Determination of thresholds for minimally important difference and clinically important response on the functional outcomes of sleep questionnaire short version in adults with narcolepsy or obstructive sleep apnea

Terri E Weaver, Diane M Menno, Morgan Bron, Ross D Crosby, Susan Morris, Susan D Mathias, Terri E Weaver, Diane M Menno, Morgan Bron, Ross D Crosby, Susan Morris, Susan D Mathias

Abstract

Purpose: This study estimated thresholds for clinically important responses and minimally important differences for two indicators of improvement for the 10-item version of the functional outcomes of sleep questionnaire (FOSQ-10).

Methods: Participants with excessive daytime sleepiness with narcolepsy or obstructive sleep apnea received 12 weeks of solriamfetol treatment. Participants completed the FOSQ-10 and other patient-reported outcome measures, including the single-item patient global impression of change (PGI-C) assessment. Clinicians completed the single-item clinician global impression of change (CGI-C) for each participant. Data from the two studies were analyzed separately, both without regard to treatment assignment. In total, 690 participants (47% female, mean age 48 years, 77% Caucasian, 91% from North America) were enrolled. Two clinically important changes, defined as a minimally important difference and a clinically important response, were determined using distribution and anchor-based analyses. A receiver operating characteristic analysis was used to determine the optimal FOSQ-10 change threshold.

Results: Spearman correlations between change in FOSQ-10 scores and PGI-C and CGI-C were - 0.57 and - 0.49 for participants with narcolepsy and - 0.42 and - 0.37 for participants with obstructive sleep apnea. Receiver operating characteristic analysis suggested minimally important difference and clinically important response estimates of 1.7 and 2.5 and 1.8 and 2.2 points in narcolepsy and obstructive sleep apnea, respectively.

Conclusions: Minimally important difference and clinically important response estimates for the FOSQ-10 for adults with excessive daytime sleepiness in narcolepsy or obstructive sleep apnea will be helpful for interpreting changes over time and defining a clinical responder. CLINICALTRIALS.

Gov identifiers: NCT02348593 (first submitted January 15, 2015) and NCT02348606 (first submitted January 15, 2015).

Keywords: Clinical significance; Clinically important difference; Hypersomnolence disorders; Quality of life; Sleep-disordered breathing; Treatment outcome.

Conflict of interest statement

TEW has received royalty fees for the use of the FOSQ-10 from Jazz Pharmaceuticals, ResMed, Bayer AD, Cook Medical, Nyxoah, RWS, Merck & Co. Inc., Verily Life Science, WCG MedAvante Prophase, Stratevi, Evidation Health, and Philips Respironics. RDC is a consultant to Health Outcomes Solutions, which received funding from Jazz Pharmaceuticals for conducting these analyses. DMM, MB, and SM are employees of Jazz Pharmaceuticals who, in the course of their employment, have received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals, LLC. SDM is an employee of Health Outcomes Solutions, which received funding from Jazz Pharmaceuticals for conducting these analyses.

© 2021. The Author(s).

Figures

Fig. 1
Fig. 1
Change in FOSQ-10 by PGI-C rating. FOSQ-10, Functional Outcomes of Sleep Questionnaire-10; PGI-C, Patient Global Impression of Change; mITT, modified intent-to-treat; OSA, obstructive sleep apnea
Fig. 2
Fig. 2
ROC curves for PGI-C response = (a) “much improved or better and (b) “minimally improved or better” ROC, receiver operating characteristic; PGI-C, Patient Global Impression of Change; OSA, obstructive sleep apnea
Fig. 3
Fig. 3
ROC curves for CGI-C response = (a) “much improved or better” and (b) “minimally improved or better” ROC, receiver operating characteristic; CGI-C, Clinician Global Impression of Change; OSA, obstructive sleep apnea

