Improvement In Symptoms Research Articles

Cognitive-Behavioural Social Skills Training: Mediation of Treatment Outcomes in a Randomized Controlled Trial for Youth at Risk of Psychosis: L’entraînement aux compétences sociales cognitivo-comportementales : variables médiatrices des résultats thérapeutiques dans le cadre d’un essai clinique randomisé pour les jeunes présentant un risque de psychose

Objectives Currently, there are no effective treatments for functional outcomes (i.e., role and social) and negative symptoms for youth at clinical high-risk (CHR) for psychosis. Investigations into possible mechanisms that may contribute to the improvement of functioning and negative symptoms are needed in CHR research to help inform psychosocial treatments. The present study examined whether functioning and negative symptoms were mediated by asocial beliefs, defeatist beliefs, self-efficacy, maladaptive schemas, anxiety, depression, social cognition, or attenuated psychotic symptoms (APS) in a large clinical trial. Methods CHR participants ( n = 203; 104 females; 99 males) were recruited as part of a three-site randomized control trial comparing group cognitive-behavioural social skills training (CBSST) versus a supportive therapy group. Mediation analyses were conducted to test the relationships between treatment group, mediators (asocial beliefs, defeatist beliefs, self-efficacy, maladaptive schemas, anxiety, depression, social cognition, and APS), and outcome (social and role functioning, and negative symptoms). The mediation analyses employed conditional process path analysis via ordinary least squares regression. Results At the end of treatment, but not 12-month follow-up, more severe APS were found to mediate the impact of treatment on negative symptoms, and social and role functioning. The greater the severity of APS, the less likely that CBSST would result in improvement in negative symptoms and social and role functioning. Many of the other variables showed significant associations with social (less for role) functioning and negative symptoms but did not mediate the effect of treatment on these outcomes at the end of treatment or 12-month follow-up. Conclusions There were no significant mediators except for APS at the end of treatment. Since more severe APS may result in participants being unable to fully participate in therapy and thus limit their gains, clinical implications may include offering some individual therapy to prepare these young people to benefit from the group treatment.

Catheter ablation of atrial fibrillation in women with heart failure with preserved ejection fraction

BackgroundHeart failure with preserved ejection fraction (HFpEF) and atrial fibrillation (AF) often coincide. Female sex is associated with both increased prevalence of HFpEF and reduced therapeutic efficacy of catheter ablation of AF. This sub-analysis of the previously published AFFECT-study evaluates outcome after cryoballoon-ablation in women with and without HFpEF.MethodsOne-hundred-and-two patients (LVEF ≥ 50%) scheduled for cryoballoon-ablation of AF were prospectively enrolled. Forty-two were female. Comprehensive baseline assessment included echocardiography, stress echocardiography, six-minute-walk-test, biomarker- and quality-of-life-assessment (QoL, SF-36), and was repeated at follow-up ≥12 months after AF-ablation. Baseline parameters, procedural characteristics and outcome after AF-ablation were compared between women with and without HFpEF.ResultsWomen with HFpEF (n = 20) were characterized by higher median left atrial volume index (35.8 ml/m2 vs. 25.8 ml/m2, P < 0.001), left ventricular hypertrophy (median left ventricular mass index: 92.0 g/m2 vs. 83.0 g/m2, P = 0.027), reduced distance in the 6-min-walk-test (median: 453 m vs. 527 m, P = 0.008) and higher left atrial pressures (median: 14.0 mmHg vs.9.5 mmHg, P = 0.008) compared to women without HFpEF (n = 21). During follow-up, HFpEF-patients more often experienced AF-related re-hospitalization (36.8% vs. 9.1%, P = 0.039) and numerically higher AF-recurrence-rates (57.9% vs. 31.1%, P = 0.109). There was no significant improvement of heart failure-related symptoms, echocardiographic parameters and cardiac biomarkers levels. QoL showed no significant improvement in both subgroups. Women with HFpEF still exhibited a lower SF-36 Physical Component Summary Score vs. women without HFpEF (median: 41.2 vs. 52.1, P < 0.001).ConclusionWomen with HFpEF constitute a distinct subgroup with high rates of AF-related events after AF-ablation, and persistence of both symptoms and functional hallmarks of HFpEF. Consideration of sex-specific cardiac co-morbidities is crucial for personalization and optimization of AF-therapy.Clinical Trial RegistrationClinicalTrials.gov Identifier NCT05603611.

