Transplantation and Cellular Therapy最新文献

{"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}

{"title":"Nephrotoxicity in CAR-T Cell Therapy","authors":"Karol Sadowski ,&nbsp;Weronika Ploch ,&nbsp;Alicja Downar ,&nbsp;Wiktoria Giza ,&nbsp;Dorota Szcześ ,&nbsp;Wioletta Olejarz ,&nbsp;Wiesław W. Jędrzejczak ,&nbsp;Jolanta Małyszko ,&nbsp;Grzegorz Basak","doi":"10.1016/j.jtct.2025.03.007","DOIUrl":"10.1016/j.jtct.2025.03.007","url":null,"abstract":"<div><div>Chimeric antigen receptor (CAR)-T cell therapy is a novel therapy for the treatment of different hematologic malignancies. Besides its efficiency, CAR-T cell therapy is associated with significant toxicity, primarily manifested as cytokine release syndrome (CRS) and neurotoxicity. However, there are reports that CAR-T cell therapy is also nephrotoxic and this aspect has attracted less attention to date. In this review, we focus on the incidence and association between CAR-T cell therapy and kidney injury. Here, we describe risk factors, biomarkers, and potential reasons for acute kidney injury (AKI) and chronic kidney disease (CKD) related to CAR-T cell therapy to shed light on pathomechanisms leading to renal impairment as well as to the association of kidney failure with other side effects of CAR-T cell therapy. We also review the toxicity of different types of CAR-T cell products, the impact of nephrotoxicity on CAR-T cell therapy efficacy, and the safety of lymphodepletion in patients with baseline AKI or CKD.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 407-418"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143664619","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":"G-CSF-Primed Peripheral Blood Stem Cell Haploidentical Transplantation Could Achieve Satisfactory Clinical Outcomes for Severe Aplastic Anemia Patients","authors":"Xiao-Di Ma ,&nbsp;Zheng- Li Xu ,&nbsp;Yun He ,&nbsp;Yi-Fei Cheng ,&nbsp;Ting-Ting Han ,&nbsp;Yuan-Yuan Zhang ,&nbsp;Jing-Zhi Wang ,&nbsp;Xi-Dong Mo ,&nbsp;Feng-Rong Wang ,&nbsp;Xin Zhao ,&nbsp;Yu Wang ,&nbsp;Xiao-Hui Zhang ,&nbsp;Xiao-Jun Huang ,&nbsp;Lan-Ping Xu","doi":"10.1016/j.jtct.2025.04.008","DOIUrl":"10.1016/j.jtct.2025.04.008","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;div&gt;Allogeneic hematopoietic stem cell transplantation (allo-HSCT) continues to be a cornerstone in the treatment of severe aplastic anemia (SAA). The advancement of haploidentical hematopoietic stem cell transplantation (haplo-HSCT) has broadened therapeutic possibilities, particularly for patients lacking fully human leukocyte antigen (HLA)-matched donors. However, it still remains unclear which type of graft source is better for SAA patients underwent haplo-HSCT.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Objectives&lt;/h3&gt;&lt;div&gt;This study aimed to assess the clinical outcomes of haplo-HSCT using granulocyte colony-stimulating factor (G-CSF)-primed peripheral blood (G-PB) as the graft source, comparing them to a control group receiving G-CSF-primed bone marrow (BM) plus G-PB (BM+PB).&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Study design&lt;/h3&gt;&lt;div&gt;This was a single-center, retrospective, case-pair cohort study. Between January 2020 and December 2023, a total of 278 consecutive SAA patients received haplo-HSCT in Peking University People’s Hospital. In total, 22 patients receiving haplo-HSCT using PB were included in this study. To minimize the impact of potential confounders in this study, we used the propensity score matching (PSM) method to match patients who underwent haplo-HSCT with G-PB plus G-BM at the same time with a 3:1 ratio using nearest-neighbor matching. In the end, 88 patients were included in this study. A total of 22 patients received PB stem cells as graft and 66 patients received G-CSF-primed BM plus PB as graft.