Pub Date : 2025-12-27DOI: 10.1038/s41409-025-02790-0
Fabio Serpenti, Nicole Galli, Francesca Cavallaro, Kordelia Barbullushi, Nicola Cecchi, Maria Goldaniga, Francesco Passamonti, Giorgia Saporiti
{"title":"Danazol treatment for poor graft function after hematopoietic stem cell transplantation: a single centre experience","authors":"Fabio Serpenti, Nicole Galli, Francesca Cavallaro, Kordelia Barbullushi, Nicola Cecchi, Maria Goldaniga, Francesco Passamonti, Giorgia Saporiti","doi":"10.1038/s41409-025-02790-0","DOIUrl":"10.1038/s41409-025-02790-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 3","pages":"360-363"},"PeriodicalIF":5.2,"publicationDate":"2025-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846311","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}
Pub Date : 2025-12-27DOI: 10.1038/s41409-025-02792-y
Marco Cioce, Simona Sica, Sarah Jayne Liptrott, Fabio Lamberti, Simona Calza, Chiara Cannici, Orejeta Diamanti, Alfonso Parisi, Laura Orlando, Stefano Botti, Carmen Nuzzo, Giuseppe Vetrugno, Patrizia Laurenti, Chiara Visintini
{"title":"Differences between Physicians’ and Nurses’ Perspectives in Defining Core Nursing-Sensitive Outcomes in Adult HSCT and CAR-T Therapy: An Interdisciplinary Italian Delphi Consensus","authors":"Marco Cioce, Simona Sica, Sarah Jayne Liptrott, Fabio Lamberti, Simona Calza, Chiara Cannici, Orejeta Diamanti, Alfonso Parisi, Laura Orlando, Stefano Botti, Carmen Nuzzo, Giuseppe Vetrugno, Patrizia Laurenti, Chiara Visintini","doi":"10.1038/s41409-025-02792-y","DOIUrl":"10.1038/s41409-025-02792-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 3","pages":"364-368"},"PeriodicalIF":5.2,"publicationDate":"2025-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846338","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}
Pub Date : 2025-12-27DOI: 10.1038/s41409-025-02787-9
Abdul-Hamid Bazarbachi, Divaya Bhutani, Michael Sang Hughes, Suzanne Lentzsch, Markus Mapara, Pawel Muranski, Ran Reshef, Tamna Wangjam, Rajshekhar Chakraborty
The treatment landscape for multiple myeloma is rapidly evolving, driven by T-cell-mediated tumor responses through CAR T-cell therapies and bispecific antibodies. Since KarMMa and CARTITUDE-1 established the activity of idecabtagene vicleucel and ciltacabtagene autoleucel in heavily pretreated disease, efforts have intensified to move them earlier, culminating in KarMMa-3 and CARTITUDE-4, which demonstrated superiority to standard regimens in earlier lines and prompted label expansions. Yet randomized data alone do not capture real-world effectiveness. Emerging cohorts highlight attrition before infusion, product-specific safety signatures with infection-driven non-relapse mortality, and heterogeneous outcomes in high-risk biology such as extramedullary disease, adverse cytogenetics, and early relapse. Access and delivery constraints, including bridging intensity, vein-to-vein intervals, out-of-specification release, and outpatient feasibility, further determine who benefits. This review synthesizes updated randomized and real-world evidence for idecabtagene vicleucel and ciltacabtagene autoleucel, examines challenging populations and emerging toxicities, and delineates operational factors that shape outcomes at scale. We outline where CAR T should displace existing standards, where caution is warranted, and which innovations in targets, constructs, and manufacturing are most likely to advance the start line.
