Transplantation and Cellular Therapy最新文献

{"title":"CD7 CART Therapy Bridging Allo-HSCT Remarkably Improves Long-Term DFS in Refractory/Relapsed T-ALL/LBL","authors":"Zhihui Li ,&nbsp;Qinlong Zheng ,&nbsp;Keyan Yang ,&nbsp;Teng Xu ,&nbsp;Lei Wang ,&nbsp;Xianxuan Wang ,&nbsp;Wiaopei Wen ,&nbsp;Jingjing Wang ,&nbsp;Yongqiang Zhao ,&nbsp;Yanzhi Song ,&nbsp;Chen Chen ,&nbsp;Qi Zhou ,&nbsp;Tong Wu","doi":"10.1016/j.jtct.2024.11.009","DOIUrl":"10.1016/j.jtct.2024.11.009","url":null,"abstract":"<div><div>T-ALL is caused by abnormal proliferation of T cells. It comprises 25%-50% of ALL cases in children and adults. Outlook for R/R T-ALL/LBL and patients over 60 is even dimmer. The treatment is challenging due to its biological and genetic diversity, limiting the development of effective targeted and immunotherapeutic strategies. Salvaged allo-HSCT offers only 20% to 30% DFS. This current study retrospectively analyzed 90 patients with R/R T-ALL (40, 44.4%) or T-LBL (50, 55.6%) treated at Beijing Gobroad Boren Hospital from February 2018 to January 2023. The median age was 14 (range: 2–65) y old. Somatic and germline gene mutations were detected by sequencing pretransplant. Thirty-two (35.6%) patients were sensitive to chemotherapy and achieved CR before transplant (CR group), and 58 (64.4%) cases were resistant to chemotherapy and in non-remission (NR) pre-HSCT. Forty-one of 58 patients in NR received CD7 CAR-T before allo-HSCT (CART group) and the rest 17 patients in NR underwent salvaged transplant (NR group). The results indicate that CD7 CAR-T group have OS (<em>p</em> = .029; 2-y OS rates: 54.4% [95% CI: 38.9% to 76%]) and DFS (<em>p</em> = .00032; 2-y DFS: 51.0% (95% CI: 36.9% to 70.7%)) similar to those in the CR group, but better than those in the NR group. The CIR for CD7 CAR-T group and CR group was significantly lower than NR group after 1 y (<em>p</em> = .0016; CAR-T group 2-y CIR: 31.67% (95% CI: 19.3% to 49.2%)). Our study examined the somatic and germline gene mutations in R/R T-ALL/LBL and evaluated the prognosis after transplantation. Based on our limited study, we found that using CD7 CAR T cells followed by allo-HSCT greatly enhanced the long-term DFS of chemo resistant T-ALL/LBL patients.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 73.e1-73.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142740068","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 Adverse Event Landscape of Stem Cell Transplant: Evidence for AGVHD Driving Early Transplant Associated Toxicities","authors":"Takuto Takahashi ,&nbsp;Benjamin Watkins ,&nbsp;Brandi Bratrude ,&nbsp;Donna Neuberg ,&nbsp;Kyle Hebert ,&nbsp;Kayla Betz ,&nbsp;Alison Yu ,&nbsp;Sung W Choi ,&nbsp;Jeffrey Davis ,&nbsp;Christine Duncan ,&nbsp;Roger Giller ,&nbsp;Michael Grimley ,&nbsp;Andrew C. Harris ,&nbsp;David Jacobsohn ,&nbsp;Nahal Lalefar ,&nbsp;Nosha Farhadfar ,&nbsp;Michael A. Pulsipher ,&nbsp;Shalini Shenoy ,&nbsp;Aleksandra Petrovic ,&nbsp;Kirk R. Schultz ,&nbsp;Muna Qayed","doi":"10.1016/j.jtct.2024.03.030","DOIUrl":"10.1016/j.jtct.2024.03.030","url":null,"abstract":"<div><div>Although unrelated-donor (URD) hematopoietic cell transplantation (HCT) is associated with many toxicities, a detailed analysis of adverse events, as defined by the Common Terminology Criteria for Adverse Events (CTCAE), has not previously been curated. This represents a major unmet need, especially as it relates to assessing the safety of novel agents. We analyzed a detailed AE database from the “ABA2” randomized, double-blind, placebo-controlled clinical trial of abatacept for acute graft-versus-host disease (AGVHD) prevention, for which the FDA mandated a detailed AE assessment through Day +180, and weekly neutrophil and platelet counts through Day +100. These were analyzed for their relationship to key transplant outcomes, with a major focus on the impact of AGVHD on the development/severity of AEs. A total of 2102 AEs and 1816 neutrophil/platelet