Evidence-based Treatment Options Research Articles

Early treatment for children with mental health problems and genetic conditions through a parenting intervention (The GAP): study protocol for a pragmatic randomized controlled trial

BackgroundChildren with genetic conditions are at increased risk for mental health and neurodevelopmental problems, often accompanied by significant parental distress. Genetic and family factors can impact children and parents’ mental health. Early parenting interventions, like the Incredible Years® programs, have demonstrated to improve parental distress and children’s mental health. The recent version for young children with language delays or autism spectrum disorder (IY-ASLD®) has shown to be feasible and effective to support parents in their children’s developmental trajectories. The effectiveness of treatments for children with genetic conditions and neurodevelopmental problems is largely unexplored, leaving significant gaps in evidence-based options. Clinicians lack guidance, especially when patients exhibit language or social communication impairments but do not meet diagnostic criteria for a full-blown autism spectrum disorder (ASD). We aim to fill this gap, providing evidence on the feasibility and effectiveness of the IY-ASLD® intervention for such patients.MethodsWe designed a prospective multicenter pragmatic randomized controlled trial including approximately 68 children aged 3 to 7 years, recruited from three tertiary care reference hospitals. Inclusion criteria will necessitate genetic confirmation of a neurodevelopmental disorder along with language, communication, or socialization difficulties. Individuals with an ASD diagnosis will be excluded. All subjects are included in a territorial register for rare conditions (ReMin, Registre de Malalties Minoritàries de Catalunya). Families will randomly be assigned to the intervention or the control group. The intervention will be held online by clinical psychologists and child and adolescent psychiatrists.DiscussionOur group has recently piloted the online implementation of the IY-ASLD® intervention for the first time in Spain, for parents of children with language delays, socialization difficulties, or ASD, but not genetically determined. Our multicenter research consortium is well-positioned to recruit patients with rare conditions and implement efficient treatment pathways within the National Health System. Given the geographical dispersion of families affected by rare conditions, the online format offers logistical advantages and improved therapy access, enhancing homogeneity across all patients. The results of this study will inform clinicians and policymakers about evidence-based treatment options for this vulnerable and overlooked group of young children.Trial registrationClinicalTrials.gov NCT06125093. Date of registration: first submitted 2023–10-23; first posted 2023–11-09. URL of trial registry record.

Surgical systematic reviews: best available evidence or disposable waste?

Abstract Evidence-based medicine demands treatment options for patients to be based on the current best available evidence. Systematic reviews (SRs) with meta-analyses allow surgeons to make therapeutical decisions in accordance with the highest level of evidence. Also, high-quality SRs support physicians to challenge the colossal amount of new research data created daily. The systematic review working group of the Study Center of the German Society of Surgery (SDGC) has created specific methodological literature regarding surgical SRs, giving recommendations to assess critical risk of bias and to prevent the creation of SRs that do not provide any new insights to the field. SRs should only be considered if there is new clinically relevant data available that allows the SR to create novel evidence. To address the dilemma of new SRs generated without adding new evidence, living systematic reviews and evidence mapping represent an innovative approach, in which SRs are regularly updated with new research data.

Treatment of bipolar depression: clinical practice vs. adherence to guidelines-data from a Bavarian drug surveillance project.

Pharmacotherapy of bipolar depression (BPD) is confronted with major clinical challenges, like limited evidence-based treatment options, regular cases of treatment resistance, and risk of treatment-emergent affective switches. Medical guidelines can support practitioners to make decisions based on current scientific evidence. The objective of this study is to evaluate to what extent recommendations of the 2019 German S3 guidelines "Diagnosis and Treatment of Bipolar Disorders" are reflected in clinical practice in inpatient treatment. We conducted a descriptive analysis of prescription numbers in 2,627 patients with BPD in a naturalistic inpatient setting analyzing data from the ongoing Bavarian multicenter drug safety project Pharmaco-Epidemiology and Vigilance (Pharmako-EpiVig) from the years 2014-2022. Of the patients, 38% were not administered any drug explicitly recommended for treatment of BPD, that is, quetiapine, lamotrigine, carbamazepine, or olanzapine. Only 6% of the patients received monotherapy with one of those drugs. Of the patients, 34% were administered ≥4 psychotropic drugs simultaneously. Patients received 912 different therapy regimens of mono or combination therapy with mood stabilizers (MS), atypical antipsychotics (AAP), and antidepressants. Of the patients, 72% received an antidepressant and 6% without concomitant prescription of an AAP or MS. Prescription rates of venlafaxine (21% to 14%) and tricyclic antidepressants (9% to 6%) decreased significantly from the first (2014-2016) to the last (2020-2022) observed time period. Of the patients, 60% received an MS. Prescription rate of valproate (22% to 14%) decreased significantly, while lithium prescription increased significantly (29% to 35%). Of the patients, 71% were administered an AAP. Quetiapine was the most prescribed drug overall (43%). Only two patients were administered a combination of olanzapine and fluoxetine. Our results demonstrate a substantial gap between guideline recommendations and current clinical practice. The remarkable heterogeneity in treatment regimens, with no discernible dominant treatment approach, is in part a reflection of the complexity of bipolar disorder but also substantiates the need of comprehensive recommendations regarding combination therapies. Increase in lithium prescription is an encouraging development due to its unique efficacy in maintenance treatment. To improve the quality of clinical practice guideline implementation, more randomized controlled trials shouldbeconducted in the future to prospectively investigate different implementation strategies.

