September 2019

Editorial Board
Published in issue: September 2019
ii

Editorials

Important steps in the journey to highly effective CFTR modulator access for people with CF
• D.R. VanDevanter,
• N. Mayer-Hamblett
Published in issue: September 2019
p577-578
Taskforce recommends coordinated effort to improve clinical research conduct and find highly effective CFTR-directed treatment for rare mutations
• G.M. Solomon,
• D.P. Nichols
Published online: July 04, 2019
p579-580
Inhaled therapies for NTM disease – The way forward?
• Rachel Thomson
Published in issue: September 2019
p581-583

News

News
Published online: August 10, 2019
p584-586

Perspective

Transcript from Stuart Elborn's address at the ECFC award in June 2019
Published in issue: September 2019
p587-589

Review

Physical activity and associations with clinical outcome measures in adults with cystic fibrosis; a systematic review
• James Shelley,
• Lynne M. Boddy,
• Zoe R. Knowles,
• Claire E. Stewart,
• Ellen A. Dawson
Published online: March 26, 2019
p590-601

CFTR/Basic Science

Lumacaftor-rescued F508del-CFTR has a modified bicarbonate permeability
• Loretta Ferrera,
• Debora Baroni,
• Oscar Moran
Published online: February 07, 2019
p602-605

Decreased mRNA and protein stability of W1282X limits response to modulator therapy
• M.A. Aksit,
• A.D. Bowling,
• T.A. Evans,
• ...
• P.R. Sosnay,
• G.R. Cutting,
• N. Sharma
Published online: February 22, 2019
p606-613

Diagnosis/Screening

International approaches for delivery of positive newborn bloodspot screening results for CF
• J. Chudleigh,
• C.L. Ren,
• J. Barben,
• K.W. Southern
Published online: April 29, 2019
p614-621

Immunology

AR-13 reduces antibiotic-resistant bacterial burden in cystic fibrosis phagocytes and improves cystic fibrosis transmembrane conductance regulator function
• Kaivon Assani,
• Chandra L. Shrestha,
• Hannah Rinehardt,
• ...
• Susan D. Reynolds,
• Larry S. Schlesinger,
• Benjamin T. Kopp
Published online: October 23, 2018
p622-629

Pseudomonas aeruginosa colonization causes PD-L1 overexpression on monocytes, impairing the adaptive immune response in patients with cystic fibrosis
• José Avendaño-Ortiz,
• Emilio Llanos-González,
• Víctor Toledano,
• ...
• Luis A. Aguirre,
• Francisco García-Río,
• Eduardo López-Collazo
Published online: November 12, 2018
p630-635

Systemic levels of anti-PAD4 autoantibodies correlate with airway obstruction in cystic fibrosis
• Ruchi Yadav,
• Dae-goon Yoo,
• J. Michelle Kahlenberg,
• ...
• Hanwen Huang,
• Arlene Stecenko,
• Balázs Rada
Published online: January 10, 2019
p636-645

Microbiology

Differences in the lower airway microbiota of infants with and without cystic fibrosis
• Katherine B. Frayman,
• Kristine M. Wylie,
• David S. Armstrong,
• ...
• Keith Grimwood,
• Gregory A. Storch,
• Sarath C. Ranganathan
Published online: December 20, 2018
p646-652

Metagenome – Inferred bacterial replication rates in cystic fibrosis airways
• Katarzyna Pienkowska,
• Lutz Wiehlmann,
• Burkhard Tümmler
Published online: January 23, 2019
p653-656

Hyperbaric oxygen treatment increases killing of aggregating Pseudomonas aeruginosa isolates from cystic fibrosis patients
• Signe Agnete Møller,
• Peter Østrup Jensen,
• Niels Høiby,
• ...
• Kasper Nørskov Kragh,
• Thomas Bjarnsholt,
• Mette Kolpen
Published online: January 30, 2019
p657-664

Epidemiology/Models of Care

Non-invasive ventilation and clinical outcomes in cystic fibrosis: Findings from the UK CF registry
• Olga Archangelidi,
• Siobhán B. Carr,
• Nicholas J. Simmonds,
• ...
• Winston Banya,
• Paul Cullinan
• on behalf of CF-EpiNet
Published online: November 28, 2018
p665-670

Interprofessional provider educational needs and preferences regarding the provision of sexual and reproductive health care in cystic fibrosis
• Traci M. Kazmerski,
• Eliza B. Nelson,
• Lori R. Newman,
• ...
• Elizabeth Miller,
• S. Jean Emans,
• Gregory S. Sawicki
Published online: February 19, 2019
p671-676

