Chronic daily respiratory care needs in people with cystic fibrosis treated with highly effective cystic fibrosis transmembrane conductance regulator modulators.
Journal
Current opinion in pulmonary medicine
ISSN: 1531-6971
Titre abrégé: Curr Opin Pulm Med
Pays: United States
ID NLM: 9503765
Informations de publication
Date de publication:
01 11 2023
01 11 2023
Historique:
medline:
5
10
2023
pubmed:
23
8
2023
entrez:
23
8
2023
Statut:
ppublish
Résumé
Cystic fibrosis is a genetic disease that increases risk of death from respiratory failure because of impairment in mucociliary clearance. Complex daily care regimens including medications and airway clearance techniques (ACTs) aim to preserve lung function and alleviate symptoms for people with cystic fibrosis (pwCF). The success of highly effective modulator therapy (HEMT) permits evaluation of treatment simplification. In this review, we evaluate adjustments made in daily respiratory care among pwCF taking HEMT and the feasibility of treatment simplification. Treatment simplification has been identified as a top priority among pwCF, with recent studies showing pwCF are willing to sacrifice mild to moderate amounts of lung function and longevity to reduce treatment burden. Retrospective studies have shown that patients taking HEMT with better baseline lung function have lower adherence to and prescription of inhaled medications. A randomized, controlled trial found that short-term discontinuation of dornase alfa or hypertonic saline was clinically noninferior to continuation of these medications. Major knowledge gaps remain about withdrawing ACTs. This review highlights trials evaluating the feasibility of treatment simplification among pwCF taking HEMT. More data is needed to evaluate approaches to simplification in this phenotypically diverse patient population.
Identifiants
pubmed: 37611027
doi: 10.1097/MCP.0000000000001006
pii: 00063198-990000000-00103
doi:
Substances chimiques
Cystic Fibrosis Transmembrane Conductance Regulator
126880-72-6
Types de publication
Review
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
580-586Informations de copyright
Copyright © 2023 Wolters Kluwer Health, Inc. All rights reserved.
Références
Shteinberg M, Haq IJ, Polineni D, Davies JC. Cystic fibrosis. Lancet 2021; 397:2195–2211.
Haq IJ, Gray MA, Garnett JP, et al. Airway surface liquid homeostasis in cystic fibrosis: pathophysiology and therapeutic targets. Thorax 2016; 71:284–287.
Ehre C, Hansson GC, Thornton DJ, Ostedgaard LS. Mucus aberrant properties in CF: insights from cells and animal models. J Cyst Fibros 2023; 22 Suppl 1:S23–S26.
Batson BD, Zorn BT, Radicioni G, et al. Cystic fibrosis airway mucus hyperconcentration produces a vicious cycle of mucin, pathogen, and inflammatory interactions that promotes disease persistence. Am J Respir Cell Mol Biol 2022; 67:253–265.
Hill DB, Button B, Rubinstein M, Boucher RC. Physiology and pathophysiology of human airway mucus. Physiol Rev 2022; 102:1757–1836.
Nichols DP, Chmiel JF. Inflammation and its genesis in cystic fibrosis. Pediatr Pulmonol 2015; 50: (Suppl 40): S39–S56.
Yonker LM, Marand A, Muldur S, et al. Neutrophil dysfunction in cystic fibrosis. J Cyst Fibros 2021; 20:1062–1071.
Wang G, Nauseef WM. Neutrophil dysfunction in the pathogenesis of cystic fibrosis. Blood 2022; 139:2622–2631.
Aaron SD, Stephenson AL, Cameron DW, Whitmore GA. A statistical model to predict one-year risk of death in patients with cystic fibrosis. J Clin Epidemiol 2014; 68:1336–1345.
Belkin RA, Henig NR, Singer LG, et al. Risk factors for death of patients with cystic fibrosis awaiting lung transplantation. Am J Respir Crit Care Med 2006; 173:659–666.
Mogayzel PJ Jr, Naureckas ET, Robinson KA, et al. Cystic fibrosis pulmonary guidelines. Chronic medications for maintenance of lung health. Am J Respir Crit Care Med 2013; 187:680–689.
Flume PA, Robinson KA, O'Sullivan BP, et al. Clinical Practice Guidelines for Pulmonary Therapies Committee. Cystic fibrosis pulmonary guidelines: airway clearance therapies. Resp Care 2009; 54:522–537.
Donaldson SH, Bennett WD, Zeman KL, et al. Mucus clearance and lung function in cystic fibrosis with hypertonic saline. New Engl J Med 2006; 354:241–250.
Elkins MR, Robinson M, Rose BR, et al. National Hypertonic Saline in Cystic Fibrosis (NHSCF) Study Group. A controlled trial of long-term inhaled hypertonic saline in patients with cystic fibrosis. New Engl J Med 2006; 354:229–240.
Henke MO, Ratjen F. Mucolytics in cystic fibrosis. Paediatr Resp Rev 2007; 8:24–29.