QOL-25. DIGITAL PHENOTYPING OF PERIOPERATIVE MOBILITY METRICS IN PATIENTS UNDERGOING SURGICAL RESECTION OF SKULL BASE TUMORS

Abstract Current methods to assess quality of life and recovery after surgery for skull base tumors are limited and often biased by subjective patient-reported assessments, while objective, in-situ assessments are lacking. Smartphone-based digital phenotyping has been increasingly studied across pathologies, utilizing built-in technologies to measure behavioral patterns pertaining to sleep, physical mobility, social interactions, and cognitive functioning, among others. Here, we report our initial experience with smartphone-based digital phenotyping in patients who underwent resection of skull base lesions. Patients seen at our multi-disciplinary skull base center were enrolled under an institutional board-approved protocol and installed the Beiwe application, which passively collected GPS data 20 days pre- and up to 140 days post-surgery. Variables measured included time spent at home, number of separate significant places visited outside home, daily walking time, and daily number of steps taken. 26 patients were enrolled, comprised of 14 pituitary adenomas, 8 meningiomas, and 4 vestibular schwannomas. All patients demonstrated increased time spent at home, fewer number of significant locations visited, decreased walking time, and decreased total steps in the immediate post-operative period. For pituitaries, mean return to baseline for these variables was 25.7, 24.3, 80.2, and 88.9 days, respectively. For meningiomas, return to baseline was 56.5, 214.4, 45.0, and 45.6 days, respectively. For vestibular schwannomas, return to baseline was 219, 39.9, 35.1, and 36.4 days, respectively. This study demonstrates initial efficacy of digital phenotyping for a cohort of patients undergoing surgical resection of skull base lesions, based on passively collected smartphone GPS data during the peri-operative period. This is part of a larger, ongoing study enrolling surgical patients as well as non-operative controls for comparison. Such peri-operative mobility metrics may potentially be refined to predict postoperative outcomes following surgery such as improvement of neurological symptoms, postoperative CSF leak, infection, dysnatremia, and hospital length of stay.

QOL-27. PATIENT REPORTED OUTCOME AND PREFERENCE AFTER CRANIOTOMY AND LASER THERMAL ABLATION: A PILOT STUDY

Abstract BACKGROUND Laser interstitial thermal therapy (LITT) is a minimally invasive procedure that allows cytoreduction in the treatment of brain tumors and can be considered by both surgeons and patients as an alternative to craniotomy. OBJECTIVE We surveyed 27 patients who underwent both craniotomy and LITT during distinct stages of their oncology journey to assess patient reported outcomes comparing both procedures. METHODS A nine question survey was developed and validated to assess patient reported post-operative recovery, pain level, narcotic use, and procedure preference. The survey was administered to patients with WHO II-IV gliomas who underwent both craniotomy and LITT. RESULTS The survey was reviewed by independent surgeons, patient advocates, and patients in terms of face validity and showed >90% correlation in test-retest reliability. Our cohort had a mean age of 57 (+/- 12 years), 78% had glioblastoma. There was no significant difference in symptomatic improvement post-craniotomy or post-LITT (30% versus 4%, p=0.17). Similarly, no significance was detected in patient-reported recovery time from craniotomy (time required to return to pre-operative state, 4.3 +/- 9.1 weeks, median 2 weeks) or LITT (2 ± 2.3 weeks, median 1 week)(p=0.21). Notably, post-surgical pain (1-10 pain scale) and need for narcotic use in the first week (yes/no) after the procedure were significantly lower post-LITT (average pain score 1.7 versus 5 points, narcotic use 4% versus 81%, p<0.0005 for both comparisons). When asked which procedure they would choose – having experienced both craniotomy and LITT, surveyed patients overwhelmingly chose LITT over craniotomy (89% versus 11%, p<0.0005). Of note, the patients who preferred craniotomy experienced improved neurologic function post-craniotomy or suffered new deficits post-LITT. CONCLUSION In this pilot study, patients reported less pain and narcotic use post-LITT relative to craniotomy and generally preferred the former procedure if given the choice. Validation of these results in future studies can help inform decision making in clinical scenarios where there is equipoise between LITT and craniotomy.

