Transplantation and Cellular Therapy最新文献

{"title":"What's Best? Transformative Therapies for Adults with Sickle Cell Disease","authors":"John J Strouse , Adetola Kassim","doi":"10.1016/j.jtct.2025.04.004","DOIUrl":"10.1016/j.jtct.2025.04.004","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 279-281"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143903372","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":"Unravelling T Cell Immunity in CAR-T Therapy: Implications for Influenza Vaccination","authors":"Adam G Stewart , Roy F Chemaly","doi":"10.1016/j.jtct.2025.04.003","DOIUrl":"10.1016/j.jtct.2025.04.003","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 282-284"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143903403","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":"Masthead (Purpose and Scope)","authors":"","doi":"10.1016/S2666-6367(25)01161-3","DOIUrl":"10.1016/S2666-6367(25)01161-3","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages A1-A2"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143904500","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":"Outcome of Patients With IDH-Mutated AML Following Allogeneic Stem Cell Transplantation—A Retrospective Analysis on Behalf of the German Registry for Hematopoietic Stem Cell Transplantation and Cell Therapy, DRST","authors":"Thomas Schroeder ,&nbsp;Sarah Flossdorf ,&nbsp;Claudia Schuh ,&nbsp;Caroline Pabst ,&nbsp;Michael Stadler ,&nbsp;Johannes Schetelig ,&nbsp;Claudia Wehr ,&nbsp;Matthias Stelljes ,&nbsp;Elisa Sala ,&nbsp;Andreas Burchert ,&nbsp;Julia Winkler ,&nbsp;H. Christian Reinhardt ,&nbsp;Nicolaus Kröger ,&nbsp;Katharina Fleischhauer ,&nbsp;Christina Rautenberg","doi":"10.1016/j.jtct.2025.02.018","DOIUrl":"10.1016/j.jtct.2025.02.018","url":null,"abstract":"<div><div>Mutations in isocitrate dehydrogenase 1 and 2 genes (<em>IDH1</em> and <em>IDH2</em>) are found in 15% to 20% of patients with acute myeloid leukemia (AML). <em>IDH</em> inhibitors have been introduced as targeted treatment and are currently under investigation as maintenance therapy after allogeneic transplantation (allo-SCT). Since reports about the outcome of <em>IDH1</em>- and <em>IDH2</em>-mutated (<em>IDH</em>mut) AML after allo-SCT are limited, we retrospectively analyzed 356 <em>IDH</em>-mutated AML patients (<em>IDH1</em> 40%, <em>IDH2</em> 60%). Ten patients (4%) had received an <em>IDH</em> inhibitor prior transplantation, but none had received maintenance with <em>IDH</em> inhibitors. After a median follow-up of 24 months 3-year probabilities of overall (OS) and event-free (EFS) survival, relapse and nonrelapse mortality (NRM) for the entire cohort were 73%, 60%, 27% and 13% respectively. While 3-year OS (78% versus 70%), EFS (56% versus 63%) and NRM (10% versus 14%) rates were similar for <em>IDH1</em>mut and <em>IDH2</em>mut patients, relapse incidence was numerically higher in <em>IDH1</em>mut patients (34% versus 24%) and landmark analysis suggested a continuous rise of relapse incidence preferentially in <em>IDH1</em>mut AML also beyond the first year. Concordantly, <em>IDH2</em> mutation was associated with superior EFS and by trend with lower relapse incidence. The strongest risk factor for adverse outcomes, however, was AML not in CR. This analysis provides benchmarks for interpretation of results emerging from post-transplant maintenance trials in <em>IDH</em>mut AML and suggest that maintenance strategies may further optimize the promising outcome in this molecularly defined subgroup by reducing relapse risk, especially for patients whose AML is not in remission at time of alloHCT.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 303.e1-303.e9"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143587139","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":"Plasma Nucleosome Levels and Risk of Acute Graft-Versus-Host Disease After Myeloablative Allogeneic Hematopoietic Stem Cell Transplantation: A Single-Center Cohort Study","authors":"Sune Holm Hansen ,&nbsp;Sisse Rye Ostrowski ,&nbsp;Niels Smedegaard Andersen ,&nbsp;Lone Smidstrup Friis ,&nbsp;Brian Kornblit ,&nbsp;Søren Lykke Petersen ,&nbsp;Ida Schjødt ,&nbsp;Henrik Sengeløv ,&nbsp;Lars Klingen Gjærde","doi":"10.1016/j.jtct.2025.02.015","DOIUrl":"10.1016/j.jtct.2025.02.015","url":null,"abstract":"<div><div>Circulating nucleosomes are representative of cell death, which is a feature of acute graft-versus-host disease (GVHD) following allogeneic hematopoietic stem cell transplantation (allo-HSCT). We explored whether plasma nucleosome levels were prognostic for acute GVHD. We examined the level of circulating nucleosomes in 131 patients who underwent a myeloablative allo-HSCT between June 2015 and August 2018. The measurements were made using quantitative photometric sandwich-ELISA on stored plasma samples obtained pretransplantation (at a median of day –23) and around days +7, +14, and +28 after allo-HSCT. The median plasma nucleosome level remained constant until day +28, where they