{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,26]],"date-time":"2026-04-26T04:14:32Z","timestamp":1777176872328,"version":"3.51.4"},"reference-count":32,"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><jats:sec><jats:title>Background<\/jats:title><jats:p>The aims of this study are to report our experience with treosulfan\u2010based conditioning regimens for patients with non\u2010malignant hematologic conditions, correlating clinical outcomes at different time points post\u2010transplant with treosulfan exposure (AUC).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This study was a single\u2010center observational study investigating overall survival (OS), disease\u2010free survival (DFS), and event\u2010free survival (EFS) end\u2010points post\u2010transplant. The consequences of treosulfan AUC with respect to toxicity, correction of underlying disease, and long\u2010term chimerism were also explored using pharmacokinetic analysis.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Forty\u2010six patients received 49 transplants with treosulfan and fludarabine\u2010based conditioning between 2005 and 2023. Twenty\u2010four patients also received thiotepa. Donor chimerism was assessed on either whole blood or sorted cell lines at different time points post\u2010transplant. Thirty\u2010nine patients received treosulfan pharmacokinetic assessment to evaluate cumulative AUC, with five infants receiving real\u2010time assessment to facilitate daily dose adjustment. OS, DFS, and EFS were 87%, 81%, and 69%, respectively. Median follow\u2010up was 32.1\u2009months (range 0.82\u2013160\u2009months) following transplant. Lower EFS was associated with patient age (&lt;1\u2009year; <jats:italic>p<\/jats:italic>\u2009=\u2009.057) and lower cumulative treosulfan dose (&lt;42\u2009g\/m<jats:sup>2<\/jats:sup>; <jats:italic>p<\/jats:italic>\u2009=\u2009.003). Stable donor chimerism in B\u2010cell, NK\u2010cell, and granulocyte lineages at 1\u2010year post\u2010transplant were more prevalent in patients receiving thiotepa conditioning. Two infants required daily dose adjustment to treosulfan to avoid high AUC.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Excellent clinical outcomes and stable chimerism were observed in this patient series. The addition of thiotepa conferred no significant toxicity and trended toward sustained ongoing donor engraftment. Correlating treosulfan AUC with long\u2010term patient outcomes is required.<\/jats:p><\/jats:sec>","DOI":"10.1111\/petr.14780","type":"journal-article","created":{"date-parts":[[2024,5,20]],"date-time":"2024-05-20T09:23:46Z","timestamp":1716197026000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Outcomes from hematopoietic stem cell transplantation following treosulfan\u2010based conditioning: A clinical and pharmacokinetic analysis"],"prefix":"10.1111","volume":"28","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0203-4904","authenticated-orcid":false,"given":"Sebastian P. 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