Determining the minimal clinically important difference for the questionnaire of olfactory disorders in people with cystic fibrosis and factors associated with improvement after highly effective modulator therapy.
chronic rhinosinusitis
cystic fibrosis
highly effective modulator therapy
olfaction
olfactory disorders
olfactory dysfunction
olfactory test
patient-reported outcome measure
quality of life
Journal
International forum of allergy & rhinology
ISSN: 2042-6984
Titre abrégé: Int Forum Allergy Rhinol
Pays: United States
ID NLM: 101550261
Informations de publication
Date de publication:
25 Dec 2023
25 Dec 2023
Historique:
revised:
21
11
2023
received:
27
08
2023
accepted:
05
12
2023
medline:
25
12
2023
pubmed:
25
12
2023
entrez:
25
12
2023
Statut:
aheadofprint
Résumé
Olfactory dysfunction (OD) is common among people with cystic fibrosis (PwCF). The Questionnaire of Olfactory Disorders (QOD) is a validated instrument that evaluates olfactory-specific quality-of-life. The QOD minimal clinically important difference (MCID) and factors associated with olfactory improvement after elexacaftor/tezacaftor/ivacaftor have not been determined for PwCF. Prospective observational data were pooled from three studies that enrolled adult PwCF with chronic rhinosinusitis (CRS). QOD scores and disease characteristics were assessed. To evaluate internal consistency and calculate the QOD MCID, Cronbach's alpha and four distribution-based methods were employed. For participants who enrolled prior to elexacaftor/tezacaftor/ivacaftor, QOD scores were obtained at baseline and after elexacaftor/tezacaftor/ivacaftor initiation. Multivariable regression was used to identify factors associated with QOD improvement. Of 129 PwCF included, 65 had QOD scores before and 3-6 months after starting elexacaftor/tezacaftor/ivacaftor. Mean baseline QOD score was 6.5 ± 7.9. Mean Cronbach's alpha was ≥0.85. The MCID estimates were as follows: Cohen's effect size = 1.6, standard error of measurement = 2.5, ½ baseline standard deviation = 4.0, and minimal detectable change = 6.9. Mean MCID was 3.7. Of those with pre/post elexacaftor/tezacaftor/ivacaftor QOD scores, the mean change in QOD was -1.3 ± 5.4. After elexacaftor/tezacaftor/ivacaftor, QOD improvement surpassed the MCID in 22% of participants (14/65). Worse baseline QOD scores and nasal polyps were associated with improved QOD scores after elexacaftor/tezacaftor/ivacaftor (both p < 0.04). The QOD MCID in PwCF was estimated to be 3.7. Elexacaftor/tezacaftor/ivacaftor led to qualitative but not clinically meaningful improvements in QOD score for most PwCF; PwCF with worse baseline QOD scores and nasal polyps improved in a clinically significant manner.
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Subventions
Organisme : Cystic Fibrosis Foundation
ID : BESWIC20A0
Organisme : Cystic Fibrosis Foundation
ID : TAYLOR19A0
Organisme : Cystic Fibrosis Foundation
ID : BESWIC22Y5
Organisme : Marshall and Margherite McComb Foundation
Informations de copyright
© 2023 ARS-AAOA, LLC.
Références
Soler ZM, Smith TL, Alt JA, Ramakrishnan VR, Mace JC, Schlosser RJ. Olfactory-specific quality of life outcomes after endoscopic sinus surgery. Int Forum Allergy Rhinol. 2016;6(4):407-413. doi:10.1002/alr.21679
Liu DT, Prem B, Sharma G, Kaiser J, Besser G, Mueller CA. Depression symptoms and olfactory-related quality of life. Laryngoscope. 2022;132(9):1829-1834. doi:10.1002/lary.30122
Sivam A, Wroblewski KE, Alkorta-Aranburu G, et al. Olfactory dysfunction in older adults is associated with feelings of depression and loneliness. Chem Senses. 2016;41(4):293-299. doi:10.1093/chemse/bjv088
Soler ZM, Kohli P, Storck KA, Schlosser RJ. Olfactory impairment in chronic rhinosinusitis using threshold, discrimination, and identification scores. Chem Senses. 2016;41(9):713-719. doi:10.1093/chemse/bjw080
Rudmik L, Smith TL. Olfactory improvement after endoscopic sinus surgery. Curr Opin Otolaryngol Head Neck Surg. 2012;20(1):29-32. doi:10.1097/MOO.0b013e32834dfb3d
Jiang RS, Lu FJ, Liang KL, et al. Olfactory function in patients with chronic rhinosinusitis before and after functional endoscopic sinus surgery. Am J Rhinol. 2008;22(4):445-448. doi:10.2500/ajr.2008.22.3195
Laing DG, Armstrong JE, Aitken M, et al. Chemosensory function and food preferences of children with cystic fibrosis. Pediatr Pulmonol. 2010;45(8):807-815. doi:10.1002/ppul.21261
Di Lullo AM, Iacotucci P, Comegna M, et al. Cystic fibrosis: the sense of smell. Am J Rhinol Allergy. 2020;34(1):35-42. doi:10.1177/1945892419870450
