Early Treatment with Vigabatrin Does Not Decrease Focal Seizures or Improve Cognition in Tuberous Sclerosis Complex: The PREVeNT Trial.

OBJECTIVE: This study was undertaken to test the hypothesis that early vigabatrin treatment in tuberous sclerosis complex (TSC) infants improves neurocognitive outcome at 24 months of age. METHODS: A phase IIb multicenter randomized double-blind placebo-controlled trial was conducted of vigabatrin at first epileptiform electroencephalogram (EEG) versus vigabatrin at seizure onset in infants with TSC. Primary outcome was Bayley Scales of Infant and Toddler Development, Third Edition (Bayley-III) cognitive assessment score at 24 months. Secondary outcomes were prevalence of drug-resistant epilepsy, additional developmental outcomes, and safety of vigabatrin. RESULTS: Of 84 infants enrolled, 12 were screen failures, 4 went straight to open label vigabatrin, and 12 were not randomized (normal EEG throughout). Fifty-six were randomized to early vigabatrin (n = 29) or placebo (n = 27). Nineteen of 27 in the placebo arm transitioned to open label vigabatrin, with a median delay of 44 days after randomization. Bayley-III cognitive composite scores at 24 months were similar for participants randomized to vigabatrin or placebo. Additionally, no significant differences were found between groups in overall epilepsy incidence and drug-resistant epilepsy at 24 months, time to first seizure after randomization, and secondary developmental outcomes. Incidence of infantile spasms was lower and time to spasms after randomization was later in the vigabatrin group. Adverse events were similar across groups. INTERPRETATION: Preventative treatment with vigabatrin based on EEG epileptiform activity prior to seizure onset does not improve neurocognitive outcome at 24 months in TSC children, nor does it delay onset or lower the incidence of focal seizures and drug-resistant epilepsy at 24 months. Preventative vigabatrin was associated with later time to onset and lower incidence of infantile spasms. ANN NEUROL 2023.

Treatment Practices and Outcomes in Continuous Spike and Wave during Slow Wave Sleep: A Multicenter Collaboration.

OBJECTIVES: To determine how continuous spike and wave during slow wave sleep (CSWS) is currently managed and to compare the effectiveness of current treatment strategies using a database from 11 pediatric epilepsy centers in the US. STUDY DESIGN: This retrospective study gathered information on baseline clinical characteristics, CSWS etiology, and treatment(s) in consecutive patients seen between 2014 and 2016 at 11 epilepsy referral centers. Treatments were categorized as benzodiazepines, steroids, other antiseizure medications (ASMs), or other therapies. Two measures of treatment response (clinical improvement as noted by the treating physician; and electroencephalography improvement) were compared across therapies, controlling for baseline variables. RESULTS: Eighty-one children underwent 153 treatment trials during the study period (68 trials of benzodiazepines, 25 of steroids, 45 of ASMs, 14 of other therapies). Children most frequently received benzodiazepines (62%) or ASMs (27%) as first line therapy. Treatment choice did not differ based on baseline clinical variables, nor did these variables correlate with outcome. After adjusting for baseline variables, children had a greater odds of clinical improvement with benzodiazepines (OR 3.32, 95%CI 1.57-7.04, P = .002) or steroids (OR 4.04, 95%CI 1.41-11.59, P = .01) than with ASMs and a greater odds of electroencephalography improvement after steroids (OR 3.36, 95% CI 1.09-10.33, P = .03) than after ASMs. CONCLUSIONS: Benzodiazepines and ASMs are the most frequent initial therapy prescribed for CSWS in the US. Our data suggests that ASMs are inferior to benzodiazepines and steroids and support earlier use of these therapies. Multicenter prospective studies that rigorously assess treatment protocols and outcomes are needed.

Diagnosing and managing childhood absence epilepsy by telemedicine.

