Matteo Chiurlo, Laura Galli, Francesca Farina, Andrea Acerbis, Giovanni Mori, Elisabetta Xue, Daniela Clerici, Sara Mastaglio, Simona Piemontese, Maria T Lupo Stanghellini, Marco Ripa, Chiara Tassan Din, Consuelo Corti, Jacopo Peccatori, Paolo Scarpellini, Antonella Castagna, Fabio Ciceri, Raffaella Greco, Chiara Oltolini
{"title":"Risk stratification and impact of donor type on breakthrough invasive fungal infections in haematopoietic cell transplant with post-transplant cyclophosphamide and mould-active prophylaxis.","authors":"Matteo Chiurlo, Laura Galli, Francesca Farina, Andrea Acerbis, Giovanni Mori, Elisabetta Xue, Daniela Clerici, Sara Mastaglio, Simona Piemontese, Maria T Lupo Stanghellini, Marco Ripa, Chiara Tassan Din, Consuelo Corti, Jacopo Peccatori, Paolo Scarpellini, Antonella Castagna, Fabio Ciceri, Raffaella Greco, Chiara Oltolini","doi":"10.1038/s41409-025-02605-2","DOIUrl":"https://doi.org/10.1038/s41409-025-02605-2","url":null,"abstract":"<p><p>Incidence of breakthrough proven-probable invasive fungal infections (b-PP-IFIs) in allogeneic haematopoietic cell transplant recipients (allo-HCT-r) receiving mould-active prophylaxis (MAP) and post-transplant cyclophosphamide (PT-Cy) is largely unknown. Retrospective study on allo-HCT-r, classified at high-risk for IFIs whether ≥1 of the following conditions was met: 1] active disease; 2] cord-blood; 3] previous transplant; 4] acute graft-versus-host-disease (a-GVHD) grade≥3; 5] mismatched-related or unrelated donor with neutropenia before transplant or grade-2 a-GVHD or Cytomegalovirus infection. Objectives were to estimate cumulative incidence function (CIF) of b-PP-IFIs, evaluate infection-related mortality (IRM) and predictive factors of b-PP-IFIs. Overall, 473 allo-HCT-r (n = 286 posaconazole, n = 187 voriconazole) were analysed: 64.7% were at high-risk, 81.6% received PT-Cy. Fifteen b-PP-IFIs occurred: 14/306 in high-risk, 1/167 in non-high-risk group. CIF of b-PP-IFIs in high-risk group was 2.0% (95%CI = 0.8-4.1%) at 30-day and 5.1% (95%CI = 2.9-8.2%) at 1-year post-transplant. The 1-year CIF of IRM was higher in allo-HCT-r with b-PP-IFIs compared to those without [46.7% (95%CI = 19.6-70%) vs. 8.2% (95%CI = 5.3-9.2%), Gray's test: p < 0.001]. In allo-HCT-r receiving PT-Cy, neutropenia before transplant [sHR 7.54 (95%CI = 1.81-31.43)] and chronic myeloproliferative disorders versus AML/MDS [sHR 7.72 (95%CI = 1.68-35.42)] increased risk of b-PP-IFIs, while donor type did not. MAP effectively prevented IFIs. PT-Cy conferred a comparable risk of b-PP-IFIs in matched compared to mismatched-transplants.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973823","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 Benavente, Juan Montoro, Aitana Balaguer-Roselló, Marta Villalba, Pedro Chorão, Pedro Asensi, Pablo Granados, Pilar Lloret, Inés Gómez-Seguí, Pilar Solves, Marta Santiago, Brais Lamas, Ana Bataller, Juan Eirís, David Martinez, Alberto Louro, Paula Rebollar, Aurora Perla, Javier de la Rubia, Miguel Á Sanz, Jaime Sanz
{"title":"Cytokine release syndrome: implications for transplant outcomes in haploidentical and HLA-matched HSCT using PTCy.","authors":"Rafael Benavente, Juan Montoro, Aitana Balaguer-Roselló, Marta Villalba, Pedro Chorão, Pedro Asensi, Pablo Granados, Pilar Lloret, Inés Gómez-Seguí, Pilar Solves, Marta Santiago, Brais Lamas, Ana Bataller, Juan Eirís, David Martinez, Alberto Louro, Paula Rebollar, Aurora Perla, Javier de la Rubia, Miguel Á Sanz, Jaime Sanz","doi":"10.1038/s41409-025-02594-2","DOIUrl":"https://doi.org/10.1038/s41409-025-02594-2","url":null,"abstract":"<p><p>This study assessed 552 allogeneic hematopoietic cell transplantation (HCT) recipients with posttransplant cyclophosphamide (PTCy) to evaluate the incidence, characteristics, risk factors, and impact of early posttransplant cytokine release syndrome (CRS) on outcomes. The