Intestinal failure (IF) is a broad term encompassing various conditions that hinder the body’s ability to absorb nutrients for growth and maintenance. These conditions can significantly affect child’s well-being, leading to physical limitations, psychological distress, and social isolation. We aimed to evaluate the available data on health-related quality of life (HRQoL) in pediatric patients with IF and without neurodevelopmental delay. For this systematic review and meta-analysis, we searched CINAHL, EMBASE, PsycINFO, PubMed, and Web of Science. All observational studies of pediatric patients (< 18 years) with IF which measured HRQOL and with evidence of absence of neurodevelopmental delay were included, without language or date restrictions, up to June 2024. We did separate random-effects meta-analyses for overall HRQOL and subgroup domains. Evidence from observational studies was synthesised as differences between standardised mean differences (SMDs) for all subgroup domains. Heterogeneity was assessed using the I² statistic and the Cochran Q test. The quality of the evidence was assessed with the Newcastle-Ottawa scale. This study is registered on PROSPERO, number CRD42024561812. Of 491 records identified, 14 were eligible and data were available for 12 studies, all of which had a fair/good quality. The included studies involved a pooled sample of 510 participants (mean age = 7.0 ± 3.6 years). The analysis disclosed that compared to healthy children, pediatric patients with IF had lower overall quality of life in both child- and parent-report (Standardized Mean Difference [SMD]= -0.62; 95

BACKGROUND: Telemedicine and tele monitoring represent an emerging-study area in several and chronic diseases. Tele-rehabilitation during COVID-19 disease became an essential tool to promote physical activity in total safety. In fact, for pwCF physical activity was fundamental not only for the respiratory program but also. The aim of the study was to evaluate the feasibility of home web-based program of home exercise training program in patients with cystic fibrosis (CF) during COVID-19 pandemic. METHODS: Thirty-two patients (12 M/ 20 F) age 20.52 (+/- 9.3); FEV1 mean was 84.88% (+/- 21.1) and Ph angle mean at T0 was 6.11 (+/- 0.83). Patients performed at the beginning spirometry, CF questionnaire-revised, bioimpedance analysis and handgrip-test. At the end of study, patients replaced the same examinations with also satisfaction and utility Likert Scales. RESULTS: Adherence average rate was 61.5%. Satisfaction and utility scores were respectively 4.5 and 4.3(Likert Scale); about QoL the Body-domain had a statistically significant increase (P value <0.05). CONCLUSIONS: Web-based rehabilitation could be a good tool but more studies are needed to confirm optimal values of adherence. Although data are based on a small sample size and longer periods of treatment are requested to analyze especially the physiological answer about heart rate and lung volumes.

Literature has highlighted a significant relationship between chronic disease and mental health, particularly in inflammatory bowel disease (IBD). The aim of the study was to evaluate the prevalence of anxiety and depressive symptoms and suicidal ideation in a sample of adolescents with IBD and identify associated risk factors. A retrospective analysis of demographic, clinical, and mental health data was conducted using validated instruments (PHQ-9 for depressive symptoms; GAD-7 for anxiety symptoms; PHQ-9 > 0 for suicidal ideation) on 147 pediatric patients (mean age = 15.7 +/- 2.6; males = 77 [52.4%]) with IBD treated at the Bambino Ges & ugrave; Children's Hospital in Rome. A regression analysis was conducted to assess the influence of risk factors (age, sex, previous surgery, time since diagnosis, body mass index, type [Crohn's disease vs. ulcerative colitis], and disease activity). Approximately one-fifth of the sample of adolescents with IBD exhibited clinically significant signs of anxiety and depression (moderate to severe symptoms of both anxiety and depression were observed in 22.4% and 20.4% of patients, respectively) and approximately one-sixth of patients reported suicidal ideation (15.6%), suggesting a higher prevalence in all three areas compared to the general population. Females demonstrated higher levels of anxiety and depression compared to male. Among the risk factors considered, maintaining a healthy body weight has a positive effect in reducing anxiety symptoms. Adopting a multidisciplinary approach that integrates psychological prevention and interventions into the treatment of these patients appears essential, particularly in adolescence.

