Medical Research Council Sum Score Research Articles

Prediction of mechanical ventilation in Guillain-Barré syndrome at admission: Construction of a nomogram and comparison with the EGRIS model

BackgroundRespiratory failure requiring mechanical ventilation (MV) is a common and severe complication of Guillain-Barré syndrome (GBS) with a reported incidence ranging from 20 % to 30 %. Thus, we aim to develop a nomogram to evaluate the risk of MV in patients with GBS at admission and tailor individualized care and treatment. MethodsA total of 633 patients with GBS (434 in the training set, and 199 in the validation set) admitted to the First Hospital of Jilin University, Changchun, China from January 2010 to January 2021 were retrospectively enrolled. Subjects (n = 71) from the same institution from January 2021 to May 2022 were prospectively collected and allocated to the testing set. Multivariable logistic regression analysis was applied to build a predictive model incorporating the optimal features selected in the least absolute shrinkage and selection operator (LASSO) in the training set. The predictive model was validated using internal bootstrap resampling, an external validation set, and a prospective testing set, and the model's performance was assessed by using the concordance index (C-index), calibration curves, and decision curve analysis (DCA). Finally, we established a multivariable logistic model by using variables of the Erasmus GBS Respiratory Insufficiency Score (EGRIS) and did the same analysis to compare the performance of our predictive model with the EGRIS model. ResultsVariables in the final model selected by LASSO included time from onset to admission, facial and/or bulbar weakness, Medical Research Council sum score at admission, neutrophil-to-lymphocyte ratio, and platelet-lymphocyte ratio. The model presented as a nomogram displaying favorable discriminative ability with a C-index of 0.914 in the training set, 0.903 in the internal validation set, 0.953 in the external validation set, and 0.929 in the testing set. The model was well-calibrated and clinically useful as assessed by the calibration curve and DCA. As compared with the EGRIS model, our predictive model displayed satisfactory performance. ConclusionsWe constructed a nomogram for early prediction of the risk of MV in patients with GBS. This model had satisfactory performance and appeared more efficient than the EGRIS model in Chinese patients with GBS.

Takotsubo cardiomyopathy in Guillain-Barré syndrome.

Takotsubo cardiomyopathy (TCM) is a serious autonomic complication of Guillain-Barré syndrome (GBS). However, the association between TCM and GBS has not been investigated in detail. We investigated the characteristics of GBS patients with TCM (GBS-TCM). Clinical features and anti-ganglioside antibody between the GBS-TCM patients and 62 classical GBS patients without TCM as control patients were compared. Eight GBS-TCM patients were identified, in whom TCM was diagnosed at a mean of 6.5 [range 3-42] days after the onset of GBS. The age at onset of GBS was elder in the GBS-TCM patients than in the control GBS patients (76.5 [56-87] vs. 52 [20-88] years, p < 0.01). Notably, cranial nerve deficits, particularly in the lower cranial nerves, were observed in all GBS-TCM patients (100% vs. 41.9%, p < 0.01). Additionally, the GBS-TCM patients showed a higher GBS disability score at nadir (5 [4-5] vs. 4 [1-5], p < 0.01), and lower Medical Research Council sum scores at admission and nadir (37 [30-44] vs. 48 [12-60] at admission, p < 0.05, and 20 [12-44] vs. 40 [0-60] at nadir, p < 0.05, respectively). Mechanical ventilation was more frequently required in the GBS-TCM patients (62.5% vs. 11.3%, p < 0.01). Three GBS-TCM patients were positive for anti-ganglioside antibodies. TCM occurred at a relatively early phase of GBS. The characteristics of GBS-TCM were the elder, lower cranial nerve involvements, severe limb weakness, and respiratory failure.

Effects of the High-Intensity Early Mobilization on Long-Term Functional Status of Patients with Mechanical Ventilation in the Intensive Care Unit.

