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Clinical usefulness of patient-reported-outcome-measurement information system in Pediatric Crohn’s Disease: a cross-sectional study

Health and Quality of Life Outcomes volume 22, Article number: 112 (2024) Cite this article

Abstract

Objectives

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.

Study design

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.

Results

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.

Conclusion

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.

Introduction

The incidence and prevalence of pediatric-onset IBD are increasing globally [1,2,3], with approximately 10–25% of patients being diagnosed during childhood or adolescence [1, 4,5,6]. Early-onset IBD (< 10 years) encompasses a longer period of life with a chronic disease and typically has a more complicated and extensive course than diagnosed in adulthood [7,8,9]. Recent advancements in the management of pediatric CD patients have aimed to improve disease control [9,10,11] by focusing on mucosal and transmural healing [10], and HRQOL and disability are recognized as long-term treatment targets according to the STRIDE II (Selecting Therapeutic Targets in Inflammatory Bowel Disease) study, which also emphasizes the development of patient-reported outcomes (PROs) as essential tools for clinical practice and future research [11].

PROs are health experiences and evaluations that are assessed by patients and/or caregivers [12] and represent measures of treatment outcomes and disease management [6, 12,13,14,15]. They are becoming important tools in healthcare management, driving healthcare quality [13,14,15,16]. The PRO Measurement Information System (PROMIS®) has developed several pediatric self-reported item banks for individuals aged 8–17 years across five general health domains (physical function, pain, fatigue, emotional health, and social health). The use of PROMIS® in pediatric IBD has emerged [17, 18], as PROMIS® scores are significantly associated with CD/UC activity in a linear and clinically meaningful manner; however, most studies included internet-based cohorts and were not performed in a real clinical setting [17,18,19].

This study aimed to assess whether the PROMIS® instrument reflects global health status similarly to current clinical assessment tools (anchor assessments of disease) in a clinical setting. Specifically, the objective of this study was to evaluate the correlation between PROMIS® and current clinical assessment tools, including clinical assessments, laboratory tests, endoscopy/histology/imaging, and activity scores. Additionally, the present study aimed to evaluate the associations between PROMIS® scores and HRQOL, sociodemographic data, and medical history, including patient self-perception of disease.

Methods

Study population

This cross-sectional study had a prospective recruitment period of eight months (January to August 21) and included all pediatric patients with CD (aged 8–17 years) who provided consent or consent to participate. Patients were consecutively enrolled from an outpatient setting at a single reference center of pediatric gastroenterology during their scheduled appointments. Patients were stratified into two groups based on the Pediatric Crohn’s Disease Activity Index (PCDAI) [20, 21]: remission group, PCDAI < 10 (G1); and active disease group, PCDAI > 10 (G2).An analysis comparing patients with shorter (> 1 month to diagnosis) and longer (< 1 month to diagnosis) time to diagnosis, was also performed.

Informed assent (patients < 16 years) and consent (patients > 16 years and caregivers of patients < 16 years) were obtained prior to enrollment. The exclusion criteria included unwillingness to participate, limitations in verbal or written comprehension of Portuguese, recent diagnosis of IBD (< 1 month), and hospitalization.

Data collection

All the data were collected during the clinical assessment of the patients at the scheduled appointment. The questionnaires were completed in the waiting room before medical appointments or during treatment infusions. Clinical and demographic data, including academic and extracurricular/social activity data, were collected from patient records and completed a brief questionnaire. The collected data included demographic data [sex, birth date, age at diagnosis, school level according to the International Standard Classification of Education [22] (ISCED 2011) and extracurricular activities] and disease-related data [time of diagnosis, years of disease, disease phenotype [23] (Paris Classification), need for hospitalization/surgery, induction of remission treatment, current treatment, treatment compliance and need for treatment escalation]. Compliance with oral treatment was directly inquired of patients and their parents whether there had been any forgetfulness or missed treatments lasting more than one week in the preceding six months. Compliance with biological treatment was monitored by the hospital pharmacy.