The American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index‐Daily Diary (ANMS GCSI‐DD): Psychometric validation and meaningful change threshold in patients with idiopathic or diabetic gastroparesis

AbstractIntroductionThe American Neurogastroenterology and Motility Society Gastroparesis Cardinal Symptom Index‐Daily Diary (ANMS GCSI‐DD) is a patient‐reported outcome (PRO) instrument for gastroparesis. This study evaluated the psychometric properties of the ANMS GCSI‐DD and determined a meaningful change threshold using the data from a phase 2b trial in adults with moderate‐to‐severe idiopathic or diabetic gastroparesis (DG).MethodsThe psychometric properties of ANMS GCSI‐DD were analyzed using other clinician‐ and patient‐reported outcomes from 242 patients during the 12‐week trial. Trial exit interviews were conducted in a cohort of 32 patients to capture their perspectives on meaningful change from the trial.ResultsANMS GCSI‐DD demonstrated good performance: unidimensionality scores exhibited good fit; internal consistency reliability was >0.70; test–retest reliability was above 0.90, and convergent validity showed strong correlations (0.70–0.90) with overall severity rating and moderate correlations (0.30–0.70) with other outcomes. ANMS GCSI‐DD scores discriminated among groups with varying severity of gastroparesis symptoms in known‐groups validity (p < 0.001) and were responsive to symptom improvement. Triangulation of the quantitative anchor‐based estimates of 0.9–1.4 category points as a meaningful change in the ANMS GCSI‐DD composite score, and the qualitative exit interview findings of 0.5–1.5 as meaningful change, supports a one‐point change on a five‐point scale (0–4) as the meaningful change threshold.ConclusionThe ANMS GCSI‐DD is a reliable and valid PRO instrument to employ in clinical development for patients with moderate‐to‐severe idiopathic or DG, and a one‐point score change in the ANMS GCSI‐DD composite score is recommended as a meaningful change threshold of symptom change.

Recalcitrant Torticollis: A Formidable Treatment Challenge

Introduction There are few evidence-based practice guidelines for recalcitrant torticollis (RT), defined as torticollis that persists past 1 year of age despite conservative therapy. Available evidence recommends a combination of physical therapy (PT), occupational therapy (OT), botulinum toxin injection (BTI), and in the most severe cases, surgical release. Methods This is a single-center, retrospective study conducted with Institutional Review Board approval. Inclusion criteria were diagnosis of congenital muscular torticollis before 1 year old and persistence of symptoms past 1 year despite conservative treatment between January 1, 2005, and January 30, 2023. Patients with incomplete data, acquired torticollis, confounding diagnoses, or who did not receive a diagnosis or conservative therapy before 1 year of age were excluded. Results Seventy-seven patients met the inclusion criteria. The average age at diagnosis was 4.5 ± 2.5 months. Initial treatment consisted of PT for 61 patients (79.2%), stretching exercises for 11 patients (14.3%), and OT for 4 patients (5.2%). Regardless of timing, 74 patients in total (96.1%) received PT for an average of 13.3 ± 8.6 months. Conservative treatment modalities led to the resolution of symptoms for 40 patients (51.2%) and improvement of symptoms for 35 patients (45.5%). Two patients received at least 1 BTI which led to further improvement of symptoms. One patient underwent operative intervention which consisted of sternocleidomastoid release. Conclusions RT is a challenging clinical entity that requires diligent multidisciplinary care. Patients who present and begin conservative therapy before 1 year of age are likely to have improvement or resolution of symptoms without more advanced therapies.

Holistic Treatment of Acne Vulgaris: Integrating the Complete Repertory for Quality-of-life Enhancement

Acne vulgaris, a common dermatological condition, significantly impacts the quality of life, particularly in adolescents and young adults. This study investigates the effectiveness of individualized homeopathic treatments selected using the Complete Repertory in alleviating acne symptoms and enhancing patients' quality of life. Over a period of 4-6 months, 101 patients, aged 15 to 25, were treated using the Global Acne Grading System (GAGS) and the Cardiff Acne Disability Index (CADI) to evaluate progress. Significant improvements in both physical symptoms and psychological well-being were observed, suggesting that personalized homeopathic regimens could be a viable holistic approach (Schroyens, 2018).

