James Fan Wu, Noel Estrada-Merly, Yuhong Zhou, Bethany Canales, Tina W F Yen, Anita D'Souza
{"title":"Multiple myeloma incidence, transplant utilization, and mortality- impact of social vulnerability.","authors":"James Fan Wu, Noel Estrada-Merly, Yuhong Zhou, Bethany Canales, Tina W F Yen, Anita D'Souza","doi":"10.1038/s41409-025-02733-9","DOIUrl":"https://doi.org/10.1038/s41409-025-02733-9","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145285609","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}
M J M Janssen, M A de Witte, L G M Daenen, F Verduyn Lunell, D van Baarle, J Kuball, A H W Bruns
{"title":"Preserved measles immunity after allogeneic hematopoietic stem cell transplantation in a cohort of mainly αβ T-cell-depleted graft recipients.","authors":"M J M Janssen, M A de Witte, L G M Daenen, F Verduyn Lunell, D van Baarle, J Kuball, A H W Bruns","doi":"10.1038/s41409-025-02731-x","DOIUrl":"https://doi.org/10.1038/s41409-025-02731-x","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238114","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}
Liang-Piu Koh, Yeh Ching Linn, Yang Liang Boo, Victor Ling, Zi Yi Lim, Michelle Poon, Jeffrey Quek, Hein Than, Colin Phipps Diong, Balamurugan Vellayappan, Aloysius Ho, Francesca Lim, William Hwang, Lip Kun Tan, Jean Rachel M Catapia, Joanne Lee, Ian Wu, Kheng Wei Yeoh, Bryan Ho, Wen Shen Looi, Lawrence Ng, Tertius Tansloan Tuy, Yvonne Loh, Teck Guan Soh, Gina Gan, Kee Khiang Heng, Yin Jie Koh, Wing Leung
{"title":"Upfront memory T cell add-back with haploidentical TCRαβ-depleted graft in adults with haematological malignancies: a nationwide, multicentre, single-arm, prospective study.","authors":"Liang-Piu Koh, Yeh Ching Linn, Yang Liang Boo, Victor Ling, Zi Yi Lim, Michelle Poon, Jeffrey Quek, Hein Than, Colin Phipps Diong, Balamurugan Vellayappan, Aloysius Ho, Francesca Lim, William Hwang, Lip Kun Tan, Jean Rachel M Catapia, Joanne Lee, Ian Wu, Kheng Wei Yeoh, Bryan Ho, Wen Shen Looi, Lawrence Ng, Tertius Tansloan Tuy, Yvonne Loh, Teck Guan Soh, Gina Gan, Kee Khiang Heng, Yin Jie Koh, Wing Leung","doi":"10.1038/s41409-025-02728-6","DOIUrl":"https://doi.org/10.1038/s41409-025-02728-6","url":null,"abstract":"<p><p>In haploidentical hematopoietic cell transplantation (Haplo-HCT), in vivo or ex vivo T-cell depletion (TCD) can prevent graft-versus-host disease (GVHD) but increase risk of infection and relapse. We hypothesized that TCRαβ-depleted allograft together with upfront infusion with CD45RA-depleted memory T cells (αβTCD + T<sub>M</sub>DLI) may result in favourable GVHD-free and relapse-free survival (GRFS). Between January 2017 and July 2023, 145 adult patients with various haematological malignancies received αβTCD + T<sub>M</sub>DLI. All except 2 patients had robust engraftment at a median of 12 days for neutrophil and 11 days for platelet. The cumulative incidence (CI) of CMV, EBV, HHV6 or ADV infection was only 43% (n = 63) at day+120. CI of grade II-IV and III-IV acute GVHD at 180 days was 31% and 8% respectively. Chronic GVHD was seen in only 5 patients with a 2-year CI of 4%. CI of non-relapse mortality and relapse at 2 years were 17% and 22% respectively. At a median follow up of 28 months, 3-year overall (OS), event-free (EFS), and GRFS were 67%, 62%, and 59%, respectively. This was significantly improved over a propensity score-matched contemporary cohort (n = 53) who received PTCy as GVHD prophylaxis. This first multi-center study demonstrated the potential benefits of the αβTCD + TMDLI approach for Haplo-HCT.