P Ahmadi, W Alsdorf, L Leypoldt, R Kosch, C Schaefers, N Gagelmann, F Ayuk, F Kron, K Weisel
{"title":"T cell redirection as a new standard of care for relapsed multiple myeloma: impact on inpatient capacity, financial burden and infrastructural requirements in Germany.","authors":"P Ahmadi, W Alsdorf, L Leypoldt, R Kosch, C Schaefers, N Gagelmann, F Ayuk, F Kron, K Weisel","doi":"10.1038/s41409-024-02505-x","DOIUrl":"https://doi.org/10.1038/s41409-024-02505-x","url":null,"abstract":"","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":"143022055","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":"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":"https://doi.org/10.1038/s41409-025-02511-7","url":null,"abstract":"<p><p>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.</p>","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}
{"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":"<p><p>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.</p>","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}
Andrew P Jallouk, Olalekan Oluwole, Bhagirathbhai Dholaria
{"title":"\"Are They Ready Yet?\": release criteria for autologous CAR T cells.","authors":"Andrew P Jallouk, Olalekan Oluwole, Bhagirathbhai Dholaria","doi":"10.1038/s41409-024-02482-1","DOIUrl":"https://doi.org/10.1038/s41409-024-02482-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143000034","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}
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":"<p><p>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.</p>","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}
Silke Heidenreich, Katharina Egger-Heidrich, Jörg P Halter, Lasse Jost, Friedrich Stölzel, Markus Perl, Alexander Denk, Matthias Edinger, Wolfgang Herr, Nicolaus Kröger, Daniel Wolff, Francis Ayuk, Matthias A Fante
{"title":"Safety and efficacy of the ROCK-2-inhibitor Belumosudil in cGvHD treatment - a retrospective, German-Swiss multicenter real-world data analysis.","authors":"Silke Heidenreich, Katharina Egger-Heidrich, Jörg P Halter, Lasse Jost, Friedrich Stölzel, Markus Perl, Alexander Denk, Matthias Edinger, Wolfgang Herr, Nicolaus Kröger, Daniel Wolff, Francis Ayuk, Matthias A Fante","doi":"10.1038/s41409-024-02507-9","DOIUrl":"https://doi.org/10.1038/s41409-024-02507-9","url":null,"abstract":"<p><p>Belumosudil is a first in class ROCK2-inhibitor approved by the FDA for the 3rd line treatment of chronic graft-versus-host disease (cGvHD). In this retrospective real-world analysis, we report safety and efficacy data of belumosudil treatment from 5 German/Swiss transplant centers. A total of 33 adult patients (median age 59 years) with moderate (n = 2) or severe (n = 31) cGvHD were treated on individual request due to lack of EMA approval. The patient cohort had a long history of cGvHD (median 44 months) and was heavily pretreated (median 4 prior lines). The overall response rate was 42% (95%CI, 25-60%) including organ responses in all organs except the liver (n = 2). The median time to response was 3 months (range, 1-9 months) and 8 of 14 patients (57%) had a durable response at last follow-up. One-third of patients had at least a 50% reduction in concomitant corticosteroid dosage. Median failure-free survival and median overall survival were 16.5 and 23.1 months, respectively. Adverse events ≥CTCAE grade 3 were reported in 27% of patients, with a predominance of infectious events, including one fatal course. The results are consistent with previous prospective trials including a favorable safety profile, while acknowledging the challenges of a heavily pretreated patient cohort.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142982684","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}
Christian J Puzo, Stuart Seropian, Henry Rinder, Christopher A Tormey, Alexa J Siddon
{"title":"Defining a lineage-specific chimerism threshold for the use of donor lymphocyte infusions in treating myeloid malignancies.","authors":"Christian J Puzo, Stuart Seropian, Henry Rinder, Christopher A Tormey, Alexa J Siddon","doi":"10.1038/s41409-025-02510-8","DOIUrl":"https://doi.org/10.1038/s41409-025-02510-8","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142977560","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":"Editorial: Molecular MRD testing in patients with acute myeloid leukemia.","authors":"Amanda L Blackmon, Michael R Grunwald","doi":"10.1038/s41409-024-02493-y","DOIUrl":"https://doi.org/10.1038/s41409-024-02493-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142969662","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}
Marco Maria Sindoni, Francesca Limido, Anita Toso, Giovanna Lucchini, Giuseppe Gaipa, Adriana Balduzzi, Silvia Nucera
{"title":"aGvHDtrackR and cGvHDtrackR: shiny applications for graft versus host disease management and clinical data collection.","authors":"Marco Maria Sindoni, Francesca Limido, Anita Toso, Giovanna Lucchini, Giuseppe Gaipa, Adriana Balduzzi, Silvia Nucera","doi":"10.1038/s41409-025-02509-1","DOIUrl":"https://doi.org/10.1038/s41409-025-02509-1","url":null,"abstract":"<p><p>Graft-versus-host disease (GvHD) is one of the most common and troublesome complications after allogeneic hematopoietic stem cell transplantation (HSCT). Despite adequate GvHD prophylaxis, 30-50% of the patients still develop acute or chronic GvHD, often requiring multiple lines of therapy. Therefore, it is crucial to closely monitor the onset and the response of GvHD to therapies to identify the best available treatment for each patient. Currently, some applications (desktop or mobile) that allow to score GvHD severity at the bedside are available. However, none of the published systems is designed to record ongoing therapies and to upload data in a database, which can support both the clinical decision-making process as well as data collection. To this aim, we developed two Shiny apps: aGvHDtrackR for acute GvHD and cGvHDtrackR for chronic GvHD. These applications record GvHD grading alongside the therapies used for each patient and allow to export of the data in a longitudinal patient-specific database. This is of help for the clinical management of patients and for future multicentric studies on GvHD.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-01-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142944722","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}