Longitudinal change in symptom severity in children with ASD: Results from the ELENA cohort.

Autism spectrum disorder (ASD) is a lifelong neurodevelopmental condition and understanding the changes in autism symptoms over time is crucial for tailoring support and interventions. This study therefore aimed to investigate the changes in symptom severity in a large cohort of children with ASD over a three-year follow-up period and identify factors that influence these changes. The study included 575 children diagnosed with ASD, ranging in age from 2 to 12 years, who were assessed at baseline and again 3 years later using the Autism Diagnostic Observational Schedule-2 (ADOS-2). ASD severity changes were investigated using the ADOS calibrated severity score (CSS) scores for total, social affect (SA) and restricted and repetitive behaviors (RRB). Results highlight four distinct patterns: stable high, stable low, increased, and decreased severity. The ADOS CSS total score changed for half of the sample, reflecting an increase in ASD severity for 21.9% and a decrease for 29.1% of children. For the other half, the ADOS CSS score remained stable, either high (34.4%) or low (14.6%). While the majority of previous studies reported stability in ASD severity, our findings revealed significant variability with frequent improvements in SA symptoms whereas RRBs remained stable or worsened. Our findings also showed that an improvement in SA was associated with the youngest group and early diagnosis. However, no clinical or sociodemographic factors were linked to changes in RRB, emphasizing the necessity for RRB-specific therapies. The third six-year follow-up point of the ongoing ELENA cohort study will map the long-term trajectories of the severity of ASD symptoms and their potential risk factors.

Allogenic bone marrow-derived mesenchymal stromal cell-based therapy for patients with chronic low back pain: a prospective, multicentre, randomised placebo controlled trial (RESPINE study).

OBJECTIVES: To assess the efficacy of a single intradiscal injection of allogeneic bone marrow mesenchymal stromal cells (BM-MSCs) versus a sham placebo in patients with chronic low back pain (LBP). METHODS: Participants were randomised in a prospective, double-blind, controlled study to receive either sham injection or intradiscal injection of 20 million allogeneic BM-MSC, between April 2018 and December 2022. The first co-primary endpoint was the rate of responders defined by improvement of the Visual Analogue Scale (VAS) for pain of at least 20% and 20 mm, or improvement of the Oswestry Disability Index (ODI) of 20% between baseline and month 12. The secondary structural co-primary endpoint was assessed by the disc fluid content measured by quantitative MRI T2, between baseline and month 12. Secondary endpoints included pain VAS, ODI, the Short Form (SF)-36 and the minimal clinically important difference in all timepoints (1, 3, 6, 12 and 24 months). We determined the immune response associated with allogeneic cell injection between baseline and 6 months. Serious adverse events (SAEs) were recorded. RESULTS: 114 patients were randomised (n=58, BM-MSC group; n=56, sham placebo group). At 12 months, the primary outcome was not reached (74% in the BM-MSC group vs 69% in the placebo group; p=0.77). The groups did not differ in all secondary outcomes. No SAE related to the intervention occurred. CONCLUSIONS: While our study did not conclusively demonstrate the efficacy of allogeneic BM-MSCs for LBP, the procedure was safe. Long-term outcomes of MSC therapy for LBP are still being studied. TRIAL REGISTRATION NUMBER: EudraCT 2017-002092-25/ClinicalTrials.gov: NCT03737461.

Validation of the French version of the preschool form of the social responsiveness scale-second edition (SRS-2-P).

OBJECTIVES: To evaluate the psychometric properties of the French form of the preschool social responsiveness scale-2 (SRS-2-P-Fr). PARTICIPANTS: A sample of French children aged 2 to 4 years comprising a group of 93 children with autism spectrum disorder (ASD) and a community control group of 201 children. MATERIALS AND METHODS: Study of internal consistency using Cronbach's α coefficient, convergent validity using correlations with the ADI-R and ADOS-2, and diagnostic accuracy using the receiver operating characteristic curve to determine the best threshold scores useful for screening ASD in preschool children. RESULTS: Good internal consistency (0.78 to 0.98) according to Cronbach's alpha coefficients for the total score and the sub-scores of the SRS-2-P scale. The study of convergent validity showed strong correlations with the total score of the ADOS-2 Toddlers and the communication and reciprocal social interaction sub-scores of the ADI-R. Diagnostic accuracy indicated that the best cut-off score for detecting ASDs was 56 (area under the curve=0.97, sensitivity 0.925, specificity 0.98). CONCLUSION: The SRS-2-P-Fr is a valid instrument for the early detection of ASD in preschool children in France and can facilitate diagnosis.

