scholarly journals Evolving Cognitive Dysfunction in Children with Neurologically Stable Opsoclonus–Myoclonus Syndrome

Children ◽  
2020 ◽  
Vol 7 (9) ◽  
pp. 103
Author(s):  
En Lin Goh ◽  
Kate Scarff ◽  
Stephanie Satariano ◽  
Ming Lim ◽  
Geetha Anand
Keyword(s):  
Cognitive Dysfunction ◽  
Disease Onset ◽  
Clinical Relapse ◽  
Disease Course ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Mild Disease ◽  
Severity Scores ◽  
Full Neurological Recovery ◽  
Opsoclonus Myoclonus Syndrome

Cognitive and acquired neurodevelopmental deficits have been reported in children with opsoclonus–myoclonus syndrome (OMS) and are known to be associated with more severe and relapsing disease course. However, there is a paucity of data regarding cognitive dysfunction in children with stable neurological disease. We report three children with OMS and evolving cognitive dysfunction in the context of a mild disease course. The children’s ages at disease onset were between 17 and 35 months and they were followed up for 4–10 years. Neuroblastoma was identified in one child. OMS severity scores ranged between 8 and 12/15 at presentation. They underwent immunotherapy and all were in remission by 7 months (range 4–13 months), with treatment maintained for 1 year. One child remained relapse-free, while two others had one clinical relapse each and were immunotherapy-responsive again. In all cases, evolving cognitive dysfunction was reported despite being in remission and stable off treatment for a median of 20 months (range of 12–31 months; two OMS scores of 0/15 and one of 2/15). In children with OMS who have completed treatment and have made full or near full neurological recovery, concerns remain regarding long-term outcome in terms of future learning and cognitive development.

Subgroup stratification and outcome in recently diagnosed generalized myasthenia gravis

Neurology ◽  
2020 ◽  
Vol 95 (10) ◽  
pp. e1426-e1436 ◽  
Author(s):  
Matthias Tomschik ◽  
Eva Hilger ◽  
Jakob Rath ◽  
Eva-Maria Mayer ◽  
Michael Fahrner ◽  
...  
Keyword(s):  
Myasthenia Gravis ◽  
Late Onset ◽  
Disease Onset ◽  
Early Response ◽  
Response To Treatment ◽  
Disease Course ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Treatment Regimens ◽  
Year 2000

ObjectiveTo describe disease outcomes of myasthenia gravis (MG) subgroups and which factors influence outcomes by reviewing individual patient records of a representative cohort.MethodsWe performed a retrospective analysis of 199 patients with generalized MG and disease onset after the year 2000 who were treated at 2 tertiary referral centers in Austria. We stratified patients as early- and late-onset acetylcholine receptor antibody-positive, muscle-specific tyrosine kinase (MuSK) antibody-positive, and seronegative patients and patients with thymoma regardless of antibody status. We evaluated patients' symptom severity and treatment regimens and the occurrence of life-threatening events at yearly time points for up to 10 years.ResultsMinimal manifestation status or better was eventually achieved and sustained for >1 year by 125 (63%) patients. Forty percent (66 of 165 patients) showed an early response to treatment, which predicted a benign disease course later on. In contrast, 19% of patients, who remained symptomatic for 2 years after disease onset despite immunosuppressive therapy, were more treatment resistant in the following years. The strongest predictor of outcome was the diagnostic subgroup. Patients with MuSK-MG had a much better outcome than previously reported.ConclusionOur data give an update on the disease course of generalized MG in the new century. Diagnostic subgroups and response to treatment within the first 2 years help to predict the long term outcome.

