Prospective Longitudinal Study Research Articles

Actigraphy measures show sleep improvement after parathyroidectomy for primary hyperparathyroidism

ImportanceThe symptoms of primary hyperparathyroidism are often subtle, such as fatigue, mood changes, and sleep disturbances. After parathyroidectomy, patients often report improvement in sleep and mood; however, objective data supporting these improvements is lacking. ObjectiveThis prospective study uses standard measures to objectively and subjectively assess sleep in patients with primary hyperparathyroidism before and after parathyroidectomy. DesignA longitudinal prospective study was conducted over three one-week-long periods: pre-parathyroidectomy, 1-week post-parathyroidectomy, and three months post-parathyroidectomy. During each time point, patients wore an actigraphy device, recorded a sleep diary, and completed the Pittsburgh Sleep Quality Index (PSQI), Insomnia Severity Index (ISI), and Depression Anxiety Stress Scale (DASS). Statistical analysis was performed using repeated measures models to compare the average measures among the three time points and test for trends over time. SettingSingle institution, tertiary care center. ParticipantsPatients with primary hyperparathyroidism from ages 18 to 89 years old. ExposureParathyroidectomy between September 2020 and January 2024. Main outcomes and measuresActigraphy data, consensus sleep diary, Pittsburgh Sleep Quality Index (PSQI), Insomnia Severity Index (ISI), Depression Anxiety Stress Scales – 21 Items (DASS). ResultsThirty-six patients were enrolled, and 34 patients completed the study. Actigraphy data showed a significant negative trend in average sleep latency (p = 0.045) and average time in bed (p = 0.046). Sleep diary data showed additional differences in the number of awakenings (p = 0.002), wake after sleep onset (p < 0.001), sleep quality (p < 0.001), and sleep efficiency (p = 0.02) among the three time points and/or as a significant negative trend. PSQI and ISI scores were significantly different among the three time points (p = 0.002 and p < 0.001, respectively) and also declined significantly over time (p = 0.008 and p = 0.007, respectively). DASS depression, anxiety, and stress scores were significantly different among the three time points (p < 0.001, p = 0.01, and p < 0.001, respectively), and stress also declined significantly over time (p = 0.005). Conclusion and relevanceThis study represents the most extensive prospective study demonstrating objective and subjective sleep and mood improvement in patients with primary hyperparathyroidism after parathyroidectomy.

Using Odih-Score (Modified Klotz Score) for quality of defecation after anorectal malformation following posterior sagittal anorectoplasty (PSARP): Study of validation

Abstract* Objective The diagnostic scale or score is a clinical assessment tool used to determine diagnosis and evaluate further therapy results. The Klotz Score is a previous score modified to create a new instrument, the Odih-score. This new instrument is specifically designed to evaluate the quality of defecation success after operative anorectal malformation and is expected to be simpler and easier to apply in research hospitals. Its primary use is to evaluate the quality of defecation in post-PSARP anorectal malformation patients. Method The study conducted between 2016 and 2021, a longitudinal prospective study was conducted at RSUD Arifin Achmad Pekanbaru Riau Indonesia. The study aimed to validate the reliability of the Odih-Score (Modified Klotz Score) research instrument for evaluating defecation quality in post-PSARP anorectal malformation patients. Pearson validation test and Cronbach alpha reliability test were used as parameters for the validation, while the sensitivity and specificity of diagnostic tests were evaluated using research instrument (IBM SPSS Statistics version 25) The research was approved by the Ethical Clearance Board of the Faculty of Medicine, University of Riau, under reference number No.031/UN.19.5.1.1.8/UEPKK/2019. Results The analysis of the validation test results indicates that the Odih-Score is statistically significant (Sig.&lt;0.05) and has a greater r-count value (0.573-0.790) than the r-table value (0.202). The research reliability test also shows a high level of reliability (Cronbach alpha 0.814) for all five items of the Odih-Score instrument. Further, the sensitivity and specificity test as a diagnostic test resulted in an AUC value of 0.798, with an Odih-Score cut-off point of 11. Conclusion Odih-Score (Modified Klotz’s score) is considered valid and reliable and can be used as a diagnostic test instrument to evaluate the quality of post-anorectal Malformation defecation with PSARP.

