Eduard Schulz, Filip Pirsl, Kathy McGraw, Darryl Nousome, Yi Liu, Sencer Goklemez, Alain Mina, Noa G. Holtzman, Najla El Jurdi, Steven Z. Pavletic
{"title":"Prevalence of clonal hematopoiesis of indeterminate potential in patients with severe chronic graft-versus-host disease","authors":"Eduard Schulz, Filip Pirsl, Kathy McGraw, Darryl Nousome, Yi Liu, Sencer Goklemez, Alain Mina, Noa G. Holtzman, Najla El Jurdi, Steven Z. Pavletic","doi":"10.1038/s41409-025-02537-x","DOIUrl":"10.1038/s41409-025-02537-x","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"725-728"},"PeriodicalIF":4.5,"publicationDate":"2025-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143584203","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}
Mohamed A. Kharfan-Dabaja, Razan Mohty, Neela Easwar, Patrick Johnston, Madiha Iqbal, Narendranath Epperla, Jean Yared, Nausheen Ahmed, Mehdi Hamadani, Amer Beitinjaneh, Mayur Narkhede, Deepa Jagadeesh, Praveen Ramakrishnan Geethakumari, Bhagirathbhai Dholaria, Usama Gergis, Javier Munoz, Jose Sandoval-Sus, Frederick L. Locke, Joshua Fein, Arushi Khurana, Ernesto Ayala, Kaitlin Annunzio, Aaron P. Rapoport, Forat Lutfi, Othman Salim Akhtar, Lazaros Lekakis, Amitkumar Mehta, Olalekan O. Oluwole, Jennifer Logue, Michael D. Jain, Tsiporah Shore, Urshila Durani, Muhamad Alhaj Moustafa, Joseph McGuirk, Yi Lin, Samuel Yamshon, Julio C. Chavez
{"title":"Chimeric antigen receptor T cell therapy in octogenarians with B cell lymphoma: a real-world US multicenter collaborative study","authors":"Mohamed A. Kharfan-Dabaja, Razan Mohty, Neela Easwar, Patrick Johnston, Madiha Iqbal, Narendranath Epperla, Jean Yared, Nausheen Ahmed, Mehdi Hamadani, Amer Beitinjaneh, Mayur Narkhede, Deepa Jagadeesh, Praveen Ramakrishnan Geethakumari, Bhagirathbhai Dholaria, Usama Gergis, Javier Munoz, Jose Sandoval-Sus, Frederick L. Locke, Joshua Fein, Arushi Khurana, Ernesto Ayala, Kaitlin Annunzio, Aaron P. Rapoport, Forat Lutfi, Othman Salim Akhtar, Lazaros Lekakis, Amitkumar Mehta, Olalekan O. Oluwole, Jennifer Logue, Michael D. Jain, Tsiporah Shore, Urshila Durani, Muhamad Alhaj Moustafa, Joseph McGuirk, Yi Lin, Samuel Yamshon, Julio C. Chavez","doi":"10.1038/s41409-025-02541-1","DOIUrl":"10.1038/s41409-025-02541-1","url":null,"abstract":"Older patients with lymphoma are typically underrepresented in clinical trials with chimeric antigen receptor T cell (CAR T) therapy. In this multicenter, observational study we aimed to assess the safety and efficacy of standard CD19 CAR T in patients 80 years of age or older. At total of 88 patients, median age 82 (range, 80-89) years, were included. Diffuse large B cell lymphoma (DLBCL) (N = 60, 68.2%) represented the most common histology. Patients were treated mostly with axicabtagene ciloleucel (N = 41, 46.6%) followed by lisocabtagene maraleucel (N = 25, 28.4%). Cytokine release syndrome (CRS) (any grade) was seen in 68 (77.3%) and 51 (58%) developed immune effector cell-associated neurotoxicity syndrome (ICANS). Incidence of grade 3-4 CRS and ICANS were 7.4% and 31.4%, respectively. For patients with DLBCL/tFL, the 1-year NRM, relapse, PFS, and OS were 11.6%, 40.8%, 47.6%, and 61.2%, respectively. We conclude that CAR T is feasible and effective in patients 80 years or older with B cell lymphomas. These patients must be provided the opportunity to be evaluated for this curative approach.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"632-639"},"PeriodicalIF":4.5,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-025-02541-1.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143536505","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}
