{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,7,30]],"date-time":"2025-07-30T12:42:22Z","timestamp":1753879342886,"version":"3.41.2"},"reference-count":30,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2021,3,6]],"date-time":"2021-03-06T00:00:00Z","timestamp":1614988800000},"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":[[2021,6]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Unmanipulated haploid HSCT for SAA has resulted in improved outcomes over recent years. However, studies related to unmanipulated haploid HSCs combined with tp\u2010UCB transplantation for other types of NMD are rare. Accordingly, we present the outcomes of 109 pediatric patients with life\u2010threatening NMD undergoing unmanipulated haploid HSCs combined with tp\u2010UCB transplantation.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedure<\/jats:title><jats:p>We retrospectively investigated 109 pediatric patients with life\u2010threatening NMD treated with unmanipulated haploid HSCs combined with tp\u2010UCB transplantation in a single center.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The median days of neutrophil and platelet engraftment were +13 and +22\u00a0days, respectively. None of the cases experienced PGF. The incidence rates for grade I\u2010II, III\u2010IV aGVHD and cGVHD were 44.9%, 24.8%, and 9.3%, respectively. The incidence rates of CMV and EBV viremia were 46.7% and 39.4%, respectively. The median follow\u2010up duration was 997\u00a0days. In total, 106 patients survived, including 104 cases with FFS and 2 cases with SGF. Three patients died. The 5\u2010year TRM, OS, and FFS were 2.8%, 97.2%, and 96.2%, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>The results of unmanipulated haploid HSCs combined with tp\u2010UCB in pediatric patients with life\u2010threatening NMD were promising. However, further research is now needed to determine specific factors that might influence the engraftment of HSCs.<\/jats:p><\/jats:sec>","DOI":"10.1111\/petr.13995","type":"journal-article","created":{"date-parts":[[2021,3,6]],"date-time":"2021-03-06T16:06:47Z","timestamp":1615046807000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["Improved outcomes using unmanipulated haploidentical hematopoietic stem cells combined with third\u2010party umbilical cord blood transplantation for non\u2010malignant diseases in children: The experience of a single center"],"prefix":"10.1111","volume":"25","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-0672-6983","authenticated-orcid":false,"given":"Xiangfeng","family":"Tang","sequence":"first","affiliation":[{"name":"Department of Pediatrics Chinese PLA General Hospital\u2010Sixth Medical Center  Beijing 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