Pub Date : 2025-12-01DOI: 10.1038/s41409-025-02748-2
A. Bertaina, M. Maffeis, G. Lucchini, J. E. Galimard, A. Dalissier, K. Kleinschmidt, M. Ansari, M. Benakli, O. C. Mirci-Danicar, D. Pagliara, J. H. Dalle, A. Al Ahmari, E. Skorobogatova, C. Renard, J. Styczynski, R. Rihani, R. Formankova, A. Balduzzi, C. Jubert, E. Goussetis, M. Ifversen, A. Lankester, B. Versluijs, M. Aljurf, P. Bader, S. Corbacioglu, K. Kalwak
The interplay between graft-versus-host disease (aGVHD) and the graft-versus-leukemia (GVL) effect in children with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (HSCT) remains complex. This EBMT Pediatric Diseases Working Party (PDWP) retrospective analysis of 2374 pediatric AML patients evaluated the impact of aGVHD on relapse incidence (RI), non-relapse mortality (NRM), overall survival (OS), and leukemia-free survival (LFS). Grade III/IV aGVHD significantly reduced RI (HR: 0.61, p = 0.01) while significantly increasing NRM (HR: 4.51, p < 0.001), offsetting any benefits in OS and LFS. Grade II aGVHD increased NRM (HR: 2.07, p = 0.002) without affecting RI or OS, while grade I aGVHD had no significant impact on these outcomes. Patients with Grade II or higher aGVHD were at greater risk of both chronic (c)GvHD (HR: 1.98 for Grade II; HR: 4.33 for Grade III/IV, p < 0.001) and extensive cGVHD (HR: 2.52 for Grade II; HR: 4.91 for Grade III/IV, p < 0.001). These findings highlight the challenge of mitigating NRM while preserving the GVL effect to optimize disease control and long-term survival in pediatric AML. This study provides critical insights for refining post-transplant strategies in this population.
在接受同种异体造血干细胞移植（HSCT）的急性髓性白血病（AML）患儿中，移植物抗宿主病（aGVHD）和移植物抗白血病（GVL）效应之间的相互作用仍然很复杂。EBMT儿科疾病工作组（PDWP）对2374名儿科AML患者进行了回顾性分析，评估了aGVHD对复发率（RI）、非复发死亡率（NRM）、总生存期（OS）和无白血病生存期（LFS）的影响。III/IV级aGVHD显著降低RI (HR: 0.61, p = 0.01)，显著增加NRM （HR: 4.51, p = 0.01）
{"title":"Interplay between acute graft-versus-host disease and graft-versus-leukemia effect in pediatric acute myeloid leukemia patients undergoing allogeneic hematopoietic stem cell transplantation: implications for relapse incidence and survival – an EBMT PDWP retrospective study","authors":"A. Bertaina, M. Maffeis, G. Lucchini, J. E. Galimard, A. Dalissier, K. Kleinschmidt, M. Ansari, M. Benakli, O. C. Mirci-Danicar, D. Pagliara, J. H. Dalle, A. Al Ahmari, E. Skorobogatova, C. Renard, J. Styczynski, R. Rihani, R. Formankova, A. Balduzzi, C. Jubert, E. Goussetis, M. Ifversen, A. Lankester, B. Versluijs, M. Aljurf, P. Bader, S. Corbacioglu, K. Kalwak","doi":"10.1038/s41409-025-02748-2","DOIUrl":"10.1038/s41409-025-02748-2","url":null,"abstract":"The interplay between graft-versus-host disease (aGVHD) and the graft-versus-leukemia (GVL) effect in children with acute myeloid leukemia (AML) undergoing allogeneic hematopoietic stem cell transplantation (HSCT) remains complex. This EBMT Pediatric Diseases Working Party (PDWP) retrospective analysis of 2374 pediatric AML patients evaluated the impact of aGVHD on relapse incidence (RI), non-relapse mortality (NRM), overall survival (OS), and leukemia-free survival (LFS). Grade III/IV aGVHD significantly reduced RI (HR: 0.61, p = 0.01) while significantly increasing NRM (HR: 4.51, p < 0.001), offsetting any benefits in OS and LFS. Grade II aGVHD increased NRM (HR: 2.07, p = 0.002) without