Evidence map›Paper›PMID 40312718›Full record

ReviewPediatric rheumatology online journal2025

Developing consensus outcome measures in juvenile systemic sclerosis: a global survey of pediatric rheumatologists and literature review.

Natalia Vasquez-Canizares, Clare E Pain, Francesco Zulian, Amra Adrovic Yildiz, Simone Appenzeller, Edoardo Marrani, Emanuela Del Giudice, Antonella Petaccia, Francesca Tirelli, Gabriele Simonini and 28 more

Erratum issuedAbstract readReview
In one paragraph

Review in Pediatric rheumatology online journal, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing papers in PubMed
–field-weighted citation impact
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

3 citing papers in PubMed.

  1. Article
  2. Article
  3. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

38 authors.

Natalia Vasquez-CanizaresDepartment of Pediatrics, Division of Pediatric Rheumatology, Children's Hospital at Montefiore/ Albert Einstein College of Medicine, 3415 Bainbridge Ave, Bronx, NY, 10467, USA. navasque@montefiore.org.
Clare E PainDepartment of Rheumatology, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, UK.
Francesco ZulianRheumatology Unit, Department of Woman and Child Health, University Hospital of Padova, Padua, Italy.
Amra Adrovic YildizDepartment of Pediatric Rheumatology, Cerrahpasa Medical School, Istanbul University-Cerrahpasa, Istanbul, Türkiye.
Simone AppenzellerDepartment of Orthopedics, Rheumatology and Traumatology, School of Medical Science, University of Campinas, Campinas, Brazil.
Edoardo MarraniPediatric Rheumatology Unit, Meyer Children Hospital IRCCS, Florence, Italy.
Emanuela Del GiudicePediatrics and Neonatology Unit, Department of Maternal Infantile and Urological Sciences, Sapienza University of Rome, Polo Pontino, Santa Maria Goretti Hospital, Latina, Italy.
Antonella PetacciaFondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Italy.
Francesca TirelliRheumatology Unit, Department of Woman and Child Health, University Hospital of Padova, Padua, Italy.
Gabriele SimoniniPediatric Rheumatology Unit, Meyer Children Hospital IRCCS, Florence, Italy.
Mustafa ÇakanDepartment of Pediatric Rheumatology, Zeynep Kamil Women and Children's Diseases Training and Research Hospital, Istanbul, Türkiye.
Marco CattaliniPediatrics Clinic, ERN RITA Centre, University of Brescia and ASST Spedali Civili Di Brescia, Brescia, Italy.
Paulo Rogerio JulioDepartment of Child and Adolescent Health, Universidade Estadual de Campinas, Campinas, São Paulo, Brazil.
Kathryn TorokPediatric Rheumatology, UPMC Children's Hospital of Pittsburgh, Pittsburgh, PA, USA.
Raju KhubchandaniPediatric Rheumatology, SRCC Children's Hospital, Mumbai, India.
Ozgur KasapcopurDepartment of Pediatric Rheumatology, Cerrahpasa Medical School, Istanbul University-Cerrahpasa, Istanbul, Türkiye.
Lauren RobinsonDepartment of Pediatrics, Division of Pediatric Rheumatology, Hospital for Special Surgery, New York, NY, USA.
Eslam Al-AbadiChildhood Arthritis and Rheumatic Diseases Unit, Birmingham Women's and Children's Hospital NHSFT, Birmingham, UK.
Aybuke GunalpDepartment of Pediatric Rheumatology, Cerrahpasa Medical School, Istanbul University-Cerrahpasa, Istanbul, Türkiye.
Meiping LuDepartment of Rheumatology Immunology and Allergy, Children's Hospital, Zhejiang University School of Medicine, Zhejiang, China.
Hanna LythgoeDepartment of Paediatric and Adolescent Rheumatology, Royal Manchester Children's Hospital, Manchester, UK.
Amanda RobinsonPediatrics, Division of Pediatric Rheumatology, University of Utah, Salt Lake City, UT, USA.
Betul SozeriHealthy Sciences University, Istanbul Umraniye Training and Research Hospital, Istanbul, Türkiye.
Susan ShenoiSeattle Children's Hospital and Research Center / University of Washington, Seattle, WA, USA.
Emily WillisDepartment of Paediatric and Adolescent Rheumatology, Royal Manchester Children's Hospital, Manchester, UK.
Katherine ClarkeDepartment of Rheumatology, Alder Hey Children's NHS Foundation Trust, Eaton Road, Liverpool, UK.
Rongjun ZhengDepartment of Rheumatology Immunology and Allergy, Children's Hospital, Zhejiang University School of Medicine, Zhejiang, China.
Biagio CastaldiPediatric and Congenital Cardiology Unit, Department for Women and Children's Health, University Hospital of Padova, Padua, Italy.
Valentina LeoneDepartment of Paediatrics, Istituto Mediterraneo Per I Trapianti E Terapie Ad Alta Specializzazione-IRCCS ISMETT, University of Pittsburgh Medical Centre (UPMC), Palermo, Italy.
Valerio ManiscalcoRheumatology Unit, ERN-ReCONNET Center, Meyer Children's Hospital IRCCS, Florence, Italy.
Lucy SteadWomen's Hospital Liverpool, Neonatology, Liverpool, UK.
Lusine AmbartsumyanDivision of Gastroenterology and Hepatology, Seattle Children's Hospital, University of Washington School of Medicine, Seattle, WA, USA.
Franziska RosserDepartment of Pediatrics, Division of Pulmonary Medicine, University of Pittsburgh School of Medicine, Pittsburgh, PA, USA.
Phuoc DuongDepartment of Cardiac Surgery and Cardiology, Alder Hey Children's Hospital, Liverpool, UK.
Aurelia MinutiD. Samuel Gottesman Library, Albert Einstein College of Medicine, Bronx, NY, USA.
Suzanne C Li *Hackensack Meridian School of Medicine, Joseph M. Sanzari Children's Hospital, Hackensack, NJ, USA.
Marinka Twilt *Department of Pediatrics, Alberta Children's Hospital, Cumming School of Medicine, University of Calgary, Calgary, AB, Canada.
CARRA Juvenile Scleroderma Workgroup

