Transplantation and Cellular Therapy最新文献

{"title":"Officers and Directors of ASTCT","authors":"","doi":"10.1016/S2666-6367(25)01237-0","DOIUrl":"10.1016/S2666-6367(25)01237-0","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Page A6"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144548738","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":"Semper Progrediens","authors":"Effie W. Petersdorf","doi":"10.1016/j.jtct.2025.06.005","DOIUrl":"10.1016/j.jtct.2025.06.005","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 399-400"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144549483","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":"Umbilical Cord Blood: Expanding Transplant Options","authors":"Amy K. Keating","doi":"10.1016/j.jtct.2025.06.008","DOIUrl":"10.1016/j.jtct.2025.06.008","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 405-406"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144548740","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":"Recent Advances in the Prognosis of HHV-6 Encephalitis Following Allogeneic Hematopoietic Stem Cell Transplantation","authors":"Kazuya Kurihara ,&nbsp;Daichi Sadato ,&nbsp;Takashi Toya ,&nbsp;Chizuko Hirama ,&nbsp;Kana Kato ,&nbsp;Kaori Kondo ,&nbsp;Yasutaka Sadaga ,&nbsp;Chika Kato ,&nbsp;Masashi Shimabukuro ,&nbsp;Atsushi Jinguji ,&nbsp;Fumihiko Ouchi ,&nbsp;Hiroaki Shimizu ,&nbsp;Yuho Najima ,&nbsp;Yuka Harada ,&nbsp;Noriko Doki","doi":"10.1016/j.jtct.2025.04.016","DOIUrl":"10.1016/j.jtct.2025.04.016","url":null,"abstract":"<div><div>Human herpesvirus-6 (HHV-6) encephalitis is a rare but fatal complication following allogeneic hematopoietic stem cell transplantation (allo-HSCT). Despite advancements in transplantation outcomes and supportive care, knowledge regarding the clinical features and prognostic factors of HHV-6 encephalitis remains limited. This study aims to clarify the clinical characteristics of HHV-6 encephalitis in allo-HSCT recipients and to identify prognostic factors. This is a single-center retrospective study that analyzed the patients with HHV-6 encephalitis who underwent allo-HSCT at our institute between 2004 and 2023. The diagnosis of HHV-6 encephalitis was confirmed by the presence of neurological symptoms and the detection of HHV-6 DNA in the cerebrospinal fluid using real-time polymerase chain reaction. Fifty-three patients were included in this study. The median time from allo-HSCT to HHV-6 encephalitis onset was 24 days (range: 3 to 189). Of the 53 patients, 38 (71.7%) received systemic steroids, with a median interval of 11 days (range: 2 to 46) from steroid initiation to encephalitis onset. One-year overall survival and non-relapse mortality (NRM) after encephalitis diagnosis were 33.5% and 46.5%, respectively. While HHV-6 encephalitis directly caused one death, infections unrelated to HHV-6 were the leading cause of mortality (47.5%). The interval from the onset to the initiation of antiviral therapy was significantly shorter in recent cases (0.0 after 2018 versus 1.5 days before 2017: <em>P</em> = .0086), and 1-year NRM was significantly lower (26.8 versus 62.1%; <em>P</em> = .024). Multivariate analysis revealed that allo-HSCT before 2017 (hazard ratio [HR] 3.13, <em>P</em> = .012) and haploidentical transplantation (HR 3.07, <em>P</em> = .001) were independent prognostic factors for NRM. Among the 32 patients who survived for over 100 days after the initiation of HHV-6 encephalitis treatment, neurological sequelae persisted in 16 (50.0%) cases, including short-term memory impairment in 11. Overall, our study indicates that recent improvements in HHV-6 encephalitis outcomes after allo-HSCT likely result from earlier initiation of antiviral treatment.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 463.e1-463.e12"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144014937","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":"Out-of-Pocket Expenditures and Financial Hardship Among Patients With Myelodysplastic Syndrome Undergoing Allogeneic Transplant or Hypomethylating Agent / Supportive Care (BMT CTN 1102)","authors":"Christopher T. Su ,&nbsp;Wael Saber ,&nbsp;Aasthaa Bansal ,&nbsp;Li Li ,&nbsp;Ryotaro Nakamura ,&nbsp;Corey Cutler ,&nbsp;Joshua A. Roth ,&nbsp;Winona Wright ,&nbsp;Lotte Steuten ,&nbsp;Scott D. Ramsey","doi":"10.1016/j.jtct.2025.04.015","DOIUrl":"10.1016/j.jtct.2025.04.015","url":null,"abstract":"<div><h3>Introduction</h3><div>The Blood and Marrow Transplant Clinical Trials Network (BMT CTN) 1102 trial demonstrated that allogeneic hematopoietic cell transplantation (HCT) was associated with superior overall survival compared to non-HCT approaches among elderly patients with higher-risk myelodysplastic syndrome (MDS). The trial included an ancillary cost diary component to assess the out-of-pocket (OOP) expenditures and financial hardship in the post-HCT period through 3 phased surveys for up to 19 months after enrollment.