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Editorial Board
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iii
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| Editorial |
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EuroCareCF: Working together to improve patient care and therapy development
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David N. Sheppard
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S1-S4
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| Commentary |
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EuroCareCF: A mighty effort and a huge success
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J. Stuart Elborn
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S5-S6
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| Workpackage 1. Optimising Patient Care & CF Team Work |
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The implementation of standards of care in Europe: State of the art
Abstract: The care and condition of people with cystic fibrosis (CF) in 34 European countries is reported using data obtained from publications, registries and professionals providing CF patient care....
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Carla Colombo,
James Littlewood
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S7-S15
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European cystic fibrosis bone mineralisation guidelines
Abstract: Patients with cystic fibrosis (CF) are at risk of developing low bone mineral density (BMD) and fragility fractures. This paper presents consensus statements that summarise current knowledge...
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Isabelle Sermet-Gaudelus,
Maria Luisa Bianchi,
Michèle Garabédian,
Robert M. Aris,
Alison Morton,
Dana S. Hardin,
Sarah L. Elkin,
Juliet E. Compston,
Steven P. Conway,
Mireille Castanet,
Susan Wolfe,
Charles S. Haworth
et al.
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S16-S23
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Guidelines for the diagnosis and management of distal intestinal obstruction syndrome in cystic fibrosis patients
Abstract: Complete or incomplete intestinal obstruction by viscid faecal material in the terminal ileum and proximal colon – distal intestinal obstruction syndrome (DIOS) – is a common complication in...
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Carla Colombo,
Helmut Ellemunter,
Roderick Houwen,
Anne Munck,
Chris Taylor,
Michael Wilschanski,
on behalf of the ECFS
et al.
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S24-S28
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Best practice guidance for the diagnosis and management of cystic fibrosis-associated liver disease
Abstract: Approximately 5–10% of cystic fibrosis (CF) patients develop multilobular cirrhosis during the first decade of life. Most CF patients later develop signs of portal hypertension with complica...
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Dominique Debray,
Deirdre Kelly,
Roderick Houwen,
Birgitta Strandvik,
Carla Colombo
et al.
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S29-S36
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End of life care for patients with cystic fibrosis
Abstract: Palliative care is an approach that improves quality of life for patients and their families facing problems associated with a life-threatening illness. Care planning is particularly importa...
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Dorota Sands,
Teresa Repetto,
Lieven J. Dupont,
Aleksandra Korzeniewska-Eksterowicz,
Paola Catastini,
Susan Madge
et al.
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S37-S44
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Guiding principles on how to manage relevant psychological aspects within a CF team: Interdisciplinary approaches
Abstract: Managing CF can be emotionally and physically challenging for patients and their relatives. The disease and its treatment influence the ability to tackle normal tasks of daily living and une...
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Rita M. Nobili,
Alistair J.A. Duff,
Gerald Ullrich,
Ulrike Smrekar,
Trudy Havermans,
Mandy Bryon,
Ula Borawska-Kowalczyk,
Maria Sandberg Malmborg
et al.
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S45-S52
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| Workpackage 3. Coordination of Clinical Research |
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New clinical diagnostic procedures for cystic fibrosis in Europe
Abstract: In the majority of cases, there is no difficulty in diagnosing Cystic Fibrosis (CF). However, there may be wide variation in signs and symptoms between individuals which encourage the scient...
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K. De Boeck,
N. Derichs,
I. Fajac,
H.R. de Jonge,
I. Bronsveld,
I. Sermet,
F. Vermeulen,
D.N. Sheppard,
H. Cuppens,
M. Hug,
P. Melotti,
P.G. Middleton,
M. Wilschanski,
on behalf of the ECFS Diagnostic Network Working Group and the EuroCareCF WP3 Group on CF diagnosis
et al.
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S53-S66
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Guideline on the design and conduct of cystic fibrosis clinical trials: The European Cystic Fibrosis Society–Clinical Trials Network (ECFS-CTN)
Abstract: We describe the rationale for disease specific research networks in general as well as the aims and function of the European Cystic Fibrosis Society–Clinical Trials Network (ECFS-CTN) specif...
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K. De Boeck,
V. Bulteel,
H. Tiddens,
T. Wagner,
I. Fajac,
S. Conway,
F. Dufour,
A.R. Smyth,
T. Lee,
I. Sermet,
B. Kassai,
S. Elborn,
on behalf of all ECFS-CTN network partners
et al.
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S67-S74
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Chronic Pseudomonas aeruginosa infection definition: EuroCareCF Working Group report
Abstract: Chronic pulmonary infection with P. aeruginosa develops in most patients with cystic fibrosis (CF); by adulthood 80% of patients are infected and chronic P. aeruginosa infection is the prima...
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T. Pressler,
C. Bohmova,
S. Conway,
S. Dumcius,
L. Hjelte,
N. Høiby,
H. Kollberg,
B. Tümmler,
V. Vavrova
et al.
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S75-S78
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Pulmonary exacerbation: Towards a definition for use in clinical trials. Report from the EuroCareCF Working Group on outcome parameters in clinical trials
Abstract: Pulmonary exacerbations represent a key outcome variable in clinical trials of cystic fibrosis (CF). As there is variation in the trigger for use of intravenous antibiotics compared to the u...
