Effect of Brain-Gut Behavioral Treatments on Abdominal Pain in Irritable Bowel Syndrome: Systematic Review and Network Meta-Analysis.

BACKGROUND & AIMS: Some brain-gut behavioral treatments (BGBTs) are beneficial for global symptoms in irritable bowel syndrome (IBS). United States management guidelines suggest their use in patients with persistent abdominal pain, but their specific effect on this symptom has not been assessed systematically. METHODS: We searched the literature through December 16, 2023, for randomized controlled trials (RCTs) assessing efficacy of BGBTs for adults with IBS, compared with each other or a control intervention. Trials provided an assessment of abdominal pain resolution or improvement at treatment completion. We extracted data as intention-to-treat analyses, assuming dropouts to be treatment failures and reporting pooled relative risks (RRs) of abdominal pain not improving with 95% confidence intervals (CIs), ranking therapies according to the P score. RESULTS: We identified 42 eligible randomized controlled trials comprising 5220 participants. After treatment completion, the BGBTs with the largest numbers of trials and patients recruited demonstrating efficacy for abdominal pain, specifically, included self-guided/minimal contact cognitive behavioral therapy (CBT) (RR, 0.71; 95% CI, 0.54-0.95; P score, 0.58), face-to-face multicomponent behavioral therapy (RR, 0.72; 95% CI, 0.54-0.97; P score, 0.56), and face-to-face gut-directed hypnotherapy (RR, 0.77; 95% CI, 0.61-0.96; P score, 0.49). Among trials recruiting only patients with refractory global IBS symptoms, group CBT was more efficacious than routine care for abdominal pain, but no other significant differences were detected. No trials were low risk of bias across all domains, and there was evidence of funnel plot asymmetry. CONCLUSIONS: Several BGBTs, including self-guided/minimal contact CBT, face-to-face multicomponent behavioral therapy, and face-to-face gut-directed hypnotherapy may be efficacious for abdominal pain in IBS, although none was superior to another.

Behavioral Therapy for Functional Heartburn: Recommendation Statements.

BACKGROUND & AIMS: Brain-gut behavior therapies (BGBT) are increasingly recognized as effective therapeutic interventions for functional heartburn. However, recommendations regarding candidacy for treatment, initial treatment selection, and navigating treatment non-response have not been established for functional heartburn specifically. The aim of this study was to establish expert-based recommendations for behavioral treatment in patients with functional heartburn. METHODS: The validated RAND/University of California, Los Angeles Appropriateness Method was applied to develop recommendations. A 15-member panel composed of 10 gastrointestinal psychologists and 5 esophageal specialists ranked the appropriateness of a series of statements on a 9-point interval scale over 2 ranking periods. Statements were within the following domains: pre-therapy evaluation, candidacy criteria for BGBT, selection of initial BGBT, role of additional therapy for initial non-response to BGBT, and role of pharmacologic neuromodulation. The primary outcome was appropriateness of each intervention based on the recommendation statements. RESULTS: Recommendations for psychosocial assessment (eg, hypervigilance, symptom-specific anxiety, health-related quality of life), candidacy criteria (eg, motivated for BGBT, acknowledges the role of stress in symptoms), and treatment were established. Gut-directed hypnotherapy or cognitive behavioral therapy were considered appropriate BGBT for functional heartburn. Neuromodulation and/or additional BGBT were considered appropriate in the context of non-response. CONCLUSIONS: Gut-directed hypnotherapy and/or cognitive behavioral therapy are recommended as appropriate behavioral interventions for heartburn symptoms, depending on clinical indication, specific gut-brain targets, and preferred treatment modality (pharmacologic vs non-pharmacologic). Pre-therapy evaluation of psychosocial processes and candidacy for BGBT are important to determine eligibility for referral to psychogastroenterology services.

Accuracy, Reliability, and Comprehensibility of ChatGPT-Generated Medical Responses for Patients With Nonalcoholic Fatty Liver Disease.