References

    1. Fernandez-Mendoza J, Vgontzas AN, Kritikou I, Calhoun SL, Liao D, Bixler EO. Natural history of excessive daytime sleepiness: role of obesity, weight loss, depression, and sleep propensity. Sleep. 2015;38(3):351–360. doi: 10.5665/sleep.4488.
    1. Zhang J, Han F. Sleepiness in narcolepsy. Sleep Med Clin. 2017;12(3):323–330. doi: 10.1016/j.jsmc.2017.03.008.
    1. Young T, Peppard PE, Gottlieb DJ. Epidemiology of obstructive sleep apnea: a population health perspective. Am J Respir Crit Care Med. 2002;165(9):1217–1239. doi: 10.1164/rccm.2109080.
    1. Young T, Palta M, Dempsey J, Skatrud J, Weber S, Badr S. The occurrence of sleep-disordered breathing among middle-aged adults. N Engl J Med. 1993;328(17):1230–1235. doi: 10.1056/NEJM199304293281704.
    1. Fu Y, Xu H, Xia Y, Qian Y, Li X, Zou J, Wang Y, Meng L, Tang X, Zhu H, Zhou H, Su K, Yu D, Yi H, Guan J, Yin S. Excessive daytime sleepiness and metabolic syndrome in men with obstructive sleep apnea: a large cross-sectional study. Oncotarget. 2017;8(45):79693–79702. doi: 10.18632/oncotarget.19113.
    1. Gasa M, Tamisier R, Launois SH, Sapene M, Martin F, Stach B, Grillet Y, Levy P, Pepin JL, Scientific Council of the Sleep Registry of the French Federation of Pneumology-FFP Residual sleepiness in sleep apnea patients treated by continuous positive airway pressure. J Sleep Res. 2013;22(4):389–397. doi: 10.1111/jsr.12039.
    1. Dean B, Aguilar D, Shapiro C, Orr WC, Isserman JA, Calimlim B, Rippon GA. Impaired health status, daily functioning, and work productivity in adults with excessive sleepiness. J Occup Environ Med. 2010;52(2):144–149. doi: 10.1097/JOM.0b013e3181c99505.
    1. Bioulac S, Micoulaud-Franchi JA, Arnaud M, Sagaspe P, Moore N, Salvo F, Philip P (2017) Risk of motor vehicle accidents related to sleepiness at the wheel: a systematic review and meta-analysis. Sleep 40(10). 10.1093/sleep/zsx134
    1. Weaver TE, Laizner AM, Evans LK, Maislin G, Chugh DK, Lyon K, Smith PL, Schwartz AR, Redline S, Pack AI, Dinges DF. An instrument to measure functional status outcomes for disorders of excessive sleepiness. Sleep. 1997;20(10):835–843.
    1. Chasens ER, Ratcliffe SJ, Weaver TE. Development of the FOSQ-10: a short version of the functional outcomes of sleep questionnaire. Sleep. 2009;32(7):915–919. doi: 10.1093/sleep/32.7.915.
    1. Brozek JL, Guyatt GH, Schunemann HJ. How a well-grounded minimal important difference can enhance transparency of labelling claims and improve interpretation of a patient reported outcome measure. Health Qual Life Outcomes. 2006;4:69. doi: 10.1186/1477-7525-4-69.
    1. Schunemann HJ, Akl EA, Guyatt GH. Interpreting the results of patient reported outcome measures in clinical trials: the clinician's perspective. Health Qual Life Outcomes. 2006;4:62. doi: 10.1186/1477-7525-4-62.
    1. Coon CD, Cappelleri JC. Interpreting change in scores on patient-reported outcome instruments. Ther Innov Regul Sci. 2016;50(1):22–29. doi: 10.1177/2168479015622667.
    1. Thorpy MJ, Shapiro C, Mayer G, Corser BC, Emsellem H, Plazzi G, Chen D, Carter LP, Wang H, Lu Y, Black J, Dauvilliers Y. A randomized study of solriamfetol for excessive sleepiness in narcolepsy. Ann Neurol. 2019;85(3):359–370. doi: 10.1002/ana.25423.
    1. Schweitzer PK, Rosenberg R, Zammit GK, Gotfried M, Chen D, Carter LP, Wang H, Lu Y, Black J, Malhotra A, Strohl KP. Solriamfetol for excessive sleepiness in obstructive sleep apnea (TONES 3): a randomized controlled trial. Am J Respir Crit Care Med. 2019;199(11):1421–1431. doi: 10.1164/rccm.201806-1100OC.
