Publications
Alam F, Munidasa S, Zanette B, Braganza S, Li D, Jensen R, Dumas MP, Ratjen F, Santyr G. Assessing 129Xe multi-breath washout MRI response to elexacaftor/tezacaftor/ivacaftor intervention in pediatric CF. J Cyst Fibros. 2024 Sep 30:S1569-1993(24)01789-2. doi: 10.1016/j.jcf.2024.09.021. Epub ahead of print. PMID: 39353741.
Walkup LL, Roach DJ, Plummer JW, Willmering MM, Zanette B, Santyr G, Fain SB, Rock MJ, Mata J, Froh D, Stanojevic S, Cleveland ZI, Ratjen F, Woods JC. Same-Day Repeatability and 28-Day Reproducibility of Xenon MRI Ventilation in Children With Cystic Fibrosis in a Multi-Site Trial. J Magn Reson Imaging. 2024 Sep 11. doi: 10.1002/jmri.29605. Epub ahead of print. PMID: 39257323.
2023:
Goralski JL, Hoppe JE, Mall MA, McColley SA, McKone E, Ramsey B, Rayment JH, Robinson P, Stehling F, Taylor-Cousar JL, Tullis E, Ahluwalia N, Chin A, Chu C, Lu M, Niu T, Weinstock T, Ratjen F, Rosenfeld M. Phase 3 Open-Label Clinical Trial of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged 2-5 Years with Cystic Fibrosis and at Least One F508del Am J Respir Crit Care Med. 2023 Jul 1;208(1):59-67. doi: 10.1164/rccm.202301-0084OC. PMID: 36921081; PMCID: PMC10870849.
Wainwright C, McColley SA, McNally P, Powers M, Ratjen F, Rayment JH, Retsch-Bogart G, Roesch E, Ahluwalia N, Chin A, Chu C, Lu M, Menon P, Waltz D, Weinstock T, Zelazoski L, Davies JC. Long-Term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Children Aged ⩾6 Years with Cystic Fibrosis and at Least One F508delAllele: A Phase 3, Open-Label Clinical Trial. Am J Respir Crit Care Med. 2023 Jul 1;208(1):68-78. doi: 10.1164/rccm.202301-0021OC. PMID: 37154609; PMCID: PMC10870850.
2022:
Mall MA, Brugha R, Gartner S, Legg J, Moeller A, Mondejar-Lopez P, Prais D, Pressler T, Ratjen F, Reix P, Robinson PD, Selvadurai H, Stehling F, Ahluwalia N, Arteaga-Solis E, Bruinsma BG, Jennings M, Moskowitz SM, Noel S, Tian S, Weinstock TG, Wu P, Wainwright CE, Davies JC. Efficacy and Safety of Elexacaftor/Tezacaftor/Ivacaftor in Children 6 Through 11 Years of Age with Cystic Fibrosis Heterozygous for F508deland a Minimal Function Mutation: A Phase 3b, Randomized, Placebo-controlled Study. Am J Respir Crit Care Med. 2022 Dec 1;206(11):1361-1369. doi: 10.1164/rccm.202202-0392OC. PMID: 35816621; PMCID: PMC9746869.
Mayer-Hamblett N, Ratjen F, Russell R, Donaldson SH, Riekert KA, Sawicki GS, Odem-Davis K, Young JK, Rosenbluth D, Taylor-Cousar JL, Goss CH, Retsch-Bogart G, Clancy JP, Genatossio A, O’Sullivan BP, Berlinski A, Millard SL, Omlor G, Wyatt CA, Moffett K, Nichols DP, Gifford AH; SIMPLIFY Study Group. Discontinuation versus continuation of hypertonic saline or dornase alfa in modulator treated people with cystic fibrosis (SIMPLIFY): results from two parallel, multicentre, open-label, randomised, controlled, non-inferiority trials. Lancet Respir Med. 2023 Apr;11(4):329-340. doi: 10.1016/S2213-2600(22)00434-9. Epub 2022 Nov 4. PMID: 36343646; PMCID: PMC10065895.
