Fabio Andreozzi, Jacques-Emmanuel Galimard, Johan Maertens, Mahmoud Aljurf, Katherine Clesham, Jaime Sanz, Sebastien Maury, Sandrine Loron, Jakob Passweg, Thomas Schroeder, Urpu Salmenniemi, Gwendolyn van Gorkom, Regis Peffault de Latour, Peter A von dem Borne, Franco Locatelli, Robert F Wynn, Jean-Hugues Dalle, Jordi Esteve, Arnon Nagler, Katharina Kleinschmidt, Mohamad Mohty, Krzysztof Kalwak, Fabio Ciceri
{"title":"Outcomes of 544 Patients with t(6;9)/DEK::NUP214 acute myeloid leukemia undergoing allogeneic stem cell transplantation: an EBMT study on behalf of the acute leukemia working party (ALWP) and the pediatric diseases working party (PDWP).","authors":"Fabio Andreozzi, Jacques-Emmanuel Galimard, Johan Maertens, Mahmoud Aljurf, Katherine Clesham, Jaime Sanz, Sebastien Maury, Sandrine Loron, Jakob Passweg, Thomas Schroeder, Urpu Salmenniemi, Gwendolyn van Gorkom, Regis Peffault de Latour, Peter A von dem Borne, Franco Locatelli, Robert F Wynn, Jean-Hugues Dalle, Jordi Esteve, Arnon Nagler, Katharina Kleinschmidt, Mohamad Mohty, Krzysztof Kalwak, Fabio Ciceri","doi":"10.1038/s41409-026-02857-6","DOIUrl":"https://doi.org/10.1038/s41409-026-02857-6","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) with translocation (6;9)(p23;q34) is a rare and high-risk disease, frequently co-occurring with FLT3-ITD alteration. We retrospectively analyzed 544 patients with t(6;9) AML undergoing allogeneic hematopoietic stem cell transplantation (allo-HSCT) and reported to the EBMT registry, between 2000 and 2022. At 2-years, overall survival (OS), leukemia-free survival (LFS), relapse incidence (RI), non-relapse mortality (NRM) and GVHD-free/relapse-free survival (GRFS) were 65.7%, 59.1%, 23.0%, 17.9% and 45.6% respectively. The 431 patients transplanted in first complete remission (CR1) had more favorable OS (71.7%), LFS (65.8%) and RI (18.2%). Pediatric and adolescent/young adult (AYA, ≤ 39 years) patients in CR1 had improved outcomes. In patients ≥ 40 years old, OS, LFS, and NRM gradually worsened, whereas significant increase in RI and decrease in GRFS were observed after 53 years. In a matched-pair analysis performed on 76 FLT3-ITD positive and 76 negative CR1 patients, FLT3-ITD was associated with an approximately three-fold higher RI risk, without differences in OS. In conclusion, allo-HSCT in t(6;9) AML provides relatively favorable outcomes especially when performed in CR1. Pediatric and AYA patients derived the greatest benefit from transplant. FLT3-ITD positivity remains a relevant risk factor for relapse, though without impact on OS.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147762162","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}
Kordelia Barbullushi, Nico Gagelmann, Kristin Rathje, Anita Badbaran, Johanna Richter, Mathias Schäfersküpper, Franziska E Marquard, Radwan Massoud, Evgeny Klyuchnikov, Normann Steiner, Mirjam Reichard, Ina Rudolph, Silke Heidenreich, Christian Niederwieser, Catherina Lück, Dietlinde Janson, Christine Wolschke, Francis Ayuk, Nicolaus Kröger
{"title":"Outcomes of allogeneic transplantation in blast-phase myeloproliferative neoplasms: one-third achieve long-term survival.","authors":"Kordelia Barbullushi, Nico Gagelmann, Kristin Rathje, Anita Badbaran, Johanna Richter, Mathias Schäfersküpper, Franziska E Marquard, Radwan Massoud, Evgeny Klyuchnikov, Normann Steiner, Mirjam Reichard, Ina Rudolph, Silke Heidenreich, Christian Niederwieser, Catherina Lück, Dietlinde Janson, Christine Wolschke, Francis Ayuk, Nicolaus Kröger","doi":"10.1038/s41409-026-02842-z","DOIUrl":"https://doi.org/10.1038/s41409-026-02842-z","url":null,"abstract":"<p><p>Philadelphia-negative myeloproliferative