A Case of Acute Polyneuropathy with Nephrotic Syndrome Showing Transient Proximal Sensory Conduction Defects

Acute sensorimotor polyneuropathy that resembles Guillain-Barré syndrome (GBS) is rarely accompanied with nephrotic syndrome, and its underlying immunological mechanisms are unclear. A 56-year-old man presented with simultaneous acute progressive symmetric sensorimotor polyneuropathy and proteinuria. A kidney biopsy revealed focal segmental glomerulosclerosis. Serial electrophysiologic studies showed only a transient proximal conduction block in the median nerve, stimulated somatosensory evoked potential and prolonged terminal latencies of the median and peroneal nerves. The patient's neurologic deficits and kidney dysfunction recovered with corticosteroid treatment. Our case showed that somatosensory evoked potential study can be an important objective tool in the diagnosis of acute polyneuropathy with normal distal nerve conduction and that corticosteroids should be considered in the initial treatment of GBS-resembling polyneuropathy associated with nephrotic syndrome

Efficacy and safety of givosiran for acute hepatic porphyria: 24-month interim analysis of the randomized phase 3 ENVISION study

Background & Aims Upregulation of hepatic delta-aminolevulinic acid synthase 1 with accumulation of potentially toxic heme precursors delta-aminolevulinic acid and porphobilinogen is fundamental to the pathogenesis of acute hepatic porphyria. Aims: evaluate long-term efficacy and safety of givosiran in acute hepatic porphyria. Methods Interim analysis of ongoing ENVISION study (NCT03338816), after all active patients completed their Month 24 visit. Patients with acute hepatic porphyria (≥12 years) with recurrent attacks received givosiran (2.5 mg/kg monthly) (n=48) or placebo (n=46) for 6 months (double-blind period); 93 received givosiran (2.5 mg or 1.25 mg/kg monthly) in the open-label extension (continuous givosiran, n=47/48; placebo crossover, n=46/46). Endpoints included annualized attack rate, urinary delta-aminolevulinic acid and porphobilinogen levels, hemin use, daily worst pain, quality of life, and adverse events. Results Patients receiving continuous givosiran had sustained annualized attack rate reduction (median 1.0 in double-blind period, 0.0 in open-label extension); in placebo crossover patients, median annualized attack rate decreased from 10.7 to 1.4. Median annualized days of hemin use were 0.0 (double-blind period) and 0.0 (open-label extension) for continuous givosiran patients and reduced from 14.98 to 0.71 for placebo crossover patients. Long-term givosiran led to sustained lowering of delta-aminolevulinic acid and porphobilinogen and improvements in daily worst pain and quality of life. Safety findings were consistent with the double-blind period. Conclusions Long-term givosiran has an acceptable safety profile and significantly benefits acute hepatic porphyria patients with recurrent attacks by reducing attack frequency, hemin use, and severity of daily worst pain while improving quality of life

Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis

BACKGROUND Patisiran, an investigational RNA interference therapeutic agent, specifically inhibits hepatic synthesis of transthyretin. METHODS In this phase 3 trial, we randomly assigned patients with hereditary transthyretin amyloidosis with polyneuropathy, in a 2:1 ratio, to receive intravenous patisiran (0.3 mg per kilogram of body weight) or placebo once every 3 weeks. The primary end point was the change from baseline in the modified Neuropathy Impairment Score+7 (mNIS+7; range, 0 to 304, with higher scores indicating more impairment) at 18 months. Other assessments included the Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QOL-DN) questionnaire (range, −4 to 136, with higher scores indicating worse quality of life), 10-m walk test (with gait speed measured in meters per second), and modified body-mass index (modified BMI, defined as [weight in kilograms divided by square of height in meters]×albumin level in grams per liter; lower values indicated worse nutritional status). RESULTS A total of 225 patients underwent randomization (148 to the patisiran group and 77 to the placebo group). The mean (±SD) mNIS+7 at baseline was 80.9±41.5 in the patisiran group and 74.6±37.0 in the placebo group; the least-squares mean (±SE) change from baseline was −6.0±1.7 versus 28.0±2.6 (difference, −34.0 points; P<0.001) at 18 months. The mean (±SD) baseline Norfolk QOL-DN score was 59.6±28.2 in the patisiran group and 55.5±24.3 in the placebo group; the least-squares mean (±SE) change from baseline was −6.7±1.8 versus 14.4±2.7 (difference, −21.1 points; P<0.001) at 18 months. Patisiran also showed an effect on gait speed and modified BMI. At 18 months, the least-squares mean change from baseline in gait speed was 0.08±0.02 m per second with patisiran versus −0.24±0.04 m per second with placebo (difference, 0.31 m per second; P<0.001), and the least-squares mean change from baseline in the modified BMI was −3.7±9.6 versus −119.4±14.5 (difference, 115.7; P<0.001). Approximately 20% of the patients who received patisiran and 10% of those who received placebo had mild or moderate infusion-related reactions; the overall incidence and types of adverse events were similar in the two groups. CONCLUSIONS In this trial, patisiran improved multiple clinical manifestations of hereditary transthyretin amyloidosis

