{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,8]],"date-time":"2026-06-08T12:56:37Z","timestamp":1780923397460,"version":"3.54.1"},"reference-count":42,"publisher":"Wiley","issue":"10","license":[{"start":{"date-parts":[[2021,6,4]],"date-time":"2021-06-04T00:00:00Z","timestamp":1622764800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100012524","name":"American Lebanese Syrian Associated Charities","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100012524","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100001422","name":"American Society of Hematology","doi-asserted-by":"publisher","award":["ASH Scholar Award"],"award-info":[{"award-number":["ASH Scholar Award"]}],"id":[{"id":"10.13039\/100001422","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2021,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Bone marrow failure in dyskeratosis congenita (DKC) is progressive, and allogeneic hematopoietic cell transplantation (HCT) is the only curative treatment. However, outcomes after HCT are suboptimal because of mucosal, vascular, pulmonary, and hepatic fragility, which can be exacerbated by chemotherapy conditioning and graft\u2010versus\u2010host disease (GVHD). These toxicities can be mitigated by reducing the intensity of the conditioning regimen.<\/jats:p><\/jats:sec><jats:sec><jats:title>Procedures<\/jats:title><jats:p>We performed a retrospective analysis on pediatric patients with DKC who underwent HCT at our institution between 2008 and 2019.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>We identified nine patients (median age, 5.7\u00a0years) who underwent HCT with a fludarabine\u2010based reduced\u2010intensity conditioning (RIC) regimen. GVHD prophylaxis consisted of tacrolimus plus mycophenolate mofetil (MMF) (<jats:italic>n<\/jats:italic>\u2009\u00a0=\u00a0\u20098), tacrolimus\/pentostatin (<jats:italic>n<\/jats:italic>\u2009\u00a0=\u00a0\u20091), or cyclosporine\/MMF (<jats:italic>n<\/jats:italic>\u2009\u00a0=\u00a0\u20091). The median time to neutrophil engraftment was 19 days (range, 13\u201026 days), and the median time to platelet engraftment was 18 days (range, 17\u201043 days). Lung function, as measured by spirometry in six patients, remained stable during post\u2010HCT observation. Six patients (67%) remain alive, with a median follow\u2010up of 73.5 months.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Because of toxicity after myeloablative conditioning, RIC is becoming standard for HCT in DKC. These results suggest that RIC regimen is feasible and safe for patients with DKC and does not accelerate pulmonary damage in the short\u2010to\u2010medium term after HCT.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.29177","type":"journal-article","created":{"date-parts":[[2021,6,4]],"date-time":"2021-06-04T15:27:57Z","timestamp":1622820477000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":18,"title":["Reduced\u2010intensity conditioning\u2013based hematopoietic cell transplantation for dyskeratosis congenita: Single\u2010center experience and literature review"],"prefix":"10.1002","volume":"68","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-1402-6549","authenticated-orcid":false,"given":"Senthil Velan","family":"Bhoopalan","sequence":"first","affiliation":[{"name":"Department of Hematology St. Jude Children's Research Hospital  Memphis 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