Tiddens HAWM, Chen Y, Andrinopoulou ER, Davis SD, Rosenfeld M, Ratjen F, Kronmal RA, Hinckley Stukovsky KD, Dasiewicz A, Stick SM; SHIP-CT Study Group. The effect of inhaled hypertonic saline on lung structure in children aged 3-6 years with cystic fibrosis (SHIP-CT): a multicentre, randomised, double-blind, controlled trial. Lancet Respir Med. 2022 Jul;10(7):669-678. doi: 10.1016/S2213-2600(21)00546-4. Epub 2022 Mar 11. PMID: 35286860.
Nichols DP, Paynter AC, Heltshe SL, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Rosenfeld M, Sagel SD, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Clancy JP, Kirby S, Van Dalfsen JM, Kloster MH, Rowe SM; PROMISE Study group. Clinical Effectiveness of Elexacaftor/Tezacaftor/Ivacaftor in People with Cystic Fibrosis: A Clinical Trial. Am J Respir Crit Care Med. 2022 Mar 1;205(5):529-539. doi: 10.1164/rccm.202108-1986OC. PMID: 34784492; PMCID: PMC8906485.
2021:
Chilvers MA, Davies JC, Milla C, Tian S, Han Z, Cornell AG, Owen CA, Ratjen F. Long-term safety and efficacy of lumacaftor-ivacaftor therapy in children aged 6-11 years with cystic fibrosis homozygous for the F508del-CFTR mutation: a phase 3, open-label, extension study. Lancet Respir Med. 2021 Jul;9(7):721-732. doi: 10.1016/S2213-2600(20)30517-8. Epub 2021 Jan 28. Erratum in: Lancet Respir Med. 2021 Apr;9(4):e38. doi: 10.1016/S2213-2600(21)00093-X. PMID: 33516285.
Sagel SD, Khan U, Heltshe SL, Clancy JP, Borowitz D, Gelfond D, Donaldson SH, Moran A, Ratjen F, VanDalfsen JM, Rowe SM. Clinical Effectiveness of Lumacaftor/Ivacaftor in Patients with Cystic Fibrosis Homozygous for F508del-CFTR. A Clinical Trial. Ann Am Thorac Soc. 2021 Jan;18(1):75-83. doi: 10.1513/AnnalsATS.202002-144OC. PMID: 32644818; PMCID: PMC7780982.
2020:
Bernasconi N, Kieninger E, Shaw M, Kurz J, Moeller A, Ratjen F, Rochat I, Stanojevic S, Singer F. CFTR-function and ventilation inhomogeneity in individuals with cystic fibrosis. J Cyst Fibros. 2021 Jul;20(4):641-647. doi: 10.1016/j.jcf.2020.12.009. Epub 2020 Dec 19. PMID: 33349584.
O’Neill K, Ferguson K, Cosgrove D, Tunney MM, De Soyza A, Carroll M, Chalmers JD, Gatheral T, Hill AT, Hurst JR, Johnson C, Loebinger MR, Angyalosi G, Haworth CS, Jensen R, Ratjen F, Saunders C, Short C, Davies JC, Elborn JS, Bradley JM. Multiple breath washout in bronchiectasis clinical trials: is it feasible? ERJ Open Res. 2020 Oct 13;6(4):00363-2019. doi: 10.1183/23120541.00363-2019. PMID: 33083441; PMCID: PMC7553113.
Shaw M, Khan U, Clancy JP, Donaldson SH, Sagel SD, Rowe SM, Ratjen F; PROSPECT Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network. Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: Results from the prospect study. J Cyst Fibros. 2020 Nov;19(6):931-933. doi: 10.1016/j.jcf.2020.05.010. Epub 2020 Jun 6. PMID: 32513528; PMCID: PMC9125683.
2019:
Saunders C, Jensen R, Robinson PD, Stanojevic S, Klingel M, Short C, Davies JC, Ratjen F. Integrating the multiple breath washout test into international multicentre trials. J Cyst Fibros. 2020 Jul;19(4):602-607. doi: 10.1016/j.jcf.2019.11.006. Epub 2019 Nov 24. PMID: 31771900.
