Mean Change Research Articles

Changes in blood pressure after catheter-based renal denervation in South Africa.

Renal denervation (RDN) is an interventional treatment for patients with uncontrolled hypertension. The Global SYMPLICITY Registry (GSR) is a prospective, all-comer, world-wide registry designed to assess the safety and efficacy of RDN. We evaluated the outcomes in South African patients in the GSR over 12 months. Eligible patients with hypertension had a daytime mean blood pressure (BP) > 135/85 mmHg or night-time mean BP > 120/70 mmHg. Office and 24-hour ambulatory systolic BP reduction and adverse events over 12 months were evaluated. South African patients (n = 36) in the GSR had a mean age of 54.4 ± 9.9 years with a median of four prescribed antihypertensive medication classes. At 12 months, mean changes in office and 24-hour ambulatory systolic BP were -16.9 ± 24.2 and -15.3 ± 18.5 mmHg, respectively, with only one adverse event recorded. RDN safety and efficacy in South African patients were consistent with world-wide GSR results.

Cariprazine Augmentation in Treatment-Resistant Bipolar Depression: Data from a Retrospective Observational Study.

Treatment-resistant bipolar depression is one of the leading problems in psychiatry with serious consequences on patients functioning, quality of life and resource utilization. Despite this, there is a lack of consensus on diagnostic criteria and treatment algorithms. The objective of the present study is to assess the acute effectiveness and tolerability of cariprazine in the management of treatment resistant bipolar depression. This is a four weeks retrospective multicentric observational study on patients with treatment resistant bipolar depression receiving cariprazine in augmentation to the current treatment. Cariprazine dosage changed during the follow-up period according to clinical judgment. Since data followed a non-normal distribution, non-parametric tests were used to pursue the analysis. The effectiveness of cariprazine was assessed through the mean change in Hamilton Depression rating scale (HAM-D) scores from baseline to endpoint. For missing values, a "Last Observation Carried Forward" approach was applied. Fifty-one patients were enrolled. Four patients (7.8%) discontinued cariprazine mainly due to adverse events. Mean cariprazine dose was 1.7 mg/day. The mean HAM-D score decreased significantly from baseline (T0) to week 4 (T4) at each evaluation point. Fourty-five one percent of the patients benefited of cariprazine add-on strategy: 23.5% achieved a clinical response and 21.6% were remitters. Among the completers, 70.6% experienced at least one adverse event. All side effects were mild to moderate. Cariprazine seems to be an effective and well tolerated option in the management of patients with treatment resistant bipolar depression.

A novel multifamily treatment targeting symptoms related to Latinx caregiver deportation.

Latinx youth who have experienced caregiver deportation show higher rates of posttraumatic stress disorder (PTSD) and internalizing symptoms compared to Latinx youth who have not. Thus, there is a need for culturally and linguistically appropriate therapeutic approaches to address psychiatric needs related to caregiver deportation. Positive Adaptations for Trauma and Healing (PATH) is a manualized 10-week group therapy for Latinx youth and their caregivers that integrates trauma-informed interventions with positive psychology and resilience interventions. The present study examined PATH for reducing trauma symptomatology for Latinx youth who endured at least three traumatic experiences in their caregiving system as well as differences for Latinx youth who experienced their caregiver's deportation. Self-identified Latinx youth (N = 31) and their Spanish-speaking caregivers were recruited from two urban hospitals. All youth experienced three or more traumatic events. Pre- to postintervention mean change was measured with t tests; mixed-effects analysis of variance assessed whether the program was feasible for youth who had experienced a caregiver deportation versus Latinx youth who did not. Assessment with the Trauma Symptom Checklist and the UCLA PTSD Index for Youth showed statistically significant reductions on Anxiety, Depression, Anger, and PTSD subscales. Latinx youth who experienced a caregiver's deportation exhibited larger reductions in anger symptoms and PTSD symptoms. Study results suggest that the novel group therapy intervention was feasible and acceptable for Latinx youth and their caregivers. Despite a small sample size, the intervention proved feasible for Latinx youth who experienced caregiver deportation. (PsycInfo Database Record (c) 2024 APA, all rights reserved).

Predictors of response in PROMIS-global in a chronic low back pain specialty clinic: STarTBack and chronic overlapping pain conditions.

