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Introduction The Amyotrophic Lateral Sclerosis (ALS)-Specific Quality of Life instrument and its revised version (ALSSQOL and ALSSQOL-R) have strong psychometric properties, and have demonstrated research and clinical utility. In this study we aimed to develop a short form (ALSSQOL-SF) suitable for limited clinic time and patient stamina. Methods The ALSSQOL-SF was created using Item Response Theory and confirmatory factor analysis on 389 patients. A cross-validation sample of 162 patients assessed convergent, divergent, and construct validity of the ALSSQOL-SF compared with psychosocial and physical functioning measures. Results The ALSSQOL-SF consisted of 20 items. Compared with the ALSSQOL-R, optimal precision was retained, and completion time was reduced from 15-25 minutes to 2-4 minutes. Psychometric properties for the ALSSQOL-SF and its subscales were strong. Discussion The ALSSQOL-SF is a disease-specific global QOL instrument that has a short administration time suitable for clinical use, and can provide clinically useful, valid information about persons with ALS. Muscle Nerve 58: 646-654, 2018.

Amyotrophic lateral sclerosis (ALS) is a progressive, incurable, and fatal neurodegenerative disease associated with average survival of 2 to 5 years. ALS is characterized by the progressive loss of motor neurons resulting in extremity weakness, dysarthria, dysphagia, and respiratory failure. Despite the serious nature of ALS, the mental health of patients with ALS can be relatively preserved; however, unrecognized and untreated mental illness can have a significant impact on quality of life and outcomes. Cognitive and behavioral changes can be prevalent and affect the management of patients. There are also important mental health implications for the caregiver. This article reviews the current understanding of psychiatric disease in patients with ALS. [ Psychiatr Ann . 2017;47(5):258–262. ]

Purpose of Review: This review describes the most common motor neuron disease, ALS. It discusses the diagnosis and evaluation of ALS and the current understanding of its pathophysiology, including new genetic underpinnings of the disease. This article also covers other motor neuron diseases, reviews how to distinguish them from ALS, and discusses their pathophysiology. Recent Findings: In this article, thespectrum of cognitiveinvolvementinALS, new concepts aboutprotein synthesis pathologyinthe etiologyofALS, and new geneticassociations will be covered. This concept has changed over the past 3 to 4 years with the discovery of new genes and genetic processes that may trigger the disease. As of 2014, two-thirds of familial ALS and 10% of sporadic ALS can be explained by genetics. TAR DNA binding protein 43 kDa (TDP-43), for instance, has been shown to cause frontotemporal dementia as well as some cases of familial ALS, and is associated with frontotemporal dysfunction in ALS. Summary: The anterior horn cells control all voluntary movement: motor activity, respiratory, speech, and swallowing functions are dependent upon signals from the anterior horn cells. Diseases that damage the anterior horn cells, therefore, have a profound impact. Symptoms of anterior horn cell loss (weakness, falling, choking) lead patients to seek medical attention. Neurologists are the most likely practitioners to recognize and diagnose damage or loss of anterior horn cells. ALS, the prototypical motor neuron disease, demonstrates the impact of this class of disorders. ALS and other motor neuron diseases can represent diagnostic challenges. Neurologists are often called upon to serve as a ‘‘medical home’’ for these patients: coordinating care, arranging for durable medical equipment, and leading discussions about end-of-life care with patients and caregivers. It is important for neurologists to be able to identify motor neuron diseases and to evaluate and treat patients affected by them. Continuum (Minneap Minn) 2014;20(5):1185–1207.

In a healthcare environment that is trying to achieve better clinical outcomes and patient satisfaction, shared decision-making is a well-established concept that is gaining more interest. Multiple sclerosis is a preference-sensitive condition and provides the opportunity to implement decision aids at various decision points in the disease process. Literature about patient education and outcomes of shared decision aids in multiple sclerosis has been growing over the last decade. In this topical review, we present an overview of the current literature on shared decision-making in multiple sclerosis. While limitations to the generalizability and applicability of decision aids exist, there is evidence that decision aids and shared decision-making can be valuable tools in the clinical care of multiple sclerosis patients.

