Sara Azevedo

Background: Iron deficiency (ID) and iron deficiency anemia (IDA) are common complications of pediatric inflammatory bowel disease (IBD). Owing to questions regarding optimal iron formulation, dosage, route of administration, and safety, these complications are frequently overlooked and undertreated, negatively impacting patient development and quality of life.Purpose: To assess the safety and efficacy of iron sucrose (IS) and ferric carboxymaltose (FCM) in the treatment of ID and IDA in pediatric IBD.Methods: We retrospectively reviewed the medical records of pediatric patients with IBD treated for 10 years with IS (age <14 years) or FCM (age ≥14 years) in a single regional referral center. The Ganzoni formula was used to calculate the iron dose administered. Adverse reactions were monitored during treatment and after discharge. Efficacy was defined as a ≥2 g/dL rise in Hb or anemia resolution within 12 weeks after treatment in cases of IDA and transferrin saturation or ferritin normalization in cases of ID.Results: Sixty-three patients were treated with IV iron (41 with Crohn disease, 15 with ulcerative colitis, 7 with IBD-unclassified; median age, 14.6 years; 104 treatment courses [63 FCM, 41 IS during the 10-year study period]). Retreatment was necessary after a median 1.4 years in 26 patients (41.3%). The median activity scores of patients with recurrent ID indicated inactive disease. The treatment efficacy was 66.7% (FCM) and 67.6% (IS) in patients with IDA and 77.8% in patients with ID but without anemia. One adverse reaction (hypotension and rash) was associated with IS treatment.Conclusion: In one of the largest and longest follow-up cohorts, FCM and IS were safe and effective for correcting ID in pediatric patients with IBD. As ID recurs frequently, proactive screening and treatment are important.

Background: Pediatric Crohn’s Disease (CD) affects more than physical health, influencing emotional well-being, social integration, and developmental milestones, with an impact on disease management. This study aimed to explore adolescents’ lived experiences with CD and identify factors influencing their motivation for self-management. Methods: A descriptive, cross-sectional qualitative study was conducted using a semi-structured, self-administered online questionnaire. Participants (n = 10) were adolescents with CD who had been diagnosed for over three years and were recruited from a tertiary pediatric gastroenterology center. Data included demographics, clinical characteristics, IMPACT-III (HRQOL), and PROMIS short forms. Open-ended responses underwent thematic analysis using the framework developed by Braun and Clarke. Results: Participants (80% female, median age 16.2 years, median disease duration 4.6 years) were all in clinical remission (median PCDAI = 2) and with good quality of life (median IMPACT-III = 80.7). Six themes emerged: (1) disease knowledge, (2) emotional responses, (3) coping and adaptation, (4) social support, (5) daily life and school impact, and (6) transition to adult care. Most participants demonstrated strong disease literacy and reported effective coping strategies. Emotional responses to diagnosis ranged from relief (60%) to distress (40%); relapses commonly triggered anxiety and fear. Therapeutic changes and disease monitoring were perceived as beneficial (100%) but with concern. Diagnostic procedures were viewed as burdensome by 70% of respondents. School performance and extracurricular participation were negatively affected in 40% during flares. Concerns regarding the future were reported by 40% of participants, with 30% believing that CD might limit life aspirations. While 60% managed their disease independently, 30% relied on parental support. All acknowledged the need for transition to adult care, though readiness varied. Conclusions: This study illustrates the overall impact of disease on pediatric CD patients. It reports significant emotional challenges and difficulties, as well as an impact on daily life, despite good disease knowledge. The findings underscore the importance of psychosocial well-being, ongoing mental health assessment, non-invasive monitoring, and holistic care, emphasizing the patient perspective, in managing pediatric CD.