The diagnosis of childhood absence epilepsy (CAE) is typically based on history and description of spells, supported by an office-based positive hyperventilation test and confirmed by routine electroencephalography (EEG). In the current coronavirus disease 2019 (COVID-19) pandemic, many pediatric neurologists have switched to telemedicine visits for nonemergent outpatient evaluations. We present a series of children diagnosed as having CAE on the basis of a positive hyperventilation test performed during remote televisits. Several of these children were begun on treatment for CAE prior to obtaining an EEG, with significant seizure reduction. Our series documents the feasibility of CAE diagnosis and management by telemedicine.

Testing a Tailored Social-Ecological Model for Autism Spectrum Disorders.

OBJECTIVE: To propose a tailored social ecological model for Autism Spectrum Disorders and explore relationships between variables in a large nationally-representative dataset. METHODS: A tailored social-ecological model was developed and examined across variables in the 2016/2017 National Survey of Children's Health. A series of iterative multivariable logistic regressions were performed including individual, family, and community/neighborhood variables. A multivariable logistic regression using state-level fixed effects was performed to understand dynamics related to macro-level policies. RESULTS: In the full model, gender, disability severity, certain types of insurance coverage and household income were significantly related to ASD diagnosis. Females had lower odds of a diagnosis compared to males (aOR: 0.27; CI:0.18-0.41). Children with at least one other moderate/severe disability had odds 7.61 higher (CI:5.36-10.82) of a diagnosis than children without moderate/severe disabilities. Children with public insurance only (aOR:1.66; CI:1.14-2.41) or both private and public insurance coverage (aOR: 2.62; CI:1.6-4.16) had higher odds of a diagnosis compared to children with private insurance only. For those who reported it was "somewhat" or "very often" hard to cover basics with their income, odds of a diagnosis were higher compared to those who reported it was "never" or "hardly ever" hard to cover basics (aOR: 1.676; CI:0.21-2.56). CONCLUSIONS FOR PRACTICE: Patterns of ASD diagnosis are related to individual and family characteristics. There is some evidence that a child's environment has some relationship to reported ASD diagnosis. Professionals should be aware of an individual's environmental factors or context when assessing for ASD.

Comorbid Internalizing and Externalizing Symptoms Among Children with ADHD: The Influence of Parental Distress, Parenting Practices, and Child Routines.

Emotional/behavioral concerns are common among children with ADHD. Familial factors (e.g., parental adjustment, parenting behaviors) are linked to the presence of comorbid internalizing/externalizing symptoms among children with ADHD. The purpose of the present study was to evaluate a model that includes multiple familial variables and their direct and indirect effects on child emotional and behavioral problems among children with ADHD. Participants included parents of children (6-12 years of age; M = 8.87, SD = 1.92) with a diagnosis of ADHD (N = 300). Participants completed measures of child emotional/behavioral concerns, parental distress, routines, and parenting behaviors. Path analyses revealed direct effects for parental distress, parent behavior and routines on child adjustment, after controlling for the other variables. A significant indirect relation between parental distress, routines, and externalizing behavior was observed. These findings highlight one specific path through which parental distress appears to influence specific behavioral concerns that are commonly observed in children with ADHD.

Scalp EEG spikes predict impending epilepsy in TSC infants: A longitudinal observational study.