counts were analyzed from 142 8/8-HLA-matched URD HCT recipients on ABA2 (placebo cohort, n = 69, abatacept cohort, n = 73). This analysis resulted in 2 major observations. (1) Among graft source, conditioning intensity, age, and Grade 2 to 4 AGVHD, only AGVHD impacted Grade 3 to 5 AE acquisition after the first month post-transplant. (2) The development of Grade 3 to 4 AGVHD was associated with thrombocytopenia. We have created a detailed resource for the transplant community by which to contextualize clinical toxicities after transplant. It has identified AGVHD as a major driver of post-HCT Grade 3 to 5 AEs, and underscored a link between AGVHD and thrombocytopenia. This establishes a critical safety framework upon which the impact of novel post-transplant AGVHD therapeutics should be evaluated. This trial was registered at <span><span>www.clinicaltrials.gov</span><svg><path></path></svg></span> (#NCT01743131).</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 109.e1-109.e13"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140615399","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":"Early Mixed Donor Chimerism is a Strong Negative Prognostic Indicator in Allogeneic Stem Cell Transplant for AML and MDS","authors":"Michael Radford ,&nbsp;Alejandro Garcia-Horton ,&nbsp;Rohail Badami ,&nbsp;Elaine Jin ,&nbsp;Nida Usmani ,&nbsp;Daria Grafodatskaya ,&nbsp;Elizabeth McCready ,&nbsp;Dina Khalaf ,&nbsp;Irwin Walker ,&nbsp;Brian Leber ,&nbsp;Kylie Lepic ,&nbsp;Gregory Pond ,&nbsp;Tobias Berg","doi":"10.1016/j.jtct.2024.11.006","DOIUrl":"10.1016/j.jtct.2024.11.006","url":null,"abstract":"&lt;div&gt;&lt;h3&gt;Background&lt;/h3&gt;&lt;div&gt;Allogeneic bone marrow transplantation remains the most potent curative therapy for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) due to the graft-versus-tumor effect provided by donor cells. Donor chimerism is utilized early after transplantation to evaluate engraftment and to monitor the persistence of donor hematopoiesis.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Objective(s)&lt;/h3&gt;&lt;div&gt;Literature is conflicting regarding to the prognostic utility of early mixed donor chimerism, chimerism kinetic patterns as well as factors associated with it and we sought to clarify this uncertainty.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Study Design&lt;/h3&gt;&lt;div&gt;In this single-centre retrospective analysis, 141 adults aged 18 years of age or older with AML (n = 104) and MDS (n = 37) who received their first transplant from HLA matched related, matched unrelated or mismatched related (haploidentical) donors between 2016 and 2022 and had at least day 30 chimerism measured were included. Approximately 30% received post-transplant cyclophosphamide for graft-versus-host disease (GVHD) prophylaxis and 67% of subjects received reduced-intensity conditioning. Chimerism was measured using STR-PCR from unfractionated peripheral blood mononuclear cells (whole blood; WB) and CD3+ (T cell; TC) compartment at each time point. Complete donor chimerism was defined as ≥95% whereas &lt;95% defined as mixed. Competing risk analysis was used to estimate cumulative incidence of relapse with kinetic calculations completed using an increment factor. Kaplan–Meier was used for overall survival (OS) and relapse-free survival (RFS). Cox proportional hazards regression was used to explore prognostic factors for OS and RFS.