Introduction and objectives: Pediatric inflammatory Bowel Disease (IBD) patients often face challenges in coping and disease management, which can impact self-management. This study investigates these patients' conceptualization of the disease and motivations for self-management. Methods: Descriptive cross-sectional study using a self-administered, semi-structured online questionnaire applied to pediatric Crohn’s Disease (CD) patients diagnosed>3 years, aiming to assess the impact of living with CD. Results: 10 patients included, 80% female, mean age 15.4 years, mean disease duration of 5.2 years. All patients were in remission with mean PCDAI of 3 (±4.8) and a mean IMPACT III score of 81.1). All patients reported good disease knowledge and adequate coping. Emotional responses at diagnosis included relief (60%) and negative emotions (40%). During relapses, anxiety and fear were prevalent, with 40% struggling with relapses. Therapeutic changes and monitoring were viewed as beneficial (100%) but with concern; 70% found monitoring tests a negative experience. Daily life impact was reported by 20%, with school accomplishments affected during relapses (40%). Extracurricular activities were limited during active disease (40%). Concerns about the future were noted by 40%, with 30% believing CD might limit their future. Most patients described appropriate self-efficacy: 60% self-managed, while 30% relied on parents. Transition to adult care was deemed necessary. Conclusion: This study illustrates the overall impact of disease on pediatric CD patients. It reports significant emotional and daily life challenges. The findings underscore the importance of psychosocial well-being, ongoing mental health assessment, non-invasive monitoring, and holistic care, emphasizing the patient perspective in managing pediatric CD.

Background: This study aims to evaluate and compare the perspectives of pediatric Crohn’s disease (CD) patients and their parents/caregivers concerning global physical, emotional, and social health as well as health-related quality of life (HRQQL), using both the Patient-Reported Outcomes Measurement Information System (PROMIS) and the IMPACT III questionnaire. Methods: In a cross-sectional study, 31 dyads of pediatric CD patients (aged 8–17 years) and their parents/caregivers were recruited from an outpatient Pediatric Gastroenterology Center. Participants completed PROMIS (Global Health, Depressive Symptoms, Anxiety, Meaning and Purpose Pain Interference Life Satisfaction, Peer Relationships, Physical Activity and Fatigue) and IMPACT III measures. Comparative analyses using t-tests and multivariate analyses assessed the impact of demographic factors on score differences. Cohen’s Kappa analysis evaluated the alignment between parent and child perceptions of disease status. Results: The sample comprised 58% females with a mean age of 15.2 (± 2) years and a mean disease duration of 2.7 (± 2.7) years. Most patients were in disease remission (83.9%) and perceived their disease as better or unchanged in the past 6 months. Concerning PROMIS scores, parents reported significantly lower global health scores (p < 0.001) and higher meaning and purpose scores (p = 0.029) compared to their children. Parental education and professional status significantly influenced PROMIS score differences. Specifically, mothers with specialized professions showed smaller differences in PROMIS depression and pain interference, although greater differences in PROMIS meaning and purpose, as compared to their respective children’s scores. Fathers with specialized professions demonstrated greater differences in PROMIS anxiety scores but smaller differences in PROMIS life satisfaction scores. A significant misalignment between parent and child subjective perceptions of disease status was observed (p = 0.004), suggesting that parents may overestimate symptom severity or underestimate improvements compared to their children’s experiences. Conclusion: This study highlights the importance of integrating patient and parental perspectives in the clinical management of pediatric CD. The observed discrepancies in disease-related perceptions, influenced by parental educational and professional background, underscore the need for comprehensive assessments to ensure accurate, patient-centered care. For broader generalization, further research should explore these dynamics in newly diagnosed and hospitalized patients.

Background: Inflammatory bowel disease (IBD) may adversely affect physical, psychological, and social well-being. Integrating patient-reported outcomes (PROs) into clinical practice is crucial for comprehensive disease management.Objective: To evaluate the responsiveness and clinical utility of Patient-Reported Outcomes Measurement Information System (PROMIS) instruments, compared with standard clinical assessment tools in pediatric CD patients.Methods: A longitudinal, prospective study with 31 pediatric Crohn's disease (CD) patients aged 8–17 years recruited from a Pediatric Gastroenterology Unit. Data were collected at baseline and every 6 months over 18 months. PROMIS pediatric measures assessed PROs. Disease activity was evaluated using the pediatric Crohn's disease activity index (PCDAI) and clinical markers. IMPACT-III was also applied. Linear mixed-effects models (LMMs) and bivariate analyses were used to assess changes over time.Results: PROMIS Global Health scores showed significant improvement over time, indicating enhanced overall health perceptions among patients. Notable reductions were observed in PROMIS Pain Interference and Fatigue scores, indicating better physical health. PROMIS depression scores generally decreased, suggesting improved mental health. PCDAI scores, hemoglobin, and platelet count significantly changed and correlated with PROMIS measures. Globally, the study demonstrated significant and clinically relevant changes in multiple PROMIS measures, confirming their responsiveness to changes in disease activity.Conclusion: PROMIS instruments are clinically useful in managing pediatric CD, providing valuable insights into global health and quality of life. Integrating PROMIS measures into routine clinical practice may enhance disease management and treatment strategies for pediatric IBD patients.