Fuchs HJ, Borowitz DS, Christiansen DH, et al. Effect of aerosolized recombinant human DNase on exacerbations of respiratory symptoms and on pulmonary function in patients with cystic fibrosis. The Pulmozyme Study Group. N Engl J Med 1994; 331:637–642.
Ramsey BW, Pepe MS, Quan JM, et al. Intermittent administration of inhaled tobramycin in patients with cystic fibrosis. Cystic Fibrosis Inhaled Tobramycin Study Group. N Engl J Med 1999; 340:23–30.
Konstan MW, Flume PA, Kappler M, et al. Safety, efficacy and convenience of tobramycin inhalation powder in cystic fibrosis patients: The EAGER trial. J Cyst Fibros 2011; 10:54–61.
McCoy KS, Quittner AL, Oermann CM, et al. Inhaled aztreonam lysine for chronic airway Pseudomonas aeruginosa in cystic fibrosis. Am J Respir Crit Care Med 2008; 178:921–928.
Schuster A, Haliburn C, Doring G, et al. Freedom Study Group. Safety, efficacy and convenience of colistimethate sodium dry powder for inhalation (Colobreathe DPI) in patients with cystic fibrosis: a randomised study. Thorax 2013; 68:344–350.
Burnham P, Stanford G, Stewart R. Autogenic drainage for airway clearance in cystic fibrosis. Cochrane Database Syst Rev 2021; 12:CD009595.
Heinz KD, Walsh A, Southern KW, et al. Exercise versus airway clearance techniques for people with cystic fibrosis. Cochrane Database Syst Rev 2022; 6:CD013285.
McIlwaine M, Button B, Nevitt SJ. Positive expiratory pressure physiotherapy for airway clearance in people with cystic fibrosis. Cochrane Database Syst Rev 2019; 2019:CD003147.
Gifford AH, Taylor-Cousar JL, Davies JC, McNally P. Update on clinical outcomes of highly effective modulator therapy. Clin Chest Med 2022; 43:677–695.
Van Goor F, Yu H, Burton B, Hoffman BJ. Effect of ivacaftor on CFTR forms with missense mutations associated with defects in protein processing or function. J Cyst Fibros 2014; 13:29–36.
Ramsey BW, Davies J, McElvaney NG, et al. VX08-770-102 Study Group. A CFTR potentiator in patients with cystic fibrosis and the G551D mutation. New Engl J Med 2011; 365:1663–1672.
Rosenfeld M, Wainwright CE, Higgins M, et al. Ivacaftor treatment of cystic fibrosis in children aged 12 to <24 months and with a CFTR gating mutation (ARRIVAL): a phase 3 single-arm study. Lancet Respir Med 2018; 6:545–553.
Keating D, Marigowda G, Burr L, et al. VX16-445-001 Study Group. VX-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two phe508del alleles. New Engl J Med 2018; 379:1612–1620.
Middleton PG, Mall MA, Drevinek P, et al. VX17-445-102 Study Group. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. New Engl J Med 2019; 381:1809–1819.
Sawicki GS, Sellers DE, Robinson WM. High treatment burden in adults with cystic fibrosis: challenges to disease self-management. J Cyst Fibros 2009; 8:91–96.
Sawicki GS, Ren CL, Konstan MW, et al. Treatment complexity in cystic fibrosis: trends over time and associations with site-specific outcomes. J Cyst Fibros 2013; 12:461–467.
Sawicki GS, Tiddens H. Managing treatment complexity in cystic fibrosis: challenges and opportunities. Pediatr Pulmonol 2012; 47:523–533.
George M, Rand-Giovannetti D, Eakin MN, et al. Perceptions of barriers and facilitators: self-management decisions by older adolescents and adults with CF. J Cyst Fibros 2010; 9:425–432.
Hente E, Weiland J, Mullen L, et al. Assessment of treatment burden and complexity in cystic fibrosis: a quality improvement project. Pediatr Pulmonol 2021; 56:1992–1999.
Rowbotham NJ, Smith S, Leighton PA, et al. The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers. Thorax 2018; 73:388–390.
Gifford AH, Mayer-Hamblett N, Pearson K, Nichols DP. Answering the call to address cystic fibrosis treatment burden in the era of highly effective CFTR modulator therapy. J Cyst Fibros 2020; 19:762–767.
Hollin IL, Donaldson SH, Roman C, et al. Beyond the expected: identifying broad research priorities of researchers and the cystic fibrosis community. J Cyst Fibros 2019; 18:375–377.
Cameron RA, Office D, Matthews J, et al. Treatment preference among people with cystic fibrosis: the importance of reducing treatment burden. Chest 2022; 162:1241–1254.
Kapnadak SG, Dimango E, Hadjiliadis D, et al. Cystic fibrosis foundation consensus guidelines for the care of individuals with advanced cystic fibrosis lung disease. J Cyst Fibros 2020; 19:344–354.