Comparison of early patient-reported outcomes between uniportal thoracoscopic segmentectomy and wedge resection for peripheral small-sized non-small-cell lung cancer

BackgroundAnalysis of patient-reported outcomes (PROs) offers valuable insights into distinguishing the effects of closely related medical procedures from the patient’s perspective. In this study we compared symptom burden in patients undergoing uniportal thoracoscopic segmentectomy and wedge resection for peripheral small-sized non-small cell lung cancer (NSCLC).MethodsThis study included patients with peripheral NSCLC from an ongoing longitudinal prospective cohort study (CN-PRO-Lung 3) who underwent segmentectomy or wedge resection with tumor diameter ≤ 2 cm and consolidation tumor ratio (CTR) ≤ 0.5. PROs data were collected using the Perioperative Symptom Assessment for Lung Surgery questionnaire pre-operatively, daily post-surgery up to the fourth hospitalization day, and weekly post-discharge up to the fourth week. Propensity score matching and a generalized estimation equation model were employed to compare symptom severity. In addition, short-term clinical outcomes were compared.ResultsIn total, data of 286 patients (82.4%) undergoing segmentectomy and 61 patients (17.6%) undergoing wedge resection were extracted from the cohort. No statistically significant differences were found in the proportion of moderate-to-severe symptoms and mean scores for pain, cough, shortness of breath, disturbed sleep, fatigue, drowsiness, and distress during the 4-day postoperative hospitalization or the 4-week post-discharge period before or after matching (all p > 0.05). Compared with segmentectomy, wedge resection showed better short-term clinical outcomes, including shorter operative time (p = 0.001), less intraoperative bleeding (p = 0.046), and lower total hospital costs (p = 0.002).ConclusionsThe study findings indicate that uniportal thoracoscopic segmentectomy and wedge resection exert similar early postoperative symptom burden in patients with peripheral NSCLC (tumor diameter ≤ 2 cm and CTR ≤ 0.5).Clinical trial registrationNot applicable.

Children’s orthopaedics

The April 2024 Children’s orthopaedics Roundup360 looks at: Ultrasonography or radiography for suspected paediatric distal forearm fractures?; Implant density in scoliosis: an important variable?; Gait after paediatric femoral shaft fracture treated with intramedullary nail fixation: a longitudinal prospective study; The opioid dilemma: navigating pain management for children’s bone fractures; 12- to 20-year follow-up of Dega acetabuloplasty in patients with developmental dysplasia of the hip; Physeal fractures of the distal ulna: incidence and risk factors for premature growth arrest; Analysis of growth after transphyseal anterior cruciate ligament reconstruction in children; Management of lateral condyle humeral fracture associated with elbow dislocation in children: a retrospective international multicentre cohort study.

Study protocol for a prospective cohort study describing the injury characteristics in elite gymnasts in TeamGym: the Swedish TeamGym Injury Cohort – STIC

IntroductionGymnastics consists of several different disciplines, whereof TeamGym is one. TeamGym is a young discipline with sparse research. The aim of the study is to investigate the injury characteristics in...

Effect of a person-centred goals-of-care form and clinical communication training on shared decision-making and outcomes in an acute hospital: a prospective longitudinal interventional study.

Patients with a life-limiting illness (LLI) requiring hospitalisation have a high likelihood of deterioration and 12-month mortality. To avoid non-aligned care, we need to understand our patients' goals and values. To describe the association between the implementation of a shared decision-making (SDM) programme and documentation of goals of care (GoC) for hospitalised patients with LLI. A prospective longitudinal interventional study of patients admitted to acute general medicine wards in an Australian tertiary hospital over 5 years was conducted. A SDM programme with a new GoC form, communication training and clinical support was implemented. The primary outcome was the proportion of patients with a documented person-centred GoC discussion (PCD). Clinical outcomes included hospital utilisation and 90-day mortality. 1343 patients were included. The proportion of patients with PCDs increased from 0% to 35.4% (adjusted odds ratio (aOR), 2.38; 95% confidence interval (CI), 2.01-2.82; P < 0.001). During this time, median hospital length of stay decreased from 8 days (interquartile range (IQR), 4-14) to 6 days (IQR, 3-11) (adjusted estimate effect, -0.38; 95% CI, -0.64 to -0.11; P = 0.005) and rapid response team activation from 28% to 13% (aOR, 0.87; 95% CI, 0.78-0.97; P value = 0.01). Documented treatment preference of high-dependency unit care decreased from 39.7% to 24.4% (aOR, 0.81; 95% CI, 0.73-0.89; P value < 0.001), and ward-based care increased from 31.9% to 55.1% (aOR, 1.24; 95% CI, 1.14-1.36; P value < 0.001). The implementation of a SDM programme was associated with increased documentation of person-centred GoC, changed patient treatment preference to lower intensity care and reduced hospital utilisation.