This study evaluated the clinical utility of the Patient-Reported Outcomes Measurement Information System (PROMIS®) by comparing it with objective clinical data and validated health-related quality of life (HRQOL) measures in pediatric Crohn’s disease (CD) patients. Cross-sectional study. Pediatric CD patients (aged 8–17 years) were enrolled prospectively over eight months from an outpatient pediatric gastroenterology center. We assessed the associations between PROMIS® Pediatric short-form measures, demographic and disease-related data, global clinical assessments, and HRQOL measures. A subanalysis according to the PCDAI (remission versus active disease) was also conducted. Thirty-one patients (mean age: 15.3; 58% female) with a mean disease duration of 2.7 years were included; 80.6% were in remission or had mild disease. The PROMIS® score was significantly correlated with several factors: age was negatively correlated with the PROMIS® Global Health Scale (r=-0.399; p = 0.026) and Life Satisfaction (r=-0.359; p = 0.047); sex was associated with the PROMIS® Cognitive Function Scale (t = 2.20; p = 0.038), favoring males; and school level was inversely related to the PROMIS® Peer Relationships (F = 3.90; p = 0.003). Clinical assessments also revealed significant correlations between hemoglobin and PROMIS® Global Health (r = 0.356; p = 0.049) and pain interference (r=-0.360; p = 0.046) and between ferritin and PROMIS® Meaning and Purpose (r = 0.435; p = 0.016) and cognitive function (r = 0.450; p = 0.011). Disease activity assessments correlated significantly with multiple PROMIS® measures, with better scores in patients in remission. Treatment changes, particularly corticosteroid treatment, negatively impacted the PROMIS® Anxiety and Life Satisfaction scores. IMPACT-III scores correlated positively with PROMIS® Global Health, Meaning and Purpose, Life Satisfaction, and peer relationships scores and negatively with Depression, Anxiety, Pain interference, and Fatigue scores. Group analysis indicated better PROMIS® scores and HRQOL scores in remission than in active disease remission. Consistent with recent evidence, PROMIS® scores reliably reflect disease activity and HRQOL. The meaningful associations with clinical assessment and treatment efficacy reinforce the clinical relevance and utility of PROs in the patient-centered management of pediatric IBD and highlight the importance of self-reports as a gold standard tool for assessing health status.

Inflammatory bowel disease (IBD) is an immune-mediated chronic disease with a significant impact on quality of life. In pediatric patients, diagnosing and managing IBD is particularly challenging, and IBD often presents as a more severe and progressive disease. Patient-reported outcomes (PROs) are measures of treatment and disease management outcomes reported by patients and/or caregivers. These measures evaluate several aspects of disease management from the patient/caregiver perspective, emphasizing the patient’s real-life experience with the disease and its treatment. PROs represent a model of patient-centered care, facilitating better-informed healthcare decisions. The Patient-Reported Outcomes Measurement Information System (PROMIS) was developed to promote the use of PROs among patients with chronic conditions. Its primary objective is to provide PROs for research and clinical practice throughout the lifespan. The PROMIS is a non-disease-specific instrument for both adults and pediatric patients assessing domains of physical, psychological, and social health, as well as quality of life (QOL). These instruments are designed to be applicable to a wide range of chronic diseases. Despite the initial expectation concerning PROs in assessing pediatric IBD outcomes, objective data in this area have only recently begun to emerge. This narrative review, based on a selection of reliable articles recognized by PubMed and Cochrane Library, aimed to identify and summarize previously published evidence of the usefulness of PROs, particularly the PROMIS, in IBD patients and in the pediatric population. We present an updated perspective, including identification of their general applications and most relevant previous studies, in the mentioned areas and identify knowledge gaps.

