{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T06:27:14Z","timestamp":1786602434414,"version":"build-2736575974"},"reference-count":24,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T00:00:00Z","timestamp":1786579200000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Pediatr."],"abstract":"<jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Pediatric aplastic anemia (AA), a rare and potentially fatal disease, demonstrates significant heterogeneity in pathogenesis, disease severity, therapeutic regimens, and clinical outcomes.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      In this study, clinical features and outcomes of 70 children with AA, including severe AA (SAA,\n                      <jats:italic>n<\/jats:italic>\n                      = 30), very severe AA (vSAA,\n                      <jats:italic>n<\/jats:italic>\n                      = 21) and nonsevere AA (nSAA,\n                      <jats:italic>n<\/jats:italic>\n                      = 19), were retrospectively analyzed, with a median follow-up of 60.7 months (range, 0.4-136.5 months).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Patients with nSAA were mainly treated with cyclosporine A, whereas SAA\/vSAA patients primarily received allogeneic hematopoietic stem cell transplantation (HSCT) followed by standard immunosuppressive therapy (IST). Ultimate therapy regimens differed significantly between patients with SAA\/vSAA and nSAA (\n                      <jats:italic>p<\/jats:italic>\n                      = 0.001). SAA\/vSAA group exhibited a higher overall response rate at the 2-year follow-up (85.5% vs. 55.6%,\n                      <jats:italic>p<\/jats:italic>\n                      = 0.019). The 2-year overall survival (OS) and event-free survival (EFS) for the entire cohort were 97.1% and 48.5%, respectively. In patients with SAA\/vSAA, HSCT was associated with higher and faster cumulative complete response (CR) rate (\n                      <jats:italic>p<\/jats:italic>\n                      &amp;lt; 0.0001) and superior EFS (\n                      <jats:italic>p<\/jats:italic>\n                      = 0.0297) compared with IST. Two IST-resistant patients were observed to achieved CR with eltrombopag (EPAG) salvage therapy.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>pediatric AA carries excellent OS but suboptimal EFS. HSCT tends to yield more favorable EFS compared with IST in SAA\/vSAA patients, and EPAG may act as an effective salvage option for appropriately selected IST-resistant individuals. Further multicenter prospective research is warranted prior to implementing these findings in routine clinical practice.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.3389\/fped.2026.1915688","type":"journal-article","created":{"date-parts":[[2026,8,13]],"date-time":"2026-08-13T05:33:07Z","timestamp":1786599187000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":0,"title":["Real-world retrospective analysis of clinical outcomes in pediatric acquired aplastic anemia: a single-center 10-year cohort study"],"prefix":"10.3389","volume":"14","author":[{"given":"Shanshan","family":"Li","sequence":"first","affiliation":[{"name":"Department of Hematology and oncology, Shanghai Children\u2019s Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kai","family":"Chen","sequence":"additional","affiliation":[{"name":"Department of Hematology and oncology, Shanghai Children\u2019s Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hui","family":"Jiang","sequence":"additional","affiliation":[{"name":"Department of Hematology and oncology, Shanghai Children\u2019s Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Na","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Hematology and oncology, Shanghai Children\u2019s Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jingwei","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Hematology and oncology, Shanghai Children\u2019s Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xuelian","family":"Liao","sequence":"additional","affiliation":[{"name":"Department of Hematology and oncology, Shanghai Children\u2019s Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ting","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Gastroenterology, Hepatology and Nutrition, Shanghai Children's Hospital, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]},{"name":"Gut Microbiota and Metabolic Research Center, Institute of Pediatric Infection, Immunity and Critical Care Medicine, School of Medicine, Shanghai Jiao Tong University","place":["Shanghai, China"]}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shayi","family":"Jiang","sequence":"additional","affiliation":[{"name":"Department of Hematology and oncology, Shanghai 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