Gabrielle Meyers, Mehdi Hamadani, Michael Martens, Haris Ali, Patrice Chevallier, Hannah Choe, Andrew C. Harris, Ernst Holler, Eric van Hooren, Willem Klaassen, Eric Leifer, Ypke van Oosterhout, Lia Perez, Iskra Pusic, Matthias Stelljes, Walter van der Velden, Emanuele Ammatuna, David Beauvais, Jérôme Cornillon, Richard T. Maziarz, Johannes Schetelig, Jennifer Romeril, Margaret L. MacMillan, John E. Levine, Gérard Socié
{"title":"Correction: Anti-CD3/CD7 immunoconjugate (T-Guard) for severe, steroid-refractory GVHD: final report of BMT CTN 2002","authors":"Gabrielle Meyers, Mehdi Hamadani, Michael Martens, Haris Ali, Patrice Chevallier, Hannah Choe, Andrew C. Harris, Ernst Holler, Eric van Hooren, Willem Klaassen, Eric Leifer, Ypke van Oosterhout, Lia Perez, Iskra Pusic, Matthias Stelljes, Walter van der Velden, Emanuele Ammatuna, David Beauvais, Jérôme Cornillon, Richard T. Maziarz, Johannes Schetelig, Jennifer Romeril, Margaret L. MacMillan, John E. Levine, Gérard Socié","doi":"10.1038/s41409-025-02534-0","DOIUrl":"10.1038/s41409-025-02534-0","url":null,"abstract":"","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"746-746"},"PeriodicalIF":4.5,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-025-02534-0.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143522598","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}
Paul B. Koller, Tamer Othman, Dongyun Yang, Sally Mokhtari, Yazeed Samara, Amanda Blackmon, Vaibhav Agrawal, Hoda Pourhassan, Brian J. Ball, Idoroenyi Amanam, Shukaib Arslan, Salman Otoukesh, Karamjeet S. Sandhu, Ibrahim Aldoss, Haris Ali, Amandeep Salhotra, Ahmed Aribi, Andrew Artz, Pamela S. Becker, Vinod Pullarkat, Forest Marc Stewart, Eileen P. Smith, Anthony Stein, Guido Marcucci, Stephen J. Forman, Ryotaro Nakamura, Monzr M. Al Malki
{"title":"Melphalan-based conditioning with post-transplant cyclophosphamide for peripheral blood stem cell transplantation: donor effect","authors":"Paul B. Koller, Tamer Othman, Dongyun Yang, Sally Mokhtari, Yazeed Samara, Amanda Blackmon, Vaibhav Agrawal, Hoda Pourhassan, Brian J. Ball, Idoroenyi Amanam, Shukaib Arslan, Salman Otoukesh, Karamjeet S. Sandhu, Ibrahim Aldoss, Haris Ali, Amandeep Salhotra, Ahmed Aribi, Andrew Artz, Pamela S. Becker, Vinod Pullarkat, Forest Marc Stewart, Eileen P. Smith, Anthony Stein, Guido Marcucci, Stephen J. Forman, Ryotaro Nakamura, Monzr M. Al Malki","doi":"10.1038/s41409-025-02523-3","DOIUrl":"10.1038/s41409-025-02523-3","url":null,"abstract":"Fludarabine and melphalan (FM) conditioning offers effective disease control with an acceptable toxicity profile. Post-transplant cyclophosphamide (PTCy) for graft-versus-host disease (GVHD) prophylaxis has improved transplant outcomes. We retrospectively reviewed patients receiving FM-based transplants with PTCy at City of Hope. Of 248 patients included, 89 (35.9%) received hematopoietic cell transplant (HCT) from a matched related/unrelated donor (MRD/MUD), 118 (47.6%) from a haploidentical (HID) donor, and 49 (19.8%) from a mismatched unrelated donor (MMUD). There were no differences in acute and chronic GVHD based on donor type. The 2-year overall survival (OS) for patients receiving HID, MMUD, and MRD/MUD was 58%, 55%, and 70%; disease-free survival (DFS) was 52%, 48%, and 66%; and graft-versus-host/relapse-free survival (GRFS) were 48%, 40%, and 59%, respectively. OS, DFS, and GRFS were similar regardless of donor type on multivariable analysis. However, donor age ≥35 years was associated with lower OS and GRFS and higher 2-year non-relapse mortality (NRM) on multivariable analysis across all patients, regardless of donor type. FM with PTCy appears to produce similar outcomes between MRD/MUD, MMUD, and HID when adjusting for donors <35 years, and donor age seems to be the most important factor when selecting a donor with this regimen.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"625-631"},"PeriodicalIF":4.5,"publicationDate":"2025-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-025-02523-3.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143522600","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}
