{"title":"Allogeneic hematopoietic stem cell transplantation in elderly patients with AML and MDS: navigating risks and optimizing outcomes.","authors":"Mingyang Wang, Jieya Luo, Didier Blaise, Raynier Devillier, Erlie Jiang","doi":"10.1038/s41409-026-02872-7","DOIUrl":"https://doi.org/10.1038/s41409-026-02872-7","url":null,"abstract":"<p><p>The growing burden of acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) among aging populations underscores the critical need for tailored treatment strategies for elderly patients. While allogeneic hematopoietic stem cell transplantation (allo-HSCT) remains the only potentially curative option for eligible patients, its application in older adults is fraught with significant challenges. This review explores recent advancements in optimizing allo-HSCT for elderly patients with AML and MDS. Key considerations include assessing patient fitness, predicting transplant outcomes, refining conditioning regimens, and designing personalized treatment strategies. Optimizing the comprehensive management of elderly patients across the pre-transplant, transplant, and post-transplant phases is expected to broaden access to allo-HSCT and enhance its curative potential.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-05-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147855949","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}
Roser Velasco, Martin Schmidt-Hieber, Olaf Penack, Dina Averbuch, Aitana Balaguer-Roselló, Francesco Baccelli, Luis Bataller, Simone Cesaro, Chiara Briani, Rafael de la Cámara, Petra Huehnchen, Thushan de Silva, Alberto Mussetti, Per Ljungman, Zinaida Perić, Malgorzata Mikulska, Alexey Polushin, David Navarro Ortega, Albert Pons-Escoda, Dionysios Neofytos, Hélène Schoemans, José Luis Piñana, Francesco Onida, Annalisa Ruggeri, Isabel Sanchez-Ortega, Ibrahim Yakoub-Agha, Jan Styczynski
{"title":"Symptom-guided approach to central nervous system complications in allogeneic hematopoietic cell transplant recipients: Best practice recommendations from the EBMT Practice Harmonisation and Guidelines committee.","authors":"Roser Velasco, Martin Schmidt-Hieber, Olaf Penack, Dina Averbuch, Aitana Balaguer-Roselló, Francesco Baccelli, Luis Bataller, Simone Cesaro, Chiara Briani, Rafael de la Cámara, Petra Huehnchen, Thushan de Silva, Alberto Mussetti, Per Ljungman, Zinaida Perić, Malgorzata Mikulska, Alexey Polushin, David Navarro Ortega, Albert Pons-Escoda, Dionysios Neofytos, Hélène Schoemans, José Luis Piñana, Francesco Onida, Annalisa Ruggeri, Isabel Sanchez-Ortega, Ibrahim Yakoub-Agha, Jan Styczynski","doi":"10.1038/s41409-026-02866-5","DOIUrl":"https://doi.org/10.1038/s41409-026-02866-5","url":null,"abstract":"<p><p>Central nervous system complications (CNSC) after allogeneic hematopoietic cell transplantation (allo-HCT) are relatively common and may have a major impact on patients´ outcomes. Neurological symptoms may occur at any point during the post-transplant course, although they are more common early after HCT, with a wide range of possible presentations. As many of CNSC symptoms are non-specific, the initial assessment is often difficult, and early decisions regarding the diagnostic work-up can influence the identification of the underlying cause. Blood tests, neuroimaging and cerebrospinal fluid (CSF) analyses are particularly important, as they frequently provide the first clues that help narrow the very wide differential diagnoses. In view of these challenges, an international EBMT working group composed of haematologists, infectious disease specialists, neurologists, a neuroradiologist, and a microbiologist developed symptom-based recommendations for the early evaluation of CNSC after allo-HCT. The work summarizes key aspects of the clinical presentation and outlines practical steps for the initial diagnostic approach. These recommendations are intended to support haematologists, who are usually the first to assess these patients, in promptly recognizing CNSC so to start timely appropriate management.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147833611","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}