Nonalcoholic fatty liver disease (NAFLD) is an increasing global health problem and is expected to become the leading indication for liver transplantation.1 There are no approved NAFLD-specific pharmacotherapies, and lifestyle modification is the primary recommended therapy.2 Innovative approaches to facilitate the implementation and long-term maintenance of lifestyle changes are needed to address the challenging and complex nature of the management of NAFLD, which recently was renamed as metabolic dysfunction-associated steatotic liver disease, to overcome the limitations and stigma of the previous name.3,4 Artificial intelligence (AI)-powered chatbots have been shown to provide effective personalized support and education to patients, with the potential to complement health care resources. The OpenAI Foundation's AI chatbot, Chat Generative Pretrained Transformer (ChatGPT), has attracted worldwide attention for its remarkable performance in question-answer tasks.5-7 This study evaluated the accuracy, completeness, and comprehensiveness of chatGPT's responses to NAFLD-related questions, with the aim of assessing its performance in addressing patients' queries about the disease and lifestyle behaviors.

Development and Current State of Digital Therapeutics for Irritable Bowel Syndrome.

BACKGROUND & AIMS: Irritable bowel syndrome (IBS) is a common, debilitating disorder characterized by abdominal pain and disordered bowel habits. Current pharmacologic treatments often provide incomplete symptom relief and may be poorly tolerated. Furthermore, alleviation of gastrointestinal symptoms does not always translate into improved quality of life for IBS patients. Current treatment guidelines recommend brain-gut behavior therapy (BGBT) in conjunction with other IBS therapies, and, in randomized controlled trials, BGBT has been shown to improve symptoms, patient satisfaction, functioning, and quality of life. Access to BGBT is limited by lack of adequately trained gastrointestinal psychologists, patient time constraints, and cost. Furthermore, clinician knowledge that BGBT is specific, and different from psychotherapy approaches for common mental health disorders, may limit referrals even where available. This review provides an overview of the pathophysiology of IBS, disease burden, unmet therapeutic needs, evidence base of novel digital therapeutics for IBS, and guidance on the introduction and appropriateness of these interventions for patients. METHODS: We searched the literature for available published data relating to the use of novel digital therapeutics to provide cognitive behavioral therapy and gut-directed hypnotherapy in the treatment of irritable bowel syndrome. RESULTS: Clinical trial data support the development and utility of digital therapeutics designed to deliver self-guided cognitive behavioral therapy and hypnotherapy for the treatment of IBS. CONCLUSIONS: BGBTs are effective, guideline-recommended treatments for IBS. Digital therapeutic devices offer accessible, cost-effective treatment options for delivery of adjunctive BGBT for the treatment of IBS. The decision to recommend digital BGBTs should be guided by careful patient assessment that includes mental health screening and risk assessment.

Predictors of parent self-monitoring patterns in a family-based behavioral weight loss treatment program.

OBJECTIVE: Self-monitoring dietary intake is a critical component of family-based intensive health behavior and lifestyle treatment for pediatric obesity, but adherence rates are often low. This study identifies predictors of parent self-monitoring rates during treatment. METHODS: A secondary analysis of parent self-monitoring data from a randomized controlled trial involving 150 parent-child dyads. Patterns of self-monitoring were identified using a latent class mixed model approach. Logistic regression analyses evaluated predictors of self-monitoring patterns. RESULTS: Latent class models identified two trajectory groups: a high consistent self-monitoring group and a low-decreasing self-monitoring group. When compared to parents in the low group, parents in the high group lost more weight throughout treatment. Children in the high group had a similar trajectory for weight loss; however, the groups were not statistically different. Higher levels of family chaos and poorer family problem-solving skills were associated with higher odds of being in the low group. CONCLUSION: This study identified two patterns of rates of parent self-monitoring, which were associated with parent weight loss and were differentiated by family chaos and poor problem-solving. These findings suggest that families with high levels of chaos and poor problem-solving could benefit from early intervention to improve outcomes in pediatric obesity treatment programs. TRIAL REGISTRATION: Clinicaltrials.gov Identifier: NCT01197443.

Psychotropic Medication Use and Polypharmacy Among Children and Adolescents Initiating Intensive Behavioral Therapy for Severe Challenging Behavior.

