{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,28]],"date-time":"2026-04-28T16:17:41Z","timestamp":1777393061224,"version":"3.51.4"},"reference-count":24,"publisher":"Wiley","issue":"7","license":[{"start":{"date-parts":[[2021,3,10]],"date-time":"2021-03-10T00:00:00Z","timestamp":1615334400000},"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":[[2021,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>Outcomes after relapse remain poor in pediatric patients with acute myeloid leukemia (AML), and new therapeutic approaches are needed. Lenalidomide has demonstrated activity in adults with lower risk myelodysplastic syndromes and older adults with relapsed or refractory (R\/R) AML.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>In this phase 2 study (NCT02538965), pediatric patients with R\/R AML who received two or more prior therapies were treated with lenalidomide (starting dose 2\u00a0mg\/kg\/day on days 1\u201321 of each 28\u2010day cycle) for a maximum of 12 cycles. The primary endpoint was rate of complete response (CR) and CR with incomplete blood count recovery (CRi) within the first four cycles.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Seventeen patients enrolled and received one or more dose of lenalidomide. Median age was 12\u00a0years (range 5\u201318 years), median white blood cell count was 3.7 \u00d7 10<jats:sup>9<\/jats:sup>\/L, and median peripheral blood blast count was 1.0 \u00d7 10<jats:sup>9<\/jats:sup>\/L. One patient (5.9%) with a complex karyotype including del(5q) achieved CRi after two cycles of lenalidomide. This responder proceeded to a second hematopoietic stem cell transplantation and has remained without evidence of disease for 3\u00a0years. All patients experienced one or more of grades 3\u20134 treatment\u2010emergent adverse event (TEAE). The most common grades 3\u20134 TEAEs were thrombocytopenia (58.8%), febrile neutropenia (47.1%), anemia (41.2%), and hypokalemia (41.2%).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>In this population of pediatric patients with R\/R AML, safety data were consistent with the known safety profile of lenalidomide. As only one patient responded, further evaluation of lenalidomide at the dose and schedule studied is not warranted in pediatric AML, with the possible exception of patients with del(5q).<\/jats:p><\/jats:sec>","DOI":"10.1002\/pbc.28946","type":"journal-article","created":{"date-parts":[[2021,3,11]],"date-time":"2021-03-11T06:48:53Z","timestamp":1615445333000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":7,"title":["Results of a phase 2, multicenter, single\u2010arm, open\u2010label study of lenalidomide in pediatric patients with relapsed or refractory acute myeloid leukemia"],"prefix":"10.1002","volume":"68","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-9054-4321","authenticated-orcid":false,"given":"Maureen M.","family":"O'Brien","sequence":"first","affiliation":[{"name":"Cancer and Blood Diseases Institute, Cincinnati Children's Hospital Medical Center University of Cincinnati College of Medicine  Cincinnati Ohio USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Todd A.","family":"Alonzo","sequence":"additional","affiliation":[{"name":"Keck School of Medicine University of Southern California  Los Angeles California USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Todd M.","family":"Cooper","sequence":"additional","affiliation":[{"name":"Seattle Children's Cancer and Blood Disorders Center University of Washington  Seattle Washington USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"John E.","family":"Levine","sequence":"additional","affiliation":[{"name":"Bone Marrow and Stem Cell Transplantation Program Mount Sinai School of Medicine  New York New York USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patrick A.","family":"Brown","sequence":"additional","affiliation":[{"name":"The Johns Hopkins Hospital  Baltimore Maryland USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tamra","family":"Slone","sequence":"additional","affiliation":[{"name":"Department of Pediatrics University of Texas Southwestern Medical Center  Dallas Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Keith J.","family":"August","sequence":"additional","affiliation":[{"name":"Children's Mercy Hospital Kansas City  Kansas City Missouri USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Bouchra","family":"Benettaib","sequence":"additional","affiliation":[{"name":"Formerly Bristol Myers Squibb  Princeton New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Noha","family":"Biserna","sequence":"additional","affiliation":[{"name":"Formerly Bristol Myers Squibb  Princeton New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"Poon","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb  Princeton New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Meera","family":"Patturajan","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb  Princeton New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nianhang","family":"Chen","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb  Princeton New Jersey USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mathew","family":"Simcock","sequence":"additional","affiliation":[{"name":"Celgene Ltd., a Bristol\u2010Myers Squibb Company  Uxbridge UK"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Linda","family":"Zimmerman","sequence":"additional","affiliation":[{"name":"Formerly Bristol Myers Squibb  Overland Park Kansas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"E. 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