cohort included 36% matched sibling donors (MSD), 34% matched unrelated donors (MUD), 27% haploidentical donors, and 4% mismatched unrelated donors (MMUD). CRS was observed in 182 patients, with the highest incidence in haploidentical transplants (80%) compared to MMUD (32%), MUD (23%), and MSD (8%). Most CRS cases were mild, with 93% classified as grade 1 and 6% as grade 2, with only one severe case of grade 3. In haploidentical transplants, CRS was linked to a lower risk of severe chronic graft-versus-host disease (GVHD) and non-relapse mortality (NRM), leading to improved overall survival. In contrast, among HLA-matched recipients (MSD and MUD), there were no significant differences in outcomes between those with or without CRS. However, subgroup analysis revealed that CRS in patients with myeloid malignancies, including acute myeloid leukemia, myelodysplastic syndromes, and myeloproliferative neoplasms, was associated with a reduced relapse rate, improving survival outcomes. In conclusion, while CRS is typically mild and short-lived, it significantly impacts survival, particularly in haploidentical transplants and HLA-matched patients with myeloid malignancies.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143972069","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}
Sara Tarantino, Myriam Labopin, Robert Zeiser, Matthias Stelljes, Thomas Schroeder, Nicolaus Kröger, Wolfgang Bethge, Jakob Passweg, Martin Bornhäuser, Christoph Schmid, Johanna Tischer, Matthias Eder, Eolia Brissot, Jordi Esteve, Arnon Nagler, Mohamad Mohty, Fabio Ciceri
{"title":"Allogeneic stem cell transplantation in de novo core-binding factor acute myeloid leukemia in active disease: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation.","authors":"Sara Tarantino, Myriam Labopin, Robert Zeiser, Matthias Stelljes, Thomas Schroeder, Nicolaus Kröger, Wolfgang Bethge, Jakob Passweg, Martin Bornhäuser, Christoph Schmid, Johanna Tischer, Matthias Eder, Eolia Brissot, Jordi Esteve, Arnon Nagler, Mohamad Mohty, Fabio Ciceri","doi":"10.1038/s41409-025-02596-0","DOIUrl":"https://doi.org/10.1038/s41409-025-02596-0","url":null,"abstract":"<p><p>Core-binding factor acute myeloid leukemia (CBF-AML) generally has a favorable prognosis, with allogeneic hematopoietic stem cell transplantation (allo-SCT) recommended for relapsed/ refractory (R/R) cases achieving second complete remission (CR). However, clinical outcomes remain suboptimal for patients who relapse or fail to achieve CR following induction chemotherapy. Allo-SCT in non-CR is a potential strategy for such patients, though supporting evidence in CBF-AML is limited. To assess outcomes and prognostic factors of allo-SCT in R/R CBF-AML with active disease, we conducted a retrospective analysis of 610 patients with CBF-AML in non-CR undergoing allo-SCT from 2010 to 2021 across 174 centers within the European Society for Blood and Marrow Transplantation. Graft sources included matched sibling (MSD, n = 151), unrelated (UD, n = 368), and haploidentical donors (Haplo, n = 91). Among patients, 124 had inv(16), and 486 had t(8;21). Two-year overall survival (OS) and leukemia-free survival (LFS) were 53.6% and 42.7%, respectively. Haplo-SCT showed inferior OS compared to MSD (HR 1.79, p = 0.003) and UD (HR 1.64, p = 0.004) and reduced chronic graft-versus-host disease. Patients with t(8;21) exhibited higher relapse incidence (HR 2.04, p = 0.002) and poorer survival outcomes than those with inv(16). These findings confirm the therapeutic role of allo-SCT in R/R CBF-AML in non-CR, supporting its favorable risk profile.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143977830","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}
