Machine Learning Prediction of Pulmonary Function in Children With Cystic Fibrosis

NCT07770854 | Completed

• 1 other identifier: Gazi2907
• Study Type: observational
• Target: 31
• Locations: 1 country
• Sites: 1

Brief Summary

Cystic fibrosis is a multisystem genetic disorder characterized by progressive pulmonary impairment and extrapulmonary limitations. This retrospective observational study will perform a secondary analysis of previously collected data from children with cystic fibrosis. The primary objective is to determine the relative contribution of peripheral muscle oxygenation and postural balance parameters to the prediction of pulmonary function, including forced expiratory volume in 1 second, forced vital capacity, and peak expiratory flow, using machine-learning regression methods. The additional contribution of exercise-related oxygen saturation responses, symptoms, nutritional and anthropometric characteristics, physical activity, respiratory muscle function, quality of life, treatment burden, and disease-related clinical variables will also be examined. No new assessment, intervention, or participant contact will occur.

Conditions

Cystic Fibrosis (CF)

Keywords

pulmonary function; machine learning; FEV1; FVC; peak expiratory flow; peripheral muscle oxygenation; postural balance; six-minute walk test; physical activity; children

Outcome Measures

Primary Outcomes (6)

• Forced Expiratory Volume in 1 Second (FEV1), Absolute Value

  FEV1 was measured by spirometry according to ATS/ERS standards and recorded in liters. The highest value from technically acceptable forced expiratory maneuvers was retained. FEV1 will be used as a prespecified pulmonary function prediction target

  At the single archived cross-sectional assessment (data originally collected between April 2021 and September 2022)

• Forced Expiratory Volume in 1 Second (FEV1), Percent Predicted

  FEV1 expressed as percent of the age-, sex-, and body-size-appropriate predicted value will be used as a prespecified pulmonary function prediction target. Higher values indicate better pulmonary function.

  At the single archived cross-sectional assessment (data originally collected between April 2021 and September 2022)

• Forced Vital Capacity (FVC), Absolute Value

  FVC was measured by spirometry according to ATS/ERS standards and recorded in liters. The highest value from technically acceptable forced expiratory maneuvers was retained. FVC will be used as a prespecified pulmonary function prediction target.

  At the single archived cross-sectional assessment (data originally collected between April 2021 and September 2022)

• Forced Vital Capacity (FVC), Percent Predicted

  FVC expressed as percent of the age-, sex-, and body-size-appropriate predicted value will be used as a prespecified pulmonary function prediction target. Higher values indicate better pulmonary function.

  At the single archived cross-sectional assessment (data originally collected between April 2021 and September 2022)

• Peak Expiratory Flow (PEF), Absolute Value

  PEF was measured by spirometry according to ATS/ERS standards and recorded in \[ L/min\]. The highest value from technically acceptable forced expiratory maneuvers was retained. PEF will be used as a prespecified pulmonary function prediction target.

  At the single archived cross-sectional assessment (data originally collected between April 2021 and September 2022)

• Peak Expiratory Flow (PEF), Percent Predicted

  PEF expressed as percent of the age-, sex-, and body-size-appropriate predicted value will be used as a prespecified pulmonary function prediction target. Higher values indicate better pulmonary function.

  At the single archived cross-sectional assessment (data originally collected between April 2021 and September 2022)

Secondary Outcomes (3)

• Cross-Validated Predictive Performance of the FEV1 Models

  During analysis of the archived dataset, through study completion (up to 12 months)

• Cross-Validated Predictive Performance of the FVC Models

  During analysis of the archived dataset, through study completion (up to 12 months)

• Cross-Validated Predictive Performance of the PEF Models

  During analysis of the archived dataset, through study completion (up to 12 months)

Other Outcomes (6)

• Mean Cross-validated Normalized Importance Score of Peripheral Muscle Oxygen Saturation

  During analysis of the archived dataset, through study completion (up to 12 months)

• Selection Stability of Peripheral Muscle Oxygen Saturation Across Repeated Cross-validation

  During analysis of the archived dataset, through study completion (up to 12 months)

• Mean Cross-validated Normalized Importance Score of the Postural Balance Predictor Domain

  During analysis of the archived dataset, through study completion (up to 12 months)

Study Arms (1)

Children With Cystic Fibrosis

Archived records of children aged 6 to 18 years with cystic fibrosis who underwent multidimensional clinical and functional assessments at the Gazi University Cardiopulmonary Rehabilitation Unit between April 2021 and September 2022. The present study involves secondary data analysis only; no new intervention or assessment will be performed.

Other: Archived Multidimensional Clinical and Functional Assessment Data

Interventions

Archived Multidimensional Clinical and Functional Assessment Data OTHER

Secondary analysis of archived spirometry, near-infrared spectroscopy, postural balance, exercise capacity, respiratory muscle function, physical activity, quality-of-life, anthropometric, and clinical data. No new assessment or intervention is administered.