Clinical Trials

Speeding up access to new drugs for CF: Considerations for clinical trial design and delivery
• Jane C. Davies,
• Pavel Drevinek,
• J. Stuart Elborn,
• ...
• J. Stuart Elborn,
• Eitan Kerem,
• Tim Lee
Published online: July 11, 2019
p677-684

Theranostics by testing CFTR modulators in patient-derived materials: The current status and a proposal for subjects with rare CFTR mutations
• Margarida D. Amaral,
• Kris de Boeck
• on behalf of the ECFS Strategic Planning Task Force on ‘Speeding up access to new drugs for CF’
Published online: July 17, 2019
p685-692

GLPG1837, a CFTR potentiator, in p.Gly551Asp (G551D)-CF patients: An open-label, single-arm, phase 2a study (SAPHIRA1)
• Jane C. Davies,
• Olivier Van de Steen,
• Silke van Koningsbruggen-Rietschel,
• ...
• Florence Namour,
• Herman de Kock,
• Katja Conrath
Published online: May 27, 2019
p693-699

CFTR activity is enhanced by the novel corrector GLPG2222, given with and without ivacaftor in two randomized trials
• Scott C. Bell,
• Peter J. Barry,
• Kris De Boeck,
• ...
• Katja Conrath,
• Olivier Van de Steen,
• Kors van der Ent
Published online: May 03, 2019
p700-707

A phase 3 study of tezacaftor in combination with ivacaftor in children aged 6 through 11 years with cystic fibrosis
• Seth Walker,
• Patrick Flume,
• John McNamara,
• ...
• Caroline A. Owen,
• Gregory Sawicki
• on behalf of the VX15-661-113 Investigator Group
Published online: June 27, 2019
p708-713

Pharmachology

Clofazimine inhalation suspension for the aerosol treatment of pulmonary nontuberculous mycobacterial infections
• Brandon Banaschewski,
• Deepshikha Verma,
• Lian J. Pennings,
• ...
• Stefan Ufer,
• Kevin Stapleton,
• Thomas Hofmann
Published online: May 25, 2019
p714-720

Pulmonology

Abnormal preschool Lung Clearance Index (LCI) reflects clinical status and predicts lower spirometry later in childhood in cystic fibrosis
• K.M. Hardaker,
• H. Panda,
• K. Hulme,
• ...
• S. Towns,
• H. Selvadurai,
• P.D. Robinson
Published online: March 01, 2019
p721-727

Imaging

A two-center analysis of hyperpolarized ¹²⁹Xe lung MRI in stable pediatric cystic fibrosis: Potential as a biomarker for multi-site trials
• Marcus J. Couch,
• Robert Thomen,
• Nikhil Kanhere,
• ...
• Felix Ratjen,
• Jason Woods,
• Giles Santyr
Published online: March 25, 2019
p728-733

Endocrinology

The impact of cystic fibrosis-related diabetes on health-related quality of life
• Eugenie Kwong,
• Sameer Desai,
• Louise Chong,
• ...
• Jie Zheng,
• Pearce G. Wilcox,
• Bradley S. Quon
Published online: March 29, 2019
p734-736

Psychosocial

Measuring recovery in health-related quality of life during and after pulmonary exacerbations in patients with cystic fibrosis
• Patrick A. Flume,
• Ellison D. Suthoff,
• Mark Kosinski,
• Gautham Marigowda,
• Alexandra L. Quittner
Published online: December 23, 2018
p737-742

Online Only Publications

Who and why; sharing our experiences of developing a standard operating procedure (SOP) to allocate screening slots for highly competitive cystic fibrosis trials
• Rebecca Dobra,
• Sandra Scott,
• Jane C. Davies,
• Nicholas J. Simmonds
Published online: May 03, 2019
e45-e46

Fair subject selection in cystic fibrosis trials
• Camila Strassle
Published online: June 10, 2019
e47

Fair selection of participants in clinical trials: The challenge to push the envelope further
• J.C. Davies,
• S. Scott,
• R. Dobra,
• ...
• G. Ruiz,
• M. Waller,
• L. Baker
Published online: August 09, 2019
e48-e50

Mild cystic fibrosis in carriers of two nonsense mutations – a case of genetic compensation response?
• Burkhard Tümmler
Published online: June 29, 2019
e51-e52


Total pancreatectomy with islet autotransplantation in a pancreatic-sufficient cystic fibrosis patient
• Ina St Onge,
• Jaimie D. Nathan,
• Maisam Abu-El-Haija,
• Barbara A. Chini
Published online: August 13, 2019
e53-e55