A Randomized, Placebo-Controlled Clinical Trial of Efficacy and Safety: Modafinil in the Treatment of Fatigue in Patients With Primary Biliary Cirrhosis. (March 2017)

• Record Type: Journal Article
• Authors: Silveira, Marina G.; Gossard, Andrea A.; Stahler, Alisha C.; Jorgensen, Roberta A.; Petz, Janice L.; Ali, Ahmad H.; Lindor, Keith D.
• Is Part Of: American journal of therapeutics. Volume 24:Number 2(2017)
• Publication Date: 2017-03
• Subjects: primary biliary cirrhosis -- fatigue -- ursodeoxycholic acid -- liver transplantation; Chemotherapy -- Periodicals; Pharmacology -- Periodicals; 615.58
• DOI: 10.1097/MJT.0000000000000387
• Languages: English
• ISSNs: 1075-2765

Abstract

Abstract : Background and Aims: Fatigue is a common symptom of primary biliary cirrhosis (PBC), and is associated with an impaired quality of life. Study Question: No studies have assessed the use of modafinil in fatigue related to PBC in a controlled manner. Study Design, Measures, and Outcomes: A randomized, double-blind, placebo-controlled study was conducted to determine the safety and efficacy of modafinil for the treatment of fatigue in PBC. Forty patients were randomized to modafinil (n = 20) or placebo (n = 20) for 12 weeks. A verbal report of fatigue for at least 6 months was required for enrollment. Modafinil was administered at 100 mg by mouth once daily; a change by 50 mg every 2 weeks (maximum: 200 mg once daily) was allowed, depending on the subject's response to treatment. The primary outcome was defined as a ≥50% improvement in fatigue severity [quantified by the Fisk Fatigue Impact Scale (FFIS)] after 12 weeks of treatment, compared with baseline values. Results: Thirty-three PBC patients completed the study. After 12 weeks of therapy, only 5 patients had a ≥50% reduction in FFIS scores: 3 patients (17.6%) in the modafinil arm and 2 (12.5%) in the placebo arm ( P = 1.00). Change in median FFIS score was not statistically different between patients in the 2 treatment groups ( P = 0.36). Modafinil was associated with minimal adverse events (headaches, diarrhea, and rash). Conclusions: In patients with PBC who have fatigue, treatment with modafinil for 12 weeks was safe and fairly well tolerated; however, it did not result in beneficial effects on fatigue compared with patients treated with placebo (CONSORT Table 1).ClinicalTrials.gov identifier NCT00943176. Abstract : Supplemental Digital Content is Available in the Text.