OBJECTIVE: To evaluate the prevalence, trends, and factors associated with psychotropic medication use and polypharmacy among children and adolescents initiating intensive behavioral therapy for severe challenging behavior over a 10-year period. STUDY DESIGN: In this retrospective observational study, we examined data from caregiver interviews and patient medical records on the number and types of psychotropic medications prescribed to patients initiating intensive behavioral therapy between January 1, 2013, and December 31, 2022. Trends in medication use and polypharmacy across the 10-year period were analyzed using regression analysis, while differences in demographics and clinical factors for patients with use and polypharmacy were analyzed using nonparametric statistical analysis with odds ratios presented for significant factors. RESULTS: Data from all 302 pediatric patients initiating intensive behavioral therapy across the 10-year period were analyzed. Among all patients and all years, 83.8% were taking at least 1 psychotropic medication and 68.2% experienced polypharmacy. There were no changes in the prevalence of use, mean number of medications taken, or polypharmacy across the 10-year period. Patients diagnosed with attention-deficit/hyperactivity disorder or anxiety disorder, as well as those exhibiting self-injurious behavior had higher use of psychotropic medication and polypharmacy and were taking more medications overall. CONCLUSIONS: Psychotropic medication use and polypharmacy were extremely high for children and adolescents with severe challenging behavior, but use and polypharmacy did not change over the 10-year period of data collection. Further research is needed to establish the generality of these findings to other regions of the US.

Gamified Closed-Loop Intervention Enhances Tic Suppression in Children: A Randomized Trial.

BACKGROUND: Gamification of behavioral intervention for tic disorders (TDs) potentially enhances compliance and offers key clinical advantages. By delivering immediate positive feedback upon tic-suppression, games may counteract negative reinforcement, which presumably contribute to tic consolidation by relieving uncomfortable premonitory urges. OBJECTIVES: We developed a gamified protocol (XTics), which leverages this potential by combining gamified tic-triggering with immediate feedback, and evaluated its clinical value in enhancing tic suppression. METHODS: XTics encompasses two conditions: Immediate and Contingent Reward (ICR), where game progression is contingent upon successful tic suppression, and Delayed Reward (DR), where game events' outcomes are random. Employing a randomized crossover design, 35 participants (aged 7-15 years) underwent daily gaming sessions over a week per condition. Improvements in our primary measures, including the inter-tic interval (ITI) and tic severity assessment by blinded evaluators (Yale Global Tic Severity-Total Tic Score [YGTSS-TTS], Rush), and parents (Parent Tic Questionnaire [PTQ]), were compared between ICR and DR, and assessed across conditions for the 4-week protocol. RESULTS: No participant voluntarily left the study before completing its two-phase protocol. As expected, ITI showed significantly larger improvement (Z = 4.19, P = 2.85 × 10-5) after ICR (1442 ± 2250%) versus DR (242 ± 493%) training, increasing at a higher pace (t(67) = 3.15, P = 0.0025). Similarly, Rush tic severity scores reduced more post-ICR versus DR (t(47) = 3.47, P = 0.002). We observed a clinically significant reduction of 25.69 ± 23.39% in YGTSS-TTS following a f4-week protocol including both conditions. Parent-reported tic severity decreased by 42.99 ± 31.69% from baseline to 3 months post-treatment. CONCLUSIONS: The combination of gamified tic-triggering with immediate and contingent rewards demonstrates a promising approach for enhancing treatment efficacy in TDs, boosting traditional therapeutic methods. © 2024 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

Pretreatment cognitive performance is associated with differential self-harm outcomes in 6 v. 12-months of dialectical behavior therapy for borderline personality disorder.

BACKGROUND: Recent findings suggest that brief dialectical behavior therapy (DBT) for borderline personality disorder is effective for reducing self-harm, but it remains unknown which patients are likely to improve in brief v. 12 months of DBT. Research is needed to identify patient characteristics that moderate outcomes. Here, we characterized changes in cognition across brief DBT (DBT-6) v. a standard 12-month course (DBT-12) and examined whether cognition predicted self-harm outcomes in each arm. METHODS: In this secondary analysis of 240 participants in the FASTER study (NCT02387736), cognitive measures were administered at pre-treatment, after 6 months, and at 12 months. Self-harm was assessed from pre-treatment to 2-year follow-up. Multilevel models characterized changes in cognition across treatment. Generalized estimating equations examined whether pre-treatment cognitive performance predicted self-harm outcomes in each arm. RESULTS: Cognitive performance improved in both arms after 6 months of treatment, with no between-arm differences at 12-months. Pre-treatment inhibitory control was associated with different self-harm outcomes in DBT-6 v. DBT-12. For participants with average inhibitory control, self-harm outcomes were significantly better when assigned to DBT-12, relative to DBT-6, at 9-18 months after initiating treatment. In contrast, participants with poor inhibitory control showed better self-harm outcomes when assigned to brief DBT-6 v. DBT-12, at 12-24 months after initiating treatment. CONCLUSIONS: This work represents an initial step toward an improved understanding of patient profiles that are best suited to briefer v. standard 12 months of DBT, but observed effects should be replicated in a waitlist-controlled study to confirm that they were treatment-specific.

