Pediatric IBD patients experience several also limitations in daily activities and restrictions in school, professional, and social interactions (26, 27). Impaired HRQOL, with significant impacts on physical, psychological, and autonomy domains linked to disease activity, is well document (3, 14, 18, 26–29), in both the short and long term (14).
Healthcare recommendations for improving care in pediatric IBD 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, SES-CD score, imaging data, PCDAI, clinical assessment of disease, and the IBD-specific HRQOL tool (IMPACT III). The study also included patient self-perception of disease, socio-demographic data, and past clinical history related to CD in the association analysis.
Consistent with previous studies in pediatric chronic conditions, including IBD (17, 18, 30), we found that mean PROMIS® measures are related to disease activity and HRQOL. In our research, gender, patients' age, and age at diagnosis were significantly associated with global 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(26, 27), 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(31, 32).
Although not all PROMIS® measures 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(13–16).
In our study, PROMIS® scores reflecting better health were associated with better laboratory (Hb and serum ferritin) and endoscopic scores (SES-CD), clinical perceptions of disease remission, and self-reported perceptions of disease (patients perceiving improvements showed higher scores than those with unchanged disease).
The correlation between PROMIS® and global and physical health and laboratory markers of inflammation, particularly fecal calprotectin and CRP in the subgroup of patients with active disease and a correlation Hb and serum ferritin and global, mental, and physical health, independent of disease activity, may suggest that anemia is an important, and independent factor affecting adversely patients and global health and HRQOL(33). This underscores the importance of closely monitoring biochemical markers of inflammation in asymptomatic patients(11).
Interestingly, PROMIS® correlations with PCDAI were not entirely consistent with previous studies, where PROMIS® scores were significantly associated with self-reported SCDAI in a linear and clinically meaningful manner(17). This discrepancy may be due to the study population consisting primarily of patients with a disease largely in remission.
A novel finding in our study was the agreement between clinical physician global assessment, representing real-world clinical assessment, and health outcomes reported by 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 perceiving their disease as worsening showed the most significant impairment in physical health. These findings underscore the relevance and accuracy of the patient perspective (34–36)
Regarding HRQOL the study results were consistent with published data(17–19), with strong and expected correlations between IMPACT-III scores 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, consisting of patients with relatively well-controlled disease.
In the Arvanitis et al. study(17), 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. Physical Activity was expected to correlate with HRQOL and disease activity, as previously reported in other studies using PROMIS® pediatric measures(18, 19, 30, 37, 38). However, no other study using PROMIS® in IBD patients has applied this measure to evaluate physical health specifically, suggesting that physical activity may be less affected in IBD patients than in other conditions. This result should be interpreted with caution, considering the small number of patients with active disease.
PROMIS® correlations with treatment and treatment experiences were interesting findings of our study. We found that some treatment options (corticosteroids), treatment changes, and the number of treatment changes adversely affected 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 corticosteroids' negative impact on several health domains evaluated by PROMIS® were described by by Kappleman et al. (16)in a cross-sectional study of an adult IBD patients web-based cohort.
Our study's results align with the management recommendations of pediatric IBD (9, 10, 39) emphasizing the importance of involving patients in the decision-making process regarding the best treatment options to achieve mucosal healing(11). These findings upport the usefulness of including the patient's perspective in healthcare to provide more patient-centered care.
Self-reporting is associated with better health status and self-management (12, 35, 36, 40) and is being recognized as a gold standard tool for assessing individual health status during clinical visits (12). STRIDE II (11) recommends frequent assessing of well-being domains during the disease course, independently of objective markers of inflammation.
PROMIS® instruments have consistently proven effective in measuring QOL, physical function, psychological and social health in IBD, particularly in the pediatric setting and from a patient-centered perspective. Our study further reinforces the usefulness of PRO assessment as a valid and important tool in 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 PROMIS®'s ability 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 doctor's assessments and patients' subjective perceptions, yielding good correlations with other tools. Most prior studies using PROMIS® in pediatric IBD 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 limitations. The sample size was small and primarily composed of well-controlled patients with low disease activity/remission, limiting generalization but demonstrating certain tools' potential discriminatory value even in the absence of active disease. The cross-sectional nature of the study is another limitation, with a prospective analysis being more appropriate for dynamically assessing disease course and its impact on patient health. Additionally, the clinical assessment was always conducted by the same researcher, though integrating objective and validated clinical data helped limit this bias.