Two types of vidian neurectomy show efficacy in treating allergic rhinitis and vasomotor rhinitis

This study aimed to evaluate the efficacies of endoscopic vidian neurectomy (VN) and highly selective vidian neurectomy (HSVN) for the treatment of allergic rhinitis (AR) and vasomotor rhinitis (VMR). All AR and VMR patients were divided into two groups, Group VN and Group HSVN. The efficacy evaluation methods were visual analog scale (VAS), rhinoconjunctivitis quality of life questionnaire (RQLQ), and medication score. The efficacy evaluations were used to assess patient rhinitis symptoms, quality of life and drug usage. The time points of follow-up period were preoperative, 3 months, 6 months, 1 year, 2 years, and 3 years post operation. By analyzing pre- and postoperative VAS, medication score and RQLQ score, the results showed that rhinitis symptoms and quality of life in both VN and HSVN groups were significantly improved at 3 years, and the rate of improvement decreased gradually with time. Post operation, there were no significant differences in VAS and medication scores between the VN and HSVN groups. With respect to RQLQ, postoperative 2 years and 3 years improvements in sleep disorders, non-nasal symptoms and eye symptoms were significantly greater in the HSVN group than in the VN group. Improvements in rhinitis symptoms and quality of life in AR and VMR were sustained by VN and HSVN until 3 years post operation, with greater quality of life improvements in the HSVN group.

The Relationship Between Mercury Exposure and Membranous Nephropathy: Case Reports and Meta‐Analysis

ABSTRACTTo investigate the clinical characteristics of patients with membranous nephropathy (MN) and the therapeutic efficacy of Sodium Dimercaptosulphonate (DMPS), as well as the relationship between mercury (Hg) exposure and MN, we investigated the clinical manifestations and treatment outcomes of six patients with MN and searched the China National Knowledge Internet (CNKI), PubMed, Web Of Science, and Embase databases for relevant studies on Hg exposure and MN published from the inception of the databases to April 2024. Data analysis was performed using SPSS 26.0 and Stata 16.0. We found that (1) the clinical symptoms of MN patients were mainly characterized by proteinuria, edema, hypoproteinemia, and hyperlipidemia. Comparative analysis before and after DMPS treatment showed a decrease in 24‐h urinary Hg, 24‐h urinary protein, and total cholesterol levels, as well as an increase in serum albumin levels (p < 0.05). (2) Two MN patients received DMPS for sole Hg detoxification treatment, whereas four patients received Hg detoxification combined with hormone therapy, and all patients showed significant improvement in symptoms after treatment. (3) Among the 564 articles, four met the inclusion criteria. The results showed that Hg exposure increased the incidence of MN by 5.74 times (95% confidence interval [CI] = 2.57, 12.83). The clinical symptoms of MN patients are mostly manifested as proteinuria, edema, hypoproteinemia, and hyperlipidemia. DMPS Hg detoxification treatment is effective for Hg‐induced MN. Hg exposure can increase the prevalence of MN, therefore making it necessary to take prudent measures to reduce the risk of Hg exposure.

DUPILUMAB: A NEW ALLY FOR THE TREATMENT OF EOSINOPHILIC ESOPHAGITIS?

