{"title":"The prognostic impact of CEBPA mutation in acute myeloid leukemia patients undergoing allogeneic hematopoietic stem cell transplantation.","authors":"Xianjing Zhang, Dan Feng, Yigeng Cao, Mingyue Xu, Yuqing Liu, Wei Zhang, Rongli Zhang, Jialin Wei, Weihua Zhai, Aimin Pang, Donglin Yang, Yi He, Sizhou Feng, Mingzhe Han, Junren Chen, Erlie Jiang","doi":"10.1038/s41409-026-03068-9","DOIUrl":"https://doi.org/10.1038/s41409-026-03068-9","url":null,"abstract":"<p><p>The presence of mutations in basic leucine zipper (bZIP) region of the CEBPA gene in patients with acute myeloid leukemia (AML) is defined as a favorable risk marker according to the guidelines. A total of 173 CEBPA-mutated AML patients receiving allogeneic hematopoietic stem cell transplantation (allo-HSCT) were retrospectively recruited, compared with 345 control patients selected by propensity score matching. Patients carrying CEBPA<sup>mut</sup> had a better 5-year PFS than CEBPA<sup>wt</sup> group (60.8% vs. 55.0%, P = 0.016). Failure to achieve complete remission after first induction chemotherapy, the presence of CEBPA mutation before allo-HSCT and the absence of maintenance therapy were proven to be statistically significant factors for worse survival (all P < 0.001). Surprisingly, survival analysis revealed that patients with nontypical in-frame bZIP mutations or harboring both FLT3-ITD and CEBPA mutations had a favorable outcome of PFS (CEBPA<sup>in-frame bZIP</sup> 59.9% vs. CEBPA<sup>no in-frame bZIP</sup> 63.3% vs. CEBPA<sup>wt</sup> 55.0%, P = 0.045; CEBPA<sup>mut</sup>/FLT3-ITD<sup>mut</sup> 75.9% vs. CEBPA<sup>mut</sup>/FLT3-ITD<sup>wt</sup> 57.3% vs. CEBPA<sup>wt</sup> 55.0%, P = 0.02). Conversely, the presence of GATA2 mutations showed no benefit. Our findings suggest that CEBPA<sup>mut</sup> patients with nontypical in-frame shift in bZIP region or with the co-mutation of FLT3-ITD could benefit from allo-HSCT.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149206720","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}
Fabio Andreozzi, Aspasia Georgala, Juana Schwartz, Nina Knelange, Gloria Tridello, Malgorzata Mikulska, Jan Styczynski, Dina Averbuch
{"title":"Gaps in guidelines implementation and need for harmonization in toxoplasmosis management after allogeneic stem cell transplantation across EBMT centers: an Infectious Diseases Working Party survey.","authors":"Fabio Andreozzi, Aspasia Georgala, Juana Schwartz, Nina Knelange, Gloria Tridello, Malgorzata Mikulska, Jan Styczynski, Dina Averbuch","doi":"10.1038/s41409-026-03055-0","DOIUrl":"https://doi.org/10.1038/s41409-026-03055-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149179369","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}
Arnon Nagler, Norbert Claude Gorin, Christian Chabannon, Guillermo Puigventós, Romée Heemskerk, Isabel Sánchez-Ortega, Tuula Rintala, Yann Mahé, Emanuele Angelucci, Silvia Montoto, Michelle Kenyon, Hilda Mekelenkamp, Donal P McLornan, Antonio M Risitano, Mette Hazenberg, Anna Sureda, Ibrahim Yakoub Agha, Annalisa Ruggeri, Fabio Ciceri, Raffaella Greco
{"title":"Publisher Correction: defining global priorities in hematopoietic transplantation and cellular therapy: a statement paper from the EBMT global committee.","authors":"Arnon Nagler, Norbert Claude Gorin, Christian Chabannon, Guillermo Puigventós, Romée Heemskerk, Isabel Sánchez-Ortega, Tuula Rintala, Yann Mahé, Emanuele Angelucci, Silvia Montoto, Michelle Kenyon, Hilda Mekelenkamp, Donal P McLornan, Antonio M Risitano, Mette Hazenberg, Anna Sureda, Ibrahim Yakoub Agha, Annalisa Ruggeri, Fabio Ciceri, Raffaella Greco","doi":"10.1038/s41409-026-03046-1","DOIUrl":"10.1038/s41409-026-03046-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149163737","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":"Pleuroparenchymal