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

Jessa E Miller¹, Jennifer L Taylor-Cousar^{2

3}, Jonathan B Overdevest⁴, Aastha Khatiwada⁵, Jess C Mace⁶, Jeremiah A Alt⁷, Todd E Bodner⁸, Naweed I Chowdhury⁹, Emily A DiMango¹⁰, Patricia H Eshaghian¹¹, Anne E Getz¹², David A Gudis⁴, Ethan J Han¹, Peter H Hwang¹³, Claire L Keating¹⁰, Ashoke Khanwalkar¹², Adam J Kimple¹⁴, Jivianne T Lee¹, Douglas Li¹¹, Karolin Markarian¹⁵, Meghan Norris¹⁴, Jayakar V Nayak¹³, Cameran Owens¹², Zara M Patel¹³, Katie Poch², Rodney J Schlosser¹⁶, Kristine A Smith⁷, Timothy L Smith⁶, Zachary M Soler¹⁶, Jeffrey D Suh¹, Jeremy P Tervo¹⁷, Grant A Turner¹¹, Marilene B Wang¹, Milene T Saavedra², Daniel M Beswick¹

Affiliations

¹ Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, California, USA.
² Department of Medicine, National Jewish Health, Denver, Colorado, USA.
³ Department of Pediatrics, National Jewish Health, Denver, Colorado, USA.
⁴ Department of Otolaryngology-Head and Neck Surgery, Columbia University, New York, New York, USA.
⁵ Department of Biostatistics, National Jewish Health, Denver, Colorado, USA.
⁶ Department of Otolaryngology-Head and Neck Surgery, Oregon Health Sciences University, Portland, Oregon, USA.
⁷ Department of Otolaryngology-Head and Neck Surgery, University of Utah, Salt Lake City, Utah, USA.
⁸ Department of Psychology, Portland State University, Portland, Oregon, USA.
⁹ Department of Otolaryngology-Head and Neck Surgery, Vanderbilt Health, Nashville, Tennessee, USA.
¹⁰ Department of Medicine, Columbia University, New York, New York, USA.
¹¹ Department of Pulmonary Medicine, University of California, Los Angeles, California, USA.
¹² Department of Otolaryngology-Head and Neck Surgery, University of Colorado, Aurora, Colorado, USA.
¹³ Department of Otolaryngology-Head and Neck Surgery, Stanford Medicine, Palo Alto, California, USA.
¹⁴ Department of Otolaryngology-Head and Neck Surgery, University of North Carolina, Chapel Hill, North Carolina, USA.
¹⁵ David Geffen School of Medicine, University of California, CTSI, Los Angeles, California, USA.
¹⁶ Department of Otolaryngology-Head and Neck Surgery, Medical University of South Carolina, Charleston, South Carolina, USA.
¹⁷ Vagelos College of Physicians & Surgeons, Columbia University, New York, New York, USA.

PMID: 38145393
DOI: 10.1002/alr.23312

Observational Study

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

Jessa E Miller et al. Int Forum Allergy Rhinol. 2024 Jun.

. 2024 Jun;14(6):1079-1087.

doi: 10.1002/alr.23312. Epub 2023 Dec 25.

Authors

Affiliations

¹ Department of Otolaryngology-Head and Neck Surgery, University of California, Los Angeles, California, USA.
² Department of Medicine, National Jewish Health, Denver, Colorado, USA.
³ Department of Pediatrics, National Jewish Health, Denver, Colorado, USA.
⁴ Department of Otolaryngology-Head and Neck Surgery, Columbia University, New York, New York, USA.
⁵ Department of Biostatistics, National Jewish Health, Denver, Colorado, USA.
⁶ Department of Otolaryngology-Head and Neck Surgery, Oregon Health Sciences University, Portland, Oregon, USA.
⁷ Department of Otolaryngology-Head and Neck Surgery, University of Utah, Salt Lake City, Utah, USA.
⁸ Department of Psychology, Portland State University, Portland, Oregon, USA.
⁹ Department of Otolaryngology-Head and Neck Surgery, Vanderbilt Health, Nashville, Tennessee, USA.
¹⁰ Department of Medicine, Columbia University, New York, New York, USA.
¹¹ Department of Pulmonary Medicine, University of California, Los Angeles, California, USA.
¹² Department of Otolaryngology-Head and Neck Surgery, University of Colorado, Aurora, Colorado, USA.
¹³ Department of Otolaryngology-Head and Neck Surgery, Stanford Medicine, Palo Alto, California, USA.
¹⁴ Department of Otolaryngology-Head and Neck Surgery, University of North Carolina, Chapel Hill, North Carolina, USA.
¹⁵ David Geffen School of Medicine, University of California, CTSI, Los Angeles, California, USA.
¹⁶ Department of Otolaryngology-Head and Neck Surgery, Medical University of South Carolina, Charleston, South Carolina, USA.
¹⁷ Vagelos College of Physicians & Surgeons, Columbia University, New York, New York, USA.

PMID: 38145393
DOI: 10.1002/alr.23312

Abstract

Introduction: 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.

Methods: 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.

Results: 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).

Conclusion: 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.

Keywords: chronic rhinosinusitis; cystic fibrosis; highly effective modulator therapy; olfaction; olfactory disorders; olfactory dysfunction; olfactory test; patient‐reported outcome measure; quality of life.

PubMed Disclaimer

References

REFERENCES

1. 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
1. 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
1. 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
1. 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
1. 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

Publication types

Actions

MeSH terms

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Substances

Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions
Actions

Grants and funding

LinkOut - more resources

Full Text Sources
- Ovid Technologies, Inc.
- Wiley
Medical
- Genetic Alliance
- MedlinePlus Health Information

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

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

Affiliations

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

Authors

Affiliations

Abstract

References

REFERENCES

Publication types

MeSH terms

Substances

Grants and funding

LinkOut - more resources

Full Text Sources

Medical