INTRODUCTION: Eosinophilic esophagitis is a chronic, immune-mediated disease of the gastrointestinal tract, resulting from the interaction between genetic, environmental and immunological factors of the affected patient. The clinical picture is characterized by esophageal dysfunction, which may present as dysphagia, precordial pain, upper abdominal pain or food impaction. Treatment aims to improve clinical symptoms and prevent disease progression and subsequent complications. In this context, immunobiologicals are emerging and promising therapies, with Dupilumab being the first specific treatment approved by the FDA. It is an IgG4 monoclonal antibody that targets IL-4R alpha and, from there, reduces protein transphorylation, transcription and the T helper 2-regulated response. The objective of this systematic review is to evaluate the efficacy of this drug in the treatment of eosinophilic esophagitis, in comparison with the options already available on the market. METHOD: PubMed searches were performed using the keywords “eosinophilic esophagitis dupilumab” and “dupilumab treatment esophagitis eosinophilic”. Initially, 60 results were obtained, but after exclusion by title, abstract and full text, 14 articles were included. RESULT: all articles analyzed demonstrated improvement in patients using Dupilumab, with 13 of them indicating a reduction in eosinophil counts, 8 showing clinical improvement in symptoms, 8 indicating histological improvement, 7 indicating improvement in the endoscopic pattern, according to the Endoscopic Reference for Eosinophilic Esophagitis, and 4 indicating a reduction in the Dysphagia Symptom Questionnaire (DQQ) score. DISCUSSION: eosinophilic esophagitis is a rare disease, but with potential for progression, negatively interfering with the patient's quality of life. Therefore, since current drug therapy is nonspecific, the use of Dupilumab presents itself as an innovative and optimistic solution for the future, because it changes the understanding of disease management, expands therapeutic options and improves prognosis. CONCLUSION: the use of Dupilumab in the treatment of eosinophilic esophagitis has proven to be effective. However, for the treatment to be well established with regard to the doses used, frequency of administration and longitudinal monitoring, further studies are still required.

Efficacy of the combination of transcutaneous tibial nerve stimulation and mirabegron in women with overactive bladder in a prospective randomized controlled trial

The management of overactive bladder (OAB) in women encompasses a range of strategies, from behavioral modifications to pharmacotherapy and nerve stimulation techniques. This prospective, randomized, controlled trial evaluates the efficacy of the combination of TTNS and mirabegron in symptom improvement over three months in women diagnosed with OAB. The study was designed as a randomized controlled trial. A total of 40 patients were prospectively randomized into two groups. Twenty patients in the combination group accepted TTNS and mirabegron therapy, and the other 20 patients as control only accepted mirabegron therapy. Primary outcomes were changes from baseline in the lower urinary tract symptoms. The severity of symptoms and quality of life (QoL) were assessed using the validated OAB questionnaire (OAB-q). TTNS reduced the clinical symptoms of OAB, and the difference was statistically significant at all study sites (p < 0.05). Regarding secondary outcomes, the OAB-q symptom bother score was lower in the combination group than in the mirabegron group (p < 0.05). The OAB-q score in the combination group was statistically superior to that in the mirabegron group (p < 0.05). The incidence of complications was not statistically significant between the two groups.The combination of TTNS and mirabegron represents a promising therapeutic strategy for women with overactive bladder, significantly improving symptoms and quality of life with a favorable safety profile. Further research with a larger sample size and long-term follow-up is warranted to confirm these findings and explore the underlying mechanisms of this combination therapy’s efficacy.

Impact of the COVID-19 pandemic and lockdown measures on clinical visits and subjective symptoms in childhood allergic rhinitis induced by house dust mites in Shanghai

BackgroundEstablishing the interaction between aeroallergens and air pollution in children with house dust mite induced allergic rhinitis (HDM-AR) remains challenging, especially in urban areas. The coronavirus disease 2019 (COVID-19) pandemic and the subsequent lockdown measures provided a valuable opportunity.MethodsWe analyzed the clinical data of HDM-AR children between March and August in 2018–2020, and classified the children according to the type and the degree of HDM sensitization. The records of patients’ hospital visits, allergic rhinitis symptoms assessments, and air pollution measurements in Shanghai were used to assess the differences before (2018–2019) and during the pandemic (2020), as well as during lockdown (March-June) and unlockdown (July-August) period in 2020.ResultsThe study included 1570 HDM-AR children aged 2 to 8 years old, 815 (51.9%) were monosensitized to HDM (mono-HDM-AR), and 755 (48.1%) were polysensitized to HDM (poly-HDM-AR). There was a significant increase in the rate of clinical visits among children with HDM-AR during the COVID-19 pandemic compared to pre-pandemic (P < 0.001), particularly among older children aged 7–8 years (P = 0.01). During the unlockdown period, there was a notable decrease in clinical visits for children with poly-HDM-AR (P < 0.001). Children with high levels of HDM sensitization exhibited significant symptom improvement in unlockdown period (P < 0.001). Although the air pollutants concentration had improved during the study, there was no effect on the improvement of HDM-AR children as expected.ConclusionsThe COVID-19 pandemic and its associated lockdown measures provided a unique context to observe the dynamics of management in children with HDM-AR. The findings underscore the complexity of managing allergic conditions in pediatric populations, highlighting the influence of environmental and lifestyle changes on disease presentation and the need for tailored approaches to treatment during periods of societal disruption.