fibroelastosis after allogeneic hematopoietic stem cell transplantation: Risk factors and longitudinal pulmonary function trajectories in a multicenter cohort.","authors":"Yuki Kondo, Koji Sakamoto, Seitaro Terakura, Akira Ando, Yuta Mori, Ryo Hanajiri, Shingo Iwano, Fumie Kinoshita, Tomonori Sato, Hiromichi Aso, Yasuhiro Tomita, Masashi Sawa, Yoshitaka Hibino, Kazutaka Ozeki, Masahide Oki, Hiroatsu Iida, Toshihiko Yokoyama, Tetsuya Nishida, Hitoshi Kiyoi, Makoto Ishii","doi":"10.1038/s41409-026-03069-8","DOIUrl":"https://doi.org/10.1038/s41409-026-03069-8","url":null,"abstract":"<p><p>Pleuroparenchymal fibroelastosis (PPFE) is an uncommon but serious late pulmonary complication after hematopoietic stem cell transplantation (HSCT), frequently resulting in the need for lung transplantation and contributing substantially to mortality. However, its risk factors and prediagnostic pulmonary function trajectory remain poorly defined. This multicenter retrospective cohort study included 1818 patients who underwent allogeneic HSCT between 2005 and 2023. PPFE was diagnosed using established clinical criteria. Cumulative incidence was estimated treating death as a competing event, risk factors were evaluated using cause-specific Cox models, and prediagnostic pulmonary function trajectories were analyzed using mixed-effects and segmented regression models. During a median follow-up of 42.5 months, 306 patients developed late-onset noninfectious pulmonary complications (LONIPCs), including 19 with PPFE. Although the overall incidence of LONIPCs did not differ by graft source, umbilical cord blood transplantation (UCBT) was associated with higher cumulative incidence of PPFE. In an adjusted cause-specific Cox analysis, UCBT remained associated with PPFE, whereas chronic graft-versus-host disease was not significantly associated. PPFE occurred late after transplantation and was preceded by accelerated %FVC decline approximately four years before clinical diagnosis. These findings suggest that extended pulmonary function surveillance may support earlier detection of PPFE in selected high-risk HSCT recipients.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149164611","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}
Víctor Galán-Gómez, Odelaisy León-Triana, Anna Alonso-Saladrigues, Nazaret Sanchez-Sierra, Águeda Molinos-Quintana, Blanca Herrero, Mi Kwon, Pere Barba, Anna Torrent, Mónica Cabrero, Jaime Sanz, Rafael Hernani-Duarte, Berta González-Martínez, Isabel Martínez-Romera, Wing Hong Kwan, Susana Rives, Gema Casado, José María Sánchez-Pina, Pascual Balsalobre, Antonio Pérez-Martínez
{"title":"Bridging therapy in pediatric and AYA patients under 25 years with relapsed/refractory B-ALL undergoing anti-CD19 CAR-T therapy: a multicenter retrospective study by the Spanish group GETH-TC.","authors":"Víctor Galán-Gómez, Odelaisy León-Triana, Anna Alonso-Saladrigues, Nazaret Sanchez-Sierra, Águeda Molinos-Quintana, Blanca Herrero, Mi Kwon, Pere Barba, Anna Torrent, Mónica Cabrero, Jaime Sanz, Rafael Hernani-Duarte, Berta González-Martínez, Isabel Martínez-Romera, Wing Hong Kwan, Susana Rives, Gema Casado, José María Sánchez-Pina, Pascual Balsalobre, Antonio Pérez-Martínez","doi":"10.1038/s41409-026-03067-w","DOIUrl":"https://doi.org/10.1038/s41409-026-03067-w","url":null,"abstract":"<p><p>CAR-T therapy is a powerful treatment for pediatric B-cell acute lymphoblastic leukemia (B-ALL). However, the impact of bridging therapy prior to CAR-T infusion on patient outcomes remains insufficiently explored. In this retrospective study of patients with relapse/refractory B-ALL (N = 103), we compared outcomes