Bone Marrow Transplantation最新文献

{"title":"Correction: Placental expanded mesenchymal-like cells (PLX-R18) for poor graft function after hematopoietic cell transplantation: A phase I study.","authors":"Joseph P McGuirk, Leland Metheny, Luis Pineiro, Mark Litzow, Scott D Rowley, Batia Avni, Roni Tamari, Hillard M Lazarus, Jacob M Rowe, Michal Sheleg, Daniel Rothenstein, Nitsan Halevy, Tsila Zuckerman","doi":"10.1038/s41409-024-02469-y","DOIUrl":"https://doi.org/10.1038/s41409-024-02469-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-12-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142766310","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":"Efficacy and prognostic assessment of chemotherapy-bridged transplantation in pediatric patients with advanced myelodysplastic syndromes.","authors":"Xingchen Wang, Chenmeng Liu, Yunlong Chen, Yang Wan, Wenbin An, Xiaolan Li, Lipeng Liu, Fang Liu, Li Zhang, Yao Zou, Xiaojuan Chen, Yumei Chen, Ye Guo, Xiaofan Zhu, Wenyu Yang","doi":"10.1038/s41409-024-02488-9","DOIUrl":"https://doi.org/10.1038/s41409-024-02488-9","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142766334","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":"Outcomes of allogeneic hematopoietic stem cell transplantation versus intensive chemotherapy in patients with myeloid sarcoma: a nationwide representative multicenter study.","authors":"Jie Sun, Yi-Cheng Zhang, Jia Wei, Ya-Jing Xu, Yue Zhang, Yu-Hua Li, An-Qin Wu, Lei Fan, Yu Zhu, Feng-Qi Liu, Zhong-Xing Jiang, Chao Liu, Ming Jiang, Jian-Hua Qu, Peng-Cheng He, Jie Wang, Xiao-Bing Huang, Rong Xiao, Su-Jun Gao, Qiang Guo, San-Bin Wang, Xiao-Ping Li, Sheng-Jin Fan, Li-Li Sun, Lan-Ping Xu, Xiao-Jun Huang, Xiao-Hui Zhang","doi":"10.1038/s41409-024-02485-y","DOIUrl":"https://doi.org/10.1038/s41409-024-02485-y","url":null,"abstract":"<p><p>Myeloid sarcoma (MS) is a rare hematological neoplasm with poor prognosis, posing a significant clinical challenge due to the absence of effective and standardized treatments. We conducted a retrospective analysis of 162 MS patients treated at 12 centers to compare outcomes between intensive chemotherapy and allogeneic hematopoietic stem cell transplantation (allo-HSCT). Our analysis revealed that allo-HSCT demonstrated superior overall survival (OS) within the initial 36 months compared to intensive chemotherapy alone (p = 0.037). However, beyond 36 months (36-60 months), a reverse trend was observed (p = 0.056). Subgroup analysis revealed potential benefit for isolated MS patients with allo-HSCT, but not for those with leukemic MS. Additionally, in patients achieving first complete remission (CR1) after induction chemotherapy, allo-HSCT did not significantly improve 5-year OS compared with intensive chemotherapy alone (p = 0.25). Conversely, allo-HSCT significantly improved 5-year OS in non-CR1 patients (p < 0.001). Notably, HLA-matched HSCT and haploidentical HSCT showed comparable outcomes in terms of OS, disease-free survival, and cumulative incidence of relapse. In conclusion, allo-HSCT improved outcomes for MS patients within 36 months of disease onset, and haploidentical HSCT emerged as a viable treatment option for patients without matched donors.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-12-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142766341","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":"High level serum IL-6 predicts occurrence of TA-TMA.","authors":"Renzhi Zheng, Lin Li, Jianzhi Zhao, Diange Jin, Jimin Shi, Yanmin Zhao, Jian Yu, Xiaoyu Lai, Lizhen Liu, Huarui Fu, Congxiao Zhang, Jia Shen, He Huang, Yi Luo, Yishan Ye","doi":"10.1038/s41409-024-02478-x","DOIUrl":"https://doi.org/10.1038/s41409-024-02478-x","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142738257","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":"A multifactorial risk