Variation in noninvasive ventilation use in amyotrophic lateral sclerosis

Neurology ◽  
2019 ◽  
Vol 93 (3) ◽  
pp. e306-e316 ◽  
Author(s):  
Nimish J. Thakore ◽  
Brittany R. Lapin ◽  
Erik P. Pioro ◽  
Loutfi S. Aboussouan
Keyword(s):  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Noninvasive Ventilation ◽  
Rating Scale ◽  
Factors Affecting ◽  
Cox Proportional Hazard ◽  
Bulbar Onset ◽  
Cox Proportional Hazard Models ◽  
R Factors ◽  
Lateral Sclerosis

ObjectiveWe sought to examine prevalence and predictors of noninvasive ventilation (NIV) in a composite cohort of patients with amyotrophic lateral sclerosis (ALS) followed in a clinical trials setting (Pooled Resource Open-Access ALS Clinical Trials database).MethodsNIV initiation and status were ascertained from response to question 12 of the revised ALS Functional Rating Scale (ALSFRS-R). Factors affecting NIV use in patients with forced vital capacity (FVC) ≤50% of predicted were examined. Predictors of NIV were evaluated by Cox proportional hazard models and generalized linear mixed models.ResultsAmong 1,784 patients with 8,417 simultaneous ALSFRS-R and FVC% measures, NIV was used by 604 (33.9%). Of 918 encounters when FVC% ≤50%, NIV was reported in 482 (52.5%). Independent predictors of NIV initiation were lower FVC% (hazard ratio [HR] 1.27, 95% confidence interval [CI] 1.17–1.37 for 10% drop), dyspnea (HR 2.62, 95% CI 1.87–3.69), orthopnea (HR 4.09, 95% CI 3.02–5.55), lower bulbar and gross motor subscores of ALSFRS-R (HRs 1.09 [95% CI 1.03–1.14] and 1.13 [95% CI 1.07–1.20], respectively, per point), and male sex (HR 1.73, 95% CI 1.31–2.28). Adjusted for other variables, bulbar onset did not significantly influence time to NIV (HR 0.72, 95% CI 0.47–1.08). Considerable unexplained variability in NIV use was found.ConclusionNIV use was lower than expected in this ALS cohort that was likely to be optimally managed. Absence of respiratory symptoms and female sex may be barriers to NIV use. Prospective exploration of factors affecting adoption of NIV may help bridge this gap and improve care in ALS.

Risk factors for cognitive impairment in amyotrophic lateral sclerosis: a systematic review and meta-analysis

2021 ◽  
pp. jnnp-2020-325701
Author(s):  
Tianmi Yang ◽  
Yanbing Hou ◽  
Chunyu Li ◽  
Bei Cao ◽  
Yangfan Cheng ◽  
...  
Keyword(s):  
Risk Factors ◽  
Amyotrophic Lateral Sclerosis ◽  
Cognitive Impairment ◽  
Family History ◽  
Rating Scale ◽  
Or Education ◽  
Increased Risk ◽  
Bulbar Onset ◽  
History Of ◽  
Lateral Sclerosis

ObjectiveCognitive impairment is a common, far-reaching but imperceptible manifestation in patients with amyotrophic lateral sclerosis (ALS). We aimed to identify the risk factors for cognitive impairment in ALS.MethodsWe searched PubMed and EMBASE for cross-sectional, case–control and cohort studies that reported predictors of cognitive impairment in ALS. The obtained data were meta-analysed to generate overall ORs and 95% CIs.ResultsTwenty-seven eligible articles reporting on 6799 individuals were included out of 20 501 records. Nine predictors were identified: C9orf72 (OR 3.62, 95% CI 1.76 to 7.45), dysarthria (OR 2.25, 95% CI 1.20 to 4.22), family history of ALS (OR 1.76, 95% CI 1.18 to 2.61), predominant upper motor neuron (PUMN) phenotype (OR 1.73, 95% CI 1.09 to 2.73) and bulbar onset (OR 1.54, 95% CI 1.28 to 1.87) increased risk factors for cognitive impairment in ALS. ALS Functional Rating Scale-Revised scores, sex, age or education level were not significantly associated with cognitive impairment in ALS. In addition, C9orf72 (OR=5.94) and bulbar onset (OR=2.08) were strong predictors of ALS-frontotemporal dementia. Female sex conferred more susceptibility to executive cognitive impairment than male sex (OR=1.82).ConclusionsPatients with C9orf72 repeat expansion, dysarthria, family history of ALS, PUMN phenotype and bulbar onset had a high risk for cognitive impairment in ALS. These associations may contribute to understanding the heterogeneity of ALS.PROSPERO registration numberCRD42020201085.

scholarly journals How COVID-19 pandemic changed our management strategies for amyotrophic lateral sclerosis (ALS) patients: Egyptian study

2021 ◽  
Vol 57 (1) ◽  
Author(s):  
Hebatallah R. Rashed
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Disease Progression ◽  
North Africa ◽  
Rating Scale ◽  
Management Strategies ◽  
Office Visits ◽  
Bulbar Onset ◽  
A Prospective Study ◽  
Als Patients ◽  
Lateral Sclerosis

