Publications
164 results found
Archangelidi O, Carr SB, Simmonds NJ, et al., 2019, Non-invasive ventilation and clinical outcomes in cystic fibrosis: Findings from the UK CF registry, Journal of Cystic Fibrosis, Vol: 18, Pages: 665-670, ISSN: 1569-1993
Background: Non-invasive ventilation (NIV) for respiratory failure and airway clearance is an established intervention in cystic fibrosis (CF), but its therapeutic benefit on lung function and survival remains under-investigated. Methods: Using data from the UK CF Registry between 2007 and 2015, we explored the patterns of NIV use, and assessed changes in mean percent predicted FEV1 (ppFEV1) prior to and after NIV use, and the survival of patients on NIV. Results: Among 11,079 patients, 1107 had at least one record of NIV treatment. Incidence and prevalence of NIV was lower in children and followed non-linear temporal patterns. Adjusting for other risk factors, ppFEV1 rose by 0.70 (95%CI: -0.83, 2.24) after first NIV use in children. In adults with a low ppFEV1 (<40%) at initiation of treatment, NIV increased mean ppFEV1 by 2.60 (95% CI: 0.93, 4.27). Our analysis showed that NIV initiation is associated with an increased risk of death/transplant in both children (HR = 2.47; 95%CI: 1.20–5.08) and adults (HR = 1.96; 95% CI: 1.63–2.36) but effect was attenuated in children with low ppFEV1 (<40%). Conclusions: NIV usage in CF improves spirometric values but does not benefit survival. Further studies are required to better understand survival outcomes and ultimately improve NIV outcomes in CF.
Di Paolo M, Teopompi E, Savi D, et al., 2019, Reduced exercise ventilatory efficiency in adults with cystic fibrosis and normal to moderately impaired lung function, JOURNAL OF APPLIED PHYSIOLOGY, Vol: 127, Pages: 501-512, ISSN: 8750-7587
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- Citations: 7
Dobra R, Madge S, Martin I, et al., 2019, "Fortunate are those who take the first steps"? The psychosocial impact of novel drug development., Paediatric Respiratory Reviews, Vol: 31, Pages: 9-11, ISSN: 1526-0542
Novel drug development offers people with cystic fibrosis exciting opportunities but is not without challenges. Currently, there is an understandable emphasis on protecting patients' physical health when developing treatments. However, there appears to be little consideration of how novel drug development impacts on psychosocial wellbeing, or the downstream consequences of this. Using an illustrative case and reviewing the literature we explore themes regarding the psychosocial impact of trial participation and novel drug development and identify areas requiring further research. Through this, we hope to prepare healthcare professionals to better understand the needs of their patients in this rapidly evolving landscape.
Simmonds NJ, 2019, Is it cystic fibrosis? The challenges of diagnosing cystic fibrosis, PAEDIATRIC RESPIRATORY REVIEWS, Vol: 31, Pages: 6-8, ISSN: 1526-0542
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- Citations: 12
Vijayasingam A, Frost E, Wilkins J, et al., 2019, S140 Interim results from a prospective study of tablet and web-based audiometry to detect ototoxicity in adults with cystic fibrosis (vol 73, pg A87, 2018), THORAX, Vol: 74, Pages: 723-723, ISSN: 0040-6376
Savi D, Schiavetto S, Simmonds NJ, et al., 2019, Effects of Lumacaftor/Ivacaftor on physical activity and exercise tolerance in three adults with cystic fibrosis, JOURNAL OF CYSTIC FIBROSIS, Vol: 18, Pages: 420-424, ISSN: 1569-1993
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- Citations: 19
Stanford G, Parrott H, Bilton D, et al., 2019, A randomised crossover trial evaluating the short-term effects of non-invasive ventilation as an adjunct to airway clearance techniques in adults with cystic fibrosis, BMJ Open Respiratory Research, Vol: 6, ISSN: 2052-4439
Introduction Non-invasive ventilation (NIV) is used in cystic fibrosis (CF) to support airway clearance techniques (ACTs) by augmenting tidal volumes and reducing patient effort. However, the evidence base for this is limited. We hypothesised that NIV, in addition to usual ACT, would increase sputum clearance. In addition, we investigated ease of sputum clearance (EoC), work of breathing (WoB) and NIV tolerability.Methods Adults with CF (16+ years) at the end of hospitalisation for a pulmonary exacerbation were randomised to a cross-over trial of NIV-supported ACT or ACT alone in two consecutive days. No other changes to standard care were made. The primary outcome was the total 24-hour expectorated sputum wet weight after the intervention. Spirometry was completed pre-treatment and post-treatment. Oxygen saturations were measured pre-treatment, during treatment and post-treatment. EoC and WoB were assessed using Visual Analogue Scale.Results 14 subjects completed the study (7 male, mean age 35 [SD 17] years, mean forced expiratory volume in 1 s [FEV1] 49 [20] % predicted). The difference between treatment regimens was −0.98 g sputum (95% CI −11.5 to 9.6, p=0.84) over 24 hours. During treatment oxygen saturations were significantly higher with NIV-supported ACT (mean difference 2.0, 95% CI 0.9 to 2.6, p=0.0004). No other significant differences were found in post-treatment FEV1, EoC, WoB, oxygen saturations or subject preference.Conclusions There was no difference in treatment effect between NIV-supported ACT and ACT alone, although the study was underpowered. Oxygen saturations were significantly higher during NIV-supported ACT, but with no effect on post-treatment saturations. NIV was well tolerated.
