Transplantation and Cellular Therapy最新文献

{"title":"Real-World Outcomes of Upfront Autologous Hematopoietic Stem Cell Transplantation in Patients With Newly Diagnosed Multiple Myeloma With Deletion 17p","authors":"Curtis Marcoux ,&nbsp;Oren Pasvolsky ,&nbsp;Denái R. Milton ,&nbsp;Mark R. Tanner ,&nbsp;Qaiser Bashir ,&nbsp;Samer Srour ,&nbsp;Neeraj Saini ,&nbsp;Paul Lin ,&nbsp;Jeremy Ramdial ,&nbsp;Yago Nieto ,&nbsp;Guilin Tang ,&nbsp;Hans C. Lee ,&nbsp;Krina K. Patel ,&nbsp;Partow Kebriaei ,&nbsp;Amna Ahmed ,&nbsp;Yosra Aljawai ,&nbsp;Sheeba K. Thomas ,&nbsp;Robert Z. Orlowski ,&nbsp;Elizabeth J. Shpall ,&nbsp;Richard E. Champlin ,&nbsp;Muzaffar H. Qazilbash","doi":"10.1016/j.jtct.2024.10.011","DOIUrl":"10.1016/j.jtct.2024.10.011","url":null,"abstract":"<div><div>Despite tremendous advancements in multiple myeloma (MM) therapeutics, outcomes remain heterogeneous, heavily influenced by clinical and cytogenetic factors. Among these, deletion of the short arm of chromosome 17 (del(17p)) is a strong predictor of poor prognosis. The aim of this study was to evaluate real-world outcomes in patients with newly diagnosed MM (NDMM) with del(17p) undergoing upfront autologous hematopoietic stem cell transplantation (auto-HCT). We conducted a single-center retrospective analysis of patients with NDMM who underwent upfront auto-HCT at MD Anderson Cancer Center between 2008 and 2018. Primary endpoints were progression-free survival (PFS) and overall survival (OS), with secondary endpoints being hematological response and measurable residual disease (MRD) status postauto-HCT. MRD status in the bone marrow biopsy was evaluated using 8-color next-generation flow cytometry with a sensitivity of 1/10^–5 cells. One hundred and fifteen patients were included (55% male). Median age at auto-HCT was 62 years (range 34 to 83). The median del(17p) clone size was 20%, with 51 (53%) patients having clone sizes &gt;20% and 15 (15%) patients having clone sizes &gt;55%. Additional high-risk cytogenetic abnormalities included t(4;14) in 15 (13%) patients, t(14;16) in 8 (7%) patients, and 1q21+ in 25 (22%) patients. After induction, 10% of patients achieved ≥ CR, and 50% achieved ≥ VGPR, with 25% having MRD-negative ≥ VGPR. Post-transplant, 42% achieved ≥ CR, and 83% achieved ≥ VGPR as best response, with 55% (48/87) having MRD-negative ≥ VGPR. With a median follow-up of 31.4 months (range 3.1 to 199.1), median PFS and OS for the entire cohort were 19.9 and 71.5 months, respectively, and 5-year OS was 53%. Concurrent del(17p) and t(4;14) were associated with significantly worse outcomes, with median PFS and OS of 11.5 and 22.4 months, respectively. In multivariable analysis (MVA), female sex was associated with worse PFS (HR [95% CI] 2.87 [1.75 to 4.72], <em>P</em> &lt; .001), while MRD negative CR post-transplant (0.35 [0.18 to 0.68], <em>P</em> = .002) and maintenance therapy (0.46 [0.27 to 0.77], <em>P</em> = 0.003) were associated with better PFS. In MVA for OS, female sex (2.22 [1.18 to 4.17], <em>P</em> = 0.013) and the presence of t(4;14) (2.55 [1.09 to 5.95], <em>P</em> = 0.030) were associated with worse OS, whereas Karnofsky Performance Status of ≥90 (0.47 [0.23 to 0.94], <em>P</em> = 0.034) was associated with better OS. This study affirms del(17p) as a high-risk abnormality with unfavorable outcomes despite modern therapies. The co-occurrence of del(17p) and t(4;14) was associated with particularly poor outcomes. Novel approaches are needed for this high-risk subgroup.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 12.e1-12.