{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,5,13]],"date-time":"2025-05-13T14:19:50Z","timestamp":1747145990404,"version":"3.40.5"},"reference-count":30,"publisher":"Wiley","issue":"2","license":[{"start":{"date-parts":[[2023,12,15]],"date-time":"2023-12-15T00:00:00Z","timestamp":1702598400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"funder":[{"DOI":"10.13039\/100004336","name":"Novartis","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004336","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["P30 CA016672"],"award-info":[{"award-number":["P30 CA016672"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["American J Hematol"],"published-print":{"date-parts":[[2024,2]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Improvement of autologous stem\u2010cell transplantation (ASCT) for myeloma is needed. Building on our prior work, we prospectively evaluated panobinostat and gemcitabine\/busulfan\/melphalan (GemBuMel) with ASCT in this population. Patients aged 18\u201365\u2009years with relapsed\/refractory or high\u2010risk myeloma and adequate end\u2010organ function were eligible. Treatment included panobinostat (20\u2009mg\/day, days \u22129 to \u22122) and GemBuMel (days \u22128 to \u22122). Patients were enrolled in 1st (ASCT\u20101) or 2nd ASCT (ASCT\u20102) cohorts. We compared their outcomes with all our other concurrent ASCT patients who met eligibility criteria but received melphalan or BuMel off study, matched for age, prior therapy lines, high\u2010risk cytogenetics, and response at ASCT. We enrolled 80 patients, 48 and 32 in the ASCT\u20101 and ASCT\u20102 cohorts, respectively; in these two cohorts, high\u2010risk cytogenetics were noted in 33 and 15 patients, respectively; unresponsive disease in 12 and 11 patients, respectively, after a median of 2 and 3 therapy lines, respectively. Transplant\u2010related mortality (TRM) occurred in two ASCT\u20102 patients. One\u2010year PFS rates were 69% (ASCT\u20101) and 72% (ASCT\u20102); 1\u2010year OS rates were 79% (ASCT\u20101) and 84% (ASCT\u20102). Minimal residual disease negativity improved after ASCT\u20101 (8.5%\u201323%, <jats:italic>p<\/jats:italic>\u2009&lt;\u2009.0001) and ASCT\u20102 (34%\u201355%, <jats:italic>p<\/jats:italic>\u2009=\u2009.02), which correlated with improved outcomes. Trial patients and controls (<jats:italic>N<\/jats:italic>\u2009=\u2009371) had similar TRM and post\u2010ASCT maintenance. Trial patients had better PFS after either a 1st (<jats:italic>p<\/jats:italic>\u2009=\u2009.02) or a 2nd ASCT (<jats:italic>p<\/jats:italic>\u2009=\u2009.04) than matched\u2010paired control patients. In conclusion, panobinostat\/GemBuMel is effective for relapsed\/refractory or high\u2010risk myeloma patients, with better PFS than concurrent matched controls receiving melphalan or BuMel.<\/jats:p>","DOI":"10.1002\/ajh.27168","type":"journal-article","created":{"date-parts":[[2023,12,15]],"date-time":"2023-12-15T18:37:00Z","timestamp":1702665420000},"page":"245-253","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":0,"title":["Safety and efficacy of a new high\u2010dose regimen of panobinostat, gemcitabine, busulfan, and melphalan for 1st or 2nd salvage <scp>ASCT<\/scp> for refractory\/relapsed or high\u2010risk myeloma: Matched\u2010pair comparisons with concurrent control cohorts"],"prefix":"10.1002","volume":"99","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6034-8939","authenticated-orcid":false,"given":"Yago","family":"Nieto","sequence":"first","affiliation":[{"name":"Department of Stem Cell Transplantation and Cellular Therapy The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zixi","family":"Yang","sequence":"additional","affiliation":[{"name":"Department of Biostatistics The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Benigno C.","family":"Valdez","sequence":"additional","affiliation":[{"name":"Department of Stem Cell Transplantation and Cellular Therapy The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Suprateek","family":"Kundu","sequence":"additional","affiliation":[{"name":"Department of Biostatistics The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-4086-6453","authenticated-orcid":false,"given":"Qaiser","family":"Bashir","sequence":"additional","affiliation":[{"name":"Department of Stem Cell Transplantation and Cellular Therapy The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeremy","family":"Ramdial","sequence":"additional","affiliation":[{"name":"Department of Stem Cell Transplantation and Cellular Therapy The University of Texas MD Anderson Cancer Center  Houston Texas USA"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6693-2025","authenticated-orcid":false,"given":"Samer","family":"Srour","sequence":"additional","affiliation":[{"name":"Department of Stem Cell Transplantation and Cellular Therapy The University of Texas MD Anderson Cancer Center  Houston Texas 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