Intensive care unit (ICU)-acquired weakness often occurs in patients with invasive mechanical ventilation (IMV). Early active mobility may reduce ICU-acquired weakness, improve functional status, and reduce disability. The aim of this study was to investigate whether high-intensity early mobility improves post-ICU discharge functional status of IMV patients. 132 adult patients in the ICU who were undergoing IMV were randomly assigned into two groups with a ratio of 1 : 1, with one group received high-intensity early mobility (intervention group, IG), while the other group received conventional treatment (control group, CG). The functional status (Barthel Index (BI)), capacity of mobility (Perme score and ICU Mobility Scale (IMS)), muscle strength (Medical Research Council sum scores (MRC-SS)), mortality, complication, length of ICU stay, and duration of IMV were evaluated at ICU discharge or after 3-month of ICU discharge. The patient's functional status was improved (BI scores 90.6 ± 18.0 in IG vs. 77.7 ± 27.9 in CG; p=0.005), and capacity of mobility was increased (Perme score 17.6 ± 7.1 in IG vs. 12.2 ± 8.5 in CG, p < 0.001; IMS 4.7 ± 2.6 in IG vs. 3.0 ± 2.6 in CG, p < 0.001). The IG had a higher muscle strength and lower incidence of ICU-acquired weakness (ICUAW) than that in the CG. The incidence of mortality and delirium was also lower than CG at ICU discharge. However, there were no differences in terms of length of ICU stay, duration of IMV, ventilator-associated pneumonia, and venous thrombosis. High-intensity early mobility improved the patient's functional status and increased capacity of mobility with IMV. The benefits to functional status remained after 3 month of ICU discharge. Other benefits included higher muscle strength, lower incidence of ICUAW, mortality, and delirium in IG.

Post-COVID-19 physical and cognitive impairments and associations with quality of life: a cross-sectional study.

This study aimed to compare physical and cognitive functions between post-coronavirus disease 2019 (COVID-19) participants and healthy matched controls and investigate associations between physical and cognitive impairments with quality of life. Twenty-three post-COVID-19 participants and 23 age and sex-matched healthy people without a history of COVID-19 were included. Physical function was assessed using the Medical Research Council Sum Score (MRC-SS), 2 min Step Test, Modified Borg Scale, and Short Physical Performance Battery (SPPB) Test. Cognitive function was assessed using the Montreal Cognitive Assessment (MoCA), Trail Making Test (TMT), and Stroop test, and the quality of life was evaluated using the Euro Quality of Life-5 Dimensions-3 Levels (EQ-5D-3l) questionnaire. Assessments were performed by a physical therapist in one session. Mann-Whitney U test showed that in the post-COVID-19 group, compared to the control group, the number of steps in the 2 min Step Test (p < 0.001, ES = 0.57) and the scores of the SPPB (p = 0.03, ES = 0.32), MoCA (p = 0.003, ES = 0.44), Stroop test (p < 0.001, ES = 0.75), and the EQ-5D-3l visual analog scale (p = 0.027, ES = 0.32) were significantly lower. In addition, the Modified Borg Scale score (p < 0.001, ES = 0.6), TMT-A (p = 0.013, ES = 0.36) and TMT-B (p = 0.016, ES = 0.35) times, and the Stroop time (p < 0.001, ES = 0.61) were significantly higher in the post-COVID-19 group. There were no significant between-group differences in the MRC-SS score (p = 0.055, ES = 0.28). Furthermore, there were significant moderate to high associations between physical and cognitive functions and the quality of life in post-COVID-19 participants. On average 4 months after symptomatic COVID-19, post-COVID-19 participants had significant impairments in physical and cognitive functions compared to healthy matched controls that were significantly correlated with the quality of life. These findings highlight the need for a comprehensive assessment to plan appropriate management strategies.

Effects of acute phase intensive electrical muscle stimulation in COVID-19 patients requiring invasive mechanical ventilation: an observational case-control study

We investigated the effects of acute-phase intensive electrical muscle stimulation (EMS) on physical function in COVID-19 patients with respiratory failure requiring invasive mechanical ventilation (IMV) in the intensive care unit (ICU). Consecutive COVID-19 patients requiring IMV admitted to a university hospital ICU between January and April 2022 (EMS therapy group) or between March and September 2021 (age-matched historical control group) were included in this retrospective observational case–control study. EMS was applied to both upper and lower limb muscles for up to 2 weeks in the EMS therapy group. The study population consisted of 16 patients undergoing EMS therapy and 16 age-matched historical controls (median age, 71 years; 81.2% male). The mean period until initiation of EMS therapy after ICU admission was 3.2 ± 1.4 days. The EMS therapy group completed a mean of 6.2 ± 3.7 EMS sessions, and no adverse events occurred. There were no significant differences between the two groups in Medical Research Council sum score (51 vs. 53 points, respectively; P = 0.439) or ICU mobility scale at ICU discharge. Addition of upper and lower limb muscle EMS therapy to an early rehabilitation program did not result in improved physical function at ICU discharge in severe COVID-19 patients.

