Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration最新文献

{"title":"Percutaneous endoscopic gastrostomy, body weight loss and survival in amyotrophic lateral sclerosis: a population-based registry study","authors":"Antonio Fasano, N. Fini, D. Ferraro, L. Ferri, M. Vinceti, J. Mandrioli","doi":"10.1080/21678421.2016.1270325","DOIUrl":"https://doi.org/10.1080/21678421.2016.1270325","url":null,"abstract":"Abstract Objective: To assess the role of percutaneous endoscopic gastrostomy (PEG) insertion, and its timing, on ALS survival, and to study prognostic factors of survival before and after PEG placement in a population-based setting. Methods: In this observational population-based, registry study, we enrolled patients with newly- diagnosed ALS, according to the El Escorial revised criteria, who were resident in the Emilia Romagna Region, and who developed severe dysphagia needing enteral nutritional support. The primary outcome measure was tracheostomy-free survival after PEG recommendation. Results: There were 210 patients needing PEG, out of an incident cohort of 545 patients from the Emilia Romagna Registry for ALS, who were diagnosed between 2009 and 2013. One hundred and ninety-three patients were included in the study, and 17 were excluded because they were already tracheostomized at the time of PEG placement. Of the 193 patients included in the study, 152 underwent PEG, whereas 41 did not undergo the procedure. Patients who did not undergo PEG, among the eligible ones, had the same tracheostomy-free survival from onset as patients who did (25 vs. 32 months, p = 0.21). Tracheostomy-free survival from PEG recommendation was greater in patients who underwent PEG placement than in patients who did not (6 vs. 2 months, p = 0.008). Median tracheostomy-free survival from PEG insertion was eight months (95% CI5–12); 30 days after PEG placement, survival was 89.60%. At Cox multivariable analysis, the hazard of death or tracheostomy after PEG insertion was significantly influenced by the difference between BMI at the time of the PEG procedure and BMI at diagnosis (HR 1.05, 95% CI 1.02–1.08; p = 0.002). The hazard of death or tracheostomy was not affected by the timing of PEG insertion. Conclusions: The present study, although it has some limitations, suggests a gain of tracheostomy-free survival from the time of PEG recommendation for patients who undergo PEG placement, and, among patients who undergo PEG, a greater survival if PEG is inserted before a significant weight loss occurs, and if nutritional support avoids further weight loss. Should this association between prevention of weight loss and better clinical outcome be confirmed by further studies, it would have important implications for disease management.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1270325","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"43789070","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A novel mutation of the C-terminal amino acid of FUS (Y526C) strengthens FUS gene as the most frequent genetic factor in aggressive juvenile ALS","authors":"P. Corcia, V. Danel, A. Lacour, S. Beltran, C. Andres, P. Couratier, H. Blasco, P. Vourc'h","doi":"10.1080/21678421.2016.1265564","DOIUrl":"https://doi.org/10.1080/21678421.2016.1265564","url":null,"abstract":"Abstract Although amyotrophic lateral sclerosis (ALS) typically occurs around 60 years, numerous publications report an onset of ALS before the age of 25 years that define juvenile ALS (jALS). Over the last decade, growing literature mentioned jALS with an aggressive evolution which are mainly linked to the FUS gene. We report here the case of a 25-year-old woman with a bulbar onset ALS that progressed in less than 12 months to invasive ventilation due to respiratory failure; Genetic screening identified a new mutation in the FUS gene that lies within the last codon. After reading the literature, it might be legitimate to consider that jALS linked to FUS mutations represent a specific entity different from both classical jALS and adult ALS linked to FUS gene. This should encourage clinician to firstly screen the FUS gene in the presence of a sporadic ALS that occurs before the age of 25 and with an aggressive profile of evolution.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1265564","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41251898","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diagnostic and prognostic significance of neurofilament light chain NF-L, but not progranulin and S100B, in the course of amyotrophic lateral sclerosis: Data from the German MND-net","authors":"P. Steinacker, A. Huss, B. Mayer, T. Grehl, J. Grosskreutz, G. Borck, J. Kuhle, D. Lulé, T. Meyer, Patrick Oeckl, S. Petri, Jochen H Weishaupt, A. Ludolph, M. Otto","doi":"10.1080/21678421.2016.1241279","DOIUrl":"https://doi.org/10.1080/21678421.2016.1241279","url":null,"abstract":"Abstract There is a need for diagnostic, prognostic, and monitoring blood biomarkers for ALS. We aimed to analyse and compare proposed candidate markers for disease progression in the course of ALS. Blood samples were taken from 