Cognitive dysfunction and mortality in multiple sclerosis: Long-term retrospective review

2021 ◽  
pp. 135245852110665
Author(s):  
Sara Cavaco ◽  
Inês Ferreira ◽  
Inês Moreira ◽  
Ernestina Santos ◽  
Raquel Samões ◽  
...  
Keyword(s):  
Multiple Sclerosis ◽  
Cognitive Dysfunction ◽  
Retrospective Review ◽  
Progressive Disease ◽  
Disease Course ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Study Results ◽  
Clinical Records

Background: Cognitive dysfunction as a predictor of clinical progression and mortality in multiple sclerosis (MS) is still a matter of debate. Objective: The aim of this study was to explore the long-term outcome associated with neuropsychological performance in a cohort of patients with MS. Methods: A series of 408 MS patients had previously undergone a comprehensive neuropsychological assessment and a contemporaneous neurological evaluation (T1). A retrospective review of the clinical records was conducted 102–192 months after T1. Demographic and clinical data regarding the last clinical appointment with EDSS measurement (T2) were collected and the date of the last clinical contact or death (TS) was recorded. Results: This review revealed that cognitive dysfunction (T1) was associated with higher odds of transitioning from relapsing–remitting course to a progressive disease course (adjusted odds ratio (OR) = 2.29, p = 0.043) and higher hazard of death in the total sample (adjusted hazard ratio (HR) = 3.07, p = 0.006) and the progressive disease course subgroup (adjusted HR = 3.68, p = 0.007), even when adjusting for other covariates. Discussion: The study results demonstrate that cognitive dysfunction in MS is predictive of poorer prognosis and mortality.

scholarly journals Validation of prediction models of severe disease course and non-achievement of remission in juvenile idiopathic arthritis: part 1—results of the Canadian model in the Nordic cohort

2019 ◽  
Vol 21 (1) ◽  
Author(s):  
Veronika Rypdal ◽  
◽  
Jaime Guzman ◽  
Andrew Henrey ◽  
Thomas Loughin ◽  
...  
Keyword(s):  
Juvenile Idiopathic Arthritis ◽  
Prediction Model ◽  
Prediction Models ◽  
Severe Disease ◽  
Disease Onset ◽  
Fine Tuning ◽  
Disease Course ◽  
Internal Validation ◽  
Long Term Outcome

Abstract Background Models to predict disease course and long-term outcome based on clinical characteristics at disease onset may guide early treatment strategies in juvenile idiopathic arthritis (JIA). Before a prediction model can be recommended for use in clinical practice, it needs to be validated in a different cohort than the one used for building the model. The aim of the current study was to validate the predictive performance of the Canadian prediction model developed by Guzman et al. and the Nordic model derived from Rypdal et al. to predict severe disease course and non-achievement of remission in Nordic patients with JIA. Methods The Canadian and Nordic multivariable logistic regression models were evaluated in the Nordic JIA cohort for prediction of non-achievement of remission, and the data-driven outcome denoted severe disease course. A total of 440 patients in the Nordic cohort with a baseline visit and an 8-year visit were included. The Canadian prediction model was first externally validated exactly as published. Both the Nordic and Canadian models were subsequently evaluated with repeated fine-tuning of model coefficients in training sets and testing in disjoint validation sets. The predictive performances of the models were assessed with receiver operating characteristic curves and C-indices. A model with a C-index above 0.7 was considered useful for clinical prediction. Results The Canadian prediction model had excellent predictive ability and was comparable in performance to the Nordic model in predicting severe disease course in the Nordic JIA cohort. The Canadian model yielded a C-index of 0.85 (IQR 0.83–0.87) for prediction of severe disease course and a C-index of 0.66 (0.63–0.68) for prediction of non-achievement of remission when applied directly. The median C-indices after fine-tuning were 0.85 (0.80–0.89) and 0.69 (0.65–0.73), respectively. Internal validation of the Nordic model for prediction of severe disease course resulted in a median C-index of 0.90 (0.86–0.92). Conclusions External validation of the Canadian model and internal validation of the Nordic model with severe disease course as outcome confirm their predictive abilities. Our findings suggest that predicting long-term remission is more challenging than predicting severe disease course.

scholarly journals Long-term outcome of ten children with opsoclonus-myoclonus syndrome