Prediction of Physical Frailty at Hospital Discharge in Critically Ill Older Patients

Aim To investigate predicting factors for physical frailty development at hospital discharge in older patients admitted to the intensive care unit (ICU). Methods This case–control study focused on ICU-admitted patients aged ≥ 65 years. This study assessed several independent variables, including functional status score for the ICU, Medical Research Council (MRC) sum score, and handgrip strength at ICU discharge. Physical frailty was the primary outcome of this study. Results In total, 61 participants were included: 29 in the non-frailty group and 32 in the frailty group. Logistic regression analysis was used to extract only the MRC sum score at ICU discharge, with a cutoff score of 46 to predict physical frailty onset at hospital discharge. The functional status score for the ICU and handgrip strength were not extracted. Conclusions An MRC sum score ≤46 at hospital discharge can predict physical frailty in critically ill older patients admitted to the ICU.

Tele-Rehabilitation for Boys with Duchenne Muscular Dystrophy in India Amidst the COVID-19 Pandemic: An Implementation Study

Purpose: Duchenne muscular dystrophy (DMD) is X-linked progressive neuromuscular disorder of childhood onset which leads to loss of ambulation, followed by respiratory and cardiac muscle weakness. Physiotherapy is integral to the multidisciplinary care plan for boys with DMD. During the COVID-19 pandemic, Tele-medicine was increasingly used to provide healthcare to patients. The study aimed to assess the efficacy and feasibility of Tele-rehabilitation among ambulatory boys with DMD. Materials and Methods: Ambulatory boys aged 5 to 12 years with DMD were recruited using purposive sampling. The Tele-rehabilitation program was designed by a neuromuscular team and provided by a physiotherapist through telephonic video calls twice a month for three months. A compliance diary was provided to fill out at home after each session. The outcome was measured with a change in Medical Research Council (MRC) sum score and gower's times at 12 weeks using paired t- test. Results: 92 boys with DMD were enrolled and 66 (71%) boys completed the program and had follow-up at 12 weeks. The baseline MRC sum score was 38.2±3.7, and the gower's time was 9.2±6.7 seconds. At 12 weeks MRC sum score was 38.8±4.2, and gower's time was 8.59±7.7 seconds (p-value- 0.03 and p-value- 0.001), respectively. The Vigno's and Brook's scores didn't show a significant change. Compliance of &amp;gt;50% to the Tele-rehabilitation exercise programs was present in 89% of subjects. Conclusion: A short-term Tele-rehabilitation program was feasible with a high compliance rate and efficacious in motor strengthening among boys with DMD.

Long-term Health-related Quality of Life and Physical Function of COVID-19 Survivors with ICU-acquired Weakness.

This study examined the long-term health-related quality of life (HRQOL) and physical function of coronavirus 2019 (COVID-19) survivors diagnosed with intensive care unit-acquired weakness (ICU-AW). The correlation between muscle weakness at ICU discharge and HRQOL was assessed. A retrospective study was conducted on COVID-19 patients admitted to the ICU at Hyogo Medical University Hospital between January 2021 and November 2021. The HRQOL was evaluated using the SF-36 questionnaire, and physical function, including muscle strength assessed by the Medical Research Council Sum Score (MRC-SS), grip strength, and the 6-min walk distance (6MWD), were assessed 18 months after the onset. ICU-AW was diagnosed in patients with an MRC-SS of less than 48 at ICU discharge. We investigated the correlations between the MRC-SS at ICU discharge and the long-term clinical outcomes. We included 26 patients, with 13 having ICU-AW. In the long-term follow-up, the ICU-AW group had significantly lower scores than the no ICU-AW group in the SF-36 subscales such as Physical Functioning (PF), Role Limitation-Physical (RP), Bodily Pain (BP), Vitality (VT), Social Functioning (SF), and Role Limitation-Emotional (RE), as well as in the Physical Component Summary Score (PCS). The muscle strength was also decreased in the ICU-AW group. The MRC-SS at ICU discharge was positively correlated with PF, RP, BP, SF, RE, and PCS in SF-36 at the 18-month follow-up. COVID-19 survivors with ICU-AW experienced a long-term decline in HRQOL, and muscle weakness at ICU discharge was correlated with the long-term HRQOL.