Contributions of the ELENA Cohort to Study Autism Spectrum Disorder in Children and Adolescents from a Biopsychosocial Framework.

Autism spectrum disorder (ASD) is a complex neurodevelopmental condition influenced by a myriad of developmental, biological, psychological, and socio-demographic factors. The ELENA cohort seeks to delineate the intricate interplay of these factors, facilitating the identification of risk factors and the development of targeted interventions. This paper emphasizes the clinical profiles of children and outlines key findings from a biopsychosocial perspective. The ELENA cohort, a multicenter initiative across French regional centers, conducted a systematic prospective analysis on children newly diagnosed with DSM-5 ASD between 2012 and 2019. This encompassed direct assessments and parent-reported questionnaires covering a broad spectrum of developmental, biological, psychological and socio-demographic measures. Embedded case-control studies further examined risk and protective factors, alongside specific environmental and psychosocial influences during pregnancy and early childhood. A subset of participants also contributed biospecimens, with data enhancement via linkage to French National Administrative Healthcare Databases. The study unveils baseline clinical characteristics for 876 children, average age 6 (SD ± 3.3) previously unreported in protocol descriptions. It highlights the study's developmental biopsychosocial approach and its novel findings on children's socio-adaptive functioning, ASD severity, comorbidities, quality of life and interventions. Employing developmental biopsychosocial insights offers a promising pathway to integrating health, social care, and experiential insights, ultimately aiming to enhance the future well-being and outcomes for children with ASD. This approach underscores the need of a holistic, interdisciplinary strategy in encouraging and supporting the ASD community.

Attainment of LDL-cholesterol target in high cardiovascular risk type 1 diabetic French people.

AIMS: As people with type 1 diabetes have increased risk of cardiovascular morbi-mortality, management of cardiovascular risk factors is of crucial importance. We assessed the prevalence and factors associated with LDL-cholesterol (LDL-c) target achievement in patients with type 1 diabetes at high and very-high cardiovascular risk. METHODS: In this observational multicenter study, we included hospitalized patients with type 1 diabetes who had a fasting blood lipid analysis at admission. Cardiovascular risk level and LDL-c target values were defined according to ESC/EAS guidelines into force at admission: LDL-c target for very-high risk (VHR) and high risk (HR) patients was 1.4 and 1.8 mmol/l respectively for patients included from September 2019 (2019 guidelines) and 1.8 and 2.6 mmol/l respectively for patients included in 2016-2019 (2016 guidelines). LDL-c target attainment was assessed in HR and VHR patients, and factors associated with attainment were identified with multivariable analysis. RESULTS: We included 85 HR patients (median age 37y [interquartile range: 27;45], 64 % females) and 356 VHR patients (49 [35;61] years, 42 % females). In HR patients, 7 % were treated with statins, and 35.3 % achieved the LDL-c target. Increasing age (odds ratio 0.58 [95 % confidence interval: 0.38;0.89]), body mass index (0.86 [0.75;0.98]), and HbA1c (0.69 [0.50;0.94]) were independently associated with lower odds of attaining LDL-c target. In VHR patients, 36 % were treated with statins, and 17.4 % achieved LDL-c target. Statin treatment (2.33 [1.22;4.43]), secondary prevention (2.33 [1.21;4.48]) and chronic renal failure (2.82 [1.42;5.61]) were associated with higher odds of attaining LDL-c target. CONCLUSION: Control of LDL-c is highly insufficient in both HR and VHR patients. Cardiovascular risk evaluation and better control of risk factors may help decrease cardiovascular morbi-mortality in patients with type 1 diabetes. REGISTRATION NUMBER: NCT03449784.

Impact of a bundle of care (intravenous iron, erythropoietin and transfusion metabolic adjustment) on post-operative transfusion incidence in cardiac surgery: a single-centre, randomised, open-label, parallel-group controlled pilot trial.

