Transplantation and Cellular Therapy最新文献

{"title":"Feasibility of a Total Body Irradiation-Augmented Reduced-Toxicity Conditioning Regimen with an Antithymocyte Globulin/Post-Transplantation Cyclophosphamide 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":"10.1016/j.jtct.2025.05.017","url":null,"abstract":"<p><p>Haploidentical donor transplantation (HIDT) with post-transplantation cyclophosphamide (PTCy)-based graft-versus-host disease (GVHD) prophylaxis is a promising alternative donor option for adults with high-risk acute lymphoblastic leukemia (ALL). The optimal conditioning regimen and GVHD prophylaxis strategy in this patient population remain unclear, however. We evaluated a newly optimized reduced-toxicity conditioning regimen consisting of fludarabine 150 mg/m², melphalan 100 mg/m², and low-dose (400 cGy) total body irradiation (FMTBI) with GVHD prophylaxis using an antithymocyte globulin (ATG)/PTCy combination in 26 adult patients with ALL undergoing HIDT. We compared the recipients of the new regimen to 52 historical controls who received fludarabine 150 mg/m² plus busulfan 9.6 mg/kg (FB group) with ATG. Key endpoints included disease-free survival (DFS), overall survival (OS), GVHD-and relapse-free survival (GRFS), relapse, nonrelapse mortality (NRM), and post-transplantation immune reconstitution. At 1 year post-transplantation, the FMTBI group had higher DFS (80.4% versus 51.9%; P = .024) and a trend toward improved GRFS (61.0% versus 34.6%; P = .073). Relapse incidence was slightly lower (11.9% versus 32.7%; P = .059) in the FMTBI group, particularly in the central nervous system. The cumulative incidence of moderate to severe chronic GVHD was lower (0.0% versus 11.5%; P = .074) in the FMTBI group. The rates of OS (82.9% versus 78.8%; P = .465) and NRM (7.7% versus 15.4%; P = .342) were similar in the 2 groups. Natural killer/natural killer T cell recovery was transiently delayed at 3 months after the FMTBI regimen but normalized by 6 months. Compared to the historical FB with ATG alone group, our newly optimized FMTBI and ATG/PTCy combination showed improved DFS and relapse control while reducing chronic GVHD 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 Chimeric Antigen Receptor T Cell Therapy 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":"10.1016/j.jtct.2025.05.012","url":null,"abstract":"<p><p>Chimeric antigen receptor T cell (CAR-T) therapy is a genetically engineered cellular therapy that is currently integrated into the management of hematologic 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 postinfusion care. A CAR-T therapy program, with its unique requirements, needs to be delivered by a multidisciplinary team. 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 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 therapy that may incorporate not only industry-sponsored trials, but also in-house manufacturing of investigational CAR-T products. 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":"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, containing 4 or 5 doses of PEG-Asp. We evaluated the impact of PEG-Asp on transplantation outcomes in 2 cohorts: the prospective PDT-ALL-2016 cohort (2016-2021, N = 218) and a retrospective adult regimen cohort (2008-2015, N = 279). In the 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 the prospective cohort was 68.8% (95% confidence interval [CI], 62.4%-75.8%). Patients receiving the full 4-5 doses of PEG-Asp exhibited superior survival compared with those receiving fewer doses in the prospective cohort, with a 5-year OS of 75.3% (95% CI, 68.2%-83.1%) versus 59.5% (95% CI, 48.8%-72.5%), respectively. As anticipated, the PEG-Asp-intensified prospective cohort demonstrated superior 5-year OS compared with the 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 with low-dose (≤1 doses; N = 206; 41.3%) or medium-dose (2-3 doses; N = 137; 27.5%), as validated by multivariate analysis. Herein, we have presented that PEG-Asp is associated with the 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":"10.1016/j.jtct.2025.05.013","url":null,"abstract":"<p><p>The long-term financial toxicity for patients who received Chimeric Antigen Receptor (CAR) T-cell therapy and their caregivers remains under-explored. 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. 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. 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. 