Changes in Resting-State Functional Connectivity and Cognitive-Affective Symptoms in Patients With Post-Concussion Syndrome Treated With N-Acetyl Cysteine

Objective: Concussion accounts for more than 80% of people experiencing traumatic brain injury. Acute concussion is associated with characteristic cognitive and functional deficits that may persist for weeks to months. A subgroup of these patients (from 10% to 50%) have persistent symptoms referred to as chronic post-concussion syndrome (PCS). There are limited treatment options for these patients and the pathophysiology is poorly understood, though oxidative stress is thought to be a contributing factor. The purpose of this study was to evaluate whether an antioxidant, N-acetylcysteine (NAC), might be beneficial in patients with PCS. Setting: Outpatient medicine center. Participants: Fifty patients with chronic PCS for at least 3 months post injury. Design: The patients with PCS were enrolled in this randomized unblinded clinical trial to receive the antioxidant NAC as a combination of daily oral and weekly intravenous infusions, or assigned to a waitlist control group where they would continue to receive standard of care. Main Measures: Resting-state functional connectivity (FC) magnetic resonance imaging (rsFC-MRI) was performed pre and post either NAC or the waitlist period along with cognitive, emotional, and sensory symptom assessments. Results: The results demonstrated significant (P &lt; .05) improvements in symptoms as determined by the Rivermead Post-Concussion Symptoms Questionnaire, Spielberger State-Trait Anxiety Inventory, and Profile of Mood Scale in the PCS group receiving NAC as compared to patients receiving ongoing standard care. Importantly, there were significant (P&lt; .01) changes in FC in the NAC group, particularly in networks such as the default mode network, salience network, and executive control network. These changes in FC also correlated with improvements in symptoms. Conclusions: In patients with chronic PCS, NAC treatment was associated with significant changes in resting state FC and improvement in a variety of symptoms, particularly cognitive and affective symptoms.

Facilitating Thought Progression to Reduce Depressive Symptoms: Randomized Controlled Trial.

The constant rise in the prevalence of major depressive disorder calls for new, effective, and accessible interventions that can rapidly and effectively reach a wide range of audiences. Recent developments in the digital health domain suggest that dedicated online platforms may potentially address this gap. Focusing on targeting ruminative thought, a major symptomatic hallmark of depression, in this study we hypothesized that delivering a digital health-based intervention designed to systematically facilitate thought progression would substantially alleviate depression. The study aims to investigate the efficacy of a novel digital intervention on the reduction of depressive symptoms. This intervention was designed as an easy-to-use gamified app specifically aimed to facilitate thought progression through intense practicing of associative, semantically broad, fast, and creative thought patterns. A randomized clinical trial was conducted, comparing changes in depression symptoms between participants who used the app in the intervention group (n=74) and waitlist control group (n=27) over the course of 8 weeks. All participants filled out a battery of clinical questionnaires to assess the severity of depression at baseline and 4 and 8 weeks after starting the study. These primarily included the Montgomery-Åsberg Depression Rating Scale (MADRS) and the Patient Health Questionnaire-9 as well as the Positive Affect Negative Affect Scale-Negative Affect Score, Ruminative Response Scale, and Symptoms of Depression Questionnaire. Additional questionnaires were implemented to assess anxiety, positive affect, anhedonia, and quality of life. The results indicate that across multiple clinical measurements, participants in the intervention group who played the gamified app showed greater and faster improvement in depressive symptoms compared with their waitlist control counterparts. The difference between the groups in MADRS improvement was -7.01 points (95% CI -10.72 to -3.29; P<.001; Cohen d=0.67). Furthermore, the difference in improvement between groups persisted up to 4 weeks posttrial (MADRS differences at week 12: F49,2=6.62; P=.003; ηp2=0.21). At the end of the trial, participants who played the app showed high interest in continuing using the app. The results demonstrate that a gamified app designed to facilitate thought progression is associated with improvement in depressive symptoms. Given its innovative and accessibility features, this gamified method aiming to facilitate thought progression may successfully complement traditional treatments for depression in the future, providing a safe and impactful way to enhance the lives of individuals experiencing depression and anxiety. ClinicalTrials.gov NCT05685758; https://clinicaltrials.gov/study/NCT05685758.