{"title":"CAR T-cells in multiple myeloma: the race to the start line","authors":"Abdul-Hamid Bazarbachi, Divaya Bhutani, Michael Sang Hughes, Suzanne Lentzsch, Markus Mapara, Pawel Muranski, Ran Reshef, Tamna Wangjam, Rajshekhar Chakraborty","doi":"10.1038/s41409-025-02787-9","DOIUrl":"10.1038/s41409-025-02787-9","url":null,"abstract":"The treatment landscape for multiple myeloma is rapidly evolving, driven by T-cell-mediated tumor responses through CAR T-cell therapies and bispecific antibodies. Since KarMMa and CARTITUDE-1 established the activity of idecabtagene vicleucel and ciltacabtagene autoleucel in heavily pretreated disease, efforts have intensified to move them earlier, culminating in KarMMa-3 and CARTITUDE-4, which demonstrated superiority to standard regimens in earlier lines and prompted label expansions. Yet randomized data alone do not capture real-world effectiveness. Emerging cohorts highlight attrition before infusion, product-specific safety signatures with infection-driven non-relapse mortality, and heterogeneous outcomes in high-risk biology such as extramedullary disease, adverse cytogenetics, and early relapse. Access and delivery constraints, including bridging intensity, vein-to-vein intervals, out-of-specification release, and outpatient feasibility, further determine who benefits. This review synthesizes updated randomized and real-world evidence for idecabtagene vicleucel and ciltacabtagene autoleucel, examines challenging populations and emerging toxicities, and delineates operational factors that shape outcomes at scale. We outline where CAR T should displace existing standards, where caution is warranted, and which innovations in targets, constructs, and manufacturing are most likely to advance the start line.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 3","pages":"260-274"},"PeriodicalIF":5.2,"publicationDate":"2025-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02787-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846294","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-12-26DOI: 10.1038/s41409-025-02794-w
Valerie Greco-Stewart, Sigal Manor, Simona Pollichieni, Arnim Kathke, Gabi Rall, Irina Evseeva, Paul Johnson, Abby Martfeld, Martine Schuit, Ingrid Tistl
{"title":"Identification and management of the backup donor: recommendations from the World Marrow Donor Association","authors":"Valerie Greco-Stewart, Sigal Manor, Simona Pollichieni, Arnim Kathke, Gabi Rall, Irina Evseeva, Paul Johnson, Abby Martfeld, Martine Schuit, Ingrid Tistl","doi":"10.1038/s41409-025-02794-w","DOIUrl":"10.1038/s41409-025-02794-w","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 3","pages":"255-259"},"PeriodicalIF":5.2,"publicationDate":"2025-12-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02794-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145843617","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-12-24DOI: 10.1038/s41409-025-02780-2
Alexandros Rampotas, Jose Maria Aspa-Cilleruelo, Linda Koster, Daniele Avenoso, Jakob Passweg, Elisa Sala, Marie Robin, Anders Eivind Myhre, Moniek de Witte, Erfan Nur, Patrice Chevallier, Thomas Schroeder, Micha Srour, Patrizia Chiusolo, Urpu Salmenniemi, Mareike Verbeek, Maria Chiara Finazzi, Cristina Castilla-Llorente, Marie Therese Rubio, Patryk Sobieralski, Katja Sockel, Ahmad Alabdulkarim, Joanna Drozd-Sokolowska, Kavita Raj, Giorgia Battipaglia, Tomasz Czerw, Nicola Polverelli, Juan Carlos Hernández-Boluda, Donal P. McLornan
Allogeneic haematopoietic cell transplantation (allo-HCT) remains the only potentially curative option for patients with myelofibrosis (MF), yet the integration of JAK inhibitors (JAKi) and novel agents into transplant pathways has created increasing complexity. To capture current real-world practice, the EBMT Chronic Malignancies Working Party conducted a survey of 19 high-volume European centres performing MF allo-HCT. Most centres (68%) routinely initiated JAKi, primarily ruxolitinib, in transplant-eligible patients prior to conditioning, with goals of splenomegaly reduction and symptom control. Management of ruxolitinib intolerance or resistance was heterogeneous, with strategies including switching to alternative JAKi, proceeding directly to allo-HCT, or enroling in clinical trials. Peri-transplant approaches also varied: over half of centres continued ruxolitinib throughout conditioning, while others employed tapering or abrupt discontinuation. Experience with newer JAKi and investigational therapies was limited. Post-transplant, most centres did not routinely reintroduce JAKi, although some used them for relapse or GVHD mitigation. Notably, many centres reported transplant delays due to prolonged medical therapy, with adverse consequences including disease progression. These findings highlight significant heterogeneity in practice, which is likely to increase as more novel agents are integrated in treatment algorithms. Harmonised, multidisciplinary guidelines to optimise timing and outcomes for MF patients eligible for allo-HCT are needed.
