Expanding the US Food and Drug Administration–approved indications for immune checkpoint inhibitors in patients with cancer has resulted in therapeutic success and immune-related adverse events (irAEs). Neurologic irAEs (irAE-Ns) have an incidence of 1%–12% and a high fatality rate relative to other irAEs. Lack of standardized disease definitions and accurate phenotyping leads to syndrome misclassification and impedes development of evidence-based treatments and translational research. The objective of this study was to develop consensus guidance for an approach to irAE-Ns including disease definitions and severity grading. A working group of four neurologists drafted irAE-N consensus guidance and definitions, which were reviewed by the multidisciplinary Neuro irAE Disease Definition Panel including oncologists and irAE experts. A modified Delphi consensus process was used, with two rounds of anonymous ratings by panelists and two meetings to discuss areas of controversy. Panelists rated content for usability, appropriateness and accuracy on 9-point scales in electronic surveys and provided free text comments. Aggregated survey responses were incorporated into revised definitions. Consensus was based on numeric ratings using the RAND/University of California Los Angeles (UCLA) Appropriateness Method with prespecified definitions. 27 panelists from 15 academic medical centers voted on a total of 53 rating scales (6 general guidance, 24 central and 18 peripheral nervous system disease definition components, 3 severity criteria and 2 clinical trial adjudication statements); of these, 77% (41/53) received first round consensus. After revisions, all items received second round consensus. Consensus definitions were achieved for seven core disorders: irMeningitis, irEncephalitis, irDemyelinating disease, irVasculitis, irNeuropathy, irNeuromuscular junction disorders and irMyopathy. For each disorder, six descriptors of diagnostic components are used: disease subtype, diagnostic certainty, severity, autoantibody association, exacerbation of pre-existing disease or de novo presentation, and presence or absence of concurrent irAE(s). These disease definitions standardize irAE-N classification. Diagnostic certainty is not always directly linked to certainty to treat as an irAE-N (ie, one might treat events in the probable or possible category). Given consensus on accuracy and usability from a representative panel group, we anticipate that the definitions will be used broadly across clinical and research settings.
Introduction Improvements in quality of life, tremor, and other motor features have been recognized as superior in patients with advanced Parkinson's disease (PD) treated with deep brain stimulation (DBS) surgery versus best medical therapy. We studied a group of patients with PD after undergoing DBS surgery in regard to expectations and satisfaction with DBS outcomes to determine gaps in patient education. Methods This study was a retrospective, single academic center chart review and outcome questionnaire sent to patients with PD who had undergone DBS surgery between 2007 and 2014. Results All patients surveyed indicated that benefit from DBS surgery met their overall expectations at least partially, but only 46.4% (SE: 9.6%) were in complete agreement. 3.6% (SE: 3.6%) of participants strongly disagreed that preoperative education prepared them adequately for the procedure and 17.9% (SE: 7.4%) only somewhat agreed. Conclusions Our findings demonstrate that patients' expectations of DBS surgery in PD were at least partially met. However, there was a considerable percentage of patients who did not feel adequately prepared for the procedure. A structured, multidisciplinary team approach in educating PD patients throughout the different stages of DBS surgery may be helpful in optimizing patients' experience and satisfaction with surgery outcomes.
ObjectiveTo describe the retinal nerve fiber layer (RNFL) with the demographic and clinical profile in patients with glutamic acid decarboxylase 65 (GAD65) and glycine receptor (GlyR) neurological autoimmunity.BackgroundGAD65 and GlyR autoimmunity can cause a wide range of clinical phenomena, including stiff-person spectrum disorder (SPSD) and epilepsy. Both GAD65, through γ-aminobutyric acid-ergic neurons, and GlyR interact in the retina. Optical coherence tomography (OCT) has previously been used in a variety of neurological disorders to establish baseline characteristics and monitor disease course. This presents a noninvasive opportunity to evaluate for a biomarker that may assist with the treatment of these rare but debilitating disorders.Design/MethodsOCT measures of RNFL by sectors were studied in patients with GAD65 and GlyR neurological autoimmunity and compared to that of 148 healthy control eyes. Patients' baseline characteristics were also reviewed retrospectively from medical records.ResultsOf the 14 patients included in this study, 12 patients were female, and the mean age was 52.6 ± 16.8 (22-79) years when OCT was performed. Ten had GAD65 autoimmunity and 4 had GlyR autoimmunity. Patients with GAD or GlyR autoimmunity showed lower RNFL thickness in multiple sectors compared to the healthy control group. This result was most apparent in the anti-GAD65 antibody subgroup. Eleven patients had SPSD, one patient had epilepsy, and two had non-specific symptoms.ConclusionsThis study provides insight into baseline RNFL thickness in a group with GAD65 and GlyR autoimmunity, two conditions that may produce varied symptoms. While limited by sample size, RNFL thinning was seen in the GAD65 and GlyR autoimmunity groups, and it was most evident in the anti-GAD65 subgroup. This provides a baseline characterization and suggests that future studies should be conducted to determine the utility of OCT as a biomarker for these conditions.
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