Anchor assessments of disease

Disease activity scores included the PCDAI at diagnosis and recruitment (patients scoring < 10 considered in remission), the Simple Endoscopic Score for Crohn’s Disease (SES-CD) [24] from endoscopic assessment within a 6–12 month interval, abdominal magnetic resonance imaging (A-MRI) within a 3–6 month interval and laboratory test data (hemoglobin, erythrocyte sedimentation rate, serum C-reactive protein, serum ferritin, and fecal calprotectin) from the appointment day.

Clinical physician global assessment of disease

At the scheduled visit, the responsible physician provided an assessment of current treatment and disease severity categorizing it as remissive, mild, moderate, and severe disease, based on the presence of symptoms/signs of active disease (abdominal pain, diarrhea, bloody stools, weight loss, fatigue, impairment of normal activities, abdominal tenderness or mass, fistula, toxic appearance), need of treatment escalation, corticosteroid use, hospitalization (within a 6 months interval), treatment compliance and missed appointments.

The variables represent a composite clinical score to reflect the regular clinical observation of these patients. This score was based on clinical studies on pediatric and adult IBD patients [25, 26].

Measure of health-related quality of life and PROs

HRQOL was measured using an Impact III [27]. The IMPACT-III is a 35-item self-report, IBD-specific measure, with lower scores indicating poorer HRQOL. The PROMIS® included 10 short forms for evaluating significant PROs in IBD patients: global health, meaning and purpose, cognitive function, life satisfaction, peer relationships, depression, anxiety, pain interference, physical activity, and fatigue. In this study, short forms were used, including a fixed set of 4–10 items or questions for each domain-short form. The PROMIS® instrument was calibrated using a T score metric, with the mean of the original calibration population equal to 50 and the standard deviation (SD) in the calibration population equal to 10 [22].

Patient self-perception of disease status

A semiquantitative scale was applied to patients: “feeling better/good”, “feeling the same/not (god) good not bad”, or “feeling bad/worse”.

Statistical analyses

Descriptive statistics were calculated, including means and standard deviations, as well as absolute and relative frequencies. To assess the possible relationships between the PROMIS® score and standard assessment tools, sociodemographic data and other variables, we conducted bivariate analysis. Spearman’s rank correlation and Pearson’s correlation were employed to examine correlations between variables, while independent samples t tests and one-way ANOVA, or their nonparametric equivalents, were used when comparing groups, as appropriate. All analyses were conducted using R version 4.3.0 (R Core Team 2023), and statistical significance was assessed at a threshold of α = 0.05.

Results

Description of the sample

The study included 31 participants with a mean age of 15.3 ± 2 years and 58% females (n = 18). The mean age at CD diagnosis was 12.7 (± 3.4) years, and the mean disease duration was 2.7 (± 2.7). Complete demographic and clinical data are reported in Table 1.

Table 1 Demographic and clinical data of the study population
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Table 2 reports the global assessment of disease at enrollment. Most of the participants were in clinical remission or had mild active disease [n = 25 (80.6%)]. Patient self-perception of disease was consistent with these results, as 28 (90.3%) patients reported “feeling better or the same”. Complete biochemical, SES-CD score and imagological A-MRI data are shown in Table 2.

Table 2 Global assessment of disease incidence
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Patients reported good HRQOL [mean IMPACT III 73,2(± 13.1)]. The mean (SD) PROMIS® score (Table 2), however, indicated poor global health, poor physical health, and good social health [Peer relationship 50.6 (9.5)].

PROMIS® scores and associations

Demographic data

Age was negatively correlated with the PROMIS® Global Health Scale (r=-0.399; p = 0.026) and Life Satisfaction Scale (r=-0.359; p = 0.047) (Table 3). Sex was associated with PROMIS® Cognitive Function (t = 2.20; p = 0.038), favoring males. School level was inversely associated with the PROMIS® Peer Relationships Scale (F = 3.90; p = 0.003).

