Transplantation and Cellular Therapy最新文献

{"title":"Recent Advances in the Prognosis of HHV-6 Encephalitis Following Allogeneic Hematopoietic Stem Cell Transplantation.","authors":"Kazuya Kurihara, Daichi Sadato, Takashi Toya, Chizuko Hirama, Kana Kato, Kaori Kondo, Yasutaka Sadaga, Chika Kato, Masashi Shimabukuro, Atsushi Jinguji, Fumihiko Ouchi, Hiroaki Shimizu, Yuho Najima, Yuka Harada, Noriko Doki","doi":"10.1016/j.jtct.2025.04.016","DOIUrl":"10.1016/j.jtct.2025.04.016","url":null,"abstract":"<p><p>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: P = .0086), and 1-year NRM was significantly lower (26.8 versus 62.1%; P = .024). Multivariate analysis revealed that allo-HSCT before 2017 (hazard ratio [HR] 3.13, P = .012) and haploidentical transplantation (HR 3.07, P = .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.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-28","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":"Heterogeneity of Survival Benefit Conferred By Letermovir.","authors":"Yu Akahoshi, Hideki Nakasone, Katsuto Takenaka, Takahide Ara, Yuma Tada, Noriko Doki, Naoyuki Uchida, Masatsugu Tanaka, Yuta Hasegawa, Wataru Takeda, Tetsuya Nishida, Jun Ishikawa, Naoki Kurita, Masashi Sawa, Makoto Onizuka, Shinichi Kako, Shin-Ichiro Fujiwara, Keisuke Kataoka, Koji Kawamura, Takahiro Fukuda, Yoshiko Atsuta, Kimikazu Yakushijin, Yoshinobu Kanda","doi":"10.1016/j.jtct.2025.04.010","DOIUrl":"10.1016/j.jtct.2025.04.010","url":null,"abstract":"<p><p>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 (>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.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-28","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":"Dose Effect of Antithymocyte Globulin or Post-transplant Cyclophosphamide in Haploidentical Peripheral Blood Stem Cell Transplantation: A Comparative Study.","authors":"Yosuke Nakaya, Hirohisa Nakamae, Hideki Nakasone, Hiroshi Okamura, Naonori Harada, Koji Kawamura, Seitaro Terakura, Yuta Hasegawa, Tetsuya Eto, Takahiro Fukuda, Nobuhiro Hiramoto, Koji Nagafuji, Shuichi Ota, Yumiko Maruyama, Toshiro Kawakita, Ken-Ichi Matsuoka, Noriko Doki, Tomohiko Kamimura, Toshihiko Ando, Takashi Akasaka, Yoshiko Atsuta, Junya Kanda","doi":"10.1016/j.jtct.2025.04.007","DOIUrl":"10.1016/j.jtct.2025.04.007","url":null,"abstract":"","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143996452","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":"Cytokine Release Syndrome and Neurotoxicity Following CD19 CAR-T in B-Cell Lymphoma.","authors":"Roni Shouval, Christopher Strouse, Soyoung Kim, Temitope Oloyede, Sairah Ahmed, Farrukh T Awan, Danny Luan, Veronika Bachanova, Talha Badar, Merav Bar, Pere Barba, Amer M Beitinjaneh, Amanda Cashen, Bhagirathbhai Dholaria, Mahmoud Elsawy, Siddhartha Ganguly, Praveen Ramakrishnan Geethakumari, Uri Greenbaum, Hamza Hashmi, LaQuisa C Hill, Michael D Jain, Tania Jain, Partow Kebriaei, Adam S Kittai, Frederick L Locke, Premal D Lulla, Elena Mead, Joseph P McGuirk, Alberto Mussetti, Taiga Nishihori, Amanda L Olson, Martina Pennisi, Miguel-Angel Perales, Peter A Riedell, Wael Saber, Abu-Sayeef Mirza, Margarida Magalhaes-Silverman, Elizabeth J Shpall, Mohamed Sorror, Kitsada Wudhikarn, Cameron J Turtle, Amy Moskop, Marcelo C Pasquini","doi":"10.1016/j.jtct.2025.03.011","DOIUrl":"10.1016/j.jtct.2025.03.011","url":null,"abstract":"<p><p>Chimeric antigen receptor T cell (CAR-T) therapy is an effective treatment for relapsed-refractory large B-cell lymphoma (LBCL). However, toxicities, particularly cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), remain significant concerns. Analyze temporal trends, risk factors, and associations between these toxicities and their severity. In this registry study by the Center for International Blood and Marrow Transplant Research, we studied CRS and ICANS in 1916 LBCL patients treated with commercial CAR-T therapies (axicabtagene ciloleucel 74.9%, tisagenlecleucel 25.1%) between 2018 and 2020. Outcomes include development of CRS/ICANS, timing and severity according to ASTC grading, overall survival (OS). Risk factors were assessed using Cox proportional hazards model. Among patients developing CRS (75.2%), 11.3% had grade ≥3 CRS. Among patients developing ICANS (43.5%), 47.7% had grade ≥3 ICANS. Among patients developing CRS, severe CRS rates decreased from 14.0% in 2018 to 9.2% in 2020 (P< .01). However, the proportion of severe ICANS in patients who developed ICANS remained statistically unchanged (41.5% in 2018 to 53.7% in 2020, P= .10). CRS and ICANS were correlated: 57.1% of patients with CRS also experienced ICANS, and CRS was reported in 97.5% of ICANS cases, suggesting a potential continuum between toxicities. Axicabtagene ciloleucel was associated with higher risk of any grade CRS (OR, 4.6; 95% CI, 3.65 to 5.81) and ICANS (OR, 5.85; 95% CI, 4.48 to 7.64) as well as early and severe forms of both complications. Older age, lower performance status, and elevated lactate dehydrogenase levels prior to infusion also variably predicted these toxicities. In a landmark analysis starting 30 days postinfusion, patients with severe CRS or severe ICANS had shorter OS compared to those without these toxicities. High grades of CRS improved over time likely related to earlier intervention, development of ICANS is intrinsically related with CRS. These findings underscore the need for effective strategies to mitigate these toxicities and improve CAR-T safety.