Evaluation of the thyroid gland vascularity during pregnancy using 2-dimensional color Superb Microvascular İmaging vascularization index technique

PurposeWe aimed to investigate maternal thyroid parenchymal vascularity with 2-dimensional color superb microvascular imaging vascularization index (2D-cSMIVI) levels and thyroid gland volume in the first, second and third trimesters. MethodsThis longitudinal prospective study was carried out with participants selected from 30 healthy asymptomatic pregnant women. Ultrasonography (US) for the thyroid gland was performed in each trimester. The vascularization index (VI) values obtained by manually drawing the contours of the thyroid parenchyma in the longutidinal plane, using the free region of interest (ROI) with 2DcSMIVI mode. VI values obtained in each trimester, thyroid hormone levels (TSH, T3, T4) and thyroid volumes were compared. ResultsWe have detected that thyroid gland vascularity increased significantly as pregnancy progresses. The mean VI values of thyroid gland in third trimester were significantly higher than first and second trimester (p < 0.001), and the mean VI values of the thyroid gland in the second trimester were significantly higher than first trimester (p < 0.001). During pregnancy, we detected the increase in VI values, TSH levels and thyroid gland volumes. ConclusionMaternal thyroid gland gray scale findings, parenchymal vascularization, thyroid volumes should be evaluated routinely for the healthy fetal development. 2D-cSMIVI method allows us to evaluate vascularization with quantitative numerical values objectively. We have detected that the VI values and volume of the thyroid gland increases as pregnancy progresses. In the complex situation of pregnancy process, the thyroid gland can be evaluated quantitatively with SMI method effectively.

Elevated Risk of Dementia Diagnosis in Older Adults with Low Frequencies and Durations of Social Conversation.

Social networks and social participation have protective effects on cognitive function maintenance and Alzheimer's disease and general dementia development. We aimed to investigate the association between conversations and dementia incidence in older adults. This longitudinal prospective cohort study used population data from the National Center for Geriatric and Gerontology-Study of Geriatric Syndromes (NCGG-SGS) from September 2015 to February 2017. The database included 4,167 individuals in Japan aged ≥60 years who were generally healthy and without major cognitive impairment. Participants were classified into two groups according to six daily conversation measures at baseline. The conversation index was calculated as a composite score for these measures. Participants were tracked monthly over 60 months for new-onset dementia. Data from 2,531 participants were analyzed (72.7±6.7 years; range: 60-96 years). Dementia incidence per 1,000 person-years was 15.7 (95% confidence interval, 13.6-18.1). The Youden index determined the cut-off point for dementia incidence, with a conversation index of 16/17 points. The low conversation group included more participants with new-onset dementia. Cox proportional hazards regression crude models showed remarkable relationships between dementia onset and specific conversation measurements, including conversation index. According to the Cox regression adjusted model, the cut-off point of the conversation index showed only a remarkable relationship with dementia onset. Dementia risk was extensively associated with low daily conversation statuses. The assessment of conversational factors may be useful as a risk indicator for the development of Alzheimer's disease and general dementia.

