PEDSpace
Welcome to PEDSpace, a public data bank repository powered by PEDSnet. PEDSpace serves as a centralized hub where digital assets generated during PEDSnet studies are made readily accessible to researchers, clinicians, and stakeholders worldwide.
In PEDSpace, users can explore a wealth of resources to facilitate impactful research endeavors. Among these assets are meticulously defined variables, curated code sets, and modules for assessing data quality. Each component is designed to empower researchers with the tools necessary to navigate complex pediatric healthcare data effectively.
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Explore PEDSpace Collections
Browse a comprehensive set of functions to aid in data quality assessment of clinical datasets.
Browse a comprehensive index of PEDSnet projects, their associated publications, and related research materials.
Browse documentation, ETL specifications, and resources for participating PEDSnet sites.
Resources for use in defining cohorts and other analytic tasks for investigating EHR data.
Recent Submissions
- Accurate and Automated Multi-Center TCD Velocity Analysis Using Natural Language Processing [Abstract]Affiliation:Children's Hospital of Philadelphia; Perelman School of Medicine at the University of Pennsylvania; Nemours Children's HealthUp to 10% of US children with sickle cell disease (SCD) develop abnormal transcranial Doppler (TCD) velocities, indicating abnormal cerebral vasculature and a high risk of stroke. Chronic red cell transfusions decrease stroke risk in patients with abnormal TCD. However, transfusions are resource-intensive and burdensome to patients, and they may not fully reverse existing damage. Prevention of TCD abnormalities is therefore ideal. The impact of real-world SCD care practices on TCD results is largely unknown due to an inability to detect TCD outcomes without labor-intensive manual review. We present a novel method for identification and interpretation of TCD results across multiple children's hospitals.
- High Body Mass Index and Response to Anti-Tumor Necrosis Factor Therapy in Pediatric Crohn's DiseaseCreated:2024-06Affiliation:University of Iowa; University of Alabama at Birmingham; Boston Children's Hospital; Nemour Children's Health; University of Oklahoma; University of Vermont; Ann & Robert Lurie Children's Hospital; Icahn School of Medicine at Mount Sinai; Yale University; SUNY Upstate Medical Center; Mott Children's Hospital; Stanford Medicine Children's Health; Riley Hospital for Children; University of North Carolina at Chapel Hill; Children's Hospital of The King's Daughters; Wright State University; Children's Hospital of Wisconsin; Cincinnati Children's Medical Center**Introduction:** Obesity is common among patients with pediatric Crohn's disease (PCD). Some adult studies suggest obese patients respond less well to anti-tumor necrosis factor (TNF) treatment. This study sought compares anti-TNF response and anti-TNF levels between pediatric patients with normal and high body mass index (BMI). **Methods:** The COMBINE trial compared anti-TNF monotherapy with combination therapy with methotrexate in patients with PCD. In this secondary analysis, a comparison of time-to-treatment failure among patients with normal BMI vs BMI Z -score >1, adjusting for prescribed anti-TNF (infliximab [IFX] or adalimumab [ADA]), trial treatment assignment (combination vs monotherapy), and relevant covariates. Median anti-TNF levels across BMI category was also examined. **Results:** Of 224 participants (162 IFX initiators and 62 ADA initiators), 111 (81%) had a normal BMI and 43 (19%) had a high BMI. High BMI was associated with treatment failure among ADA initiators (7/10 [70%] vs 12/52 [23%], hazard ratio 0.29, P = 0.007) but not IFX initiators. In addition, ADA-treated patients with a high BMI had lower ADA levels compared with those with normal BMI (median 5.8 vs 12.8 μg/mL, P = 0.02). IFX trough levels did not differ between BMI groups. **Discussion:** Overweight and obese patients with PCD are more likely to experience ADA treatment failure than those with normal BMI. Higher BMI was associated with lower drug trough levels. Standard ADA dosing may be insufficient for overweight children with PCD. Among IFX initiators, there was no observed difference in clinical outcomes or drug levels, perhaps due to weight-based dosing and/or greater use of proactive drug monitoring.