Background: Italy initiated elexacaftor/tezacaftor/ivacaftor (ETI) for people with cystic fibrosis (pwCF) in July 2021. It has led to dramatic improvements in lung function, BMI, sweat chloride, and respiratory symptoms. However, few data are available on side effects or effects on a broad range of outcomes. Research question: How does ETI affect mental health, cognitive processing, neuropsychological side effects, GI symptoms, and health-related quality of life over time? Study design and methods: This was a prospective, "real-world" longitudinal study. Participants were recruited consecutively and evaluated at initiation (T0) and after 1 month, 3 months, and 6 months of starting treatment. Assessments included depression (nine-item Patient Health Questionnaire), anxiety (seven-item Generalized Anxiety Disorder), cognition (Symbol Digit Modalities Test), GI Symptom Tracker, and health-related quality of life (Cystic Fibrosis Questionnaire-Revised). Based on literature, an ad hoc questionnaire was developed to assess side effects: insomnia, headache, memory problems, "brain fog," and concentration problems. Following descriptive analyses, longitudinal data were analyzed by using mixed models for repeated measures, controlling for age and sex when appropriate. Results: Ninety-two consecutive pwCF (female/male, 46/46; mean age, 25.4 years) participated. FEV1 increased initially and then remained stable. BMI also increased significantly from T0 to 6 months (P < .01). Depression improved from T0 to 1 month (P < .001); however, no changes in anxiety were found. Cognitive processing improved from T0 to subsequent assessments. Positive changes were reported on the GI Symptom Tracker for stools and adherence challenges, although no changes were found for abdominal pain and digestion. Side effects occurred in 10% to 29%, with no reduction over time; insomnia increased significantly across time. Female participants reported more side effects than male participants (ie, insomnia, headache, concentration problems, brain fog). Interpretation: This prospective study evaluated the effects of ETI using multiple measures. Significant improvements were found in many domains; however, side effects were reported by a substantial proportion of pwCF, with no improvements over time. Female participants reported more side effects than male participants. pwCF should be followed up systematically to assess the frequency of side effects after starting this new modulator.

Background Inflammatory bowel diseases [IBD] are chronic and pervasive conditions of the gastrointestinal tract with a rising incidence in paediatric and young adult populations. Evidence suggests that psychological disorders might be associated with relapse of disease activity. This study aims to evaluate the efficacy of short-term psychodynamic psychotherapy [STPP] in addition to standard medical therapy [SMT] in maintaining clinical remission in adolescents and young adults [AYA] with quiescent IBD, compared with SMT alone.Methods A two-arm, single-centre, randomised, controlled trial was conducted in 60 IBD AYA in clinical remission. Patients were randomised to receive an 8-week STPP + SMT [n = 30] or SMT alone [n = 30]. The primary outcome was the steroid-free remission rate at 52 weeks after treatment. Secondary outcomes included the overall hospitalisation rate within 52 weeks after treatment, and medication adherence obtained from patient's electronic medical records.Results Intention-to-treat analysis showed significant improvement in maintaining disease remission rates in the 8-week STPP + SMT group compared with the control one. The proportion of patients maintaining steroid-free remission at 52 weeks was higher in patients in STTP group [93.1%] compared with patients randomised to control group [64.3%; p = 0.01]. There were no significant differences in secondary outcomes, except for depression reduction in STPP + SMT group.Conclusions An 8-week STPP intervention in addition to SMT effectively increases the steroid-free remission rates in AYA with quiescent IBD. Results do not support effects for other secondary outcomes, except for depression reduction.

Advancements in early diagnosis and paediatric cardiac surgery have improved the long-term survival of patients with congenital heart disease, necessitating a thorough assessment of their health-related quality of life (HRQoL). This study aimed to assess HRQoL in paediatric patients with coarctation of the aorta (CoA) (both as reported by patients and caregivers), and to evaluate associated factors. Patients aged 5-18 years diagnosed with CoA and their parents were enrolled at Bambino Ges & ugrave; Children's Hospital between September 2016 and December 2017. Socio-demographic characteristics were recorded using a family form, and the Pediatric Quality of Life Inventory (PedsQL) 3.0 cardiac module was used to evaluate HRQoL. Clinical data were retrieved from medical chart reviews. In this observational study, sixty-five pediatric patients (39 males, median [IQR] age 12 [9-14]) with CoA and their parents (65 mothers and 65 fathers) were enrolled. These patients exhibited overall good HRQoL. Mothers reported significantly lower total HRQoL scores compared to patient self-reports (p = .037), as well as treatment anxiety (p = .033), and cognitive problems (p = .021). Pediatric patients with CoA perceived their HRQoL better than their mothers did. Female sex and older age were associated with lower HRQoL scores.