scoring system for the prediction of early relapse in CMML patients with allo-HSCT: a nationwide representative multicenter study.","authors":"Jian-Ying Zhou, Yu-Xiu Chen, Hai-Long Yuan, Ya-Jing Xu, Xiao-Bing Huang, Su-Jun Gao, Yi-Cheng Zhang, Fang Zhou, Xian-Min Song, Yi Luo, Jian-Min Yang, Yu-Hua Li, Shun-Qing Wang, Yu-Jun Dong, Xi Zhang, Yi-Mei Feng, Xin Du, Han Zhu, Zun-Min Zhu, Ke-Hong Bi, Ming Jiang, Ting Niu, Ding-Ming Wan, Yi Chen, Li Liu, Hai Yi, Yu-Hong Chen, Feng-Rong Wang, Yuan-Yuan Zhang, Xiao-Dong Mo, Wei Han, Jing-Zhi Wang, Yu Wang, Huan Chen, Xiang-Yu Zhao, Ying-Jun Chang, Kai-Yan Liu, Xiao-Jun Huang, Xiao-Hui Zhang","doi":"10.1038/s41409-024-02480-3","DOIUrl":"https://doi.org/10.1038/s41409-024-02480-3","url":null,"abstract":"<p><p>Chronic myelomonocytic leukemia (CMML) is a clonal hematopoietic stem cell malignancy and the only curable therapy is allogeneic hematopoietic stem cell transplantation (allo-HSCT). However, allo-HSCT is not appropriate for all CMML patients, and relapse is the leading cause of treatment failure. This project conducted a nationwide multicenter real-world study to develop a novel prediction scoring system for early relapse. A total of 238 CMML patients from twenty-seven medical centers treated with allo-HSCT, and 307 adult patients with CMML who underwent allo-HSCT in a publicly available research dataset from the Center for International Blood and Marrow Transplantation Registry (CIBMTR) database were included. Independent prognostic factors for the early relapse of CMML posttransplantation were identified according to competing risk regression methods. Four prognostic factors were identified: bone marrow blasts >10% (hazard ratio [HR], 4.262; P = 0.014), age >60 years (HR, 6.221; P = 0.007), hemoglobin level <100 g/L (HR, 3.695; P = 0.004), and non TET2 gene mutation (HR, 3.425; P = 0.017). A risk-grading scoring system was developed based on the regression coefficients and patients were stratified into low-risk (0-1 point), intermediate-risk (1.5-2 points) and high-risk ( > 2 points) groups. The validated internal c-statistic was 0.767 (95% confidence interval [CI], 0.674-0.860), and the external c-statistic was 0.769 (95% CI, 0.703-0.836). In the derivation cohort, the cumulative incidence rates of early relapse in the low-risk, intermediate-risk, and high-risk groups were 1.35% (95% CI: 1-4%), 10.40% (95% CI: 4-16%), and 29.54% (95% CI: 16-39%) (P < 0.001), respectively. This scoring system can be utilized to early identification of patients at a high risk of relapse and contributing to the implementation of urgent medical support.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142715366","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":"Utilization of hematopoietic cell transplantation and cellular therapy technology in Europe and associated Countries. Using the 2022 activity survey data to correlate with economic and demographic factors. A report from the EBMT","authors":"Jakob R. Passweg,&nbsp;Helen Baldomero,&nbsp;Tobias Alexander,&nbsp;Emanuele Angelucci,&nbsp;Dina Averbuch,&nbsp;Ali Bazarbachi,&nbsp;Fabio Ciceri,&nbsp;Greco Raffaella,&nbsp;Mette D. Hazenberg,&nbsp;Krzysztof Kalwak,&nbsp;Donal P. McLornan,&nbsp;Antonio M. Risitano,&nbsp;Annalisa Ruggeri,&nbsp;John A. Snowden,&nbsp;Anna Sureda","doi":"10.1038/s41409-024-02459-0","DOIUrl":"10.1038/s41409-024-02459-0","url":null,"abstract":"We looked at treatment rates and center density across countries for patients treated in 2022; 46,143 HCTs (19,011 (41.2%) allogeneic, 27,132 (58.8%) autologous) reported by 689 centers. 