Transplantation and Cellular Therapy最新文献

{"title":"Prognostic implications of splenomegaly in BCMA-directed CAR T-Cell therapy for relapsed myeloma.","authors":"Thomas Wiemers, Maximilian Ferle, Jonas Ader, Veronika Sotikova, David Fandrei, Nora Grieb, Luise Fischer, Patrick Born, Heike Weidner, Song Yau Wang, Madlen Jentzsch, Georg-Nikolaus Franke, Carmen Herling, Klaus Metzeler, Marco Herling, Simone Heyn, Timm Denecke, Kristin Reiche, Uwe Platzbecker, Vladan Vucinic, Thomas Neumuth, Hans-Jonas Meyer, Maximilian Merz","doi":"10.1016/j.jtct.2025.07.003","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.07.003","url":null,"abstract":"<p><strong>Background: </strong>B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell therapy has shown significant promise for patients with relapsed or refractory multiple myeloma (RRMM). Despite its efficacy, treatment is frequently complicated by adverse events such as cytokine release syndrome and hematologic toxicities, including severe thrombocytopenia. Identifying reliable prognostic markers is essential to improve patient risk stratification, optimizing treatment strategies, and managing complications effectively. While various prognostic markers have been explored, spleen size has not been extensively studied in this context.</p><p><strong>Objective: </strong>This study aims to evaluate spleen size as a prognostic marker in RRMM patients receiving CAR T-cell therapy. Specifically, we examine its association with thrombocytopenia, metabolic tumor volume, soluble BCMA (sBCMA) levels, progression-free survival (PFS), and overall survival (OS). Additionally, we compare spleen size to other established prognostic markers, including sBCMA, EASIX, and CAR-HEMATOTOX scores, to determine its predictive value.</p><p><strong>Study design: </strong>Data from RRMM patients (N=73) treated with either Idecabtagene vicleucel or Ciltacabtagen autoleucel were analyzed. The association of spleen size, assessed via computed tomography imaging, with clinical outcomes was analyzed.</p><p><strong>Results: </strong>Splenomegaly (spleen size > 340 cm³) was found to be significantly associated with severe and prolonged thrombocytopenia, higher metabolic tumor volumes, and elevated sBCMA levels. In our cohort, splenomegaly emerged as an independent prognostic factor for both PFS and OS, showing stronger associations than other markers such as sBCMA, EASIX, and CAR-HEMATOTOX scores.</p><p><strong>Conclusions: </strong>Spleen size may serve as a promising prognostic marker in CAR T-cell therapy for RRMM patients, providing a simple and readily accessible tool for enhancing risk stratification. This finding could inform monitoring strategies and optimize healthcare resource management for these patients.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144668598","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":"A ten-year follow-up study of children with thalassemia major post transplantation using treosulfan, thiotepa, and fludarabine-based conditioning regimen and its impact on growth and puberty.","authors":"Kavitha Ganesan, Vijayashree Muthukumar, Anupama Nair, Nithya Seshadri, Minakshi Balwani, Anuraag Nalla Reddy, Soundaram Valliyappan, Ramya Uppuluri, Revathi Raj","doi":"10.1016/j.jtct.2025.07.011","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.07.011","url":null,"abstract":"<p><strong>Background: </strong>We present a uniform cohort of children with thalassemia major who underwent treosulfan-based conditioning for hematopoietic stem cell transplantation (HSCT) and its impact on growth and puberty.</p><p><strong>Patients and methods: </strong>The study included retrospective analysis of children up to 18 years of age who underwent allogeneic HSCT for transfusion-dependent thalassemia major between 2010 to 2020 with a minimum follow-up period of two years.