A comprehensive physical functional assessment of survivors of critical care unit stay due to COVID-19.

To examine the physical function and respiratory muscle strength of patients - who recovered from critical COVID-19 - after intensive care unit discharge to the ward on Days one (D1) and seven (D7), and to investigate variables associated with functional impairment. This was a prospective cohort study of adult patients with COVID-19 who needed invasive mechanical ventilation, non-invasive ventilation or high-flow nasal cannula and were discharged from the intensive care unit to the ward. Participants were submitted to Medical Research Council sum-score, handgrip strength, maximal inspiratory pressure, maximal expiratory pressure, and short physical performance battery tests. Participants were grouped into two groups according to their need for invasive ventilation: the Invasive Mechanical Ventilation Group (IMV Group) and the Non-Invasive Mechanical Ventilation Group (Non-IMV Group). Patients in the IMV Group (n = 31) were younger and had higher Sequential Organ Failure Assessment scores than those in the Non-IMV Group (n = 33). The short physical performance battery scores (range 0 - 12) on D1 and D7 were 6.1 ± 4.3 and 7.3 ± 3.8, respectively for the Non-Invasive Mechanical Ventilation Group, and 1.3 ± 2.5 and 2.6 ± 3.7, respectively for the IMV Group. The prevalence of intensive care unit-acquired weakness on D7 was 13% for the Non-IMV Group and 72% for the IMV Group. The maximal inspiratory pressure, maximal expiratory pressure, and handgrip strength increased on D7 in both groups, but the maximal expiratory pressure and handgrip strength were still weak. Only maximal inspiratory pressure was recovered (i.e., > 80% of the predicted value) in the Non-IMV Group. Female sex, and the need and duration of invasive mechanical were independently and negatively associated with the short physical performance battery score and handgrip strength. Patients who recovered from critical COVID-19 and who received invasive mechanical ventilation presented greater disability than those who were not invasively ventilated. However, they both showed marginal functional improvement during early recovery, regardless of the need for invasive mechanical ventilation. This might highlight the severity of disability caused by SARS-CoV-2.

THE EFFECT OF IN-BED LEG CYCLING EXERCISES ON MUSCLE STRENGTH IN PATIENTS WITH INTENSIVE CARE UNIT-ACQUIRED WEAKNESS: A SINGLE-CENTER RETROSPECTIVE STUDY.

To examine the effect of in-bed leg cycling exercise on patients with intensive care unit-acquired weakness (ICU-AW). Single-center retrospective study. Patients admitted to the ICU between January 2019 and March 2023 were enrolled in the ergometer group, and those admitted to the ICU between August 2017 and December 2018 were enrolled in the control group. The ergometer group performed in-bed leg cycling exercises 5 times per week for 20 min from the day of ICU-AW diagnosis. Furthermore, the ergometer group received 1 early mobilization session per day according to the early mobilization protocol, whereas the control group received 1 or 2 sessions per day. The number of patients with recovery from ICU-AW at ICU discharge and improvement in physical functions were compared. Significantly more patients in the ergometer group recovered from ICU-AW than in the control group (87.0% vs 60.6%, p = 0.039). Regarding physical function, the ergometer group showed significantly higher improvement efficiency in Medical Research Council sum score (1.0 [0.7-2.1] vs 0.1 [0.0-0.2], p < 0.001). In-bed leg cycling exercise, in addition to the early mobilization protocol, reduced the number of patients with ICU-AW at ICU discharge.

Effectiveness of massage and range of motion exercises on muscle strength and intensive care unit-acquired weakness in Iranian patients with COVID-19: a randomized parallel-controlled trial.

Muscle weakness is prevalent in intensive care patients. This study focused on comparing the effects of massage and range of motion (ROM) exercises on muscle strength and intensive care unit-acquired weakness (ICU-AW) among patients with coronavirus disease 2019 (COVID-19). This study was a randomized clinical trial that recruited patients (n=45) with COVID-19 admitted to the ICU and divided them into three groups (ROM exercises, massage, and control). We evaluated muscle strength and ICU-AW in the arms and legs using a hand dynamometer. The Medical Research Council sum score was determined before and after the intervention. The study findings were that 0%, 20%, and 100% of the participants in the ROM exercises, massage, and control groups had ICU-AW on the 7th day of ICU admission. The ROM exercise group had greater muscle strength in the hands and legs than the massage and control groups, and the massage group had greater muscle strength than the control group. Massage and ROM exercises could improve muscle strength and reduce ICU-AW in COVID-19 patients admitted to the ICU.