The TOTAL trial for weight management participation: A randomized controlled trial protocol

BackgroundThe three types of evidence-based treatment options for adults with overweight and obesity – behavioral weight management, anti-obesity medications (AOM), and bariatric surgery – are underutilized in the Veterans Health Administration (VHA) system. Our objective in this manuscript is to describe the study protocol for an adequately powered randomized controlled trial (RCT) of a behavioral intervention: TOTAL (Teaching Obesity Treatment Options to Adult Learners) to increase patient uptake of obesity treatment. MethodsIn this multi-site, parallel, RCT, eligible Veterans with a body mass index [BMI] ≥ 27 who had not received obesity treatment within the past 12 months were randomly assigned to TOTAL or usual care. TOTAL involves watching an 18-min video that highlights obesity health risks, pros/cons of all three evidence-based obesity treatments, and expected treatment outcomes. It also includes motivational sessions delivered via televideo at 2 weeks, 6 months, and 12 months after the video (target n = 494 participants). The primary outcome is initiation of behavioral weight management treatment within 18 months of randomization. Secondary outcomes include sustained behavioral weight management treatment, initiation of AOM, bariatric surgery referral, and weight change across 18 months. ConclusionTOTAL, which seeks to increase delivery of weight management treatment within the largest integrated health system in the U.S., combines patient education with motivational interviewing components. If efficacious in this trial, further evaluation of intervention effectiveness and implementation throughout the VHA and other healthcare systems would be warranted.

AAPM&R consensus guidance on spasticity assessment and management.

The American Academy of Physical Medicine and Rehabilitation (AAPM&R) conducted a comprehensive review in 2021 to identify opportunities for enhancing the care of adult and pediatric patients with spasticity. A technical expert panel (TEP) was convened to develop consensus-based practice recommendations aimed at addressing gaps in spasticity care. To develop consensus-based practice recommendations to identify and address gaps in spasticity care. The Spasticity TEP engaged in a 16-month virtual meeting process, focusing on formulating search terms, refining research questions, and conducting a structured evidence review. Evidence quality was assessed by the AAPM&R Evidence, Quality and Performance Committee (EQPC), and a modified Delphi process was employed to achieve consensus on recommendation statements and evidence grading. The Strength of Recommendation Taxonomy (SORT) guided the rating of individual studies and the strength of recommendations. The TEP approved five recommendations for spasticity management and five best practices for assessment and management, with one recommendation unable to be graded due to evidence limitations. Best practices were defined as widely accepted components of care, while recommendations required structured evidence reviews and grading. The consensus guidance statement represents current best practices and evidence-based treatment options, intended for use by PM&R physicians caring for patients with spasticity. This consensus guidance provides clinicians with practical recommendations for spasticity assessment and management based on the best available evidence and expert opinion. Clinical judgment should be exercised, and recommendations tailored to individual patient needs, preferences, and risk profiles. The accompanying table summarizes the best practice recommendations for spasticity assessment and management, reflecting principles with little controversy in care delivery.

