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Impact of guideline-recommended dietitian assessments on weight gain in infants with cystic fibrosis.
Ong, T, Onchiri, FM, Britto, MT, Heltshe, SL, Kessler, LG, Seid, M, Ramsey, BW
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2022;(1):115-122
Abstract
BACKGROUND Cystic fibrosis (CF)-specialized nutrition care strives to meet normal infant growth, but the relationship of dietitian assessments to weight outcomes is unknown. We characterize nutrition management for inadequate weight gain and assess association of dietitian assessments and center-level weight-for-age Z-scores (WAZ). METHODS We used encounter data from 226 infants across 28 US CF Centers from the Baby Observational Nutritional study between January 2012 through December 2017. We identified dietitian assessments and consensus guideline-recommended responses to inadequate weight gain: calorie increases, pancreatic enzyme replacement therapy (PERT) increases, or shortened time to next visit. We compared center assessments by funnel plot and summarize median WAZ by center. RESULTS Of 2,527 visits, 808 (32%) visits had identified inadequate weight gain, distributed in 216 infants. Assessments occurred in 1953 visits (77%), but varied widely between centers (range 17% - 98%). For inadequate weight gain, most and least common responses were calorie increase (64%) and PERT increase (21%). Funnel plot analysis identified 4 high-performers for frequent dietitian assessments (range 92% - 98%) and 4 under-performers (range 17% - 56%). High-performers treated inadequate weight gain more often with adequate calories (24/30, 80% v. 12/23, 52%) and closer follow up (104/164, 63% v. 60/120, 49%) compared to under-performers. Three of 4 high-performing sites met center nutrition goals for positive median WAZ at 2 years old unlike 3 under-performers (WAZHigh 0.33 v. WAZLow -0.15), despite similar patient characteristics. CONCLUSION We characterized multicenter variation in dietitian assessments, identifying opportunities to improve care delivery to target early nutrition outcomes.
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Overweight and obesity in adults with cystic fibrosis: An Italian multicenter cohort study.
Gramegna, A, Aliberti, S, Contarini, M, Savi, D, Sotgiu, G, Majo, F, Saderi, L, Lucidi, V, Amati, F, Pappalettera, M, et al
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2022;(1):111-114
Abstract
BACKGROUND Over the last decades aggressive interventions have been successful to improve nutritional outcomes in people with cystic fibrosis (CF). As a result, with improvement of life expectancy and new CFTR modulators, overweight and obesity are progressively becoming a source of concern for adult population and in developed countries. METHODS This was a multicenter, observational, cross-sectional study of 321 adults with CF at three large CF centers in Italy. Patients were divided into three groups according to BMI classes, overweight and obesity (OW) group including patients with BMI ≥25 kg/m2, normal weight (NW) group with BMI 18.6-24.9 kg/m2 and underweight (UW) group with BMI ≤18.5 kg/m2. RESULTS We demonstrated that prevalence of OW in adults with CF in Italy is 22%. OW status is independently associated with male sex (OR 3.520, P = 0.001), pancreatic sufficiency (OR 2.873, P = 0.014) and older age at diagnosis (1.015, P = 0.042). BMI correlated with ppFEV1 (r = 0.337; P<0.0001) with median ppFEV1 significantly higher in patients with OW than comparisons. We also reported preliminary data on unfavorable cardiovascular risk factors in a subgroup of patients, where median blood levels [IQR] of cholesterol and systemic hypertension [%] were significantly higher in the OW group than in the NW and UW. CONCLUSIONS People with CF and OW is a relevant patient group that might deserve better definition and proper clinical management.
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Measuring the impact of CFTR modulation on sweat chloride in cystic fibrosis: Rationale and design of the CHEC-SC study.
Zemanick, ET, Konstan, MW, VanDevanter, DR, Rowe, SM, Clancy, JP, Odem-Davis, K, Skalland, M, Mayer-Hamblett, N
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2021;(6):965-971
Abstract
BACKGROUND The Characterizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes (CHEC-SC) study is a large epidemiologic study designed to determine the relationship between sweat chloride response and clinical outcomes in people with cystic fibrosis (CF) on commercially approved CFTR modulators. A challenge to study feasibility was capturing sweat chloride measurements before modulator initiation. We tested the hypothesis that historic sweat chloride approximated contemporary pre-modulator values to estimate CFTR modulator-induced changes, allowing a single-visit study design. METHODS GOAL and PROSPECT were multi-center prospective studies of individuals initiating ivacaftor or lumacaftor-ivacaftor. At enrollment, pre-modulator sweat chloride was measured and historic results recorded. Post-modulator sweat chloride was measured at 1, 3 and 6 months. For this analysis, differences between historic and pre-modulator sweat chloride were estimated. CFTR modulator-induced sweat chloride mean changes were compared using historic and pre-modulator sweat chloride. RESULTS Paired historic and pre-modulator sweat chloride (n=406 participants) revealed a non-significant mean change of -1.0 mmol/L (95% CI: -2.71, 0.66) over an average of 17.2 years. Calculating sweat response to ivacaftor or lumacaftor-ivacaftor using historic or pre-modulator values resulted in similar estimates of modulator response. Based on these results, the CHEC-SC study was designed with a single, post-modulator sweat chloride measurement. CONCLUSIONS Historic sweat chloride values provide a reliable estimate of pre-modulator sweat chloride for people starting on modulator therapy. The CHEC-SC study anticipates capturing approximately 5,000 sweat chloride values, providing an unprecedented understanding of sweat chloride across the CF population in the era of CFTR modulators.
