Bone Marrow Transplantation最新文献

{"title":"Late complications after autologous HSCT for autoimmune diseases: a retrospective survey from the autoimmune diseases working party (ADWP), transplant complications working party (TCWP) and paediatric diseases working party (PDWP) of the european society for blood and marrow transplantation (EBMT)","authors":"Kirill Kirgizov, Raffaella Greco, Manuela Badoglio, Myriam Labopin, Riccardo Saccardi, Montserrat Rovira, Dominique Farge, Mieke W. H. Roeven, John Moore, Varun Mehra, Francesco Onida, Alexey Polushin, Jürgen Finke, Tobias Alexander, Joachim Burman, Nicoletta Del Papa, Diana M. Greenfield, Majid Kazmi, Alicia Rovo, Nina Salooja, Helene Schoemans, Basil Sharrack, Chiara Nozzoli, André Tichelli, Peter Bader, Selim Corbacioglu, Zinaida Peric, Elisa Roldan, Grzegorz W. Basak, John A. Snowden","doi":"10.1038/s41409-026-02983-1","DOIUrl":"10.1038/s41409-026-02983-1","url":null,"abstract":"Autologous hematopoietic stem cell transplantation (aHSCT) has evolved as a treatment for severe autoimmune diseases (ADs). Advances in transplant procedures and supportive care have led to improvements in long-term survival but there is limited data on ‘late effects’. The aim of this retrospective EBMT registry study is to assess the incidence of ‘late effects’ after aHSCT for ADs. All EBMT centres were invited and data were received for 579 patients (median age 37 years, range 2.7–73.8), who received aHSCT between 1997 and 2016. Median follow-up was 89 months (IQR 82.4–95.5). Indications included multiple sclerosis (MS, 46.8%), systemic sclerosis (SSc, 28.7%), Crohn’s disease (CD, 12.6%), systemic lupus erythematosus (SLE, 2.4%) and other ADs (9.5%). Conditioning was mainly based on Cy/ATG (51.9%) or BEAM/ATG (25.3%). At 10-years, the cumulative incidence of secondary ADs was 10.3% (95% CI: 7.7–13.3), new post-transplant cancers 4.1% (95% CI: 2.2–6.8), endocrine complications 16.2% (95% CI: 12.6–20.1), and cardiovascular complications 14.7% (95% CI: 11.2–18.6). Median time to endocrine events (15.4 months) was generally sooner than median times to secondary ADs (24.5 months) and cardiovascular (25.3 months), with new cancers occurring relatively late (median time 81.7 months). Overall, at 10-years, progression-free survival (PFS) was 40.1% (95% CI: 34.7–45.4), overall survival (OS) 83.5% (95% CI: 79.3–86.9) and non-relapse mortality (NRM) 5.0% (95% CI: 3.2–7.4). By multivariate analysis, secondary ADs were relatively higher in CD vs SSc (HR = 4.93, p = 0.01) and MS (HR = 2.05, p = 0.055), while cardiovascular complications were significantly increased in SSc compared to MS (HR = 8.85, p < 10−3) or CD (HR = 3.45, p = 0.016). The incidence of post-aHSCT cancers was related to increasing age (HR per 10 y = 2.26, p < 10−3), without significant association with disease type. Incidence of endocrine complications were significantly lower in SSc compared to MS (HR = 0.35, p = 0.027) and CD (HR = 0.33, p = 0.041) and significantly more frequent in females (HR = 4.27, p < 10-3). Autologous HSCT for ADs is associated with a cumulative burden of non-infectious late complications, including thyroid dysfunction, osteoporosis, cardiovascular complications, gonadal dysfunction, secondary ADs and subsequent cancers. These impact all ages of patient and vary depending on AD indication, reflecting disease characteristics and other treatments. This study highlights the importance of screening, monitoring, and treatment of late effects in this setting.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1416-1422"},"PeriodicalIF":5.1,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148599140","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":"Impacts of IPSS-M and transplant conditioning intensity in allogeneic stem cell transplant for myelodysplastic neoplasms","authors":"Zilu Zhang, Xiaoqian Xu, Qi Wen, Ruoxuan Zhang, Hongru Chen, Minli Hu, Chupeng Zhang, Chuanhe Jiang, Luxiang Wang, Jiayu Huang, Zengkai Pan, Hua Jin, Yanmin Zhao, Wei Shi, Yang Cao, Xiaodong Mo, Xiaoxia Hu","doi":"10.1038/s41409-026-02939-5","DOIUrl":"10.1038/s41409-026-02939-5","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1456-1458"},"PeriodicalIF":5.1,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148599196","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":"Graft-versus-host disease prophylaxis with abatacept plus intermediate dose cyclophosphamide for GVHD prophylaxis after peripheral blood stem transplantation: a single institution study","authors":"Pallavi Surana, Mendel Goldfinger, Ioannis Mantzaris, Marina