Background: Hypoxic-ischemic encephalopathy (HIE) is a severe pathology, and no unique predictive biomarker has been identified. Our aims are to identify associations of perinatal and outcome parameters with morphological anomalies and ADC values from MRI. The secondary aims are to define a predictive ADC threshold value and detect ADC value fluctuations between MRIs acquired within 7 days (MR0) and at 1 year (MR1) of birth in relation to perinatal and outcome parameters. Methods: Fifty-one term children affected by moderate HIE treated with hypothermia and undergoing MRI0 and MRI1 were recruited. Brain MRIs were evaluated through the van Rooij score, while ADC maps were co-registered on a standardized cerebral surface, on which 29 ROIs were drawn. Statistical analysis was performed in Matlab, with the statistical significance value at 0.05. Results: ADC0 < ADC1 in the left and right thalami, left and right frontal white matter, right visual cortex, and the left dentate nucleus of children showing abnormal perinatal and neurodevelopmental parameters. At ROC analysis, the best prognostic ADC cut-off value was 1.535 mm2/s × 10−6 (sensitivity 80%, specificity 86%) in the right frontal white matter. ADC1 > ADC0 in the right visual cortex and left dentate nucleus, positively correlated with multiple abnormal perinatal and neurodevelopmental parameters. The van Rooij score was significantly higher in children presenting with sleep disorders. Conclusions: ADC values could be used as prognostic biomarkers to predict children’s neurodevelopmental outcomes. Further studies are needed to address these crucial topics and validate our results. Early and multidisciplinary perinatal evaluation and the subsequent re-assessment of children are pivotal to identify physical and neuropsychological disorders to guarantee early and tailored therapy.

Abstract Background and aims Anxiety and depression symptoms are common in IBD population, both adult and pediatric patients. Increased psychological distress might contribute to initiation and switching to biologic therapy in adult patients with IBD or other chronic inflammatory diseases. Aim of the present study are to evaluate anxiety and depression symptoms in IBD pediatric patients with disease remission and investigate their role in initiation or switching to biologic therapy. Methods We performed a retrospective analysis on IBD pediatric patients, assessing for anxiety (GAD-7) and depression (PHQ-9) symptoms. Demographic and disease characteristics were obtained from medical records. Results Eighty-six patients [31 (36%) females - mean age = 15.6 (SD = 2.8) years] were included. Patients scored above cut-off (> 10) on PHQ-9 and GAD-7 were 17 (19.7%) and 18 (20.9%), respectively. No differences were found between UC and CD patients. Baseline clinically relevant depression symptoms were significantly associated with the odds of initiating or switching to biologic therapy within 2 years [OR = 4.5 (1.4–14.3)], even after confounders adjustment [4.2 (1.2–14.9)]. Relationship was not significant with anxiety symptoms. Conclusion Anxiety and depression symptoms is relatively common in pediatric IBD population, even with disease remission. Pediatric IBD patients with high depression symptoms are at increased risk of initiating or switching to biologic therapy. Mental health screening programs should be incorporated in routine clinical practice, especially for depression, regardless of disease activity and disease type. Early diagnosis and proper intervention for mental illness should be part of routine IBD management.