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145238067","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":"Comparison of single-unit umbilical cord blood transplantation and haploidentical transplantation using posttransplant cyclophosphamide during first complete remission of acute myeloid leukemia.","authors":"Masamitsu Yanada, Satoshi Yamasaki, Shohei Mizuno, Junichi Sugita, Takahiro Fujino, Yukiko Misaki, Masatsugu Tanaka, Naoyuki Uchida, Makoto Onizuka, Noriko Doki, Shuichi Ota, Masashi Sawa, Toshiro Kawakita, Yuta Hasegawa, Hirohisa Nakamae, Kazuya Ishiwata, Nobuhiro Hiramoto, Fumihiko Ishimaru, Junya Kanda, Marie Ohbiki, Yoshiko Atsuta, Hideki Nakasone, Takaaki Konuma","doi":"10.1038/s41409-025-02729-5","DOIUrl":"https://doi.org/10.1038/s41409-025-02729-5","url":null,"abstract":"<p><p>Choosing an optimal alternative donor is an important clinical concern in allogeneic hematopoietic cell transplantation (HCT). In Japan, single-unit umbilical cord blood transplantation (UCBT) has been widely used in the last two decades, whereas HCT from HLA-haploidentical related donors (haplo-HCT) has been increasingly used following the advent of posttransplant cyclophosphamide (PTCY) for graft-versus-host disease (GVHD) prophylaxis. This registry-based study aimed to compare outcomes between single-unit UCBT (n = 848) and PTCY-based haplo-HCT (n = 241) performed during first complete remission in patients with acute myeloid leukemia. UCBT was associated with a lower likelihood of engraftment (P < 0.001), a higher risk of grade 2-4 and grade 3-4 acute GVHD (P = 0.003 each), and a lower risk of extensive chronic GVHD (P = 0.048). The UCBT and haplo-HCT groups did not significantly differ in 3-year probabilities of overall survival (68% versus 69%, P = 0.686), GVHD/relapse-free survival (55% versus 54%, P = 0.866), relapse (14% versus 16%, P = 0.463), and non-relapse mortality (21% versus 19%, P = 0.403), respectively, which were confirmed with multivariate analysis. These results indicate that both procedures should be considered viable options for patients lacking a matched donor.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145228454","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}
Gagan Raju, Moneeza Walji, David Nemirovsky, Sean Devlin, Ilan Goldstein, Amandeep Singh, Pamela Susman, Natasia Rodriguez, Miguel-Angel Perales, Doris M Ponce
{"title":"Real-world experience of belumosudil and belumosudil/ruxolitinib combination in steroid-refractory chronic graft-versus-host disease.","authors":"Gagan Raju, Moneeza Walji, David Nemirovsky, Sean Devlin, Ilan Goldstein, Amandeep Singh, Pamela Susman, Natasia Rodriguez, Miguel-Angel Perales, Doris M Ponce","doi":"10.1038/s41409-025-02721-z","DOIUrl":"https://doi.org/10.1038/s41409-025-02721-z","url":null,"abstract":"<p><p>Belumosudil is approved after failure of ≥2 lines of therapy in chronic graft-versus-host disease cGVHD. However, real-world data is limited. We conducted a retrospective analysis of 67 patients with steroid-refractory or dependent (SR/SD) cGVHD. At baseline, most patients had advanced multi-organ cGVHD. The 6- and 12-month overall response rate (ORR) was 61%. However, a subset of patients achieved deeper responses with ongoing therapy at 12 months. The 6-month failure-free survival (FFS) was 75% (95%CI: 65-86) whereas the 12-month FFS was 66% (95%CI: 55-78). A low incidence of drug-related grade ≥3 toxicities was observed. A cohort of patients with immune function analysis showed gradual improvement in immune subsets at 1-year post-treatment. The combined bel-rux cohort (n = 14) showed a 6- and 12-month ORR of 64% and 57%, respectively. Overall, belumosudil was associated with high treatment response and survival outcomes. Notably, deeper responses were observed with ongoing therapy, and it was overall well tolerated. In a cohort of patients, immune cell populations had preserved to improved values throughout treatment. Patients who received bel-rux demonstrated efficacy and safety as well. Overall, our real-world study indicates similar findings to the clinical trial and supports the use of belumosudil in cGVHD.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145228457","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}