neoplasms (MPNs) can progress to blast phase (MPN-BP), a biologically distinct and highly lethal entity with a median survival typically under six months. Allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only potentially curative approach, yet relapse and non-relapse mortality limit durable benefit. We retrospectively analyzed post-transplant outcomes in 51 consecutive adults undergoing 53 allo-HSCTs for MPN-BP. Median age was 62 years; most cases evolved from myelofibrosis, and JAK2 was the predominant driver mutation. Neutrophil engraftment occurred in all but two patients (median 12 days). At 1-year, cumulative incidences were 35.8% for grade II-IV acute GVHD, 7.5% for moderate-severe chronic GVHD, 44.3% for relapse, and 25.8% for non-relapse mortality. One-year overall survival (OS) and disease-free survival were 43.4% and 37.7%, respectively; relapse was the leading cause of death. In multivariable analysis, TP53 mutations and higher peripheral blast burden adversely affected OS, while CALR mutations appeared to be associated with improved OS, peripheral blasts also independently predicted relapse. These data underscore the cure rate of approximately one-third of MPN-BP and highlight peripheral blasts and TP53 as actionable risk markers for transplant strategies.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147697533","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}
Claudia Langebrake, Eva-Maria A Wansing, Dietlinde Janson, Christine Wolschke, Catherina Lueck, Francis Ayuk, Nicolaus M Kröger, Adrin Dadkhah
{"title":"Busulfan pharmacokinetics in adults - a real-world evaluation of intra-individual variability and the impact of obesity and deferasirox.","authors":"Claudia Langebrake, Eva-Maria A Wansing, Dietlinde Janson, Christine Wolschke, Catherina Lueck, Francis Ayuk, Nicolaus M Kröger, Adrin Dadkhah","doi":"10.1038/s41409-026-02833-0","DOIUrl":"https://doi.org/10.1038/s41409-026-02833-0","url":null,"abstract":"<p><p>To avoid under- or overexposure caused by a high variability of busulfan's pharmacokinetic parameters, therapeutic drug monitoring (TDM) is highly recommended. Nevertheless, it is not widely used in European transplant centers. We retrospectively analyzed real-world busulfan TDM data in adults in our center between 9/2016 and 11/2024 with special focus on overweight/obesity and comedication with deferasirox. Overall, 287 patients (161 male/126 female), median age 59.6 years (19.4-77.7), were included. Median calculated AUC after the first TDM was 17.1 mg*h/L with high interindividual variability (range: 9.6-37.9). Target attainment without TDM would only have been achieved in 59 patients (20.6%) using the standard dose, even if adjusted for overweight/obesity and comedication with deferasirox. Busulfan clearance (CL) was significantly reduced in patients receiving deferasirox (0.13 L/kg<sub>dosing weight</sub> [0.07-0.22] vs 0.20 [0.12-0.33], p < 0.001), while no influence of body weight was detectable. Overall, there were no Intra-individual changes in both CL (-0.65% [-41.72-57.79]) and V<sub>d</sub> (-1.40% [-36.85-69.04) between doses. However, in one third of patients relevant deviations were observed, regardless of either body weight or comedication with deferasirox. In contrast to some published data, no general decrease in CL over time was observed.