Background: Red blood cell (RBC) transfusions are frequent in patients after cardiac surgery. This study assessed whether a bundle of care including pre-operative and post-operative administration of erythropoietin (EPO) with intravenous iron supplementation, and restrictive transfusion adjusted for ScvO2 could result in reduced postoperative transfusions. Methods: In this single-centre, randomised, open-label, parallel-group controlled pilot study, patients undergoing elective cardiac surgery with high risk of transfusion in a University Hospital were enrolled by the investigator and the randomisation procedure using a central internet-based system was made by the clinical research assistant. Since the trial was open-label, no masking was used. Patients were assigned (1:1) to either the STOP group (40,000 IU subcutaneous EPO combined with 20 mg/kg intravenous ferric carboxymaltose if Hb < 13 g/dL the day before surgery or at ICU admission, and RBC transfusion if Hb ≤ 8 g/dL and ScvO2 ≤ 65%, or additional EPO dose if 8 < Hb < 13 g/dL) or to the control group (RBC transfusion if Hb ≤ 8 g/dL, or, if 8 < Hb < 13 g/dL, intravenous iron sucrose 200 mg or 300 mg according to weight). Primary outcome was the incidence of postoperative RBC transfusion up to hospital discharge or postoperative day 28. The trial is registered with ClinicalTrials.gov, NCT04141631. Findings: Between Jan 20, 2020, and Sept 6, 2022, among 128 patients enrolled, 123 (male, 54.4%, 67/123) were included in the full analysis set: 62 in the STOP group and 61 in the control group. Nine patients (14.5%, 9/62) in the STOP group required RBC transfusion vs 19 (31.2%, 19/61) in the control group (odds ratio 0.37 [95% CI: 0.15-0.91], p = 0.03). The median length of follow up to transfusion was 2.6 days (1.5; 4.6) and 3.3 (1.6; 4.2) in control and STOP groups respectively (p = 0.61). Interpretation: The bundle of care may reduce postoperative RBC transfusion. The findings should be taken with caution due to the unblinded and exploratory nature of the study. Funding: University of Montpellier Hospital and Vifor Pharma.

Implications of right aortic arch prenatal diagnosis: the multicentric nationwide ARCADE cohort.

OBJECTIVES: This study aims to describe the various presentations of the prenatally diagnosed isolated right aortic arch (RAA), that is, without associated congenital heart defect and to evaluate the impact of prenatal diagnosis of isolated RAA in terms of postnatal outcome. METHOD: In this multicentric retrospective study, from 2010 to 2019, all live births with a prenatal ultrasound diagnosis of isolated RAA were included, with a 1-year postnatal follow-up. The concordance between the different diagnostic steps (prenatal ultrasound, postnatal ultrasound and postnatal CT scan) was evaluated using Gwet's AC1 coefficient. RESULTS: A total of 309 cases of prenatally diagnosed RAA were analysed, most of which had a left ductus arteriosus (83%). The concordance between prenatal and postnatal ultrasound diagnosis was excellent regarding the RAA type (AC1=0.97, 95% CI=(0.94 to 0.99)). The rare discrepancies mainly involved non-diagnosed or misdiagnosed double aortic arch (2%). CT scan was performed in 108 neonates (35%) and the concordance between prenatal ultrasound and postnatal CT scan was good regarding the RAA diagnosis (AC1=0.80, 95% CI=(0.69 to 0.90)) but poor regarding the distribution of brachiocephalic vessels (AC1=0.21, 95% CI=(0.06 to 0.36)). An associated genetic anomaly was sought for in half of the cases and identified in 4% of the cohort. During the first year of life, 50 (18%) infants presented with vascular ring symptoms and 24 (8%) underwent aortic arch surgery. CONCLUSION: This multicentric nationwide cohort of 309 prenatally diagnosed isolated RAA demonstrated the reliability of prenatal screening, highlighted the rare cases of discrepancies between prenatal and postnatal diagnosis and underlined the value of CT scan to improve the postnatal follow-up. TRIAL REGISTRATION NUMBER: NCT04029064.

Efficacy of a Transition Program in Adolescents and Young Adults With Congenital Heart Disease: The TRANSITION-CHD Randomized Controlled Trial.