Burgel PR, Durieu I, Chiron R, et al. French Cystic Fibrosis Reference Network Study Group. Rapid improvement after starting elexacaftor-tezacaftor-ivacaftor in patients with cystic fibrosis and advanced pulmonary disease. Am J Respir Crit Care Med 2021; 204:64–73.
O'Shea KM, O’Carroll OM, Carroll C, et al. Efficacy of elexacaftor/tezacaftor/ivacaftor in patients with cystic fibrosis and advanced lung disease. Eur Respir J 2021; 57:2003079.
Taylor-Cousar J, Niknian M, Gilmartin G, Pilewski JM. investigators VX. Effect of ivacaftor in patients with advanced cystic fibrosis and a G551D-CFTR mutation: safety and efficacy in an expanded access program in the United States. J Cyst Fibros 2016; 15:116–122.
Salvatore D, Terlizzi V, Francalanci M, et al. Ivacaftor improves lung disease in patients with advanced CF carrying CFTR mutations that confer residual function. Respir Med 2020; 171:106073.
Batalden M, Batalden P, Margolis P, et al. Coproduction of healthcare service. BMJ Qual Saf 2016; 25:509–517.
Homa K, Stevens G, Forcino R, et al. Assessing shared decision-making in cystic fibrosis care using collaboRATE: a cross-sectional study of 159 programs. J Patient Exp 2021; 8: 23743735211034032.
Almulhem M, Harnett N, Graham S, et al. Exploring the impact of elexacaftor-tezacaftor-ivacaftor treatment on opinions regarding airway clearance techniques and nebulisers: TEMPO a qualitative study in children with cystic fibrosis, their families and healthcare professionals. BMJ Open Respir Res 2022; 9:e001420.
Granger E, Davies G, Keogh RH. Treatment patterns in people with cystic fibrosis: have they changed since the introduction of ivacaftor? J Cyst Fibros 2022; 21:316–322.
Guimbellot JS, Baines A, Paynter A, et al. Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation. J Cyst Fibros 2021; 20:213–219.
Nichols DP, Paynter AC, Heltshe SL, et al. Clinical effectiveness of elexacaftor/tezacftor/ivacaftor in people with cystic fibrosis: a clinical trial. Am J Respir Crit Care Med 2022; 205:529–539.
Song JT, Desai S, Franciosi AN, et al. Research letter: The impact of elexacaftor/tezacaftor/ivacaftor on adherence to nebulized maintenance therapies in people with cystic fibrosis. J Cyst Fibros 2022; 21:1080–1081.
Mayer-Hamblett N, Nichols DP, Odem-Davis K, et al. Evaluating the impact of stopping chronic therapies after modulator drug therapy in cystic fibrosis: the SIMPLIFY Clinical Trial Study Design. Ann Am Thorac Soc 2021; 18:1397–1405.
Mayer-Hamblett N, Ratjen F, Russell R, et al. 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, noninferiority trials. Lancet Respir Med 2023; 11:329–340.
Robinson PD, Douglas TA, Wainwright CE. Reducing treatment burden in the era of CFTR modulators. Lancet Respir Med 2023; doi: 10.1016/S2213-2600(23)00223-0. [Epub ahead of print].
doi: 10.1016/S2213-2600(23)00223-0.
Griese M, Costa S, Linnemann RW, et al. Safety and efficacy of elexacaftor/tezacaftor/ivacaftor for 24 weeks or longer in people with cystic fibrosis and one or more F508del alleles: interim results of an open-label phase 3 clinical trial. Am J Respir Crit Care Med 2021; 203:381–385.
Bower JK, Volkova N, Ahluwalia N, et al. Real-world safety and effectiveness of elexacaftor/tezacaftor/ivacaftor in people with cystic fibrosis: Interim results of a long-term registry-based study. J Cyst Fibros 2023; doi: 10.1016/j.jcf.2023.03.002. [Epub ahead of print].
doi: 10.1016/j.jcf.2023.03.002.
Gifford AH, Mayer-Hamblett N, Nichols DP. Reducing treatment burden in the era of CFTR modulators - authors’ reply. Lancet Respir Med 2023; doi: 10.1016/S2213-2600(23)00224-2. [Epub ahead of print].
doi: 10.1016/S2213-2600(23)00224-2.
Rowbotham NJ, Daniels TE. Airway clearance and exercise for people with cystic fibrosis: balancing longevity with life. Pediatr Pulmonol 2022; 57: (Suppl 1): S50–S59.
VanDevanter DR, Zemanick ET, Konstan MW, et al. CHEC-SC Study Group. Willingness of people with cystic fibrosis receiving elexacaftor/tezacaftor/ivacaftor (ETI) to participate in randomized modulator and inhaled antimicrobial clinical trials. J Cyst Fibros 2023; doi: 10.1016/j.jcf.2023.04.007. [Epub ahead of print].
doi: 10.1016/j.jcf.2023.04.007.