OBJECTIVE: To determine if routine electroencephalography (EEG) in seizure-naive infants with tuberous sclerosis complex (TSC) can predict epilepsy and subsequent neurocognitive outcomes. METHODS: Forty infants 7 months of age or younger and meeting the genetic or clinical diagnostic criteria for tuberous sclerosis were enrolled. Exclusion criteria included prior history of seizures or treatment with antiseizure medications. At each visit, seizure history and 1-hour awake and asleep video-EEG, standardized across all sites, were obtained until 2 years of age. Developmental assessments (Mullen and Vineland-II) were completed at 6, 12, and 24 months of age. RESULTS: Of 40 infants enrolled (mean age of 82.4 days), 32 completed the study. Two were lost to follow-up and six were treated with antiepileptic drugs (AEDs) due to electrographic seizures and/or interictal epileptiform discharges (IEDs) on their EEG studies prior to the onset of clinical seizures. Seventeen of the 32 remaining children developed epilepsy at a mean age of 7.5 months (standard deviation [SD] = 4.4). Generalized/focal slowing, hypsarrhythmia, and generalized/focal attenuation were not predictive for the development of clinical seizures. Presence of IEDs had a 77.3% positive predictive value and absence a 70% negative predictive value for developing seizures by 2 years of age. IEDs preceded clinical seizure onset by 3.6 months (mean). Developmental testing showed significant decline, only in infants with ongoing seizures, but not infants who never developed seizures or whose seizures came under control. SIGNIFICANCE: IEDs identify impending epilepsy in the majority (77%) of seizure-naive infants with TSC. The use of a 1-hour awake and asleep EEG can be used as a biomarker for ongoing epileptogenesis in most, but not all, infants with TSC. Persistent seizures, but not history of interictal epileptiform activity or history of well-controlled seizures, correlated with low scores on the Vineland and Mullen tests at 2 years of age.

Infantile Spasms-Have We Made Progress?

PURPOSE OF THE REVIEW: The care of children with infantile spasms is full of areas of ambiguity, despite practice parameters and randomized trials. This review is to update the current care of children with infantile spasms. RECENT FINDINGS: Several recent studies have generated new data to guide management including a randomized trial supporting combination therapy of hormone treatment and vigabatrin as an initial treatment for infantile spasms. Studies have suggested that we are not consistent in our recognition of hypsarrhythmia, while additional studies suggest that the presence or absence of this pattern may not have any predictive value. Additionally, the use of appropriate medications as well as an early diagnosis has the most impact on short-term outcomes. Children with infantile spasms benefit most from early diagnosis and early treatment with appropriate standard medications such as hormonal therapy (ACTH or prednisolone) or vigabatrin.

Bottom-of-sulcus focal cortical dysplasia presenting as epilepsia partialis continua multimodality characterization including 7T MRI.

INTRODUCTION: Bottom-of-sulcus focal cortical dysplasias are an under recognized, surgically treatable cause of focal epilepsy. Resection can dramatically reduce the seizure burden for children with refractory epilepsy, or eliminate seizures altogether. MATERIAL AND METHODS: We report the case and present the results of multimodality evaluation of a 15-year-old young man who presented with long-standing partial epilepsy affecting his right leg, which over the years became refractory to therapy. RESULTS: High-resolution 3T MRI images acquired as a dedicated epilepsyprotocol were initially interpreted as unremarkable. On further review by an experienced specialist aware of clinical and electroencephalographic findings, a subtle focal cortical dysplasia was identified at the bottom of a sulcus near the medial aspect of the left precentral gyrus. After confirmation of the extent of the lesion with PET and ultra-high field 7T MRI, the patient underwent cortical mapping and focal resection and remains free of seizures. COCLUSIONS: This case emphasizes the need for a multidisciplinary approach to the evaluation of refractory focal epilepsy in children and highlights the potential role of ultra-high field 7T MRI in identifying the often subtle causative anatomic abnormalities.

Knowledge, attitudes and behaviours related to dietary salt among adults in the state of Victoria, Australia 2015.