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Results&lt;/h3&gt;&lt;div&gt;Both day 30 mixed WB and TC donor chimerism were individually associated with an increased risk of relapse and worse overall and relapse-free survival at days 30, 60 and 90 post-transplant. Day 30 mixed WB was more specific for relapse (86%), while mixed TC was more sensitive (67%). Complete day 30 chimerism had a negative predictive value of 63% and 70% and positive predictive value of 57% and 67% for WB and TC, respectively. Day 30 WB and TC donor chimerism of &lt;88.92% and 89.29% had specificities of 79.17% and 82.19% although sensitivities only approximated 50%. Evaluating the kinetics of chimerism over the first 90 days provided additional information for prognosticating relapse than absolute chimerism values at individual time points in both WB day 30 to 90 [HR, 1.75 (95% CI, 1.04 to 2.94); &lt;em&gt;P&lt;/em&gt; &lt; .035] and TC day 60 to 90 [HR, 1.32 (95% CI, 1.03, 1.69); &lt;em&gt;P&lt;/em&gt; &lt; .29]. Twice as many patients with complete chimerism developed acute GVHD compared to those with mixed chimerism. Factors that were found to be associated with day 30 mixed TC chimerism were donor source, ATG GVHD prophylaxis, myeloablative conditioning and female sex, while only donor source was associated with mixed WB.&lt;/div&gt;&lt;/div&gt;&lt;div&gt;&lt;h3&gt;Conclu","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 77.e1-77.e20"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142640000","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":"Comparison of Nonrelapse Mortality After Haploidentical Hematopoietic Stem Cell Transplantation With Post-transplant Cyclophosphamide Versus Single Umbilical Cord Blood Transplantation in Hematologic Disease","authors":"Kaito Harada ,&nbsp;Junya Kanda ,&nbsp;Masahiro Hirayama ,&nbsp;Fumiya Wada ,&nbsp;Naoyuki Uchida ,&nbsp;Masatsugu Tanaka ,&nbsp;Hirohisa Nakamae ,&nbsp;Masahito Tokunaga ,&nbsp;Kazuya Ishiwata ,&nbsp;Makoto Onizuka ,&nbsp;Yuta Hasegawa ,&nbsp;Takahiro Fukuda ,&nbsp;Tetsuya Eto ,&nbsp;Naoki Kurita ,&nbsp;Toshiro Kawakita ,&nbsp;Atsushi Jinguji ,&nbsp;Fumihiko Ishimaru ,&nbsp;Yoshiko Atsuta ,&nbsp;Hideki Nakasone","doi":"10.1016/j.jtct.2024.11.011","DOIUrl":"10.1016/j.jtct.2024.11.011","url":null,"abstract":"&lt;div&gt;&lt;div&gt;Unrelated cord blood transplantation (UCBT) and haploidentical transplantation using posttransplant cyclophosphamide (PTCy-haplo) are alternatives for patients lacking a human leukocyte antigen-matched donor. CD34&lt;sup&gt;+&lt;/sup&gt; cell counts in cord blood affect transplantation outcomes, particularly nonrelapse mortality (NRM). The primary objective of this study was to compare the transplantation outcomes after UCBT and PTCy-haplo focusing on CD34&lt;sup&gt;+&lt;/sup&gt; cell counts in cord blood. This retrospective study used data from 2014 to 2020 from a Japanese nationwide database. UCBT cases were divided into those with UCBT with higher (UCB-H; ≥.84 × 10&lt;sup&gt;5&lt;/sup&gt;/kg) and lower (UCB-L; &lt;.84 × 10&lt;sup&gt;5&lt;/sup&gt;/kg) CD34&lt;sup&gt;+&lt;/sup&gt; cell counts, depending on the median CD34&lt;sup&gt;+&lt;/sup&gt; cell count. The study cohort comprised cases of PTCy-haplo (n = 1142), UCB-H (n = 3185), and UCB-L (n = 3172). In the multivariate analysis, neutrophil engraftment was significantly better in the PTCy-haplo than in the UCB-H (hazard ratio [HR], .64; 95% confidence interval [CI], .57 to .70; &lt;em&gt;P&lt;/em&gt; &lt; .001) and UCB-L groups (HR, .45; 95% CI, .41 to .50; &lt;em&gt;P&lt;/em&gt; &lt; .001). The UCB-H group showed similar NRM (HR, 1.19, 95% CI, 1.00 to 1.43, &lt;em&gt;P&lt;/em&gt; = .051) and OS (HR, 1.05, 95% CI, .94 to 1.18, &lt;em&gt;P&lt;/em&gt; = .38) compared with PTCy-haplo, whereas UCB-L was significantly associated with poor NRM (HR, 1.35, 95% CI, 1.13 to 1.61, &lt;em&gt;P&lt;/em&gt; = .001) and OS (HR, 1.13, 95% CI, 1.01 to 1.26, &lt;em&gt;P&lt;/em&gt; = .038). In contrast, the UCB-H (HR, .86; 95% CI, .75 to .98; &lt;em&gt;P&lt;/em&gt; = .027) and UCB-L groups (HR, .80; 95% CI, .70 to .92; &lt;em&gt;P&lt;/em&gt; = .001) were associated with lower relapse rate. Regarding the graft-versus-host disease (GVHD), the UCB-H and UCB-L groups were identified as significant risk factors for the development of grade II–IV acute GVHD (UCB-H: HR, 1.73; 95% CI, 1.51 to 1.99; &lt;em&gt;P&lt;/em&gt; &lt; .001; UCB-L: HR, 1.55; 95% CI, 1.35 to 1.78; &lt;em&gt;P&lt;/em&gt; &lt; .001) and