Efficacy and safety of incobotulinumtoxin/A in the treatment of 2- to 5-year-old children with chronic sialorrhea associated with neurological disorders and/or intellectual disability

Toxicon ◽  
2021 ◽  
Vol 190 ◽  
pp. S8-S9
Author(s):  
Steffen Berweck ◽  
Marcin Bonikowski ◽  
Marta Banach ◽  
Angelika Hanschmann ◽  
Michael Althaus ◽  
...  
Keyword(s):  
Intellectual Disability ◽  
Neurological Disorders ◽  
Efficacy And Safety ◽  
Incobotulinumtoxin A

scholarly journals Placebo-Controlled Clinical Trial of IncobotulinumtoxinA for Sialorrhea in Children: SIPEXI

Neurology ◽  
2021 ◽  
pp. 10.1212/WNL.0000000000012573
Author(s):  
Steffen Berweck ◽  
Marcin Bonikowski ◽  
Heakyung Kim ◽  
Michael Althaus ◽  
Birgit Flatau-Baqué ◽  
...  
Keyword(s):  
Intellectual Disability ◽  
Adverse Events ◽  
Active Treatment ◽  
Neurological Disorders ◽  
Efficacy And Safety ◽  
Search Query ◽  
Open Label ◽  
Link Type ◽  
Open Label Extension ◽  
Main Period

Background and Objectives:This prospective phase III study (SIPEXI) investigated efficacy and safety of repeated injections of incobotulinumtoxinA (incoBoNT/A) for treatment of chronic sialorrhea (drooling) associated with neurological disorders (e.g., cerebral palsy, traumatic brain injury) and/or intellectual disability in children/adolescents.Methods:The study enrolled 2-17-year-olds with sialorrhea due to neurological disorders and/or intellectual disability. Patients received body weight-dependent doses of incoBoNT/A (20 U to 75 U). A main period with 1 injection cycle (placebo-controlled, double-blind, 6-17-year-olds) was followed by an open-label extension with up to 3 further cycles. An additional cohort of 2-5-year-olds received active treatment throughout the study. Co-primary endpoints were the change in unstimulated salivary flow rate (uSFR) from baseline to week 4, and the carers’ global impression of change scale (GICS) rating at week 4. Adverse events were recorded.Results:In the main period, 220 patients aged 6-17 years were randomized and treated (148 patients in incoBoNT/A group, 72 patients in placebo group). 35 patients aged 2-5 years received incoBoNT/A (no placebo). 214 patients aged 6-17 years and 33 patients aged 2-5 years continued treatment in the open-label extension period. For the 6-17-year-olds, a significant difference between incoBoNT/A and placebo was seen in the mean uSFR decrease (difference: -0.06 g/min; p = 0.0012) and the carers’ GICS rating (difference: 0.28 points; p = 0.032) at week 4, in favor of active treatment. The secondary endpoints consistently supported these results. A sustained benefit was observed during the extension. Incidences of adverse events were comparable between incoBoNT/A and placebo and did not increase notably with repeated injections. The most common adverse events were respiratory infections. Efficacy and safety were also favorable in the uncontrolled cohort of 2-5-year-olds.Discussion:Both co-primary efficacy endpoints were reached and superiority of incoBoNT/A over placebo was confirmed. IncoBoNT/A (up to 75 U, up to 4 cycles) is an effective and well-tolerated treatment for sialorrhea associated with neurological disorders in children.Study registrations:Clinicaltrials.gov: NCT02270736 (www.clinicaltrials.gov/ct2/show/results/NCT02270736); EU Clinical Trials Register: 2013-004532-30 (www.clinicaltrialsregister.eu/ctr-search/search?query=2013-004532-30).Classification of evidence:This study provides Class I evidence that injection of incobotulinumtoxinA decreases drooling in children aged 6-17 years with neurological disorders.

