Transplantation and Cellular Therapy最新文献

{"title":"Herombopag Versus rhTPO in Stem Cell Transplantation: A Comparative Study of Efficacy, Safety, and Cost-Effectiveness.","authors":"Fenglian Luo, Zhiming Luo, Yun Luo, Hanqing Zeng, Ying Chen, Chun Cao","doi":"10.1016/j.jtct.2025.06.023","DOIUrl":"10.1016/j.jtct.2025.06.023","url":null,"abstract":"<p><strong>Background: </strong>Thrombocytopenia remains a significant clinical challenge following peripheral blood stem cell transplantation (PBSCT), with delayed platelet engraftment (DPE) associated with increased morbidity and mortality. While current strategies including platelet transfusions and recombinant human thrombopoietin (rhTPO) are widely used, their clinical utility is constrained by suboptimal efficacy, high costs, and administration challenges. Herombopag, a novel second-generation thrombopoietin receptor agonist (TPO-RA), presents potential advantages due to its oral bioavailability and unique mechanism of action. This retrospective study aimed to comprehensively compare herombopag with rhTPO in terms of hematopoietic recovery, safety profile, and economic impact in both autologous (auto-) and allogeneic (allo-) PBSCT settings.</p><p><strong>Methods: </strong>We conducted a retrospective cohort analysis of 106 consecutive PBSCT patients (56 auto-PBSCT, 50 allo-PBSCT) treated between January 2022 and October 2024. Patients were stratified into two intervention groups: oral herombopag (5 mg/day) versus subcutaneous rhTPO (15,000 U/day), initiated when platelet counts dropped below 30 × 10⁹/L. Primary endpoints included time to neutrophil and platelet engraftment, while secondary endpoints encompassed bleeding events, transfusion requirements, infectious complications (CMV, EBV, BKV), GVHD incidence, drug-related toxicities, and overall treatment costs. Rigorous statistical analyses were performed using SPSS v26.0, employing appropriate parametric and nonparametric tests with statistical significance set at P < .05.</p><p><strong>Results: </strong>Engraftment Kinetics: In auto-PBSCT, comparable results were observed between groups for both neutrophil (herombopag: 12.25 ± 1.30 days versus rhTPO: 12.43 ± 1.35 days; P = .615) and platelet engraftment (13.32 ± 1.31 versus 13.61 ± 1.23 days; P = .403). Allo-PBSCT patients receiving herombopag demonstrated significantly faster hematopoietic recovery: neutrophil engraftment (12.76 ± 1.81 versus 13.80 ± 1.71 days; P = .042) and platelet engraftment (15.76 ± 2.59 versus 17.72 ± 2.81 days; P = .013).</p><p><strong>Clinical outcomes: </strong>Bleeding events were markedly reduced with herombopag in both auto-PBSCT (7.1% versus 42.9%; P = .005) and allo-PBSCT (8.0% versus 40.0%; P = .020) cohorts. Platelet transfusion requirements were significantly lower in herombopag-treated patients (auto-PBSCT: 1.11 ± 1.10 versus 2.04 ± 1.07 units, P = .002; allo-PBSCT: 1.64 ± 1.85 versus 3.56 ± 1.96 units, P = .001).</p><p><strong>Safety profile: </strong>Comparable rates of viral reactivation (CMV, EBV, BKV), GVHD, and manageable liver function abnormalities were observed between groups. No thrombotic complications or significant survival differences were noted at 2-year follow-up (OS/PFS, P>0.05).</p><p><strong>Economic evaluation: </strong>Herombopag demonstrated substantial cost savings, reducing med","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529718","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":"Improving Patient Access through Value-Based CAR T Delivery: Examples Using a Micro-costing Tool.","authors":"Mona Shafey, Olivia Dickinson, Helen Lilley, Brett Doble","doi":"10.1016/j.jtct.2025.06.028","DOIUrl":"10.1016/j.jtct.2025.06.028","url":null,"abstract":"<p><p>The true costs of chimeric antigen receptor T-cell (CAR T) delivery (i.e., all costs except acquisition costs) remain uncertain, with little guidance available to guide accurate estimation. Providers therefore may face challenges in estimating the costs of establishing or expanding CAR T services, particularly if they have limited business expertise or CAR T experience. This risks over- or under-estimation of CAR T delivery