Global Impressions-Improvement Scale Research Articles

Mandibular Advancement vs Combined Airway and Positional Therapy for Snoring

Approximately 150 million individuals in the US snore in the absence of obstructive sleep apnea (primary snoring), but few studies have examined the efficacy of treatments for snoring or evaluated the effect of snoring in sleeping partners. To evaluate the efficacy of 2 treatments for primary snoring. This pilot randomized clinical trial that included a convenience sample of people who snore without sleep apnea and their sleeping partner who underwent 4 weeks of snoring treatment was conducted at an academic medical center between October 3, 2022, and July 3, 2023. Fifty couples were randomized to either use a mandibular advancement device (MAD) or receive combined airway and positional therapy (CAPT; external nasal dilator, nasal saline lavage with mometasone, mouth taping, and lateral positional therapy). Percentage of sleeping partners who reported that their partner's snoring was either very much improved or much improved (responder) on the Clinical Global Impression of Improvement scale. A total of 42 dyads completed the study; 23 (55%) were randomized to MAD and 19 (45%) to CAPT. Among people who snore, 26 (62%) were female, and the mean (SD) age was 48 (14) years. Of 23 dyads randomized to MAD, 21 people who snore (91%) were rated by the sleeping partner as a responder, while 11 of the 19 dyads (58%) randomized to CAPT were rated by the sleeping partner as responder, resulting in a difference of 33 percentage points (95% CI, 8-58) and a number needed to treat of 3. Of the 10 participants who were withdrawn, 4 were withdrawn due to adverse effects of the treatment that were evenly distributed between the MAD (n = 2) and CAPT (n = 2) groups. The results of this randomized clinical trial showed that the MAD may be more effective than CAPT for treating primary snoring, while both treatment options were found to reduce primary snoring. Physicians should have a patient-centered discussion to determine which treatment is best for individual patients with primary snoring, weighing convenience, adverse effects, and cost as factors. ClinicalTrials.gov Identifier: NCT05756647.

Real life retrospective study of cannabidiol therapy in alternating hemiplegia of childhood

BackgroundMany alternating hemiplegia of childhood (AHC) patients have received Cannabidiol (CBD) but, to our knowledge, there are no published data available. GoalsTest the hypothesis that CBD has favorable effects on AHC spells. MethodsRetrospective review of available data of AHC patients who received CBD. Primary analysis: Clinical Global Impression Scale of Improvement (CGI-I) score for response of AHC spells to CBD with calculation of 95% confidence interval (CI) for rejection of the null hypothesis. Secondary analyses, performed to achieve an understanding of the effect of CBD as compared to flunarizine, were CGI-I scores of 1) epileptic seizures to CBD, 2) AHC spells to flunarizine, 3) epileptic seizures to flunarizine. Also, Mann-Whitney test was done for comparison of CGI-I scores of CBD and flunarizine to both AHC spells and seizures. ResultsWe studied 16 AHC patients seen at Duke University and University of Lyon. CI of CGI-I scores for AHC spells in response to CBD and to flunarizine, each separately, indicated a positive response to each of these two medications: neither overlapped with the null hypothesis score, 4, indicating significant positive responses with p < 0.05 for both. These two scores also did not differ (p = 0.84) suggesting similar efficacy of both: CBD score was 2 ± 1.1 with a 95% CI of 1.5–2.6 and flunarizine score was 2.3 ± 1.3 with a 95% CI of 1.7–3.1. In patients who had seizures, CI calculations indicated a positive effect of CBD on seizure CGI scores but not of flunarizine on seizure scores. CBD was well tolerated with no patients discontinuing it due to side effects and with some reporting positive behavioral changes. ConclusionOur study indicates a real-life positive effect of CBD on AHC type spells.

Readiness for treatment predicts depression outcomes in a partial hospital program.

Evidence-based interventions vary in effectiveness for individuals with depression, which has a large public health burden. Readiness for change or treatment can be an important individual difference predictor of depression outcomes. To inform public service initiatives targeting readiness for treatment, characterizing readiness across settings and levels of care is key. However, limited data exist on the role of readiness for treatment in acute psychiatric settings and in particular, partial hospital programs which are key points in the continuity of inpatient and outpatient care. The present study assessed readiness for treatment in terms of importance, confidence, and motivation to engage in a partial hospital program and tested whether higher levels of readiness were associated with better treatment outcomes among clients with depression. Participants (N = 192) with major depressive disorder rated their readiness for treatment (Readiness Rulers), depression (Patient Health Questionnaire-9), and global improvement (Clinical Global Impression Scale-Improvement Self-Report) while enrolled in a partial hospital program. Generalized linear regression models assessed the effect of baseline readiness on outcomes at discharge, adjusted for baseline level of the outcome, age, sex, race, and ethnicity. Greater baseline readiness predicted reduced depression and better global improvement at discharge. Higher confidence and motivation to engage in treatment, but not importance, were associated with better depression outcomes. Identifying and addressing readiness for treatment by leveraging public health systems and services (e.g., help lines, family interventions) prior to or upon starting a partial hospital program may be useful to maximize gains in treatment. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

Long-term treatment with ganaxolone for seizures associated with cyclin-dependent kinase-like 5 deficiency disorder: Two-year open-label extension follow-up.

