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Feasibility of Conducting Comparative Effectiveness Research and Validation of a Clinical Disease Activity Score for Chronic Nonbacterial Osteomyelitis
Journal article   Peer reviewed

Feasibility of Conducting Comparative Effectiveness Research and Validation of a Clinical Disease Activity Score for Chronic Nonbacterial Osteomyelitis

Eveline Y Wu, Melissa Oliver, Joshua Scheck, Sivia Lapidus, Ummusen Kaya Akca, Shima Yasin, Sara M Stern, Antonella Insalaco, Manuela Pardeo, Gabriele Simonini, …
Journal of rheumatology, Vol.50(10), pp.1333-1340
10/01/2023
DOI: 10.3899/jrheum.2022-1323
PMCID: PMC10543471
PMID: 37399459

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Abstract

OBJECTIVE Prospective comparative effectiveness research (CER) in chronic nonbacterial osteomyelitis (CNO) is lacking. Our objectives were:1) determine the use and safety of each consensus treatment plan (CTP) regimen for CNO, 2) assess the feasibility of using chronic nonbacterial osteomyelitis international registry (CHOIR) data for CER, and 3) develop and validate a CNO clinical disease activity score (CDAS) using CHOIR. METHODS Consenting children or young adults with CNO were enrolled into CHOIR. Demographic, clinical, and imaging data were prospectively collected. The CNO CDAS was developed through a Delphi survey and nominal group technique. External validation surveys were administered to CHOIR participants. RESULTS 140 (76%) CHOIR participants enrolled between August 2018 and September 2020 received at least one CTP regimen. Baseline characteristics from different CTP groups were well matched. Patient pain, patient global assessment, and clinical CNO lesion count were key variables included in the CNO CDAS. The CDAS showed a strong correlation with patient/parent report of difficulty using a limb, back, or jaw and patient/parent report of disease severity, but a weak correlation with patient/parent report of fatigue, sadness, and worry. The change in CDAS was significant in patients reporting disease worsening or improvement (p<0.001). The CDAS significantly decreased after initiating second-line treatments from median 12 (8-15.5) to 5 (3-12) (p=0.002). While second-line treatments were well tolerated, psoriasis was the most common adverse event. CONCLUSION The CNO CDAS was developed and validated for disease monitoring and assessment of treatment effectiveness. CHOIR provided a comprehensive framework for future comparative effectiveness research.

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