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143996473","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":"Coronary Artery Calcification as a Predictor of Survival in Patients Receiving Post-transplant Cyclophosphamide for GVHD Prophylaxis.","authors":"Christopher Graham, Anmol Baranwal, Bas Kietselaer, Chadi Ayoub, Carolyn Larsen, Kimberly J Langer, Mohamed A Kharfan-Dabaja, Ernesto Ayala, James Foran, Hemant Murthy, Vivek Roy, Madiha Iqbal, Jeanne Palmer, Lisa Z Sproat, Saurabh Chhabra, Nandita Khera, Talal Hilal, Urshila Durani, Aasiya Matin, Mehrdad Hefazi Torghabeh, Abhishek Mangaonkar, Mithun V Shah, Mark R Litzow, William J Hogan, David Dingli, Hassan B Alkhateeb","doi":"10.1016/j.jtct.2025.03.014","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.03.014","url":null,"abstract":"<p><p>Post-transplant Cyclophosphamide (PTCy) is becoming the new standard of care for graft-versus-host disease (GVHD) prophylaxis in patients undergoing allogeneic hematopoietic stem cell transplantation (alloHCT). High-dose cyclophosphamide has been associated with cardiac dysfunction through multiple mechanisms. Assess if patients with evidence of coronary artery calcification (CAC) are at higher risk for non-relapse mortality (NRM) and inferior survival after receiving PTCy for GVHD prophylaxis. We retrospectively reviewed patients with hematologic diseases undergoing alloHCT using PTCy for GVHD prophylaxis in the Mayo Clinic Enterprise from 2018 to 2022. Patients with non-contrast CT imaging for review for CAC within 1 year (yr) of alloHCT were included in this study, with imaging analyzed by a blinded independent reviewer. Of 204 patients who received PTCy for GVHD prophylaxis, 144 (70.5%) had available CT imaging available for CAC review. Seventy-three (50.7%) patients were positive for CAC (+) and 71 (49.3%) were negative for CAC (-). Compared to CAC-, CAC+ patients were older (64 versus 45 years, P < .001) and more likely to have known coronary artery disease (CAD) before transplant (16.4% versus 1.4%, P = .004), but had comparable HCT-CI (P=0.17). NRM was higher among patients with CAC+ compared to CAC- at 1 and 2-yr (26.5% versus 8.9%, P = .008), (28.0% versus 8.9%, P = .005), respectively. Univariate competing risk analysis showed that CAC was significantly associated with 2-yr NRM (HR 3.41, 95% CI 1.36-8.54, P = .009) and inferior post-alloHCT survival (2-yr OS rate 51.6% versus 72.3%, P = .01). Multivariate analysis (MVA) confirmed that CAC+ was associated with 2-yr NRM (HR 4.37, 95% CI 1.71-11.18, P = .002). While CAC+ did not impact OS in the whole cohort, among elderly patients age ≥60 and without a history of CAD, MVA confirmed that CAC+ was associated with an inferior 2-yr OS (HR 3.67, 95% CI 1.007-13.38, P = .049) and higher NRM (35.5% versus 0%, P = .006). Coronary artery calcification is readily assessable in imaging studies during pretransplant evaluation. Among patients receiving PTCy, CAC was associated with a higher NRM. CAC+ was associated with inferior OS, particularly in elderly patients without a history of coronary artery disease.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144048128","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, Leslie Lehmann, Christopher E Dandoy, Jeffery J Auletta, Priscila Badia, Sheri A Ballard, Robyn Blacken, Nancy M Daraiseh, Catherine Desmond, Chloe Dunseath, Preston Epling, Taylor J Fitch, Laura Flesch, David Hartley, John Huber, Kari Jenssen, Georgia Kent, Anna Klunk, Malika Kapadia, Katilyn Kusnier, Nicole Liberio, Steffani Maier, Kasiani C Myers, Gabby O'Connor, Sarah Tarquini, Rachel Phelan, Ahna Pai, Seth Rotz","doi":"10.1016/j.jtct.2025.04.013","DOIUrl":"10.1016/j.jtct.2025.04.013","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-21","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}