Objectives: Italy started elexacaftor/tezacaftor/ivacaftor (ETI) in July 2021. This study evaluated the global effects of ETI in a longitudinal study, assessing patients prior to and for 6 months after starting ETI. Measures focused on the "whole person" and included neurocognitive side-effects, physical, mental and health-related quality of life (HRQoL) outcomes. Methods: This was a prospective observational study that enrolled 92 consecutive pwCF, ages 12–59 years (F/M = 46/47; M age = 25.4). An ad-hoc measure, based on published literature, was developed to assess common neurocognitive side-effects on a 4-point Likert scale (e.g., headache, memory, concentration, insomnia, brain fog). Measures were administered within 2 weeks of initiating ETI and at 1, 3 and 6 months: FEV1, BMI, PHQ-9, GAD-7, GI Symptom Tracker (4 scales: Abdominal Symptoms, Stools, Eating Challenges, Adherence Challenges), Symbol Digit Modality Test (SDMT), neurocognitive side-effects, and CFQ-R. Results: As expected, improvements in FEV1 and BMI were observed across time. Neurocognitive side-effects were reported most frequently for headaches (27–30%) and memory (15%-20%), endorsed more often by females than males. Cognitive processing on the SDMT improved significantly by 6 months, but performance at baseline was 1 standard deviation below the mean. Few improvements in GI symptoms were reported over time. Depression, but not anxiety, improved by 3-months post-ETI and were sustained at 6 months. HRQoL improved on a majority of domains, except Body Image and Digestion. Improvements were most significant from baseline to 3-months, with decreases in some scales at 6-months. Conclusion: This is the first study to evaluate the effects of ETI on the whole person over time. A substantial percentage of women with CF reported neurocognitive side-effects. No improvements in GI symptoms were found. Improvements in other domains of functioning were reported for cognitive processing, depression, and HRQoL.

Introduction: Telemedicine and tele-monitoring represent an emerging-study area in several and chronic diseases. Tele-rehabilitation during Covid-19 disease became an essential tool to promote physical activity in total safety. Infact, for pwCF physical activity was fundamental not only for the respiratory program but also Aim: To evaluate the feasibility of home web-based program of home exercise training program in patients with cystic fibrosis during COVID-19 pandemic. Subjects and methods: 32 patients (12 M/ 20 F) age 20,52 (±9,3); FEV1 mean was 84,88% and Ph angle mean at T0 was 6,11 (sd 0,83). Results: Adherence average rate was 61,5 %. Satisfaction and utility scores were respectively 4.5 and 4.3(Likert Scale); about QoL the Body-domain had a statistically significant increase (pvalue <0.05). Conclusion: Web-based rehabilitation could be a good tool but more studies are needed to confirm optimal values of adherence. Although data are based on a small sample size and longer periods of treatment are requested to analyze especially the physiological answer about heart rate and lung volumes.

BACKGROUND:Elexacaftor-tezacaftor-ivacaftor (ETI) represents a significant step forward in cystic fibrosis (CF) care and could change the course of CF lung disease and quality of life for many people with CF (PwCF). However, several PwCF cannot benefit from these modulators because their rare mutations are not eligible for treatment. This study aimed to investigate the lived experiences of PwCF who are not eligible for ETI.METHODS:Data were collected through semi-structured interviews with 13 individuals with CF that were not eligible for ETI. Thematic analysis was used to identify the key themes of their experiences.RESULTS:Two main themes and six subthemes were identified. The first main theme (being deemed ineligible for ETI) had four subthemes (disappointment, information, happiness, and concerns). The second main theme (coping with a life without ETI) had two subthemes (lack of hope and continued hope).CONCLUSIONS:PwCF who are not eligible for ETI experience intense disappointment and conflicting emotions that can influence their decision-making linked to diminishing/renewal hope. Integrated care, including mental health monitoring programs, should be provided to these patients to aid them in overcoming their disappointment and to improve their coping.

AIMS:To synthesize qualitative studies of subjective experience of pregnancy in people with cystic fibrosis. DESIGN:Thematic synthesis of qualitative studies. DATA SOURCES:We searched PUBMED, CINAHL, EMBASE. PsicINFO and Social Sciences Citation Index for qualitative studies published in English, which reported on pregnancy in people with cystic fibrosis. Searches were carried out in March 2021, updated in June 2022. REVIEW METHODS:Studies that met the inclusion criteria were appraised for quality using the Critical Appraisal Skills Programme (CASP) for qualitative research. Data were extracted from the studies, analyse and synthesise using thematic synthesis approach. RESULTS:Thirteen studies were included in the review and 'Walking on a wire' framework was conceptualized. We found three analytical themes: (1) desire for information related to pregnancy in cystic fibrosis, (2) factors at play in decision-making related to pregnancy for people with cystic fibrosis and (3) pregnancy experience and eight descriptive themes: (1) information topics, (2) CF healthcare team/PwCF as a provider of reproductive health information, (3) information timing, (4) barriers to information delivery, (5) barriers to decision-making and stresses relating to the process of trying to conceive, (6) environmental factors, (7) coping with challenges, and (8) moving towards parenthood. CONCLUSION:For people with cystic fibrosis, pregnancy is a complex pathway: the amount of knowledge about cystic fibrosis and sexual and reproductive health, barriers to pregnancy and environmental factors provides the background to decision-making. Moreover, coping with pregnancy is a challenging experience, where they have to mediate between the physical and emotional implications of planning a pregnancy and the limitations imposed by the chronic health conditions. IMPACT:Understanding the psychological experiences of people with cystic fibrosis (PwCF) will improve future research and practice. Education about sexual and reproductive health and psychosocial care programmes are necessary to help PwCF deal with the challenges related to pregnancy. Hospitals should enhance the development of specific programmes to promote the well-being of individuals with CF who are planning a pregnancy.