Tools, such as the STarTBack Screening Tool (SBT), have been developed to identify risks of progressing to chronic disability in low back pain (LBP) patients in the primary care population. However, less is known about predictors of change in function after treatment in the specialty care population. We pursued a retrospective observational cohort study involving LBP patients seen in a multidisciplinary specialty clinic to assess which features can predict change in function at follow-up. The SBT was administered at initial visit, and a variety of patient characteristics were available in the chart including the presence of chronic overlapping pain conditions (COPCs). Patient Reported Outcomes Measurement Information System-10 (PROMIS-10) global physical health (PH) and global mental health (MH) were measured at baseline and at pragmatic time points during follow-up. Linear regression was used to estimate adjusted associations between available features and changes in PROMIS scores. 241patients were followed for a mean of 17.0 ± 7.5months. Mean baseline pain was 6.7 (SD 2.1), PROMIS-10 global MH score was 44.8 (SD 9.3), and PH score was 39.4 (SD 8.6). 29.7% were low-risk on the SBT, 41.8% were medium-risk, and 28.5% were high-risk. Mean change in MH and PH scores from baseline to the follow-up questionnaire were 0.86 (SD 8.11) and 2.39 (SD 7.52), respectively. Compared to low-risk patients, high-risk patients had a mean 4.35points greater improvement in their MH score (p= 0.004) and a mean 3.54points greater improvement in PH score (p= 0.006). Fewer COPCs also predicted greater improvement in MH and PH. SBT and the presence of COPC, which can be assessed at initial presentation to a specialty clinic, can predict change in PROMIS following treatment. Effort is needed to identify other factors that can help predict change in function after treatment in the specialty care setting.

Adjunct role of potassium-rich vegetarian diet and a novel potassium food supplement to improve pain in chronic rheumatoid arthritis on supervised standard care: a randomised controlled study

IntroductionAn earlier food survey showed dietary potassium deficiency in rheumatoid arthritis (RA).ObjectiveTo evaluate an adjunct role of oral potassium to reduce joint pain in RA.Methods172 consenting eligible symptomatic patients (median...

Real-world Impact of 3 and 4.5 mg Doses of Dulaglutide on Weight and Hemoglobin A1c in Patients With Type 2 Diabetes Mellitus.

Limited real-world data on the benefits and risks associated with 3 and 4.5 mg doses of dulaglutide currently exists, making it difficult to determine the impact of dose titration for patients currently managed with dulaglutide 1.5 mg weekly. To determine the impact of dulaglutide 3 and 4.5 mg doses on weight and hemoglobin A1c (HbA1c) in patients with type 2 diabetes mellitus (T2DM), in clinical practice. Retrospective, observational study of adult T2DM patients receiving dulaglutide 3 or 4.5 mg weekly within a large, university-affiliated, primary care network. The primary outcome was change in weight and HbA1c from baseline to 24 weeks. Secondary outcomes included incremental changes in weight and HbA1c, and describing trends related to dose reductions. Ninety-five patients were included, 62 in the dulaglutide 3 mg group and 33 in the dulaglutide 4.5 mg group. After 24 weeks, the mean changes in weight and HbA1c from baseline were -1.8 kg (P < 0.01) and -0.4% (P < 0.01) in the 3 mg group, and -4.2 kg (P < 0.01) and -0.4% (P = 0.119) in the 4.5 mg group. Incremental change in weight and HbA1c among patients who were titrated from dulaglutide 3 to 4.5 mg weekly were -2.6 kg (P < 0.01) and -0.2% (P = 0.04), respectively. Titration from dulaglutide 1.5 to 3 mg resulted in significant reductions in weight and HbA1c after 24 weeks. Additional, statistically significant, reductions in weight and HbA1c were seen when patients were further titrated to dulaglutide 4.5 mg weekly.

Cross-Education Effects of Hip Strengthening in Individuals with Chronic Ankle Instability

