Cystic-fibrosis related-diabetes (CFRD) is preceded by and associated with growth failure and deteriorating lung function

2017 ◽  
Vol 30 (8) ◽  
Author(s):  
Nicolas Terliesner ◽  
Mandy Vogel ◽  
Anna Steighardt ◽  
Ruth Gausche ◽  
Constance Henn ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Children And Adolescents ◽  
Pancreatic Insufficiency ◽  
Growth Failure ◽  
Forced Expiratory Volume ◽  
Exocrine Pancreatic Insufficiency ◽  
Retrospective Case ◽  
Poor Growth ◽  
Impaired Lung Function

AbstractBackground:Impaired glucose metabolism and cystic fibrosis (CF)-related diabetes (CFRD) are associated with insufficient weight gain and impaired lung function in children and adolescents with CF. We have asked whether imminent CFRD may be a cause of poor growth in children and adolescents.Methods:A retrospective case control study including 32 patients with CF with or without diabetes was conducted. Sixteen pairs, matched according to age, gender and exocrine pancreatic insufficiency, were analysed. Standard deviation scores (SDS) of height, growth, weight, body mass index (BMI), forced vital capacity (FVC), forced expiratory volume in the first second (FEV1) and forced expiratory flow at 75% of expired FVC (FEF75) were recorded during a mean observation period of 13 years per patient.Results:SDS of height and weight were reduced in CF patients with diabetes compared to those without, not only at the point of diagnosis (both p<0.05) but years before the evidence of diabetes. Afterwards there was a significant decline in height (p<0.001) and weight (p<0.01) SDS in CFRD patients and an increasing difference between the height and weight of CF patients with or without diabetes. In contrast, no significant reduction of BMI-SDS was observed in CFRD patients. All analysed lung function parameters showed a marked decline in CFRD patients starting 1 year prior to the diagnosis of diabetes.Conclusions:Deteriorating growth, reduced weight and impaired lung function are related to the development of CFRD and are obvious several years before the actual diagnosis of diabetes.

scholarly journals Lung function over the life course of paediatric and adult patients with cystic fibrosis from a large multi-centre registry

2020 ◽  
Vol 10 (1) ◽  
Author(s):  
Arul Earnest ◽  
Farhad Salimi ◽  
Claire E. Wainwright ◽  
Scott C. Bell ◽  
Rasa Ruseckaite ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Mixed Model ◽  
Linear Mixed Model ◽  
Pancreatic Insufficiency ◽  
Forced Expiratory Volume ◽  
Rate Of Change ◽  
Data Registry ◽  
Mortality Outcome ◽  
The Impact

Abstract A key measure of lung function in people with Cystic Fibrosis (CF) is Forced Expiratory Volume in the first second FEV1 percent predicted (FEV1pp). This study aimed to address challenges in identifying predictors of FEV1pp, specifically dealing with non-linearity and the censoring effect of death. Data was obtained from a large multi-centre Australian Cystic Fibrosis Data Registry (ACFDR). A linear mixed model was used to study FEV1pp as the endpoint. There were 3655 patients (52.4% male) included in our study. Restricted cubic splines were used to fit the non-linear relationship between age of visit and FEV1pp. The following predictors were found to be significant in the multivariate model: age of patient at visit, BMI z-score, age interaction with lung transplantation, insulin dependent diabetes, cirrhosis/portal hypertension, pancreatic insufficiency, Pseudomonas aeruginosa infection and baseline variability in FEV1pp. Those with P. aeruginosa infection had a lower mean difference in FEV1pp of 4.7 units, p < 0.001 compared to those who did not have the infection. Joint modelling with mortality outcome did not materially affect our findings. These models will prove useful for to study the impact of CFTR modulator therapies on rate of change of lung function among patients with CF.

scholarly journals Heart rate variability, exercise capacity and levels of daily physical activity in children and adolescents with mild-to-moderate cystic fibrosis

2021 ◽  
pp. 1-10
Author(s):  
Pitiguara de Freitas Coelho ◽  
Roberta Ribeiro Batista Barbosa ◽  
Rodrigo dos Santos Lugao ◽  
Fernanda Mayrink Gonçalves Liberato ◽  
Pâmela Reis Vidal ◽  
...  
Keyword(s):  
Physical Activity ◽  
Cystic Fibrosis ◽  
Heart Rate ◽  
Heart Rate Variability ◽  
Lung Function ◽  
Children And Adolescents ◽  
Exercise Capacity ◽  
Pancreatic Insufficiency ◽  
Daily Physical Activity ◽  
Rr Intervals