</p><p><strong>Results: </strong>Of 202 children in the study, 59% were males and 41% were females and 110/202 (54%) had a matched family donor (MFD), 62/202 (31%) haploidentical and 30/202 (15%) matched unrelated donor (MUD). Seventy-three (36%) were <5 years of age at HSCT, 90 (45%) between 5 to 10 years, and 39 (19%) were over 10 years of age. The mean height SDS at HSCT was -0.574 and at the current assessment, the mean height SDS was -0.669 (p=0.391). There was no significant reduction in growth potential. Twenty-nine (14.4%) were short at the time of HSCT (height SDS <-2) and at the current assessment, six (20.7%) continued to remain short, while 23 (79.3%) had catch-up growth and moved to height SDS ≥-2. The mean height SDS during HSCT in Class 1 thalassemia was -0.216, -0.478 in Class 2, and -0.898 in Class 3 respectively (p=0.026). The current height SDS in these classes are -0.115, -0.710, and -0.929 respectively, confirming that children in Class 1 and 2 were able to catch up on their growth; however, Class 3 patients failed to catch up growth after HSCT (p=0.010). In the children currently above 10 years of age, 17 (43.6%) were in Tanner stage 5. Of the 83 female children, 45 (54.2%) attained spontaneous menarche. Fourteen (6.9%) children required growth hormone supplementation.</p><p><strong>Conclusion: </strong>Our study demonstrates the impact of treosulfan-based conditioning on growth and puberty in children with thalassemia major. Despite the higher cost of treosulphan, growth potential was maintained and pubertal growth was per age for majority of the children in the cohort.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144668597","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":"Effect of Anti-thymocyte Globulin and Post-Transplant Cyclophosphamide on GvHD Outcomes in Female Donor-to-Male Recipient Matched Unrelated Donor Allogeneic Stem Cell Transplantation for Acute Leukemia.","authors":"Nihar Desai, Sergio Rodriguez Rodriguez, Mats Remberger, Eshrak Al-Shaibani, Tommy Alfaro Moya, Igor Novitzky-Basso, Ivan Pasic, Fotios V Michelis, Auro Viswabandya, Dennis Dong Hwan Kim, Rajat Kumar, Jonas Mattsson, Arjun D Law","doi":"10.1016/j.jtct.2025.07.010","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.07.010","url":null,"abstract":"<p><p>Female-to-male (F→M) sex-mismatched allogeneic hematopoietic stem cell transplantation (HSCT) is known to increase the risk of graft-versus-host disease (GvHD). We evaluated the impact of donor-recipient sex mismatch on GvHD incidence and assessed the efficacy of combined low-dose antithymocyte globulin (ATG) (2.5 mg/kg) and post-transplant cyclophosphamide (PTCy) as GvHD prophylaxis compared to calcineurin inhibitor-methotrexate/mycophenolate mofetil (CNI-MTX/MMF). We retrospectively analyzed 861 HSCT recipients, with AML as the predominant indication (82%). Among the cohort, 39% of transplants were sex-mismatched (M→F: 26%, F→M: 13%), while 61% were sex-matched (M→M: 42%, F→F: 19%). The primary outcomes were cumulative incidences of acute and chronic GvHD, relapse, and non-relapse mortality (NRM). F→M HSCT were associated with higher rates of grade II-IV acute GvHD at day +100 (42.2% vs. 27.0%, HR: 1.54; p<0.01) and chronic GvHD at 2 years (54.2% vs. 43.4%, HR: 1.33; p=0.05). In the overall cohort, ATG-PTCy was associated with a reduced risk of grade III-IV acute GvHD (HR: 0.42; p<0.01) and chronic GvHD (HR: 0.22; p<0.001) compared to CNI-MTX/MMF, without an increased risk of relapse (HR: 0.86, p=0.39) or NRM (HR: 0.59, p=0.35). A subgroup multivariable analysis of F→M recipients (n=114) confirmed a reduced risk of grade II-IV (HR: 0.48, p=0.05), grade III-IV acute GvHD (HR: 0.25; p=0.04), and chronic GvHD (HR: 0.33; p<0.01) with ATG-PTCy. F→M sex mismatch is associated with increased GvHD risk after HSCT. The combination of low-dose ATG and PTCy may help reduce GvHD in this high-risk group without an increase in disease relapse or NRM.