Sina Alexandra Beer, Johanna Blättel, Kristina Reuß, Claus-Philipp Maier, Christoph Faul, Wichard Vogel, Wolfgang Bethge, Claudia Lengerke
{"title":"Long-term patient-reported outcomes following allogeneic hematopoietic cell transplantation","authors":"Sina Alexandra Beer, Johanna Blättel, Kristina Reuß, Claus-Philipp Maier, Christoph Faul, Wichard Vogel, Wolfgang Bethge, Claudia Lengerke","doi":"10.1038/s41409-025-02540-2","DOIUrl":"10.1038/s41409-025-02540-2","url":null,"abstract":"Therapeutic progress has improved the overall survival of patients treated with allogeneic hematopoietic cell transplantation (alloHCT). Thus, the impact on quality of life (QoL) becomes increasingly relevant. However, QoL is not monitored regularly in clinical practice, and most trials stop QoL assessments early post-alloHCT, missing long-term dynamics. To address this knowledge gap, we conducted a cross-sectional survey of 214 adult alloHCT recipients (average age 53 y, 42.5% female, median follow-up 56 months) to evaluate QoL using patient-reported outcome measurements (PROMs), spanning a period from 30 days to over 10 years post-transplant. Participants completed the EORTC QLQ-C30 and FACT-BMT at a single follow-up timepoint to investigate QoL-related factors. Comparing long-term follow-up patients (beyond year 3, n = 125) with short-term follow-up patients (day 30 to month 12, n = 89) shows significantly better long-term QoL outcomes (P = 0.016). However, PROM symptom scales indicate moderate fatigue and insomnia rates in long-term survivors. Better QoL was associated with male gender, lower ECOG, RIC conditioning, no relapse, no ongoing immunosuppression and full-time work. Summarized, while we observe encouraging long-term outcomes, our data suggest that QoL recovery remain highly individual. We strongly recommend the use of PROMs to enhance our understanding of long-term survivorship post-alloHCT.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"617-624"},"PeriodicalIF":4.5,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-025-02540-2.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143514517","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}
Angela Vidal, Cristina Bora, Andrea Jarisch, Janna Pape, Susanna Weidlinger, Tanya Karrer, Michael von Wolff
{"title":"Impact of haematopoietic stem cell transplantation for benign and malignant haematologic and non-haematologic disorders on fertility: a systematic review and meta-analysis","authors":"Angela Vidal, Cristina Bora, Andrea Jarisch, Janna Pape, Susanna Weidlinger, Tanya Karrer, Michael von Wolff","doi":"10.1038/s41409-025-02520-6","DOIUrl":"10.1038/s41409-025-02520-6","url":null,"abstract":"Haematopoietic stem cell transplantation (HSCT) exposes patients to long-term complications like gonadal dysfunction and infertility. The European Society for Blood and Marrow Transplantation advised in 2015 that fertility preservation should be considered for children and adolescents requiring HSCT. This systematic review and meta-analysis is part of the FertiTOX project, which aims to close the data gap regarding the