Pub Date : 2026-01-03DOI: 10.1038/s41409-025-02783-z
Patricia López-Pereira, Ali Bazarbachi, Ambroise Marçais, Maud Ngoya, Irma Khvedelidze, Arian Laurence, Victoria Potter, Flore Sicre de Fontbrune, Alina Daniela Tanase, Lucy Cook, Sridhar Chaganti, Emma Nicholson, Yasmina Serroukh, Jennifer Clay, Hélène Labussière-Wallet, Fabio Ciceri, Bertram Glass, Olivier Hermine, Anna Sureda Balari
Adult T-cell leukemia/lymphoma (ATLL) is a rare HTLV-1-associated malignancy with a dismal prognosis due to intrinsic chemoresistance and immunosuppression. This retrospective EBMT registry analysis evaluated outcomes of allogeneic stem cell transplantation (allo-SCT) in 73 ATLL patients transplanted between 2004 and 2021. With a median follow-up of 3.9 years, 2-year overall survival (OS) and progression-free survival (PFS) were 49% (95% CI 37-60) and 43% (95% CI 31-54), respectively. Non-relapse mortality was 7% at 2 years, while relapse incidence (RI) was 49.8%. Multivariate analysis identified haploidentical donors as associated with improved OS, while non-CR status at transplant was independently associated with significantly worse PFS (HR 2.93, p = 0.01), OS (HR 4.12, p = 0.002), and RI (HR 2.66, p = 0.04) compared to CR. Our findings suggest that allo-SCT represents a feasible and potentially curative consolidation strategy for ATLL patients, with CR at transplant, younger age, and haploidentical donors being independent predictors of improved survival, supporting the importance of disease control, patient selection, and alternative donor strategies.
成人t细胞白血病/淋巴瘤(ATLL)是一种罕见的htlv -1相关的恶性肿瘤,由于内在的化疗耐药和免疫抑制,预后不佳。这项回顾性EBMT登记分析评估了2004年至2021年间移植的73例ATLL患者的同种异体干细胞移植(allo-SCT)的结果。中位随访时间为3.9年,2年总生存率(OS)和无进展生存率(PFS)分别为49% (95% CI 37-60)和43% (95% CI 31-54)。2年非复发死亡率为7%,而复发率(RI)为49.8%。多因素分析表明,单倍体相同的供体与改善的OS相关,而移植时的非CR状态与显著较差的PFS (HR 2.93, p = 0.01)、OS (HR 4.12, p = 0.002)和RI (HR 2.66, p = 0.04)相关。我们的研究结果表明,对于移植时CR、年龄较小的ATLL患者,同种异体sct是一种可行且具有潜在治愈性的巩固策略。单倍体供体是提高生存率的独立预测因素,支持疾病控制、患者选择和替代供体策略的重要性。
{"title":"Outcomes of patients with HTLV-1 adult T-cell leukemia/lymphoma (ATLL) after allogeneic SCT: results of the EBMT LWP.","authors":"Patricia López-Pereira, Ali Bazarbachi, Ambroise Marçais, Maud Ngoya, Irma Khvedelidze, Arian Laurence, Victoria Potter, Flore Sicre de Fontbrune, Alina Daniela Tanase, Lucy Cook, Sridhar Chaganti, Emma Nicholson, Yasmina Serroukh, Jennifer Clay, Hélène Labussière-Wallet, Fabio Ciceri, Bertram Glass, Olivier Hermine, Anna Sureda Balari","doi":"10.1038/s41409-025-02783-z","DOIUrl":"https://doi.org/10.1038/s41409-025-02783-z","url":null,"abstract":"<p><p>Adult T-cell leukemia/lymphoma (ATLL) is a rare HTLV-1-associated malignancy with a dismal prognosis due to intrinsic chemoresistance and immunosuppression. This retrospective EBMT registry analysis evaluated outcomes of allogeneic stem cell transplantation (allo-SCT) in 73 ATLL patients transplanted between 2004 and 2021. With a median follow-up of 3.9 years, 2-year overall survival (OS) and progression-free survival (PFS) were 49% (95% CI 37-60) and 43% (95% CI 31-54), respectively. Non-relapse mortality was 7% at 2 years, while relapse incidence (RI) was 49.8%. Multivariate analysis identified haploidentical donors as associated with improved OS, while non-CR status at transplant was independently associated with significantly worse PFS (HR 2.93, p = 0.01), OS (HR 4.12, p = 0.002), and RI (HR 2.66, p = 0.04) compared to CR. Our findings suggest that allo-SCT represents a feasible and potentially curative consolidation strategy for ATLL patients, with CR at transplant, younger age, and haploidentical donors being independent predictors of improved survival, supporting the importance of disease control, patient selection, and alternative donor strategies.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-01-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145899459","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/s41409-025-02790-0
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":"https://doi.org/10.1038/s41409-025-02790-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"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/s41409-025-02787-9
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":"https://doi.org/10.1038/s41409-025-02787-9","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","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":"https://doi.org/10.1038/s41409-025-02792-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"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/s41409-025-02784-y
Ihunayachi Ohuabunwa, Bhagirathbhai Dholaria, Megan Wanca, Grace Johnson Mercadante, Phavina Akhom, Brittney Baer, Ali Nur, Leslie Mader, Sarah Moseley, Eden Biltibo, Andrew Jallouk, Reena Jayani-Kosarzycki, James Jerkins, Adetola Kassim, Nancy Long, David Morgan, Vivek Patel, Shakthi Bhaskar, Bipin Savani, Salyka Sengsayadeth, Muhamed Baljevic, Olalekan O Oluwole
{"title":"Feasibility of outpatient administration of ciltacabtagene autoleucel and reduction in healthcare utilization cost: the Vanderbilt experience.","authors":"Ihunayachi Ohuabunwa, Bhagirathbhai Dholaria, Megan Wanca, Grace Johnson Mercadante, Phavina Akhom, Brittney Baer, Ali Nur, Leslie Mader, Sarah Moseley, Eden Biltibo, Andrew Jallouk, Reena Jayani-Kosarzycki, James Jerkins, Adetola Kassim, Nancy Long, David Morgan, Vivek Patel, Shakthi Bhaskar, Bipin Savani, Salyka Sengsayadeth, Muhamed Baljevic, Olalekan O Oluwole","doi":"10.1038/s41409-025-02784-y","DOIUrl":"https://doi.org/10.1038/s41409-025-02784-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145846345","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 : 2025-12-26DOI: 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":"https://doi.org/10.1038/s41409-025-02794-w","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-12-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","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":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}