ABSTRACT Introduction/Purpose Chronic ankle instability (CAI) is associated with reduced balance, lumbopelvic stability, and hip strength. Accordingly, hip strengthening is an effective intervention for individuals with CAI. Cross-education through unilateral exercise is also beneficial to individuals with CAI, but cross-education effects of hip strengthening remain unknown. The purpose of this study was to determine cross-education effects of hip strengthening in individuals with CAI. Methods Thirty individuals with CAI were randomized into control (CON) and hip strengthening (HIP) groups. Baseline and postintervention tests of the uninvolved limb included lumbopelvic stability (unilateral hip bridge), handheld dynamometry (isometric hip extension, abduction, and external rotation strength), and balance (Star Excursion Balance Test (SEBT) in anterior, posterolateral (PL), and posteromedial directions). After baseline testing, HIP completed progressive hip strengthening of the involved limb three times per week for 8 wk, and CON did not. Repeated-measures analyses of variance assessed the effects of group and time on each outcome. Each group’s mean changes from baseline to postintervention were assessed via effect sizes. Statistical significance was set a priori at P &lt; 0.05. Results Of the 30 participants enrolled, 10 did not complete the postintervention data collection. Multiple imputations estimated missing data, and an intention-to-treat analysis was conducted. There were no significant group-by-time interactions for hip strength, balance, or lumbopelvic stability. HIP exhibited moderate effect sizes for SEBT-PL and hip bridge. All other effect sizes were small or negligible. Conclusions We did not identify a clear cross-education effect following unilateral hip strengthening in a cohort with CAI. Examination of baseline to postintervention effect sizes indicate a potential trend toward improved balance and lumbopelvic stability of the HIP group.

Abstract 3404: Body weight trajectory among patients with non-small-cell lung cancer receiving immune checkpoint inhibitors

Abstract Objectives: Body weight is an important prognostic factor in patients with lung cancer. As immunotherapy involves toxicities, body weight may be affected during the treatment course. The objective of this study is to examine the trajectory of weight changes at 180 and 365 days after the initiation of immune checkpoint inhibitors (ICIs) among patients with NSCLC. Methods: Retrospective chart review and data extraction were performed among patients receiving ICIs at the Ohio State University Comprehensive Cancer Center between 2015 and 2021. Patients with NSCLC were included in this study if their initial treatment entailed a PD-1/PD-L1 ICI), either alone or in combination with chemotherapy/targeted cancer drugs, and if they also had their body weights recorded on ICI initiation and 180 days after (± 30 days) treatment initiation. A subset of these patients who had body weight data on day 365 of ICI initiation were included in additional analysis. Percentages of change in body weight relative to pre-ICI were calculated ([weight at 180 or 360 days minus pre-ICI weight] divided by pre-ICI weight x 100%) and examined by treatment regimen and pre-ICI body mass index (BMI) using t-tests. A sensitivity analysis was performed by excluding 11 patients with cachexia defined by the presence of ICD-9 (799.4) or 10 (R64) codes in their medical records. Results: A total of 554 patients with NSCLC received any ICI; 139 patients met the inclusion criteria, of which 96 had body weight data on day 365. The mean age of diagnosis was 63.8 (SD=10.1); 80% were White and 18.7% were Black. The median time on ICI was 195 days (inter-quartile range, 55-392 days) among patients with ICI only and 255 days (inter-quartile range, 126-479 days) among patients with ICI combined with chemotherapy or targeted cancer drugs. Among patients receiving ICI only, body weight increased among those with normal weight (BMI &amp;lt;25.0; mean change = +7.57%, P=0.013) but decreased among those classified as overweight (BMI = 25.0 to 29.9; −5.2%, P=0.037) or obese (BMI ≥30; −6.5%, P=0.025). Decreases in weight were also observed for patients with obesity and receiving a combined treatment (−9.65%, P=0.043). After one year of treatment, weight changes among patients receiving ICI only remained significant in the normal weight group (+15.6%, P=0.007), but not among patients with other BMIs and treatment regimens. These associations remained similar after excluding the patients with cachexia. Conclusions: Body weight change during ICI therapy may vary by treatment regimen and pre-ICI BMI among patients with NSCLC. Whether the weight changes are related to toxicities and survival warrants further research to shed light on weight management, ICI efficacy, and prevention of treatment-related adverse events. Funding: The study was in part funded by NIH/NCI R37CA248371, OSUCCC CCSG P30CA016058, and Pelotonia Institute for Immuno-Oncology (PIIO) at OSUCCC. Citation Format: Ting-Yuan David Cheng, Ben Kinder, Rand T. Akasheh, Cuthbert Mario Mahenge, Carolyn J. Presley, Peter G. Shields, Xuan Nguyen, Aditi Shendre, Zihai Li, Lang Li. Body weight trajectory among patients with non-small-cell lung cancer receiving immune checkpoint inhibitors [abstract]. In: Proceedings of the American Association for Cancer Research Annual Meeting 2024; Part 1 (Regular Abstracts); 2024 Apr 5-10; San Diego, CA. Philadelphia (PA): AACR; Cancer Res 2024;84(6_Suppl):Abstract nr 3404.