In the placebo-controlled, double-blind phase of the Marigold study (NCT03572933), ganaxolone significantly reduced major motor seizure frequency (MMSF) in patients with cyclin-dependent kinase-like 5 deficiency disorder (CDD). We report 2-year safety and clinical outcomes data from the open-label extension (OLE) phase of Marigold. Patients with CDD who completed the double-blind phase were eligible to continue in the OLE. Efficacy assessments included MMSF reduction from prerandomization baseline, responder rates, and Clinical Global Impression-Improvement scores, including assessment of seizure intensity and duration (CGI-CSID). Safety assessments included treatment-emergent adverse events (TEAEs) and TEAEs leading to discontinuation. Of 101 patients who enrolled in Marigold, 88 (87.1%) entered the OLE (median age = 5 years, 79.5% female). Median 28-day MMSF at baseline was 50.6. At 2 years in the OLE (months 22-24), MMSF was reduced by a median of 48.2% (n = 50); when missing data were imputed, median reduction in MMSF was 43.8% using a mixed effects model and 27.4% using a last observation carried forward model. During months 22-24, 23 of 50 (46.0%) patients experienced reductions in MMSF of ≥50%; 12 of 50 (24.0%) patients experienced MMSF reductions of ≥75%. During months 22-24, 40 of 49 (81.6%) patients were rated by caregivers as having improvement in seizure-related outcomes based on CGI-CSID scores. Thirty-seven patients discontinued ganaxolone due to lack of efficacy (n = 13), withdrawal by caregiver (n = 12), adverse event (n = 10), physician decision (n = 1), or death (n = 1; unrelated to study drug). The most common treatment-related TEAEs were somnolence (17.0%), seizure (11.4%), and decreased appetite (5.7%). Patients reported serious TEAEs (n = 28, 31.8%); those reported in ≥3% of patients were seizure (n = 6), pneumonia (n = 5), acute respiratory failure (n = 3), aspiration pneumonia (n = 3), and dehydration (n = 3). Sustained reductions in MMSF at 2 years in the OLE support the efficacy of ganaxolone in seizures associated with CDD. Safety findings in the OLE were consistent with the double-blind phase.

Plum-blossom needle therapy for Tourette syndrome: A case report

IntroductionTourette syndrome (TS) is a neurodevelopmental disorder characterized by the childhood onset of motor and phonic tics, frequently accompanied by behavioral and psychiatric comorbidities. Existing treatments for tics raise safety concerns. In China, acupuncture has been a widely utilized approach for TS, and one specific traditional acupuncture method, plum-blossom needle therapy (PBNT), has exhibited favorable tolerance among pediatric patients. In this report, we present a case of TS treated using PBNT. Case presentationAn 11-year-old boy diagnosed with Tourette syndrome for over five years presented with frequent loud throat clearing, eye blinking, severe abdominal tensing, shoulder shrugging, and upper limb twitching. Plum-blossom needle therapy (PBNT) was administered for 8 weeks, involving two sessions per week. The boy's tic symptoms gradually ameliorated, ultimately becoming minimal following the 8-week treatment period. Specifically, the Yale Global Tic Severity Scale (YGTSS) score decreased from 71 to 20, and the TS Clinical Global Impression Scale of Improvement (CGI-I) score reached 1, signifying 'very much improved' post-treatment. However, the Children and Adolescents' Quality of Life Scale (CAQOL) score exhibited minimal variation, moving from 146 to 150. ConclusionThis case suggests that plum-blossom needle therapy (PBNT) is well-tolerated by children and may be effective in alleviating tics associated with Tourette syndrome (TS). However, further research, particularly large-sample randomized controlled trials, is imperative to establish the efficacy and effectiveness of PBNT.

Patient Outcomes After Robotic Ventral Rectopexy With Sacrocolpopexy.

As few studies exist examining postoperative functional outcomes in patients undergoing robotic sacrocolpopexy and ventral rectopexy, results from this study can help guide surgeons in counseling patients on their outcomes. The aim of the study was to evaluate functional outcomes and overall postoperative satisfaction as measured by the Pelvic Floor Disability Index 20 (PFDI-20), Pelvic Organ Prolapse/Urinary Incontinence Sexual Questionnaire (PISQ-12), and Patient Global Impression of Improvement Scale (PGI-I) in patients who underwent combined robotic ventral rectopexy and sacrocolpopexy for concomitant pelvic organ prolapse (POP) and rectal prolapse or intussusception (RP/I). This was a retrospective cohort and survey study of patients with combined POP and RP/I who underwent the previously mentioned surgical repair between January 2018 and July 2021. Each patient was contacted to participate in a survey evaluating postoperative symptoms related bother, sexual function, and overall satisfaction using the PFDI-20, PISQ-12, and PGI-I. A total of 107 patients met study inclusion criteria with 67 patients completing the surveys. The mean age and body mass index were 63.7 ± 11.5 years and 25.0 ± 5.4, respectively. Of the patients, 19% had a prior RP repair and 23% had a prior POP repair. Rectal prolapse or intussusception recurrence was reported in 10.4% of patients and objective POP recurrence was found in 7.5% of patients. Sixty-seven patients (62%) completed the surveys. The median time to survey follow-up was 18 (8.8-51.8) months. At the time of survey, the mean PFDI-20 score was 95.7 ± 53.7. The mean PISQ-12 score for all patients was 32.8 ± 7.2 and the median PGI-I score was 2.0 (interquartile range, 1.0-3.0). In this cohort of patients who underwent a combined robotic ventral rectopexy and sacrocolpopexy, patient-reported postoperative symptom bother was low, sexual function was high, and their overall condition was much improved.