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;The PB group demonstrated greater neutrophil (100% vs. 93.9%, &lt;em&gt;P&lt;/em&gt; = .04) and platelet engraftment (95.5% vs. 89.0%, &lt;em&gt;P&lt;/em&gt; = .03) incidence compared with the BM+PB group. There were no significant differences in the cumulative incidences of grades II–IV (13.6% vs. 25.8%, &lt;em&gt;P&lt;/em&gt; = .28) or grades III–IV acute graft-versus-host disease (aGVHD; 4.5% vs. 4.6%, &lt;em&gt;P&lt;/em&gt; = .99) between the PB group and BM+PB group. The PB group (36.7%) exhibited a trend toward a higher incidence of chronic GVHD compared to BM+PB group (24.1%). However, the difference between the two groups was not statistically significant. Moreover, the immune reconstitution of CD3+T cells, CD4+T cells, CD8+T cells and CD19+B cells were also comparable between two groups. At 3 years post-haplo-HSCT, the probabilities of overall survival (OS), failure-free survival (FFS), and GVHD-free/failure-free survival (GFFS) were 86.1% versus 87.9% (&lt;em&gt;P&lt;/em&gt; = .90), 86.1% versus 83.3% (&lt;em&gt;P&lt;/em&gt; = .73) and 76.5% versus 75.2% (&lt;em&gt;P&lt;/em&gt; = .70) for PB and BM+PB group, respectively. In univariate analysis, the graft source did not influence the clinical outcomes after HSCT.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Conclusions&lt;/h3&gt;&lt;div&gt;This study illustrated the safety and efficacy of haplo-HSCT with PB being the single graft source as the treatment for SAA, providing a basis for further potential optimization of the current protocol. In the future, this concl","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 454.e1-454.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144041168","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":"Changes in Chronic Graft-versus-Host Disease Treatment Over Time: A 15-Years Survey Within Allogeneic Hematopoietic Stem Cell Transplant Centers in Germany, Austria, and Switzerland","authors":"Alexander Denk ,&nbsp;Matthias Alexander Fante ,&nbsp;Silke Heidenreich ,&nbsp;Hildegard T. Greinix ,&nbsp;Eva-Maria Wagner Drouet ,&nbsp;Katharina Egger-Heidrich ,&nbsp;Julia Marx ,&nbsp;Darina Kodzhabasheva ,&nbsp;William Krüger ,&nbsp;Gesine Bug ,&nbsp;Claudia Wehr ,&nbsp;Joerg Halter ,&nbsp;Irene Teichert von Lüttichau ,&nbsp;Lutz Peter Mueller ,&nbsp;Olaf Penack ,&nbsp;Inken Hilgendorf ,&nbsp;Guido Kobbe ,&nbsp;Stefan Klein ,&nbsp;Anita Lawitschka ,&nbsp;Jochen Frietsch ,&nbsp;Daniel Wolff","doi":"10.1016/j.jtct.2025.04.017","DOIUrl":"10.1016/j.jtct.2025.04.017","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;div&gt;Chronic graft-versus-host disease (cGVHD) represents a major complication after allogeneic stem cell transplantation (alloHSCT). In 2009 and 2018 a survey among German, Austrian, and Swiss transplant centers showed a homogeneous 1&lt;sup&gt;st&lt;/sup&gt;-line treatment practice, while 2nd-line treatment as well as management of progressive onset type and bronchiolitis obliterans syndrome (BOS) displayed significant heterogeneity. Since the last survey, ruxolitinib (rux) has been approved and other new agents are explored in treatment of cGVHD.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Objective&lt;/h3&gt;&lt;div&gt;We conducted a follow-up survey in 2024 to document the impact of recent approvals and new agents on treatment pattern focusing on management of 2nd-line treatment, progressive onset type, BOS, and sclerotic manifestations.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Study design&lt;/h3&gt;&lt;div&gt;A paper-and-pencil-based questionnaire was sent electronically to 60 German speaking centers performing alloHSCT. 20 centers responded, representing 45% of the patients receiving an alloHSCT in 2023 in Germany, Austria, and Switzerland.