</div></div><div><h3>Objective</h3><div>The purpose of the study is to assess the OOP costs and financial hardship experienced by participants of BMT CTN 1102.</div></div><div><h3>Study Design</h3><div>BMT CTN 1102 assigned participants to Donor and No-Donor arms based on donor availability. Participants could additionally enroll in the ancillary cost diary component, with a total of 138 participants returning 267 surveys across 3 survey waves at 1-, 7-, and 19-months after enrollment. As participants who underwent HCT returned 78% (207/267) of the total surveys, we report on the collected data descriptively.</div></div><div><h3>Results</h3><div>Participants who underwent HCT had high levels of monthly OOP expenditure ($1126, $812, $442) and financial hardship (47%, 53%, 57%) across the 3 survey waves. For reference, participants who did not undergo HCT generally reported lower levels of OOP expenditure ($478, $845, $256) and financial hardship (37%, 55%, 46%).</div></div><div><h3>Conclusion</h3><div>Among BMT CTN 1102 participants, those who underwent HCT reported high levels of OOP expenditures and financial hardship for up to 19 months after enrollment. Ongoing routine assessment of patient-level OOP expenditures and financial burden may be helpful in the post-HCT survivorship period.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 459.e1-459.e6"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144020440","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 Quality of Life and Symptoms in Transplantation and CAR-T Recipients: Expert Panel Recommendations from the Survivorship Special Interest Group of ASTCT.","authors":"Rahul Banerjee, Hermioni L Amonoo, Anna Barata, Neel S Bhatt, Manuel R Espinoza-Gutarra, Reena V Jayani-Kosarzycki, Hannah Katz, Vanessa E Kennedy, Mariam Nawas, Angela Steineck, Chris Wanjiku, Erin Costanzo, Rachel N Cusatis, Jennifer M Knight, Helene Schoemans, Surbhi Sidana, William A Wood, Anthony D Sung, Catherine J Lee, Betty K Hamilton","doi":"10.1016/j.jtct.2025.06.030","DOIUrl":"10.1016/j.jtct.2025.06.030","url":null,"abstract":"<p><p>Patient-reported outcomes (PROs) to measure quality of life (QOL) and other symptoms play an increasingly important role in clinical trials and regulatory approvals for hematopoietic cell transplantation (HCT) and chimeric antigen receptor T-cell (CAR-T) therapy. However, their adoption has been hindered by wide heterogeneity in the choice of PRO measures for clinical research, including the Functional Assessment of Cancer Therapy Bone Marrow Transplantation (FACT-BMT) and the European Organization for Research and Treatment of Cancer Quality of Life Core Questionnaire (EORTC QLQ-C30) inventories. In addition, the potential for PRO integration into routine standard-of-care (SOC) practice for patients undergoing HCT or CAR-T therapy has not yet been realized. As part of a coordinated effort by 3 American Society for Transplantation and Cellular Therapy Special Interest Groups, we developed best practices for PRO integration in adult and pediatric recipients of HCT and CAR-T therapy. We strongly encourage the use of the Patient-Reported Outcomes Measurement Information System (PROMIS) or the PRO version of Common Terminology Criteria for Adverse Events (PRO-CTCAE) instruments as the primary PRO measures for most HCT and CAR-T trials. Measures such as the PROMIS-29 inventory can be used for QOL assessments, while PRO-CTCAE item banks can be used for specific symptoms. Rationales for our strong recommendation to move from FACT-BMT and EORTC QLQ-C30 to PROMIS/PRO-CTCAE instruments include: (1) free licensing and ease of implementation, including in electronic medical records; (2) psychometric validation in a variety of oncologic settings, including during inpatient hospitalizations; (3) translation into multiple languages, with validation in both adult and pediatric settings; and (4) adoption into centrally collected PRO protocols from the Center for International Blood and Marrow Transplant Research for both HCT and CAR-T recipients. Steps to operationalize these PRO measures are discussed, as are methods to migrate existing data from legacy PRO instruments. We similarly recommend the consideration of PRO integration into SOC clinical practice, including the development of threshold-based workflows to both personalize and standardize care in this setting. Other panel recommendations include the use of standardized timepoints for longitudinal PRO assessments and the inclusion of patient advocates when implementing PRO measures. Implementing these steps will improve the ability of PROs to improve outcomes for patients undergoing HCT or CAR-T therapy, both in trials and-more importantly-in SOC practice.