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":"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 Human Herpesvirus 6B After Hematopoietic Cell Transplantation and Chimeric Antigen Receptor-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":"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 herpesvirus 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-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":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12270546/pdf/","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":"OA","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":"10.1016/j.jtct.2025.05.018","url":null,"abstract":"<p><p>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. 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. 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. 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 to 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 nonrelapse mortality. 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":"Low Peripheral Blood Counts and Elevated Proinflammatory Cytokines Signal a Poor CD19 Chimeric Antigen Receptor T-cell Response in Acute Lymphoblastic Leukemia.","authors":"Katelyn Burleigh, Kelly G Stratton, Jenny L Smith, Michael C Jensen, Cameron J Turtle, Camille Keenan, Colleen Annesley, Corinne Summers, Bobbie-Jo Webb-Robertson, Alexandre V Hirayama, Rebecca A Gardner, Heather H Gustafson","doi":"10.1016/j.jtct.2025.05.003","DOIUrl":"10.1016/j.jtct.2025.05.003","url":null,"abstract":"<p><p>CD19 chimeric antigen receptor T-cell (CAR-T) therapy has significantly improved outcomes for patients with relapsed/refractory B-cell acute lymphoblastic leukemia (R/R B-ALL). However, approximately 20% of patients fail to achieve a complete remission (CR), and some develop severe, life-threatening toxicities. Understanding the biological mechanisms underlying both dysfunctional responses and severe toxicity is essential for optimizing patient management and improving therapeutic efficacy. This study aimed to (1) characterize cytokine profiles associated with dysfunctional responses and severe toxicity following CAR-T infusion, (2) examine the timing and trajectory of cytokine changes in relation to treatment outcomes, and evaluate potential strategies for mitigating toxicity and treatment failure. We conducted a comprehensive analysis of serum cytokine profiles in 86 adult and pediatric patients undergoing autologous CD19 CAR-T therapy for B-ALL. Patients were categorized into three groups: (1) Dysfunctional response-Patients who failed to achieve a minimal residual disease-negative CR (MRD-CR) by Day 63 or who experienced recurrence of CD19+ disease in the setting ongoing CAR-T cell detection before Day 63. (2) Functional response with severe cytokine release syndrome (CRS) and/or neurotoxicity (NTX)-Patients with best response of MRD-CR by Day 63 who experienced grade 3 or higher CRS or NTX. (3) Functional response without severe CRS or NTX-Patients with best response of MRD-CR by Day 63 who did not experience grade ≥3 CRS or NTX. Cytokine levels were measured during the first-week postinfusion and correlated with treatment efficacy, toxicity outcomes, complete blood counts, and CAR-T expansion dynamics. This analysis aimed to better understand how cytokine profiles relate to patient outcomes and immune responses in CAR-T therapy. Patients with dysfunctional response exhibited decreased neutrophils, platelets, and levels of granulocytic cytokines (suggestive of low bone marrow reserve) alongside elevated pro-inflammatory cytokines by Day 1. Functional response with severe toxicity patients showed a progressive rise in proinflammatory cytokines, reaching similar levels to dysfunctional response patients by Day 7. We observed that high cytokines at both the Day 1 and Day 7 time points were associated with poor survival. These findings remained significant when adjusting for high disease burden, a known predictor of severe inflammatory toxicity and lack of response. Early post-CAR-T infusion inflammation is associated with both dysfunctional response and severe toxicity-even after adjusting for disease burden. This suggests that inflammation, in addition to disease burden, plays a role in determining patient outcome. Therefore, strategies aimed at reducing the pro-inflammatory state prior to or early after CAR-T cell infusion may improve outcomes for R/R B-ALL patients.