Bone Marrow Transplantation最新文献_第3页

Enhanced antileukemic effect of NKp30b isoform of donor-derived NK cells infused after HLA-haploidentical allogeneic hematopoietic cell transplantation in high-risk AML and MDS.

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-31 DOI: 10.1038/s41409-025-02518-0

Eun-Ji Choi, Hyeon Ho Lee, Suk Ran Yoon, Inpyo Choi, Hyunkyung Park, Han-Seung Park, Yunsuk Choi, Jung-Hee Lee, Je-Hwan Lee, Hun Sik Kim, Kyoo-Hyung Lee

引用次数: 0

Relationship between age, conditioning intensity, and outcome after allografting in adults age ≥60 years with AML.

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-29 DOI: 10.1038/s41409-025-02516-2

Phuong T Vo, Brenda M Sandmaier, Megan Othus, Naveed Ali, Eduardo Rodríguez-Arbolí, Corentin Orvain, Chris Davis, Ryan S Basom, Rainer Storb, Roland B Walter

{"title":"Relationship between age, conditioning intensity, and outcome after allografting in adults age ≥60 years with AML.","authors":"Phuong T Vo, Brenda M Sandmaier, Megan Othus, Naveed Ali, Eduardo Rodríguez-Arbolí, Corentin Orvain, Chris Davis, Ryan S Basom, Rainer Storb, Roland B Walter","doi":"10.1038/s41409-025-02516-2","DOIUrl":"10.1038/s41409-025-02516-2","url":null,"abstract":"Methodological advancements now allow older adults with AML to receive allografts although conflicting data exist regarding relative outcomes across age groups and benefits of different conditioning intensities. We retrospectively analyzed 495 adults aged 60-64 (n = 184), 65-69 (n = 189), or ≥70 (n = 122) allografted for AML in remission at our institution from 2006 to 2023. There were no significant differences in relapse or relapse-free survival (RFS) among the 3 age cohorts after multivariable adjustment. Patients aged ≥70 years had higher non-relapse mortality (NRM) than those aged ≥60-64 (P = 0.022) but their overall survival (OS) was only statistically non-significantly shorter (P = 0.11). There was an important interplay between age, conditioning intensity, and outcomes. Relative to age 60-64, age ≥70 years was associated with a higher risk of relapse (hazard ratio [HR] = 3.47; P = 0.012) and NRM (HR = 3.88; P = 0.001) with reduced intensity conditioning (RIC), leading to shorter RFS (HR = 3.79; P < 0.001) and OS (HR = 3.46; P < 0.001), while no such associations were found with nonmyeloablative (NMA) conditioning. Underlying, patients aged 60-64 and 65-69, but not those aged ≥70, had a significantly lower relapse risk with RIC relative to NMA conditioning, whereas NRM risks increased across all age cohorts. Our findings support allografting for adults ≥70 with AML in remission, especially with NMA conditioning.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143063637","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}

引用次数: 0

Dynamics of neoantigen-specific T-cells in post-transplant relapse: do leukemia neoantigens elicit immune responses in transplant recipients?

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-29 DOI: 10.1038/s41409-025-02515-3

Biswas Neupane, Kedwin Ventura, Kayla Parr, Jui-En Ray Lee, Kevin Quann, Cai Chen, A John Barrett, Sawa Ito

引用次数: 0

Hematopoietic stem cell transplantation in inborn errors of metabolism-a retrospective analysis on behalf of the pediatric disease working party from the Brazilian Society of Bone Marrow Transplantation and Cellular Therapy.

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-26 DOI: 10.1038/s41409-025-02512-6

Adriana Mello Rodrigues, Juliana Folloni Fernandes, Lauro Gregianin, Samantha Nichele, Joanna Trennepohl, Rafaela Muratori, Lara Maria Miranda de Gouvêa, Gisele Loth, Polliany Pelegrina, Cilmara Kuwahara, Fernanda Benini, Carolina Almeida Peixoto, Juliana Bach, Adriana Koliski, Rebeca Toasa Gomes, Júlia Lopes Garcia, Gabriele Zamperlini Netto, Alessandra Araújo Gomes, Ana Beatriz Bechara Mafra, Fernanda Fetter Scherer, Cláudio Galvão de Castro Junior, Alberto Cardoso M Lima, Nelson Hamerschlak, Ricardo Pasquini, Liane Esteves Daudt, Carmem Bonfim