affecting RI or OS, while grade I aGVHD had no significant impact on these outcomes. Patients with Grade II or higher aGVHD were at greater risk of both chronic (c)GvHD (HR: 1.98 for Grade II; HR: 4.33 for Grade III/IV, p < 0.001) and extensive cGVHD (HR: 2.52 for Grade II; HR: 4.91 for Grade III/IV, p < 0.001). These findings highlight the challenge of mitigating NRM while preserving the GVL effect to optimize disease control and long-term survival in pediatric AML. This study provides critical insights for refining post-transplant strategies in this population.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"197-205"},"PeriodicalIF":5.2,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145653436","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}
Pub Date : 2025-11-29DOI: 10.1038/s41409-025-02751-7
Marta Villalba, Allain-Thibeault Ferhat, Tobias Gedde-Dahl, Gerard Socie, Anne Huynh, Igor Wolfgang Blau, Ibrahim Yakoub-Agha, Didier Blaise, Matthias Eder, Edouard Forcade, Renato Fanin, Jakob Passweg, Charles Crawley, Matthias Stelljes, Friedrich Stölzel, Nicolaus Kroeger, Henrik Sengeloev, Patrice Ceballos, Helene Labussiere-Wallet, Xavier Poiré, Jordi Esteve, Bipin Savani, Arnon Nagler, Simona Piemontese, Jaime Sanz, Mohamad Mohty, Fabio Ciceri
We evaluated the influence of donor type in 3006 adults with adverse-risk cytogenetic acute myeloid leukemia (AML) in first complete remission undergoing allogeneic hematopoietic cell transplantation (HCT). Donor types included matched sibling (MSD), matched unrelated (MUD), mismatched unrelated (MMUD), and haploidentical donors. At 2 years, leukemia-free survival, overall survival (OS), and graft-versus-host disease (GVHD)-free/relapse-free survival were 47%, 55%, and 36%. Compared with MSD, OS was inferior with MUD (HR 1.36; 95% CI, 1.1–1.67), MMUD (HR 1.4; 95% CI, 1.07–1.83), and haploidentical grafts (HR 1.33; 95% CI, 1.02–1.73). Haploidentical was associated with lower relapse risk (HR 0.72; 95% CI, 0.53–0.97), but higher non-relapse mortality (NRM) (HR 3.85; 95% CI, 2.44–6.08). All alternative donors showed higher rates of grade II–IV acute GVHD. In 702 patients receiving post-transplant cyclophosphamide (PTCy), survival differences attenuated. However, haploidentical and MMUD showed higher risk of grade III–IV acute GVHD (HR 2.61; 95% CI, 1.04–6.54 and HR 3.74; 95% CI, 1.14–12.24), and haploidentical had increased NRM (HR 3.22; 95% CI, 1.23–8.44), without significant relapse. Findings support safety of alternative donors and reinforce MSD as preferred choice in adverse-risk AML. PTCy mitigates but does not eliminate the risk of donor mismatch.
{"title":"Impact of donor selection in adverse-risk AML undergoing hematopoietic cell transplantation: A study from the EBMT Acute Leukemia Working Party","authors":"Marta Villalba, Allain-Thibeault Ferhat, Tobias Gedde-Dahl, Gerard Socie, Anne Huynh, Igor Wolfgang Blau, Ibrahim Yakoub-Agha, Didier Blaise, Matthias Eder, Edouard Forcade, Renato Fanin, Jakob Passweg, Charles Crawley, Matthias Stelljes, Friedrich Stölzel, Nicolaus Kroeger, Henrik Sengeloev, Patrice Ceballos, Helene Labussiere-Wallet, Xavier Poiré, Jordi Esteve, Bipin Savani, Arnon Nagler, Simona Piemontese, Jaime Sanz, Mohamad Mohty, Fabio Ciceri","doi":"10.1038/s41409-025-02751-7","DOIUrl":"10.1038/s41409-025-02751-7","url":null,"abstract":"We