Transplantation and Cellular Therapy最新文献

{"title":"LETERMOVIR PRIMARY AND SECONDARY PROPHYLAXIS IN PEDIATRIC RECIPIENTS OF ALLOGENEIC HAEMATOPOIETIC STEM CELL TRANSPLANT.","authors":"Francesca Vendemini, Paola De Lorenzo, Francesca Romani, Sandro Maria Ivano Malandrin, Marta Verna, Sonia Bonanomi, Maria Grazia Valsecchi, Giovanna Lucchini, Adriana Balduzzi","doi":"10.1016/j.jtct.2025.05.021","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.021","url":null,"abstract":"<p><p>1.</p><p><strong>Background: </strong>CMV reactivation represents one of the most frequent infectious complications post HSCT. Letermovir is a viral terminate inhibitor which has been approved in adults for the prophylaxis of post HSCT CMV reactivation. Its use in pediatric HSCT recipient was recently approved by US FDA but data on the use of letermovir in children remain limited. 2.</p><p><strong>Objective(s): </strong>We aimed at comparing the incidence of CMV reactivation in a more recent cohort of pediatric HSCT recipients receiving primary or secondary prophylaxis with letermovir with a previous cohort of children receiving no prophylaxis. We analysed the risk factors for CMV reactivation among IgG seropositive recipients and the role of CMV reactivation on treatment related mortality/overall survival. 3.</p><p><strong>Study design: </strong>This is a single centre retrospective study which enrolled all consecutive patients aged <21 years who underwent allogeneic HSCT between 1 January 1, 2014 and October 31, 2023 at the pediatric HSCT Unit of the Fondazione IRCCS San Gerardo dei Tintori in Monza 4.</p><p><strong>Results: </strong>287 patients who received 308 HSCT were analysed. Three months cumulative incidence of CMV reactivation was 29.5% (95CI 24.3-35) in the standard cohort versus 3.7% (95%CI 0.3-16.3) in the cohort of patients receiving letermovir as primary prophylaxis (p= 0.0029). The use of letermovir as well as the use of a HLA-identical donor with no serotherapy, bone marrow as a source of stem cell and the absence of acute GVHD were statistically significant protective factors against CMV reactivation at multivariate analysis. At 3 months after discontinuation of preemptive therapy, the cumulative incidence of CMV reactivation was 0% for the 15 patients receiving letermovir as secondary prophylaxis versus 34.7% (SE 5.8, 95CI 23.7 -46.0) for the 72 patients not receiving secondary prophylaxis. 5.</p><p><strong>Conclusion(s): </strong>Letermovir is safe and efficacious in preventing CMV reactivation in pediatric patients undergoing HSCT in both primary and secondary prophylaxis.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144175012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Choosing between HLA-Mismatched Unrelated and Haploidentical donors: Donor age considerations.","authors":"Rohtesh S Mehta, Gabrielle Schmidt, Kirsten Williams, Shyam A Patel, Johannes Schetelig, Bipin Savani, Medhat Askar, Effie Petersdorf, Olle Ringden, Christopher G Kanakry, Jennifer A Kanakry, Heather Stefanski, Esteban Arrieta-Bolaños, Brian Betts, Cara Benjamin, Shahinaz Gadalla, Tao Wang, Jennifer Saultz, Stephen Spellman, Najla El Jurdi, Yung-Tsi Bolon, Stephanie J Lee","doi":"10.1016/j.jtct.2025.05.019","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.019","url":null,"abstract":"<p><strong>Background: </strong>Haploidentical donors and HLA-mismatched unrelated donors (MMUDs) are increasingly utilized for hematopoietic cell transplantation (HCT), with post-transplantation cyclophosphamide (PTCy) emerging as an effective graft-versus-host disease (GVHD) prophylaxis strategy. Despite the growing use of these donor types, comparative data to guide donor selection remain limited. Donor age is a known predictor of HCT outcomes, yet its specific impact when choosing between haploidentical and MMUD donors with PTCy-based prophylaxis has not been thoroughly explored.</p><p><strong>Objectives: </strong>This study aimed to evaluate the influence of donor age on HCT outcomes in patients receiving haploidentical or MMUD HCT with PTCy-based GVHD prophylaxis, hypothesizing that younger donors (<30 years) would be associated with improved outcomes compared to older donors (≥30 years) regardless of donor type.