PURPOSE: Currently, nearly 90% of patients with congenital heart disease (CHD) reach adulthood in relatively good health. Structured transition programs have emerged to support adolescents and young adults in transitioning to adult care structures, improve their autonomy, and limit healthcare ruptures. The TRANSITION-CHD randomized controlled trial aimed to assess the impact of a transition program on health-related quality of life (HRQoL) in adolescents and young adults with CHD. METHODS: From January 2017 to February 2020, 200 subjects with a CHD, aged 13-25 years, were enrolled in a prospective, controlled, multicenter study and randomized in two balanced groups (transition program vs. standard of care). The primary outcome was the change in PedsQL self-reported HRQoL score between baseline and 12-month follow-up, using an intention-to-treat analysis. The secondary outcomes were the change in disease knowledge, physical health (cardiopulmonary fitness, physical activity), and mental health (anxiety, depression). RESULTS: The change in HRQoL differed significantly between the transition group and the control group (mean difference = 3.03, 95% confidence interval (CI) = [0.08; 5.98]; p = .044; effect size = 0.30), in favor of the intervention group. A significant increase was also observed in the self-reported psychosocial HRQoL (mean difference = 3.33, 95% CI = [0.01; 6.64]; p = .049; effect size = 0.29), in the proxy-reported physical HRQoL (mean difference = 9.18, 95% CI = [1.86; 16.51]; p = .015; effect size = 0.53), and in disease knowledge (mean difference = 3.13, 95% CI = [1.54; 4.72]; p < .001; effect size = 0.64). DISCUSSION: The TRANSITION-CHD program improved HRQoL and disease knowledge in adolescents and young adults with CHD, supporting the generalization and systematization of similar preventive interventions in pediatric and congenital cardiology.

Selective inner muscle layer myotomy is associated with lower pain and same clinical efficacy that full-thickness myotomy in patients treated by POEM for achalasia: A multicenter retrospective comparative analysis of 158 patients.

INTRODUCTION: The aim of this study was to compare the impact of the depth of myotomy (selective inner layer myotomy (SIM) vs. full-thickness myotomy (FTM)) on the outcome of patients treated with POEM for achalasia. METHODS: This was a retrospective, observational, conducted in two tertiary centers between October 2018 and September 2022. Patients were divided into two groups: SIM and FTM. The primary endpoint was clinical efficacy at 6 months, while secondary endpoints were postoperative criteria (such as pain, length of hospital stay, complications) and occurrence of gastroesophageal reflux disease (GERD) (esophagitis at 6 months, heartburn, and pH-metry). RESULTS: 158 patients were included in the study (33 in the FTM group and 125 in the SIM group). The success rates at 6 and 12 months were similar in both groups, with 84 % and 70 % in the SIM group versus 90 % and 80 % in the FTM group, respectively (p = 0.57 and p = 0.74). However, more opioid analgesics were consumed in the FTM group compared to the SIM group (41% vs 21 %, p < 0.01). The length of hospitalization was longer in the FTM group than in the SIM group (2.17 ± 2.62 vs 2.94 ± 2.33, p < 0.001). The rate of esophagitis at 6 months was comparable (16 % in the SIM group vs 12 % in the FTM group, p = 0.73). There was no significant difference in terms of heartburn at 6 or 12 months between the SIM and FTM groups (18.5% vs 3.8 %, p = 0.07 and 27% vs 12.5 %, p = 0.35, respectively). CONCLUSION: There was no significant difference in terms of clinical efficacy and GERD occurrence between FTM and SIM. However, full-thickness myotomy was associated with more postoperative pain and a longer length of hospital stay. Therefore, selective internal myotomy should be preferred over full-thickness myotomy.

Health-related quality of life in children and adolescents with Marfan syndrome or related disorders: a controlled cross-sectional study.

