Simona Iacobelli, Stefan Schönland, Linda Koster, Didier Blaise, Emma Nicholson, A E C Broers, Patrice Chevallier, Péter Reményi, František Folber, John G Gribben, Erfan Nur, Neil Rabin, Matthew Collin, Tobias Gedde-Dahl, Katharine Bailey, Paul Ferguson, Matthias Stelljes, Adrian Bloor, Meral Beksac, Joanna Drozd-Sokolowska, Kavita Raj, Patrick J Hayden, Ibrahim Yakoub-Agha, Donal P McLornan, Nicolaus Kröger
{"title":"Outcomes following different upfront stem cell transplantation strategies for multiple myeloma: a statistical perspective on behalf of the Chronic Malignancies Working Party of the EBMT.","authors":"Simona Iacobelli, Stefan Schönland, Linda Koster, Didier Blaise, Emma Nicholson, A E C Broers, Patrice Chevallier, Péter Reményi, František Folber, John G Gribben, Erfan Nur, Neil Rabin, Matthew Collin, Tobias Gedde-Dahl, Katharine Bailey, Paul Ferguson, Matthias Stelljes, Adrian Bloor, Meral Beksac, Joanna Drozd-Sokolowska, Kavita Raj, Patrick J Hayden, Ibrahim Yakoub-Agha, Donal P McLornan, Nicolaus Kröger","doi":"10.1038/s41409-025-02675-2","DOIUrl":"https://doi.org/10.1038/s41409-025-02675-2","url":null,"abstract":"<p><p>Multiple myeloma (MM) is a heterogenous malignant disease. Novel agents including bispecific antibodies and chimeric antigen receptor (CAR) T cells have improved response rates and patient outcome, but the majority of patients ultimately still relapse. High dose chemotherapy followed by autologous hematopoietic stem cell transplantation (auto-HCT) remains standard care of treatment for transplant-eligible patients. While single auto-HCT is commonly used, a planned tandem auto-HCT or auto-allo approach remains controversial, based on conflicting results from clinical trials. Here we compared the outcome of 24,936 MM patients aged between 20 and 65 years who underwent first auto-HCT during 2002-2015, reported to the EBMT registry, of whom 3683 and 878 got tandem auto-HCT and auto-allo-HCT respectively. We used non-standard statistical approaches to account for time-dependence of treatments and of their effects, including models with multiple timescales and dynamic prediction. Differences were reported by graphs of hazard functions, hazard ratios and conditional probabilities over time. For both OS and PFS, there was a limited but persistent advantage for the tandem auto-HCT group compared to single auto-HCT, and a clear advantage for the auto-allo-HCT group over both other strategies in the longer term, albeit at the cost of higher early mortality.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-07-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144752261","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}
Marta Lisa Battista, Gabriele Facchin, Maria De Martino, Patrizia Chiusolo, Domenico Russo, Michele Malagola, Francesco Saraceni, Giorgia Mancini, Alessandro Rambaldi, Irene Maria Cavattoni, Albana Lico, Chiara Nozzoli, Angela Cuoghi, Marco Ladetto, Vincenzo Federico, Maria Caterina Micò, Maria Teresa Lupo-Stanghellini, Elena Oldani, Eliana Degrandi, Anna Maria Gallina, Matteo Parma, Alessandra Biffi, Franca Fagioli, Cristina Skert, Antonella Geromin, Chiara Savignano, Miriam Isola, Renato Fanin, Fabio Ciceri, Massimo Martino, Francesca Patriarca
{"title":"Real-world study in steroid-refractory acute graft versus host disease: Comparison of efficacy and tolerability of ruxolitinib alone or ruxolitinib in association with extracorporeal photopheresis or extracorporeal photopheresis monotherapy.","authors":"Marta Lisa Battista, Gabriele Facchin, Maria De Martino, Patrizia Chiusolo, Domenico Russo, Michele Malagola, Francesco Saraceni, Giorgia Mancini, Alessandro Rambaldi, Irene Maria Cavattoni, Albana Lico, Chiara Nozzoli, Angela Cuoghi, Marco Ladetto, Vincenzo Federico, Maria Caterina Micò, Maria Teresa Lupo-Stanghellini, Elena Oldani, Eliana Degrandi, Anna Maria Gallina, Matteo Parma, Alessandra Biffi, Franca Fagioli, Cristina Skert, Antonella Geromin, Chiara Savignano, Miriam Isola, Renato Fanin, Fabio Ciceri, Massimo Martino, Francesca Patriarca","doi":"10.1038/s41409-025-02674-3","DOIUrl":"10.1038/s41409-025-02674-3","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-07-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144717487","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}