evaluated the influence of donor type in 3006 adults with adverse-risk cytogenetic acute myeloid leukemia (AML) in first complete remission undergoing allogeneic hematopoietic cell transplantation (HCT). Donor types included matched sibling (MSD), matched unrelated (MUD), mismatched unrelated (MMUD), and haploidentical donors. At 2 years, leukemia-free survival, overall survival (OS), and graft-versus-host disease (GVHD)-free/relapse-free survival were 47%, 55%, and 36%. Compared with MSD, OS was inferior with MUD (HR 1.36; 95% CI, 1.1–1.67), MMUD (HR 1.4; 95% CI, 1.07–1.83), and haploidentical grafts (HR 1.33; 95% CI, 1.02–1.73). Haploidentical was associated with lower relapse risk (HR 0.72; 95% CI, 0.53–0.97), but higher non-relapse mortality (NRM) (HR 3.85; 95% CI, 2.44–6.08). All alternative donors showed higher rates of grade II–IV acute GVHD. In 702 patients receiving post-transplant cyclophosphamide (PTCy), survival differences attenuated. However, haploidentical and MMUD showed higher risk of grade III–IV acute GVHD (HR 2.61; 95% CI, 1.04–6.54 and HR 3.74; 95% CI, 1.14–12.24), and haploidentical had increased NRM (HR 3.22; 95% CI, 1.23–8.44), without significant relapse. Findings support safety of alternative donors and reinforce MSD as preferred choice in adverse-risk AML. PTCy mitigates but does not eliminate the risk of donor mismatch.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"187-196"},"PeriodicalIF":5.2,"publicationDate":"2025-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629644","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}
Pub Date : 2025-11-29DOI: 10.1038/s41409-025-02744-6
Carlos de Miguel, Ferran Briansó, Rosalía Alonso, Guiomar Bautista, Luis Espinosa, Carlos Manchado-Perdiguero, María E. Martínez-Muñoz, Lucía Núñez, Isabel Salcedo, Ali Sánchez-Peral, Rafael F. Duarte
The use of TRECs/KRECs in allogeneic HSCT (alloHSCT) has been limited by a lack of standard technical platforms to allow comparison and validation of results between centers. We quantified absolute TRECs/KRECs on sequential samples collected prospectively (pre-transplant, 1, 3, 6 and 12 months post-transplant) in 374 alloHSCT for hematological malignancies using LightCycler 480/TREC-KREC- ACTB (Roche Diagnostics). Following prompt decrease after transplant, KRECs recover as soon as 3 months posttransplant, while TRECs recovery takes up to 1 year (p < 0.001). KRECs do not associate with outcomes. However, higher pre-transplant TRECs strongly associate with reduced non-relapse mortality (NRM) and increased overall survival (OS), and remain independent in multivariate analysis (HR 0.37, p = 0.001, and HR 0.51, p < 0.001, respectively). In addition, faster TRECs recovery measured sequentially at 1, 3, 6 and 12 months after alloHSCT associates with better OS. Furthermore, landmark analyses showed that early survivors with higher TRECs levels at 6 and 12 months after alloHSCT had significantly better subsequent long-term survival, independent from graft-versus-host disease (GVHD) and other clinical factors in multivariate analysis (HR 0.33, p = 0.016 and HR 0.13, p < 0.001, respectively). TRECs levels pre-transplant and at 6 and 12 months post-transplant provide novel biomarker measurable data that associate with alloHSCT long-term outcomes.