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Novel imaging techniques for cystic fibrosis lung disease.
Goralski, JL, Stewart, NJ, Woods, JC
Pediatric pulmonology. 2021;(Suppl 1):S40-S54
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Abstract
With an increasing number of patients with cystic fibrosis (CF) receiving highly effective CFTR (cystic fibrosis transmembrane regulator protein) modulator therapy, particularly at a young age, there is an increasing need to identify imaging tools that can detect and regionally visualize mild CF lung disease and subtle changes in disease state. In this review, we discuss the latest developments in imaging modalities for both structural and functional imaging of the lung available to CF clinicians and researchers, from the widely available, clinically utilized imaging methods for assessing CF lung disease-chest radiography and computed tomography-to newer techniques poised to become the next phase of clinical tools-structural/functional proton and hyperpolarized gas magnetic resonance imaging (MRI). Finally, we provide a brief discussion of several newer lung imaging techniques that are currently available only in selected research settings, including chest tomosynthesis, and fluorinated gas MRI. We provide an update on the clinical and/or research status of each technique, with a focus on sensitivity, early disease detection, and possibilities for monitoring treatment efficacy.
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Factors Contributing to Vitamin D Status at Hospital Admission for Pulmonary Exacerbation in Adults With Cystic Fibrosis.
Bhimavarapu, A, Deng, Q, Bean, M, Lee, N, Ziegler, TR, Alvarez, J, Tangpricha, V
The American journal of the medical sciences. 2021;(1):75-82
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Abstract
BACKGROUND Individuals with cystic fibrosis (CF) have difficulty maintaining optimal vitamin D status due to pancreatic insufficiency-induced malabsorption, inadequate sunlight exposure, and poor intake of vitamin D containing foods. Vitamin D deficiency may increase the risk of pulmonary exacerbations of CF. The objective of this study was to assess factors impacting vitamin D status in patients with CF recently hospitalized for a pulmonary exacerbation of CF. METHODS This was a pre-planned analysis of vitamin D intake in patients enrolled in a multi-center, double-blind, randomized controlled study examining vitamin D therapy for pulmonary exacerbation of CF. Demographic information, responses from a habitual sun exposure questionnaire and food frequency questionnaire, and vitamin D supplement usage were queried and compared to serum 25-hydroxyvitamin D (25(OH)D) concentrations. RESULTS A total of 48 subjects were included in this analysis. Subjects were taking approximately 1,200 IU of vitamin D daily. Reported vitamin D intake, age, race, employment, and education were not significantly associated with vitamin D status in this population. However, smoking status, sunlight exposure in the last 3 years, and skin type (in the bivariate model) were all significantly associated with vitamin D status (all p<0.05). CONCLUSIONS Sunlight exposure was the most predictive determinant of vitamin D status in patients with CF prior to pulmonary exacerbation. Subjects reported vitamin D intake below the recommended amounts. The role and mode of optimizing vitamin D status prior to a pulmonary exacerbation needs further investigation.
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Hypertonic saline in people with cystic fibrosis: review of comparative studies and clinical practice.
Terlizzi, V, Masi, E, Francalanci, M, Taccetti, G, Innocenti, D
Italian journal of pediatrics. 2021;(1):168
Abstract
Cystic fibrosis (CF) is a multisystem disorder, caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. These cause a reduced secretion of chloride, a marked absorption of sodium and, therefore, of water, through the epithelium, resulting in the formation of thickened secretions in organs such as lung or pancreas. These viscous secretions lead to airway obstruction, chronic infection and inflammation resulting in progressive lung damage, bronchiectasis and eventual respiratory failure. Although the average life expectancy has increased over the last 30 years, lung disease is the most common cause of death in people with CF. For these reasons, the improvement of sputum clearance is a major therapeutic aim in CF and early initiation of airway clearance is widely recommended and implemented. Symptomatic mucolytic therapy today is mainly based on inhalation of DNase, hypertonic saline or mannitol, in combination with physiotherapy. Mucolytic agents break down the gel structure of mucus and therefore decrease its elasticity and viscosity, reducing the pulmonary exacerbation frequency and to improve and stabilize lung function. Nevertheless, high quality studies comparing these mucolytic drugs are still few, and the individual experiences of patients and caregivers explain the high variability of their use globally. This review will summarize the current knowledge on hypertonic saline in the treatment of CF lung disease. Furthermore, we report the real-world prescription of inhaled mucolytic agents in CF.