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144643564","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":"Insurance Barriers to High-Cost Anti-Infective Medications Post Allogeneic Hematopoietic Cell Transplant.","authors":"Stephen Eng, Grashma Vadakkel, Samantha Brown, Susan Murillo, Warren Fingrut, Sean Devlin, Melinda Cook, Lauren DeRespiris, Andrew Lin, Stephanie Monaco-Cermak, Mary Nauffal, Sergio A Giralt, Miguel-Angel Perales, Juliet Barker, Christina Cho, Moneeza Walji, Gunjan L Shah","doi":"10.1016/j.jtct.2025.07.006","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.07.006","url":null,"abstract":"&lt;p&gt;&lt;p&gt;Hematopoietic cell transplant (HCT) is a potentially curative treatment modality for patients with hematologic malignancies, but it can be associated with significant financial burden and toxicity due to increased medical expenditures (i.e., prescription drugs). Prophylactic anti-infective medications are an essential component of allogeneic hematopoietic cell transplantation (allo-HCT) and help mitigate early post-transplant complications. Although these prophylactic anti-infective medications are essential, insurance barriers (i.e., prior authorizations, denials, and high out-of-pocket costs) limit access for many patients and may result in reduced access to allo-HCT. The objective of this study was to evaluate the degree and severity of insurance barriers in a large cohort of allo-HCT recipients to characterize the patient populations and transplant types experiencing these barriers. Medical records of patients who received high-cost medications (mold-active triazoles and letermovir [LTV]) during their first allo-HCT (1/1/20 - 5/1/22) were evaluated for insurance type, ancestry/ethnicity, and stem cell source. Pharmacy records were used to analyze insurance barriers in the first 100 days post-transplant. Patients were categorized as having Minimal to None, Moderate, or Extensive barriers. In the azole (n=287, voriconazole n=162, posaconazole n=64, isavuconazole n=61) and LTV (n=191) groups, the median age was 61 (range 19-81) and 61 years (range 22-81), respectively, with 44% female overall. In the azole group, 60% of patients had Minimal to None, 23% had Moderate, and 17% had Extensive barriers, while in the LTV group, 39% had Minimal to None, 28% had Moderate, and 33% had Extensive barriers. Seventy percent of patients had private insurance. The proportion of private vs. governmental insurance was equal in each barrier category in the azole group; however, among patients receiving LTV, those with government insurance were more likely to fall into the Extensive barrier category. Extensive insurance barriers impacted 17% (49/287) of patients prescribed high-cost azoles. Among these patients, 31% had projected costs exceeding $1,000, 78% required financial assistance (20% copay assistance card, 12% drug manufacturer assistance, 62% internal financial assistance), and 80% required more than 60 minutes of PharmD time to coordinate financial resources to make care affordable, compared to just 1% of patients with minimal/no barriers. Among patients prescribed LTV, one-third (63/191, 33%) faced extensive insurance barriers with 57% of these patients having projected costs exceeding $1,000, 98% required financial assistance (34% copay assistance card, 45% manufacturer assistance, 21% internal financial assistance), and 68% required more than 60 minutes of PharmD time, compared to 8% of patients with minimal/no barriers. For both the azole and LTV groups, by univariable analysis, insurance type, ancestry/ethnicity, and stem cell source were not ","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144620742","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":"MOLD