Hemodynamic effects of treatment with the ketone body 3-hydroxybutyrate in cardiogenic shock - a randomized, controlled, assessor-blinded porcine trial

Abstract Funding Acknowledgements Type of funding sources: Foundation. Main funding source(s): Novo Nordisk Foundation Independent Research Fund DK. Background Cardiogenic shock (CS) is a life-threatening disease with limited evidence-based treatment options. Treatment with the ketone body 3-hydroxybutyrate (3-OHB) induces favorable hemodynamic effects in patients with heart failure and CS. It increases cardiac output (CO) and left ventricular (LV) ejection fraction (LVEF) while reducing cardiac filling pressures. Yet, whether treatment with 3-OHB demonstrates beneficial hemodynamic effects during acute ischemic LV stunning in CS must be fully elucidated. This study aimed to examine the acute hemodynamic effects and underlying cardiovascular mechanisms of 3-OHB infusion in the acute phase of LV stunning in a porcine model of ischemic CS. Methods Sixteen 60-kg-pigs were included in a randomised, controlled, assessor-blinded crossover study. CS was achieved by repeated, slow injections of microspheres into the left main coronary artery. CS was defined as ≥30% reduction in CO and/or mixed venous saturation (SVO2). Upon the onset of CS, following a one-hour no-touch period, the pigs received an infusion with 3-OHB and isoosmolar, isovolumic saline (control) for two hours each in random order. Infusion periods were separated by a one-hour washout period. CO (primary endpoint), SVO2, and pulmonary pressures were measured by right heart catheterization. A pressure-volume admittance catheter was placed in the LV to measure LVEF, contractility (the slope of end systolic pressure-volume relationship, denoded as Ees), afterload (arterial elastance, Ea, and end-systolic pressure, ESP), preload (end-diastolic volume, EDV), and work-related parameters. Results CS was successfully achieved in all randomized animals. CO decreased by 36% from 4.5±1.0 L/min to 2.9±0.5 L/min (p<0.001) and SVO2 decreased by 31% from 55.0±8.1% to 37.7±5.8% (p<0.001). During 3-OHB infusion, CO increased by 0.9 L/min (95% CI: 0.4 to 1.3 L/min) compared with the control infusion. In parallel, Ees increased by 0.22 mmHg/mL (95% CI: 0.08 to 0.37 mmHg/mL), LVEF improved by 7 percentage points (95%CI: 1 to 13 percentage points) and cardiac efficiency increased by 6 percentage points (95%CI: 1 to 11 percentage points). Systemic vascular resistance was decreased by 479 dyn*cm5 (95% CI: 736 to 221 dyn*cm5); Ea and ESP remained similar. Heart rate was increased during 3-OHB infusion by 21 bpm (95% CI: 11 to 32 bpm) while no significant change was observed in stroke volume (p=0.6). Conclusion Treatment with 3-OHB improved CO, LVEF, and cardiac efficiency in CS. The increase was mediated through increased ventricular contractility and decreased peripheral resistance accompanied by increased heart rate.CO, Ees and cardiac efficiencyEndpoint parameters during 3-OHB

Protocol for a systematic mapping review of surgical and pharmacological interventions for the treatment of trigeminal neuralgia.