2006 ◽  
Vol 166 (4) ◽  
pp. 359-363 ◽  
Author(s):  
Andrea Klein ◽  
Bernhard Schmitt ◽  
Eugen Boltshauser
Keyword(s):  
Term Outcome ◽  
Long Term Outcome ◽  
Opsoclonus Myoclonus Syndrome

scholarly journals Epidemiology of NMOSD in Catalonia: Influence of the new 2015 criteria in incidence and prevalence estimates

2017 ◽  
Vol 24 (14) ◽  
pp. 1843-1851 ◽  
Author(s):  
Maria Sepúlveda ◽  
Marta Aldea ◽  
Domingo Escudero ◽  
Sara Llufriu ◽  
Georgina Arrambide ◽  
...  
Keyword(s):  
Disease Onset ◽  
Population Based ◽  
Reference Laboratory ◽  
Multiple Sources ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Aged People ◽  
Female Predominance ◽  
Prevalence Estimates ◽  
Elder People

Background: Population-based studies on neuromyelitis optica spectrum disorders (NMOSD) are limited, and it is unclear whether the rates have changed with the implementation of the new 2015 criteria. Objectives: To estimate the incidence and prevalence of NMOSD in Catalonia (Spain), using both the 2006 and the 2015 criteria. Methods: In this clinic-based retrospective study, patients diagnosed with NMOSD between 2006 and 2015 were identified using multiple sources, including direct contact to all Catalan hospitals, identification of cases through the Catalan Health Surveillance System, and registry of antibodies to aquaporin-4 (AQP4-IgG) and myelin oligodendrocyte glycoprotein (MOG-IgG) in a reference laboratory. The incidence rate was calculated for the period 1 January 2006–1 January 2016 and prevalence for the date 1 January 2016. Results: We identified 74 patients (by the 2015 criteria). Most patients were Caucasian (81%), and female (76%) with a median age at disease onset of 42 years (range, 10–76 years). In total, 54 (73%) patients were positive for AQP4-IgG, 11 (15%) double-seronegative, and 9 (12%) MOG-IgG-positive. Rates of incidence and prevalence (0.63/1,000,000 person-years and 0.89/100,000, respectively) were 1.5-fold higher than those reported by the 2006 criteria. Lowest rates were seen in children and elder people and highest in women and middle-aged people (40–59 years). The female predominance was lost in incident AQP4-IgG-seronegative children and AQP4-IgG-positive elder people. MOG-IgG and double-seronegativity contributed similarly but did not influence the long-term outcome. Conclusion: The new criteria increase the estimates, but NMOSD remains as a rare disease. The differences in age- and sex-specific estimates highlight the importance of the serologic classification.

scholarly journals Long-term outcome in rheumatoid arthritis: A simple algorithm of baseline parameters can predict radiographic damage, disability, and disease course at 12-year followup

2002 ◽  
Vol 47 (4) ◽  
pp. 383-390 ◽  
Author(s):  
K. W. Drossaers-Bakker ◽  
A. H. Zwinderman ◽  
T. P. M. Vliet Vlieland ◽  
D. Van Zeben ◽  
K. Vos ◽  
...  
Keyword(s):  
Rheumatoid Arthritis ◽  
Simple Algorithm ◽  
Disease Course ◽  
Term Outcome ◽  
Radiographic Damage ◽  
Long Term Outcome

scholarly journals AB0976 CAPTURING THE ENTHESITIS RELATED ARTHRITIS CONTEMPORARY PROFILE OF NORTHERN GREEK PATIENTS IN THE ERA OF BIOLOGICS

2020 ◽  
Vol 79 (Suppl 1) ◽  
pp. 1782.1-1783
Author(s):  
D. Deligeorgakis ◽  
M. Trachana ◽  
P. Pratsidou-Gertsi ◽  
D. Dimopoulou ◽  
A. B. Haidich ◽  
...  
Keyword(s):  
Clinical Remission ◽  
Disease Onset ◽  
Young Patients ◽  
Disease Status ◽  
Median Percentage ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Enthesitis Related Arthritis ◽  
Negative Prognostic Factor