{"title":"G-CSF-Primed Peripheral Blood Stem Cell Haploidentical Transplantation Could Achieve Satisfactory Clinical Outcomes for Severe Aplastic Anemia Patients.","authors":"Xiao-Di Ma, Zheng-Li Xu, Yun He, Yi-Fei Cheng, Ting-Ting Han, Yuan-Yuan Zhang, Jing-Zhi Wang, Xi-Dong Mo, Feng-Rong Wang, Xin Zhao, Yu Wang, Xiao-Hui Zhang, Xiao-Jun Huang, Lan-Ping Xu","doi":"10.1016/j.jtct.2025.04.008","DOIUrl":"10.1016/j.jtct.2025.04.008","url":null,"abstract":"<p><strong>Background: </strong>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.</p><p><strong>Objectives: </strong>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).</p><p><strong>Study design: </strong>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.</p><p><strong>Results: </strong>The PB group demonstrated greater neutrophil (100% vs. 93.9%, P = .04) and platelet engraftment (95.5% vs. 89.0%, P = .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%, P = .28) or grades III-IV acute graft-versus-host disease (aGVHD; 4.5% vs. 4.6%, P = .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% (P = .90), 86.1% versus 83.3% (P = .73) and 76.5% versus 75.2% (P = .70) for PB and BM+PB group, respectively. In univariate analysis, the graft source did not influence the clinical outcomes after HSCT.</p><p><strong>Conclusions: </strong>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 conclusion should be further tested by prospective randomized trials.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-19","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":"Proceedings From the Reimagining Caregiver Workshop: Addressing Caregiver Requirements for Hematopoietic Cell Transplant.","authors":"Jaime M Preussler, Anna M DeSalvo, Ben Tweeten, June Klaphake, Meghann R Cody, Paris M McGhee, Karla S Dawson, Katie Schoeppner, Jeffery J Auletta","doi":"10.1016/j.jtct.2025.04.006","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.04.006","url":null,"abstract":"<p><p>The NMDP-sponsored, PCORI (Patient Centered Outcomes Research Institute)-funded project, Reimagining Caregiving Together, Engagement to Address Caregiver Requirement Barriers, 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. 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.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-12","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":"Omidubicel-onlv Transplantation for Hematologic Malignancies: Results of a Multicenter Expanded Access Program.","authors":"Mitchell E Horwitz, Gary J Schiller, Stephanie B Tsai, Andrew R Rezvani, Richard T Maziarz, Uri Goshen, Stuart Levy, Aurélie Schwarzbach, Roei D Mazor, Patrick J Stiff","doi":"10.1016/j.jtct.2025.04.005","DOIUrl":"https://doi.org/10.1016/j.jtct.2025.04.005","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-08","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":"Hematopoietic Stem Cell (HSC) Graft Cryopreservation Effects on Outcomes of Patients with Hematologic Malignancies: the Multicenter United Kingdom Experience During the COVID-19 Pandemic.","authors":"Ruta Barkauskiene, Angharad Pryce, Rachel Pearce, John A Snowden, Ram Malladi, Victoria Potter, Julia Lee, Marie Wilson, Nicola Cooper, Jane F Apperley, Chloe Anthias, Antonio Pagliuca, Rachel Pawson, Robert Danby","doi":"10.1016/j.jtct.2025.03.018","DOIUrl":"10.1016/j.jtct.2025.03.018","url":null,"abstract":"<p><p>The effects of graft cryopreservation on patient outcomes in allogeneic hematopoietic cell transplantation (HCT) remains unclear. In our multicenter UK study, outcomes of 926 adults receiving an allogeneic cryopreserved peripheral blood stem cell (PBSC) graft for a malignant hematologic indication between June 2020 and September 2021 were compared with 1491 adults with hematologic malignancy transplanted June 2018 to September 2019 with fresh PBSC grafts. There were short delays in median platelet and neutrophil engraftment with cryopreserved hematopoietic stem cell (HSC) grafts: 18 versus 15 days (P < .01) for platelets and 14 versus 13 days (P < .01) for neutrophils in the cryopreserved and fresh historical control groups, respectively. Reassuringly, primary graft failure rates were similar (P = .48). Relapse rates were higher (P = .03) but non-relapse mortality was lower (P < .01) in the cryopreserved group at 12-month follow-up. There was no statistical difference in overall survival between the groups (P = .52). Cryopreservation of allogeneic HSC grafts remains an important quality consideration in HCT practice. Advancement of our clinical and scientific understanding of this aspect of laboratory processing of HCT grafts is essential in relation not only to the pandemic but also for its use in other clinical and logistical settings where fresh donations are not feasible.</p>","PeriodicalId":23283,"journal":{"name":"Transplantation and Cellular Therapy","volume":" ","pages":""},"PeriodicalIF":3.6,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144028189","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}