Trigeminal neuralgia is a painful neuropathic disorder characterized by sudden electric shock-like pain that significantly impacts patients' quality of life. Multiple treatment alternatives are available, including medical and surgical options but establishing the optimal course of action can be challenging. To enhance clinical decision-making for trigeminal neuralgia treatment, it is imperative to organize, describe and map the available systematic reviews and randomized trials. This will help identify the best treatment alternatives supported by evidence and acknowledge potential knowledge gaps where future research is needed. This systematic mapping review aims to provide up-to-date evidence on the different surgical and pharmacological treatment alternatives used for trigeminal neuralgia. A search will be systematically conducted on the Epistemonikos database to identify potentially eligible systematic reviews. Additionally, a search will be made in PubMed, CENTRAL, and EBSCO to identify randomized controlled trials assessing pharmacological and surgical treatment interventions for trigeminal neuralgia. Two independent reviewers will screen and select the studies. Data on the different treatment alternatives and reported outcomes in the included studies will be extracted using standardized forms. Following extraction, descriptive statistical methods will be used to analyze the data. The final output of this study will include an evidence map that will illustrate the connections between different treatments and their respective outcomes, providing a clear depiction of the evidence landscape. This study expects to map, describe and assess the methodological quality of the available systematic reviews and trials on pharmacological interventions and neurosurgical procedures for treating trigeminal neuralgia. It will present the results in an evidence map that organizes the available evidence based on their different interventions and outcomes. This evidence map will serve as a visual tool to assist healthcare professionals and patients to understand evidence-based treatment options and their implications for managing this medical condition. La neuralgia del trigémino es un trastorno neuropático doloroso caracterizado por un dolor súbito y agudo, similar a una descarga eléctrica, que impacta significativamente en la calidad de vida. Dada la variedad de tratamientos disponibles, médicos y quirúrgicos, es crucial organizar y mapear la evidencia proveniente de revisiones sistemáticas y ensayos clínicos para orientar las decisiones clínicas. Esto permite identificar tratamientos respaldados por evidencia y señalar áreas de investigación futura. El propósito de esta revisión sistemática de mapeo es proporcionar una visión actualizada de la evidencia existente en relación con las diversas opciones de tratamiento quirúrgico y farmacológico empleadas en el manejo de la neuralgia del trigémino. Se realizará una búsqueda sistemática en la base de datos Epistemonikos para identificar potenciales revisiones sistemáticas. Adicionalmente, se buscará en PubMed, CENTRAL y EBSCO ensayos clínicos aleatorizados que evalúen intervenciones de tratamiento farmacológico y quirúrgico para la neuralgia del trigémino. Dos revisores independientes cribarán y seleccionarán los estudios. Se extraerán datos sobre las diferentes alternativas de tratamiento y los resultados reportados en los estudios incluidos utilizando formularios estandarizados. Tras la extracción, se utilizarán métodos estadísticos descriptivos para analizar los datos. El producto final de este estudio incluirá un mapa de evidencia que ilustrará las conexiones entre los diferentes tratamientos y sus respectivos resultados, proporcionando una representación clara del panorama de la evidencia. Los resultados que se extraerán de este mapeo sistemático incluyen identificar y describir las diferentes alternativas, tanto farmacológicas como quirúrgicas, que existen para el tratamiento de la neuralgia del trigémino. Además, se planea presentar un mapa de evidencia que se basará en los ensayos clínicos aleatorizados y revisiones sistemáticas, el cual mostrará la evidencia de manera organizada entre las diferentes intervenciones y sus desenlaces. Este mapa de evidencia servirá como una herramienta visual que ayudará a los profesionales de la salud y los pacientes a comprender mejor las opciones de tratamiento respaldadas por la evidencia y sus consecuencias en el manejo de esta condición médica.

Deep Brain Stimulation for Primary Refractory Tinnitus: A Systematic Review.

tinnitus is a common and often debilitating condition with limited evidence-based treatment options. Deep brain stimulation (DBS) is an approved treatment modality for certain neurological conditions; its experimental use as a treatment modality for severe tinnitus is novel and beginning to show promise. This systematic review focuses on the current evidence for the safety and efficacy of DBS for treatment of refractory tinnitus. a systematic search in PubMed and EMBASE was performed to identify peer-reviewed studies on DBS of non-cortical structures for the primary indication of tinnitus treatment. Three studies were identified as meeting these criteria, one of which had two related sub-studies. seven patients with available data who underwent DBS for tinnitus were identified. DBS targets included nucleus accumbens (NAc), ventral anterior limb of the internal capsule (vALIC), caudate nucleus, and the medial geniculate body (MGB) of the thalamus. All studies used the Tinnitus Functional Index (TFI) as a primary outcome measure. DBS of the caudate was most commonly reported (n = 5), with a mean TFI improvement of 23.3 points. Only one subject underwent DBS targeting the NAc/vALIC (extrapolated TFI improvement 46.8) and one subject underwent DBS targeting the MGB (TFI improvement 59 points). DBS is a promising treatment option for refractory subjective tinnitus, with early data, from small patient cohorts in multiple studies, suggesting its safety and efficacy. Further studies with a larger patient population are needed to support this safety and efficacy before implementing this treatment to daily practice.

Prevalence and predictors of medication for opioid use disorder among reproductive-aged women