Toledano M, Mukherjee S, Howell J, et al., 2019, The emerging burden of liver disease in cystic fibrosis patients: a UK nationwide study, PLoS ONE, Vol: 14, ISSN: 1932-6203
ObjectiveCystic fibrosis associated liver disease (CFLD) is the third largest cause of mortality in CF. Our aim was to define the burden of CFLD in the UK using national registry data and identify risk factors for progressive disease.MethodsA longitudinal population-based cohort study was conducted. Cases were defined as all patients with CFLD identified from the UK CF Registry, 2008–2013 (n = 3417). Denominator data were derived from the entire UK CF Registry. The burden of CFLD was characterised. Regression analysis was undertaken to identify risk factors for cirrhosis and progression.ResultsPrevalence of CFLD increased from 203.4 to 228.3 per 1000 patients during 2008–2013. Mortality in CF patients with CFLD was more than double those without; cirrhotic patients had higher all-cause mortality (HR 1.54, 95% CI 1.09 to 2.18, p = 0.015). Median recorded age of cirrhosis diagnosis was 19 (range 5–53) years. Male sex, Pseudomonas airway infection and CF related diabetes were independent risk factors for cirrhosis. Ursodeoxycholic acid use was associated with prolonged survival in patients without cirrhosis.ConclusionsThis study highlights an important changing disease burden of CFLD. The prevalence is slowly increasing and, importantly, the disease is not just being diagnosed in childhood. Although the role of ursodeoxycholic acid remains controversial, this study identified a positive association with survival.
De Tarso Dalcin PR, Figueiredo da Silva G, Simmonds N, 2019, Clinical Characteristics and Outcomes in Adult Cystic Fibrosis Patients with Severe Lung Disease in Southern Brazil, International Conference of the American-Thoracic-Society, Publisher: AMER THORACIC SOC, ISSN: 1073-449X
Simmonds NJ, Pabary R, Kohlhaufl J, et al., 2018, THE ADDED VALUE OF NASAL POTENTIAL DIFFERENCE MEASUREMENT WHEN FIRST-LINE CYSTIC FIBROSIS (CF) INVESTIGATIONS ARE NON-DIAGNOSTIC, Winter Meeting of the British-Thoracic-Society, Publisher: BMJ PUBLISHING GROUP, Pages: A84-A85, ISSN: 0040-6376
Simmonds NJ, Castellani C, Colombo C, et al., 2018, CLINICAL EFFECTIVENESS RESULTS FROM THE FIRST INTERIM ANALYSIS OF THE VOCAL STUDY; AN OBSERVATIONAL STUDY OF IVACAFTOR IN PATIENTS WITH CYSTIC FIBROSIS AND SELECTED NON-G551D GATING MUTATIONS, Winter Meeting of the British-Thoracic-Society, Publisher: BMJ PUBLISHING GROUP, Pages: A43-A43, ISSN: 0040-6376
Vijayasingam A, Shah A, Simmonds NJ, et al., 2018, INTERIM RESULTS FROM A PROSPECTIVE STUDY OF TABLET AND WEB-BASED AUDIOMETRY TO DETECT OTOTOXICITY IN ADULTS WITH CYSTIC FIBROSIS, THORAX, Vol: 73, Pages: A87-A88, ISSN: 0040-6376
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- Citations: 1
Abdolrasouli A, Bercusson AC, Rhodes JL, et al., 2018, Airway persistence by the emerging multi-azole-resistant Rasamsonia argillacea complex in cystic fibrosis, Mycoses, Vol: 61, Pages: 665-673, ISSN: 0933-7407
Infections caused by Rasamsonia argillacea complex have been reported in various clinical settings. Cystic fibrosis (CF) is one of the main underlying conditions. An observational cohort study of CF patients with Rasamsonia in respiratory samples was conducted. Eight isolates from six patients were identified as R. argillacea complex and tested for antifungal susceptibility. All isolates had high MICs to voriconazole and posaconazole and low MECs to echinocandins. Four patients experienced lung function decline in the year preceding first Rasamsonia isolation. This continued in the year following first isolation in three out of four cases. Antifungal therapy was initiated in two patients, to which only one exhibited a clinical response. Three out of six patients died within three years of isolating Rasamsonia. Genotyping suggests that similar genotypes of Rasamsonia can persist in CF airways. Consistent with other fungi in CF, the clinical impact of airway colonization by Rasamsonia is variable. In certain patients, Rasamsonia may be able to drive clinical decline. In others, though a clear impact on lung function may be difficult to determine, the appearance of Rasamsonia acts as a marker of disease severity. In others it does not appear to have an obvious clinical impact on disease progression.