Εfficacy and safety of vortioxetine (Lu AA21004) in the treatment of adult patients with major depressive disorder: A systematic review and a meta‑analysis of randomized controlled trials.

Vortioxetine is a novel drug for the treatment of major depressive disorder (MDD). It has been reported that vortioxetine exhibits positive effect on the acute stage of MDD, while it can effectively prevent the recurrence of MDD during the maintenance period. Currently, the results of systematic reviews on vortioxetine are insufficient since several efficacy measures, such as the 24-Items Hamilton Rating Scale for Depression (HADRS-24) total score and other safety factors have not been evaluated. Therefore, the present study aimed to evaluate the efficacy and safety of different doses of vortioxetine on the treatment of adult patients with MDD via assessing more efficacy and safety indicators. The clinical, double-blind, parallel and randomized controlled trials (RCTs) on the effect of vortioxetine on MDD were retrieved from PubMed\Medline, EBSCO, Embase, Cochrane Library, OVID, Web of Science and clinical trial registration websites from database inception to November 2022. A total of two investigators independently screened the included references and independently evaluated their quality. The meta-analysis was performed using Revman 5.0 software. The present systematic review was registered in PROSPERO (registration no. CRD42018106343). In the present study 11 RCTs were included, with a total of 4,908 adult patients with MDD. More specifically, 1,158 patients were included in the 5-mg vortioxetine group, 736 in the 10-mg group, 298 in the 15-mg group, 864 in the 20-mg group and 1,852 in the placebo group. All 11 studies were randomized, double-blinded and parallel control trials, and all publications were evaluated as high quality. The meta-analysis results showed that patients in the 5-, 10- and 20-mg vortioxetine groups exhibited significantly higher Montgomery-Asberg Depression Rating Scale (MADRS) response (≥50%) and remission (≤10%) rates compared with the placebo group (P<0.05). The pooled analysis also revealed a statistically significant change in the total score of HADRS-24, MADRS, Sheehan Disability Scale (SDS), Clinical Global Impression Scale-Improvement (CGI-I) and HADRS-24 response rate in the 10- and 20-mg vortioxetine groups compared with the placebo group (P<0.05). However, no statistically significant changes in the total score of HADRS-24, MADRS, SDS, CGI-I and HADRS-24 response rate were obtained in the 5-mg group compared with the placebo group (P>0.05). Furthermore, the most common adverse events were nausea, hyperhidrosis, insomnia and vomiting, the incidence of which was increased with higher doses of vortioxetine. Overall, the results suggested that vortioxetine administration at doses of 5-20 mg was significantly effective and safe compared with placebo in the treatment of MDD. However, 5 mg vortioxetine displayed no difference in the HADRS-24, MADRS, SDS and CGI-I total scores, and HADRS-24 response rate. Furthermore, patient treatment with increasing vortioxetine doses was associated with good tolerance and high safety. Nevertheless, more multi-center, high-quality and long-term RCTs are still needed to support the aforementioned findings.

Comparative analysis of transvaginal natural orifice transluminal endoscopic surgery versus laparoendoscopic single-site sacrocolpopexy for pelvic organ prolapse: A propensity score matching study