Clinical anchor assessments of disease

Some significant associations were found with laboratory test results, where Hb was correlated with PROMIS® global health (r = 0.356; p = 0.049) and pain interference (r=-0-360; p = 0.046) (Table 3). The serum ferritin concentration was correlated with the PROMIS® Meaning and Purpose score (r = 0.435; p = 0.016) and cognitive function (r = 0.450; p = 0.011). No significant correlations were found with the calprotectin concentration, ESR, C-RP, PCDAI or PROMIS® score.

Clinical physician global assessment of disease and patient self-perception of disease status

The Clinical Physician Global Assessment of Disease was significantly associated with the PROMIS® Global Health score (F = 3.64, p = 0.039), where patients in remission had higher scores than those with mild disease. An association with PROMIS® Meaning and Purpose (F = 4.74, p = 0.002) was also found, with patients in remission having higher scores than those with mild illness, and with Pain Interference (F = 9.88, p = 0.007), with patients classified by their physician as in remission being found to have lower pain scores than those with mild illness.

Patient self-perception of disease was significantly associated with PROMIS® Global Health (F = 7.35, p = 0.003), with patients who perceived improvements showing higher scores than those classified as having unchanged disease; cognitive function (F = 5.43, p = 0.025), with patients perceiving their disease as worsening having lower cognitive scores than those perceiving improvements; and PROMIS® Fatigue (F = 5.17; p = 0.012) and Life Satisfaction (F = 5.83, p = 0.008). Patients who reported no changes in their disease had lower life satisfaction and lower fatigue scores than those who reported improvements.

Treatment and treatment modifications

Moreover, PROMIS® Anxiety was associated with recent treatment changes and corticosteroid treatment. PROMIS® Cognitive Function and Life Satisfaction were also associated with treatment, with corticosteroid use negatively impacting emotional health and self-perception of disease.

Patients who had changed treatment during the visit before enrollment (within 3 months) had higher PROMIS® anxiety scores (t = 3.60; p = 0.037), and patients who had received corticosteroid treatment (t = 4.63; p = 0.001) also had higher scores. Moreover, PROMIS® Cognitive Function was also associated with treatment (F = 2.833; p = 0.045). PROMIS® Life Satisfaction was associated with corticosteroid treatment (t=-4.07; p < 0.001), and corticosteroid treatment was associated with lower life satisfaction and self-perception (F = 5.83, p = 0.008).

The PROMIS® pain interference score was correlated with the number of biologics used for treatment until T0 (r = 0.40, p = 0.026), and the number of treatments changed until T0 (r=-0.43, p = 0.002) (Table 3).

IMPACT III correlations

The IMPACT-III score was positively correlated (Table 3) with the PROMIS® Global Health Scale (r = 0.687; p < 0.001), Meaning and Purpose (r = 0.430; p = 0.016), Life Satisfaction (r = 0.709; p < 0.001), and Peer Relationships (r = 0.375; p = 0.038). Negative correlations were found with PROMIS® Depression (r=-0.725; p < 0.001), Anxiety (r=-0.486; p = 0.006), Pain Interference (r=-0.544; p = 0.002), and Fatigue (r=-0.566; p = 0.001).

No statistically significant correlation was found between total IMPACT-III and PROMIS® Cognitive (r = 0.318, p = 0.082) or Physical Activity (r=-0.129, p = 0.488).

Table 3 Correlation coefficients between PROMIS measures and clinical/laboratory variables
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Group stratification by disease activity status

Table 4 presents the results according to the group stratification by disease activity. The clinical and demographic details of both groups are listed in Table 4.

Table 4 Demographic and clinical characteristics of disease and global assessment by disease activity
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Anchor assessments of disease activity were the worst in G2, reflecting poor disease control and the presence of inflammation, although these findings were not statistically significant. The serum ALB concentration (t = 3.364, p = 0.002), hematocrit level (t = 2.467, p = 0.020), number of biological treatments (t= -2.566, p = 0.016), and number of surgeries (t = 3.035, p = 0.006) before enrollment were all significantly associated with disease status.

The clinical assessment of disease was found to be significantly associated with disease status (p < 0.001). Patient self-perception of disease status revealed that 66.6% of patients in G2 considered the disease to be the same or better compared with the previous 6 months.