Di Paolo M, Felton I, Blazquez PT, et al., 2018, EFFECTS OF EXTENDED FUNGAL CULTURES ON PREVALENCE OF FILAMENTOUS FUNGI INFECTIONS IN A LARGE ADULT CYSTIC FIBROSIS CENTRE, Publisher: WILEY, Pages: 290-291, ISSN: 8755-6863
Horsley AR, Blaas S, Burr L, et al., 2018, NOVEL CFTR CORRECTOR FDL169: SAFETY, PHARMACOKINETICS AND PHARMACODYNAMICS, Publisher: WILEY, Pages: 252-252, ISSN: 8755-6863
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- Citations: 1
Bilton D, Caine N, Cunningham S, et al., 2018, Use of a rare disease patient registry in long-term post-authorisation drug studies: a model for collaboration with industry, LANCET RESPIRATORY MEDICINE, Vol: 6, Pages: 495-496, ISSN: 2213-2600
Eades CP, Armstrong-James DPH, Periselneris J, et al., 2018, Improvement in Exophiala dermatitidis airway persistence and respiratory decline in response to interferon-gamma therapy in a patient with cystic fibrosis, Journal of Cystic Fibrosis, Vol: 17, Pages: e32-e34, ISSN: 1569-1993
Castellani C, Colombo C, van der Ent CK, et al., 2018, AN OBSERVATIONAL STUDY OF IVACAFTOR IN PATIENTS WITH CYSTIC FIBROSIS AND SELECTED NON-G551D GATING MUTATIONS IN UK, ITALY AND NETHERLANDS: HEALTHCARE RESOURCE UTILIZATION FROM THE FIRST INTERIM ANALYSIS OF THE VOCAL STUDY, Publisher: ELSEVIER SCIENCE INC, Pages: S255-S255, ISSN: 1098-3015
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- Citations: 1
Savi D, Di Paolo M, Simmonds NJ, et al., 2018, Is daily physical activity affected by dynamic hyperinflation in adults with cystic fibrosis?, BMC Pulmonary Medicine, Vol: 18, ISSN: 1471-2466
Background: The aim of this study was to investigate the relationship between dynamic hyperinflation and daily physical activity (DPA) in adults with cystic fibrosis (CF).Methods: Thirty-four clinically stable CF were studied. All patients undertook incremental cardiopulmonary exercise testing (CPET). CPET-related measurements included: oxygen uptake (V’O2), carbon dioxide production (V’CO2), ventilatory profile, work rate (W), inspiratory capacity (IC), end-expiratory lung volume (EELV). PA was assessed using the accelerometer SenseWear Pro3 Armband.Results: Exercise tolerance was reduced in most of patients and the mean V’O2,peak value was 75.2% of predicted (28.5 ± 4.8 ml/min/kg). Seventy % of patients responded to CPET with dynamic hyperinflation. Higher incidence of dynamic hyperinflation was found in CF males compared to CF females (p = 0.026). Patients who developed dynamic hyperinflation during CPET had higher vigorous PA (p = 0.01) and more total energy expenditure (p = 0.006) than patients who did not. EELVΔ was related to activities requiring vigorous intensity and total energy expenditure (R = 0.46, p = 0.001; R = 0.57, p < 0.001).Conclusions:In adults with CF and mild to moderate lung impairment, DPA might not be limited by dynamic hyperinflation.