PurposeTo compare the safety, feasibility, and effectiveness of transvaginal natural orifice transluminal endoscopic sacrocolpopexy (vNOTES-SC) and laparoendoscopic single-site sacrocolpopexy (LESS-SC) for pelvic organ prolapse (POP). MethodNinety-four patients with POP who underwent vNOTES-SC or LESS-SC from October 2016 to November 2018 were included. The propensity score matching method was used for 1:1 matching between the two surgery groups. After matching, the general perioperative indicators, surgical complications, and the subjective and objective therapeutic effects of the two groups 3 years post-surgery were analyzed. ResultsAfter matching, 36 patients in each group were included, exhibiting balanced and comparable baseline data and an average follow-up of 48.6 ± 7.44 months. The operation time and postoperative hospitalization days were significantly reduced in the vNOTES-SC group (P < 0.05). However, perioperative complication incidence was not significantly different between the two groups (P > 0.05). Additionally, no significant differences were detected in de novo stress urinary incontinence (16.7% vs. 13.9%), de novo overactive bladder (de novo OAB, 8.3% vs. 0.0%), urination disorder (2.8% vs. 0.0%), defecation disorder (0.0% vs. 2.8%), lumbosacral pain (0.0% vs. 2.8%), or mesh complication (2.8% vs. 5.6%) incidences between the vNOTES-SC and LESS-SC groups (P > 0.05). Prolapse recurrence was not reported in either group. The quantitative description of pelvic organ position (POP-Q), Pelvic Floor Impact Questionnaire-7 (PFIQ-7), and Patient Global Impression of Improvement scale (PGI-I) scores showed improvement after the operation, but no significant differences were observed between the two groups (P > 0.05). ConclusionThe 3-year follow-up revealed that vNOTES-SC and LESS-SC had similar complications and efficacy rates. Compared with LESS-SC, vNOTES-SC resulted in shorter operation time and fewer postoperative hospitalization days (corresponding to the enhanced recovery after surgery [ERAS] concept), along with better cosmetic results without a scar. Therefore, our study findings suggest that clinicians should choose the surgery method based on the specific situation, and we recommend choosing vNOTES-SC when both surgeries are suitable.

The 1450-nm Diode Laser Reduces Redness and Porphyrin Density: An Image-Based, Patient-Oriented Appraisal.

Acne vulgaris remains the leading dermatological condition. The efficacy of laser treatment has been supported by many clinical studies, but studies investigating its multidimensional action are lacking. To comprehensively investigate the efficacy of 1450-nm diode laser treatment in patients with inflammatory acne and provide objective and subjective data for doctors in clinical practice. This retrospective study included patients with inflammatory acne lesions who underwent three courses of 1450-nm diode laser treatment between October 2019 and August 2020. Facial surface analysis was performed via objective computer assessments using the Canfield VISIA imaging system. Post-treatment subjective assessments were retrieved and analyzed using the clinical global impression-improvement index (CGI-I) and patient global impression of improvement scales (PGI-I). The final analysis included 20 patients. The changes in the porphyrin VISIA system scores demonstrated significant improvement, with median scores being 35.83, 48.83, and 54.83, respectively. The changes in the red area VISIA scores also showed improvement, with the median scores being 48, 50.33, and 58.83, respectively. The average CGI-I scale scores were 2.2 ± 1.01, 1.70 ± 0.80, and 1.50 ± 0.76, respectively (p = 0.001), and the average PGI-I scale scores were 3.10 ± 0.85, 3.10 ± 0.97, and 3.05 ± 0.95, respectively (p = 0.727), with no significant changes observed in sebum production. The present study is the first to provide objective and subjective evidence proving that the 1450-nm diode laser can reduce inflammatory acne lesions.

0704 A PSG Study of the Effect of Clonazepam and Melatonin on REM Sleep without Atonia in Isolated REM Sleep Behavior Disorder

Abstract Introduction Although very few prospective clinical trials are available assessing the benefits of drugs in isolated REM sleep behavior disorder (iRBD), clonazepam and melatonin are considered as the first-line treatment options. However, while evidence supporting the efficacy of these two compounds on the clinical behavioral outcomes seems to be clear, it is not clear if they are able to significantly modify the main polysomnographic (PSG) feature of iRBD, i.e. REM sleep without atonia (RSWA). For this reason, we carried-out an observational cross-sectional retrospective PSG study of the effects of clonazepam and melatonin on RSWA. Methods A total of 83 iRBD patients (70 males and 13 females, age range 50.9-83.2 years) were enrolled in this study: 43 drug-free, 21 patients taking at bedtime chronically (&amp;gt;6 months) clonazepam (0.5-2 mg), seven patients taking melatonin extended-release alone (2-3 mg), and 12 taking a combination of clonazepam and melatonin extended-release (same doses as above). PSG studies were assessed for all subjects, including the measurement of the automatic REM sleep atonia index (RAI) and periodic leg movements during sleep, as well as a series of demographic and clinical variables, such as age, age at onset, disease duration, clinical global impression scale (severity and improvement), mini-mental state evaluation, and RBD severity scale. Results None of the outcomes considered in this study showed significant differences between the groups considered. All showed low average values of RAI, as expected, and there were no differences between patients taking clonazepam, melatonin, or a combination of them (drug-free 0.778±0.184, clonazepam 0.797±0.183, clonazepam+melatonin 0.692±0.224, melatonin 0.673±0.228; ANOVA F = 1.329, p = 0.271), despite the clinical global impression scale-improvement was reported to be “much improved” or “minimally improved” in all treated patients. Conclusion This study confirms our previous findings in smaller patient series taking only clonazepam and suggest that both clonazepam and melatonin might be beneficial on some clinical manifestations of iRBD but are unable to modify its underlying neurophysiology. This might also indicate that these agents do not seem to interfere with the core mechanisms of this disease and other disease modifying drugs need to be discovered for iRBD. Support (if any)

Using the iNPH Radscale as a Radiological Predictor of Clinical Outcomes after Ventriculoperitoneal Shunt Placement in Patients with Idiopathic Normal Pressure Hydrocephalus (iNPH) (P9-11.014)

