PMID- 38145393
OWN - NLM
STAT- Publisher
LR  - 20231225
IS  - 2042-6984 (Electronic)
IS  - 2042-6976 (Linking)
DP  - 2023 Dec 25
TI  - 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.
LID - 10.1002/alr.23312 [doi]
AB  - 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, (1/2) 
      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.
CI  - (c) 2023 ARS-AAOA, LLC.
FAU - Miller, Jessa E
AU  - Miller JE
AUID- ORCID: 0000-0001-9769-187X
AD  - Department of Otolaryngology-Head and Neck Surgery, University of California, Los 
      Angeles, California, USA.
FAU - Taylor-Cousar, Jennifer L
AU  - Taylor-Cousar JL
AD  - Department of Medicine, National Jewish Health, Denver, Colorado, USA.
AD  - Department of Pediatrics, National Jewish Health, Denver, Colorado, USA.
FAU - Overdevest, Jonathan B
AU  - Overdevest JB
AUID- ORCID: 0000-0002-1152-2512
AD  - Department of Otolaryngology-Head and Neck Surgery, Columbia University, New 
      York, New York, USA.
FAU - Khatiwada, Aastha
AU  - Khatiwada A
AD  - Department of Biostatistics, National Jewish Health, Denver, Colorado, USA.
FAU - Mace, Jess C
AU  - Mace JC
AD  - Department of Otolaryngology-Head and Neck Surgery, Oregon Health Sciences 
      University, Portland, Oregon, USA.
FAU - Alt, Jeremiah A
AU  - Alt JA
AUID- ORCID: 0000-0003-0560-5028
AD  - Department of Otolaryngology-Head and Neck Surgery, University of Utah, Salt Lake 
      City, Utah, USA.
FAU - Bodner, Todd E
AU  - Bodner TE
AD  - Department of Psychology, Portland State University, Portland, Oregon, USA.
FAU - Chowdhury, Naweed I
AU  - Chowdhury NI
AD  - Department of Otolaryngology-Head and Neck Surgery, Vanderbilt Health, Nashville, 
      Tennessee, USA.
FAU - DiMango, Emily A
AU  - DiMango EA
AD  - Department of Medicine, Columbia University, New York, New York, USA.
FAU - Eshaghian, Patricia H
AU  - Eshaghian PH
AD  - Department of Pulmonary Medicine, University of California, Los Angeles, 
      California, USA.
FAU - Getz, Anne E
AU  - Getz AE
AD  - Department of Otolaryngology-Head and Neck Surgery, University of Colorado, 
      Aurora, Colorado, USA.
FAU - Gudis, David A
AU  - Gudis DA
AUID- ORCID: 0000-0002-1938-9349
AD  - Department of Otolaryngology-Head and Neck Surgery, Columbia University, New 
      York, New York, USA.
FAU - Han, Ethan J
AU  - Han EJ
AUID- ORCID: 0000-0002-0981-4858
AD  - Department of Otolaryngology-Head and Neck Surgery, University of California, Los 
      Angeles, California, USA.
FAU - Hwang, Peter H
AU  - Hwang PH
AUID- ORCID: 0000-0002-0786-4675
AD  - Department of Otolaryngology-Head and Neck Surgery, Stanford Medicine, Palo Alto, 
      California, USA.
FAU - Keating, Claire L
AU  - Keating CL
AD  - Department of Medicine, Columbia University, New York, New York, USA.
FAU - Khanwalkar, Ashoke
AU  - Khanwalkar A
AUID- ORCID: 0000-0001-9960-7758
AD  - Department of Otolaryngology-Head and Neck Surgery, University of Colorado, 
      Aurora, Colorado, USA.
FAU - Kimple, Adam J
AU  - Kimple AJ
AD  - Department of Otolaryngology-Head and Neck Surgery, University of North Carolina, 
      Chapel Hill, North Carolina, USA.
FAU - Lee, Jivianne T
AU  - Lee JT
AD  - Department of Otolaryngology-Head and Neck Surgery, University of California, Los 
      Angeles, California, USA.
FAU - Li, Douglas
AU  - Li D
AD  - Department of Pulmonary Medicine, University of California, Los Angeles, 
      California, USA.
FAU - Markarian, Karolin
AU  - Markarian K
AUID- ORCID: 0009-0002-0076-3166
AD  - David Geffen School of Medicine, University of California, CTSI, Los Angeles, 
      California, USA.