BACKGROUND: This cross-sectional controlled study aims to assess health-related quality of life (HRQoL) of children and adolescents with a molecular diagnosis of Marfan syndrome (MFS) or related disorders and to evaluate the factors associated with HRQoL in this population. Sixty-three children with MFS and 124 age- and sex-matched healthy children were recruited. HRQoL was assessed using the Pediatric Quality of Life Inventory (PedsQL™) generic questionnaire. The correlation between HRQoL scores and the different continuous parameters (age, body mass index, disease severity, systemic score, aortic sinus diameter, and aerobic physical capacity) was evaluated using Pearson's or Spearman's coefficient. A multiple linear regression analysis was performed on the two health summary self-reported PedsQL™ scores (physical and psychosocial) to identify the factors associated with HRQoL in the MFS group. RESULTS: Except for emotional functioning, all other domains of HRQoL (psychosocial and physical health, social and school functions) were significantly lower in children with MFS compared to matched healthy children. In the MFS group, the physical health summary score was significantly lower in female than in male patients (self-report: absolute difference [95%CI] = -8.7 [-17.0; -0.47], P = 0.04; proxy-report: absolute difference [95%CI] = -8.6 [-17.3; 0.02], P = 0.05) and also negatively correlated with the systemic score (self-report: R = -0.24, P = 0.06; proxy-report: R = -0.29, P = 0.03) and with the height Z-score (proxy-report: R = -0.29, P = 0.03). There was no significant difference in the physical health summary scores between the different genetic subgroups. In the subgroup of 27 patients who performed a cardiopulmonary exercise test, self- and proxy-reported physical health summary scores were highly correlated with their aerobic physical capacity assessed by peak oxygen consumption (VO2max) and ventilatory anaerobic threshold (VAT). In the multivariate analysis, the most important independent predictors of decreased physical health were increased height, decreased body mass index, decreased VAT and use of prophylactic therapy. CONCLUSIONS: This study reports an impaired HRQoL in children and adolescents with MFS or related conditions, in comparison with matched healthy children. Educational and rehabilitation programs must be developed and evaluated to improve exercise capacity and HRQoL in these patients. TRIAL REGISTRATION: ClinicalTrials.gov, NCT03236571 . Registered 28 July 2017.

Muscle strength, quantity and quality and muscle fat quantity and their association with oxidative stress in patients with facioscapulohumeral muscular dystrophy: Effect of antioxidant supplementation.

The purpose of this study was to identify causes of quadriceps muscle weakness in facioscapulohumeral muscular dystrophy (FSHD). To this aim, we evaluated quadriceps muscle and fat volumes by magnetic resonance imaging and their relationships with muscle strength and oxidative stress markers in adult patients with FSHD (n = 32) and healthy controls (n = 7), and the effect of antioxidant supplementation in 20 of the 32 patients with FSHD (n = 10 supplementation and n = 10 placebo) (NCT01596803). Compared with healthy controls, the dominant quadriceps strength and quality (muscle strength per unit of muscle volume) were decreased in patients with FSHD. In addition, fat volume was increased, without changes in total muscle volume. Moreover, in patients with FSHD, the lower strength of the non-dominant quadriceps was associated with lower muscle quality compared with the dominant muscle. Antioxidant supplementation significantly changed muscle and fat volumes in the non-dominant quadriceps, and muscle quality in the dominant quadriceps. This was associated with improved muscle strength (both quadriceps) and antioxidant response. These findings suggest that quadriceps muscle strength decline may not be simply explained by atrophy and may be influenced also by the muscle intrinsic characteristics. As FSHD is associated with increased oxidative stress, supplementation might reduce oxidative stress and increase antioxidant defenses, promoting changes in muscle function.

Cardiac magnetic resonance ventricular parameters correlate with cardiopulmonary fitness in patients with functional single ventricle.

Owing to advances in medical and surgical fields, patients with single ventricle (SV) have a greatly improved life expectancy. However, progressive functional deterioration is observed over time, with a decrease in cardiopulmonary fitness. This study aimed to identify, in patients with SV, the association between cardiac magnetic resonance imaging (CMR) parameters and change in cardiopulmonary fitness assessed by cardiopulmonary exercise test (CPET), and if certain thresholds could anticipate a decline in aerobic fitness. Patients with an SV physiology were retrospectively screened from 2011 and 2021 in a single-centre observational study. We evaluated (1) the correlation between baseline CMR and CPET parameters, (2) the association between baseline CMR results and change in peak oxygen uptake (peak VO2), and (3) the cut-off values of end-diastolic and end-systolic volume index in patients with an impaired cardiopulmonary fitness (low peak VO2 and/or high VE/VCO2 slope). 32 patients were included in the study. End-systolic volume index (r = 0.37, p = 0.03), end-diastolic volume index (r = 0.45, p = 0.01), and cardiac index (r = 0.46, p = 0.01) correlated with the VE/VCO2 slope. End-systolic ventricular volume (r = - 0.39, p = 0.01), end-diastolic ventricular volume (r = - 0.38, p = 0.01), and cardiac output (r = - 0.45, p < 0.01) inversely correlated with the peak VO2. In multivariate analysis, the cardiac index obtained from baseline CMR was inversely associated with the change in peak VO2 (p < 0.01). An end-diastolic volume index > 101 ml/m2 and an end-systolic volume index > 47 ml/m2 discriminated patients with impaired cardiopulmonary fitness. CMR parameters correlate with cardiopulmonary fitness in patients with SV and can therefore be useful for follow-up and therapeutic management of these patients.