</p><p><strong>Study design: </strong>We conducted a retrospective analysis of 7,116 patients with hematologic malignancies from the Center for International Blood and Marrow Transplant Research (CIBMTR) database, transplanted between 2013 and 2021. Donors were categorized into four groups: younger haploidentical (<30 years), older haploidentical (≥30 years), younger MMUD (<30 years), and older MMUD (≥30 years). The primary outcome was GVHD-free relapse-free survival (GRFS), defined as the absence of grade III-IV acute GVHD, chronic GVHD requiring systemic immunosuppressive therapy (IST), relapse, or death. Secondary outcomes included overall survival (OS), treatment-related mortality (TRM), relapse, grade III-IV acute GVHD, overall chronic GVHD, and chronic GVHD requiring IST. Comparisons were made between (a) younger MMUD vs. older haploidentical and (b) younger haploidentical vs. older MMUD groups using multivariable Cox proportional hazards models.</p><p><strong>Results: </strong>In multivariable analysis, the older MMUD group exhibited inferior GRFS (Hazard Ratio [HR] 1.20; 95% confidence interval [CI], 1.06-1.36; p=0.003), higher TRM (HR 1.49; 95% CI, 1.13-1.96; p=0.005), and increased grade III-IV acute GVHD (HR 2.88; 95% CI, 1.43-5.80; p=0.003) compared to the younger haploidentical group. The younger MMUD group had modest GRFS improvement over the older haploidentical group (HR 0.87; 95% CI, 0.78-0.98; p=0.02) and significantly reduced risks of grade II-IV acute GVHD (HR 0.67; 95% CI, 0.51-0.88; p=0.003) and chronic GVHD (HR 0.78; 95% CI, 0.65-0.94; p=0.009).</p><p><strong>Conclusions: </strong>Younger donor age is associated with superior HCT outcomes, emphasizing the importance of prioritizing donors aged <30 years regardless of donor type when feasible.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144151669","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Feasibility of TBI-Augmented Reduced Toxicity Conditioning Regimen with ATG/PTCy Combination for Haploidentical Donor Transplantation in Adult Acute Lymphoblastic Leukemia.","authors":"Jaehyun Ahn, Daehun Kwag, Gi June Min, Sung-Soo Park, Silvia Park, Sung-Eun Lee, Byung-Sik Cho, Ki-Seong Eom, Yoo-Jin Kim, Hee-Je Kim, Chang-Ki Min, Seok-Goo Cho, Jae-Ho Yoon","doi":"10.1016/j.jtct.2025.05.017","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.017","url":null,"abstract":"<p><strong>Background: </strong>Haploidentical donor transplantation (HIDT) with post-transplant cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis is a promising alternative donor option for adults with high-risk acute lymphoblastic leukemia (ALL). However, the optimal conditioning regimen and GVHD prophylaxis strategy in this patient population remain unclear.</p><p><strong>Methods: </strong>We evaluated a newly optimized reduced-toxicity conditioning (RTC) regimen which consisted of fludarabine 150 mg/m², melphalan 100 mg/m², and low-dose total body irradiation 400 cGy (FMTBI) with GVHD prophylaxis using ATG/PTCy combination in 26 adult patients with ALL undergoing HIDT. We compared the new regimen to 52 historical controls receiving fludarabine 150 mg/m² plus busulfan 9.6 mg/kg (FB) with ATG. Key endpoints included disease-free survival (DFS), overall survival (OS), GVHD-and-relapse-free survival (GRFS), relapse, non-relapse mortality (NRM), and post-transplantation immune reconstitution.</p><p><strong>Results: </strong>At one year, the FMTBI group had higher DFS (80.4% vs. 51.9%, p=0.024) and a trend toward improved GRFS (61.0% vs. 34.6%, p=0.073). Relapse incidence was slightly lower (11.9% vs. 32.7%, p=0.059), particularly in the CNS. The cumulative incidence of moderate to severe chronic GVHD was lower (0.0% vs. 11.5%, p=0.074) in the FMTBI group. OS (82.9% vs. 78.8%, p=0.465) and NRM (7.7% vs. 15.4%, p=0.342) rates were similar. NK/NKT cell recovery was transiently delayed at 3 months after FMTBI regimen but normalized by 6 months.</p><p><strong>Conclusions: </strong>Our newly optimized FMTBI with ATG/PTCy combination showed improved DFS and relapse control while reducing chronic GVHD compared to historical FB with ATG alone in HIDT for adult ALL.