{"title":"Hematopoietic stem cell transplantation in inborn errors of metabolism-a retrospective analysis on behalf of the pediatric disease working party from the Brazilian Society of Bone Marrow Transplantation and Cellular Therapy.","authors":"Adriana Mello Rodrigues, Juliana Folloni Fernandes, Lauro Gregianin, Samantha Nichele, Joanna Trennepohl, Rafaela Muratori, Lara Maria Miranda de Gouvêa, Gisele Loth, Polliany Pelegrina, Cilmara Kuwahara, Fernanda Benini, Carolina Almeida Peixoto, Juliana Bach, Adriana Koliski, Rebeca Toasa Gomes, Júlia Lopes Garcia, Gabriele Zamperlini Netto, Alessandra Araújo Gomes, Ana Beatriz Bechara Mafra, Fernanda Fetter Scherer, Cláudio Galvão de Castro Junior, Alberto Cardoso M Lima, Nelson Hamerschlak, Ricardo Pasquini, Liane Esteves Daudt, Carmem Bonfim","doi":"10.1038/s41409-025-02512-6","DOIUrl":"https://doi.org/10.1038/s41409-025-02512-6","url":null,"abstract":"Hematopoietic stem cell transplantation (HSCT) is an established treatment for selected patients with inborn errors of metabolism. In this first report from the PDWP-SBTMO, we included 105 patients transplanted between 1988 and 2021 across six Brazilian HSCT centers. The most prevalent diseases were X-linked adrenoleukodystrophy (n = 61) and mucopolysaccharidosis (type I n = 20; type II n = 10), with a median age at HSCT of 8.7 years and 2.1 years, respectively. Most conditioning regimens were myeloablative and busulfan-based. With a median follow-up of 6.7 years, the 5-years overall survival (OS) was 75% (95% CI, 0.65-0.82) with a superior 5-years OS for those transplanted after 2010 (87% vs. 63%, p = 0.01). Higher risk of death was associated with the use of haploidentical donor (HR8.86, p 0.021), unrelated cord blood (HR 8.76, p 0.005), unrelated donor (HR 5.91, p 0.02), and for HSCT performed before 2010 (HR 4.16, p = 0.0015). The CI of acute GVHD was 24.8%, while chronic GVHD was 9.5%. Major causes of death were infections (n = 8), GVHD (n = 6), and neurologic progression (n = 3). Despite improvements in transplant outcomes since 2011, challenges persist, emphasizing the need for early diagnosis, timely transplantation and expanding HSCT centers with expertise in the field.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143045589","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}

引用次数: 0

Identification of the conditions and minimum requirements necessary for the release of autologous fresh CAR T-cell products under hospital exemption: a position paper from the WP-bioproduction of the UNITC consortium.

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-25 DOI: 10.1038/s41409-024-02504-y

Olivier Boyer, Daniele Bensoussan, Halvard Bönig, Christian Chabannon, Béatrice Clémenceau, Alexis Cuffel, Guillaume Dachy, John de Vos, Sophie Derenne, Jean-Sébastien Diana, Aurore Dougé, Laure Deramoudt, Christophe Ferrand, Edouard Forcade, Jeanne Galaine, Anne Galy, Camille Giverne, Jérémie Martinet, Chrystel Marton, Jérôme Larghero, Loïc Reppel, Sébastien Viel, Ibrahim Yakoub-Agha, Marina Deschamps

{"title":"Identification of the conditions and minimum requirements necessary for the release of autologous fresh CAR T-cell products under hospital exemption: a position paper from the WP-bioproduction of the UNITC consortium.","authors":"Olivier Boyer, Daniele Bensoussan, Halvard Bönig, Christian Chabannon, Béatrice Clémenceau, Alexis Cuffel, Guillaume Dachy, John de Vos, Sophie Derenne, Jean-Sébastien Diana, Aurore Dougé, Laure Deramoudt, Christophe Ferrand, Edouard Forcade, Jeanne Galaine, Anne Galy, Camille Giverne, Jérémie Martinet, Chrystel Marton, Jérôme Larghero, Loïc Reppel, Sébastien Viel, Ibrahim Yakoub-Agha, Marina Deschamps","doi":"10.1038/s41409-024-02504-y","DOIUrl":"https://doi.org/10.1038/s41409-024-02504-y","url":null,"abstract":"The accessibility of CAR-T cells in centralized production models faces significant challenges, primarily stemming from logistical complexities and prohibitive costs. However, European Regulation EC No. 1394/2007 introduced a pivotal provision known as the hospital exemption. This exemption enables academic institutions to produce ATMPs, including autologous CAR-T cells, under GMP conditions tailored to specific needs. In response to this regulatory framework, our work group has launched a guideline initiative. This endeavor aims to bolster academic CAR T-cell manufacturing by implementing strategic workshops that serve a dual purpose: standardizing production processes and ensuring both efficacy and safety standards are met. This inaugural focused on delineating criteria for the release of fresh CAR-T cell batches. Key emphases included thorough product characterization, stringent safety and potency evaluations. These criteria are essential for compliance with regulatory mandates and aligning with industry best practices. Notably, the release of initial CAR T-cell batches will be facilitated through provisional certification, with final certification contingent upon the acquisition of comprehensive analytical control results. This procedural framework adheres to methodologies endorsed by the SFGM-TC and the EBMT. Such adherence underscores a commitment to harmonizing practices across academic manufacturing facilities, thus fortifying the accessibility, efficacy, and safety of point-of-care units.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143036863","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}

