{"doi":"10.1101/2022.10.03.22280351","title":"Feasibility of Conducting Comparative Effectiveness Research and Validation of a Clinical Disease Activity Score for Chronic Nonbacterial Osteomyelitis","abstract":"Abstract Introduction Prospective comparative effectiveness research in chronic nonbacterial osteomyelitis (CNO) is lacking. Objectives Study objectives were to: 1) determine the use and safety of each consensus treatment plan (CTP) regimen for CNO, 2) the feasibility of using ch ronic nonbacterial o steomyelitis international registry (CHOIR) data for comparative effectiveness research, 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 the three 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. The CDAS significantly decreased after initiating second-line treatments from median 12 (8-15.5) to 5 (3-12). 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. Key messages The ch ronic nonbacterial o steomyelitis international registry (CHOIR) provides comprehensive prospective data for comparison of treatment effectiveness The clinical disease activity score (CDAS) has content and construct validity to assess CNO","journal":"medRxiv","year":2022,"id":304511,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":1,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9396,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2022-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":788726,"name":"Melissa Oliver","orcid":"0000-0002-7689-2573","position":1,"is_corresponding":false},{"id":997943,"name":"Joshua Scheck","orcid":null,"position":2,"is_corresponding":false},{"id":858042,"name":"Sivia Lapidus","orcid":"0000-0001-8452-9937","position":3,"is_corresponding":false},{"id":572637,"name":"Ümmüşen Kaya Akça","orcid":"0000-0002-0426-9432","position":4,"is_corresponding":false},{"id":997389,"name":"Shima Yasin","orcid":"0000-0003-1520-1254","position":5,"is_corresponding":false},{"id":297057,"name":"Sara Stern","orcid":null,"position":6,"is_corresponding":false},{"id":997390,"name":"Antonella Insalaco","orcid":"0000-0003-1443-1840","position":7,"is_corresponding":false},{"id":997391,"name":"Manuela Pardeo","orcid":"0000-0003-0850-7351","position":8,"is_corresponding":false},{"id":997392,"name":"Gabriele Simonini","orcid":"0000-0002-5919-4861","position":9,"is_corresponding":false},{"id":997393,"name":"Edoardo Marrani","orcid":"0000-0002-0346-3268","position":10,"is_corresponding":false},{"id":254105,"name":"Xing Wang","orcid":"0000-0003-4313-6381","position":11,"is_corresponding":false},{"id":372514,"name":"Bin Huang","orcid":"0000-0001-9724-675X","position":12,"is_corresponding":false},{"id":997944,"name":"Leonard K. 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