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144151704","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Setting up a CAR-T Program: A Framework for Delivery from the Worldwide Network for Blood & Marrow Transplantation.","authors":"Syed Osman Ahmed, Riad El Fakih, Mohamed A Kharfan-Dabaja, Farhatullah Syed, Ghulam Mufti, Christian Chabannon, Damiano Rondelli, Mohamad Mohty, Ali A Al Ahmari, Jordan Gauthier, Marco Ruella, Miguel-Angel Perales, Shahrukh Hashmi, Feras Alfraih, Sarah Ghorashian, Mohsen Alzahrani, Zubair Abba, Mickey Koh, Marcelo Pasquini, Annalisa Ruggeri, Laurent Garderet, Abdulwahab Albabtain, Daniel Weisdorf, Hildegard Greinix, Hadeel Samarkandi, Nada Hamad, Yoshiko Atsuta, Mehdi Hamadani, Parameswaran Hari, Navneet S Majhail, Raffaella Greco, Hazzaa Alzahrani, Anna Sureda, Ibrahim Yakoub-Agha, Ali D Alahmari, Dietger Niederwieser, Mahmoud Aljurf","doi":"10.1016/j.jtct.2025.05.012","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.012","url":null,"abstract":"<p><p>Chimeric antigen receptor therapy (CAR-T therapy) is a genetically engineered cellular therapy that is currently integrated into the management of hematological malignancies. Institutions treating patients with CAR-T therapy need to establish a framework of delivery that covers all the main components of the patient journey including intake of patients into the program from referring centers, patient selection according to established eligibility criteria, apheresis, logistics, bridging therapy, infusion and post-infusion care. A CAR-T therapy program, with its unique requirements, needs to be delivered by a multidisciplinary team (MDT). Prior to the establishment of the program, a well-structured business plan should be developed with a clear financial and/or reimbursement model. Consideration should be given to the overall capacity and staffing requirements. Standard operating procedures and guidelines are vital for ensuring that quality standards are clearly defined and adhered to. Institutions should develop a research plan for CAR-T that may incorporate not only industry sponsored trials but also in-house CAR-T manufacture of investigational CAR-T constructs. This report presents recommendations from a group of international experts highlighting the priorities and considerations when developing a new CAR-T program.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143600","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cumulative Dose of Peg-asparaginase Is Associated with Outcome of Allogeneic Hematopoietic Stem Cell Transplantation for Acute Lymphoblastic Leukemia.","authors":"Junjie Chen, Jia Li, Zhixiang Wang, Zicong Huang, Jieping Lin, Jiawang Ou, Xiuli Xu, Bingqing Tang, Chenhao Ding, Zihong Cai, Ren Lin, Li Xuan, Qifa Liu, Hongsheng Zhou","doi":"10.1016/j.jtct.2025.05.015","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.015","url":null,"abstract":"<p><p>The integration of pediatric-inspired chemotherapy with allogeneic hematopoietic stem cell transplantation (HSCT) for adult acute lymphoblastic leukemia (ALL) remains marginally addressed. Based on retrospective analysis, we designed a pegylated-asparaginase (PEG-Asp) -intensified pediatric-inspired regimen, PDT-ALL-2016. In this protocol, HSCT is allocated following chemotherapy which containing 4 or 5 doses PEG-Asp. We evaluated the impact of PEG-Asp on transplantation outcomes in two cohorts: the prospective PDT-ALL-2016 cohort (2016-2021, N= 218) and a retrospective adult regimen cohort (2008-2015, N= 279). In retrospective cohort, we identified that high-dose (4-5 doses) PEG-Asp prior to HSCT was optimal for survival, which was subsequently incorporated into the PDT-ALL-2016 protocol. The 5-year overall survival (OS) in prospective cohort was 68.8% (95% CI, 62.4-75.8%). Meanwhile, patients receiving full 4-5 doses PEG-Asp exhibited superior survival compared to those receiving a fewer doses in prospective cohort, with 5-year OS of 75.3% (95% CI, 68.2-83.1%) versus 59.5% (95% CI, 48.8-72.5%), respectively. As anticipated, PEG-Asp intensified prospective cohort demonstrated superior 5-year OS compared to retrospective cohort (43.4%; 95% CI, 37.7-49.9%). Then, the effect of high-dose PEG-Asp was confirmed across the entire cohort. High-dose PEG-Asp (N=154; 31.2%) resulted in superior survival, compared to low- (≤1 doses; N=206; 41.3%) or medium-dose (2-3 doses; N=137; 27.5%), as validated by multivariate analysis. Herein we presented that PEG-Asp is associated with outcome of HSCT, suggesting that integrating PEG-Asp-intensified chemotherapy with HSCT may constitute total therapy for adult ALL.