4329 patients received advanced cellular therapies, 3205 were CAR-T. We found considerable differences in utilization of autologous, allogeneic HCT and more so for CAR-T. Differences in procedure type and for allogeneic HCT in donor use and disease indication are highlighted. For instance, countries with the highest use of unrelated donors per 10 million inhabitants were Germany (297) and the Netherlands (230), for identical sibling HCT it was Israel (148) and Lebanon (113), for haploidentical it was Israel (94) and Italy (94) and for cord blood it was the Netherlands (24) and the United Kingdom (15). We looked at HCT use for specific indications in allogeneic HCT (AML CR1, MDS, MPN and BMF). We correlated treatment rates with GNI and with demographic age structure and show correlations in HCT and CAR-T use and center density, highest in Italy for allogeneic and autologous HCT and in Switzerland for CAR-T. Resource restricted countries tend to concentrate HCT use in a limited number of centers. These data are useful for comparisons across countries.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 2","pages":"227-236"},"PeriodicalIF":4.5,"publicationDate":"2024-11-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-024-02459-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142692647","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":"The BLIND study: blinatumomab and DLI approach for management of B-ALL relapse after allogeneic stem cell transplantation. A multicentric Italian experience.","authors":"S Giammarco, R Maggi, Luca Di Marino, P Chiusolo, L Teofili, N Piccirilo, E Metafuni, M A Limongiello, S Leotta, G Milone, A Cupri, I Cutini, R Saccardi, C Nozzoli, R Boncompagni, A Gozzini, B Scappini, M Piccini, N S Fracchiolla, S Sica","doi":"10.1038/s41409-024-02475-0","DOIUrl":"https://doi.org/10.1038/s41409-024-02475-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142680095","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":"Measurable residual disease testing and allogeneic hematopoietic cell transplantation for AML: adapting Pre-MEASURE to clinical practice","authors":"Brian C. Shaffer,&nbsp;Partow Kebriaei,&nbsp;Marcos de Lima,&nbsp;Antonio M. Jimenez Jimenez","doi":"10.1038/s41409-024-02481-2","DOIUrl":"10.1038/s41409-024-02481-2","url":null,"abstract":"Measurable residual disease (MRD) testing in patients with acute myelogenous leukemia (AML) represents a heterogenous assessment process designed to quantify leukemia-specific biomarkers that are not ascertainable by routine pathologic evaluation. The most common tools used to assess MRD are multiparameter flow cytometry (MPFC), and polymerase chain reaction (PCR) based tools, including quantitative or digital droplet PCR (qPCR, ddPCR), or next-generation sequencing (NGS) technologies. Collectively, MRD assessments have become an important clinical tool in the management of patients with AML. Despite progress, significant questions remain with respect to the appropriate timing, frequency, and methodology of MRD assessment, and whether or how to adapt therapy based on MRD results. Recent data from the Pre-MEASURE study, a retrospective cohort analysis of error corrected NGS based MRD assessment prior to allogeneic hematopoietic cell transplantation (alloHCT) in patients with AML, provides additional key information with respect to the emerging role of NGS-based technology in MRD assessment. In the context of this review, we evaluate the Pre-MEASURE study as well as other recent, high-quality assessments of MRD in AML. Our focus is to provide a practical assessment of the use of emerging MRD technologies in patients with AML with an emphasis on the role of peri-transplant MRD for the practicing clinician.