Amygdala-related electrical fingerprint is modulated with neurofeedback training and correlates with deep-brain activation: proof-of-concept in borderline personality disorder.

BACKGROUND: The modulation of brain circuits of emotion is a promising pathway to treat borderline personality disorder (BPD). Precise and scalable approaches have yet to be established. Two studies investigating the amygdala-related electrical fingerprint (Amyg-EFP) in BPD are presented: one study addressing the deep-brain correlates of Amyg-EFP, and a second study investigating neurofeedback (NF) as a means to improve brain self-regulation. METHODS: Study 1 combined electroencephalography (EEG) and simultaneous functional magnetic resonance imaging to investigate the replicability of Amyg-EFP-related brain activation found in the reference dataset (N = 24 healthy subjects, 8 female; re-analysis of published data) in the replication dataset (N = 16 female individuals with BPD). In the replication dataset, we additionally explored how the Amyg-EFP would map to neural circuits defined by the research domain criteria. Study 2 investigated a 10-session Amyg-EFP NF training in parallel to a 12-weeks residential dialectical behavior therapy (DBT) program. Fifteen patients with BPD completed the training, N = 15 matched patients served as DBT-only controls. RESULTS: Study 1 replicated previous findings and showed significant amygdala blood oxygenation level dependent activation in a whole-brain regression analysis with the Amyg-EFP. Neurocircuitry activation (negative affect, salience, and cognitive control) was correlated with the Amyg-EFP signal. Study 2 showed Amyg-EFP modulation with NF training, but patients received reversed feedback for technical reasons, which limited interpretation of results. CONCLUSIONS: Recorded via scalp EEG, the Amyg-EFP picks up brain activation of high relevance for emotion. Administering Amyg-EFP NF in addition to standardized BPD treatment was shown to be feasible. Clinical utility remains to be investigated.

The immune phenotype of perinatal anxiety in an anxiety-focused behavioral intervention program in Pakistan.

BACKGROUND: Dysregulation of the immune system has been associated with psychiatric disorders and pregnancy-related complications, such as perinatal depression. However, the immune characteristics specific to perinatal anxiety remain poorly understood. In this study, our goal was to examine specific immune characteristics related to prenatal anxiety within the context of a randomized controlled trial designed to alleviate anxiety symptoms-the Happy Mother - Healthy Baby (HMHB) study in Rawalpindi, Pakistan. MATERIALS AND METHODS: Pregnant women (n = 117) were followed prospectively in the 1st, 2nd, and 3rd trimesters (T1, T2, T3) and at 6 weeks postpartum (PP6). Each visit included a blood draw and anxiety evaluation (as measured by the anxiety subscale of the Hospital Anxiety and Depression Scale - HADS -using a cutoff ≥ 8). We enrolled both healthy controls and participants with anxiety alone; those with concurrent depression were excluded. RESULTS: K-means cluster analysis revealed three anxiety clusters: Non-Anxiety, High and Consistent Anxiety, and Decreasing Anxiety. Principal components analysis revealed two distinct clusters of cytokine and chemokine activity. Women within the High and Consistent Anxiety group had significantly elevated chemokine activity across pregnancy (in trimester 1 (ß = 0.364, SE = 0.178, t = 2.040, p = 0.043), in trimester 2 (ß = 0.332, SE = 0.164, t = 2.020, p = 0.045), and trimester 3 (ß = 0.370, SE = 0.179, t = 2.070, p = 0.040) compared to Non-Anxiety group. Elevated chemokine activity was associated with low birthweight (LBW) and small for gestational age (SGA). CONCLUSION: Our findings reveal a unique pattern of immune dysregulation in pregnant women with anxiety in a Pakistani population and offer preliminary evidence that immune dysregulation associated with antenatal anxiety may be associated with birth outcomes. The dysregulation in this population is distinct from that in our other studies, indicating that population-level factors other than anxiety may play a substantial role in the differences found. (Clinicaltrials.gov # NCT04566861).

A positive affect intervention alters leukocyte DNA methylation in sexual minority men with HIV who use methamphetamine.