increased significantly (<em>P</em> &lt; .001 compared to all other times of measurement). The plasma nucleosome level at day +28 was inversely associated with the risk of later grade II to IV acute GVHD (odds ratio [OR] 0.86 per 5 arbitrary unit [AU] increase [95% confidence intervals (CI): 0.66 to 0.99], <em>P</em> = .03), also after adjustment for risk factors of acute GVHD (OR 0.78 per 5 AU increase [95% CI: 0.56 to 0.96], <em>P</em> = .01). We found no support for an association between the plasma level of nucleosomes measured pretransplantation or around day +7 or +14 and the risk of subsequent grade II to IV acute GVHD. We observed a positive correlation between nucleosomes, suppressor of tumorigenesis 2, and C-reactive protein at day +28 (Spearman's <em>ρ</em> = 0.522, <em>P</em> &lt; .001; and Spearman's <em>ρ</em> = 0.386, <em>P</em> &lt; .001; respectively). A lower level of plasma nucleosomes at day +28 after HSCT was associated with a higher risk of subsequent acute GVHD. Additional studies are needed to validate circulating nucleosomes as a prognostic biomarker of acute GVHD.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 301.e1-301.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143469358","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":"Profile of a Pioneer: Carl H. June","authors":"David L. Porter ,&nbsp;Stephan A. Grupp","doi":"10.1016/j.jtct.2025.04.002","DOIUrl":"10.1016/j.jtct.2025.04.002","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 273-278"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143903371","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":"Co-designing a Novel Ordinal Endpoint for an Adaptive Platform Trial, BANDICOOT, in Pediatric Hematopoietic Stem Cell Transplant","authors":"Hannah Walker ,&nbsp;Lorna McLeman ,&nbsp;Deborah Meyran ,&nbsp;Li-yin Goh ,&nbsp;Peter Summers ,&nbsp;Julian Stolper ,&nbsp;Diane Hanna ,&nbsp;David Hughes ,&nbsp;Stacie Wang ,&nbsp;Claudia Toro ,&nbsp;Elizabeth Williams ,&nbsp;Roxanne Dyas ,&nbsp;Lori Chait Rubinek ,&nbsp;Kaitlyn Taylor ,&nbsp;Chris J. Selman ,&nbsp;Anneke Grobler ,&nbsp;Katherine J. Lee ,&nbsp;Tom Snelling ,&nbsp;Theresa Cole ,&nbsp;Amanda Gwee ,&nbsp;Rachel Conyers","doi":"10.1016/j.jtct.2025.01.894","DOIUrl":"10.1016/j.jtct.2025.01.894","url":null,"abstract":"<div><div>An adaptive platform trial (APT) offers the ability to incorporate several research questions in the same target population across multiple domains (interventions), with the ability to add new questions in a perpetual manner. An APT is particularly appealing for pediatric hematopoietic stem cell transplant (HCT); an area of high heterogeneity, limited trial availability, and high mortality. Ideally, all domains in an APT would have the same primary endpoint. Therefore, an ordinal endpoint with multiple categories that combines various clinical outcomes into a single outcome measure is particularly appealing for APTs. Unfortunately, there is no accepted ordinal endpoint for pediatric HCT trials. This article aims to describe the methodology used to co-design a novel primary ordinal endpoint for the pediatric HCT APT — BANDICOOT. BANDICOOT is a study that aims to <em>build an adaptive novel platform design — improving the complications, cost-effectiveness, outcomes, and overall survival from hematopoietic stem cell transplantation.</em>The results of this process identified two potential ordinal endpoints that could be used, one focusing on organ support and the other on a combination of organ support, viral reactivation, and immune reconstitution. We explored the data extraction required for these endpoints from electronic medical records that we will utilize to validate the endpoints and determine which will be used in the APT BANDICOOT. In an era in which APTs are becoming increasingly utilized to answer important questions in clinical care, this article describes a reproducible strategy for the design of high-quality and meaningful ordinal endpoints.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 321.e1-321.e12"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143374705","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":"Autologous or Allogeneic Hematopoietic Stem Cell Transplantation as Front-Line Treatment for Adult Secondary Acute Myeloid Leukemia Patients: The PETHEMA Registry Experience","authors":"Josefina Serrano ,&nbsp;David Martínez-Cuadrón ,&nbsp;Cristina Gil ,&nbsp;Teresa Bernal ,&nbsp;Mar Tormo ,&nbsp;Pilar Martínez-Sánchez ,&nbsp;Carlos Rodríguez-Medina ,&nbsp;Pilar Herrera ,&nbsp;José A. Pérez Simón ,&nbsp;María J. Sayas ,&nbsp;Juan Bergua ,&nbsp;Esperanza Lavilla-Rubira ,&nbsp;Mariluz Amigo ,&nbsp;Celina Benavente ,&nbsp;José L. López Lorenzo ,&nbsp;Manuel M. Pérez-Encinas ,&nbsp;María B. Vidriales ,&nbsp;Clara Aparicio-Pérez ,&nbsp;Esther Prados de la Torre ,&nbsp;Mercedes Colorado ,&nbsp;Pau Montesinos","doi":"10.1016/j.jtct.2025.02.011","DOIUrl":"10.1016/j.jtct.2025.02.011","url":null,"abstract":"<div><div>It is widely accepted