Efficacy and safety of switching to dolutegravir/lamivudine in virologically suppressed people with HIV-1 aged ≥ 50 years: week 48 pooled results from the TANGO and SALSA studies

BackgroundAs the population of people with HIV ages, concerns over managing age-related comorbidities, polypharmacy, immune recovery, and drug-drug interactions while maintaining viral suppression have arisen. We present pooled TANGO and SALSA efficacy and safety results dichotomized by age (< 50 and ≥ 50 years).MethodsWeek 48 data from the open-label phase 3 TANGO and SALSA trials evaluating switch to once-daily dolutegravir/lamivudine (DTG/3TC) fixed-dose combination vs continuing current antiretroviral regimen (CAR) were pooled. Proportions of participants with HIV-1 RNA ≥ 50 and < 50 copies/mL (Snapshot, intention-to-treat exposed) and safety were analyzed by age category. Adjusted mean change from baseline in CD4 + cell count was assessed using mixed-models repeated-measures analysis.ResultsOf 1234 participants, 80% of whom were male, 29% were aged ≥ 50 years. Among those aged ≥ 50 years, 1/177 (< 1%) DTG/3TC participant and 3/187 (2%) CAR participants had HIV-1 RNA ≥ 50 copies/mL at 48 weeks; proportions with HIV-1 RNA < 50 copies/mL were high in both treatment groups (≥ 92%), consistent with overall efficacy and similar to observations in participants aged < 50 years (≥ 93%). Regardless of age category, CD4 + cell count increased or was maintained from baseline with DTG/3TC. Change from baseline in CD4 + /CD8 + ratio was similar across age groups and between treatment groups. One CAR participant aged < 50 years had confirmed virologic withdrawal, but no resistance was detected. In the DTG/3TC group, incidence of adverse events (AEs) was similar across age groups. Proportions of AEs leading to withdrawal were low and comparable between age groups. Although drug-related AEs were generally low, across age groups, drug-related AEs were more frequent in participants who switched to DTG/3TC compared with those who continued CAR. While few serious AEs were observed in both treatment groups, more were reported in participants aged ≥ 50 years vs < 50 years.ConclusionsAmong individuals with HIV-1, switching to DTG/3TC maintained high rates of virologic suppression and demonstrated a favorable safety profile, including in those aged ≥ 50 years despite higher prevalence of concomitant medication use and comorbidities.Trial registration number: TANGO, NCT03446573 (February 27, 2018); SALSA, NCT04021290 (July 16, 2019).

Dynamic Temporal and Tactile Cueing in Young Children With Childhood Apraxia of Speech: A Multiple Single-Case Design.

Childhood apraxia of speech (CAS) is a multivariate motor speech disorder that requires a motor-based intervention approach. There is limited treatment research on young children with CAS, reflecting a critical gap in the literature given that features of CAS are often in full expression early in development. Dynamic Temporal and Tactile Cueing (DTTC) is a treatment approach designed for children with severe CAS, yet the use of DTTC with children younger than 3 years of age has not been examined. A multiple single-case design was employed to examine the use of DTTC in seven children with CAS (aged 2.5-5 years) over the course of 6 weeks of intervention. Changes in word accuracy were measured in treated words from baseline to posttreatment and from baseline to maintenance (6 weeks posttreatment). Generalization of word accuracy changes to matched untreated words was also examined. A linear mixed-effects model was used to estimate the change in word accuracy for treated and untreated words across all children from baseline to posttreatment and to maintenance. A quasi-Poisson regression model was used to estimate mean change and calculate effect sizes for treated and untreated words. Group-level analyses revealed significant changes in word accuracy for treated and untreated words at posttreatment and maintenance. At the child level, six of seven children displayed medium-to-large effect sizes where word accuracy increased in an average of 3.4/5 words across all children. Each child displayed some degree of generalization to untreated targets, specifically for words with the same syllable shape as the treated words. These results demonstrate that DTTC can yield positive change in some young children with CAS. Key differences in each child's performance are highlighted.

