Abstract
BackgroundPlasma exchange (PE) is used to treat a range of neurological disorders. Based on results demonstrated in Alzheimer’s disease, we theorized that PE with albumin replacement (PE-A) might alter the metabolic profile of plasma and cerebrospinal fluid in patients with amyotrophic lateral sclerosis (ALS) by removing disease-inducing molecules. The aim of this study was to evaluate the effect of PE-A on disease progression in ALS.MethodsIn this open-label, non-controlled, single-arm, prospective pilot study, 13 adults with ALS had 6 months’ treatment with PE-A 5% and 6 months’ follow-up. Primary endpoints were changes from baseline in the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score and forced vital capacity (FVC) through 48 weeks. A post hoc analysis compared individual patient data with the expected ALSFRS-R progression slope.ResultsThe median ALSFRS-R score declined throughout the study, although the rate of decline was slower than expected in seven patients at treatment end and in five patients at study end. Six patients remained in the same baseline slope progression category, and four patients improved their slope category at treatment end. Median FVC decreased significantly during the study. Treatment was well tolerated. Of 330 PE-A procedures, 0.9% were associated with potentially related adverse events.ConclusionAlthough functional impairment progressed, about two-thirds of patients showed a slower than expected rate of decline at treatment end. Most patients had unaltered (54.5%) or reduced (36.4%) ALSFRS-R slope progression at treatment end. Further evaluation of PE-A in controlled studies involving more patients is warranted.EudraCT number2013-004842-40.Trial registrationClinicalTrials.gov identifier: NCT02479802.
Highlights
Amyotrophic lateral sclerosis (ALS) is a neurogenerative disease characterized by progressive loss of the upper and lower motor neurons [1]
As investigated in Alzheimer’s disease [31,32,33], Plasma exchange (PE) with albumin replacement (PE-A) may alter the metabolic profile of the plasma and cerebrospinal fluid in amyotrophic lateral sclerosis (ALS) patients by removing disease-inducing molecules and providing benefits related to the multiple functions of albumin. We considered it appropriate to reexamine the potential therapeutic role of PE-A in patients with ALS
The three patients who violated inclusion criteria and one patient who withdrew before completing half the scheduled treatment were excluded from the per-protocol population
Summary
Amyotrophic lateral sclerosis (ALS) is a neurogenerative disease characterized by progressive loss of the upper and lower motor neurons [1]. The pathophysiology of ALS is not fully understood but is thought to reflect a complex interaction of genetic mutations and environmental factors that leads to dysfunction of various molecular pathways affecting motor neurons, resulting in neurodegeneration [1, 5] Among these pathways, oxidative stressinduced lipid peroxidation may alter lipid metabolism and signalling leading to the accumulation of toxic compounds and disruption of the blood–brain barrier [6,7,8,9,10,11,12,13,14]. Conclusion functional impairment progressed, about two-thirds of patients showed a slower than expected rate of decline at treatment end.
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