125 ALS patients, including nine patients with C9orf72 or SOD1 mutation, at regular intervals of six months. ALS patients were characterized by the ALS functional rating scale (ALSFRS-R) and the Edinburgh Cognitive and Behavioural ALS Screen (ECAS). We quantified neurofilament light chain (NF-L), S100B, and progranulin (PGRN) and analysed it in relation to disease progression. Results showed that, at baseline, serum concentrations of NF-L but not PGRN or S100B discriminated significantly between ALS and controls. Within 24 months follow-up the marker concentrations remained stable. Baseline serum NF-L levels correlated with survival time, which was confirmed in subgroups with fast, intermediate, and slow disease progression and there was a weak association with disease duration. For S100B and PGRN we found an association with ALSFRS-R score changes and a trend for decreased levels in the fast progressor subgroup. In conclusion, serum NF-L in any ALS disease stage is a promising marker to support diagnosis and predict outcome, while serum PGRN and S100B are only of minor prognostic value.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1241279","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45658932","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Initiation of non-invasive ventilation in amyotrophic lateral sclerosis and clinical practice guidelines: Single-centre, retrospective, descriptive study in a national reference centre","authors":"M. Georges, J. Golmard, C. Llontop, A. Shoukri, F. Salachas, T. Similowski, C. Morélot-Panzini, J. Gonzalez-Bermejo","doi":"10.1080/21678421.2016.1236817","DOIUrl":"https://doi.org/10.1080/21678421.2016.1236817","url":null,"abstract":"Abstract In amyotrophic lateral sclerosis (ALS), respiratory muscle weakness leads to respiratory failure. Non-invasive ventilation (NIV) maintains adequate ventilation in ALS patients. NIV alleviates symptoms and improves survival. In 2006, French guidelines established criteria for NIV initiation based on limited evidence. Their impact on clinical practice remains unknown. Our objective was to describe NIV initiation practices of the main French ALS tertiary referral centre with respect to guidelines. In this retrospective descriptive study, 624 patients followed in a single national reference centre began NIV between 2005 and 2013. We analysed criteria used to initiate NIV, including symptoms, PaCO2, forced vital capacity, maximal inspiratory pressures and time spent with SpO2 <90% at night. At NIV initiation, 90% of patients were symptomatic. Median PaCO2 was 48 mmHg. The main criterion to initiate NIV was ‘symptoms’ followed by ‘hypercapnia’ in 42% and 34% of cases, respectively. NIV was initiated on functional parameters in only 5% of cases. Guidelines were followed in 81% of cases. In conclusion, despite compliance with French guidelines, the majority of patients are treated at the stage of symptomatic daytime hypoventilation, which suggests that NIV is initiated late in the course of ALS. Whether this practice could be improved by changing guidelines or increasing respiratory-dedicated resources remains to be determined.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1236817","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"47632015","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Translation and Psychometric Evaluation of a Korean Version of the Amyotrophic Lateral Sclerosis-Specific Quality of Life – Revised","authors":"Ju-Yong Oh, G. Hong, S. H. Kim, Jung A Kim","doi":"10.1080/21678421.2016.1249885","DOIUrl":"https://doi.org/10.1080/21678421.2016.1249885","url":null,"abstract":"Abstract Objective: The objective was to translate the Amyotrophic Lateral Sclerosis-Specific Quality of Life - Revised instrument (ALSSQOL-R) into Korean and to examine the psychometric properties of the Korean amyotrophic lateral sclerosis (ALS) population. Methods: The translation involved forward and backward translation. The psychometric properties of the Korean version of the ALSSQOL-R (K-ALSSQOL-R) were tested by patients in an ALS multidisciplinary clinic in Korea (n = 120). The internal consistency, test-retest reliability, content validity, criterion-related validity, and construct validity were evaluated. Results: With regard to the reliability estimate, the internal consistency (Cronbach’s alpha = 0.92) and the test–retest reliability (ICC = 0.89) were good. With regard to the validity estimate, the K-ALSSQOL-R demonstrated concurrent validity with the McGill Quality of Life Single-Item Scale (r = 0.62) and the first question of the World Health Organisation QOL Instrument-Brief (WHOQOL-BREF) (r = 0.64). The convergent validity and discriminant validity were acceptable. The confirmatory factor analysis supported a six-factor model. Conclusion: The K-ALSSOQL-R can be used as a reliable and valid measure of QOL among Korean ALS patients.