Abstract Background There are several studies that have discussed the efficacy of telemedicine with amyotrophic lateral sclerosis (ALS) patients; however, this approach is still preliminary in Egypt and in North Africa. The objective of the current study is to discuss current experience with telemedicine in monitoring patients in the specialized ALS clinic in Egypt. Efficacy of Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) in monitoring disease progression remotely will be discussed. Results This is a prospective study. Forty-three ALS patients were included in this study in the period between July 1, 2020, and February 6, 2021. Fifty-three telemedicine encounters and 13 post-telemedicine office visits were available. None of the participating patients had COVID-19 infection. Eight patients showed decline in ALSFRS score. ALSFRS-R score reported during telemedicine encounters was confirmed during office visits. Three bulbar onset ALS patients had gastrostomy, and 2 bulbar onset ALS patients had Botox injection for drooling. All eight patients with declining ALSFRS-R were maintained on non-invasive ventilation (NIV) based on their symptoms. Conclusion This is the first study discussing telemedicine in the field of ALS in Egypt and North Africa. ALSFRS-R showed feasibility and reliability in detecting disease progression remotely.

scholarly journals Monitoring disease progression with plasma creatinine in amyotrophic lateral sclerosis clinical trials

2017 ◽  
Vol 89 (2) ◽  
pp. 156-161 ◽  
Author(s):  
Ruben P A van Eijk ◽  
Marinus J C Eijkemans ◽  
Toby A Ferguson ◽  
Stavros Nikolakopoulos ◽  
Jan H Veldink ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Amyotrophic Lateral Sclerosis ◽  
Muscle Strength ◽  
Sample Size ◽  
Disease Progression ◽  
Rating Scale ◽  
Plasma Creatinine ◽  
Rate Of Decline ◽  
Lateral Sclerosis

ObjectivesPlasma creatinine is a predictor of survival in amyotrophic lateral sclerosis (ALS). It remains, however, to be established whether it can monitor disease progression and serve as surrogate endpoint in clinical trials.MethodsWe used clinical trial data from three cohorts of clinical trial participants in the LITRA, EMPOWER and PROACT studies. Longitudinal associations between functional decline, muscle strength and survival with plasma creatinine were assessed. Results were translated to trial design in terms of sample size and power.ResultsA total of 13 564 measurements were obtained for 1241 patients. The variability between patients in rate of decline was lower in plasma creatinine than in ALS functional rating scale–Revised (ALSFRS-R; p<0.001). The average rate of decline was faster in the ALSFRS-R, with less between-patient variability at baseline (p<0.001). Plasma creatinine had strong longitudinal correlations with the ALSFRS-R (0.43 (0.39–0.46), p<0.001), muscle strength (0.55 (0.51–0.58), p<0.001) and overall mortality (HR 0.88 (0.86–0.91, p<0.001)). Using plasma creatinine as outcome could reduce the sample size in trials by 21.5% at 18 months. For trials up to 10 months, the ALSFRS-R required a lower sample size.ConclusionsPlasma creatinine is an inexpensive and easily accessible biomarker that exhibits less variability between patients with ALS over time and is predictive for the patient’s functional status, muscle strength and mortality risk. Plasma creatinine may, therefore, increase the power to detect treatment effects and could be incorporated in future ALS clinical trials as potential surrogate outcome.

scholarly journals Prevalence and Factors Related to Pathological Laughter and Crying in Patients With Amyotrophic Lateral Sclerosis

2021 ◽  
Vol 12 ◽  
Author(s):  
Qian-Qian Wei ◽  
Ruwei Ou ◽  
Junyu Lin ◽  
Lingyu Zhang ◽  
Yanbing Hou ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Regression Analysis ◽  
Rating Scale ◽  
Cox Proportional Hazards ◽  
Disease Stage ◽  
Female Patients ◽  
Pathological Laughter ◽  
Bulbar Onset ◽  
Late Disease Stage ◽  
Lateral Sclerosis

Objective: This study aimed to explore the prevalence and clinical correlates of pathological laughter and crying (PLC) in patients with amyotrophic lateral sclerosis (ALS).Methods: A total of 1,031 ALS patients were enrolled between August 2012 and August 2019. The PLC was recorded by a face-to-face interview. Other characteristics of patients, including depression, anxiety, cognition, and behavior function, were also evaluated. The potential associated factors of PLC were explored using forward binary regression analysis. Survival was analyzed in groups using propensity score matching (PSM) and Cox proportional hazards models.Results: The prevalence of PLC was 11.4% in all patients at baseline. Bulbar-onset and female patients had higher prevalence of PLC. The multivariate regression analysis indicated that PLC in ALS was associated with bulbar onset (p &lt; 0.001), late disease stage (p &lt; 0.001), and higher score in the Hamilton Depression Rating Scale (HDRS) (p = 0.012). The higher score of HDRS was significantly and independently associated with PLC occurrence in bulbar-onset patients (p = 0.032). The late disease stage was related to PLC occurrence in spinal-onset patients (p &lt; 0.001). After comparison with matched pairs by using PSM, PLC at baseline had no impact on survival.Conclusion: PLC was not uncommon in ALS, especially in bulbar-onset and female patients. We highlighted that the emotional state other than cognitive function had possible relationship with PLC in ALS.