Ratjen F, Davis SD, Stanojevic S, Kronmal RA, Hinckley Stukovsky KD, Jorgensen N, Rosenfeld M; SHIP Study Group. Inhaled hypertonic saline in preschool children with cystic fibrosis (SHIP): a multicentre, randomised, double-blind, placebo-controlled trial. Lancet Respir Med. 2019 Sep;7(9):802-809. doi: 10.1016/S2213-2600(19)30187-0. Epub 2019 Jun 6. PMID: 31178421.
Perrem L, Stanojevic S, Shaw M, Pornillos M, Guido J, Sanders DB, Solomon M, Grasemann H, Sweezey N, Waters V, Davis SD, Ratjen F. Comparative analysis of respiratory symptom scores to detect acute respiratory events in children with cystic fibrosis. J Cyst Fibros. 2023 Mar;22(2):296-305. doi: 10.1016/j.jcf.2022.06.007. Epub 2022 Jun 24. PMID: 35753986.
Perrem L, Stanojevic S, Solomon M, Grasemann H, Sweezey N, Waters V, Sanders DB, Davis SD, Ratjen F. Evaluation of clinically relevant changes in the lung clearance index in children with cystic fibrosis and healthy controls. Thorax. 2023 Apr;78(4):362-367. doi: 10.1136/thoraxjnl-2021-218347. Epub 2022 Apr 15. PMID: 35428702.
Jensen R, Shaw M, Phanthanourak A, Ratjen F. Re: Impact of spiroware re-analysis method on multiple-breath washout outcomes in children with cystic fibrosis; M.A. Oestreich, F. Wyler, P. Latzin et al. J Cyst Fibros. 2022 May;21(3):e210-e211. doi: 10.1016/j.jcf.2022.04.001. Epub 2022 Apr 9. PMID: 35414443.
2021:
Robinson PD, Jensen R, Seeto RA, Stanojevic S, Saunders C, Short C, Davies JC, Ratjen F. Impact of cross-sensitivity error correction on representative nitrogen-based multiple breath washout data from clinical trials. J Cyst Fibros. 2022 May;21(3):e204-e207. doi: 10.1016/j.jcf.2021.08.033. Epub 2021 Sep 12. PMID: 34526221.
Stanojevic S, Davis SD, Perrem L, Shaw M, Retsch-Bogart G, Davis M, Jensen R, Clem CC, Isaac SM, Guido J, Jara S, France L, McDonald N, Solomon M, Sweezey N, Grasemann H, Waters V, Sanders DB, Ratjen FA. Determinants of lung disease progression measured by lung clearance index in children with cystic fibrosis. Eur Respir J. 2021 Jul 8;58(1):2003380. doi: 10.1183/13993003.03380-2020. PMID: 33542049.
Perrem L, Stanojevic S, Shaw M, Jensen R, McDonald N, Isaac SM, Davis M, Clem C, Guido J, Jara S, France L, Solomon M, Grasemann H, Waters V, Sweezey N, Sanders DB, Davis SD, Ratjen F. Lung Clearance Index to Track Acute Respiratory Events in School-Age Children with Cystic Fibrosis. Am J Respir Crit Care Med. 2021 Apr 15;203(8):977-986. doi: 10.1164/rccm.202006-2433OC. PMID: 33030967.
2020:
Anagnostopoulou P, Latzin P, Jensen R, Stahl M, Harper A, Yammine S, Usemann J, Foong RE, Spycher B, Hall GL, Singer F, Stanojevic S, Mall MA, Ratjen F, Ramsey KA. Normative data for multiple breath washout outcomes in school-aged Caucasian children. Eur Respir J. 2020 Apr 3;55(4):1901302. doi: 10.1183/13993003.01302-2019. Erratum in: Eur Respir J. 2022 Aug 18;60(2):1951302. doi: 10.1183/13993003.51302-2019. PMID: 31862765.
