Tim Richardson, Hishan Tharmaseelan, Lukas Frenzel, Philipp Gödel, Moritz Fürstenau, Pascal Nieper, Till Braun, Daniel Schütte, Michael Hallek, Christof Scheid, Udo Holtick
{"title":"Post-transplant-cyclophosphamide plus everolimus as GvHD prophylaxis in refractory T- and B-cell lymphoma.","authors":"Tim Richardson, Hishan Tharmaseelan, Lukas Frenzel, Philipp Gödel, Moritz Fürstenau, Pascal Nieper, Till Braun, Daniel Schütte, Michael Hallek, Christof Scheid, Udo Holtick","doi":"10.1038/s41409-024-02472-3","DOIUrl":"https://doi.org/10.1038/s41409-024-02472-3","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638368","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Utility of the 2024 best practice recommendations from the EBMT Cellular Therapy and Immunobiology Working Party for use of donor lymphocyte infusions after allogeneic hematopoietic stem cell transplantation.","authors":"Anne-Claire Mamez, Amandine Pradier, Sarah Morin, Federica Giannotti, Chiara Bernardi, Stavroula Masouridi-Levrat, Yves Chalandon, Federico Simonetta","doi":"10.1038/s41409-024-02458-1","DOIUrl":"https://doi.org/10.1038/s41409-024-02458-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638369","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Barbara Jeker, Laura Thalmann, Ulrike Bacher, Henning Nilius, Gaëlle Rhyner, Martin Sökler, Susanne Soltermann, Annette Winkler, Corinne Vorburger, Michael Daskalakis, Michèle Hoffmann, Thomas Pabst
{"title":"Comparing stem cell mobilization with chemotherapy and cytokine (G-CSF) versus cytokine alone in myeloma patients (MOCCCA): a randomized phase II, open-label, non-inferiority trial.","authors":"Barbara Jeker, Laura Thalmann, Ulrike Bacher, Henning Nilius, Gaëlle Rhyner, Martin Sökler, Susanne Soltermann, Annette Winkler, Corinne Vorburger, Michael Daskalakis, Michèle Hoffmann, Thomas Pabst","doi":"10.1038/s41409-024-02468-z","DOIUrl":"https://doi.org/10.1038/s41409-024-02468-z","url":null,"abstract":"<p><p>In fit patients with newly diagnosed myeloma, high-dose chemotherapy (HDCT) followed by autologous stem cell transplantation (ASCT) is considered standard of care. For mobilization of CD34+ cells for ASCT, combined cytotoxic chemotherapy and G-CSF is commonly used. However, the importance of cytostatic chemotherapy for reliable mobilization remains unclear. This prospective randomized phase II non-inferiority trial compared G-GSF only (G) compared to standard chemotherapy/G-CSF (CG) for CD34+ mobilization. The primary endpoint was a less than 15% difference in successful stem cell collection ( ≥ 5.0 × 10<sup>6</sup> CD34+ cells/kg b.w. in a single day collection procedure without additional stimulation with plerixafor) with the G regimen. 136 patients were 1:1 randomized. With an 18% difference in favor of the CG therapy, the non-inferiority margin was not maintained (95% CI 1%, 34%, p = 0.04). The median total CD34+ yield was 9.99 × 10<sup>6</sup>/kg b.w. in CG patients and 7.42 × 10<sup>6</sup>/kg b.w. in patients with G-CSF alone (p < 0.001). Ultimately, 130 (96%) patients proceeded to HDCT with ASCT. There were no differences in adverse events, hematologic engraftment, quality of life, or pain perception between the groups. Our data indicate that G-CSF only is inferior to chemotherapy with G-CSF for peripheral CD34+ stem cell mobilization. Trial registration SNCTP #: SNCTP000002952; Trials.gov #: NCT03442673.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142638366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Julia Möhlmann, Lisanne van der Ploeg, Jurgen Langenhorst, Tim Bognàr, Kim van der Elst, Marc Bierings, Alwin Huitema, Aurelia de Vries Schultink, Caroline Lindemans
{"title":"Evaluation of standard fludarabine dosing and corresponding exposures in infants and young children undergoing hematopoietic cell transplantation.","authors":"Julia Möhlmann, Lisanne van der Ploeg, Jurgen Langenhorst, Tim Bognàr, Kim van der Elst, Marc Bierings, Alwin Huitema, Aurelia de Vries Schultink, Caroline Lindemans","doi":"10.1038/s41409-024-02467-0","DOIUrl":"https://doi.org/10.1038/s41409-024-02467-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142614598","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Molecular measurable residual disease monitoring and transplant indications in NPM1 mutated acute myeloid leukemia.","authors":"Mary R Christopher, Mariam T Nawas, John L