{"title":"Sequential quantification of T-cell receptor excision circles (TRECs) and K-deleting recombination excision circles (KRECs) and overall survival after allogeneic HSCT","authors":"Carlos de Miguel, Ferran Briansó, Rosalía Alonso, Guiomar Bautista, Luis Espinosa, Carlos Manchado-Perdiguero, María E. Martínez-Muñoz, Lucía Núñez, Isabel Salcedo, Ali Sánchez-Peral, Rafael F. Duarte","doi":"10.1038/s41409-025-02744-6","DOIUrl":"10.1038/s41409-025-02744-6","url":null,"abstract":"The use of TRECs/KRECs in allogeneic HSCT (alloHSCT) has been limited by a lack of standard technical platforms to allow comparison and validation of results between centers. We quantified absolute TRECs/KRECs on sequential samples collected prospectively (pre-transplant, 1, 3, 6 and 12 months post-transplant) in 374 alloHSCT for hematological malignancies using LightCycler 480/TREC-KREC- ACTB (Roche Diagnostics). Following prompt decrease after transplant, KRECs recover as soon as 3 months posttransplant, while TRECs recovery takes up to 1 year (p < 0.001). KRECs do not associate with outcomes. However, higher pre-transplant TRECs strongly associate with reduced non-relapse mortality (NRM) and increased overall survival (OS), and remain independent in multivariate analysis (HR 0.37, p = 0.001, and HR 0.51, p < 0.001, respectively). In addition, faster TRECs recovery measured sequentially at 1, 3, 6 and 12 months after alloHSCT associates with better OS. Furthermore, landmark analyses showed that early survivors with higher TRECs levels at 6 and 12 months after alloHSCT had significantly better subsequent long-term survival, independent from graft-versus-host disease (GVHD) and other clinical factors in multivariate analysis (HR 0.33, p = 0.016 and HR 0.13, p < 0.001, respectively). TRECs levels pre-transplant and at 6 and 12 months post-transplant provide novel biomarker measurable data that associate with alloHSCT long-term outcomes.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"179-186"},"PeriodicalIF":5.2,"publicationDate":"2025-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02744-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629753","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-11-29DOI: 10.1038/s41409-025-02779-9
Florent Malard, Ernst Holler, Zinaida Peric, Varun Mehra, Raphael Duarte, Jaime Sanz, Alexandros Spyridonidis, Xavier Poiré, Linde Morsink, Johannes Clausen, Ali Bazarbachi, Arnon Nagler, Fabio Ciceri, Annalisa Ruggeri, Mohamad Mohty
{"title":"Microbiotherapy and fecal microbiota transplantation in hematology-oncology: a European clinical perspective to navigate the evolving regulatory framework and the emergence of a new therapeutic class","authors":"Florent Malard, Ernst Holler, Zinaida Peric, Varun Mehra, Raphael Duarte, Jaime Sanz, Alexandros Spyridonidis, Xavier Poiré, Linde Morsink, Johannes Clausen, Ali Bazarbachi, Arnon Nagler, Fabio Ciceri, Annalisa Ruggeri, Mohamad Mohty","doi":"10.1038/s41409-025-02779-9","DOIUrl":"10.1038/s41409-025-02779-9","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"125-127"},"PeriodicalIF":5.2,"publicationDate":"2025-11-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02779-9.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629750","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-11-27DOI: 10.1038/s41409-025-02772-2
P. López-Pereira, S. Romero Domínguez, F. Martin-Moro, A. C. Caballero, A. López García, B. de la Cruz Benito, M. Franch Sarto, F. de la Cruz Vicente, J. López Marín, S. Pinzón Marino, A. Jiménez-Ubieto, A. Cabirta, N. C. Gutiérrez, M. J. Lis Chulvi, A. Puyuelo, A. Muntañola Prat, A. Penedo Coello, E. García-Lerma, S. Mercadal Vilchez
{"title":"Outcomes of de novo primary central nervous system lymphoma using the MATRix regimen for induction followed by thiotepa-based conditioning: a GELTAMO analysis","authors":"P. López-Pereira, S. Romero Domínguez, F. Martin-Moro, A. C. Caballero, A. López García, B. de la Cruz Benito, M. Franch Sarto, F. de la Cruz Vicente, J. López Marín, S. Pinzón Marino, A. Jiménez-Ubieto, A. Cabirta, N. C. Gutiérrez, M. J. Lis Chulvi, A. Puyuelo, A. Muntañola Prat, A. Penedo Coello, E. García-Lerma, S. Mercadal Vilchez","doi":"10.1038/s41409-025-02772-2","DOIUrl":"10.1038/s41409-025-02772-2","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"229-231"},"PeriodicalIF":5.2,"publicationDate":"2025-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629767","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}
Pub Date : 2025-11-27DOI: 10.1038/s41409-025-02730-y
Johanna Richter, Nico Gagelmann, Felix Fischbach, Kristin Rathje, Lena Kristina Pfeffer, Boris Fehse, Anita Badbaran, Susanna Carolina Berger, Rolf Krause, Evgeny Klyuchnikov, Christine Wolschke, Catherina Lueck, Francis Ayuk, Manuel A. Friese, Christoph Heesen, Nicolaus Kröger
Autologous hematopoietic stem cell transplantation is an effective therapeutic option for patients with treatment-refractory multiple sclerosis (MS) and may be considered as first line treatment in aggressive forms. Currently, a variety of conditioning and serotherapy regimens are employed across transplant centers. In this study, we compared immune reconstitution at days 30 and 100 post-transplant in MS patients undergoing AHSCT with cyclophosphamide-based conditioning, combined with in vivo T-cell depletion using either polyclonal rabbit anti-thymocyte globulin (ATG; Thymoglobulin, Genzyme-Sanofi) or rabbit anti-T-lymphocyte globulin (ATLG; Grafalon, Neovii). We observed a significantly faster immune reconstitution for CD3+, CD3+HLA-DR+, CD3+CD4+, CD4+CD45RA+, CD4+CD45RO+, CD3+CD8+, CD8+CD45RA+, CD8+CD45RO+, and CD4+CD25+CD127low cells in patients receiving ATLG compared to ATG at day 30 post-transplant. Although infections resulting in rehospitalization by day 180 were similarly distributed between groups, viral reactivations occurred exclusively in patients receiving ATG. No sign of high grade infectious complications or death was noted.