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;In 1&lt;sup&gt;st&lt;/sup&gt;-line treatment of classic standard risk cGVHD, single agent prednisone represents standard of care (14/20 centers) which may be combined with calcineurin inhibitor (CNI) (4/20), while rux is used in selected cases only. In 2nd-line treatment rux is now used by the majority of centers (19/20). In the presence of cytopenia, rux remains the preferred agent (12/20) while use of extracorporeal photopheresis (ECP) is considered by 8 of 20 centers. In case of active infections, ECP is preferred by 15 of 20 centers and both agents are regarded as steroid-sparing agents in 2nd-line treatment of steroid-dependent cGVHD. Rux would be applied in the presence of active infections by 5/20 centers only. Moreover, rux (15/20) and ECP (6/20) are also preferred treatment modalities in treatment of progressive onset cGVHD. For BOS, systemic and inhalative corticosteroids, montelukast and azithromycin (FAM, 13/20), rux (15/20), ECP (17/20) and CNI (10/20) are frequently applied agents, while abatacept (8/20), belumosudil (7/20), imatinib (5/20), mycophenolate mofetil (MMF) (5/20), everolimus (4/20) and ibrutinib (3/20) are used as salvage options in selected patients only. In case of new sclerotic manifestations after failure of 2nd-line treatment including steroids, CNI and rux, most centers would use ECP (14/20), whereas subsequent or alternative salvage treatment of sclerotic manifestations remains heterogenous comprising belumosudil (13/20), ibrutinib (5/20), imatinib (5/20), rituximab (4/20), cyclosporine (3/20), tacrolimus (3/20), everolimus (3/20), sirolimus (3/20), methotrexate (3/20) and MMF (3/20). The preferred taper sequence of immunosuppressive agents in case of response applied in 12/20 centers is initial taper of steroids, followed by taper of CNI and final termination of rux.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Conclusion&lt;/","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 450.e1-450.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036410","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":"Impact of Cytomegalovirus Reactivation Following Allogeneic Hematopoietic Cell Transplantation on the Relapse of Acute Leukemia","authors":"Hyunkyung Park ,&nbsp;Je-Hwan Lee ,&nbsp;Heungsup Sung ,&nbsp;Jung-Hee Lee ,&nbsp;Han-Seung Park ,&nbsp;Eun-Ji Choi ,&nbsp;Kyoo-Hyung Lee ,&nbsp;Sung-Han Kim ,&nbsp;Young-Shin Lee ,&nbsp;Young-Ah Kang ,&nbsp;Mijin Jeon ,&nbsp;Ji Min Woo ,&nbsp;Hyeran Kang ,&nbsp;Yunsuk Choi","doi":"10.1016/j.jtct.2025.04.021","DOIUrl":"10.1016/j.jtct.2025.04.021","url":null,"abstract":"<div><h3>Background</h3><div>Cytomegalovirus (CMV) reactivation following allogeneic hematopoietic cell transplantation (HCT) is a significant complication; however, its impact on relapse remains controversial.</div></div><div><h3>Objectives</h3><div>This study aimed to evaluate the clinical impact of CMV reactivation on relapse and survival after HCT in patients with acute leukemia.</div></div><div><h3>Study Design</h3><div>We conducted a retrospective analysis of 1258 patients diagnosed with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) who underwent their first HCT between 2000 and 2020.</div></div><div><h3>Results</h3><div>Our cohort included 871 patients with AML and 387 patients with ALL. Among all patients, 751 (61.6%) experienced CMV reactivation within one year post-HCT. CMV reactivation was associated with HCT from unrelated donors, anti-thymocyte globulin use, and the occurrence of acute graft-versus-host disease. Notably, CMV reactivation was associated with a lower risk of relapse (HR, 0.713; <em>P</em> = .001) and improved event-free survival (HR, 0.743; <em>P</em> = .001) in both AML and ALL patients. However, there was no significant difference in non-relapse mortality or overall survival according to CMV reactivation.</div></div><div><h3>Conclusion</h3><div>These results suggest that CMV reactivation may prevent post-HCT relapse and enhance event-free survival in AML and ALL patients. Therefore, a CMV prophylaxis strategy is warranted to establish a safe range of CMV reactivation titers that can yield beneficial effects.