Browsing by Author "Zhao, Yongdong"

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    Disease burden and social impact of pediatric chronic nonbacterial osteomyelitis from the patient and family perspective
    (Biomed Central, 2018-12-14) Oliver, Melissa; Lee, Tzielan C.; Halpern-Felsher, Bonnie; Murray, Elizabeth; Schwartz, Rebecca; Zhao, Yongdong; Pediatrics, School of Medicine
    BACKGROUND: Chronic nonbacterial osteomyelitis (CNO) is an autoinflammatory bone disorder that if left untreated can result in bone destruction and severe continuing pain due to persistent inflammation. The impact this chronic disease has on the daily lives of affected children and their families is not well known. The purpose of this study is to understand the disease burden and socioeconomic and psychological impact of CNO from the patients' and families' perspectives and identify areas of improvement for patient care and reduced disease burden based on patients' and families' responses. METHODS: Participants were invited through a social media platform group and at clinic visits at Stanford Children's Health. An online survey was administered to patients with a diagnosis of CNO made at < 22 years of age and/or the parent/guardian of a patient with CNO diagnosis made at < 22 years of age. RESULTS: There was a total of 284 survey participants. The median age at CNO diagnosis was 10 years (range 2-22+). Median time from first CNO symptom to diagnosis was 2 years. Antibiotics were used in 35% of patients prior to CNO diagnosis; of these, 24% received antibiotics for greater than 6 months. Between 25 and 61% reported a negative effect of CNO on relationships, school/work performance, or finances; and 19-50% reported effects on psychosocial well-being. The majority agreed patients' performance with daily tasks and hobbies was challenged by pain, fatigue and physical limitation related to CNO. CONCLUSIONS: Patients with CNO experienced on average a 2-year delay in diagnosis and receiving effective treatments. At least 25% reported problems with relationships, school, work, finances and well-being due to CNO. Recognition of these challenges emphasizes the need to increase awareness of this disease and address the socioeconomic stressors and mental health issues in order to provide optimal care of children with CNO.
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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.