The mean IMPACT III I score was greater in G1 (77.1) than in G2 (65), reflecting differences in HRQOL.

Overall, compared with those in G2, the PROMIS® scores (Table 4) in G1 were greater; PROMIS® scores, which reflect better health (global health, meaning and purpose, life satisfaction, cognitive function, and peer relationship); and lower PROMIS® scores, which reflect worse health (depression, anxiety, pain interference, fatigue, physical activity); however, these two scores were not significantly different.

Remission group (G1, n = 26/31)

Several significant results were found for G1, all consistent with better disease control and better general health and well-being, according to the PROMIS® scores and its correlation with demographic and anchor assessments of disease.

Years of diagnosis were strongly correlated with PROMIS® Life Satisfaction score (r = 0.936, p = 0.006), and age at diagnosis was strongly negatively correlated with this measure (r = -0.845, p = 0.034).

Concerning laboratory indicators of active disease, a positive correlation was found with Hb and PROMIS® Global Health (r = 0.514, p = 0.009) and Life Satisfaction (r = 0.512, p = 0.009), and a negative correlation was found with PROMIS® Fatigue (r = -0.486, p = 0.014). Calprotectin was negatively correlated with PROMIS® Fatigue (r= -0.863, p = 0.027), and C-RP was correlated with PROMIS® Life Satisfaction (r = 0.812, p = 0.049). The PCDAI was positively correlated with the PROMIS® Global Health Scale (r = 0.961, p = 0.002) and the Meaning and Purpose scale (r = 0.834, p = 0.039).

The clinical assessment of disease was positively associated with the PROMIS® Global Health Scale (t = 3.559, p = 0.011) and negatively associated with the PROMIS® Depression Scale (W = 2, p = 0.009) and Pain Interference Scale (W = 6.5, p = 0.021).

Patient self-perception of disease was also correlated with PROMIS® Global Health (F = 7.654, p = 0.003), Life Satisfaction (F = 9.661, p = 0.001), and Fatigue (F = 6.41, p = 0.006).

Finally, correlations were observed between the IMPACT-III score and all the PROMIS® scores, except for physical activity.

IMPACT-III was negatively associated with PROMIS® anxiety (r= -0.865, p = 0.026).

Active disease group (G2, n = 5/31)

In G2, significant associations were also found, although the anchor measures were not always correlated with the same PROMIS® measures as in G1.

Sex and PROMIS® Depression score were significantly associated (t= -3.309, p = 0.030), with male patients scoring lower on the PROMIS® Depression Scale. Extracurricular activities were positively associated with PROMIS® Meaning and Purpose scores (t = 5.580, p = 0.008).

Like in G1, years of diagnosis were positively associated with PROMIS® Life Satisfaction (r = 0.936, p = 0.006), and age at diagnosis was negatively associated with Life Satisfaction (r= -0.845, p = 0.036).

Correlations were found with laboratory markers of inflammation: positive correlations were found with serum ferritin and PROMIS® global health (r = 0.951, p = 0.003) and with R-CP and PROMIS® life satisfaction (r = 0.812, p = 0.049), and negative correlations were found with calprotectin and PROMIS® fatigue (r= -0.863, p = 0.027).

The PCDAI was correlated with the PROMIS® Meaning and Purpose (r = 0.834, p = 0.039) and PROMIS® Global Health (r = 0.961, p = 0.002) scores.

The SES-CDs were associated with the PROMIS® Meaning and Purpose Scale (F = 15126.333, p = 0.006) and the PROMIS® Pain Interference Scale (F = 445.885, p = 0.035).

Regarding the clinical assessment of disease, a significant correlation was observed only with the PROMIS® Meaning and Purpose score (t= -5.580, p = 0.008). A significant negative relationship was observed between patient self-perception and PROMIS® Cognition score (F = 33.809, p = 0.009).

The final IMPACT-III score was significantly and negatively correlated with the PROMIS® Anxiety score (r = -0.865, p = 0.026), with patients having lower IMPACT-III scores experiencing greater PROMIS® Anxiety.