Children With Cystic Fibrosis

Eligibility Criteria

• Age: 6 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)
• Sampling Method: Non-Probability Sample

Study Population

The study population will consist of archived records from children aged 6 to 18 years with cystic fibrosis who were referred by the Division of Pediatric Pulmonology, Gazi University Faculty of Medicine, and evaluated at the Cardiopulmonary Rehabilitation Unit, Department of Physiotherapy and Rehabilitation, Gazi University Faculty of Health Sciences, between April 2021 and September 2022. Only records meeting the prespecified eligibility criteria and containing the variables required for the principal analyses will be included. No new recruitment or participant contact will occur.

You may qualify if:

• Age 6 to 18 years at the time of the source assessment
• Diagnosis of cystic fibrosis according to the criteria specified in the Cystic Fibrosis Foundation consensus report
• Clinically stable at the time of the source assessment
• Receiving standard medical treatment
• Written informed consent/assent from the participant and parent or legal guardian for participation in the source study
• Availability of archived data for the principal variables required for the present analysis

You may not qualify if:

• Diagnosed visual, hearing, vestibular, or neurological disorder that could affect balance
• Hospitalization within the month preceding the source assessment
• Participation in a structured exercise training program within the 3 months preceding the source assessment
• History of COVID-19 or tobacco use
• Orthopedic disorder affecting mobility or history of musculoskeletal surgery
• Acute pulmonary exacerbation at the time of assessment
• Allergic bronchopulmonary aspergillosis
• History of lung or liver transplantation
• Systemic corticosteroid use
• Pulmonary hypertension or cardiovascular instability
• Missing archived records required for the principal analyses

Sponsors & Collaborators

• Gazi University: lead

Study Sites (1)

Gazi University Faculty of Health Sciences Department of Cardiopulmonary Physiotherapy and Rehabilitation, Ankara, Çankaya 06490

Ankara, Ankara, 06560, Turkey (Türkiye)

Location

Related Publications (16)

• Dwyer TJ, Alison JA, McKeough ZJ, Elkins MR, Bye PT. Evaluation of the SenseWear activity monitor during exercise in cystic fibrosis and in health. Respir Med. 2009 Oct;103(10):1511-7. doi: 10.1016/j.rmed.2009.04.013. Epub 2009 May 23.

  PMID: 19464863 RESULT

• Feldmann A, Schmitz R, Erlacher D. Near-infrared spectroscopy-derived muscle oxygen saturation on a 0% to 100% scale: reliability and validity of the Moxy Monitor. J Biomed Opt. 2019 Nov;24(11):1-11. doi: 10.1117/1.JBO.24.11.115001.

  PMID: 31741352 RESULT

• Laveneziana P, Albuquerque A, Aliverti A, Babb T, Barreiro E, Dres M, Dube BP, Fauroux B, Gea J, Guenette JA, Hudson AL, Kabitz HJ, Laghi F, Langer D, Luo YM, Neder JA, O'Donnell D, Polkey MI, Rabinovich RA, Rossi A, Series F, Similowski T, Spengler CM, Vogiatzis I, Verges S. ERS statement on respiratory muscle testing at rest and during exercise. Eur Respir J. 2019 Jun 13;53(6):1801214. doi: 10.1183/13993003.01214-2018. Print 2019 Jun.

  PMID: 30956204 RESULT

• American Thoracic Society/European Respiratory Society. ATS/ERS Statement on respiratory muscle testing. Am J Respir Crit Care Med. 2002 Aug 15;166(4):518-624. doi: 10.1164/rccm.166.4.518. No abstract available.

  PMID: 12186831 RESULT

• Graham BL, Steenbruggen I, Miller MR, Barjaktarevic IZ, Cooper BG, Hall GL, Hallstrand TS, Kaminsky DA, McCarthy K, McCormack MC, Oropez CE, Rosenfeld M, Stanojevic S, Swanney MP, Thompson BR. Standardization of Spirometry 2019 Update. An Official American Thoracic Society and European Respiratory Society Technical Statement. Am J Respir Crit Care Med. 2019 Oct 15;200(8):e70-e88. doi: 10.1164/rccm.201908-1590ST.

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• Collins GS, Moons KGM, Dhiman P, Riley RD, Beam AL, Van Calster B, Ghassemi M, Liu X, Reitsma JB, van Smeden M, Boulesteix AL, Camaradou JC, Celi LA, Denaxas S, Denniston AK, Glocker B, Golub RM, Harvey H, Heinze G, Hoffman MM, Kengne AP, Lam E, Lee N, Loder EW, Maier-Hein L, Mateen BA, McCradden MD, Oakden-Rayner L, Ordish J, Parnell R, Rose S, Singh K, Wynants L, Logullo P. TRIPOD+AI statement: updated guidance for reporting clinical prediction models that use regression or machine learning methods. BMJ. 2024 Apr 16;385:e078378. doi: 10.1136/bmj-2023-078378.