This stroke code simulation was developed as an educational tool for neurology junior residents, emergency department residents, medical students, nurses, and any medical learners involved in stroke management. The goal is to offer hands-on practice in running a stroke code effectively and efficiently as leaders of a team, ensuring appropriate critical medical decision-making and patient care. Duration of the simulation is 20 minutes, with a minimum of 20 minutes dedicated to a debriefing. Standardized patients are utilized for role playing to enable presentation of neurologic deficits and use of the National Institute of Health Stroke Scale. Residents ideally participate in these simulations in groups of two or three, giving one person the opportunity to be the team leader running the stroke code and others to take the roles of team members. In addition, emergency medicine residents and intensive care unit nurses are included to act as confederates in the simulations and participate in the debrief. Debriefing sessions are held immediately after each simulation with intent of formative feedback using the “good judgment” and “advocacy inquiry” method. Pre- and postsimulation learner surveys help assess effectiveness and provide critical feedback for future considerations. Out of 13 participating neurology residents during our implementation, all agreed that these simulation sessions met the stated clinical objectives. All residents also agreed that the overall quality and utility of debriefing sessions were good. Approximately 84% agreed that the simulation experience was more useful than reading a chapter or attending a lecture about the clinical scenario. After completing the sessions, all surveyed residents agreed that they will be able to better facilitate future acute stroke care as compared to before the training.

Impaired graft function in the postoperative course after lung transplantation (LTx) may in part be due to alterations in pulmonary surfactant. Animal data provide increasing evidence for surfactant abnormalities in the early course after graft reperfusion. However, little is known about the integrity of the surfactant system in human lung transplant recipients. We therefore investigated surfactant properties in bronchoalveolar lavage fluid (BALF) of patients with lung transplants (n = 60) in comparison to that of healthy subjects (n = 10). The phospholipid concentrations of BALF and of surfactant subfractions were measured, and total protein was analyzed. Surface activity was measured with a pulsating bubble surfactometer (PBS). Minimum surface tension was 15.8 +/- 1.1 mN/m in lung transplant recipients, whereas healthy subjects had minimum surface tensions of 3.4 +/- 1.9 mN/m (p = 0.0004). As a marker for potential surfactant inhibition, protein-to-phospholipid (PL) ratios showed no significant differences in the two major study groups. The ratio of small surfactant aggregates to large surfactant aggregates was increased in patients with lung transplants (p = 0.043). Episodes of infection or rejection did not change surface activities, nor did they induce altered ratios of protein to PL or of small to large surfactant aggregates. Surfactant activity did not correlate with pulmonary-function data. Moreover, surface tension showed no correlation with the time after transplantation. Our results suggest a persistent impairment of biophysical surfactant properties after LTx, possibly due to type-II-cell malfunction.

Amyotrophic lateral sclerosis (ALS) is a devastating, and currently incurable, neuromuscular disease in which oxidative stress and mitochondrial impairment are contributing to neuronal loss. Coenzyme Q10 (CoQ10), an antioxidant and mitochondrial cofactor, has shown promise in ALS transgenic mice, and in clinical trials for neurodegenerative diseases other than ALS. Our aims were to choose between two high doses of CoQ10 for ALS, and to determine if it merits testing in a Phase III clinical trial.We designed and implemented a multicenter trial with an adaptive, two-stage, bias-adjusted, randomized, placebo-controlled, double-blind, Phase II design (n = 185). The primary outcome in both stages was a decline in the ALS Functional Rating Scale-revised (ALSFRSr) score over 9 months. Stage 1 (dose selection, 35 participants per group) compared CoQ10 doses of 1,800 and 2,700 mg/day. Stage 2 (futility test, 75 patients per group) compared the dose selected in Stage 1 against placebo.Stage 1 selected the 2,700 mg dose. In Stage 2, the pre-specified primary null hypothesis that this dose is superior to placebo was not rejected. It was rejected, however, in an accompanying prespecified sensitivity test, and further supplementary analyses. Prespecified secondary analyses showed no significant differences between CoQ10 at 2,700 mg/day and placebo. There were no safety concerns.CoQ10 at 2,700 mg daily for 9 months shows insufficient promise to warrant Phase III testing. Given this outcome, the adaptive Phase II design incorporating a dose selection and a futility test avoided the need for a much larger conventional Phase III trial.

We congratulate Sorenson et al.1 for their well-executed IGF-1 trial in patients with amyotrophic lateral sclerosis (ALS). While the title appropriately reflects the subcutaneous mode of IGF-1 delivery, the article fails to distinguish this critical limitation. What is the bioavailability in CSF and brain parenchyma in humans treated twice daily with 0.05 mg/kg IGF-1? Animal studies indicate that subcutaneous delivery of ∼0.8 mg/kg IGF-1 results in a maximal CSF concentration of 3 ng/mL IGF-1 at 2 hours postinjection.2 This equates to 0.4 nM IGF-1 in CSF at peak. The Sorenson et al. trial dose was 16-fold lower, suggesting that the maximal CSF concentration in patients would be 0.025 nM. The Kd of the IGF-1 receptor is 0.15 nM.3 Therefore, at 0.025 nM, only 14% of high-affinity and less than 1% of low-affinity receptors will be occupied. Likewise, the concentration of IGF-1 in CSF overestimates the bioavailable concentration within the brain parenchyma. IGF-1 injected into the lateral ventricle of rats exhibits a half-life of only 12 minutes in the CSF and penetrates …