Reagan","doi":"10.1038/s41409-024-02465-2","DOIUrl":"https://doi.org/10.1038/s41409-024-02465-2","url":null,"abstract":"<p><p>NPM1 mutated acute myeloid leukemia (AML) comprises roughly 30% of all AML cases and is mainly classified as favorable or intermediate-risk according to the European Leukemia Net stratification. Some patients, however, either have a poor response to initial intensive chemotherapy or ultimately relapse. NPM1 mutations are common, generally stable at early relapse and AML specific, features which make them ideal targets for measurable residual disease (MRD) monitoring. MRD monitoring via molecular analysis during the course of treatment can inform the role of allogeneic stem cell transplantation (HCT) in first remission in patients with NPM1 mutated AML with high-risk co-occurring mutations, particularly FLT3-ITD, and in favorable risk patients who do not achieve defined molecular milestones. In this review, we evaluate the prognostic role of MRD monitoring in NPM1 mutated AML and its use as a predictive biomarker to refine risk stratification and inform decision making regarding treatment. We explore the impact of pre-HCT MRD positivity on post-HCT outcomes in this AML subset, and how HCT-related factors such as conditioning intensity may influence this risk.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142614619","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Xiaotian Zhang, Kailin Xu, Robert Peter Gale, Bin Pan
{"title":"Strategies following failure of CAR-T-cell therapy in non-Hodgkin lymphoma.","authors":"Xiaotian Zhang, Kailin Xu, Robert Peter Gale, Bin Pan","doi":"10.1038/s41409-024-02463-4","DOIUrl":"https://doi.org/10.1038/s41409-024-02463-4","url":null,"abstract":"<p><p>Several CD19 CAR-T-cell drugs are approved for safety and efficacy in advanced B-cell cancers with encouraging results. However, primary refractory and relapse are common. We critically analyze long-term data on efficacy of CD19 CAR-T-cell therapies in B-cell non-Hodgkin lymphomas from clinical trials with those of so-called real world data. We identify co-variates associated with efficacy, discuss mechanisms of relapse, summarize the data on the results of post-failure therapy including allotransplants, monoclonal and bi-specific antibodies, antibody-drug conjugates, immune checkpoint-inhibitors and repeat infusions of CAR-T-cells. We conclude, save for allotransplants, there are few data strongly supporting any of these interventions. Most trial are with few heterogeneously-treated subjects with diverse interventions and brief follow-up. Interventions need to be tailored to the cause(s) of CAR-T-cell failure. Prestly, there is not a convincingly safe and effective therapy of people failing initial CAR-T-cell therapy of B-cell non-Hodgkin lymphoma.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142614629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Matjaz Sever, Joanna Drozd-Sokolowska, Luuk Gras, Linda Koster, Frantisek Folber, Stephan Mielke, Roland Fenk, Grzegorz Basak, Jane Apperley, Jennifer Byrne, Alessandro Rambaldi, Mark Ringhoffer, Matthias Eder, Marek Trneny, Didier Blaise, Stig Lenhoff, Cecilia Isaksson, Jakob Passweg, Anu Partanen, Ioanna Sakellari, Stefan Schönland, Curly Morris, Meral Beksac, Kavita Raj, Patrick J Hayden, Donal P McLornan
{"title":"Satisfactory outcomes following a second autologous hematopoietic cell transplantation for multiple myeloma in poor stem cell mobilizers: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT.","authors":"Matjaz Sever, Joanna Drozd-Sokolowska, Luuk Gras, Linda Koster, Frantisek Folber, Stephan Mielke, Roland Fenk, Grzegorz Basak, Jane Apperley, Jennifer Byrne, Alessandro Rambaldi, Mark Ringhoffer, Matthias Eder, Marek Trneny, Didier Blaise, Stig Lenhoff, Cecilia Isaksson, Jakob Passweg, Anu Partanen, Ioanna Sakellari, Stefan Schönland, Curly Morris, Meral Beksac, Kavita Raj, Patrick J Hayden, Donal P McLornan","doi":"10.1038/s41409-024-02460-7","DOIUrl":"https://doi.org/10.1038/s41409-024-02460-7","url":null,"abstract":"<p><p>Autologous hematopoietic cell transplants (auto-HCTs) remain the standard of care for transplant-eligible MM patients. The general practice has been to undergo upfront apheresis following induction to collect sufficient number of CD34+ cells to facilitate two auto-HCTs. However, 5-30% of MM patients do not initially mobilise a sufficient number of hematopoietic stem cells and are classified as poor mobilizers (PM). We compared the baseline characteristics and outcomes of 61 PMs and 816 non-PM patients who underwent a second auto-HCT and who were enrolled in the non-interventional CALM study (NCT01362972). Only patients who collected CD34+ prior to auto-HCT1 were included. Auto-HCT2 comprised both tandem and salvage transplants. PMs were re-mobilized with plerixafor (n = 24, 39.3%) or non-plerixafor-based regimens (n = 37, 60.7%). There were no significant differences in engraftment, progression-free survival (PFS) or overall survival (OS) after the second auto-HCT between PM and non-PM patients. There was a trend to shorter PFS in PM patients undergoing salvage auto-HCT (median 9.6 vs. 12.9 months; p = 0.08) but no significant difference in OS. The median OS was 41.1 months for PM and 41.2 months for non-PM patients (p = 0.86). These data suggest that salvage mobilization is effective and does not affect overall outcomes after a second auto-HCT.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142614621","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Kavita Raj, Diderik-Jan Eikema, Sarah Lawless, Linda Koster, Desiree Kunadt, Nicolaus Kröger, Uwe Platzbecker, Matthias Stelljes, Wolfgang Bethge, Tobias Holderried, Renato Fanin, Robert Zeiser, Jürgen Kuball, Véronique Leblond, Emma Nicholson, Jakob Passweg, Victoria Potter, Jacques-Olivier Bay, Ali Bazarbachi, Lucía López Corral, Carmelo Gurnari, Christof Scheid, Joanna Drozd-Sokolowska, Treen Curly Morris, Patrick Hayden, Ibrahim Yakoub-Agha, Marie Robin, Donal P McLornan
{"title":"Allogeneic hematopoietic cell transplantation for therapy-related myeloid neoplasms arising following treatment for multiple myeloma: a retrospective study on behalf of the Chronic Malignancies Working Party of the EBMT.","authors":"Kavita Raj, Diderik-Jan Eikema, Sarah Lawless, Linda Koster, Desiree Kunadt, Nicolaus Kröger, Uwe Platzbecker, Matthias Stelljes, Wolfgang Bethge, Tobias Holderried, Renato Fanin, Robert Zeiser, Jürgen Kuball, Véronique Leblond, Emma Nicholson, Jakob Passweg, Victoria Potter, Jacques-Olivier Bay, Ali Bazarbachi, Lucía López Corral, Carmelo Gurnari, Christof Scheid, Joanna Drozd-Sokolowska, Treen Curly Morris, Patrick Hayden, Ibrahim Yakoub-Agha, Marie Robin, Donal P McLornan","doi":"10.1038/s41409-024-02462-5","DOIUrl":"https://doi.org/10.1038/s41409-024-02462-5","url":null,"abstract":"<p><p>Therapy-related myeloid neoplasms (t-MN) are a complication of multiple myeloma (MM) treatment. Our retrospective, EBMT registry study included 157 such patients allografted (allo-HCT) between 2006 and 2018. Most patients (130) had a prior autologous HCT. Fifty-seven (36.4%) were transplanted for t-AML and 100 (63.6%) for t-MDS. Median times from MM and t-MN diagnoses to allo-HCT were 72.6 (interquartile range (IQR), 46.1-102.9) and 6.4 (IQR, 3.9-9.4) months. Fifty-eight (38.4%) t-MN patients were in complete remission (CR) at allo-HCT predominantly conditioned with reduced intensity (70.3%). With a median follow-up of 64.9 (95% CI: 39-76) months, relapse incidence (RI) from MM at 1 and 5 years was 4% (0-10%) and 12% (2-22%), respectively, with few deaths (n = 3) only due to MM disease progression, whereas t-MN RI and non-relapse mortality (NRM) at 1 and 5 years were 35% (95% CI 28-43%) and 45% (95% CI: 36-53%) and 20% (95% CI 13-26%) and 31% (95% CI: 23-39%). Overall survival (OS) and progression-free survival (PFS) estimates at 1 and 5 years were 55% (95% CI: 47-63%) and 27% (95% CI: 19-35%) and 45% (95% CI 36-53%) and 24% (95% CI 16-32%). Older (>65 years) t-MN patients with high-risk cytogenetics do not benefit from allo-HCT.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142614534","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Emily C Liang, Kai Rejeski, Teng Fei, Aya Albittar, Jennifer J Huang, Andrew J Portuguese, Qian Wu, Sandeep Raj, Marion Subklewe, Roni Shouval, Jordan Gauthier
{"title":"Correction: Development and validation of an automated computational approach to grade immune effector cell-associated hematotoxicity.","authors":"Emily C Liang, Kai Rejeski, Teng Fei, Aya Albittar, Jennifer J Huang, Andrew J Portuguese, Qian Wu, Sandeep Raj, Marion Subklewe, Roni Shouval, Jordan Gauthier","doi":"10.1038/s41409-024-02453-6","DOIUrl":"https://doi.org/10.1038/s41409-024-02453-6","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142614587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}