Residents need competence not confidence: A retrospective evaluation of the new competency education program for Korean neurology residents.

The objective of our study was to scrutinize the learning experiences of Korean neurology residents, with an emphasis on the implications of the novel competency-based curriculum implemented in 2021. We hypothesized that this revised curriculum could modulate residents' cognitive conduct, primarily the manifestation of overconfidence, in distinctive ways across different stages of training. Our investigative framework was three-fold. Initially, we began with a qualitative inquiry involving in-depth interviews with a purposively selected cohort of eight residents from four training sites. This approach facilitated comprehensive insight into their perceptions of their competence and confidence across the continuum of a four-year residency program. Subsequently, we incorporated the K-NEPA13 assessment instrument, administered to the residents and their overseeing supervisors. This stage aimed to dissect potential cognitive biases, particularly overconfidence and consistency, within the resident population. The final study involved a comprehensive survey administered to a group of 97 Korean neurology residents, allowing us to consolidate and validate our preceding findings. Our findings revealed that junior residents portrayed heightened confidence in their clinical capabilities compared to their senior peers. Intriguingly, junior residents also displayed a stronger inclination towards reevaluating their clinical judgments, a behavior we hypothesize is stimulated by the recently introduced competency-based curriculum. We identified cognitive divergence between junior and senior residents, with the latter group favoring more consistent and linear cause-and-effect reasoning, while the former demonstrated receptiveness to introspection and reconsideration. We speculate this adaptability might be engendered by the supervisor assignment protocol intrinsic to the new curriculum. Our study highlights the essentiality of incorporating cognitive behaviors when devising medical education strategies. Acknowledging and addressing these diverse cognitive biases, and instilling a spirit of adaptability, can nurture a culture that persists in continuous learning and self-reflection among trainee doctors

Changes in Hepcidin Serum Levels Correlate with Clinical Improvement in Idiopathic Restless Legs Syndrome Patients

Background: Restless legs syndrome (RLS) is a common sensory motor neurological disorder that is related to iron&ndash;dopamine dysregulation and immune system alteration. We aimed to assess the effects of serum hepcidin, an iron-regulating hormone, in drug-naive RLS patients compared to healthy controls and to evaluate its role in helping to predict clinical improvement after treatment with dopamine agonist. Methods: Nonanemic and drug-naive RLS patients (n = 18) and healthy controls (n = 15) were enrolled. The serum hepcidin and iron-related values in the serum were measured upon the first visit in both groups and 12 weeks later after dopaminergic treatment in 12 patients. Information about sociodemographic characteristics, sleep-related profiles, mood and anxiety was obtained upon the first visit in all participants as well as after treatment in RLS patients. Results: Serum hepcidin levels exhibited no significant differences between patients with drug-na&iuml;ve RLS and healthy controls at diagnosis (7.1 &plusmn; 2.4 vs. 7.0 &plusmn; 3.2 ng/mL, p = 0.357). Decreased hepcidin levels were significantly associated with decreased RLS severity (&beta; = 0.002, 95% CI = 0.00&minus;0.00, p = 0.005) and improved quality of life (&beta; = 0.002, 95% CI = 0.00&minus;7.01, p = 0.044) in a dose-dependent manner after 12 weeks of treatment with a dopamine agonist. This association was independent of age, sex, inflammatory markers, sleep quality, insomnia, daytime sleepiness, depression and anxiety. Conclusions: This study demonstrates the role of hepcidin in evaluating the positive therapeutic response in RLS

The survey question on consistency bias: The senior residents trust more their initial decisions.