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144143559","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Long-term Financial Toxicity after CAR T-cell Therapy among Patients in Remission and Their Caregivers.","authors":"Lucy P Andersen, Ryan J Quinn, Heather Difilippo, Alfred L Garfall, David L Porter, Salimah H Meghani, Jie Deng","doi":"10.1016/j.jtct.2025.05.013","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.013","url":null,"abstract":"<p><strong>Background: </strong>The long-term financial toxicity for patients who received Chimeric Antigen Receptor (CAR) T-cell therapy and their caregivers remains under-explored.</p><p><strong>Aims: </strong>The aim of this research is to describe the financial toxicity of patients who are in remission one to five years after receiving CAR T-cell therapy and their caregivers and explore associations between social determinants of health (SDoH), clinical factors, and health-related quality of life (HRQoL) with financial toxicity.</p><p><strong>Methods: </strong>This cross-sectional study included adults who had received CAR T-cell therapy for a hematologic malignancy and their current or former informal caregivers. Patients and caregivers completed measures of financial toxicity, HRQoL, and a demographic survey, while patients also completed cognitive function and symptom burden measures. Descriptive and bivariate statistics were used in this exploratory analysis.</p><p><strong>Results: </strong>There were 58 patients and 31 caregivers study participants. Financial toxicity was relatively low, 25% of patients reported mild to moderate and 18% of caregivers reported mild to severe financial toxicity. Patient financial toxicity was significantly associated with patient income, HRQoL domains, the mental HRQoL summary score, and symptom burden. Caregiver financial toxicity was significantly associated with caregiver age, employment status, HRQoL domains, and the mental HRQoL summary score.</p><p><strong>Conclusions: </strong>Patients and caregivers reported low levels of financial toxicity in the present study: A majority of patients (75%) and caregivers (81%) experienced zero to minimal financial toxicity. Certain patients and caregivers at higher risk for financial toxicity may benefit from targeted interventions coupled with supportive care to address other HRQoL needs.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144133287","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"NIH Chronic Graft-versus-Host Disease Consensus Conference 2025 Update.","authors":"Stephanie J Lee, Kirsten M Williams, Stefanie Sarantopoulos, Carrie L Kitko, Corey Cutler, Joseph Pidala, Geoffrey R Hill, Zachariah DeFilipp, Hildegard T Greinix, Daniel Wolff, Sophie Paczesny, Geoffrey D E Cuvelier, Kirk R Schultz, Steven Z Pavletic","doi":"10.1016/j.jtct.2025.05.016","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.016","url":null,"abstract":"<p><p>In 2020, the third NIH Consensus Development Project on Criteria for Chronic Graft-versus-Host Disease (GVHD) Clinical Trials was held with the goals of identifying gaps in understanding, prevention and treatment of chronic graft-versus-host disease (GVHD) and making actionable recommendations that would advance the field. An interim meeting was held in October 2024 to review progress on the 2020 recommendations. Each group was charged with reviewing their previous recommendations, assessing whether the field is on track to eventually achieve the goals, and considering whether recommendations should be modified in light of new data or insufficient progress. This manuscript summarizes the Working Groups' reports and helps define the research agenda for future studies in chronic GVHD. Overall, modest progress has been made on most initiatives. Some studies in progress will address key recommendations and results are eagerly anticipated.