BACKGROUND: Information on consumer's knowledge, attitudes and behaviours (KABs) related to salt can be used to inform awareness and education campaigns and serve as a baseline measure to monitor changes in KABs over time. The aim of this study was to determine KABs related to salt intake among Victorian adults. METHODS: Cross-sectional survey conducted in Victorian adults aged 18-65 years. Participants were recruited from shopping centres located in Melbourne and Geelong and via online methods (Facebook and Consumer Research Panel) to complete an online survey assessing KABs related to dietary salt. Descriptive statistics (mean (SD) or n (%)) were used to report survey findings. RESULTS: A total of 2398 participants provided a valid survey (mean age 43 years (SD 13), 57% female). The majority (80%) were born in Australia and 63% were the main household grocery shopper. The majority (89%) were aware of the health risks associated with a high salt intake. Eighty three percent believed that Australians eat too much salt. Three quarters (75%) correctly identified salt from processed foods as being the main source of salt in the diet. Less than a third (29%) of participants believed their own individual salt intake exceeded dietary recommendations and only 28% could correctly identify the maximum recommended daily intake for salt. Just under half (46%) of participants were concerned about the amount of salt in food. Almost two thirds (61%) of participants believed that there should be laws which limit the amount of salt added to manufactured foods and 58% agreed that it was difficult to find lower salt options when eating out. CONCLUSIONS: The findings of this study serve as a baseline assessment of KABs related to salt intake in Victorian adults and can be used to assess changes in salt related KABs over time. Public concern about salt is low as many people remain unaware of their own salt intake. An increased awareness of the excessive amount of salt consumed and increased availability of lower salt foods are likely to reduce population salt intake.

Women at extreme risk for obesity-related carcinogenesis: Baseline endometrial pathology and impact of bariatric surgery on weight, metabolic profiles and quality of life.

OBJECTIVES: The study objectives were to determine baseline endometrial histology in morbidly obese women undergoing bariatric surgery and to assess the surgical intervention's impact on serum metabolic parameters, quality of life (QOL), and weight. METHODS: Women undergoing bariatric surgery were enrolled. Demographic and clinicopathologic data, serum, and endometrium (if no prior hysterectomy) were collected preoperatively and serum collected postoperatively. Serum global biochemical data were assessed pre/postoperatively. Welch's two sample t-tests and paired t-tests were used to identify significant differences. RESULTS: Mean age of the 71 women enrolled was 44.2 years, mean body mass index (BMI) was 50.9 kg/m(2), and mean weight loss was 45.7 kg. Endometrial biopsy results: proliferative (13/30; 43%), insufficient (8/30; 27%), secretory (6/30; 20%) and hyperplasia (3/30; 10%-1 complex atypical, 2 simple). QOL data showed significant improvement in physical component scores (PCS means 33.9 vs. 47.2 before/after surgery; p<0.001). Twenty women underwent metabolic analysis which demonstrated significantly improved glucose homeostasis, improved insulin responsiveness, and free fatty acid levels. Significant perturbations in tryptophan, phenylalanine and heme metabolism suggested decreased inflammation and alterations in the intestinal microbiome. Most steroid hormones were not significantly impacted with the exception of decreased DHEAS and 4-androsten metabolites. CONCLUSIONS: Bariatric surgery is accompanied by an improved physical quality of life as well as beneficial changes in glucose homeostasis, insulin responsiveness, and inflammation to a greater extent than the hormonal milieu. The potential cancer protective effects of bariatric surgery may be due to other mechanisms other than simply hormonal changes.

Perceptions of obesity and cancer risk in female bariatric surgery candidates: highlighting the need for physician action for unsuspectingly obese and high risk patients.

OBJECTIVES: To determine: 1) whether obese women perceive themselves to be obese or at risk for malignancy, 2) perceived impact of obesity on cancer risks, 3) compliance with cancer screening, and 4) rates of menstrual dysfunction. METHODS: Surveys were administered to female patients presenting for bariatric weight loss surgery. Demographics, gynecologic history, perception of cancer risk, and screening history were collected/analyzed. Women were categorized as obese (BMI: 30-39kg/m(2)), morbidly obese (40-49kg/m(2)), super obese (≥50kg/m(2)) and compared. RESULTS: Ninety-three women (mean age: 44.9 years, mean BMI: 48.7kg/m(2)) participated and 45.7% felt they were in 'good', 'very good', or 'excellent' health despite frequent medical comorbidities. As BMI increased, women were more likely to correctly identify themselves as obese (23% of obese vs. 77% of morbidly obese vs. 85% of super obese; p<0.001) but there were no significant differences in comorbidities. Two-thirds of women correctly identified obesity as a risk factor for uterine cancer, yet 48% of those retaining a uterus perceived that it was "not likely/not possible" to develop uterine cancer. Menstrual irregularities were common as was evaluation and interventions for the same; 32% had prior hysterectomy. Participation in cancer screening was robust. CONCLUSIONS: Women presenting for bariatric surgery have high rates of menstrual dysfunction. While they perceive that obesity increases uterine cancer risk, they often do not perceive themselves to be at risk. This disconnect may stem from the fact that many failed to identify themselves as obese perhaps because overweight/obesity has become the norm in U.S. society.

