Michelle M Chin, John S Tamaresis, Laura J Johnston, Robert Lowsky, Everett Meyer, Lori Muffly, Parveen Shiraz, Matthew J Frank, Andrew R Rezvani, Sushma Bharadwaj, Wen-Kai Weng, Judith A Shizuru, Sally Arai
{"title":"Belumosudil combination therapy for chronic graft-versus-host-disease in real-world clinical practice.","authors":"Michelle M Chin, John S Tamaresis, Laura J Johnston, Robert Lowsky, Everett Meyer, Lori Muffly, Parveen Shiraz, Matthew J Frank, Andrew R Rezvani, Sushma Bharadwaj, Wen-Kai Weng, Judith A Shizuru, Sally Arai","doi":"10.1038/s41409-024-02476-z","DOIUrl":"https://doi.org/10.1038/s41409-024-02476-z","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142667189","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}
Águeda Molinos-Quintana, Nuria Martínez-Cibrian, Anna Alonso-Saladrigues, Víctor Galán-Gómez, Rebeca Bailén, Susana Buendía-López, Carolina Fuentes-Socorro, Mi Kwon, Marta González-Vincent, Concepción Pérez de Soto, Berta González-Martínez, Susana Rives, José María Pérez-Hurtado, Valentín Ortiz-Maldonado, José Antonio Pérez-Simón
{"title":"Successful allogeneic CD34<sup>+</sup> hematopoietic stem cell boost for prolonged cytopenias following CAR T-cell therapy in B-cell acute lymphoblastic leukemia. On behalf of the Spanish Group for Hematopoietic Transplantation and Cellular Therapy (GETH-TC).","authors":"Águeda Molinos-Quintana, Nuria Martínez-Cibrian, Anna Alonso-Saladrigues, Víctor Galán-Gómez, Rebeca Bailén, Susana Buendía-López, Carolina Fuentes-Socorro, Mi Kwon, Marta González-Vincent, Concepción Pérez de Soto, Berta González-Martínez, Susana Rives, José María Pérez-Hurtado, Valentín Ortiz-Maldonado, José Antonio Pérez-Simón","doi":"10.1038/s41409-024-02473-2","DOIUrl":"10.1038/s41409-024-02473-2","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142646843","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":"Pretransplantation risk factors for positive MRD after allogeneic stem cell transplantation in AML patients: a prospective study.","authors":"Si-Qi Li, Chun-Zi Yu, Lan-Ping Xu, Yu Wang, Xiao-Hui Zhang, Huan Chen, Yu-Hong Chen, Feng-Rong Wang, Yu-Qian Sun, Chen-Hua Yan, Meng Lv, Xiao-Dong Mo, Yan-Rong Liu, Kai-Yan Liu, Xiao-Su Zhao, Xiang-Yu Zhao, Xiao-Jun Huang, Ying-Jun Chang","doi":"10.1038/s41409-024-02466-1","DOIUrl":"https://doi.org/10.1038/s41409-024-02466-1","url":null,"abstract":"<p><p>We aimed to prospectively explore the risk factors for measurable residual disease (MRD) positivity after allogeneic stem cell transplantation (allo-SCT) in AML patients (n = 478). The cumulative incidences (CIs) of post-SCT MRD positivity at 100 days, 360 days and 3 years were 4.6%, 12.1% and 18.3%, respectively. Positive pre-SCT MRD and pre-SCT active disease were risk factors for post-SCT MRD positivity at both 360 days and 3 years (P < 0.001). European LeukemiaNet (ELN) 2017 risk stratification was a risk factor for positive post-SCT MRD at 360 days (P = 0.044). A scoring system for predicting post-SCT MRD positivity at 360 days was established by using pre-SCT MRD, pre-SCT active disease and ELN 2017 risk stratification. The CI of positive post-SCT MRD at 3 years was 13.2%, 23.7%, and 43.9% for patients with scores of 0, 1, and 2, respectively (P < 0.001). Multivariate analysis demonstrated that the scoring system was associated with a higher CI of post-SCT MRD positivity, leukemia relapse and inferior survival. Our data indicate that positive pre-SCT MRD status, pre-SCT active disease, and ELN 2017 risk stratification are risk factors for positive post-SCT MRD status in AML patients.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142643708","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}
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}