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Longitudinal Evaluation of Patient-reported Outcomes Measurement Information System (PROMIS) Scores in Children Under
Joseph N Charla1, Leila Mehraban Alvandi1, Zachariah Samuel1
1Montefiore Einstein Department of Orthopaedic Surgery, Division of Pediatric Orthopaedic Surgery, Bronx, NY.
Insights
Pediatric Legg-Calvé-Perthes Disease (LCPD) outcomes improve with disease stage. PROMIS scores in children under 6 showed significant gains in mobility, pain, and emotional well-being as the condition progressed.
Area of Science:
- Pediatric Orthopedics
- Rehabilitation Medicine
- Child Health Outcomes
Background:
- Legg-Calvé-Perthes Disease (LCPD) is an idiopathic ischemic hip disorder impacting children.
- Children under 6 with LCPD are understudied, despite being a significant patient group.
- This study focuses on longitudinal outcomes in this young demographic.
Purpose of the Study:
- To evaluate longitudinal Patient-Reported Outcomes Measurement Information System (PROMIS) scores in children diagnosed with LCPD before age 6.
- To understand the evolution of functional and emotional well-being in relation to disease progression.
- To provide data for setting realistic expectations for families.
Main Methods:
- A multicenter prospective cohort study included 181 children (188 hips) diagnosed with LCPD at age 6 or younger.
- PROMIS T-scores (mobility, pain, fatigue, depression, anxiety, anger, peer relationships) were assessed.
- Linear mixed-effects regression models analyzed PROMIS scores against Waldenström stages, adjusting for covariates.
Main Results:
- PROMIS scores were analyzed across Waldenström stages in 181 children (median age 4.6 years).
- At stage I, most domains showed mild impairment, except normal depression and fatigue.
- Pain interference and anger normalized by stage IIa, mobility by stage IIIa, and anxiety by stage IIIb.
Conclusions:
- PROMIS T-scores demonstrated significant improvements across multiple domains as LCPD patients under 6 advanced through Waldenström stages.
- These findings offer valuable insights into the natural history of LCPD in young children.
- The data can assist clinicians in guiding families regarding prognosis and expected recovery trajectories.
Background:
Legg-Calvé-Perthes Disease (LCPD) is an idiopathic ischemic hip disorder that can lead to significant impairments in children. Despite representing a substantial portion of LCPD patients, children under 6 remain understudied. This study evaluates longitudinal PROMIS scores in children diagnosed with LCPD before age 6.
Methods:
This multicenter prospective cohort included children diagnosed with LCPD at age 6 or younger between February 2017 and July 2023. PROMIS T-scores (mobility, pain interference, fatigue, depression, anxiety, anger, and peer relationships) were analyzed alongside the Waldenström stage at the time of assessment using linear mixed-effects regression models, adjusting for demographics and interventions.
Results:
Among 188 hips in 181 children (median age 4.6 y, 81% male, 79% Caucasian) across 11 institutions, 810 PROMIS surveys were included. The mean and median follow-up was 1.9 years. At stage I, all domains showed mild impairment except depression and fatigue, which remained normal. Pain interference and anger normalized by stage iia, mobility by stage IIIa, and anxiety by stage IIIb (P<0.05).
Conclusions:
In children under 6, PROMIS T-scores showed significant improvements across domains as patients progressed through Waldenström stages. These findings provide insight into how PROMIS T-scores evolve with disease stage, helping clinicians guide families on expected outcomes and set realistic expectations.
Level Of Evidence:
Level II-prognostic.
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