Konopleva, R. Alejandro Sica, Noah Kornblum, Aditi Shastri, Lauren C. Shapiro, Stephen Z. Peeke, Ritesh Aggarwal, Kira Gritsman, Eric J. Feldman, Alyssa de Castro, Jhannine Verceles, Royston Browne, Ridhi Gupta, David Levitz, Amanda Lombardo, Kith Pradhan, Plabani Sahu, Brittany Moavero, Angela Czavar, Amit Verma, Dennis L. Cooper","doi":"10.1038/s41409-026-03000-1","DOIUrl":"10.1038/s41409-026-03000-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1459-1460"},"PeriodicalIF":5.1,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148599182","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":"Secondary autoimmune and inflammatory diseases after allogeneic hematopoietic stem cell transplantation: a retrospective study from the EBMT Complications Working Party (CWP)","authors":"Arsene Mekinian, Artem Oganesyan, Zinaida Perić, Myriam Labopin, Urpu Salmenniemi, José Antonio Pérez Simón, Anke Verlinden, Ioanna Sakellari, Mohsen Alzahrani, Massimo Martino, Frédéric Baron, John A. Snowden, Hakan Ozdogu, Alexander Kulagin, Edgar Jost, Anna Torrent, Fabio Benedetti, Anna De Becker, Sylvie François, Montserrat Rovira, Hans Christian Reinhardt, Alessandra Biffi, Mark Ringhoffer, Pascal Ambron, Jose Luis López Lorenzo, Nabil Yafour, Alexandre Galvagni, Manuela Badoglio, Ivan Moiseev, Tobias Alexander, Mohamad Mohty, Olivier Fain, Raffaella Greco, Olaf Penack","doi":"10.1038/s41409-026-02981-3","DOIUrl":"10.1038/s41409-026-02981-3","url":null,"abstract":"Secondary autoimmune and inflammatory diseases (SAIDs) are underrecognized and poorly described after allogeneic hematopoietic stem cell transplantation (allo-HSCT). The standardization of management is hindered by the scarcity of data on epidemiology, pathogenesis, and clinical outcomes. To improve the evidence base, we performed a retrospective multicenter EBMT database study of patients who underwent allo-HSCT between 2005 and 2019 for either hematological malignancy or severe aplastic anemia with available information on SAIDs. We included 129 cases of SAIDs and 14,617 controls. The 5-year incidence of SAIDs was 0.9% (95% confidence interval (CI): 0.7–1.1), and the 10-year incidence was 1.1% (95% CI: 0.9–1.3). The median time from allo-HSCT to the diagnosis of SAIDs was 442 days [interquartile range (IQR): 242–1082]. Overall survival after the onset of SAIDs was 83.4% (95% CI 74.6–89.3) at 2 years and 73.4% (95% CI: 62–82) at 5 years. In multivariate analysis, risk factors significantly associated with SAIDs were bone marrow failure (Hazard Ratio (HR): 3.41 [95% CI, 1.55–7.52], p = 0.002), female donor to male patient (HR 1.77 [95% CI, 1.15–2.73], p = 0.009), and the presence of chronic GvHD (HR 1.61 [95% CI, 1.04–2.51], p = 0.034). SAIDs after allo-HSCT are rare but clinically significant entities requiring further investigations, exploring treatment and prevention strategies, as well as improving diagnostic approaches.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1405-1415"},"PeriodicalIF":5.1,"publicationDate":"2026-07-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148599126","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":"Autologous stem cell transplantation in POEMS syndrome: Multicenter real-world treatment patterns and outcomes","authors":"Luis Gerardo Rodríguez-Lobato, Anna de Daniel, Itziar Carro Arostegui, Iago Arribas Castro, Mercedes Gironella, Jordi Verge, Lourdes Escoda, Carles Tolosa, Josep María Martí-Tutusaus, Cristina Baca, Eugènia Abella, Cristina Motlló, Jordi López-Pardo, Albert Oriol, Carlos Castillo-Girón, Natalia Tovar, Laura Rosiñol, M. Teresa Cibeira, Anna Sureda, Carlos Fernández de Larrea","doi":"10.1038/s41409-026-02984-0","DOIUrl":"10.1038/s41409-026-02984-0","url":null,"abstract":"POEMS syndrome is a rare multisystem plasma cell disorder for which evidence guiding management is largely derived from single-center retrospective series. We conducted a retrospective multicenter study including all consecutive patients diagnosed between January 1995 and June 2024 across nine referral hospitals in Catalonia, Spain, within the GEMMAC collaborative network. Clinical characteristics, treatment strategies, transplant outcomes, and long-term survival were analyzed. Forty patients were included, with a median age of 65 years; 25 underwent autologous stem cell transplantation (ASCT). Hematologic response to first-line therapy was achieved in 77.5% of patients. After a median follow-up of 94.2 months, median progression-free survival was 102.6 months and median overall survival was 146.9 months, with 5-year rates of 69.0% and 87.6%, respectively. ASCT was associated with deep and durable hematologic and clinical responses. Baseline ECOG performance status and depth of hematologic response correlated with outcomes. VEGF levels declined markedly after treatment, particularly following ASCT. Transplant-related toxicity was manageable, although late complications were observed. These findings support ASCT as a central therapeutic strategy in eligible patients and underscore the need for long-term follow-up.