Reading comprehension improvement in autism.

Introduction: A subset of autistic children excel at word decoding but have difficulty with reading comprehension (i.e., the discrepant poor comprehender reading profile). Prior research suggests the Visualizing and Verbalizing (V/V) for language comprehension and thinking intervention helps improve reading comprehension in autistic children with this reading profile. Previous studies have demonstrated the role of vocabulary, memory, and social functioning in reading comprehension; however, predictors and moderators of reading comprehension within this specific profile of autistic readers have not been thoroughly explored. Methods: In this study, we examined the effectiveness of the V/V intervention by comparing reading comprehension scores between groups and across time. Participants included a sample of autistic children (AUT-EXP; n=22) and a waitlist control group of autistic children (AUT-WLC; n=17) with reading comprehension difficulties, as well as a sample of non-autistic children (Non-AUT; n=26) (all age 8-13 years). AUT-EXP and AUT-WLC groups completed a battery of cognitive assessments during pre and post tests. We also analyzed whether cognitive assessment scores predicted reading comprehension, and examined the moderating effects of group (AUT-EXP vs. AUT-WLC) on these relationships. Results: The AUT-EXP group significantly improved in their pre to post reading comprehension scores (t(21)=4.19, p<.001, d=.89), whereas the AUT-WLC group did not. Verbal memory significantly predicted reading comprehension, though group did not moderate relationships between cognitive test performance and reading comprehension. Discussion: Results suggest that the V/V intervention may help improve reading comprehension for autistic children with the discrepant poor comprehender reading profile. Additionally, strategies for improving verbal memory may indirectly enhance reading comprehension in autistic children with this reading profile.

Structure of Restricted Repetitive Behaviors of Individuals Referred for Autism Spectrum Disorder Assessment.

PURPOSE: Restricted and/or repetitive displays of behavior, interests, or activities (RRBs) are one of the core symptom domains of autism spectrum disorder (ASD). Current and past research indicates two 'clusters' of RRBs in children with ASD: repetitive sensorimotor (e.g., hand/finger and more complex motor mannerisms) and insistence on sameness (e.g., resistance to changes in the environment) behaviors. The current study aims to fill a gap by examining how RRBs may diverge in individuals with ASD and with other neurodevelopmental disorders (ONDD) in a clinical sample. METHODS: A total of 558 individuals were seen at a tertiary care clinic for a comprehensive clinical assessment of ASD. The sample was split into ASD (n = 292 individuals) and ONDD (n = 266) groups based on clinical diagnosis. Exploratory factor analyses were conducted using Autism Diagnostic Interview-Revised (ADI-R) RRB item scores for the overall sample, the ASD group, and the ONDD group. RESULTS: Exploratory factor analysis of ADI-R RRB items indicated a 2-factor solution for the full sample and ASD group. Items loaded onto two factors comprised of "Repetitive Sensorimotor" and "Insistence on Sameness" behaviors, consistent with previous literature. Results demonstrated a unique loading pattern for the non-ASD group, with items clustering into "Higher Order" (e.g., circumscribed interests) and "Lower Order" (e.g., hand and finger mannerisms) behaviors. CONCLUSION: The results of the current study may point towards using RRBs to guide screening of children who are referred for an ASD evaluation to better identify children who are at higher risk of having ASD.

IL-10R inhibition reprograms tumor-associated macrophages and reverses drug resistance in multiple myeloma.