Do changes in the frailty score differ by the type of group sports and exercises participated in? A 3-year longitudinal study

BackgroundOlder adults who engage in group sports and exercises achieve greater health benefits than those who exercise by themselves. The benefits of group participation may vary depending on the type of sports/exercise they engage in. The present study aimed to identify the association between specific sports and exercise types performed in groups and evaluate the longitudinal changes in multidimensional frailty scores among community-dwelling older adults in Japan.MethodsWe used 3-year follow-up data from the Japan Gerontological Evaluation Study and analyzed 33,746 men and 36,799 women aged ≥ 65 years. To elucidate the relationship between participation in 20 types of group sports/exercises in 2016 (baseline) and the change in frailty score (using the Kihon Checklist, KCL) from 2016 to 2019, we performed linear regression analyses through multivariate adjustments for age group, self-rated health, marital status, living alone, occupational status, years of education, alcohol drinking status, smoking status, equivalent income, and disease status using an inverse probability weighting method. P < 0.05 was considered statistically significant.ResultsThe mean change in KCL scores over 3 years was + 0.62 and + 0.61 points in men and women, respectively, implying the degree of frailty worsened. The sports/exercise types that significantly prevented increments in KCL scores for both sexes compared to non-participants were hiking (men: B, − 0.36; women: B, − 0.29), walking (men: B, − 0.26; women: B, − 0.24), tennis (men: B, − 0.23; women: B, − 0.24), ground golf (men: B, − 0.21; women: B, − 0.19), and weight exercises (men: B, − 0.19; women: B, − 0.16).ConclusionParticipation in specific sports and exercise groups offer significant physical and psychological benefits for frailty prevention among older adults in Japan. The results of this study may offer substantive evidence to encourage older adults to participate in group activities for the prevention of multidimensional frailty. It will also help public health stakeholders to decide which type of sports and exercise groups to promote in a community.

How does a motor or cognitive dual-task affect our sense of upper limb proprioception?

Daily upper limb activities require multitasking and our division of attention. How we allocate our attention can be studied using dual-task interference (DTi). Given the vital role proprioception plays in movement planning and motor control, it is important to investigate how conscious upper limb proprioception is impacted by DTi through cognitive and motor interference. To examine how dual-task interference impacts conscious upper limb proprioception during active joint repositioning tasks (AJRT). Forty-two healthy participants, aged between 18 and 35, took part in this cross-sectional study. Participants completed two AJRT during three conditions: baseline (single task), dual-cognitive task (serial subtractions), and dual-motor task (non-dominant hand movements). The proprioceptive error (PE; difference between their estimation and targeted position) was measured using an AJRT of 75% and 90% of maximum internal rotation using the Biodex System IIITM and the Upper Limb Proprioception Reaching Test (PRO-Reach). To determine if PEs differed during dual-task interference, interference change scores from baseline were used with one sample t-tests and analyses of variance. The overall mean PE with the Biodex was 4.1° ± 1.9 at baseline. Mean change scores from baseline reflect a mean improvement of 1.5° ± 1.0 (p < .001) during dual-cognitive task and of 1.5° ± 1.2 (p < .001) during dual-motor task. The overall mean PE with the PRO-Reach was 4.4cm ± 1.1 at baseline. Mean change scores from baseline reflect a mean worsening of 1.0cm ± 1.1 (p < .001) during dual-cognitive task and improvement of 0.8cm ± 0.6 (p < .001) during dual-motor task. Analysis of variance with the Biodex PEs revealed an interference effect (p < .001), with the cognitive condition causing greater PEs compared to the motor condition and a criterion position effect (p = .006), where 75% of maximum IR produced larger PEs during both interference conditions. An interference effect (p = .022) with the PRO-Reach PEs was found highlighting a difference between the cognitive and motor conditions, with decreased PEs during the contralateral motor task. Interference tasks did impact proprioception. Cognitive interference produced mixed results, whereas improved proprioception was seen during motor interference. Individual task prioritization strategies are possible, where each person may choose their own attention strategy when faced with dual-task interference.

Effect of outpatient cardiac rehabilitation on motor function and health-related quality of life in stroke survivors