OBJECTIVE: This epigenomics sub-study embedded within a randomized controlled trial examined whether an evidenced-based behavioral intervention model that decreased stimulant use altered leukocyte DNA methylation (DNAm). METHODS: Sexual minority men with HIV who use methamphetamine were randomized to a five-session positive affect intervention (n = 32) or an attention-control condition (n = 21), both delivered during three months of contingency management for stimulant abstinence. All participants exhibited sustained HIV virologic control - an HIV viral load less than 40 copies/mL at baseline and six months post-randomization. The Illumina EPIC BeadChip measured leukocyte methylation of cytosine-phosphate-guanosine (CpG) sites mapping onto five a priori candidate genes of interest (i.e., ADRB2, BDNF, FKBP5, NR3C1, OXTR). Functional DNAm pathways and soluble markers of immune dysfunction were secondary outcomes. RESULTS: Compared to the attention-control condition, the positive affect intervention significantly decreased methylation of CpG sites on genes that regulate ß2 adrenergic and oxytocin receptors. There was an inconsistent pattern for the direction of the intervention effects on methylation of CpG sites on genes for glucocorticoid receptors and brain-derived neurotrophic factor. Pathway analyses adjusting for the false discovery rate (padj < 0.05) revealed significant intervention-related alterations in DNAm of Reactome pathways corresponding to neural function as well as dopamine, glutamate, and serotonin release. Positive affect intervention effects on DNAm were accompanied by significant reductions in the self-reported frequency of stimulant use. CONCLUSIONS: There is an epigenetic signature of an evidence-based behavioral intervention model that reduced stimulant use, which will guide the identification of biomarkers for treatment responses.

Can the delivery of behavioural support be improved in the NHS England Low-Calorie Diet Programme? An observational study of behaviour change techniques.

BACKGROUND: Previous research has illustrated a drift in the fidelity of behaviour change techniques (BCTs) during the design of the pilot NHS England Low-Calorie Diet (NHS-LCD) Programme. This study evaluated a subsequent domain of fidelity, intervention delivery. Two research questions were addressed: (1) To what extent were BCTs delivered with fidelity to providers programme plans? (2) What were the observed barriers and facilitators to delivery? METHODS: A mixed-methods sequential explanatory design was employed. Remote delivery of one-to-one and group-based programmes were observed. A BCT checklist was developed using the BCT Taxonomy v1; BCTs were coded as present, partially delivered, or absent during live sessions. Relational content analysis of field notes identified observed barriers and facilitators to fidelity. RESULTS: Observations of 122 sessions across eight samples and two service providers were completed. Delivery of the complete NHS-LCD was observed for five samples. Fidelity ranged from 33% to 70% across samples and was higher for group-based delivery models (64%) compared with one-to-one models (46%). Barriers and facilitators included alignment with the programme's target behaviours and outcomes, session content, time availability and management, group-based remote delivery, and deviation from the session plan. CONCLUSIONS: Overall, BCTs were delivered with low-to-moderate fidelity. Findings indicate a dilution in fidelity during the delivery of the NHS-LCD and variation in the fidelity of programmes delivered across England. Staff training could provide opportunities to practice the delivery of BCTs. Programme-level changes such as structured activities supported by participant materials and with sufficient allocated time, might improve the delivery of BCTs targeting self-regulation.

The NHS Low-Calorie Diet Digital Programme: Fidelity of behaviour change technique delivery.

AIMS: NHS England commissioned independent service providers to deliver the NHS Low-Calorie Diet Programme pilot. Previous research has illustrated a drift in the fidelity of behaviour change techniques (BCTs) during the delivery of the programme provided through face-to-face group or one-to-one behavioural support. The aim of this study was to assess the delivery fidelity of the BCT content in the digital delivery of the programme. METHODS: Online, app chat and phone call support content was coded using The Behaviour Change Technique Taxonomy. BCTs delivered by each service provider (N = 2) were calculated and compared to the BCTs specified in the NHS service specification and those specified in the providers' programme plans. RESULTS: Between 78% and 83% of the BCTs identified in the NHS service specification were delivered by the service providers. The fidelity of BCT delivery to those specified in providers' programme plans was 60%-65% for provider A, and 82% for provider B. CONCLUSIONS: The BCT content of the digital model used in the NHS-LCD programme adhered well to the NHS service specification and providers' plans. It surpassed what has been previously observed in face-to-face services provided through group or one-on-one behavioural support models.

Understanding how self-management education and support programmes for type 2 diabetes are expected to change behaviour: A document analysis of two programmes.