grade III–IV acute GVHD (UCB-H: HR, 2.28; 95% CI, 1.78 to 2.91; &lt;em&gt;P&lt;/em&gt; &lt; .001; UCB-L: HR, 1.85; 95% CI, 1.44 to 2.37; &lt;em&gt;P&lt;/em&gt; &lt; .001), but neither were associated with the incidence of all-grade GVHD (UCB-H: HR, 1.12; 95% CI, .95 to 1.32; &lt;em&gt;P&lt;/em&gt; = .16; UCB-L: HR, 1.08; 95% CI, .91 to 1.27; &lt;em&gt;P&lt;/em&gt; = .37) or extensive chronic GVHD (UCB-H: HR, .86; 95% CI, .68 to 1.09; &lt;em&gt;P&lt;/em&gt; = .21; UCB-L: HR, .88; 95% CI, .69 to 1.12; &lt;em&gt;P&lt;/em&gt; = .31). Furthermore, higher NRM in UCB-L was attributed to higher infection-related mortality (HR, 1.50; 95% CI, 1.15 to 1.95; &lt;em&gt;P&lt;/em&gt; = .003) but not GVHD-related mortality (HR, 1.15; 95% CI, .82 to 1.62; &lt;em&gt;P&lt;/em&gt; = .43), whereas UCB-H was not a significant risk factor for both infection-related mortality (HR, 1.29; 95% CI, .99 to 1.69; &lt;em&gt;P&lt;/em&gt; = .06) and GVHD-related mortality (HR, 1.28; 95% CI, .90 to 1.80; &lt;em&gt;P&lt;/em&gt; = .16). UCB-H offered similar NRM and OS to PTCy-haplo, whereas UCB-L had worse outcomes. Our results can provide useful","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 103.e1-103.e13"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142740093","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":"Real-world Outcomes of Commercial Tisagenlecleucel for Children, Adolescents, and Young Adults With Acute Lymphoblastic Leukemia in Japan","authors":"Itaru Kato ,&nbsp;Daisuke Tomizawa ,&nbsp;Motohiro Kato ,&nbsp;Shinsuke Hirabayashi ,&nbsp;Atsushi Manabe ,&nbsp;Masahiro Irie ,&nbsp;Yoji Sasahara ,&nbsp;Yuki Arakawa ,&nbsp;Katsuyoshi Koh ,&nbsp;Hirotoshi Sakaguchi ,&nbsp;Masanaka Sugiyama ,&nbsp;Chitose Ogawa ,&nbsp;Takahiro Kamiya ,&nbsp;Shoji Saito ,&nbsp;Yozo Nakazawa ,&nbsp;Nobuhiro Nishio ,&nbsp;Yoshiyuki Takahashi ,&nbsp;Naoko Iwai ,&nbsp;Souichi Adachi ,&nbsp;Junko Takita ,&nbsp;Hidefumi Hiramatsu","doi":"10.1016/j.jtct.2024.11.016","DOIUrl":"10.1016/j.jtct.2024.11.016","url":null,"abstract":"<div><div>Chimeric antigen receptor (CAR) T cells are a major new treatment option for children, adolescents, and young adults (CAYA) patients with relapsed and refractory (R/R) B cell acute lymphoblastic leukemia (B-ALL). Therefore, accumulating evidence from real-world experiences of CAR-T outcomes in various regions worldwide is important, particularly when comparing outcomes of patients with differing medical and ethnic backgrounds. More than 5 years have passed since tisagenlecleucel was approved in Japan. Here, we report a retrospective, multi-institutional investigation examining the association between baseline parameters and clinical outcomes. The aim was to investigate real-world experience and to better comprehend the efficacy of commercial tisagenlecleucel. A nationwide consortium called the Japan CAR-T Consortium conducted a retrospective, multicenter study of CAYA patients who received CAR-T cell treatment with commercial tisagenlecleucel. Forty-two patients with R/R B-ALL whose leukapheresis samples were shipped to Novartis for commercial tisagenlecleucel manufacture were included in the analysis. All infused patients were included in the response, toxicity, and survival analyses. The best overall response rate was 93%. The 1-year overall survival and event-free survival (EFS) rates after infusion were 82% and 56%, respectively. Twenty-seven (64%) had low disease burden (LB, defined as &lt;5% bone marrow [BM] lymphoblasts) prior to tisagenlecleucel infusion. LB was associated with superior outcomes, with a 1-year EFS rate of 80% compared with 24% in high disease burden (≧5% BM lymphoblasts). Multivariate analysis identified an association between prior hematopoietic stem cell transplantation (HSCT) (<em>n</em> = 23, 55%) and superior outcomes, with a 1-year EFS rate of 75% compared with 24% for patients without prior HSCT. This first analysis of CAYA patients with R/R B-ALL undergoing treatment with commercial tisagenlecleucel in Japan reports an efficacy similar to that in clinical trials and other real-world studies and confirms that LB and prior HSCT are associated with superior EFS.