gonadotoxicity of anticancer therapies to provide more accurate advice regarding fertility preservation. This review were conducted in November 2023, covering articles since 2000. In total, 56 studies were included in the meta-analysis, comprising 1853 female malignant, 241 female benign, 1871 male malignant, and 226 male benign cases. The analysis, using a random-effects model, estimated the prevalence and its 95% confidence interval, revealing that overall infertility exceeded 30% in all groups. Female malignant cases had a prevalence of 65% (95% CI: 0.58–0.71), while in females with benign disease, it was 61% (CI: 0.48–0.73). Males with malignant disease had a prevalence of 41% (CI: 0.32–0.51), and those with benign disease had 31% (CI: 0.19–0.46). The > 30% overall prevalence indicates a clinical need for fertility preservation counseling in both genders undergoing HSCT. Further prospective studies are necessary to address HSCT’s individual impact on gonadal function. This systematic review is registered with the International Prospective Register of Systematic Reviews (PROSPERO) under CRD42023486928.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"645-672"},"PeriodicalIF":4.5,"publicationDate":"2025-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-025-02520-6.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143613331","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}
Joanna Drozd-Sokołowska, Gloria Tridello, Inge Verheggen, Musa Karakukcu, Nour Ben Abdeljelil, Anca Colita, Mahmoud Aljurf, Nicolaus Kröger, Gulyuz Ozturk, Jakob Passweg, Massimiliano Gambella, Marina Popova, Lucía López Corral, Alina Tanase, Agnieszka Piekarska, Mohsen Al Zahrani, Muhlis Cem Ar, Grzegorz Basak, Annoek E. C. Broers, Kristina Carlson, Andrew Clark, Maura Faraci, Pavel Jindra, Gergely Kriván, Sophie Ducastelle Lepretre, Stephan Mielke, Judith Niederland, Fabrizio Pane, Katharine Patrick, John A. Snowden, Irfan Yavasoglu, Marco Zecca, Anna Waszczuk-Gajda, Lotus Wendel, Nina Knelange, Rafael de la Camara, Lidia Gil, Malgorzata Mikulska, Dina Averbuch, Jan Styczynski
{"title":"Tuberculosis after hematopoietic cell transplantation: retrospective study on behalf of the Infectious Diseases Working Party of the EBMT","authors":"Joanna Drozd-Sokołowska, Gloria Tridello, Inge Verheggen, Musa Karakukcu, Nour Ben Abdeljelil, Anca Colita, Mahmoud Aljurf, Nicolaus Kröger, Gulyuz Ozturk, Jakob Passweg, Massimiliano Gambella, Marina Popova, Lucía López Corral, Alina Tanase, Agnieszka Piekarska, Mohsen Al Zahrani, Muhlis Cem Ar, Grzegorz Basak, Annoek E. C. Broers, Kristina Carlson, Andrew Clark, Maura Faraci, Pavel Jindra, Gergely Kriván, Sophie Ducastelle Lepretre, Stephan Mielke, Judith Niederland, Fabrizio Pane, Katharine Patrick, John A. Snowden, Irfan Yavasoglu, Marco Zecca, Anna Waszczuk-Gajda, Lotus Wendel, Nina Knelange, Rafael de la Camara, Lidia Gil, Malgorzata Mikulska, Dina Averbuch, Jan Styczynski","doi":"10.1038/s41409-025-02530-4","DOIUrl":"10.1038/s41409-025-02530-4","url":null,"abstract":"Tuberculosis (TB) is rare following hematopoietic cell transplantation (HCT). In this multinational retrospective study, we report the frequency, characteristics, and outcome of TB following HCT performed during 2000–2019. Fifty-two patients (35 (67%) males, 15 (29%) children) from 24 centers developed TB following allogeneic (n = 47) or autologous (n = 5) HCT; with the relative frequency of 0.21% and 0.025%, respectively. Forty (77%) were bacteriologically, 12 (23%) clinically confirmed. The median time from HCT to TB was 135 (range, 16–3225) days. Eighteen (35%) patients with extrapulmonary