across different bridging therapy groups: Low-Intensity Chemotherapy (LIC, N = 50), Immunotherapy (N = 20), or High-Intensity Chemotherapy (HIC, N = 33). At day +28 post-infusion, LIC and Immunotherapy showed significantly higher rates of Complete Remission with negative Minimal Residual Disease compared with HIC (96% vs 85% vs 75%), along with lower rates of disease progression (4% vs 10% vs 15.2%) and treatment-related mortality (0% vs 5% vs 9.1%). Additionally, patients who received three or more prior lines of therapy before CAR-T infusion exhibited significantly lower response rates. Despite potential biases, such as intrinsic disease aggressiveness or unidentified genomic alterations, present findings suggest that using LIC or immunotherapy as bridging strategies, in patients with fewer prior treatment lines, may be associated with a lower overall disease and treatment burden and could potentially contribute to improved CAR-T cell efficacy and clinical outcomes. Nevertheless, additional investigations are required to elucidate biological and clinical mechanisms by which bridging approaches and treatment history modulate CAR-T responses.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149165330","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}
Mehmet Sevki Uyanik, Joseph Pidala, Mohammed A Kharfan-Dabaja
{"title":"Hyperacute Graft-Versus-Host Disease.","authors":"Mehmet Sevki Uyanik, Joseph Pidala, Mohammed A Kharfan-Dabaja","doi":"10.1038/s41409-026-03059-w","DOIUrl":"https://doi.org/10.1038/s41409-026-03059-w","url":null,"abstract":"<p><p>Hyperacute graft-versus-host disease (HA-GVHD) is a fulminant form of acute GVHD that develops early after allogeneic hematopoietic cell transplantation, before neutrophil engraftment ensues, and is associated with increased risk of non-relapse mortality. While incidence of HA-GVHD appears to be decreasing owing to in vivo or ex vivo T-cell depletion strategies, it still contributes with life-threatening side effects in the setting of human leukocyte antigen (HLA) mismatch or T-cell replete grafts with insufficient immune prophylaxis. Clinically, HA-GVHD presents almost always with severe skin involvement accompanied by fever, rash, diarrhea, and hepatic dysfunction. Differential diagnosis includes engraftment syndrome, adverse drug reactions, immunogenic skin reactions, and infectious exanthems. Therapeutic approaches mirror those prescribed for post-transplant acute GVHD, centered on high-dose systemic corticosteroids. Although there is no consensus on managing corticosteroid-refractory cases, ruxolitinib could be considered the therapy of choice based on data from post-engraftment GVHD. Further studies are needed to assess effectiveness of novel agents in corticosteroid-refractory HA-GVHD. The main purpose of this review is to summarize available literature on pathophysiology, incidence, risk factors, differential diagnosis, and treatment considerations for HA-GVHD. Knowledge gaps, particularly pertaining to use of post-transplant cyclophosphamide and treatment of corticosteroid-refractory HA-GVHD, are emphasized to highlight future research opportunities.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126565","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}
Nirav N Shah, Hannah Choe, Nancy M Hardy, Patrice Chevallier, Marie-Therese Rubio, Mark A Schroeder, Carlos Solano, Amelia Langston, Patrick Stiff, Kevin Hou, Michael Arbushites, Michael Pratta, Rodica Morariu-Zamfir, Miguel-Angel Perales