Aerobic physical capacity and health-related quality of life in children with sickle cell disease.

BACKGROUND: Aerobic fitness is a predictor of cardiovascular health which correlates with health-related quality of life in the general population. The aim is to evaluate the aerobic capacity by cardiopulmonary exercise test (CPET) in children with sickle cell disease in comparison with healthy matched controls. METHODS: Controlled cross-sectional study. RESULTS: A total of 72 children (24 with sickle cell disease and 48 healthy controls), aged 6-17 years old were enrolled. Children with sickle cell disease had a poor aerobic capacity, with median VO2max Z-score values significantly lower than matched controls (-3.55[-4.68; -2.02] vs. 0.25[-0.22; 0.66], P < 0.01, respectively), and a high proportion of 92% children affected by an impaired aerobic capacity (VO2max Z-score < -1.64). The VO2max decrease was associated with the level of anemia, the existence of a homozygote HbS/S mutation, restrictive lung disease and health-related quality of life. CONCLUSION: Aerobic capacity is poor in children with sickle cell disease. VO2max decrease is associated with the level of anemia, the existence of a homozygote HbS/S mutation, lung function, and health-related quality of life. These results represent a signal in favor of early initiation of cardiac rehabilitation in patients with sickle cell disease. CLINICAL TRIALS: NCT05995743. IMPACT: Aerobic fitness is a predictor of cardiovascular health which correlates with health-related quality of life in the general population. Aerobic capacity (VO2max) is poor in children with sickle cell disease, despite the absence of any pattern of heart failure. VO2max decrease was associated with the level of anemia, the existence of a homozygote HbS/S mutation, restrictive lung disease, and health-related quality of life. These results are in favor of early initiation of cardiac rehabilitation in children with sickle cell disease.

Early hybrid cardiac rehabilitation in congenital heart disease: the QUALIREHAB trial.

BACKGROUND AND AIMS: Cardiopulmonary fitness in congenital heart disease (CHD) decreases faster than in the general population resulting in impaired health-related quality of life (HRQoL). As the standard of care seems insufficient to encourage and maintain fitness, an early hybrid cardiac rehabilitation programme could improve HRQoL in CHD. METHODS: The QUALIREHAB multicentre, randomized, controlled trial evaluated and implemented a 12-week centre- and home-based hybrid cardiac rehabilitation programme, including multidisciplinary care and physical activity sessions. Adolescent and young adult CHD patients with impaired cardiopulmonary fitness were randomly assigned to either the intervention (i.e. cardiac rehabilitation) or the standard of care. The primary outcome was the change in HRQoL from baseline to 12-month follow-up in an intention-to-treat analysis. The secondary outcomes were the change in cardiovascular parameters, cardiopulmonary fitness, and mental health. RESULTS: The expected number of 142 patients was enroled in the study (mean age 17.4 ± 3.4 years, 52% female). Patients assigned to the intervention had a significant positive change in HRQoL total score [mean difference 3.8; 95% confidence interval (CI) 0.2; 7.3; P = .038; effect size 0.34], body mass index [mean difference -0.7 kg/m2 (95% CI -1.3; -0.1); P = .022; effect size 0.41], level of physical activity [mean difference 2.5 (95% CI 0.1; 5); P = .044; effect size 0.39], and disease knowledge [mean difference 2.7 (95% CI 0.8; 4.6); P = .007; effect size 0.51]. The per-protocol analysis confirmed these results with a higher magnitude of differences. Acceptability, safety, and short-time effect of the intervention were good to excellent. CONCLUSIONS: This early hybrid cardiac rehabilitation programme improved HRQoL, body mass index, physical activity, and disease knowledge, in youth with CHD, opening up the possibility for the QUALIREHAB programme to be rolled out to the adult population of CHD and non-congenital cardiac disease.

A non-randomized controlled study to assess the impact of the "Appui Parental", an early and intensive support programme for vulnerable families: Study protocol.