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1249885","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46622255","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Identifying behavioural changes in ALS: Validation of the Beaumont Behavioural Inventory (BBI)","authors":"M. Elamin, Marta Pinto-Grau, T. Burke, P. Bede, J. Rooney, M. O'Sullivan, K. Lonergan, E. Kirby, Emma Quinlan, Nadia Breen, A. Vajda, M. Heverin, N. Pender, O. Hardiman","doi":"10.1080/21678421.2016.1248976","DOIUrl":"https://doi.org/10.1080/21678421.2016.1248976","url":null,"abstract":"Abstract Objective: Behavioural changes are an important part of amyotrophic lateral sclerosis (ALS). However, most tools do not account for the influence of motor impairment. Furthermore, they do not fully measure the broad range of behavioural changes specific to ALS. This study aimed to develop and validate an ALS specific behavioural inventory, the Beaumont Behavioural Inventory (BBI). Methods: The BBI was validated in a cohort of ALS patients (n = 85) and 78 age-, gender-, and education-matched controls. The scale was validated against the Frontal Systems Behaviour Scale (FrSBe) and The Frontal Assessment Battery (FAB) for convergent validity, and against other non-behavioural measures to assess discriminant validity. Reliability was assessed with Cronbach's alpha. Results: The instrument showed high internal consistency (Cronbach’s alpha value =0.891). BBI scores highly correlated with the FrSBe and moderately with the FAB. However, the measure was independent from non-behavioural measures. Using a cut-off score of 7 for mild behavioural changes, the BBI displayed high sensitivity and specificity (87.9% and 78.85%, respectively). The cut-off score for moderate changes, consistent with a diagnosis of ALS-FTD, is set at 22.5, showing 90% sensitivity and 96% specificity. Discussion: The BBI is a sensitive and specific tool to assess the entire behavioural spectrum of ALS.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1248976","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"45971024","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The measurement and estimation of total energy expenditure in Japanese patients with ALS: a doubly labelled water method study","authors":"Toshio Shimizu, K. Ishikawa-Takata, Akiko Sakata, U. Nagaoka, N. Ichihara, C. Ishida, Y. Nakayama, T. Komori, M. Nishizawa","doi":"10.1080/21678421.2016.1245756","DOIUrl":"https://doi.org/10.1080/21678421.2016.1245756","url":null,"abstract":"Abstract Appropriate nutritional therapy has not been established for patients with amyotrophic lateral sclerosis (ALS). Our objective was to measure the total energy expenditure (TEE) and determine an equation to estimate the energy requirements for Japanese patients with ALS. Twenty-six Japanese patients with ALS participated in the study. The TEE was measured using the doubly labelled water (DLW) method for a 14-day period. Using a range of clinical parameters and multiple regression analyses, we determined an adequate equation to calculate TEE. Results showed that the median value of total energy intake (TEI) was 1581 (interquartile 1278–1782) kcal/d. TEE and TEE/body weight were 1628 kcal/d (1352–1865) and 31.3 kcal/kg (29.2–34.4), respectively. The ratio of TEE/estimated TEE by the Harris-Benedict equation was 1.14 (1.09–1.26). The difference between TEI and TEE was –63 kcal (–221 – 122), and 15 patients (57.7%) showed a negative balance. From regression analyses, we determined an equation to estimate TEE using the resting metabolic rate estimated by the Harris-Benedict equation (RMR-HB) and scores of the revised ALS Functional Rating Scale (ALSFRS-R): TEE = (1.67 × RMR-HB) + (11.8 × ALSFRS-R) – 680 (p < 0.0001). In conclusion, energy expenditure of Japanese patients with ALS was higher than expected, and we proposed a preliminary equation to estimate TEE for future nutritional intervention.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1245756","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42092090","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ALSUntangled No. 36: Accilion","authors":"R. Bedlack, E. Fixsen, C. Quinn, C. Karam, A. Sherman, L. Ostrow, O. Hardiman, T. Heiman-Patterson, L. Gutmann, M. Bromberg, Gregory T. Carter, E. Kabashi, T. Bertorini, T. Mozaffar, P. Andersen, Josep Gamez, M. Dimachkie, Yunxia Wang, P. Wicks, J. Heywood, Steven P Novella, L. Rowland, E. Pioro, Lisa Kinsley, K. Mitchell, J. Glass, S. Sathornsumetee, J. Baker, N. Atassi, D. Forshew, J. Ravits, R. Conwit, C. Jackson, Kate Dalton, K. Tindall, Ginna Gonzalez, J. Robertson, Larry Phillips, M. Benatar, E. Sorenson, C. Shoesmith, S. Nash, N. Maragakis, D. Moore, J. Caress, K. Boylan, C. Armon, M. Grosso, B. Gerecke, J. Wymer, B. Oskarsson, R. Bowser, V. Drory, J. Shefner, N. Lechtzin, Melanie L Leitner, Robert G. Miller, T. Levine, J. Russell, K. Sharma, D. Saperstein, L. McClusky, D. Macgowan, J. Licht, A. Verma, M. Strong, C. Lomen‐Hoerth, R. Tandan, Michael H. Rivner, S. Kolb, M. Polak, S. Rudnicki, Pamela Kittrell, Muddasir Quereshi, G. Sachs, G. Pattee, M. Weiss, J. Kissel, Jonathan M. Goldstein, J. Roths","doi":"10.1080/21678421.2016.1215400","DOIUrl":"https://doi.org/10.1080/21678421.2016.1215400","url":null,"abstract":"Accilion is a topical mineral cream advertised by