Shaw M, Oppelaar MC, Jensen R, Stanojevic S, Davis SD, Retsch-Bogart G, Ratjen FA. The utility of moment ratios and abbreviated endpoints of the multiple breath washout test in preschool children with cystic fibrosis. Pediatr Pulmonol. 2020 Mar;55(3):649-653. doi: 10.1002/ppul.24618. Epub 2020 Jan 3. PMID: 31899855.
2019:
Ratjen F, Jensen R, Klingel M, McDonald R, Moore C, Benseler N, Wilson D, Stanojevic S. Effect of changes in tidal volume on multiple breath washout outcomes. PLoS One. 2019 Jul 3;14(7):e0219309. doi: 10.1371/journal.pone.0219309. PMID: 31269068; PMCID: PMC6608950.
2024:
Waters V, Shaw M, Perrem L, Quon BS, Tullis E, Solomon M, Rayment JH, Lavoie A, Tse SM, Daigneault P, Bilodeau L, Price A, Nicholson M, Chin M, Parkins M, McKinney ML, Tam JS, Stanojevic S, Grasemann H, Ratjen F; (PIPE Study Investigators). A randomised trial of oral prednisone for cystic fibrosis pulmonary exacerbation treatment. Eur Respir J. 2024 Jun 6;63(6):2302278. doi: 10.1183/13993003.02278-2023. PMID: 38697648.
Gatt D, Shaw M, McCoy J, Kritzinger F, Solomon M, Dell S, Ratjen F. Disease Manifestations in Siblings with Primary Ciliary Dyskinesia. Ann Am Thorac Soc. 2024 Jan;21(1):173-175. doi: 10.1513/AnnalsATS.202308-747RL. PMID: 37903339.
Martin I, McDonald N, Wilson D, Ratjen F, Grasemann H. Elexacaftor-tezacaftor-ivacaftor increases airway nitric oxide in children with cystic fibrosis. J Cyst Fibros. 2024 Jan;23(1):109-111. doi: 10.1016/j.jcf.2023.05.015. Epub 2023 Oct 21. PMID: 37867075.
2023:
Grasemann H, Ratjen F. Cystic Fibrosis. N Engl J Med. 2023 Nov 2;389(18):1693-1707. doi: 10.1056/NEJMra2216474. PMID: 37913507.
Gatt D, Shaw M, Waters V, Kritzinger F, Solomon M, Dell S, Ratjen F. Treatment response to pulmonary exacerbation in primary ciliary dyskinesia. Pediatr Pulmonol. 2023 Oct;58(10):2857-2864. doi: 10.1002/ppul.26599. Epub 2023 Jul 14. PMID: 37449771.
Gatt D, Shaw M, Wee W, Solomon M, Dell SD, Ratjen F. Efficacy of Antibiotic Eradication Therapy of Early Pseudomonas aeruginosa Infection in Children with Primary Ciliary Dyskinesia. Ann Am Thorac Soc. 2023 Jun;20(6):854-860. doi: 10.1513/AnnalsATS.202210-858OC. PMID: 36753426.
Licht JC, Seidl E, Slingers G, Waters V, de Vries R, Post M, Ratjen F, Grasemann H. Exhaled breath profiles to detect lung infection with Staphylococcus aureus in children with cystic fibrosis. J Cyst Fibros. 2023 Sep;22(5):888-893. doi: 10.1016/j.jcf.2023.02.010. Epub 2023 Feb 26. PMID: 36849333.
Alam FS, Zanette B, Munidasa S, Braganza S, Li D, Woods JC, Ratjen F, Santyr G. Intra- and Inter-visit Repeatability of 129 Xenon Multiple-Breath Washout MRI in Children With Stable Cystic Fibrosis Lung Disease. J Magn Reson Imaging. 2023 Sep;58(3):936-948. doi: 10.1002/jmri.28638. Epub 2023 Feb 14. PMID: 36786650.
2022:
Grasemann H, McDonald N, Yuan XZ, Dell S, Waters V, Ratjen F. Lower Airway Nitrogen Oxide Levels in Children with Primary Ciliary Dyskinesia Is Linked to Neutrophilic Inflammation. J Pediatr. 2022 May;244:230-233. doi: 10.1016/j.jpeds.2022.01.040. Epub 2022 Feb 2. PMID: 35120987.