INVASIVE FUNGAL INFECTION IN HEMATOPOIETIC CELL TRANSPLANT WITH POST-TRANSPLANT CYCLOPHOSPHAMIDE AND POSACONAZOLE PROPHYLAXIS.","authors":"Pedro Chorão, Pablo Granados, Pilar Calvillo-Batllés, Pedro Asensi, Marta Villalba, Aitana Balaguer-Roselló, Santiago de Cossio, Eva María González, María Dolores Gómez, Inés Gómez, Pilar Solves, Christian Tejada, Alberto Louro, Aurora Perla, Miguel Salavert, Javier de la Rubia, Miguel A Sanz, Jaime Sanz, Juan Montoro","doi":"10.1016/j.jtct.2025.07.005","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.07.005","url":null,"abstract":"<p><strong>Background: </strong>Post-transplantation cyclophosphamide (PTCy) graft-versus-host disease (GVHD) prophylaxis regimens may increase the incidence of mold invasive fungal infections (IFI). Nevertheless, data on prophylaxis strategies and donor modalities are heterogenous and reports on posaconazole prophylaxis remain limited.</p><p><strong>Objectives: </strong>Characterize mold IFI incidence, risk factors and impact on outcomes in patients with hematologic malignancies undergoing PTCy-based HCT from matched sibling, matched unrelated, and haploidentical donors under posaconazole prophylaxis.</p><p><strong>Study design: </strong>Single-center retrospective study of mold IFI episodes in 435 patients undergoing HCT at a single institution. Daily 300 mg posaconazole dosage was given between days +7 and +90 or during GVHD on steroids.</p><p><strong>Results: </strong>Mold IFI affected 6% of patients (16 possible, eight probable, and two proven), with 73% breakthrough infections. Only Aspergillus spp. was identified. With a median onset of 88 days, the two-year cumulative incidence was 5.6% for overall and 2.1% for probable/proven mold IFI. Grade II-IV acute GVHD and higher HCT comorbidity index increased the risk for probable/proven mold IFI. One patient died from mold IFI, although 7% of deaths occurred with active mold IFI. Probable/proven mold IFI negatively impacted on the overall survival (OS).</p><p><strong>Conclusions: </strong>Mold IFI is an infrequent complication in allogeneic HCT with PTCy under posaconazole prophylaxis, although acute GVHD significantly increases its risk. While direct mortality is low, OS is negatively impacted by probable/proven mold IFI.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144620656","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":"Corrigendum to: \"Combining three different pre-transplant scores improves predictive value in patients after haploidentical stem cell transplantation with TBF conditioning and PTCy\" Journal: Transplant Cell Ther. 2021 Jul;27(7):614.e1-614.e8 doi: 10.1016/j.jtct.2021.03.021.","authors":"Albert Esquirol, Maria Jesus Pascual, Irene Garcia-Cadenas, Beatriz Herruzo, Christelle Ferrà, Ariadna Pérez, Alberto Torio, Anna Torrent, Marian Cuesta, Rodrigo Martino, Jorge Sierra","doi":"10.1016/j.jtct.2025.06.010","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.010","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144584965","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":"Incidence and Risk Factors of Skin Cancer and Preneoplastic Lesions after Autologous and Allogeneic Hematopoietic Cell Transplantation.","authors":"Miguel Mansilla-Polo, Juan Montoro, Javier López-Davia, Aitana Balaguer-Rosello, Marta Villalba-Montaner, Pedro Chorão, Pedro Asensi, Javier De la Rubia, Blanca de Unamuno-Bustos, Daniel Martín-Torregrosa, Carlos Abril-Pérez, Vicent Martínez-Cózar, Margarita Llavador-Ros, Miguel Ángel Sanz, Rafael Botella-Estrada, Jaime Sanz","doi":"10.1016/j.jtct.2025.06.031","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.06.031","url":null,"abstract":"<p><strong>Background: </strong>Although an increased risk of skin cancer has been documented in recipients of allogeneic hematopoietic stem cell transplantation (HCT), there is still a paucity of information regarding its specific risk factors in the contemporaneous era, as well as its frequency after autologous HCT.