同种异体造血细胞移植（Allogeneic hematopoietic cell transplantation, allo-HCT）仍然是骨髓纤维化（MF）患者唯一潜在的治疗选择，然而，将JAK抑制剂（JAKi）和新型药物整合到移植途径中，使其变得越来越复杂。为了了解当前的现实世界实践，EBMT慢性恶性肿瘤工作组对19个高容量的欧洲中心进行了MF - all - hct调查。大多数中心（68%）在适应前对符合移植条件的患者常规启动JAKi，主要是ruxolitinib，目的是减少脾肿大和控制症状。ruxolitinib不耐受或耐药的处理是异质性的，策略包括切换到替代JAKi，直接进行allo-HCT，或参加临床试验。移植前后的治疗方法也各不相同：超过一半的中心在整个调节过程中继续使用鲁索利替尼，而其他中心则逐渐减少或突然停药。新的JAKi和研究性治疗的经验有限。移植后，大多数中心没有常规地重新引入JAKi，尽管有些中心将其用于复发或缓解GVHD。值得注意的是，许多中心报告说，由于长期的医疗治疗导致移植延迟，造成了包括疾病进展在内的不良后果。这些发现突出了实践中显著的异质性，随着更多的新型药物被纳入治疗算法，这种异质性可能会增加。需要统一的多学科指南来优化有资格接受同种异体hct治疗的MF患者的时机和结果。
{"title":"A European survey on allogeneic haematopoietic cell transplantation for myelofibrosis on behalf of the Chronic Malignancies Working Party of the EBMT: focus on ‘real world’ experience of JAK inhibitors, splenomegaly management and novel agents in the transplant algorithm","authors":"Alexandros Rampotas, Jose Maria Aspa-Cilleruelo, Linda Koster, Daniele Avenoso, Jakob Passweg, Elisa Sala, Marie Robin, Anders Eivind Myhre, Moniek de Witte, Erfan Nur, Patrice Chevallier, Thomas Schroeder, Micha Srour, Patrizia Chiusolo, Urpu Salmenniemi, Mareike Verbeek, Maria Chiara Finazzi, Cristina Castilla-Llorente, Marie Therese Rubio, Patryk Sobieralski, Katja Sockel, Ahmad Alabdulkarim, Joanna Drozd-Sokolowska, Kavita Raj, Giorgia Battipaglia, Tomasz Czerw, Nicola Polverelli, Juan Carlos Hernández-Boluda, Donal P. McLornan","doi":"10.1038/s41409-025-02780-2","DOIUrl":"10.1038/s41409-025-02780-2","url":null,"abstract":"Allogeneic haematopoietic cell transplantation (allo-HCT) remains the only potentially curative option for patients with myelofibrosis (MF), yet the integration of JAK inhibitors (JAKi) and novel agents into transplant pathways has created increasing complexity. To capture current real-world practice, the EBMT Chronic Malignancies Working Party conducted a survey of 19 high-volume European centres performing MF allo-HCT. Most centres (68%) routinely initiated JAKi, primarily ruxolitinib, in transplant-eligible patients prior to conditioning, with goals of splenomegaly reduction and symptom control. Management of ruxolitinib intolerance or resistance was heterogeneous, with strategies including switching to alternative JAKi, proceeding directly to allo-HCT, or enroling in clinical trials. Peri-transplant approaches also varied: over half of centres continued ruxolitinib throughout conditioning, while others employed tapering or abrupt discontinuation. Experience with newer JAKi and investigational therapies was limited. Post-transplant, most centres did not routinely reintroduce JAKi, although some used them for relapse or GVHD mitigation. Notably, many centres reported transplant delays due to prolonged medical therapy, with adverse consequences including disease progression. These findings highlight significant heterogeneity in practice, which is likely to increase as more novel agents are integrated in treatment algorithms. Harmonised, multidisciplinary guidelines to optimise timing and outcomes for MF patients eligible for allo-HCT are needed.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 3","pages":"333-340"},"PeriodicalIF":5.2,"publicationDate":"2025-12-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02780-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145826817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-12-23DOI: 10.1038/s41409-025-02765-1
Iman Abou Dalle, Jacques-Emmanuel Galimard, Xavier Poire, Jaime Sanz, Anne Huynh, Nicolaus Kröger, Eva Maria Wagner-Drouet, David Burns, Matthias Eder, Bruno Lioure, Depei Wu, Jiri Mayer, Kristina Carlson, Matthias Stelljes, Matthew Collin, Mahmoud Aljurf, Arnon Nagler, Jordi Esteve, Fabio Ciceri, Ali Bazarbachi, Mohamad Mohty
