{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,15]],"date-time":"2026-10-15T06:58:35Z","timestamp":1792047515033,"version":"4.4.0"},"reference-count":50,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T00:00:00Z","timestamp":1697673600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2024,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>The incidence of secondary malignancies associated with busulfan exposure is considered low, but has been poorly characterized. Because this alkylating agent is increasingly utilized as conditioning prior to gene therapy in nonmalignant hematologic and related disorders, more precise characterization of busulfan's potential contribution to subsequent malignant risk is warranted.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Procedure<\/jats:title>\n                    <jats:p>We conducted a literature\u2010based assessment of busulfan and subsequent late effects, with emphasis on secondary malignancies, identifying publications via PubMed searches, and selecting those reporting at least 3\u00a0years of follow\u2010up.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>We identified eight pediatric and 13 adult publications describing long\u2010term follow\u2010up in 570 pediatric and 2076 adult hematopoietic cell transplant (HCT) recipients. Secondary malignancies were reported in 0.5% of pediatric HCT recipients, with no cases of myelodysplastic syndrome (MDS) or acute myelocytic leukemia (AML). Fatal secondary malignancies were reported in 0.8% of 1887 evaluable adult HCT recipients, and an overall incidence of secondary malignancies of 4.8% was reported in a subset of 389 evaluable adult patients. We also reviewed long\u2010term results from eight publications evaluating lentiviral\u2010 and human promotor\u2010based HSC\u2010targeted gene therapy in 215 patients with nonmalignant conditions, in which busulfan\/treosulfan monotherapy or busulfan\/fludarabine was the only conditioning. Two malignancies were reported in patients with sickle cell disease (SCD), one of which was potentially busulfan\u2010related. No additional malignancies were reported in 173 patients with follow\u2010up of 5\u201312\u00a0years.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>The incidence of busulfan\u2010related secondary malignancies is low, and likely to be substantially less than 1% in pediatric transplant recipients, especially those receiving busulfan monotherapy for nonmalignant conditions other than SCD.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pbc.30738","type":"journal-article","created":{"date-parts":[[2023,10,19]],"date-time":"2023-10-19T11:29:32Z","timestamp":1697714972000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":8,"title":["Busulfan and subsequent malignancy: An evidence\u2010based risk assessment"],"prefix":"10.1002","volume":"71","author":[{"given":"Janel R.","family":"Long\u2010Boyle","sequence":"first","affiliation":[{"name":"University of California  San Francisco California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Donald B.","family":"Kohn","sequence":"additional","affiliation":[{"name":"University of California  Los Angeles California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ami J.","family":"Shah","sequence":"additional","affiliation":[{"name":"Lucile Packard Children's Hospital Stanford University School of Medicine  Stanford California USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sueli Marques","family":"Spencer","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals Inc.  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Cranbury New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Arpita","family":"Shah","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals Inc.  Cranbury New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-4584-6854","authenticated-orcid":false,"given":"Meredith","family":"Reatz","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals Inc.  Cranbury New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joana","family":"Matos","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals Inc.  Cranbury New Jersey USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2211-4151","authenticated-orcid":false,"given":"Jonathan D.","family":"Schwartz","sequence":"additional","affiliation":[{"name":"Rocket Pharmaceuticals Inc.  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