Rafael Colmenares, Eva Barragán, Rebeca Rodríguez-Veiga, Laura Torres-Miñana, Joaquín Sánchez-García, Mar Tormo, Teresa Bernal, Pilar Martínez-Sánchez, Eduardo Rodríguez-Arbolí, Cristina Gil, Elena Soria-Saldise, Josefina Serrano, Mercedes Colorado, María García-Fortes, Cristina Bilbao, José Luis López-Lorenzo, María José Larráyoz, Esther Pérez-Santaolalla, Esperanza Lavilla-Rubira, Lorenzo Algarra, María Carmen García-Garay, Carmen Chillón, Melissa Torres-Ochando, Carmen Couto, Raimundo García-Boyero, Ágata Almela, Víctor Noriega, Marta Callejas, Manuel Barrios, Soledad Casado, Amaia Balerdi, Ana Cabello, Jorge Labrador, María Carmen Mateos, María Luz Amigo, Manuel Pérez-Encinas, María José García-Pérez, Lissette Costilla, Juan Bergua, Gonzalo Carreño-Tarragona, Joaquín Martínez-López, Rosa Ayala, Pau Montesinos
{"title":"Impact of molecular profiling in patients with acute myeloid leukemia undergoing allogeneic transplantation in first remission: a study by the PETHEMA group.","authors":"Rafael Colmenares, Eva Barragán, Rebeca Rodríguez-Veiga, Laura Torres-Miñana, Joaquín Sánchez-García, Mar Tormo, Teresa Bernal, Pilar Martínez-Sánchez, Eduardo Rodríguez-Arbolí, Cristina Gil, Elena Soria-Saldise, Josefina Serrano, Mercedes Colorado, María García-Fortes, Cristina Bilbao, José Luis López-Lorenzo, María José Larráyoz, Esther Pérez-Santaolalla, Esperanza Lavilla-Rubira, Lorenzo Algarra, María Carmen García-Garay, Carmen Chillón, Melissa Torres-Ochando, Carmen Couto, Raimundo García-Boyero, Ágata Almela, Víctor Noriega, Marta Callejas, Manuel Barrios, Soledad Casado, Amaia Balerdi, Ana Cabello, Jorge Labrador, María Carmen Mateos, María Luz Amigo, Manuel Pérez-Encinas, María José García-Pérez, Lissette Costilla, Juan Bergua, Gonzalo Carreño-Tarragona, Joaquín Martínez-López, Rosa Ayala, Pau Montesinos","doi":"10.1038/s41409-026-02871-8","DOIUrl":"https://doi.org/10.1038/s41409-026-02871-8","url":null,"abstract":"<p><p>Acute myeloid leukemia (AML) is a heterogeneous malignancy with a poor prognosis. Genetic and molecular profiling help guide treatment decisions, including the use of allogeneic hematopoietic stem cell transplantation (allo-HSCT), to reduce relapse risk. This study evaluated the impact of individual and co-mutational genetic profiles in AML patients in first complete remission after receiving allo-HSCT using data from the PETHEMA registry. A retrospective analysis assessed overall survival and relapse-free survival (RFS). Cox regression identified significant variables used to develop a risk score based on hazard ratios, incorporating age, AML type, transplant timing, and genetic/molecular alterations. A total of 717 patients (median age 56.5 years) were included, most classified as adverse risk by ELN2022 criteria. Both ELN2017 and ELN2022 risk classifications were validated. Multivariate analysis showed that DNMT3A, SF3B1, TP53, and WT1 mutations were linked to shorter RFS, whereas FLT3-ITD mutations correlated with prolonged RFS. These findings were integrated into a proposed prognostic score, which was validated. Therefore, this study highlights the prognostic importance of genetic mutations in AML patients undergoing allo-HSCT. These insights could inform pre-transplant strategies, including donor selection and conditioning regimens, as well as post-transplant maintenance therapy.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-05-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147833602","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}
R Van Dijck, B van der Holt, A E C Broers, M D Hazenberg, T Devos, M W M van der Poel, D Breems, L Deleu, B A G Schuthof, C M C van Hooije, J J Cornelissen, N P M Schaap, P A W Te Boekhorst
{"title":"Correction: Pacritinib in transplant-eligible myelofibrosis: final analysis of the phase II HOVON-134 trial.","authors":"R Van Dijck, B van der Holt, A E C Broers, M D Hazenberg, T Devos, M W M van der Poel, D Breems, L Deleu, B A G Schuthof, C M C van Hooije, J J Cornelissen, N P M Schaap, P A W Te Boekhorst","doi":"10.1038/s41409-026-02900-6","DOIUrl":"https://doi.org/10.1038/s41409-026-02900-6","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147833590","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}