Immune profiles of pre-frail people living with HIV-1: a prospective longitudinal study

BackgroundPeople living with HIV (PLWH) are at risk of frailty, which is predictive for death. As an overactivity of the immune system is thought to fuel frailty, we characterized the immune activation profiles linked to frailty.MethodsWe quantified twenty-seven activation markers in forty-six virological responders (four females and forty-two males; median age, 74 years; median duration of infection, 24 years; median duration of undetectability, 13 years), whose frailty was determined according to the Fried criteria. T cell and NK cell activation was evaluated by flow cytometry, using a panel of cell surface markers. Soluble markers of inflammation, and monocyte activation and endothelial activation were measured by ELISA. The participants’ immune activation was profiled by an unsupervised double hierarchical clustering analysis. We used ANOVA p-values to rank immunomarkers most related to Fried score. A Linear Discriminant Analysis (LDA) was performed to link immune activation markers to frailty.Results41% of the participants were pre-frail, including 24% with a Fried score of 1, and 17% with a Fried score of 2. ANOVA identified the 14 markers of T cell, monocyte, NK cell, endothelial activation, and inflammation the most linked to Fried 3 classes. The LDA performed with these 14 markers was capable of discriminating volunteers according to their Fried score. Two out of the 5 immune activation profiles revealed by the hierarchical clustering were linked to and predictive of pre-frailty. These two profiles were characterized by a low percentage of CD4 T cells and a high percentage of CD8 T cells, activated CD4 T cells, CD8 T cells, and NK cells, and inflammation.ConclusionsWe identified a particular immune activation profile associated with pre-frailty in PLWH. Profiling participants at risk of developing frailty might help to tailor the screening and prevention of medical complications fueled by loss of robustness. Further studies will indicate whether this frailty signature is specific or not of HIV infection, and whether it also precedes frailty in the general population.

Impact effects of COVID-19 pandemic on chronic disease patients: A longitudinal prospective study.

To assess the effects of COVID-19 pandemic on clinical variables as part of the routine clinical monitoring of patients with chronic diseases in primary care. A prospective longitudinal study was conducted in primary care centres of the Andalusian Health Service. Data were recorded before the pandemic (T1), during the declaration of the state of emergency (T2) and in the transition phase (T3). The Barthel index and the Short Portable Mental Status Questionnaire (SPMSQ) were used to analyse functional and cognitive changes at the three time points. HbA1c, systolic and diastolic blood pressure, heart rate, BMI and lipid levels were assessed as clinical variables. Descriptive statistics and non-parametric chi-square test were used for analysis. STROBE checklist was used for the preparation of this paper. A total fo148 patients with chronic conditions were included in the analysis. Data analysis revealed in T2 only significant reductions in BMI, total levels of cholesterol and HDL during the onset of the pandemic. Barthel Index, SPMSQ, blood pressure and triglycerides and LDL levels worsened in T2, and the negative effects were maintained in T3. Compared to pre-pandemic values, HbA1c levels improved in T3, but HDL levels worsened. COVID-19 has drastically disrupted several functional, cognitive and biological variables. These results may be useful in identifying clinical parameters that deserve closer attention in the case of a new health crisis. Further studies are needed to assess the potential impacts of each specific chronic condition. Cognitive and functional status, blood pressure and triglycerides and LDL levels worsen in short term, maintaining the negative effects in medium-term.

B-flow/spatiotemporal image correlation M-mode ultrasound provides novel method to quantify spiral artery remodeling during normal human pregnancy.

During human pregnancy placental extravillous trophoblasts replace the vascular smooth muscle and elastic tissue within the walls of the uterine spiral arteries, thereby remodeling these arteries into distensible low resistance vessels to promote placental perfusion. The present study, determined whether B-flow/ spatio-temporal image correlation (STIC) M-mode ultrasonography provides an in vivo imaging method to digitally quantify spiral artery luminal distensibility, as a physiological index of spiral artery remodeling, during advancing stages of normal human pregnancy. A prospective longitudinal observational study was conducted to quantify spiral artery distensibility, i.e. vessel luminal diameter at systole minus diameter at diastole, by B-flow/STIC M-mode ultrasonography during the first, second and third trimesters in 290 women exhibiting normal pregnancy. Maternal serum levels of placental growth factor (PlGF) and soluble fms-like tyrosine kinase (sFlt-1), growth factors that modulate events important in spiral artery remodeling, were quantified in a subset of the subjects at the first, second and third semesters. Median [first quartile, third quartile] spiral artery distensibility progressively increased (P < 0.0001) between the first trimester (0.17 [0.14, 0.21]), second (0.23 [0.18, 0.28]) and third (0.26 [0.21, 0.35]) trimesters of pregnancy. Spiral artery volume flow (ml/cardiac cycle) progressively increased (P < 0.001) between the first 2.49 [1.38, 4.99], second 3.86 [2.06, 6.91] and third 7.79 [3.83, 14.98] trimesters. Coinciding with the elevation in spiral artery distensibility, the median ratio of serum PlGF/sFlt-1 levels increased (P < 0.001) between the first (7.2 [4.5, 10], second (22.7 [18.6, 42.2]) and third (56.2 [41.9, 92.5] trimesters. The present study shows that B-flow/STIC M-mode ultrasonography provides an in vivo imaging technology to digitally quantify structural/physiological expansion of the walls of the spiral arteries during the cardiac cycle as a consequence of their transformation into compliant vessels during advancing stages of normal human pregnancy. This article is protected by copyright. All rights reserved.