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 86-96"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142772504","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":"Health-Related Values Discussions with Patients Undergoing Allogeneic and Autologous Stem Cell Transplant: Feasibility and Acceptability of an Early Primary Palliative Care Intervention","authors":"Abigail G. Cohen ,&nbsp;Christina Cho ,&nbsp;Emily Patterson ,&nbsp;Jessica Magaldi ,&nbsp;Tara Doga ,&nbsp;Kristine Naputo ,&nbsp;Kelsey Alvarez ,&nbsp;Elizabeth Giles ,&nbsp;Grace Yang ,&nbsp;Afshana Hoque ,&nbsp;Dana Kramer ,&nbsp;Sean Devlin ,&nbsp;David Nemirovsky ,&nbsp;William E. Rosa ,&nbsp;Jessica I. Goldberg ,&nbsp;Miguel-Angel Perales ,&nbsp;Andrew S. Epstein ,&nbsp;Judith E. Nelson ,&nbsp;Heather Landau","doi":"10.1016/j.jtct.2024.12.009","DOIUrl":"10.1016/j.jtct.2024.12.009","url":null,"abstract":"<div><h3>Background</h3><div>Hematopoietic stem cell transplant (HSCT) has curative potential but also relatively high morbidity and mortality. Patients have multidimensional palliative care (PC) needs throughout the transplant process. However, PC is not routinely offered to patients with hematologic malignancies. National guidelines recommend PC concurrent with curative hematologic disease treatment, including HSCT.</div></div><div><h3>Objectives</h3><div>Our goal was to determine the feasibility and acceptability of incorporating early and ongoing discussions of patients’ core health-related values (HRVs) for patients with hematologic malignancies undergoing HSCT.</div></div><div><h3>Study Design</h3><div>We designed and implemented a pilot study evaluating the transplant team's use of a brief, structured guide with eight open-ended questions to support patients’ articulation of their HRVs. All English-speaking patients undergoing HSCT from March 2021 to March 2022 in two outpatient HSCT clinics were eligible and offered enrollment. HRV discussions were planned pretransplant, and then at 5 time points post-transplant (Day 10-14, Day 30, Day 100, 6 months, 1 year). Clinicians and patients were surveyed to assess the feasibility and acceptability of this primary PC intervention.</div></div><div><h3>Results</h3><div>31 patients, mostly male (61%) and white (68%), with plasma cell (58%) and myeloid (42%) diseases participated in 149 values discussions. Initial discussions averaged 17.7 minutes; subsequent discussions were 13.3 minutes. Most patients were comfortable discussing their values and indicated it was important and helpful for them, as well as beneficial for their caregivers. Patients reported feeling heard and understood by their care team following values discussions. Clinicians were comfortable having the discussions, felt they were beneficial, and indicated learning new information about their patients beyond their diagnosis.</div></div><div><h3>Conclusions</h3><div>Incorporating discussions of patients’ HRVs into routine HSCT care was found to be feasible and acceptable in this pilot study. Feedback from patients and providers was overwhelmingly positive. Based on these results, the program has been refined and expanded to include all patients receiving HSCT and chimeric antigen receptor T cell (CAR-T) therapy, with plans to study the clinical impact of this approach.