{
  "abstract": "Objectives Clinical trial data are lacking for treatment of patients with juvenile systemic sclerosis (jSSc). Three published recommendations exist for jSSc but real-world data on treatment patterns are lacking. The aim of this study was to analyse treatments used in the jSSc inception cohort (jSSci) and compare to published recommendations on the treatment of jSSc.Methods Data was extracted for patients with 24 months follow-up visits in the jSSci up until June 2023. Medications used and their association with clinical characteristics were analysed. Logistic regression analyses were performed to compare treatments between limited and diffuse cutaneous jSSc subtypes, organ involvement and time of initiation of treatment. Multilevel mixed effects logistic regression analyses were used to evaluate the change in medication use in follow-up. Treatment patterns were compared against published recommendations.Results 93 patients had 24 months follow-up data. 77% of patients were receiving disease-modifying treatment (DMARD) at enrolment, which increased to 91% at 24 months (p<0.001). Patients with diffuse cutaneous jSSc subtype had significantly more frequently active ulcerations, skin involvement and received any kind of treatment more often compared with limited (97% vs 91%, p=0.047). Methotrexate was used in 52% of patients at enrolment which decreased to 37% at 24 months (p=0.001). Mycophenolate mofetil use increased from 24% to 46% (p<0.001). Biological DMARDs increased from 5% to 22% (p<0.001). The treatment pattern strongly overlapped with the published paediatric guidance.Conclusion This is the first report regarding the pattern of medication use in real life in the currently largest patient cohort of patients with jSSc. The observed pattern overlaps with the published recommendations.",
  "authors": [
    {
      "affiliations": [
        "Dr Hamburg Centre for Pediatric and Adolescence Rheumatology, DLR Hamburg, Hamburg, Germany"
      ],
      "name": "Ivan Foeldvari"
    },
    {
      "affiliations": [
        "Pediatric Rheumatology, University of Pittsburgh, Pittsburgh, Pennsylvania, USA"
      ],
      "name": "Kathryn Torok"
    },
    {
      "affiliations": [
        "Istanbul University Cerrahpasa Faculty of Medicine, Fatih, Turkey"
      ],
      "name": "Ozgur Kasapcopur"
    },
    {
      "affiliations": [
        "Istanbul University Cerrahpasa Faculty of Medicine, Fatih, Turkey"
      ],
      "name": "Amra Adrovic"
    },
    {
      "affiliations": [
        "The Hospital for Sick Children, Toronto, Ontario, Canada"
      ],
      "name": "Brian Feldman"
    },
    {
      "affiliations": [
        "Universidade do Estado do Rio de Janeiro, Rio de Janeiro, Brazil"
      ],
      "name": "Flavio Sztajnbok"
    },
    {
      "affiliations": [
        "Universidade Federal de São Paulo, Sao Paulo, Brazil"
      ],
      "name": "Maria Teresa Terreri"
    },
    {
      "affiliations": [
        "Universidade Federal de São Paulo, Sao Paulo, Brazil"
      ],
      "name": "Ana Paula Sakamoto"
    },
    {
      "affiliations": [
        "Toronto Western Hospital, Toronto, Ontario, Canada"
      ],
      "name": "Sindhu Johnson"
    },
    {
      "affiliations": [
        "Hospital Sant Joan de Déu, Barcelona, Spain"
      ],
      "name": "Jordi Anton"
    },
    {
      "affiliations": [
        "Riga Stradins University, Riga, Latvia"
      ],
      "name": "Valda Stanevicha"
    },
    {
      "affiliations": [
        "Jaslok Hospital and Research Centre, Mumbai, Maharashtra, India"
      ],
      "name": "Archana Khan"
    },
    {
      "affiliations": [
        "Emma Children’s Hospital, Amsterdam, The Netherlands"
      ],
      "name": "Dieneke Schonenberg-Meinema"
    },
    {
      "affiliations": [
        "Birmingham Women’s and Children’s Hospital NHSFT, Birmingham, UK"
      ],
      "name": "Eslam Al-Abadi"
    },
    {
      "affiliations": [
        "I M Sechenov First Moscow State Medical University, Moskva, Russian Federation"
      ],
      "name": "Ekaterina Alexeeva"
    },
    {
      "affiliations": [
        "Hospital de Pediatria Prof Dr Juan P Garrahan, Buenos Aires, Argentina"
      ],
      "name": "Maria Katsicas"
    },
    {
      "affiliations": [
        "Sir Ganga Ram Hospital, New Delhi, Delhi, India"
      ],
      "name": "Sujata Sawhney"
    },
    {
      "affiliations": [
        "Department of Internal Medicine, Ghent University, Ghent, Belgium",
        "Department of Rheumatology, Ghent University Hospital, Ghent, Belgium"
      ],
      "name": "Vanessa Smith"
    },
    {
      "affiliations": [
        "State University of Campinas Faculty of Medical Sciences, Campinas, Brazil"
      ],
      "name": "Simone Appenzeller"
    },
    {
      "affiliations": [