Funding

Childhood Arthritis and Rheumatology Research Alliance App_207
6 · The paper itself

Abstract

backgroundJuvenile systemic sclerosis (JSSc) is a rare multisystemic disease with high morbidity and mortality rates. Treatment options remain limited, and there is a significant unmet need for effective therapies. This study aims to address this gap by investigating current JSSc management practices and identifying key outcome measures that can be used to inform the development of standardized assessment tools for future clinical trials.

methodsA web-based survey was distributed to pediatric rheumatologists to assess cardiopulmonary assessment standard of care practices and immunosuppressive treatment use in JSSc. Respondents were categorized by region (North America, Europe, Latin America, and Asia/Africa), and country income level. A scoping literature review was conducted using the PRISMA-SCR framework to identify outcome measures for six domains in SSc.

resultsOne hundred forty-one pediatric rheumatologists from 26 countries completed the survey. Significant variations in JSSc cardiopulmonary assessment practices across regions and income levels were noted. Respondents in North America and Europe reported using pulmonary function tests (PFTs) with diffusing capacity of the lungs for carbon monoxide (DLCO) more frequently than those in Latin America, or Asia/Africa (p < 0.001). The 6-min walk test (6MWT) was used less frequently by respondents in North America than other regions (p = 0.004). Use of oral corticosteroid and cyclophosphamide for treatment of JSSc varies significantly based on country income level, with higher usage in low- and middle-income nations. The scoping review identified 848 relevant articles for data extraction (ranging from 36 to 156 per domain) from a pool of 31,825 records, which were screened in multiple stages by 39 investigators.

conclusionWe found significant variability in JSSc assessment and treatment preferences, influenced by geography and income. This highlights the urgent need for international collaboration and standardized approaches in JSSc care.

Indexed as

Outcome Assessment, Health CarePractice Patterns, Physicians'RheumatologistsScleroderma, SystemicChildConsensus Statements as TopicGlobal HealthHumansImmunosuppressive AgentsMaleRespiratory Function TestsSurveys and QuestionnairesImmunosuppressive AgentsAssessmentConsensusJuvenile sclerodermaJuvenile systemic sclerosisOutcome measureSclerodermaScoping reviewSurveySystemic sclerosisTreatment

Identifiers

PMID40312718
PMCPMC12046749

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.