Multiple myeloma (MM) is the cancer of plasma cells within the bone marrow and remains incurable. Tumor-associated macrophages (TAMs) within the tumor microenvironment often display a pro-tumor phenotype and correlate with tumor proliferation, survival, and therapy resistance. IL-10 is a key immunosuppressive cytokine that leads to recruitment and development of TAMs. In this study, we investigated the role of IL-10 in MM TAM development as well as the therapeutic application of IL-10/IL-10R/STAT3 signaling inhibition. We demonstrated that IL-10 is overexpressed in MM BM and mediates M2-like polarization of TAMs in patient BM, 3D co-cultures in vitro, and mouse models. In turn, TAMs promote MM proliferation and drug resistance, both in vitro and in vivo. Moreover, inhibition of IL-10/IL-10R/STAT3 axis using a blocking IL-10R monoclonal antibody and STAT3 protein degrader/PROTAC prevented M2 polarization of TAMs and the consequent TAM-induced proliferation of MM, and re-sensitized MM to therapy, in vitro and in vivo. Therefore, our findings suggest that inhibition of IL-10/IL-10R/STAT3 axis is a novel therapeutic strategy with monotherapy efficacy and can be further combined with current anti-MM therapy, such as immunomodulatory drugs, to overcome drug resistance. Future investigation is warranted to evaluate the potential of such therapy in MM patients.

Drug-Resistant Epilepsy in Tuberous Sclerosis Complex Is Associated With TSC2 Genotype: More Findings From the Preventing Epilepsy Using Vigatrin (PREVeNT) Trial.

BACKGROUND: Children with tuberous sclerosis complex (TSC) are at high risk for drug-resistant epilepsy (DRE). The ability to stratify those at highest risk for DRE is important for counseling and prompt, aggressive management, necessary to optimize neurocognitive outcomes. Using the extensively phenotyped PREVeNT cohort, we aimed to characterize whether the TSC genotype was associated with DRE. METHODS: The study group (N = 70) comprised participants with TSC enrolled at age less than or equal to six months with detailed epilepsy and other phenotypic and genotypic data, prospectively collected as part of the PREVeNT trial. Genotype-phenotype correlations of DRE, time to first abnormal electroencephalography, and time to epilepsy onset were compared using Fisher exact test and regression models. RESULTS: Presence of a TSC2 pathogenic variant was significantly associated with DRE, compared with TSC1 and participants with no pathogenic mutation identified. In fact, all participants with DRE had a TSC2 pathogenic variant. Furthermore, TSC2 variants expected to result in no protein product were associated with higher risk for DRE. Finally, TSC1 pathogenic variants were associated with later-onset epilepsy, on average 21.2 months later than those with other genotypes. CONCLUSIONS: Using a comprehensively phenotyped cohort followed from infancy, this study is the first to delineate genotype-phenotype correlations for epilepsy severity and onset in children with TSC. Patients with TSC2 pathogenic variants, especially TSC2 pathogenic variants predicted to result in lack of TSC2 protein, are at highest risk for DRE, and are likely to have earlier epilepsy onset than those with TSC1. Clinically, these insights can inform counseling, surveillance, and management.

Happily Distant or Bitter Medicine? The Impact of Social Distancing Preferences, Behavior, and Emotional Costs on Subjective Wellbeing During the Epidemic.