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147687748","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 antiviral therapies on survival in patients with HHV-6B encephalitis after cord blood transplantation: a real-world study.","authors":"Toshiki Terao, Wataru Kitamura, Takahiro Fujino, Takami Haruyama, Yuri Okazoe, Naoyuki Uchida, Masatsugu Tanaka, Atsushi Wake, Masahito Tokunaga, Keisuke Kataoka, Masashi Sawa, Makoto Onizuka, Masako Toyosaki, Koichi Onodera, Tetsuya Nishida, Yuta Katayama, Takeshi Maeda, Yuta Hasegawa, Shunya Arai, Makoto Yoshimitsu, Takahiro Fukuda, Marie Ohbiki, Yoshiko Atsuta, Kimikazu Yakushijin, Masao Ogata, Hideki Nakasone","doi":"10.1038/s41409-026-02856-7","DOIUrl":"https://doi.org/10.1038/s41409-026-02856-7","url":null,"abstract":"<p><p>In this registry-based study, we evaluated the efficacy of different antiviral therapies for adult patients who developed human herpesvirus-6B (HHV-6B) encephalitis after their first cord blood transplantation (CBT). Among 3,862 CBT recipients registered in Japan between January 2019 and December 2023, HHV-6B encephalitis occurred in 289 patients within 100 days post-transplant (7.5%). Antiviral treatments included foscarnet (FCN) monotherapy (n = 190), ganciclovir (GCV) monotherapy (n = 12), and FCN/GCV combination therapy (n = 74). The FCN/GCV group was associated with a potential improvement in 1-year survival compared with the FCN and GCV groups (63.2% vs. 52.5% vs. 41.7%, p = 0.38). Moreover, FCN/GCV therapy was identified as an independent predictor of improved survival compared with FCN monotherapy (HR, 0.60; 95% CI, 0.38-0.95; p = 0.031). Furthermore, the combination FCN/GCV therapy showed better survival than FCN monotherapy in the patients after neutrophil engraftment (1-year survival 68.1% vs. 51.6%), but worse survival in patients before engraftment (1-year survival 34.1% vs. 54.6%). Together, these findings provide real-world evidence linking antiviral regimen selection to post-transplant outcomes in HHV-6B encephalitis and suggest that FCN/GCV combination therapy may offer a survival advantage over FCN monotherapy in selected CBT patients, with treatment strategies potentially tailored according to hematologic recovery.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147687942","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}
Anthony D Ho, Peter Dreger, Christopher Juttner, Robert Peter Gale
{"title":"Blood cell-derived transplants at 40.","authors":"Anthony D Ho, Peter Dreger, Christopher Juttner, Robert Peter Gale","doi":"10.1038/s41409-026-02865-6","DOIUrl":"https://doi.org/10.1038/s41409-026-02865-6","url":null,"abstract":"<p><p>February, 2026 is the 40th anniversary of the 1st publication of rapid recovery of bone marrow function after high-dose chemotherapy and infusion of an autologous blood- rather than a bone-marrow-derived graft. The recipient, a man with advanced Burkitt lymphoma, is in remission and alive. In the next 40 years transplants of blood-derived progenitor cells have become the most common graft for auto- and allotransplants. The rapid hematopoietic recovery after blood cell-derived grafts is associated with fewer short-term complications and better outcomes in many settings compared with bone marrow-derived grafts but may be associated with more graft-versus-host disease (GvHD) after allotransplants. Using blood-derived grafts also reduced costs, enabled the development of cell-based immune therapy and in vitro manipulation of blood-derived cells for gene and chimeric antigen receptor (CAR)-T-cell therapies.