BackgroundWomen of reproductive age would benefit from treatment of opioid use disorder (OUD) prior to pregnancy to improve maternal and infant outcomes. In this study, we aimed to identify the prevalence of medication for OUD (MOUD) and characterize correlates of MOUD receipt among 12–49-year-old women with OUD seeking treatment in publicly funded substance use disorder treatment programs at the time of their first treatment episode. MethodsThis cross-sectional study explores the demographic and clinical characteristics of women of reproductive age with OUD receiving publicly funded substance use treatment services. We used data from the concatenated 2015–2021 Treatment Episode Data Set–Admissions (TEDS-A), which documents demographic and clinical characteristics of patient admissions to publicly funded substance use treatment services in the United States. ResultsIn the sample of females aged 12–49 with no prior treatment admissions and primary OUD (n=325,512), 40.53% received MOUD (n=131,930), including 39.40% of non-pregnant women (n=115,315) and 52.79% of pregnant women (n=8423). Pregnant women had significantly higher odds of receiving MOUD (aOR = 2.42, 95%CI: 2.30, 2.54) compared to non-pregnant women. Non-white race, treatment setting, and treatment self-referral were also associated with higher levels of MOUD. ConclusionsWe identified a significant unmet need among both pregnant and non-pregnant women with OUD seeking care in publicly funded treatment clinics. While women who are pregnant are significantly more likely to receive evidence-based treatment with MOUD, still 47.21% of pregnant women did not receive MOUD. All reproductive-aged women with OUD should be offered evidence-based treatment options, including MOUD.

State of the Science: The Unified Protocol for Transdiagnostic Treatment of Emotional Disorders

Before the development of the Unified Protocol for the Transdiagnostic Treatment of Emotional Disorders (UP), evidence-based treatment options for commonly co-occurring anxiety, mood, and related disorders consisted of numerous single-disorder protocols that shared many similarities, reflecting the overlap among these disorders themselves. The UP distilled common elements of cognitive behavioral protocols into a unified intervention directly targeting core temperamental features underlying these disorders of emotion, namely neuroticism and associated emotion dysregulation. The UP has since become a leading “transdiagnostic” treatment for emotional disorders, which is now available in several formats (e.g., individual, group, digital) and has accumulated a strong evidence base, leading to international implementation. There is now also research evidence that the UP can be flexibly applied to a range of clinical presentations, including borderline personality disorder, trauma- and stressor-related disorders, eating disorders, alcohol use disorder, and comorbid chronic physical health conditions. Yet additional research is needed to evaluate the UP in routine clinical settings, with more heterogeneous patient populations, and under circumstances that mirror actual clinical practice. Thus, we must also continue to explore the benefits of large-scale UP training initiatives and implementation in major healthcare systems.

Long-Term Trends in Decisional Regret Among Men with Localized Prostate Cancer

Purpose: There are several evidence-based treatment options for localized prostate cancer (PCa). Decisional regret (DR), or regret based on the treatment a patient chooses, is poorly understood in patients with PCa. We describe long-term trends in DR for patients with localized PCa and factors associated with regret. Materials and Methods: We surveyed an established prospective cohort of patients with PCa in North Carolina who were diagnosed between 2011 and 2013. DR was assessed by a validated instrument at 12, 24, 36, 48, 60, 72, 84, and 120 months after treatment or active surveillance (AS). We estimated the overall trend of DR and examined how DR was associated with treatment and demographic data using generalized linear mixed-model regressions. Results: The sample included 1456 men. At 12 months, 88% (n = 1100) of patients did not regret their treatment. With all time points included, DR increased slightly in early months and the speed of increase slowed over time (P = .003 for time, .02 for time-squared). Divorced men were more likely to have DR compared with married men (odds ratio [OR] = 1.5; 95% confidence interval [CI] 0.9-2.7). African Americans (OR = 1.5; 95% CI 1.0-2.1) and those who underwent surgery had significantly more regret (14.6%; 95% CI 11.3-18.0, P = .01) than patients of other racial groups or who underwent AS, respectively. Conclusions: Although DR is low overall in this cohort, being married and AS were associated with lower regret than those who were not married or who underwent surgery, respectively. Understanding factors that affect DR can guide urologists to more effectively direct resources and counseling.

Effect of Sodium-Glucose Cotransporter 2 Inhibitors in Adults With Congenital Heart Disease