INTRODUCTION: Early home visit programmes have been developed to help parents build an adequate relationship with their baby and to prevent child developmental delays and affective disorders. The "Appui Parental" programme is an intervention carried out by nursery nurses to provide intensive parental support to vulnerable families. Before extending this programme, it seemed necessary to evaluate its impact objectively. OBJECTIVES: The main aim is to determine the impact of the "Appui Parental" programme on the change in the child's symptoms. The secondary objectives are to evaluate its effects on mother-child interactions, self-assessed parental competence, perception of social support, primary caregiver's anxiety-depression symptoms, alliance with the nursery nurse, frequency of out-of-home placements, and nursery nurses' stress. METHOD: This non-randomized prospective multicentre study would include 44 families who receive the "Appui Parental" intervention for a one to 20-month-old child (intervention group) and 44 families with the same vulnerability criteria who receive care as usual by the maternal and child protection services (control group). The child, parents, mother-child interaction, nursery nurse-mother alliance, and nursery nurse's stress will be assessed at month one and month 18 after inclusion. Comparisons between groups will be performed. CONCLUSION: This study should provide the public authorities with objective data on this programme's impact and allow them to pursue its generalization. For professionals, the study should confirm the interest in close early parental support through home visits or should lead to rethinking some aspects of the programme.

Association of difficulties in motor skills with longitudinal changes in social skills in children with autism spectrum disorder: findings from the ELENA French Cohort.

Developmental coordination disorder is a frequently co-occurring condition with autism spectrum disorder (ASD). Several cross-sectional studies have reported that children with difficulties in motor skills have a higher severity of ASD symptoms. This study aims to examine the association of difficulties in motor skills with longitudinal changes in social skills in children with ASD. Participants were drawn from the ELENA cohort, a French longitudinal cohort of children with ASD. Motor skills were assessed using the Movement Assessment Battery for Children at baseline, while social skills were measured using the Autism Diagnostic Observation Schedule (ADOS-2) and the Vineland Adaptive Behavior Scales (VABS-II) at both the baseline and a follow-up assessment conducted 3 years later. A composite score of social skills was created at baseline and at both time points. Linear regression models were performed to assess the association between difficulties in motor skills and changes in social skills, considering potential confounders such as IQ, age, and gender. The sample included 162 children with ASD. Children with difficulties in global motor skills (N = 114) showed less favorable trajectories in social skills compared to those without motor difficulties. The results were consistent when examining the ADOS-2 and the VABS-II separately. This study provides evidence for the negative impact of difficulties in motor skills on the longitudinal development of social skills in children with ASD. Interventions targeting motor difficulties may have broader benefits, extending beyond motor function to improve socialization.

Prospective evaluation of transoral outlet reduction (TORe) after Roux-en-Y gastric bypass (RYGB) failure.

BACKGROUND AND AIMS: Weight regain after RYGB is multifactorial including dilatation of the gastro-jejunal anastomosis. Transoral outlet reduction (TORe) procedure is a minimally invasive alternative to surgical anastomotic revision. METHODS: We conducted a prospective, multicenter, simple blind, randomized study in patients with weight regain following RYGB, comparing the efficacy of conventional nutritional and behavioral management associated with a TORe procedure (TORe group) with conventional management alone and a Sham procedure (Sham group). The main objective of this study was to evaluate the percentage of excess weight loss (%EWL) at 12 months after endoscopy. RESULTS: From January 2015 to January 2019, 73 subjects were randomized in four French Bariatric centers. The final analysis involved 50 subjects, 25 in each group, 44 women, 6 men, with an average BMI of 40.6 kg/m2. At 12 months, the average %EWL was significantly higher in the TORe group than in the Sham group (13.5 ± 14.1 vs. - 0.77 ± 17.1; p = 0.002). Cohen's d was 0.91, indicating a large effect size of the procedure on the %EWL. There was no significant difference between groups concerning the improvement of obesity-related comorbidities (diabetes and dyslipidemia) and quality of life at 12 months. We report frequent adverse events in the TORe group (20% had adverse events related to the procedure). Three adverse events were serious, including two perforations of the gastro-jejunal anastomosis after TORe group that led to the premature termination of the study. CONCLUSIONS: After RYGBP failure linked to the dilatation of the gastro-jejunal anastomosis, TORe procedure with nutritional management results in significantly higher %EWL at 12 months compared to patients with nutritional management alone. As surgery, this minimally invasive endoscopic procedure can be associated with severe adverse events.