</p><p><strong>Objectives: </strong>The objective of this single-center study was to analyze separately the incidence, characteristics, and risk factors of skin cancer and its precursor actinic keratosis (AK) in a large contemporary cohort of allogeneic and autologous HCT recipients during a study period spanning from 2007 to 2023.</p><p><strong>Study design: </strong>This observational, unicentric cohort study included all patients who underwent autologous or allogeneic HCT at a tertiary hospital.</p><p><strong>Results: </strong>The study included 2,042 patients, 1,182 allogeneic and 860 autologous. The median follow-up for surviving patients was 61 months (interquartile range, 25-91). Sixty-seven skin cancers were recorded, with a median interval to diagnosis of 35.4 months. The most common cancer lesions detected were basal cell carcinoma (51%) followed by squamous cell carcinoma (31%), and melanoma (13%). The cumulative incidence of skin cancer at 6 years was 4% (95% CI, 2.9 - 5.3), being 6.1% (95% CI, 3.5 - 9.6) in autologous and 3.2% (95% CI, 2.2 - 4.6) in allogeneic HCT recipients (p < 0.001). Increased recipient age (hazard ratio [HR] 1.6, 95% CI 1.3-2.0, p < 0.001), male sex (HR 2.2, 95% CI 1.4-3.6, p = 0.001), and a history of prior skin cancer (HR 6.3, 95% CI 3.3-12.2, p <0.001) were identified as independent risk factors for skin cancer. Additionally, for allogeneic HCT, graft-versus-host disease (GVHD) with mucocutaneous involvement showed higher risk (HR = 3.21, 95% CI = 1.76-5.86, p<0.001). Voriconazole use was specifically associated with invasive squamous cell carcinomas (SCC) (p=0.007). Twenty-nine additional cases developed AK with a median interval to diagnosis of 31 months following HCT. The 6-year cumulative incidence of AK was 2.2% (95% CI, 2.9 - 5.3).</p><p><strong>Conclusions: </strong>Both autologous and allogeneic HCT recipients are at risk of skin cancer, especially those with older age, male sex, chronic GVHD with mucocutaneous involvement, and pretransplant history of skin cancer. Voriconazole was particularly associated to higher invasive SCC rates. These findings underscore the need for ongoing vigilance and implementation of risk-based screening and prevention practices.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144576341","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":"Allogeneic Hematopoietic Cell Donor Selection: Contemporary Guidelines from the NMDP/CIBMTR.","authors":"Antonio M Jimenez Jimenez, Stephen R Spellman, Ioannis Politikos, Shannon R McCurdy, Steven M Devine, Monzr M Al Malki, Yung-Tsi Bolon, Stephanie J Lee, Jason Dehn, Joseph Pidala, Martin Maiers, Medhat Askar, Craig Malmberg, Jeffery J Auletta, Heather Stefanski, Larisa Broglie, Muna Qayed, Mitchell Horwitz, Jennifer S Wilder, Mahasweta Gooptu, Rohtesh S Mehta, Marcelo Fernandez-Viña, Bronwen E Shaw, Brian C Shaffer","doi":"10.1016/j.jtct.2025.07.004","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.07.004","url":null,"abstract":"<p><p>Allogeneic hematopoietic cell transplantation (HCT) remains a curative therapy for many patients with hematologic malignancies, bone marrow failure syndromes, inborn errors of immunity and metabolic disorders. Current donor selection strategies typically prioritize the selection of an HLA-matched donor over HLA mismatched (\"alternative\") donor sources, with a hierarchical approach to the donor search. More recent data challenge this rubric, particularly in the context of novel graft versus host disease (GVHD) prophylaxis strategies that demonstrate improved outcomes in alternative donor HCT recipients. In this setting, an increased emphasis on non-HLA factors (both donor characteristics and systemic factors) in determining donor selection is now feasible. In this guideline, we review recent evidence from prospective clinical trials as well as high-quality observational studies and provide expert panel recommendations on donor selection algorithms and prioritization in the era of novel GVHD prophylaxis. We then highlight important questions still to be answered in our field.