Background:Enthesitis-Related Arthritis (ERA) is a subtype of Juvenile Idiopathic Arthritis (JIA) subtype with an estimated prevalence ranging from 8% to 37.4%. The improvement of the disease course and outcome has been related with the introduction of biologic Disease-Modifying Antirheumatic Drugs (bDMARDs) and the uninterrupted monitoring following the transition of young patients to adult rheumatology settings.Objectives:To capture the contemporary ERA profile in Northern Greek patients by analyzing the characteristics and treatment outcome in the era of bDMARDs.Methods:This retrospective cohort study included patients who had been monitored on a 3-month schedule for ≥12 months, from 2000 to 2017. The periodic metric assessment included the disease status and burden by applying contemporary tools in respect to activity, clinical remission (CR) and damage (cJADAS, JSpADA, Wallace criteria for CR and JADI, respectively).Results:Forty-three patients, mainly male (60%) with a mean age at disease onset of 10.75 (SD:2.75) years were enrolled. The predominant joints were the hip, ankle and sacroiliac (56%, 49% and 46%, respectively). Median lag time from diagnosis to bDMARDs initiation was 8.5 months. Patients with sacroiliitis were more likely to receive bDMARDs (hazard ratio [HR]:3.26, 95% confidence interval [CI]: 1.35, 7.88). Thirty six patients (84%) achieved clinical remission (CR) on medication (CRONM), within a median time of 11 months and correlated with compliance (HR:3.62, 95%CI: 1.34, 9.76). Twenty patients (47%) experienced a flare following CR, mainly as a single episode (75%). The median flare-free survival following remission on and off medication (CROFFM) was 42 and 34 months, respectively. At the last evaluation, both median baseline cJADAS (8), and JSpADA (2) dropped to 0, while 13 patients (30%) were in CROFFM, 17 (40%) in CRONM, and 13 (30%) had persistent disease activity. The median percentage of CR per patient was 54% and no patient had JADI >0.Conclusion:Early administration of bDMARDs and compliance to monitoring and treatment improved the long-term outcome in ERA. Axial involvement emerged as a negative prognostic factor with an increased need for bDMARDs and diminished rates of CR.Disclosure of Interests:Dimitrios Deligeorgakis: None declared, Maria Trachana: None declared, Polyxeni Pratsidou-Gertsi: None declared, Despoina Dimopoulou: None declared, Anna Bettina Haidich: None declared, Alexandros Garyfallos Grant/research support from: MSD, Aenorasis SA, Speakers bureau: MSD, Novartis, gsk

scholarly journals Takotsubo Cardiomyopathy in Patients Suffering From Acute Non-traumatic Subarachnoid Hemorrhage - A Single Center Follow-up Study

2021 ◽  
Author(s):  
Csilla Molnár ◽  
Judit Gál ◽  
Dorottya Szántó ◽  
László Fülöp ◽  
Andrea Szegedi ◽  
...  
Keyword(s):  
Subarachnoid Hemorrhage ◽  
Takotsubo Cardiomyopathy ◽  
Troponin T ◽  
Disease Onset ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Entire Cohort ◽  
Follow Up Study ◽  
Transcranial Duplex Sonography