Background: Autonomic nervous system balance is altered in cystic fibrosis (CF), although its influence on physical fitness has been poorly explored. Objective: This study aimed to evaluate the association of heart rate variability (HRV) with exercise capacity and levels of daily physical activity in children and adolescents with mild-to-moderate CF. Methods: A cross-sectional study including individuals with CF aged 6–18 years, not under CFTR modulator therapy, was performed. Sociodemographic (age, sex) and clinical information (airway colonization, pancreatic insufficiency, and genotyping) were collected. In addition, exercise capacity (modified shuttle test — MST), lung function (spirometry), body composition (bioimpedance), levels of daily physical activity (5-day accelerometer), and HRV (both at rest and during the MST) were evaluated. Results: 30 individuals (20 females) aged [Formula: see text] years, mean FEV[Formula: see text]%, were included. A sympathovagal balance (LF/HF) increase ([Formula: see text]) during the MST was shown, indicating a predominance of sympathetic modulation. The standard deviation of all RR intervals (SDNN) and the high frequency (HF) index during exercise correlated significantly with FEV1 ([Formula: see text], [Formula: see text] and [Formula: see text], [Formula: see text]; respectively). MST distance also correlated positively and significantly with SDNN ([Formula: see text], [Formula: see text]), square root of the mean of the sums of squares of frequencies between RR intervals greater than 50[Formula: see text]ms — RMSSD ([Formula: see text], [Formula: see text]), low frequency — LF ([Formula: see text], [Formula: see text]), HF ([Formula: see text], [Formula: see text]), dispersion of points perpendicular to the short-term identity line — SD1 ([Formula: see text], [Formula: see text]) and negatively with LF/HF ([Formula: see text], [Formula: see text]). Regarding daily physical activity, SDNN at rest ([Formula: see text], [Formula: see text]) and exercise ([Formula: see text], [Formula: see text]) showed positive correlations with time in moderate-to-vigorous activities. When normalizing the SDNN and classifying individuals as normal or altered, those presenting altered SDNN showed poorest FEV1 ([Formula: see text]) and lower exercise capacity ([Formula: see text]). Conclusion: HRV correlates with lung function, exercise capacity and levels of daily physical activity in children and adolescents with CF. The study highlights the influence of CF on autonomic function and suggests HRV measurement as an easy tool to be used in clinical settings as an alternative marker to monitor CF individuals.

scholarly journals Lung function and disease severity in cystic fibrosis patients heterozygous for p.Arg117His

2017 ◽  
Vol 3 (1) ◽  
pp. 00056-2016 ◽  
Author(s):  
Michal Shteinberg ◽  
Damian G. Downey ◽  
Diane Beattie ◽  
John McCaughan ◽  
Alastair Reid ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Function ◽  
Disease Severity ◽  
Pancreatic Insufficiency ◽  
Forced Expiratory Volume ◽  
Class I ◽  
Lung Function Decline ◽  
Diagnostic Features ◽  
Sweat Chloride ◽  
In Trans

Expression of p.Arg117His cystic fibrosis (CF) transmembrane conductance regulator is influenced by a polythymidine (poly-T) tract and a thymidine–guanine (TG) repeat on intron 9, which vary in length and affect exon 10 skipping.We compared clinical characteristics and the rate of progression of lung disease of CF patients carrying the p.Arg117His mutation with different intron 9 varying sequences (poly-T) and mutation classes in trans.Data were collected from patients in Northern Ireland, UK, including diagnostic features, sweat chloride, nutritional status, sputum microbiology, CF-related complications and lung function. Poly-T and TG repeats were determined by PCR. Forced expiratory volume in 1 s (FEV1) decline was determined from linear regression of FEV1 measurements of patients over time.We identified 62 patients with p.Arg117His, 55 with a class I/II mutation in trans and six with p.Arg117His/p.Gly551Asp. 42 patients had 5T and 13 had 7T. All patients had 12 TG repeats. Patients with p.Arg117His-5T had greater lung function decline, sweat chloride concentrations, pancreatic insufficiency and prevalence of Pseudomonas aeruginosa infection compared with patients with p.Arg117His-7T.Lung function decline and disease severity in p.Arg117His is determined by the poly-T tract length and identity of the mutation in trans. Patients with p.Arg117His-5T and a second class I/II mutation have a severity similar to p.Phe508del homozygous patients, although lung function decline is delayed to an older age. There may be linkage disequilibrium between p.Arg117His and 12 TG repeats.

scholarly journals Digestive enzyme replacement relieves growth failure in preterm infants with poor exocrine pancreatic function: a retrospective case series

