Burden of disease, treatment utilization, and the impact on education and employment in patients with sickle cell disease: A comparative analysis of high- and low- to middle-income countries for the international Sickle Cell World Assessment Survey.

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Abstract

[Indexed for MEDLINE] Conflict of interest statement: I.O. reports consultancy for Agios, Cheisi, Cyclerion, FORMA Therapeutics, Global Blood Therapeutics, HCP Live, Novartis, and Takeda; speakers' bureau for Emmaus, Global Blood Therapeutics, and Novartis; advisory board for Acceleron, Cyclerion, Cheisi, Emmaus, FORMA therapeutics, Global Blood Therapeutics, Novartis, and Novo‐Nordisk; grants from Centers for Disease Control (CDC), Department for Public Health (DPH), Health Resources and Services Administration (HRSA), Patient Centered Outcomes Research Institute (PCORI), and NC; Data and Safety Monitoring Board (DSMB) membership for Micella Biopharma; and Editor‐In‐Chief for Hematology News. J.J. reports employment by the Sickle Cell Society and honoraria from Novartis. F.E.‐R. reports research funding from Cyclerion, Novartis, and Pfizer; and advisory board participation for Novartis, Global Blood Therapeutics, and bluebird bio. A.N. reports consultancy/expert testimony for bluebird bio, Global Blood Therapeutics, and Novartis. C.P.M. reports consultancy for Agios, bluebird bio, Emmaus, Forma Therapeutics, Global Blood Therapeutics, Novartis, Roche, and Sanguine. C.T. reports consultancy/expert testimony for Novartis, Cyclerion, and Global Blood Therapeutics. J.P. is an employee of Novartis Pharmaceuticals Corporation. N.R. is an employee of Novartis Pharma AG. O.R.‐H. and T.B. are employees of Adelphi Real World, which received payment from Novartis Pharmaceuticals as part of this research. B.A. reports consultancy or membership on an advisory committee for Agios, Aruvant, bluebird bio, CRISPR/Vertex, Cyclerion, Emmaus, Forma Therapeutics, Global Blood Therapeutics, Hemanext, Novartis, Novo Nordisk, Sanofi Genzyme, and Terumo BCT; and research funding from Forma Therapeutics, Global Blood Therapeutics, Hemanext, Imara, and Novartis. 19. J Rheumatol. 2023 Feb;50(2):236-239. doi: 10.3899/jrheum.220382. Epub 2022 Oct 1. Reliability of the Pediatric Specific Musculoskeletal Ultrasound Scoring Systems for the Elbow, Wrist, and Finger Joints. Vega-Fernandez P(1), Esteban Y(2), Oberle E(3), Proulx-Gauthier JP(4), Clark M(5), Shenoi S(6), Thatayatikom A(7), Benham H(8), Brunner EJ(9), Woolnough L(10), Henrickson M(2), Pratt LR(11), De Ranieri D(12), Hoffmann S(13), Janow G(14), Bukulmez H(15), Altaye M(16), Cassedy A(16), Ting TV(2), Roth J; CARRA JIA Ultrasound Workgroup. Author information: (1)P. Vega-Fernandez, MD, MSc, Y. Esteban, MD, M. Henrickson, MD, MPH, T.V. Ting, MD, MSc, Department of Pediatrics, University of Cincinnati, Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA; patricia.vegafernandez@cchmc.org. (2)P. Vega-Fernandez, MD, MSc, Y. Esteban, MD, M. Henrickson, MD, MPH, T.V. Ting, MD, MSc, Department of Pediatrics, University of Cincinnati, Division of Rheumatology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. (3)E. Oberle, MD, Nationwide Children's Hospital, Columbus, Ohio, USA. (4)J.P. Proulx-Gauthier, MD, FRCPC, Department of Pediatrics, CHU de Québec-Université Laval, Quebec City, Quebec, Canada. (5)M. Clark, MD, Department of Pediatrics, Vanderbilt University Medical Center, Nashville, Tennessee, USA. (6)S. Shenoi, MBBS, MS, Seattle Children's Hospital and Research Center University of Washington, Seattle, Washington, USA. (7)A. Thatayatikom, MD, AdventHealth for Children, Orlando, Florida, USA. (8)H. Benham, DNP, APRN, Scottish Rite for Children Dallas, Dallas, Texas, USA. (9)E.J. Brunner, DO, Geisinger Medical Center, Danville, Pennsylvania, USA. (10)L. Woolnough, MD, MSCS, Department of Pediatrics, UFHealth, Gainesville, Florida, USA. (11)L.R. Pratt, MD, University of Nebraska Medical Center, Omaha, Nebraska, USA. (12)D. De Ranieri, MD, Department of Pediatrics, Northwestern Feinberg School of Medicine, Division of Rheumatology, Ann and Robert H. Lurie Children's Hospital, Chicago, Illinois, USA. (13)S. Hoffmann, MD, Children's Hospital of Richmond, Virginia, USA. (14)G. Janow, MD, MPH, Joseph M. Sanzari Children's Hospital, Hackensack, New Jersey, USA. (15)H. Bukulmez, MD, Department of Pediatrics, Division of Pediatric Rheumatology, Metro Health Medical System, Case Western Reserve University, Cleveland, Ohio, USA. (16)M. Altaye, PhD, A. Cassedy, PhD, Department of Pediatrics, University of Cincinnati, Division of Biostatistics and Epidemiology, Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, USA. OBJECTIVE: Musculoskeletal ultrasound (MSUS) is increasingly being used in the evaluation of pediatric musculoskeletal diseases. In order to provide objective assessments of arthritis, reliable MSUS scoring systems are needed. Recently, joint-specific scoring systems for arthritis of the pediatric elbow, wrist, and finger joints were proposed by the Childhood Arthritis and Rheumatology Research Alliance (CARRA) MSUS workgroup. This study aimed to assess the reliability of these scoring systems when used by sonographers with different levels of expertise. METHODS: Members of the CARRA MSUS workgroup attended training sessions for scoring the elbow, wrist, and finger. Subsequently, scoring exercises of B mode and power Doppler (PD) mode still images for each joint were performed. Interreader reliability was determined using 2-way single-score intraclass correlation coefficients (ICCs) for synovitis and Cohen [Formula: see text] for tenosynovitis. RESULTS: Seventeen pediatric rheumatologists with different levels of MSUS expertise (1-15 yrs) completed a 2-hour training session and calibration exercise for each joint. Excellent reliability (ICC > 0.75) was found after the first scoring exercise for all the finger and elbow views evaluated on B mode and PD mode, and for all of the wrist views on B mode. After a second training session and a scoring exercise, the wrist PD mode views reached excellent reliability as well. CONCLUSION: The preliminary CARRA MSUS scoring systems for assessing arthritis of the pediatric elbow, wrist, and finger joints demonstrate excellent reliability among pediatric MSUS sonographers with different levels of expertise. With further validation, this reliable joint-specific scoring system could serve as a clinical tool and scientific outcome measure. Copyright © 2023 by the Journal of Rheumatology. DOI: 10.3899/jrheum.220382 PMCID: PMC9898106