Browsing by Author "Ozen, Seza"

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    Feasibility of Conducting Comparative Effectiveness Research and Validation of a Clinical Disease Activity Score for Chronic Nonbacterial Osteomyelitis
    (The Journal of Rheumatology, 2023) Wu, Eveline Y.; Oliver, Melissa; Scheck, Joshua; Lapidus, Sivia; Akca, Ummusen Kaya; Yasin, Shima; Stern, Sara M.; Insalaco, Antonella; Pardeo, Manuela; Simonini, Gabriele; Marrani, Edoardo; Wang, Xing; Huang, Bin; Kovalick, Leonard K.; Rosenwasser, Natalie; Casselman, Gabriel; Liau, Adriel; Shao, Yurong; Yang, Claire; Mosa, Doaa Mosad; Tucker, Lori; Girschick, Hermann; Laxer, Ronald M.; Akikusa, Jonathan D.; Hedrich, Christian; Onel, Karen; Dedeoglu, Fatma; Twilt, Marinka; Ferguson, Polly J.; Ozen, Seza; Zhao, Yongdong; Pediatrics, School of Medicine
    Objective: Prospective comparative effectiveness research (CER) in chronic nonbacterial osteomyelitis (CNO) is lacking. Our objectives were to (1) determine the use and safety of each consensus treatment plan (CTP) regimen for CNO, (2) assess the feasibility of using the 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: One hundred forty (78.2%) CHOIR participants enrolled between August 2018 and September 2020 received at least 1 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 12.0 (IQR 8.0-15.5) to 5.0 (IQR 3.0-12.0; P = 0.002). Although 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 CER.
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    The 2021 European Alliance of Associations for Rheumatology/American College of Rheumatology points to consider for diagnosis and management of autoinflammatory type I interferonopathies: CANDLE/PRAAS, SAVI and AGS
    (BMJ, 2022) Cetin Gedik, Kader; Lamot, Lovro; Romano, Micol; Demirkaya, Erkan; Piskin, David; Torreggiani, Sofia; Adang, Laura A.; Armangue, Thais; Barchus, Kathe; Cordova, Devon R.; Crow, Yanick J.; Dale, Russell C.; Durrant, Karen L.; Eleftheriou, Despina; Fazzi, Elisa M.; Gattorno, Marco; Gavazzi, Francesco; Hanson, Eric P.; Lee-Kirsch, Min Ae; Montealegre Sanchez, Gina A.; Neven, Bénédicte; Orcesi, Simona; Ozen, Seza; Poli, M. Cecilia; Schumacher, Elliot; Tonduti, Davide; Uss, Katsiaryna; Aletaha, Daniel; Feldman, Brian M.; Vanderver, Adeline; Brogan, Paul A.; Goldbach-Mansky, Raphaela; Pediatrics, School of Medicine
    Objective: Autoinflammatory type I interferonopathies, chronic atypical neutrophilic dermatosis with lipodystrophy and elevated temperature/proteasome-associated autoinflammatory syndrome (CANDLE/PRAAS), stimulator of interferon genes (STING)-associated vasculopathy with onset in infancy (SAVI) and Aicardi-Goutières syndrome (AGS) are rare and clinically complex immunodysregulatory diseases. With emerging knowledge of genetic causes and targeted treatments, a Task Force was charged with the development of 'points to consider' to improve diagnosis, treatment and long-term monitoring of patients with these rare diseases. Methods: Members of a Task Force consisting of rheumatologists, neurologists, an immunologist, geneticists, patient advocates and an allied healthcare professional formulated research questions for a systematic literature review. Then, based on literature, Delphi questionnaires and consensus methodology, 'points to consider' to guide patient management were developed. Results: The Task Force devised consensus and evidence-based guidance of 4 overarching principles and 17 points to consider regarding the diagnosis, treatment and long-term monitoring of patients with the autoinflammatory interferonopathies, CANDLE/PRAAS, SAVI and AGS. Conclusion: These points to consider represent state-of-the-art knowledge to guide diagnostic evaluation, treatment and management of patients with CANDLE/PRAAS, SAVI and AGS and aim to standardise and improve care, quality of life and disease outcomes.