Figure 1.Responses to difference in current perceived stress level before and durring the pandemicConclusions: This is the first known descriptive study to look at effects of a worldwide pandemic on caregivers of young PwCF.It also is one of few studies examining caregiver perceived self-efficacy in CF.It was limited by the number of responses, skewed demographics of those who responded, and being administered at a single CF center.We found that caregivers of PwCF at our center are experiencing higher rates of stress, anxiety, and anger.This did not seem to affect their perceived self-efficacy, although there was no pre-pandemic measurement.We learned that we need to find ways to reach a larger demographic and those who are underrepresented.This study shows the need to better understand caregiver emotional distress, especially during times of ambiguity.Understanding caregiver stress and perceived self-efficacy has the potential to provide insight for the medical team on supportive ways to abate negative outcomes in young PwCF.

Introduction: COVID-19 emerged in China leading to worldwide morbidity and mortality, including depression and anxiety. As the pandemic spread throughout Italy and Europe, mental health concerns emerged for people with cystic fibrosis (pwCF), who are at increased risk. This led to development of a Telehealth Psychological Support Intervention to help adolescents/young adults with CF and caregivers cope with the stress and emotional challenges of the lockdown which began in Italy in March 2020. Methods: This intervention utilized cognitive behavioral skills (e.g., relaxation training, cognitive reframing). Participants included 16 adolescents/adults and 14 parents, who completed 4 individual video sessions with a psychologist. Stress ratings, PHQ-8 and GAD-7 were completed electronically. Feasibility and Satisfaction were rated. Results: Ratings of stress significantly decreased from pre to post-testing for both pwCF (paired t=-4.06 (14), p<.01) and parents (paired t=-5.2, p< 001). Most participants reported clinically elevated depression and anxiety at the pre-test, with statistically significant reductions in depression for pwCF (pre: M=8.0 to post M=4.7; paired t (14)=2.8, p <.05) but not anxiety (pre: M=6.9 to post: M=5.6, t (14)=1.2, p=NS). Reductions in depression were found for parents (pre: M=6.4 to post: M 5.1, (14) t=-2.5, p<.05) but not anxiety (pre: M=8.1 to post: M=7.9, t (14)=-0.2, p=ns). Positive ratings of Feasibility and Satisfaction were documented. Conclusion: This brief telehealth intervention yielded significant reductions in stress and depression for participants. Anxiety was not reduced, possibly because COVID was ongoing. This intervention was effective for improving mental health and was highly feasible and satisfactory.

Introduction Psychological problems are common among the pediatric population suffering from a chronic illness, especially compared to the normal population. Stressful life events, such as social distancing measures implemented to counter the COVID-19 pandemic emergency, can strongly influence their epidemiology. Objectives The aim of this study was to assess the prevalence rate of depressive and anxiety symptoms among an Italian pediatric population affected by chronic illness and already under a Children Hospital psychological follow-up program during COVID-19 lockdown. Methods We conducted a cross-sectional study among 54 Italian children and adolescents affected by chronic illness (mean age: 15y; range: 8.9-18y) during the COVID-19 epidemic period. We assessed depressive and anxiety symptoms with the Patient Health Questionnaire (PHQ-9) and the Generalized Anxiety Disorders (GAD-7) questionnaire during scheduled follow-up checks or teleconsulting. Results Preliminary results showed an elevated prevalence of depressive and anxiety symptoms (51% and 48% respectively) among chronically ill children during the COVID-19 outbreak. When compared with a non-ill pediatric population (Zhou et al. 2020), rates are + 7.3% higher for depression and + 10.6% for anxiety. Conclusions Chronic ill pediatric patients are a vulnerable group and require careful consideration. For this reason, the healthcare system should be able to implement and guarantee adequate mental health support programs and continuity of care. Further research is necessary since the COVID-19 outbreak could be repeated.