Beswick DM, Humphries SM, Balkissoon CD, et al. Olfactory dysfunction in people with cystic fibrosis with at least one copy of F508del. Int Forum Allergy Rhinol. 2022;12(7):963-966. doi:10.1002/alr.22946
Keating D, Marigowda G, Burr L, et al. VX-445-tezacaftor-ivacaftor in patients with cystic fibrosis and one or two Phe508del alleles. N Engl J Med. 2018;379(17):1612-1620. doi:10.1056/NEJMoa1807120
Middleton PG, Mall MA, Dřevínek P, et al. Elexacaftor-tezacaftor-ivacaftor for cystic fibrosis with a single Phe508del allele. N Engl J Med. 2019;381(19):1809-1819. doi:10.1056/NEJMoa1908639
Heijerman HGM, McKone EF, Downey DG, et al. Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation: a double-blind, randomised, phase 3 trial. Lancet. 2019;394(10212):1940-1948. doi:10.1016/S0140-6736(19)32597-8
DiMango E, Overdevest J, Keating C, Francis SF, Dansky D, Gudis D. Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis. J Cyst Fibros. 2021;20(3):460-463. doi:10.1016/j.jcf.2020.07.002
Stapleton AL, Kimple AJ, Goralski JL, et al. Elexacaftor-tezacaftor- ivacaftor improves sinonasal outcomes in cystic fibrosis. J Cyst Fibros. 2022;21(5):792-799. doi:10.1016/j.jcf.2022.03.002
Beswick DM, Humphries SM, Balkissoon CD, et al. Impact of cystic fibrosis transmembrane conductance regulator therapy on chronic rhinosinusitis and health status: deep learning CT analysis and patient-reported outcomes. Ann Am Thorac Soc. 2022;19(1):12-19. doi:10.1513/AnnalsATS.202101-057OC
Beswick DM, Khatiwada A, Miller JE, et al. Impact of highly effective modulator therapy on chronic rhinosinusitis and health status: 2-year follow-up. J Cyst Fibros. 2023. doi:10.1016/j.jcf.2023.09.013
Tervo JP, DiMango E, Gudis DA, et al. Olfaction, body mass index, and quality of life with cystic fibrosis combination therapy. Int Forum Allergy Rhinol. 2023;13:1-7. doi:10.1002/alr.23209
Bacon DR, Stapleton A, Goralski JL, et al. Olfaction before and after initiation of elexacaftor-tezacaftor-ivacaftor in a cystic fibrosis cohort. Int Forum Allergy Rhinol. 2022;12(2):223-226. doi:10.1002/alr.22891
Beswick DM, Humphries SM, Balkissoon CD, et al. Olfactory dysfunction in cystic fibrosis: impact of CFTR modulator therapy. J Cyst Fibros. 2022;21(2):e141-e147. doi:10.1016/j.jcf.2021.09.014
Castellanos CX, Osterbauer B, Hasday S, Keens TG, Koempel J, Ference EH. Improvement in sinonasal quality-of-life indicators for pediatric patients with cystic fibrosis treated with elexacaftor-tezacaftor-ivacaftor. Int Forum Allergy Rhinol. 2023;13(1):72-75. doi:10.1002/alr.23036
Patel ZM, Holbrook EH, Turner JH, et al. International consensus statement on allergy and rhinology: olfaction. Int Forum Allergy Rhinol. 2022;12(4):327-680. doi:10.1002/alr.22929
Simopoulos E, Katotomichelakis M, Gouveris H, Tripsianis G, Livaditis M, Danielides V. Olfaction-associated quality of life in chronic rhinosinusitis: adaptation and validation of an olfaction-specific questionnaire. Laryngoscope. 2012;122(7):1450-1454. doi:10.1002/lary.23349
Mattos JL, Schlosser RJ, Mace JC, Smith TL, Soler ZM. Establishing the minimal clinically important difference for the Questionnaire of Olfactory Disorders. Int Forum Allergy Rhinol. 2018;8(9):1041-1046. doi:10.1002/alr.22135
Beswick DM. Impact of sinus surgery on individuals with cystic fibrosis. ClinicalTrials.gov identifier: NCT04469439. May 19, 2022. Accessed August 23, 2023. https://clinicaltrials.gov/study/NCT04469439?term=NCT04469439&rank=1
Chowdhury NI, Mace JC, Bodner TE, et al. Investigating the minimal clinically important difference for SNOT-22 symptom domains in surgically managed chronic rhinosinusitis. Int Forum Allergy Rhinol. 2017;7(12):1149-1155. doi:10.1002/alr.22028
Norman GR, Sloan JA, Wyrwich KW. Interpretation of changes in health-related quality of life: the remarkable universality of half a standard deviation. Med Care. 2003;41(5):582-592. doi:10.1097/01.MLR.0000062554.74615.4C
Wyrwich KW, Nienaber NA, Tierney WM, Wolinsky FD. Linking clinical relevance and statistical significance in evaluating intra-individual changes in health-related quality of life. Med Care. 1999;37(5):469-478. doi:10.1097/00005650-199905000-00006
Samsa G, Edelman D, Rothman ML, Williams GR, Lipscomb J, Matchar D. Determining clinically important differences in health status measures: a general approach with illustration to the Health Utilities Index Mark II. Pharmacoeconomics. 1999;15(2):141-155. doi:10.2165/00019053-199915020-00003
Copay AG, Subach BR, Glassman SD, Polly DW, Schuler TC. Understanding the minimum clinically important difference: a review of concepts and methods. Spine J. 2007;7(5):541-546. doi:10.1016/j.spinee.2007.01.008