriers and facilitators related to engagement in ACP.
METHODS: We systematically searched 12 electronic databases for empirical studies on ACP in adults with chronic respiratory diseases. Identified studies underwent full review and data extraction.
RESULTS: Of 2509 studies, 21 were eligible: 10 were quantitative studies. Although a majority of patients was interested in engaging in ACP, ACP was rarely carried out. Many HCPs acknowledged the importance of ACP but were hesitant to initiate it. Barriers to engagement in ACP were the complex disease course of patients with chronic respiratory diseases, HCPs' concern of taking away patients' hopes and lack of continuity of care. The identification of trigger points and training of HCPs on how to communicate sensitive topics were identified as facilitators to engagement in ACP.
CONCLUSIONS: In conclusion, ACP is surprisingly uncommon in chronic respiratory disease, possibly due to the complex disease course of chronic respiratory diseases and ambivalence of both patients and HCPs to engage in ACP. Providing patients with information about their disease can help meeting their needs. Additionally, support of HCPs through identification of trigger points, training and system-related changes can facilitate engagement in ACP.
SYSTEMATIC REVIEW REGISTRATION NUMBER: CRD42016039787.

PMID: 29109233 [PubMed - as supplied by publisher]

Synthetic host defense peptide IDR-1002 reduces inflammation in Pseudomonas aeruginosa lung infection.

PLoS One. 2017;12(11):e0187565

Authors: Wuerth KC, Falsafi R, Hancock REW

Abstract
Pseudomonas aeruginosa is a frequent cause of lung infections, particularly in chronic infections in cystic fibrosis patients. However, treatment is challenging due to P. aeruginosa evasion of the host immune system and the rise of antibiotic resistant strains. Host defense peptides (HDPs) and synthetic derivatives called innate defense regulators (IDRs) have shown promise in several infection models as an alternative to antibiotic treatment. Here we tested peptide IDR-1002 against P. aeruginosa in vitro and in vivo. Treatment of bronchial epithelial cells and macrophages with IDR-1002 or in combination with live P. aeruginosa or its LPS led to the reduction of agonist-induced cytokines and chemokines and limited cell killing by live P. aeruginosa. In an in vivo model using P. aeruginosa combined with alginate to mimic a chronic model, IDR-1002 did not reduce the bacterial burden in the lungs, but IDR-1002 mice showed a significant decrease in IL-6 in the lungs and in gross pathology of infection, while histology revealed that IDR-1002 treated mice had reduced alveolar macrophage infiltration around the site of infection and reduced inflammation. Overall, these results indicate that IDR-1002 has promise for combating P. aeruginosa lung infections and their resulting inflammation.

PMID: 29107983 [PubMed - in process]

Genetic therapies for cystic fibrosis lung disease.

Curr Opin Pharmacol. 2017 Nov 03;34:119-124

Authors: Hart SL, Harrison PT

Abstract
Gene therapy for cystic fibrosis (CF) has been the subject of intense research over the last twenty-five years or more, using both viral and liposomal delivery methods, but so far without the emergence of a clinical therapy. New approaches to CF gene therapy involving recent improvements to vector systems, both viral and non-viral, as well as new nucleic acid technologies have led to renewed interest in the field. The field of therapeutic gene editing is rapidly developing with the emergence of CRISPR/Cas9 as well as chemically modified mRNA therapeutics. These new types of nucleic acid therapies are also a good fit with delivery by non-viral delivery approaches which has led to a renewed interest in lipid-based and other nanoformulations.

PMID: 29107808 [PubMed - as supplied by publisher]

Inflammation and host-pathogen interaction: Cause and consequence in cystic fibrosis lung disease.

J Cyst Fibros. 2017 Oct 26;:

Authors: Bragonzi A, Horati H, Kerrigan L, Lorè NI, Scholte BJ, Weldon S

Abstract
Cystic Fibrosis (CF) lung disease is associated with dysregulation of host defence systems, which ultimately disrupts the balance between inflammation and resolution and leaves the host susceptible to repeated infection. However, the mechanisms underlying these defects are complex and continue to garner significant interest among the CF research community. This review explores emerging data on novel aspects of innate host defence with promising biomarker and therapeutic potential for CF lung disease. Improved understanding of inflammation and host defence against pathogens in patients and animal models during the progression of CF lung disease is pivotal for the discovery of new therapeutics that can limit and/or prevent damage from birth.

PMID: 29107600 [PubMed - as supplied by publisher]

Faecal calprotectin concentrations in young children with cystic fibrosis.