<h3>Objective:</h3> To determine the utility of the iNPH Radscale in predicting clinical outcomes of VPS placement in patients with NPH. <h3>Background:</h3> NPH is treated by placement of a VPS; however, there is no standardized approach to determining surgical candidacy. The iNPH Radscale is an established scoring system for CT/MRI scans that evaluates seven radiographic features characteristic of iNPH. A Radscale score of ≥8 suggests a high probability of an iNPH diagnosis. This study aimed to assess whether the pre-surgical iNPH Radscale could be used to help determine surgical candidacy. <h3>Design/Methods:</h3> Patients with a clinical diagnosis of NPH who underwent VPS placement between 1/1/10–6/30/21 were included in the study. The seven Radscale parameters were measured on patients’ pre-surgical MRIs. Patient charts were reviewed to determine clinical ambulation, cognition, and urination outcomes closest to one year post-VPS and were graded using the Clinical Global Impression Scale-Improvement (CGI-I). Chi-square and ANOVA were used to determine the relationship between total Radscale scores and CGI-I scores. The Fisher’s Exact Test was used to determine the association between each Radscale sub-score and CGI-I scores. <h3>Results:</h3> Out of 180 patients, 134 had complete Radscale scores and were included in this analysis. Fifty-nine percent had scores of ≥8, and 41% had scores &lt;8. Forty-seven percent had CGI-I ambulation scores that reflected significant improvement, and 45% demonstrated insignificant improvement or worse outcomes. There was no evidence of a significant association between the total Radscale score and CGI-I scores after VPS; nor was there evidence of a significant association between the Radscale sub-scores and CGI-I outcomes. <h3>Conclusions:</h3> This study suggests both total and sub-scores of the iNPH Radscale ratings based on pre-VPS placement imaging have no clear association with clinical improvement after VPS placement. Further research is needed to identify preoperative parameters that are predictive of positive shunt response. <b>Disclosure:</b> Miss Ghosh has nothing to disclose. Dr. Gilson has nothing to disclose. Dr. Kundrick has nothing to disclose. Mr. Baugher has nothing to disclose. The institution of Dr. Liao has received research support from American Parkinson Disease Association. Dr. Salles has nothing to disclose. Ms. Sonneborn has nothing to disclose. Olivia Hogue has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Stoparkinson Healthcare Systems, LLC. Mr. Fellows has nothing to disclose. Dr. Fernandez has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbvie. Dr. Fernandez has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cerevel. Dr. Fernandez has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amneal. Dr. Fernandez has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Elsevier. The institution of Dr. Fernandez has received research support from Biogen. The institution of Dr. Fernandez has received research support from Michael J Fox Founda. The institution of Dr. Fernandez has received research support from Roche. The institution of Dr. Fernandez has received research support from Parkinson Foundation. The institution of Dr. Fernandez has received research support from UCB. Dr. Fernandez has received publishing royalties from a publication relating to health care. Dr. Fernandez has received personal compensation in the range of $10,000-$49,999 for serving as a Steering Committee/Advisory Committee Member with Parkinson Study Group. Sean Nagel has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Abbott. Sean Nagel has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Medtronic. The institution of Sean Nagel has received research support from Hydrocephalus Association.

26 Mandibular Advancement vs Home Treatment for Primary Snoring: A Randomized Trial

OBJECTIVES/GOALS: The primary aim is to evaluate the efficacy of a Mandibular Advancement Device (MAD) vs conservative treatment for adults with non-apneic snoring, as measured by the sleeping partner. The secondary aim is to evaluate the effectiveness of treatment of snoring on the sleeping partner’s sleep quality. METHODS/STUDY POPULATION: We plan to enroll 60 pairs of primary snorers and their sleeping partners in our randomized clinical trial. Snorers will be randomized to either 4 weeks of conservative therapy, consisting of nightly Mometasone nasal rinse, breathe-rite strips, mouth taping, and lateral positional therapy, or 4 weeks of Mandibular Advancement Device therapy (MAD). 30 pairs of snorers and their partners will be in each arm. At follow up the primary outcome measure, the Clinical Global Impression of Improvement Scale (CGI-I), will be assessed by the sleeping partner to evaluate the response to snoring treatment. RESULTS/ANTICIPATED RESULTS: To date, there is no study reporting the rate of response in participants using MAD in Primary Snoring. Due to lack of preliminary data and effect size, we hypothesize that the rate of the responders in the MAD group will be 20% higher than the rate of responders in the active control group based on literature studies and preliminary results. A responder will be classified as someone whose sleeping partner rates on the CGI-I scale that the snoring was much improved or very much improved. MAD has been shown previously to be an effective therapy at treating sleep apnea and reducing snoring, and we anticipate it will continue to be so for patients who do not have sleep apnea. DISCUSSION/SIGNIFICANCE: Snoring is a nearly ubiquitous problem that prevents restful sleep for spouses of snorers, which is known to have detrimental health effects. Yet it does not have scientifically proven treatments. Our study will evaluate these treatments in an effort to improve the health of the sleeping partners.