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 2","pages":"128-134"},"PeriodicalIF":4.5,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-024-02481-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142675089","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":"The role of daratumumab in complications post-allogeneic hematopoietic stem cell transplantation: a single-center prospective study on PRCA and AIHA.","authors":"S Giammarco, M A Limongiello, Luca Di Marino, Elisabetta Metafuni, Luciana Teofili, Patrizia Chiusolo, Simona Sica","doi":"10.1038/s41409-024-02479-w","DOIUrl":"https://doi.org/10.1038/s41409-024-02479-w","url":null,"abstract":"<p><p>Pure red cell aplasia (PRCA) and autoimmune hemolytic anemia (AIHA) post-hematopoietic stem cell transplantation (HSCT) are an unmet medical need with no established standard of care, significantly affecting the patient quality of life and posing a challenge for clinicians. The anti-CD38 IgG-kappa Daratumumab appears to be a safe and efficace treatment compared to prior drugs. Our study is a prospective monocentric investigation assessing the use of daratumumab in these complications following allo-HSCT. Here we describe our experience on six patients with a median age of 65 years. All treated patients, except one, who died because of sepsis during GVHD exacerbation, reached transfusion independence with erythropoietin suspension. Poor graft function remains a management challenge for clinicians and has a significant impact on the patient's quality of life. Currently, therapeutic options for PRCA and for the least common AIHA, appear ineffective, making it difficult to address the diverse needs of post-transplant patients. Although our data and those previously reported in the literature are preliminary, daratumumab prompts further reflection on its use in this setting of patients.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142680394","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":"High-dose chemotherapy with autologous haematopoietic stem cell transplantation in patients with isolated vitreoretinal lymphoma: a LOC network study.","authors":"Adam Mainguy, Carole Soussain, Valérie Touitou, Amin Bennedjai, Laurent Kodjikian, Hervé Ghesquières, Gandhi Damaj, Rémy Gressin, Jean-Baptiste Ducloyer, Olivier Chinot, Anaïs Vautier, Cécile Moluçon-Chabrot, Guido Ahle, Luc Taillandier, Jean Pierre Marolleau, Adrien Chauchet, Fabrice Jardin, Nathalie Cassoux, Denis Malaise, Adélaïde Toutée, Sara Touhami, Magali Le Garff-Tavernier, Khê Hoang-Xuan, Sylvain Choquet, Caroline Houillier","doi":"10.1038/s41409-024-02477-y","DOIUrl":"https://doi.org/10.1038/s41409-024-02477-y","url":null,"abstract":"<p><p>Despite its indolent evolution, vitreoretinal lymphoma (VRL) has a poor prognosis due to a major risk of relapse in the central nervous system (CNS) and may necessitate aggressive therapy. However, the use of high-dose chemotherapy with autologous stem cell transplantation (HCT-ASCT) is poorly documented. We retrospectively analysed from the French LOC network database the adult immunocompetent patients treated with HCT-ASCT for isolated VRL. Thirty-eight patients underwent consolidation with HCT-ASCT for isolated VRL between 2008 and 2019 after induction chemotherapy. Twenty patients had primary VRL, and 18 had an isolated VRL relapse of a primary CNS lymphoma. Three patients underwent HCT-ASCT in first-line treatment, 24 in second-line treatment, and 11 in subsequent lines. At HCT-ASCT, the median age was 61 years, and the median KPS was 90. Thirty-two patients (84%) received high-dose thiotepa-based HCT. One patient (3%) died from HCT-ASCT toxicity. Nineteen (50%) patients relapsed after HCT-ASCT, including 17 cases occurring in the brain. The median progression-free survival, brain-free survival and overall survival from HCT-ASCT were 96, 113 and 92 months, respectively. HCT-ASCT represents an effective therapeutic strategy for select VRL patients, with a tolerable safety profile. However, the risk of subsequent brain relapse remains significant.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2024-11-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142674734","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}