Randomized trial of interferon β-1a in chronic inflammatory demyelinating polyradiculoneuropathy

Abstract

Objective: To test the safety and efficacy of interferon β-1a (IFN-β) in treatment-resistant chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). Background: Current treatment regimens leave 4% to 30% of patients with CIDP with moderate or severe disability. IFN-β has been reported as beneficial in one treatment-resistant patient. Methods: Ten consecutive treatment-resistant patients were randomized in a double-blind, crossover design to receive placebo or IFN-β (3 MIU for 2 weeks and then 6 MIU for 10 weeks) subcutaneously three times weekly, followed by 4 weeks without treatment, and then the opposite treatment for 12 weeks. The primary outcome measure was “clinically important” improvement by specified amounts in any three of eight clinical measures: timed 10-m walk, Ambulation Index, expanded Medical Research Council sum score, nine-hole peg test time, Functional Independence Measure, Hammersmith Motor Ability, a new Guy’s Neurological Disability Scale, and the EuroQoL quality-of-life scale. These and motor median nerve conduction studies were measured before and after 12 weeks of each treatment. Results: Clinically important improvement was observed in one patient while taking IFN-β and two patients while taking placebo. There was no significant difference between IFN-β and placebo in the change in any of the individual clinical or neurophysiological measures between the beginning and end of treatment. There were no serious adverse events. Conclusion: This trial shows that IFN-β is safe but not efficacious in treatment-resistant CIDP.