{"doi":"10.1002/ajh.27168","title":"Safety and efficacy of a new high‐dose regimen of panobinostat, gemcitabine, busulfan, and melphalan for 1st or 2nd salvage <scp>ASCT</scp> for refractory/relapsed or high‐risk myeloma: Matched‐pair comparisons with concurrent control cohorts","abstract":"Improvement of autologous stem-cell 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 18-65 years with relapsed/refractory or high-risk myeloma and adequate end-organ function were eligible. Treatment included panobinostat (20 mg/day, days -9 to -2) and GemBuMel (days -8 to -2). Patients were enrolled in 1st (ASCT-1) or 2nd ASCT (ASCT-2) 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-risk cytogenetics, and response at ASCT. We enrolled 80 patients, 48 and 32 in the ASCT-1 and ASCT-2 cohorts, respectively; in these two cohorts, high-risk 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-related mortality (TRM) occurred in two ASCT-2 patients. One-year PFS rates were 69% (ASCT-1) and 72% (ASCT-2); 1-year OS rates were 79% (ASCT-1) and 84% (ASCT-2). Minimal residual disease negativity improved after ASCT-1 (8.5%-23%, p < .0001) and ASCT-2 (34%-55%, p = .02), which correlated with improved outcomes. Trial patients and controls (N = 371) had similar TRM and post-ASCT maintenance. Trial patients had better PFS after either a 1st (p = .02) or a 2nd ASCT (p = .04) than matched-paired control patients. In conclusion, panobinostat/GemBuMel is effective for relapsed/refractory or high-risk myeloma patients, with better PFS than concurrent matched controls receiving melphalan or BuMel.","journal":"American Journal of Hematology","year":2023,"id":415031,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":0,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.9575,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2023-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":654378,"name":"Zixi Yang","orcid":null,"position":1,"is_corresponding":false},{"id":418650,"name":"Benigno C. Valdez","orcid":"0000-0003-2471-8247","position":2,"is_corresponding":false},{"id":525487,"name":"Suprateek Kundu","orcid":"0000-0002-1767-4875","position":3,"is_corresponding":false},{"id":339779,"name":"Qaiser Bashir","orcid":"0000-0002-4086-6453","position":4,"is_corresponding":false},{"id":730116,"name":"Jeremy Ramdial","orcid":"0000-0002-8984-3420","position":5,"is_corresponding":false},{"id":652227,"name":"Samer A. Srour","orcid":"0000-0002-6693-2025","position":6,"is_corresponding":false},{"id":407651,"name":"Muzaffar H. Qazilbash","orcid":"0000-0003-2876-2886","position":7,"is_corresponding":false},{"id":104774,"name":"Yago Nieto","orcid":"0000-0001-6034-8939","position":0,"is_corresponding":true}],"reference_count":29,"raw_metadata":null,"created_at":"2026-07-19T01:22:08.814145Z","pmid":"38100199","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}