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; Edoardo Marrani; Xing Wang; Bin Huang; Leonard K Kovalick; Natalie Rosenwasser; Gabriel Casselman; Adriel Liau; Yurong Shao; Claire Yang; Doaa Mosad Mosa; Lori Tucker; Hermann Girschick; Ronald M Laxer; Jonathan D Akikusa; Christian M Hedrich; Karen Onel; Fatma Dedeoglu; Marinka Twilt; Polly J Ferguson; Seza Ozen; Yongdong Zhao

doi:10.3899/jrheum.2022-1323

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

J Rheumatol. 2023 Oct;50(10):1333-1340. doi: 10.3899/jrheum.2022-1323. Epub 2023 Jul 1.

Authors

Eveline Y Wu¹, Melissa Oliver², Joshua Scheck³, Sivia Lapidus⁴, Ummusen Kaya Akca⁵, Shima Yasin⁶, Sara M Stern⁷, Antonella Insalaco⁸, Manuela Pardeo⁸, Gabriele Simonini⁹, Edoardo Marrani⁹, Xing Wang¹⁰, Bin Huang¹¹, Leonard K Kovalick¹, Natalie Rosenwasser³, Gabriel Casselman³, Adriel Liau³, Yurong Shao³, Claire Yang³, Doaa Mosad Mosa¹², Lori Tucker¹³, Hermann Girschick¹⁴, Ronald M Laxer¹⁵, Jonathan D Akikusa¹⁶, Christian M Hedrich¹⁷, Karen Onel¹⁸, Fatma Dedeoglu¹⁹, Marinka Twilt²⁰, Polly J Ferguson⁶, Seza Ozen⁵, Yongdong Zhao²¹

Affiliations

¹ E.Y. Wu, MD, MSCR, L.K. Kovalick, PNP, Division of Rheumatology, Department of Pediatrics, The University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
² M. Oliver, MD, MS, Division of Pediatric Rheumatology, Indiana University School of Medicine, Indianapolis, Indiana, USA.
³ J. Scheck, BS, N. Rosenwasser, MD, G. Casselman, BS, A. Liau, BS, Y. Shao, BS, C. Yang, BS, Y. Zhao, MD, PhD, Division of Pediatric Rheumatology, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA.
⁴ S. Lapidus, MD, Division of Rheumatology, Department of Pediatrics, Joseph M. Sanzari Children's Hospital, Hackensack University Medical Center and Hackensack Meridian School of Medicine, Hackensack, New Jersey, USA.
⁵ U. Kaya Akca, MD, S. Ozen, MD, Division of Rheumatology, Department of Pediatrics, Hacettepe University, Ankara, Turkey.
⁶ S. Yasin, MD, MSc, P.J. Ferguson, MD, Division of Rheumatology, Allergy and Immunology, Department of Pediatrics, University of Iowa Carver College of Medicine, Iowa City, Iowa, USA.
⁷ S.M. Stern, MD, Division of Rheumatology, Department of Pediatrics, The University of Utah, Salt Lake City, Utah, USA.
⁸ A. Insalaco, MD, M. Pardeo, MD, Division of Rheumatology, ERN RITA Center, IRCCS Ospedale Pediatrico Bambino Gesù, Roma, Italy.
⁹ G. Simonini, MD, E. Marrani, MD, Rheumatology Unit, ERN ReCONNET Center, Meyer Children's Hospital IRCCS, Florence, Italy.
¹⁰ X. Wang, PhD, Biostatistics Epidemiology and Analytics in Research, Seattle Children's Research Institute, Seattle, Washington, USA.
¹¹ B. Huang, PhD, Division of Biostatistics and Epidemiology, Cincinnati Children's Hospital Medical Center, University of Cincinnati College of Medicine, Cincinnati, Ohio, USA.
¹² D.M. Mosa, MD, Rheumatology and Rehabilitation Department, Mansoura University Hospitals, Mansoura University, Mansoura City, Egypt.
¹³ L. Tucker, MD, Division of Rheumatology, BC Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.
¹⁴ H. Girschick, Department of Pediatrics, Vivantes Clinic Friedrichshain, Berlin, Germany.
¹⁵ R.M. Laxer, The Hospital for Sick Children and the University of Toronto, Toronto, Ontario, Canada.
¹⁶ J.D. Akikusa, Royal Children's Hospital, Melbourne, Australia.
¹⁷ C.M. Hedrich, MD, PhD, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, UK.
¹⁸ K. Onel, MD, Division of Pediatric Rheumatology, Hospital for Special Surgery, New York, New York, USA.
¹⁹ F. Dedeoglu, MD, Division of Immunology, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA.
²⁰ M. Twilt, MD, Division of Rheumatology, Department of Paediatrics, Alberta Children's Hospital, Calgary, Alberta, Canada.
²¹ J. Scheck, BS, N. Rosenwasser, MD, G. Casselman, BS, A. Liau, BS, Y. Shao, BS, C. Yang, BS, Y. Zhao, MD, PhD, Division of Pediatric Rheumatology, Seattle Children's Hospital, University of Washington, Seattle, Washington, USA; ydzhao@uw.edu.

PMID: 37399459
PMCID: PMC10543471 (available on 2024-10-01)
DOI: 10.3899/jrheum.2022-1323

Abstract

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.

Keywords: chronic nonbacterial osteomyelitis; chronic recurrent multifocal osteomyelitis; clinical disease activity score; comparative effectiveness research; consensus treatment plans.

Publication types

Research Support, N.I.H., Extramural
Research Support, Non-U.S. Gov't

MeSH terms

Child
Chronic Disease
Comparative Effectiveness Research*
Feasibility Studies
Humans
Osteomyelitis* / drug therapy
Osteomyelitis* / pathology
Prospective Studies
Young Adult

Supplementary concepts

Chronic recurrent multifocal osteomyelitis

Grants and funding

KL2 TR002490/TR/NCATS NIH HHS/United States