J Cyst Fibros. 2017 Oct 26;:

Authors: Ellemunter H, Schüller K, Steinkamp G

PMID: 29107599 [PubMed - as supplied by publisher]

Occurrence of Pseudomonas aeruginosa in taps: Implications for patients with cystic fibrosis (CF).

J Hosp Infect. 2017 Oct 26;:

Authors: Caskey S, Stirling J, Moore JE, Rendall JC

PMID: 29107077 [PubMed - as supplied by publisher]

Loss of β Epithelial Sodium Channel Function in Meibomian Glands Produces Pseudohypoaldosteronism 1-Like Ocular Disease in Mice.

Am J Pathol. 2017 Oct 26;:

Authors: Yu D, Saini Y, Chen G, Ghio AJ, Dang H, Burns KA, Wang Y, Davis RM, Randell SH, Esther CR, Paulsen F, Boucher RC

Abstract
Human subjects with pseudohypoaldosteronism-1 due to loss of function mutations in epithelial Na(+) channel (ENaC) subunits exhibit meibomian gland (MG) dysfunction. A conditional βENaC MG knockout mouse model was generated to elucidate the pathogenesis of absent ENaC function in the MG and associated ocular surface disease. βENaC MG KO mice exhibited a striking age dependent, female predominant MG dysfunction phenotype, with white toothpaste-like secretions observed obstructing MG orifices at seven weeks of age. There were compensatory increases in tear production but higher tear Na(+) and indices of mucin concentration in βENaC MG KO mice. Histologically, MG acinar atrophy was observed with ductal enlargement and ductal epithelial hyperstratification. Inflammatory cell infiltration was observed in both MG and conjunctiva of βENaC MG KO mice. In older βENaC MG KO mice (five to 11 months), significant ocular surface pathologies were noted, including corneal opacification, ulceration, neovascularization, and ectasia. Inflammation in MG and conjunctiva was confirmed by increased cytokine gene and protein expression and positive Ly-6B.2 immunostaining. Cell proliferation assays revealed lower proliferation rates of MG cells derived from βENaC MG KO than control mice, suggesting that βENaC plays a role in cell renewal of mouse MG. Loss of βENaC function resulted in MG disease and severe ocular surface damage that phenocopied aspects of human pseudohypoaldosteronism-1 MG disease and was sex dependent.

PMID: 29107074 [PubMed - as supplied by publisher]

Evaluating the sinus and Nasal Quality of Life Survey in the pediatric cystic fibrosis patient population.

Int J Pediatr Otorhinolaryngol. 2017 Nov;102:133-137

Authors: Xie DX, Wu J, Kelly K, Brown RF, Shannon C, Virgin FW

Abstract
INTRODUCTION: The Sinus and Nasal Quality of Life Survey (SN-5) is a validated quality of life (QOL) questionnaire for chronic rhinosinusitis in patients age 2-12. Its utility in the cystic fibrosis (CF) has been studied, but not yet validated. The purpose of this study is to determine the effectiveness of the SN-5 for evaluation of sinonasal symptoms in the pediatric CF population.
METHODS: This retrospective study analyzed SN-5 surveys completed between 2012 and 2015 by pediatric CF patients and caregivers. Baseline and follow-up overall QOL scores and specific symptom scores were obtained from surveys completed in the three-year span. Non-parametric statistics were conducted to identify differences in survey data.
RESULTS: A total of 165 patients completed baseline and follow-up surveys. The overall QOL of the patient cohort did not change over the duration of the study (p = 0.660). Thirty-seven patients indicated higher overall QOL, with all five symptom scores showing significant improvement. Analysis by age group showed that QOL was significantly correlated with all five symptoms for children ages 0-4. In patients 5-12 years, overall QOL was only correlated with sinus infection (r = -0.3090, p = 0.01). QOL was significantly correlated with sinus infection (r = -0.2903, p = 0.04) and allergy symptoms (r = -0.5644, p < 0.01) in patients >12 years of age.
CONCLUSION: There remains a need for a validated CRS QOL tool for children with CF. Though the SN-5 has previously been described as a potential instrument, our data suggest that it may be more valuable in children ages 0-4.

PMID: 29106860 [PubMed - in process]

Letter to the editors: Cystic fibrosis liver disease in adults: limits of non invasive tests of fibrosis.

Hepatology. 2017 Nov 03;:

Authors: Hillaire S, Cazals-Hatem D, Erlinger S, Paradis V

PMID: 29105109 [PubMed - as supplied by publisher]

Reply: Cystic Fibrosis Liver Disease in Adults: limits of non-invasive tests of fibrosis.

Hepatology. 2017 Nov 03;:

Authors: Koh C, Sakiani S, Heller T

PMID: 29105100 [PubMed - as supplied by publisher]