Phase 3 Open-Label Study Evaluating the Efficacy and Safety of Mavacamten in Japanese Adults With Obstructive Hypertrophic Cardiomyopathy - The HORIZON-HCM Study.

Mavacamten, a cardiac myosin inhibitor, significantly improved symptoms and cardiac function vs. placebo in patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in EXPLORER-HCM. However, the efficacy and safety profiles of mavacamten in Japanese patients are unclear. HORIZON-HCM is a Phase 3 single-arm study in Japanese patients with symptomatic obstructive HCM. The mavacamten starting dose was 2.5 mg; individualized dose titration occurred in Weeks 6-20 based on Valsalva left ventricular outflow tract (LVOT) gradient and resting left ventricular ejection fraction (LVEF). Overall, 38 patients were treated; 36 completed the 30-week primary treatment analysis period. Clinically significant improvements in postexercise LVOT gradient were observed after 30 weeks of treatment (mean change from baseline -60.7 mmHg). Improvements in N-terminal pro B-type natriuretic peptide, New York Heart Association class, and Kansas City Cardiomyopathy Questionnaire-23 Clinical Summary Score were observed over 30 weeks, and mean LVEF was ≥74% at all visits. Treatment-emergent adverse events (TEAEs) and serious TEAEs were reported in 63.2% and 7.9% of patients, respectively; none resulted in treatment discontinuation. One patient experienced a transient asymptomatic reduction in LVEF to <50%. No deaths occurred during the study. In Japanese patients with obstructive HCM, mavacamten was associated with similar improvements in LVOT gradients, cardiac biomarkers, and symptoms to those observed in EXPLORER-HCM. Treatment was well tolerated with no new safety concerns.

Efgartigimod: A Review in Generalised Myasthenia Gravis.

Efgartigimod (Vyvgart®; Vyvgart® Hytrulo) is a neonatal fragment crystallizable receptor (FcRn) antagonist indicated for the treatment of generalised myasthenia gravis (gMG) in adults who are acetylcholine receptor (AChR) antibody positive (Ab+). Efgartigimod is approved for both intravenous (IV) and subcutaneous (SC) use. In a pivotal phaseIII trial, IV efgartigimod was associated with significant and clinically meaningful improvements in myasthenia gravis symptoms and reductions in disease burden. The beneficial effects of IV efgartigimod were reproducible, durable and maintained over the long term. IV efgartigimod also improved health-related quality of life (HRQOL). In another phaseIII trial, SC efgartigimod PH20 was noninferior to IV efgartigimod in reducing total immunoglobulin G levels. Clinical improvement with SC efgartigimod PH20 was consistent with that of IV efgartigimod and was reproducible over the long term. Efgartigimod was generally well tolerated; the most common adverse events were headache and infections (with IV efgartigimod) and injection-site reactions (with SC efgartigimod PH20). Although further long-term data are required, IV and SC formulations of efgartigimod provide effective, generally well-tolerated and flexible treatment options for adults with AChR Ab+ gMG.

Psychiatric considerations of multiple sclerosis

Multiple sclerosis is a degenerative neuroinflammatory disease that affects the central nervous system with a wide range of neurological and psychiatric symptoms. A correct differential diagnosis is crucial for the management of pathology and there are specific diagnostic methods. This study presents the case of a 53-year-old patient diagnosed with Strumpell-Lorrain disease resistant to psychopharmacological treatment for depression and anxiety who, after the change of diagnosis and therapy to multiple sclerosis, showed clinical improvement in psychiatric symptoms.

Robotic versus laparoscopic revision to Toupet fundoplication for failed Nissen fundoplication: a single-center experience.