Abstract Background: Takotsubo cardiomyopathy (TTC) is an important complication of subarachnoid hemorrhage (SAH), that may delay surgical or endovascular treatment and may influence patient outcome. This prospective follow-up study intended to collect data on the prevalence, severity, influencing factors and long-term outcome of TTC in patients suffering from non-traumatic SAH. Methods: Consecutive patients admitted with the diagnosis of non-traumatic SAH were included. Intitial assessment consisted of cranial CT, Hunt-Hess, Fisher and WFNS scoring, 12-lead ECG, transthoracic echocardiography (TTE), transcranial duplex sonography and collecting laboratory parameters (CK, CK-MB, cardiac troponin T, NT-proBNP and urine metanephrine and normetanephrine). Diagnosis of TTC was based on modified Mayo criteria. TTC patients were dichotomized to mild and severe forms. Follow-up of TTE, Glasgow Outcome Scale assessment, Barthel’s and Karnofsky scoring occurred on days 30 and 180.Results: One hundred thirty six patients were included. The incidence of TTC in the entire cohort was 28.7 %; of them, 20.6% and 8.1% were mild and severe, respectively. TTC was more frequent in females (30/39; 77%) than in males (9/39; 23%) and was more severe. The occurrence of TTC was related to mFisher scores and WFNS scores. Although the severity of TTC was related to mFisher score, Hunt-Hess score, WFNS score and GCS, multivariate analysis showed the strongest relationship with mFisher scores. Ejection fraction differences between groups were present on day 30, but disappeared by day 180, whereas wall motion score index was still higher in the severe TTC group at day 180. By the end of the follow-up period (180 days), 70 (74.5%) patients survived in the non-TTC, 22 (81.5%) in the mild TTC and 3 (27%) in the severe TTC group (n = 11) (p = 0.002). At day 180, GOS, Barthel, and Karnofsky outcome scores were higher in patients in the control (non-TTC) and the mild TTC groups than in the severe TTC group. Conclusions: Takotsubo cardiomyopathy is a frequent finding in patients with SAH, and severe TTC may be present in 8% of SAH cases. The severity of TTC may be an independent predictor of mortality and outcome at 6 months after disease onset. Therefore, a regular follow-up of ECG and TTE abnormalities is warranted in patients with subrachnoid hemorrhage for early detection of TTC. Trial registration: The study was registered at the Clinical Trials Register under the registration number of NCT02659878 (date of registration: January 21, 2016)

scholarly journals Rituximab Treatment and Long-term Outcome of Patients With Autoimmune Encephalitis

2021 ◽  
Vol 8 (6) ◽  
pp. e1088
Author(s):  
Franziska S. Thaler ◽  
Luise Zimmermann ◽  
Stefan Kammermeier ◽  
Christine Strippel ◽  
Marius Ringelstein ◽  
...  
Keyword(s):  
Real World ◽  
Independent Living ◽  
Disease Onset ◽  
Autoimmune Encephalitis ◽  
Acid Decarboxylase ◽  
Real World Data ◽  
Term Outcome ◽  
Long Term Outcome ◽  
Score Improvement

Background and ObjectivesTo determine the real-world use of rituximab in autoimmune encephalitis (AE) and to correlate rituximab treatment with the long-term outcome.MethodsPatients with NMDA receptor (NMDAR)-AE, leucine-rich glioma-inactivated-1 (LGI1)- AE, contactin-associated protein-like-2 (CASPR2)-AE, or glutamic acid decarboxylase 65 (GAD65) disease from the GErman Network for Research on AuToimmune Encephalitis who had received at least 1 rituximab dose and a control cohort of non–rituximab-treated patients were analyzed retrospectively.ResultsOf the 358 patients, 163 (46%) received rituximab (NMDAR-AE: 57%, CASPR2-AE: 44%, LGI1-AE: 43%, and GAD65 disease: 37%). Rituximab treatment was initiated significantly earlier in NMDAR- and LGI1-AE (median: 54 and 155 days from disease onset) compared with CASPR2-AE or GAD65 disease (median: 632 and 1,209 days). Modified Rankin Scale (mRS) scores improved significantly in patients with NMDAR-AE, both with and without rituximab treatment. Although being more severely affected at baseline, rituximab-treated patients with NMDAR-AE more frequently reached independent living (mRS score ≤2) (94% vs 88%). In LGI1-AE, rituximab-treated and nontreated patients improved, whereas in CASPR2-AE, only rituximab-treated patients improved significantly. No improvement was observed in patients with GAD65 disease. A significant reduction of the relapse rate was observed in rituximab-treated patients (5% vs 13%). Detection of NMDAR antibodies was significantly associated with mRS score improvement. A favorable outcome was also observed with early treatment initiation.DiscussionWe provide real-world data on immunosuppressive treatments with a focus on rituximab treatment for patients with AE in Germany. We suggest that early and short-term rituximab therapy might be an effective and safe treatment option in most patients with NMDAR-, LGI1-, and CASPR2-AE.Class of EvidenceThis study provides Class IV evidence that rituximab is an effective treatment for some types of AE.

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