{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,6]],"date-time":"2026-10-06T14:49:08Z","timestamp":1791298148454,"version":"4.1.0"},"reference-count":36,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2013,9,9]],"date-time":"2013-09-09T00:00:00Z","timestamp":1378684800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"name":"Office of Orphan Products Development of the Federal Drug Administration","award":["R01 FD002612"],"award-info":[{"award-number":["R01 FD002612"]}]},{"name":"National Center for Research Resources, National Center for Advancing Translational Sciences, National Institutes of Health","award":["8 UL1 TR000077-04"],"award-info":[{"award-number":["8 UL1 TR000077-04"]}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Pediatric Blood &amp; Cancer"],"published-print":{"date-parts":[[2014,1]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>A majority of Fanconi anemia (FA) patients will experience bone marrow failure (BMF) and androgen therapy (most often oxymetholone) may be utilized as a treatment to improve BMF\u2010related cytopenias. However, oxymetholone is associated with toxicities making identification of other agents of interest. In this study we aimed to evaluate the toxicity profile and hematologic response in patients with FA who are treated with low\u2010dose oxandrolone, a synthetic non\u2010fluorinated anabolic steroid, similar to oxymetholone, with known dosing thresholds for virilization.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Procedure<\/jats:title>\n                    <jats:p>A single arm, Phase I\/II study was designed to treat patients on low\u2010dose oxandrolone. If no toxicity or hematologic response was noted at 16 weeks, a single dose escalation was offered. Subjects were regularly assessed for toxicity, including determinations of virilization, behavioral changes, and liver and kidney function. At 32 weeks, those who demonstrated hematologic response were allowed to continue study treatment, and those without improvement were deemed non\u2010responsive.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>Nine subjects completed the study and were followed for a median of 99 weeks (46\u2013136 weeks). Three (33.3%) subjects developed mild sub\u2010clinical virilization and continued treatment with a dose reduction. None (0%) had adverse behavioral changes. Two (22.2%) developed elevated liver function tests at 42 and 105 weeks. Seven (77.8%) subjects had a hematologic response.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion<\/jats:title>\n                    <jats:p>Oxandrolone appears to be well\u2010tolerated, has limited toxicities at the administered doses in FA with patients, and may be an alternative androgen for the treatment of BMF in FA. Pediatr Blood Cancer 2014;61:11\u201319. \u00a9 2013 Wiley Periodicals, Inc.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/pbc.24617","type":"journal-article","created":{"date-parts":[[2013,9,9]],"date-time":"2013-09-09T03:22:45Z","timestamp":1378696965000},"page":"11-19","source":"Crossref","is-referenced-by-count":31,"title":["Oxandrolone for the treatment of bone marrow failure in Fanconi anemia"],"prefix":"10.1002","volume":"61","author":[{"given":"Susan R.","family":"Rose","sequence":"first","affiliation":[{"name":"Department of Pediatrics University of Cincinnati College of Medicine, Cincinnati Children's Hospital Medical Center Cincinnati Ohio"},{"name":"Division of Endocrinology Stanford University School of Medicine Stanford California"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mi\u2010Ok","family":"Kim","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Cincinnati College of Medicine, Cincinnati Children's 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