Efficacy and safety of duloxetine in painful diabetic peripheral neuropathy: a systematic review and meta-analysis of randomized controlled trials

BackgroundPainful diabetic peripheral neuropathy (PDPN) is a key concern in clinical practice. In this systematic review and meta-analysis, we compared duloxetine and placebo treatments in terms of their efficacy and safety in patients with PDPN.MethodsFollowing the PRISMA guidelines, we searched the Cochrane Library, PubMed, and Embase databases for relevant English articles published before January 11, 2021. Treatment efficacy and safety were assessed in terms of pain improvement, patient-reported health-related performance, and patients’ quality of life.ResultsWe reviewed a total of 7 randomized controlled trials. Regarding pain improvement, duloxetine was more efficacious than placebo (mean difference [MD] − 0.89; 95% confidence interval [CI] − 1.09 to − 0.69; P < .00001). Furthermore, duloxetine significantly improved the patients’ quality of life, which was assessed using the Clinical Global Impression severity subscale (MD − 0.48; 95% CI − 0.61 to − 0.36; P < .00001), Patient Global Impression of Improvement scale (MD − 0.50; 95% CI − 0.64 to − 0.37; P < .00001), and European Quality of Life Instrument 5D version (MD 0.04; 95% CI 0.02 to 0.07; P = .0002). Severe adverse events were rare, whereas nausea, somnolence, dizziness, fatigue, constipation, and decreased appetite were common; approximately, 12.6% of all patients dropped out because of the common symptoms.ConclusionsDuloxetine is more efficacious than placebo treatments in patients with PDPN. The rarity of severe adverse events indicates that duloxetine is safe. When a 60-mg dose is insufficient, 120 mg of duloxetine may improve PDPN symptoms. Our findings may help devise optimal treatment strategies for PDPN.Systematic review registrationPROSPERO CRD42021225451

Determining the optimal initial dose for Japanese patients with nocturnal polyuria using an initial dose of desmopressin 50 μg.

There is no consistent opinion on the optimal initial dose of desmopressin for patients with nocturnal polyuria. Over a period of 12 weeks, we investigated the safety and efficacy of an initial dose of 50 μg of desmopressin for elderly men. Eighty patients (mean age: 78.8 years) were started on an initial dose of 50 μg of desmopressin for nocturia associated with nocturnal polyuria. Safety and efficacy were evaluated after 1, 4, and 12 weeks using a frequency-volume chart, Athens Insomnia Scale, Patient Global Impression of Improvement scale, physical examination, blood tests, and a body composition analyzer. Along with reduction in the frequency and volume of night-time urination, improvements in hours of undisturbed sleep, nocturnal polyuria index, and International Prostate Symptom Score, and Overactive Bladder Symptom Scores on quality of life measures were also observed. Hyponatremia was observed in 15 patients (18.7%). However, only 5.0% of patients had hyponatremia after the dose was reduced to 25 μg, and the continuation rate at 12 weeks was high at 87.5%. Age and other physical factors, such as body mass index, body water content, body fat mass, and muscle mass were not significant predictors of adverse events. Our study suggests that an initial dose of 50 μg is more effective than a uniformly minimum dose based on factors such as age and physique. Furthermore, a high continuation rate can be achieved by appropriately reducing the dose, if adverse events occur.

Interprofessional Case Conference Enhances Group Learning and the Quality, Safety, Value, and Equity of Team-Based Care.

Patients seeking treatment for complex conditions require coordinated care from interprofessional clinicians. Collaborative engagement in an interprofessional community of practice is crucial to the collective competence of a team and the provision of high-quality, safe health care leading to improved patient outcomes. The objective of this descriptive, cross-sectional study was to describe interprofessional communication, coordination, and collaboration of participants in an integrated practice unit that was structured to include weekly case conferences as part of routine practice. Data were collected from October 2019 to February 2020. Web-based surveys were administered to a convenience sample that included 33 questions and followed the CHERRIES checklist for reporting results. Items focused on team knowledge, impact on patient care, and communication, and conference focus and effectiveness. Descriptive and survey item analysis included frequency, percentage, means and standard deviation, Chi-square, and Pearson correlation analysis. Patient outcome data were collected via a Patient Global Impression of Improvement scale and were analyzed using a paired sample t test. Survey respondents (n = 161) included clinicians and administrative staff. Results demonstrated that interprofessional case conferences improved the collective competence of the team, including team knowledge and communication. Participants viewed case conferences as a means to enhance care delivery quality, value, safety, and equity. In the study period, there was also a statistically significant improvement between the patient's first follow-up and last visits. Survey respondents indicated that case conferences were an effective means to deliver high-quality, patient-centered care through interprofessional collaboration and education.

