Objectives To assess the validity of the Patient-Reported Outcomes Measurement Information System® (PROMIS®) Pediatric measures in patients with chronic nonbacterial osteomyelitis (CNO).

Methods Within the longitudinal patient registry of CNO, English-speaking patients aged 8 years and older self-reported PROMIS Pediatric measures of fatigue, pain interference (PI), pain behavior (PB), mobility, upper extremity (UE), physical activity (PA) and strength impact (SI), and external validation measures. Log-transformed linear mixed-effects models with random patient intercepts were used to assess PROMIS T-score changes. Wilcoxon signed-rank test was performed to determine the PROMIS T-score changes among the improved, worsened, and unchanged groups. Spearman rank correlation test determined the relationship of PROMIS T-scores with disease status reported by patients/families.

Results More than 1,000 clinical visits from 184 patients included PROMIS Pediatric measures entries. All PROMIS T-scores correlated significantly (p<0.01) with patient-reported variables and physician global assessment (PHGA). The correlation between function and mobility, PB, and PI was good (r=0.4-0.6). The correlation of patient-reported disease status was strong with PHGA (r=0.75), moderate with mobility, PB, PI, and weak with Fatigue, SI, UE, and PA. The changes of PROMIS T-scores over time for mobility, PB, PA, and PI compared to the self-reported status change were significant (p<0.05). After effective treatment, when clinical disease activity score improved by at least 2.5 points (n=18), the change of PROMIS T-scores for mobility, PB, PI, UE were significant (p<0.05).

Conclusion This study provided evidence supporting the use of PROMIS Pediatric mobility, pain behavior, and pain interference measures for CNO clinical disease monitoring.

Key Messages PROMIS Pediatric measures are valid tools for assessing disease burden in patients with Chronic Nonbacterial osteomyelitis (CNO); T-scores across all domains showed significant correlations with patient-reported outcomes and physician global assessments, with particularly strong correlations for mobility, pain behavior, and pain interference.

PROMIS Pediatric T-scores for mobility, pain behavior, physical activity, and pain interference were sensitive to patient-reported changes in disease status over time, supporting their use in tracking disease progression in CNO.

Following effective treatment, defined by at least a 2.5-point improvement in clinical disease activity score, PROMIS Pediatric T-scores for mobility, pain behavior, pain interference, and upper extremity function showed significant improvement, highlighting their responsiveness to treatment in CNO.

M. Eckert: None; E. Wu: Pharming Healthcare, Inc, 1, 6, Sumitoma Pharma America, Inc, 1; M. Oliver: None; J. Scheck: None; S. Lapidus: None; U. Akca: None; S. Yasin: None; A. Lenert: None; S. Stern: None; A. insalaco: None; M. Pardeo: None; G. Simonini: None; E. Marrani: None; X. Wang: None; B. Huang: None; L. Kovalick: None; N. Rosenwasser: None; E. Balay-Dustrude: None; G. Casselman: None; L. Adriel: None; A. Klein: None; Y. Shao: None; C. Yang: None; M. Briggs: None; E. Deng: None; I. Hamilton: None; E. Mueller: None; E. Machrone: None; P. Trunnel: None; D. Mosa: None; L. Tucker: None; H. Girschick: None; R. Laxer: Akros pharma, 2, Eli Lilly canada, 2, Novartis, 2, Sanofi, 2, sobi, 2, UpToDate (royalties for chapter writing); G. Tiller: None; J. Akikusa: Pfizer, 1; C. Hedrich: Merck, 5; K. Onel: None; F. Dedeoglu: UpToDate (royalties for chapter writing), Sobi (honorarium for a symposium talk) 9; M. Twilt: None; S. Ozen: Novartis, 2, 6, SOBI, 6; and Amgen (consultancy) P. Ferguson: None; L. Schanberg: Bristol-Myers Squibb(BMS), 5, Sanofi, 12, DSMB member, UCB, 12, DSMB Chair; B. Reeve: Novartis, 12, Guest speaker on advisory board, Observer-Reported Communication Ability (ORCA) measure, 12, part inventor for the Observer-Reported Communication Ability (ORCA) measure, receive licensing fees., Pro Pharma NH00006 US Meeting, 6; Y. Zhao: Bristol-Myers Squibb(BMS), 5.

This study was funded by Kaila Komfort

I confirm all relevant ethical guidelines have been followed, and any necessary IRB and/or ethics committee approvals have been obtained.

Yes

The details of the IRB/oversight body that provided approval or exemption for the research described are given below:

The Institutional Review Board of Seattle Childrens Hospital gave ethical approval for this work

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Yes

Disclosure: Y Zhao receives research fund from AF-CARRA, Bristol-Myers Squibb, NIAMS of NIH (Award Number 1R56AR080771.

Funding/Support: Dr A. Lenert is supported by the National Institute Of Arthritis And Musculoskeletal And Skin Diseases of the National Institutes of Health (Award Number K23AR082966).

Disclosures: M. Eckert: None; E. Wu: Pharming Healthcare, Inc, 1, 6, Sumitoma Pharma America, Inc, 1; M. Oliver: None; J. Scheck: None; S. Lapidus: None; U. Akca: None; S. Yasin: None; A. Lenert: None; S. Stern: None; A. insalaco: None; M. Pardeo: None; G. Simonini: None; E. Marrani: None; X. Wang: None; B. Huang: None; L. Kovalick: None; N. Rosenwasser: None; E. Balay-Dustrude: None; G. Casselman: None; L. Adriel: None; A. Klein: None; Y. Shao: None; C. Yang: None; M. Briggs: None; E. Deng: None; I. Hamilton: None; E. Mueller: None; E. Machrone: None; P. Trunnel: None; D. Mosa: None; L. Tucker: None; H. Girschick: None; R. Laxer: Akros pharma, 2, Eli Lilly canada, 2, Novartis, 2, Sanofi, 2, sobi, 2, UpToDate (royalties for chapter writing); G. Tiller: None; J. Akikusa: Pfizer, 1; C. Hedrich: Merck, 5; K. Onel: None; F. Dedeoglu: UpToDate (royalties for chapter writing), Sobi (honorarium for a symposium talk) 9; M. Twilt: None; S. Ozen: Novartis, 2, 6, SOBI, 6; and Amgen (consultancy) P. Ferguson: None; L. Schanberg: Bristol-Myers Squibb(BMS), 5, Sanofi, 12, DSMB member, UCB, 12, DSMB Chair; B. Reeve: Novartis, 12, Guest speaker on advisory board, Observer- Reported Communication Ability (ORCA) measure, 12, part inventor for the Observer-Reported Communication Ability (ORCA) measure, receive licensing fees., Pro Pharma NH00006 US Meeting, 6; sY. Zhao: Bristol-Myers Squibb(BMS), 5.

All data produced in the present study are available upon reasonable request to the authors