Prevalence and trajectories of neuropsychological post-COVID-19 symptoms in initially hospitalized patients.

To investigate the prevalence and trajectories of post-COVID-19 neuropsychological symptoms. Prospective longitudinal multicentre cohort study. A total of 205 patients initially hospitalized with SARS-CoV-2 (COVID-19). Validated questionnaires were administered at 9 months (T1) and 15 months (T2) post-hospital discharge to assess fatigue, cognitive complaints, insomnia, anxiety, depression, and post-traumatic stress symptoms. Analyses included 184 out of 205 patients. Approximately 50% experienced high cognitive complaints at T1 and T2, while severe fatigue affected 52.5% at T1 and 55.6% at T2. Clinically relevant insomnia scores were observed in 25% of patients at both time-points. Clinically relevant anxiety scores were present in 18.3% at T1 and 16.7% at T2, depression in 15.0% at T1 and 18.9% at T2, and PTSD in 12.4% at T1 and 11.8% at T2. Most symptoms remained stable, with 59.2% of patients experiencing at least 1 persistent symptom. In addition, 31.5% of patients developed delayed-onset symptoms. Post-COVID-19 cognitive complaints and fatigue are highly prevalent and often persist. A subgroup develops delayed symptoms. Emotional distress is limited. Screening can help identify most patients experiencing long-term problems. Future research should determine risk factors for persistent and delayed onset symptoms.

Fishing for the evidence on community water fluoridation: a commentary on the CATFISH study.

Goodwin M, Emsley R, Kelly M P et al. Evaluation of water fluoridation scheme in Cumbria: the CATFISH prospective longitudinal cohort study. Southampton: National Institute for Health and Care Research, 2022. This longitudinal, prospective cohort study, recruited two populations - a birth cohort and older school cohort - assessing the effects of community water fluoridation (CWF) over a five-year period. The control group received non-fluoridated drinking water in the East of Cumbria, while the intervention group received fluoridated drinking water in the West of Cumbria. The primary outcome measures were the presence or absence of decay into dentine in primary and permanent teeth. Cost effectiveness was assessed by quality-adjusted life-years. Birth Cohort: Modest beneficial effect (17.4% with decay, 21.4% in control). Adjusted Odd Ratios (AOR): 0.74 (95% CI: 0.56 to 0.98). Older Cohort: Insufficient evidence of difference (19.1% with decay and 21.9% in control). AOR: 0.80 (95% CI: 0.58 to 1.09). The authors concluded that CWF was effective in the birth cohort and that the modest absolute reduction in caries should be considered in the context of other preventive measures. A longer follow-up period was recommended to fully understand the benefits and risks of CWF in contemporary low-prevalence populations.

Disposable E-Cigarette Use and Subsequent Use Patterns in Adolescents and Young Adults.