costs, with potential for inadequate provider reimbursement, resource challenges, and restricted patient access. Accurate estimation of CAR T delivery costs is an essential component of ensuring value-based healthcare that achieves the best possible patient outcomes, while also ensuring that providers are adequately compensated for service delivery. The purpose of this paper is to demonstrate how value-based CAR T service delivery can be facilitated using an Excel-based micro-costing framework (\"tool\") to derive accurate and transparent cost estimates that consider the full patient pathway from initial assessment through to 100 days post CAR T infusion. Micro-costing can be used to support robust CAR T business case development and ensure all individual components of the treatment pathway have been accounted for. The tool's flexibility allows providers to model different future scenarios (e.g., changes in the case mix) and test the impact of resource changes (e.g., adjusting staff experience levels) to make informed resourcing decisions. This facilitates identification of opportunities for efficiencies, resource reallocation, and service improvements, and supports providers in delivering CAR T therapy to as many eligible patients as possible. A clear and detailed understanding of CAR T delivery costs can also support validation of reimbursement levels, such as CAR T tariffs, mitigating the risk of provider underpayment for services. The publicly available micro-costing tool is the first to support costing across the full CAR T treatment pathway from initial patient assessment to long-term follow-up. It has the potential to transform provider approaches to CAR T program development, reducing reliance on assumptive costs and uncertainty, and instead offering an evidence-driven approach. Overall, the tool supports value-based and sustainable CAR T therapy delivery, with the aim of ensuring adequate reimbursement for providers while facilitating patient access to this potentially life-saving therapy.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529719","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 #11: Updated Cytomegalovirus Guidelines in Hematopoietic Cell Transplant and Cellular Therapy Recipients.","authors":"Fareed Khawaja, Danniel Zamora, Michelle K Yong, Morgan Hakki, Breana K Goscicki, Lara Danziger-Isakov, Andrew Lin, Paul A Carpenter, Michael Boeckh, Genovefa A Papanicolaou, Sanjeet S Dadwal, Roy F Chemaly","doi":"10.1016/j.jtct.2025.06.025","DOIUrl":"10.1016/j.jtct.2025.06.025","url":null,"abstract":"<p><p>The Practice Guidelines Committee of the American Society of Transplantation and Cellular Therapy partnered with the society's Transplant Infectious Diseases Special Interest Group to update its previous infectious disease guidelines for the prevention and management of cytomegalovirus (CMV) infection and disease following hematopoietic cell transplantation (HCT). The two updates, published in 2021, focused on the prevention and management of CMV infection, respectively, including refractory and resistant CMV infections. To best serve clinical providers, each standalone topic in the infectious diseases series has been published in a concise format of frequently asked questions (FAQs). Adult and pediatric infectious diseases and HCT content experts developed the FAQs and the answers; recommendations were graded according to their strength (A-E) and the level of the supporting evidence (I-III). Several advances in CMV prevention and management since 2021 warranted an update to the original third and fourth topics in the series. This eleventh topic in the series focuses on new antiviral treatments for CMV, expanded indications of existing antiviral therapy for the prevention of CMV, and the treatment of CMV in special populations such as CAR T-cell therapy recipients and pediatric transplant recipients.