AIMS: Attendance at diabetes self-management education and support (DSMES) programmes for type 2 diabetes is associated with positive outcomes, but the impact on some outcomes is inconsistent and tends to decline over time. Understanding the active ingredients of effective programmes is essential to optimise their effectiveness. This study aimed to (1) retrospectively identify behaviour change techniques (BCTs), mechanisms of action (MoAs) and intervention functions in two DSMES programmes, the Community-Oriented Diabetes Education and the Diabetes Education and Self-Management for Ongoing and Newly Diagnosed and (2) examine variation in content between programmes. METHODS: A multiple case study approach, including a documentary analysis of the programme materials, was conducted. Materials were coded using the BCT Taxonomy v1, the Mode of Delivery Ontology v1 and the Intervention Source Ontology v1. The Behaviour Change Wheel guidance and the Theory and Techniques tool were used to identify intervention functions and MoAs. Programme stakeholders provided feedback on the findings. RESULTS: Thirty-four BCTs were identified across the programmes, with 22 common to both. The identified BCTs were frequently related to 'goals and planning', 'feedback and monitoring' and 'natural consequences'. BCTs were linked with 15 MoAs, predominantly related to reflective motivation ('beliefs about capabilities' and 'beliefs about consequences') and psychological capability ('knowledge'). BCTs served six intervention functions, most frequently 'education', 'enablement' and 'persuasion'. CONCLUSIONS: Although both programmes included several BCTs, some BCTs were rarely or never used. Additional BCTs could be considered to potentially enhance effectiveness by addressing a wider range of barriers.

Policy Interventions to Enhance Medical Care for People With Obesity in the United States-Challenges, Opportunities, and Future Directions.

Policy Points Health policymakers have insufficiently addressed care for people with obesity (body mass index ≥ 30 kg/m2) in the United States. Current federal policies targeting obesity medications reflect this unfortunate reality. We argue for a novel policy framework to increase access to effective obesity therapeutics and care, recognizing that, though prevention is critical, the epidemic proportions of obesity in the United States warrant immediate interventions to augment care. Reducing barriers to and improving the quality of existing anti-obesity medications, intensive behavioral therapy, weight management nutrition and dietary counseling, and bariatric surgery are critical. Moreover, to ensure continuity of care and patient-clinician trust, combating physician and broader weight stigma must represent a central component of any viable obesity care agenda.

Which terms should be used to describe medications used in the treatment of seizure disorders? An ILAE position paper.

A variety of terms, such as "antiepileptic," "anticonvulsant," and "antiseizure" have been historically applied to medications for the treatment of seizure disorders. Terminology is important because using terms that do not accurately reflect the action of specific treatments may result in a misunderstanding of their effects and inappropriate use. The present International League Against Epilepsy (ILAE) position paper used a Delphi approach to develop recommendations on English-language terminology applicable to pharmacological agents currently approved for treating seizure disorders. There was consensus that these medications should be collectively named "antiseizure medications". This term accurately reflects their primarily symptomatic effect against seizures and reduces the possibility of health care practitioners, patients, or caregivers having undue expectations or an incorrect understanding of the real action of these medications. The term "antiseizure" to describe these agents does not exclude the possibility of beneficial effects on the course of the disease and comorbidities that result from the downstream effects of seizures, whenever these beneficial effects can be explained solely by the suppression of seizure activity. It is acknowledged that other treatments, mostly under development, can exert direct favorable actions on the underlying disease or its progression, by having "antiepileptogenic" or "disease-modifying" effects. A more-refined terminology to describe precisely these actions needs to be developed.

Single institution experience with efgartigimod in patients with myasthenia gravis: Patient selection, dosing schedules, treatment response, and adverse events.

INTRODUCTION/AIMS: Efgartigimod is a neonatal Fc receptor blocker and was the first approved medication in its class for the treatment of generalized myasthenia gravis (gMG). As a novel therapy, little is known about the use of efgartigimod in clinical practice. This study aims to describe how efgartigimod is being incorporated into the current therapeutic landscape of MG. METHODS: We reviewed the charts of 17 patients with gMG treated with efgartigimod at the University of Pennsylvania between January 2022 and June 2023. RESULTS: Efgartigimod was selected mainly for patients who were treatment refractory, had side effects to other treatments, and/or required quick improvement in their symptoms. All patients had been previously treated with at least one medication for MG and had an average baseline Myasthenia Gravis Activities of Daily Living (MG-ADL) score of 9.1. The patients treated with efgartigimod improved their MG-ADL score by an average of 5.5 points at 3 months (p < .001) and 7.1 points by 6 months (p < .001). Forty percent of patients achieved minimal symptom expression. Adverse events (AEs) were reported in 43.7% of patients on efgartigimod, the most common being mild infection (urinary tract infection and thrush). There were no serious AEs. DISCUSSION: This study found efgartigimod to be efficacious, well tolerated, and safe in patients with MG. Efgartigimod should be considered as an add-on therapy, a bridge therapy, or as a monotherapy if patients have difficulty tolerating other treatments.