    1. Mitler MM, Gujavarty KS, Browman CP. Maintenance of wakefulness test: a polysomnographic technique for evaluation treatment efficacy in patients with excessive somnolence. Electroencephalogr Clin Neurophysiol. 1982;53(6):658–661. doi: 10.1016/0013-4694(82)90142-0.
    1. Johns MW. A new method for measuring daytime sleepiness: the Epworth sleepiness scale. Sleep. 1991;14(6):540–545. doi: 10.1093/sleep/14.6.540.
    1. Cohen J (1988) Statistical power analysis for the behavioral sciences, 2nd edn. Lawrence Erlbaum, Hillsdale, NJ
    1. Guyatt GH, Bombardier C, Tugwell PX. Measuring disease-specific quality of life in clinical trials. CMAJ. 1986;134(8):889–895.
    1. Scrima L, Emsellem HA, Becker PM, Ruoff C, Lankford A, Bream G, Khayrallah M, Lu Y, Black J. Identifying clinically important difference on the Epworth sleepiness scale: results from a narcolepsy clinical trial of JZP-110. Sleep Med. 2017;38:108–112. doi: 10.1016/j.sleep.2017.07.006.
    1. Steffen AD, Lai C, Weaver TE. Criteria for gauging response to sodium oxybate for narcolepsy. J Sleep Res. 2018;27(4):e12628. doi: 10.1111/jsr.12628.
    1. Crosby RD, Kolotkin RL, Williams GR. Defining clinically meaningful change in health-related quality of life. J Clin Epidemiol. 2003;56(5):395–407. doi: 10.1016/S0895-4356(03)00044-1.
    1. Lydick E, Epstein RS. Interpretation of quality of life changes. Qual Life Res. 1993;2(3):221–226. doi: 10.1007/BF00435226.
    1. Hanley JA. Receiver operating characteristic (ROC) methodology: the state of the art. Crit Rev Diagn Imaging. 1989;29(3):307–335.
    1. Patrick DL, Gagnon DD, Zagari MJ, Mathijs R, Sweetenham J. Assessing the clinical significance of health-related quality of life (HrQOL) improvements in anaemic cancer patients receiving epoetin alfa. Eur J Cancer. 2003;39(3):335–345. doi: 10.1016/s0959-8049(02)00628-7.
    1. 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.
    1. Wyrwich KW, Tierney WM, Wolinsky FD. Further evidence supporting an SEM-based criterion for identifying meaningful intra-individual changes in health-related quality of life. J Clin Epidemiol. 1999;52(9):861–873. doi: 10.1016/S0895-4356(99)00071-2.
    1. Jowett SL, Seal CJ, Barton JR, Welfare MR. The short inflammatory bowel disease questionnaire is reliable and responsive to clinically important change in ulcerative colitis. Am J Gastroenterol. 2001;96(10):2921–2928. doi: 10.1111/j.1572-0241.2001.04682.x.
    1. Kosinski M, Zhao SZ, Dedhiya S, Osterhaus JT, Ware JE., Jr Determining minimally important changes in generic and disease-specific health-related quality of life questionnaires in clinical trials of rheumatoid arthritis. Arthritis Rheum. 2000;43(7):1478–1487. doi: 10.1002/1529-0131(200007)43:7<1478::AID-ANR10>;2-M.
    1. Wyrwich KW, Tierney WM, Wolinsky FD. Using the standard error of measurement to identify important changes on the asthma quality of life questionnaire. Qual Life Res. 2002;11(1):1–7. doi: 10.1023/A:1014485627744.
    1. Weaver TE, Mathias SD, Crosby RD, Bron M, Bujanover S, Menno D, Villa KF, Drake C (2020) Relationship between sleep efficacy endpoints and measures of functional status and health-related quality of life in participants with narcolepsy or obstructive sleep apnea treated for excessive daytime sleepiness. J Sleep Res:e13210. 10.1111/jsr.13210

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