Marina Gomez-Llobell, Carlos Serra Smith, Daniel Gómez-Costas, Ignacio Gómez-Centurión, Jose Manuel García Domínguez, Vicente Escudero-Vilaplana, Yolanda Fernández Bullido, Diego Carbonell, Marjorie Pion, Rebeca Bailén, Sara Gil-Perotin, María Luisa Martínez Ginés, José Luis Revuelta-Herrero, Paula Fernández-Caldas, Verónica Astrid Pérez Fernández, Roberto Collado-Borrell, Mariana Bastos-Oreiro, Cristina Villanueva-Bueno, Ramón García-Sanz, Mi Kwon
{"title":"ICANS risk model in CD19 CAR-T therapy: insights from serum and CSF cytokine profiling.","authors":"Marina Gomez-Llobell, Carlos Serra Smith, Daniel Gómez-Costas, Ignacio Gómez-Centurión, Jose Manuel García Domínguez, Vicente Escudero-Vilaplana, Yolanda Fernández Bullido, Diego Carbonell, Marjorie Pion, Rebeca Bailén, Sara Gil-Perotin, María Luisa Martínez Ginés, José Luis Revuelta-Herrero, Paula Fernández-Caldas, Verónica Astrid Pérez Fernández, Roberto Collado-Borrell, Mariana Bastos-Oreiro, Cristina Villanueva-Bueno, Ramón García-Sanz, Mi Kwon","doi":"10.1038/s41409-025-02679-y","DOIUrl":"https://doi.org/10.1038/s41409-025-02679-y","url":null,"abstract":"<p><p>Immune Effector Cell-Associated Neurotoxicity Syndrome (ICANS) is a common and potentially severe complication of CD19 CAR-T therapy. While some clinical risk factors have been described, the contribution of cytokines, particularly in plasma and cerebrospinal fluid (CSF), remains limited. This study aimed to identify predictors and characterize cytokine patterns associated with ICANS to develop a multivariable risk model. We retrospectively analyzed 101 adult patients treated with axicabtagene ciloleucel (axi-cel) or tisagenlecleucel (tisa-cel) between 2019 and 2023. Cytokines (interleukin (IL)-1β, IL-6, IL-15, GM-CSF) were measured in plasma pre- and post-infusion, and in CSF during neurotoxicity. ICANS occurred in 36% of patients, more frequently with axi-cel (46% vs. 21%, p < 0.05). Autoimmune disease history and elevated IL-6 and IL-15 were associated with increased risk. CSF cytokines were also elevated during ICANS episodes. A multivariate model predicting any-grade ICANS included CAR-T product, time to cytokine release syndrome (CRS) onset, IL-6 at day 3, and pre-infusion D-dimer (AUC = 0.83). The model for grade 2-4 ICANS included number of prior therapies, grade ≥2 CRS, autoimmune disease, IL-15 at day 0, and GM-CSF (AUC = 0.80). Integrating cytokine profiles with clinical parameters may enable early ICANS risk stratification and improve personalized monitoring in CAR-T recipients.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144706284","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}
Daniela Morales, Gisele Loth, Vergílio Antonio Rensi Colturato, Rita Barbosa Tavares, Simone Lermontov, Adriana Mello Rodrigues, Polliany Roberta Dorini Pelegrina, Cilmara Cristina Kuwahara, Nathalia Kersting, Ciliana Rechenmacher, Priscila de Oliveira da Silva, Yasmine Monteiro, Mariana Bohns Michalowski, Mario Bernardes Wagner, Carmem Bonfim, Liane Esteves Daudt