BackgroundHeart failure (HF) is the principal cause of morbidity and mortality in adults with congenital heart disease (ACHD). Robust evidence-based treatment options are lacking. ObjectivesThis study aims to evaluate the safety, tolerability, and short-term HF-related effects of sodium-glucose cotransporter 2 inhibitors (SGLT2i) in a real-world ACHD population. MethodsAll patients with ACHD treated with SGLT2i in 4 European ACHD centers were included in this retrospective study. Data were collected from 1 year before starting SGLT2i to the most recent follow-up. Data on side effects, discontinuation, mortality, and hospitalizations were collected. ResultsIn total, 174 patients with ACHD were treated with SGLT2i from April 2016 to July 2023. The mean age was 48.7 ± 15.3 years, 72 (41.4%) were female, and 29 (16.7%) had type 2 diabetes mellitus. Ten (5.7%) patients had mild, 75 (43.1%) moderate, and 89 (51.1%) severe congenital heart disease. HF was the most frequent starting indication (n = 162, 93.1%), followed by type 2 diabetes (n = 11, 6.3%) and chronic kidney disease (n = 1, 0.6%). At median follow-up of 7.7 months (Q1-Q3: 3.9–13.2 months), 18 patients (10.3%) reported side effects, 12 (6.9%) permanently discontinued SGLT2i, and 4 (2.3%) died of SGLT2i-unrelated causes. A significant reduction in the HF hospitalization rate was observed from 6 months before to 6 months after starting SGLT2i (relative rate = 0.30; 95% CI: 0.14-0.62; P = 0.001). ConclusionsSGLT2i generally seem safe, well-tolerated, and potentially beneficial in patients with ACHD. SGLT2i was associated with a 3-fold reduction in the 6-month HF hospitalization rate. These results warrant prospective randomized investigation of the potential benefits of SGLT2i for patients with ACHD.

Experts' perspectives on the impact of visual impairment and comorbid mental disorders on functioning in essential life domains.

Visual impairment (VI) with comorbid mental disorders (MDs) are expected to have a major impact on people's daily functioning, for which tailored support is needed. However, this has been barely investigated. Therefore, this study aimed to (1) determine the impact of VI and comorbid MDs on functioning in essential life domains, (2) gain insight into best-practices that are currently used to support this target group, and (3) determine strategies to optimize care in the future. A four-step qualitative Delphi method was used to obtain input from 31 Dutch professionals who work with this target group (84% female, mean age 46 years, on average 11 years of experience in working with the target group). The Self-Sufficiency Matrices were used to determine the impact on various aspects of daily living, for people with VI and (1) autism spectrum disorder, (2) psychotic disorders, (3) obsessive-compulsive disorder, (4) antisocial personality disorder, (5) borderline personality disorder, (6) dependent personality disorder. Experts describe a frail and vulnerable population, in which the VI and MD often have a cumulative negative impact on people's physical and mental health. People frequently experience anxiety, depression, fatigue and sleep disturbances. Also, many tend to neglect self-care and substance abuse is common. They often experience difficulty in trusting others while at the same time being dependent on them. Social interaction and relationships are complicated because of communication restrictions (e.g. no facial recognition) and social incompetence or withdrawal. Experts advise taking transdiagnostic factors into account, using evidence-based psychological treatment options based on an intermittent approach, and offering multidisciplinary care. They stress the importance of building trust, showing patience and empathy, stimulating empowerment, involving the informal network and building on positive experiences. VI and comorbid MD have a major impact on people's daily functioning on a mental, physical, social and environmental level. This study provides insight into best-practices to support this target group. According to experts, more research is needed which could be aimed at investigating tailored diagnostic approaches and treatment options and include clients' perspectives.

The Management of Phaeochromocytomas and Paragangliomas in the Era of Precision Medicine: Where Are We Now? Evidence-Based Systemic Treatment Options and Future Cluster Oriented Perspectives.

Pheochromocytomas (PCCs) and Paragangliomas (PGLs), commonly known as PPGLs to include both entities, are rare neuroendocrine tumors that may arise in the context of hereditary syndromes or be sporadic. However, even among sporadic PPGLs, identifiable somatic alterations in at least one of the known susceptibility genes can be detected. Therefore, about 3/4 of all PPGL patients can be assigned to one of the three molecular clusters that have been identified in the last years with difference in the underlying pathogenetic mechanisms, biochemical phenotype, metastatic potential, and prognosis. While surgery represents the mainstay of treatment for localized PPGLs, several therapeutic options are available in advanced and/or metastatic setting. However, only few of them hinge upon prospective data and a cluster-oriented approach has not yet been established. In order to render management even more personalized and improve the prognosis of this molecularly complex disease, it is undoubtable that genetic testing for germline mutations as well as genome profiling for somatic mutations, where available, must be improved and become standard practice. This review summarizes the current evidence regarding diagnosis and treatment of PPGLs, supporting the need of a more cluster-specific approach in clinical practice.

Practical Approach to Diagnosis and Management of IL-1-Mediated Autoinflammatory Diseases (CAPS, TRAPS, MKD, and DIRA).