BackgroundOutpatient cardiac rehabilitation (CR) is a promising tool for improving functional outcome in stroke survivors, however, evidence for improving emotional health is limited. We aimed to clarify the effects of outpatient CR following in-hospital stroke rehabilitation on health-related quality of life (HRQOL) and motor function. MethodsPatients with acute ischemic stroke or transient ischemic attack discharged directly home were recruited, and 128 patients who fulfilled criteria for insurance coverage of CR were divided into the CR (+) group (n = 46) and CR (-) group (n = 82). All patients underwent in-hospital stroke rehabilitation, and within 2 months after stroke onset, patients in the CR (+) group started a 3-month outpatient CR program of supervised sessions. Changes of motor function and HRQOL assessed by the short form-36 version 2 (SF-36) from discharge to 3 months post-discharge were compared between the two groups. ResultsTwenty-six patients in the CR (+) group completed the program and 66 patients in the CR (-) group were followed up at a 3-month examination. Least-square mean changes in 6-minute walk distance and isometric knee extension muscle strength were significantly higher in the CR (+) group than the CR (-) group (52.6 vs. 16.3 m; 10.1 vs. 3.50 kgf/kg). Improvement of HRQOL at 3 months was not observed in the CR (+) group. ConclusionsOutpatient CR following in-hospital stroke rehabilitation within 2 months after stroke onset improved exercise tolerance and functional strength but not HRQOL assessed by the SF-36 after completion of CR in the present cohort.

Use of activity trackers to improve blood pressure in young people at risk for cardiovascular disease: a pilot randomized controlled trial.

Promoting physical activity among young individuals with cardiovascular disease (CVD) risk factors such as hypertension, diabetes, or chronic kidney disease can lower systolic blood pressure (BP). We sought to determine whether a 6-month intervention using a physical activity tracker was feasible and effective, compared with usual care. Participants were recruited at a single academic medical center. Those aged 8-30years were randomized in a 2:1 ratio to either the intervention (use of a Fitbit physical activity tracker coupled with feedback regarding the participant's step count) or usual care. The primary feasibility outcomes were screening-to-enrollment ratio and 6-month retention rates; the primary clinical outcome was a change in systolic BP from 0-6months. Sixty-three participants were enrolled (57% male; mean age: 18 ± 4years). The screening-to-enrollment ratio was 1.8:1. Six-month retention was 62% in the intervention group and 86% in the control group (p = 0.08). Mean change in systolic BP in the intervention group was not significantly different from the control group at 6months (- 2.3mmHg; 95% CI - 6.5, 1.8 vs. 3.0mmHg; 95% CI - 2.5, 8.4, respectively, p = 0.12). Among children and young adults at elevated CVD risk, the use of a physical activity tracker coupled with tailored feedback regarding their step count progress was feasible but not sustained over time. Physical activity tracker use did not have a statistically significant effect on BP after 6months. Augmented strategies to mitigate risk in young patients at high risk for early-onset CVD should be explored. This trial is registered at ClinicalTrials.gov (NCT03325426).

Phase 2, Double-Blind, Placebo-controlled Trial of a c-Jun N-Terminal Kinase Inhibitor in Idiopathic Pulmonary Fibrosis.

Rationale: Idiopathic pulmonary fibrosis is a fatal and progressive disease with limited treatment options. Objectives: We sought to assess the efficacy and safety of CC-90001, an oral inhibitor of c-Jun N-terminal kinase 1, in patients with idiopathic pulmonary fibrosis. Methods: In a Phase 2, randomized (1:1:1), double-blind, placebo-controlled study (ClinicalTrials.gov ID: NCT03142191), patients received CC-90001 (200 or 400 mg) or placebo once daily for 24 weeks. Background antifibrotic treatment (pirfenidone) was allowed. The primary endpoint was change in the percentage of predicted FVC (ppFVC) from baseline to Week 24; secondary endpoints included safety. Measurements and Main Results: In total, 112 patients received at least one dose of study drug. The study was terminated early because of a strategic decision made by the sponsor. Ninety-one patients (81%) completed the study. The least-squares mean changes from baseline in ppFVC at Week 24 were -3.1% (placebo), -2.1% (200 mg), and -1.0% (400 mg); the differences compared with placebo were 1.1% (200 mg; 95% confidence interval: -2.1, 4.3; P = 0.50) and 2.2% (400 mg; 95% confidence interval: -1.1, 5.4; P = 0.19). Adverse event frequency was similar in patients in the combined CC-90001 arms versus placebo. The most common adverse events were nausea, diarrhea, and vomiting, which were more frequent in patients in CC-90001 arms versus placebo. Fewer patients in the CC-90001 arms than in the placebo arm experienced cough and dyspnea. Conclusions: Treatment with CC-90001 over 24 weeks led to numerical improvements in ppFVC in patients with idiopathic pulmonary fibrosis compared with placebo. CC-90001 was generally well tolerated, which was consistent with previous studies. Clinical trial registered with www.clinicaltrials.gov (NCT03142191).

Efficacy and safety of tibial nerve stimulation using a wearable device for overactive bladder.