Jan Styczynski, Gloria Tridello, Inge Verheggen, Nina Knelange, Lotus Wendel, Per Ljungman, Jose Luis Piñana, Malgorzata Mikulska, Lidia Gil, Simone Cesaro, Fabio Ciceri, Raffaella Greco, Depei Wu, Didier Blaise, Raffaella Cerretti, Patrice Chevallier, Edouard Forcade, Flore Sicre de Fontbrune, Mohamad Mohty, Sophie Ducastelle Lepretre, Claude Eric Bulabois, Stephanie Nguyen Quoc, Ibrahim Yakoub-Agha, Rafael de la Camara, Dina Averbuch
{"title":"Impact of donor and recipient EBV serostatus on transplant outcomes in patients with acute leukemia after haploidentical hematopoietic cell transplantation with post-transplant cyclophosphamide: Infectious Diseases Working Party EBMT retrospective study.","authors":"Jan Styczynski, Gloria Tridello, Inge Verheggen, Nina Knelange, Lotus Wendel, Per Ljungman, Jose Luis Piñana, Malgorzata Mikulska, Lidia Gil, Simone Cesaro, Fabio Ciceri, Raffaella Greco, Depei Wu, Didier Blaise, Raffaella Cerretti, Patrice Chevallier, Edouard Forcade, Flore Sicre de Fontbrune, Mohamad Mohty, Sophie Ducastelle Lepretre, Claude Eric Bulabois, Stephanie Nguyen Quoc, Ibrahim Yakoub-Agha, Rafael de la Camara, Dina Averbuch","doi":"10.1038/s41409-026-02898-x","DOIUrl":"https://doi.org/10.1038/s41409-026-02898-x","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-05-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147833548","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}
Reem Alasbali, Mohsen Alzahrani, Karina Wilkerson, Phavina Akhom, Fahad Alotaibi, Bader Alahmari, Josu de la Fuente, Michael R DeBaun, Walid Rasheed, Hadeel Samarkandi, Riad El Fakih, Mostafa Saleh, Tusneem Elhassan, Hazza Alzahrani, Mahmoud Aljurf, Fahad Almohareb, Adetola A Kassim, Ali AlAhmari
{"title":"Excellent outcomes using a novel reduced intensity conditioning with thiotepa and post-transplant cyclophosphamide for HLA-matched related donor transplant in adolescents and adults with sickle cell disease.","authors":"Reem Alasbali, Mohsen Alzahrani, Karina Wilkerson, Phavina Akhom, Fahad Alotaibi, Bader Alahmari, Josu de la Fuente, Michael R DeBaun, Walid Rasheed, Hadeel Samarkandi, Riad El Fakih, Mostafa Saleh, Tusneem Elhassan, Hazza Alzahrani, Mahmoud Aljurf, Fahad Almohareb, Adetola A Kassim, Ali AlAhmari","doi":"10.1038/s41409-026-02876-3","DOIUrl":"https://doi.org/10.1038/s41409-026-02876-3","url":null,"abstract":"<p><p>Myeloablative HLA-matched related donor (MRD) hematopoietic stem cell transplantation (HSCT) is a potentially curative treatment for children with sickle cell disease (SCD), achieving survival rates over 90%. However, optimal conditioning for older SCD patients undergoing MRD transplantation is unknown. We evaluated a novel conditioning regimen comprising thymoglobulin, thiotepa, cyclophosphamide, fludarabine, and total-body irradiation (200 cGy). Graft-versus-host disease (GVHD) prophylaxis included post-transplant cyclophosphamide (100 mg/kg), mycophenolate mofetil, and sirolimus. We aimed to improve donor engraftment rates, decrease GVHD, and minimize toxicity. A total of 25 patients were prospectively recruited in a multicentre study across three sites; 22/25 (88%) were fully matched, and 3/25 (12%) received a one-antigen-mismatched sibling transplant. 12% (3/25) of patients had experienced a second graft failure from a previous HCT. 11 participants (44%) received bone marrow, while 14 (56%) received peripheral blood as stem cell sources. The median age was 26.7 years (IQR, 18.53-32.02), and the median follow-up was 42 months (IQR, 30-49). The estimated 5-year event-free survival rate was 96%, and the 2-year overall survival rate was 100%. No graft failure was observed. Median whole blood chimerism at day +28 and day +365 engraftment was 100% and 97%, respectively. Among evaluable participants, 17/21 (81%) were off immunosuppression at 1-year post-transplant. The 1-year grade III-IV acute GVHD rate was 4% (n = 1), with no chronic GVHD. This regimen produced high engraftment and survival in this cohort using our novel conditioning regimen for MRD HSCT in adolescents and adults with severe SCD, including those undergoing a second HSCT. Optimal donor engraftment is associated with a lower risk of secondary myeloid neoplasms.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147762196","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}