{"title":"Comparison of anti-human T cell globulins on immune reconstitution and early infections after autologous transplant in patients with multiple sclerosis","authors":"Johanna Richter, Nico Gagelmann, Felix Fischbach, Kristin Rathje, Lena Kristina Pfeffer, Boris Fehse, Anita Badbaran, Susanna Carolina Berger, Rolf Krause, Evgeny Klyuchnikov, Christine Wolschke, Catherina Lueck, Francis Ayuk, Manuel A. Friese, Christoph Heesen, Nicolaus Kröger","doi":"10.1038/s41409-025-02730-y","DOIUrl":"10.1038/s41409-025-02730-y","url":null,"abstract":"Autologous hematopoietic stem cell transplantation is an effective therapeutic option for patients with treatment-refractory multiple sclerosis (MS) and may be considered as first line treatment in aggressive forms. Currently, a variety of conditioning and serotherapy regimens are employed across transplant centers. In this study, we compared immune reconstitution at days 30 and 100 post-transplant in MS patients undergoing AHSCT with cyclophosphamide-based conditioning, combined with in vivo T-cell depletion using either polyclonal rabbit anti-thymocyte globulin (ATG; Thymoglobulin, Genzyme-Sanofi) or rabbit anti-T-lymphocyte globulin (ATLG; Grafalon, Neovii). We observed a significantly faster immune reconstitution for CD3+, CD3+HLA-DR+, CD3+CD4+, CD4+CD45RA+, CD4+CD45RO+, CD3+CD8+, CD8+CD45RA+, CD8+CD45RO+, and CD4+CD25+CD127low cells in patients receiving ATLG compared to ATG at day 30 post-transplant. Although infections resulting in rehospitalization by day 180 were similarly distributed between groups, viral reactivations occurred exclusively in patients receiving ATG. No sign of high grade infectious complications or death was noted.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"172-178"},"PeriodicalIF":5.2,"publicationDate":"2025-11-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02730-y.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Long-term follow-up of belumosudil as second or subsequent line of therapy for steroid-dependent/resistant cGVHD: clinical outcomes from a Japanese study","authors":"Koji Kato, Yoshihiro Inamoto, Toshiro Kawakita, Yasushi Onishi, Ken-ichi Matsuoka, Soichi Shiratori, Kazuhiro Ikegame, Nobuhiro Hiramoto, Masako Toyosaki, Yuta Katayama, Yuhki Koga, Shun Murayama, Yuji Sasagawa, Mami Shindo, Takanori Teshima, Kiyohiko Hatake, Yoshinobu Maeda","doi":"10.1038/s41409-025-02775-z","DOIUrl":"10.1038/s41409-025-02775-z","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"225-228"},"PeriodicalIF":5.2,"publicationDate":"2025-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02775-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2025-11-26DOI: 10.1038/s41409-025-02742-8
Giorgia Battipaglia, Claudia Wehr, Rick Admiraal, Stefan Nierkens, Florent Malard, Robert Zeiser, Francesco Onida, Isabel Sanchez-Ortega, Annalisa Ruggeri, Ibrahim Yakoub-Agha, Mohamad Mohty, Jürgen Kuball
Rabbit anti-thymocyte globulin (ATG) and anti-T-lymphocyte globulin (ATLG) are widely used in allogeneic hematopoietic cell transplantation (allo-HCT) to prevent graft rejection and reduce the risk of graft-versus-host disease. Despite their broad application, clinical practice varies substantially in terms of product selection, dosing, and timing. Distinct differences in manufacturing, pharmacokinetics, and immunological effects between ATG and ATLG complicate efforts to standardize their use across transplant platforms. This review presents the most recent insights into the pharmacology, mechanisms of action, clinical efficacy, and toxicity profiles of both agents. It highlights their differential impact across various allo-HCT settings. Key topics such as re-exposure, immune reconstitution, adverse events, and individualized dosing strategies guided by pharmacokinetic modeling are discussed. These analyses informed consensus recommendations developed through a dedicated expert workshop within the EBMT to support optimized, context-specific use of rabbit ATG and ATLG in allo-HCT for hematologic malignancies.