Effect of Pessary vs Surgery on Patient-Reported Improvement in Patients With Symptomatic Pelvic Organ Prolapse

Pelvic organ prolapse is a prevalent condition among women that negatively affects their quality of life. With increasing life expectancy, the global need for cost-effective care for women with pelvic organ prolapse will continue to increase. To investigate whether treatment with a pessary is noninferior to surgery among patients with symptomatic pelvic organ prolapse. The PEOPLE project was a noninferiority randomized clinical trial conducted in 21 participating hospitals in the Netherlands. A total of 1605 women with symptomatic stage 2 or greater pelvic organ prolapse were requested to participate between March 2015 through November 2019; 440 gave informed consent. Final 24-month follow-up ended at June 30, 2022. Two hundred eighteen participants were randomized to receive pessary treatment and 222 to surgery. The primary outcome was subjective patient-reported improvement at 24 months, measured with the Patient Global Impression of Improvement scale, a 7-point Likert scale ranging from very much better to very much worse. This scale was dichotomized as successful, defined as much better or very much better, vs nonsuccessful treatment. The noninferiority margin was set at 10 percentage points risk difference. Data of crossover between therapies and adverse events were captured. Among 440 patients who were randomized (mean [SD] age, 64.7 [9.29] years), 173 (79.3%) in the pessary group and 162 (73.3%) in the surgery group completed the trial at 24 months. In the population, analyzed as randomized, subjective improvement was reported by 132 of 173 (76.3%) in the pessary group vs 132 of 162 (81.5%) in the surgery group (risk difference, -6.1% [1-sided 95% CI, -12.7 to ∞]; P value for noninferiority, .16). The per-protocol analysis showed a similar result for subjective improvement with 52 of 74 (70.3%) in the pessary group vs 125 of 150 (83.3%) in the surgery group (risk difference, -13.1% [1-sided 95% CI, -23.0 to ∞]; P value for noninferiority, .69). Crossover from pessary to surgery occurred among 118 of 218 (54.1%) participants. The most common adverse event among pessary users was discomfort (42.7%) vs urinary tract infection (9%) following surgery. Among patients with symptomatic pelvic organ prolapse, an initial strategy of pessary therapy, compared with surgery, did not meet criteria for noninferiority with regard to patient-reported improvement at 24 months. Interpretation is limited by loss to follow-up and the large amount of participant crossover from pessary therapy to surgery. Netherlands Trial Register Identifier: NTR4883.

A preliminary study of comprehensive behavioral intervention for tics in Chinese children with chronic tic disorder or Tourette syndrome.

To investigate the adaptability of Comprehensive Behavioral Intervention for Tics (CBIT) for a Chinese population, and evaluate the efficacy of combined CBIT and pharmacotherapy (CBIT + PT) compared to CBIT or pharmacotherapy (PT) alone for reducing tics and for improving the quality of life (QoL) in a sample of Chinese children with chronic tic disorders (CTD) and Tourette syndrome (TS). In this 10-week randomized controlled pilot trial, 37 outpatients aged between 6 and 16 years affected by TS and CTD were randomly assigned to receive CBIT (n = 22) or PT alone (n = 15). Considering the feasibility, the patients allocated to the CBIT treatment group could further choose whether to simultaneously take medicine voluntarily, resulting in a CBIT alone group (n = 12) and a CBIT + PT group (n = 10). At baseline, no significant difference was found between the three groups in the demographic and clinical characteristics (p > 0.05). All three groups showed a significant reduction in tic severity after treatment assessed by the Yale Global Tic Severity Scale (YGTSS) severity score [F (2,33) = 35.05, p < 0.001, η p 2 = 0.51], the score of the Clinical Global Impression scale for Improvement (CGI-I) [F (1,34) = 13.87, p = 0.001, η p 2 = 0.29], and YGTSS impairment score [F (2,33) = 31.71, p < 0.001, η p 2 = 0.48]. Significant interactions were found between the time-point and group in emotional functioning [F (2,29) = 4.39, p = 0.02, η p 2 = 0.23], psychosocial functioning [F (2,29) = 5.93, p = 0.007, η p 2 = 0.29], and total QoL score [F (1,34) = 3.72, p = 0.04, η p 2 = 0.20] of Pediatric Quality of Life Inventory (PedsQL 4.0) for children suggesting a significantly larger improvement in emotional functioning, psychosocial functioning, and total QoL score of the life quality in the CBIT group for children self-report. PedsQL for proxy report only showed a significant main effect of time-point in physical functioning [F (1,33) = 8.35, p = 0.01, η p 2 = 0.2], emotional functioning [F (1,33) = 10.75, p = 0.002, η p 2 = 0.25], psychosocial functioning [F (1,34) = 11.38, p = 0.002, η p 2 = 0.26], and total Qol score [F (1,34) = 13.21, p = 0.001, η p 2 = 0.29]. CBIT is probably effective in reducing tic severity in Chinese children with tic disorders. CBIT + PT may not be superior to CBIT alone in reducing tic severity and improving quality of life. CBIT alone showed advantages in improving quality of life over CBIT + PT and PT alone. CBIT might be an appropriate treatment option for patients with tic disorder in Chinese mainland.

Effect of fenfluramine on seizures and comorbidities in SCN8A-developmental and epileptic encephalopathy: A case series.

