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Introduction: Consensus is lacking on the optimal management of asymptomatic congenital pulmonary airway malformation (CPAM). For future studies, the CONNECT consortium (the COllaborative Neonatal Network for the first European CPAM Trial)-an international collaboration of specialised caregivers-has established consensus on a core outcome set of outcome parameters concerning respiratory insufficiency, surgical complications, mass effect and multifocal disease. These outcome parameters have been incorporated in the CONNECT trial, a randomised controlled trial which, in order to develop evidence-based practice, aims to compare conservative and surgical management of patients with an asymptomatic CPAM., Methods and Analysis: Children are eligible for inclusion after the CPAM diagnosis has been confirmed on postnatal chest CT scan and they remain asymptomatic. On inclusion, children are randomised to receive either conservative or surgical management. Subsequently, children in both groups are enrolled into a standardised, 5-year follow-up programme with three visits, including a repeat chest CT scan at 2.5 years and a standardised exercise tolerance test at 5 years.The primary outcome is exercise tolerance at age 5 years, measured according to the Bruce treadmill protocol. Secondary outcome measures are molecular genetic diagnostics, validated questionnaires-on parental anxiety, quality of life and healthcare consumption-, repeated imaging and pulmonary morbidity during follow-up, as well as surgical complications and histopathology. This trial aims to end the continuous debate surrounding the optimal management of asymptomatic CPAM., Ethics and Dissemination: This study is being conducted in accordance with the Declaration of Helsinki. The Medical Ethics Review Board of Erasmus University Medical Centre Rotterdam, The Netherlands, has approved this protocol (MEC-2022-0441). Results will be disseminated through peer-reviewed scientific journals and conference presentations., Trial Registration Number: NCT05701514., Competing Interests: Competing interests: None declared., (© Author(s) (or their employer(s)) 2023. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.)

Cystic fibrosis (CF) centres can assess the quality of the care they deliver by benchmarking their practices and outcomes against those of other CF centres. This is most easily done using summaries of electronic patient records, such as are generated by patient registries. All centres should assess their compliance with standards of care, as determined by consensus documents and evidence-based medicine, and continually seek out and implement ways to improve their clinical outcomes. This may imply changes to routine centre practice as well as to treatment strategies.

Early detection of progressive lung disease in cystic fibrosis (CF) may lead to better treatment and prognosis. Routine lung function indices may be relatively insensitive markers of peripheral airway obstruction and alveolar collapse. We hypothesized that the single-breath diffusion capacity of the lung for carbon monoxide (DLCO) would change before tests of airway function in patients with CF. We assessed lung function longitudinally in 53 children with CF during a mean period of 3.8 years to determine whether the diffusion capacity of the lung becomes abnormal before more conventional indices of lung function do. Within patients, DLCO was slightly elevated and remained stable, while forced expired volume in 1 sec (FEV1) and forced vital capacity (FVC) declined progressively (mean individual decline, -1.8% and -0.8% of predicted). Cross-sectionally, this decline was faster (mean group decline -3.8% and -2.8% of predicted), indicating an additional cohort effect. Normalized diffusion capacity at an early stage of CF is slightly elevated and is preserved in spite of progressive obstructive lung disease. This can be attributed to alterations in pulmonary and bronchial circulation due to loss of function and/or number of alveolar units. Diffusion capacity at rest does not appear to be a suitable early marker of progressive deterioration of CF lung disease., (Copyright 2004 Wiley-Liss, Inc.)