{"title":"Usage of rabbit anti-thymocyte / anti-T-lymphocyte globulins (ATG / ATLG) for hematological malignancies in allogeneic hematopoietic cell transplantation: Best practice recommendations from the EBMT Practice Harmonisation and Guidelines Committee","authors":"Giorgia Battipaglia, Claudia Wehr, Rick Admiraal, Stefan Nierkens, Florent Malard, Robert Zeiser, Francesco Onida, Isabel Sanchez-Ortega, Annalisa Ruggeri, Ibrahim Yakoub-Agha, Mohamad Mohty, Jürgen Kuball","doi":"10.1038/s41409-025-02742-8","DOIUrl":"10.1038/s41409-025-02742-8","url":null,"abstract":"Rabbit anti-thymocyte globulin (ATG) and anti-T-lymphocyte globulin (ATLG) are widely used in allogeneic hematopoietic cell transplantation (allo-HCT) to prevent graft rejection and reduce the risk of graft-versus-host disease. Despite their broad application, clinical practice varies substantially in terms of product selection, dosing, and timing. Distinct differences in manufacturing, pharmacokinetics, and immunological effects between ATG and ATLG complicate efforts to standardize their use across transplant platforms. This review presents the most recent insights into the pharmacology, mechanisms of action, clinical efficacy, and toxicity profiles of both agents. It highlights their differential impact across various allo-HCT settings. Key topics such as re-exposure, immune reconstitution, adverse events, and individualized dosing strategies guided by pharmacokinetic modeling are discussed. These analyses informed consensus recommendations developed through a dedicated expert workshop within the EBMT to support optimized, context-specific use of rabbit ATG and ATLG in allo-HCT for hematologic malignancies.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"128-141"},"PeriodicalIF":5.2,"publicationDate":"2025-11-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02742-8.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145629779","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Graft failure (GF) is a barrier to successful allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with myelofibrosis (MF). We investigated the incidence, risk factors, and prognosis for GF after allo-HSCT for MF. Two hundred and eleven patients with MF who underwent allo-HSCT across 32 hematology centers in China between December 2008 and December 2024 were retrospectively analyzed. Among them, 66 underwent matched sibling donor HSCT, 127 haploidentical HSCT, and 18 unrelated donor HSCT. The overall GF incidence was 12.5%. GF incidence was significantly associated with donor type (matched sibling, 4.8%; alternative, 13.3%; P = 0.024). Pretransplant massive splenomegaly increased GF incidence (non-massive splenomegaly, 8.5%; massive, 18.5%; P = 0.034). In multivariate analysis, massive splenomegaly (HR = 3.047; P = 0.007) and alternative donors (HR = 3.528; P = 0.041) increased GF risk. With median follow-up of 734 days, 3-year OS, DFS, relapse rate and NRM was 65.5%, 60.8%, 10.1% and 27.8%, respectively. Multivariate analysis showed pretransplant splenomegaly reduced 3-year DFS (HR = 1.671; P = 0.025), and alternative donors reduced 3-year OS (HR = 2.033; P = 0.015). In conclusion, Allo-HSCT provides curative outcomes for MF patients. However, GF remains a significant challenge, particularly in haploidentical HSCT and those with massive pretransplant splenomegaly.