Pathogenic variants in nucleoporin TPR (translocated promoter region, nuclear basket protein) cause severe intellectual disability in humans

2021 ◽  
Author(s):  
Nicole J Van Bergen ◽  
Katrina M Bell ◽  
Kirsty Carey ◽  
Russell Gear ◽  
Sean Massey ◽  
...  
Keyword(s):  
Intellectual Disability ◽  
Neurological Disorders ◽  
Promoter Region ◽  
Nuclear Pore ◽  
Valuable Insight ◽  
Amino Acid Deletion ◽  
Pathogenic Variants ◽  
Severe Intellectual Disability ◽  
Functional Analyses ◽  
Insight Into

Abstract The nuclear pore complex (NPC) is a multi-protein complex that regulates the trafficking of macromolecules between the nucleus and cytoplasm. Genetic variants in components of the NPC have been shown to cause a range of neurological disorders, including intellectual disability and microcephaly. Translocated promoter region, nuclear basket protein (TPR) is a critical scaffolding element of the nuclear facing interior of the NPC. Here we present two siblings with biallelic variants in TPR who present with a phenotype of microcephaly, ataxia and severe intellectual disability. The variants result in a premature truncation variant, and a splice variant leading to a 12-amino acid deletion respectively. Functional analyses in patient fibroblasts demonstrate significantly reduced TPR levels, and decreased TPR-containing NPC density. A compensatory increase in total NPC levels was observed, and decreased global RNA intensity in the nucleus. The discovery of variants that partly disable TPR function provide valuable insight into this essential protein in human disease, and our findings suggest that TPR variants are the cause of the siblings’ neurological disorder.

scholarly journals Intellectual disability among Bhutanese refugees referred to psychiatric service of BPKIHS

2017 ◽  
Vol 13 (1) ◽  
pp. 23-29
Author(s):  
Binod Kumar Deo ◽  
L Mallick ◽  
D R Shakya ◽  
P M Shyangwa ◽  
N Sapkota ◽  
...  
Keyword(s):  
Mental Retardation ◽  
Intellectual Disability ◽  
Neurological Disorders ◽  
Psychiatric Service ◽  
Mild Mental Retardation ◽  
Intellectually Disabled ◽  
Bhutanese Refugees ◽  
Seizure Disorders ◽  
Co Morbidity ◽  
Age Range

Background: Intellectual disability plays a significant role, especially in the field of rehabilitation. It becomes more important when people live in refugee camp for a long duration.Objective: To assess the level of intelligence and disability; and to sort out the illnesses comorbid with mental retardation in the Bhutanese refugees in eastern Nepal.Methods: Hospital based descriptive study was done using purposive sampling. Instruments used were: vineland social maturity scale, developmental screening test, seguin form board, standard progressive matrices and colored progressive matrices.Results: Samples were 42 with 64% females. Age range was 11-20 years. Among these referred cases, 45% had speech disorder and hearing loss and 31% had neurological disorders, including seizure disorders. Sixty-six percent had mild, 9% moderate and 19% severe mental retardation.Conclusion: Majority of the intellectually disabled Bhutanese refugees had mild mental retardation, followed by severe one. Seizure was a common co-morbidity in mental retardation. Health Renaissance 2015;13 (1): 

Efficacy and safety of rituximab therapy in patients with autoimmune neurological disorders

2021 ◽  
Vol 429 ◽  
pp. 118822
Author(s):  
Maria Pia Giannoccaro ◽  
Vincenzo Donadio ◽  
Veria Vacchiano ◽  
Patrizia Avoni ◽  
Rocco Liguori
Keyword(s):  
Neurological Disorders ◽  
Efficacy And Safety

scholarly journals Efficacy of Acupuncture for Health Conditions in Children: A Review

2008 ◽  
Vol 8 ◽  
pp. 670-682 ◽  
Author(s):  
Jean Libonate ◽  
Subhadra Evans ◽  
Jennie C. I. Tsao
Keyword(s):  
Neurological Disorders ◽  
Large Scale ◽  
Health Conditions ◽  
Empirical Literature ◽  
Efficacy And Safety ◽  
Methodological Issues ◽  
Control Groups ◽  
Limited Sample ◽  
Randomized Controlled ◽  
Childhood Problems

Acupuncture has been used to treat a variety of childhood problems; however, the efficacy and safety of pediatric acupuncture remains unclear. This article reviews the existing empirical literature relating to the use of acupuncture for medical conditions in children. A systematic search of the literature revealed that acupuncture has been used to treat five main conditions in children, including pain, nocturnal enuresis, postoperative nausea/vomiting, laryngospasm/stridor, and neurological disorders. Despite a number of methodological issues, including limited sample sizes, lack of randomization, and inappropriate control groups, it is concluded that acupuncture represents a promising intervention for a variety of pediatric health conditions. To further address the safety, effectiveness, and acceptability of acupuncture in children, large-scale randomized controlled trials are needed.