Inflammatory bowel disease (IBD) is an immune-mediated chronic disease with a significant impact on quality of life. In pediatric patients, diagnosing and managing IBD is particularly challenging, and IBD often presents as a more severe and progressive disease. Patient-reported outcomes (PROs) are measures of treatment and disease management outcomes reported by patients and/or caregivers. These measures evaluate several aspects of disease management from the patient/caregiver perspective, emphasizing the patient’s real-life experience with the disease and its treatment. PROs represent a model of patient-centered care, facilitating better-informed healthcare decisions. The Patient-Reported Outcomes Measurement Information System (PROMIS) was developed to promote the use of PROs among patients with chronic conditions. Its primary objective is to provide PROs for research and clinical practice throughout the lifespan. The PROMIS includes no disease-specific instruments for both adults and pediatric patients assessing domains of physical, psychological and social health, as well as quality of life (QoL). These instruments are designed to be applicable to a wide range of chronic diseases. Despite the initial expectation concerning PROs in assessing pediatric IBD outcomes, objective data in this area have only recently begun to emerge. This narrative review, based on a selection of reliable articles recognized by PubMed and Cochrane Library, aimed to identify and summarize previously published evidence of the usefulness of PROs, particularly PROMIS, in IBD patients and in the pediatric population. We present an updated perspective, including identification of their general applications and most relevant previous studies, in the mentioned areas and identifying knowledge gaps.

Anemia is a common complication of inflammatory bowel disease (IBD), usually recognized at diagnosis and during flare‐ups. However, the exact prevalence of anemia associated to IBD (IBD‐A) is unknown. Despite its major clinical relevance and quality of life impact in both adult and pediatric IBD patients, it has been for long time neglected. It is mostly multifactorial, being a unique example of the combination of chronic iron deficiency (ID) and anemia of chronic disease (ACD). The current management of IBD‐A represents a paradigm shift in clinical practice, involving several challenges. A pro‐active approach is recommended and with the new generation of available iron compounds and recent guidelines, the ultimate goal will be the improvement of the patients’ quality of life. Sound data are still lacking, concerning the best treatment/prevention approach for IDA/ID. Based on current evidence, oral iron therapy might be preferred in mild IDA, whereas intravenous iron may be advantageous in more severe IDA/flaring IBD. Long‐term prospective clinical trials are needed, to optimize treatment schedule and to better define the clinical and hematological long‐term outcomes, both in adults and in children. They should demonstrate the efficacy, safety, and tolerance profile of different available iron formulas, as well as their cost‐efficacy ratio.

Background and aims: Increasing evidence in adults demonstrates efficacy and safety of IV iron in inflammatory Bowel disease (IBD) associated iron deficiency anemia; however, evidence in pediatric patients is yet scarce and no previous study has included a long follow-up. This study aimed to evaluate safety and efficacy of IV iron (primary end point), and the need of re-treatment (secondary end point), in this setting. Methods: Prospective recruitment (40 months); PCDAI determined before and after treatment; anemia defined according to WHO criteria; IV iron treatment included iron sucrose and ferric carboxymaltose. Primary and secondary endpoints included hemoglobin, serum ferritin, transferrin saturation at baseline and 4-6 weeks after treatment; and the need of re-treatment during the median follow-up period (18 months), respectively. Results: Nineteen patients (median age: 15.5 years) with remissive/mild disease were included. At recruitment, the median hemoglobin was 10.5 g/dl, (median s-ferritin: 20.1 ug/l, median transferrin saturation; 6%) and 4-6 weeks after treatment was 12.7 g/dl. Median hemoglobin according to age groups before vs. after treatment: <12 years:11 vs. 12.0 g/dl; females ≥12 years:9.9 vs. 12.6 g/dl; and males ≥12 years:11.1 vs. 13.3 g/dl. Patients with remissive vs. mild disease had median Hb of 10.5 g/dl vs. 10.6 g/dl, and median s-ferritin: 6.8 ug/dl vs. 43.3 ug/dl, respectively). Nine patients were treated with iron sucrose (median dose 672.6 mg/dl) and 10 patients with ferric carboxymaltose (median dose 811.5 mg/dl). No major adverse reactions occurred. Six patients needed re-treatment after a median 15.5 months period. Conclusions: Our prospective study, concerning pediatric IBD anemia patients with remission/mild disease and a significant follow-up, emphasizes efficacy and safety of IV-iron and the importance of long-term follow-up of iron status. Summary: In pediatric IBD iron anemia, the evidence supporting the efficacy and safety of IV-iron is scare. This prospective study aims to evaluate the safety and efficacy (short and long term) of IV-iron in these patients. Nineteen pediatric CD patients were evaluated before and after IV iron treatment (40-month period).The median Hb before and after IV iron was 10.5 and 12.7 g/dl, respectively. No major adverse reactions were documented. Six patients needed re-treatment (median period of 15.5 months). This study further demonstrates the efficacy and safety of IV iron. It reinforces the importance of long-term follow-up of the iron status in pediatric CD patients.