  PMID: 38626948 RESULT

• Zhou GC, Wang Z, Palipana AK, Andrinopoulou ER, Miranda Afonso P, McPhail GL, Siracusa CM, Gecili E, Szczesniak RD. Predicting lung function decline in cystic fibrosis: the impact of initiating ivacaftor therapy. Respir Res. 2024 Apr 27;25(1):187. doi: 10.1186/s12931-024-02794-2.

  PMID: 38678203 RESULT

• Alaa AM, van der Schaar M. Prognostication and Risk Factors for Cystic Fibrosis via Automated Machine Learning. Sci Rep. 2018 Jul 26;8(1):11242. doi: 10.1038/s41598-018-29523-2.

  PMID: 30050169 RESULT

• Dawson N, Dzurino D, Karleskint M, Tucker J. Examining the reliability, correlation, and validity of commonly used assessment tools to measure balance. Health Sci Rep. 2018 Oct 27;1(12):e98. doi: 10.1002/hsr2.98. eCollection 2018 Dec.

  PMID: 30623052 RESULT

• Kenis-Coskun O, Karadag-Saygi E, Bahar-Ozdemir Y, Gokdemir Y, Karadag B, Kayhan O. The involvement of musculoskeletal system and its influence on postural stability in children and young adults with cystic fibrosis. Ital J Pediatr. 2017 Nov 21;43(1):106. doi: 10.1186/s13052-017-0426-0.

  PMID: 29162121 RESULT

• Yoleri B, Bosnak Guclu M, Sismanlar Eyuboglu T, Aslan AT. Peripheral muscle oxygenation during upper extremity functional exercise and balance in pediatric patients with cystic fibrosis: a cross-sectional study. Eur J Appl Physiol. 2026 Jun;126(6):3589-3602. doi: 10.1007/s00421-026-06130-y. Epub 2026 Mar 7.

  PMID: 41793435 RESULT

• Nixon PA, Orenstein DM, Kelsey SF, Doershuk CF. The prognostic value of exercise testing in patients with cystic fibrosis. N Engl J Med. 1992 Dec 17;327(25):1785-8. doi: 10.1056/NEJM199212173272504.

  PMID: 1435933 RESULT

• Sovtic A, Minic P, Markovic-Sovtic G, Trajkovic GZ. Respiratory Muscle Strength and Exercise Performance in Cystic Fibrosis-A Cross Sectional Study. Front Pediatr. 2018 Sep 4;6:244. doi: 10.3389/fped.2018.00244. eCollection 2018.

  PMID: 30234080 RESULT

• Vendrusculo FM, Heinzmann-Filho JP, Piva TC, Marostica PJ, Donadio MV. Inspiratory Muscle Strength and Endurance in Children and Adolescents with Cystic Fibrosis. Respir Care. 2016 Feb;61(2):184-91. doi: 10.4187/respcare.04231. Epub 2015 Nov 24.

  PMID: 26604327 RESULT

• Werkman M, Jeneson J, Helders P, Arets B, van der Ent K, Velthuis B, Nievelstein R, Takken T, Hulzebos E. Exercise oxidative skeletal muscle metabolism in adolescents with cystic fibrosis. Exp Physiol. 2016 Mar;101(3):421-31. doi: 10.1113/EP085425.

  PMID: 26707538 RESULT

• Castellani C, Duff AJA, Bell SC, Heijerman HGM, Munck A, Ratjen F, Sermet-Gaudelus I, Southern KW, Barben J, Flume PA, Hodkova P, Kashirskaya N, Kirszenbaum MN, Madge S, Oxley H, Plant B, Schwarzenberg SJ, Smyth AR, Taccetti G, Wagner TOF, Wolfe SP, Drevinek P. ECFS best practice guidelines: the 2018 revision. J Cyst Fibros. 2018 Mar;17(2):153-178. doi: 10.1016/j.jcf.2018.02.006. Epub 2018 Mar 3.

  PMID: 29506920 RESULT

MeSH Terms

Conditions

Cystic Fibrosis; Motor Activity

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases; Behavior

Study Officials

• Elif KELES GULNERMAN, MD, PhD

  Department of Radiology, Feinberg School of Medicine, Northwestern University

  PRINCIPAL INVESTIGATOR

• Meral BOŞNAK GÜÇLÜ, Prof.

  Gazi University

  STUDY DIRECTOR

• Betül Yoleri, MSc

  Gazi University

  STUDY CHAIR

• Ulas BAGCI, Prof.

  Department of Radiology, Feinberg School of Medicine, Northwestern University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof.

Study Record Dates

• First Submitted: August 10, 2026
• First Posted: August 18, 2026
• Study Start: January 1, 2021
• Primary Completion: February 1, 2025
• Study Completion: March 1, 2025
• Last Updated: August 18, 2026

Record last verified: 2026-08

Data Sharing

• IPD Sharing: Will not share

Locations

TU (1)