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144529717","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 Haploidentical Stem Cell Transplantation for Acute Lymphoblastic Leukemia Patients: A Single-Center Experience in Mexico.","authors":"Ángel Carrillo Jurado, Alberto Olaya Vargas, Yadira Berenice Melchor Vidal, Carmen Torres Alarcón, Carolina García Castillo, Gilberto Barranco Lampón","doi":"10.1016/j.jtct.2025.06.027","DOIUrl":"10.1016/j.jtct.2025.06.027","url":null,"abstract":"<p><p>Access to allogeneic hematopoietic stem cell transplantation (allo-HSCT) for acute lymphoblastic leukemia (ALL) remains limited in low- and middle-income countries (LMICs). Haploidentical stem cell transplantation (haplo-SCT) with post-transplant cyclophosphamide (PTCy) has expanded donor availability, but data from Latin America are scarce. To evaluate clinical outcomes of haplo-SCT in pediatric and adult ALL patients in a single center in Mexico using a standardized protocol. This retrospective observational study included 28 MRD-negative patients with ALL who received haplo-SCT at Centro Médico ABC between 2019 and 2023. Conditioning regimens include total body irradiation, cyclophosphamide, and etoposide. PTCy-based GVHD prophylaxis was used in all cases. Outcomes included overall survival (OS), event-free survival (EFS), GVHD incidence, and transplant-related complications. The median age was 9 yr (range: 1 to 55); 82% were pediatric and 18% were adult. Blinatumomab was used in 36% of patients prior to transplant; all Ph+ cases received tyrosine kinase inhibitors. The 60-month OS was 64.1% (95% CI, 53.8% to 74.4%) and EFS was 55.6% (95% CI, 44.6% to 66.6%). Neutrophil engraftment occurred in 96.4% of patients. Acute GVHD occurred in 54% and chronic GVHD in 32%. Nonrelapse mortality at 100 d was 7%. No cases of cytokine release syndrome were observed. This study demonstrates that haplo-SCT using modern protocols yields favorable outcomes in a resource-limited Mexican setting, supporting its broader adoption in LMICs. Long-term survival was comparable to outcomes reported in high-income countries, highlighting the feasibility and scalability of this strategy.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":4.4,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144512537","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, Risk Factors, and Outcomes of Bloodstream Infection during Conditioning Phase before Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Ling Pan, Jia Li, Qingsong Lin, Xiaomeng Feng, Xueyuan Li, Guixin Zhang, Sisi Zhen, Yuqing Cui, Jieru Wang, Yuping Fan, Tingting Zhang, Yigeng Cao, Wenbin Cao, Aiming Pang, Donglin Yang, Xin Chen, Rongli Zhang, Jialin Wei, Qiaoling Ma, Weihua Zhai, Yi He, Mingzhe Han, Erlie Jiang, Sizhou Feng","doi":"10.1016/j.jtct.2025.05.027","DOIUrl":"10.1016/j.jtct.2025.05.027","url":null,"abstract":"<p><p>Bloodstream infections (BSI) pose critical risks during allogeneic hematopoietic stem cell transplantation (allo-HSCT), yet data on BSI during the conditioning phase remain limited. To assess the characteristics, risk factors, and outcomes of BSI during conditioning in allo-HSCT patients with hematological diseases. We conducted a single-center retrospective cohort study including 2395 consecutive allo-HSCT recipients between September 2013 and September 2023. BSI occurred in 113 patients (5.7% cumulative incidence), with median onset on day -2. Monomicrobial Gram-negative bacteria (73.5%, 83/113) and Gram-positive bacteria (18.6%, 21/113) predominated, with polymicrobial BSI in 8.0% (9/113). Carbapenem-resistant Gram-negative organisms (CRO) constituted 15.9% (18/113) of infections. Independent BSI risk factors included antithymocyte globulin (ATG)-mediated conditioning (96.5% of BSI cases), aplastic anemia (AA), hematopoietic cell transplantation-comorbidity index (HCT-CI) ≥2, and preconditioning neutropenia ≥7 days. Patients were stratified by risk based on cumulative incidence: low (0 factor ± ATG, 3.3%), intermediate (ATG + 1 factor, 9.3%), and high (ATG + ≥ 2 factors, 21.4%) (P < .001). BSI significantly reduced 28-day survival (94.7% versus 99.7%, P < .001), with 5.3% mortality (6/113). CRO BSI exhibited lower survival than non-CRO cases (76.5% versus 97.9%, P < .001). Prior CRO colonization independently predicted CRO BSI (P = .003). Appropriate empirical therapy and ceftazidime/avibactam (CAZ-AVI)-based definitive regimens improved early survival. BSI surveillance should prioritize patients undergoing ATG-based conditioning, particularly those with AA, HCT-CI ≥ 2, or preconditioning neutropenia ≥7 days. Given the dominant mortality risk of CRO BSI during conditioning, pre-HSCT CRO screening is imperative, and targeted therapies such as CAZ-AVI are critical.