Mutations in the DNMT3A gene are not yet classified as a distinct prognostic group in the latest European Leukemia Net (ELN) 2022 genetic risk classification of AML. We analyzed 1888 adult AML patients with ELN 2022 intermediate- or poor-risk cytogenetics who received their first allo-transplant in first complete remission between 2015 and 2022. Among patients with cytogenetically normal AML, the triple-positive mutation group (DNMT3A, NPM1, and FLT3-ITD) was the most frequent (n = 340, 29%), while DNMT3A co-occurrence with either FLT3 or NPM1 mutations alone was less common (4% and 9%, respectively). Patients with DNMT3A mutations were less likely to have a secondary AML (14% versus 24%, p < 0.001). DNMT3A mutations negatively affected post-transplant leukemia-free survival (LFS) in patients with normal karyotype and NPM1 mutation without FLT3-ITD (2-year LFS: 70% versus 90%, hazard ratio [HR]: 3.3, p = 0.006), and increased relapse incidence (RI) in FLT3-ITD and wild-type NPM1 subgroup (2-year RI: 30% versus 18%, HR: 2.32, p = 0.03). Notably, patients with normal karyotype and triple-positive mutation exhibited excellent 2-year LFS and OS (61% and 70%), indicating that allo-transplant overcomes the dismal outcome of this group. The impact of DNMT3A mutations on post-transplant outcomes in AML patients in first remission varies based on karyotype and co-mutations.
{"title":"The impact of DNMT3A mutation on survival of AML patients receiving allotransplant in first remission depends on the karyotype and co-occurring mutations","authors":"Iman Abou Dalle, Jacques-Emmanuel Galimard, Xavier Poire, Jaime Sanz, Anne Huynh, Nicolaus Kröger, Eva Maria Wagner-Drouet, David Burns, Matthias Eder, Bruno Lioure, Depei Wu, Jiri Mayer, Kristina Carlson, Matthias Stelljes, Matthew Collin, Mahmoud Aljurf, Arnon Nagler, Jordi Esteve, Fabio Ciceri, Ali Bazarbachi, Mohamad Mohty","doi":"10.1038/s41409-025-02765-1","DOIUrl":"10.1038/s41409-025-02765-1","url":null,"abstract":"Mutations in the DNMT3A gene are not yet classified as a distinct prognostic group in the latest European Leukemia Net (ELN) 2022 genetic risk classification of AML. We analyzed 1888 adult AML patients with ELN 2022 intermediate- or poor-risk cytogenetics who received their first allo-transplant in first complete remission between 2015 and 2022. Among patients with cytogenetically normal AML, the triple-positive mutation group (DNMT3A, NPM1, and FLT3-ITD) was the most frequent (n = 340, 29%), while DNMT3A co-occurrence with either FLT3 or NPM1 mutations alone was less common (4% and 9%, respectively). Patients with DNMT3A mutations were less likely to have a secondary AML (14% versus 24%, p < 0.001). DNMT3A mutations negatively affected post-transplant leukemia-free survival (LFS) in patients with normal karyotype and NPM1 mutation without FLT3-ITD (2-year LFS: 70% versus 90%, hazard ratio [HR]: 3.3, p = 0.006), and increased relapse incidence (RI) in FLT3-ITD and wild-type NPM1 subgroup (2-year RI: 30% versus 18%, HR: 2.32, p = 0.03). Notably, patients with normal karyotype and triple-positive mutation exhibited excellent 2-year LFS and OS (61% and 70%), indicating that allo-transplant overcomes the dismal outcome of this group. The impact of DNMT3A mutations on post-transplant outcomes in AML patients in first remission varies based on karyotype and co-mutations.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 3","pages":"326-332"},"PeriodicalIF":5.2,"publicationDate":"2025-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145817830","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}
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