Dunwoody R, Steel A, Landy J, et al., 2018, <i>Clostridium difficile</i> and cystic fibrosis: management strategies and the role of faecal transplantation, PAEDIATRIC RESPIRATORY REVIEWS, Vol: 26, Pages: 16-18, ISSN: 1526-0542
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- Citations: 8
Simmonds NJ, Sherrard LJ, Bell SC, 2018, Cystic fibrosis, Essentials of Clinical Pulmonology, Pages: 303-325, ISBN: 9781498715799
Cystic fibrosis (CF) is the most common lethal inherited condition affecting Caucasians, with an occurrence of -1 in 3, 000 live births, and it has been estimated that CF gene carrier rates range from 1 in 20-30 of the population. However, the incidence of CF varies considerably across the globe, and differences are also observed in populations with a similar origin, including Northern Europe. Incidence diverges between races and has been estimated as 1 in 4, 00010, 000 live births in Latin Americans and 1 in 20, 000 live births in African Americans, with a much lower incidence reported in people with an Asian background (<1 in 80, 000 live births). Furthermore, it was previously thought that a diagnosis of CF was unlikely in some regions of the world, including the Middle East and the Indian subcontinent; however, recently, increasing numbers of patients diagnosed with CF have been identified in these populations.
Castellani C, Simmonds NJ, 2018, Identifying undiagnosed cystic fibrosis in adults with bronchiectasis, BRONCHIECTASIS, Editors: Chalmers, Polverino, Aliberti, Bals, Publisher: EUROPEAN RESPIRATORY SOCIETY, Pages: 29-44, ISBN: 978-1-84984-097-2
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- Citations: 1
Hippolyte SS, Simmonds NJ, Bilton D, et al., 2017, ARE GIRLS ALWAYS THINNER THAN BOYS? USING UK CYSTIC FIBROSIS (CF) REGISTRY DATA (2008-2013) TO EXAMINE WEIGHT CHANGES BETWEEN THE SEXES FROM CHILDHOOD AND BEYOND, Winter Meeting of the British-Thoracic-Society, Publisher: BMJ PUBLISHING GROUP, Pages: A76-A77, ISSN: 0040-6376
Hippolyte SS, Simmonds NJ, Bilton D, et al., 2017, DIABETES AND PSEUDOMONAS, A TERRIBLE COMBINATION? EXAMINING THE UK CYSTIC FIBROSIS REGISTRY FOR A SEX DIFFERENCE IN OUTCOMES (2008-2013), Winter Meeting of the British-Thoracic-Society, Publisher: BMJ PUBLISHING GROUP, Pages: A2-A3, ISSN: 0040-6376
Archangelidi O, Simmonds NJ, Carr SB, et al., 2017, A national study of non-Invasive ventilation and clinical outcomes in Cystic Fibrosis, British Thoracic Society Annual Conference, Publisher: BMJ Publishing Group, Pages: A219-A220, ISSN: 1468-3296
Simmonds NJ, Bush A, 2017, The Man in the Paper Mask: One (Mask) for All and All for…..Cystic Fibrosis?, American Journal of Respiratory and Critical Care Medicine, Vol: 197, Pages: 281-283, ISSN: 1073-449X
Kayria M, Chris O, Dhasmana DJ, et al., 2017, Burkholderia cepacia complex and limited cutaneous vasculitis in patients with cystic fibrosis: a case series., JRSM Open, Vol: 8, Pages: 1-4, ISSN: 2054-2704
There is a high association of reactive skin presentations, mainly limited cutaneous vasculitis in patients with cystic fibrosis and Burkholderia cepcia complex chronic infection. This may be due to raised levels of circulating inflammatory mediators.
Cathcart F, Parrott H, Jones A, et al., 2016, PHYSIOTHERAPY MANAGEMENT OF ADULT PATIENTS WITH CYSTIC FIBROSIS ON INTENSIVE CARE UNITS (ICU) - A SURVEY OF UK PHYSIOTHERAPISTS, THORAX, Vol: 71, Pages: A135-A135, ISSN: 0040-6376
Turnbull AR, Lund-Palau H, Simbo A, et al., 2016, CHALLENGES CORRECTLY CLASSIFYING <i>PSEUDOMONAS AERUGINOSA</i> INFECTION STATUS IN PATIENTS ON INHALED ANTIBIOTICS: ARE NEGATIVE CULTURES CLINICALLY MEANINGFUL?, PEDIATRIC PULMONOLOGY, Vol: 51, Pages: 323-323, ISSN: 8755-6863
Waller MD, Simmonds NJ, 2016, Phenotypic variability of <i>R117H</i>-<i>CFTR</i> expression within monozygotic twins, PAEDIATRIC RESPIRATORY REVIEWS, Vol: 20, Pages: 21-23, ISSN: 1526-0542
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- Citations: 6
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