To evaluate the efficacy and safety of a wearable, smartphone-controlled, rechargeable transcutaneous tibial nerve stimulation (TTNS) device in patients with overactive bladder (OAB). This multicentre, prospective, single-blind, randomised clinical trial included eligible patients with OAB symptoms who were randomly assigned to the stimulation group or sham group. The primary efficacy outcome was change from baseline in voiding frequency/24 h after 4 weeks of treatment. The secondary efficacy outcomes included changes in bladder diary outcomes (urgency score/void, nocturia episodes/day, micturition volume/void, and incontinence episodes/day), questionnaires on Overactive Bladder Symptom Score (OABSS), Patient Perception of Bladder Condition (PPBC), and American Urological Association Symptom Index Quality of Life Score (AUA-SI-QoL) at baseline and after 4 weeks of treatment. Device-related adverse events (AEs) were also evaluated. In the full analysis set (FAS), the mean (sd) change of voiding frequency/24 h in the stimulation group and sham group at 4 weeks were -3.5 (2.9) and -0.6 (2.4), respectively (P < 0.01). Similar results were obtained in the per-protocol set (PPS): -3.5 (2.9) vs -0.4 (2.3) (P < 0.01). In the FAS and PPS, micturition volume/void significantly improved at 4 weeks (P = 0.01 and P = 0.02). PPBC improvement almost reached significance in the FAS (P = 0.05), while it was significant in the PPS (P = 0.02). In the FAS and PPS, AUA-SI-QoL significantly improved at 4 weeks in the two groups (P < 0.01 and P < 0.01), whereas there were no significant differences in urgency score/void, nocturia episodes/day or OABSS between the groups. Also, no device-related serious AEs were reported. The non-invasive neuromodulation technique using the novel ambulatory TTNS device is effective and safe for treating OAB. Its convenience and easy maintenance make it a new potential home-based treatment modality. Future studies are warranted to confirm its longer-term efficacy.

A Randomized Crossover Trial of Mixed Meal Tolerance Test Response in People with Type1 Diabetes on Insulin Pump Therapy and YG1699 or Dapagliflozin.

YG1699 is a novel inhibitor of sodium-glucose cotransporter 1 (SGLT1) and SGLT2. This double-blind, 3-way crossover trial compared YG1699 to dapagliflozin as an adjunct to insulin in people with type 1 diabetes (T1D) on insulin pump therapy. Treatment periods included four mixed meal tolerance tests (MMTTs) and insulin withdrawal tests per person. Nineteen adults with T1D were randomized to YG1699 10 mg, YG1699 25 mg, and dapagliflozin 10 mg once daily for 1 week in different orders. The primary end point was the difference in area under the curve (AUC) in plasma glucose (AUC0-120min) after an MMTT between treatment groups. Mean change in plasma glucose after an MMTT (AUC0-120min) was lower for YG1699 10 mg vs. dapagliflozin (89.51% of baseline vs. 102.13%, 90% confidence interval (CI) vs. dapagliflozin, -6% to -16%, P = 0.0003) and for YG1699 25 mg (84.83% vs. 102.13%, 90% CI vs. dapagliflozin -13% to -22%, P < 0.0001). At 120 minutes, mean glucose values on no treatment, dapagliflozin, YG1699 10 mg, and YG1699 25 mg were 149 (SE 7.6), 141 (SE 6.1), 128 (SE 6.9), and 115 (SE 7.8) mg/dL, respectively. Insulin dose requirements were lower for YG1699 10 mg and 25 mg vs. dapagliflozin for bolus insulin, and for YG1699 10 mg vs. dapagliflozin for total daily insulin. Safety profiles were similar between treatment groups. YG1699 reduced post-prandial glucose more than dapagliflozin in people with T1D on insulin pump therapy. The results were consistent with dual SGLT1/SGLT2 inhibition by YG1699.

Cataract surgery in children using intracameral mydriatic.