Practitioner Review: A core competencies perspective on the evidence-based treatment of child conduct problems.

BACKGROUND: The effective treatment of child conduct problems is understood to rely on a range of therapist competencies, yet these have rarely been an explicit focus of research. In this practitioner review, we examine core competencies for the delivery of evidence-based parenting interventions for conduct problems in early-to-middle childhood. These are examined in light of research into the common elements shared by these interventions, literature regarding common challenges in these interventions, and conceptualisations of such competencies in other fields of mental health. METHODS: We report on the development of a novel consensus-based model of core competencies for evidence-based practice in this field, based on consultation with an international expert panel. This includes competencies as they apply to complex presentations of conduct problems. RESULTS: Despite considerable variation among widely disseminated programmes in terms of content, format and skills-training practices, there is strong consensus among practitioners regarding core competencies. These relate to three broad domains: (a) generic therapeutic competencies; (b) parenting intervention competencies; (c) specific parenting skills/techniques. CONCLUSIONS: Practitioners working with conduct problems, particularly complex presentations thereof, require competencies for engaging not only mothers, but fathers and diverse/non-traditional caregivers and other stakeholders, in evidence-based parenting interventions. Moreover, the successful delivery of these interventions necessitates competencies that extend beyond behaviour management and encompass broader aspects of the family system and the wider ecology of the child.

Commentary: Holy grails, personalized medicine, and the public health burden of psychopathology - a reflection on Ahuvia et al. (2023).

Clinical psychology and psychiatry have many 'holy grails' or research findings that are widely sought after but remain elusive. The use of machine learning (ML) models for treatment selection is one of these holy grails. Ahuvia et al. (Journal of Child Psychology and Psychiatry, 2023) recently analyzed a large trial (n = 996) of two distinct single-session interventions (SSIs) for internalizing distress and found little evidence that an ML model could predict differential treatment response. I discuss potential avenues for advancing SSI research. One avenue is the dissemination and implementation of SSIs, including how they interact with other treatments in routine care. Quantifying and critically questioning the promises of holy grails like ML models is sorely needed. Using simulation modeling to evaluate the relative merits of using ML models for treatment selection or using SSIs versus other treatment strategies may be another path forward.

A predictive model for medium-term weight loss response in people with type 2 diabetes engaging in behavioural weight management interventions.

AIMS: To develop and evaluate prediction models for medium-term weight loss response in behavioural weight management programmes. MATERIALS AND METHODS: We conducted three longitudinal analyses using the Action for HEalth in Diabetes (LookAHEAD) trial, Weight loss Referrals for Adults in Primary care (WRAP) trial, and routine data from the National Health Service Greater Glasgow and Clyde Weight Management Service (NHS-GGCWMS). We investigated predictors of medium-term weight loss (>5% body weight) over 3 years in NHS-GGCWMS and, separately, predictors of weight loss response in LookAHEAD over 4 years. We validated predictors in both studies using WRAP over 5 years. Predictors of interest included demographic and clinical variables, early weight change in-programme (first 4 weeks) and overall in-programme weight change. RESULTS: In LookAHEAD and WRAP the only baseline variables consistently associated with weight loss response were female sex and older age. Of 1152 participants in NHS-GGCWMS (mean age 57.8 years, 60% female, type 2 diabetes diagnosed for a median of 5.3 years), 139 lost weight over 3 years (12%). The strongest predictor of weight loss response was early weight change (odds ratio 2.22, 95% confidence interval 1.92-2.56) per 1% weight loss. Losing 0.5% weight in the first 4 weeks predicted medium-term weight loss (sensitivity 89.9%, specificity 49.5%, negative predictive value 97.3%). Overall in-programme weight change was also associated with weight loss response over 3 years in NHS-GGCWMS and over 5 years in WRAP. CONCLUSIONS: Not attaining a weight loss threshold of 0.5% early in weight management programmes may identify participants who would benefit from alternative interventions.