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 107.e1-107.e12"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142865541","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":"Chronic Graft-Versus-Host Disease Adversely Impacts School Performance in Children and Young Adults","authors":"Zahra Hudda ,&nbsp;Amanda Flannery ,&nbsp;Patricia Dillhoff ,&nbsp;Kristen Webster ,&nbsp;Jodi Jacobs ,&nbsp;Sarah Strong ,&nbsp;Jennifer Detzel ,&nbsp;Stella M. Davies ,&nbsp;Pooja Khandelwal","doi":"10.1016/j.jtct.2024.12.005","DOIUrl":"10.1016/j.jtct.2024.12.005","url":null,"abstract":"<div><h3>Background</h3><div>Chronic graft-versus-host disease (cGVHD) adversely impacts return to work for adult allogeneic hematopoietic stem cell transplant (HSCT) survivors, but no data exist on children with cGVHD transitioning back to school. We hypothesized that cGVHD adversely impacts broad aspects of school experience of children compared to their allogeneic-HSCT peers without cGVHD.</div></div><div><h3>Methods</h3><div>We conducted a single center cross-sectional pilot study using a 42-item questionnaire, investigating academic performance and social-emotional aspects of schooling pre- and post-HSCT. Forty allogeneic-HSCT patients of school-age completed the questionnaire, and responses were compared between patients with and without cGVHD.</div></div><div><h3>Results</h3><div>Twenty patients had cGVHD while 20 age or gender matched allogeneic-HSCT patients without cGVHD were controls. Ten of the 20 cGVHD patients experienced academic delays, of whom 2 were unable to resume or commence school due to cGVHD. All controls resumed/commenced school post-HSCT. Patients with cGVHD were chronically absent, as 8 of the 18 cGVHD patients missed ≥4 days of school per month post-HSCT compared to 0/20 controls (<em>P</em> ≤ .001). Profound barriers to school participation specific to cGVHD were appreciated with an average of 3 concurrent barriers (range 1-7) ranging from physical appearance to clothing discomfort. Significant gaps in school services were identified as only 6 (33%) cGVHD had school accommodations post-HSCT.</div></div><div><h3>Conclusions</h3><div>Academic challenges and emotional and psychosocial impacts are profound. Future studies evaluating the feasibility of standardizing early school-based interventions are required.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 99.e1-99.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142829512","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Retrospective Analysis of Fresh versus Cryopreserved Allogenic Bone Marrow Transplant within a Pediatric Population: A Change in Practice Due to the COVID-19 Pandemic","authors":"Rhea Hans ,&nbsp;Charlotte Schwalbach ,&nbsp;Roberta H. Adams ,&nbsp;Holly Miller ,&nbsp;Dana Salzberg ,&nbsp;Mohamad Sinno ,&nbsp;Kristen Beebe ,&nbsp;Daniela Giralt ,&nbsp;Jennifer Stahlecker ,&nbsp;Jeff Crosby ,&nbsp;Jefferson Lin ,&nbsp;Lucia Mirea ,&nbsp;Kevin Land ,&nbsp;Alexander Ngwube","doi":"10.1016/j.jtct.2024.12.004","DOIUrl":"10.1016/j.jtct.2024.12.004","url":null,"abstract":"<div><h3>Background</h3><div>Several adult studies show mixed reports in clinical outcomes between cryopreserved and fresh stem cell products, with majority reporting no significant differences and others report that there are differences in outcomes. There is limited literature reporting its impact on outcomes in pediatric hematopoietic cell transplantation (HSCT).</div></div><div><h3>Objective</h3><div>To compare clinical outcomes between fresh vs cryopreserved stem cell treatment in pediatric HSCT.</div></div><div><h3>Study Design</h3><div>A retrospective chart review was conducted on allogenic HSCT at Phoenix Children's Hospital between January 1, 2016, and March 31, 2023. The study included 181 patients, with 105 receiving fresh stem cell products and 76 receiving cryopreserved products. Clinical outcomes including, neutrophil and platelet recovery, graft versus host disease, immune reconstitution and survival outcome were compared.</div></div><div><h3>Results</h3><div>Study subjects had median follow-up of 997 (range 12-2642) days. 92 patients were treated for a malignant disease (leukemia/lymphoma) and 89 were treated for a non-malignant disease (hemoglobinopathies, immunodeficiency/immune dysregulation, and bone marrow failure). 