Systemic autoinflammatory diseases (SAIDs) are a group of rare genetic and nongenetic immune dysregulatory disorders associated with high morbidity and mortality if left untreated. Therefore, early diagnosis and initiation of targeted treatment is vital in SAID patients to control the disease activity and prevent long-term immune-mediated damage. A specific group of genetically defined SAIDs is associated with increased inflammasome-mediated production of active interleukin (IL)-1. Even though progress in immunobiology and genetics has brought forth diagnostic tools and novel treatments that have been described in the literature extensively, many challenges remain in the clinical setting. Some challenges that health care providers may face on a day-to-day basis include the requirement of a multidisciplinary approach due to the complexity of these diseases, limited evidence-based treatment options, and barriers to access available therapies. Primarily, IL-1 inhibitors anakinra, canakinumab, and rilonacept are used to control the inflammation in these patients, with the goal of achieving sustainable remission. Recently published provisional points to consider from the European Alliance of Associations for Rheumatology (EULAR) and American College of Rheumatology (ACR) provide diagnosis, management, and monitoring recommendations for four IL-1-mediated autoinflammatory diseases: cryopyrin-associated periodic syndromes (CAPS), tumour necrosis factor receptor-associated periodic syndrome (TRAPS), mevalonate kinase deficiency (MKD), and deficiency of the IL-1 receptor antagonist (DIRA). The goal of this paper is to aid health care professionals by providing a practical approach to diagnosis and management of these four IL-1 mediated SAIDs on the basis of the recent EULAR/ACR recommendations.

An early-curricular team learning activity to foster integration of biochemical concepts and clinical sciences in undergraduate medical education.

The ability to connect key concepts of biochemistry with clinical presentations is essential for the development of clinical reasoning skills and adaptive expertise in medical trainees. To support the integration of foundational and clinical sciences in our undergraduate health science curricula, we developed a small group active learning exercise during which interprofessional groups of students use clinical cases to explore the biochemistry, diagnostic strategy, and evidence-based treatment options of inborn errors of metabolism (IEM). We designed multistage learning modules consisting of (1.) low-fidelity case simulations of pediatric patients presenting with IEMs, (2.) guided group discussions on clinical biochemistry, differential diagnoses, and diagnostic strategies, (3.) oral presentations of clinical reasoning strategies, and (4.) discussion of relevant evidence-based medicine topics related to the cases. These modules Scientific Knowledge Integrated in Patient Presentations (SKIPPs) were added to a first-semester foundational sciences course serving five health professions programs. The assessment of learning outcomes by students and faculty shows that SKIPPs sessions are well-received activities that significantly improve trainees' ability to integrate foundational science concepts into clinical scenarios, to practice interprofessional teamwork and to develop clinical reasoning skills.

SIFT IT: A feasibility and preliminary efficacy randomized controlled trial of a social cognition group treatment programme for people with acquired brain injury

ABSTRACT Making sense of social situations requires social cognitive skills, which can be impaired after acquired brain injury (ABI), yet few evidence-based treatment options are available. This study aimed to evaluate the feasibility of a multi-faceted social cognition group treatment programme, SIFT IT, for people after ABI using an RCT design. Twenty-eight participants were recruited, and 23 were randomized into either Treatment or Waitlist. SIFT IT consisted of 14 weekly 90-minute small group sessions facilitated by a Clinical Psychologist. Topics included: emotion self-awareness, emotion perception, perspective taking, and choosing adaptive social responses. Preliminary efficacy outcomes were assessed at baseline, post-treatment, and three-month follow-up. Demand for treatment was evident with 61% recruitment and 91% post-treatment retention rates, with 63% attending at least 13/14 sessions. Large between-group treatment effects (with non-zero 95% confidence intervals) were observed for emotion perception, detecting hints, and informant ratings of social cognitive deficits. Implementation challenges recruiting to groups and maintaining group allocation fidelity, with a small sample size does, however, raise questions about the appropriateness of an RCT design in a future efficacy trial. Overall, this study showed there is demand for social cognitive interventions after ABI and the SIFT IT programme was practicable and acceptable to participants. Trial registration: Australian New Zealand Clinical Trials Registry identifier: ACTRN12617000405314.