移植失败（GF）是骨髓纤维化（MF）患者成功进行同种异体造血干细胞移植（allo-HSCT）的障碍。我们调查了同种异体造血干细胞移植治疗MF后GF的发病率、危险因素和预后。回顾性分析了2008年12月至2024年12月期间，中国32个血液学中心211例接受同种异体造血干细胞移植的MF患者。其中，66例接受了匹配的兄弟姐妹供体HSCT， 127例接受了单倍相同的HSCT， 18例接受了无血缘关系的供体HSCT。GF的总发病率为12.5%。GF发病率与供体类型显著相关（配对兄弟姐妹，4.8%；替代，13.3%;P = 0.024）。移植前脾肿大增加GF发生率（非脾肿大8.5%；脾肿大18.5%;P = 0.034）。在多变量分析中，脾肿大（HR = 3.047; P = 0.007）和其他供体（HR = 3.528; P = 0.041）增加GF的风险。中位随访734天，3年OS、DFS、复发率和NRM分别为65.5%、60.8%、10.1%和27.8%。多因素分析显示，移植前脾肿大可降低3年DFS (HR = 1.671, P = 0.025)，替代供体可降低3年OS （HR = 2.033, P = 0.015）。总之，同种异体造血干细胞移植为MF患者提供了治疗效果。然而，GF仍然是一个重大的挑战，特别是在单倍体HSCT和移植前脾肿大的患者中。
{"title":"Incidence and risk factors for graft failure after allogeneic hematopoietic stem cell transplantation in patients with myelofibrosis","authors":"Yuqian Sun, Jun Zhu, Jia Chen, Erlie Jiang, Borui Tang, Xinchuan Chen, Yanmin Zhao, Wei Shi, Xi Zhang, Fang Zhou, Mingfeng Zhao, Jinsong Yan, Yehui Tan, Shuangnian Xu, Guanchen Bai, Weijie Cao, Yang Cao, Xinhong Fei, Jian Zhou, Sanbin Wang, Peng Zhao, Kourong Miao, Ying Lu, Li Ding, Zhiling Yan, Liping Dou, Mei Lan, Shunqing Wang, Zhiguo Wang, Hai Yi, Hailong Yuan, Xiaosheng Fang, Hao Zhang, Xiaojun Huang","doi":"10.1038/s41409-025-02746-4","DOIUrl":"10.1038/s41409-025-02746-4","url":null,"abstract":"Graft failure (GF) is a barrier to successful allogeneic hematopoietic stem cell transplantation (allo-HSCT) in patients with myelofibrosis (MF). We investigated the incidence, risk factors, and prognosis for GF after allo-HSCT for MF. Two hundred and eleven patients with MF who underwent allo-HSCT across 32 hematology centers in China between December 2008 and December 2024 were retrospectively analyzed. Among them, 66 underwent matched sibling donor HSCT, 127 haploidentical HSCT, and 18 unrelated donor HSCT. The overall GF incidence was 12.5%. GF incidence was significantly associated with donor type (matched sibling, 4.8%; alternative, 13.3%; P = 0.024). Pretransplant massive splenomegaly increased GF incidence (non-massive splenomegaly, 8.5%; massive, 18.5%; P = 0.034). In multivariate analysis, massive splenomegaly (HR = 3.047; P = 0.007) and alternative donors (HR = 3.528; P = 0.041) increased GF risk. With median follow-up of 734 days, 3-year OS, DFS, relapse rate and NRM was 65.5%, 60.8%, 10.1% and 27.8%, respectively. Multivariate analysis showed pretransplant splenomegaly reduced 3-year DFS (HR = 1.671; P = 0.025), and alternative donors reduced 3-year OS (HR = 2.033; P = 0.015). In conclusion, Allo-HSCT provides curative outcomes for MF patients. However, GF remains a significant challenge, particularly in haploidentical HSCT and those with massive pretransplant splenomegaly.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 2","pages":"1-7"},"PeriodicalIF":5.2,"publicationDate":"2025-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145602202","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}
Pub Date : 2025-11-25DOI: 10.1038/s41409-025-02759-z
Edward A. Copelan, Robert P. Gale
{"title":"Hematopoietic cell transplantation in first relapse of (CBF- and other) AML","authors":"Edward A. Copelan, Robert P. Gale","doi":"10.1038/s41409-025-02759-z","DOIUrl":"10.1038/s41409-025-02759-z","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"61 1","pages":"1-2"},"PeriodicalIF":5.2,"publicationDate":"2025-11-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.comhttps://www.nature.com/articles/s41409-025-02759-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145602184","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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