Gülyüz Öztürk, Mehmet Akif Yeşilipek, Arzu Akçay, Vedat Uygun, Gülcihan Özek, Gülsün Karasu, Ebru Yılmaz, Fatma Demir Yenigürbüz, Seda Öztürkmen, Serap Aksoylar, İkbal Ok Bozkaya, Koray Yalçın, Başak Adaklı Aksoy, Ekrem Ünal, Burcu Akıncı, Hayriye Daloğlu, Barbaros Şahin Karagün, Savaş Kansoy, Namık Özbek, Elif İnce, Hacı Ahmet Demir, Müge Gündoğdu, Barış Malbora, Musa Karakükçü, Murat Elli, Arzu Akyay, Adalet Meral Güneş, Sinan Akbayram, Nazan Sarper, Buket Erer Del Castello, Volkan Hazar, Bülent Antmen
{"title":"Effect of genetic mutations on outcomes of stem cell transplantation in children with hemophagocytic lymphohistiocytosis.","authors":"Gülyüz Öztürk, Mehmet Akif Yeşilipek, Arzu Akçay, Vedat Uygun, Gülcihan Özek, Gülsün Karasu, Ebru Yılmaz, Fatma Demir Yenigürbüz, Seda Öztürkmen, Serap Aksoylar, İkbal Ok Bozkaya, Koray Yalçın, Başak Adaklı Aksoy, Ekrem Ünal, Burcu Akıncı, Hayriye Daloğlu, Barbaros Şahin Karagün, Savaş Kansoy, Namık Özbek, Elif İnce, Hacı Ahmet Demir, Müge Gündoğdu, Barış Malbora, Musa Karakükçü, Murat Elli, Arzu Akyay, Adalet Meral Güneş, Sinan Akbayram, Nazan Sarper, Buket Erer Del Castello, Volkan Hazar, Bülent Antmen","doi":"10.1038/s41409-025-02592-4","DOIUrl":"https://doi.org/10.1038/s41409-025-02592-4","url":null,"abstract":"<p><p>Primary hemophagocytic lymphohistiocytosis (p-HLH) can be cured with allogeneic haematopoietic stem cell transplantation (allo-HSCT). It remains unclear whether HSCT outcomes are affected by the presence of different genetic mutations. We used data obtained from children who underwent allo-HSCT for HLH to examine the effects of genetic mutations on HSCT outcomes. Data from 153 paediatric patients in 18 paediatric stem cell centres were retrospectively evaluated. Patients were divided into four groups: 1) with PRF1 mutation (n = 46), 2) with UNC13D mutation (n = 38), 3) with STX11/STXBP2 mutation (n = 25) and 4) with Griscelli syndrome type 2/ Chediak-Higashi syndrome (GS2/CHS) diagnosis (n = 44). Statistical analysis showed no difference between the subgroups in terms of engraftment, VOD, acute GVHD, chronic GVHD, TRM, OS and EFS rates. The most important factor affecting OS and EFS in all genetic subgroups was remission status before HSCT. The 5-year EFS values for children with mutations in PRF1, UNC13D, STX11/STXBP2 and GS2/CHS were 71%, 66.6%, 74% and 66.7, respectively (log-rank >0.05). However, with prospective studies covering more patients, and creating different genetic subgroups by performing more detailed genetic analyses, special approaches for different genetic subgroups can be revealed in the future.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143981463","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}
Marion Larue, Florent Malard, Ahmed S Alaskar, Mahmoud Aljurf, Mutlu Arat, Marie Balsat, Frédéric Baron, Grzegorz Basak, Ali Bazarbachi, Francesca Bonifazi, Eolia Brissot, Fabio Ciceri, Selim Corbacioglu, Fiona Dignan, Anne Huynh, Michelle Kenyon, Jürgen Kuball, Silvy Lachance, Tamas Masszi, Arnon Nagler, Shinichiro Okamoto, Antonio Pagliuca, Annalisa Ruggeri, Tapani Ruutu, Ibrahim Yakoub-Agha, Yishan Ye, Rafael F Duarte, Zinaida Perić, Enric Carreras, Mohamad Mohty
{"title":"Management of liver sinusoidal obstruction syndrome/veno-occlusive disease in adults: a 2025 perspective from an international expert group.","authors":"Marion Larue, Florent Malard, Ahmed S Alaskar, Mahmoud Aljurf, Mutlu Arat, Marie Balsat, Frédéric Baron, Grzegorz Basak, Ali Bazarbachi, Francesca Bonifazi, Eolia Brissot, Fabio Ciceri, Selim Corbacioglu, Fiona Dignan, Anne Huynh, Michelle Kenyon, Jürgen Kuball, Silvy Lachance, Tamas Masszi, Arnon Nagler, Shinichiro Okamoto, Antonio Pagliuca, Annalisa Ruggeri, Tapani Ruutu, Ibrahim Yakoub-Agha, Yishan Ye, Rafael F Duarte, Zinaida Perić, Enric Carreras, Mohamad Mohty","doi":"10.1038/s41409-025-02598-y","DOIUrl":"https://doi.org/10.1038/s41409-025-02598-y","url":null,"abstract":"<p><p>Sinusoidal obstruction syndrome (SOS) formerly known as Veno-occlusive disease (VOD) is a potentially fatal