Gandhi Damaj, Adèle de Masson, Peter Dreger, Ali Bazarbachi, Philipp Berning, Rafael F Duarte, Christopher P Fox, Emmanuella Guenova, Olivier Hermine, Charalampia Kyriakou, Alina Tanase, Olivier Tournilhac, Wojiech Jurczak, Imke E Karsten, Stéphanie Nguyen Quoc, Yasmina Serroukh, Federico Stella, Gerald Wulf, Anna Sureda, Evangelia Papadavid, Pablo L Ortiz-Romero, Annalisa Ruggeri, Isabel Sánchez-Ortega, Julia J Scarisbrick, Ibrahim Yakoub-Agha, Francesco Onida, Norbert Schmitz
{"title":"Allogeneic hematopoietic cell transplant in cutaneous T-cell lymphomas: recommendations from the EBMT PH&G Committee.","authors":"Gandhi Damaj, Adèle de Masson, Peter Dreger, Ali Bazarbachi, Philipp Berning, Rafael F Duarte, Christopher P Fox, Emmanuella Guenova, Olivier Hermine, Charalampia Kyriakou, Alina Tanase, Olivier Tournilhac, Wojiech Jurczak, Imke E Karsten, Stéphanie Nguyen Quoc, Yasmina Serroukh, Federico Stella, Gerald Wulf, Anna Sureda, Evangelia Papadavid, Pablo L Ortiz-Romero, Annalisa Ruggeri, Isabel Sánchez-Ortega, Julia J Scarisbrick, Ibrahim Yakoub-Agha, Francesco Onida, Norbert Schmitz","doi":"10.1038/s41409-025-02696-x","DOIUrl":"https://doi.org/10.1038/s41409-025-02696-x","url":null,"abstract":"<p><p>This manuscript provides expert recommendations on the role of allogeneic hematopoietic cell transplantation (allo-HCT) for cutaneous T-cell lymphoma (CTCL), specifically Mycosis Fungoides (MF) and Sezary Syndrome (SS). Critical aspects such as patient selection, timing, and bridging therapy are addressed, as well as donor source, conditioning regimens and post-transplant management. These consensus guidelines are based on a thorough literature review and discussions among leading dermatologists and hematologists. These recommendations aim to harmonise clinical practice towards improving patient outcomes in these rare but aggressive lymphomas. It is of critical importance to consider allo-HCT early in the management of eligible patients with high-risk disease. Advanced stage, large-cell transformation, relapsed or refractory disease following systemic treatment, and N3-stage lymph node involvement are indicators that should trigger consultation with a transplant hematologist in parallel with a donor search. Early interaction between dermatologists and transplant hematologists is vital to avoiding delays, which can significantly impact post-transplant outcomes and survival. This EBMT Practice Harmonisation & Guidelines Committee consensus provides practical recommendations for the selection, timing, and conduct of allogeneic transplantation in advanced-stage mycosis fungoides and Sézary syndrome, aiming to optimize outcomes through early multidisciplinary collaboration and evidence-based decision making.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145205529","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}
Radwan Massoud, Evgeny Klyuchnikov, Normann Steiner, Maroly Bohorquez Manjarres, Gaby Zeck, Rolf Krause, Silke Heidenreich, Claudia Langebrake, Adrin Dadkhah, Ina Rudolph, Rusudan Sabauri, Christian Niederwieser, Tetiana Perekhrestenko, Mirjam Reichard, Mathias Schäfersküpper, Franziska E Marquard, Sofia Oechsler, Gunnar Weise, Kristin Rathje, Maraike Harfmann, Nico Gagelmann, Catherina Lück, Christine Wolschke, Francis Ayuk, Nicolaus Kröger
{"title":"Sequential versus standard conditioning in untreated MDS patients with blasts undergoing allogeneic HSCT.","authors":"Radwan Massoud, Evgeny Klyuchnikov, Normann Steiner, Maroly Bohorquez Manjarres, Gaby Zeck, Rolf Krause, Silke Heidenreich, Claudia Langebrake, Adrin Dadkhah, Ina Rudolph, Rusudan Sabauri, Christian Niederwieser, Tetiana Perekhrestenko, Mirjam Reichard, Mathias Schäfersküpper, Franziska E Marquard, Sofia Oechsler, Gunnar Weise, Kristin Rathje, Maraike Harfmann, Nico Gagelmann, Catherina Lück, Christine Wolschke, Francis Ayuk, Nicolaus Kröger","doi":"10.1038/s41409-025-02711-1","DOIUrl":"https://doi.org/10.1038/s41409-025-02711-1","url":null,"abstract":"<p><p>Myelodysplastic syndromes (MDS) can progress to AML and often require allogeneic hematopoietic stem cell transplantation (allo-SCT). The sequential FLAMSA-FB regimen, featuring a cytoreductive FLAMSA phase followed by