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 465.e1-465.e14"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144036411","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":"Heterogeneity of Survival Benefit Conferred By Letermovir","authors":"Yu Akahoshi ,&nbsp;Hideki Nakasone ,&nbsp;Katsuto Takenaka ,&nbsp;Takahide Ara ,&nbsp;Yuma Tada ,&nbsp;Noriko Doki ,&nbsp;Naoyuki Uchida ,&nbsp;Masatsugu Tanaka ,&nbsp;Yuta Hasegawa ,&nbsp;Wataru Takeda ,&nbsp;Tetsuya Nishida ,&nbsp;Jun Ishikawa ,&nbsp;Naoki Kurita ,&nbsp;Masashi Sawa ,&nbsp;Makoto Onizuka ,&nbsp;Shinichi Kako ,&nbsp;Shin-Ichiro Fujiwara ,&nbsp;Keisuke Kataoka ,&nbsp;Koji Kawamura ,&nbsp;Takahiro Fukuda ,&nbsp;Yoshinobu Kanda","doi":"10.1016/j.jtct.2025.04.010","DOIUrl":"10.1016/j.jtct.2025.04.010","url":null,"abstract":"<div><div>Variation in treatment effects based on individual patient characteristics—known as treatment effect heterogeneity or effect modification—has recently gained significant attention. A previous clinical trial and its post hoc analysis suggested that letermovir (LTV) may reduce mortality more in some patients than in others. We hypothesized that the survival benefit of LTV differs according to each patient’s specific characteristics. This study aimed to identify patient characteristics that are associated with significant survival benefits from LTV. Patients who underwent transplantation between 2018 and 2022 were randomly divided into training (n = 5779) and validation groups (n = 2865). We developed two models: one using a proportional hazards model with interaction terms (PI), and another using a modern machine learning (ML) approach to detect heterogeneity in the survival benefit—specifically, to identify patient characteristics associated with greater benefit from LTV. In our cohort, 60% of patients received LTV as prophylaxis. In the training cohort, the final PI model, using additive interactions, identified advanced age (≥60), high comorbidities (HCT-CI ≥3), umbilical cord blood (UCB), and haploidentical HCT with post cyclophosphamide (PTCy Haplo) as highly beneficial factors. Meanwhile, the ML model, using a causal forest algorithm, classified the top 60% of patients based on the estimated individual treatment effect as the high benefit group. In the validation group, 67.1% and 59.9% of patients were considered to be high benefit by the PI and ML models, respectively. The absolute difference in 6-month NRM (LTV versus no LTV) in the high benefit group (PI model: 9.8% versus 16.3%; ML model: 11.3% versus 16.3%) was greater than that in the low benefit group (PI model: 4.3% versus 6.9%; ML model: 4.1% versus 6.2%). Most patients (&gt;80%) with advanced age, high comorbidities, or UCB were classified as high benefit by the ML model, supporting the robustness of the PI model. Our models successfully identified patients who could be expected to experience lower NRM with LTV prophylaxis, underscoring the importance of personalized medicine.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 461.e1-461.e12"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144011699","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":"Proceedings From the Reimagining Caregiver Workshop: Addressing Caregiver Requirements for Hematopoietic Cell Transplant","authors":"Jaime M. Preussler ,&nbsp;Anna M. DeSalvo ,&nbsp;Ben Tweeten ,&nbsp;June Klaphake ,&nbsp;Meghann R. Cody ,&nbsp;Paris M. McGhee ,&nbsp;Karla S. Dawson ,&nbsp;Katie Schoeppner ,&nbsp;Jeffery J. Auletta","doi":"10.1016/j.jtct.2025.04.006","DOIUrl":"10.1016/j.jtct.2025.04.006","url":null,"abstract":"<div><div>The NMDP-sponsored, PCORI (Patient Centered Outcomes Research Institute)-funded project, <em>Reimagining Caregiving Together, Engagement to Address Caregiver Requirement Barriers</em>, is a series of workshops aimed to convene and engage a diverse group of stakeholders to promote discussion of challenges and solutions to caregiver requirements and to develop a PCOR/comparative effectiveness research (CER) agenda to generate evidence on alternative post-allogeneic hematopoietic cell transplant (alloHCT) models to improve access to care. This paper reviews the proceedings from the first workshop and provides an overview of the workshop’s efforts to begin to address caregiver requirement barriers.