Recognition and management of children and adolescents with conduct disorder: a real-world data study from four western countries

BackgroundConduct disorders (CD) are among the most frequent psychiatric disorders in children and adolescents, with an estimated worldwide prevalence in the community of 2–4%. Evidence-based psychological outpatient treatment leads to significant improvement in about two-thirds of cases. However, there seems to be considerable variation in rates of CD diagnoses and implementation of evidence-based interventions between nations. The aim of this study was to compare administrative prevalence and treatment patterns for CD in children and adolescents seen in health care systems across four Western countries (Denmark, Germany, Norway, and the USA).MethodsCross-sectional observational study using healthcare data to identify children and adolescents (aged 0–19 years) with an ICD-10 code for CD within the calendar year 2018. Within each country’s study population, the prevalence of CD, psychiatric comorbidity, psychopharmacological treatment, and psychiatric hospitalisation was calculated.ResultsThe prevalence of diagnosed CD differed 31-fold between countries: 0.1% (Denmark), 0.3% (Norway), 1.1% (USA) and 3.1% (Germany), with a male/female ratio of 2.0–2.5:1. The rate of psychiatric comorbidity ranged from 69.7 to 86.1%, with attention-deficit/hyperactivity disorder being most common. Between 4.0% (Germany) and 12.2% (USA) of youths with a CD diagnosis were prescribed antipsychotic medication, and 1.2% (Norway) to 12.5% (Germany) underwent psychiatric hospitalisation.ConclusionRecognition and characteristics of youths diagnosed with CD varied greatly by country. In some countries, the administrative prevalence of diagnosed CD was markedly lower than the average estimated worldwide prevalence. This variation might reflect country-specific differences in CD prevalence, referral thresholds for mental health care, diagnostic tradition, and international variation in service organisation, CD recognition, and availability of treatment offers for youths with CD. The rather high rates of antipsychotic prescription and hospitalisation in some countries are remarkable, due to the lack of evidence for these therapeutic approaches. These findings stress the need of prioritising evidence-based treatment options in CD. Future research should focus on possible reasons for inter-country variation in recognition and management of CD, and also address possible differences in patient-level outcomes.

Hidradenitis Suppurativa Patient Referrals to a Canadian Community Dermatology Practice: A Retrospective Chart Review.

Awareness of hidradenitis suppurativa (HS) among non-dermatology healthcare providers is essential to facilitate prompt diagnosis, treatment, and referral to dermatology for further management. The purpose of this study was to analyze recent referrals to a Canadian community dermatology practice and compare the diagnostic concordance rates for HS between dermatologists and non-dermatologists. This study was a single-centre, retrospective chart review that was completed at Beacon Dermatology in Calgary, AB. Patients who were referred by a non-dermatologist for suspicion of HS and/or were diagnosed with HS by a dermatologist at Beacon Dermatology for the first time between May 2020 and May 2023 were included. Referral letters and dermatology clinic notes were analyzed to extract patient demographics, suspected pre-referral and post-referral diagnoses, and interim management plans that were initiated by the referring provider. A total of 451 patient charts with suspected and/or confirmed HS were retrieved from the clinic database. The median wait time from referral to the first dermatology appointment was 9.1 weeks. The average duration of HS symptoms was 7.3 years. HS was suspected by the referring provider and confirmed by a dermatologist in 286 cases (63%). Preliminary management was initiated in 60% of mild and 66% of moderate-to-severe cases. Given the prolonged time to diagnosis of HS, increasing awareness among healthcare providers is essential. Furthermore, this study highlighted the low implementation rates for evidence-based treatment options in preliminary management plans. Ultimately, this study demonstrates the need for increased interdisciplinary education on HS management in Canada.

Pandemic Effects on the Perception of Eating Disorder X Posts Among Adolescents and Transitional Age Youth

Eating disorders (EDs) are serious mental health conditions that can have devastating physical and psychological effects. They are significantly underdiagnosed and undertreated, and many individuals suffer in silence due to stigma, shame, and lack of access to appropriate care. Adolescents and transitional age youth (TAY) suffer from high prevalence of EDs, highlighting the need for effective prevention and intervention strategies, and improved access to evidence-based treatment options. Estimates suggest that at least 9% of the global population suffers from EDs, with the average age of onset being 18 years.1 Adolescents aged 13 to 18 are particularly vulnerable, with approximately 2.7% affected. TAY likely have an even higher number of ED cases.2 EDs have the highest fatality rates of any mental illness, with an estimated 4% of patients with anorexic nervosa dying from complications.3