Group stratification by time to diagnosis

When analyzing the comparison between patients with shorter and longer time to diagnosis, we found that there was no statistically significant evidence of differences in all laboratory measures, in the IMPACT-III score, and in most PROMIS measures, except for PROMIS Anxiety (p = 0.016), and PROMIS Depression (p = 0.036) where patients with longer time to diagnosis have lower anxiety and depression scores.”

Discussion

Pediatric IBD patients also experience several limitations in daily activities and restrictions in school, professional, and social interactions [28, 29]. Impaired HRQOL, which has significant impacts on physical, psychological, and autonomy domains linked to disease activity, is well documented [3, 14, 18, 28,29,30,31] in both the short and long term [14].

Healthcare recommendations for improving care in pediatric IBD patients are increasingly comprehensive, with a focus on patient-reported data to assess physical and psychosocial functioning and well-being [10, 11].

This study evaluated the correlations and associations of several short-form pediatric PROMIS® measures with current clinical tools used in the real clinical setting for managing pediatric IBD, referred to as anchor assessments of disease, including laboratory biomarkers of inflammation, the SES-CD score, imaging data, the PCDAI, clinical assessment of disease, and the IBD-specific HRQOL tool (IMPACT III). The study also included patient self-perception of disease, sociodemographic data, and past clinical history related to CD in the association analysis.

Consistent with the findings of previous studies of pediatric chronic conditions, including IBD [17, 18, 32], we found that the mean PROMIS® score was related to disease activity and HRQOL. In our research, sex, patient age, and age at diagnosis were significantly associated with overall mental and social health. In patients in remission, disease duration and age at diagnosis were positively correlated with emotional health. These findings may be related to the experience of living with a chronic condition and the patient’s family/social support. School may act as a protective factor in social health, as significant associations were found between school level and peer relationships.

Our results align with the established knowledge of coping strategies for pediatric CD [28, 29], where younger patients and those with more severe disease at presentation and during follow-up may have more difficulty adapting to a CD diagnosis and may experience psychological comorbidities [33, 34].

Although not all PROMIS® scores were consistently associated with all-anchor disease scores, most selected PROMIS® measures showed associations with the overall anchor assessments of pediatric CD in a linear manner, consistent with clinical experience. This reflects the usefulness and reliability of PROs in a clinical setting and highlights the importance of evaluating general health domains (physical function, pain, fatigue, emotional health, social health) in daily clinical decisions and driving healthcare quality [13,14,15,16].

In our study, PROMIS® scores reflecting better health were associated with better laboratory (Hb and serum ferritin) and endoscopic (SES-CD) scores, clinical perceptions of disease remission, and self-reported perceptions of disease (patients perceiving improvements had higher scores than did those with unchanged disease). Concerning laboratory markers of inflammation, correlations between fecal calprotectin and CRP levels were only found between PROMIS® score and global and physical health in patients with active disease. The correlations between Hb and serum ferritin with global, mental, and physical health, irrespective of disease activity and time to diagnosis, suggest that anemia may be a significant and independent factor adversely impacting these patients’ overall health and HRQOL [35]. Furthermore, the results underscore the importance of closely monitoring biochemical markers of inflammation, even in asymptomatic patients [11].

Interestingly, the correlations of PROMIS® scores with PCDAI scores were not entirely consistent with those of previous studies, where PROMIS® scores were significantly associated with self-reported SCDAI scores in a linear and clinically meaningful manner [17]. This discrepancy may be because the study population consisted primarily of patients with a disease largely in remission.

A novel finding in our study was the agreement between the clinical physician global assessment, which represents real-world clinical assessment, and the health outcomes reported by the PROMIS® scores. Better health in all assessed PROMIS® domains was associated with better-controlled disease. Additionally, patient self-perception of disease showed similar associations, with those perceiving improvements having higher scores reflecting better health in all but social domains. Patients who perceived their disease as worsening had the most significant impairment in physical health. These findings underscore the relevance and accuracy of the patient perspective [36,37,38].