Advanced Mineral Compounds, LLC (AMC, 2). It is one of several products with different names and websites (Table I) that trace their origin to a botanist named John Wayne Kennedy (20) and his patent entitled ‘Bioavailable minerals for plant health’ (19). Three of these products are topical mineral creams while a fourth is a mineral supplement marketed to be sprayed on agricultural crops to promote disease resistance and improve growth. These products appear to have similar ingredients and similar proposed mechanisms, and nearly identical description by which they claim to distinguish themselves from other mineral compounds (Table I). A representative from AMC has told us that these products are different in important ways including ‘‘zinc/copper ratios’’ and ‘‘redox’’, that there is a legal dispute underway between the current owners, and that ‘‘efforts to have these compounds independently assessed and thoroughly verified are now routinely obstructed’’ (21). Interestingly, the same cancer-patient testimonials appear for three of these products and the same ALS-patient testimonial appears for two of them (Table I).","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1215400","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"46952959","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Serum urate at trial entry and ALS progression in EMPOWER","authors":"Éilis J. O’Reilly, Dawei Liu, D. Johns, M. Cudkowicz, S. Paganoni, M. Schwarzschild, Melanie L Leitner, A. Ascherio","doi":"10.1080/21678421.2016.1214733","DOIUrl":"https://doi.org/10.1080/21678421.2016.1214733","url":null,"abstract":"Abstract Our objective was to determine whether serum urate predicts ALS progression. A study population comprised adult participants of EMPOWER (n = 942), a phase III clinical trial to evaluate the efficacy of dexpramipexole to treat ALS. Urate was measured in blood samples collected during enrollment as part of the routine block chemistry. We measured outcomes by combined assessment of function and survival rank (CAFs), and time to death, by 12 months. Results showed that in females there was not a significant relation between urate and outcomes. In males, outcomes improved with increasing urate (comparing highest to lowest urate quartile: CAFS was 53 points better with p for trend = 0.04; and hazard ratio for death was 0.60 with p for trend = 0.07), but with adjustment for body mass index (BMI) at baseline, a predictor of both urate levels and prognosis, associations were attenuated and no longer statistically significant. Overall, participants with urate levels equal to or above the median (5.1 mg/dl) appeared to have a survival advantage compared to those below (hazard ratio adjusted for BMI: 0.67; 95% confidence interval 0.47–0.95). In conclusion, these findings suggest that while the association between urate at baseline and ALS progression is partially explained by BMI, there may be an independent beneficial effect of urate.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1214733","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41798707","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Longitudinal evaluation of upper extremity reachable workspace in ALS by Kinect sensor","authors":"Evan de Bie, B. Oskarsson, N. Joyce, A. Nicorici, G. Kurillo, Jay J. Han","doi":"10.1080/21678421.2016.1241278","DOIUrl":"https://doi.org/10.1080/21678421.2016.1241278","url":null,"abstract":"Abstract Our objective was to evaluate longitudinal changes in Microsoft Kinect measured upper extremity reachable workspace relative surface area (RSA) versus the revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R), ALSFRS-R upper extremity sub-scale and Forced Vital Capacity (FVC) in a cohort of patients diagnosed with amyotrophic lateral sclerosis (ALS). Ten patients diagnosed with ALS (ages 52–76 years, ALSFRS-R: 8–41 at entry) were tested using single 3D depth sensor, Microsoft Kinect, to measure reachable workspace RSA across five visits spanning one year. Changes in RSA, ALSFRS-R, ALSFRS-R upper extremity sub-scale, and FVC were assessed using a linear mixed model. Results showed that upper lateral quadrant RSA declined significantly in one year by approximately 19% (p <0.01) while all other quadrants and total RSA did not change significantly in this time-period. Simultaneously, ALSFRS-R upper extremity sub-scale worsened significantly by 25% (p <0.01). In conclusion, upper extremity reachable workspace RSA as a novel ALS outcome measure is capable of objectively quantifying declines in upper extremity ability over time in patients with ALS with more granularity than other common outcome measures. RSA may serve as a clinical endpoint for the evaluation of upper extremity targeted therapeutics.","PeriodicalId":7740,"journal":{"name":"Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration","volume":null,"pages":null},"PeriodicalIF":2.8,"publicationDate":"2017-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/21678421.2016.1241278","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42312723","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}