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12243979/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561270","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":"Outcomes of Unrelated Donor Stem Cell Transplantation with Partial T Cell Depletion for Pediatric Patients with Hemoglobinopathies.","authors":"Nora M Gibson, Caitlin W Elgarten, Joseph H Oved, Lisa Wray, Jason Freedman, Eugene Khandros, Elizabeth Worster, Peter Nicholas, Stephan Kadauke, Yongping Wang, Stephan Grupp, Janet L Kwiatkowski, Timothy S Olson","doi":"10.1016/j.jtct.2025.06.029","DOIUrl":"10.1016/j.jtct.2025.06.029","url":null,"abstract":"<p><p>Optimal methods of alternative donor allogeneic transplant for pediatric patients with sickle cell disease (SCD) and beta thalassemia major (BTM) lacking matched related donors have remained elusive. Most studies demonstrate unacceptable rates of graft rejection, graft-versus-host disease, and/or organ toxicity in children. Ex vivo partial T cell depletion (pTCD) of unrelated donor peripheral stem cell (URD-PSC) grafts has the potential to facilitate durable engraftment while preventing GVHD. We present a single-center analysis of URD pTCD-PSCT for pediatric patients with SCD and BTM. Sixteen patients underwent URD-SCT with 10/10 or 9/10 HLA-matched donors across 3 clinical trial protocols. Conditioning included hydroxyurea, thymoglobulin, fludarabine, thiotepa, and busulfan. Graft manipulation included CD3⁺/CD19⁺ depletion with 1 × 10⁵ CD3⁺ cells/kg addback in 4 patients, and TCRαβ⁺ T cell/CD19⁺ depletion in 12 patients. Median follow-up is 36 months (range 10 to 57). One- and 3-year overall survival is 93.8%, and 3-year graft failure-free survival is 81.3%. One patient with preexisting severe cerebrovasculopathy died from cerebral hemorrhage. All patients demonstrated rapid trilinear engraftment, and notably rapid platelet engraftment. No patients developed Grades III and IV acute GVHD or moderate-to-severe chronic GVHD. All 6 patients with SCD receiving 10/10 matched donor grafts and all patients with BTM (all 5 had 9/10 matched donors) exhibited durable engraftment with median total donor chimerism of 95% (range 88% to 100%) at last follow-up. 3 of 5 patients with SCD receiving 9/10 HLA-matched donor grafts experienced graft failure. Two of these patients had lasting engraftment and resolution of disease phenotype with a second transplant. URD-PSCT with pTCD is associated with excellent engraftment, overall survival, and minimal GVHD in patients with SCD receiving 10/10 URD grafts and patients with BTM with ≥9/10 URD grafts.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561271","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":"Omidubicel-onlv Transplantation for Hematologic Malignancies: Results of a Multicenter Expanded Access Program","authors":"Mitchell E. Horwitz ,&nbsp;Gary J. Schiller ,&nbsp;Stephanie B. Tsai ,&nbsp;Andrew R. Rezvani ,&nbsp;Richard T. Maziarz ,&nbsp;Uri Goshen ,&nbsp;Stuart Levy ,&nbsp;Aurélie Schwarzbach ,&nbsp;Roei D. Mazor ,&nbsp;Patrick J. Stiff","doi":"10.1016/j.jtct.2025.04.005","DOIUrl":"10.1016/j.jtct.2025.04.005","url":null,"abstract":"<div><div>Omidubicel-onlv is an FDA-approved, nicotinamide-modified, allogeneic hematopoietic progenitor cell therapy derived from umbilical cord blood (UCB). A phase 3 study demonstrated improved hematopoietic recovery and decreased infections with omidubicel compared with UCB allogeneic transplantation. We report results of an Expanded Access Program evaluating clinical outcomes in patients with hematologic malignancies following transplantation with omidubicel. Between August 2020 and May 2023, 29 patients were transplanted at 5 US sites. Patients received myeloablative conditioning, prophylactic and therapeutic medications, and supportive care per institutional guidelines, and were monitored for engraftment, infections, and graft-versus-host-disease (GVHD) for up to 2 years post-transplant. Results were compared