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Vitamin D supplementation in patients with cystic fibrosis: A systematic review and meta-analysis.
Juhász, MF, Varannai, O, Németh, D, Szakács, Z, Kiss, S, Izsák, VD, Martonosi, ÁR, Hegyi, P, Párniczky, A
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2021;(5):729-736
Abstract
Despite routine supplementation, vitamin D insufficiency is often seen in cystic fibrosis (CF) patients on account of pancreatic insufficiency. Vitamin D is a crucial component of bone health and affects nearly all cells of the immune system. However, clinical benefits or harms associated with supplementation are poorly documented. In this systematic review, we included randomized controlled trials (RCTs) that compared vitamin D supplementation with placebo (i.e. 'non-increased dose') in CF patients. Analysing the 8 included RCTs, the intervention group had significantly higher serum 25-hydroxyvitamin D (se25OHD) levels, but there were no significant differences found in the quantitative synthesis of clinical outcomes, including bone disease-, respiratory status- and immunological status-related outcomes. Based on our current results, while a higher vitamin D dose elevates se25OHD, it does not seem to influence clinical outcomes. Future RCTs should include outcomes of past studies and apply longer follow-up periods to document long-term patient-important outcomes.
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Reversible tremor in an infant with vitamin E deficiency and cystic fibrosis.
Lo Barco, T, Ghobert, AL, Lucca, F, Galati, G, Proietti, J, Cantalupo, G
Lancet (London, England). 2021;(10295):156
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Minimising the risk of cross infection between siblings with cystic fibrosis (CF) within the home: Successful domestic steam disinfection of CF bacterial and foodborne pathogens on common household cutlery and crockery utensils.
Millar, BC, Moore, JE
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2021;(4):708-711
Abstract
Avoidance of cross infection is important between CF siblings living in the same household. In this study, we examined the ability of domestic steam disinfection to eradicate 16 species of CF bacterial and foodborne pathogens from the surface of contaminated crockery and cutlery. Domestic steam disinfection employing baby bottle disinfector devices, when performed properly under manufacturer's instructions, eradicated all organisms tested and offers a relatively inexpensive, simple, versatile and widely available technology for the elimination of common CF bacterial and foodborne pathogens from contaminated crockery and cutlery utensils. Most CF households may already have a baby bottle disinfector device, in order to disinfect nebulizers, respiratory equipment and toothbrushes and therefore employment of this device to disinfect crockery and cutlery would be easily achieved. We therefore advocate the employment of such devices to disinfect such common household utensils, as a critical control in the elimination of these organisms from these sources, thereby enhancing CF sibling safety.
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Gastroesophageal Reflux Disease and Need for Antireflux Surgery in Children with Cystic Fibrosis: A Systematic Review on Incidence, Surgical Complications, and Postoperative Outcomes.
Ng, J, Friedmacher, F, Pao, C, Charlesworth, P
European journal of pediatric surgery : official journal of Austrian Association of Pediatric Surgery ... [et al] = Zeitschrift fur Kinderchirurgie. 2021;(1):106-114
Abstract
INTRODUCTION Gastroesophageal reflux disease (GERD) is associated with accelerated decline in lung health in children with cystic fibrosis (CF). Thus, antireflux surgery (ARS) is offered to a selected CF cohort with refractory GERD, but outcomes remain poorly investigated. This study aimed to determine the incidence of GERD in children with CF and to evaluate complications and outcomes of ARS. MATERIALS AND METHODS A systematic literature-based search was conducted using various online databases according to Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines. The number of GERD cases in pediatric CF cohorts who underwent diagnostic investigation(s) was recorded. Data on postoperative complications and outcomes (including symptoms, lung function, and nutritional status) following ARS were analyzed. RESULTS Ten articles (n = 289 patients) met the defined inclusion criteria (51% male; age range, 0.5 month-36 years). The overall incidence of GERD was 46% (range, 19-81%), derived from seven studies (n = 212 patients). Four publications (n = 82 patients) reported on ARS due to uncontrolled GERD. All ARSs were Nissen fundoplication (majority with gastrostomy placement). Major postoperative complications occurred in 15 (18%) patients, two required redo-ARS. Median follow-up time was 2 years (range, 3 months-6 years); 59% showed symptom improvement, and pulmonary exacerbations and decline in lung function were reduced. Nutritional status mainly improved in milder CF cases. There were no deaths related to ARS. CONCLUSION Approximately half of pediatric CF patients have GERD. Published data for children with CF are limited and heterogeneous in terms of GERD diagnosis and outcomes following ARS. However, ARS has shown to slow the deterioration of lung function in CF.