</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":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12237277/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144120939","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","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":"Stephen R Spellman, Ke Xu, Temitope Oloyede, Kwang Woo Ahn, Othman Akhtar, Yung-Tsi Bolon, Larisa Broglie, Jenni Bloomquist, Caitrin Bupp, Min Chen, Steven M Devine, Najla El-Jurdi, Mehdi Hamadani, Mary Hengen, Anna H Huppler, Samantha Jaglowski, Michelle Kuxhausen, Stephanie J Lee, Amy Moskop, Kristin M Page, Marcelo C Pasquini, Waleska Perez, Rachel Phelan, Doug Rizzo, Wael Saber, Heather E Stefanski, Patricia Steinert, Eileen Tuschl, Alexis Visotcky, Rebecca Vogel, Jeffery J Auletta, Bronwen E Shaw, Mariam Allbee-Johnson","doi":"10.1016/j.jtct.2025.05.014","DOIUrl":"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) therapy from 2016 and 2023, reported to the CIBMTR. A 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 the 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- to 74-year-old age group. Overall, matched unrelated donors (MUDs) continue to be the most common allogeneic donor source (45%) followed by haploidentical related donors (Haplo; 21%), matched related donors (MRDs; 18%), mismatched unrelated donors (MMUDs; (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-transplantation 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 use. Autologous HCT continued to decline slightly, whereas 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 MRDs and MUDs, PTCy use differs by conditioning intensity, with non-myeloablative/reduced-intensity conditioning (NMA/RIC) higher (58% and 64%, respectively), reflecting the standard of care established by BMT CTN 1703, compared with myeloablative (MAC; 43% and 46%, respectively). In pediatrics, calcineurin inhibitor ± others remains the most common GVHD prevention strategy for use of MRDs (88%) and MUDs (68%). Although common in the pediatric Haplo HCT setting at 68% in 2023, use of PTCy is less common across other mismatched donor types in which use of abatacept or ex-vivo T-cell depletion/CD34 selection accounts for 28% and 17% in MMUDs, respectively. Three-year overall survival continues to significan","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}

{"title":"Outcomes of Multiple Myeloma Patients With Prior Solid Tumors Undergoing Autologous Transplantation.","authors":"Oren Pasvolsky, Curtis Marcoux, Denái R Milton, Natalie Rafaeli, Mark R Tanner, Qaiser Bashir, Samer Srour, Neeraj Saini, Paul Lin, Jeremy Ramdial, Yago Nieto, Guilin Tang, Ali H Mohamedi, Abdullah F Deen, Yosra Aljawai, Hans C Lee, Krina K Patel, Melody R Becnel, Partow Kebriaei, Sheeba K Thomas, Robert Z Orlowski, Richard Champlin, Elizabeth J Shpall, Muzaffar H Qazilbash","doi":"10.1016/j.jtct.2025.05.008","DOIUrl":"10.1016/j.jtct.2025.05.008","url":null,"abstract":"<p><p>Upfront autologous hematopoietic cell transplantation (autoHCT) remains standard of care for eligible patients with newly diagnosed multiple myeloma (MM). Comorbidities are routinely evaluated to determine eligibility and estimate mortality after autoHCT, including the history of prior solid tumor (PST). While PST is considered high-risk for worse survival based on widely used risk indices, its independent impact on transplant outcomes in MM remains unclear. To elucidate the prognostic impact of PST in patients with MM undergoing upfront autoHCT. We conducted a single-center retrospective analysis of consecutive MM patients who underwent upfront autoHCT between 1997 and 2021, categorizing them into those with (PST+) and without (PST-) prior solid organ malignancy. Primary endpoints were progression-free survival (PFS) and overall survival (OS). Among 2853 patients included in this analysis, 274 (10%) were PST+ and 2579 (90%) were PST-. The PST+ patients were older (67 vs. 60 years; P < .001), predominantly male (66% vs. 58%; P = .010), were more often transplanted in the year 2010 or later (78% vs. 69%; P = .003) and were more likely to have high-risk cytogenetic abnormalities (30% vs. 24%; P = .06). There was no significant difference in pre-transplant hematologic response (P = .33), day-100 post-transplant (P = .35) or the best post-transplant response (P = .27) between the PST+ and PST- groups. Similarly, there were no differences in pre-transplant (P = .34) or best post-transplant (P = .44) MRD status between the two groups. After a median follow-up of 53.8 months (range 0.2-262), the median PFS was comparable (36.7 months in PST+ vs. 39.9 months in PST-, P = .31), yet the median OS was significantly shorter in the PST+ group (81.3 months vs. 104.0 months, P = .020). Multivariable analysis confirmed PST as an independent predictor of inferior OS (HR 1.34, P = .011). MM patients undergoing upfront autoHCT with PST had worse OS compared to those without PST, despite similar response rates and PFS.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144095028","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}