2021:
Gonska T, Keenan K, Au J, Dupuis A, Chilvers MA, Burgess C, Bjornson C, Fairservice L, Brusky J, Kherani T, Jober A, Kosteniuk L, Price A, Itterman J, Morgan L, Mateos-Corral D, Hughes D, Donnelly C, Smith MJ, Iqbal S, Arpin J, Reisman J, Hammel J, van Wylick R, Derynck M, Henderson N, Solomon M, Ratjen F. Outcomes of Cystic Fibrosis Screening-Positive Infants With Inconclusive Diagnosis at School Age. Pediatrics. 2021 Dec 1;148(6):e2021051740. doi: 10.1542/peds.2021-051740. PMID: 34814176.
Filipow N, Davies G, Main E, Sebire NJ, Wallis C, Ratjen F, Stanojevic S. Unsupervised phenotypic clustering for determining clinical status in children with cystic fibrosis. Eur Respir J. 2021 Aug 5;58(2):2002881. doi: 10.1183/13993003.02881-2020. PMID: 33446607.
Pollak M, Shaw M, Wilson D, Solomon M, Ratjen F, Grasemann H. Bronchodilator responsiveness in cystic fibrosis children treated for pulmonary exacerbations. Pediatr Pulmonol. 2021 Jul;56(7):2036-2042. doi: 10.1002/ppul.25409. Epub 2021 Apr 20. PMID: 33830642.
Nichols DP, Donaldson SH, Frederick CA, Freedman SD, Gelfond D, Hoffman LR, Kelly A, Narkewicz MR, Pittman JE, Ratjen F, Sagel SD, Rosenfeld M, Schwarzenberg SJ, Singh PK, Solomon GM, Stalvey MS, Kirby S, VanDalfsen JM, Clancy JP, Rowe SM. PROMISE: Working with the CF community to understand emerging clinical and research needs for those treated with highly effective CFTR modulator therapy. J Cyst Fibros. 2021 Mar;20(2):205-212. doi: 10.1016/j.jcf.2021.02.003. Epub 2021 Feb 19. PMID: 33619012; PMCID: PMC8686210.
2020:
Faughnan ME, Mager JJ, Hetts SW, Palda VA, Lang-Robertson K, Buscarini E, Deslandres E, Kasthuri RS, Lausman A, Poetker D, Ratjen F, Chesnutt MS, Clancy M, Whitehead KJ, Al-Samkari H, Chakinala M, Conrad M, Cortes D, Crocione C, Darling J, de Gussem E, Derksen C, Dupuis-Girod S, Foy P, Geisthoff U, Gossage JR, Hammill A, Heimdal K, Henderson K, Iyer VN, Kjeldsen AD, Komiyama M, Korenblatt K, McDonald J, McMahon J, McWilliams J, Meek ME, Mei-Zahav M, Olitsky S, Palmer S, Pantalone R, Piccirillo JF, Plahn B, Porteous MEM, Post MC, Radovanovic I, Rochon PJ, Rodriguez-Lopez J, Sabba C, Serra M, Shovlin C, Sprecher D, White AJ, Winship I, Zarrabeitia R. Second International Guidelines for the Diagnosis and Management of Hereditary Hemorrhagic Telangiectasia. Ann Intern Med. 2020 Dec 15;173(12):989-1001. doi: 10.7326/M20-1443. Epub 2020 Sep 8. PMID: 32894695.
Eber E, Trawinska-Bartnicka M, Sands D, Bellon G, Mellies U, Bolbás K, Quattrucci S, Mazurek H, Widmann R, Schoergenhofer C, Jilma B, Ratjen F. Aerosolized lancovutide in adolescents (≥12 years) and adults with cystic fibrosis – a randomized trial. J Cyst Fibros. 2021 Jan;20(1):61-67. doi: 10.1016/j.jcf.2020.08.014. Epub 2020 Sep 1. PMID: 32888826.
