Therapeutic plasma exchange for neuromyelitis optica spectrum disorder: A multicenter retrospective study by the ASFA neurologic diseases subcommittee.

IMPORTANCE: Neuromyelitis optica/neuromyelitis optica spectrum disorder patients' response to therapeutic plasma exchange (TPE) is currently incompletely characterized. OBJECTIVE: Our study aims to understand the clinical status improvement of neuromyelitis optica/neuromyelitis optica spectrum disorder patients treated with TPE. DESIGN, SETTING, AND PARTICIPANTS: This is a multicenter retrospective study conducted between 1 January 2003 and 31 July 2017 at 13 US hospitals performing apheresis procedures. Subjects studied were diagnosed with neuromyelitis optica/neuromyelitis optica spectrum disorder who received TPE during presentation with acute disease. MAIN OUTCOMES AND MEASURES: The primary outcome was clinical status improvement in patients treated with TPE. Secondary measures were procedural and patient characteristics associated with response to treatment. RESULTS: We evaluated 114 patients from 13 institutions. There was a female predilection. The largest ethnic group affected was non-Hispanic Caucasian. The average age of diagnosis was 43.1 years. The average time to diagnosis was 3.1 years. On average, five procedures were performed during each treatment series. The most commonly performed plasma volume exchange was 1.0 to 1.25 using 5% albumin as replacement fluid. Most patients (52%) did not require an additional course of TPE and noted "mild" to "moderate" clinical status improvement. Maximal symptom improvement appeared by the fourth or fifth TPE treatment. CONCLUSION AND RELEVANCE: TPE improved the clinical status of patients. Adults responded more favorably than children. Procedural characteristics, including number of TPEs, plasma volume exchanged, and replacement fluid used, were similar between institutions. TPE was well-tolerated and had a low severe adverse event profile.

Therapeutic plasma exchange in neuromyelitis optica: a case series.

Neuromyelitis optica (NMO) is a relapsing inflammatory disease of the central nervous system that predominantly affects the spinal cord and optic nerves. The clinical hallmark of the disease is a step-wise deterioration of visual and spinal cord function. This study reviews patients with steroid resistant relapsing NMO presenting for therapeutic plasma exchange (TPE) at our institution from December 2005 to December 2012. A total of five patients were treated with single volume TPE. Both subjective and objective clinical response to TPE was estimated by three different sources (the patient, a Transfusion Medicine physician, and the treating Neurologist) with the patient and Transfusion Medicine physician's final assessment of response made at the time of the last TPE in the series and the treating neurologist's assessment of response made at the time of the next neurological exam after the last TPE. A total of 17 TPE series were performed with the average course of therapy being three series (ranged 1-5) with five TPE (ranged 3-7) per series. All patients demonstrated improvement with each series of TPE and all procedures were well tolerated with only transient and well-described reactions all of which were successfully resolved with minor or no sequelae.

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.

Therapeutic plasma exchange for fulminant hepatic failure secondary to Wilson's disease.

Wilson's disease (WD) is an autosomal-recessive disorder of impaired copper metabolism resulting in accumulation of copper primarily in the liver but ultimately in many organs and tissues. A small number of patients with WD initially present with fulminant hepatic failure (FHF), hypercupremia, and intravascular hemolysis. The therapeutic goals for these patients include quickly removing the copper and preparing the patient for liver transplantation. Here, we report on a 6-year-old male with WD in FHF with anemia, renal insufficiency, and coagulopathy. The patient received a series of therapeutic plasma exchanges (TPE) as adjunctive therapy to remove copper and stabilize his coagulopathy and anemia until a transplant was possible. A total of five single plasma volume (1500 mL) TPE were performed over the course of 11 days with plasma as the replacement fluid. Laboratory results demonstrated temporary improvement after each procedure. Liver transplantation was performed 12 days after beginning TPE and 35 days after admission to the hospital. TPE was a successful adjunctive therapy to bridge this patient with WD to transplantation.