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1396-1404"},"PeriodicalIF":5.1,"publicationDate":"2026-07-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-026-02984-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148590366","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}

{"title":"EASIX and sC5b-9 for early identification of adults at high risk of developing TA-TMA after allo-HCT","authors":"Wenwen Guo, Shulian Chen, Xiaoyu Zhang, Mingyue Xu, Xianjing Zhang, Jiali Wang, Yawei Zheng, Yuyan Shen, Qingzhen Liu, Xiaofei Ni, Mengnan Lv, Tingting Zhang, Yuanyuan Shi, Fengjiao Wang, Xiaoli Zhao, Jialin Wei, Rongli Zhang, Xin Chen, Yigeng Cao, Yong Li, Yueyi Mu, Aiming Pang, Mingzhe Han, Guoqing Zhu, Wenbin Cao, Erlie Jiang","doi":"10.1038/s41409-026-02994-y","DOIUrl":"10.1038/s41409-026-02994-y","url":null,"abstract":"Transplant-associated thrombotic microangiopathy (TA-TMA) remains a severe complication after allogeneic hematopoietic cell transplantation (allo-HCT). Early prediction of high-risk patients remains challenging, particularly in adults. This prospective observational study consecutively enrolled patients aged >14 years undergoing allo-HCT. TA-TMA was screened and diagnosed according to the harmonization criteria. The 100-day cumulative incidence of TA-TMA was 6.5% (95% CI 4.5–8.9%). Multivariable analyses identified elevated baseline endothelial activation and stress index (EASIX, HR 1.33, p = 0.011) and day 14 sC5b-9 (HR 2.84, p < 0.001) were independently associated with TA-TMA. The optimal cutoff was 217 ng/mL for sC5b-9 and 6.2 for EASIX. Using the cutoffs for the combined EASIX and sC5b-9, patients were stratified into low-, intermediate-, and high-risk groups, with corresponding 100-day incidences of 2.2%, 7.1%, and 32.4%. Compared with the low-risk group, the intermediate- and high-risk groups showed increased risk of TA-TMA (HR 3.29, 95% CI 1.39–7.79; HR 14.32, 95% CI 5.62–36.50, respectively). The risk stratification system showed moderate discrimination, with a time-dependent AUC of 0.75 (95% CI 0.66–0.84). Therefore, baseline EASIX and day14 sC5b-9 were significantly associated with TA-TMA, and their combination may improve early identification of adult patients at high-risk of developing TA-TMA. Name of the registry: ClinicalTrials.gov. Trial registration number: NCT06102694. The date that we registered the trial: 10/22/2023. URL of trial registry record: https://clinicaltrials.gov/study/NCT06102694?cond=NCT06102694&rank=1 .","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1388-1395"},"PeriodicalIF":5.1,"publicationDate":"2026-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148577139","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":"Baseline liver stiffness predicts SOS/VOD after allogeneic hematopoietic stem cell transplantation","authors":"Lorenzo Lazzari, Anna Francesio, Alessandro Bruno, Simona Piemontese, Andrea Acerbis, Raffaella Greco, Fabio Giglio, Andrea Assanelli, Maria Teresa Lupo-Stanghellini, Fabio Ciceri, Annalisa Ruggeri","doi":"10.1038/s41409-026-02999-7","DOIUrl":"10.1038/s41409-026-02999-7","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1453-1455"},"PeriodicalIF":5.1,"publicationDate":"2026-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148583486","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":"Are all mismatched donors the same in allogeneic hematopoietic cell transplantation for secondary acute myeloid leukemia in the posttransplant cyclophosphamide era?","authors":"Baldeep Wirk, John McCarty","doi":"10.1038/s41409-026-02996-w","DOIUrl":"10.1038/s41409-026-02996-w","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 9","pages":"1131-1132"},"PeriodicalIF":5.1,"publicationDate":"2026-07-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-026-02996-w.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148583478","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}

