{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,8]],"date-time":"2026-04-08T03:23:08Z","timestamp":1775618588780,"version":"3.50.1"},"reference-count":25,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2024,11,1]],"date-time":"2024-11-01T00:00:00Z","timestamp":1730419200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Veno\u2010occlusive disease (VOD) is a life\u2010threatening endotheliopathy that can occur after stem cell transplant (SCT). Numerous risk factors contribute to the development of VOD during SCT, and the role of prophylactic defibrotide (DF) in mitigating these risks remains unclear.<\/jats:p><\/jats:sec><jats:sec><jats:title>Objective<\/jats:title><jats:p>We compare not only the incidence of VOD development, but also the severity of VOD and survival outcomes between patients who did and did not develop VOD and did or did not receive prophylactic DF.<\/jats:p><\/jats:sec><jats:sec><jats:title>Study design<\/jats:title><jats:p>In this single\u2010center retrospective study of 58 pediatric SCT patients from 2008 to 2022, we compare the demographics, risk profiles, and outcomes within three cohorts: Group 1: prophylactic DF and no VOD (<jats:italic>n<\/jats:italic>\u00a0=\u00a05), Group 2: prophylactic DF and development of VOD (<jats:italic>n<\/jats:italic>\u00a0=\u00a06), and Group 3: treatment DF for patients who developed VOD (<jats:italic>n<\/jats:italic>\u00a0=\u00a047).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Patients with VOD who did not receive prophylactic DF had higher severity classification of disease at onset (very severe 80.9% vs. 66.7%, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.592) and at maximum severity (very severe 89.4% vs. 83.3%, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.532), as opposed to mild, moderate, or severe categorization compared to those who did not receive prophylactic DF. Patients who developed VOD and did not receive prophylactic DF had a lower 1\u2010year survival probability compared to those who received prophylactic DF and still developed VOD (51.1% vs. 75% alive at 1\u00a0year, excluding the two subjects without adequate follow\u2010up time, <jats:italic>p<\/jats:italic>\u00a0=\u00a0.266).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Although, not statistically significant in our small retrospective study, there is potential overall survival and decreased VOD severity benefits of prophylactic DF.<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.31396","type":"journal-article","created":{"date-parts":[[2024,11,2]],"date-time":"2024-11-02T05:19:38Z","timestamp":1730524778000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":2,"title":["The benefits of prophylactic defibrotide: Are the tides turning?"],"prefix":"10.1002","volume":"72","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-3851-1979","authenticated-orcid":false,"given":"Mahvish Q.","family":"Rahim","sequence":"first","affiliation":[{"name":"Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics Indiana University School of Medicine  Indianapolis Indiana USA"},{"name":"Riley Hospital for Children at Indiana University Health  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"April L.","family":"Rahrig","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics Indiana University School of Medicine  Indianapolis Indiana USA"},{"name":"Riley Hospital for Children at Indiana University Health  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Devin","family":"Dinora","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics Indiana University School of Medicine  Indianapolis Indiana USA"},{"name":"Riley Hospital for Children at Indiana University Health  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jessica","family":"Harrison","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics Indiana University School of Medicine  Indianapolis Indiana USA"},{"name":"Riley Hospital for Children at Indiana University Health  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ryanne","family":"Green","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics Indiana University School of Medicine  Indianapolis Indiana USA"},{"name":"Riley Hospital for Children at Indiana University Health  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Allie","family":"Carter","sequence":"additional","affiliation":[{"name":"Department of Biostatistics and Health Data Science Indiana University School of Medicine  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7653-5920","authenticated-orcid":false,"given":"Jodi","family":"Skiles","sequence":"additional","affiliation":[{"name":"Pediatric Hematology Oncology and Stem Cell Transplant, Department of Pediatrics Indiana University School of Medicine  Indianapolis Indiana USA"},{"name":"Riley Hospital for Children at Indiana University Health  Indianapolis Indiana USA"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"311","published-online":{"date-parts":[[2024,11]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2020.00489"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2010.02.009"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.bbmt.2018.07.039"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41409-020-0918-1"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1038\/bmt.2017.161"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.13215"},{"issue":"3","key":"e_1_2_7_8_1","first-page":"737","article-title":"Treatment of severe veno\u2010occlusive disease with defibrotide: compassionate use results in response without significant toxicity in a high\u2010risk population","volume":"92","author":"Richardson PG","year":"1998","journal-title":"Blood"},{"key":"e_1_2_7_9_1","unstructured":"Jazz Pharmaceuticals announces FDA approval of defitelio (defibrotide sodium) for the treatment of hepativ veno\u2010occlusive disease (VOD) with renal of pulmonary dysfunction following hematopoietic stem\u2010cell transplantation (HSCT).Jazz Pharmaceuticals;2016. 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