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147687768","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":"Chronic graft-versus-host disease after haploidentical transplantation in aplastic anemia: clinical characteristics, risk factors, and survival outcomes.","authors":"Ming-Hao Lin, Sheng-Nan Gao, Xiao-Jun Huang, Lan-Ping Xu, Hai-Xia Fu, Yi-Fei Cheng, Ting-Ting Han, Yuan-Yuan Zhang, Xiao-Dong Mo, Yu-Qian Sun, Yu Wang, Xiao-Hui Zhang, Zheng-Li Xu","doi":"10.1038/s41409-026-02855-8","DOIUrl":"https://doi.org/10.1038/s41409-026-02855-8","url":null,"abstract":"<p><p>To investigate the clinical characteristics, risk factors, and impacts of chronic graft-versus-host disease (cGvHD) on survival outcomes in patients with severe aplastic anemia (SAA) following haploidentical hematopoietic stem cell transplantation (haplo-HSCT), a cohort of 530 patients was analyzed. The findings revealed that 156 patients developed cGvHD, with a 5-year cumulative incidence of 29.6% (72.4% mild, 17.9% moderate, 9.6% severe). Multivariate analysis identified patient age ≥18 years (hazard ratio [HR] = 1.38, P = 0.044) and a history of grade II-IV acute GvHD (HR = 1.69, P = 0.002) as independent risk factors for cGvHD development. For severe cGvHD, risk factors included previous antithymocyte globulin treatment (HR = 2.99, P = 0.035) and a history of grade III-IV acute GvHD (HR = 4.37, P = 0.010). No significant difference in OS was observed between cGvHD and non-cGvHD groups, but the 5-year OS rate was significantly lower in patients with moderate to severe cGvHD compared to those without the condition (84.3% vs. 95.1%, P = 0.006). Among patients with cGvHD, patients with severe cGvHD had significantly lower 5-year OS (80.0%) than those with mild (97.3%) or moderate (96.4%) cGvHD (P = 0.007). These findings contribute to understanding cGvHD in SAA patients after haplo-HSCT, aiding in personalized management to improve outcomes.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147670415","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}
Aude-Marie Fourmont, David Beauvais, Marie Balsat, Johan Maertens, Edouard Forcade, Anne Huynh, Xavier Poiré, Raynier Devillier, Cristina Castilla-Llorente, Patrice Chevallier, Cécile Pochon, Etienne Daguindau, Philippe Lewalle, Claude-Eric Bulabois, Eolia Brissot, Nicole Raus, Jérôme Lambert, Stéphanie Nguyen, Nathalie Dhédin, Loïc Vasseur
{"title":"Alternative versus matched unrelated donor in adolescents and young adults transplanted for acute myeloid leukemia in first complete remission: A report from the SFGM-TC.","authors":"Aude-Marie Fourmont, David Beauvais, Marie Balsat, Johan Maertens, Edouard Forcade, Anne Huynh, Xavier Poiré, Raynier Devillier, Cristina Castilla-Llorente, Patrice Chevallier, Cécile Pochon, Etienne Daguindau, Philippe Lewalle, Claude-Eric Bulabois, Eolia Brissot, Nicole Raus, Jérôme Lambert, Stéphanie Nguyen, Nathalie Dhédin, Loïc Vasseur","doi":"10.1038/s41409-026-02853-w","DOIUrl":"https://doi.org/10.1038/s41409-026-02853-w","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147670367","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}
Allison O Taylor, Joshua S Mercer, Claire I Bradbury, Samantha M Thomas, Edwin Alyea, Nelson J Chao, Taewoong Choi, Cristina Gasparetto, Mitchell E Horwitz, Yubin Kang, Gwynn D Long, Richard D Lopez, Sendhilnathan Ramalingam, Stefanie Sarantopoulos, Anthony D Sung, Sanghee Hong
{"title":"Sociodemographic factors and timing of allogeneic hematopoietic stem cell transplantation in myeloproliferative neoplasms.","authors":"Allison O Taylor, Joshua S Mercer, Claire I Bradbury, Samantha M Thomas, Edwin Alyea, Nelson J Chao, Taewoong Choi, Cristina Gasparetto, Mitchell E Horwitz, Yubin Kang, Gwynn D Long, Richard D Lopez, Sendhilnathan Ramalingam, Stefanie Sarantopoulos, Anthony D Sung, Sanghee Hong","doi":"10.1038/s41409-026-02836-x","DOIUrl":"https://doi.org/10.1038/s41409-026-02836-x","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147662158","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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