To study the pupil dynamics with premixed intracameral anesthetic mydriatic combination of phenylephrine (0.31%), tropicamide (0.02%), and lidocaine (1%) in pediatric cataract surgery. Consecutive children aged ≤12 years planned for cataract surgery were recruited. A commercially available premixed combination of phenylephrine (0.31%), tropicamide (0.02%), and lidocaine (1%) was injected at the beginning of surgery without any topical/infusion drugs for mydriasis. Pupil sizes at various points of surgery were studied. We recruited 75 patients with a mean age of 24.3 ± 33.4 months (range: 1 month-11 years). Adequate mydriasis with a single injection was achieved in 93.5% (n = 73 eyes of 70 patients) without additional pharmacotherapy or intervention. The mean pupillary diameter increased from 1.8 ± 0.79 to 6.1 ± 1.4 mm after injection (mean change of 4.2 ± 1.25 mm from baseline). The mean variability in pupillary diameter was 0.73 ± 1.3 mm. In five eyes, good dilatation was not possible even after repeat injection. Fixed-dose premixed intracameral injection is effective in pupil dilatation. It alleviates the need for any topical dilators or additional intraoperative supplementation for pediatric cataract surgery.

Use of improved memory type control charts for monitoring cancer patients recovery time censored data

Control charts are a statistical approach for monitoring cancer data that can assist discover patterns, trends, and unusual deviations in cancer-related data across time. To detect deviations from predicted patterns, control charts are extensively used in quality control and process management. Control charts may be used to track numerous parameters in cancer data, such as incidence rates, death rates, survival time, recovery time, and other related indicators. In this study, CDEC chart is proposed to monitor the cancer patients recovery time censored data. This paper presents a composite dual exponentially weighted moving average Cumulative sum (CDEC) control chart for monitoring cancer patients recovery time censored data. This approach seeks to detect changes in the mean recovery time of cancer patients which usually follows Weibull lifetimes. The results are calculated using type I censored data under known and estimated parameter conditions. We combine the conditional expected value (CEV) and conditional median (CM) approaches, which are extensively used in statistical analysis to determine the central tendency of a dataset, to create an efficient control chart. The suggested chart's performance is assessed using the average run length (ARL), which evaluates how efficiently the chart can detect a change in the process mean. The CDEC chart is compared to existing control charts. A simulation study and a real-world data set related to cancer patients recovery time censored data is used for results illustration. The proposed CDEC control chart is developed for the data monitoring when complete information about the patients are not available. So, instead of doping the patients information we can used the proposed chart to monitor the patients information even if it is censored. The authors conclude that the suggested CDEC chart is more efficient than competitor control charts for monitoring cancer patients recovery time censored data. Overall, this study introduces an efficient new approach for cancer patients recovery time censored data, which might have significant effect on quality control and process improvement across a wide range of healthcare and medical studies.

Tolerability, safety and efficacy of a novel phosphate binder VS-505 (AP301): a Phase 2 dose-escalation and dose-ranging study in patients undergoing maintenance hemodialysis.

VS-505 (AP301), an acacia and ferric oxyhydroxide polymer, is a novel fiber-iron-based phosphate binder. This two-part Phase 2 study evaluated the tolerability, safety and efficacy of oral VS-505 administered three times daily with meals in treating hyperphosphatemia in chronic kidney disease (CKD) patients receiving maintenance hemodialysis (MHD). In Part 1, patients received dose-escalated treatment with VS-505 2.25, 4.50 and 9.00g/day for 2weeks each, guided by serum phosphorus levels. In Part 2, patients received randomized, open-label, fixed-dosage treatment with VS-505 (1.50, 2.25, 4.50 or 6.75g/day) or sevelamer carbonate 4.80g/day for 6weeks. The primary efficacy endpoint was the change in serum phosphorus. The study enrolled 158 patients (Part 1: 25; Part 2: 133), with 130 exposed to VS-505 in total. VS-505 was well tolerated. The most common adverse events were gastrointestinal disorders, mainly feces discolored (56%) and diarrhea (15%; generally during Weeks 1-2 of treatment). Most gastrointestinal disorders resolved without intervention, and none was serious. In Part 1, serum phosphorus significantly improved (mean change -2.0mg/dL; 95% confidence interval -2.7, -1.4) after VS-505 dose escalation. In Part 2, serum phosphorus significantly and dose-dependently improved in all VS-505 arms, with clinically meaningful reductions with VS-505 4.50 and 6.75g/day, and sevelamer carbonate 4.80g/day [mean change -1.6 (-2.2, -1.0), -1.8 (-2.4, -1.2) and -1.4 (-2.2, -0.5) mg/dL, respectively]. In both parts, serum phosphorus reductions occurred within 1week of VS-505 initiation, returning to baseline within 2weeks of VS-505 discontinuation. VS-505, a novel phosphate binder, was well tolerated with a manageable safety profile, and effectively and dose-dependently reduced serum phosphorus in CKD patients with hyperphosphatemia receiving MHD. NCT04551300 .