{"title":"Conditioning regimen for hematopoietic cell transplant in transfusion dependent thalassemia: Recommendations from the EBMT practice harmonisation and guidelines committee","authors":"Mohammed Essa, Mattia Algeri, Mohsen Alzahrani, Francoise Bernaudin, Victoria Bordon, Sonali Choudhury, Josu de la Fuente, Fabio Giglio, Erfan Nur, Valeria Maria Pinto, Francesco Onida, Annalisa Ruggeri, Isabel Sánchez-Ortega, Akif Yesilipek, Ibrahim Yakoub-Agha, Emanuele Angelucci","doi":"10.1038/s41409-026-02977-z","DOIUrl":"10.1038/s41409-026-02977-z","url":null,"abstract":"Allogeneic hematopoietic cell transplantation (HCT) has represented a curative therapy for transfusion-dependent thalassemia (TDT) for over four decades. Despite major advances in conditioning regimens, graft-versus-host disease (GVHD) prophylaxis, and supportive care, substantial heterogeneity persists in patient preparation strategies and transplant protocols across centers. To address these practice variations and provide harmonized guidance, the European Society for Blood and Marrow Transplantation (EBMT) Practice Harmonization and Guidelines Committee convened an international expert workshop in Berlin (Germany) on September 29–30, 2025. A structured literature review was conducted in advance. Evidence was presented, critically appraised, and discussed in consensus-based sessions. Recommendations were formulated using the GRADE framework and approved by panel members and external reviewers. These guidelines summarize contemporary evidence regarding pre-transplant optimization, conditioning regimens according to age and donor type, management of iron overload and ineffective erythropoiesis, and approaches for alternative donor transplantation and gene therapy. The aim of this document is to provide practical, evidence-informed recommendations to standardize HCT practice and optimize outcomes for children and adults with TDT globally, including in resource-limited settings.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1379-1387"},"PeriodicalIF":5.1,"publicationDate":"2026-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-026-02977-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148560861","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}

{"title":"Nonmyeloablative and reduced-intensity conditioning for allogeneic hematopoietic stem cell transplantation in sickle cell disease: a narrative review of safety, engraftment, and graft-versus-host-disease outcomes in adults and high-risk populations","authors":"Ghada Elgohary, Mohamed Alsharif, Riad El Fakih, Chokri Mohammed Ben Lamine, Marwan Shaheen, Mahmoud Al Jurf","doi":"10.1038/s41409-026-02982-2","DOIUrl":"10.1038/s41409-026-02982-2","url":null,"abstract":"Allogeneic hematopoietic stem cell transplantation (HSCT) is the only widely available cure for sickle cell disease (SCD), yet its use in adults has historically been limited by transplant-related toxicity. Non-myeloablative (NMA) and reduced-intensity conditioning (RIC) regimens have addressed this by maintaining durable engraftment at considerably lower risk. The NIH NMA protocol, alemtuzumab plus 300 cGy total body irradiation, achieves overall survival (OS) above 90% in matched sibling transplants, with no chronic graft-versus-host disease (GVHD) and preserved fertility. Haploidentical transplantation has likewise matured; a thiotepa-based regimen with post-transplant cyclophosphamide (PTCy) yielded 2-year OS of 95% and graft failure of just 7% in a phase 2 multicenter trial, with mixed donor chimerism of at least 20% myeloid cells sufficient for phenotypic cure. Chronic GVHD incidence differs across platforms, from 0% with NMA conditioning, 13–22% with RIC, to 22% in the haploidentical plus PTCy setting, and non-relapse mortality follows a similar pattern at 0–5% and 5–10% across these groups. Survivors report meaningful improvements in quality of life, though infectious complications and secondary malignancies continue to warrant close follow-up. Taken together, the data support NMA and RIC transplantation as genuinely curative options carrying an acceptable risk profile for adults living with SCD, and advances in haploidentical conditioning have largely put to rest the graft failure rates that once made unrelated or mismatched transplantation prohibitive. How to best target chimerism levels, reduce late toxicity, and weigh transplant against the rapidly advancing field of gene therapy are questions the field has yet to fully resolve.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 10","pages":"1308-1315"},"PeriodicalIF":5.1,"publicationDate":"2026-07-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-026-02982-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148560879","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}