SCN8A‐developmental and epileptic encephalopathy is caused by pathogenic variants in the SCN8A gene encoding the Nav1.6 sodium channel, and is characterized by intractable multivariate seizures and developmental regression. Fenfluramine is a repurposed drug with proven antiseizure efficacy in Dravet syndrome and Lennox–Gastaut syndrome. The effect of fenfluramine treatment was assessed in a retrospective series of three patients with intractable SCN8A epilepsy and severe neurodevelopmental comorbidity (n = 2 females; age 2.8–13 years; 8–16 prior failed antiseizure medications [ASM]; treatment duration: 0.75–4.2 years). In the 6 months prior to receiving fenfluramine, patients experienced multiple seizure types, including generalized tonic–clonic, focal and myoclonic seizures, and status epilepticus. Overall seizure reduction was 60%–90% in the last 3, 6, and 12 months of fenfluramine treatment. Clinically meaningful improvement was noted in ≥1 non‐seizure comorbidity per patient after fenfluramine, as assessed by physician‐ratings of ≥“Much Improved” on the Clinical Global Impression of Improvement scale. Improvements included ambulation in a previously non‐ambulant patient and better attention, sleep, and language. One patient showed mild irritability which resolved; no other treatment‐related adverse events were reported. There were no reports of valvular heart disease or pulmonary arterial hypertension. Fenfluramine may be a promising ASM for randomized clinical trials in SCN8A‐related disorders.

Minimal clinically important difference of the King’s Parkinson’s disease Pain Scale

Objective Pain is a common and debilitating symptom of Parkinson's disease (PD) and has no specific treatment. King's Parkinson's disease Pain Scale (KPPS) is the only specific scale for pain measurement in PD with established psychometric properties. The minimal clinically important difference (MCID) of KPPS, an important parameter for the design and interpretation of therapeutic interventions, has not yet been measured. The aim of our study was to assess the MCID of KPPS. Methods Two hundred and seven PD patients were evaluated by KPPS before and after receiving the intervention. The Clinical Global Impression of Improvement Scale was used as an anchor, and a Receiver Operating Characteristic (ROC) curve was used to determine the optimal MCID cut-off point for KPPS. The distribution-based approach applied one-third standard deviation (SD), 0.5 SD, and one standard error of measurement (SEM) of the total score of KPPS to determine the MCID. Results The MCID achieved from the ROC curve was 3 points (sensitivity: 74.4%; specificity: 81.9%). For the distribution-based method, the MCIDs corresponding to 0.3 SD, 0.5 SD, and one SEM were 5.65, 9.41, and 2.54 points, respectively. Conclusion KPPS is a valid scale for measuring pain in PD with demonstrable MCID. IMPLICATIONS FOR REHABILITATION The King's Parkinson's disease Pain Scale (KPPS) is a valid scale for measuring pain in patients with Parkinson's disease (PD) with demonstrable minimal clinically important difference (MCID). The MCID obtained in the current study will assist clinicians and researchers when interpreting KPPS change score to determine clinically meaningful changes of pain in both PD progression and response to interventions.

Findings from a pilot open-label trial of N-acetylcysteine for the treatment of pediatric mania and hypomania

BackgroundPediatric bipolar disorder is a highly prevalent and morbid disorder and is considered a prevalent public health concern. Currently approved treatments often pose the risk of serious side effects. Therefore, this study assessed the efficacy and tolerability of N-acetylcysteine (NAC), in children and adolescents with bipolar spectrum disorder.MethodsWe conducted a 12-week open-label trial of NAC for treatment of mania and hypomania in children and adolescents ages 5–17 with bipolar spectrum disorder including participants with full and subthreshold manic symptoms, accepting those with and without mixed states with co-occurring depression, and Young Mania Rating Scale scores ≥ 20 and < 40. Symptoms of mania and depression were assessed using the Young Mania Rating Scale (YMRS), Hamilton Depression Rating Scale (HDRS), Children’s Depression Rating Scale (CDRS), and Clinical Global Impression (CGI) Severity (CGI-S) and Improvement (CGI-I) scales for mania and depression.ResultsThis study had a high drop-out rate with only 53% completing all 12 weeks. There was a significant reduction in YMRS, HDRS, and CDRS mean scores from baseline to endpoint. Of the 24 exposed participants, 54% had an anti-manic response measured by a reduction in YMRS ≥ 30% and 46% had a CGI-I mania score ≤ 2 at endpoint. Additionally, 62% of participants had an anti-depressive response measured by a reduction in HDRS ≥ 30%, 31% had an anti-depressive response measured by a reduction in CDRS ≥ 30%, and 38% had a CGI-I depression score ≤ 2 at endpoint.ConclusionsThese pilot open-label findings in a small sample provide preliminary data supporting the tolerability and safety of NAC in a pediatric population. The findings of this pilot scale study indicating improvement in mania and depression are promising, but require replication with a monotherapy randomized placebo controlled clinical trial and larger sample.Trial RegistrationClinicalTrials.gov Identifier: NCT02357290. First Registration 06/02/2015.