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144512536","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":"High-Dose Melphalan Followed by Busulfan and Fludarabine Conditioning Prior to Allogeneic Stem Cell Transplantation in Elderly Patients with Active Acute Myeloid Leukemia-A Retrospective Single-Center Study.","authors":"Julian Ronnacker, Marc-Andre Urbahn, Christian Reicherts, Simon Call, Eva Eßeling, Mathias Floeth, Julia Marx, Jörn Albring, Jan-Henrik Mikesch, Christoph Schliemann, Georg Lenz, Matthias Stelljes","doi":"10.1016/j.jtct.2025.06.019","DOIUrl":"10.1016/j.jtct.2025.06.019","url":null,"abstract":"<p><p>In patients with relapsed or refractory (r/r) acute myeloid leukemia (AML), long-term survival in most cases can only be achieved with allogeneic hematopoietic stem cell transplantation. The introduction of sequential conditioning regimens has contributed to the steadily improving prognosis of r/r AML patients. As most studies mainly included younger patients, feasibility of sequential conditioning in elderly or comorbid patients remains subject of debate. We retrospectively investigated outcomes of 103 patients aged ≥55 years with active r/r AML receiving sequential melphalan-based conditioning followed by fludarabine and busulfan at our center from 2014 to 2022. The median (range) age in our cohort was 67 (55 to 76) years. Thirty-one percent of the patients had hematopoietic cell transplantation comorbidity index scores >3 points. Overall survival (OS) and relapse-free survival estimates at 3 years were 44% (95% confidence interval [CI], 35% to 55%) and 40% (95% CI, 31% to 51%), respectively. Cumulative incidences of relapse and nonrelapse mortality (NRM) at 3 years were 28% (95% CI, 19% to 37%) and 32% (95% CI, 23% to 41%), respectively. Transplantation from a mismatched donor was identified as major risk factor for OS (adjusted hazard ratio [HR_adj] 3.03; 95% CI, 1.72 to 5.35; P < .001) and NRM (HR_adj 2.86; 95% CI, 1.38 to 5.93; P = .005). A high leukemic burden before conditioning (20% to 49% versus 5% to 19% bone marrow blasts, HR_adj 3.00; 95% CI, 1.10 to 8.18; P = .032) was associated with an increased relapse risk. In summary, our data suggest that sequential conditioning with melphalan followed by busulfan and fludarabine is effective and tolerable in elderly patients with active AML, allowing for 3-year OS of >40%. At the same time, our data highlight the need to refine treatment strategies for elderly patients with active AML who receive transplants from <10/10 matched donors, or patients with high leukemic burden.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144508399","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":"Real-World Treatment Patterns and Out-of-Pocket Costs After CAR T-Cell Therapy in Commercially Insured Patients With B-Cell Non-Hodgkin Lymphoma in the United States.","authors":"Mohammed Zuber, Shaimaa Elshafie, Shifa Taj, Lorenzo Villa Zapata","doi":"10.1016/j.jtct.2025.06.018","DOIUrl":"10.1016/j.jtct.2025.06.018","url":null,"abstract":"<p><p>CAR T-cell therapy has transformed the treatment of B-cell non-Hodgkin Lymphoma (NHL), yet treatment failure remains a major concern. Clinical trial data indicate relapse rates up to 50% within 6 months, but real-world evidence on salvage therapy patterns and financial burdens is limited. This study aims to assess the risk of CAR T failure risk, characterize subsequent therapeutic strategies, and quantify patient out-of-pocket (OOP) health care costs. We conducted a retrospective cohort study using the Merative MarketScan database (2017-2022), identifying adult patients with diffuse large B-cell lymphoma (DLBCL), follicular lymphoma (FL), mantle cell lymphoma (MCL), or primary mediastinal large B-cell lymphoma (PMBCL) who received CAR-T therapy. Additional therapy post-index indicated relapse, with cumulative risk estimated via Kaplan-Meier analysis. OOP costs, including copayments, coinsurance, and deductibles, were calculated. Among 224 eligible patients (median age: 57 years, 70% male, 83% DLBCL), 85 (38%) initiated subsequent therapy, with a cumulative failure risk of 36% at 6 months and 48% at 12 months. Lenalidomide was the most common salvage therapy in DLBCL. Six-month total OOP costs distributed across 121 patients were $273,676, with outpatient claims comprising 67%. Patients requiring additional therapy had higher mean OOP costs ($3,221 versus $1,806), with some reaching $38,889. This study underscores the persistent need for effective salvage therapy options and the financial burden of treatment failure. As CAR T-cell therapy is increasingly utilized in earlier treatment lines, future research should focus on optimizing post-CAR T-cell management and mitigating financial toxicity.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144498097","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":"The Caregiver Paradigm in Hematopoietic Cell Transplant: Current and Future Directions.","authors":"Ben Tweeten, Jill Randall, Anna Barata, Nandita Khera, Melody A Griffith, Anna M DeSalvo, Katie Schoeppner, Jaime M Preussler","doi":"10.1016/j.jtct.2025.06.022","DOIUrl":"10.1016/j.jtct.2025.06.022","url":null,"abstract":"<p><p>Many transplant centers (TCs) require a patient to have a caregiver 24 hours a day, 7 days a week for a minimum timeframe to proceed with an allogeneic hematopoietic cell transplant (alloHCT). However, this requirement varies across TCs, with timing of requirements varying from no requirements up to 180 days, and there is inconclusive evidence to support the need for this requirement. The current caregiver requirement paradigm can limit access for many patients, besides causing a significant physical, emotional, and financial burden on caregivers. This article reviews literature on the current alloHCT caregiver paradigm and identifies barriers to change. Lastly, it highlights alternative caregiving models that are currently being implemented, as well as opportunities for future directions to improve access, including the need for research on interventions such as remote monitoring, community partnerships, policy changes, and enhanced screening. There is a need for evidence-based patient-centered care models to transform the caregiver paradigm to ensure that all patients can receive the treatment they need, irrespective of whether they have a caregiver. The future of post-alloHCT care demands a shift towards patient-centered, flexible caregiving models that accommodate the diverse needs and circumstances of patients. Such evidence-based changes in the paradigm can help improve access to, and outcomes for, alloHCT.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144369251","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":"Assessing physical function in transplantation and CAR-T recipients: expert recommendations from the survivorship, aging and biobehavioral special interest groups of ASTCT.","authors":"Shatha Farhan, Vanessa E Kennedy, Manuel R Espinoza-Gutarra, Hannah Lust, Maria Silvina Odstrcil Bobillo, Adam Yuh Lin, Rebecca L Olin, Richard J Lin, Kelly E Rentscher, Mallory R Taylor, Lathika Mohanraj, William A Wood, Hemant S Murthy, Nuasheen Ahmed, Amylou C Dueck, Rachel Phelan, Debra Lynch Kelly, Carrie Yuen, Pashna N Munshi, Hélène Schoemans, Betty K Hamilton, Catherine Lee, Anthony D Sung","doi":"10.1016/j.jtct.2025.06.017","DOIUrl":"10.1016/j.jtct.2025.06.017","url":null,"abstract":"<p><p>The past few decades have witnessed significant advancements in stem cell transplantation and cell therapy (TCT). This has allowed their expanded use in older patients and those with comorbidities with favorable outcomes. However, these procedures carry significant risks, such as graft-versus-host disease, infection, cytokine release syndrome, and immune effector cell-associated neurotoxicity. Therefore, physical function assessment is crucial to assess patient