Predictors of postoperative physical functional decline at hospital discharge in elderly patients with prolonged intensive care unit stay after cardiac surgery

BackgroundA prolonged stay in the intensive care (ICU) is associated with physical function decline following cardiac surgery. To predict physical function decline after cardiac surgery, it may be important to evaluate physical function in the ICU. ObjectivesThis study aimed to determine that physical function examination at ICU discharge was independently associated with physical functional decline at hospital discharge in elderly patients who had undergone cardiac surgery and prolonged the ICU stay. MethodsWe assessed physical function before and after cardiac surgery in elderly patients who had spent ≥72 h in the ICU in this retrospective cohort study using the short physical performance battery (SPPB). At hospital discharge, a decrease of at least 1 point on the SPPB was considered a postoperative physical functional decline. Postoperative physical functional decline at hospital discharge was predicted using multiple logistic regression. ResultsWe revealed postoperative physical functional deterioration in 28.0% of patients who spent ≥72 h in the ICU following cardiac surgery. The Medical Research Council sum score (MRC-SS) (OR: 0.96, 95% CI: 0.82–0.99) and mechanical ventilation days (OR: 1.27, 95% CI: 1.01–1.64) were independently associated with physical functional decline at hospital discharge. ConclusionsPhysical function at ICU discharge and mechanical ventilation days were predictors of postoperative physical functional decline at hospital discharge in patients. MRC-SS was more accurate in predicting postoperative physical functional decline at hospital discharge when performed at the time of ICU discharge.

Association between language function and body composition characteristics in patients with subacute left hemispheric stroke

Background &amp; Objectives: Stroke-related sarcopenia and language disorder are main sequelae of stroke. The aim of this study is to evaluate the association between language function and body composition characteristics in patients with subacute left hemiplegic stroke. Methods: Body mass index, skeletal muscle index (SMI), and body fat percentage using bioimpedance analysis, hand grip strength, and Medical Research Council (MRC) sum score were evaluated. Language function was evaluated using the Western Aphasia Battery (WAB). A partial correlation analysis was used to assess the relationship between body composition and language function. Results: A total of 54 patients with subacute left hemispheric stroke were enrolled in the study. The aphasia quotient (AQ) of WAB was significantly correlated with SMI, body fat percentage, MRC-sum score, MRC-sum score of unaffected side and hand grip strength. In the multiple regression analysis after adjusting for age and sex, the AQ of WAB was significantly correlated with SMI. Conclusions: Skeletal muscle index showed highest correlation with language function in patients with left hemispheric stroke.

High-dose pharmaceutical-grade biotin in patients with demyelinating neuropathies: a phase 2b open label, uncontrolled, pilot study

BackgroundWe proposed to investigate high-dose pharmaceutical-grade biotin in a population of demyelinating neuropathies of different aetiologies, as a proof-of-concept.MethodsPhase IIb open label, uncontrolled, single center, pilot study in 15 patients (three groups of five patients) with chronic demyelinating peripheral neuropathy, i.e. chronic inflammatory demyelinating polyradiculoneuropathy, anti-myelin-associated glycoprotein neuropathy and Charcot-Marie-Tooth 1a or 1b. The investigational product was high-dose pharmaceutical-grade biotin (100 mg taken orally three times a day over a maximum of 52 weeks.The primary endpoint was a 10% relative improvement in 2 of the following 4 electrophysiological variables: motor nerve conduction velocity, distal motor latency, F wave latency, duration of the compound muscle action potential. The secondary endpoints included Overall Neuropathy Limitations Scale (ONLS) score, Medical Research Council (MRC) sum score, Inflammatory Neuropathy Cause and Treatment (INCAT) sensory sum score, 10-m walk test, 6-min walk test, posturography parameters, and nerve excitability variables.ResultsThe primary endpoint was reached in one patient. In the full population analysis, some secondary endpoints parameters improved: MRC score, INCAT sensory sum score, 6-min walk distance, strength-duration time constant, and rheobase. There was a positive correlation between the improvement in the 6-min walk distance and the strength-duration time constant. Regarding the safety results, 42 adverse events occurred, of which three were of severe intensity but none was considered as related to the investigational product.ConclusionsEven if the primary endpoint was not met, administration of high-dose pharmaceutical-grade biotin led to an improvement in various sensory and motor parameters, gait abilities, and nerve excitability parameters. The tolerance of the treatment was satisfactory.Trial registrationClinicalTrials.gov Identifier: NCT02967679; date 2016/12/05.

