TY - JOUR
T1 - Assessing the impact of sodium oxybate treatment on functioning, productivity, and health-related quality of life in patients with narcolepsy
T2 - findings from the Nexus Narcolepsy Registry (waves 1–4)
AU - Thorpy, Michael J.
AU - Ohayon, Maurice M.
AU - Carls, Ginger
AU - Black, Jed
AU - Pasta, David J.
AU - Hyman, Danielle L.
AU - Villa, Kathleen F.
N1 - Funding Information:
This study was supported by Jazz Pharmaceuticals.The authors acknowledge and thank Invitae Corporation and the Invitae CONNNECT PIN program as the source of this data. Under the direction of the authors, Lauren Fink, PhD of MedErgy (Yardley, PA, USA) and employees of Peloton Advantage, LLC, an OPEN Health company, provided medical writing support, which was funded by Jazz Pharmaceuticals in accordance with Good Publication Practice (GPP3) guidelines (http://www.ismpp.org/gpp3). Although Jazz Pharmaceuticals reviewed the content of this manuscript, the ultimate interpretation and the decision to submit it for publication was made by the authors independently.MJT has received research/grant support and consultancy fees from Jazz Pharmaceuticals, Merck & Co, Inc., Balance Therapeutics, Axsome, Avadel, and Harmony Biosciences. MMO has received consulting fees from Jazz Pharmaceuticals; participated in advisory boards for Jazz Pharmaceuticals and Pfizer; and has received research grants from Jazz Pharmaceuticals and Sanofi. GC and KFV are former employees of Jazz Pharmaceuticals who, in the course of this employment, have received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals plc; GC is currently a consultant to Jazz Pharmaceuticals. JB is a part-time employee of Jazz Pharmaceuticals and a shareholder of Jazz Pharmaceuticals plc. DJP is a former employee of ICON Clinical Research, which received funding from Jazz to manage the Nexus Narcolepsy Registry and conduct this analysis. DLH is a full-time employee of Jazz Pharmaceuticals who, in the course of this employment, has received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals plc.
Funding Information:
MJT has received research/grant support and consultancy fees from Jazz Pharmaceuticals , Merck & Co, Inc. , Balance Therapeutics , Axsome , Avadel , and Harmony Biosciences . MMO has received consulting fees from Jazz Pharmaceuticals ; participated in advisory boards for Jazz Pharmaceuticals and Pfizer; and has received research grants from Jazz Pharmaceuticals and Sanofi . GC and KFV are former employees of Jazz Pharmaceuticals who, in the course of this employment, have received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals plc; GC is currently a consultant to Jazz Pharmaceuticals. JB is a part-time employee of Jazz Pharmaceuticals and a shareholder of Jazz Pharmaceuticals plc. DJP is a former employee of ICON Clinical Research, which received funding from Jazz to manage the Nexus Narcolepsy Registry and conduct this analysis. DLH is a full-time employee of Jazz Pharmaceuticals who, in the course of this employment, has received stock options exercisable for, and other stock awards of, ordinary shares of Jazz Pharmaceuticals plc.
Publisher Copyright:
© 2021 The Authors
PY - 2021/8
Y1 - 2021/8
N2 - Background: The aim of this study was to evaluate the impact of different therapy regimens, including sodium oxybate (SXB)-containing regimens, on patient-reported outcomes (PROs) in people with narcolepsy. Methods: Online surveys were used to collect information from persons with narcolepsy in the Nexus Narcolepsy Registry. Surveys contained questionnaires assessing self-reported sleep quality (SQ; via single question), daytime sleepiness and function (Epworth Sleepiness Scale and Functional Outcomes of Sleep Questionnaire), health-related quality of life (HRQoL; 36-Item Short Form Health Survey [SF-36]), work productivity and impairment (Work Productivity and Activity Impairment: Specific Health Problem), and history of injuries or motor vehicle accidents. Treatment with SXB (including monotherapy or combination therapy; SXB group) was compared with non-SXB therapy (No SXB group). The P values presented are nominal, as there are no adjustments for multiplicity. Results: From June 2015 through December 2017, 983 participants completed 1760 surveys. SQ and daytime functioning scores were better in the SXB group compared with the No SXB group (all P < 0.001). HRQoL scores were better for the SXB group compared with the No SXB group for the SF-36 Physical Component (P = 0.016), Mental Component (P < 0.001), and all 8 subscales. Additionally, PROs were better for the SXB group for presenteeism, overall work and activity impairment, and risk of motor vehicle accidents (all P ≤ 0.001). Conclusion: Based on participants’ self-assessments, treatment regimens with SXB were associated with better outcomes than regimens not containing SXB across many PROs, including SQ, HRQoL, work and activities, and risk of traffic accidents. Clinicaltrials.gov identifier: NCT02769780.
AB - Background: The aim of this study was to evaluate the impact of different therapy regimens, including sodium oxybate (SXB)-containing regimens, on patient-reported outcomes (PROs) in people with narcolepsy. Methods: Online surveys were used to collect information from persons with narcolepsy in the Nexus Narcolepsy Registry. Surveys contained questionnaires assessing self-reported sleep quality (SQ; via single question), daytime sleepiness and function (Epworth Sleepiness Scale and Functional Outcomes of Sleep Questionnaire), health-related quality of life (HRQoL; 36-Item Short Form Health Survey [SF-36]), work productivity and impairment (Work Productivity and Activity Impairment: Specific Health Problem), and history of injuries or motor vehicle accidents. Treatment with SXB (including monotherapy or combination therapy; SXB group) was compared with non-SXB therapy (No SXB group). The P values presented are nominal, as there are no adjustments for multiplicity. Results: From June 2015 through December 2017, 983 participants completed 1760 surveys. SQ and daytime functioning scores were better in the SXB group compared with the No SXB group (all P < 0.001). HRQoL scores were better for the SXB group compared with the No SXB group for the SF-36 Physical Component (P = 0.016), Mental Component (P < 0.001), and all 8 subscales. Additionally, PROs were better for the SXB group for presenteeism, overall work and activity impairment, and risk of motor vehicle accidents (all P ≤ 0.001). Conclusion: Based on participants’ self-assessments, treatment regimens with SXB were associated with better outcomes than regimens not containing SXB across many PROs, including SQ, HRQoL, work and activities, and risk of traffic accidents. Clinicaltrials.gov identifier: NCT02769780.
KW - Excessive daytime sleepiness
KW - Health-related quality of life
KW - Motor vehicle accidents
KW - Narcolepsy
KW - Sodium oxybate
KW - Work productivity
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U2 - 10.1016/j.sleep.2021.06.010
DO - 10.1016/j.sleep.2021.06.010
M3 - Article
C2 - 34247126
AN - SCOPUS:85109449148
VL - 84
SP - 380
EP - 388
JO - Sleep Medicine
JF - Sleep Medicine
SN - 1389-9457
ER -