The effect of discontinuing hypertonic saline or dornase alfa on mucociliary clearance in elexacaftor/tezacaftor/ivacaftor treated people with cystic fibrosis: The SIMPLIFY-MCC Study
The availability of highly effective CFTR modulators, especially elexacaftor/tezacaftor/ivacaftor (ETI), has changed the lives of people with cystic fibrosis (pwCF) via marked improvements in lung function, nutrition, exacerbation frequency, and symptoms [1 –3]. As a result, pwCF and their providers have questioned the need for other supportive therapies. Because ETI substantially improves mucociliary clearance (MCC) [4], the additive benefit of hypertonic saline (HS) and dornase alfa (DA) are particularly uncertain [5]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - February 13, 2024 Category: Respiratory Medicine Authors: Scott H. Donaldson, Timothy E. Corcoran, Joseph M. Pilewski, Beth L. Laube, Peter Mogayzel, Agathe Ceppe, Jihong Wu, Kirby Zeman, Steven M. Rowe, David P. Nichols, Alex H. Gifford, William D. Bennett, Nicole Mayer-Hamblett, SIMPLIFY MCC Study teams Tags: Short Communication Source Type: research
Nebuliser systems for drug delivery in cystic fibrosis
CONCLUSIONS: Newer technologies e.g. AAD and VMT have advantages over conventional systems in terms of treatment time, deposition as a percentage of priming dose, preference and adherence. Data are lacking for all varieties of medications which are used in CF care, including different inhaled antibiotics or hypertonic saline, with all delivery (nebuliser system) possibilities. Long-term RCTs are needed to evaluate different nebuliser systems to determine patient-focused outcomes (such as QoL and burden of care), safe and effective dosing levels of a wide variety of medications, clinical outcomes (such as hospitalisations a...
Source: Cochrane Database of Systematic Reviews - November 9, 2023 Category: General Medicine Authors: Gemma Stanford Lisa Morrison Catherine Brown Source Type: research
Decreased mucolytic prescriptions in Phe508del homozygous Cystic Fibrosis Adults on Elexacaftor-tezacaftor-ivacaftor (Kaftrio/Trikafta)
Conclusion: Kaftrio reduces the prescription of Dornase alfa and HTS and overall mucolytic prescription in these patients. No significant reduction in carbocisteine presciption, though limited by sample size. This reinforces the role of Kaftrio in reducing the treatment burden in cystic fibrosis patients. (Source: European Respiratory Journal)
Source: European Respiratory Journal - October 27, 2023 Category: Respiratory Medicine Authors: Zahid, M. A., Hartley, J., Sur Roy, A., Lau, C., Jarvis, F., Mukhtar, K., Khan, M. Tags: Adult CF Source Type: research
Dornase Alfa and Hypertonic Saline: Pass the Salt?
Chest. 2023 Oct;164(4):807-808. doi: 10.1016/j.chest.2023.06.036.NO ABSTRACTPMID:37805234 | DOI:10.1016/j.chest.2023.06.036 (Source: Chest)
Source: Chest - October 7, 2023 Category: Respiratory Medicine Authors: Marc A Sala Manu Jain Source Type: research
113 Effect of discontinuing hypertonic saline or dornase alfa on mucociliary clearance in people with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor: The SIMPLIFY-MCC Study
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - October 1, 2023 Category: Respiratory Medicine Authors: S. Donaldson, T. Corcoran, J. Pilewski, B. Laube, P. Mogayzel, A. Ceppe, J. Wu, K. Zeman, S. Rowe, D. Nichols, A. Gifford, W. Bennett, N. Hamblett Tags: CLINICAL TRIALS & OUTCOME MEASURES Source Type: research
106 Impact of discontinuing hypertonic saline and dornase alfa in people with cystic fibrosis established on elexacaftor-tezacaftor-ivacaftor: A SIMPLIFY ancillary study
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - October 1, 2023 Category: Respiratory Medicine Authors: N. Hamblett, A. Gifford, M. Kloster, R. Russell, A. Braun, R. Gibson, J. Hoppe, R. Jain, R. Linnemann, T. Liou, J. Lysinger, C. Milla, J. Young, D. Nichols Tags: CLINICAL TRIALS & OUTCOME MEASURES Source Type: research
Emulated trial investigating effects of multiple treatments: estimating combined effects of mucoactive nebulisers in cystic fibrosis using registry data
Conclusion
