{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,23]],"date-time":"2026-08-23T17:16:38Z","timestamp":1787505398586,"version":"build-2736575974"},"reference-count":19,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2024,5,20]],"date-time":"2024-05-20T00:00:00Z","timestamp":1716163200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Transplantation"],"published-print":{"date-parts":[[2024,6]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>The goal of this study was to assess the effect of donor type and pre\u2010transplant immunotherapy (IST) on outcomes of hematopoietic stem cell transplantation (HSCT) for children and young adults with severe aplastic anemia (SAA).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>This retrospective, multi\u2010center study included 52 SAA patients, treated in 5 pediatric transplant programs in Florida, who received HSCT between 2010 and 2020 as the first\u2010 or second\u2010line treatment.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      The median age at HSCT for all 52 patients was 15\u2009years (range 1\u201325). The 3\u2010year overall survival (OS) by donor type were as follows: 95% [95% CI 85.4\u201399] for matched related donors (MRD) (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200924), 84% [95% CI 63.5\u201399] for haploidentical (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200913), and 71% [95% CI 36\u201399] for matched unrelated donors (MUD) (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u20097). The 3\u2010year OS was 81% [95% CI 69.7\u201099] for all patients, 90.5% [95% CI 79.5\u201099] for non\u2010IST patients (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200927), and 70% [95% CI 51\u201399] for IST patients (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200924) (log\u2010rank\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u2009.04). Survival of haploidentical HSCT (haplo\u2010HSCT) recipients with post\u2010transplant cyclophosphamide (PTCy) (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200913) was excellent for both groups: 100% for non\u2010IST patients (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u20093) and 80% for IST patients (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200910). The 3\u2010year OS for patients with previous IST by donor type in groups where &gt;5 patients were available was 78.8% [95% CI 52.3\u201399] for haplo\u2010HSCT (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u200910) and 66.7% [95% CI 28.7\u201399] for MUD (\n                      <jats:italic>N<\/jats:italic>\n                      \u2009=\u20096). Although it appears that patients receiving HSCT \u22656\u2009months after the start of IST had worse survival, the number of patients in each category was small and log\u2010rank was not significant(\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u2009.65).\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Patients receiving MUD and haplo\u2010HSCT with PTCy had similar outcomes, suggesting that haplo\u2010HSCT with PTCy could be included in randomized trials of upfront IST versus alternative donor HSCT.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1111\/petr.14784","type":"journal-article","created":{"date-parts":[[2024,5,20]],"date-time":"2024-05-20T05:19:38Z","timestamp":1716182378000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["The role of donor type and pre\u2010transplant immunosuppression on outcomes of hematopoietic stem cell transplantation in children and young adults with severe aplastic anemia"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0009-0001-4570-9799","authenticated-orcid":false,"given":"Reema","family":"Kashif","sequence":"first","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Biljana","family":"Horn","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jordan","family":"Milner","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Joyce","sequence":"additional","affiliation":[{"name":"Nemours Children's Health &amp; Wolfson Children's Hospital  Jacksonville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mansi","family":"Dalal","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin\u2010Ju","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kevin","family":"McNerney","sequence":"additional","affiliation":[{"name":"Center for Cell &amp; Gene Therapy for Non\u2010Malignant Conditions John Hopkins All Children's Hospital  St. Petersburg Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Cline","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John","family":"Fort","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Castillo","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; BMT University of Florida  Gainesville Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jorge","family":"Galvez","sequence":"additional","affiliation":[{"name":"Pediatric Blood &amp; Marrow Transplantation Program Nicklaus Children's Hospital  Miami Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1802-5025","authenticated-orcid":false,"given":"Warren","family":"Alperstein","sequence":"additional","affiliation":[{"name":"School of Medicine University of Miami  Miami Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5513-1233","authenticated-orcid":false,"given":"John","family":"Ligon","sequence":"additional","affiliation":[{"name":"Department of Pediatrics, Division of Pediatric Hematology\u2010Oncology &amp; 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