Stanojevic S, Filipow N, Ratjen F. Paediatric reproducibility limits for the forced expiratory volume in 1 s. Thorax. 2020 Oct;75(10):891-896. doi: 10.1136/thoraxjnl-2020-214817. Epub 2020 Aug 11. PMID: 32788261.
Mayer-Hamblett N, van Koningsbruggen-Rietschel S, Nichols DP, VanDevanter DR, Davies JC, Lee T, Durmowicz AG, Ratjen F, Konstan MW, Pearson K, Bell SC, Clancy JP, Taylor-Cousar JL, De Boeck K, Donaldson SH, Downey DG, Flume PA, Drevinek P, Goss CH, Fajac I, Magaret AS, Quon BS, Singleton SM, VanDalfsen JM, Retsch-Bogart GZ. Building global development strategies for cf therapeutics during a transitional cftr modulator era. J Cyst Fibros. 2020 Sep;19(5):677-687. doi: 10.1016/j.jcf.2020.05.011. Epub 2020 Jun 7. PMID: 32522463; PMCID: PMC7492419.
Perrem L, Stanojevic S, Shaw M, Davis S, Retsch-Bogart G, Ratjen F. Changes in the parent cystic fibrosis questionnaire-revised (CFQ-R) with respiratory symptoms in preschool children with cystic fibrosis. J Cyst Fibros. 2020 May;19(3):492-498. doi: 10.1016/j.jcf.2020.02.017. Epub 2020 Mar 2. PMID: 32139196; PMCID: PMC7329610.
Grasemann H, Klingel M, Avolio J, Prentice C, Gonska T, Tullis E, Ratjen F. Long-term effect of CFTR modulator therapy on airway nitric oxide. Eur Respir J. 2020 Jan 9;55(1):1901113. doi: 10.1183/13993003.01113-2019. PMID: 31601715.
2019:
Bell SC, Mall MA, Gutierrez H, Macek M, Madge S, Davies JC, Burgel PR, Tullis E, Castaños C, Castellani C, Byrnes CA, Cathcart F, Chotirmall SH, Cosgriff R, Eichler I, Fajac I, Goss CH, Drevinek P, Farrell PM, Gravelle AM, Havermans T, Mayer-Hamblett N, Kashirskaya N, Kerem E, Mathew JL, McKone EF, Naehrlich L, Nasr SZ, Oates GR, O’Neill C, Pypops U, Raraigh KS, Rowe SM, Southern KW, Sivam S, Stephenson AL, Zampoli M, Ratjen F. The future of cystic fibrosis care: a global perspective. Lancet Respir Med. 2020 Jan;8(1):65-124. doi: 10.1016/S2213-2600(19)30337-6. Epub 2019 Sep 27. Erratum in: Lancet Respir Med. 2019 Dec;7(12):e40. doi: 10.1016/S2213-2600(19)30408-4. PMID: 31570318; PMCID: PMC8862661.
Klingel M, Stanojevic S, Tullis E, Ratjen F, Waters V. Oral Azithromycin and Response to Pulmonary Exacerbations Treated with Intravenous Tobramycin in Children with Cystic Fibrosis. Ann Am Thorac Soc. 2019 Jul;16(7):861-867. doi: 10.1513/AnnalsATS.201811-774OC. PMID: 30874447.
Perrem L, Stanojevic S, Solomon M, Carpenter S, Ratjen F. Incidence and risk factors of paediatric cystic fibrosis-related diabetes. J Cyst Fibros. 2019 Nov;18(6):874-878. doi: 10.1016/j.jcf.2019.04.015. Epub 2019 May 6. PMID: 31072797.
Rayment JH, Couch MJ, McDonald N, Kanhere N, Manson D, Santyr G, Ratjen F. Hyperpolarised 129Xe magnetic resonance imaging to monitor treatment response in children with cystic fibrosis. Eur Respir J. 2019 May 2;53(5):1802188. doi: 10.1183/13993003.02188-2018. PMID: 30819815.