TB (mainly involving lymph nodes and liver/spleen) were significantly younger, developed TB shorter after HCT, more often had inherited underlying disease, and received immunosuppressive therapy at TB diagnosis as compared to pulmonary TB. Five (22%) of 23 patients with drug-susceptibility testing performed, were resistant to at least one anti-TB drug. Treatment success was achieved in 38/50 (76%) of treated patients. One-year overall survival reached 75.7% and the 1-year cumulative incidence of TB-associated death was 18.1%. Concluding, TB is a rare, albeit severe complication, which can develop any time after HCT, frequently involves extrapulmonary sites, and results in high mortality rates. High proportion of drug-resistant TB warrants routine susceptibility testing.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"603-616"},"PeriodicalIF":4.5,"publicationDate":"2025-02-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490516","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}
Eli Muchtar, Angela Dispenzieri, Vaishali Sanchorawala, Hamza Hassan, Raphael Mwangi, Matthew Maurer, Francis Buadi, Hans C. Lee, Muzaffar Qazilbash, Andrew Kin, Jeffrey Zonder, Sally Arai, Michelle M. Chin, Rajshekhar Chakraborty, Suzanne Lentzsch, Hila Magen, Eden Shkury, Caitlin Sarubbi, Heather Landau, Stefan Schönland, Ute Hegenbart, Morie Gertz
{"title":"A model for predicting day-100 stem cell transplant-related mortality in AL amyloidosis","authors":"Eli Muchtar, Angela Dispenzieri, Vaishali Sanchorawala, Hamza Hassan, Raphael Mwangi, Matthew Maurer, Francis Buadi, Hans C. Lee, Muzaffar Qazilbash, Andrew Kin, Jeffrey Zonder, Sally Arai, Michelle M. Chin, Rajshekhar Chakraborty, Suzanne Lentzsch, Hila Magen, Eden Shkury, Caitlin Sarubbi, Heather Landau, Stefan Schönland, Ute Hegenbart, Morie Gertz","doi":"10.1038/s41409-025-02535-z","DOIUrl":"10.1038/s41409-025-02535-z","url":null,"abstract":"Autologous stem cell Transplant (ASCT)-related mortality (TRM) in AL amyloidosis remains elevated. AL amyloidosis patients (n = 1718) from 9 centers, transplanted 2003–2020 were included. Pre-ASCT variables of interest were assessed for association with day-100 all-cause mortality. A random forest (RF) classifier with 10-fold cross-validation assisted in variable selection. The final model was fitted using logistic regression. The median age at ASCT was 58 years. Day-100 TRM occurred in 75 patients (4.4%) with the predominant causes being shock, high-grade arrhythmia, and organ failure. Ten factors were associated with day-100 TRM on univariate analysis. RF classifier using these variables generated a model with an area under the curve (AUC) of 0.72 ± 0.12. To refine the model selection using importance hierarchy function, a 4-variable model [NT-proBNP/BNP, serum albumin, ECOG performance status (PS), and systolic blood pressure] was built with an AUC of 0.70 ± 0.12. Based on logistic regression coefficients, ECOG PS 2/3 was assigned two points while other adverse predictors 1-point each. The model score range was 0–5, with a day-100 TRM of 0.46%, 3.2%, 5.8%, and 14.5% for 0, 1, 2, and ≥3 points, respectively. This model to predict day-100 TRM in AL amyloidosis allows better-informed decision-making in this heterogeneous disease.","PeriodicalId":9126,"journal":{"name":"Bone Marrow Transplantation","volume":"60 5","pages":"595-602"},"PeriodicalIF":4.5,"publicationDate":"2025-02-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.nature.com/articles/s41409-025-02535-z.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143490511","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}