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D. Bilton,
G. Canny,
S. Conway,
S. Dumcius,
L. Hjelte,
M. Proesmans,
B. Tümmler,
V. Vavrova,
K. De Boeck
et al.
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S79-S81
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Measuring health-related quality of life in clinical trials in cystic fibrosis
Abstract: The inclusion of health-related quality of life (HRQoL) as an outcome measure in cystic fibrosis (CF) clinical trials can supply important patient-reported information not captured by other ...
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J. Abbott,
A. Hart,
T. Havermans,
A. Matossian,
L. Goldbeck,
C. Barreto,
A. Bergsten-Brucefors,
T. Besier,
P. Catastini,
F. Lupi,
D. Staab
et al.
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S82-S85
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Recommendations for the classification of diseases as CFTR-related disorders
Abstract: Several diseases have been clinically or genetically related to cystic fibrosis (CF), but a consensus definition is lacking. Here, we present a proposal for consensus guidelines on cystic fi...
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C. Bombieri,
M. Claustres,
K. De Boeck,
N. Derichs,
J. Dodge,
E. Girodon,
I. Sermet,
M. Schwarz,
M. Tzetis,
M. Wilschanski,
C. Bareil,
D. Bilton,
C. Castellani,
H. Cuppens,
G.R. Cutting,
P. Drevínek,
P. Farrell,
J.S. Elborn,
K. Jarvi,
B. Kerem,
E. Kerem,
M. Knowles,
M. Macek,
A. Munck,
D. Radojkovic,
M. Seia,
D.N. Sheppard,
K.W. Southern,
M. Stuhrmann,
E. Tullis,
J. Zielenski,
P.F. Pignatti,
C. Ferec
et al.
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S86-S102
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| Workpackage 4. Small and Medium-Sized Enterprises (SMEs) Group |
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Developing new products in cystic fibrosis: Needs and obstacles for activities of small and middle-sized companies
Abstract: Small and middle-sized enterprises (SMEs) can make important contributions to medical progress through the development of new safe and effective drugs that address the greatest unmet needs o...
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Miriam Schlangen,
Andreas L.G. Reimann
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S103-S109
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Medical needs of cystic fibrosis patients and policies for fair co-operation between small and middle-sized companies and patient organizations
Abstract: Background and methods: Workpackage 4 of EuroCareCF brought together a group of small and middle-size companies (SMEs) with strong interest in drug development for cystic fibrosis (CF). The ...
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Miriam Schlangen,
Andreas L.G. Reimann
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S110-S113
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| Workpackage 5. Novel Therapies |
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Gene and cell therapy for cystic fibrosis: From bench to bedside
Abstract: Clinical trials in cystic fibrosis (CF) patients established proof-of-principle for transfer of the wild-type cystic fibrosis transmembrane conductance regulator (CFTR) gene to airway epithe...
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Massimo Conese,
Fiorentina Ascenzioni,
A. Christopher Boyd,
Charles Coutelle,
Ida De Fino,
Stefaan De Smedt,
Joanna Rejman,
Joseph Rosenecker,
Dirk Schindelhauer,
Bob J. Scholte
et al.
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S114-S128
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Pharmacological therapy for cystic fibrosis: From bench to bedside
Abstract: With knowledge of the molecular behaviour of the cystic fibrosis transmembrane conductance regulator (CFTR), its physiological role and dysfunction in cystic fibrosis (CF), therapeutic strat...
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Frédéric Becq,
Marcus A. Mall,
David N. Sheppard,
Massimo Conese,
Olga Zegarra-Moran
et al.
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S129-S145
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Antibiotic treatment of CF lung disease: From bench to bedside
Abstract: Chronic infection of the respiratory tract is a hallmark of cystic fibrosis (CF). Antibiotic treatment has been used as one of the mainstays of therapy and together with other treatment moda...
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Robert Bals,
Dominique Hubert,
Burkhard Tümmler
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S146-S151
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| Workpackage 6. Animal Models |
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Mouse models of cystic fibrosis: Phenotypic analysis and research applications
Abstract: Genetically modified mice have been studied for more than fifteen years as models of cystic fibrosis (CF). The large amount of experimental data generated illuminates the complex multi-organ...
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Martina Wilke,
Ruvalic M. Buijs-Offerman,
Jamil Aarbiou,
William H. Colledge,
David N. Sheppard,
Lhousseine Touqui,
Alice Bot,
Huub Jorna,
Hugo R. De Jonge,
Bob J. Scholte
et al.
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S152-S171
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The ENaC-overexpressing mouse as a model of cystic fibrosis lung disease
Abstract: Chronic lung disease remains the major cause of morbidity and mortality of cystic fibrosis (CF) patients. Cftr mutant mice developed severe intestinal obstruction, but did not exhibit the ch...
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Zhe Zhou,
Julia Duerr,
Bjarki Johannesson,
Susanne C. Schubert,
Diana Treis,
Maria Harm,
Simon Y. Graeber,
Alexander Dalpke,
Carsten Schultz,
Marcus A. Mall
et al.
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S172-S182
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| Workpackage 8. Ethical/Legal/Social Issues |
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Diversified harmony: Supranational and domestic regulation of pediatric clinical trials in the European Union
Abstract: Over the past decades, considerable legislative effort has been made to facilitate and encourage clinical research in the European Union (EU). Hereby, specific attention has been paid to the...
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Wim Pinxten,
Kris Dierickx,
Herman Nys
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S183-S198
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