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142508737","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of the Type of Tyrosine Kinase Inhibitor (imatinib or dasatinib) Used Before allo-HCT on Outcome of Patients with Philadelphia-Positive Acute Lymphoblastic Leukemia. A Study on Behalf of the Acute Leukemia Working Party of the EBMT","authors":"Sebastian Giebel ,&nbsp;Myriam Labopin ,&nbsp;Zinaida Peric ,&nbsp;Jakob Passweg ,&nbsp;Didier Blaise ,&nbsp;Urpu Salmenniemi ,&nbsp;David Beauvais ,&nbsp;Péter Reményi ,&nbsp;Patrice Chevallier ,&nbsp;Stephan Mielke ,&nbsp;Tobias Gedde-Dahl ,&nbsp;Jan J. Cornelissen ,&nbsp;Marie Balsat ,&nbsp;Gesine Bug ,&nbsp;Ali Bazarbachi ,&nbsp;Eolia Brissot ,&nbsp;Arnon Nagler ,&nbsp;Fabio Ciceri ,&nbsp;Mohamad Mohty","doi":"10.1016/j.jtct.2024.07.016","DOIUrl":"10.1016/j.jtct.2024.07.016","url":null,"abstract":"<div><div>The use of tyrosine kinase inhibitors (TKIs) during induction and consolidation, followed by allogeneic hematopoietic cell transplantation (allo-HCT), is a standard of care for patients with Philadelphia (Ph)-positive acute lymphoblastic leukemia (ALL). The goal of this study was to compare results of allo-HCT according to the type of TKI used pre-transplant, either imatinib, dasatinib or both. This was a retrospective, registry-based analysis including adult patients with Ph-positive ALL treated with allo-HCT between years 2010-2022. The analysis included 606 patients pre-treated with imatinib, 163 with dasatinib and 94 with both imatinib and dasatinib. Allo-HCTs were performed in first complete remission from either unrelated (56%), matched sibling (36%) or haploidentical donors (8%). Relapse incidence at 2 years was 26% in the imatinib group and 21% in the dasatinib group and 19% in the imatinib + dasatinib group (<em>P = .</em>06) while non-relapse mortality was 19%, 15%, and 23%, respectively (<em>P = .</em>37). No significant differences were found for leukemia-free survival (55% vs. 63% vs. 58%, <em>P = .</em>11) and overall survival (72% vs. 76% vs. 65%, <em>P = .</em>32). The incidence of grade 2-4 acute graft-versus-host disease (GVHD) and chronic GVHD was comparable across study groups, while the incidence of grade 3-4 acute GVHD was significantly increased for patients pre-treated with dasatinib alone (20%) than in the imatinib group (10%) or imatinib + dasatinib group (13%) (<em>P = .</em>002). On multivariate analysis a chance of GVHD and relapse-free survival (GRFS) was significantly decreased while the risk of grade 3-4 acute GVHD was increased for the dasatinib compared to imatinib group (hazard ratio, HR = 1.27, <em>P</em> = .048 and HR = 2.26, <em>P</em> = .0009, respectively). This study provides no evidence for the advantage of one TKI over another in terms of LFS and OS. However, the use of dasatinib is associated with increased risk of severe acute GVHD and decreased GRFS.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 14.e1-14.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141789116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Novel Drug Combinations and Donor Lymphocyte Infusions Allow Prolonged Disease Control in Multiple Myeloma Patients Relapsing after Allogeneic Transplantation","authors":"Chiara Nozzoli ,&nbsp;Martina Pucillo ,&nbsp;Luisa Giaccone ,&nbsp;Alessandro Rambaldi ,&nbsp;Maria Teresa Lupo Stanghellini ,&nbsp;Edoardo Benedetti ,&nbsp;Domenico Russo ,&nbsp;Nicola Mordini ,&nbsp;Silvia Mangiacavalli ,&nbsp;Paolo Bernasconi ,&nbsp;Matteo Parma ,&nbsp;Paola Carluccio ,&nbsp;Piero Galieni ,&nbsp;Paolo Rivela ,&nbsp;Massimo Martino ,&nbsp;Patrizia Chiusolo ,&nbsp;Miriam Isola ,&nbsp;Maria De Martino ,&nbsp;Elena Oldani ,&nbsp;Eliana Degrandi ,&nbsp;Francesca Patriarca","doi":"10.1016/j.jtct.2024.10.015","DOIUrl":"10.1016/j.jtct.2024.10.015","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Although allogeneic stem cell transplantation (allo-SCT) is curative for only a minority of patients with multiple myeloma (MM), patients who relapse after allo-SCT can experience long-term survival, suggesting a synergy between antimyeloma drugs administered after allo-SCT and donor T cells. We retrospectively evaluated the outcome of MM patients reported to the Gruppo Italiano Trapianto Midollo Osseo e Terapia Cellulare (GITMO) network who underwent allo-SCT between 2009 and 2018, to identify predictors of long-term outcome in the whole population (242 patients) and predictors of prolonged overall survival (OS) after relapse in the subgroup of relapsed patients (118 patients). In the whole population, at a median follow-up of 40.9 months after allo-SCT, the median duration of OS and progression-free survival (PFS) were 39.4 and 19.0 months after allo-SCT, respectively. The cumulative incidence of nonrelapse mortality (NRM) was 10.3% at 1 year and 27.6% at 5 years. The cumulative incidence of grade II-IV acute graft-versus-host disease (GVHD) was 19.8%, and the 5-year cumulative incidence of moderate or severe chronic GVHD was 31.8%. In the multivariate model, older age at transplantation (&lt;em&gt;P&lt;/em&gt; = .020), receipt of &gt;2 lines of therapy before allo-SCT (&lt;em&gt;P&lt;/em&gt; = .003), and transplantation from an unrelated or haploidentical donor (&lt;em&gt;P&lt;/em&gt; = .025) were significant factors associated with reduced OS. Relapse after allo-SCT occurred in 118 patients (59%) at a median of 14.3 months (interquartile range, 7.2 to 26.9 months). Twenty patients (17%) received only steroids, radiotherapy, or supportive care; 41 (35%) received 1 line of salvage treatment; 23 (19%) received 2 lines of salvage treatment; and 34 (29%) received 3 or 4 lines of salvage treatment. Nine patients were treated exclusively with chemotherapy, 9 received at least 1 salvage treatment including immunomodulating agents, 43 patients were treated with at least 1 rescue therapy including proteasome inhibitors, and 37 patients received at least 1 salvage treatment including monoclonal antibodies (33 with daratumumab, 1 with elotuzumab, 1 with isatuximab, and 2 with belantamab). The median OS of relapsed patients was 38.5 months from allo-SCT and 20.2 months from relapse. In multivariate analysis, OS after relapse was significantly prolonged in patients with a longer time to relapse after allo-SCT (time to relapse 6 to 24 months, &lt;em&gt;P&lt;/em&gt; = .016; time to relapse ≥24 months, &lt;em&gt;P&lt;/em&gt; &lt; .001) and in those who had received at least 3 lines of salvage treatment (&lt;em&gt;P&lt;/em&gt; &lt; .036) and donor lymphocyte infusion (DLI) (&lt;em&gt;P&lt;/em&gt; = .020). In this study, patients who underwent transplantation in early phases of disease and with an HLA-identical sibling donor had the best chance of long-term survival. Late relapse after allo-SCT, multiple courses of salvage treatment, and an association with DLI could allow for long-term disease control in patients who experienced rel","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 26.e1-26.e13"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142590776","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Officers and Directors of ASTCT","authors":"","doi":"10.1016/S2666-6367(24)00820-0","DOIUrl":"10.1016/S2666-6367(24)00820-0","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Page A5"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143150145","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Training in Transplantation and Cellular Therapy in Latin America: A Cross-Sectional Study of the LABMT","authors":"Andrés Noyola-Pérez ,&nbsp;Rafaella Ribas-Muratori ,&nbsp;Marco A. Vargas-Hernández ,&nbsp;Laura Saavedra-Salazar ,&nbsp;Cristóbal Frutos ,&nbsp;Carmem Bonfim ,&nbsp;Fernando Barroso-Duarte ,&nbsp;Sebastián Galeano ,&nbsp;Gregorio Jaimovich ,&nbsp;Amado Karduss ,&nbsp;Andrés Gómez-De León ,&nbsp;Latin-American Bone Marrow Transplantation Group (LABMT)","doi":"10.1016/j.jtct.2024.09.025","DOIUrl":"10.1016/j.jtct.2024.09.025","url":null,"abstract":"<div><div>Hematopoietic cell transplantation (HCT) is a complex and resource-intensive procedure that has become a critical treatment for certain hematologic conditions. However, in Latin America, access to HCT is limited compared to high-income countries, in part due to a lack of standardized training programs for HCT professionals. To address this gap, the Latin-American Bone Marrow Transplantation Group conducted a cross-sectional study to assess the current state of training programs in HCT and cellular therapy across the region. This study aimed to describe and analyze the