The HRQOL data obtained in the present study were consistent with published data [17,18,19], with strong and expected correlations between the IMPACT-III score and several PROMIS® measures. However, PROMIS® Cognitive Function and Physical Activity did not correlate with HRQOL. This may be due to the characteristics of our study population, which consisted of patients with relatively well-controlled disease. Physical Activity was expected to correlate with HRQOL and disease activity, as previously reported in other studies using PROMIS pediatric measures [18, 19, 32, 39, 40]. However, no other study using PROMIS in IBD patients has specifically applied this measure to evaluate physical health, suggesting that physical activity may be less affected in IBD patients than in other conditions. A comparison may be drawn with the study of Arvanitis et al. [17], where changes in Crohn’s disease activity and HRQOL were not associated with differences in PROMIS Peer Relationships except when the disease was very active or QOL was very impaired. This result should be interpreted cautiously, considering the small number of patients with active disease.

The correlations of the PROMIS® score with treatment and treatment experience were interesting findings in our study. We found that several treatment options (corticosteroids), treatment changes, and the number of treatment changes adversely affect individuals’ emotional health and self-perception of disease. Corticosteroids, as expected, had the most significant negative impact on emotional health, with patients presenting high anxiety and low life satisfaction scores. Similar data regarding the negative impact of corticosteroids on several health domains evaluated by the PROMIS® were described by Kappleman et al. [16]in a cross-sectional study of an adult IBD patient web-based cohort.

Our study’s results align with the management recommendations of pediatric IBD [9, 10, 41], emphasizing the importance of involving patients in the decision-making process regarding the best treatment options to achieve mucosal healing [11]. These findings support the usefulness of including the patient’s perspective in healthcare to provide more patient-centered care.

Self-reports are associated with better health status and self-management [12, 37, 38, 42] and are recognized as the gold standard tool for assessing individual health status during clinical visits [12]. The STRIDE II [11] recommends frequent assessment of well-being domains during the disease course, independent of objective markers of inflammation.

The PROMIS® instrument has consistently proven effective in measuring QOL, physical function, and psychological and social health in patients with IBD, particularly in the pediatric setting and from a patient-centered perspective. Our study further reinforces the usefulness of the PRO assessment as a valid and important tool for managing pediatric IBD.

There are several strengths to our study. We performed a comprehensive analysis integrating current validated tools for managing pediatric CD and assessing the ability of the PROMIS® to reflect patient disease and well-being (responsiveness). Our study underscores its validity and feasibility as an everyday clinical tool. We used a broader set of PROMIS® measures encompassing all health domains (global, physical, mental, and social) to better assess the usefulness of PROs, not only compared to standard clinical evaluation but also for detailed evaluations of patients’ general well-being. Another strength is the inclusion of both doctors’ assessments and patients’ subjective perceptions, which yielded good correlations with the other tools. Most prior studies using PROMIS® in pediatric IBD patients were not performed in real-world scenarios, where time-consuming questionnaire applications are less feasible. In our study, we used short-form PROMIS® measures with lower respondent burdens, increasing their applicability in clinical settings.

However, our study has several limitations. The sample size was small and was primarily composed of well-controlled patients with low disease activity/remission, limiting generalization but demonstrating the potential discriminatory value of certain tools even in the absence of active disease. The cross-sectional nature of the study is another limitation, as a prospective analysis is more appropriate for dynamically assessing disease course and its impact on patient health. Additionally, clinical assessments were always conducted by the same researcher, although integrating objective and validated clinical data helped limit this bias.

Conclusion

Despite the limitations of our study, our preliminary findings reinforce the validity and feasibility of the PROMIS® in a real-world clinical setting. Consistent with previously published data, this study demonstrated that PROMIS® scores reliably correlate with disease activity and HRQOL. Additionally, PROMIS® scores showed meaningful associations with clinical subjective assessments and treatment experiences, highlighting its utility as a reliable marker of disease assessment and treatment efficacy.