Disposable electronic cigarettes (e-cigarettes) are widely used by adolescents and young adults (AYAs). Whether using disposable devices is associated with future e-cigarette use patterns is unknown but important for informing e-cigarette regulation. Prospective longitudinal study combining data from adolescent (14-17 years) and young adult (21-24 years) cohorts from Southern California surveyed at baseline and approximately 8-month follow-up during 2021 to 2022. The analyses included AYAs who used e-cigarettes in the past 30 days at baseline and had exposure and outcome data (N = 403; adolescent n = 124, young adult n = 279). In the pooled sample of AYAs who used e-cigarettes at baseline (57.2% cis-gender female, 56.2% Hispanic), 278 (69.0%) reported past 30-day disposable e-cigarette use, and 125 (31.0%) used only nondisposable e-cigarettes. Baseline use of disposable (versus only nondisposable) devices was associated with higher odds of continued e-cigarette use (adjusted odds ratio = 1.92; 95% confidence interval = 1.09-3.42) and a greater number of times used e-cigarettes per day at follow-up (adjusted incidence rate ratio = 1.29; 95% confidence interval = 1.02-1.63). In supplemental analyses, disposable e-cigarette use was associated with greater odds of no changes (versus reductions) in e-cigarette use frequency and puffs per episode from baseline to follow-up but was not associated with increases in use frequency and intensity. No differences in e-cigarette use outcomes were found between those with poly-device (disposable and nondisposable) versus only disposable device use. Use of disposable e-cigarette devices among AYAs may be associated with higher risks for persistent e-cigarette use patterns, which should be considered in tobacco product regulation designed to protect AYAs.

Olfactory threshold as a biomarker of long-term relapse activity in multiple sclerosis

BackgroundOlfactory threshold (OT) is a marker of short-term inflammatory activity in multiple sclerosis (MS). ObjectiveTo investigate whether OT predicts long-term MS clinical disease course. MethodsThis was a 6-year prospective longitudinal study on MS patients at the MS clinic Innsbruck. Clinical visits assessing the occurrence of relapses, Expanded Disability Status Scale (EDSS) scores, and disease-modifying treatment (DMT), were conducted biannually. OT testing was performed at baseline (BL), year 1 (Y1), year 2 (Y2) and year 6 (Y6), using the threshold subscore of the “Sniffin’ Sticks” test. Cognitive function was assessed by the Symbol Digit Modalities Test. ResultsOf 139 MS patients, 92 were eligible for Y6 follow-up. 68% experienced relapses, 53% EDSS worsening, 29% progression independent of relapse activity (PIRA) and 41% cognitive deterioration. OT scores were lower at BL, Y1 and Y2 in patients requiring DMT escalation. In multivariable analysis, higher OT scores at BL, Y1, Y2 and Y6 were associated with lower risk of relapse (hazard ratio, HR: 0.65–0.92) and EDSS worsening (HR: 0.86–0.89), while no associations were found for PIRA and cognitive deterioration. ConclusionsOT is a potential surrogate marker for long-term inflammatory disease activity and DMT failure in MS.

Feasibility Value of Right Ventricular Longitudinal Shortening Fraction and the Prognostic Implications in Patients With Heart Transplantation.

Right ventricular longitudinal shortening fraction (RVLSF) is a 2-dimensional speckle tracking echocardiography parameter based on tricuspid annular displacement analysis that could be used to assess right ventricular (RV) systolic function. The value of RVLSF in the assessment of RV systolic function in recipients of heart transplantation (HT) and whether RVLSF can replace strain parameters remains unknown. A total of 153 adult patients who underwent HT were consecutively enrolled in this prospective longitudinal study. All subjects were examined by conventional transthoracic 2-dimensional echocardiography and 2-dimensional speckle tracking echocardiography to evaluate the RV end-diastolic basal diameter, RV end-diastolic area, fractional area change, peak systolic velocity of tricuspid annulus, tricuspid annular plane systolic excursion, RV free wall strain, and RVLSF. Cox proportional hazards regression was used to test if the parameters of interest had independent prognostic value for adverse outcome prediction in patients who underwent HT. A significant positive correlation was found between the measurements of RVLSF and RV free wall strain (r=0.927, P<0.001). Compared with the event-free group, the adverse outcome group displayed reduced RVLSF and RV free wall strain and higher age (P<0.001, <0.001, =0.016, respectively) in patients who underwent HT. RVLSF and RV free wall strain were independently associated with poor prognosis in multivariable analysis (both P<0.001). RVLSF assessment provides an effective evaluation of RV longitudinal systolic function in the transplanted hearts and has prognostic value for adverse outcomes in patients undergoing HT.

Treatment of Symptomatic Spinal Muscular Atrophy with Nusinersen: A Prospective Longitudinal Study on Scoliosis Progression.

