{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,20]],"date-time":"2026-07-20T22:02:00Z","timestamp":1784584920673,"version":"3.55.0"},"reference-count":15,"publisher":"BMJ","issue":"2","license":[{"start":{"date-parts":[[2024,11,8]],"date-time":"2024-11-08T00:00:00Z","timestamp":1731024000000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2024,11,8]],"date-time":"2024-11-08T00:00:00Z","timestamp":1731024000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Scleroderma and Related Disorders"],"published-print":{"date-parts":[[2025,6]]},"abstract":"<jats:sec>\n                    <jats:title>Objective:<\/jats:title>\n                    <jats:p>Juvenile-onset systemic sclerosis (jSSc) is a rare and life-threatening disease with no formal studies evaluating the indications for, access to, or benefits of autologous stem cell transplantation (ASCT). As a first step toward understanding pediatric jSSc specialist thoughts and experiences with ASCT, we conducted a multinational survey.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>An electronic survey was developed and distributed in November 2023 to members of the Pediatric Rheumatology European Society (PRES) and\/or Childhood Arthritis and Rheumatology Research Alliance (CARRA) pediatric scleroderma workgroups.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>Twenty-nine (69%) jSSc specialists completed the survey. All participants have considered or would consider ASCT referral for a jSSc patient. Nearly all respondents indicated disease-modifying anti-rheumatic drugs (DMARDs) should be trialed prior to ASCT referral, with most indicating two to four DMARDs. The most common reasons selected for referral were rapidly progressive disease (despite DMARD) (90%), followed by severe disease status (83%), and significant impact on quality of life (83%). All respondents selected pulmonary disease as an indication for referral, followed by cardiac (93%), gastrointestinal (72%), and skin disease (66%). While pulmonary and cardiac involvement were considered individually sufficient for referral for ASCT, only a minority considered musculoskeletal involvement (28%) sufficient on its own.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>This survey is the first explore thoughts and experience with ASCT for jSSc. Results indicate pediatric rheumatologists were aware of and would consider ASCT for their patients. Our results indicate there is likely some variability in clinical practice regarding who is referred for ASCT, and further research is needed to guide development of evidence-based clinical care guidelines.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/23971983241293297","type":"journal-article","created":{"date-parts":[[2024,11,8]],"date-time":"2024-11-08T03:15:14Z","timestamp":1731035714000},"page":"163-169","update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["What are Juvenile-onset systemic sclerosis providers thoughts, experiences, and reasons for autologous stem cell transplant? Result of a multinational survey"],"prefix":"10.1136","volume":"10","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0659-5298","authenticated-orcid":false,"given":"Ivan","family":"Foeldvari","sequence":"first","affiliation":[{"name":"Hamburger Zentrum f\u00fcr Kinder-und Jugenrheumatologie, Hamburg, Germany"},{"name":"Hamburg Centre for Pediatric and Adolescence Rheumatology, Centre for Treatment of Scleroderma and Uveitis in Childhood and Adolescence, Teaching Unit of the Asklepios Campus of the Semmelweis Medical School, Budapest, Sch\u00f6n Klinik Hamburg Eilbek, Hamburg, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samantha","family":"Branton","sequence":"additional","affiliation":[{"name":"UPMC Children\u2019s Hospital of Pittsburgh, Pittsburgh, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Suzanne C","family":"Li","sequence":"additional","affiliation":[{"name":"Joseph M. Sanzari Children\u2019s Hospital, Hackensack Meridian School of Medicine, Hackensack, NJ, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Franziska J","family":"Rosser","sequence":"additional","affiliation":[{"name":"Division of Pulmonary Medicine, Department of Pediatrics, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1662-143X","authenticated-orcid":false,"given":"Kathryn S","family":"Torok","sequence":"additional","affiliation":[{"name":"Division of Rheumatology, Department of Pediatrics, School of Medicine, University of Pittsburgh, Pittsburgh, PA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2024,11,8]]},"reference":[{"key":"e_1_3_2_2_2","doi-asserted-by":"publisher","DOI":"10.1177\/2397198318763701"},{"key":"e_1_3_2_3_2","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/ken388"},{"key":"e_1_3_2_4_2","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/ker269"},{"key":"e_1_3_2_5_2","doi-asserted-by":"publisher","DOI":"10.1080\/1744666X.2023.2298354"},{"key":"e_1_3_2_6_2","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(11)60982-3"},{"key":"e_1_3_2_7_2","doi-asserted-by":"publisher","DOI":"10.1001\/jama.2014.6368"},{"key":"e_1_3_2_8_2","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa1703327"},{"key":"e_1_3_2_9_2","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/keaa584"},{"key":"e_1_3_2_10_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.41153"},{"key":"e_1_3_2_11_2","doi-asserted-by":"publisher","DOI":"10.1002\/acr.24609"},{"key":"e_1_3_2_12_2","doi-asserted-by":"publisher","DOI":"10.1164\/rccm.202306-1113ST"},{"key":"e_1_3_2_13_2","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/39.5.556"},{"key":"e_1_3_2_14_2","doi-asserted-by":"publisher","DOI":"10.1136\/ard.2009.114264"},{"key":"e_1_3_2_15_2","doi-asserted-by":"publisher","DOI":"10.1093\/rheumatology\/kead457"},{"key":"e_1_3_2_16_2","doi-asserted-by":"publisher","DOI":"10.1002\/art.42850"}],"container-title":["Journal of Scleroderma and Related Disorders"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/23971983241293297","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/23971983241293297","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/23971983241293297","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,5,5]],"date-time":"2026-05-05T11:53:03Z","timestamp":1777981983000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/23971983241293297"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,11,8]]},"references-count":15,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2025,6]]}},"alternative-id":["10.1177\/23971983241293297"],"URL":"https:\/\/doi.org\/10.1177\/23971983241293297","relation":{},"ISSN":["2397-1983","2397-1991"],"issn-type":[{"value":"2397-1983","type":"print"},{"value":"2397-1991","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,11,8]]}}}