complication that occurs mainly after haematopoietic cell transplantation, especially allogeneic transplantation. The liver is the principal organ affected, though other organs, such as the lungs, may also be involved to a lesser extent. The condition is characterised by obstruction of the hepatic venules, leading to sinusoidal congestion, hepatic ischaemia and, in severe cases, fulminant liver failure. Recent refined diagnostic criteria, published by the European Society for Blood and Marrow Transplantation in 2023, provide a more accurate method of detecting SOS/VOD, allowing earlier intervention and better stratification of patients according to the severity of their disease. This article focuses on liver SOS/VOD and discussing key risk factors, new diagnostic methods and therapeutic strategies, with an emphasis on the early use of defibrotide, which remains the reference treatment for severe SOS/VOD.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143960023","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}
Piyatida Chumnumsiriwath, Pongthep Vittayawacharin, Jorge Ramos-Perez, Deepa Jeyakumar, Benjamin J Lee, Jean Doh, Shawn Griffin, Matthew Nguyen, Ramy M Hanna, Minh-Ha Tran, Piyanuch Kongtim, Stefan O Ciurea
{"title":"Narsoplimab for refractory transplantation-associated thrombotic microangiopathy (TA-TMA) in adult patients receiving allogeneic hematopoietic stem cell transplantation (AHSCT).","authors":"Piyatida Chumnumsiriwath, Pongthep Vittayawacharin, Jorge Ramos-Perez, Deepa Jeyakumar, Benjamin J Lee, Jean Doh, Shawn Griffin, Matthew Nguyen, Ramy M Hanna, Minh-Ha Tran, Piyanuch Kongtim, Stefan O Ciurea","doi":"10.1038/s41409-025-02595-1","DOIUrl":"https://doi.org/10.1038/s41409-025-02595-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143973365","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":"Feasibility and efficacy of partial replacement of post transplantation cyclophosphamide with bendamustine on day +4 for graft versus host disease prophylaxis in patients undergoing allogeneic hematopoietic cell transplantation.","authors":"Velu Nair, Manthan Kathrotiya, Vijaykumar Shirure, Shruti Bhise, Sandip Kheni, Jayani Patel, Neha Motwani, Dhara Shah, Grishma Sukhwal, Chaitrangi Paranjape, Vivek Nair, Anshul Warman, Seema Patrikar, Uday Yanamandra","doi":"10.1038/s41409-025-02581-7","DOIUrl":"https://doi.org/10.1038/s41409-025-02581-7","url":null,"abstract":"<p><p>PT-CY use in T cell-replete haploidentical HCT has significantly improved outcomes. However, hyperhydration with MESNA in CY administration poses a challenge, in patients with cardiac/ renal problems. PT-CY also increases VOD risk with prior exposure to hepatotoxic drugs. Katsanis et al. in a phase Ia trial in patients undergoing HCT for hematological malignancies showed that partially replacing PT-CY with PT-BEN had comparable outcomes to conventional PT-CY. We conducted an ambispective study in 54 patients [haplo (39), MSD(14), and MUD(1)] with nonmalignant hematological disorders and hematological malignancies in pediatric and adult patients undergoing HCT (MAC/RIC) from February 2019 to May 2024. GvHD prophylaxis comprised of PT-CY/BEN (PT-CY 50 mg/kg Day +3; PT-BEN 90 mg/m<sup>2</sup> Day +4) in a prospective arm (n = 21) and PT-CY/CY (50 mg/kg on Days +3, +4; comparator arm) in ambispective (prospective 12; retrospective 21) arm. In both groups, immunosuppression with CNI and MMF was also given. PT-CY/BEN was comparable to PT-CY/CY in terms of safety, efficacy, and GVHD prevention. In the PT-CY/BEN group, there was earlier neutrophil (0.008) and platelet (0.0057) engraftment with significantly lower BK viremia. Incidence of bacterial infection, TRM, EFS, and OS were comparable in both groups.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143977197","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}