Nusinersen treatment has demonstrated efficacy in improving clinical outcomes for spinal muscular atrophy (SMA), yet its impact on scoliosis progression remains unclear. This study aimed to assess the progression of scoliosis in pediatric patients with SMA undergoing nusinersen treatment. In this prospective study, data were systematically collected from Hong Kong pediatric SMA patients receiving nusinersen between 2018 and 2023. All patients had longitudinal radiographic studies pre-nusinersen, and at half-yearly or yearly intervals during treatment based on the scoliosis severity. Motor function evaluations were conducted pre-nusinersen, and after starting treatment at 6- and 12-month intervals. Twenty-three patients ((SMA type 1 (SMA1) = 8, SMA type 2 (SMA2) = 7, SMA type 3 (SMA3) = 8)) with a median age of 5.8 years (range: 0.4-17.5 years) at nusinersen initiation, and median follow-up duration of 3.4 years (range: 1.1-5.2 years) were included. During the study period, motor scores remained stable or improved in 83% of patients. However, scoliosis progressed across all subtypes, with mean annual progression rates of 5.2, 11.9, and 3.6 degrees in SMA1, SMA2, and SMA3 respectively. Patients initiating nusinersen between ages 5 and 11 years exhibited the most rapid progression, with rates of 11.8, 16.5, and 7.3 degrees per year in SMA1, SMA2, and SMA3 respectively. Positive correlations were observed between the difference in CHOP-INTEND score post-nusinersen and scoliosis progression in SMA1 (rs = 0.741, p = 0.041). Conversely, negative correlations were found between the difference in HFMSE score post-nusinersen and scoliosis progression in SMA2 (rs =-0.890, p = 0.012) and SMA3 (rs =-0.777, p = 0.028). This study reveals that nusinersen treatment in symptomatic pediatric SMA patients with motor improvement is linked to increased scoliosis progression in SMA1, whereas it is associated with decreased progression in SMA2 and SMA3. Age, baseline Cobb angle, and motor milestone improvement are influential factors in scoliosis progression.

Fetal Left Ventricle Function Evaluated by Two-Dimensional Speckle-Tracking Echocardiography across Clinical Stages of Severity in Growth-Restricted Fetuses.

Fetal growth restriction (FGR) can result in adverse perinatal outcomes due to cardiac dysfunction. This study used 2D speckle-tracking echocardiography to assess left ventricle (LV) longitudinal strain across FGR severity stages. A prospective longitudinal cohort study measured global (GLS) and segmental LV longitudinal strain in FGR fetuses, with evaluations conducted at various time points. FGR was classified into subtypes based on published criteria using fetal weight centile and Doppler parameters. A linear mixed model was employed to analyze repeated measures and compare Z-score measurements between groups throughout gestational age. The study included 40 FGR fetuses and a total of 107 evaluations were performed: 21 from small for gestational age (SGA), 74 from the FGR stage I, and 12 from the FGR stage ≥ II. The results indicate that SGA and stage I FGR fetuses exhibit higher LV GLS than stages ≥ II. Throughout gestation, SGA and FGR stage I fetuses showed similar behavior with consistently better LV GLS values when compared to FGR stages ≥ II. No significant differences were observed in LV GLS strain behavior between SGA and FGR stage I. In conclusion, all FGRs show signs of early cardiac dysfunction, with severe cases demonstrating significantly a lower LV GLS when compared to mild cases, suggesting deterioration of cardiac dysfunction with progression of fetal compromise.

Opportunistic screening for COPD among socially marginalized patients

BackgroundChronic obstructive pulmonary disease (COPD) is a common disease associated with premature death. Tobacco exposure is the main risk factor, but lower socioeconomic status, early life insults, and occupational exposures are also important risk factors. Socially marginalized people, facing homelessness, substance use disorder, and mental illness, are likely to have a higher risk of developing COPD, and, furthermore, experience barriers to healthcare access and consequently poorer outcomes.ObjectiveThis study aims to assess COPD prevalence and the impact of opportunistic screening among hospitalized patients who are in contact with hospital social nurses. These patients constitute a group of patients with a high prevalence of psychiatric and somatic diseases, substance use, low life expectancy, and are socially marginalized.MethodsThe present prospective longitudinal study includes a clinical examination at baseline. Participants will have spirometry done and be interviewed regarding risk factors, socioeconomic conditions, and respiratory symptoms. The 5-year follow-up assessment incorporates data from baseline and register data over the 5 years, including information on morbidity, use of COPD medication, hospital contacts, mortality, and socioeconomic factors.Anticipated resultsReferral for further diagnostic work-up and management after the screening, including COPD treatment and smoking cessation support, is expected to improve survival rates. The study is still enrolling patients.Trial registrationThe study is registered at ClinicalTrials.gov , NCT04754308 with study status: “enrolling”.