Anne Pralong, Steffen T Simon, Marco Herling, Alinda Reimer, Berenike Schoerger, Lisa Pusch, Christoph Scheid, Michael Hallek, Jithmi Weliwitage, Martin Hellmich, Raymond Voltz, Udo Holtick
{"title":"Goals-of-care transition after an allogeneic hematopoietic stem-cell transplantation: A cross-sectional survey among hematologists on associated challenges.","authors":"Anne Pralong, Steffen T Simon, Marco Herling, Alinda Reimer, Berenike Schoerger, Lisa Pusch, Christoph Scheid, Michael Hallek, Jithmi Weliwitage, Martin Hellmich, Raymond Voltz, Udo Holtick","doi":"10.1038/s41409-026-02873-6","DOIUrl":"https://doi.org/10.1038/s41409-026-02873-6","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147762216","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}
Fernando Barroso Duarte, Yhasmine Delles Oliveira Garcia, Vanderson Rocha, Vaneuza Araújo Moreira Funke, Maria Claudia Rodrigues Moreira, Alessandra Aparecida Paz, Jayr Schmidt Filho, Claudia Caceres Astigarraga, Roberto Luiz da Silva, Vinícius Campos de Molla, Alexandre Silvério, João Victor Piccolo Feliciano, George Maurício Navarro Barros, Vergílio Antônio Rensi Colturato, Samir Kanaan Nabhan, João Samuel de Holanda Farias, Ana Carolina Arrais Maia, Ângelo Atalla, Ricardo Chiattone, Maria Cristina Martins de Almeida Macedo, Milton Alexandre Ferreira Aranha, Yana Augusta Novis Zogbi, Décio Lener, Rodolfo Daniel de Almeida Soares, Phillip Scheinberg, Rodolfo Froes Calixto, Gustavo Machado Teixeira, Marcos Paulo Colella, Celso Arrais Rodrigues, Anderson João Simione, Cinthya Corrêa da Silva, Nelson Hamerschlak
{"title":"Patients with myelodysplastic/myeloproliferative neoplasms have higher rates of relapse following allogeneic hematopoietic cell transplantation compared to those with MDS: A Brazilian SBTMO/CIBMTR registry analysis.","authors":"Fernando Barroso Duarte, Yhasmine Delles Oliveira Garcia, Vanderson Rocha, Vaneuza Araújo Moreira Funke, Maria Claudia Rodrigues Moreira, Alessandra Aparecida Paz, Jayr Schmidt Filho, Claudia Caceres Astigarraga, Roberto Luiz da Silva, Vinícius Campos de Molla, Alexandre Silvério, João Victor Piccolo Feliciano, George Maurício Navarro Barros, Vergílio Antônio Rensi Colturato, Samir Kanaan Nabhan, João Samuel de Holanda Farias, Ana Carolina Arrais Maia, Ângelo Atalla, Ricardo Chiattone, Maria Cristina Martins de Almeida Macedo, Milton Alexandre Ferreira Aranha, Yana Augusta Novis Zogbi, Décio Lener, Rodolfo Daniel de Almeida Soares, Phillip Scheinberg, Rodolfo Froes Calixto, Gustavo Machado Teixeira, Marcos Paulo Colella, Celso Arrais Rodrigues, Anderson João Simione, Cinthya Corrêa da Silva, Nelson Hamerschlak","doi":"10.1038/s41409-026-02877-2","DOIUrl":"https://doi.org/10.1038/s41409-026-02877-2","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147762159","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}
Miles Horton, Becky Leung, Ricky Nelles, Matthew Rubic, Christopher McKinnely, Alia Cibich, Mark R Dowling, Stewart Hunt, Lynette C Y Chee, Ashleigh P Scott, David S Ritchie, Devendra Hiwase, Duncan Purtill, Abir Bhattacharya, Glen A Kennedy, Ashish Bajel
{"title":"Pre-transplant azacitidine does not improve survival in allogeneic HSCT for higher-risk MDS.","authors":"Miles Horton, Becky Leung, Ricky Nelles, Matthew Rubic, Christopher McKinnely, Alia Cibich, Mark R Dowling, Stewart Hunt, Lynette C Y Chee, Ashleigh P Scott, David S Ritchie, Devendra Hiwase, Duncan Purtill, Abir Bhattacharya, Glen A Kennedy, Ashish Bajel","doi":"10.1038/s41409-026-02874-5","DOIUrl":"https://doi.org/10.1038/s41409-026-02874-5","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2026-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147762266","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}