Nissen fundoplication (NF) is a common surgical procedure to treat gastroesophageal reflux disease; however, a subset of patients may continue to experience symptoms or develop symptom recurrence despite a successful procedure. This study aims to compare laparoscopic and robotic approaches for treating failed NF and evaluate the outcomes after converting to Toupet fundoplication (TF). We conducted a retrospective analysis of patients who underwent robotic or laparoscopic revision to TF for failed NF between 2016 and 2023. The data collected included demographics, pre-operative workup, and peri- and post-operative outcomes. Symptom analysis and anti-reflux medication usage were collected using a patient questionnaire. Failed fundoplication was defined as the need for an additional operation due to unresolved GERD symptoms or the emergence of a new issue. Eighty-eight patients (56 laparoscopic, 32 robotic) were included. Mean operative time was 148.71 ± 53.64min for the total cohort and was significantly longer in the robotic group (RG) 167.43min vs 138.01min in the Laparoscopic group (LG) (p value = 0.012). The LG had a length of hospital stay of 2.16 ± 1.69days vs RG 2.21 ± 1.28days (p value = 0.867). The LG had a higher number of early readmissions (5.4%, p value = 0.629) and both the LG and the RG had 1 patient that required an early reintervention. Symptoms of dysphagia and reflux decreased in both groups at last follow-up, but the reduction in PPI use was not significant. Surgical revision to TF for failed NF provides significant symptom improvement with low rates of complications and recurrences. Our study shows that both approaches are safe and feasible and have comparable surgical and symptom outcomes.

Improvement in Persistent Post-Concussive Symptoms After Treatment with Neuromodulation: A Case Study

AbstractUp to 30% of youth with concussion experience persistent post-concussive symptoms (PPCS) impacting cognitive, social, mental, and physical health. Limited evidence-based therapies exist to treat PPCS. We present a case of an adolescent female with multiple sports-related concussions with persistent symptoms, including headache, nausea, dizziness, brain fog, and anxiety. After 4 weeks of treatment with an auricular percutaneous electrical nerve field stimulation (PENFS) device, she had near-complete resolution of her symptoms, allowing a return to her previous level of academic achievement, as well as physical activity. Emerging evidence suggests that various forms of neuromodulation, such as repetitive transcranial magnetic stimulation (rTMS) and transcranial direct current stimulation (tDCS), can benefit patients with post-concussive symptoms. This adolescent female’s improvement with PENFS contributes to this expanding field.

Outcomes of patients with intermediate-risk neuroblastoma presenting with motor deficits relating to intraspinal tumor extension: A report from the Children's Oncology Group study ANBL0531.

Tumor invasion of the spinal canal is detected radiographically in approximately 15% of patients with newly diagnosed neuroblastoma (NB). The optimal clinical approach to maintain excellent survival outcomes while minimizing long-term sequelae is yet to be defined. Patients with intermediate-risk neuroblastoma (IR-NB) and radiographically identified intraspinal tumors who were treated on the Children's Oncology Group study ANBL0531 were studied prospectively to evaluate neurologic outcomes related to cord compression. Patients were defined as being symptomatic versus asymptomatic based on reporting of neurologic motor deficits at diagnosis. Patient characteristics, tumor biology, chemotherapy treatment, surgical interventions, and neurologic and disease outcomes are reported. Of the 92 patients with intraspinal tumors, 42 (46%) were symptomatic and most (73%) had complete resolution of symptoms. Age, degree of motor deficit, and duration of symptoms at diagnosis were not associated with complete resolution. While symptomatic patients were more likely to undergo upfront laminectomy, laminectomy was not associated with improvement of motor symptoms. Administration of additional chemotherapy beyond initial treatment assigned per protocol to achieve the treatment end point was not associated with achieving symptom resolution. Patients presenting with motor deficits due to intraspinal tumor had excellent survival and favorable neurologic outcomes, with the majority reporting complete resolution of motor symptoms regardless of severity and duration of symptoms at diagnosis or neurosurgical intervention. Prompt diagnosis and initiation of first-line chemotherapy treatment remain priority, while neurosurgical intervention should be reserved for patients with rapid neurologic deterioration. Biology-based therapy and tumor response should continue to be used to maintain favorable outcomes.