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144133288","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"American Society for Transplantation and Cellular Therapy Series #9: Management of HHV-6B After Hematopoietic Cell Transplantation and Chimeric Antigen Receptor (CAR)-T-Cell Therapy.","authors":"Eleftheria Kampouri, Guy Handley, Tuan L Phan, Yeon Joo Lee, Ryan Shaw, Paul A Carpenter, Sanjeet S Dadwal, Roy F Chemaly, Genovefa A Papanicolaou, Masao Ogata, Michael Boeckh, Danielle M Zerr, Joshua A Hill","doi":"10.1016/j.jtct.2025.05.001","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.001","url":null,"abstract":"<p><p>The Practice Guidelines Committee and the Transplant Infectious Disease Special Interest Group of the American Society for Transplantation and Cellular Therapy developed guidelines focusing on human herpes virus 6B (HHV-6B). A compendium-style approach was used to address a series of standalone frequently asked questions (FAQs), supported by tables and figures to spotlight key concepts. Adult and pediatric infectious disease and hematopoietic cell transplantation (HCT) content experts developed these FAQs, and finalized recommendations after consensus was reached. This ninth topic in the series focuses on the relevant risk factors, diagnostic considerations, prophylaxis, and treatment approaches relevant to HHV-6B infections after HCT and Chimeric Antigen Receptor (CAR)-T-Cell Therapy.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144133285","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sinusoidal Obstruction Syndrome after Allogeneic Hematopoietic Cell Transplantation with Post-Transplant Cyclophosphamide.","authors":"Aitana Balaguer-Roselló, Juan Montoro, Marta Villalba, Patricia Lizama, Lisseth Torres, Pedro Chorão, Pedro Asensi Cantó, Pablo Granados, Juan Eirís, Christian Tejada, Marta Moreno-Torres, Vicente Navarro-Aguilar, Alexandre Pérez-Girbés, Erika Moro, Inés Gómez-Seguí, Pilar Solves, Ana Bataller, Brais Lamas, Alberto Louro, José Vicente Castell, Javier de la Rubia, Miguel Ángel Sanz, Jaime Sanz","doi":"10.1016/j.jtct.2025.05.018","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.018","url":null,"abstract":"<p><strong>Backround: </strong>Sinusoidal obstruction syndrome/veno-occlusive disease (SOS/VOD) is a serious complication following allogeneic hematopoietic cell transplantation (HCT). Although post-transplant cyclophosphamide (PTCy) is increasingly used for graft-versus-host disease prophylaxis, data on its impact in the context of SOS/VOD remain limited.</p><p><strong>Objectives: </strong>This study aimed to assess the incidence, clinical characteristics, prognostic factors, treatment approaches, and outcomes of SOS/VOD in HCT recipients receiving GVHD prophylaxis with PTCY, sirolimus or tacrolimus, and mycophenolate mofetil (MMF) across all donor types.</p><p><strong>Study design: </strong>This single-center observational study included all 532 consecutive adults who underwent HCT with PTCy between January 2017 and February 2024. Patient demographics, transplant procedures, toxicities, and complications were prospectively collected. Clinical charts were reviewed as needed to address inconsistencies or missing information.</p><p><strong>Results: </strong>Myeloablative conditioning was administered to 96% of recipients, who received grafts from matched sibling donors (MSD, 36%), matched unrelated donors (MUD, 34%), haploidentical donors (26%), or mismatched unrelated donors (MMUD, 4%). SOS/VOD was diagnosed in 35 patients and classified according to EBMT criteria as probable (n=10), clinical (n=23), or proven (n=2). Classical SOS/VOD occurred in 21 patients (60%), while 14 (40%) had late-onset disease. EBMT severity grading showed 3% mild, 20% moderate, 37% severe, and 40% very severe cases. The 100-day cumulative incidence was 6.6% (95% CI:4.7-8.9). Multivariable analysis identified prior transplantation, prior antibody-drug conjugates and higher CD3+ cell dose as risk factors. Patients with very severe or severe SOS/VOD (based on clinical features but not those upgraded solely due to the presence of risk factors) received defibrotide. Four patients (11.4%) died from SOS/VOD; all classified as very severe, and three of them had undergone prior transplantation (two autologous, one allogeneic). Despite high severity, SOS/VOD analyzed as a time-dependent variable showed no association with overall survival or non-relapse mortality.