Federico Ravaioli, Antonio Colecchia, Jacopo Peccatori, Daria Pagliara, Anna Grassi, Francesco Barbato, Riccardo Masetti, Barbara Sarina, Simona Sica, Simone Cesaro, Chiara Nozzoli, Giovanni Manfredi Assanto, Lucia Prezioso, Stella Santarone, Francesco Saglio, Ester Vanni, Attilio Olivieri, Mario Delia, Edoardo Benedetti, Francesco Zallio, Fabrizio Pane, Cristina Skert, Mariacristina Menconi, Fabio Benedetti, Francesco De Felice, Luigi Colecchia, Tamara Belotti, Luigina Vanessa Alemanni, Margherita Ursi, Giovanni Marasco, Marcello Roberto, Amanda Vestito, Elton Dajti, Matteo Garcovich, Stefania Bramanti, Daniela Taurino, Francesco Quagliarella, Fabio Ciceri, Arcangelo Prete, Andrea Pession, Davide Festi, Francesca Bonifazi
{"title":"Diagnostic accuracy of liver stiffness measurement for the diagnosis of veno-occlusive disease/sinusoidal obstruction syndrome after hematopoietic stem cell transplantation (HSCT), the ELASTOVOD STUDY: an investigator-initiated, prospective, multicentre diagnostic clinical trial.","authors":"Federico Ravaioli, Antonio Colecchia, Jacopo Peccatori, Daria Pagliara, Anna Grassi, Francesco Barbato, Riccardo Masetti, Barbara Sarina, Simona Sica, Simone Cesaro, Chiara Nozzoli, Giovanni Manfredi Assanto, Lucia Prezioso, Stella Santarone, Francesco Saglio, Ester Vanni, Attilio Olivieri, Mario Delia, Edoardo Benedetti, Francesco Zallio, Fabrizio Pane, Cristina Skert, Mariacristina Menconi, Fabio Benedetti, Francesco De Felice, Luigi Colecchia, Tamara Belotti, Luigina Vanessa Alemanni, Margherita Ursi, Giovanni Marasco, Marcello Roberto, Amanda Vestito, Elton Dajti, Matteo Garcovich, Stefania Bramanti, Daniela Taurino, Francesco Quagliarella, Fabio Ciceri, Arcangelo Prete, Andrea Pession, Davide Festi, Francesca Bonifazi","doi":"10.1038/s41409-025-02570-w","DOIUrl":"https://doi.org/10.1038/s41409-025-02570-w","url":null,"abstract":"<p><p>Hepatic Veno-occlusive disease/sinusoidal obstruction syndrome (VOD/SOS) is a severe complication following hematopoietic stem cell transplantation (HSCT), traditionally diagnosed based on clinical criteria. This study aimed to evaluate the diagnostic performance of liver stiffness measurement (LSM) as a non-invasive tool for non invasive diagnosis of VOD/SOS. A multicentre clinical trial was conducted in Italy from April 2018 to December 2021, screening 1089 patients across 25 centers. VOD/SOS diagnosis followed established clinical guidelines, and patients underwent comprehensive clinical, laboratory, and imaging evaluations up to +100 days post-HSCT or until VOD/SOS diagnosis. LSM was measured pre-HSCT and on specific post-transplant days (ClinicalTrials.gov: NCT03426358). The study enrolled 774 adults and 167 children. The +100-day incidence of VOD/SOS HSCT was 5.53 and 5.26 in the overall and allo-HSCT population, higher in children (14.3%) than in adults (3.68%). The 100-day overall survival (OS) probability was 89.5% (overall) and 89.0% (allo-HSCT) while one-yr OS 79% and 78%, respectively, with outcomes varying by VOD/SOS occurrence and severity. LSM significantly differed between VOD/SOS patients and non-affected individuals at all post-HSCT time points, correlating with disease severity. A diagnostic algorithm was proposed, achieving ≥95% sensitivity and specificity, with a 6 kPa rule-out and 25 kPa rule-in cut-off, enhanced by the \"three-time pre-HSCT rule.\" Survivors showed declining LSM over time, while non-survivors did not. Fully recovered patients had lower LSM than non-improvers. LSM also distinguished VOD/SOS from other liver complications within +100 days post-HSCT in both adults and children. In conclusion, LSM is a reliable, non-invasive diagnostic tool for VOD/SOS. LSM contribute to differential diagnosis and to treatment response as well. This study underscores the potential of LSM, combined with multidisciplinary expertise, to guide VOD/SOS diagnosis and management in HSCT patients, improving potentially the clinical outcomes.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143980423","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}