scholarly journals Milano–Torino Staging and Long-Term Survival in Chinese Patients with Amyotrophic Lateral Sclerosis

Cells ◽  
2021 ◽  
Vol 10 (5) ◽  
pp. 1220
Author(s):  
Ruojie He ◽  
Minying Zheng ◽  
Ling Lian ◽  
Xiaoli Yao
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Disease Progression ◽  
Rating Scale ◽  
Age At Onset ◽  
Chinese Patients ◽  
Term Survival ◽  
Long Term Survival ◽  
Factors Affecting ◽  
Lateral Sclerosis

(1) Background: The aim of this longitudinal study was to evaluate the association between disease progression according to the Milano–Torino staging (MITOS) system and long-term survival in Chinese patients with amyotrophic lateral sclerosis (ALS). We also examined factors affecting MITOS progression. (2) Methods: Patients were enrolled and underwent follow-up at 6, 12, 18, and 24 months, and their demographic and clinical data, including the Milano–Torino stage, Amyotrophic Lateral Sclerosis Functional Rating Scale—Revised (ALSFRS-R) score and neuropsychiatric data, were evaluated. The sensitivity and specificity of predicting survival outcomes based on MITOS progression and ALSFRS-R score decline from baseline to 6 months were compared. The associations between MITOS progression from baseline to 6 months and survival outcome at 12, 18 and 24 months were examined, and factors associated with disease progression were evaluated with subgroup analyses. (3) Results: Among the 100 patients included, 74% were in stage 0 at baseline, and approximately 95% progressed to a higher stage of the MITOS system at 24 months. MITOS progression from baseline to 6 months and ALSFRS-R decline showed comparable value for predicting survival at 12, 18, and 24 months. MITOS progression from baseline to 6 months is strongly associated with death outcomes. Older age at onset and increased depression and anxiety scores may be related to disease progression. (4) Conclusions: MITOS progression during the early disease course could serve as a prognostic marker of long-term survival and may have utility in clinical trials. Age at onset and diagnosis and neuropsychiatric factors might be associated with disease progression.

scholarly journals Predictors of outcome in pleomorphic xanthoastrocytoma

2020 ◽  
Author(s):  
Antonio Dono ◽  
Victor Lopez-Rivera ◽  
Ankush Chandra ◽  
Cole T Lewis ◽  
Rania Abdelkhaleq ◽  
...  
Keyword(s):  
Tumor Size ◽  
Postoperative Radiotherapy ◽  
Treatment Strategies ◽  
Pleomorphic Xanthoastrocytoma ◽  
Survival Outcomes ◽  
Factors Affecting ◽  
Cox Proportional Hazard ◽  
Kaplan Meier ◽  
Cox Proportional Hazard Models ◽  
Male Patients

Abstract Background Pleomorphic xanthoastrocytomas (PXA) are circumscribed gliomas that typically have a favorable prognosis. Limited studies have revealed factors affecting survival outcomes in PXA. Here, we analyzed the largest PXA dataset in the literature and identify factors associated with outcomes. Methods Using the Surveillance, Epidemiology, and End Results (SEER) 18 Registries database, we identified histologically confirmed PXA patients between 1994 and 2016. Overall survival (OS) was analyzed using Kaplan-Meier survival and multivariable Cox proportional hazard models. Results In total, 470 patients were diagnosed with PXA (males = 53%; median age = 23 years [14-39 years]), the majority were Caucasian (n = 367; 78%). The estimated mean OS was 193 months [95% CI: 179-206]. Multivariate analysis revealed that greater age at diagnosis (≥39 years) (3.78 [2.16-6.59], P &lt; .0001), larger tumor size (≥30 mm) (1.97 [1.05-3.71], P = .034), and postoperative radiotherapy (RT) (2.20 [1.31-3.69], P = .003) were independent predictors of poor OS. Pediatric PXA patients had improved survival outcomes compared to their adult counterparts, in which chemotherapy (CT) was associated with worse OS. Meanwhile, in adults, females and patients with temporal lobe tumors had an improved survival; conversely, tumor size ≥30 mm and postoperative RT were associated with poor OS. Conclusions In PXA, older age and larger tumor size at diagnosis are risk factors for poor OS, while pediatric patients have remarkably improved survival. Postoperative RT and CT appear to be ineffective treatment strategies while achieving GTR confer an improved survival in male patients and remains the cornerstone of treatment. These findings can help optimize PXA treatment while minimizing side effects. However, further studies of PXAs with molecular characterization are needed.

Sign in / Sign up

Export Citation Format

Share Document