2021 ◽  
Author(s):  
Annette Münch ◽  
Christoph Bührer ◽  
Ann Carolin Longardt
Keyword(s):  
Weight Gain ◽  
Preterm Infants ◽  
Digestive Enzyme ◽  
Pancreatic Insufficiency ◽  
Growth Failure ◽  
Exocrine Pancreatic Function ◽  
Exocrine Pancreatic Insufficiency ◽  
Very Preterm Infants ◽  
Enzyme Replacement ◽  
Fecal Pancreatic Elastase

AbstractIn orally fed preterm infants, poor weight gain may be linked to low fecal pancreatic elastase-1 (FPE-1) activity, indicative of exocrine pancreatic insufficiency. The objective of this study was the retrospective assessment of the effect of exogenous digestive enzyme replacement by gavage in preterm infants with growth failure and low FPE-1 (<200 μg/g). We analyzed weight gain relative to baseline and caloric intake during 14-day periods before and after institution of digestive enzyme replacement containing 6000 U lipase and 240 U protease kg−1 d−1. Among 46 of 132 preterm infants < 1250g birth weight surviving to at least 14 days in whom FPE-1 was determined, 38 infants had low FPE-1 (< 200 μg/g), and 33 infants received exogenous digestive enzyme replacement. Average daily weight gain significantly increased from 14.4 [range 2.6–22.4] g kg−1 d−1 to 17.4 [8.4–29.0] g kg−1 d−1 (P = 0.001), as did weight gain per kcal, from 0.08 [0.02–0.13] g kcal−1 d−1 to 0.11 [0.05–0.18] g kcal−1 d−1.Conclusion: In preterm infants with signs and symptoms of exocrine pancreatic insufficiency, exogenous digestive enzyme replacement is associated with improved growth. What is Known:• Very preterm infants on full enteral nutrition may display growth failure linked to transient poor exocrine pancreatic function.• Porcine pancreatic enzymes covered with an acid-resistant coating are too large to pass the internal diameter of most gavage tubes used in very preterm infants.What is New:• Administration of a liquid formulation of acid-resistant microbial digestive enzymes in preterm infants with growth failure and low fecal pancreatic elastase-1 values was associated with improved weight gain.• Response to exogenous digestive enzyme replacement was associated with the prior extent of growth failure.

scholarly journals 2 The syndrome of exocrine pancreatic insufficiency – cystic fibrosis or other disease?

2012 ◽  
Vol 11 ◽  
pp. S55
Author(s):  
A. Sobczynska-Tomaszewska ◽  
K. Czerska ◽  
A. Szpecht-Potocka ◽  
D. Popielarz ◽  
K. Wertheim-Tysarowska ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Pancreatic Insufficiency ◽  
Exocrine Pancreatic Insufficiency

scholarly journals Efficacy and Safety of a New Formulation of Pancrelipase (Ultrase MT20) in the Treatment of Malabsorption in Exocrine Pancreatic Insufficiency in Cystic Fibrosis

2010 ◽  
Vol 2010 ◽  
pp. 1-7 ◽  
Author(s):  
Michael W. Konstan ◽  
Theodore G. Liou ◽  
Steven D. Strausbaugh ◽  
Richard Ahrens ◽  
Jamshed F. Kanga ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Pancreatic Insufficiency ◽  
Pancreatic Enzyme ◽  
Gastrointestinal Symptoms ◽  
Signs And Symptoms ◽  
Exocrine Pancreatic Insufficiency ◽  
Efficacy And Safety ◽  
Protein Nitrogen ◽  
Enzyme Replacement ◽  
New Formulation

Background. Pancreatic enzyme replacement therapy is the standard of care for treatment of malabsorption in patients with cystic fibrosis (CF) and exocrine pancreatic insufficiency (PI).Aim. To evaluate efficacy and safety of a new formulation of pancrelipase (Ultrase MT20) in patients with CF and PI. Coefficients of fat absorption (CFA%) and nitrogen absorption (CNA%) were the main efficacy parameters. Safety was evaluated by monitoring laboratory analyses, adverse events (AEs), and overall signs and symptoms.Methods. Patients (n=31) were randomized in a crossover design comparing this pancrelipase with placebo during 2 inpatient evaluation periods (6-7 days each). Fat and protein/nitrogen ingestion and excretion were measured from food diaries and 72-hour stool collections. CFA% and CNA% were calculated for each period and compared.Results. Twenty-four patients provided analyzable data. This pancrelipase increased mean CFA% and CNA% (+34.7% and +25.7%, resp.,P<.0001for both), reduced stool frequency, and improved stool consistency compared with placebo. Placebo-treated patients reported more AEs, with gastrointestinal symptoms being the most frequently reported AE.Conclusions. This pancrelipase is a safe and effective treatment for malabsorption associated with exocrine PI in patients with CF.

Sign in / Sign up

Export Citation Format

Share Document