Sebastian Galeano, Carmem Bonfim, Amado Karduss, Gregorio Jaimovich, Andrés Gómez-De León, Gustavo Bettarello, Anderson Simione, Cinthya Correa, Helen Baldomero, Daniel Neumann, Ana Lisa Basquiera, Mariano Berro, Guillermina Remaggi, Ariel Amaru, Fernando Barroso, Adriana Seber, Francisco Barriga, Julia Palma, Bárbara Puga, Matías Sánchez, Juan Manuel Herrera, Calixto Hernández, David Gómez-Almaguer, Félix Gaytán Morales, Guillermo J Ruiz-Argüelles, Ninotzka Mendoza, María Liz Benítez, Alfredo Wong, Carolina Pagés, Marcos Hernández, Dietger Niederwieser, Damiano Rondelli, Cristóbal Frutos
{"title":"Results of the Latin American Bone Marrow Transplantation Society (LABMT) activity survey 2019-2022: the impact of the COVID-19 pandemic and the increase in related haploidentical donors.","authors":"Sebastian Galeano, Carmem Bonfim, Amado Karduss, Gregorio Jaimovich, Andrés Gómez-De León, Gustavo Bettarello, Anderson Simione, Cinthya Correa, Helen Baldomero, Daniel Neumann, Ana Lisa Basquiera, Mariano Berro, Guillermina Remaggi, Ariel Amaru, Fernando Barroso, Adriana Seber, Francisco Barriga, Julia Palma, Bárbara Puga, Matías Sánchez, Juan Manuel Herrera, Calixto Hernández, David Gómez-Almaguer, Félix Gaytán Morales, Guillermo J Ruiz-Argüelles, Ninotzka Mendoza, María Liz Benítez, Alfredo Wong, Carolina Pagés, Marcos Hernández, Dietger Niederwieser, Damiano Rondelli, Cristóbal Frutos","doi":"10.1038/s41409-025-02600-7","DOIUrl":"https://doi.org/10.1038/s41409-025-02600-7","url":null,"abstract":"<p><p>A total of 6767 first hematopoietic cell transplants (HCT), 4121 autologous (61%) and 2646 allogeneic (39%), were reported by 166 teams from 12 Latin American countries that answered the 2022 LABMT/WBMT activity survey. The transplant rate (TR) for Latin America in 2022 was 103 HCT/10 million inhabitants with a wide variation between the different countries. The main indication for allogeneic (allo)-HCT was acute lymphoblastic leukaemia (41%) for the pediatric population and acute myeloid leukemia (32%) for adults. The main indication for autologous (auto)-HCT was neuroblastoma (33%) in children and plasma cell disorders (57%) in adults. In alloHCT, the most used hematopoietic cell source was the bone marrow (54%) in pediatric while peripheral blood stem cells (PBSC) (87%) was in adults. PBSC was the source of choice for autoHCT in both ages. The main trends observed in the period 2019-2022 was a decrease in the number of procedures in 2020 in association with the start of the COVID-19 pandemic, resuming growth in the following years. AlloHCT had a greater growth compared to autoHCT, and it was mainly driven by the utilization of haploidentical related donors, which became the main source from 2020 onwards.</p>","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143953731","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}
Birgitte S Bergland, Mats Remberger, Geir Erland Tjønnfjord, Camilla Dao Vo, Anders E Myhre, Ingerid W Abrahamsen, Tobias Gedde-Dahl, Tor Henrik Anderson Tvedt
{"title":"Should a young matched unrelated donor be preferred over an older matched related donor in allogeneic stem cell transplantation for acute leukemia and myelodysplastic syndrome?","authors":"Birgitte S Bergland, Mats Remberger, Geir Erland Tjønnfjord, Camilla Dao Vo, Anders E Myhre, Ingerid W Abrahamsen, Tobias Gedde-Dahl, Tor Henrik Anderson Tvedt","doi":"10.1038/s41409-025-02579-1","DOIUrl":"https://doi.org/10.1038/s41409-025-02579-1","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":" ","pages":""},"PeriodicalIF":4.5,"publicationDate":"2025-04-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143977978","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}