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144576340","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":"Corrigendum to 'Clinical Practice Recommendations for Hematopoietic Cell Transplantation.' Transplant Cell Ther. 2024 Sep;30(9):832-843.","authors":"Madiha Iqbal, Ambuj Kumar, Peter Dreger, Julio Chavez, Craig S Sauter, Anna M Sureda, Veronika Bachanova, Richard T Maziarz, Martin Dreyling, Sonali M Smith, Caron Jacobson, Bertram Glass, Carla Casulo, Olalekan O Oluwole, Silvia Montoto, Ranjana Advani, Jonathon Cohen, Gilles Salles, Nada Hamad, John Kuruvilla, Brad S Kahl, Mazyar Shadman, Abraham S Kanate, Lihua Elizabeth Budde, Manali Kamdar, Christopher Flowers, Mehdi Hamadani, Mohamed A Kharfan-Dabaja","doi":"10.1016/j.jtct.2025.05.022","DOIUrl":"10.1016/j.jtct.2025.05.022","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144486002","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":"Comparative Study of Bu/Cy/ATG and Flu/Cy/ATG Conditioning Regimens for Unrelated Donor Hematopoietic Stem Cell Transplantation in Severe Aplastic Anemia.","authors":"Liangliang Wu, Xiaowei Chen, Ming Zhou, Wenjian Mo, Ruiqing Zhou, Yumiao Li, Shilin Xu, Caixia Wang, Shiyi Pan, Wei Zhou, Tingfen Deng, Yuling Zhang, Yuping Zhang, Shunqing Wang","doi":"10.1016/j.jtct.2025.06.014","DOIUrl":"10.1016/j.jtct.2025.06.014","url":null,"abstract":"<p><p>Unrelated donor hematopoietic stem cell transplantation (URD-HSCT) is a curative option for severe aplastic anemia (SAA), but the optimal conditioning regimen remains unclear. This retrospective study compares Busulfan/Cyclophosphamide/Anti-thymocyte globulin (Bu/Cy/ATG) and Fludarabine/Cyclophosphamide/Anti-thymocyte globulin (Flu/Cy/ATG) protocols to identify the best regimen for SAA patients. We retrospectively analyzed the clinical outcomes of 107 SAA patients who underwent URD-HSCT with Flu/Cy/ATG (n = 63) or Bu/Cy/ATG (n = 44) between November 2012 and December 2022. No significant differences were observed in the cumulative incidence of neutrophil/platelet engraftment, graft failure, graft-versus-host disease (GVHD), or CMV viremia. Overall survival (OS) at 7 years was 95.5% (95% CI: 89.5-100) with Bu/Cy/ATG vs. 85.5% (95% CI: 77.1-94.7) with Flu/Cy/ATG, and failure-free survival (FFS) at 7 years was 95.5% (95% CI: 89.5-100) vs. 83.9% (95% CI: 75.2-93.6). Multivariate analysis identified Bu/Cy/ATG protocol as favorable for OS (Hazard ratio, HR 0.122, 95% CI: 0.021-0.715, P = .020) and FFS (HR 0.090, 95% CI: 0.015-0.538, P = .008). Moreover, multivariate analysis confirmed that the Bu/Cy/ATG regimen significantly reduced the risk of EBV viremia (Relative risk, RR 0.175, 95% CI: 0.026-0.717, P = .032) and post-transplant lymphoproliferative disorder (RR 0.031, 95% CI: 0-0.536, P = .012). Subgroup analysis through multivariate modeling further demonstrated that the Bu/Cy/ATG regimen demonstrated superior OS, FFS and EBV infection outcomes in patients older than 30 years. The Bu/Cy/ATG regimen, compared to Flu/Cy/ATG protocol, offers superior outcomes, including improved OS/FFS and reduced EBV infection, suggesting it may be the preferred choice for SAA patients undergoing URD-HSCT, especially for patients older than 30 years. Larger cohorts and prospective trials are needed to validate these findings.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144295007","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}