{"title":"Outcomes of hematopoietic stem cell transplantation in pediatric acute lymphoblastic leukemia: a multicenter Brazilian cohort study.","authors":"Daniela Morales, Gisele Loth, Vergílio Antonio Rensi Colturato, Rita Barbosa Tavares, Simone Lermontov, Adriana Mello Rodrigues, Polliany Roberta Dorini Pelegrina, Cilmara Cristina Kuwahara, Nathalia Kersting, Ciliana Rechenmacher, Priscila de Oliveira da Silva, Yasmine Monteiro, Mariana Bohns Michalowski, Mario Bernardes Wagner, Carmem Bonfim, Liane Esteves Daudt","doi":"10.1038/s41409-025-02676-1","DOIUrl":"https://doi.org/10.1038/s41409-025-02676-1","url":null,"abstract":"<p><p>Acute leukemias are prevalent among children and adolescents, with survival rates exceeding 90%, but high-risk cases often require hematopoietic stem cell transplantation (HSCT). Limited data are available on HSCT outcomes for pediatric acute lymphoblastic leukemia (ALL) in low- and middle-income countries. This Brazilian multicenter retrospective cohort study analyzed outcomes of ALL patients aged up to 18 years who underwent HSCT from January 2010 to June 2020. A total of 439 patients were included (69% male, 78.9% B-ALL). Unrelated donor transplants accounted for 49.9%, and haploidentical transplants for 18.9%. Bone marrow (82.6%) was the predominant stem cell source. Transplants occurred in the first or second complete remission (CR) in 38.7% and 44.2% of cases, respectively. The 100-day cumulative incidence of acute GvHD was 46.1%, and chronic GvHD after 100 days occurred in 25.5%. At five years, overall survival and event-free survival were 52.4% and 51.7%. By day +100, sepsis was the leading cause of mortality (42.3%), whereas relapse accounted for 76.3% of deaths thereafter. Findings align with HSCT literature, demonstrating similar outcomes despite challenges in developing countries and providing a basis for further research.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144706285","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}
Agni Hatzakis, Bernard D Maybury, Sridhar Chaganti, Andrea Stevens, Bethanie Rooke, Elizabeth Peterknecht, Farida Shah, Usha Alluri, Duncan J Murray, Julia J Scarisbrick
{"title":"Allogeneic haematopoietic stem cell transplantation in advanced cutaneous T-cell lymphomas offers curative potential.","authors":"Agni Hatzakis, Bernard D Maybury, Sridhar Chaganti, Andrea Stevens, Bethanie Rooke, Elizabeth Peterknecht, Farida Shah, Usha Alluri, Duncan J Murray, Julia J Scarisbrick","doi":"10.1038/s41409-025-02683-2","DOIUrl":"https://doi.org/10.1038/s41409-025-02683-2","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144697558","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}
Olaf Penack, Andrea Bacigalupo, Eleni Gavriilaki, Hildegard Greinix, Florent Malard, David Michonneau, Attilio Olivieri, Zinaida Peric, Elisa Sala, Carlos Solano, Daniel Wolff, Robert Zeiser
{"title":"Treatment patterns of extracorporeal photopheresis in steroid-refractory graft versus host disease: A delphi study.","authors":"Olaf Penack, Andrea Bacigalupo, Eleni Gavriilaki, Hildegard Greinix, Florent Malard, David Michonneau, Attilio Olivieri, Zinaida Peric, Elisa Sala, Carlos Solano, Daniel Wolff, Robert Zeiser","doi":"10.1038/s41409-025-02687-y","DOIUrl":"https://doi.org/10.1038/s41409-025-02687-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144688811","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}
Alessandro Criscimanna, Jacques Emmanuel Galimard, Maria Chiara Auriemma, Lorenzo Lazzari, Elisa Diral, Alessandro Bruno, Simona Piemontese, Daniela Clerici, Sara Mastaglio, Luca Vago, Raffaella Greco, Francesca Farina, Antonella Capasso, Daniele Sannipoli, Andrea Acerbis, Gregorio Maria Bergonzi, Sarah Marktel, Consuelo Corti, Massimo Bernardi, Andrea Assanelli, Fabio Ciceri, Maria Teresa Lupo-Stanghellini, Annalisa Ruggeri
{"title":"Validation of Manhattan risk to predict aGvHD severity in Rapamycin and PTCy-based prophylaxis: a study on 459 consecutive HCT patients.","authors":"Alessandro Criscimanna, Jacques Emmanuel Galimard, Maria Chiara Auriemma, Lorenzo Lazzari, Elisa Diral, Alessandro Bruno, Simona Piemontese, Daniela Clerici, Sara Mastaglio, Luca Vago, Raffaella Greco, Francesca Farina, Antonella Capasso, Daniele Sannipoli, Andrea Acerbis, Gregorio Maria Bergonzi, Sarah Marktel, Consuelo Corti, Massimo Bernardi, Andrea Assanelli, Fabio Ciceri, Maria Teresa Lupo-Stanghellini, Annalisa Ruggeri","doi":"10.1038/s41409-025-02677-0","DOIUrl":"https://doi.org/10.1038/s41409-025-02677-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144682016","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}