- Comparative Effectiveness of Anti-TNF in Combination With Low-Dose Methotrexate vs Anti-TNF Monotherapy in Pediatric Crohn's Disease: A Pragmatic Randomized TrialCreated:2023-07Affiliation:University of North Carolina at Chapel Hill; University of Michigan; Children's Hospital of The King's Daughters; Cincinnati Children's Hospital Medical Center; Lucile Packard Children's Hospital; Children's Mercy Kansas City; Nationwide Children's Hospital; Children's Hospital of Wisconsin; Esoterix Specialty Laboratory; Nemours Children's Health; University of Iowa; Progenika Biopharma, a Grifols Company; Children's Hospital of Philadelphia; University of Oklahoma; University of Alabama at Birmingham; Mass General for Children; Emory University; University of Nebraska Medical Center; Children's National Medical Center; Levine Children's Hospital; Saint Louis University School of Medicine; Yale University; Washington University School of Medicine; Wright State University; Riley Hospital for Children; The University of Vermont; State University of New York Upstate Medical University; Boston Children's Hospital**Background & Aims:** Tumor necrosis factor inhibitors, including infliximab and adalimumab, are a mainstay of pediatric Crohn's disease therapy; however, nonresponse and loss of response are common. As combination therapy with methotrexate may improve response, we performed a multicenter, randomized, double-blind, placebo-controlled pragmatic trial to compare tumor necrosis factor inhibitors with oral methotrexate to tumor necrosis factor inhibitor monotherapy. **Methods:** Patients with pediatric Crohn's disease initiating infliximab or adalimumab were randomized in 1:1 allocation to methotrexate or placebo and followed for 12-36 months. The primary outcome was a composite indicator of treatment failure. Secondary outcomes included anti-drug antibodies and patient-reported outcomes of pain interference and fatigue. Adverse events (AEs) and serious AEs (SAEs) were collected. **Results:** Of 297 participants (mean age, 13.9 years, 35% were female), 156 were assigned to methotrexate (110 infliximab initiators and 46 adalimumab initiators) and 141 to placebo (102 infliximab initiators and 39 adalimumab initiators). In the overall population, time to treatment failure did not differ by study arm (hazard ratio, 0.69; 95% CI, 0.45-1.05). Among infliximab initiators, there were no differences between combination and monotherapy (hazard ratio, 0.93; 95% CI, 0.55-1.56). Among adalimumab initiators, combination therapy was associated with longer time to treatment failure (hazard ratio, 0.40; 95% CI, 0.19-0.81). A trend toward lower anti-drug antibody development in the combination therapy arm was not significant (infliximab: odds ratio, 0.72; 95% CI, 0.49-1.07; adalimumab: odds ratio, 0.71; 95% CI, 0.24-2.07). No differences in patient-reported outcomes were observed. Combination therapy resulted in more AEs but fewer SAEs. **Conclusions:** Among adalimumab but not infliximab initiators, patients with pediatric Crohn's disease treated with methotrexate combination therapy experienced a 2-fold reduction in treatment failure with a tolerable safety profile.
- Use of Patient-Reported Outcomes Measurement Information System Pediatric Measures as Clinical Trial Endpoints: Experience from a Multicenter Pragmatic Trial in Children with Crohn's DiseaseCreated:2022-03Affiliation:University of North Carolina at Chapel Hill; Children's Hospital of Philadelphia; Cincinnati Children's Hospital Medical Center**Objectives:** To evaluate whether Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome (PRO) measures can serve as valid endpoints in a clinical trial of a chronic pediatric illness. **Study Design:** We evaluated the responsiveness of PROMIS pediatric measures collected through the Clinical Outcomes of Methotrexate Binary Therapy in Practice (COMBINE) trial, a multicenter, randomized, double-blind, placebo-controlled, pragmatic clinical trial in pediatric patients with Crohn's disease (CD). We examined the relationships between changes in PROMIS pediatric measures and changes in disease activity by evaluating PRO score changes among patients who did and patients who did not experience improvement in disease activity. **Results:** Participants included 266 children and adolescents with CD from a total of 35 institutions. Over the course of follow-up, participants showed improvement in most PRO domains, with the largest effect sizes observed for the clinically improved group. Patients who maintained steroid-free remission showed significantly lower PRO scores for the Pain Interference, Fatigue, and inflammatory bowel disease (IBD) Symptoms domains and higher scores for the Positive Affect domain. **Conclusions:** This study demonstrates the responsiveness of the PROMIS pediatric measures of Fatigue and Pain Interference as study endpoints in a large, multicenter pragmatic trial in pediatric CD, extending a growing body of research supporting the use of PROMIS pediatric measures as reliable PRO endpoints for clinical trials.
- Interpreting Patient-Reported Outcome Scores: Pediatric Inflammatory Bowel Disease as a Use CaseCreated:2022-12Affiliation:Children's Hospital of Philadelphia; Cincinnati Children's Hospital Medical Center; University of Cincinnati; University of North Carolina at Chapel Hill; Temple University**Objective:** To demonstrate how to interpret Patient-Reported Outcomes Measurement Information System (PROMIS) pediatric patient-reported outcome measure (PROM) scores for patients with pediatric inflammatory bowel disease (IBD). **Methods:** Using data from a prospective cohort study of patients ages 8 to 23 years with IBD (n = 1049), we established disease-specific percentiles and computed the minimal clinically important difference (MCID) change score for 6 pediatric PROMs. We applied these results, general population percentiles, and the reliable change index to interpret PROM scores in a clinical trial sample of patients ages 8 to 20 years with IBD (n = 294) in which PROMIS PROMs were obtained at baseline and 3 months later. **Results:** Application of general population percentiles showed that the clinical trial sample at baseline had moderately worse self-reported health than the general population (22% of patients at or above the 95th percentile on Fatigue; 21% on Pain Interference). IBD-specific percentiles showed that the sample was somewhat worse than the reference IBD sample (8% of patients at or above the 95th percentile on Fatigue; 11% on Pain Interference). Application of the MCID threshold indicated that among the subgroup of patients that improved by 15 or more on the short Pediatric Crohn's Disease Activity Index (n = 38), 45% also improved on IBD Symptoms, 47% for Fatigue, and 65% for Pain Interference. **Conclusion:** This study established IBD-specific percentiles for 6 pediatric PROMIS measures and demonstrated the application of percentiles and other methods for interpreting PROM scores.