availability, characteristics, and challenges of HCT training programs in Latin America, with a focus on identifying barriers and proposing solutions for improvement. A cross-sectional survey was sent to 127 recognized HCT centers across 14 Latin-American countries in December 2022. The survey collected data on institutional characteristics, training program structure, costs, and barriers to program development. Descriptive statistics were used to summarize the data, and comparative analyses were performed using Chi-square and Mann–Whitney tests. Of the 127 centers surveyed, 50 (39%) responded, with the majority located in Brazil (34%) and Mexico (30%). Among the respondents, 64% (<em>n</em> = 32) offered formal training programs lasting 6 months or longer. The most significant barriers reported were lack of funding (<em>n</em> = 21), limited number of transplant procedures (<em>n</em> = 15), and a shortage of qualified professors (<em>n</em> = 11). Proposed solutions included increasing student mobility opportunities (<em>n</em> = 28), enhancing program quality (<em>n</em> = 27), and improving access to funding (<em>n</em> = 15). Only 6% of programs offered exposure to CAR-T therapy, and fewer than half of the centers provided international rotations. This study highlights significant disparities in HCT training programs across Latin America, with most countries lacking access to formalized training. While Brazil and Mexico serve as regional hubs, other nations have limited or no training opportunities. Addressing these gaps through increased funding, international collaborations, and standardized curricula is essential to improving HCT training and ultimately patient care in the region.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 47.e1-47.e9"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475655","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Shift from Widespread to Tailored Antifungal Prophylaxis in Lymphoma Patients Treated with CD19 CAR T Cell Therapy: Results from a Large Retrospective Cohort","authors":"Giovanna Melica ,&nbsp;Alejandro Luna de Abia ,&nbsp;Gunjan L. Shah ,&nbsp;Sean Devlin ,&nbsp;Magdalena Corona ,&nbsp;Joshua Fein ,&nbsp;Parastoo B. Dahi ,&nbsp;Sergio A. Giralt ,&nbsp;Richard J. Lin ,&nbsp;M. Lia Palomba ,&nbsp;Allison Parascondola ,&nbsp;Jae Park ,&nbsp;Gilles Salles ,&nbsp;Amethyst Saldia ,&nbsp;Michael Scordo ,&nbsp;Roni Shouval ,&nbsp;Miguel-Angel Perales ,&nbsp;Susan K. Seo","doi":"10.1016/j.jtct.2024.10.010","DOIUrl":"10.1016/j.jtct.2024.10.010","url":null,"abstract":"<div><div>Patients undergoing CD19 chimeric antigen receptor (CAR)–T cell therapy exhibit multiple immune deficits that may increase their susceptibility to infections. Invasive fungal infections (IFIs) are life-threatening events in the setting of hematologic diseases. However, there is ongoing debate regarding the optimal role and duration of antifungal prophylaxis in this specific patient population. The objective of this study was to provide a comprehensive overview of the evolution of IFI prophylactic strategies over time and to assess IFI incidence rates in a cohort of patients with relapsed or refractory (R/R) lymphoma treated with CAR-T cell therapy. A single-center retrospective study was conducted on a cohort of patients with R/R B cell lymphoma treated with CD19 CAR-T cell therapy between April 2016 and March 2023. Group A (April 2016–August 2020) consisted of patients primarily treated with fluconazole, irrespective of their individual IFI risk profile. In Group B (September 2020–March 2023) antifungal prophylaxis was recommended only for high-risk patients. Overall, 330 patients were included. Antifungal prophylaxis was prescribed to 119/142 (84%) patients in Group A and 58/188 (31%) in Group B (<em>P</em> &lt; .001). Anti-mold azoles were prescribed to 8 (5.6%) patients in Group A and 21 (11.2%) patients in Group B. In Group A, 42 (29%) patients were switched to another antifungal, 9 (21%) because of toxicity, with 6 cases of transaminitis and 3 cases of prolonged QTc. In