fludarabine-busulfan (FB) conditioning, may enhance disease control. We retrospectively analyzed 106 untreated MDS patients with blasts 5-19% at the University Medical Center Hamburg who received either FLAMSA-FB (n = 45) or standard conditioning (n = 61: Thiotepa-Busulfan (n = 30), Fludarabine-Busulfan (n = 16), Treosulfan-Fludarabine (n = 15)). Median follow-up was 24 months. The FLAMSA group was younger (median age 56 vs. 62, p = 0.02), but baseline IPSS risk scores (p = 0.16) and donor types (p = 0.43) were comparable. Engraftment rates were similar. At two years, overall survival (OS) was 62% with FLAMSA and 68% with standard conditioning (p = 0.92), while progression-free survival (PFS) was 56% vs. 59% (p = 0.92). Non-relapse mortality (22% vs. 25%, p = 0.78) and cumulative incidence of relapse (22% vs. 13%, p = 0.12) did not differ significantly, nor did grade II-IV acute graft-versus-host disease (GVHD). Propensity score matching in 18 pairs confirmed no significant differences in OS, PFS, NRM, or CIR. However, moderate-to-severe chronic GVHD was higher with FLAMSA-FB (50% vs. 17%, p = 0.04). Thus, FLAMSA-FB did not improve transplant outcomes over standard conditioning but was linked to an increased risk of chronic GVHD.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145205542","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}
Anna Torrent, Mireia Morgades, Gloria Iacoboni, Juan Luis Reguera, Mi Kwon, Lucia López Corral, Rafael Hernani, Valentín Ortiz-Maldonado, Jaime Sanz, Ana Carolina Caballero Gonzalez, Luisa Guerra, José María Sánchez Pina, Alberto Mussetti, Alejandro Martin Garcia-Sancho, Mariana Bastos-Oreiro, Javier Delgado, Cecilia Carpio, Juan-Manuel Sancho, Antonio Pérez-Martinez, Pere Barba
{"title":"Patient selection and treatment outcomes over time in patients with large B-cell lymphoma receiving car T-cell therapy.","authors":"Anna Torrent, Mireia Morgades, Gloria Iacoboni, Juan Luis Reguera, Mi Kwon, Lucia López Corral, Rafael Hernani, Valentín Ortiz-Maldonado, Jaime Sanz, Ana Carolina Caballero Gonzalez, Luisa Guerra, José María Sánchez Pina, Alberto Mussetti, Alejandro Martin Garcia-Sancho, Mariana Bastos-Oreiro, Javier Delgado, Cecilia Carpio, Juan-Manuel Sancho, Antonio Pérez-Martinez, Pere Barba","doi":"10.1038/s41409-025-02722-y","DOIUrl":"https://doi.org/10.1038/s41409-025-02722-y","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-09-30","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145198159","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}
Raffaella Greco, Riccardo Saccardi, Marta Ponzano, Manuela Badoglio, Grzegorz Helbig, Marek Smilowski, Alice Mariottini, Joachim Burman, Kristina Carlson, Majid Kazmi, Paolo A Muraro, Ian Gabriel, Barbara Withers, Jennifer Massey, Riccardo Varaldo, Matilde Inglese, Jaime Sanz, Sara Gil-Perotin, Basil Sharrack, Elisa Roldan, Chiara Nozzoli, Alessio Signori, Maria Pia Sormani, Tobias Alexander, John A Snowden
{"title":"BEAM/ATG or cyclophosphamide/ATG as conditioning regimen in autologous haemopoietic stem cell transplantation for multiple sclerosis: a retrospective analysis of the EBMT autoimmune diseases working party.","authors":"Raffaella Greco, Riccardo Saccardi, Marta Ponzano, Manuela Badoglio, Grzegorz Helbig, Marek Smilowski, Alice Mariottini, Joachim Burman, Kristina Carlson, Majid Kazmi, Paolo A Muraro, Ian Gabriel, Barbara Withers, Jennifer Massey, Riccardo Varaldo, Matilde Inglese, Jaime Sanz, Sara Gil-Perotin, Basil Sharrack, Elisa Roldan, Chiara Nozzoli, Alessio Signori, Maria Pia Sormani, Tobias Alexander, John A Snowden","doi":"10.1038/s41409-025-02715-x","DOIUrl":"https://doi.org/10.1038/s41409-025-02715-x","url":null,"abstract":"<p><p>Multiple Sclerosis (MS) is the most frequent autoimmune disease (AD) treated with autologous hematopoietic stem cell transplantation (HSCT). There are no prospective trials comparing the most used conditioning regimens, BEAM/Anti-thymocyte globulin (ATG) and cyclophosphamide (CYC)/ATG. Herein, we report a retrospective