fitness and potential optimization before and after TCT. The existence of diverse assessment tools makes implementation, comparison, and sharing knowledge among centers difficult. This paper proposes a tiered approach aiming to harmonize physical assessment in the TCT. This allows healthcare facilities to prioritize recommended assessments based on their current capabilities and resources. TCT patients should receive comprehensive physical assessments pre- and post-TCT using a combination of both patient-reported and objective measures. For patient-reported measures, the Patient-Reported Outcomes Measurement Information System should be considered. For objective measures, we recommend considering a physical performance assessment (eg, gait speed) or muscle strength assessment (eg, hand grip), if feasible. Albumin and C reactive protein are also informative in predicting the risk of nonrelapse mortality. Other composite tools, questionnaire libraries, biomarkers, imaging, and wearables can be added according to research and clinical needs. A care workflow needs to be in place in case any impairment is found during the evaluation with goals of increasing physiologic reserve and mitigating stressors. This tiered approach will increase awareness and adoption of these tools and hence improve patient care, facilitate data sharing, and enhance collaboration in this field.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144369250","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":"Inflammatory Markers and Microbiome Dysbiosis in Hematopoietic Cell Transplant Recipients with Lung Graft-versus-Host Disease.","authors":"Victoria Brehm, Ziyi Wang, Luis Rocha, Breanna Jones, Robert R Jenq, Chia-Chi Chang, Guang-Shing Cheng, Joe Hsu, Husham Sharifi, Gregory Yanik, Luis Luna, Anum Waqar, Jhankruti Zaveri, Burton F Dickey, Lara Bashoura, Elizabeth J Shpall, Matt Zinter, David O'Dwyer, Richard E Champlin, George Chen, Amin Alousi, Sophie Paczesny, Christine B Peterson, Ajay Sheshadri","doi":"10.1016/j.jtct.2025.06.020","DOIUrl":"10.1016/j.jtct.2025.06.020","url":null,"abstract":"<p><p>Bronchiolitis obliterans (BOS) is a manifestation of pulmonary chronic graft-versus-host disease (cGVHD) and is a devastating complication of allogeneic hematopoietic stem cell transplantation (HCT). Early detection and treatment of BOS may improve outcomes, but biomarkers that accurately identify BOS early are lacking. We aimed to determine whether certain validated cGVHD markers could also accurately diagnose BOS as compared with patients without BOS and with or without extrapulmonary cGVHD. In addition, we sought to determine whether dysbiosis of the gut or oral microbiomes was associated with BOS or with inflammatory biomarkers. We enrolled 43 recipients of allogeneic HCT, 16 of whom had BOS. For each patient, we obtained pulmonary function tests, measured the levels of 9 serum biomarkers utilizing enzyme-linked immunosorbent assays, and analyzed both the oral and gut microbiome using microbial DNA amplification and sequencing. We compared biomarker levels to lung function, both at baseline and over time, as well as to microbiome diversity. Higher IL1RL1 (P = .002) and IL-17 (P = .041) at enrollment were negatively correlated with FEV1% (forced expiratory volume in 1 second) lung function over time. Increases in IL1RL1 (P = .035), IL-17 (P = .009), and WFDC2 (P = .045) levels over time were associated with worsened lung function/FEV1% over time. There were minimal correlations between gut microbiome diversity and lung function or serum biomarkers. Oral microbiome alpha diversity was lower in subjects with BOS than without (P = .00057), and oral beta diversity was associated with FEV1% and with levels of several biomarkers. Our pilot study suggests that certain serum cGVHD markers may identify recipients of allogeneic HCT at higher risk for pulmonary impairment over time and that these markers should be followed with robust, controlled studies.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-06-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144340431","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}