Group B, 21 (11.2%) patients were switched to the antifungal drug, mainly from fluconazole or micafungin to a mold-active agent following revised guidelines. No difference was found in liver toxicity between the two groups at infusion, day 10, and day 30. No significant differences were observed between the groups. IFIs following CAR-T cell therapy were rare, with 1 case of cryptococcal meningoencephalitis in group A (.7%) and 1 case of invasive aspergillosis in Group B (.5%), both occurring in patients on micafungin prophylaxis. In this large single-center cohort of patients with R/R lymphoma treated with CAR-T cells, we show that individualized prophylaxis, alongside careful management of CAR-T cell–related toxicities such as CRS, was associated with a very low IFI rate, avoiding the risk of unnecessary toxicities, drug–drug interactions, and high costs.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 36-44"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11780678/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142508738","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Representativeness of Blood and Marrow Transplant Clinical Trials Network (BMT CTN) Trial Participants","authors":"Manmeet Kaur ,&nbsp;Mary M. Horowitz ,&nbsp;Adam Mendizabal ,&nbsp;Min Chen ,&nbsp;Amy Foley ,&nbsp;Jeffery J. Auletta ,&nbsp;Steven Devine ,&nbsp;Anita D'Souza","doi":"10.1016/j.jtct.2024.10.014","DOIUrl":"10.1016/j.jtct.2024.10.014","url":null,"abstract":"<div><div>Underrepresentation by race and ethnicity in oncology clinical trials, including those of hematopoietic cell transplantation (HCT), is a known challenge. This analysis studied accrual on Blood and Marrow Transplant Clinical Trials Network (BMT CTN) trials conducted in 2014 to 2020 by race/ethnicity, age, and sex, comparing these characteristics with those of potentially eligible patients identified from the Surveillance, Epidemiology, and End Results (SEER) and Center for International Blood and Marrow Transplant Research (CIBMTR) databases for the disease, age, and years of interest of BMT CTN studies. Five BMT CTN trials met the inclusion criteria, including 1 autologous HCT trial and 4 allogeneic HCT trials. Two studies focused on multiple myeloma (BMT CTN 1302 and 1401), 2 studies focused on graft-versus-host disease (GVHD) treatment (BMT CTN 1301 and 1501), and 1 study focused on post-HCT maintenance therapy in FLT3⁺ acute myelogenous leukemia (BMT CTN 1506). A decline in the proportion of patients from minority racial and ethnic groups was seen from the SEER population to trial enrollees, with the largest drop seen between the SEER population and all patients who underwent HCT (on or off trial) at US transplant centers. Allogeneic HCT trials that allowed alternative donor graft sources had less decrease from the SEER population. No decrease in clinical trial enrollment was seen with respect to older age and female HCT recipients. This study provides insight into the underrepresentation of racial and ethnic minority patients in BMT CTN clinical trials, owing largely to lack of access to HCT in general. Pathways expanding access to donors and improving the outreach of HCT programs to underserved populations are needed to improve access to clinical trials.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 49-57"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142569178","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Challenging the Status Quo: Multi-level Solutions for Equitable Hematopoietic Cell Transplantation Clinical Trial Representation","authors":"Kristine A. Karvonen","doi":"10.1016/j.jtct.2024.12.014","DOIUrl":"10.1016/j.jtct.2024.12.014","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 7-9"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143012349","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Profile of a Pioneer: Eliane Gluckman","authors":"Joanne Kurtzberg","doi":"10.1016/j.jtct.2024.12.012","DOIUrl":"10.1016/j.jtct.2024.12.012","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 