with previously reported phase 3 outcomes. Omidubicel recipients had a median age of 39 (range 20-73, 62% male); 45% were non-White and 65.5% had acute leukemia. Median follow-up was 11.8 (range: .3-27.7) months. Median neutrophil and platelet engraftment times were 12 and 33.5 days, respectively. Acute GVHD (grade 3-4) at day 100 occurred in 19% of patients, with chronic GVHD at 1 year in 9% of patients, all of which were mild. First grade 2 to 3 bacterial infections through 100 days post-transplant and first grade 3 viral infection 1 year post-transplant occurred in 18% and 12% of patients, respectively. One-year disease-free survival and overall survival rates were 76% and 87%, respectively. This real-world study of omidubicel transplantation for hematologic malignancies finds that this graft source is commonly used for non-White allogeneic transplant recipients. The rapid engraftment kinetics observed following transplantation with omidubicel appears to have addressed excessive nonrelapse mortality that has been previously observed following myeloablative umbilical cord blood transplantation.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 436-447"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144051201","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":"Understanding Telomere Biology in Hematopoietic Cell Transplantation: A Dynamical Systems Perspective.","authors":"Amir A Toor, Morgan Horton, Haniya Khalid, Elizabeth Krieger, Tsung-Po Lai, Stephen R Spellman, John E Levine, Wael Saber, Valerie Stewart, Shahinaz M Gadalla","doi":"10.1016/j.jtct.2025.06.024","DOIUrl":"10.1016/j.jtct.2025.06.024","url":null,"abstract":"<p><strong>Background: </strong>T cell proliferation and repertoire reconstitution is a hallmark of successful hematopoietic cell transplantation (HCT). This process may be modeled as a dynamical system and in such a system, precise telomere length (TL) measurement may reflect the proliferative capacity of donor T cells. TL for different chromosomes span a few orders of magnitude, and different blood cell populations including T cell clones display variable TL; these differences across the cell populations are not represented when examining average leukocyte TL. This study aims to develop a method that integrates the entire spectrum of TL observed within a sample to better understand the influence on clinical outcomes following HCT.</p><p><strong>Methods: </strong>To better reflect the entire span of TL, we used data generated using the Telomere Shortest Length Assay (TeLSA) that provides discrete measurements of individual telomeres for each blood DNA sample. TeSLA leukocyte TL (LTL) measurements were performed on 72 paired samples collected from the donor pretransplant (D-LTL) and the recipient 90 days following HCT (post-HCT LTL). Area under the curve (AUC) calculations were used to incorporate the full distribution of measured LTL from each sample. The magnitude of LTL shortening after HCT was calculated as the difference between the AUCs for D-LTL and corresponding post-HCT LTL, and referred to as AUC delta-TL.</p><p><strong>Results: </strong>Telomere band lengths ranged from 350 base pairs to 16.7 kilobases with a logarithmically declining distribution in all samples when arrayed in descending order. The AUC delta-TL predicted patient overall survival (OS; P-log rank <.0001); HCT recipients with an intermediate degree of TL shortening (25^th to 75^th percentile/Q2&3) post-HCT experienced the best outcomes (2 years OS = 92%), whilst donors with minimal (<25^th percentile/Q1; 2 years OS = 33%; adjusted HR versus intermediate shortening = 9.3, P = .001) or maximal (>75^th percentile/Q4; 2 years OS = 59%; adjusted HR = 6.0, P = .01) TL shortening had worse outcomes.</p><p><strong>Conclusion: </strong>The findings described herein suggest that the degree of donor telomere attrition may correlates with clinical outcomes following transplant, possibly reflecting alloreactive T cell expansion. Accounting for the entire span of telomere lengths, may better identify post-transplant risk groups.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144561272","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 Art and Images of Reflections: 2023-2025","authors":"Keith M. Sullivan,&nbsp;Stephan J. Forman","doi":"10.1016/j.jtct.2025.06.006","DOIUrl":"10.1016/j.jtct.2025.06.006","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 395-398"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144549482","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}