Abstract Background From March 10 to May 3, 2020, Coronavirus disease 2019 (COVID-19) and subsequent enforced lockdown in Italy has led to an increased general psychological distress1. In particular, people with both compromised immune function and pre-existing physical or psychiatric problems are at increased risk of adverse psychosocial outcomes2. The purpose of this study was to compare mental health burden in Inflammatory Bowel Disease (IBD) paediatric patients with and without COVID-19 lockdown exposure. Methods We conducted a retrospective case-control study comparing mental health outcomes of two matched 1:1 IBD paediatric groups: Group 1 - before the exposure (January 2019-September 2019) and Group 2 - after COVID-19 pandemic lockdown (April 2020-December 2020). Matching criteria included gender, age, disease duration, IBD subtype, Body Mass Index and disease activity (remission or active). This study included patients with Crohn Disease (CD) and Ulcerative Colitis (UC), >12 years, attending outpatient visits at Bambino Gesù Children Hospital. Data were collected using the Generalised Anxiety Disorder (GAD-7) for anxiety and the Patient Health Questionnaire (PHQ-9) for depression. We have estimated the prevalence of anxiety and depression between the two groups. Both groups were also compared with respect to the average score of anxiety and depression symptoms. Sociodemographic and clinical data were recorded from patients’ medical chart. Results A total of 108 IBD paediatric patients (62 males, 54 with CD and 54 with UC, mean age=16±2,5 years) was enrolled in this study. Out of these, 54 patients were enrolled in Group 1 and 54 in Group 2. Using a cut-off score ≥10, we found similar prevalence of anxiety (13.0% vs 22.2%, P=.206) and depression (13.0% vs 18.5%, P=.428) among the groups. Average scores were quite similar among the two groups, both for anxiety (5.78 vs 5.96, P=.924) and depression (5.89 vs 5.87, P=. 704). Symptom severity levels were mild (≃6) in both groups, according to standardized instrument cut-offs. No significant associations were found between disease activity and symptoms of anxiety and depression in both groups. Conclusion IBD-related symptoms (i.e. weight loss, persistent diarrhoea, abdominal pain and fatigue) lead to severe psychosocial impairment in paediatric IBD patients. COVID-19 pandemic outbreak and enforced isolation (i.e., lockdown, long distance learning, and social restrictions) limited patients’ stressful social situations engagement and paradoxically prevented a worsening of their mental health. References

BACKGROUND:Cystic fibrosis (CF) is a chronic, life-threatening condition that results in life-long morbidity and premature mortality. CF has a significant impact on healthy siblings' adaptation and well-being. Siblings of patients with a chronic disease may experience psychological difficulties, such as anxiety, depression symptoms and troubles in adaptation. This study aimed to explore the participants' experiences of growing up with a CF patient diagnosed at a paediatric age and their adaptation strategies.METHODS:We enrolled eight CF siblings (adolescents and young adults) in a 6-month focus group sessions programme. Each session had two phases (psychoeducational and experiential). Transcripts were analysed and grouped through grounded theory analysis to elaborate on data-driven theory.RESULTS:We identified 14 subthemes by which the raw data could be organized. All the subthemes were gathered together according to the axial coding process into six themes (illness, changes, communication, avoidance, normalization, and protection and care). We then grouped the six themes into two main themes ('Growing up with a CF brother or sister' and 'Finding the right distance') and conceptualized the grounded theory 'Keeping the right distance'. The participants described the evolving process of maintaining a balance between the illness of their brother or sister, family organization and their own needs.CONCLUSIONS:Knowing siblings' experiences and their common strategies to deal with the experience of having a brother or sister with a chronic health condition may be useful to ensure more tailored and specific interventions.