Our findings underscore the importance of incorporating PROs into the management of pediatric IBD, emphasizing the impact of disease management on emotional well-being, social functioning, and overall HRQOL. Furthermore, the study highlights the necessity of considering patient perspectives in treatment decision-making processes to optimize clinical outcomes.

This study advocates for a patient-centered approach to pediatric CD care, ensuring comprehensive and effective management strategies. By integrating patient-reported outcomes, healthcare providers can better understand and address the multifaceted aspects of pediatric IBD, ultimately improving the quality of life for young patients.

In conclusion, the PROMIS® instrument has proven to be an effective, valid, and reliable tool for assessing quality of life, physical function, psychological health, and social health in pediatric IBD patients. Their integration into routine clinical practice can enhance disease monitoring and patient care, supporting a holistic approach to managing chronic conditions in pediatric populations.

Data availability

No datasets were generated or analysed during the current study.

Abbreviations

A-MRI:

Abdominal magnetic resonance imaging

CD:

Crohn’s disease

C-RP:

C-reactive protein

ESR:

the erythrocyte sedimentation rate

Hb:

hemoglobin

HRQOL:

health-related quality of life

IBD:

inflammatory bowel disease

ISCED:

the International Standard Classification of Education

PROs:

Patient Reported Outcomes

PROMIS®:

the Patient-Reported Outcome-Measurement Information System

PCDAI:

the Pediatric Crohn’s Disease Activity Index

QOL:

the Quality of Life

SES-CD:

Simple Endoscopic Score for Crohn’s Disease

STRIDE II:

Select Therapeutic Targets in Inflammatory Bowel Disease

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Authors and Affiliations

  1. Gastroenterology Unit, Pediatric Department, Santa Maria University Hospital - CHLN, Academic Medical Centre of Lisbon, Lisbon, Portugal

    Sara Azevedo & Ana Isabel Lopes

  2. Medical School, University of Lisbon, Lisbon, Portugal

    Sara Azevedo, Maria Miguel Oliveira, Paulo Nogueira & Ana Isabel Lopes

  3. Área Disciplinar Autónoma de Bioestatística (Laboratório de Biomatemática) Medical School, University of Lisbon, Lisbon, Portugal

    Maria Miguel Oliveira & Paulo Nogueira

  4. Laboratório Associado TERRA, Instituto de Saúde Ambiental. Medical School, University of Lisbon, Lisbon, Portugal

    Maria Miguel Oliveira & Paulo Nogueira

  5. Centro de Investigação, Inovação e Desenvolvimento em Enfermagem de Lisboa (CIDNUR), Escola Superior de Enfermagem de Lisboa, Lisbon, Portugal

    Paulo Nogueira

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Contributions

All authors read and approved the final manuscript. Conceptualization, data curation, investigation, methodology (SA), formal analysis (SA, MO and PN) supervision: (AIL), validation: (AIL and PN) The first draft of the manuscript was written by (SA) and all authors commented on previous versions of the manuscript.

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Correspondence to Sara Azevedo.

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Ethics approval and consent to participate

Ethics approval and consent to participate: The study was conducted in accordance with the ethical principles set forth in the Declaration of Helsinki of the World Medical Association and in accordance with the additional requirements set forth in the national legislation. Prior to the commencement of the study, ethical approval was obtained from the Ethical Committee of Santa Maria University Hospital - CHLN, Academic Medical Centre of Lisbon, Portugal.

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Informed consent to participate in the study and for publication was obtain prior to enrollment. All pediatric CD patients with 8–17 years of age signed an informed assent (< 16 years) and consent (patients with16 years). Informed consent was also obtain from all caregivers of all pediatric patients.

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The authors declare no competing interests.

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Azevedo, S., Oliveira, M.M., Nogueira, P. et al. Clinical usefulness of patient-reported-outcome-measurement information system in Pediatric Crohn’s Disease: a cross-sectional study. Health Qual Life Outcomes 22, 112 (2024). https://doiorg.publicaciones.saludcastillayleon.es/10.1186/s12955-024-02330-2

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Health and Quality of Life Outcomes

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