The top clinical trial opportunities in therapeutic apheresis and neurology.

OBJECTIVE

The National Heart, Lung, and Blood Institute, of The National Institutes of Health, convened the 2012 State-of-the-Science Symposium in therapeutic apheresis (TA) with the goals of identifying and prioritizing future research concept proposals to optimize the use of TA over the next decade.

METHODS

Six subcommittees, including neurology, were formed based on organ system, pathophysiology, and technology/special considerations. The subcommittees consisted of physicians, clinical subject matter experts, and basic scientists. Each subcommittee developed concept proposals that were presented, evaluated, and prioritized based on scientific importance, clinical significance, and feasibility.

RESULTS

The neurology subcommittee developed eight concept proposals. The proposals include therapeutic plasma exchange (TPE) in neuromyelitis optica; TPE versus intravenous immunoglobulin (IVIG) in anti-muscle specific kinase associated myasthenia gravis, severe acute disseminated encephalomyelitis, and anti-NMDA encephalitis; extracorporeal photopheresis in relapsing remitting multiple sclerosis and polymyositis; fibrinogen/low-density lipoprotein apheresis in idiopathic sudden sensorineural hearing loss; and creation of a rare neurologic disease registry and biorepository.

CONCLUSIONS

Key clinical research priorities to evaluate and optimize the use of TA on selected neurologic disorders exist. The research opportunities if addressed would provide evidence-based data to inform the care of patients with these selected neurologic diseases.