引用次数: 0

T cell redirection as a new standard of care for relapsed multiple myeloma: impact on inpatient capacity, financial burden and infrastructural requirements in Germany.

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-22 DOI: 10.1038/s41409-024-02505-x

P Ahmadi, W Alsdorf, L Leypoldt, R Kosch, C Schaefers, N Gagelmann, F Ayuk, F Kron, K Weisel

引用次数: 0

Impact of TP53 alteration on allogeneic hematopoietic cell transplantation outcomes for myelodysplastic syndromes. TP53改变对骨髓增生异常综合征异基因造血细胞移植结果的影响。

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-22 DOI: 10.1038/s41409-025-02511-7

Cuiyan Zhou, Lanping Xu, Xiaohui Zhang, Yingjun Chang, Xiaodong Mo, Yuqian Sun, Xiaojun Huang, Yu Wang

{"title":"Impact of TP53 alteration on allogeneic hematopoietic cell transplantation outcomes for myelodysplastic syndromes.","authors":"Cuiyan Zhou, Lanping Xu, Xiaohui Zhang, Yingjun Chang, Xiaodong Mo, Yuqian Sun, Xiaojun Huang, Yu Wang","doi":"10.1038/s41409-025-02511-7","DOIUrl":"10.1038/s41409-025-02511-7","url":null,"abstract":"The poor outcome of TP53 alteration has been reported in myelodysplastic syndrome (MDS) patients. However, the role of allogeneic hematopoietic stem cell transplantation (allo-HSCT) in TP53 alteration patients remains debated. Previous studies showed that TP53 mutations had no effect on the prognosis of patients with acute leukemia after haploidentical HSCT (haplo-HSCT). The effect of haplo-HSCT on MDS patients with TP53 alterations remains to be further elucidated. We aimed to reveal the role of TP53 alterations in the prognosis of MDS patients undergoing allo-HSCT, especially haplo-HSCT. 261 MDS patients with known TP53 status were enrolled, including thirty-seven patients with TP53 mutation/deletion (TP53mut/del). TP53mut/del patients showed a worse rate of 2-year cumulative incidence of relapse (CIR) and 2-year disease-free survival (DFS) than TP53 wild type (TP53wt) patients (46.2% vs 17.0%, P < 0.001; 41.8% vs 68.9, P < 0.001) after allo-HSCT, even for those with haplo-HSCT (CIR: P < 0.001; DFS: P = 0.002). However, the prognostic effect of TP53 alteration on overall survival (OS) was not observed in patients with haplo-HSCT (66.7% vs 75.2%, P = 0.108). Positivity of post-transplantation measurable residual disease (post-MRD) and time from diagnosis to transplantation were independent risk factors for MDS patients. TP53 alterations do not affect OS in patients undergoing haplo-HSCT requires further validation.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142999998","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}

引用次数: 0

Myeloablative conditioning in cord blood transplantation for acute myeloid leukemia patients is efficacious only until age 55. 急性髓系白血病患者脐带血移植的清髓调节仅在55岁前有效。

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-21 DOI: 10.1038/s41409-025-02508-2

Shinichiro Oshima, Yasuyuki Arai, Tadakazu Kondo, Shingo Yano, Shigeki Hirabayashi, Naoyuki Uchida, Makoto Onizuka, Shigesaburo Miyakoshi, Masatsugu Tanaka, Satoshi Takahashi, Masayuki Hayashi, Toshiro Kawakita, Yasufumi Uehara, Shuichi Ota, Toru Izumi, Masashi Sawa, Tetsuya Nishida, Yuta Katayama, Koji Nagafuji, Koji Kato, Tatsuo Ichinohe, Yoshiko Atsuta, Masamitsu Yanada