analysis of the EBMT database aimed to assess their risk/benefit ratio. We included 1114 MS patients who were conditioned with either BEAM/ATG or CYC/ATG. Neutrophil engraftment in BEAM/ATG- and CYC/ATG-treated patients occurred at a median time of 11 and 10 days after HSCT (p = 0.079). Overall, 2.0% of patients conditioned with BEAM/ATG and 1.0% with CYC/ATG died within 100 days from HSCT (p = 0.19). Overall, the incidence of NEDA (No-Evidence of Disease Activity) failure in BEAM/ATG and CYC/ATG at 5 years was 42.3% (95% CI: 36.3-48.8%) and 44.1% (95% CI: 30.8-60.1%), respectively (p = 0.081). No statistically significant differences between the two treatments [HR = 0.90 (95% CI = 0.61; 1.34), p = 0.60] were confirmed, when adjusting for disease type, disability at baseline and year of transplant. A clear difference between the regimens is lacking, both in terms of toxicity and efficacy, in this large population. Type of disease (relapsing/remitting vs progressive) is still the major determinant of neurological outcome.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145190946","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}
Benedetta Elena Di Majo, Mouad Abouqateb, Jacques-Emmanuel Galimard, Arnaud Dalissier, Giovanna Lucchini, Birgitta Versluys, Ludmila Zubarovskaya, Oana Mirci-Danicar, Charlotte Jubert, Sanjay Tewari, Robert Wynn, Sarah Lawson, Gerard Michel, Adriana Balduzzi, Rose-Marie Hamladji, Anne Sirvent, Ali Al-Ahmari, Fanny Rialland, Maura Faraci, Marc Ansari, Jean-Hugues Dalle, Charlotte Calvo, Vanderson Rocha, Eliane Gluckman, Katharina Kleinschmidt, Selim Corbacioglu, Krzysztof Kalwak, Annalisa Ruggeri
{"title":"Single unrelated umbilical cord blood versus unmanipulated haploidentical HCT using PTCy in pediatric AML: a retrospective study on behalf of the EBMT PDWP and CTIWP.","authors":"Benedetta Elena Di Majo, Mouad Abouqateb, Jacques-Emmanuel Galimard, Arnaud Dalissier, Giovanna Lucchini, Birgitta Versluys, Ludmila Zubarovskaya, Oana Mirci-Danicar, Charlotte Jubert, Sanjay Tewari, Robert Wynn, Sarah Lawson, Gerard Michel, Adriana Balduzzi, Rose-Marie Hamladji, Anne Sirvent, Ali Al-Ahmari, Fanny Rialland, Maura Faraci, Marc Ansari, Jean-Hugues Dalle, Charlotte Calvo, Vanderson Rocha, Eliane Gluckman, Katharina Kleinschmidt, Selim Corbacioglu, Krzysztof Kalwak, Annalisa Ruggeri","doi":"10.1038/s41409-025-02720-0","DOIUrl":"https://doi.org/10.1038/s41409-025-02720-0","url":null,"abstract":"<p><p>Hematopoietic stem cell transplantation (HCT) is the standard consolidation therapy for children with high-risk or relapsed acute myeloid leukemia (AML). In the absence of an human leukocyte antigen (HLA)-matched donor, both unrelated cord blood transplantation (UCBT) and haploidentical HCT with post-transplant cyclophosphamide (haplo-PTCy) serve as viable alternatives. This study analyzed outcomes of 254 pediatric AML patients (CR1/CR2) who underwent either single-unit UCBT (N = 127) without serotherapy or haplo-PTCy (N = 127) between 2011 and 2021. Propensity score weighting was applied to minimize baseline differences. With a median follow-up of 2.9 years, no significant differences were observed in overall survival, leukemia-free survival, relapse incidence, non-relapse mortality (NRM), or graft-versus-host disease (GVHD)-free relapse-free survival. However, UCBT recipients had a lower risk of chronic GVHD (7.1% vs. 19.8%; P = 0.019). These findings confirm comparable survival outcomes between UCBT and Haplo-PTCy. Given the higher risk of cGVHD in haplo-PTCy, further research is needed to refine GVHD prevention strategies while maintaining relapse-free survival. Both approaches remain safe and readily available alternatives for pediatric AML patients lacking matched donors.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":5.2,"publicationDate":"2025-09-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145190932","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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