Accuracy of the tuberculosis molecular bacterial load assay to diagnose and monitor response to anti-tuberculosis therapy: a longitudinal comparative study with standard-of-care smear microscopy, Xpert MTB/RIF Ultra, and culture in Uganda

In 2018, the tuberculosis molecular bacterial load assay (TB-MBLA), a ribosomal RNA-based test, was acknowledged by WHO as a molecular assay that could replace smear microscopy and culture for monitoring tuberculosis treatment response. In this study, we evaluated the accuracy of TB-MBLA for diagnosis and monitoring of treatment response in comparison with standard-of-care tests. For this longitudinal prospective study, patients aged 18 years or older with presumptive tuberculosis (coughing for at least 2 weeks, night sweats, and weight loss) were enrolled at China-Uganda Friendship Hospital Naguru (Kampala, Uganda). Participants were evaluated for tuberculosis by TB-MBLA in comparison with Xpert MTB/RIF Ultra (Xpert-Ultra) and smear microscopy, with Mycobacteria Growth Indicator Tube (MGIT) culture as a reference test. Participants who were positive on Xpert-Ultra were enrolled on a standard 6-month anti-tuberculosis regimen, and monitored for treatment response at weeks 2, 8, 17, and 26 after initiation of treatment and then 3 months after treatment. Between Nov 15, 2019, and June 15, 2022, 210 participants (median age 35 years [IQR 27-44]) were enrolled. 135 (64%) participants were male and 72 (34%) were HIV positive. The pretreatment diagnostic sensitivities of TB-MBLA and Xpert-Ultra were similar (both 99% [95% CI 95-100]) but the specificity was higher for TB-MBLA (90%[83-96]) than for Xpert-Ultra (78% [68-86]). Ten participants were Xpert-Ultra trace positive, eight (80%) of whom were negative by TB-MBLA and MGIT culture. Smear microscopy had lower diagnostic sensitivity (75%[65-83]) but higher specificity (98% [93-100]) than TB-MBLA and Xpert-Ultra. Among participants who were smear microscopy negative, the sensitivity of TB-MBLA was 96% (95 CI 80-100) and was 100% (95% CI 86-100) in those who were HIV positive. 129 (61%) participants were identified as tuberculosis positive by Xpert-Ultra and these individuals were enrolled in the treatment group and monitored for treatment response. According to TB-MBLA, 19 of these patients cleared bacillary load to zero by week 2 of treatment and remained negative throughout the 6-month treatment follow-up. Positivity for tuberculosis decreased with treatment as measured by all tests, but therate was slower with Xpert-Ultra. Consequently, 31 (33%) of 95 participants were still Xpert-Ultra positive at the end of treatment but were clinically well and negative on TB-MBLA and culture at 6 months of treatment. Two patients were still Xpert-Ultra positive with a further 3 months of post-treatment follow-up. The rate of conversion to negative of the DNA-based Xpert-Ultra was 3·3-times slower than that of the rRNA-based TB-MBLA. Consequently for the same patient, it would take 13 weeks and 52 weeks to reach complete tuberculosis negativity by TB-MBLA and Xpert-Ultra, respectively. Participants who were positive on smear microscopy at 8weeks, who received an extra month of intensive treatment, had a similar TB-MBLA-measured bacillary load at 8weeks to those who were smear microscopy negative. TB-MBLA has a similar performance to Xpert-Ultra for pretreatment diagnosis of tuberculosis, but is more accurate at detecting and characterising the response to treatment than Xpert-Ultra and standard-of-care smear microscopy. European and Developing Countries Clinical Trials Partnership, Makerere University Research and Innovation Fund, US National Institutes of Health.