To inhibit the spread of COVID-19 Public health officials stress, and governments often require, restrictions on social interaction ("social distancing"). While the medical benefits are clear, important questions remain about these measures' downsides: How bitter is this medicine? Ten large non-probability internet-based surveys between April and November 2020, weighted statistically to reflect the US population in age, education, and religious background and excluding respondents who even occasionally role-played rather than giving their own true views; N = 6,223. Pre-epidemic data from 2017-2019, N = 4,032. Reliable multiple-item scales including subjective wellbeing (2 European Quality of Life Survey items, Cronbach's alpha = .85); distancing attitudes (5 items, alpha = .87); distancing behavior e.g., standing 6' apart in public (5 items, alpha = .80); emotional cost of distancing and restrictions on social interaction (8-12 items, alpha = .94); and an extensive suite of controls (19 variables). Descriptive statistics, OLS regression, structural equation models. Subjective wellbeing is greater for those who approve of distancing, for those who practice distancing, and particularly for those whose distancing attitudes and behavior are congruent, either both in favor or both opposed (multiplicative interaction). The emotional cost of distancing is strongly tied to wellbeing and is heterogeneous, with some disliking distancing much more than others. An SEM model suggests causality: that emotional costs strongly reduce wellbeing but not vice-versa. During the epidemic, COVID issues constitute two of the top 5 influences on wellbeing, behind only subjective health and religious belief and tied with income. All this is net of family background, religious origins, age, ethnicity, race, gender, rural residence, education, occupational status, marriage, unemployment, income, health, religion, and political party. Supplementary Information: The online version contains supplementary material available at 10.1007/s11482-023-10149-0.

Evaluation of the Simplified Score to Predict Early Relapse in Multiple Myeloma (S-ERMM) in the MMRF CoMMpass study.

BACKGROUND: Zaccaria and colleagues recently proposed a new risk score to identify patients at high risk for relapse within 18 months of diagnosis (ER18), the Score for Early Relapse in Multiple Myeloma (S-ERMM). We performed external validation of the S-ERMM using data from the CoMMpass study. PATIENTS AND METHODS: Clinical data was obtained from the CoMMpass study. Patients were assigned S-ERMM risk scores and risk categories by the three iterations of the International Staging System (ISS): ISS, R-ISS and R2-ISS. Patients with missing data or early mortality in remission were excluded. Our primary endpoint was the relative predictive ability of the S-ERMM versus other risk scores for ER18 as assessed by area-under-the-curve (AUC). RESULTS: 476 patients had adequate data to assign all four risk scores. 65%, 25% and 10% were low, intermediate and high risk by S-ERMM. 17% experienced ER18. All four risk scores stratified patients by risk for ER18. S-ERMM (AUC: 0.59 [95% CI 0.53-0.65]) was similar to R-ISS (0.63 [95% CI 0.58-0.69]) and statistically inferior to ISS (0.68 [95% CI 0.62-0.75]) and R2-ISS (0.66 [95% CI 0.61-0.72]) for prediction of ER18. Sensitivity analyses were performed and did not significantly impact results. CONCLUSION: The S-ERMM risk score is not superior to existing risk stratification systems for predicting early relapse in NDMM and further studies are needed to identify the optimal approach.

Classifying children with ASD by service utilization and treatment type: A cluster analysis of a nationally representative United States survey.

BACKGROUND: If there are patterns of the distribution of services and treatments across the population of people with ASD, these patterns should be based along clinical characteristics or other service needs and not sociodemographic characteristics unrelated to evidence-based care. We examined how individuals in a broad, nationally representative sample "grouped together" based on service utilization and services needed but not covered by insurance. By understanding various treatment patterns, clinicians, researchers, policymakers, and self-advocates and their families can better advocate for high-quality, evidence-based services to be provided equitably. METHODS: Using the 2011 Survey of Pathways to Diagnosis and Services, a cluster analysis was performed to explore patterns in this population based on medication use, private services use, school-based service use, and services not covered by insurance. Differences in clusters were then explored through multinomial logistic regression. RESULTS: Six clusters emerged, showing differences in the level of service/medication usage and insurance coverage. Differences across clusters were associated with the level of functional limitation and age at ASD diagnosis. Disparities by insurance type, functional limitation, and age at diagnosis exist among patterns of ASD service provision. CONCLUSIONS: Our analysis showed that intervention for children with ASD can be across several scales - high and low users of services (both private and school-based), high and low users of medications, and high and low levels of reported non-covered services. The differences were clustered in multiple ways. Further research should incorporate longitudinal and nationally representative data to explore these relationships further.