{"title":"Myeloablative conditioning in cord blood transplantation for acute myeloid leukemia patients is efficacious only until age 55.","authors":"Shinichiro Oshima, Yasuyuki Arai, Tadakazu Kondo, Shingo Yano, Shigeki Hirabayashi, Naoyuki Uchida, Makoto Onizuka, Shigesaburo Miyakoshi, Masatsugu Tanaka, Satoshi Takahashi, Masayuki Hayashi, Toshiro Kawakita, Yasufumi Uehara, Shuichi Ota, Toru Izumi, Masashi Sawa, Tetsuya Nishida, Yuta Katayama, Koji Nagafuji, Koji Kato, Tatsuo Ichinohe, Yoshiko Atsuta, Masamitsu Yanada","doi":"10.1038/s41409-025-02508-2","DOIUrl":"https://doi.org/10.1038/s41409-025-02508-2","url":null,"abstract":"Umbilical cord blood transplantation (CBT) is accepted as an effective treatment for acute myeloid leukemia (AML), and reduced-intensity conditioning (RIC), rather than myeloablative conditioning (MAC) regimens allowed elderly patients to be treated safely. However, appropriate intensities of conditioning regimens are still unclear, especially for middle-aged patients. To compare outcomes after RIC and MAC regimens, we analyzed AML patients aged 16 years or older in the Japanese registry database, who underwent single cord unit CBT between 2010-2019. Median ages of the RIC group (n = 1353) and the MAC group (n = 2101) were 59 and 51 years (P < 0.001), respectively. 5-year overall survival (OS) after MAC was superior to that of RIC (38.3% vs 27.7%, P < 0.001) with lower incidence of relapse (33.9% vs 37.4%, P = 0.029) and better neutrophil engraftment (84.7% vs 75.9%, P < 0.001). Detailed subgroup analysis revealed that age at transplantation is the most important factor affecting 5-year OS in RIC and MAC. This analysis identified a threshold of 55 years, beyond which the superiority of MAC disappeared, irrespective of other factors such as disease status or performance status. In conclusion, RIC may be preferable for patients aged 56 or older in CBT for AML due to higher potential toxicities.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143000060","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}

引用次数: 0

"Are They Ready Yet?": release criteria for autologous CAR T cells. “他们准备好了吗？”：自体CAR - T细胞释放标准。

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-17 DOI: 10.1038/s41409-024-02482-1

Andrew P Jallouk, Olalekan Oluwole, Bhagirathbhai Dholaria

引用次数: 0

Incidence, risk factors, and outcomes of transplant-associated thrombotic microangiopathy in pediatric patients after allogeneic hematopoietic cell transplantation: a single-institution prospective study. 异基因造血细胞移植后儿科患者移植相关血栓性微血管病的发病率、危险因素和结局：一项单机构前瞻性研究

IF 4.5 2区医学

Bone Marrow Transplantation Pub Date : 2025-01-15 DOI: 10.1038/s41409-024-02506-w

Su Hyun Yoon, Sung Han Kang, Hyery Kim, Eun Seok Choi, Ho Joon Im, Kyung-Nam Koh

{"title":"Incidence, risk factors, and outcomes of transplant-associated thrombotic microangiopathy in pediatric patients after allogeneic hematopoietic cell transplantation: a single-institution prospective study.","authors":"Su Hyun Yoon, Sung Han Kang, Hyery Kim, Eun Seok Choi, Ho Joon Im, Kyung-Nam Koh","doi":"10.1038/s41409-024-02506-w","DOIUrl":"https://doi.org/10.1038/s41409-024-02506-w","url":null,"abstract":"Transplant-associated thrombotic microangiopathy (TA-TMA) is an increasingly recognized complication in hematopoietic cell transplantation (HCT). Given the rarity of prospective pediatric studies on TA-TMA, this study aimed to evaluate the incidence, survival outcomes, and risk factors for predicting early the development of TA-TMA in a pediatric population following allogeneic HCT. We conducted a prospective analysis of 173 pediatric patients to evaluate the incidence, survival outcome, and risk factors of TA-TMA. The cumulative incidence of TA-TMA at one-year post-HCT was 4.7% (95% CI, 2.2-8.6%). Patients with TA-TMA showed significantly poorer 1-year overall survival (OS) rate, 50.0% ± 17.7% compared to 85.4% ± 2.8% in those without TA-TMA (p = 0.008). Additionally, the non-relapse mortality (NRM) rate was higher in the TA-TMA group at 12.5% (95% CI, 3.7-55.8%) versus 7.0% (95% CI, 2.8-10.1%) (p = 0.598). A urine protein/creatinine ratio ≥ 1 mg/mg on day 30 post-HCT was significantly associated with TA-TMA occurrence (adjusted HR, 9.5; [95% CI], 1.28-70.39; p = 0.028). This study showed the significantly unfavorable clinical outcomes associated with TA-TMA in pediatric patients and emphasized the importance of early identification of patients at risk. Further research is needed to explore additional strategies for early detection and intervention to improve outcomes.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143000056","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}

引用次数: 0