Rituximab has demonstrated utility in treating other autoimmune diseases, and this phase 2, double-blind, randomized, controlled trial compared rituximab with placebo in 57 patients with primary biliary cholangitis (PBC).
A study was recently published in Efficacy and Mechanism Evaluation that investigated the potential clinical benefit of rituximab for the treatment of fatigue in primary biliary cholangitis (PBC), an autoimmune liver disease that is characterized by the loss of the intrahepatic bile ducts accompanied by progressive cholestasis. About half of patients with PBC experience fatigue.
Rituximab has demonstrated utility in treating other autoimmune diseases, and this phase 2, double-blind, randomized, controlled trial compared rituximab with placebo in 57 patients with PBC aged 18 years or older with moderate to severe fatigue as measured by a score of greater than 33 on the PBC-40 fatigue domain scale. The trial took place at a single center in the United Kingdom in Newcastle upon Tyne Hospitals National Health Service Foundation Trust.
Patients were randomized in to receive either rituximab or a placebo. Participants received 2 transfusions of rituximab (1000 mg) or placebo on days 1 and 15 of the trial and were followed up at 3-month intervals for a year following treatment.
The primary outcome measure was the PBC-40 fatigue domain evaluation at 3 months, which was assessed on an intention-to-treat basis. Although there were no serious adverse events linked to the drug, there was also no statistically significant difference in fatigue score measured at 3 months between the rituximab and placebo arms (adjusted mean difference —0.9; 95% CI, –4.6-3.1). However, the anaerobic threshold improved significantly in the rituximab arm with an adjusted mean difference at 3 months of 1.41 (95% CI, 0.03-2.80).
Overall, researchers found that rituximab is ineffective for the treatment of fatigue in patients with PBC, despite an increase in anaerobic threshold.
Khanna A, Jopson L, Howel D, et al. Rituximab for the treatment of fatigue in primary biliary cholangitis (formerly primary biliary cirrhosis): a randomized controlled trial. [Published online April 2018]. EME. PMID: 29733563.