ABSTRACT
BACKGROUND AND PURPOSE: The aim was to investigate whether neurofilament light chain (NfL) and profilin-1 (PFN-1) might qualify as surrogate disease and treatment-response biomarkers by correlating their concentrations dynamic with clinical status in a cohort of 30 adult spinal muscular atrophy type 3 patients during nusinersen therapy up to 34 months. METHODS: Neurofilament light chain was measured in cerebrospinal fluid at each drug administration with a commercial enzyme-linked immunosorbent assay (ELISA); PFN-1 concentrations were tested in serum sampled at the same time points with commercial ELISA assays. Functional motor scores were evaluated at baseline, at the end of the loading phase and at each maintenance dose and correlated to biomarker levels. The concurrent effect of age and clinical phenotype was studied. RESULTS: Neurofilament light chain levels were included in the reference ranges at baseline; a significant increase was measured during loading phase until 1 month. PFN-1 was higher at baseline than in controls and then decreased during therapy until reaching control levels. Age had an effect on NfL but not on PFN-1. NfL was partially correlated to functional scores at baseline and at last time point, whilst no correlation was found for PFN-1. CONCLUSION: Cerebrospinal fluid NfL levels did not qualify as an optimal surrogate treatment biomarker in adult spinal muscular atrophy patients with a long disease duration, whilst PFN-1 might to a greater extent represent lower motor neuron pathological processes. The observed biomarker level variation during the first 2 months of nusinersen treatment might suggest a limited effect on axonal remodeling or rearrangement.
ABSTRACT
Background: In 2020, the emergence of the new Coronavirus has put health professionals under enormous pressure, as they had to work in difficult and often disadvantaged situations. Prevention of symptoms such as stress, anxiety and burnout therefore become important health management goals. Aim: The aim of this pilot cross-sectional study was to assess the reliability and feasibility of a tool on Occupational Health Nurses after a Pandemic Period such as the COVID-19 pandemic (Salute Oc-cupazionale negli Infermieri in Periodo Pandemico Covid19 - SOIC) that aims to assess the occupational health and psychological wellbeing of nurses during periods of health crisis. Methods: This study was conducted from September to November 2022. The SOIC tool is composed by two preliminary sections and a third part including five validated questionnaires (measuring burnout, work engagement, psychological symptoms, resilience, and mindful awareness). An opportunistic sample of 202 nurses working in a Teaching Hospital of Rome and members of NurSind union were invited to participate: of these, 24 nurses completed the SOIC in two subsequent occasions (T1 and T2). Results: The test-retest assessment showed no differences between the two waves (T1 and T2) in terms of median scores for all questionnaires included in the SOIC tool. The Cronbach alphas, considering all items of each questionnaire included in the SOIC tool, showed good or excellent internal consistencies. Conclusion: The test-retest assessments and the reliability analyses encouraged the usability of the SOIC tool. Furthermore, consistent associations between the five questionnaires were obtained.