The survey question on consistency bias: The senior residents trust more their initial decisions.</p

K-NEPA 13 stands for Korea Neurologist’s Entrustable Professional Activities 13.

It is an assessment tool that measures residents’ competence in their ability to perform 13 critical clinical tasks (competencies-Communication, Collaboration, Clinical knowledge, independence, Community support and understanding care System). (DOCX)</p

Ranked core competence: Mean ranks (s.d).

The objective of our study was to scrutinize the learning experiences of Korean neurology residents, with an emphasis on the implications of the novel competency-based curriculum implemented in 2021. We hypothesized that this revised curriculum could modulate residents’ cognitive conduct, primarily the manifestation of overconfidence, in distinctive ways across different stages of training. Our investigative framework was three-fold. Initially, we began with a qualitative inquiry involving in-depth interviews with a purposively selected cohort of eight residents from four training sites. This approach facilitated comprehensive insight into their perceptions of their competence and confidence across the continuum of a four-year residency program. Subsequently, we incorporated the K-NEPA13 assessment instrument, administered to the residents and their overseeing supervisors. This stage aimed to dissect potential cognitive biases, particularly overconfidence and consistency, within the resident population. The final study involved a comprehensive survey administered to a group of 97 Korean neurology residents, allowing us to consolidate and validate our preceding findings. Our findings revealed that junior residents portrayed heightened confidence in their clinical capabilities compared to their senior peers. Intriguingly, junior residents also displayed a stronger inclination towards reevaluating their clinical judgments, a behavior we hypothesize is stimulated by the recently introduced competency-based curriculum. We identified cognitive divergence between junior and senior residents, with the latter group favoring more consistent and linear cause-and-effect reasoning, while the former demonstrated receptiveness to introspection and reconsideration. We speculate this adaptability might be engendered by the supervisor assignment protocol intrinsic to the new curriculum. Our study highlights the essentiality of incorporating cognitive behaviors when devising medical education strategies. Acknowledging and addressing these diverse cognitive biases, and instilling a spirit of adaptability, can nurture a culture that persists in continuous learning and self-reflection among trainee doctors.</div

The perceived level of preparedness mean (s.d).

The objective of our study was to scrutinize the learning experiences of Korean neurology residents, with an emphasis on the implications of the novel competency-based curriculum implemented in 2021. We hypothesized that this revised curriculum could modulate residents’ cognitive conduct, primarily the manifestation of overconfidence, in distinctive ways across different stages of training. Our investigative framework was three-fold. Initially, we began with a qualitative inquiry involving in-depth interviews with a purposively selected cohort of eight residents from four training sites. This approach facilitated comprehensive insight into their perceptions of their competence and confidence across the continuum of a four-year residency program. Subsequently, we incorporated the K-NEPA13 assessment instrument, administered to the residents and their overseeing supervisors. This stage aimed to dissect potential cognitive biases, particularly overconfidence and consistency, within the resident population. The final study involved a comprehensive survey administered to a group of 97 Korean neurology residents, allowing us to consolidate and validate our preceding findings. Our findings revealed that junior residents portrayed heightened confidence in their clinical capabilities compared to their senior peers. Intriguingly, junior residents also displayed a stronger inclination towards reevaluating their clinical judgments, a behavior we hypothesize is stimulated by the recently introduced competency-based curriculum. We identified cognitive divergence between junior and senior residents, with the latter group favoring more consistent and linear cause-and-effect reasoning, while the former demonstrated receptiveness to introspection and reconsideration. We speculate this adaptability might be engendered by the supervisor assignment protocol intrinsic to the new curriculum. Our study highlights the essentiality of incorporating cognitive behaviors when devising medical education strategies. Acknowledging and addressing these diverse cognitive biases, and instilling a spirit of adaptability, can nurture a culture that persists in continuous learning and self-reflection among trainee doctors.</div