</div><div>The first workshop, “Defining the Problem and Developing Key Messages” was held in-person in Minneapolis, MN, October 3-4^th, 2024. Discussion focused on caregiver requirements, identifying a vision for safe-post-alloHCT care and barriers to that vision as well as planning for communication and next steps. Pre- and post-surveys were conducted for evaluation. Survey results showed a significant decrease in the perception of the need for a 24/7 caregiver, reflecting the influence of shared discussion, understanding and problem solving. Materials from this workshop and continued engagement between this workshop and the second workshop in May 2025 will be used to develop a comprehensive strategy and research agenda to enable more patients to receive alloHCT who might currently be unable to do so due to caregiver barriers.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 458.e1-458.e10"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144062100","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":"Multicenter Study on Caregiver Experiences in Pediatric Hematopoietic Stem Cell Transplantation Part I: Integrative Analysis of Mental Health, Psychosocial Stressors, and Support Mechanisms","authors":"Neel S. Bhatt ,&nbsp;Leslie Lehmann ,&nbsp;Christopher E. Dandoy ,&nbsp;Jeffery J. Auletta ,&nbsp;Priscila Badia ,&nbsp;Sheri A. Ballard ,&nbsp;Robyn Blacken ,&nbsp;Nancy M. Daraiseh ,&nbsp;Catherine Desmond ,&nbsp;Chloe Dunseath ,&nbsp;Preston Epling ,&nbsp;Taylor J. Fitch ,&nbsp;Laura Flesch ,&nbsp;David Hartley ,&nbsp;John Huber ,&nbsp;Kari Jenssen ,&nbsp;Georgia Kent ,&nbsp;Anna Klunk ,&nbsp;Malika Kapadia ,&nbsp;Katilyn Kusnier ,&nbsp;Seth Rotz","doi":"10.1016/j.jtct.2025.04.013","DOIUrl":"10.1016/j.jtct.2025.04.013","url":null,"abstract":"<div><div>Caregivers of children undergoing allogeneic hematopoietic stem cell transplantation (HSCT) face substantial psychological, social, and logistical challenges throughout the transplant journey. This multicenter, longitudinal qualitative study explored the evolving mental health experiences, stressors, and coping strategies of 49 caregivers interviewed across four key time points: transplant (d 0), d +30, d +100, and d +180. Participants reported acute distress early in the process, exacerbated by restrictive hospital environments, the demands of hypervigilant caregiving, financial strain, and the emotional toll of family separation. As care transitioned to the outpatient setting, challenges shifted toward navigating complex home care, managing lingering uncertainty, and balancing the needs of other family members. Throughout the process, caregivers expressed heightened anxiety related to fear of relapse, infection, and long-term complications. Despite these burdens, many caregivers described powerful sources of resilience. Children’s emotional strength, honest communication, and a desire to return to normal life helped sustain caregiver optimism. Support from the healthcare team, financial assistance, and access to professional mental health services further alleviated stress. Caregivers emphasized the need for enhanced inpatient environments, clearer outpatient guidance, structured mental health resources, and practical tools like caregiver handbooks. These findings underscore the need for holistic, family-centered care that addresses caregiving's psychological and practical dimensions during pediatric HSCT. Tailored, time-sensitive support strategies are essential to improving caregiver well-being and, in turn, optimizing patient outcomes across the transplant continuum.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 7","pages":"Pages 456.e1-456.e16"},"PeriodicalIF":3.6,"publicationDate":"2025-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144038778","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}