For individuals with CF prescribed DNase, we found no evidence that adding HS had an effect on FEV1% or prescription of intravenous antibiotics. Our study illustrates the emulated target trial approach using CF Registry data. (Source: Thorax)
Source: Thorax - September 15, 2023 Category: Respiratory Medicine Authors: Granger, E., Davies, G., Keogh, R. H. Tags: Open access, Thorax Cystic fibrosis Source Type: research
Bladder cancer patients have increased NETosis and impaired DNaseI-mediated NET degradation that can be therapeutically restored < em > in vitro < /em >
CONCLUSION: To the best of our knowledge, this is the first report demonstrating that BC patients have an increased NETosis systemically and in the tumor microenvironment, in part caused by an impaired DNaseI-mediated NET degradation. Remarkably, this defect can be therapeutically restored in vitro with the approved Dornase alfa, thus Pulmozyme® could become a potential therapeutic tool to locally reduce BC progression.PMID:37275882 | PMC:PMC10237292 | DOI:10.3389/fimmu.2023.1171065 (Source: Cancer Control)
Source: Cancer Control - June 5, 2023 Category: Cancer & Oncology Authors: Raquel Herranz Julia Oto Marta Hueso Emma Plana Fernando Cana Mar ía Castaño Lourdes Cord ón David Ramos-Soler Santiago Bonanad C ésar D Vera-Donoso Manuel Mart ínez-Sarmiento Pilar Medina Source Type: research
WS05.05 Randomised withdrawal of hypertonic saline in those with lower lung function after receiving elexacaftor/tezacaftor/ivacaftor; a sub-study of the SIMPLIFY Trial
Objectives: SIMPLIFY tested withdrawal of hypertonic saline (HS) or dornase alfa for 6 weeks in PwCF taking ETI and showed non-inferiority (compared with continuing HS or dornase alfa) in both lung function and respiratory symptom scores. Inclusion in either trial required ppFEV1 ≥60%, and the mean ppFEV1 was 97%. A pre-planned lower lung function (LLF) sub-study was conducted to test HS withdrawal in PwCF with ppFEV1 of 40–59%, hypothesizing that continuing HS is superior to discontinuing HS with respect to 6-week change in ppFEV1. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2023 Category: Respiratory Medicine Authors: D. Nichols, A. Gifford, M. Kloster, R. Russell, J. Young, R. Amaro-Galvez, J. Billings, Z. Mukadam, J. Clancy, N. Mayer-Hamblett Tags: Workshops Source Type: research
Effect of Hypertonic Saline on Lung Function as Add-On Treatment in People with Cystic Fibrosis Receiving Dornase-alfa: A Cystic Fibrosis Foundation Patient Registry Analysis
Chest. 2023 May 25:S0012-3692(23)00783-3. doi: 10.1016/j.chest.2023.05.024. Online ahead of print.ABSTRACTBACKGROUND: Introduction of novel therapies for cystic fibrosis (CF), raises the question whether traditional treatments can be withdrawn. Nebulized hypertonic saline (HS) could potentially be discontinued in patients receiving Dornase alfa (DA).RESEARCH QUESTION: In the pre-modulator era, did people with CF who were F508del homozygous (CFF508del) and received DA and HS have better preserved lung function than those treated with DA only?STUDY DESIGN: Retrospective analysis of CF Foundation Patient Registry data (2006-2...
Source: Chest - May 27, 2023 Category: Respiratory Medicine Authors: Athanasios G Kaditis James Acton Connie Fenton Leila Kheirandish-Gozal Zarah Ner Rebekah Nevel David Gozal Adrienne Ohler Source Type: research
Effect of Hypertonic Saline on Lung Function as Add-On Treatment in People with Cystic Fibrosis Receiving Dornase-alfa: A Cystic Fibrosis Foundation Patient Registry Analysis
Chest. 2023 May 25:S0012-3692(23)00783-3. doi: 10.1016/j.chest.2023.05.024. Online ahead of print.ABSTRACTBACKGROUND: Introduction of novel therapies for cystic fibrosis (CF), raises the question whether traditional treatments can be withdrawn. Nebulized hypertonic saline (HS) could potentially be discontinued in patients receiving Dornase alfa (DA).RESEARCH QUESTION: In the pre-modulator era, did people with CF who were F508del homozygous (CFF508del) and received DA and HS have better preserved lung function than those treated with DA only?STUDY DESIGN: Retrospective analysis of CF Foundation Patient Registry data (2006-2...