Neuropsychological assessment fails to predict relapse among cigarette smokers: A prospective study of neurocognitive abilities.

OBJECTIVES: Understanding the factors that lead to relapse is a major challenge for the clinical support of smoking cessation. Neurocognitive abilities such as attention, executive functioning and working memory, are possible predictors of relapse and can be easily assessed in everyday clinical practice. In this prospective longitudinal study, we investigated the relationship between pre-smoking cessation neurocognitive performance and relapse at six months in a sample of patients being treated for their tobacco dependence. METHODS: 130 tobacco consumers were included in the study. They completed a comprehensive neuropsychological and clinical assessment before smoking cessation. The targeted abilities were intelligence, inhibition, shifting, working memory updating, verbal fluency and decision-making. RESULTS: The rate of tobacco relapse at 6 months was 58%. Logistic regressions were used to assess which variables best explained relapse. None of the neuropsychological tests was a significant predictor of relapse at either 1, 3 or 6 months, either alone, or controlling for other covariates acting as significant predictors of relapse. CONCLUSIONS: Common neuropsychological tests, even those specifically targeting executive functioning such as inhibition, are not useful predictors of the success of a smoking cessation program in a clinical setting. Other variables, such as motivation to quit smoking or the presence of comorbid depression or anxiety disorders, appear to be more useful predictors of relapse.

High prevalence of malnutrition in systemic sclerosis: Results from a French monocentric cross-sectional study.

OBJECTIVES: Systemic sclerosis (SSc) can cause malnutrition due to frequent gastrointestinal involvement. However, prevalence of malnutrition in SSc is poorly known. The aim of this study was to evaluate the prevalence of malnutrition in SSc and its potential associations with disease features in patients from a tertiary referral center. METHODS: All patients meeting American College of Rheumatology/European Alliance of Associations for Rheumatology criteria for SSc followed between January 1, 1985, and January 1, 2019, at the Department of Internal Medicine, Saint Eloi University Hospital, were included. Malnutrition was assessed using the 2020 French recommendations for SSc and the malnutrition universal screening tool score. Severe malnutrition was defined via the French Haute Autorité de Santé (National Health Authority) 2007 criteria. RESULTS: A total of 120 patients were included, with mean age 64 (± 15) y and a female-to-male sex ratio of 5:1. According to 2020 French recommendations, 71 patients (59.2%) were malnourished and 30 (25%) had at least one criterion of severe malnutrition. With the malnutrition universal screening tool score, 41.7%, 20%, and 38.3%, respectively, had low, medium, and high risk of malnutrition. Multivariate analysis revealed the following results: 1) malnutrition was associated with cardiac involvement (P < 0.01); 2) a high malnutrition universal screening tool score was also associated with specific cardiac involvement (P < 0.01); and 3) severe malnutrition was strongly correlated with interincisal distance <35 mm (P = 0.02). CONCLUSIONS: Malnutrition affects more than half of SSc patients and is associated with specific cardiac involvement. Interincisal distance <35 mm could be a red flag for severe malnutrition in SSc.

Effect of Nutritional Deprivation after Sleeve Gastrectomy on Bone Mass, Periostin, Sclerostin and Semaphorin 4D: A Two-Year Longitudinal Study.

Bariatric surgery induces bone loss, but the exact mechanisms by which this process occurs are not fully known. The aims of this 2-year longitudinal study were to (i) investigate the changes in areal bone mineral density (aBMD) and bone turnover markers following sleeve gastrectomy (SG) and (ii) determine the parameters associated with the aBMD variations. Bone turnover markers, sclerostin, periostin and semaphorin 4D were assessed before and 1, 12 and 24 months after SG, and aBMD was determined by DXA at baseline and after 12 and 24 months in 83 patients with obesity. Bone turnover increased from 1 month, peaked at 12 months and remained elevated at 24 months. Periostin and sclerostin presented only modest increases at 1 month, whereas semaphorin 4D showed increases only at 12 and 24 months. A significant aBMD decrease was observed only at total hip regions at 12 and 24 months. This demineralisation was mainly related to body weight loss. In summary, reduced aBMD was observed after SG in the hip region (mechanical-loading bone sites) due to an increase in bone turnover in favour of bone resorption. Periostin, sclerostin and semaphorin 4D levels varied after SG, showing different time lags, but contrary to weight loss, these biological parameters did not seem to be directly implicated in the skeletal deterioration.