FAU - Norris, Meghan
AU  - Norris M
AD  - Department of Otolaryngology-Head and Neck Surgery, University of North Carolina, 
      Chapel Hill, North Carolina, USA.
FAU - Nayak, Jayakar V
AU  - Nayak JV
AUID- ORCID: 0000-0003-4454-0861
AD  - Department of Otolaryngology-Head and Neck Surgery, Stanford Medicine, Palo Alto, 
      California, USA.
FAU - Owens, Cameran
AU  - Owens C
AD  - Department of Otolaryngology-Head and Neck Surgery, University of Colorado, 
      Aurora, Colorado, USA.
FAU - Patel, Zara M
AU  - Patel ZM
AUID- ORCID: 0000-0003-2072-982X
AD  - Department of Otolaryngology-Head and Neck Surgery, Stanford Medicine, Palo Alto, 
      California, USA.
FAU - Poch, Katie
AU  - Poch K
AD  - Department of Medicine, National Jewish Health, Denver, Colorado, USA.
FAU - Schlosser, Rodney J
AU  - Schlosser RJ
AD  - Department of Otolaryngology-Head and Neck Surgery, Medical University of South 
      Carolina, Charleston, South Carolina, USA.
FAU - Smith, Kristine A
AU  - Smith KA
AD  - Department of Otolaryngology-Head and Neck Surgery, University of Utah, Salt Lake 
      City, Utah, USA.
FAU - Smith, Timothy L
AU  - Smith TL
AUID- ORCID: 0000-0002-6424-7083
AD  - Department of Otolaryngology-Head and Neck Surgery, Oregon Health Sciences 
      University, Portland, Oregon, USA.
FAU - Soler, Zachary M
AU  - Soler ZM
AD  - Department of Otolaryngology-Head and Neck Surgery, Medical University of South 
      Carolina, Charleston, South Carolina, USA.
FAU - Suh, Jeffrey D
AU  - Suh JD
AD  - Department of Otolaryngology-Head and Neck Surgery, University of California, Los 
      Angeles, California, USA.
FAU - Tervo, Jeremy P
AU  - Tervo JP
AUID- ORCID: 0000-0002-8883-8566
AD  - Vagelos College of Physicians & Surgeons, Columbia University, New York, New 
      York, USA.
FAU - Turner, Grant A
AU  - Turner GA
AD  - Department of Pulmonary Medicine, University of California, Los Angeles, 
      California, USA.
FAU - Wang, Marilene B
AU  - Wang MB
AD  - Department of Otolaryngology-Head and Neck Surgery, University of California, Los 
      Angeles, California, USA.
FAU - Saavedra, Milene T
AU  - Saavedra MT
AD  - Department of Medicine, National Jewish Health, Denver, Colorado, USA.
FAU - Beswick, Daniel M
AU  - Beswick DM
AUID- ORCID: 0000-0001-8612-5442
AD  - Department of Otolaryngology-Head and Neck Surgery, University of California, Los 
      Angeles, California, USA.
LA  - eng
GR  - BESWIC20A0/Cystic Fibrosis Foundation/
GR  - TAYLOR19A0/Cystic Fibrosis Foundation/
GR  - BESWIC22Y5/Cystic Fibrosis Foundation/
GR  - Marshall and Margherite McComb Foundation/
PT  - Journal Article
DEP - 20231225
PL  - United States
TA  - Int Forum Allergy Rhinol
JT  - International forum of allergy & rhinology
JID - 101550261
SB  - IM
OTO - NOTNLM
OT  - chronic rhinosinusitis
OT  - cystic fibrosis
OT  - highly effective modulator therapy
OT  - olfaction
OT  - olfactory disorders
OT  - olfactory dysfunction
OT  - olfactory test
OT  - patient-reported outcome measure
OT  - quality of life
EDAT- 2023/12/25 12:42
MHDA- 2023/12/25 12:42
CRDT- 2023/12/25 08:53
PHST- 2023/11/21 00:00 [revised]
PHST- 2023/08/27 00:00 [received]
PHST- 2023/12/05 00:00 [accepted]
PHST- 2023/12/25 12:42 [medline]
PHST- 2023/12/25 12:42 [pubmed]
PHST- 2023/12/25 08:53 [entrez]
AID - 10.1002/alr.23312 [doi]
PST - aheadofprint
SO  - Int Forum Allergy Rhinol. 2023 Dec 25. doi: 10.1002/alr.23312.