Dharshan Sivaraj, Wendi Bacon, Gwynn D Long, David A Rizzieri, Michael E Horwitz, Keith M Sullivan, Yubin Kang, Zighou Li, Nelson J Chao, Cristina Gasparetto
{"title":"Correction: High-dose BCNU/Melphalan conditioning regimen before autologous stem cell transplantation in newly diagnosed multiple myeloma.","authors":"Dharshan Sivaraj, Wendi Bacon, Gwynn D Long, David A Rizzieri, Michael E Horwitz, Keith M Sullivan, Yubin Kang, Zighou Li, Nelson J Chao, Cristina Gasparetto","doi":"10.1038/s41409-025-02663-6","DOIUrl":"https://doi.org/10.1038/s41409-025-02663-6","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144682015","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}
Moazzam Shahzad, Aleenah Mohsin, Ibrahim Khamees, Aqeeb Ur Rehman, Muhammad Kashif Amin, Sibgha Gull Chaudhary, Iqra Anwer, Kayla Pfannenstiel, Al-Ola Abdallah, Mehdi Hamadani, Joseph P Mcguirk, Muhammad Umair Mushtaq
{"title":"Impact of body mass index on outcomes following allogeneic hematopoietic stem cell transplantation.","authors":"Moazzam Shahzad, Aleenah Mohsin, Ibrahim Khamees, Aqeeb Ur Rehman, Muhammad Kashif Amin, Sibgha Gull Chaudhary, Iqra Anwer, Kayla Pfannenstiel, Al-Ola Abdallah, Mehdi Hamadani, Joseph P Mcguirk, Muhammad Umair Mushtaq","doi":"10.1038/s41409-025-02680-5","DOIUrl":"https://doi.org/10.1038/s41409-025-02680-5","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144658384","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":"Characteristics and prognosis of central nervous system relapse after allogeneic hematopoietic stem cell transplantation in adult patients with leukemia.","authors":"Li-Ping Guo, Li-Juan Hu, Xiao-Dong Mo, Yu-Qian Sun, Yi-Fei Cheng, Lan-Ping Xu, Xiao-Hui Zhang, Xiao-Jun Huang, Yu Wang","doi":"10.1038/s41409-025-02673-4","DOIUrl":"https://doi.org/10.1038/s41409-025-02673-4","url":null,"abstract":"<p><p>Central nervous system(CNS) is a common site of extramedullary involvement after allogeneic hematopoietic stem cell transplantation(allo-HSCT). It may confer inferior survival when combined with bone marrow(BM) relapse in leukemia patients. We performed a retrospective study to investigate the associated risk factors and prognosis in adult patients with post-transplant CNS relapse. Between 2016 and 2024 in our center, 74 leukemia patients developed CNS relapse after allo-HSCT, with 23 cases of isolated CNS(iCNS) relapse and 51 cases of combined CNS and BM(CNS&BM) relapse. Patients with CNS&BM relapse after allo-HSCT conferred inferior 5-year overall survival (OS) compared with those with iCNS relapse, and independent risk factors related with CNS&BM relapse were identified, including male gender(relative risk ratio[RR] = 4.09, 95% confidence interval [CI] = 1.29-13.02, p = 0.017), HLA-identical transplantation (RR = 4.11, 95% CI = 1.10-15.32, p = 0.035), and without chronic graft-versus-host disease occurrence (RR = 3.73, 95% CI = 1.00-13.84, p = 0.049). Early CNS relapse was identified as an independent predictor of worse 5-year OS from HSCT for patients with CNS&BM relapse (n = 51, <12 months after allo-HSCT, hazard ratio=7.35, p = 0.003). Systemic therapy after CNS relapse reduced subsequent BM relapse for ALL and CML patients (p = 0.038). These findings demonstrate the outcome of patients with post-transplant CNS&BM relapse remains very poor. HLA-haploindentical allo-HSCT and systemic therapy after CNS relapse may better reduce BM relapse.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-07-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144658383","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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