Efficacy and Safety of Clozapine in Patients with Intellectual Disability

2017 ◽  
Vol 41 (S1) ◽  
pp. S91-S91
Author(s):  
P. Ifteni ◽  
A. Teodorescu
Keyword(s):  
Intellectual Disability ◽  
Challenging Behavior ◽  
Randomized Trials ◽  
Behavioral Problem ◽  
Efficacy And Safety ◽  
Global Assessment Of Functioning ◽  
Day Range ◽  
Duration Of Hospitalization ◽  
Competing Interest ◽  
Overt Aggression Scale

IntroductionAggression is common and a major behavioral problem in patients with intellectual disability (ID). Antipsychotics are frequently used for psychosis or challenging behavior. There is little literature regarding utilization of clozapine in patients with ID for aggressive behavior.Aims and objectivesThe aims of the study were the evaluation of efficacy and safety of clozapine in treatment of aggression in patients with ID.MethodsA longitudinal naturalistic study including a cohort of 225 consecutive patients with intellectual disability admitted to an acute psychiatric unit between 1 January 2014 and 31 December 2015. Severity of symptoms was assessed at admission with Modified Overt Aggression Scale (MOAS) and Global Assessment of Functioning Scale (GAFS). The data included: demographics, main psychiatric diagnosis, IQ, alcohol/smoking, institutionalization, antipsychotics and another psychotropics, restraint, readiness to discharge (RDQ), side-effects and length of stay.ResultsOf 225 potentially eligible individuals, 205 (92.7%) were treated with antypschotics and 110 male (53.56%) with mean age 32.37 (SD = 9.9). Thirty-seven patients (18%), 18 male (48.65%) were treated with clozapine, mean dose 309.45 mg/day (range 100–450 mg/day). Clozapine reduced need for restraint and duration of hospitalization compared with haloperidol (P < 0.05).ConclusionsClozapine was efficient and safety for treating persistent aggression in patients with intellectual disability. There were no seizures, myocarditis or agranulocytosis during study. Larger and randomized trials are needed to fully explore the anti-aggressive benefit of clozapine.Disclosure of interestThe authors have not supplied their declaration of competing interest.

scholarly journals Subgrouping School-Aged Children with Autism Spectrum Disorder Based on Co-Occurring Psychopathology

2021 ◽  
Author(s):  
Antonia M. H. Piergies ◽  
Tomoya Hirota ◽  
Rei Monden ◽  
Shuting Zheng
Keyword(s):  
Autism Spectrum Disorder ◽  
Intellectual Disability ◽  
Internalizing Problems ◽  
Neurological Disorders ◽  
Externalizing Problems ◽  
Autism Spectrum ◽  
Spectrum Disorder ◽  
Gastrointestinal Disorders ◽  
School Aged Children ◽  
Children With Asd

Background: Symptom heterogeneity within autism spectrum disorder (ASD) is compounded by co-occurring psychopathology. Considering that some psychiatric disorders present together more often than others, deriving subgroups of individuals with ASD based on co-occurring psychopathology could better our understanding of symptom patterns existing within this population. This study's purpose was to derive subgroups of school-aged children with ASD based on co-occurring psychopathology while controlling for age and sex and to examine correlates of subgroup membership. Method: Six latent class models were estimated with a sample from the Simons Simplex Collection (n = 2,092), using dichotomized categories (borderline/clinical versus normal range) from five of the DSM-Oriented Scales of the Child Behavior Checklist for Ages 6 to 18 as indicator variables. We evaluated the predictive value of intellectual disability and three groups of medical conditions (allergies/autoimmune disorders, gastrointestinal disorders, and neurological disorders) on subgroup membership using multinomial logistic regression. Results: Four subgroups emerged based on the combined presence/absence of psychiatric symptoms: "Low Psychopathology," "Externalizing Problems," "Internalizing Problems," and "High Psychopathology." Gastrointestinal disorders, the strongest predictor, was associated with increased odds of belonging to the "Externalizing Problems," "Internalizing Problems," and "High Psychopathology" subgroups over the "Low Psychopathology" subgroup, whereas intellectual disability was associated with decreased odds. Neurological disorders was also associated with increased odds of belonging to the "Internalizing Problems" and "High Psychopathology" subgroups. Conclusion: Patterns of psychopathology exist within school-aged children with ASD and are correlated with intellectual disability as well as specific medical conditions, providing guidance for clinical practice and etiology-driven research.

Sign in / Sign up

Export Citation Format

Share Document