{"title":"Itacitinib plus calcineurin inhibitor-based therapy for prophylaxis of graft-versus-host disease: GRAVITAS-119 results.","authors":"Nirav N Shah, Hannah Choe, Nancy M Hardy, Patrice Chevallier, Marie-Therese Rubio, Mark A Schroeder, Carlos Solano, Amelia Langston, Patrick Stiff, Kevin Hou, Michael Arbushites, Michael Pratta, Rodica Morariu-Zamfir, Miguel-Angel Perales","doi":"10.1038/s41409-026-03056-z","DOIUrl":"10.1038/s41409-026-03056-z","url":null,"abstract":"<p><p>GRAVITAS-119 (NCT03320642), a single-arm, open-label, phase 1, proof-of-concept study, assessed addition of itacitinib to standard-of-care graft-versus-host disease (GVHD) prophylaxis regimens in reduced-intensity conditioning peripheral blood allogeneic hematopoietic stem cell transplant. Day 28 hematologic recovery (percentage of patients demonstrating neutrophil recovery and platelet recovery) was the primary endpoint. Of 84 enrolled patients, 41 received itacitinib plus tacrolimus/methotrexate (nine with antithymocyte globulin [ATG]), 24 received itacitinib plus cyclosporine A/mycophenolate mofetil (16 with ATG), and 19 received itacitinib plus post-transplant cyclophosphamide/tacrolimus. Hematologic recovery was achieved by 51/55 (92.7%) evaluable patients (neutrophil recovery, 98.7%; platelet recovery, 94.6%). Estimated 1-year GVHD-free, relapse-free survival was 39.7%; estimated 1-year overall survival was 78.3%. Cumulative incidences for grade 2‒4 and grade 3‒4 acute GVHD at Day 180 were 13.2% and 4.8%, respectively; cumulative incidence of chronic GVHD at 1 year was 37.8%. Most common grade ≥3 adverse events were cytopenias; grade ≥3 infections occurred in 23 (27.4%) patients. Most patients receiving itacitinib plus standard-of-care GVHD prophylaxis achieved hematologic recovery, with any-grade GVHD and infection rates similar to previous reports with standard-of-care prophylaxis. Addition of itacitinib to GVHD prophylaxis permits timely hematologic recovery and does not increase GVHD or infection rates, although unmet need still remains for improved regimens.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126554","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}
A B Bocanegra, C De Miguel, G Bautista, C Mosquera, A Segura, M Valdenebro, Muñoz-Sánchez Ana, I Lipperheide, M Pérez-Redondo, J Portolés, D Ballesteros, R F Duarte
{"title":"Narsoplimab in critically ill patients with high-risk transplant-associated thrombotic microangiopathy requiring intensive care.","authors":"A B Bocanegra, C De Miguel, G Bautista, C Mosquera, A Segura, M Valdenebro, Muñoz-Sánchez Ana, I Lipperheide, M Pérez-Redondo, J Portolés, D Ballesteros, R F Duarte","doi":"10.1038/s41409-026-03070-1","DOIUrl":"https://doi.org/10.1038/s41409-026-03070-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126589","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}
Anniek Stuut, Flores Weverling, Anke Janssen, Frances Verheij, Iris Brinkman, Klaartje Nijssen, Pleun Schonewille, Christien Oudbier, Anna van Rhenen, Lotte van der Wagen, Laura Daenen, Reinier Raymakers, Kasper Westinga, Tim Hutten, Lisette van de Corput, Julia Drylewicz, Jurgen Kuball, Moniek de Witte
{"title":"Peri-transplant ruxolitinib in myelofibrosis patients undergoing αβTCR and CD19-depleted stem cell transplantation.","authors":"Anniek Stuut, Flores Weverling, Anke Janssen, Frances Verheij, Iris Brinkman, Klaartje Nijssen, Pleun Schonewille, Christien Oudbier, Anna van Rhenen, Lotte van der Wagen, Laura Daenen, Reinier Raymakers, Kasper Westinga, Tim Hutten, Lisette van de Corput, Julia Drylewicz, Jurgen Kuball, Moniek de Witte","doi":"10.1038/s41409-026-03063-0","DOIUrl":"https://doi.org/10.1038/s41409-026-03063-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.1,"publicationDate":"2026-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149103915","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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