that allogeneic hematopoietic stem cell transplantation (allo-HSCT) is the sole potentially curative option available for secondary acute myeloid leukemia (sAML). However, clinical factors impacting outcomes after allo-HSCT and the potential role of autologous HSCT (auto-HSCT) in real-life series are needed. Previously, the PETHEMA group reported a series of 2310 patients with sAML in the nationwide registry. Of these, 876 were candidates to receive chemotherapy and 274 underwent HSCT (55 auto-HSCT and 219 allo-HSCT). In this study, we analyzed the role of auto-HSCT or allo-HSCT as front-line treatment for sAML patients included in the Spanish PETHEMA AML registry. Here we report an analysis of outcomes as well as prognostic variables in this series of patients undergoing auto-HSCT or allo-HSCT as part of the front-line treatment for sAML. We used the multinational PETHEMA AML registry (Clincial Trials.gov identifier NCT02607059) to identify adult patients (age ≥18 years) with a diagnosis of sAML who underwent auto- or allo-HSCT as front-line treatment in Spanish and Portuguese institutions between August, 1, 1992, and July, 31, 2020. Patient characteristics, diagnostic findings, and management, including treatments, characteristics of HSCT, and outcomes, were retrieved from the PETHEMA AML registry in this retrospective multicenter analysis. With a median follow-up of 32.7 months, better 5-year overall survival (OS) and leukemia-free survival (LFS) were obtained with allo-HSCT in first complete response (CR) (44.5% and 39.9%, respectively) compared with auto-HSCT in CR1 (30% and 20.5%, respectively) but without reaching statistical differences for OS <em>(P</em> = .22 and .03, respectively). The higher incidence of relapse in auto-HSCT is counterbalanced with the significantly lower nonrelapse mortality rate. For allo-HSCT recipients, 5-year outcomes were significantly influenced by the cytogenetic/genetic risk. In multivariate analysis, the adverse cytogenetic/genetic risk group retained statistical significance for all endpoints. We confirmed the role of allo-HSCT as a potential curative option for patients and report that auto-HSCT in CR can still provide a 5-year LFS of 20% in sAML patients. Finally, our results confirm adverse cytogenetic/genetic risk category as an independent negative factor in sAML patients undergoing HSCT.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 299.e1-299.e14"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143432767","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":"Officers and Directors of ASTCT","authors":"","doi":"10.1016/S2666-6367(25)01125-X","DOIUrl":"10.1016/S2666-6367(25)01125-X","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Page A5"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143903369","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":"Pediatric Transplant and Cellular Therapy Consortium RESILIENT Conference on Pediatric Chronic Graft-versus-Host Disease Survivorship after Hematopoietic Cell Transplantation: Part III. Long-Term Impact of Chronic Graft-versus-Host Disease on Endocrinologic, Cardiovascular, and Metabolic Outcomes in Survivors of Pediatric Hematopoietic Cell Transplantation","authors":"Pooja Khandelwal ,&nbsp;Jonathan D. Fish ,&nbsp;Lev Gorfinkel ,&nbsp;Gregory M.T. Guilcher ,&nbsp;Jonathan Howell ,&nbsp;Miki Nishitani ,&nbsp;Brandon Nuechterlein ,&nbsp;Esther Obeng ,&nbsp;Ami J. Shah ,&nbsp;Geoffrey D.E. Cuvelier ,&nbsp;Seth Rotz ,&nbsp;Kirsten M Williams ,&nbsp;Christine N. Duncan","doi":"10.1016/j.jtct.2025.01.891","DOIUrl":"10.1016/j.jtct.2025.01.891","url":null,"abstract":"<div><div>Chronic graft-versus-host disease (cGVHD) has a profound impact on the endocrinologic and cardiovascular health of survivors of transplantation performed in childhood. The impact of cGVHD is long-lasting and contributes to morbidity and early mortality through multiple mechanisms. Organs and tissues may be direct targets of alloreactive donor-derived immune cells. Corticosteroids and other cGVHD-directed therapies influence hormonal actions, alter bone metabolism, and negatively impact cardiometabolic health. Pediatric survivors are particularly vulnerable to the endocrinologic and cardiovascular effects of cGVHD as it develops during periods of intense growth and development, although little is known about the direct contribution to late effects. The Research and Education Toward Solutions for Late Effects to Innovate, Excel, and Nurture after cGVHD (RESILIENT after cGVHD) effort brought together content experts to determine the state of the science, develop clinical recommendations, and propose a research agenda in endocrine, cardiovascular, and metabolic cGVHD survivorship, which are detailed in this report.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 5","pages":"Pages 297.e1-297.e15"},"PeriodicalIF":3.6,"publicationDate":"2025-05-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143426377","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}