TY - JOUR
T1 - Reduced Patient-Reported Outcome Scores Associate With Level of Fibrosis in Patients With Nonalcoholic Steatohepatitis
AU - Younossi, Zobair M.
AU - Stepanova, Maria
AU - Anstee, Quentin M.
AU - Lawitz, Eric J.
AU - Wai-Sun Wong, Vincent
AU - Romero-Gomez, Manuel
AU - Kersey, Kathryn
AU - Li, Georgia
AU - Subramanian, G. Mani
AU - Myers, Robert P.
AU - Djedjos, C. Stephen
AU - Okanoue, Takeshi
AU - Trauner, Michael
AU - Goodman, Zachary
AU - Harrison, Stephen A.
N1 - Funding Information:
Funding Supported by Gilead Sciences (Foster City, CA). Conflicts of interest These authors disclose the following: Z.M.Y. has received research funds or served as consultant to Gilead Sciences, Intercept, NovoNordisk, BMS, Allergan/Tobira, Terns, Viking, AbbVie, Novartis, and Quest Diagnostics. Q.M.A. has received research grant funding from AbbVie, Allergan/Tobira, AstraZenica, GlaxoSmithKline, Novartis Pharma AG, Pfizer Ltd, Vertex; has served as a consultant to Abbott Laboratories, Allergan/Tobira, E3Bio, Eli Lilly & Company Ltd, Galmed, Genfit SA, Gilead, Grunthal, Imperial Innovations, Intercept, Inventiva, Janssen, Kenes, Madrigal, MedImmune, NewGene, NGMBio, Novartis, Novo Nordisk, Pfizer, Raptor Pharma, and Servier; and received lecture fees from Abbott Laboratories, Allergan/Tobira, BMS, Clinical Care Options, Falk, Genfit SA, and Gilead. V.W. has served as a consultant or advisory board member for AbbVie, Allergan, Echosens, Gilead Sciences, Janssen, Perspectum Diagnostics, Pfizer, and Terns and has received lecture fees from Bristol-Myers Squibb, Echosens, Gilead Sciences, and Merck. E.L., M.R.G., and T.O. have received research funds from Gilead Sciences. K.K., G.L., G.M.S., R.P.M., and C.S.D. are employees of Gilead Sciences. M.T. has received speaker honoraria from BMS, Falk Foundation, Gilead, and MSD; advisory board fees from Albireo, Falk Pharma GmbH, Genfit, Gilead, Intercept, MSD, Novartis, and Phenex; has also received travel grants from AbbVie, Falk, Gilead, and Intercept and unrestricted research grants from Albireo, Cymabay, Falk, Gilead, Intercept, MSD, and Takeda. He is also co-inventor of a patent on the medical use of norUDCA. Z.G. has received research funds from Gilead Sciences, Intercept, BMS, Novartis, Galectin, Conatus, and Allergan/Tobira. S.A.H. has received research funds or consulted for Gilead, Intercept, Genfit, HistoIndex, Novartis, Terns, Cirius, Cymabay, Perspectum, Madrigal, NGM Bio, CiVi, Hightide, Akero, Corcept, Innovate, IQVIA, Medpace, Galectin, Metacrine, Consynance, Echosens, Viking, Fibronostics, Pfizer, Conatus, and Galmed. The remaining authors disclose no conflicts.
Publisher Copyright:
© 2019 AGA Institute
PY - 2019/11
Y1 - 2019/11
N2 - Background: Patient-reported outcomes (PROs) are used to measure patients’ experience with their disease. However, there are few PRO data from patients with NASH. We collected data from the STELLAR clinical trials to assess PROs for NASH and advanced fibrosis. Methods: We analyzed data from 1667 patients (58 ± 9 years, 40% male, 52% with cirrhosis, 74% with diabetes) with NASH and bridging fibrosis or compensated cirrhosis (metavir scores, F3 or F4) enrolled in the phase 3 STELLAR trials of selonsertib (NCT03053050 and NCT03053063) who completed PRO questionnaires (SF-36, CLDQ-NASH, EQ-5D, or WPAI:SHP) before treatment initiation. Results: Compared with patients with F3 fibrosis, higher proportions of patients with F4 fibrosis were female, were white, had more hematologic and gastrointestinal comorbidities, and had type 2 diabetes (P ≤ .01). Mean physical health-related PRO scores were significantly lower than those of the general population: patients with F4 fibrosis had score reductions of 4.4% to 12.9% in 6/8 SF-36 domains and patients with F3 fibrosis had score reductions of 3.9% to 11.7% in 4/8 domains (P < .01). Compared to patients with F3 fibrosis, those with F4 fibrosis had lower scores in all but 1 domains of CLDQ-NASH, Role Physical, Bodily Pain, and Social Functioning domains of the SF-36, and EQ-5D (P ≤ 01). In multivariate regression analysis, factors independently associated with lower PRO scores included having cirrhosis, female sex, higher body mass index, history of smoking, and diabetes or other comorbidities (P < .01). Conclusions: PROs are significantly lower in patients with NASH with advanced fibrosis who participated in the STELLAR clinical trials. Treatment of patients with NASH should focus on improving not only clinical outcomes but also quantifiable symptom burden and health-related quality of life.
AB - Background: Patient-reported outcomes (PROs) are used to measure patients’ experience with their disease. However, there are few PRO data from patients with NASH. We collected data from the STELLAR clinical trials to assess PROs for NASH and advanced fibrosis. Methods: We analyzed data from 1667 patients (58 ± 9 years, 40% male, 52% with cirrhosis, 74% with diabetes) with NASH and bridging fibrosis or compensated cirrhosis (metavir scores, F3 or F4) enrolled in the phase 3 STELLAR trials of selonsertib (NCT03053050 and NCT03053063) who completed PRO questionnaires (SF-36, CLDQ-NASH, EQ-5D, or WPAI:SHP) before treatment initiation. Results: Compared with patients with F3 fibrosis, higher proportions of patients with F4 fibrosis were female, were white, had more hematologic and gastrointestinal comorbidities, and had type 2 diabetes (P ≤ .01). Mean physical health-related PRO scores were significantly lower than those of the general population: patients with F4 fibrosis had score reductions of 4.4% to 12.9% in 6/8 SF-36 domains and patients with F3 fibrosis had score reductions of 3.9% to 11.7% in 4/8 domains (P < .01). Compared to patients with F3 fibrosis, those with F4 fibrosis had lower scores in all but 1 domains of CLDQ-NASH, Role Physical, Bodily Pain, and Social Functioning domains of the SF-36, and EQ-5D (P ≤ 01). In multivariate regression analysis, factors independently associated with lower PRO scores included having cirrhosis, female sex, higher body mass index, history of smoking, and diabetes or other comorbidities (P < .01). Conclusions: PROs are significantly lower in patients with NASH with advanced fibrosis who participated in the STELLAR clinical trials. Treatment of patients with NASH should focus on improving not only clinical outcomes but also quantifiable symptom burden and health-related quality of life.
KW - BMI
KW - Emotional
KW - NAFLD
KW - Physical Function
KW - Utilities
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U2 - 10.1016/j.cgh.2019.02.024
DO - 10.1016/j.cgh.2019.02.024
M3 - Article
C2 - 30779990
AN - SCOPUS:85073628167
SN - 1542-3565
VL - 17
SP - 2552-2560.e10
JO - Clinical Gastroenterology and Hepatology
JF - Clinical Gastroenterology and Hepatology
IS - 12
ER -