124 stem cell products were from bone marrow and 57 were from peripheral blood. Comparisons between fresh vs cryopreserved treatments found no significant difference in days to neutrophil engraftment (<em>P</em> = .47) or platelet engraftment (p=0.94). No difference in the incidence of acute graft versus host disease or chronic graft versus host disease (p = 0.70) between both groups. Immune reconstitution at 365 days post-transplant did not vary significantly between treatment groups for CD4+ T cells, CD8+ T cells, CD19+ B cells, and CD56/16+ NK cells. Overall survival at 2 years was similar in the fresh vs cryopreserved (86.7% vs 84.2%; <em>P</em> = .64).</div></div><div><h3>Conclusion</h3><div>These observations suggest that cryopreserved stem cell product is a reasonable alternative with comparable efficacy and potentially offering logistical advantages. Further research with larger pediatric cohorts is recommended to confirm non-inferiority of cryopreserved treatments in pediatric HSCT.</div></div>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Pages 97.e1-97.e11"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142839445","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)00039-9","DOIUrl":"10.1016/S2666-6367(25)00039-9","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":"31 2","pages":"Page A4"},"PeriodicalIF":3.6,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143168670","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 bone marrow transplantation experience in 350 matched sibling donor grafts for Severe Thalassemia.","authors":"Rajat Kumar Agarwal, Rakesh Dhanya, Dr Deepa Trivedi, Dr Vaibhav Shah, Dr Mohan Reddy, Dr Priya Marwah, Amit Sedai, Kumari Ankita, Lalith Parmar, Dr Lawrence Faulkner","doi":"10.1016/j.jtct.2025.01.890","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.01.890","url":null,"abstract":"<p><strong>Background: </strong>For matched related hematopoietic cell donor transplantation (HCT) for non-malignant diseases most centres prefer bone marrow (BM) over peripheral stem cell (PBSC) grafts due to increased risk of chronic GVHD associated with the latter. BM generally entails delayed neutrophil and platelet recovery compared to PBSC transplants. G-CSF-primed bone marrow (G-BM) has been associated with faster hematological recovery while retaining a decreased risk of GVHD. Moreover, it may allow for reduced marrow collection volumes.</p><p><strong>Objectives: </strong>We retrospectively analysed our experience with G-BM as graft source from July 2015 to February 2023 across 350 consecutive first matched sibling transplants in children with severe thalassemia in four centres in India.</p><p><strong>Findings: </strong>We observed that G-BM is associated with rapid hematological recovery with relatively low rates of CMV reactivation (16%), low rates of moderate to severe GVHD (grade 3-4 acute GVHD was 5% and moderate to severe chronic GVHD was 3%), reduced marrow collection volumes (12.5 ml/Kg of donor's weight), and thus is potentially safer for both donors and recipients compared with standard bone marrow. This observation was made in a relatively homogenous cohort of multiply transfused patients with thalassemia who are at high risk of rejection. None of the donors required third-party blood transfusion irrespective of donor-recipient weight discrepancy.</p><p><strong>Conclusion: </strong>Our experience suggests that G-BM is associated with prompt engraftment and very low rates of moderate or severe GVHD. It also appears to be safe for donors and decrease the risk for third-party red cell transfusions. Finally, its relatively easy and inexpensive to collect. G-BM should be strongly considered as a preferable graft source in matched-related donor transplantations for thalassemia and potentially other transplant indications.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-01-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143075529","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}