Establishing core domain sets for Chronic Nonbacterial Osteomyelitis (CNO) and Synovitis, Acne, Pustulosis, Hyperostosis, Osteitis (SAPHO): A report from the OMERACT 2020 special interest group

Oliver, Melissa; Jayatilleke, Arundathi; Wu, Eveline Y.; Nuruzzaman, Farzana; Aguiar, Cassyanne L.; Lenert, Aleksander; Romano, Micol; Simonini, Gabriele; Shah, Samir A.; Hollander, Matthew; Alsaleem, Alhanouf; Fox, Emily; Theos, Alexander C.; Ramanan, Athimalaipet V; Schnabel, Anja; Lapidus, Sivia; Tucker, Lori B.; Onel, Karen; Özen, Seza; Dedeoğlu, Fatma; Ferguson, Polly J.; Shea, Beverley; Akikusa, Jonathan; Hedrich, Christian M.; Mease, Philip J.; Zhao, Yichao

doi:10.1016/j.semarthrit.2021.05.015

Cited by 8 publications

(7 citation statements)

References 12 publications

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“…Our workgroup has followed The OMERACT (Outcome Measures in Rheumatoid Arthritis Clinical Trials) filter 2.1 (24) to develop outcome measurements for CNO (25). A scoping review showed life impact and pathophysiological manifestations (25) to be the main core areas reported in the literature.…”

Section: Methodsmentioning

confidence: 99%

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

Wu¹,

Oliver²,

Scheck³

et al. 2022

Preprint

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Objectives: Study objectives were to: 1) determine the frequency of use and safety of each consensus treatment plan (CTP) regimen for chronic nonbacterial osteomyelitis (CNO) and the feasibility of using chronic nonbacterial osteomyelitis international registry (CHOIR) data for comparative effectiveness research and 2) develop and validate a CNO clinical disease activity score (CDAS) using CHOIR. Methods: Consenting CNO children or young adults 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 12 (8-15.5) to 5 (3-12). All 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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Section: Methodsmentioning

confidence: 99%

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

Wu¹,

Oliver²,

Scheck³

et al. 2022

Preprint

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“…At this time, the field of ABDs lacks validated outcomes measures that would facilitate comparative effectiveness of different therapies. The OMERACT (Outcome Measures in Rheumatoid Arthritis Clinical Trials) CNO/SAPHO working group was established in 2019 to develop a core outcome measurement set through a data driven and consensus process for CNO and SAPHO for all clinical trials ( 86 ).…”

Section: Future Directionsmentioning

confidence: 99%

Pediatric autoinflammatory bone disorders—a mini review with special focus on pathogenesis and inborn errors of immunity

Hetrick

Oliver

2023

Front. Pediatr.

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Autoinflammatory bone disorders are a group of diseases characterized by sterile osteomyelitis. This includes chronic nonbacterial osteomyelitis and the monogenic forms, Majeed syndrome and deficiency of the interleukin-1 receptor antagonist. These disorders result from innate immune system dysregulation and cytokine imbalance that triggers inflammasome activation causing downstream osteoclastogenesis and excessive bone remodeling. In this review, we will summarize the immunopathogenesis of pediatric autoinflammatory bone diseases with a special focus on the genetics and inborn errors of immunity, while briefly touching on the clinical manifestations and management of each disease as well as areas for future research.

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“…A subgroup of the CNO investigators participated with ‘Outcome Measures in Rheumatology’ (OMERACT) to lead a ‘scoping review’ with the goal of developing a core domain set for CNO that fulfills the requirements of the OMERACT 2.1 Filter [53]which can be used in clinical trials and observational studies. The ‘scoping review’ yielded 4821 articles including 273 articles that met inclusion criteria and were reviewed [54 ▪▪ ]. Preliminary disease-related manifestations and impacts reported from the scoping review were mapped to the OMERACT core areas, with most items categorized under the ‘Life Impact’ and ‘Pathophysiology Manifestation’ core areas included physical well-being, function, and pain, radiographic changes, inflammatory markers, and physical exam findings, such as limb swelling 58 .…”

Section: Clinical and Radiologic Outcome Measuresmentioning

confidence: 99%

Update on treatment responses and outcome measure development in chronic nonbacterial osteomyelitis

Ramachandran

Zhao

Ferguson

2023

Current Opinion in Rheumatology

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Purpose of review To review recent trends in treatment and recent progress in developing outcome measures needed for chronic nonbacterial osteomyelitis (CNO) clinical trials. Recent findings CNO is an autoinflammatory bone disease. In a minority of patients, the disease is genetically driven, and diagnosis can be made by DNA sequencing. However, for nonsyndromic CNO there is no diagnostic test. The number of children with CNO appears to be increasing and damage is common. Increases in CNO diagnosis is due to raised awareness, increased availability of whole-body magnetic resonance imaging and rising incidence. Treatment remains empiric and it is unclear which second line treatment is superior. Tumor necrosis factor inhibitors (TNFi) and bisphosphonates continue to be used as second line agents for nonsteroidal anti-inflammatory drugs (NSAID) refractory CNO; newer immune modulatory medications are used if this fails. Validated classification criteria, clinical outcome measures and imaging scoring standards are needed for successful clinical trials. Summary Best treatment for NSAID refractory CNO remains unclear. Classification criteria, clinical outcomes measures and standardized imaging scoring have been developed or are near completion. This will facilitate robust clinical trials in CNO with the goal of having approved medications for this painful disease.

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Establishing core domain sets for Chronic Nonbacterial Osteomyelitis (CNO) and Synovitis, Acne, Pustulosis, Hyperostosis, Osteitis (SAPHO): A report from the OMERACT 2020 special interest group

Cited by 8 publications

References 12 publications

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

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

Pediatric autoinflammatory bone disorders—a mini review with special focus on pathogenesis and inborn errors of immunity

Update on treatment responses and outcome measure development in chronic nonbacterial osteomyelitis

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