Predictors of long-term health-related quality of life in Guillain-Barré syndrome: A hospital-based study

ObjectiveThis study aimed to determine the impact of patients’ baseline clinical, neurophysiological data, and management plan of Guillain-Barré syndrome (GBS) on long-term quality of life (QoL) and to identify its potential predictors. MethodsSeventy-nine GBS patients were recruited. On admission, participants were evaluated using the Medical Research Council (MRC) sumscore, GBS disability scale (GDS), and Erasmus GBS Respiratory Insufficiency Score (EGRIS). Neurophysiological data were collected, and a management plan was devised. MRC sumscore was repeated at nadir. MRC, GDS and Short Form Survey (SF-36) were assessed at first-year follow-up. ResultsThe mean age was 37.84 ± 17.26 years, with 43 male patients (54.4%). QoL at one year correlated significantly with baseline clinical variables (age, number of days between weakness and admission, MRC sumscore at onset and nadir, high GDS, and EGRIS scores). Antecedent events, especially diarrhoea, neck muscle weakness, autonomic dysfunction, cranial nerve involvement, and mechanical ventilation (MV), associated with worse QoL. Axonal GBS patients had lower QoL than AIDP patients, and PE patients exhibited lower QoL than IVIG patients. Multiple regression analysis showed that older age, diarrhoea, number of days between weakness and admission, neck muscle weakness, cranial nerve involvement, autonomic dysfunction, early MV, and MRC at onset and nadir and high GDS could predict poor QoL. ConclusionOlder age, more days between weakness and admission, neck muscle weakness, cranial nerve involvement, autonomic dysfunction, early MV, diarrhoea, low MRC at onset and nadir, high GDS at onset, axonal type, and PE treatment were potential predictors of poor QoL in GBS.

Efficacy of Teleconsultation-Based Rehabilitation in Children with Landry-Guillain-Barré Syndrome: An Open Label Randomized Controlled Trial (Tele Rehab-LGBS Trial).

To evaluate the feasibility and efficacy of teleconsultation-based rehabilitation in children with Landry-Guillain-Barré syndrome(LGBS), measured with Medical Research Council (MRC) sum score and Hughes score. A pragmatic, prospective, parallel open label randomized controlled trial was conducted among a total of 50 childrenwith LGBS. The children were randomized using computer generated block randomization into 2 groups (25 in each group): Standard ofcare (Group A) and teleconsultation-based rehabilitation (Group B). Primary and secondary outcomes were measured with MRC sum scoreand Hughes score at 12- and 24-wkfollow-up. The mean age was 6.4 ± 3.3 y; 29 (58%) were boys. Baseline MRC sum score (median, IQR), and Hughes score (median, IQR) in group A was 24 (7-31) and 5 (4-5) respectively; and in group B was 18 (9-24) and 4 (4-5) respectively. At discharge, median (IQR) MRC sum score in group A and B was 34 (28-41), and 30 (25-43) (p value = 0.31) respectively. Tele-rehabilitation was provided to group B for 24 wk with 96% compliance. At 12 wk, median MRC sum score in group A and group B were similar [48 (IQR 44-54) vs. 52 (IQR 46-60), p value = 0.08]. At 12 wk and 24 wk, median Hughes score in group A and B were similar. At 24 wk, 15 out of 23 subjects of group A and 18 out of 25 subjects of group B were ambulatory (p value = 0.61). Teleconsultation-based rehabilitation was feasible with a high compliance rate. The functional outcomes measured with MRC sum score and Hughes score were similar in both the groups at 12 and 24 wk follow-up. Teleconsultation-based rehabilitation has similar efficacy as physical rehabilitation.