Source: Chest - May 27, 2023 Category: Respiratory Medicine Authors: Athanasios G Kaditis James Acton Connie Fenton Leila Kheirandish-Gozal Zarah Ner Rebekah Nevel David Gozal Adrienne Ohler Source Type: research
Effect of Hypertonic Saline on Lung Function as Add-On Treatment in People with Cystic Fibrosis Receiving Dornase-alfa: A Cystic Fibrosis Foundation Patient Registry Analysis
Chest. 2023 May 25:S0012-3692(23)00783-3. doi: 10.1016/j.chest.2023.05.024. Online ahead of print.ABSTRACTBACKGROUND: Introduction of novel therapies for cystic fibrosis (CF), raises the question whether traditional treatments can be withdrawn. Nebulized hypertonic saline (HS) could potentially be discontinued in patients receiving Dornase alfa (DA).RESEARCH QUESTION: In the pre-modulator era, did people with CF who were F508del homozygous (CFF508del) and received DA and HS have better preserved lung function than those treated with DA only?STUDY DESIGN: Retrospective analysis of CF Foundation Patient Registry data (2006-2...
Source: Chest - May 27, 2023 Category: Respiratory Medicine Authors: Athanasios G Kaditis James Acton Connie Fenton Leila Kheirandish-Gozal Zarah Ner Rebekah Nevel David Gozal Adrienne Ohler Source Type: research
Effect of Hypertonic Saline on Lung Function as Add-On Treatment in People with Cystic Fibrosis Receiving Dornase-alfa: A Cystic Fibrosis Foundation Patient Registry Analysis
Chest. 2023 May 25:S0012-3692(23)00783-3. doi: 10.1016/j.chest.2023.05.024. Online ahead of print.ABSTRACTBACKGROUND: Introduction of novel therapies for cystic fibrosis (CF), raises the question whether traditional treatments can be withdrawn. Nebulized hypertonic saline (HS) could potentially be discontinued in patients receiving Dornase alfa (DA).RESEARCH QUESTION: In the pre-modulator era, did people with CF who were F508del homozygous (CFF508del) and received DA and HS have better preserved lung function than those treated with DA only?STUDY DESIGN: Retrospective analysis of CF Foundation Patient Registry data (2006-2...
Source: Chest - May 27, 2023 Category: Respiratory Medicine Authors: Athanasios G Kaditis James Acton Connie Fenton Leila Kheirandish-Gozal Zarah Ner Rebekah Nevel David Gozal Adrienne Ohler Source Type: research
Effect of Hypertonic Saline on Lung Function as Add-On Treatment in People with Cystic Fibrosis Receiving Dornase-alfa: A Cystic Fibrosis Foundation Patient Registry Analysis
Chest. 2023 May 25:S0012-3692(23)00783-3. doi: 10.1016/j.chest.2023.05.024. Online ahead of print.ABSTRACTBACKGROUND: Introduction of novel therapies for cystic fibrosis (CF), raises the question whether traditional treatments can be withdrawn. Nebulized hypertonic saline (HS) could potentially be discontinued in patients receiving Dornase alfa (DA).RESEARCH QUESTION: In the pre-modulator era, did people with CF who were F508del homozygous (CFF508del) and received DA and HS have better preserved lung function than those treated with DA only?STUDY DESIGN: Retrospective analysis of CF Foundation Patient Registry data (2006-2...
Source: Chest - May 27, 2023 Category: Respiratory Medicine Authors: Athanasios G Kaditis James Acton Connie Fenton Leila Kheirandish-Gozal Zarah Ner Rebekah Nevel David Gozal Adrienne Ohler Source Type: research