        "University Medical Centre Ljubljana, Ljubljana, Slovenia"
      ],
      "name": "Tadej Avcin"
    },
    {
      "affiliations": [
        "Saint Petersburg State Pediatric Medical University of the Ministry of Health of the Russian Federation, Saint Petersburg, Russian Federation"
      ],
      "name": "Mikhail Kostik"
    },
    {
      "affiliations": [
        "Hospital for Special Surgery, New York, New York, USA"
      ],
      "name": "Thomas Lehman"
    },
    {
      "affiliations": [
        "Motol University Hospital, Prague, Czech Republic"
      ],
      "name": "Hana Malcova"
    },
    {
      "affiliations": [
        "Florence, AOU Meyer Children’s Hospital, Florence, Italy"
      ],
      "name": "Edoardo Marrani"
    },
    {
      "affiliations": [
        "Alder Hey Children’s NHS Foundation Trust, Liverpool, UK"
      ],
      "name": "Clare E Pain"
    },
    {
      "affiliations": [
        "University of Missouri Columbia Health Care, Columbia, Missouri, USA"
      ],
      "name": "Anjali Patwardhan"
    },
    {
      "affiliations": [
        "Hospital Ramón y Cajal, Madrid, Spain"
      ],
      "name": "Walter-Alberto Sifuentes-Giraldo"
    },
    {
      "affiliations": [
        "Children’s Hospital at Montefiore, Bronx, New York, USA"
      ],
      "name": "Natalia Vasquez-Canizares"
    },
    {
      "affiliations": [
        "Hospital de Santa Maria, Lisbon, Portugal"
      ],
      "name": "Patricia Costa-Reis"
    },
    {
      "affiliations": [
        "Sri Ramachandra University Medical College, Chennai, Tamil Nadu, India"
      ],
      "name": "Mahesh Janarthanan"
    },
    {
      "affiliations": [
        "Pediatric Rheumatology, University of Tübingen, Tübingen, Germany"
      ],
      "name": "Monika Moll"
    },
    {
      "affiliations": [
        "Charles University, Prague, Czech Republic"
      ],
      "name": "Dana Nemcova"
    },
    {
      "affiliations": [
        "Hospital Garcia de Orta EPE, Almada, Portugal"
      ],
      "name": "Maria Jose Santos"
    },
    {
      "affiliations": [
        "Department of Pediatrics, Faculty of Medicine, Makassed Hospital, Al-Quds University, East-Jerusalem, Palestine"
      ],
      "name": "Sima Abu Al-Saoud"
    },
    {
      "affiliations": [
        "Hospital de Niños Dr Orlando Alassia, Santa Fe, Argentina"
      ],
      "name": "Cristina Battagliotti"
    },
    {
      "affiliations": [
        "Uppsala Universitet, Uppsala, Sweden"
      ],
      "name": "Lillemor Berntson"
    },
    {
      "affiliations": [
        "Universidade Federal do Rio de Janeiro Hospital Universitário Clementino Fraga Filho, Rio de Janeiro, Brazil"
      ],
      "name": "Blanca Bica"
    },
    {
      "affiliations": [
        "Medical University of Innsbruck, Innsbruck, Austria"
      ],
      "name": "Juergen Brunner"
    },
    {
      "affiliations": [
        "Great Ormond Street Hospital for Children NHS Foundation Trust, London, UK"
      ],
      "name": "Despina Eleftheriou"
    },
    {
      "affiliations": [
        "Schneider Children’s Medical Center of Israel Faculty of Medicine Tel Aviv University, Petah-Tikva, Israel"
      ],
      "name": "Liora Harel"
    },
    {
      "affiliations": [
        "Asklepios Klinik Sankt Augustin GmbH, Sankt Augustin, Germany",
        "University Hospital Cologne, Cologne, Germany"
      ],
      "name": "Gerd Horneff"
    },
    {
      "affiliations": [
        "Luzerner Kantonsspital, Lucerne, Switzerland"
      ],
      "name": "Daniela Kaiser"
    },
    {
      "affiliations": [
        "Charite - Universitatsmedizin Berlin, Berlin, Germany"
      ],
      "name": "Tilmann Kallinich"
    },
    {
      "affiliations": [
        "University Clinical Centre of Niš, Niš, Serbia"
      ],
      "name": "Dragana Lazarevic"
    },
    {
      "affiliations": [
        "Charite - Universitatsmedizin Berlin, Berlin, Germany"
      ],
      "name": "Kirsten Minden"
    },
    {
      "affiliations": [
        "Copenhagen University Hospital Rigshospitalet, Copenhagen, Denmark"
      ],
      "name": "Susan Nielsen"
    },
    {
      "affiliations": [
        "Stony Brook Children’s Hospital, Stony Brook, New York, USA"
      ],
      "name": "Farzana Nuruzzaman"
    },
    {
      "affiliations": [
        "Oslo University Hospital, Oslo, Norway"
      ],
      "name": "Siri Opsahl Hetlevik"
    },
    {
      "affiliations": [
        "Meir Medical Center, Kefar Sava, Israel"
      ],
      "name": "Yosef Uziel"
    },
    {
      "affiliations": [
        "German Rheumatism Research Center Berlin, Berlin, Germany"
      ],
      "name": "Jens Klotsche"
    }
  ],
  "title": "What is the treatment pattern of juvenile systemic sclerosis? Results from an international cohort",
  "uid": "8d6fbf6b-f0f2-5f20-be5a-db85fbcfe52a"
}