Proximity extension assay-based discovery of biomarkers for disease activity in chronic inflammatory demyelinating polyneuropathy

BackgroundObjective disease activity biomarkers are lacking in chronic inflammatory demyelinating polyneuropathy (CIDP), impacting treatment decisions in clinical care and outcomes in clinical trials. Using a proximity extension assay, we aimed...

Prognosis and outcome in patients with chronic inflammatory demyelinating polyradiculoneuropathy in a tertiary center in Oman.

Background: Chronic inflammatory demyelinating polyradiculoneuropathy (CIDP) is an immune-mediated condition with variable clinical characteristics and different treatment modalities. We aim to present different clinical and demographic features of all patients with CIDP presented to the neuromuscular clinic within four years and their follow-up results. Methods: A retrospective study from a hospital database of 23 patients met the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) diagnostic criteria for CIPD. Complete demographic and clinical data were collected. Progress and outcome were assessed using two clinical score systems at regular intervals at 6, 12, and 18 months. Results: Mean age of patients was 43.4 ± 20.9 years (male-to-female ratio was 1.6:1). Age of onset was 39.7 ± 18.0 years. At the presentation, the Medical Research Council sum score (MRCss) was 50 (39.7-51.3), and the modified Rankin Scale (mRS) was 3 (2.2-3.4). There was a significant improvement in MRCss during four periods (P < 0.001). Multiple comparisons revealed a significant difference in MRCss between the baseline and 12 and 18 months but no significant change between the baseline and 6 months. Likewise, mRS showed a significant improvement between the baseline and 18 months (no significant change between the baseline and 6 months or 12 months). Conclusion: The clinical characteristics of CIDP in our cohort were similar to other reported studies, and most of the studied patients had good outcomes. Our results could be utilized as baseline data for a better understanding of the characteristics of CIDP in Oman and, consequently, for better management of the disease.

Neuroprognostication: Guillain-Barré Syndrome.

Guillain-Barré syndrome is an immune-mediated disease of the peripheral nerves characterized by rapidly progressing symmetric weakness, areflexia, and albuminocytological dissociation. Most patients reach their nadir within 2 weeks. Disease severity can be mild to severe, with 20% of patients requiring mechanical ventilation. Intravenous immunoglobulin and plasma exchange are equally effective treatments. Monitoring strength, respiratory function, blood pressure, and heart rate, as well as pain management and rehabilitative therapy are important aspects of management. About 20% of patients require assistance to walk at 6 months. Older age, preceding diarrhea, and lower Medical Research Council (MRC) sum scores predict poor outcome. Death from cardiovascular and respiratory complications can occur in the acute or recovery phases of the illness in 3 to 7% of the patients. Risk factors for mortality include advanced age and disease severity at onset. Neuropathic pain, weakness, and fatigue can be residual symptoms; risk factors for these include axonal loss, sensory involvement, and severity of illness.

Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome.

Stiff-person syndrome (SPS) is a rare autoimmune neurological disorder characterized by high titers of antibodies against glutamic acid decarboxylase (GAD) causing impaired GABAergic inhibitory neurotransmission. To date, there is not a defined therapy for such condition, but immunomodulating therapies, such as plasma exchange, intravenous immunoglobulins, and rituximab, have been widely used in clinical practice. However, the efficacy and tolerability of these treatments is not well established. Efgartigimod, a new neonatal Fc receptor (FcRn) blocker, is a human IgG1 antibody Fc fragment engineered with increased affinity for FcRn binding, leadingto areduction in IgGs levels, includingpathogenic IgG autoantibody showing promising results in neurological autoimmune disorders and has been approved for the treatment of AChR-seropositive generalizedmyasthenia gravis (MG). In this study, we report and describe the first data on treatment with efgartigimod in three patients affected by both AChR-seropositive generalized MG and anti-GAD-seropositive SPS. Patients were followed since the start of efgartigimod and for the whole treatment period (12weeks). MG symptoms were assessed with the "MG activity of daily living score" and the Quantitative Myasthenia Gravis score, while SPS ones were assessed with the "SPS activity of daily living score"; muscle strength was assessed with the Medical Research Council Sum score; the overall disability from MG and SPS was assessed by the modified Rankin Scale. All patients showed an improvement in symptoms of both SPS and MG after 2 cycles of treatment. Our data suggest that efgartigimod may be considered as a candidate drug for SPS and other autoantibody-mediated neurological disorders.