1-3"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143012351","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Pilot Study of UM171-Expanded Cord Blood Grafts for Tandem Auto/Allogeneic Hematopoietic Cell Transplant in High and Ultra-High-Risk Myeloma Patients","authors":"Jean Roy ,&nbsp;Sandra Cohen ,&nbsp;Guy Sauvageau ,&nbsp;Imran Ahmad ,&nbsp;Valentyn Fournier ,&nbsp;Rafik Terra ,&nbsp;Pierre Caudrelier ,&nbsp;Stéphanie Thiant ,&nbsp;Gabrielle Thauvette ,&nbsp;Nadia Bambace ,&nbsp;Jean-Sébastien Delisle ,&nbsp;Silvy Lachance ,&nbsp;Thomas Kiss ,&nbsp;Léa Bernard ,&nbsp;Denis Claude Roy ,&nbsp;Olivier Veilleux ,&nbsp;Richard LeBlanc","doi":"10.1016/j.jtct.2024.10.008","DOIUrl":"10.1016/j.jtct.2024.10.008","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Multiple myeloma (MM) remains associated with a poor outcome, particularly in patients with advanced disease and high-risk (HR) cytogenetics. To date, the only curative treatment is allogeneic (allo) hematopoietic cell transplantation (HCT), but high incidences of graft versus host disease (GVHD), nonrelapse mortality (NRM) and disease progression remain important obstacles. Cord blood (CB) transplantation has been associated with low rates of relapse and chronic (c) GVHD, but its use has declined because of high incidences of infections, severe acute GVHD and high NRM. In other hematologic malignancies, UM171-expanded CB transplants have led to improved outcomes, allowing for the selection of smaller, better HLA-matched units. We aimed to investigate the safety and feasibility of single UM171-expanded single CB unit transplantation in frontline tandem auto/allo HCT for HR/ultra-HR MM patients. Newly diagnosed MM patients ≤ 65 years with an ISS stage II/III and del(17p), t(4;14), t(14;16), t(14;20), del(1p) or +1q, R-ISS 3, ≥ 2 cytogenetic abnormalities, or plasma cell leukemia without a sibling donor and availability of a 5-7/8 matched CB graft with ≥ 0.5 x 10&lt;sup&gt;5&lt;/sup&gt; CD34+/kg and ≥ 1.5 x 10&lt;sup&gt;7&lt;/sup&gt; TNCs/kg were eligible to this phase I/II prospective study (ClinicalTrials.gov NCT03441958). After induction and autologous HCT, patients received a reduced intensity conditioning regimen and were infused with 7-day UM171-expanded CD34+ cells, along with the lymphocytes contained in the CD34-negative fraction. The primary endpoints were feasibility of UM171 expansion, safety, kinetics of engraftment, incidences and maximum grades of acute and cGVHD at 1 and 2 years, assessment of measurable residual disease (MRD) and quality of life (QoL). Between 05/2018 and 11/2021, 20 patients were enrolled. One patient had an unsuccessful CB expansion with UM171, leaving 19 patients with a median age of 56 years. Median CD34+ cell dose infused after expansion was 4.62 x 10&lt;sup&gt;6&lt;/sup&gt;/kg (range: 0.79 to 5.76). Median times to achieve absolute neutrophil counts of 0.1 and 0.5 x 10&lt;sup&gt;9&lt;/sup&gt;/L were D+6 and D+10.5; median time to reach ≥ 20 x 10&lt;sup&gt;9&lt;/sup&gt;/L platelets was D+36. Full donor chimerism was achieved in all cell lineages by D+120 in recipients of reduced intensity conditioning. Cumulative incidences of grade II-IV, grade III-IV acute GVHD and moderate/severe cGVHD at 12 months were 68.4% (95% CI: 46 to 90), 5.3% (95% CI: 0% to 16%), and 10.5% (95% CI: 0% to 25%), respectively. With a median follow-up of 2.9 years (range: 0.46 to 5.3), cumulative incidences of relapse, PFS, OS and NRM at 3 years were 36.8% (95% CI: 14 to 59), 47.4% (95% CI: 29 to 76), 68.4% (95% CI: 50 to 93) and 15.8% (95%CI: 0 to 33), respectively. Median time to complete immunosuppression discontinuation was D+238. No unexpected adverse events were observed. Only one of 7 patients alive at 2 years with negative MRD at transplant has relapsed. Non-relapsing patients ","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 1","pages":"Pages 34.e1-34.e14"},"PeriodicalIF":3.6,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142475642","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}