</p><p><strong>Conclusions: </strong>SOS/VOD remains a challenging but clinically manageable complication with a modest incidence following HCT with PTCy. Although many cases were classified as severe or very severe, the associated mortality rate was relatively low. The identification of key risk factors, such as prior transplantation, antibody-drug conjugate exposure, and higher CD3⁺ cell doses, along with the notable proportion of late-onset cases, underscores the need for vigilant monitoring and individualized management strategies.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144128855","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Current Activity Trends and Outcomes in Hematopoietic Cell Transplantation and Cellular Therapy - A report from the CIBMTR.","authors":"Spellman Stephen R, Xu S, Oloyede Temitope, Ahn Kwang Woo, Akhtar Othman, Bolon Yung-Tsi, Broglie Larisa, Bloomquist Jenni, Bupp Caitrin, Chen Min, Devine Steven M, El-Jurdi Najla, Hamadani Mehdi, Hengen Mary, Huppler Anna H, Jaglowski Samantha, Kuxhausen Michelle, Lee Stephanie J, Moskop Amy, Page Kristin M, Pasquini Marcelo C, Perez Waleska, Phelan Rachel, Rizzo Doug, Saber Wael, Stefanski Heather E, Steinert Patricia, Tuschl Eileen, Visotcky Alexis, Vogel Rebecca, Auletta Jeffery J, Shaw Bronwen E, Allbee-Johnson Mariam","doi":"10.1016/j.jtct.2025.05.014","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.05.014","url":null,"abstract":"&lt;p&gt;&lt;p&gt;The Center for International Blood and Marrow Transplant Research (CIBMTR) compiles annual summary slides describing trends in hematopoietic cell transplantation (HCT) and cellular therapy (CT) practice and outcomes. This year's report includes all patients receiving their first autologous and/or allogeneic HCT/CT in the United States between 2013 and 2023 or chimeric antigen receptor T-cell (CAR-T) from 2016 and 2023, reported to CIBMTR. Relative proportion of allogeneic and autologous HCT/CT was generated as percentage of total for donor type and for patient age, disease indication, graft-versus-host disease (GVHD) prophylaxis, and race and ethnicity. Causes of death were summarized using frequencies, and the Kaplan-Meier estimator was used for estimating overall survival. New for this year, disease risk stratification reflects European LeukemiaNet cytogenetic risk score for acute myeloid leukemia (AML) and the Revised International Prognostic Scoring System for myelodysplastic syndromes (MDS). Use of allogeneic HCT increased substantially in 2023, recovering from a decline in activity during the COVID-19 pandemic, with growth predominately in the 65-74 year-old age group. Overall, matched unrelated donors (MUD) continue as the most common allogeneic donor source (45%) followed by haploidentical related donors (Haplo) (21%), matched related donors (MRD) (18%), mismatched unrelated donors (MMUD) (12%) and cord blood (Cord) (3%). These trends hold in the adult patient population with a notable doubling of MMUD utilization since 2020 driven by the rapid shift to post-transplant cyclophosphamide based GVHD prophylaxis (PTCy) in this setting. In the pediatric setting, Haplo was the most common donor source surpassing MRD use in 2023 followed by MUD, Cord and MMUD. Autologous HCT continued to decline slightly while use of CAR-T therapy has rapidly increased since commercial approval in 2017 with lymphoma and multiple myeloma reaching 45% and 16%, respectively in 2023. Significant recent changes in GVHD prophylaxis in the adult allogeneic HCT setting have occurred. PTCy is most common in Haplo HCT with &gt;90% since 2016. Among other donor sources, the most rapid adoption is in MMUD HCT at 82% in 2023. In MRD and MUD, PTCy use differs by conditioning intensity with RIC/NMA higher (58% and 64%, respectively), reflecting the standard of care established by BMT CTN 1703, compared to MAC (43% and 46%, respectively). In pediatrics, calcineurin inhibitor ± others remains the most common GVHD prevention strategy for MRD (88%) and MUD (68%). Although common in the pediatric Haplo HCT setting at 68% in 2023, use of PTCy is less common across other mismatched donor types where use of abatacept or ex-vivo T cell depletion/CD34 selection accounts for 28% and 17% in MMUD, respectively. Three-year overall survival continues to significantly improve among patients receiving allogeneic (62.1% vs 55.8%) and autologous (82.6% vs 79.6%) HCT when comparing HCT from 20","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120938","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}